Phase 3
N=131
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne
Bottom Line
View on ClinicalTrials.gov: NCT03703882 ↗Enrolled (actual)
131
Serious AEs
1.5%
Results posted
Jun 2022
Primary outcome: Primary: Change From Baseline in North Star Ambulatory Assessment (NSAA) — -1.5; -1.8 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Edasalonexent (Drug); Placebo (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- Male
- Sponsor
- Catabasis Pharmaceuticals
- Primary completion
- Sep 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in North Star Ambulatory Assessment (NSAA) |
-1.5; -1.8 | — |
| SECONDARY Change From Baseline in 10-meter Walk/Run Test |
-0.0058; -0.0093 | — |
| SECONDARY Change From Baseline in Time to Stand From Supine |
-0.0389; -0.0459 | — |
| SECONDARY Change From Baseline in 4-stair Climb |
-0.0220; -0.0392 | — |
| SECONDARY Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
85; 41; 61; 14; 1; 1 | — |
Summary
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled.
Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the 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.
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT03703882). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.