Vasodilator and Exercise Study for DMD (VASO-REx)
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.
Conditions:
🦠 Duchenne Muscular Dystrophy 🦠 Duchenne Disease 🦠 Muscular Dystrophy 🦠 Muscular Dystrophy in Children 🦠 Vasodilation 🦠 Exercise 🦠 DMD
🗓️ Study Start (Actual) 5 June 2024
🗓️ Primary Completion (Estimated) November 2026
✅ Study Completion (Estimated) November 2026
👥 Enrollment (Estimated) 50
🔬 Study Type INTERVENTIONAL
📊 Phase PHASE2
Locations:
📍 Gainesville, Florida, United States

📋 Eligibility Criteria

Description

    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
Ages Eligible for Study: 6 Years to N/A (CHILD, ADULT, OLDER_ADULT)
Sexes Eligible for Study: MALE
Accepts Healthy Volunteers: No

🗓️ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 9 February 2024
  • First Submitted that Met QC Criteria 1 March 2024
  • First Posted 4 March 2024

Study Record Updates

  • Last Update Submitted that Met QC Criteria 21 June 2024
  • Last Update Posted 25 June 2024
  • Last Verified June 2024