A Study of the Experimental Medicine Edasalonexent in Boys With Duchenne Muscular Dystrophy

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Age: 4 to 7 years old
Gender:
Male
Healthy Volunteers: No
Keywords: DMD, duchenne muscular dystrophy
Type: Drug study, Phase 3
Target:
4 Participants
Investigator:
Description
This research study will test the effectiveness and safety of experimental edasalonexent. It will be studied in patients with Duchenne Muscular Dystrophy (DMD). An experimental drug is one that has not been approved by the FDA to treat this condition.

A mutation (error) in the gene for dystrophin causes DMD. In DMD, dystrophin is not present in the muscle cells. Dystrophin is a protein that helps muscle cells keep their structure. Without dystrophin, muscle contractions damage the membrane around individual muscle cells.

This results in cellular degeneration (decline) and inflammation (swelling). It also prevents cellular regeneration (new cells). Over time, the cycle will cause the muscle to be replaced with fibrotic tissue (scar tissue) and fat.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

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
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid for taking part in this research. However, you will be reimbursed for travel cost.
Resources
Schedule
Study duration and period
You will be in the study for about 58 weeks. There will be a Screening Period of up to 4 weeks, a 52-week Treatment Period, and a 2-week safety follow-up. Subjects who complete this study may choose to continue in the extension study if available.
Recruitment period
From Feb. 25, 2019
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Research Topic
Conditions:
  • Muscular Dystrophy
  • Duchenne