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

Ki vehet részt

• 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

Ki nem vehet részt

• 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
• Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments
• 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
• History of anaphylaxis
• Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments
• 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
• 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%
• 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