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Tadalafil as Adjuvant Therapy for DMD
Sponsor: University of Florida
Summary
This project will assess the vascular responsiveness in leg muscles of boys with Duchenne muscular dystrophy (DMD) to one single dose of tadalafil, a common vasodilator drug, using non-invasive techniques (MRI or Doppler ultrasound) and exercise testing. These findings will provide proof of concept for a subsequent intervention study to demonstrate efficacy of longer-term tadalafil to counter sympathetic vasoconstriction and slow disease progression in DMD. It will also inform whether a group of patients do not respond to the drug.
Official title: Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy
Key Details
Gender
MALE
Age Range
7 Years - 13 Years
Study Type
INTERVENTIONAL
Enrollment
25
Start Date
2021-12-14
Completion Date
2025-09-05
Last Updated
2025-02-27
Healthy Volunteers
No
Conditions
Interventions
Tadalafil
Patient will be randomized to one dose of 0.6 mg/kg tadalafill on one of two study visits. On both visits, assessments (MRI, exercise testing, clinical function) will be performed 3 hours after time of dosing.
Tadalafil
Patient will be randomized to one dose of 0.6 mg/kg tadalafill on one of two study visits. On both visits, assessments (Doppler US, exercise testing, clinical function) will be performed 3 hours after time of dosing.
Locations (1)
University of Florida
Gainesville, Florida, United States