Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (NCT05481879)
A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
🩺 Plain-English Summary (8th-Grade Level)
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.
Who can join: Review the eligibility criteria below.
Location: Stanford University — Stanford, California
Age range: 18 Years to 65 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 (20 Sites)
- Stanford University — Stanford, California
- University of Florida College of Medicine — Gainesville, Florida
- Indiana University School of Medicine — Indianapolis, Indiana
- University of Iowa — Iowa City, Iowa
- Washington University in St. Louis — St Louis, Missouri
- University of Rochester Medical Center — Rochester, New York
- Neurology Rare Disease Center — Denton, Texas
- Virginia Commonwealth University (VCU) — Richmond, Virginia
- St. Vincent's Hospital — Fitzroy, Victoria
- CHU de Nantes — Nantes,
- Institut de Myologie — Paris,
- Charité - Universitätsmedizin Berlin — Berlin,
- Ludwig Maximilians University, Munich - Friedrich Baur Institut — Munich,
- Centro Clinico Nemo — Milan,
- Fondazione Policlinico Universitario A Gemelli-Rome — Rome,
- Radboud Medical Center — Nijmegen,
- NZCR Auckland — Auckland,
- University College London Hospitals — London,
- John Walton Muscular Dystrophy Research Centre — Newcastle upon Tyne,
- Salford Royal Hospital — Salford,
📞 Contact the Study Coordinator
Phone: (781) 317-1919
Email: clinicaltrials@dyne-tx.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 Type 1 (DM1)
- A Remote Physical Activity Program in the Population Suffering from Type 1 Myotonic Dystrophy — Jonquière, Quebec
- A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) — San Carlos, California
- Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 — La Jolla, California
- Remote Assessments and Genetic Determinants of Myotonic Dystrophy — Rochester, New York
Explore by condition: All Myotonic Dystrophy Type 1 (DM1) 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.