Clinical trial

Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy

NCT05195775

Name

IRB202102391

Description

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.

Trial arms

Trial start

2021-12-14

Estimated PCD

2024-10-20

Trial end

2025-01-20

Status

Recruiting

Phase

Early phase I

Treatment

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.

Arms:

Tadalafil plus BOLD-MRI (Schedule A)

Other names:

Cialis

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.

Arms:

Tadalafil plus Doppler ultrasound (Schedule B)

Other names:

Cialis

Size

Primary endpoint

Change in post-contractile BOLD response after tadalafil dosing

MRI will be done 3 hours after dosing/no-dosing on two separate study visits

Change in post-exercise hyperemia after tadalafil dosing

Doppler ultrasound will be done 3 hours after dosing/no-dosing on two separate study visits

Eligibility criteria

Inclusion Criteria: * 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 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, other PDE5A inhibitors)

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2', 'PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Pre/post single dose tadalafil', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 25, 'type': 'ESTIMATED'}}

Updated at

2024-01-31

1 organization

1 product

1 indication

Organization

University of Florida

Product

Tadalafil

Indication

Duchenne muscular dystrophy