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GAIN-CTNNB1 gene therapy trial in pediatric CTNNB1 syndrome
2025-522719-40-00Phase 2Authorised
- Sponsor
- CTNNB1 Foundation
- Start
- Nov 2025
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label trial of fosfomycin in CTNNB1 syndrome (CTNNB1) (2 arms), sponsored by CTNNB1 Foundation. Primary endpoints: Adverse events and serious adverse events, Clinical laboratory and CSF abnormalities and 4 more. Planned start Nov 2025; status: authorised.
02
Drugs
fosfomycin
03
Conditions
CTNNB1 syndrome (CTNNB1)
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Study design, results and competitive context are available in the Gosset platform where reported.
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