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 (4) 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
Georgia
Rare Disease Research, LLC
RECRUITING
Atlanta
North Carolina
Rare Disease Research, LLC
RECRUITING
Hillsborough
Texas
The University of Texas Health Science Center at San Antonio
RECRUITING
San Antonio
Other Locations
Japan
Yamaguchi University Hospital
RECRUITING
Ube-shi
Contact Information
Primary
Dyne Clinical Trials
clinicaltrials@dyne-tx.com
+1-781-317-1919
Time Frame
Start Date: 2026-04
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