An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD) | DuchenneXchange

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enrolling by invitation

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

key information

study id #: NCT03917719

condition: Duchenne Muscular Dystrophy

status: enrolling by invitation

purpose:

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.

intervention: Edasalonexent

mechanism of action: NF-KB inhibitor to prevent muscle damage and promote muscle regeneration

results: https://clinicaltrials.gov/ct2/show/results/NCT03917719

last updated: April 18, 2019

study details

start date: March 14, 2019

estimated completion: June 2022

phase of development: Phase 3

size / enrollment: 100

study 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.

primary outcomes:

  • 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 ]

secondary outcomes:

  • 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 ]

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

study contacts

sponsor: Catabasis Pharmaceuticals

investigators: Joanne M Donovan, MD, PhD

locations: United States