Tadalafil and Sildenafil for Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01359670
• Date of registration: 23/05/2011
• Prospective Registration: No
• Primary sponsor: Cedars-Sinai Medical Center
• Public title: Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
• Scientific title: Functional Muscle Ischemia and PDE5A Inhibition in Duchenne Muscular Dystrophy
• Date of first enrolment: May 2011
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01359670
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Early Phase 1

Countries of recruitment

United States

Contacts

• Name: Ronald Victor, MD
• Affiliation: Cedars-Sinai Medical Center

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. 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)



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

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Tadalafil

Drug: Sildenafil

Primary Outcome(s)

Functional muscle ischemia [Time Frame: For 5 study visits]

Secondary Outcome(s)

EKG Monitoring [Time Frame: 5 times over about 6 weeks]

6 Minute Walk Test [Time Frame: For 5 study visits]

Cardiac Function [Time Frame: For 5 study visits]

Physical Activity [Time Frame: 5 times over about 6-weeks]

Quality of Life [Time Frame: For 5 study visits]

Secondary ID(s)

PPMD

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Parent Project Muscular Dystrophy