At a glance
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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
In Brief
A Phase 2 clinical trial evaluating Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), and 2 other interventions for Adenosine Deaminase Deficiency and Severe Combined Immunodeficiencies (SCID). Completed, enrolled 36 participants across 1 site.
Detailed 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.
Study Details
Timeline
Interventions
Autologous EFS-ADA LV CD34+ cells (OTL-101\*) are infused intravenously
Historical data from a database of ADA-SCID patients treated with allogeneic HSCT from GOSH will be collected as comparator group.
Busulfan is used for non-myeloablative conditioning
Peg-Ada enzyme replacement therapy is discontinued at Day +3- (-3/+15 days) after successful engraftment