Duchenne Muscular Dystrophy
Conditions
Keywords
Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, DMD, dystrophin, dystrophy, Duchenne
Brief summary
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.
Detailed description
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.
Interventions
DRUGEdasalonexent
100 mg/kg/day
Sponsors
Catabasis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
4 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
For Patients who Completed CAT-1004-201 or CAT-1004-301: Inclusion Criteria: * Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements * Completion of either CAT-1004-201 or CAT-1004-301
Exclusion criteria
* In the Investigator's opinion, unwilling or unable for any reason to complete all study assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study procedures For Siblings of Patients who Completed CAT-1004-201 or CAT-1004-301: Inclusion Criteria: * Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements * A sibling of a patient who completed either CAT-1004-201 or CAT-1004-301 * Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase (CK) and documentation of mutation(s) in the dystrophin gene known to be associated with a DMD phenotype * Followed by a doctor or medical professional who coordinates Duchenne care on a regular basis and willingness to disclose patient's study participation with medical professionals
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | 104 Weeks |
Secondary
| Measure | Time frame |
|---|---|
| Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA) | 104 Weeks |
| Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test | 104 Weeks |
| Durability of effects of edasalonexent on physical function as measured by the time to stand from supine | 104 Weeks |
| Durability of effects of edasalonexent on physical function as measured by the 4-stair climb | 104 Weeks |
Countries
Australia, Canada, Germany, Sweden, United Kingdom, United States
Outcome results
None listed