Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 27 June 2022
• Main ID: NCT03703882
• Date of registration: 08/10/2018
• Prospective Registration: No
• Primary sponsor: Catabasis Pharmaceuticals
• Public title: Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy PolarisDMD
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
• Date of first enrolment: October 2, 2018
• Target sample size: 131
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03703882
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Germany, Ireland, Israel, Sweden, United Kingdom, United States

Contacts

• Name: Joanne M Donovan, Chief Medical Officer, MD, PhD
• Affiliation: Catabasis Pharmaceuticals

Key inclusion & exclusion criteria

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

- 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

- Able to perform stand from supine without assistance in = 10 seconds

- Able to perform the 10MWT and 4-stair climb

- 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 corticosteroids within 24 weeks prior to Day 1; 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 have received at least 24 weeks of a stable dose of
eteplirsen prior to Day 1, and expected to continue treatment, will be eligible

- Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy,
warfarin, phenytoin, S mephenytoin, cyclosporine, dihydroergotamine, ergotamine,
fentanyl, alfentanil, pimozide, quinidine, sirolimus, tacrolimus, or paclitaxel

- Use of human growth hormone within 3 months prior to Day 1

- Other prior or ongoing significant medical conditions



Age minimum: 4 Years
Age maximum: 7 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: Placebo

Drug: Edasalonexent

Primary Outcome(s)

Change From Baseline in North Star Ambulatory Assessment (NSAA) [Time Frame: Baseline (Day 1) to Week 52]

Secondary Outcome(s)

Change From Baseline in Time to Stand From Supine [Time Frame: Baseline (Day 1) to Week 52]

Change From Baseline in 4-stair Climb [Time Frame: Baseline (Day 1) to Week 52]

Change From Baseline in 10-meter Walk/Run Test [Time Frame: Baseline (Day 1) to Week 52]

Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time Frame: Up to Week 52]

Secondary ID(s)

CAT-1004-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 21/06/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03703882