Rare Disease Research, LLC | Atlanta, GA
Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
About
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Exclusion criteria
Other inclusion and exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
88 participants in 3 patient groups
Loading...
Central trial contact
Dyne Clinical Trials
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal