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Gene therapy for Wiskott-Aldrich syndrome
ISRCTN30718752Phase 2Completed
- Sponsor
- Généthon
- Start
- May 2011
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
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Overview
Phase 2, open-label, single-arm trial of etuvetidigene autotemcel in Wiskott-Aldrich syndrome (WAS), sponsored by Généthon. Primary endpoints: Safety of conditioning regimen, Safety of the transduction procedure and 3 more. Started May 2011; status: completed.
02
Drugs
etuvetidigene autotemcel
03
Conditions
Wiskott-Aldrich syndrome (WAS)
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