NCT03703882

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 131 (actual)
• Sponsor: Catabasis Pharmaceuticals · Industry
• Sex: Male
• Age: 4 Years – 7 Years
• Healthy volunteers: Not accepted

Summary

The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the 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 52-week, randomized, double-blind, placebo-controlled period, followed by a 2-week follow- up. Approximately 125 boys with DMD will be enrolled in this trial, with 2 boys receiving edasalonexent for every 1 boy receiving placebo. Following completion of the treatment period, patients may elect to continue in a separate open-label extension study.

Conditions

• Muscular Dystrophy, Duchenne

Interventions

Type	Name	Description
DRUG	Edasalonexent	100 mg/kg/day
DRUG	Placebo	Placebo

Timeline

• Start date: 2018-10-02
• Primary completion: 2020-09-22
• Completion: 2020-09-22
• First posted: 2018-10-12
• Last updated: 2022-06-21
• Results posted: 2022-06-21

Locations

40 sites across 8 countries: United States, Australia, Canada, Germany, Ireland, Israel, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study