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

Recruiting now · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Myotonic Dystrophy Type 1 (DM1)

In brief

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.

Key facts

Study ID

NCT05481879

Run by

Dyne Therapeutics

People needed

116

Starts

2022-09-05

Expected to finish

2029-07-01

Last updated by the study team

2026-05-12

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of DM1 with trinucleotide repeat size >100.
• Age of onset of DM1 muscle symptoms ≥12 years.
• Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator.
• Hand grip strength and ankle dorsiflexion strength.
• Able to complete 10-MWRT, stair ascend/descend (MAD cohorts only), and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses.

You may not qualify if…

• History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study.
• History of anaphylaxis.
• Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments.
• Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments.
• Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is clinically significant in the opinion of the Investigator.
• Percent predicted forced vital capacity (FVC) <50%.
• History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies during study period for reasons unrelated to the study.
• Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) or who, in the opinion of the Investigator, is at significant risk to commit suicide.
• Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.
• Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers.
• Note: Other inclusion and exclusion criteria may apply.

Where it is running

• Stanford University — Stanford, California, United States (enrolling)
• University of Florida College of Medicine — Gainesville, Florida, United States (enrolling)
• Indiana University School of Medicine — Indianapolis, Indiana, United States (enrolling)
• University of Iowa — Iowa City, Iowa, United States (enrolling)
• Washington University in St. Louis — St Louis, Missouri, United States (enrolling)
• University of Rochester Medical Center — Rochester, New York, United States (enrolling)
• Neurology Rare Disease Center — Denton, Texas, United States (enrolling)
• Virginia Commonwealth University (VCU) — Richmond, Virginia, United States (enrolling)
• St. Vincent's Hospital — Fitzroy, Victoria, Australia (enrolling)
• CHU de Nantes — Nantes, France (enrolling)
• Institut de Myologie — Paris, France (enrolling)
• Charité - Universitätsmedizin Berlin — Berlin, Germany (enrolling)
• Ludwig Maximilians University, Munich - Friedrich Baur Institut — Munich, Germany (enrolling)
• Centro Clinico Nemo — Milan, Italy (enrolling)
• Fondazione Policlinico Universitario A Gemelli-Rome — Rome, Italy (enrolling)
• Radboud Medical Center — Nijmegen, Netherlands (enrolling)
• NZCR Auckland — Auckland, New Zealand (enrolling)
• University College London Hospitals — London, United Kingdom (enrolling)
• John Walton Muscular Dystrophy Research Centre — Newcastle upon Tyne, United Kingdom (enrolling)
• Salford Royal Hospital — Salford, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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