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ClinicalTrials.gov ID

NCT07486934

Lead Sponsor

Dyne TherapeuticsINDUSTRY

Overall Status

Recruiting

Study Type

Interventional

Phase

Phase 3

Enrollment

150Estimated

Last Update Posted

Aug 18, 2026Actual

Start Date

May 14, 2026Actual

Primary Completion Date

Jul 2028Estimated

Completion Date

Jan 2029Estimated

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

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).

Detailed Description

The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks).

An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.

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

• zeleciment basivarsen (DYNE-101)
• Placebo

Ages Eligible for Study

Child (0-17)Adult (18-64)Older Adult (65+)

Sexes Eligible for Study

All

Accepts Healthy Volunteers

No

Eligibility Criteria

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.

Primary Purpose

Treatment

Allocation

Randomized

Interventional Model

Parallel Assignment

Masking

Quadruple

Lead Sponsor

Dyne TherapeuticsINDUSTRY

Lauren.Meers@uza.be+32 3 821 30 00