Trials / Recruiting
RecruitingNCT07486934
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
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
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 150 (estimated)
- Sponsor
- Dyne Therapeutics · Industry
- Sex
- All
- Age
- 16 Years
- Healthy volunteers
- Not accepted
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).
Detailed 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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | zeleciment basivarsen (DYNE-101) | Administered by IV infusion |
| DRUG | Placebo | Administered by IV infusion |
Timeline
- Start date
- 2026-04-01
- Primary completion
- 2028-07-01
- Completion
- 2029-01-01
- First posted
- 2026-03-23
- Last updated
- 2026-04-16
Locations
4 sites across 2 countries: United States, Japan
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07486934. Inclusion in this directory is not an endorsement.