CLINICAL TRIALS PROFILE FOR PEGADEMASE BOVINE

All Clinical Trials for pegademase bovine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Human Genome Research Institute (NHGRI)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	University of California, Los Angeles	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	Orchard Therapeutics	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.

Clinical Trial Conditions for pegademase bovine

Condition Name

Condition Name for pegademase bovine
Intervention	Trials
ADA-SCID	2
Severe Combined Immunodeficiency Due to ADA Deficiency	2

Condition MeSH

Condition MeSH for pegademase bovine
Intervention	Trials
Severe Combined Immunodeficiency	2
Immunologic Deficiency Syndromes	2

Clinical Trial Locations for pegademase bovine

Trials by Country

Trials by Country for pegademase bovine
Location	Trials
United States	3
United Kingdom	1

Trials by US State

Trials by US State for pegademase bovine
Location	Trials
California	2
Maryland	1

Clinical Trial Progress for pegademase bovine

Clinical Trial Phase

Clinical Trial Phase for pegademase bovine
Clinical Trial Phase	Trials
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for pegademase bovine
Clinical Trial Phase	Trials
Completed	2
Active, not recruiting	1

Clinical Trial Sponsors for pegademase bovine

Sponsor Name

Sponsor Name for pegademase bovine
Sponsor	Trials
Orchard Therapeutics	3
University of California, Los Angeles	2
National Heart, Lung, and Blood Institute (NHLBI)	1

Sponsor Type

Sponsor Type for pegademase bovine
Sponsor	Trials
Other	4
NIH	3
Industry	3