NCT03765632View on ClinicalTrials.gov

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

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

13

Participants

Timeline

Start Date

January 3, 2018

Primary Completion Date

September 13, 2021

Study Completion Date

September 28, 2022

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

Peg-Ada Enzyme Replacement Therapy (ERT) is discontinued at Day +30 (-3/+15 days) after successful engraftment

Trial Locations (1)

WC1N 3JH

Great Ormond Street Hospital for Children NHS Foundation Trust, London

Sponsors

Collaborators (1)

Orchard Therapeutics
All Listed Sponsors
collaborator

Orchard Therapeutics

INDUSTRY

lead

Great Ormond Street Hospital for Children NHS Foundation Trust

OTHER

NCT03765632 - Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Biotech Hunter | Biotech Hunter