65. 原発性免疫不全症候群
［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］
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. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT03597594 (ClinicalTrials.gov) | December 2020 | 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 | Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte Infusion | St. Jude Children's Research Hospital | NULL | Recruiting | 2 Months | N/A | All | 42 | Phase 1;Phase 2 | United States |
| 2 | NCT04558736 (ClinicalTrials.gov) | November 2020 | 9/9/2020 | Haploidentical HCT for Severe Aplastic Anemia | Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia | Aplastic Anemia;Bone Marrow Failure Syndrome;Dyskeratosis Congenita | Drug: Anti-Thymocyte Globulin (Rabbit);Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Mesna;Drug: G-CSF;Radiation: Total Lymphoid Irradiation (TLI);Device: CliniMACS;Biological: HPC, A Infusion;Biological: CD45RA-depleted DLI | St. Jude Children's Research Hospital | NULL | Recruiting | N/A | 21 Years | All | 21 | Phase 2 | United States |
| 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) | 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-jRCTs031180398 | 30/12/2017 | 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 | Kato Motohiro | NULL | Recruiting | Not applicable | < 25age old | Both | 22 | Phase 2 | None (Japan only);Japan |
| 5 | 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 |
| 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 |
| 6 | NCT01220531 (ClinicalTrials.gov) | September 2010 | 22/9/2010 | 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 | Available | N/A | N/A | All | United States | ||
| 7 | NCT00579527 (ClinicalTrials.gov) | December 19, 2005 | 20/12/2007 | 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 | Biological: Cultured Thymus Tissue for Implantation (CTTI);Other: Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Daclizumab;Drug: Mycophenolate mofetil | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbH;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | N/A | N/A | All | 14 | Phase 1;Phase 2 | United States |
| 8 | 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 |
| 9 | 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 |
| 10 | 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 |
| 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 |
| 11 | 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 | |
| 12 | NCT00006054 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | 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 | NULL | Terminated | N/A | 35 Years | Both | N/A | United States |