PDE Inhibitors in DMD Study (Acute Dosing Study)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01580501
• Date of registration: 17/04/2012
• Prospective Registration: No
• Primary sponsor: Cedars-Sinai Medical Center
• Public title: PDE Inhibitors in DMD Study (Acute Dosing Study)
• Scientific title: Functional Muscle Ischemia and PDE5 Inhibition in Duchenne Muscular Dystrophy: Acute Dosing Study
• Date of first enrolment: March 2012
• Target sample size: 12
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01580501
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion 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)



Age minimum: 7 Years
Age maximum: 15 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Tadalafil and Sildenafil

Primary Outcome(s)

Pre vs. post treatment change in functional sympatholysis by NIR for each dose of each drug. [Time Frame: No]

Secondary Outcome(s)

Sympatholysis measured by brachial artery Doppler ultrasound [Time Frame: No]

Secondary ID(s)

PRO 27521

Source(s) of Monetary Support

Please refer to primary and secondary sponsors