welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
A Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)
study id #: NCT03703882
condition: Muscular Dystrophy, Duchenne
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.
intervention: Edasalonexent, Placebo
mechanism of action: NF-KB inhibitor to prevent muscle damage and promote muscle regeneration
last updated: February 15, 2019
start date: October 2, 2018
estimated completion: June 2020
phase of development: Phase 3
size / enrollment: 125
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.
- Change from baseline in North Star Ambulatory Assessment (NSAA) [Time Frame: 52 Weeks]
- Safety and tolerability measured by number of treatment- emergent adverse events (TEAEs) and serious adverse events (SAEs) [Time Frame: 52 Weeks]
- Change from baseline in 10-meter walk/run test [Time Frame: 52 Weeks]
- Change from baseline in time to stand from supine [Time Frame: 52 Weeks]
- Change from baseline in 4-stair climb [Time Frame: 52 Weeks]
• 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
• 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
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)The primary objective of this Expanded A...
How Facebook and Twitter could be the next disruptive force in clinical trialsAmber Sapp was browsing the Internet lat...
Mother-caregivers of Children With Duchenne Muscular DystrophyThe incidence of Duchenne Muscular Dystr...
Rare diseases require unique approaches in clinical trial designThere are 7,000 known rare diseases and ...
Clinical trial shows some promise for Duchenne muscular dystrophy drugThe results of an international clinical...
DMD clinical therapies II: P.135 DMD-HUB: expanding clinical trial capacity for Duchenne muscular dystrophy, 1 year ...The need to increase capacity for Duchen...
Edasalonexent could reduce functional decline in boys with DMD, MoveDMD phase 2 results suggestResults of the Phase 2 MoveDMD trial sho...