Recruiting
Phase 3

Zeleciment Basivarsen

Sponsor:

Dyne Therapeutics

Code:

NCT07486934

Conditions

Myotonic Dystrophy Type 1 (DM1)

DM1

Myotonic Dystrophy

Steinert Disease

Steinert

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Interventions

zeleciment basivarsen (DYNE-101)

Placebo

Study Details

Brief summary:

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).

Conditions

Myotonic Dystrophy Type 1 (DM1)

DM1

Myotonic Dystrophy

Steinert Disease

Steinert

Study ID

NCT07486934

Start date

May 14, 2026

Status verified date

Apr, 2026

Completion date

Jan, 2029

Anticipated

Primary completion date

Jul, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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).

Exclusion Criteria:

  • A known diagnosis of congenital DM1.
  • 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.

Study Design

Enrollment

150 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Placebo-Controlled Period: DYNE-101

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

placebo comparator: Placebo-Controlled Period: Placebo

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

experimental: Long-Term Extension Period: DYNE-101

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.

Interventions

zeleciment basivarsen (DYNE-101)

Administered by IV infusion

Placebo

Administered by IV infusion

Primary outcome measure

  • 5 Times Sit-To-Stand (5×STS) Time [ Time Frame: Baseline, Week 49 ]

Central Contacts and Locations

Central contacts

Dyne Clinical Trials

+1-781-317-1919clinicaltrials@dyne-tx.com

Locations

UCSD - Altman Clinical and Translational Research Institute

Recruiting

La Jolla, California, United States, 92037

Contacts

Christian Farfan

cfarfan@health.ucsd.edu

UCLA Medical Center (Department of Neurology)

Recruiting

Los Angeles, California, United States, 90095

Contacts

Dennis Fernando

310-825-3264defernando@mednet.ucla.edu

Stanford Neuroscience Health Center

Recruiting

Palo Alto, California, United States, 94304

Contacts

Anita Chacko

650-272-8986neuromuscularresearch@stanford.edu

Rare Disease Research, LLC

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

Julia Zhu

678-883-6897julia.zhu@rarediseaseresearch.com

IU Health Neuroscience Center

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Jennifer Terrell

317-278-6266jkramey@iu.edu

Roy Blunt NextGen Precision Health Institute

Recruiting

Columbia, Missouri, United States, 65211

Contacts

Heather McHatton

573-882-7619heathermchatton@health.missouri.edu

Rare Disease Research, LLC

Recruiting

Hillsborough, North Carolina, United States, 27278

Contacts

Hannah Nation

984-314-2252hannah.nation@rarediseaseresearch.com

The University of Texas Health Science Center at San Antonio

Recruiting

San Antonio, Texas, United States, 78229

Contacts

Tonya D Randolph

210-450-8460Randolpht@uthscsa.edu

University of Utah Clinical Neurosciences Center

Recruiting

Salt Lake City, Utah, United States, 84132

Contacts

Michael Papadakis

8015814474m.papadakis@utah.edu

Virginia Commonwealth University - Biotech One

Recruiting

Richmond, Virginia, United States, 23298

Contacts

Levi Headrick

levi.headrick@vcuhealth.org

More Information

Sponsor

Dyne Therapeutics

Last update posted

Sep 3, 2026

Last verified

Apr, 2026

Keywords

  • DM1
  • Myotonic Dystrophy
  • Myotonic Dystrophy 1
  • Myotonia
  • Myotonic Dystrophy Type 1 (DM1)
  • Dystrophy Myotonic
  • Myotonic Disorders
  • Steinert Disease
  • Steinert
  • Myotonic Muscular Dystrophy
  • HARMONIA
  • Dyne Therapeutics
  • Dyne
  • DYNE-101
  • zeleciment basivarsen
  • z-basivarsen

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Dyne Therapeutics on 2026-09-03.