Tadalafil and Sildenafil for Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2013-05-01
• Target enrollment: 0
• Participant gender: Male

Summary

This study, supported by Parent Project Muscular Dystrophy, will determine if tadalafil or sildenafil can improve muscle blood flow during exercise in boys with Duchenne muscular dystrophy.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cedars-Sinai Medical Center
• Collaborator: Parent Project Muscular Dystrophy
• Treatments: Sildenafil Citrate; Tadalafil

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)