Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Adenosine deaminase (ADA) enzyme deficiency results in severe combined immunodeficiency (SCID), a fatal autosomal recessive inherited immune disorder. Strimvelis (or GSK2696273) is a gene therapy intended for patients with ADA-SCID and for whom no suitable human leukocyte antigen (HLA) matched related stem cell donor is available. This therapy aims to restore ADA function in hematopoietic cell lineages, and in doing so prevents the pathology caused by purine metabolites (i.e., impaired immune function). This registry evaluates the long term safety and effectiveness outcomes of subjects who have received Strimvelis and is conducted as a post approval safety study associated with EMA marketing authorisation of Strimvelis™. In this study will be also included patients for whom the gene therapy medicinal product has been prepared starting from mobilized peripheral blood (mPB)-derived CD34+ cells (mPB-GT).

NCT Number	NCT03478670
Lead Sponsor	Fondazione Telethon
Conditions	Immunologic Deficiency Syndromes
Enrollment	50 participants
Start Date	2017-05-05
Primary Completion	2045-12-31 (estimated)
Study Completion	2045-12-31 (estimated)
Updated on ClinicalTrials.gov	2026-05-22