Oral Ifetroban for Duchenne Muscular Dystrophy
(DMD Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD.Funding Source - FDA OOPD
Will I have to stop taking my current medications?
The trial allows participants to continue taking certain heart medications like ACE inhibitors, beta-blockers, and angiotensin receptor blockers if they have been on a stable dose for at least three months. However, no changes to these medications are allowed during the study unless there's a significant decline in heart function. The protocol does not specify about other medications, so it's best to discuss with the trial team.
How is the drug Ifetroban unique in treating Duchenne Muscular Dystrophy?
Ifetroban is unique because it is an oral medication specifically being studied for Duchenne Muscular Dystrophy, a condition with limited treatment options. Unlike other treatments, Ifetroban is known for its potential to target specific pathways involved in muscle damage and inflammation, which may offer a novel approach to managing this condition.12345
Who Is on the Research Team?
Larry Markham, MD
Principal Investigator
Riley Children's Hospital
Are You a Good Fit for This Trial?
This trial is for males aged 7 and older with Duchenne Muscular Dystrophy (DMD). Participants must have stable heart function, be on certain heart medications for a set time without changes, and can't have had recent major surgery or other clinical trials. They need to be able to undergo MRI scans and not require specific heart failure treatments recently.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive oral ifetroban or placebo once daily for 12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of treatment with high-dose ifetroban long-term
What Are the Treatments Tested in This Trial?
Interventions
- Ifetroban
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Cumberland Pharmaceuticals
Lead Sponsor
A.J. Kazimi
Cumberland Pharmaceuticals
Chief Executive Officer since 1999
B.S. from the University of Notre Dame, MBA from Vanderbilt University Owen Graduate School of Management
Ines Macias-Perez
Cumberland Pharmaceuticals
Chief Medical Officer
Ph.D. in Cancer Biology from Vanderbilt University
Vanderbilt University Medical Center
Collaborator