Tadalafil and Sildenafil for Duchenne Muscular Dystrophy

Launched by CEDARS-SINAI MEDICAL CENTER · May 24, 2011

Keywords

ClinConnect Summary

Duchenne muscular dystrophy (DMD) is a rare, progressive and fatal muscle disease affecting boys and accounts for 80% of muscular dystrophy cases. Tadalafil and sildenafil are medications approved by the FDA for the treatment of erectile dysfunction and pulmonary hypertension. This class of medication improves muscle blood flow in a mouse model of muscular dystrophy, but their benefit to boys with DMD is unknown. The purpose of this study is to 1) determine if tadalafil or sildenafil can improve muscle blood flow during exercise in boys with DMD; and 2) to inform the design of a subsequent,...

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 1. Diagnosis of DMD confirmed by muscle biopsy or DNA analysis
• • 2. Age 7-15y
• • 3. Ambulatory
• • 4. No clinical evidence of heart failure
• Exclusion Criteria:
• • 1. Hypertension, diabetes, or heart failure by standard clinical criteria
• • 2. Elevated BNP level (\>100 pg/ml)
• • 3. LVEF \< 50%
• • 4. Wheelchair bound
• • 5. Cardiac rhythm disorder, specifically: rhythm other than sinus, SVT, atrial fibrillation, ventricular tachycardia
• • 6. Continuous ventilatory support
• • 7. Liver disease
• • 8. Renal impairment
• • 9. Contraindications to tadalafil or sildenafil (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or other PDE5A inhibitors)