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SAR446268 in non-congenital myotonic dystrophy type 1
NCT06844214Phase 2Recruiting
- Sponsor
- Sanofi
- Start
- Jul 2025
- Primary completion
- Feb 2029
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 6
01
Overview
Phase 2, open-label, single-arm trial of SAR446268 in myotonic dystrophy type 1 (DM1), sponsored by Sanofi. Primary endpoints: Incidence of treatment-emergent adverse events at 52 weeks and Proportion of participants with at least 40% DMPK mRNA knockdown in muscle biopsy at 52 weeks. Started Jul 2025; status: recruiting.
02
Drugs
SAR446268
03
Conditions
myotonic dystrophy type 1 (DM1)
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