Study Details
Testing the Drug DYNE-251 for People with Duchenne Muscular Dystrophy
(IRB#: IRB_00151486)
Duchenne Muscular Dystrophy is a disease that prevents muscles in the body from being protected from stress and damage. This can cause falling, making it hard to get up and run. This study will test the drug DYNE-251 for people with this disease. The study wants to see if the drug will help people with this disease and is safe. People in the study will be randomly chosen to be treated with the study drug DYNE-251 or a placebo. A placebo looks like the drug but without the medication. Being in the study requires up to 42 study visits for about 35 months. The visits will be in person at a study clinic. Medical tests will be done to track the health of participants.
- Male Only
- Under 18 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: Male Only
Age: Under 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- Male participants who are 4-16 years old
- Diagnosis of Duchenne muscular dystrophy
- Currently taking regular medication of glucocorticoids, such as prednisone, prednisolone, and deflazacort for at least 12 weeks
- Low level of blood leaving the left ventricle of the heart each time the heart pumps
Exclusion Criteria
- Congestive heart failure that is not under control
- Major surgery within 12 weeks before study participation or might have surgery during the study
- Need a ventilator (a machine that gives oxygen) to breathe during the day
- History of suicide attempt, behavior, or thoughts of suicide 6 months before study participation
Will I be paid for my time?
Yes
IRB#: IRB_00151486
PI: Russell Butterfield
Department: PEDIATRIC NEUROLOGY
Approval Date: 2023-01-11 07:00:00
Study Categories: Brain Health Studies
Specialties: Pediatric Neurology