Oral Ifetroban for Duchenne Muscular Dystrophy (DMD Trial)

Palo Alto (17 mi)

Overseen byLarry Markham, MD

Age: Any Age

Sex: Male

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Cumberland Pharmaceuticals

Prior Safety Data

Trial Summary

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

Eligibility Criteria

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

I am a male, 7 or older, diagnosed with DMD based on symptoms, genetic tests, or muscle biopsy.

I haven't taken corticosteroids for the last 30 days or have been on a stable dose for 8 weeks.

Exclusion Criteria

I do not have any major illnesses besides DMD.

I haven't had major surgery in the last 6 weeks and don't plan any that would affect this study.

I am allergic to gadolinium contrast or have kidney problems.

Treatment Details

The study tests Ifetroban, a potential new treatment for the cardiomyopathy associated with DMD. It's compared against a placebo in boys and men who meet strict health criteria. The goal is to find out if Ifetroban can help improve cardiac outcomes in DMD patients.

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Oral Ifetroban - Low DoseExperimental Treatment1 Intervention

Weight based, once daily oral ifetroban

Group II: Oral Ifetroban - High DoseExperimental Treatment1 Intervention

Weight based, once daily oral ifetroban

Group III: PlacebosPlacebo Group1 Intervention

Matching Placebo