Actively Recruiting

Phase 2
Age: 6Years +
MALE
ID06290713

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

Led by University of Florida · Updated on 2026-05-15

50

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

Sponsors

U

University of Florida

Lead Sponsor

N

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Collaborating Sponsor

AI-Summary

What this Trial Is About

This research aims to evaluate two potential additional therapies for boys with Duchenne muscular dystrophy (DMD) aged 6 years and older. The study focuses on aerobic exercise training and tadalafil, an FDA-approved drug that dilates blood vessels, to improve muscle function and blood flow. Researchers want to see if combining exercise with tadalafil can better address muscle deterioration and blood vessel problems common in DMD. Participants will first take a single dose of tadalafil to test how their muscles respond in terms of oxygen levels. Those who respond will be randomly assigned to either a daily tadalafil treatment or a placebo for six months. Both groups will also take part in structured cycling exercise sessions at home up to four times a week, using provided exercise equipment. The research team will monitor exercise through live video and heart rate checks. Throughout the study, participants will undergo various assessments including muscle health scans, exercise tests, and questionnaires to measure muscle fat, fatigue, lung function, physical activity, and quality of life. The main measurements include muscle blood vessel response after exercise and cycling endurance. The study also includes long-term follow-up over about three years to track ongoing effects and safety. Total participation involves medication, exercise, and regular evaluations.

CONDITIONS

Brief Title

Vasodilator and Exercise Study for DMD (VASO-REx)

Who Can Participate

Age: 6Years +
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of Duchenne muscular dystrophy confirmed by genetic report
  • Minimum entry age of 6 years old
  • Ambulatory (able to walk)
  • On stable glucocorticoid regimen for more than 3 months
Not Eligible

You will not qualify if you...

  • Contraindication to MRI (e.g., severe claustrophobia, magnetic implants, unable or unwilling to perform test)
  • Unstable medical problems including severe cardiomyopathy or left ventricular ejection fraction below 45%
  • Cardiac conduction abnormalities shown on ECG
  • Uncontrolled seizure disorder
  • Uncontrolled low or high blood pressure
  • Secondary condition affecting muscle function or metabolism (e.g., myasthenia gravis, endocrine disorder, mitochondrial disease)
  • Secondary condition causing developmental delay or impaired motor control (e.g., cerebral palsy) or history of unprovoked rhabdomyolysis
  • Contraindications to phosphodiesterase 5 inhibitors or medications affecting blood flow or muscle metabolism
  • Participation in other FDA-approved or investigational clinical trials during this study period

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Run-in Period

Duration - Up to 4 weeks

Participants receive a single dose of tadalafil to test for drug responsiveness by measuring muscle oxygenation after muscle contraction.

1 to 2 visits for drug responsiveness testing

Treatment

Duration - 6 months (26 weeks)

Participants take daily tadalafil or placebo for 6 months combined with structured home-based cycling exercise training up to four times per week with live video and heart rate monitoring.

Weekly exercise sessions with ongoing remote monitoring

Follow-up

Duration - 6 months

Participants attend follow-up visits to assess cycling performance, muscle health, and other health measures up to 6 months after treatment.

Baseline and 6-month follow-up visits

Trial Site Locations

Total: 1 location

1

University of Florida Clinical and Translational Research Building

Gainesville, Florida, United States, 32603

Actively Recruiting

Loading map...

Research Team

T

Tanja Taivassalo, Ph.D.

R

Ruby Sullivan, MS

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

Similar Trials

The SPARK Trial: A Digital Exercise Intervention for Patient...

Spondyloarthritis

Actively Recruiting

1 location

CP-EXCEL: A Feasibility Randomised Controlled Trial of an On...

Cerebral Palsy

Actively Recruiting

1 location

Frequently Asked Questions

Have more questions? Get in touch with our team for quick support

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here

Published Research Related To This Trial

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.

Jerry R Mendell, Zarife Sahenk, Kelly Lehman...

https://pubmed.ncbi.nlm.nih.gov/32539076

Evaluation of the therapeutic utility of phosphodiesterase 5A inhibition in the mdx mouse model of duchenne muscular dystrophy.

Justin M Percival, Candace M Adamo, Joseph A Beavo...

https://pubmed.ncbi.nlm.nih.gov/21695647