Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

Completed · Phase 3 · Has a placebo group

Conditions studied: Muscular Dystrophy, Duchenne

In brief

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.

Key facts

Study ID

NCT03703882

Run by

Catabasis Pharmaceuticals

People needed

131

Starts

2018-10-02

Expected to finish

2020-09-22

Last updated by the study team

2022-06-21

Who can join

Age: 4 and older, up to 7. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify 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

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Children's Hospital of Los Angeles — Los Angeles, California, United States
• UC Davis — Sacramento, California, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Rare Disease Research, LLC — Atlanta, Georgia, United States
• Rush University Children's Hospital — Chicago, Illinois, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• University of Kansas Medical Center — Fairway, Kansas, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Johns Hopkins School of Medicine — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Las Vegas Clinic — Las Vegas, Nevada, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• MetroHealth Medical Center — Cleveland, Ohio, United States
• Shriners Hospitals for Children — Portland, Oregon, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Cook Children's Medical Center — Fort Worth, Texas, United States
• University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
• Children's Hospital of Richmond at VCU — Richmond, Virginia, United States
• The Children's Hospital at Westmead — Westmead, New South Wales, Australia

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.