Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Start Date
9/5/2022
Completion Date
7/1/2029
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.
Eligibility Criteria
Age Range: 18 years to 65 years
Interventions
DYNE-101
Placebo
Conditions
Locations
Stanford University
Stanford, California 94305
United States
University of Florida College of Medicine
Gainesville, Florida 32610
United States
Indiana University School of Medicine
Indianapolis, Indiana 46202
United States
University of Iowa
Iowa City, Iowa 52242
United States
Washington University in St. Louis
St Louis, Missouri 63110
United States
University of Rochester Medical Center
Rochester, New York 14642
United States
Neurology Rare Disease Center
Denton, Texas 76208
United States
Virginia Commonwealth University (VCU)
Richmond, Virginia 23298
United States
St. Vincent's Hospital
Fitzroy, Victoria 3065
Australia
CHU de Nantes
Nantes, 44093
France
Institut de Myologie
Paris, 75013
France
Charité - Universitätsmedizin Berlin
Berlin, 10117
Germany
Ludwig Maximilians University, Munich - Friedrich Baur Institut
Munich, 80336
Germany
Centro Clinico Nemo
Milan, 20162
Italy
Fondazione Policlinico Universitario A Gemelli-Rome
Rome, 00168
Italy
Radboud Medical Center
Nijmegen,
Netherlands
NZCR Auckland
Auckland, 1023
New Zealand
University College London Hospitals
London, NW1 2BU
United Kingdom
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne,
United Kingdom
Salford Royal Hospital
Salford, M6 8HD
United Kingdom