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 | NCT04965597 (ClinicalTrials.gov) | March 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Research Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
| 2 | NCT03597594 (ClinicalTrials.gov) | September 2, 2021 | 19/6/2018 | 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 | Device: CliniMACS;Other: Donor Lymphocyte Infusion;Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa | St. Jude Children's Research Hospital | NULL | Recruiting | 2 Months | N/A | All | 42 | Phase 1/Phase 2 | United States |
| 3 | NCT04339777 (ClinicalTrials.gov) | September 22, 2020 | 8/4/2020 | Allogeneic Hematopoietic Stem Cell Transplant for People With Primary Immunodeficiency Diseases | A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immunodeficiency Diseases | Lymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable Immunodeficiency;Primary T-cell Immunodeficiency Disorders | Drug: Busulfan test dose;Drug: Fludarabine;Drug: Busulfan;Drug: Alemtuzumab;Radiation: Total body Irradiation;Procedure: Allogeneic HSCT;Drug: Tacrolimus (Tacro);Drug: Mycophenolate mofetil (MMF);Drug: Cyclophosphamide (Cytoxan) | National Cancer Institute (NCI) | NULL | Suspended | 4 Years | 69 Years | All | 10 | Phase 2 | United States |
| 4 | 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 | ||
| 5 | EUCTR2018-001489-41-SE (EUCTR) | 02/07/2018 | 08/05/2018 | 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 INN or Proposed INN: Alemtuzumab Other descriptive name: ALEMTUZUMAB Trade Name: Fludarabine INN or Proposed INN: FLUDARABINE | Karolinska Universitetssjukhuset | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Sweden | ||
| 6 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | 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 | 3 Months | 39 Years | All | 40 | Phase 1/Phase 2 | United States |
| 7 | JPRN-UMIN000030806 | 2018/01/17 | 15/01/2018 | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - 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 | NULL | Complete: follow-up continuing | Not applicable | Not applicable | Male | 3 | Phase 1,2 | Japan |
| 8 | NCT03333486 (ClinicalTrials.gov) | December 7, 2017 | 2/11/2017 | 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 | NULL | Recruiting | 1 Year | 75 Years | All | 58 | Phase 2 | United States |
| 9 | JPRN-UMIN000019532 | 2015/12/01 | 01/12/2015 | Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases | Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases - HSCT for ataxia telangiectasia and related diseases | ataxia telangiectasiaDNA damage response disorder | fludarabine 30 mg/m2x5, cyclophosphamide 5mg/kgx4, ATG 1.25mg/kgx4 | Graduate Schoool,Dept. of Community Pediatrics,Perinatal and Maternal Medicine,Tokyo Medical and Dental University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 5 | Phase 2 | Japan |
| 10 | NCT02162420 (ClinicalTrials.gov) | January 2015 | 10/6/2014 | 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 | NULL | Recruiting | N/A | 70 Years | All | 50 | N/A | United States |
| 11 | NCT02282904 (ClinicalTrials.gov) | October 23, 2014 | 4/11/2014 | 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) | NULL | Terminated | 2 Years | 65 Years | All | 7 | Phase 1/Phase 2 | United States |
| 12 | NCT01962415 (ClinicalTrials.gov) | February 4, 2014 | 10/10/2013 | 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 =55 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 | NULL | Recruiting | 2 Months | 55 Years | All | 100 | Phase 2 | United States |
| 13 | NCT01821781 (ClinicalTrials.gov) | March 2013 | 19/3/2013 | 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 | NULL | Recruiting | N/A | 21 Years | All | 20 | Phase 2 | United States |
| 14 | NCT01652092 (ClinicalTrials.gov) | September 4, 2012 | 25/7/2012 | 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: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg;Drug: Fludarabine phosphate 30 mg;Drug: MESNA | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 50 Years | All | 30 | N/A | United States |
| 15 | NCT01659606 (ClinicalTrials.gov) | July 2012 | 6/8/2012 | 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;University of Wisconsin, Madison;Karolinska University Hospital;Hackensack Meridian Health;Duke University;Oslo University Hospital;Children's Mercy Hospital Kansas City;Mayo Clinic;University of Chicago;Massachusetts General Hospital | Recruiting | N/A | 65 Years | All | 40 | Phase 2 | United States;Norway;Sweden |
| 16 | NCT01529827 (ClinicalTrials.gov) | February 28, 2012 | 6/2/2012 | 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 | 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;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;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 | 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 | NULL | Completed | 3 Years | 75 Years | All | 94 | Phase 2 | United States |
| 17 | NCT01338675 (ClinicalTrials.gov) | January 2011 | 4/3/2011 | Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD) | Chronic Granulomatous Disease | Drug: Busulfan | Seoul National University Hospital | NULL | Recruiting | N/A | N/A | Both | 5 | Phase 1/Phase 2 | Korea, Republic of | |
| 18 | EUCTR2009-017346-32-IT (EUCTR) | 15/03/2010 | 26/04/2010 | HAEMATOPOIETIC STEM CELL GENE THERAPY | A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS | Wiskott-Aldrich Syndrom 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] | Trade Name: FLUDARABINA TEVA - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 1 FLACONCINO DI VETRO DA 2 ML Product Name: na Product Code: [na] INN or Proposed INN: FLUDARABINA Other descriptive name: FLUDARABINA Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - FLACONCINO (VETRO) - 24 MG/1.2 ML 1 FLACONCINO Product Name: PLERIXAFOR Product Code: [na] INN or Proposed INN: PLERIXAFOR Other descriptive name: Plerixafor Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML Product Name: na Product Code: [na] INN or Proposed INN: LENOGRASTIM Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML Product Name: RITUXIMAB Product Code: [na] INN or Proposed INN: RITUXIMAB Other descriptive name: RITUXIMAB Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI Product Name: na Product Code: [na] | Orchard Therapeutics (Europe) Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 8 | Phase 1;Phase 2 | Italy | ||
| 19 | NCT00579137 (ClinicalTrials.gov) | October 2007 | 19/12/2007 | 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 | Terminated | N/A | N/A | All | 3 | Phase 1/Phase 2 | United States |
| 20 | NCT00455312 (ClinicalTrials.gov) | August 2007 | 30/3/2007 | 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 | NULL | Completed | N/A | 70 Years | All | 36 | Phase 2/Phase 3 | United States |
| 21 | NCT00885833 (ClinicalTrials.gov) | February 2007 | 21/4/2009 | 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 | NULL | Completed | 1 Year | 25 Years | Both | 5 | Phase 1/Phase 2 | Korea, Republic of |
| 22 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | 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) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| 23 | NCT00160355 (ClinicalTrials.gov) | May 2005 | 8/9/2005 | 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 | NULL | Completed | N/A | 18 Years | Male | 4 | Phase 1 | United States |
| 24 | NCT00295971 (ClinicalTrials.gov) | April 2005 | 23/2/2006 | 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) | Completed | 1 Year | 17 Years | Both | 21 | Phase 1 | United States |
| 25 | NCT00301834 (ClinicalTrials.gov) | January 2005 | 9/3/2006 | 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) | Completed | N/A | 21 Years | All | 35 | Phase 2 | United States |
| 26 | NCT00228852 (ClinicalTrials.gov) | April 2004 | 27/9/2005 | 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 | NULL | Completed | N/A | N/A | Both | Phase 1/Phase 2 | United States | |
| 27 | NCT00578643 (ClinicalTrials.gov) | March 2004 | 19/12/2007 | 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 | NULL | Completed | N/A | N/A | All | 15 | Phase 2 | United States |
| 28 | NCT01019876 (ClinicalTrials.gov) | June 2002 | 23/11/2009 | 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 | NULL | Recruiting | N/A | 30 Years | Both | 50 | Phase 2/Phase 3 | United States |
| 29 | NCT00176852 (ClinicalTrials.gov) | June 2002 | 12/9/2005 | 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 | Completed | N/A | 50 Years | All | 22 | Phase 2/Phase 3 | United States |
| 30 | NCT00305708 (ClinicalTrials.gov) | August 2000 | 21/3/2006 | 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) | Completed | N/A | 17 Years | Both | 40 | Phase 1/Phase 2 | United States |
| 31 | NCT00176878 (ClinicalTrials.gov) | June 2000 | 12/9/2005 | 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 | NULL | Completed | N/A | 35 Years | All | 10 | Phase 2/Phase 3 | United States |