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

Launched by DYNE THERAPEUTICS · Jul 28, 2022

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment called DYNE-101 for people with Myotonic Dystrophy Type 1 (DM1), a genetic condition that affects muscle function. The main goal of the study is to see how safe and tolerable this treatment is when given through an intravenous (IV) method. Participants will go through different phases over about 2 years, including a screening period, a treatment phase where some people will receive the actual drug while others might receive a placebo (a substance with no active effect), and a long-term extension to gather more data.

To be eligible for the trial, participants must be diagnosed with DM1 and show certain muscle symptoms. They should be aged between 18 and 96, and able to perform specific physical tasks without assistance. However, individuals with certain medical conditions or recent surgeries, or those taking specific medications, will not be able to participate. Throughout the study, participants will be closely monitored to assess how the treatment affects them and to ensure their safety. This trial is currently recruiting, and it aims to provide valuable information that could help improve treatments for DM1 in the future.

Gender

ALL

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.