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Long-term post-authorisation safety and efficacy study of Waskyra gene therapy in Wiskott-Aldrich syndrome
2024-518369-92-00Phase 4Authorised
- Sponsor
- Fondazione Telethon
- Start
- Jun 2025
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 2
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Overview
Phase 4, open-label trial of etuvetidigene autotemcel in Wiskott-Aldrich syndrome (WAS) (2 cohorts), sponsored by Fondazione Telethon. Primary endpoints: Proportion of patients with engraftment failure, Proportion of patients with insertional mutagenesis and oncogenesis and 5 more. Planned start Jun 2025; status: authorised.
02
Drugs
etuvetidigene autotemcel
03
Conditions
Wiskott-Aldrich syndrome (WAS)
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