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An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)
study id #: NCT03917719
condition: Duchenne Muscular Dystrophy
status: enrolling by invitationpurpose:
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 4-10 years of age (up to 11th 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.
mechanism of action: NF-KB inhibitor to prevent muscle damage and promote muscle regeneration
last updated: April 18, 2019
start date: March 14, 2019
estimated completion: June 2022
phase of development: Phase 3
size / enrollment: 100
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.
- 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 ]
• 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
• 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
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