Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age16+
SponsorDyne Therapeutics
About this trial
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 criteria
Qualifiers
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).
Disqualifiers
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.
Trial design
Treatments tested in this trial
- zeleciment basivarsen (DYNE-101)
- Placebo
Treatment groups
150 Participants
are divided into 3 treatment groupsLocations
14Belgium
UZ Leuven3000, Leuven Belgium
Denmark
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)2100, Copenhagen Denmark
France
Pitié-Salpêtrière Hospital75013, Paris France
Germany
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt80336, MunichBavaria, Germany
Sponsors and collaborators
Dyne Therapeutics
Lead sponsor
Source ClinicalTrials.gov