NCT02999984View on ClinicalTrials.gov

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

December 16, 2016

Primary Completion Date

October 11, 2018

Study Completion Date

September 26, 2019

Conditions
Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions
GENETIC

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

autologous cryopreserved 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 (1)

90095

University of California, Los Angeles, Los Angeles

Sponsors

Collaborators (1)

Orchard Therapeutics
All Listed Sponsors
collaborator

California Institute for Regenerative Medicine (CIRM)

OTHER

collaborator

Orchard Therapeutics

INDUSTRY

lead

University of California, Los Angeles

OTHER

NCT02999984 - Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Biotech Hunter | Biotech Hunter