Combining exercise and tadalafil for treating Duchenne muscular dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.