This study evaluates a gene therapy (Strimvelis) in which the ADA gene is inserted into the patient's own blood stem cells, for Severe Combined Immunodeficiency caused by ADA enzyme deficiency (ADA-SCID). It included pediatric patients without an HLA-matched sibling donor for whom enzyme replacement was insufficient. It was a Phase 2 study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * ADA-SCID with no HLA-identical sibling donor available * pediatric age and at least one of the following criteria: * inadequate immune response after PEG-ADA for \> 6 months * patients who discontinued PEG-ADA due to intolerance, allergy or auto-immunity * patients for whom enzyme replacement therapy is not a life long therapeutic option Exclusion Criteria: * HIV infection * history or current malignancy * Patients who received a previous gene therapy treatment in the 12 months prior to receiving Strimvelis * any other conditions dangerous for the patients according to the investigator