PEG-ADA ERT ( DrugBank: - )

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1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
65	原発性免疫不全症候群	2

65. 原発性免疫不全症候群

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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

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 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
2	NCT01852071 (ClinicalTrials.gov)	August 2, 2013	7/5/2013	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA Gene	Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)	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 Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los Angeles	Completed	1 Month	17 Years	All	20	Phase 1/Phase 2	United States