An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
- Registration Number
- NCT03917719
- Lead Sponsor
- Catabasis Pharmaceuticals
- Brief 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 130
- 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
For Siblings of Patients who Completed CAT-1004-201 or CAT-1004-301:
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
- A sibling of a patient who completed either CAT-1004-201 or CAT-1004-301
- Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase (CK) and documentation of mutation(s) in the dystrophin gene known to be associated with a DMD phenotype
- Followed by a doctor or medical professional who coordinates Duchenne care on a regular basis and willingness to disclose patient's study participation with medical professionals
Exclusion Criteria:
- Use of oral corticosteroids at screening; use of inhaled, intranasal, and topical corticosteroids is permitted
- Use of another investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks. Exception: Patients who are currently on or plan to initiate treatment with approved oligonucleotide exon-skipping therapies, and expected to continue treatment throughout the study, will be eligible
- Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, anticoagulants, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus or tacrolimus
- Use of human growth hormone within 3 months prior to Day 1
- Other prior or ongoing significant medical conditions
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Dose 1 Edasalonexent Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
- Primary Outcome Measures
Name Time Method Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) 104 Weeks
- Secondary Outcome Measures
Name Time Method Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA) 104 Weeks Durability of effects of edasalonexent on physical function as measured by the 4-stair climb 104 Weeks Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test 104 Weeks Durability of effects of edasalonexent on physical function as measured by the time to stand from supine 104 Weeks
Trial Locations
- Locations (23)
Johns Hopkins School of Medicine
🇺🇸Baltimore, Maryland, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Las Vegas Clinic
🇺🇸Las Vegas, Nevada, United States
Shriners Hospital for Children
🇺🇸Portland, Oregon, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
UC Davis
🇺🇸Sacramento, California, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
University of Iowa Children's Hospital
🇺🇸Iowa City, Iowa, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
University of Texas Health Science Center at San Antonio
🇺🇸San Antonio, Texas, United States
Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia
Queen Silvia Children's Hospital
🇸🇪Gothenburg, Sweden
Children's Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
University of Hamburg
🇩🇪Hamburg, Germany
Bristol Children's Hospital
🇬🇧Bristol, United Kingdom
University of Munich
🇩🇪Munich, Germany
Great Ormond Street Hospital (GOSH)
🇬🇧London, United Kingdom
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom
University of Kansas Medical Center
🇺🇸Fairway, Kansas, United States