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RECRUITING
NCT07486934
PHASE3

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

Sponsor: Dyne Therapeutics

View on ClinicalTrials.gov

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).

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

Key Details

Gender

All

Age Range

16 Years - Any

Study Type

INTERVENTIONAL

Enrollment

150

Start Date

2026-04

Completion Date

2029-01

Last Updated

2026-05-06

Healthy Volunteers

No

Interventions

DRUG

zeleciment basivarsen (DYNE-101)

Administered by IV infusion

DRUG

Placebo

Administered by IV infusion

Locations (5)

Rare Disease Research, LLC

Atlanta, Georgia, United States

Rare Disease Research, LLC

Hillsborough, North Carolina, United States

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

National Hospital Organization Osaka Toneyama Medical Center

Toyonaka-Shi, Osaka, Japan

Yamaguchi University Hospital

Ube-Shi, Yamaguchi, Japan