Phase 2 trial of oral ifetroban in DMD cardiomyopathy reports no safety results yet

Status: COMPLETED | Phase: PHASE2 Condition(s): Duchenne Muscular Dystrophy Cardiomyopathy, Cardiomyopathy, Dilated Intervention(s): Ifetroban (DRUG), Placebo (DRUG) 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 Detailed: This is a phase 2 randomized, double-blind, placebo-controlled, multiple dose study with an optional open-label extension to determine the safety, pharmacokinetics (PK) and efficacy of two doses of oral ifetroban in subjects with DMD. DMD patients who meet the inclusion criteria and none of the exclusion criteria will receive oral ifetroban or placebo once daily for 12 months. Subjects will be enrolled into one of three treatment groups, low-dose ifetroban, high-dose ifetroban or placebo. Each dose level will be evaluated by eight subjects with early stage (LVEF \> 45%) DMD-associated cardiomyopathy and eight subjects with more advanced stage (LVEF 35-45%) cardiac disease as there may be differences in the treatment effect based on cardiac involvement. Each subject treated will be evaluate Primary Outcome(s): Incidence of Treatment-Emergent Adverse Events Enrollment: 46 (ACTUAL) Lead Sponsor: Cumberland Pharmaceuticals Start: 2020-10-19 | Primary Completion: 2024-03-06 Results posted: 2026-01-26