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


Open to: Male

Age: 4 Years - 12 Years

Medical Conditions

Duchenne Muscular Dystrophy

This information is provided directly by researchers and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information.

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.

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Mar 2019

Oct 2020


Intervention Type : Drug
Intervention Description : 100 mg/kg/day

Intervention Arm Group : Dose 1

You can take part if:

For 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 - 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 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

You may not be able to take part if:

This is in the inclusion criteria above

Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Royal Manchester Children's Hospital
    M13 9WL
  • Bristol Children's Hospital
    BS2 8AE
  • Great Ormond Street Hospital (GOSH)
    WC1N 3JH

The study is sponsored by Catabasis Pharmaceuticals

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for Trial ID: NCT03917719

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