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

Stopped early · Phase 3

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT03917719

Run by

Catabasis Pharmaceuticals

People needed

130

Starts

2019-03-14

Expected to finish

2020-10-26

Last updated by the study team

2020-11-23

Who can join

Age: 4 and older, up to 12. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• UC Davis — Sacramento, California, United States
• Rare Disease Research, LLC — Atlanta, Georgia, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• University of Kansas Medical Center — Fairway, Kansas, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Johns Hopkins School of Medicine — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Las Vegas Clinic — Las Vegas, Nevada, United States
• Shriners Hospital for Children — Portland, Oregon, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, 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
• University of Utah — Salt Lake City, Utah, United States
• Royal Children's Hospital — Parkville, Victoria, Australia
• Children's Hospital of Eastern Ontario — Ottawa, Ontario, Canada
• University of Hamburg — Hamburg, Germany
• University of Munich — Munich, Germany
• Queen Silvia Children's Hospital — Gothenburg, Sweden
• Bristol Children's Hospital — Bristol, United Kingdom
• Great Ormond Street Hospital (GOSH) — London, United Kingdom
• Royal Manchester Children's Hospital — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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