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

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	NCT03765632 (ClinicalTrials.gov)	January 3, 2018	8/8/2018	Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID	Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1a Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency	Severe Combined Immunodeficiency Due to ADA Deficiency	Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-Ada	Great Ormond Street Hospital for Children NHS Foundation Trust	Orchard Therapeutics	Active, not recruiting	N/A	17 Years	All	13	Phase 1/Phase 2	United Kingdom
2	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 Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency	Severe Combined Immunodeficiency Due to ADA Deficiency	Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG -ADA ERT	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