Summary

for males ages 4-7 (full criteria)
at Sacramento, California and other locations
study started
estimated completion

Description

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.

Official Title

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

Details

The study includes a 52-week, randomized, double-blind, placebo-controlled period, followed by a 2-week follow- up. Approximately 125 boys with DMD will be enrolled in this trial, with 2 boys receiving edasalonexent for every 1 boy receiving placebo.

Following completion of the treatment period, patients may elect to continue in a separate open-label extension study.

Keywords

Muscular Dystrophy, Duchenne Muscular Dystrophies Musculoskeletal Diseases Neuromuscular Diseases Duchenne muscular dystrophy DMD dystrophin dystrophy Duchenne Edasalonexent Dose 1

Eligibility

You can join if…

Open to males ages 4-7

  • 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

You CAN'T join if...

  • 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

Locations

  • UC Davis
    Sacramento California 95817 United States
  • Children's Hospital of Los Angeles
    Los Angeles California 90027 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Catabasis Pharmaceuticals
Links
Sign up for this study
ID
NCT03703882
Phase
Phase 3
Study Type
Interventional
Last Updated