RECRUITINGPhase 2INTERVENTIONAL

Vasodilator and Exercise Study for DMD (VASO-REx)

Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)

About This Trial

Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment.

Who May Be Eligible (Plain English)

Who May Qualify: - Diagnosis of DMD confirmed by genetic report - Minimum entry age of 6.0 years old - Ambulatory - On stable glucocorticoid regimen (for \> 3 months) Who Should NOT Join This Trial: - Contraindication to a Magnetic resonance Imaging examination (e.g. severe claustrophobia, magnetic implants, unable/unwilling to perform test) - Presence of unstable medical problems, including severe cardiomyopathy, left ventricular ejection fraction \<45%, cardiac conduction abnormalities as evidenced on ECG, uncontrolled seizure disorder, uncontrolled hypo or hypertension - Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g., myasthenia gravis, endocrine disorder, mitochondrial disease) - Presence of a secondary condition leading to developmental delay or impaired motor control (e.g., cerebral palsy) or previous history of unprovoked rhabdomyolysis - Contraindications to phosphodiesterase 5 inhibitors (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5 inhibitors) or other medications known to modulate blood flow or muscle metabolism - Participation in currently approved FDA trials or other investigational clinical trials during the period of the study Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * Diagnosis of DMD confirmed by genetic report * Minimum entry age of 6.0 years old * Ambulatory * On stable glucocorticoid regimen (for \> 3 months) Exclusion Criteria: * Contraindication to a Magnetic resonance Imaging examination (e.g. severe claustrophobia, magnetic implants, unable/unwilling to perform test) * Presence of unstable medical problems, including severe cardiomyopathy, left ventricular ejection fraction \<45%, cardiac conduction abnormalities as evidenced on ECG, uncontrolled seizure disorder, uncontrolled hypo or hypertension * Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g., myasthenia gravis, endocrine disorder, mitochondrial disease) * Presence of a secondary condition leading to developmental delay or impaired motor control (e.g., cerebral palsy) or previous history of unprovoked rhabdomyolysis * Contraindications to phosphodiesterase 5 inhibitors (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5 inhibitors) or other medications known to modulate blood flow or muscle metabolism * Participation in currently approved FDA trials or other investigational clinical trials during the period of the study

Treatments Being Tested

DRUG

Tadalafil

Patients will be given either the intervention medication or a placebo (double-blinded, randomized trial) and will be asked to take the medication every day for 6 months in conjunction with weekly exercise sessions.

DRUG

Placebo

OTHER

Exercise Training

The home-based cycling exercise training program is designed to improve muscle strength and endurance. Participants will engage in individualized exercise sessions up to four times per week, lasting up to 40 minutes each. Live video and heart rate monitoring will ensure proper exercise performance and allow for adjustments to the program throughout the study. The participants will receive the exercise equipment for use at home.

Locations (1)

University of Florida Clinical and Translational Research Building

Gainesville, Florida, United States