Study Details
A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1
(IRB#: IRB_00199334)
This study seeks to test the safety and effectiveness of the drug DYNE-101 for the treatment of Myotonic Dystrophy Type 1
- All genders
- Over 7 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: All genders
Age: Over 7 years old
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- DM1 diagnosis with genetic confirmation
- Functional assessment scores within designated range, as determined during a study screening visit
- Body mass index (BMI) < 35kg/m2
- Willing to follow contraceptive guidelines for study duration
- Willing and able to give informed consent and comply with study visit schedule and procedures, including in-person and at-home assessments
Exclusion Criteria
- History of deep vein thrombosis or pulmonary embolism (blood clots) within last five years
- Active cancer within last five years
- Significant liver, kidney, heart, or blood disease, or other condition that could affect participant safety or impair study assessments
- Ongoing or previous treatment with a medication or therapy restricted by the study
Will I be paid for my time?
Yes
IRB#: IRB_00199334
PI: Kyle Mahoney
Department: NEUROMUSCULAR RESEARCH
Approval Date: 2026-06-24 06:00:00
Study Categories: Brain Health Studies
Specialties: Neurology
I am Interested