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Gene Therapy for Adenosine Deaminase Deficiency
NCT01279720Phase 2Completed
- Sponsor
- Great Ormond Street Hospital
- Start
- Oct 2003
- Primary completion
- Nov 2013
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of Strimvelis in adenosine deaminase deficiency, sponsored by Great Ormond Street Hospital. Primary endpoint: Immunological reconstitution at 5 years. Started Oct 2003; status: completed.
02
Drugs
Strimvelis
03
Conditions
adenosine deaminase deficiency
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