PDE Inhibitors in DMD Study (Acute Dosing Study)

• Status: Completed
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: Male

Summary

PDE5A inhibition, which boosts NO-cGMP signaling, will relieve functional muscle ischemia and restore normal blood flow regulation (i.e., functional sympatholysis) during exercise in boys with DMD. The investigators specific aim is to perform an efficient dose-titration study to inform the design of a randomized multicenter trial of PDE5A inhibition for clinical skeletal muscle and cardiac endpoints.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Cedars-Sinai Medical Center
• 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. non-ambulatory

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 sildenafil (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or other PDE5A inhibitors)