RecruitingPhase 3NCT07486934

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

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

Sponsor

Dyne Therapeutics

Enrollment

150 participants

Start Date

Apr 1, 2026

Study Type

INTERVENTIONAL

Conditions

Myotonic Dystrophy DM1 Steinert Disease Myotonic Dystrophy Type 1 (DM1)Steinert

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).

Eligibility

Min Age: 16 Years

Inclusion Criteria3

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 Criteria3

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.

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