65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 500 / Drugs : 614 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 217
| 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 | NCT03663933 (ClinicalTrials.gov) | September 4, 2018 | 7/9/2018 | 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;Drug: Reduced Intensity Conditioning;Drug: GVHD Prophylaxis;Procedure: Allogeneic HSC | National Cancer Institute (NCI) | NULL | Recruiting | 4 Years | N/A | All | 177 | Phase 2 | United States |
| 2 | JPRN-jRCTs031180398 | 30/05/2018 | 20/03/2019 | A phase II study of RIC-SCT for CGD | 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 Primary immunodeficiency;D006105 | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | Sakaguchi Hirotoshi | NULL | Recruiting | Not applicable | < 25age old | Both | 22 | Phase 2 | None (Japan only);Japan |
| 3 | JPRN-UMIN000030647 | 2017/12/30 | 31/12/2017 | 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) | 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) - A phase II study of RIC-SCT for CGD | 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 | NULL | Recruiting | Not applicable | 25years-old | Male and Female | 22 | Phase 2 | Japan |
| 4 | JPRN-UMIN000022688 | 2016/06/10 | 13/06/2016 | A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease | A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease - A pilot study of RIC-SCT for CGD | 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 | NULL | Complete: follow-up complete | Not applicable | 25years-old | Male and Female | 9 | Not selected | Japan |
| 5 | NCT02579967 (ClinicalTrials.gov) | November 19, 2015 | 16/10/2015 | 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) | NULL | Recruiting | 4 Years | 75 Years | All | 224 | Phase 2 | United States |
| 6 | 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 |
| 7 | NCT01998633 (ClinicalTrials.gov) | December 2013 | 29/10/2013 | 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 | Completed | 4 Months | 45 Years | All | 47 | Phase 2 | United States;Canada |