Tadalafil as Adjuvant Therapy for DMD

Last updated: February 26, 2025
Sponsor: University of Florida
Overall Status: Active - Not Recruiting

Phase

2/3

Condition

Muscular Dystrophy

Treatment

Tadalafil

Clinical Study ID

NCT05195775
IRB202102391
OCR41391
  • Ages 7-13
  • Male

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Diagnosis of DMD confirmed by 1) clinical history with features before the age offive, 2) physical examination, 3) elevated serum creatine kinase level and 4)absence of dystrophin expression, as determined by immunostaining or Western blot (<2%) and/or DNA confirmation of dystrophin mutation.

  • Minimum entry age of 7.0 years

  • Ambulatory

Exclusion

Exclusion Criteria:

  • Older than 13.0 years of age

  • Contraindication to an MR examination (e.g. aneurysm clip, severe claustrophobia,magnetic implants)

  • Presence of unstable medical problems

  • Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g. myasthenia gravis, endocrine disorder, mitochondrial disease)

  • Contraindications to Tadalafil (use of nitrates, alpha-adrenergic blockers, otherPDE5A inhibitors)

Study Design

Total Participants: 25
Treatment Group(s): 1
Primary Treatment: Tadalafil
Phase: 2/3
Study Start date:
December 14, 2021
Estimated Completion Date:
September 05, 2025

Study 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.

Connect with a study center

  • University of Florida

    Gainesville, Florida 32610
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.