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 (NCT06844214)
A Phase 1/Phase 2 Open-label Single Arm Study With Dose Escalation (Part A), and Dose Expansion (Part B) Parts to Evaluate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants 10 to 55 Years Old With Non-congenital Myotonic Dystrophy Type 1
🩺 Plain-English Summary (8th-Grade Level)
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 55 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 fourth 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 112 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes an optional pre-screening period, approximately 8-week screening phase and a 104-week follow-up period post-SAR446268 administration.
Who can join: Review the eligibility criteria below.
Location: University of Florida, 2004 Mowry Road - Site Number: 8400005 — Gainesville, Florida
Age range: 10 Years to 55 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (9 Sites)
- University of Florida, 2004 Mowry Road - Site Number: 8400005 — Gainesville, Florida
- University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 — Tampa, Florida
- Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 — New York, New York
- Virginia Commonwealth University Medical Center- Site Number : 8400006 — Richmond, Virginia
- Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001 — Buenos Aires,
- Investigational Site Number : 0360001 — Brisbane, Queensland
- The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001 — Montreal, Quebec
- Investigational Site Number : 3760002 — Ramat Gan,
- Investigational Site Number : 8260002 — Newcastle upon Tyne,
📞 Contact the Study Coordinator
Phone: (800) 633-1610
Email: contact-us@sanofi.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Myotonic Dystrophy
- Extracellular RNA Biomarkers of Myotonic Dystrophy — Boston, Massachusetts
- Biomarker Development for Muscular Dystrophies — Boston, Massachusetts
- Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry — Rochester, New York
- Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy — Winston-Salem, North Carolina
- Myotonic Dystrophy Family Registry — Oakland, California
Explore by condition: All Myotonic Dystrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.