NCT03917719

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 130 (actual)
• Sponsor: Catabasis Pharmaceuticals · Industry
• Sex: Male
• Age: 4 Years – 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Edasalonexent	100 mg/kg/day

Timeline

• Start date: 2019-03-14
• Primary completion: 2020-10-26
• Completion: 2020-10-26
• First posted: 2019-04-17
• Last updated: 2020-11-23

Locations

23 sites across 6 countries: United States, Australia, Canada, Germany, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study