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OTL-101 Gene Therapy for ADA-SCID
NCT01380990Phase 2Completed
- Sponsor
- Great Ormond Street Hospital
- Start
- Nov 2012
- Primary completion
- Dec 2019
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of simoladagene autotemcel in severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) (2 arms), sponsored by Great Ormond Street Hospital. Primary endpoints: Overall Survival at 12 months, Event-free Survival at 12 months and 8 more. Started Nov 2012; status: completed.
02
Drugs
simoladagene autotemcel
03
Conditions
severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)
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