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NCT06844214 · ClinicalTrials.gov registry record · Phase 1
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1
A Phase 1 study of Myotonic Dystrophy, sponsored by Sanofi.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 32
- Enrollment target
- 8
- Study locations
NCT06844214: Recruiting Phase 1 study of Myotonic Dystrophy, sponsored by Sanofi.
NCT06844214 is a Phase 1 study of Myotonic Dystrophy that is actively recruiting participants, run by Sanofi. The registered enrollment target is 32 participants, below the 852-participant average among 9 other Myotonic Dystrophy trials with a reported enrollment target (96% lower). The trial reports 8 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06844214, a Phase 1 study of Myotonic Dystrophy, is actively recruiting participants, sponsored by Sanofi.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 32 participants
- Enrollment target
- 8
- Study locations
Study Summary
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 50 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) administration of SAR446268. The study consists of a dose escalation part (Part A) during which single ascending doses of SAR446268 will be evaluated in 3 distinct cohorts and an optional 4th dose cohort. Once a safe and effective dose is identified, additional participants will be treated in Part B, the dose expansion phase of the study. The study duration will be 110 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes a 6-week screening phase and a 104-week follow-up period post-SAR446268 administration.
Primary Outcome
Number of TEAEs post-SAR446268 administration
Conditions Studied
Interventions
- BIOLOGICAL SAR446268
Study Locations (8)
Other
- Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001 - Buenos Aires
- Investigational Site Number : 3760002 - Ramat Gan
- Investigational Site Number : 8260002 - Newcastle upon Tyne
Florida
- University of Florida, 2004 Mowry Road - Site Number: 8400005 - Gainesville
- University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 - Tampa
New York
- Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 - New York
Queensland
- Investigational Site Number : 0360001 - Brisbane
Quebec
- The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001 - Montreal
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2025-07-23 |
| Est. Completion | 2029-02-28 |
| Phase | Phase 1 |
What NCT06844214 shows while recruiting
NCT06844214 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 852-participant average among 9 other Myotonic Dystrophy trials with a reported enrollment target (96% lower).
The record links to 1 condition, with Myotonic Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which SAR446268 is the first listed.
NCT06844214 lists 8 locations in 5 states (Other, Florida, New York).
Frequently Asked Questions
What is clinical trial NCT06844214 about?
NCT06844214 is a clinical study titled "A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1". This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 50 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR4462...
What is the current status of trial NCT06844214?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 32 participants. The study started on 2025-07-23. Estimated completion is 2029-02-28.
What conditions does trial NCT06844214 study?
This clinical trial studies the following conditions: Myotonic Dystrophy.
What interventions are being tested in trial NCT06844214?
The interventions under investigation include: SAR446268 (BIOLOGICAL).
Who is sponsoring clinical trial NCT06844214?
This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06844214 being conducted?
This trial has 8 study locations across Florida, New York, Queensland, Quebec. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Where NCT06844214's enrollment target sits among peer trials
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