NCT01380990 - Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

November 15, 2012

Primary Completion Date

December 23, 2019

Study Completion Date

December 23, 2019

Conditions

Adenosine Deaminase DeficiencySevere Combined Immunodeficiencies (SCID)

Interventions

GENETIC

Infusion of autologous EFS-ADA LV CD34+ cells

Autologous EFS-ADA LV CD34+ cells (OTL-101\*) are infused intravenously

OTHER

Haematopoietic Stem Cell Transplantation (HSCT)

Historical data from a database of ADA-SCID patients treated with allogeneic HSCT from GOSH will be collected as comparator group.

DRUG

Busulfan

Busulfan is used for non-myeloablative conditioning

DRUG

Peg-Ada

Peg-Ada enzyme replacement therapy is discontinued at Day +3- (-3/+15 days) after successful engraftment

Trial Locations (1)

Great Ormond Street Hospital for Children NHS Foundation Trust, London