Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy — DMD  

Cardiomyopathy, Dilated Clinical Trial

Official title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study With an Open-Label Extension to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy

Status Recruiting

Clinical Trial Summary

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

Clinical Trial Description

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 onto 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 evaluated for first-dose and steady-state exposure PK. All subjects who receive treatment will be assessed for safety. All subjects with at least one efficacy assessment post-baseline will be evaluated for efficacy. Blood and urine will be collected for standard and novel cardiac biomarkers. Target enrollment met for early-stage subjects (LVEF > 45%) and cohort is closed; enrollment remains open for the late-stage cohort (LVEF 35-45%). ;

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Dilated
• Duchenne Muscular Dystrophy Cardiomyopathy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

• NCT number: NCT03340675
• Study type: Interventional
• Source: Cumberland Pharmaceuticals
• Contact: Ines M Macias-Perez, PhD
• Phone: 6159795778
• Email: imaciasperez@cumberlandpharma.com
• Status: Recruiting
• Phase: Phase 2
• Start date: October 19, 2020
• Completion date: August 31, 2024