NCT01380990

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust · Academic / Other
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

Summary

This is a historically controlled, non-randomized Phase I/II clinical trial to assess the safety and efficacy of autologous transplantation of CD34+ hematopoietic stem/progenitor cells (HSPCs), obtained from infants affected by ADA-SCID, following transduction of the HSPCs with a lentiviral vector (LV) carrying the human ADA complementary DNA (cDNA) under the control of the elongation factor 1 alpha shortened (EFS) promoter. Subjects treated in the trial receive the infusion of autologous, transduced cells following marrow cytoreduction with busulfan. The outcomes are compared to those observed in a historical control group of patients who received an allogeneic hematopoietic stem cell transplant (HSCT). This Phase I/II clinical trial will be performed at Great Ormond Street Hospital (GOSH), London, United Kingdom.

Conditions

• Adenosine Deaminase Deficiency
• Severe Combined Immunodeficiencies (SCID)

Interventions

Type	Name	Description
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

Timeline

• Start date: 2012-11-15
• Primary completion: 2019-12-23
• Completion: 2019-12-23
• First posted: 2011-06-27
• Last updated: 2021-09-16
• Results posted: 2021-09-16

Locations

1 site across 1 country: United Kingdom