A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
← catalyst calendarNCT06844214 · readout in 924 d
Sponsored by Sanofi (industry) · SNY — their whole pipeline →.
Phase
Phase 1/2
Status
Recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
32estimated
Sites
9
Countries
Argentina, Australia, Canada +3
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2025-07-23 | actual | When the study began enrolling |
| Primary completion | 2029-02-28 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2032-04-27 | estimated | The whole study's end, after follow-up |
| First posted | 2025-02-25 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-06-01 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Myotonic Dystrophy
Intervention
- Biological: SAR446268
Primary outcomes
what the primary-completion date above is the date OFPart A and Part B: Incidence of treatment-emergent adverse events (TEAEs) following SAR446268 administration
measured Baseline to Week 52
Part B: Proportion of participants with at least 40% DMPK mRNA knockdown in muscle biopsy at Weeks 12 and 52 following SAR446268 administration
measured Weeks 12 and 52
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