Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Condition(s) studied
Investigational drug(s) / intervention(s)
DYNE-101: Administered by IV infusion
Placebo: Administered by IV infusion
Study 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 (208 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.
Eligibility
Primary outcome measure(s)
- MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — Through study completion, up to Week 257
- Dose Expansion Cohorts: Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT) — Baseline up to Week 25
Trial sites (20)
| Facility | City | Region | Status |
|---|---|---|---|
| Stanford University | Stanford | California | |
| University of Florida College of Medicine | Gainesville | Florida | |
| Indiana University School of Medicine | Indianapolis | Indiana | |
| University of Iowa | Iowa City | Iowa | |
| Washington University in St. Louis | St Louis | Missouri | |
| University of Rochester Medical Center | Rochester | New York | |
| Neurology Rare Disease Center | Denton | Texas | |
| Virginia Commonwealth University (VCU) | Richmond | Virginia | |
| St. Vincent's Hospital | Fitzroy | Victoria | |
| CHU de Nantes | Nantes | France | |
| Institut de Myologie | Paris | France | |
| Charité - Universitätsmedizin Berlin | Berlin | Germany | |
| Ludwig Maximilians University, Munich - Friedrich Baur Institut | Munich | Germany | |
| Centro Clinico Nemo | Milan | Italy | |
| Fondazione Policlinico Universitario A Gemelli-Rome | Rome | Italy | |
| Radboud Medical Center | Nijmegen | Netherlands | |
| NZCR Auckland | Auckland | New Zealand | |
| University College London Hospitals | London | United Kingdom | |
| John Walton Muscular Dystrophy Research Centre | Newcastle upon Tyne | United Kingdom | |
| Salford Royal Hospital | Salford | United Kingdom |
More Dyne Therapeutics trials in the UK
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05481879 on ClinicalTrials.gov ↗ ← All trials in the UK