A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (NCT06185764)
A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
🩺 Plain-English Summary (8th-Grade Level)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
Who can join: Review the eligibility criteria below.
Location: Stanford Neuromuscular Research — San Carlos, California
Age range: 18 Years to 64 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 (26 Sites)
Showing the first 25 of 26 sites.
- Stanford Neuromuscular Research — San Carlos, California
- University of Florida Clinical Research Center — Gainesville, Florida
- University of Kansas Medical Center — Fairway, Kansas
- Boston Children's Hospital — Boston, Massachusetts
- Washington University School of Medicine / St. Louis Children's Hospital — St Louis, Missouri
- Wake Forest Baptist Health — Winston-Salem, North Carolina
- University of Pennsylvania — Philadelphia, Pennsylvania
- Virginia Commonwealth University (Sanger Hall) — Richmond, Virginia
- Wesley Research Institute — Auchenflower,
- Neuroscience Clinical Trials Unit, Alfred Brain — Melbourne,
- Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg — Leuven,
- Hopital de Chicoutimi — Chicoutimi,
- Altasciences Montreal — Montreal,
- McGIll University — Montreal,
- University of Ottawa — Ottawa,
- CHU Research Centre of Quebec — Québec,
- Neuromuscular Reference Center Institute of Myology — Paris,
- Ludwig Maximilians Universitaet Muenchen — München,
- Centro Clinico NeMO — Milan,
- Maastricht University Medical Center — Maastricht,
- Hospital Universitario y Politécnico La Fe — Valencia,
- Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow,
- Leonard Wolfson Experimental Neurology Centre CRF — London,
- St. George's University Hospital — London,
- Salford Royal Hospital — Salford,
💰 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
- 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
- Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 — Stanford, California
Explore by condition: All Myotonic Dystrophy Type 1 (DM1) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.