Actively Recruiting
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Led by Dyne Therapeutics · Updated on 2026-05-12
116
Participants Needed
20
Research Sites
355 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Myotonic Dystrophy Type 1 with trinucleotide repeat size greater than 100
- Onset of DM1 muscle symptoms at age 12 years or older
- Clinically apparent myotonia with hand opening time of at least 2 seconds as assessed by Investigator
- Hand grip strength and ankle dorsiflexion strength
- Ability to complete 10-meter walking test, stair ascend/descend (MAD cohorts only), and five-times sit-to-stand test at screening without assistive devices such as canes, walkers, or orthoses
You will not qualify if you...
- Major surgical procedure within 12 weeks before starting study treatment or expectation of major surgery during the study
- History of anaphylaxis
- Medical conditions other than DM1 that significantly affect walking or functional assessments
- Use of medications improving myotonia within five half-lives before screening assessments
- Electrocardiogram with QTcF above 450 ms in men or 460 ms in women, PR interval above 240 ms, left bundle-branch block, or clinically significant conduction defect
- Forced vital capacity below 50% predicted
- Tibialis anterior biopsy within 3 months before Day 1 or planned biopsy during study for unrelated reasons
- History of suicide attempt, suicidal behavior, or suicidal ideation with high risk within 6 months before screening
- Use of GLP-1 agonist medications within five half-lives before screening
- Significant weight loss during study participation affecting dosing or assessments
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 20 locations
1
Stanford University
Stanford, California, United States, 94305
Actively Recruiting
2
University of Florida College of Medicine
Gainesville, Florida, United States, 32610
Actively Recruiting
3
Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202
Actively Recruiting
4
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
5
Washington University in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
6
University of Rochester Medical Center
Rochester, New York, United States, 14642
Actively Recruiting
7
Neurology Rare Disease Center
Denton, Texas, United States, 76208
Actively Recruiting
8
Virginia Commonwealth University (VCU)
Richmond, Virginia, United States, 23298
Actively Recruiting
9
St. Vincent's Hospital
Fitzroy, Victoria, Australia, 3065
Actively Recruiting
10
CHU de Nantes
Nantes, France, 44093
Actively Recruiting
11
Institut de Myologie
Paris, France, 75013
Actively Recruiting
12
Charité - Universitätsmedizin Berlin
Berlin, Germany, 10117
Actively Recruiting
13
Ludwig Maximilians University, Munich - Friedrich Baur Institut
Munich, Germany, 80336
Actively Recruiting
14
Centro Clinico Nemo
Milan, Italy, 20162
Actively Recruiting
15
Fondazione Policlinico Universitario A Gemelli-Rome
Rome, Italy, 00168
Actively Recruiting
16
Radboud Medical Center
Nijmegen, Netherlands
Actively Recruiting
17
NZCR Auckland
Auckland, New Zealand, 1023
Actively Recruiting
18
University College London Hospitals
London, United Kingdom, NW1 2BU
Actively Recruiting
19
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, United Kingdom
Actively Recruiting
20
Salford Royal Hospital
Salford, United Kingdom, M6 8HD
Actively Recruiting
Research Team
D
Dyne Clinical Trials
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
8
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