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
Dyne Therapeutics
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).
Description
The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks). An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
Eligibility
- Age range
- 16+ years
- Sex
- All
- 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…
Interventions
- Drugzeleciment basivarsen (DYNE-101)
Administered by IV infusion
- DrugPlacebo
Administered by IV infusion
Locations (5)
- Rare Disease Research, LLCAtlanta, Georgia
- Rare Disease Research, LLCHillsborough, North Carolina
- The University of Texas Health Science Center at San AntonioSan Antonio, Texas
- National Hospital Organization Osaka Toneyama Medical CenterToyonaka-Shi, Osaka
- Yamaguchi University HospitalUbe-Shi, Yamaguchi