Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- 5 Times Sit-To-Stand (5×STS) Time — Baseline, Week 49
Trial sites (43)
| Facility | City | Region | Status |
|---|---|---|---|
| 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.
More Dyne Therapeutics trials in the UK
Other trials for the same condition
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