Evaluating DYNE-101 for treating Myotonic Dystrophy Type 1

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

This study is testing a new treatment called DYNE-101 to see if it is safe and effective for people with Myotonic Dystrophy Type 1.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 116 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Dyne Therapeutics (Industry-sponsored)
• Locations: 20 sites (Stanford, California and 19 other locations)
• Trial ID: NCT05481879 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and tolerability of multiple intravenous doses of DYNE-101 in participants diagnosed with Myotonic Dystrophy Type 1 (DM1). It consists of four periods: a screening phase lasting up to 8 weeks, a 24-week placebo-controlled phase, a 24-week treatment phase, and a long-term extension period of 144 weeks. Participants will be monitored for pharmacodynamic and pharmacokinetic responses to the treatment. The study includes both multiple-ascending dose and dose expansion cohorts to evaluate the drug's efficacy.

Who should consider this trial

Good fit: Ideal candidates are individuals diagnosed with DM1, aged 12 years or older, with specific muscle symptom criteria.

Not a fit: Patients with significant comorbid conditions affecting mobility or those who have undergone major surgeries recently may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of symptoms associated with Myotonic Dystrophy Type 1.

How similar studies have performed: While this approach is novel, similar studies targeting DM1 have shown promise in exploring new treatment avenues.

Eligibility criteria

Inclusion Criteria:

* 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 Criteria:

* 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 this trial is running

Stanford, California and 19 other locations

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

Study contacts

• Study coordinator: Dyne Clinical Trials

Conditions Myotonic Dystrophy Type 1