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Taking people in United StatesPhase 3

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Key facts

Who runs it
Dyne Therapeutics
Where
Atlanta, Baltimore, Columbia, Gainesville, Hillsborough, Houston, Indianapolis, La Jolla, Los Angeles, Palo Alto, Richmond, Rochester, Salt Lake City, and San Antonio, United StatesSee the 14 sitesAlso in 11 other countries, 31 sites
Status
Recruiting
Phase
Phase 3
People taking part
150 people, planned
Started
14 May 2026
Expected to end
Jan 2029
Type of study
Interventional, 3 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • zeleciment basivarsen (DYNE-101)
  • Placebo
Record updated
13 November 2026

Who can take part

Age
16 years and older
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 6 criteriaShow less

Who can take part

  • Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (>) 100. Historical results from clinical testing are acceptable
  • Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the ankle are allowed)
  • Body mass index (BMI) less than (<) 35 kilograms per meter square (kg/m\^2)

Who cannot take part

  • History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator
  • Use of glucagon-like peptide 1 (GLP-1) agonist/incretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments; Note: Other inclusion and exclusion criteria may apply
  • A known diagnosis of congenital DM1

Where in United States

Also in 11 other countries, 31 sites

  • Rare Disease Research, LLCAtlanta, United StatesContact: Julia ZhuTaking people
  • Kennedy Krieger InstituteBaltimore, United StatesContact: Demaya StarkesTaking people
  • Roy Blunt NextGen Precision Health InstituteColumbia, United StatesContact: Heather McHattonTaking people
  • UF Health: University of Florida Clinical Research CenterGainesville, United StatesContact: Debbye LandersTaking people
  • Rare Disease Research, LLCHillsborough, United StatesContact: Hannah NationTaking people
  • Houston Methodist Neurological InstituteHouston, United StatesContact: Estefania PorrasTaking people
  • IU Health Neuroscience CenterIndianapolis, United StatesContact: Jennifer TerrellTaking people
  • UCSD Altman Clinical and Translational Research InstituteLa Jolla, United StatesContact: Christian FarfanTaking people
  • UCLA Medical Center (Department of Neurology)Los Angeles, United StatesContact: Dennis FernandoTaking people
  • Stanford Neuroscience Health CenterPalo Alto, United StatesContact: Anita ChackoTaking people
  • Virginia Commonwealth University - Biotech OneRichmond, United StatesContact: Levi HeadrickTaking people
  • University of Rochester Medical CenterRochester, United StatesContact: Emily VerdaasdonkTaking people
  • University of Utah Clinical Neurosciences CenterSalt Lake City, United StatesContact: Michael PapadakisTaking people
  • The University of Texas Health Science Center at San AntonioSan Antonio, United StatesContact: Tonya D RandolphTaking people

About the trial

What the trial wants to find out

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).

The 3 groups in the trial

  • Placebo-Controlled Period: DYNE-101Treatment being tested

    Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks.

    Treatment: zeleciment basivarsen (DYNE-101)

  • Placebo-Controlled Period: PlaceboPlacebo, for comparison

    Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.

    Treatment: Placebo

  • Long-Term Extension Period: DYNE-101Treatment being tested

    Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks. Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.

    Treatment: zeleciment basivarsen (DYNE-101) and Placebo

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
Record updated
13 November 2026