NCT05195775

Tadalafil as Adjuvant Therapy for DMD

Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 25 (estimated)
• Sponsor: University of Florida · Academic / Other
• Sex: Male
• Age: 7 Years – 13 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This project will target up to 25 ambulatory boys with DMD aged 7-13 years. Because the vascular impact of tadalafil is immediate, we will confirm that the drug target is valid target in lower extremities by assessing the change in post-exercise microvascular perfusion using Blood Oxygen Level-Dependent (BOLD) MRI or change in post-exercise hyperemia using Doppler ultrasonography. We will also assess the functional impact of the drug through exercise testing. Patients will be randomized to undergo the above-mentioned assessments with or without the study drug on two separate visits. Time function tests, MRI-based fat fraction and inflammation measurements will also be obtained in all patients, thus characterizing disease severity and provide a sampling of information on whether a subset of patients do not respond to the drug. This information may be used to inform future trials as to the appropriate target population for PDE5i as well as account for potential failures in a previously published phase 3 clinical trial.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	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.
DRUG	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.

Timeline

• Start date: 2021-12-14
• Primary completion: 2025-09-05
• Completion: 2025-09-05
• First posted: 2022-01-19
• Last updated: 2025-02-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study