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
Status: Recruiting
Location: See all (37) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
View:
• 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).
Locations
United States
California
UCSD - Altman Clinical and Translational Research Institute
RECRUITING
La Jolla
UCLA Medical Center (Department of Neurology)
RECRUITING
Los Angeles
Stanford Neuroscience Health Center
RECRUITING
Palo Alto
Georgia
Rare Disease Research, LLC
RECRUITING
Atlanta
Indiana
IU Health Neuroscience Center
RECRUITING
Indianapolis
Missouri
Roy Blunt NextGen Precision Health Institute
RECRUITING
Columbia
North Carolina
Rare Disease Research, LLC
RECRUITING
Hillsborough
Texas
The University of Texas Health Science Center at San Antonio
RECRUITING
San Antonio
Utah
University of Utah Clinical Neurosciences Center
RECRUITING
Salt Lake City
Virginia
Virginia Commonwealth University - Biotech One
RECRUITING
Richmond
Other Locations
Australia
St Vincent's Hospital, Melbourne
RECRUITING
Fitzroy
Belgium
UZA
RECRUITING
Edegem
UZ Leuven
RECRUITING
Leuven
Denmark
Aarhus University Hospital, Department of Neurology
RECRUITING
Aarhus
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)
RECRUITING
Copenhagen
France
AP-HP, Hôpital Raymond-Poincaré
RECRUITING
Garches
CHU Marseille - Hôpital de la Timone (APHM)
RECRUITING
Marseille
CHU de Nantes - Hôtel-Dieu
RECRUITING
Nantes
Pitié-Salpêtrière Hospital
RECRUITING
Paris
CHU de Toulouse - Hôpital Pierre-Paul Riquet
RECRUITING
Toulouse
Germany
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit
RECRUITING
Berlin
Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen
RECRUITING
Bonn
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt
RECRUITING
Munich
Italy
Centro Clinico Nemo Brescia, Fondazione Serena ETS
RECRUITING
Gussago
Centro Clinico Nemo Milano, Fondazione Serena ETS
RECRUITING
Milan
Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia
RECRUITING
Roma
Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile
RECRUITING
Roma
Japan
Niigata National Hospital, National Hospital Organization
RECRUITING
Kashiwazaki
National Center Hospital, National Center of Neurology and Psychiatry (NCNP)
RECRUITING
Kodaira
The University of Osaka Hospital
RECRUITING
Suita-shi
National Hospital Organization Osaka Toneyama Medical Center
RECRUITING
Toyonaka-shi
Yamaguchi University Hospital
RECRUITING
Ube-shi
Netherlands
University of Maastricht
RECRUITING
Maastricht
Radboud University Medical Center
RECRUITING
Nijmegen
Spain
Hospital Universitario Infanta Sofía
RECRUITING
San Sebastián De Los Reyes
United Kingdom
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
RECRUITING
London
Northern Care Alliance NHS Foundation Trust
RECRUITING
Salford
Contact Information
Primary
Dyne Clinical Trials
clinicaltrials@dyne-tx.com
+1-781-317-1919
Time Frame
Start Date: 2026-05-14
Estimated Completion Date: 2029-01
Participants
Target number of participants: 150
Treatments
Experimental: Placebo-Controlled Period: DYNE-101
Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks.
Placebo_comparator: Placebo-Controlled Period: Placebo
Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.
Experimental: Long-Term Extension Period: DYNE-101
Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks.~Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.
Related Therapeutic Areas
Sponsors
Leads: Dyne Therapeutics