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.
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.
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.
Sequential
DYNE-101
DrugAdministered by IV infusion
Placebo
DrugAdministered by IV infusion
8
Treatment groupsSee each treatment group below.
MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Dose Expansion Cohorts: Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT)
MAD Cohorts: Change From Baseline in Composite Alternative Splicing Index (CASI) in Skeletal Muscle Tissue
MAD Cohorts: Change From Baseline in Dystrophia Myotonica Protein Kinase (DMPK) Ribonucleic Acid (RNA) Expression in Muscle Tissue
MAD Cohorts: Change From Baseline in Hand Grip Relaxation Time
MAD Cohorts: Change From Baseline in Myotonia as Measured by vHOT
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