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

StatusEnrolling by Invitation
PhaseNot specified
Started
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Record History

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  1. Primary endpoint(s) modified
    • Secondary endpoints revised (11)
    • Study description shortened, about 6 words
    v11
  2. Primary and study completion dates: May 31, 2037 to Dec 31, 2045v10 Show 5 more changesShow less
    • Start date: Mar 28, 2017 to May 5, 2017
    • Primary endpoints: 23 to 5
    • Eligibility criteria expanded, about 6 words
    • Secondary endpoints expanded, about 77 words
    • Study description shortened, about 17 words
  3. Start date: Mar 31, 2017 to Mar 28, 2017
    • Eligibility criteria expanded, about 26 words
    • Study description expanded, about 112 words
    • Study site revised
    v9
  4. Lead sponsor: Orchard Therapeutics to Fondazione Telethon
    • Study site revised
    v8
  5. 3 versions Re-verified since May 18, 2020, no change to tracked fieldsv5-v7
Show 3 earlier versionsHide 3 earlier versions
  1. Trial status: Recruiting to Enrolling by Invitation
    • Site details revised
    v4
  2. Re-verified, no change to tracked fieldsv3
  3. Lead sponsor: GlaxoSmithKline to Orchard Therapeutics
    • Interventions: Strimvelis (or GSK2696273) (Genetic) to Strimvelis (Genetic)
    • Study site revised
    • Study title shortened, about 2 words
    v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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).
Trial Details
NCT Number NCT03478670
Lead Sponsor Fondazione Telethon
Conditions
(from sponsor)		 Immunologic Deficiency Syndromes
Related terms Immunologic Deficiency Syndromes, Severe combined immunodeficiency due to adenosine deaminase deficiency
Enrollment 50 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov