Zeleciment Basivarsen (DYNE-101) for people with myotonic dystrophy type 1

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3, interventional study enrolls adults with genetically confirmed myotonic dystrophy type 1 (DM1) to receive Zeleciment Basivarsen (DYNE-101) or placebo during a 48-week placebo-controlled period followed by a 24-week long-term extension. Eligible participants must have a CTG repeat size >100, be able to walk 10 meters and complete five sit-to-stand maneuvers independently, and have a BMI below 35 kg/m2. The protocol includes up to 8 weeks of screening and regular safety and tolerability reviews by an independent data monitoring committee. Study visits are conducted at participating U.S. clinical sites with serial clinical assessments and safety monitoring throughout the treatment periods.

Why it matters

Who can join: the full rules

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.

Where it's running

• UCSD - Altman Clinical and Translational Research Institute
  La Jolla, California, United States Recruiting
• UCLA Medical Center (Department of Neurology)
  Los Angeles, California, United States Recruiting
• Stanford Neuroscience Health Center
  Palo Alto, California, United States Recruiting
• UF Health: University of Florida Clinical Research Center
  Gainesville, Florida, United States Recruiting
• Rare Disease Research, LLC
  Atlanta, Georgia, United States Recruiting
• IU Health Neuroscience Center
  Indianapolis, Indiana, United States Recruiting

Study contacts

• Study coordinator: Dyne Clinical Trials
• Email: clinicaltrials@dyne-tx.com
• Phone: +1-781-317-1919

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Understanding long-term progression in myotonic dystrophy type 1 (DM1)

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• Remote monitoring and genetic analysis for myotonic dystrophy type 1

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• Spanish National Registry for Myotonic Dystrophy Type 1

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• Online registry for myotonic dystrophy patients

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• Quantitative MRI measurement of muscle changes in myotonic dystrophy

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• Portable markerless video system to measure walking in adults with neuromuscular conditions or healthy volunteers.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.