Vasodilator and Exercise Study for DMD (VASO-REx)

Launched by UNIVERSITY OF FLORIDA · Mar 1, 2024

Keywords

ClinConnect Summary

The Vasodilator and Exercise Study for Duchenne Muscular Dystrophy, also known as VASO-REx, is a clinical trial looking at two approaches to help improve health in boys with Duchenne muscular dystrophy (DMD). The study is exploring the effects of aerobic exercise training, which aims to strengthen muscles and improve heart health, alongside a medication called tadalafil. Tadalafil is already approved by the FDA and helps enhance blood flow, which can be a challenge for those with DMD. The goal of the trial is to see if these strategies can lead to better muscle function and overall health for patients.

To take part in this study, boys aged 6 and older who have been diagnosed with DMD and can walk independently are eligible, as long as they have been on a stable glucocorticoid treatment for at least three months. Participants will undergo assessments to monitor their health and response to the exercise and medication. It’s important to note that those with certain medical conditions or who are taking specific medications may not be able to join the study. This trial is currently recruiting participants, so it could be a great opportunity for eligible boys to contribute to research that may benefit future DMD treatments.

Gender

MALE

Eligibility 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