5481879
Last Update Posted: 2025-12-17
Recruiting
All Genders accepted | 18 Years-65 Years |
116 Estimated Participants | No Expanded Access |
Interventional Study | Does not accept healthy volunteers |
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
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 (144 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.
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.
Eligibility
Relevant conditions:
Myotonic Dystrophy Type 1 (DM1)
If you aren't sure if you meet the criteria above speak to your healthcare professional. Criteria may be updated but not reflected here, do not hesitate to contact the study if you think are close to fitting criteria.
locations
Contact Information
Overall Contact
Dyne Clinical Trials
clinicaltrials@dyne-tx.com
+1-781-317-1919
Data sourced from ClinicalTrials.gov