NCT05481879

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 116 (estimated)
• Sponsor: Dyne Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Conditions

• Myotonic Dystrophy Type 1 (DM1)

Interventions

Type	Name	Description
DRUG	DYNE-101	Administered by IV infusion
DRUG	Placebo	Administered by IV infusion

Timeline

• Start date: 2022-09-05
• Primary completion: 2029-07-01
• Completion: 2029-07-01
• First posted: 2022-08-01
• Last updated: 2026-03-05

Locations

17 sites across 8 countries: United States, Australia, France, Germany, Italy, Netherlands, New Zealand, United Kingdom

Regulatory

• FDA-regulated drug study