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Recruiting Phase 3

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

NCT07486934 · tracked via the Priya Life Science UK tracker
Phase
Phase 3
Started
2026-05-14
Last updated
2026-10-13

Condition(s) studied

Myotonic Dystrophy Type 1 (DM1)DM1Myotonic DystrophySteinert DiseaseSteinert

Investigational drug(s) / intervention(s)

zeleciment basivarsen (DYNE-101) →Placebo

zeleciment basivarsen (DYNE-101): Administered by IV infusion

Placebo: Administered by IV infusion

Study 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

Sex
ALL
Min age
16 Years
Max age
—
Healthy volunteers
No
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 outcome measure(s)

  • 5 Times Sit-To-Stand (5×STS) Time — Baseline, Week 49

Trial sites (43)

FacilityCityRegionStatus
UCSD - Altman Clinical and Translational Research Institute La Jolla California Recruiting
UCLA Medical Center (Department of Neurology) Los Angeles California Recruiting
Stanford Neuroscience Health Center Palo Alto California Recruiting
UF Health: University of Florida Clinical Research Center Gainesville Florida Recruiting
Rare Disease Research, LLC Atlanta Georgia Recruiting
IU Health Neuroscience Center Indianapolis Indiana Recruiting
Kennedy Krieger Institute Baltimore Maryland Recruiting
Roy Blunt NextGen Precision Health Institute Columbia Missouri Recruiting
University of Rochester Medical Center Rochester New York Recruiting
Rare Disease Research, LLC Hillsborough North Carolina Recruiting
Houston Methodist Neurological Institute Houston Texas Recruiting
The University of Texas Health Science Center at San Antonio San Antonio Texas Recruiting
University of Utah Clinical Neurosciences Center Salt Lake City Utah Recruiting
Virginia Commonwealth University - Biotech One Richmond Virginia Recruiting
St Vincent's Hospital, Melbourne Fitzroy Victoria Recruiting
UZA Edegem Belgium Recruiting
UZ Leuven Leuven Belgium Recruiting
Aarhus University Hospital, Department of Neurology Aarhus Denmark Recruiting
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077) Copenhagen Denmark Recruiting
AP-HP, Hôpital Raymond-Poincaré Garches France Recruiting
CHU Marseille - Hôpital de la Timone (APHM) Marseille France Recruiting
CHU de Nantes - Hôtel-Dieu Nantes France Recruiting
Pitié-Salpêtrière Hospital Paris France Recruiting
CHU de Toulouse - Hôpital Pierre-Paul Riquet Toulouse France Recruiting
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt Munich Bavaria Recruiting
Universitätsmedizin Göttingen Göttingen Lower Saxony Recruiting
Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen Bonn North Rhine-Westphalia Recruiting
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit Berlin State of Berlin Recruiting
Centro Clinico Nemo Brescia, Fondazione Serena ETS Gussago Brescia Recruiting
Centro Clinico Nemo Milano, Fondazione Serena ETS Milan Milano Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile Roma Roma Recruiting
Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia Roma Roma Recruiting
Niigata National Hospital, National Hospital Organization Kashiwazaki Niigata Recruiting
The University of Osaka Hospital Suita-shi Osaka Recruiting
National Hospital Organization Osaka Toneyama Medical Center Toyonaka-Shi Osaka Recruiting
National Center Hospital, National Center of Neurology and Psychiatry (NCNP) Kodaira Tokyo Recruiting
Yamaguchi University Hospital Ube-Shi Yamaguchi Recruiting
University of Maastricht Maastricht Netherlands Recruiting
Radboud University Medical Center Nijmegen Netherlands Recruiting
Hospital Universitari Vall d'Hebron Barcelona Barcelona Recruiting

+ 3 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07486934 on ClinicalTrials.gov ↗ ← All trials in the UK