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
Showing 1 to 8 of 8 diseases
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 | JPRN-jRCT2031210309 | 20/12/2022 | 13/09/2021 | RALPID | Phase 2 study of the efficacy and safety of sirolumus in patients with primary immunodeficiency | Primary immunodeficiency PID, ALPS, APDS, CTLA4, IPEX, lymphoproliferative disease, enterocolitis, cytopenia;D000081207 Primary immunodeficiency PID, ALPS, APDS, CTLA4, IPEX, lymphoproliferative disease, enterocolitis, c ... | Administer sirolimus tablets or granules once a daily. Initial dose: 2mg (tablet), 0.049 mg/kg or 0.035 mg/kg (granule) Administer sirolimustablets or granules once a daily. Initial dose: 2mg (tablet), 0.049 mg/kg or 0.03 ... | Imai Kohsuke | NULL | Recruiting | >= 2month old | Not applicable | Both | 10 | Phase 2 | Japan |
2 | 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 ... | 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 ... | Severe Combined Immunodeficiency (SCID) | Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradiation (TBI) Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradia ... | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Enrolling by invitation | 3 Years | 40 Years | All | 30 | Phase 1/Phase 2 | United States |
3 | NCT03910452 (ClinicalTrials.gov) | October 28, 2019 | 9/4/2019 | Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Bus ... | Haploidentical Transplant for Patients 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, B ... | Chronic Granulomatous Disease | Drug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cell Drug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradi ... | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Suspended | 4 Years | 65 Years | All | 4 | Early Phase 1 | United States |
4 | NCT02629120 (ClinicalTrials.gov) | December 17, 2015 | 10/12/2015 | 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 Patie ... | 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 Patie ... | Chronic Granulomatous Disease Transplant | Drug: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cells Drug: Alentuzumab (Campath);Drug: BusulfanIV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total ... | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 4 Years | 65 Years | All | 50 | Phase 1/Phase 2 | United States |
5 | NCT02282904 (ClinicalTrials.gov) | October 23, 2014 | 4/11/2014 | Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclop ... | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplan ... | 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 Drug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamidepost transplant ... | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 2 Years | 65 Years | All | 7 | Phase 1/Phase 2 | United States |
6 | NCT02177760 (ClinicalTrials.gov) | July 2014 | 19/6/2014 | 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) Sirolimusin Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID ... | Severe Combined Immunodeficiency;Transplacental Maternal Engraftment;Stem Cell Transplant | Drug: Sirolimus | University of California, San Francisco | NULL | Withdrawn | N/A | 1 Year | Both | 0 | Phase 2 | United States |
7 | NCT00392951 (ClinicalTrials.gov) | December 2006 | 24/10/2006 | 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 Autoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thr ... | Drug: sirolimus | Children's Hospital of Philadelphia | NULL | Completed | 1 Year | 30 Years | All | 30 | Phase 1/Phase 2 | United States |
8 | 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 Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Tr ... | 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 HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other N ... | 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 Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: FludarabinePhosphate; ... | 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 |