The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD.
The study includes a 104-week open-label treatment period with edasalonexent. Patients who completed CAT-1004-201 or CAT-1004-301 and eligible siblings of these boys will be enrolled in this trial.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
130
100 mg/kg/day
UC Davis
Sacramento, California, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Las Vegas Clinic
Las Vegas, Nevada, United States
Shriners Hospital for Children
Portland, Oregon, United States
...and 13 more locations
Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: 104 Weeks
Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)
Time frame: 104 Weeks
Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test
Time frame: 104 Weeks
Durability of effects of edasalonexent on physical function as measured by the time to stand from supine
Time frame: 104 Weeks
Durability of effects of edasalonexent on physical function as measured by the 4-stair climb
Time frame: 104 Weeks
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