Tadalafil in Becker Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01070511
• Date of registration: 17/02/2010
• Prospective Registration: No
• Primary sponsor: Cedars-Sinai Medical Center
• Public title: Tadalafil in Becker Muscular Dystrophy
• Scientific title: Functional Muscle Ischemia and PDE5A Inhibition in Becker Muscular Dystrophy
• Date of first enrolment: January 2010
• Target sample size: 48
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01070511
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Basic Science
• Phase: Phase 4

Countries of recruitment

United States

Contacts

• Name: Ronald G Victor, MD
• Affiliation: Cedars-Sinai Medical Center

Key inclusion & exclusion criteria

Becker Muscular Dystrophy Patients

- Men 18-55 years of age with a pre-existing diagnosis of Becker Muscular Dystrophy by
a clinical neurologist (based on clinical criteria plus previous muscle biopsy
analysis and/or DNA analysis).

Healthy Controls

- Men 18-55 years of age with no known medical conditions

Criteria for exclusion of subjects (both patients and controls)

- Any evidence of cardiopulmonary disease by history or by physical examination

- History of hypertension or blood pressure averaging =140/90 mmHg

- Diabetes mellitus or other systemic illness

- Heart failure by clinical exam, elevated BNP, or heart failure medication

- Serum creatinine = 1.5 mg/dL

- Any history of substance abuse (including alcohol)

- Any history of psychiatric illness

- Contraindications to tadalafil (use of nitrates, alpha-blockers, other PDE5A
inhibitors, or potent inhibitors of CYP3A4 such as ketoconazole or ritonavir)

- Contraindications to MRI (claustrophobia, metal implants, or seizure disorder)



Age minimum: 18 Years
Age maximum: 55 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Becker Muscular Dystrophy

Intervention(s)

Drug: Tadalafil

Drug: Placebo

Primary Outcome(s)

Reflex decrease in muscle tissue oxygenation (i.e., adrenergic vasoconstriction) during rhythmic handgrip exercise measured by Near Infrared Spectroscopy (NIR). [Time Frame: measured at a minimum of 2 week intervals for a minimum of 6 weeks total (for subjects with BMD)]

Secondary Outcome(s)

Change in forearm muscle water content by magnetic resonance imaging (MRI). [Time Frame: measured at a minimum of 2 week intervals for a minimum of 6 weeks total (for subjects with BMD)]

Secondary ID(s)

MDA 158944

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Muscular Dystrophy Association