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

NCT03478670 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2026-05-22

No results posted yet for this study

Summary

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).

Conditions

  • Immunologic Deficiency Syndromes

Interventions

GENETIC

Strimvelis

Strimvelis is a CD34+ cell enriched dispersion of human autologous bone marrow derived hematopoietic stem/progenitor cells transduced with a retroviral vector containing the human ADA gene. It will be administered as an intravenous infusion once only. 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, treated under hospital exemption (HE) frame, according to the Italian Decree of the Ministry of Health, January 16th 2015, "Provisions on advanced therapy drugs prepared on a non-repetitive basis".

Sponsors & Collaborators

  • Fondazione Telethon

    lead OTHER

Principal Investigators

  • Fondazione Telethon · Fondazione Telethon

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-05-05
Primary Completion
2045-12-31
Completion
2045-12-31

Countries

  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03478670 on ClinicalTrials.gov