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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
6 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03597594
(ClinicalTrials.gov)
December 202019/6/2018Haplocompatible 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 ImmunodeficiencyDrug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte InfusionSt. Jude Children's Research HospitalNULLRecruiting2 MonthsN/AAll42Phase 1/Phase 2United States
2NCT03513328
(ClinicalTrials.gov)
June 15, 201819/4/2018Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone 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;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchRecruiting3 Months39 YearsAll40Phase 1/Phase 2United States
3NCT01962415
(ClinicalTrials.gov)
February 4, 201410/10/2013Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTA 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 TransplantationPrimary Immunodeficiency (PID);Congenital Bone Marrow Failure Syndromes;Inherited Metabolic Disorders (IMD);Hereditary Anemias;Inflammatory ConditionsDrug: Hydroxyurea;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Melphalan;Drug: ThiotepaPaul SzabolcsNULLRecruiting2 Months55 YearsAll100Phase 2United States
4NCT01821781
(ClinicalTrials.gov)
March 201319/3/2013Immune Disorder HSCT ProtocolA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory RegimeImmune 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 SyndromeDrug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanWashington University School of MedicineNULLRecruitingN/A21 YearsAll20Phase 2United States
5NCT00160355
(ClinicalTrials.gov)
May 20058/9/2005Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich SyndromeHaploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot StudyWiskott-Aldrich SyndromeProcedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, ThiotepaSt. Jude Children's Research HospitalNULLCompletedN/A18 YearsMale4Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT00295971
(ClinicalTrials.gov)
April 200523/2/2006Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency DiseaseStem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency DiseasesCongenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: 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 irradiationUniversity of California, San FranciscoNational Cancer Institute (NCI)Completed1 Year17 YearsBoth21Phase 1United States