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

Study Overview
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Brief Summary
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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
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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.

Official Title
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An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: Edasalonexent


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 12 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Dose 1<br>Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day. | Drug: Edasalonexent<br>* 100 mg/kg/day<br>* Other names: CAT-1004;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 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 | 
| Durability of effects of edasalonexent on physical function as measured by the 4-stair climb |  | 104 Weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, DMD, dystrophin, dystrophy, Duchenne