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Study identifier: NCT05481879 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Myotonic Dystrophy Type 1 (DM1)  ·  Sponsor: Dyne Therapeutics

PhasePhase 1/Phase 2
Planned participants116
Who can joinAll sexes, 18 Years to 65 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Dyne Clinical Trials  ·  +1-781-317-1919  ·  clinicaltrials@dyne-tx.com

Always discuss trial participation with your own doctor first.

Locations (20)

Stanford UniversityStanford, California, United StatesRecruiting
University of Florida College of MedicineGainesville, Florida, United StatesRecruiting
Indiana University School of MedicineIndianapolis, Indiana, United StatesRecruiting
University of IowaIowa City, Iowa, United StatesRecruiting
Washington University in St. LouisSt Louis, Missouri, United StatesRecruiting
University of Rochester Medical CenterRochester, New York, United StatesRecruiting
Neurology Rare Disease CenterDenton, Texas, United StatesRecruiting
Virginia Commonwealth University (VCU)Richmond, Virginia, United StatesRecruiting
St. Vincent's HospitalFitzroy, Victoria, AustraliaRecruiting
CHU de NantesNantes, FranceRecruiting
Institut de MyologieParis, FranceRecruiting
Charité - Universitätsmedizin BerlinBerlin, GermanyRecruiting

+ 8 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT05481879