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
Recruiting · Phase 1, Phase 2 · NCT06844214
Conditions: Myotonic Dystrophy
At a glance
- Research payments reported, 2025
- $132,215
- U.S. investigators named
- 3
- Sites paid
- 4
- Planned or actual enrollment
- 32
About the trial
- Sponsor
- Sanofi
- Tested
- SAR446268 (biological)
- Dates
- Started 2025-07-23; completion 2032-04-27
- Results posted
- Not yet
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 c…
From ClinicalTrials.gov, last updated 2026-09-21. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Sanofi US Services Inc. reported $132,215 in research payments for this study in 2025 (52 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Sankarasubramoney H. Subramony, MD Neurology · Gainesville, FL
- Tuan H. Vu, MD Neurology · Tampa, FL
- Christina M. Ulane, MD Neurology · New York, NY
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of South Florida $57,500
- The Trustees of Columbia University in The City of New York $33,948
- University of Florida Contracts Grants $30,545
- $8,699
- Strong Memorial Hospital, NY $1,524
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.