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DYNE-101 for Myotonic Dystrophy Type 1

This study is testing a drug called DYNE-101 for people with Myotonic Dystrophy Type 1 (DM1). Researchers want to see how safe DYNE-101 is and if your body can tolerate it. They will also look at how it affects symptoms like myotonia (muscle stiffness). Some participants will receive DYNE-101, while others will receive a placebo (an inactive substance). You might be able to join if you are between 18 and 65 years old, have a DM1 diagnosis with a specific genetic marker (trinucleotide repeat size >100), and experienced DM1 muscle symptoms at age 12 or older. You also need to have noticeable myotonia and certain hand and ankle strengths. The study's status is currently unclear, and it aims to enroll 116 participants.

Study design
This study is interventional and involves giving participants either DYNE-101 or a placebo through an IV. It includes different groups to test various doses and will follow participants for an extended period.
What's involved
You would participate in a screening period (up to 8 weeks), a placebo-controlled period (24 weeks), a treatment period (24 weeks), and a long-term extension period (168 weeks).
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety through study completion, up to Week 217. Changes in myotonia will be measured from baseline up to Week 25.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05481879

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

Recruiting
PHASE1Ages 18–65InterventionalTreatment
Dyne Therapeutics
~116 participants
Updated 2026-05-12 on ClinicalTrials.gov
What's tested:DYNE-101Placebo

At a glance

Recruiting sites
20 of 20 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Measured over Through study completion, up to Week 217
+1 more outcome measured

Conditions

Myotonic Dystrophy Type 1 (DM1)

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Where it's being run

20 sites across 15 states
United Kingdom3
France2
Germany2
Italy2
California1
Florida1
Indiana1
Iowa1

Who to contact

Dyne Clinical Trials
[email protected]
+1-781-317-1919
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

Exclusion

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.

What this trial measures

  • MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Through study completion, up to Week 217
  • Dose Expansion Cohorts: Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT)Baseline up to Week 25