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

a study on Myotonic Dystrophy Steinert Disease Steinert

Summary

Eligibility
for people ages 16 years and up (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
study ends around

Description

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

Details

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.

Keywords

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

Eligibility

You can join if…

Open to people ages 16 years and up

  • 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/m2).

You CAN'T join if...

  • 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.

Locations

  • UCLA Medical Center (Department of Neurology) accepting new patients
    Los Angeles California 90095 United States
  • UCSD - Altman Clinical and Translational Research Institute accepting new patients
    La Jolla California 92037 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Dyne Therapeutics
Links
Sign up for this study
ID
NCT07486934
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 150 study participants
Last Updated
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