65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details

臨床試験数 : 500 / 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217

39 trials found

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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	NCT05463133 (ClinicalTrials.gov)	July 8, 2022	15/7/2022	Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an A ...	Phase I/II Study Using Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous ...	Chronic Granulomatous Disease	Drug: Sirolimus;Drug: Cyclophosphamide;Drug: Alemtuzumab;Drug: Busulfan;Biological: Pheripheral bloo ...	National Institute of Allergy and Infectious Diseases (NIAID)	NULL	Recruiting	4 Years	65 Years	All	50	Phase 1/Phase 2	United States
2	NCT03597594 (ClinicalTrials.gov)	September 8, 2021	19/6/2018	Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infus ...	Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infus ...	Severe Combined Immunodeficiency	Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: Clini ...	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 Patients With Inborn Errors of Immunity	A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of ...	Lymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable ...	Drug: Busulfan test dose;Drug: Fludarabine;Drug: Busulfan;Drug: Alemtuzumab;Radiation: Total body Irr ...	National Cancer Institute (NCI)	NULL	Recruiting	4 Years	69 Years	All	66	Phase 2	United States
4	NCT03910452 (ClinicalTrials.gov)	October 28, 2019	9/4/2019	Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Bus ...	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 Irradi ...	National Institute of Allergy and Infectious Diseases (NIAID)	NULL	Recruiting	4 Years	65 Years	All	30	Early Phase 1	United States
5	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 ( ...	A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved ...	Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodef ...	Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematolo ...	Orchard Therapeutics Ltd.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	6	Phase 3	Italy
6	NCT03601286 (ClinicalTrials.gov)	December 21, 2018	22/2/2018	Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency	Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan	Severe Combined Immunodeficiency, X-Linked	Drug: Lentiviral vector transduced CD34+ cells	Great Ormond Street Hospital for Children NHS Foundation Trust	NULL	Recruiting	8 Weeks	5 Years	Male	5	Phase 1	United Kingdom
7	NCT03619551 (ClinicalTrials.gov)	October 22, 2018	16/7/2018	Conditioning SCID Infants Diagnosed Early	A Randomized Trial of Low Versus Moderate Exposure Busulfanfor Infants With Severe Combined Immunode ...	SCID	Drug: Busulfan;Device: Cell processing for TCRaß+/CD19+ depletion	Michael Pulsipher, MD	NULL	Recruiting	0 Years	2 Years	All	64	Phase 2	United States;Canada
8	EUCTR2018-000673-68-GB (EUCTR)	09/10/2018	27/07/2018	A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with ...	Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan- Lentiviral ...	Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders cha ...	Product Name: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells INN or Proposed I ...	Great Ormond Street Hospital for Children NHS Trust	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	5	Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - ( ...	United Kingdom
9	NCT03513328 (ClinicalTrials.gov)	June 15, 2018	19/4/2018	Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation	PEDS024, Phase I/II Feasibility Study of BusulfanFludarabine and Thiotepa Conditioning Regimen for A ...	Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutro ...	Drug: Thiotepa --single daily dose;Drug: Thiotepa --escalated dose	University of Florida	Live Like Bella Pediatric Cancer Research	Active, not recruiting	3 Months	39 Years	All	6	Phase 1/Phase 2	United States
10	NCT03538899 (ClinicalTrials.gov)	May 31, 2018	3/5/2018	Autologous Gene Therapy for Artemis-Deficient SCID	A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficien ...	Severe Combined Immunodeficiency	Drug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: Busulfan	University of California, San Francisco	NULL	Recruiting	2 Months	N/A	All	25	Phase 1/Phase 2	United States

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