65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04370795 (ClinicalTrials.gov) | March 24, 2022 | 30/4/2020 | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Severe Combined Immunodeficiency (SCID) | Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradiation (TBI) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Enrolling by invitation | 3 Years | 40 Years | All | 30 | Phase 1/Phase 2 | United States |
| 2 | NCT03547830 (ClinicalTrials.gov) | April 13, 2019 | 24/5/2018 | 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 | NULL | Recruiting | 1 Month | 24 Years | All | 17 | Phase 2 | Russian Federation |
| 3 | EUCTR2018-003842-18-IT (EUCTR) | 08/01/2019 | 19/11/2018 | Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS) | A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS. | Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematological Agents Other descriptive name: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence Trade Name: Busilvex INN or Proposed INN: BUSULFAN Other descriptive name: NA Trade Name: Fludarabina Accord INN or Proposed INN: FLUDARABINE Other descriptive name: NA Trade Name: MabThera INN or Proposed INN: RITUXIMAB Other descriptive name: NA Trade Name: Mozobil, INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR Trade Name: MYELOSTIM Product Name: granulocyte colony stimulating factor (G-CSF) INN or Proposed INN: | Orchard Therapeutics Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 6 | Phase 3 | Italy | ||
| 4 | NCT03019809 (ClinicalTrials.gov) | June 2016 | 11/1/2017 | 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: G-CSF for Conditioning before HSCT.;Biological: Plerixafor for Conditioning before HSCT. | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Unknown status | 1 Month | 19 Years | All | 30 | Phase 2 | Russian Federation |
| 5 | NCT03055247 (ClinicalTrials.gov) | November 2015 | 21/7/2016 | Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD | A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD | Chronic Granulomatous Disease X-linked (X-CGD) | Drug: Ibuprofen;Drug: Myelostim;Drug: Mozobil | IRCCS San Raffaele | Fondazione Telethon | Recruiting | 18 Years | 45 Years | Male | 3 | Phase 2 | Italy |
| 6 | EUCTR2015-002356-27-IT (EUCTR) | 16/10/2015 | 29/07/2015 | 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 INN or Proposed INN: IBUPROFEN Other descriptive name: NA 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 INN or Proposed INN: LENOGRASTIM Trade Name: Mozobil 20mg/mL vial (injectable solution for subcutaneous use) Product Name: Mozobil 20mg/mL vial (injectable solution, subcutaneous use) INN or Proposed INN: Plerixafor Other descriptive name: Plerixafor Product Name: Pantoprazolo 20 mg gastro-resistant tablets INN or Proposed INN: Pantoprazole Other descriptive name: PANTOPRAZOLE | Ospedale San Raffaele | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 3 | Phase 2 | Italy | ||
| 7 | NCT02231879 (ClinicalTrials.gov) | October 14, 2014 | 3/9/2014 | 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) | NULL | Completed | 10 Years | 75 Years | All | 20 | Phase 2/Phase 3 | United States |
| 8 | EUCTR2011-001118-32-SE (EUCTR) | 03/02/2012 | 06/12/2011 | 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: 18.0;Level: PT;Classification code 10051645;Term: Idiopathic neutropenia;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 18.0;Classification code 10053176;Term: Cyclic neutropenia;Level: LLT;Classification code 10069819;Term: Congenital neutropenia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Zarzio INN or Proposed INN: FILGRASTIM Trade Name: Zarzio INN or Proposed INN: FILGRASTIM | Sandoz GmbH | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | Germany;Sweden | ||
| 9 | EUCTR2011-001118-32-DE (EUCTR) | 25/05/2011 | 27/04/2011 | 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;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 INN or Proposed INN: FILGRASTIM Trade Name: Filgrastim Hexal INN or Proposed INN: FILGRASTIM Product Name: EP2006 (Filgrastim) Product Code: EP2006 INN or Proposed INN: FILGRASTIM | Sandoz GmbH | NULL | Not Recruiting | Female: yes Male: yes | 40 | Germany;Sweden | |||
| 10 | NCT01182675 (ClinicalTrials.gov) | August 2010 | 9/8/2010 | 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 | NULL | Terminated | N/A | 3 Years | All | 7 | Phase 2 | United States |
| 11 | NCT00152100 (ClinicalTrials.gov) | February 2004 | 7/9/2005 | 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 | NULL | Completed | N/A | 2 Years | Both | 4 | Phase 1 | United States |
| 12 | NCT00006056 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | 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 | NULL | Active, not recruiting | N/A | 55 Years | Both | 40 | N/A | United States | |
| 13 | NCT00004787 (ClinicalTrials.gov) | December 1994 | 24/2/2000 | 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 | Completed | N/A | N/A | Both | 20 | Phase 2 | NULL |