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
Status: Recruiting
Location: See all (15) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• 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.
• Able to complete 10-MWRT, stair ascend/descend (MAD cohorts only), and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses.
Locations
United States
Iowa
University of Iowa
RECRUITING
Iowa City
Indiana
Indiana University School of Medicine
RECRUITING
Indianapolis
New York
University of Rochester Medical Center
RECRUITING
Rochester
Texas
Neurology Rare Disease Center
RECRUITING
Denton
Other Locations
Australia
St. Vincent's Hospital
RECRUITING
Fitzroy
France
CHU de Nantes
RECRUITING
Nantes
Institut de Myologie
RECRUITING
Paris
Germany
Ludwig Maximilians University, Munich - Friedrich Baur Institut
RECRUITING
Munich
Italy
Centro Clinico Nemo
RECRUITING
Milan
Fondazione Policlinico Universitario A Gemelli-Rome
Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q4W or Q8W for up to 24 weeks.~Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q4W or Q8W for up to 24 weeks.
Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q8W for up to 24 weeks.~Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.