65. 原発性免疫不全症候群 ［臨床試験数：273，薬物数：388（DrugBank：87），標的遺伝子数：70，標的パスウェイ数：189］
Searched query = "Primary immunodeficiency", "X SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP 70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak Higashi syndrome", "Chediak Higashi syndrome", "X linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM associated protein deficiency", "XIAP deficiency", "X linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13 4 deficiency", "Syntaxin 11 deficiency", "Munc18 2 deficiency", "Autoimmune polyendocrinopathy candidiasis ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi Pudlak syndrome type 2", "Hermanskyi Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA ID", "Interleukin 1 receptor associated kinase 4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose binding protein associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04154488 | December 11, 2019 | 11 November 2019 | A Study of Mavorixafor in Participants With Severe Congenital Neutropenia and Chronic Neutropenia Disorders | A Phase 1B, Open-Label, Multicenter Study of Mavorixafor in Patients With Severe Congenital Neutropenia and Chronic Neutropenia Disorders | Neutropenia | Drug: Mavorixafor | X4 Pharmaceuticals | Not recruiting | 12 Years | N/A | All | 45 | Phase 1 | United States | |
| 2 | NCT03814798 | December 2019 | 22 July 2019 | Study Evaluating IGSC 20% Flexible Dosing in Treatment-Experienced and Treatment-Naive Subjects With Primary Immunodeficiency | A Multicenter, Randomized, Cross-over, Open-label Study to Evaluate IGSC 20% Flexible Dosing Including Daily Push Dosing In Treatment-Experienced Subjects With Primary Immunodeficiency (PI) and Evaluation of Loading/Maintenance IGSC 20% in Treatment-Naïve Subjects With PI | Primary Immunodeficiency | Biological: IGSC 20% daily push versus every 2 weeks pump;Biological: IGSC 20% daily push versus once a week pump;Biological: IGSC 20% daily push versus 2 times per week pump;Biological: IGSC 20% 150 mg/kg | Grifols Therapeutics LLC | Not recruiting | 2 Years | 75 Years | All | 60 | Phase 3 | United States | |
| 3 | NCT04140539 | November 11, 2019 | 4 November 2019 | A Clinical Study to Enable Process Validation of Commercial Grade OTL-101 | A Single Arm, Open Label Clinical Study to Enable Process Validation of Commercial Grade Ex Vivo Hematopoietic Stem Cell Gene Therapy (OTL-101) in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | Severe Combined Immunodeficiency Due to ADA Deficiency | Biological: OTL-101 | Orchard Therapeutics | University of California, Los Angeles | Recruiting | N/A | 17 Years | All | 3 | Phase 2/Phase 3 | United States |
| 4 | NCT03597594 | November 2019 | 11 November 2019 | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Severe Combined Immunodeficiency | Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte Infusion | St. Jude Children's Research Hospital | Not recruiting | 2 Months | N/A | All | 42 | Phase 1/Phase 2 | United States | |
| 5 | NCT03896932 | November 2019 | 8 April 2019 | Minipooled-IVIG in Primary Immunodeficiency Disease | Study of Safety and Efficacy of Mini-pool Intravenous Immunoglobulin (MP-IVIG) Prepared by Assiut University Hospital Blood Bank in Primary Immunodeficiency Patients | Primary Immunodeficiency | Other: minipooled- Intravenous immunoglobulin(MP-IVIG) | Assiut University | Not recruiting | N/A | 18 Years | All | 15 | N/A | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03910452 | October 28, 2019 | 11 November 2019 | Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cell | National Institute of Allergy and Infectious Diseases (NIAID) | Recruiting | 4 Years | 65 Years | All | 30 | Early Phase 1 | United States | |
| 7 | NCT03995108 | October 17, 2019 | 4 November 2019 | Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension | WHIM Syndrome | Drug: Mavorixafor;Drug: Placebo | X4 Pharmaceuticals | Recruiting | 12 Years | N/A | All | 28 | Phase 3 | United States | |
| 8 | NCT04049084 | September 26, 2019 | 4 November 2019 | An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID | An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency (SCID) | Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV) | Orchard Therapeutics | University of California, Los Angeles;Great Ormond Street Hospital for Children NHS Foundation Trust | Recruiting | N/A | N/A | All | 70 | Phase 2 | United States;United Kingdom |
| 9 | NCT02860559 | September 2019 | 23 April 2019 | Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency | Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency | Severe Combined Immunodeficiency | Biological: TBX-1400 | Taiga Biotechnologies, Inc. | Not recruiting | 1 Month | 4 Years | All | 8 | Phase 1 | Israel | |
| 10 | NCT03939533 | September 2019 | 30 September 2019 | Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases | Clinical Phase 3 Study to Monitor the Safety, Tolerability, and Efficacy of Subcutaneous Human Immunoglobulin (CUTAQUIG®) Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder | Drug: CUTAQUIG | Octapharma | Not recruiting | 2 Years | 75 Years | All | 65 | Phase 3 | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT03974178 | September 2019 | 26 August 2019 | Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense | Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense: a Multicentre, Open-label Clinical Trial | Trypanosoma Brucei Rhodesiense; Infection | Drug: Fexinidazole | Drugs for Neglected Diseases | European and Developing Countries Clinical Trials Partnership (EDCTP) | Not recruiting | 6 Years | N/A | All | 50 | Phase 2/Phase 3 | |
| 12 | NCT03984890 | August 1, 2019 | 4 November 2019 | Vitamin D3 For CGD Patients With BCGosis/Itis | Effect of Vitamin D3 Supplementation on Chronic Granulomatous Disease Patients With BCGosis/Itis | Vitamin D3;Chronic-granulomatous Disease;BCG | Drug: Traditional treatment of CGD and TB;Drug: Vitamin D3 | Children's Hospital of Fudan University | Recruiting | N/A | 18 Years | All | 50 | Phase 2/Phase 3 | China | |
| 13 | NCT03878069 | June 2019 | 25 March 2019 | Registry Study of Revcovi Treatment in Patients With ADA-SCID | Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency | Biological: elapegademase-lvlr | Leadiant Biosciences, Inc. | Not recruiting | N/A | 65 Years | All | 20 | N/A | ||
| 14 | NCT03961009 | April 30, 2019 | 3 June 2019 | Efficacy, Safety and Pharmacokinetics Study of Kedrion IVIG 10% to Treat Participants With Primary Immunodeficiency (PID) | A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety and Pharmacokinetics of Kedrion Intravenous Immunoglobulin (IVIg) 10% in Primary Immunodeficiency Disease (PID) Patients | Primary Immunodeficiency Disease | Biological: Kedrion IVIG 10% | Kedrion S.p.A. | Recruiting | 2 Years | 70 Years | All | 50 | Phase 3 | United States;Canada | |
| 15 | NCT03825783 | April 15, 2019 | 9 September 2019 | A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene. | Leukocyte Adhesion Defect - Type I | Biological: RP-L201 | Rocket Pharmaceuticals Inc. | Recruiting | 3 Months | N/A | All | 2 | Phase 1 | Spain | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT03547830 | April 13, 2019 | 30 September 2019 | Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients | A Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Plerixafor;Drug: Gcsf | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | Recruiting | 1 Month | 24 Years | All | 17 | Phase 2 | Russian Federation | |
| 17 | NCT03601286 | December 21, 2018 | 29 April 2019 | Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency | Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan | Severe Combined Immunodeficiency, X-Linked | Drug: Lentiviral vector transduced CD34+ cells | Great Ormond Street Hospital for Children NHS Foundation Trust | Recruiting | 8 Weeks | 5 Years | Male | 5 | Phase 1 | United Kingdom | |
| 18 | NCT03812263 | November 30, 2018 | 4 February 2019 | A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Defect - Type I | Biological: RP-L201 | Rocket Pharmaceuticals Inc. | Recruiting | 3 Months | N/A | All | 9 | Phase 1/Phase 2 | United States | |
| 19 | EUCTR2018-002680-26-ES | 27/11/2018 | 7 January 2019 | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Deficiency-I (LAD-I) MedDRA version: 20.0 Level: LLT Classification code 10018137 Term: Genetic anomalies of leukocytes System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LADICell Product Code: RP-L201 Pharmaceutical Form: Suspension for injection INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS Concentration unit: IU/kg international unit(s)/kilogram Concentration type: not less then Concentration number: 4000000- | Rocket Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 2 | Phase 1 | Spain | |||
| 20 | EUCTR2018-001029-14-FR | 25/10/2018 | 6 November 2018 | HTLP | A phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients - HTLP NECKER | SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT. MedDRA version: 20.0 Level: PT Classification code 10010099 Term: Combined immunodeficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: HTLP Product Code: HTLP Pharmaceutical Form: Suspension for injection | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP) | Authorised | Female: yes Male: yes | 12 | Phase 1;Phase 2 | France | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT03619551 | October 22, 2018 | 24 June 2019 | Conditioning SCID Infants Diagnosed Early | A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRaß+/CD19+ Depleted Transplantation: A Phase II Study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) | SCID | Drug: Busulfan;Device: Cell processing for TCRaß+/CD19+ depletion | Michael Pulsipher, MD | Recruiting | N/A | 2 Years | All | 64 | Phase 2 | United States | |
| 22 | NCT03716700 | September 24, 2018 | 3 December 2018 | Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN) | Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN) | Primary Immunodeficiency Diseases (PID) | Biological: CUVITRU | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire;Baxalta US Inc., now part of Shire | Recruiting | 3 Years | N/A | All | 500 | N/A | Canada |
| 23 | EUCTR2018-000338-36-DE | 20/09/2018 | 26 November 2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1 Level: PT Classification code 10003594 Term: Ataxia telangiectasia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate Pharmaceutical Form: Solution for blood fraction modification INN or Proposed INN: Dexamethasone sodium phosphate CAS Number: 2392-39-4 Current Sponsor code: DSP Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 25- | EryDel S.p.A. | Authorised | Female: yes Male: yes | 51 | Phase 3 | United States;Poland;Belgium;Spain;Australia;Israel;Germany;India | |||
| 24 | NCT03663933 | September 4, 2018 | 4 November 2019 | Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation | Phase II Trial of Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation | Lymphoproliferative Disorders;Autoimmune Lymphoproliferative;Primary T-cell Immunodeficiency Disorders;Immune System Diseases;Common Variable Immunodeficiency | Drug: Immunosuppression Only Conditioning (IOC);Drug: Reduced Intensity Conditioning (RIC);Drug: GVHD Prophylaxis;Procedure: Allogeneic HSC | National Cancer Institute (NCI) | Recruiting | 4 Years | N/A | All | 177 | Phase 2 | United States | |
| 25 | NCT03907241 | September 1, 2018 | 24 June 2019 | Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases, Including (But Not Limited to) Those Who Have Completed the SCGAM-01 Trial | Clinical Phase III Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases, Including (But Not Limited to) Those Who Have Completed the SCGAM-01 Trial | Primary Immunodeficiency | Drug: Octanorm 16.5% | Octapharma | Not recruiting | 18 Years | 75 Years | All | 6 | Phase 3 | Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | EUCTR2018-001489-41-SE | 02/07/2018 | 23 July 2018 | A clinical trial using cell transplantation as treatment for bone marrow failure due to dyskeratosis congenita / telomere disease, inherited genetic conditions. | Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease | Dyskeratosis congenita / telomere disease MedDRA version: 20.0 Level: PT Classification code 10001756 Term: Allogenic bone marrow transplantation therapy System Organ Class: 10042613 - Surgical and medical procedures ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Lemtrada Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Alemtuzumab CAS Number: 216503-57-0 Other descriptive name: ALEMTUZUMAB Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0,2- Trade Name: Fludarabine Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: FLUDARABINE CAS Number: 21679-14-1 Concentration unit: mg/m2 milligram(s)/square meter Concentration type: equal Concentration number: 30- | Karolinska Universitetssjukhuset | Authorised | Female: yes Male: yes | 20 | Phase 2 | United States;Sweden | |||
| 27 | NCT03366142 | July 2, 2018 | 28 October 2019 | Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology | Use of Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology | LAD1 | Drug: Ustekinumab | National Institute of Allergy and Infectious Diseases (NIAID) | Recruiting | 12 Years | 65 Years | All | 20 | Phase 1/Phase 2 | United States | |
| 28 | NCT03513328 | June 15, 2018 | 3 June 2019 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders | Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;Adrenoleukodystrophy | Drug: Thiotepa--single daily dose;Drug: Thiotepa--escalated dose | University of Florida | Live Like Bella Pediatric Cancer Research | Recruiting | 6 Months | 38 Years | All | 40 | Phase 1/Phase 2 | United States |
| 29 | NCT03576469 | June 13, 2018 | 26 August 2019 | A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | CVI - Common Variable Immunodeficiency | Biological: C1-esterase inhibitor [recombinant] (C1-INH-R) | IMMUNOe Research Centers | Recruiting | 18 Years | N/A | All | 20 | Phase 4 | United States | |
| 30 | EUCTR2018-000338-36-ES | 31/05/2018 | 18 June 2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1 Level: PT Classification code 10003594 Term: Ataxia telangiectasia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate Pharmaceutical Form: Solution for blood fraction modification INN or Proposed INN: Dexamethasone sodium phosphate CAS Number: 2392-39-4 Current Sponsor code: DSP Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 25- | EryDel S.p.A. | Authorised | Female: yes Male: yes | 61 | Phase 3 | United States;Poland;Belgium;Spain;Australia;Israel;Norway;Germany;Italy;United Kingdom;India | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT03538899 | May 31, 2018 | 15 July 2019 | Autologous Gene Therapy for Artemis-Deficient SCID | A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells | Severe Combined Immunodeficiency | Drug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: Busulfan | University of California, San Francisco | Recruiting | 2 Months | N/A | All | 15 | Phase 1/Phase 2 | United States | |
| 32 | NCT03548818 | May 16, 2018 | 15 July 2019 | Role of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease | Role of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Interferon Gamma-1B | University of Colorado, Denver | Recruiting | 5 Years | 60 Years | All | 20 | Phase 3 | United States | |
| 33 | NCT03232203 | April 12, 2018 | 27 May 2019 | Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs) | Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ Treatment | Severe Combined Immunodeficiency Due to ADA Deficiency | Drug: STRIMVELIS | Orchard Therapeutics | Recruiting | 18 Years | N/A | All | 10 | N/A | Italy | |
| 34 | NCT03512314 | January 24, 2018 | 26 August 2019 | Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension | Open-label Extension Study With Tadekinig Alfa (r-hIL-18BP) to Monitor Safety and Tolerability in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP Deficiency | XIAP Deficiency;NLRC4-MAS | Drug: Tadekinig alfa | AB2 Bio Ltd. | Recruiting | N/A | N/A | All | 10 | Phase 3 | United States;Canada;Germany | |
| 35 | NCT03311503 | January 19, 2018 | 11 February 2019 | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Severe Combined Immunodeficiency, X Linked;Gene Therapy | Biological: autologous CD34+ cell transduced with G2SCID vector | David Williams | University of California, Los Angeles | Recruiting | N/A | 5 Years | Male | 10 | Phase 1/Phase 2 | United States;United Kingdom |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | JPRN-UMIN000030806 | 2018/01/17 | 2 April 2019 | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | Wiskott-Aldrich syndrome | WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and Busulfan. 1. Rituximab (day-22) 375 mg/m2 2. Preconditioning chemotherapy Fludarabine 30mg/m2 x 2 (day-3, day-2) Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours) 3. Infusion of WASP cDNA-transduced CD34 positive HSC 5 x 10^6/kg (at least 3 x 10^6/kg) | National Center for Child Heath and Development | Recruiting | Not applicable | Not applicable | Male | 3 | Phase 1,2 | Japan | ||
| 37 | JPRN-UMIN000030647 | 2017/12/30 | 2 April 2019 | Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial (CGD-RIST2) | Chronic granulomatous disease | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | National Center for Child Health and Development | Recruiting | Not applicable | 25years-old | Male and Female | 22 | Not selected | Japan | ||
| 38 | NCT03333486 | December 7, 2017 | 25 June 2018 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Recruiting | 1 Year | 75 Years | All | 58 | Phase 2 | United States |
| 39 | NCT03330795 | December 1, 2017 | 14 October 2019 | Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant | Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA Matched Cadaveric Donors (RTB-003) | Primary Immunodeficiency;PID | Biological: CD3/CD19 neg allogeneic BMT | National Institute of Allergy and Infectious Diseases (NIAID) | University of Pittsburgh | Recruiting | 10 Years | 45 Years | All | 8 | Phase 1/Phase 2 | United States |
| 40 | JPRN-UMIN000029324 | 2017/09/28 | 23 April 2019 | Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis | Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis - Clinical trial of thalidomide for CGD colitis | Chronic granulomatous disease associated colitis | Oral administration of thalidomide (1.5-3mg/kg), once a day Oral administration of placebo (1.5-3mg/kg), once a day | National Center for Child Heath and Development | Recruiting | 1years-old | Not applicable | Male and Female | 8 | Not selected | Japan | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | NCT03277313 | September 25, 2017 | 2 September 2019 | Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects | Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA;Biological: GAMMAGARD LIQUID | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire | Not recruiting | 2 Years | 15 Years | All | 44 | Phase 3 | United States |
| 42 | NCT03080480 | September 1, 2017 | 4 November 2019 | Pioglitazone Therapy for Chronic Granulomatous Disease | Efficacy and Safety of Pioglitazone Therapy for Chronic Granulomatous Disease Patients With Severe Infection. | Chronic Granulomatous Disease | Drug: Pioglitazone | Children's Hospital of Fudan University | Not recruiting | 1 Month | 18 Years | All | 3 | Phase 1/Phase 2 | China | |
| 43 | NCT03113760 | July 21, 2017 | 30 September 2019 | Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency | Multicenter, Double-blind, Placebo-controlled, Randomized Withdrawal Trial With Tadekinig Alfa (r-hIL-18BP) in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP Deficiency | NLRC4-MAS;XIAP Deficiency | Drug: Tadekinig alfa;Other: 0.9% sodium chloride | AB2 Bio Ltd. | Recruiting | N/A | 17 Years | All | 10 | Phase 3 | United States;Canada;Germany | |
| 44 | NCT03217617 | July 15, 2017 | 30 September 2019 | Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg) | Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg) | SCID, X Linked | Biological: TYF-IL-2Rg gene-modified autologous stem cells | Shenzhen Geno-Immune Medical Institute | Recruiting | 1 Month | 10 Years | Male | 10 | Phase 1/Phase 2 | China | |
| 45 | NCT03116347 | June 13, 2017 | 26 August 2019 | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric PIDD Subjects | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA;Biological: KIOVIG;Biological: Cuvitru | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire | Recruiting | 2 Years | 17 Years | All | 40 | Phase 4 | Czechia;Denmark;France;Greece;Slovakia;Sweden;United Kingdom |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | NCT03988426 | March 7, 2017 | 1 July 2019 | Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases | Clinical Phase 3 Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases. | Primary Immune Deficiency Disorder | Biological: Octanorm | Octapharma | Not recruiting | 18 Years | 70 Years | All | 25 | Phase 3 | Russian Federation | |
| 47 | NCT03112655 | February 24, 2017 | 30 September 2019 | Diagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: Early Test-of-cure | Diagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: WP4 Early Test-of-cure | African Trypanosomiasis;African; Trypanosomiasis, West;Sleeping Sickness; West African;Trypanosoma Brucei Gambiense; Infection | Diagnostic Test: RNA and neopterin detection | Institut de Recherche pour le Developpement | Institute of Tropical Medicine, Belgium;Institut National de Recherche Biomédicale. Kinshasa, République Démocratique du Congo;Ministry of Public Health, Democratic Republic of the Congo | Not recruiting | 15 Years | N/A | All | 88 | N/A | Congo, The Democratic Republic of the |
| 48 | EUCTR2016-003438-26-GB | 14/02/2017 | 19 November 2018 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru Pharmaceutical Form: Solution for injection INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | Baxalta US Inc. | Authorised | Female: yes Male: yes | 40 | Phase 4 | France;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | |||
| 49 | NCT03033745 | February 1, 2017 | 14 January 2019 | Safety and Tolerability of Higher Infusion Parameters of IgPro20 (Hizentra) in Subjects With Primary Immunodeficiency (PID) | An Open-label Multicenter Study to Evaluate the Safety and Tolerability of Higher Infusion Parameters of Immune Globulin Subcutaneous (Human), 20% Liquid (Hizentra®) in Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Drug: IgPro20 | CSL Behring | Not recruiting | 2 Years | N/A | All | 49 | Phase 4 | United States;Canada | |
| 50 | NCT03733249 | January 2017 | 28 October 2019 | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immunodeficiency;Anemia, Aplastic;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Drug: Rimiducid;Biological: rivogenlecleucel | Bellicum Pharmaceuticals | Recruiting | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;Saudi Arabia;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | NCT03054181 | December 22, 2016 | 4 March 2019 | Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO) | Facilitated Immunoglobulin Administration Registry and Outcomes Study | Primary Immunodeficiency;Secondary Immune Deficiency | Biological: HyQvia | GWT-TUD GmbH | Recruiting | N/A | N/A | All | 100 | Phase 3 | France;Germany;Italy | |
| 52 | NCT03005327 | December 2016 | 15 July 2019 | A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome | A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome | WHIM Syndrome | Drug: X4P-001 | X4 Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 15 | Phase 2 | United States;Australia | |
| 53 | NCT03037359 | December 2016 | 16 December 2017 | A Study About Low Blood Pressure in Patients With Primary Immunodeficiency Disease Treated With Immune Globulin Products | A Multicenter, Non-interventional, Observational, Prospective Study to Assess Hypotension in Patients With Primary Immunodeficiency Disease Treated With Bivigam™ (Human 10%) or Other Commercial Human 10% Immune Globulin (Intravenous) (IGIV) Products During Infusion and up to 72 Hours Post Infusion | Primary Immune Deficiency Disorder | Biological: Bivigam;Biological: Other | Biotest Pharmaceuticals Corporation | Recruiting | N/A | N/A | All | 200 | N/A | United States | |
| 54 | NCT03025789 | November 17, 2016 | 20 May 2019 | Fexinidazole in Human African Trypanosomiasis Due to T.b. Gambiense at Any Stage | An Open-label Study Assessing Effectiveness, Safety and Compliance With Fexinidazole in Patients With Human African Trypanosomiasis Due to T.b. Gambiense at Any Stage | Trypanosomiasis, African;Sleeping Sickness;Trypanosomiasis; Gambian | Drug: Fexinidazole | Drugs for Neglected Diseases | Sanofi | Recruiting | 6 Years | N/A | All | 174 | Phase 3 | Congo, The Democratic Republic of the;Guinea |
| 55 | NCT02881437 | November 11, 2016 | 15 July 2019 | IgG Level in Primary Immunodeficiency Switching From Standard SCIG to Every Other Week HyQvia | Assessment of the IgG Trough Level in Subjects With Primary Immunodeficiency Switching From Standard Subcutaneous Immunoglobulin (SCIG) to Every Other Week HyQvia | Primary Immunodeficiency | Drug: IgHy10 | University Hospital, Lille | Shire | Not recruiting | 18 Years | N/A | All | 22 | Phase 4 | France |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 56 | NCT03087955 | October 11, 2016 | 19 November 2018 | Prospective Study on Efficacy and Safety of SCYX-7158 in Patients Infected by Human African Trypanosomiasis Due to T.b. Gambiense | Efficacy and Safety Study of SCYX-7158 in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Gambiense: a Multicentre, Open-label, Prospective Study | Trypanosomiasis, African;Gambiense Trypanosomiasis;Sleeping Sickness | Drug: SCYX 7158 | Drugs for Neglected Diseases | Recruiting | 15 Years | N/A | All | 360 | Phase 2/Phase 3 | Congo, The Democratic Republic of the;Guinea | |
| 57 | EUCTR2015-003652-52-DE | 29/09/2016 | 14 May 2018 | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | Replacement therapy in patients with primary immunodeficiency disease (PID) MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: IgG Next Generation Product Code: BT595 Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Current Sponsor code: BT595 Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Biotest AG | Authorised | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;Germany;United Kingdom | |||
| 58 | EUCTR2015-005241-31-DE | 14/09/2016 | 26 November 2018 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTEST | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1 Level: PT Classification code 10003594 Term: Ataxia telangiectasia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate Pharmaceutical Form: Solution for blood fraction modification INN or Proposed INN: Dexamethasone sodium phosphate Current Sponsor code: DSP Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 25- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Intravenous use | EryDel S.p.A. | Authorised | Female: yes Male: yes | 180 | Phase 3 | United States;Spain;Costa Rica;Turkey;Israel;United Kingdom;Italy;India;Poland;Belgium;Australia;Germany;Tunisia;Norway | |||
| 59 | NCT01512888 | August 17, 2016 | 25 February 2019 | Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants | A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells | Severe Combined Immunodeficiency Disease, X-linked | Genetic: CL20-i4-EF1a-h?c-OPT;Drug: Busulfan;Device: CliniMacs | St. Jude Children's Research Hospital | National Heart, Lung, and Blood Institute (NHLBI);Assisi Foundation;California Institute for Regenerative Medicine (CIRM) | Recruiting | N/A | 24 Months | Male | 28 | Phase 1/Phase 2 | United States |
| 60 | EUCTR2015-003369-27-DE | 01/07/2016 | 11 June 2018 | A Phase I-IIa trial on low-dose IL-2 (Aldesleukin) treatment for immunological dysregulation in common variable immunodeficiency (CVID) | A Phase I-IIa trial on low-dose IL-2 (Aldesleukin) treatment for immunological dysregulation in common variable immunodeficiency (CVID) - REGAIN: REGulatory T cells and Aldesleukin for Immunodeficiency- associated eNteropathy | autoimmune enteropathy (AIE) in common variable immunodeficiency (CVID). MedDRA version: 19.0 Level: PT Classification code 10021449 Term: Immunodeficiency common variable System Organ Class: 10021428 - Immune system disorders MedDRA version: 19.0 Level: LLT Classification code 10017922 Term: Gastroenteropathy NOS System Organ Class: 100000004856 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: PROLEUKIN® S Product Name: Aldesleukin Pharmaceutical Form: Powder for solution for injection/infusion INN or Proposed INN: Aldesleukin CAS Number: 110942-02-4 Other descriptive name: ALDESLEUKIN Concentration unit: IU/ml international unit(s)/millilitre Concentration type: equal Concentration number: 18x10 6- | Universitätsklinikum Freiburg | Not Recruiting | Female: yes Male: yes | 6 | Phase 2 | Germany | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 61 | NCT02609932 | July 2016 | 25 February 2019 | Effect of IFN-? on Innate Immune Cells | Effect of Interferon-gamma 1-b on Innate Immune Cells | Chronic Granulomatous Disease | Drug: Administration of drug (Interferon-gamma 1-b) subcutaneously | University of Colorado, Denver | Not recruiting | 18 Years | 60 Years | All | 20 | Phase 1 | United States | |
| 62 | NCT02789397 | July 2016 | 11 June 2018 | Clinical Trial to Assess the Efficacy of Rituximab and Azathioprine in the Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) in Adult Patients With Common Variable Immunodeficiency (CVID) | Clinical Trial to Assess the Efficacy of Rituximab and Azathioprine in the Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) in Adult Patients With Common Variable Immunodeficiency (CVID) | Granulomatous and Lymphocytic Interstitial Lung Disease | Drug: Rituximab (RTX) and Azathioprine (AZA);Drug: Placebo Administration | Medical College of Wisconsin | Not recruiting | 18 Years | N/A | All | 0 | Phase 2 | ||
| 63 | NCT02737384 | June 14, 2016 | 11 June 2019 | Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID) | Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID): Selective Depletion of Naive Cells From the Graft | Combined Immunodeficiencies | Biological: Depletion in CD45RA graft donor | Assistance Publique - Hôpitaux de Paris | Not recruiting | 12 Months | 18 Years | All | 4 | Phase 2 | France | |
| 64 | JPRN-UMIN000022688 | 2016/06/10 | 2 April 2019 | A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease | Chronic granulomatous disease | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | National Center for Child Health and Development | Recruiting | Not applicable | 25years-old | Male and Female | 9 | Not selected | Japan | ||
| 65 | NCT02806986 | June 2016 | 22 July 2019 | Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency | A Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Biological: IGSC 20% | Grifols Therapeutics LLC | Not recruiting | 2 Years | 75 Years | All | 61 | Phase 3 | Australia;Czechia;France;Germany;Hungary;Poland;Spain;Sweden;United Kingdom;Czech Republic | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 66 | NCT02822495 | June 2016 | 15 April 2019 | Expanded Access Protocol for Tabelecleucel for Patients With Epstein-Barr Virus-Associated Viremia or Malignancies | Expanded Access Protocol for Providing Tabelecleucel to Patients With Epstein-Barr Virus-Associated Viremia or Malignancies for Whom There Are No Appropriate Alternative Therapies | Epstein-Barr Virus (EBV) Infections;Lymphoproliferative Disorders;EBV+ Associated Lymphoma;EBV+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD);Epstein-Barr Viremia;Lymphoma, AIDS-related;Epstein-Barr Virus-associated Lymphoproliferative Disease (EBV+ LPD) With Primary Immunodeficiency (PID);Leiomyosarcoma (LMS);Nasopharyngeal Carcinoma (NPC);Epstein-Barr Virus-associated Lymphoproliferative Disease (EBV+ LPD) With Acquired Immunodeficiency (AID);Solid Organ Transplant Complications;Stem Cell Transplant Complications | Biological: tabelecleucel | Atara Biotherapeutics | Not recruiting | N/A | N/A | All | Phase 1/Phase 2 | United States | ||
| 67 | NCT03019809 | June 2016 | 18 December 2018 | A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients | A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome. | Wiskott-Aldrich Syndrome;Hematopoietic Stem Cell Transplantation;Graft Failure | Biological: Plerixafor for Conditioning before HSCT.;Biological: G-CSF for Conditioning before HSCT. | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | Recruiting | 1 Month | 19 Years | All | 30 | Phase 2 | Russian Federation | |
| 68 | EUCTR2015-003290-15-GB | 12/05/2016 | 22 May 2017 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 20.0 Level: PT Classification code 10061598 Term: Immunodeficiency System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0 Level: LLT Classification code 10045792 Term: Unspecified disorder of immune mechanism System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% Pharmaceutical Form: Solution for infusion INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified Current Sponsor code: GRF6017 Concentration unit: % percent Concentration type: equal Concentration number: 20- | Grifols Therapeutics Inc. | Authorised | Female: yes Male: yes | 60 | Phase 3 | France;Hungary;Czech Republic;Poland;Spain;Australia;Germany;United Kingdom;Sweden | |||
| 69 | EUCTR2015-002491-24-DE | 11/05/2016 | 7 January 2019 | Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease | Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease - SAIL | CVID patients confirmed according to ESID/PAGID criteria or related disorders which fulfill the diagnostic criteria for CVID and interstitial lung disease or granuloma diagnosed by chest CT positive for nodules, lines or ground-glass signs MedDRA version: 18.1 Level: PT Classification code 10022611 Term: Interstitial lung disease System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders MedDRA version: 18.1 Level: PT Classification code 10021449 Term: Immunodeficiency common variable System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: ORENCIA® 125 mg solution for injection in pre-filled syringe Product Name: Abatacept Product Code: BMS-188667 Pharmaceutical Form: Solution for injection in pre-filled syringe | Universitätsklinikum Freiburg, vertreten durch den Leitenden Ärztlichen Direktor | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | Germany | |||
| 70 | NCT02627300 | March 2016 | 7 October 2019 | Study of Octanorm Subcutaneous IG in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 Trial | Clinical Phase III Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 Trial | Primary Immunodeficiency Disease | Drug: Octanorm 16.5% | Octapharma | Not recruiting | N/A | N/A | All | 21 | Phase 3 | United States;Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 71 | NCT02123615 | January 2016 | 3 August 2015 | ASIS for GAMMAGARD in Primary Immunodeficiency | ASIS for GAMMAGARD in Primary Immunodeficiency | Primary Immunodeficiency | Drug: Gadolinium For abdomen;Drug: Gadolinium For lower back;Drug: Efficacy of Gammagard subcutaneously at Week 12;Drug: Efficacy of Gammagard subcutaneously at Week 24;Drug: Efficacy of Gammagard subcutaneously at Week 36;Drug: Efficacy of Gammagard subdermally at Week 36;Drug: Efficacy of Gammagard subdermally at Week 12;Drug: Efficacy of Gammagard subdermally at Week 24;Drug: Adverse Reactions of Gammagard subcutaneously at Week 12;Drug: Adverse Reactions of Gammagard subcutaneously at Week 24;Drug: Adverse Reactions of Gammagard subcutaneously at Week 36;Drug: Adverse Reactions of Gammagard subdermally at Week 12;Drug: Adverse Reactions of Gammagard subdermally at Week 24;Drug: Adverse Reactions of Gammagard subdermally at Week 36 | ASIS Corporation | Not recruiting | 21 Years | 65 Years | Both | 60 | Phase 1/Phase 2 | United States | |
| 72 | EUCTR2016-001631-12-Outside-EU/EEA | 6 February 2017 | The pharmacokinetics and safety of intravenous IgPro10 in Japanese subjects with primary immunodeficiency | Prospective open-label single-arm study of the pharmacokinetics and safety of intravenous IgPro10 in Japanese subjects with primary immunodeficiency | Primary immunodeficiency MedDRA version: 19.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Privigen Product Name: Privigen; immunoglobulin intravenous (human) Product Code: IgPro10 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | CSL Behring KK | Not Available | Female: yes Male: yes | 10 | Phase 3 | Japan | ||||
| 73 | EUCTR2015-002001-11-DE | 22/12/2015 | 2 October 2017 | Study of MEDI4736, when used alone or in combination with Tremelimumab, as second line chemotherapy in metastatic Pancreatic Ductal Adenocarcinoma | A Phase II Open-Label, Multi-Center Study of MEDI4736 Evaluated as Single Agent or in Combination with Tremelimumab in Patients with Metastatic Pancreatic Ductal Adenocarcinoma - ALPS | Adult patients (aged =18 years) with histologically or cytologically confirmed metastatic PDAC, and had tumor progression following prior standard first-line 5-FU-containing or gemcitabine-containing chemotherapy. MedDRA version: 18.0 Level: LLT Classification code 10033605 Term: Pancreatic cancer metastatic System Organ Class: 100000004864 ;Therapeutic area: Diseases [C] - Cancer [C04] | Product Name: MEDI4736 Product Code: MEDI4736 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: durvalumab CAS Number: 1428935-60-7 Current Sponsor code: MEDI4736 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: -50 Product Name: Tremelimumab Product Code: MEDI1123 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Tremelimumab CAS Number: 745013-19-6 Current Sponsor code: MEDI1123 Other descriptive name: MEDI1123 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 20- | AstraZeneca AB | Not Recruiting | Female: yes Male: yes | 130 | Phase 2 | United States;Canada;Spain;Netherlands;Germany;Japan;Korea, Republic of | |||
| 74 | NCT02629120 | December 10, 2015 | 11 November 2019 | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease Transplant | Drug: Campath;Drug: Busulfan IV;Drug: Sirolimus;Drug: cyclophosphamide | National Institute of Allergy and Infectious Diseases (NIAID) | Recruiting | 4 Years | 65 Years | All | 50 | Phase 1/Phase 2 | United States | |
| 75 | NCT02604810 | December 2015 | 1 October 2018 | Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary Immunodeficiency | An Open-label, Multi-center Study to Evaluate the Safety and Pharmacokinetics of IGSC 20% Administered for 6 Months in Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Biological: IGIV-C 10%;Biological: IGSC 20% | Grifols Therapeutics LLC | Not recruiting | 2 Years | 75 Years | All | 53 | Phase 3 | United States;Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 76 | NCT02579967 | November 19, 2015 | 2 September 2019 | Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies | Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies | Primary T-cell Immunodeficiency Disorders;Common Variable Immunodeficiency;Immune System Diseases;Autoimmune Lymphoproliferative;Lymphoproliferative Disorders | Drug: Immunosuppression Only Conditioning - Closed with amendment L;Drug: Reduced Intensity Conditioning;Drug: Myeloablative Conditioning-Closed with amendment L;Drug: GVHD Prophylaxis;Procedure: Allo BMT | National Cancer Institute (NCI) | Recruiting | 4 Years | 75 Years | All | 224 | Phase 2 | United States | |
| 77 | NCT02593188 | November 12, 2015 | 25 February 2019 | Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global) | Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global) | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA | Baxalta now part of Shire | Recruiting | 16 Years | N/A | All | 250 | Phase 3 | United States | |
| 78 | NCT02234934 | October 29, 2015 | 11 November 2019 | Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease | A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease | Granulomatous Disease, Chronic, X-linked | Biological: Lentiviral G1XCGD Gene Therapy | University of California, Los Angeles | Boston Children’s Hospital;National Institute of Allergy and Infectious Diseases (NIAID);Genethon;California Institute for Regenerative Medicine (CIRM) | Not recruiting | 23 Months | N/A | Male | 10 | Phase 1/Phase 2 | United States |
| 79 | EUCTR2015-002356-27-IT | 16/10/2015 | 7 December 2015 | Evaluation of safety and efficacy of the combination of Ibuprofen (IBU), G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by XCGD | A multicentric, exploratory, non-randomised, non-controlled, prospective, open-label phase II, study evaluating safety and efficacy of IBU, G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by X-CGD. | X-linked chronic granulomatous disease MedDRA version: 18.0 Level: PT Classification code 10008906 Term: Chronic granulomatous disease System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Ibuprofen Pharmaceutical Form: Film-coated tablet INN or Proposed INN: IBUPROFEN CAS Number: 15687-27-1 Current Sponsor code: NA Other descriptive name: NA Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 200- Trade Name: MYELOSTIM 34 milions UI/ml, powder and solvent for solution for injection or infusion Product Name: MYELOSTIM 34 milions UI/ml - powder and solvent for solution for injection/infusion Pharmaceutical Form: Powder and solvent for solution for injection/infusion INN or Proposed INN: LENOGRASTIM CAS Number: 135968-09-1 Concentration unit: U/ml unit(s)/millilitre Concentration type: equal Concentration number: 34- Trade Name: Mozobil 20mg/mL vial (injectable solution for subcutaneous use) Product Name: Mozobil 20mg/mL vial (injectable solution, subcutaneous use) Pharmaceutical Form: Solution for injection INN or Proposed INN: Plerixafor CAS Number: 110078-46-1 Other descriptive name: Plerixafor Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 20- Product Name: Pantoprazolo 20 mg gastro-resistant tablets Pharmaceutical Form: Gastro-resistant tablet INN or Proposed INN: Pantoprazole CAS Number: 102625-70-7 Other descriptive name: PANTOPRAZOLE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- | Ospedale San Raffaele | Authorised | Female: no Male: yes | 3 | Phase 2 | Italy | |||
| 80 | NCT04136028 | September 25, 2015 | 4 November 2019 | IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous Disease | A Retrospective Analysis of Efficacy and Safety of Interleukin-1 Receptor Inhibitor for the Treatment of Granulomatous Complications in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Kineret | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | Not recruiting | N/A | 18 Years | All | 13 | Early Phase 1 | Russian Federation | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 81 | NCT01884311 | August 20, 2015 | 16 December 2017 | Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Subgam-VF in Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyperimmunoglobulin M Syndrome | Biological: Subgam | Bio Products Laboratory | Not recruiting | 2 Years | 75 Years | All | 38 | Phase 3 | United States | |
| 82 | NCT02369978 | August 2015 | 16 December 2017 | CHICAMOCHA 3 - Equivalence of Usual Interventions for Trypanosomiasis (EQUITY) | Cardiovascular Health Investigation and Collaboration From Countries of America to Assess the Markers and Outcomes of Chagas Disease (CHICAMOCHA-3) - EQUITY (Equivalence of Usual Interventions for Trypanosomiasis) | Chagas Disease | Drug: Nifurtimox;Drug: Benznidazole;Drug: Placebo | Universidad Autónoma de Bucaramanga | Fundación Cardioinfantil Instituto de Cardiología;Instituto Nacional de Salud (Colombia);Instituto Nacional de Parasitologia Dr. Mario Fatala Chaben | Recruiting | 20 Years | 55 Years | All | 500 | Phase 2/Phase 3 | Colombia |
| 83 | EUCTR2014-003746-27-DE | 15/04/2015 | 30 April 2018 | A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using an infusion pump or syringe | A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using pump or rapid push - Gammanorm in immunodeficient patients: quality of life using infusion by pump or rapid push | immunodeficiency syndrome MedDRA version: 19.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Trade Name: Gammanorm 165 mg/mL | Octapharma Pharmazeutika Produktionsges.m.b.H, Oberlaaer Strasse 235, A-1100 Vienna, Austria | Not Recruiting | Female: yes Male: yes | 40 | Phase 3;Phase 4 | Australia;Germany;Italy;United Kingdom | |||
| 84 | NCT02349906 | April 2015 | 4 November 2019 | Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases | Clinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant Diseases | Primary Immunodeficiencies;Inborn Errors of Metabolism;Haemoglobinopathies;Bone Marrow Failure Syndromes | Drug: Treosulfan;Drug: Busilvex | medac GmbH | Celerion;Venn Life Sciences;Syneos Health | Recruiting | N/A | 17 Years | All | 100 | Phase 2 | Austria;Czechia;Germany;Italy;Poland;Czech Republic |
| 85 | NCT02490956 | April 2015 | 3 August 2015 | Diagnostic Immunization With Rabies Vaccine in Patients With PID | Diagnostic Immunization With Rabies Vaccine in Patients With Primary Immunodeficiency Disorders | Primary Immunodeficiency | Biological: Verorab® (PVRV; Purified Vero Cell Vaccine) | Chulalongkorn University | Recruiting | 12 Months | 60 Years | Both | 40 | Phase 4 | Thailand | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 86 | NCT03198195 | March 10, 2015 | 16 December 2017 | Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome | Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Procedure: cyclophosphamide | Capital Research Institute of Pediatrics | Recruiting | 5 Months | 10 Years | All | 5 | N/A | ||
| 87 | NCT02571062 | March 2015 | 19 October 2015 | Bioequivalence Study - Reference Clinical Fexinidazole Tablet Versus Proposed Market Formulation | A Bioequivalence Study of the Reference Clinical Fexinidazole Tablet vs Proposed Market Formulation in Healthy Male Volunteers of African Sub-Saharan Origin:an Open-label,Randomized,Two-treatment,Single Dose,Replicate Design,Fed Condition | Trypanosomiasis, African | Drug: Fexinidazole | Drugs for Neglected Diseases | Not recruiting | 18 Years | 45 Years | Male | 30 | Phase 1 | France | |
| 88 | NCT02972281 | March 2015 | 15 July 2019 | Systematic Search for Primary Immunodeficiency in Adults With Infections | Systematic Search for Primary Immunodeficiency in Adults With Unexplained Recurrent and/or Severe Infections With Encapsulated Bacteria | Complement Deficiency;Antibody Deficiency;Chronic Sinus Infection;Meningitis, Bacterial;Pneumonia, Bacterial;Otitis Media;Streptococcal Infection;Neisseria Infections;Haemophilus Influenza;Pneumococcal Infections | Biological: Immunological diagnosis tests | University Hospital, Lille | Imagine Institute;Octapharma;CSL Behring;Laboratoire français de Fractionnement et de Biotechnologies;Air Liquide SA;The Binding Site Ltd | Recruiting | 18 Years | 65 Years | All | 240 | N/A | France |
| 89 | NCT02909244 | February 2015 | 16 December 2017 | Study of Gut Microbiota in Primary Immune Deficiency, Possibly Associated With Inflammatory Bowel Disease | Study of Gut Microbiota in Primary Immune Deficiencies, Possibly Associated With Inflammatory Bowel Disease (Chronic Granulomatous Disease, XIAP Gene Deficiency, or TTC7A Gene Deficiency) | Primary Immune Deficiencies | Biological: Biological sampling | Imagine Institute | Saint Antoine University Hospital;Assistance Publique - Hôpitaux de Paris | Not recruiting | N/A | N/A | All | 51 | N/A | France |
| 90 | NCT02162420 | January 2015 | 7 October 2019 | Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia | Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia | Dyskeratosis Congenita;Aplastic Anemia | Drug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Stem Cell Transplant;Drug: Anti-thymocyte globulin | Masonic Cancer Center, University of Minnesota | Recruiting | N/A | 70 Years | All | 50 | N/A | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 91 | NCT02244450 | December 2014 | 20 August 2018 | Generalized Neonatal Screening of Severe Combined Immunodeficiencies | Evaluation of the Clinical Utility and Cost Effectiveness Ratio of Generalized Neonatal Screening for Severe Combined Immunodeficiencies (SCID) by Quantification of TRECs on Guthrie Cards | Severe Combined Immunodeficiency, Atypical | Biological: SCID screening | Nantes University Hospital | Not recruiting | N/A | 18 Months | All | 190539 | N/A | France | |
| 92 | EUCTR2013-005508-33-PL | 17/11/2014 | 28 February 2019 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0 Level: HLT Classification code 10021606 Term: Inborn errors of metabolism NEC System Organ Class: 100000004850 MedDRA version: 20.0 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 MedDRA version: 20.0 Level: HLT Classification code 10018903 Term: Haemoglobinopathies congenital System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: TREOSULFAN CAS Number: 299-75-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- Trade Name: Ovastat 5000 (Treosulfan injection) Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: TREOSULFAN CAS Number: 299-75-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5000- Trade Name: Busilvex Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: BUSULFAN CAS Number: 55-98-1 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 6- | medac GmbH | Authorised | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | |||
| 93 | NCT02282904 | November 4, 2014 | 1 July 2019 | Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 2 Years | 65 Years | All | 12 | Phase 1/Phase 2 | United States | |
| 94 | NCT03201380 | November 1, 2014 | 16 December 2017 | Evaluation of the Practice of the TEP Choline at Patients With Prostate Cancer | Evaluation of the Practice of the TEP Choline in Provence - Alps and Côte d'Azur at Patients With Prostate Cancer - Multicentre Retrospective Study. | Prostate Cancer | Other: TEP scanner with choline | Institut Paoli-Calmettes | Not recruiting | 18 Years | N/A | Male | 300 | N/A | ||
| 95 | EUCTR2013-005508-33-DE | 29/09/2014 | 20 August 2018 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0 Level: HLT Classification code 10021606 Term: Inborn errors of metabolism NEC System Organ Class: 100000004850 MedDRA version: 20.0 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 MedDRA version: 20.0 Level: HLT Classification code 10018903 Term: Haemoglobinopathies congenital System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: TREOSULFAN CAS Number: 299-75-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- Trade Name: Ovastat 5000 (Treosulfan injection) Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: TREOSULFAN CAS Number: 299-75-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5000- Trade Name: Busilvex Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: BUSULFAN CAS Number: 55-98-1 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 6- | medac Gesellschaft für klinische Spezialpräparate mbH | Authorised | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 96 | NCT02231879 | September 3, 2014 | 3 June 2019 | Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome | A Phase III Double-Blind Randomized Crossover Study of Plerixafor Versus G-CSF in the Treatment of Patients With WHIM Syndrome. | Myelokathexis;Infections;Neutropenia;Warts;Hypogammaglobulinemia | Drug: Plerixafor;Drug: G-CSF | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 10 Years | 75 Years | All | 19 | Phase 2/Phase 3 | United States | |
| 97 | NCT02179359 | September 2, 2014 | 7 October 2019 | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders | Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic Disorders | Drug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative Regimen | Masonic Cancer Center, University of Minnesota | Recruiting | N/A | 55 Years | All | 25 | N/A | United States | |
| 98 | NCT02199496 | July 23, 2014 | 9 September 2019 | Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | An Open-Label Phase I/II Pilot Study to Assess the Safety/Tolerability and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | Gastrointestinal Inflammation Associated With CVID;CVID Enteropathy | Biological: Stelara (ustekinumab) | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 18 Years | 75 Years | All | 5 | Phase 1/Phase 2 | United States | |
| 99 | NCT02177760 | July 2014 | 14 December 2015 | Sirolimus Prophylaxis for aGVHD in TME SCID | Sirolimus in Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID) Infants Receiving Unconditioned Hematopoietic Stem Cell Transplant (HSCT) | Severe Combined Immunodeficiency;Transplacental Maternal Engraftment;Stem Cell Transplant | Drug: Sirolimus | University of California, San Francisco | Not recruiting | N/A | 1 Year | Both | 0 | Phase 2 | United States | |
| 100 | NCT02498782 | July 2014 | 3 August 2015 | Study to Evaluate Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chagas Disease | Phase 2, Randomized, Multicenter, Placebo-controlled, Safety and Efficacy Study to Evaluate Six Oral Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chronic Indeterminate Chagas Disease. | Chagas Disease;Trypanosomiasis, South American;South American Trypanosomiasis;Disease, Chagas | Drug: Fexinidazole;Drug: Placebo | Drugs for Neglected Diseases | Recruiting | 18 Years | 50 Years | Both | 140 | Phase 2 | Bolivia | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 101 | EUCTR2014-000274-20-GB | 14/05/2014 | 31 January 2017 | Long-term follow-up of the WAS gene therapy study | LONG TERM SAFETY FOLLOW UP OF PATIENTS ENROLLED IN THE PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME(GTG 002-07 AND GTG 003-08) - Long-term follow-up of the WAS gene therapy study, version 2.0 | Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages. MedDRA version: 19.1 Level: PT Classification code 10047992 Term: Wiskott-Aldrich syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Pharmaceutical Form: Solution for injection Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Concentration type: not less then Concentration number: 0.5E06 cells /Kg- | Genethon | Authorised | Female: no Male: yes | 10 | Phase 2 | United Kingdom | |||
| 102 | EUCTR2013-003257-20-GB | 02/05/2014 | 28 February 2019 | TREOSULFAN LEVELS IN CHILDREN UNDERGOING STEM CELL TRANSPLANTATION | Evaluation of Treosulfan pharmacokinetics (PK) in children undergoing allogeneic haematopoietic stem cell transplantation (HSCT) - Treosulfan PK in children | Any paediatric disease with an indication to an allogeneic stem cell transplantation (inclusing leukaemia, primary immunodeficiencies, metabolic disorders and autoimmune or genetic inflammatory bowel disorders).;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Treosulfan Product Name: TREOSULFAN Pharmaceutical Form: Injection INN or Proposed INN: Treosulfan CAS Number: 299-75-2 | GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST | Not Recruiting | Female: yes Male: yes | 90 | Phase 4 | United Kingdom | |||
| 103 | NCT02169557 | May 2014 | 17 October 2016 | Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal Study | Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal Study | Human African Trypanosomiasis (HAT) | Drug: Fexinidazole | Drugs for Neglected Diseases | Not recruiting | 15 Years | N/A | Both | 230 | Phase 2/Phase 3 | Congo, The Democratic Republic of the | |
| 104 | NCT02184689 | May 2014 | 17 October 2016 | Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study | Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study | Human African Trypanosomiasis (HAT) | Drug: fexinidazole | Drugs for Neglected Diseases | Not recruiting | 6 Years | 14 Years | Both | 125 | Phase 2/Phase 3 | Congo, The Democratic Republic of the | |
| 105 | NCT02065869 | April 2014 | 4 November 2019 | Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant | Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | Not recruiting | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;United Kingdom;Germany;Spain;United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 106 | NCT02180763 | April 2014 | 10 September 2018 | Gammanorm Quality of Life Study in Immunodeficient Patients Using Rapid Push or Pumps | A Randomized, Cross-over Study to Compare Quality of Life and Satisfaction in Primary Immunodeficient Patients Treated With Subcutaneous Injections of Gammanorm® 165 mg/mL According to the Delivery Device: Injections Using Pump or Rapid Push. | Primary Immunodeficiency (PID) | Biological: Gammanorm | Octapharma | Not recruiting | 18 Years | N/A | All | 31 | Phase 4 | France | |
| 107 | NCT02510404 | April 2014 | 25 February 2019 | Multivirus-specific Cytotoxic T Lymphocytes (mCTL) | Treatment of EBV, CMV, and Adenovirus Infections in Primary Immunodeficiency Disorders With Viral-specific Cytotoxic T-Lymphocytes | Refractory Viral Infections | Biological: mCTLs | Catherine Bollard | Children's Research Institute | Not recruiting | N/A | 45 Years | All | 1 | Phase 1 | United States |
| 108 | JPRN-UMIN000013102 | 2014/03/10 | 2 April 2019 | Efficacy of Zoledronate for RAS associated ALPS like disease (RALD) | RAS associated ALPS like disease (RALD) | intravenous drip infusion of Zoledronate | Department of Pediatrics, Tokyo Medical and Dental University | Not Recruiting | Not applicable | Not applicable | Male and Female | 5 | Not selected | Japan | ||
| 109 | NCT01888484 | March 2014 | 9 September 2019 | Study of Octanorm Subcutaneous IG in Patients With PID | Clinical Phase III Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm 16.5%) In Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder | Biological: octanorm 16.5% | Octapharma | Not recruiting | 2 Years | 75 Years | All | 64 | Phase 3 | United States;Canada;Czechia;Hungary;Poland;Russian Federation;Slovakia;Czech Republic | |
| 110 | NCT01962415 | February 4, 2014 | 4 November 2019 | Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults =40 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation | Primary Immunodeficiency (PID);Congenital Bone Marrow Failure Syndromes;Inherited Metabolic Disorders (IMD);Hereditary Anemias;Inflammatory Conditions | Drug: Hydroxyurea;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Melphalan;Drug: Thiotepa | Paul Szabolcs | Recruiting | 2 Months | 40 Years | All | 100 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 111 | NCT01814800 | February 2014 | 19 October 2017 | Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD) | An Open Label, Multicenter, Study to Evaluate the Pharmacokinetics, Efficacy and Safety of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD) | Primary Immune Deficiency Disorder | Biological: RI-002 | ADMA Biologics, Inc. | Not recruiting | 2 Years | 75 Years | All | 59 | Phase 3 | United States | |
| 112 | NCT01963143 | February 2014 | 16 December 2017 | Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyper-IgM Syndrome | Biological: Gammaplex (5%);Biological: Gammaplex 10 | Bio Products Laboratory | Not recruiting | 2 Years | 55 Years | All | 48 | Phase 3 | United States;Hungary;United Kingdom | |
| 113 | NCT01917708 | January 2014 | 19 November 2018 | BMT Abatacept for Non-Malignant Diseases | Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases | Hurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell Disease | Drug: Abatacept | Emory University | Not recruiting | N/A | 21 Years | All | 10 | Phase 1 | United States | |
| 114 | EUCTR2014-003409-13-Outside-EU/EEA | 12 January 2015 | Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects with Primary Immunodeficiency (Japan Study) | A Multicenter Study of Long-Term Clinical Outcomes of Immune Globulin Subcutaneous (Human) (SCIG) IgPro20 in Subjects with Primary Immunodeficiency | Primary Immune Deficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Hizentra® Pharmaceutical Form: Solution for injection INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: Human Normal Immunoglobulin Concentration unit: % (W/V) percent weight/volume Concentration type: equal Concentration number: 20- | CSL Behring | Not Available | Female: yes Male: yes | 22 | Japan | |||||
| 115 | NCT01420627 | December 2013 | 9 September 2019 | EZN-2279 in Patients With ADA-SCID | A Study of EZN-2279 (Polyethylene Glycol Recombinant Adenosine Deaminase [PEG-rADA]) Administered as a Weekly Intramuscular Injection in Patients With Adenosine Deaminase (ADA)-Deficient Combined Immunodeficiency | ADA-SCID;Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency | Biological: EZN-2279;Biological: Adagen | Leadiant Biosciences, Inc. | Not recruiting | N/A | N/A | All | 6 | Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 116 | NCT01998633 | December 2013 | 11 June 2018 | Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204) | Reduced-Intensity Conditioning for Children and Adults With Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (RICHI) (BMT CTN #1204) | Hemophagocytic Lymphohistiocytosis;Chronic Active Epstein-Barr Virus Infection;Chronic Granulomatous Disease;HIGM-1;Leukocyte Adhesion Deficiency;IPEX | Biological: Hematopoietic Stem Cell Transplant | Medical College of Wisconsin | National Heart, Lung, and Blood Institute (NHLBI);Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program | Not recruiting | 4 Months | 45 Years | All | 47 | Phase 2 | United States;Canada |
| 117 | EUCTR2013-000961-36-DE | 15/11/2013 | 3 April 2017 | See the full title | Tolerability and safety evaluation of the administration of Ig VENA at high infusion rates. Open label phase III study. - Ig VENA Infusion Speed | Patients affected by primary or secondary immunodeficiency (ID) or patients affected by Primary Immune Thrombocytopenia (ITP) MedDRA version: 16.1 Level: PT Classification code 10054979 Term: Secondary immunodeficiency System Organ Class: 10021428 - Immune system disorders MedDRA version: 16.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1 Level: PT Classification code 10021245 Term: Idiopathic thrombocytopenic purpura System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Ig VENA 50 g/l solution for infusion 100 ml vial + infusion set | Kedrion SpA | Not Recruiting | Female: yes Male: yes | 35 | Phase 3 | Germany;Italy | |||
| 118 | NCT02054832 | November 2013 | 6 October 2015 | Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch | A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch | Glycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0 | Dietary Supplement: Glycosade | John Mitchell | Not recruiting | 2 Years | 50 Years | Both | 11 | N/A | Canada | |
| 119 | EUCTR2013-000883-27-NO | 16/08/2013 | 26 August 2013 | The effects of the local acting antibiotic Rifaximin on markers of inflammation in the blood by changing the bacterial composition in the gut, in patients with common variable immunodeficiency. | EFFECTS OF RIFAXIMIN, BY MODULATION OF THE GUT MICROBIOTA, ON MARKERS OF SYSTEMIC INFLAMMATION IN PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY - AN EXPLORATORY OPEN-LABEL RANDOMIZED CONTROLLED TRIAL | Adult patients with the diagnosis of Common variable deficiency and fulfill the inclusion and exclusion criteria, will be invited to participate in the study.;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Xifaxan Pharmaceutical Form: Coated tablet | Oslo University hospital | Authorised | Female: yes Male: yes | Norway | |||||
| 120 | EUCTR2013-000620-34-IT | 05/08/2013 | 12 May 2014 | A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromes | A Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes - NA | Primary Immunofediciency (PID) syndromes MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Human Normal Immunoglobulin for subcutaneous administration Product Code: LFB-IgSC Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human Normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 250- | LFB Biotechnologies | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | France;Hungary;Poland;Ukraine;Germany;United Kingdom;Italy | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 121 | EUCTR2012-005727-32-NL | 25/07/2013 | 10 July 2015 | The pharmacokinetics (the behaviour in the body) and safety of the intravenous immunoglobulin product Nanogam 100 mg/ml | Pharmacokinetics and safety of the intravenous human immunoglobulin product Nanogam 100 mg/ml - PK and safety of Nanogam 100 mg/ml | Primary a- or hypogammaglobulinemia MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 100000004870 MedDRA version: 14.1 Level: LLT Classification code 10010509 Term: Congenital hypogammaglobulinemia System Organ Class: 100000004850 MedDRA version: 14.1 Level: LLT Classification code 10071133 Term: Congenital agammaglobulinemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Nanogam 100 mg/ml Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Other descriptive name: IVIG Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Trade Name: Nanogam® 50 mg/ml Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Other descriptive name: IVIG Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | Sanquin Blood Supply Foundation | Not Recruiting | Female: yes Male: yes | Netherlands | |||||
| 122 | EUCTR2011-004154-25-IT | 25/06/2013 | 20 June 2016 | Clinical study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiency | A prospective, open-label, phase I/II study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiency | Patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia. MedDRA version: 14.1 Level: PT Classification code 10016075 Term: Factor I deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Fibrinogen Concentrate from Human Plasma Product Code: BT524 Pharmaceutical Form: Powder for solution for injection/infusion INN or Proposed INN: Human Fibrinogen Concentrate CAS Number: 9001-32-5 Current Sponsor code: BT524 Other descriptive name: Human Fibrinogen Concentrate Concentration unit: mg/ml milligram(s)/millilitre Concentration type: range Concentration number: 14-26 | Biotest AG | Not Recruiting | Female: yes Male: yes | 20 | Phase 1;Phase 2 | Egypt;Lebanon;Germany;Italy | |||
| 123 | EUCTR2012-005180-27-GB | 24/06/2013 | 3 July 2017 | Roxadustat in the Treatment of Anemia in Chronic Kidney Disease Patients | A Phase 3, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of Roxadustat for the Treatment of Anemia in Chronic Kidney Disease Patients not on Dialysis - Alps | Anemia in CKD patients not on dialysis MedDRA version: 20.0 Level: LLT Classification code 10002272 Term: Anemia System Organ Class: 100000004851 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: roxadustat Product Code: FG-4592/ASP1517 - 20 mg Pharmaceutical Form: Tablet INN or Proposed INN: roxadustat CAS Number: 808118-40-3 Current Sponsor code: FG-4592 Other descriptive name: ASP1517 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Product Name: roxadustat Product Code: FG-4592/ASP1517 - 50 mg Pharmaceutical Form: Tablet INN or Proposed INN: roxadustat CAS Number: 808118-40-3 Current Sponsor code: FG-4592 Other descriptive name: ASP1517 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Product Name: roxadustat Product Code: FG-4592/ASP1517 - 100 mg Pharmaceutical Form: Tablet INN or Proposed INN: roxadustat CAS Number: 808118-40-3 Current Sponsor code: FG-4592 Other descriptive name: ASP1517 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Astellas Pharma Europe B.V. | Authorised | Female: yes Male: yes | 600 | Phase 3 | Serbia;Panama;Belarus;Estonia;Greece;Spain;Guatemala;Ukraine;Turkey;Russian Federation;Colombia;Italy;United Kingdom;Hungary;Poland;Belgium;Romania;Dominican Republic;Peru;South Africa;Bulgaria;Georgia | |||
| 124 | NCT01852370 | June 20, 2013 | 18 March 2019 | Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases | Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric Donors | Severe Combined Immunodeficiency (SCID);Immunodeficiency With Predominant T-cell Defect, Unspecified;Severe Chronic Neutropenia;Chronic Granulomatous Disease (CGD);Hyper IgE Syndromes;Hyper IgM Deficiencies;Wiskott-Aldrich Syndrome;Mendelian Susceptibility to Mycobacterial Disease;Common Variable Immune Deficiency (CVID) | Biological: CD3/CD19 negative allogeneic hematopoietic stem cells | Paul Szabolcs | Recruiting | 5 Years | 45 Years | All | 16 | Phase 1/Phase 2 | United States | |
| 125 | EUCTR2012-001725-26-DE | 12/03/2013 | 9 September 2013 | A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector | A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector - gene therapy for X-CGD | Chronic granulomatous disease (CGD) is a congenital immunodeficiency, in which neutrophil granulocytes and monocytes are not capable of producing reactive oxygen species and therefore are unable to kill phagocytized bacteria or fungi. MedDRA version: 14.1 Level: PT Classification code 10008906 Term: Chronic granulomatous disease System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: genetically modified autologous blood stem cells Product Code: somatic gene-therapy by X-CGD Pharmaceutical Form: Suspension for infusion INN or Proposed INN: G1XCG Other descriptive name: Genetically modified autologous blood stem cells Concentration unit: Other Concentration type: range Concentration number: 100-500 | Johann Wolfgang Goethe-University | Authorised | Female: yes Male: yes | 5 | Phase 1/2 | Germany | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 126 | NCT01821781 | March 2013 | 8 April 2019 | Immune Disorder HSCT Protocol | A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime | Immune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Hyper-IgM;DiGeorge Syndrome;Chediak-Higashi Syndrome;Common Variable Immune Deficiency;Immune Dysregulatory Disorders;Hemophagocytic Lymphohistiocytosis;IPEX;Autoimmune Lymphoproliferative Syndrome;X-linked Lymphoproliferative Syndrome | Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan | Washington University School of Medicine | Recruiting | N/A | 21 Years | All | 20 | Phase 2 | United States | |
| 127 | NCT01966367 | March 2013 | 25 March 2019 | CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation | CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant Disease | Bone Marrow Failure Syndrome;Severe Aplastic Anemia;Severe Congenital Neutropenia;Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Schwachman Diamond Syndrome;Primary Immunodeficiency Syndromes;Acquired Immunodeficiency Syndromes;Histiocytic Syndrome;Familial Hemophagocytic Lymphocytosis;Lymphohistiocytosis;Macrophage Activation Syndrome;Langerhans Cell Histiocytosis (LCH);Hemoglobinopathies;Sickle Cell Disease;Sickle Cell-beta-thalassemia | Biological: CD34 Stem Cell Selection Therapy | Diane George, MD | Recruiting | N/A | 40 Years | All | 25 | Early Phase 1 | United States | |
| 128 | EUCTR2012-000242-35-GB | 10/01/2013 | 14 November 2016 | Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease | A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients with X-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients | X-linked Chronic Granulomatous Disease MedDRA version: 19.0 Level: PT Classification code 10008906 Term: Chronic granulomatous disease System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Suspension of autologous CD34+cells transduced with the G1XCGD viral vector Product Code: G1XCGD transduced CD34+ cells Pharmaceutical Form: Dispersion for infusion Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR Concentration unit: Other Concentration number: >4x10e6 CD34+viab-cells/kg | Genethon | Authorised | Female: no Male: yes | 11 | Phase 1;Phase 2 | France;Germany;Switzerland;United Kingdom | |||
| 129 | NCT01218438 | January 2013 | 16 December 2017 | Phase 2/3 Study of IGSC, 20% in PIDD | A Clinical Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) for the Evaluation of Efficacy, Safety, Tolerability and Pharmacokinetics in Subjects With Primary Immunodeficiency Diseases (PIDD) | Primary Immunodeficiency Diseases (PID) | Biological: Immune Globulin Intravenous (Human), 10% Solution;Drug: Immune Globulin Subcutaneous (Human), 20% Solution | Baxalta now part of Shire | Not recruiting | 2 Years | N/A | All | 86 | Phase 2/Phase 3 | United States;Canada | |
| 130 | JPRN-UMIN000009370 | 2012/12/07 | 2 April 2019 | Thalidomide therapy clinical trial for chronic granulomatous disease-associated intractable granulomatous lesion | Chronic granulomatous disease | Oral administration of thalidomide | National Center for Child Heath and Development | Not Recruiting | 3years-old | Not applicable | Male and Female | 5 | Not selected | Japan | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 131 | NCT01581593 | November 2012 | 19 February 2015 | Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID) | Multicenter, Open-label, Historically Controlled, Phase III Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Kedrion IVIG 10% in Adult and Pediatric Subjects With Primary Immunodeficiency (PID). | Primary Immunodeficiency;Agammaglobulinemia;Hypogammaglobulinemia;Antibody Deficiency | Biological: Kedrion IVIG 10% | Kedrion S.p.A. | Not recruiting | 2 Years | 70 Years | Both | 50 | Phase 3 | United States;Canada | |
| 132 | NCT01685827 | October 2012 | 26 February 2018 | Pivotal Study of Fexinidazole for Human African Trypanosomiasis in Stage 2 | Efficacy and Safety of Fexinidazole Compared to Nifurtimox-Eflornithine Combination Therapy (NECT) in Patients With Late-stage Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: Pivotal, Non-inferiority, Multicentre, Randomised, Open-label Study | Human African Trypanosomiasis (HAT);Sleeping Sickness | Drug: Fexinidazole;Drug: Nifurtimox;Drug: Eflornithine | Drugs for Neglected Diseases | Not recruiting | 15 Years | N/A | All | 394 | Phase 2/Phase 3 | Central African Republic;Congo, The Democratic Republic of the;Congo | |
| 133 | NCT01652092 | September 4, 2012 | 28 January 2019 | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | SCID;Omenn's Syndrome;Reticular Dysgenesis;Wiskott-Aldrich Syndrome;Bare Lymphocyte Syndrome;Common Variable Immunodeficiency;Chronic Granulomatous Disease;CD40 Ligand Deficiency;Hyper IgM Syndrome;X-linked Lymphoproliferative Disease;Hemophagocytic Lymphohistiocytosis;Griscelli Syndrome;Chediak-Higashi Syndrome;Langerhan's Cell Histiocytosis | Drug: Fludarabine phosphate 30 mg;Drug: MESNA;Drug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg | Masonic Cancer Center, University of Minnesota | Recruiting | N/A | 50 Years | All | 30 | N/A | United States | |
| 134 | NCT01659606 | July 2012 | 15 April 2019 | Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita | Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease | Dyskeratosis Congenita;Hoyeraal Hreidarsson Syndrome;Revesz Syndrome;Aplastic Anemia | Biological: alemtuzumab;Drug: Fludarabine;Drug: Cyclosporins;Drug: Mycophenolate mofetil | Boston Children’s Hospital | Dana-Farber Cancer Institute;Children's Hospital Medical Center, Cincinnati;Children's Hospital Los Angeles;Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium;Baylor College of Medicine;Children's Hospital of Philadelphia;Memorial Sloan Kettering Cancer Center;University of Wisconsin, Madison;Karolinska University Hospital | Recruiting | N/A | 65 Years | All | 40 | Phase 2 | United States;Sweden |
| 135 | NCT02327351 | July 2012 | 8 August 2016 | TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID | Phase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder;Hematopoietic Stem Cell Transplantation | Other: Biological: TCR alfa beta T cell depletion | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | Recruiting | 1 Month | 18 Years | Both | 60 | Phase 2/Phase 3 | Russian Federation | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 136 | EUCTR2012-000481-38-GB | 28/06/2012 | 24 June 2013 | Assessment of the safety of Immunoglobulin and recombinant human hylaluronidase in the treatment of patients with primary immunodeficiency | Tolerability, Safety and Product Administration Evaluation of rHuPH20 Facilitated Subcutaneous Treatment with Immune Globulin (Human), 10% in Subjects with Primary Immunodeficiency Diseases – A Study in Europe - Tolerability and Safety of IG, 10% with rHuPH20 in PIDD | Primary Immunodeficiency Diseases MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: Human normal immunoglobulin Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: Human normal immunoglobulin Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Product Name: Recombinant Human Hyaluronidase (rHuPH20) Pharmaceutical Form: Solution for infusion INN or Proposed INN: HYALURONIDASE CAS Number: 757971-58-7 Other descriptive name: 36-482-Hyaluronoglucosaminidase PH20 (rHuPH20) Concentration unit: U/ml unit(s)/millilitre Concentration type: equal Concentration number: 160- Product Name: Human normal immunoglobulin Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: Human normal immunoglobulin Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Product Name: Recombinant Human Hyaluronidase (rHuPH20) Pharmaceutical Form: Solution for infusion INN or Proposed INN: HYALURONIDASE CAS Number: 757971-58-7 Other descriptive name: 36-482-Hyaluronoglucosaminidase PH20 (rHuPH20) Concentration unit: U/ml unit(s)/millilitre Concentration type: equal Concentration number: 160- | Baxter Innovations GmbH | Not Recruiting | Female: yes Male: yes | 40 | Czech Republic;Belgium;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden | ||||
| 137 | EUCTR2012-000792-16-HU | 14/06/2012 | 27 May 2013 | Study to evaluate the efficacy, metabolism and safety of human immune globulin in patients with primary immunodeficiency diseases | “CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES” | primary immunodeficiency disease (PID) MedDRA version: 14.1 Level: LLT Classification code 10049485 Term: Bruton's agammaglobulinemia System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: OCTAGAM 50 mg/ml oldatos infúzió Product Name: Octagam 5% Pharmaceutical Form: Solution for infusion CAS Number: 308067-58-5 Current Sponsor code: Octagam 5% Other descriptive name: IMMUNOGLOBULIN G Concentration unit: % percent Concentration type: equal Concentration number: 5- | OCTAPHARMA AG | Not Recruiting | Female: yes Male: yes | 23 | Czech Republic;Hungary;Germany | ||||
| 138 | NCT03315078 | April 2012 | 10 December 2018 | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-Linked Combined Immunodeficiency Diseases | Biological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | Recruiting | 2 Years | 40 Years | All | 13 | Phase 1/Phase 2 | United States | |
| 139 | NCT01529827 | February 28, 2012 | 30 September 2019 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation | Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | Roswell Park Cancer Institute | Not recruiting | 3 Years | 75 Years | All | 94 | Phase 2 | United States | |
| 140 | NCT01533961 | February 2012 | 28 September 2015 | Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the SCYX-7158 | Randomized, Double-blind, Placebo-controlled Sequential Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SCYX-7158 After Single Oral Ascending Doses in Healthy Male Volunteers | Trypanosomiasis;Trypanosomiasis, African;Protozoan Infections;Parasitic Diseases | Drug: SCYX-7158;Drug: Placebo | Drugs for Neglected Diseases | Not recruiting | 18 Years | 45 Years | Male | 136 | Phase 1 | France | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 141 | NCT03333200 | January 11, 2012 | 11 November 2019 | Longitudinal Study of Neurodegenerative Disorders | Longitudinal Study of Neurodegenerative Disorders | MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | Recruiting | N/A | N/A | All | 1500 | Phase 1 | United States | |
| 142 | NCT03354533 | January 1, 2012 | 4 February 2019 | Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II | Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II | Leukocyte Adhesion Deficiency, Type II | Drug: L-fucose | Orpha Labs | Not recruiting | N/A | 18 Years | All | 4 | Phase 1/Phase 2 | ||
| 143 | NCT01485796 | December 1, 2011 | 16 December 2017 | Tolerability and Safety of IGI, 10% With rHuPH20 in PIDD | Tolerability, Safety and Administration Mode Evaluation of Recombinant Human Hyaluronidase (rHuPH20) Facilitated Subcutaneous Treatment With Immune Globulin Infusion (Human), 10% in Subjects With Primary Immunodeficiency Diseases (PIDD) | Primary Immunodeficiency Disorders | Biological: Immune Globulin Infusion (Human), 10%;Biological: Recombinant human hyaluronidase | Baxalta now part of Shire | Not recruiting | 2 Years | N/A | All | 54 | Phase 2/Phase 3 | United States | |
| 144 | NCT01465958 | November 2011 | 19 October 2017 | Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency | An Open-label, Single-sequence, Crossover Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Subcutaneous GAMUNEX®-C in Pediatric Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Biological: GAMUNEX-C | Grifols Therapeutics Inc. | Not recruiting | 2 Years | 16 Years | All | 12 | Phase 4 | United States | |
| 145 | NCT01461018 | October 2011 | 19 February 2015 | Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects With Primary Immunodeficiency (Japan Study) | A Multicenter Study of Long-Term Clinical Outcomes of Immune Globulin Subcutaneous (Human) (SCIG) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency | Biological: Immune globulin subcutaneous (Human) | CSL Behring | Not recruiting | N/A | 75 Years | Both | 22 | Phase 3 | Japan | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 146 | NCT01220531 | September 8, 2011 | 29 July 2019 | Thymus Transplantation Safety-Efficacy | Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836 | Complete DiGeorge Anomaly;DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Thymus Tissue for Transplantation;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Basiliximab;Drug: Mycophenolate mofetil | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Not recruiting | N/A | N/A | All | Phase 3 | United States | |
| 147 | NCT01406470 | September 2011 | 19 February 2015 | Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary Immunodeficiency | An Open-Label, Single-Arm, Historically Controlled, Prospective, Multicenter Phase III Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Immune Globulin Intravenous (Human) IVIG-SN™ in Subjects With Primary Immunodeficiency | Immunologic Deficiency Syndrome | Drug: Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™ | Green Cross Corporation | Atlantic Research Group | Not recruiting | 2 Years | 70 Years | Both | 45 | Phase 3 | United States;Canada |
| 148 | NCT01483170 | September 2011 | 16 December 2017 | Multiple Dose Study to Evaluate Security, Tolerance and Pharmacokinetic of Fexinidazole (Drug Candidate for Human African Trypanosomiasis) Administered With a Loading Dose and With Food | Double-blind, Placebo Controlled, Randomized Multiple Ascending Dose Study in Fed Conditions for Ten Days Dosing Regimen With a Loading Dose to Evaluate the Safety, the Tolerability and the Pharmacokinetics of Oral Fexinidazole in 36 Healthy Male Sub-Saharan Volunteers. | Trypanosomiasis, African | Drug: Tablets Fexinidazole;Drug: Placebo | Drugs for Neglected Diseases | Not recruiting | 18 Years | 45 Years | Male | 30 | Phase 1 | France | |
| 149 | NCT01410825 | July 2011 | 20 May 2019 | Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome | Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Biological: Retrovirus-mediated gene transfer | David Williams | Not recruiting | 3 Months | 35 Years | Male | 5 | Phase 1/Phase 2 | United States | |
| 150 | NCT01412385 | June 20, 2011 | 13 May 2019 | Immune Globulin Subcutaenous (Human), 20% | A Clinical Study of Immune Globulin Subcutaneous (Human) (IGSC), 20% for the Evaluation of Efficacy, Safety, and Pharmacokinetics in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: Immune Globulin Subcutaneous (Human), 20%;Biological: Immune Globulin Intravenous (Human), 10%;Biological: Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin) | Baxalta now part of Shire | Not recruiting | 2 Years | N/A | All | 55 | Phase 2/Phase 3 | United Kingdom;Sweden;Hungary;Germany;Austria | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 151 | EUCTR2011-001118-32-DE | 25/05/2011 | 26 October 2015 | Twelve-month study on the immunogenicity (i.e. the ability of the study medication to provoke an immune response), safety and efficacy of the study medication Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia | Twelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia | Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia) MedDRA version: 14.1 Level: PT Classification code 10051645 Term: Idiopathic neutropenia System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 14.1 Level: LLT Classification code 10069819 Term: Congenital neutropenia System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1 Level: PT Classification code 10053176 Term: Cyclic neutropenia System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Filgrastim Hexal Pharmaceutical Form: Solution for injection/infusion in pre-filled syringe INN or Proposed INN: FILGRASTIM CAS Number: 121181-53-1 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 300- Trade Name: Filgrastim Hexal Pharmaceutical Form: Solution for injection/infusion in pre-filled syringe INN or Proposed INN: FILGRASTIM CAS Number: 121181-53-1 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 480- Product Name: EP2006 (Filgrastim) Product Code: EP2006 Pharmaceutical Form: Solution for injection INN or Proposed INN: FILGRASTIM CAS Number: 121181-53-1 Current Sponsor code: EP2006 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 480- | Sandoz GmbH | Not Recruiting | Female: yes Male: yes | 40 | Germany;Sweden | ||||
| 152 | NCT01313507 | May 2011 | 16 December 2017 | High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam) | Clinical Study to Evaluate the Safety and Tolerability of Immunoglobulin Intravenous (Human) 10% (NewGam) Administered at High Infusion Rates to Patients With Primary Immunodeficiency Diseases (Extension of Study NGAM-01) | Primary Immunodeficiency Disease | Biological: NewGam | Octapharma | Not recruiting | 2 Years | 75 Years | All | 21 | Phase 3 | United States | |
| 153 | NCT01458171 | April 2011 | 19 October 2017 | Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study) | A Multicenter Follow-up Study of Long-term Safety, Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency Disorder | Biological: Immune globulin subcutaneous (Human) | CSL Behring | Not recruiting | N/A | 75 Years | All | 23 | Phase 3 | Japan | |
| 154 | EUCTR2010-023483-41-HU | 01/03/2011 | 27 May 2013 | A multinational study with I10E (Human Immunoglobulin) to demonstrate the efficacy and the safety of the product in patients suffering from deficiency in their immune system | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) - I10E-0718 | a primary immunodeficiency as defined by the ESID and validated by a reference centre : • X-linked agammaglobulinemia (XLA) • Common variable immunodeficiency (CVID) MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 100000004870 MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 MedDRA version: 14.1 Level: LLT Classification code 10001471 Term: Agammaglobulinemia System Organ Class: 100000004870 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR Product Code: I10E Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | LFB BIOTECHNOLOGIES | Not Recruiting | Female: yes Male: yes | 60 | Lithuania;Ukraine;Poland;Hungary;Czech Republic;France;Serbia | ||||
| 155 | NCT01289847 | March 2011 | 19 October 2017 | A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency | A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinemia;Hyper-IgM Syndrome;Wiskott-Aldrich Syndrome | Biological: Gammaplex | Bio Products Laboratory | Not recruiting | 2 Years | 16 Years | All | 25 | Phase 4 | United States;Chile;Israel | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 156 | NCT01306019 | February 26, 2011 | 22 October 2019 | Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-linked Severe Combined Immunodeficiency;XSCID;SCID-X1;Gamma C-Deficient SCID | Other: Gene-modified CD34+ Hematopoietic stem cells;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | Recruiting | 2 Years | 40 Years | All | 20 | Phase 1/Phase 2 | United States | |
| 157 | EUCTR2010-019459-23-DE | 18/01/2011 | 28 August 2014 | CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 16.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human normal immunoglobulin CAS Number: 0 Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% Pharmaceutical Form: Solution for injection INN or Proposed INN: Human normal immunoglobulin CAS Number: 0 Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: g/l gram(s)/litre Concentration type: equal Concentration number: 160- Product Name: Immune Globulin Subcutaneos, 20% Product Code: IGSC, 20% Pharmaceutical Form: Solution for injection INN or Proposed INN: Human normal immunoglobulin CAS Number: 0 Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | Baxter Innovations GmbH | Not Recruiting | Female: yes Male: yes | 47 | Sweden;United Kingdom;Germany;Netherlands;Austria;Belgium;Hungary | ||||
| 158 | NCT01338675 | January 2011 | 19 February 2015 | Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD) | Chronic Granulomatous Disease | Drug: Busulfan | Seoul National University Hospital | Recruiting | N/A | N/A | Both | 5 | Phase 1/Phase 2 | Korea, Republic of | ||
| 159 | NCT01222247 | October 2010 | 22 July 2019 | Antenatal Late Preterm Steroids (ALPS): A Randomized Placebo-Controlled Trial | Antenatal Late Preterm Steroids (ALPS): A Randomized Placebo-Controlled Trial | Pregnancy;Respiratory Distress Syndrome;Pregnancy Outcomes;Preterm Birth | Drug: Betamethasone;Drug: Placebo | The George Washington University Biostatistics Center | National Heart, Lung, and Blood Institute (NHLBI);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Not recruiting | N/A | N/A | Female | 2831 | Phase 3 | United States |
| 160 | NCT01354587 | October 2010 | 19 February 2015 | Evaluation of Efficacy and Tolerability of Hizentra® | Evaluation of Efficacy and Tolerability of Hizentra® in Subjects Transitioning From Vivaglobin® (16% SCIG Product) to Hizentra® (20% SCIG Product) | Primary Immunodeficiency Disorders | Drug: Hizentra | University of South Florida | Recruiting | 1 Year | 75 Years | Both | 50 | N/A | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 161 | NCT01856582 | October 2010 | 11 June 2018 | CD34+ Stem Cell Infusion to Augment Graft Function | Post Transplant CD34+ Selected Stem Cell Infusion to Augment Graft Function in Children With Primary Immunodeficiency Diseases and Bone Marrow Failure Syndromes | Waning Donor Chimerism;Waning Immune Function;Primary Immunodeficiency Disease(s);Bone Marrow Failure | Biological: CD34+ | Children's Hospital Medical Center, Cincinnati | Hoxworth Blood Center | Not recruiting | N/A | 35 Years | All | 23 | Phase 2 | United States |
| 162 | JPRN-JapicCTI-101271 | 06/9/2010 | 16 July 2019 | Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study) | A Multicenter Study of Efficacy, Safety, Tolerability, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | Primary Immunodeficiency | Intervention name : Immune Globulin Subcutaneous (Human) IgPro20 INN of the intervention : Immune Globulin Subcutaneous (Human) Dosage And administration of the intervention : The weekly dose of IgPro20 should be the weekly equivalent dose of the previous IVIG treatment. | CSL Behring K.K. | Recruiting | 74 | BOTH | 15 | Phase 3 | |||
| 163 | NCT01199705 | September 2010 | 19 October 2017 | Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study) | A Multicenter Study of Efficacy, Safety, Tolerability, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency | Biological: Immune Globulin Subcutaneous (Human) (SCIG) | CSL Behring | Not recruiting | N/A | 75 Years | All | 25 | Phase 3 | Japan | |
| 164 | NCT01319851 | September 2010 | 16 December 2017 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study | Thalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia | Drug: Alefacept | Emory University | Children's Healthcare of Atlanta | Not recruiting | N/A | 21 Years | All | 3 | N/A | United States |
| 165 | EUCTR2010-019249-25-DE | 12/08/2010 | 8 May 2012 | An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID) | An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID) | Primary immunodeficiency disease (PID) MedDRA version: 14.1 Level: LLT Classification code 10010509 Term: Congenital hypogammaglobulinemia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Intratect Product Code: BT090 Pharmaceutical Form: Solution for infusion Current Sponsor code: BT090 Other descriptive name: Human normal immunoglobulin for intravenous use (IVIG) Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Biotest AG | Not Recruiting | Female: yes Male: yes | 30 | Germany;Hungary | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 166 | NCT01182675 | August 2010 | 16 December 2017 | Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim | Hematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & Filgrastim | Severe Combined Immunodeficiency | Drug: Transplant Conditioning with Mobilization Only;Drug: Transplant Conditioning with Mobilization and Alemtuzumab | University of California, San Francisco | Not recruiting | N/A | 3 Years | All | 7 | Phase 2 | United States | |
| 167 | NCT01175213 | July 2010 | 16 December 2017 | Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PID | Long-Term Tolerability and Safety of Immune Globulin Subcutaneous (IGSC) Solution Administered Subcutaneously Following Administration of Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: IGSC - rHuPH20 then IGSC or IGIV;Biological: IGIV, 10% only | Baxalta now part of Shire | Not recruiting | 2 Years | N/A | All | 66 | Phase 3 | United States | |
| 168 | NCT01147042 | May 18, 2010 | 16 December 2017 | Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease | Assessment of the Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease | IFN-Gamma Therapy;CGD Gene Mutation;CGD Response to IFNg;CGD - Chronic Granulomatous Disease;Immunodeficiency Disease | Drug: IFN-gamma | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | All | 2 | Phase 4 | United States | |
| 169 | NCT01129544 | April 2010 | 11 June 2019 | Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector | Multi-institutional Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene Transfer | Severe Combined Immunodeficiency | Biological: Gene transfer | David Williams | Boston Children’s Hospital;Children's Hospital Medical Center, Cincinnati;University of California, Los Angeles | Not recruiting | N/A | N/A | Male | 8 | Phase 1/Phase 2 | United States |
| 170 | NCT03534479 | April 2010 | 11 June 2018 | Human IgGs and Endothelial Function in Vivo in Humans | Effects of Intravenous Human Polyclonal Immunoglobulins G Infusion on Endothelial Function and Insulin Sensitivity in Humans | Common Variable Immunodeficiency | Drug: Polyclonal IgG | Federico II University | Not recruiting | 18 Years | 70 Years | All | 24 | N/A | Italy | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 171 | EUCTR2009-017346-32-IT | 15/03/2010 | 19 March 2012 | A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS | A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS | Wiskott-Aldrich Sindrome MedDRA version: 9.1 Level: PT Classification code 10061598 | Product Name: autologous CD34+ cells transduced with a lentiviral vector encoding the WASP cDNA Pharmaceutical Form: Suspension for injection Other descriptive name: autologous CD34+ cells transduced with a lentiviral vector encoding the WASP cDNA Concentration unit: % percent Concentration type: equal Concentration number: 100- Trade Name: BUSILVEX Pharmaceutical Form: Solution for infusion INN or Proposed INN: Busulfan Concentration unit: mOsm/kg milliosmol(s)/kilogram Concentration type: equal Concentration number: 1- Trade Name: FLUDARABINA TEVA Pharmaceutical Form: Solution for infusion INN or Proposed INN: Fludarabine Concentration unit: mg/m2 milligram(s)/square meter Concentration type: equal Concentration number: 30- Trade Name: THYMOGLOBULINE Pharmaceutical Form: Powder and solvent for solution for infusion INN or Proposed INN: Antithymocyte immunoglobulin (rabbit) Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 1- Trade Name: MABTHERA Pharmaceutical Form: Solution for infusion INN or Proposed INN: Rituximab Concentration unit: mg/m2 milligram(s)/square meter Concentration type: equal Concentration number: 375- | FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR | Authorised | Female: no Male: yes | Phase 1/2 | Italy | ||||
| 172 | EUCTR2009-011434-10-DE | 02/03/2010 | 24 April 2012 | CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency diseases MedDRA version: 12.0 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency MedDRA version: 12.0 Level: LLT Classification code 10049485 Term: Bruton's agammaglobulinemia | Product Name: NewGam Product Code: NewGam Pharmaceutical Form: Solution for infusion INN or Proposed INN: immunoglobulin G Other descriptive name: NewGam Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | OCTAPHARMA AG | Not Recruiting | Female: yes Male: yes | 50 | Germany | ||||
| 173 | NCT01131858 | March 2010 | 19 February 2015 | Study of Vitamin D3 Substitution to Patients With Primary Immunodeficiency | A Placebo Controlled Double Blinded Study of Vitamin D3 Substitution to Patients With Primary Immunodeficiency | Primary Immune Deficiency Disorder | Drug: Vigantol;Drug: Placebo | Karolinska University Hospital | Not recruiting | 18 Years | 75 Years | Both | 140 | Phase 1/Phase 2 | Sweden | |
| 174 | EUCTR2007-000684-16-GB | 21/01/2010 | 26 June 2012 | Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1 | Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1 | X-Linked severe combined Immunodeficiency (SCID-X1) MedDRA version: 9.1 Level: LLT Classification code 10010099 Term: Combined immunodeficiency | Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector Pharmaceutical Form: Solution for blood fraction modification Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector transduced cells Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector transduce Pharmaceutical Form: Solution for infusion | Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health | Authorised | Female: no Male: yes | 10 | United Kingdom | ||||
| 175 | EUCTR2007-004308-11-GB | 11/01/2010 | 13 February 2017 | Gene therapy for WAS | PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - Gene Therapy for WAS , version 5.0 | Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages. MedDRA version: 19.1 Level: PT Classification code 10047992 Term: Wiskott-Aldrich syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Pharmaceutical Form: Solution for injection Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Concentration unit: Other Concentration type: not less then Concentration number: 0.5E06 cells /Kg- Pharmaceutical Form: Solution for injection Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Concentration unit: Other Concentration type: not less then Concentration number: 0.5E06 cells /Kg- Pharmaceutical Form: Solution for injection Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Concentration unit: Other Concentration type: not less then Concentration number: 0.5E06 cells /Kg- | Genethon | Authorised | Female: no Male: yes | Phase 1;Phase 2 | United Kingdom | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 176 | NCT01001598 | November 2009 | 19 February 2015 | Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita | Phase I/II Dose Escalation Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita | Fanconi Anemia;Dyskeratosis Congenita | Drug: danazol | Children's Hospital Boston | Not recruiting | 3 Years | N/A | Both | 5 | Phase 1/Phase 2 | United States | |
| 177 | NCT00982904 | September 2009 | 16 December 2017 | Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the Fexinidazole | Randomized, Double-blind, Placebo-controlled Study of the Tolerability, and Pharmacokinetics of Fexinidazole After Single and Repeated Oral Ascending Doses, Completed by a Comparative Bioavailability Study of an Oral Suspension Versus a Tablet and an Exploratory Assessment of Food Effect, in Healthy Male Volunteers | Human African Trypanosomiasis | Drug: Fexinidazole/Placebo | Drugs for Neglected Diseases | Sanofi | Not recruiting | 18 Years | 45 Years | Male | 108 | Phase 1 | France |
| 178 | EUCTR2009-012036-32-FR | 30/07/2009 | 19 March 2012 | SAFETY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, WHEN ADMINISTERED TO PRIMARY IMMUNODEFICIENT PATIENTS, AT A PROGRESSIVELY INCREASED FLOW RATE | SAFETY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, WHEN ADMINISTERED TO PRIMARY IMMUNODEFICIENT PATIENTS, AT A PROGRESSIVELY INCREASED FLOW RATE | PRIMARY IMMUNODEFICIENCY MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE Product Code: IGNG Pharmaceutical Form: Solution for infusion INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | LFB BIOTECHNOLOGIES | Authorised | Female: yes Male: yes | France | |||||
| 179 | NCT00909363 | June 2009 | 1 April 2019 | Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients | Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia. | Wiskott-Aldrich Syndrome;Thrombocytopenia;Bleeding | Drug: Promacta;Diagnostic Test: blood drawing in patients with WAS;Diagnostic Test: blood drawing in healthy controls | Weill Medical College of Cornell University | Novartis Pharmaceuticals | Not recruiting | 3 Months | 80 Years | Male | 24 | Phase 2 | United States |
| 180 | NCT00906880 | April 2009 | 19 February 2015 | Clinical Study to Assess the Tolerability, Feasibility and Effectiveness of Nifurtimox and Eflornithine (NECT) for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Phase | Clinical Study to Assess the Clinical Tolerability, Feasibility and Effectiveness Under Field Conditions of the Combination of Nifurtimox and Eflornithine (NECT) for the Treatment of T.b.Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Stage | Human African Trypanosomiasis | Drug: Nifurtimox-Eflronithine Combination Treatment (NECT) | Drugs for Neglected Diseases | Ministry of Public Health, Democratic Republic of the Congo;Swiss Tropical & Public Health Institute | Not recruiting | N/A | N/A | Both | 630 | Phase 4 | Congo |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 181 | NCT00799071 | February 2009 | 19 February 2015 | Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD) | Investigation of POsaconazole Prophylaxis in Children With Chronic Granulomatous Disease (CGD): Pharmacokinetics and Tolerability (iPOD) | Chronic Granulomatous Disease | Drug: posaconazole (PSZ) | Radboud University | Not recruiting | 2 Years | 16 Years | Both | 12 | Phase 2 | Netherlands;United Kingdom | |
| 182 | EUCTR2007-003235-23-FR | 09/01/2009 | 19 March 2012 | Essai randomisé multicentrique de phase II d'évaluation immunologique d'une stratégie vaccinale de type prime boost associant une administration du vaccin conjugué anti-pneumococcique à S0 suivie de l'injection du vaccin polysaccharidique à S4 comparé à l'administration du vaccin polysaccharidique seul à S4 chez des patients ayant un déficit immunitaire commun variable - Evaluation d'une stratégie vaccinale anti-pneumococcique chez des patients aynt un DICV | Essai randomisé multicentrique de phase II d'évaluation immunologique d'une stratégie vaccinale de type prime boost associant une administration du vaccin conjugué anti-pneumococcique à S0 suivie de l'injection du vaccin polysaccharidique à S4 comparé à l'administration du vaccin polysaccharidique seul à S4 chez des patients ayant un déficit immunitaire commun variable - Evaluation d'une stratégie vaccinale anti-pneumococcique chez des patients aynt un DICV | Déficit immunitaire commun variable MedDRA version: 9.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency | Trade Name: PNEUMO 23 Pharmaceutical Form: Solution for injection Trade Name: PREVENAR Pharmaceutical Form: Suspension for injection | Inserm | Authorised | Female: yes Male: yes | Phase 2 | France | ||||
| 183 | NCT00811174 | January 2009 | 16 December 2017 | Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases | Clinical Study to Evaluate the Efficacy, Safety and Kinetics of Octagam 10% for Replacement Therapy in Primary Immunodeficiency Diseases | Immunologic Deficiency Syndromes | Drug: Octagam 10% | Octapharma | Not recruiting | 2 Years | 75 Years | All | 5 | Phase 3 | Austria | |
| 184 | NCT01289171 | January 2009 | 19 February 2015 | Acquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric Patients | Acquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric Patients: Prospective Treatment Trial With Topical Glycolic Acid and HPV Genotype Characterization | Flat Warts (Diagnosis);HIV Infections | Drug: Glycolic acid | University of Pennsylvania | NeoStrata Company, Inc.;Penn Center for AIDS Research (CFAR) | Not recruiting | 7 Years | N/A | Both | 38 | N/A | Botswana |
| 185 | EUCTR2009-011152-22-FR | 2 October 2017 | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome. An open labelled, non-randomised, phase I/II, cohort study involving a single infusion of autologous CD34+ cells transduced with the lentiviral vector w1.6_hWASP_WPRE (VSVg) in up to 5 patients with WAS. | Product Name: Autologous CD34+ cells transduced with the Lentiviral vector containing the human Wiskott Aldrich Sy Product Code: GTG003.08 Pharmaceutical Form: Solution for injection | GENETHON | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | France | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 186 | NCT00814320 | December 2008 | 16 December 2017 | Gammagard Liquid and rHuPH20 in PID | Efficacy, Tolerability and Pharmacokinetic Comparison of Immune Globulin Intravenous (Human), 10% (GAMMAGARD LIQUID/KIOVIG) Administered Intravenously or Subcutaneously Following Administration of Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV) | Baxalta now part of Shire | Not recruiting | 2 Years | N/A | All | 89 | Phase 3 | United States;Canada | |
| 187 | EUCTR2008-004518-28-NL | 19/11/2008 | 14 August 2012 | Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPOD | Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPOD | Children with chronic granulomatous disease (CGD). MedDRA version: 9.1 Level: LLT Classification code 10008906 Term: Chronic granulomatous disease | Trade Name: Noxafil 40 mg/ml oral solution Pharmaceutical Form: Oral suspension | Radboud University Nijmegen Medical Centre | Not Recruiting | Female: yes Male: yes | United Kingdom;Netherlands | |||||
| 188 | NCT00794508 | November 2008 | 19 October 2017 | MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID | MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan | Severe Combined Immunodeficiency | Biological: ADA gene transfer | Donald B. Kohn, M.D. | FDA Office of Orphan Products Development;National Institutes of Health (NIH) | Not recruiting | 1 Month | 18 Years | All | 10 | Phase 2 | United States |
| 189 | EUCTR2007-002611-27-DE | 27/10/2008 | 11 February 2013 | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Primary Immundeficiency Diseases (PID) MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome | Trade Name: Octagam 10% Product Name: Octagam 10% Pharmaceutical Form: Solution for infusion Current Sponsor code: GAM10 Concentration unit: % (W/V) percent weight/volume Concentration type: equal Concentration number: 10- | Octapharma AG | Not Recruiting | Female: yes Male: yes | 45 | France;Germany;United Kingdom | ||||
| 190 | NCT00774358 | October 2008 | 16 December 2017 | Interleukin-2 Treatment for Wiskott-Aldrich Syndrome | Reinstituting Natural Killer Cell Cytotoxicity and Cytoskeletal Dynamics in Wiskott-Aldrich Syndrome With IL-2 Therapy | Wiskott-Aldrich Syndrome (WAS);X-linked Thrombocytopenia | Drug: Interleukin-2 | Soma Jyonouchi | Texas Children's Hospital | Not recruiting | 24 Months | N/A | All | 9 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 191 | EUCTR2008-000830-30-DE | 10/07/2008 | 28 August 2012 | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency (IgPro20 EU Extension Study) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency (IgPro20 EU Extension Study) | PID (primary immuno deficiency) MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human Normal Immunoglobulin Concentration unit: % percent Concentration type: equal Concentration number: 20- | CSL Behring AG | Not Recruiting | Female: yes Male: yes | 36 | United Kingdom;Germany;France;Spain;Sweden | ||||
| 192 | EUCTR2008-003368-21-IT | 04/06/2008 | 19 March 2012 | Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndrome | Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndrome | For patients with a stabilized clinical condition, not recovered in care units for acute problems. MedDRA version: 9.1 Level: HLGT Classification code 10003816 MedDRA version: 9.1 Level: HLT Classification code 10027657 | Trade Name: ORENCIA Pharmaceutical Form: Powder for infusion* INN or Proposed INN: ABATACEPT Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- | ISTITUTO PER L`INFANZIA BURLO GAROFOLO | Not Recruiting | Female: no Male: yes | Italy | |||||
| 193 | NCT00719680 | June 2008 | 19 October 2017 | Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID) | Primary Immune Deficiency | Biological: IgPro20 | CSL Behring | Not recruiting | 2 Years | 75 Years | All | 21 | Phase 3 | United States | |
| 194 | NCT00634569 | May 2008 | 19 October 2017 | Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects | Evaluation of the Efficacy and Safety of Flebogamma 5% DIF [Immune Globulin Intravenous (Human)] for Replacement Therapy in Pediatric Subjects With Primary Immunodeficiency Diseases. | Primary Immune Deficiency Disease | Biological: Flebogamma 5% DIF | Instituto Grifols, S.A. | Not recruiting | 2 Years | 16 Years | All | 24 | Phase 4 | United States | |
| 195 | NCT00680446 | April 2008 | 19 February 2015 | Safety Study of Subcutaneous Ig NextGen 16% in Patients With Primary Immunodeficiency | An Open-Label Study of Ig NextGen 16% Administered by Subcutaneous Infusion in Patients With Primary Immunodeficiency (PID). | Primary Immune Deficiency | Drug: Immunoglobulin G (Ig NextGen 16%) | CSL Limited | Not recruiting | 3 Years | N/A | Both | 41 | Phase 3 | Australia;New Zealand | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 196 | NCT00605657 | January 2008 | 19 October 2017 | Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS) | Pilot (Phase I-II) Study of Valproic Acid (Depakote) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS) | ALPS;Hypersplenism;Lymphadenopathy | Drug: Valproic Acid;Procedure: CT Scan;Procedure: Blood Sample | Koneti Rao | Not recruiting | 2 Years | 70 Years | All | 6 | Phase 1/Phase 2 | United States | |
| 197 | NCT00546871 | October 2007 | 16 December 2017 | Comparison of Intravenous and Subcutaneous Administration of IGIV, 10% in Primary Immunodeficiency (PID) Subjects | Tolerability and Pharmacokinetic Comparison of Immune Globulin Intravenous (Human) 10% (IGIV, 10%) Administered Intravenously or Subcutaneously in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency (PID) | Drug: Immune Globulin Intravenous (Human), 10% | Baxalta now part of Shire | Not recruiting | 24 Months | N/A | All | 49 | Phase 2/Phase 3 | United States | |
| 198 | NCT00579137 | October 2007 | 19 October 2017 | Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders | CD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency Disorders | Severe Combined Immunodeficiency Disease;Severe Primary Immunodeficiency Disorder;Undefined T Cell Deficiency Disorder;Wiskott-Aldrick Syndrome | Biological: Campath -1H;Drug: Fludarabine;Biological: Anti-CD45;Procedure: Stem cell infusion | Baylor College of Medicine | Center for Cell and Gene Therapy, Baylor College of Medicine;Texas Children's Hospital | Not recruiting | N/A | N/A | All | 3 | Phase 1/Phase 2 | United States |
| 199 | EUCTR2006-006023-39-IT | 24/09/2007 | 19 March 2012 | Pharmacokinetics of Haemocomplettan P in subjects with congenital fibrinogen deficiency - ND | Pharmacokinetics of Haemocomplettan P in subjects with congenital fibrinogen deficiency - ND | PK study in patient with afibrinogemia MedDRA version: 9.1 Level: LLT Classification code 10016075 Term: Factor I deficiency | Trade Name: Haemocomplettan P Pharmaceutical Form: Powder and solvent for solution for infusion Current Sponsor code: Factor I Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1- | CSL Behring GmbH | Not Recruiting | Female: yes Male: yes | Italy | |||||
| 200 | NCT00527878 | September 2007 | 19 October 2017 | Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) Syndrome | A Double-Blind, Randomized, Placebo-Controlled Cross-Over Study Assessing the Role of Pathogen-Specific IgE and Histamine Release in the Hyper-IgE Syndrome and the Effect of Ranitidine on Laboratory and Clinical Manifestations | JOB's Syndrome;Hyper-IgE Recurrent Infection Syndrome;Immune Deficiency | Drug: Ranitidine;Drug: Placebo | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 2 Years | N/A | All | 16 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 201 | NCT00542997 | September 2007 | 19 October 2017 | Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy | A Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Common Variable Immunodeficiency;X-linked Agammaglobulinemia;Autosomal Recessive Agammaglobulinemia | Biological: Human Normal Immunoglobulin for Subcutaneous Administration (IGSC) | CSL Behring | Not recruiting | 2 Years | 65 Years | All | 51 | Phase 3 | France;Germany;Italy;Poland;Romania;Spain;Sweden;Switzerland;United Kingdom | |
| 202 | NCT00455312 | August 2007 | 16 December 2017 | Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA | Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia | Dyskeratosis Congenita;Aplastic Anemia | Drug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: Methylprednisolone | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | 70 Years | All | 36 | Phase 2/Phase 3 | United States | |
| 203 | EUCTR2006-006745-13-DE | 11/07/2007 | 19 March 2012 | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | PID (Primary Immunodeficiency) MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human Immunglobulin G (IgG) Concentration unit: % percent Concentration type: equal Concentration number: 20- | CSL Behring AG | Not Recruiting | Female: yes Male: yes | 51 | United Kingdom;Germany;France;Spain;Italy;Poland;Sweden | ||||
| 204 | NCT00490100 | June 2007 | 19 October 2017 | Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1 | Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency (XSCID): A Phase 2 Study Assessing Safety and Clinical Response to Treatment With Insulin-like Growth Factor-1 (IGF-1) | Growth Failure;X-linked Severe Combined Immunodeficiency (XSCID);Growth Hormone Resistence | Drug: Increlex | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 2 Years | 20 Years | All | 6 | Phase 1/Phase 2 | United States | |
| 205 | EUCTR2007-001410-17-FR | 31/05/2007 | 19 March 2012 | LONG-TERM SAFETY AND EFFICACY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, ADMINISTERED IN CURRENT PRACTICE TO PRIMARY IMMUNODEFICIENT PATIENTS | LONG-TERM SAFETY AND EFFICACY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, ADMINISTERED IN CURRENT PRACTICE TO PRIMARY IMMUNODEFICIENT PATIENTS | Primary Immunodeficiency MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome | Product Name: Human normal immunoglobulin for intravenous administration Product Code: IGNG Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human normal immunoglobulin for intravenous administration Current Sponsor code: IGNG Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | LFB SA | Authorised | Female: yes Male: yes | France | |||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 206 | NCT00391131 | April 2007 | 19 February 2015 | Subcutaneous Ig NextGen 16% in PID Patients | A Multi-centre, Open-label Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Subcutaneous Infusions of Ig NextGen 16% in Patients With Primary Immunodeficiency (PID). | Primary Immunodeficiency (PID) | Drug: IgNextGen 16% | CSL Limited | Not recruiting | 3 Years | N/A | Both | 35 | Phase 3 | Australia;New Zealand | |
| 207 | EUCTR2006-006522-25-DE | 01/02/2007 | 19 March 2012 | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years MedDRA version: 8.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency | Trade Name: Vivaglobin Product Name: Vivaglobin Pharmaceutical Form: Cutaneous solution INN or Proposed INN: immune globulin subcutaneous (human) Current Sponsor code: CE1200 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 160- | CSL Behring AG | Not Recruiting | Female: yes Male: yes | 28 | United Kingdom;Germany;Belgium;Spain;Italy;Greece | ||||
| 208 | NCT00885833 | February 2007 | 19 February 2015 | Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) | Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Drug: Fludarabine, Busulfan, Thymoglobulin | The Korean Society of Pediatric Hematology Oncology | Not recruiting | 1 Year | 25 Years | Both | 5 | Phase 1/Phase 2 | Korea, Republic of | |
| 209 | NCT02512679 | February 2007 | 16 December 2017 | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | Protocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological Diseases | Stem Cell Transplantation;Bone Marrow Transplantation;Peripheral Blood Stem Cell Transplantation;Allogeneic Transplantation;Genetic Diseases;Thalassemia;Pediatrics;Diamond-Blackfan Anemia;Combined Immune Deficiency;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Lymphoproliferative Disease;Metabolic Diseases | Drug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4 | Children's Hospital Los Angeles | Lucile Packard Children's Hospital | Not recruiting | 3 Months | N/A | All | 20 | Phase 2 | |
| 210 | NCT02127892 | January 2, 2007 | 16 December 2017 | SCID Bu/Flu/ATG Study With T Cell Depletion | Phase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling Donor | Severe Combined Immunodeficiency | Biological: unrelated BM with T cell depletion;Biological: unrelated cord blood;Biological: haplo BM with T cell depletion;Device: unrelated PBSC with T cell depletion | Neena Kapoor, M.D. | Not recruiting | N/A | 21 Years | All | 9 | Phase 1/Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 211 | NCT00778882 | January 2007 | 28 October 2019 | Gene Therapy for Chronic Granulomatous Disease in Korea | An Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease Patients | Chronic Granulomatous Disease | Drug: VM106 | Helixmith Co., Ltd. | Not recruiting | N/A | N/A | Male | 2 | Phase 1/Phase 2 | Korea, Republic of | |
| 212 | NCT00782106 | December 4, 2006 | 5 February 2018 | Study to Determine the Dose of Recombinant Human Hyaluronidase Needed to Infuse a Full Dose of IGIV Subcutaneously | Phase 1/2 Determination of the Dose of Recominant Human Hyaluronidase (rHuPH20) Required Enabling Up to 600 mg/kg Bodyweight of IGIV, 10% to be Administered Subcutaneously in a Single Infusion Site in Subjects With Primary Immunodeficiency (PID) | Primary Immunodeficiency Diseases (PID) | Biological: Recombinant human hyaluronidase + immune globulin intravenous | Baxalta now part of Shire | Not recruiting | 16 Years | N/A | All | 11 | Phase 1/Phase 2 | United States | |
| 213 | NCT00392951 | December 2006 | 19 November 2018 | Sirolimus for Autoimmune Disease of Blood Cells | Sirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot Series | Autoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid Arthritis | Drug: sirolimus | Children's Hospital of Philadelphia | Not recruiting | 1 Year | 30 Years | All | 30 | Phase 1/Phase 2 | United States | |
| 214 | NCT00389324 | November 2006 | 19 October 2017 | A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary Immunodeficiency | An Open-Label Single-Sequence, Crossover Trial to Evaluate the Pharmacokinetics and Safety of Subcutaneous Gamunex® 10% (Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified) in Subjects With Primary Immunodeficiency | Immunologic Deficiency Syndrome | Biological: Immune Globulin Intravenous (Human) | Grifols Therapeutics Inc. | Not recruiting | 13 Years | 75 Years | All | 35 | Phase 2 | United States;Canada | |
| 215 | NCT00419341 | November 2006 | 19 October 2017 | Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy | A Phase III Open-Label, Prospective, Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human), IgPro20 in Subjects With Primary Immunodeficiency (PID) | Primary Immune Deficiency | Biological: Human Normal Immunoglobulin for Subcutaneous Administration | CSL Behring | Not recruiting | 2 Years | 75 Years | All | 49 | Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 216 | NCT00394316 | October 30, 2006 | 16 July 2018 | Gene Therapy for Chronic Granulomatous Disease | Autologous Transplantation of Genetically Modified Cells for the Treatment of X-Linked Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 3 Years | 55 Years | Male | 3 | Early Phase 1 | United States | |
| 217 | NCT00395538 | October 30, 2006 | 2 September 2019 | Effects of PTH Replacement on Bone in Hypoparathyroidism | Effects of PTH Replacement on Bone in Hypoparathyroidism | Hypoparathyroidism;DiGeorge Syndrome | Drug: PTH 1-34 | National Institute of Dental and Craniofacial Research (NIDCR) | Not recruiting | 18 Years | 70 Years | All | 46 | Phase 3 | United States;Austria;Italy | |
| 218 | NCT00358657 | May 24, 2006 | 28 October 2019 | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders | HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| 219 | NCT00325078 | May 2006 | 19 October 2017 | Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease | Tumor Necrosis Factor Alpha Inhibitor (Lnfliximab, Adalimumab) Treatment for Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease: A Phase I/II Study Assessing Clinical and Immune Responses to Treatment and Genetic Influences | Chronic Granulomatous Disease;Crohn'S-like IBD;Inflammatory Bowel Disease (IBD) | Drug: Infliximab | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 10 Years | N/A | All | 40 | Phase 1/Phase 2 | United States | |
| 220 | NCT00579527 | March 2006 | 29 July 2019 | Phase I/II Thymus Transplantation With Immunosuppression #950 | Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950 | DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge Syndrome | Drug: Methylprednisolone or Prednisolone;Drug: Daclizumab;Drug: Mycophenolate mofetil;Drug: Tacrolimus;Drug: Cyclosporine;Drug: Rabbit anti-thymocyte globulin;Procedure: Blood Draw;Other: Thymus Tissue and Parental Parathyroid Transplantation;Biological: Thymus Tissue for Transplantation | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Not recruiting | N/A | N/A | All | 28 | Phase 1/Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 221 | NCT00576836 | February 2006 | 20 May 2019 | Thymus Transplantation Dose in DiGeorge #932 | Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1 | DiGeorge Anomaly;DiGeorge Syndrome;Complete DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Thymus Tissue for Transplantation;Other: Parathyroid Tissue for Transplantation | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Not recruiting | N/A | N/A | All | 28 | Phase 2 | United States |
| 222 | NCT00278954 | January 2006 | 19 October 2017 | Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. | A Phase III, Multicenter, Open-Label Study To Evaluate The Efficacy, Safety, and Pharmacokinetics of Gammaplex® in Primary Immunodeficiency Diseases | Primary Immunodeficiency;Common Variable Hypogammaglobulinemia;X-linked Hypogammaglobulinemia;Hypogammaglobulinemia;Immunodeficiency With Hyper-IgM;Wiskott-Aldrich Syndrome | Biological: Gammaplex (Intravenous immunoglobulin) | Bio Products Laboratory | Not recruiting | 3 Years | N/A | All | 50 | Phase 3 | United States | |
| 223 | NCT00263237 | December 2, 2005 | 16 December 2017 | STA-5326 Meslylate to Treat Gut Inflammation Associated With Common Variable Immunodeficiency | A Pilot Study of Safety and Efficacy of the Oral IL-12/23 Inhibitor, STA-5326 Mesylate, for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | Common Variable Immunodeficiency | Drug: STA-5326 | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 18 Years | 75 Years | All | 10 | Phase 1 | United States | |
| 224 | NCT00294567 | December 2005 | 19 February 2015 | Azelnidipine Anti-Coronary Atherosclerotic Trial in Hypertensive Patients by Serial Volumetric IVUS Analysis(ALPS-J) | Azelnidipine and Amlodipine Anti-Coronary Atherosclerotic Trial in Hypertensive Patients Undergoing Coronary Intervention by Serial Volumetric Intravascular Ultrasound Analysis in Junten Medical University (ALPS-J) | Hypertension;Coronary Atherosclerosis | Drug: Calcium channel blockers (amlodipine, azelnidipine) | Juntendo University Hospital | Japan Heart Foundation | Not recruiting | 20 Years | 79 Years | Both | 200 | Phase 4 | Japan |
| 225 | NCT00260702 | November 24, 2005 | 16 December 2017 | Omalizumab to Treat Hyper-IgE (Job's) Syndrome | Pilot Study of Omalizumab (Xolair) in Hyper IgE (Job's) Syndrome | Hyper-IgE Syndrome;Job's Syndrome | Drug: Omalizumab (Xolair) | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 6 Years | 76 Years | All | 1 | Phase 1 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 226 | NCT00322556 | November 2005 | 19 October 2017 | Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID) | A Multicenter Extension Study on the Safety and Efficacy of IgPro10 in Patients With Primary Immunodeficiency (PID) | Agammaglobulinemia;IgG Deficiency;Common Variable Immunodeficiency | Drug: Immunoglobulins Intravenous (Human) | CSL Behring | Not recruiting | 4 Years | 71 Years | All | 55 | Phase 3 | United States | |
| 227 | NCT00119431 | September 2005 | 19 February 2015 | Kinetics, Efficacy and Safety of C1-Esteraseremmer-N | Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema | Hereditary Angioedema Type I;Angioneurotic Edema | Drug: C1 inhibitor concentrate | Sanquin | Not recruiting | 18 Years | N/A | Both | 12 | Phase 2 | Netherlands | |
| 228 | NCT00160355 | May 2005 | 16 December 2017 | Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome | Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study | Wiskott-Aldrich Syndrome | Procedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, Thiotepa | St. Jude Children's Research Hospital | Not recruiting | N/A | 18 Years | Male | 4 | Phase 1 | United States | |
| 229 | EUCTR2004-004465-15-DE | 05/04/2005 | 19 March 2012 | A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID) | A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID) | primary immunodeficiency syndrome as congenital agammaglobulinaemia or hypogammaglobulinaemia, common variable immunodeficiency, severe combined immunodeficiencies, Wiskott Aldrich syndrome MedDRA version: 7.0 Level: HLT Classification code 10036700 | Product Name: Intratect Product Code: BT681 Pharmaceutical Form: Solution for infusion Current Sponsor code: BT681 (Intratect) Other descriptive name: human normal immunoglobulin (IVIg) Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | Biotest AG | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Germany | ||||
| 230 | NCT00295971 | April 2005 | 19 February 2015 | Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease | Stem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency Diseases | Congenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: anti-thymocyte globulin;Biological: therapeutic allogeneic lymphocytes;Drug: fludarabine phosphate;Drug: thiotepa;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: total-body irradiation | University of California, San Francisco | National Cancer Institute (NCI) | Not recruiting | 1 Year | 17 Years | Both | 21 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 231 | NCT00301834 | January 2005 | 16 December 2017 | Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders | Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation | University of California, San Francisco | National Cancer Institute (NCI) | Not recruiting | N/A | 21 Years | All | 35 | Phase 2 | United States |
| 232 | NCT00566488 | January 2005 | 20 May 2019 | Parathyroid and Thymus Transplantation in DiGeorge #931 | Parathyroid and Thymus Transplantation in DiGeorge Syndrome, #931 | DiGeorge Syndrome;Hypoparathyroidism;Complete DiGeorge Syndrome | Biological: Thymus/Parathyroid Transplantation | M. Louise Markert | Food and Drug Administration (FDA);National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Not recruiting | N/A | 24 Months | All | 25 | Phase 1 | United States |
| 233 | NCT00123916 | November 2004 | 19 February 2015 | BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease | Benznidazole Evaluation for Interrupting Trypanosomiasis - The BENEFIT Trial | Chagas Disease;Trypanosomiasis;Heart Disease | Drug: Benznidazole;Drug: Placebo | Population Health Research Institute | Canadian Institutes of Health Research (CIHR);World Health Organization;Dante Pazzanese Institute;Hospital das Clinicas de Ribeirao Preto/USP | Recruiting | 18 Years | 75 Years | Both | 1000 | Phase 3 | Argentina;Bolivia;Brazil;Colombia;El Salvador;Canada |
| 234 | NCT00146627 | September 2004 | 10 October 2016 | Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis | Clinical Study Comparing the Nifurtimox-Eflornithine Combination With the Standard Eflornithine Regimen for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis in the Meningoencephalitic Phase | Trypanosomiasis, African | Drug: Eflornithine;Drug: Nifurtimox | Drugs for Neglected Diseases | Medecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health Organization;Medecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health Organization | Not recruiting | 15 Years | 70 Years | Both | 280 | Phase 3 | Congo, The Democratic Republic of the;Congo;The Democratic Republic of the Congo;Uganda |
| 235 | NCT00168012 | September 2004 | 19 February 2015 | Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID) | An Open Study to Evaluate the Safety and Efficacy of IVIG-F10 in Patients With Primary Immunodeficiency Diseases (PID) | Agammaglobulinemia;IgG Deficiency;Common Variable Immunodeficiency | Drug: Immunoglobulins Intravenous (Human) | CSL Behring | Not recruiting | 3 Years | 70 Years | Both | 42 | Phase 3 | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 236 | NCT00168025 | September 2004 | 19 February 2015 | Efficacy and Safety of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID) | A Multicenter Study on the Efficacy, Safety and Pharmacokinetics of IgPro10 in Patients With Primary Immunodeficiency (PID) | Agammaglobulinemia;IgG Deficiency;Common Variable Immunodeficiency | Drug: Immunoglobulins Intravenous (Human) | CSL Behring | Not recruiting | 3 Years | 70 Years | Both | 89 | Phase 3 | ||
| 237 | NCT00228852 | April 2004 | 19 February 2015 | IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency | Phase I/II Trial of De-Escalation of Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency | T-Cell Immune Deficiency Diseases;Severe Combined Immunodeficiency | Drug: Busulfan, Fludarabine and ATG | Emory University | Not recruiting | N/A | N/A | Both | Phase 1/Phase 2 | United States | ||
| 238 | NCT00578643 | March 2004 | 2 July 2018 | Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease | HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Busulfan;Biological: Alemtuzumab;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Cyclosporine;Procedure: Stem Cell Infusion | Baylor College of Medicine | Texas Children's Hospital | Not recruiting | N/A | N/A | All | 15 | Phase 2 | United States |
| 239 | NCT00152100 | February 2004 | 19 February 2015 | Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome | Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome | Severe Combined Immunodeficiency | Procedure: Stem cell transplant;Drug: Filgrastim, Alemtuzumab;Device: Miltenyi CliniMACS | St. Jude Children's Research Hospital | Not recruiting | N/A | 2 Years | Both | 4 | Phase 1 | United States | |
| 240 | NCT00564759 | January 2004 | 19 February 2015 | Gene Therapy for Chronic Granulomatous Disease | Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease | Granulomatous Disease, Chronic | Drug: retroviral SF71-gp91phox transduced CD34+ cells | Johann Wolfgang Goethe University Hospitals | German Federal Ministry of Education and Research | Not recruiting | 18 Years | N/A | Male | 2 | Phase 1/Phase 2 | Germany |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 241 | NCT01279720 | October 2003 | 21 September 2015 | Gene Therapy ADA Deficiency | Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency | Adenosine Deaminase Deficiency | Biological: Intravenous infusion of transduced cells | Great Ormond Street Hospital for Children NHS Foundation Trust | Not recruiting | N/A | 18 Years | Both | 8 | Phase 1/Phase 2 | United Kingdom | |
| 242 | NCT00065390 | July 2003 | 19 February 2015 | Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome | Pilot (Phase I-II) Study of Pyrimethamine (Daraprim) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS) | Autoimmune Disease;Lymphatic Disease;Lymphoproliferative Disorder | Drug: Pyrimethamine | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | Both | 8 | Phase 1 | United States | |
| 243 | NCT00803933 | February 2003 | 19 February 2015 | Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | Phase II b Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | African Trypanosomiasis | Drug: DB289;Drug: Pentamidine | Immtech Pharmaceuticals, Inc | Bill and Melinda Gates Foundation | Not recruiting | 15 Years | 50 Years | Both | 111 | Phase 2 | Congo |
| 244 | NCT00489658 | October 2002 | 19 February 2015 | Eflornithine + Nifurtimox Late-Stage Human African Trypanosomiasis (HAT)in West Nile, Uganda | Efficacy and Safety of an Eflornithine + Nifurtimox Combination for Treatment of Late-Stage Human African Trypanosomiasis (HAT) in West Nile, Uganda | Trypanosomiasis, African | Drug: Eflornithine plus Nifurtimox combination therapy | Epicentre | Medecins Sans Frontieres | Not recruiting | N/A | N/A | Both | 31 | Phase 2/Phase 3 | Uganda |
| 245 | NCT00220766 | August 2002 | 19 February 2015 | Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients | IGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient Patients | Immunologic Deficiency Syndrome;Agammaglobulinemia;Severe Combined Immunodeficiency;Wiskott-Aldrich Syndrome;Common Variable Immunodeficiency | Drug: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified;Drug: Dextrose, 5% in Water | Grifols Therapeutics Inc. | Not recruiting | 18 Years | 75 Years | Both | 100 | Phase 3 | United States;Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 246 | NCT00579709 | July 2002 | 29 July 2019 | Thymus Transplantation With Immunosuppression | Thymus Transplantation With Immunosuppression, #884 | DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Thymus Tissue for Transplantation | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Not recruiting | N/A | N/A | All | 15 | Phase 1 | United States |
| 247 | NCT00157079 | June 25, 2002 | 29 January 2018 | Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency Disorders | A Clinical Investigation to Assess the Safety and Efficacy of Immune Globulin Intravenous (Human), 10% in Subjects With Primary Immunodeficiency Disorders | Primary Immunodeficiency Diseases (PID);Immune Thrombocytopenic Purpura (ITP);Kawasaki Syndrome | Biological: Immune Globulin Intravenous (Human), 10% | Baxalta now part of Shire | Not recruiting | 24 Months | N/A | All | Phase 3 | United States | ||
| 248 | NCT00161993 | June 13, 2002 | 26 August 2019 | Safety, Pharmacokinetic and Efficacy Study of a 10% Triple Virally Reduced Intravenous Immune Globulin Solution in Patients With Primary Immunodeficiency (Hypo- or Agammaglobulinemia) | Prospective Open-Label Study of Pharmacokinetics, Efficacy and Safety of Immune Globulin Intravenous (Human), 10% TVR Solution in Patients With Hypo- or Agammaglobulinemia | Primary Immunodeficiency Diseases (PID);Agammaglobulinemia;Hypogammaglobulinemia | Drug: Immune Globulin Intravenous (Human), 10% TVR (Triple Virally Reduced) Solution;Drug: Gammagard S/D (Solvent/Detergent) | Baxalta now part of Shire | Not recruiting | 18 Years | N/A | All | Phase 2 | Finland;Sweden | ||
| 249 | NCT00176852 | June 2002 | 1 October 2018 | Stem Cell Transplant for Hemoglobinopathy | Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism | Sickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond Syndrome | Drug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusion | Masonic Cancer Center, University of Minnesota | National Marrow Donor Program | Not recruiting | N/A | 50 Years | All | 22 | Phase 2/Phase 3 | United States |
| 250 | NCT01019876 | June 2002 | 19 February 2015 | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases | Bone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined Immunodeficiency | Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30 | Columbia University | Recruiting | N/A | 30 Years | Both | 50 | Phase 2/Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 251 | NCT00661401 | January 2002 | 19 February 2015 | Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin | Serum IgG Antibody to Streptococcus Pneumoniae, Haemophilus Influenzae Type b and Tetanus Toxoid in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin Infusions | Common Variable Immunodeficiency;Agammaglobulinemia | Biological: gammaglobulin | Federal University of São Paulo | CSL Behring | Not recruiting | 2 Years | 75 Years | Both | 5 | N/A | Brazil |
| 252 | NCT00028236 | December 10, 2001 | 16 December 2017 | Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) | Ex Vivo Retroviral Gene Transfer For Treatment of X-Linked Severe Combined Immunodeficiency (XSCID) | Severe Combined Immunodeficiency | Drug: Gene-Transduced Autologous CD34+ Stem Cells | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 18 Months | 20 Years | All | 3 | Phase 1 | United States | |
| 253 | NCT00576407 | November 2001 | 30 September 2019 | Thymus Transplantation in DiGeorge Syndrome #668 | Phase II Study of Thymus Transplantation in Complete DiGeorge Syndrome #668 | DiGeorge Syndrome;Complete Typical DiGeorge Anomaly | Biological: Thymus Tissue for Transplantation | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Not recruiting | N/A | N/A | All | 26 | Phase 2 | United States |
| 254 | NCT00024934 | October 2001 | 3 October 2016 | B-Lymphocyte Stimulator (BLyS) To Treat Selective IgA Deficiency | A Phase I Dose Escalation Study of B-Lymphocyte Stimulator (BLyS) Administered Subcutaneously in Patients With Selective IgA Deficiency | IgA Deficiency | Drug: B-Lymphocyte Stimulator (BLyS) | National Cancer Institute (NCI) | Not recruiting | N/A | N/A | Both | 20 | Phase 1 | United States | |
| 255 | NCT00023192 | August 2001 | 19 February 2015 | Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care | Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care | Chronic Granulomatous Disease | Drug: T-Cell Depleted & CD34+Select/w/StemCell Enriched Product | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | Both | 60 | Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 256 | NCT00802594 | August 2001 | 19 February 2015 | A Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | Phase II A Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | Trypanosomiasis, African | Drug: DB289 | Immtech Pharmaceuticals, Inc | Bill and Melinda Gates Foundation | Not recruiting | 16 Years | N/A | Both | 30 | Phase 2 | Angola;Congo |
| 257 | NCT00018018 | June 20, 2001 | 11 November 2019 | Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency | Treatment of SCID Due to ADA Deficiency With Autologous Cord Blood or Bone Marrow CD34+ Cells Transduced With a Human ADA Gene | Severe Combined Immunodeficiency Syndrome | Drug: CD34+ cells transduced with ADA retrovir | National Human Genome Research Institute (NHGRI) | Not recruiting | 1 Month | N/A | All | 8 | Phase 1 | United States | |
| 258 | NCT00013689 | March 2001 | 19 February 2015 | Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome | Pilot Study of Pyrimethamine and Sulfadoxine (Fansidar) for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome (ALPS) | Autoimmune Disease;Lymphoproliferative Disorder | Drug: Fansidar (pyrimethamine and sulfadoxine) | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | Both | 8 | Phase 1 | United States | |
| 259 | NCT00330148 | March 2001 | 19 February 2015 | Randomized Clinical Trial of Three Drug Combinations for Late-Stage Gambiense Human African Trypanosomiasis | Clinical Trial Comparing the Therapeutic Combinations Melarsoprol-Nifurtimox, Melarsoprol-Eflornithine and Eflornithine-Nifurtimox in the Treatment of Gambiense Human African Trypanosomiasis in the Meningo-Encephalitic Phase | Trypanosomiasis, African | Drug: melarsoprol 1.8 mg/kg/d, 10d + nifurtimox 15/20 mg/kg/d, 10d;Drug: melarsoprol 1.8 mg/kg/d, 10d + eflornithine 400 mg/kg/d, 7d;Drug: nifurtimox 15/20 mg/kg/d 10d + eflornithine 400 mg/kg/d 7d | Epicentre | Medecins Sans Frontieres;Embassy of France in Uganda;National Sleeping Sickness Control Program, Uganda | Not recruiting | N/A | N/A | Both | 435 | Phase 3 | Uganda |
| 260 | NCT00031486 | September 2000 | 19 October 2017 | Long Term Treatment of Herpes Simplex Encephalitis (HSE) With Valacyclovir | A Phase III Double-Blind, Placebo-Controlled Trial of Long Term Therapy of Herpes Simplex Encephalitis (HSE): An Evaluation of Valacyclovir (CASG-204) | Encephalitis | Drug: Valacyclovir;Drug: Placebo | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 12 Years | N/A | All | 91 | Phase 3 | United States;Canada;Sweden;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 261 | NCT00305708 | August 2000 | 19 February 2015 | Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission | Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;Thrombocytopenia | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapy | University of California, San Francisco | National Cancer Institute (NCI) | Not recruiting | N/A | 17 Years | Both | 40 | Phase 1/Phase 2 | United States |
| 262 | NCT00176878 | June 2000 | 19 October 2017 | Stem Cell Transplant for Bone Marrow Failure Syndromes | Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders | Diamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond Syndrome | Procedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulin | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | 35 Years | All | 10 | Phase 2/Phase 3 | United States | |
| 263 | NCT00006054 | March 2000 | 19 February 2015 | Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies | Immunologic Deficiency Syndromes;Chediak-Higashi Syndrome;Common Variable Immunodeficiency;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Hyper IgM Syndrome;Severe Combined Immunodeficiency;Leukocyte Adhesion Deficiency Syndrome;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow Transplantation | Fairview University Medical Center | Not recruiting | N/A | 35 Years | Both | N/A | United States | |||
| 264 | NCT00006056 | March 2000 | 19 February 2015 | Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders | Chediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantation | Fairview University Medical Center | Not recruiting | N/A | 55 Years | Both | 40 | N/A | United States | ||
| 265 | NCT00001145 | October 1999 | 19 February 2015 | Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM Syndrome | Study of Immune Responses and Safety of Recombinant CD40 Ligand in Patients With X-Linked Hyper IgM Syndrome | Immunoproliferative Disorder | Drug: Bacteriophage;Drug: rhuCD40L;Drug: KLH | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | Both | 5 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 266 | NCT00001905 | April 1999 | 19 February 2015 | Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I | Interferon Gamma Administration in Leukocyte Adhesion Deficiency Type I | Leukocyte Adhesion Deficiency Syndrome | Drug: Interferon gamma | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | Both | 5 | Phase 2 | United States | |
| 267 | NCT00004695 | September 1997 | 7 April 2015 | Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable Immunodeficiency | Common Variable Immunodeficiency | Drug: PEG-interleukin-2 | Mount Sinai School of Medicine | Not recruiting | 2 Years | N/A | Both | 48 | N/A | |||
| 268 | NCT00008450 | August 11, 1997 | 26 August 2019 | Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant | Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil | Adenosine Deaminase Deficiency;Autosomal Recessive Disorder;Immune System Disorder;Purine-Nucleoside Phosphorylase Deficiency;Severe Combined Immunodeficiency;Severe Combined Immunodeficiency With Absence of T and B Cells;X-Linked Severe Combined Immunodeficiency | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | N/A | N/A | All | 6 | Phase 1 | United States |
| 269 | NCT00001476 | June 1, 1995 | 16 December 2017 | Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up | Gene Therapy Approach for Chronic Granulomatous Disease | Chronic Granulomatous Disease;Communicable Disease | Drug: Gene Therapy Method for CGD;Device: Isolex 300i Magnetic Cell Selector | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 5 Years | N/A | All | 14 | Phase 1 | United States | |
| 270 | NCT00004787 | December 1994 | 19 February 2015 | Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes | Shwachman Syndrome;Fanconi's Anemia;Dyskeratosis Congenita;Thrombocytopenia | Drug: filgrastim | National Center for Research Resources (NCRR) | James Whitcomb Riley Hospital for Children | Not recruiting | N/A | N/A | Both | 20 | Phase 2 | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 271 | NCT00001317 | May 1992 | 19 February 2015 | A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood | A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood | Chronic Granulomatous Disease | Drug: interferon-gamma | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | Both | 100 | Phase 4 | United States | |
| 272 | NCT00001280 | January 1991 | 19 February 2015 | Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous Disease | Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous Disease | Mycoses | Drug: itraconazole | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | N/A | N/A | Both | 100 | Phase 2 | United States | |
| 273 | NCT00001255 | September 1990 | 19 February 2015 | Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study | Treatment of Severe Combined Immunodeficiency Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency With Autologous Lymphocytes of CD34+ Cells Transduced With a Human ADA Gene: A Natural History Study | Severe Combined Immunodeficiency | Drug: ADA PBSC;Drug: ADA Umbilical Cord Blood Cells;Drug: Transduced Lymphocytes | National Human Genome Research Institute (NHGRI) | Not recruiting | N/A | N/A | Both | 10 | N/A | United States |