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: To join this study, participants generally need to meet the listed inclusion criteria (9 items) and avoid the listed exclusions (25 items).
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 ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:
- Participants with neutralizing antibodies against the AAV.SAN011 capsid
- Participants with left ventricular ejection fraction \<50%
- Participants with liver or biliary disease defined as having at least one of the following:
- ALT \>3 x ULN and AST \>3 x ULN
- Alkaline phosphatase \>3 x ULN
- Total bilirubin \>1.5 x ULN (unless has a genetically confirmed diagnosis of Gilbert's syndrome)
- Direct bilirubin ≥1.5 x ULN
- Participants with International normalized ratio \>1.5
- Participants with renal disease defined as:
- Serum creatinine \>1.5 x ULN and/or estimated glomerular filtration rate \<60 mL/min/1.73 m2 as determined by Chronic Kidney Disease Epidemiology Collaboration (2021) for those age ≥18 years and Bedside Schwartz Equation for those \<18 years
- Participants with chronic respiratory insufficiency and on long term/hull-time ventilatory assistance requiring at least 6 hours per day for at least 21 consecutive days.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 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.