Combining exercise and tadalafil for treating Duchenne muscular dystrophy
Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)
This study is testing if combining exercise with a medication called tadalafil can help boys with Duchenne muscular dystrophy improve their muscle function and blood flow.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 6 Years and up |
| Sex | Male |
| Sponsor | University of Florida Academic / other |
| Locations | 1 site (Gainesville, Florida) |
| Trial ID | NCT06290713 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effectiveness of two potential therapies for boys with Duchenne muscular dystrophy (DMD): aerobic exercise training and tadalafil, an FDA-approved vasodilator. The study aims to improve muscle function and vascular health by enhancing blood flow and muscle perfusion, which are often compromised in DMD. Participants will first be assessed for drug responsiveness and then randomized to receive either tadalafil or a placebo alongside structured exercise training over six months. The trial is designed to provide insights into how these interventions can work together to benefit patients.
Who should consider this trial
Good fit: Ideal candidates are ambulatory boys aged 6 years and older with a confirmed diagnosis of Duchenne muscular dystrophy.
Not a fit: Patients with severe cardiomyopathy, unstable medical conditions, or secondary conditions affecting muscle function may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly enhance muscle function and quality of life for patients with Duchenne muscular dystrophy.
How similar studies have performed: While the combination of exercise and vasodilators is a novel approach, previous studies have shown promise in improving muscle perfusion and function in similar patient populations.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Gainesville, Florida
- University of Florida Clinical and Translational Research Building — Gainesville, Florida, United States (Recruiting)
Study contacts
- Principal investigator: Tanja Taivassalo, Ph.D. — University of Florida, College of Medicine, Department of Physiology and Aging
- Study coordinator: Tanja Taivassalo, Ph.D.
- Email: ttaivassalo@ufl.edu
- Phone: 352-294-8748
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.