An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)
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|ClinicalTrials.gov Identifier: NCT03917719|
Recruitment Status : Enrolling by invitation
First Posted : April 17, 2019
Last Update Posted : July 13, 2020
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.
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Edasalonexent||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||140 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy|
|Actual Study Start Date :||March 14, 2019|
|Estimated Primary Completion Date :||September 2022|
|Estimated Study Completion Date :||September 2022|
Experimental: Dose 1
Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03917719
|Study Chair:||Joanne M Donovan, MD, PhD||Catabasis Pharmaceuticals|