Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
Functional Muscle Ischemia and PDE5A Inhibition in Duchenne Muscular Dystrophy
1 other identifier
interventional
30
1 country
1
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for early_phase_1
Started May 2011
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2011
CompletedFirst Submitted
Initial submission to the registry
May 23, 2011
CompletedFirst Posted
Study publicly available on registry
May 25, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2013
CompletedJanuary 14, 2020
April 1, 2018
2 years
May 23, 2011
January 10, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Functional muscle ischemia
Measured by the decrease in muscle tissue oxygenation (near infrared spectroscopy) and blood flow (Doppler ultrasound) evoked by reflex sympathetic activation in exercising forearm muscle.
For 5 study visits
Secondary Outcomes (5)
Cardiac Function
For 5 study visits
EKG Monitoring
5 times over about 6 weeks
6 Minute Walk Test
For 5 study visits
Physical Activity
5 times over about 6-weeks
Quality of Life
For 5 study visits
Study Arms (2)
Tadalafil
EXPERIMENTALSildenafil
EXPERIMENTALInterventions
Escalating dose (0.25 mg/kg, 0.5 mg/kg, 1.0 mg/kg; once daily) over 2 weeks
Escalating dose (0.25 mg/kg, 0.5 mg/kg, 1.0 mg/kg; four times daily) over 2 weeks
Eligibility Criteria
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%
- Wheelchair bound
- Cardiac rhythm disorder, specifically: rhythm other than sinus, SVT, atrial fibrillation, ventricular tachycardia
- Continuous ventilatory support
- Liver disease
- Renal impairment
- Contraindications to tadalafil or sildenafil (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or other PDE5A inhibitors)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Cedars-Sinai Medical Centerlead
- Parent Project Muscular Dystrophycollaborator
Study Sites (1)
Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Ronald Victor, MD
Cedars-Sinai Medical Center
Study Design
- Study Type
- interventional
- Phase
- early phase 1
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 23, 2011
First Posted
May 25, 2011
Study Start
May 1, 2011
Primary Completion
May 1, 2013
Study Completion
May 1, 2013
Last Updated
January 14, 2020
Record last verified: 2018-04