Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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
NCT06844214 · View on ClinicalTrials.gov ↗
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.
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 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06844214
The ClinicalTrials.gov registry entry for NCT06844214 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sanofi, which has 275 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06844214 reports 8 study locations spanning 5 distinct geographic areas — top geographies include Other, Florida, New York. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06844214?
The interventions under investigation include: SAR446268 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06844214?
This trial is sponsored by Sanofi, which has 275 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.