At a glance
ClinicalIndex Comparison Record- ✓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
- ✓Hand grip strength and ankle dorsiflexion strength assessable
- ✕Major surgical procedure within 12 weeks prior or planned during study
- ✕History of anaphylaxis
- ✕Medical condition other than DM1 significantly impacting ambulation or functional assessments
- ✕QTcF ≥450 ms in men or ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or clinically significant conduction defect
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 2 clinical trial evaluating DYNE-101 and Placebo for Myotonic Dystrophy Type 1 (DM1). Currently recruiting, targeting 116 participants across 20 sites in 8 countries.
Detailed Summary
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.
Study Details
Timeline
Interventions
Administered by IV infusion
Administered by IV infusion