Actively Recruiting
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
Led by Dyne Therapeutics · Updated on 2026-05-12
116
Participants Needed
20
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of multiple intravenous doses of DYNE-101 in adults with Myotonic Dystrophy Type 1 (DM1), a condition characterized by muscle symptoms and myotonia. This randomized, placebo-controlled study involves both multiple-ascending dose and dose expansion groups. The study is designed to better understand how DYNE-101 behaves in the body and its effects on muscle function and myotonia severity. The study includes four periods: a Screening Period lasting up to 8 weeks, a Placebo-Controlled Period of 24 weeks where participants receive either DYNE-101 or placebo by IV infusion every 4 or 8 weeks, a Treatment Period of 24 weeks where all participants receive DYNE-101, and a Long-Term Extension Period of up to 168 weeks where participants continue receiving DYNE-101. Dosing schedules vary between every 4 weeks (Q4W) and every 8 weeks (Q8W). Participants will undergo various assessments throughout the study, including tests of muscle function such as hand grip relaxation time, walk/run tests, and stair climbing, as well as muscle tissue analysis for myotonia and other biomarkers. Researchers will monitor safety by tracking adverse events and drug levels in the blood. The study lasts up to about 4 years, including long-term follow-up to evaluate the effects and safety of DYNE-101 over time.
CONDITIONS
Brief 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 (DM1) with trinucleotide repeat size greater than 100
- Onset of DM1 muscle symptoms at age 12 years or older
- Clinically noticeable muscle stiffness with hand opening time of at least 2 seconds
- Hand grip and ankle dorsiflexion strength present
- Able to complete 10-meter walk/run test, stair climbing (MAD cohorts only), and sit to stand test 5 times without assistive devices such as canes, walkers, or orthoses
You will not qualify if you...
- Major surgery within 12 weeks before starting the study or planned major surgery during the study
- History of severe allergic reactions (anaphylaxis)
- Medical conditions other than DM1 that affect walking or participation in functional tests
- Use of medications that improve myotonia within 5 half-lives before screening
- Abnormal electrocardiogram with certain heart conduction issues or prolonged QT interval
- Forced vital capacity less than 50%
- Tibialis anterior muscle biopsy within 3 months before Day 1 or planned biopsy during the study for unrelated reasons
- History of suicide attempt, behavior, or significant suicidal thoughts within 6 months before screening
- Use of GLP-1 agonist medications within 5 half-lives before screening
- Significant weight loss during the study that could affect dosing or muscle function tests
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 weeks
Participants receive DYNE-101 or placebo by IV infusion once every 4 weeks or once every 8 weeks for up to 24 weeks in a randomized, placebo-controlled period. Participants who received placebo then receive DYNE-101 in a subsequent treatment period.
Infusions once every 4 or 8 weeks for up to 24 weeks
Duration - Up to 24 weeks
Participants continue to receive DYNE-101 by IV infusion once every 4 weeks or once every 8 weeks for up to 24 weeks following the placebo-controlled period.
Infusions once every 4 or 8 weeks for up to 24 weeks
Duration - Up to 168 weeks
Participants receive DYNE-101 by IV infusion once every 4 weeks or once every 8 weeks for up to 168 weeks during a long-term extension period to assess safety and efficacy.
Infusions once every 4 or 8 weeks for up to 168 weeks
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
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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