NCT01852071View on ClinicalTrials.gov

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

46

Participants

Timeline

Start Date

August 2, 2013

Primary Completion Date

August 27, 2018

Study Completion Date

August 27, 2018

Conditions
ADA-SCID
Interventions
GENETIC

Infusion of autologous EFS-ADA LV CD34+ (OTL-101)

autologous EFS-ADA LV CD34+ cells (OTL-101) are infused intravenously

DRUG

busulfan

Busulfan is used for non-myeloablative conditioning

DRUG

PEG-ADA ERT

PEG-ADA ERT is discontinued at Day +30 (-3/+15 days) after successful engraftment

Trial Locations (2)

20892

Mark O. Hatfield Clinical Research Center, NIH, Bethesda

90095

Mattel Children's Hospital, UCLA, Los Angeles

Sponsors

Collaborators (1)

Orchard Therapeutics
All Listed Sponsors
collaborator

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

collaborator

National Human Genome Research Institute (NHGRI)

NIH

collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

collaborator

Orchard Therapeutics

INDUSTRY

lead

University of California, Los Angeles

OTHER

NCT01852071 - Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene | Biotech Hunter | Biotech Hunter