Study of Tadalafil and Home-Based Aerobic Exercise as Additional Treatments for Boys with Duchenne Muscular Dystrophy Phase 2 Trial Testing Vascular and Muscle Benefits

What this Trial Is About

This research aims to evaluate two potential additional therapies for boys with Duchenne muscular dystrophy DMD aged 6 years and older. The study focuses on aerobic exercise training and tadalafil, an FDA-approved drug that dilates blood vessels, to improve muscle function and blood flow. Researchers want to see if combining exercise with tadalafil can better address muscle deterioration and blood vessel problems common in DMD. Participants will first take a single dose of tadalafil to test how their muscles respond in terms of oxygen levels. Those who respond will be randomly assigned to either a daily tadalafil treatment or a placebo for six months. Both groups will also take part in structured cycling exercise sessions at home up to four times a week, using provided exercise equipment. The research team will monitor exercise through live video and heart rate checks. Throughout the study, participants will undergo various assessments including muscle health scans, exercise tests, and questionnaires to measure muscle fat, fatigue, lung function, physical activity, and quality of life. The main measurements include muscle blood vessel response after exercise and cycling endurance. The study also includes long-term follow-up over about three years to track ongoing effects and safety. Total participation involves medication, exercise, and regular evaluations.

Vasodilator and Exercise Study for DMD (VASO-REx)