Status:
ACTIVE_NOT_RECRUITING
Tadalafil as Adjuvant Therapy for DMD
Lead Sponsor:
University of Florida
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
7-13 years
Phase:
PHASE2
PHASE3
Brief 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 techni...
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 extremi...
Eligibility Criteria
Inclusion
- Diagnosis of DMD confirmed by 1) clinical history with features before the age of five, 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
- 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, other PDE5A inhibitors)
Key Trial Info
Start Date :
December 14 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 5 2025
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT05195775
Start Date
December 14 2021
End Date
September 5 2025
Last Update
February 27 2025
Active Locations (1)
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1
University of Florida
Gainesville, Florida, United States, 32610