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Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated With Strimvelis (or GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and Effectiveness

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Trial Profile

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated With Strimvelis (or GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and Effectiveness

Status: Recruiting
Phase of Trial: Phase IV

Latest Information Update: 01 Jun 2026

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At a glance

  • Drugs GSK 2696273 (Primary)
  • Indications Adenosine deaminase deficiency
  • Focus Adverse reactions; Pharmacodynamics; Pharmacogenomic; Therapeutic Use
  • Sponsors Fondazione Telethon; GlaxoSmithKline; GSK; Orchard Therapeutics

Most Recent Events

  • 18 May 2026 Primary endpoints were streamlined from multiple efficacy, survival, immune reconstitution, growth, and quality-of-life measures to a focused long-term safety assessment. The updated endpoints primarily evaluate AESIs, AEs/SAEs, laboratory abnormalities, fertility/pregnancy outcomes, and RIS monitoring, while several efficacy-related endpoints were removed.
  • 18 May 2026 Planned End Date changed from 31 May 2037 to 31 Dec 2045.
  • 18 May 2026 Planned primary completion date changed from 31 May 2037 to 31 Dec 2045.

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