PDE Inhibitors in DMD Study (Acute Dosing Study)

Completed · Phase 1

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT01580501

Run by

Cedars-Sinai Medical Center

People needed

12

Starts

2012-03-01

Last updated by the study team

2014-01-28

Who can join

Age: 7 and older, up to 15. Sex: male. Healthy volunteers: accepted.

You may qualify if…

• diagnosis of DMD confirmed by muscle biopsy or DNA analysis
• age 7-15y
• ambulatory
• no clinical evidence of heart failure

You may not qualify if…

• hypertension, diabetes, or heart failure by standard clinical criteria
• elevated BNP level (>100 pg/ml)
• LVEF < 50%
• non-ambulatory
• cardiac rhythm disorder, specifically: rhythm other than sinus, SVT, atrial fibrillation, ventricular tachycardia
• continuous ventilatory support
• liver disease
• renal impairment
• contraindications to sildenafil (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or other PDE5A inhibitors)

Where it is running

• Cedars Sinai Medical Center — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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