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

2 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	NCT02999984 (ClinicalTrials.gov)	December 16, 2016	19/12/2016	Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID	Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transd ...	Severe Combined Immunodeficiency Due to ADA Deficiency	Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: ...	Orchard Therapeutics	California Institute for Regenerative Medicine (CIRM);University of California, Los Angeles	Completed	N/A	17 Years	All	10	Phase 1/Phase 2	United States
2	NCT01852071 (ClinicalTrials.gov)	August 2, 2013	7/5/2013	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for ...	Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Hum ...	ADA-SCID	Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG -ADA ERT	Orchard Therapeutics	National Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institu ...	Completed	1 Month	17 Years	All	20	Phase 1/Phase 2	United States

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