Trial Outcomes & Findings for Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (NCT NCT01168908)
NCT ID: NCT01168908
Last Updated: 2019-02-26
Results Overview
To determine whether a 6 month trial of oral sildenafil compared to placebo improves cardiac contractile function in DBMD as determined by a \> 10% decline in end-systolic volume as detected by CMR.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
6 months compared to baseline
Results posted on
2019-02-26
Participant Flow
Participant milestones
| Measure |
Revatio (Sildenafil)
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Double Blind, Placebo Controlled
STARTED
|
10
|
10
|
|
Double Blind, Placebo Controlled
COMPLETED
|
7
|
8
|
|
Double Blind, Placebo Controlled
NOT COMPLETED
|
3
|
2
|
|
Open Label
STARTED
|
7
|
8
|
|
Open Label
COMPLETED
|
6
|
7
|
|
Open Label
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
Revatio (Sildenafil)
n=10 Participants
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=10 Participants
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
25.5 years
STANDARD_DEVIATION 8.2 • n=5 Participants
|
22.6 years
STANDARD_DEVIATION 4.4 • n=7 Participants
|
24.05 years
STANDARD_DEVIATION 6.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 months compared to baselineTo determine whether a 6 month trial of oral sildenafil compared to placebo improves cardiac contractile function in DBMD as determined by a \> 10% decline in end-systolic volume as detected by CMR.
Outcome measures
| Measure |
Revatio (Sildenafil)
n=7 Participants
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=8 Participants
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Change in Cardiac Left Ventricular End-systolic Volume (LVESV) by Cardiac Magnetic Resonance (CMR) Imaging.
|
5.2 ml
Standard Deviation 23.61
|
-0.19 ml
Standard Deviation 5.56
|
SECONDARY outcome
Timeframe: 6 months and 12 monthsCardiac volumes and systolic ejection parameters will be measured.
Outcome measures
| Measure |
Revatio (Sildenafil)
n=7 Participants
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=8 Participants
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Change in Cardiac Systolic and Diastolic Function by CMR
LV end-diastolic volume
|
5.54 ml
Standard Deviation 18.85
|
0.24 ml
Standard Deviation 8.78
|
|
Change in Cardiac Systolic and Diastolic Function by CMR
stroke volume
|
0.34 ml
Standard Deviation 9.75
|
0.43 ml
Standard Deviation 11.46
|
SECONDARY outcome
Timeframe: 6 months and 12 monthsLeft ventricular (LV) mass will be measured by CMR .
Outcome measures
| Measure |
Revatio (Sildenafil)
n=7 Participants
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=8 Participants
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Change in Cardiac Mass
|
-3.4 grams
Standard Deviation 7.03
|
-1.05 grams
Standard Deviation 12.69
|
SECONDARY outcome
Timeframe: 6 months and 12 monthsSkeletal muscle function of the diaphragm will be measured using FVC by pulmonary function testing.
Outcome measures
| Measure |
Revatio (Sildenafil)
n=7 Participants
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=8 Participants
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Change in Forced Vital Capacity (FVC) by Pulmonary Function Testing
|
-0.13 liters
Standard Deviation 0.09
|
-0.19 liters
Standard Deviation 0.21
|
SECONDARY outcome
Timeframe: 6 months and 12 monthsSkeletal muscle strength will be assessed by pincher and grip dynamometry
Outcome measures
| Measure |
Revatio (Sildenafil)
n=7 Participants
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=8 Participants
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Change in Skeletal Muscle Strength
pinch strength
|
-0.06 pounds
Standard Deviation 0.30
|
0.12 pounds
Standard Deviation 0.45
|
|
Change in Skeletal Muscle Strength
grip strength
|
0.37 pounds
Standard Deviation 0.90
|
0.17 pounds
Standard Deviation 1.25
|
SECONDARY outcome
Timeframe: 6 monthsLeft ventricular ejection fraction by cardiac MRI was measured
Outcome measures
| Measure |
Revatio (Sildenafil)
n=7 Participants
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=8 Participants
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Ejection Fraction
|
43.25 percentage of volume
Standard Deviation 12.82
|
45.64 percentage of volume
Standard Deviation 6.82
|
Adverse Events
Revatio (Sildenafil)
Serious events: 5 serious events
Other events: 13 other events
Deaths: 1 deaths
Placebo
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Revatio (Sildenafil)
n=18 participants at risk
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=10 participants at risk
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
Cardiac disorders
Death from heart failure
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
|
Metabolism and nutrition disorders
dehydration
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
|
Respiratory, thoracic and mediastinal disorders
pneumonia
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
|
Infections and infestations
Prostate infection
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
|
Gastrointestinal disorders
fecal impaction
|
0.00%
0/18
|
10.0%
1/10 • Number of events 1
|
|
Gastrointestinal disorders
Gastroparesis
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
Other adverse events
| Measure |
Revatio (Sildenafil)
n=18 participants at risk
This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
Placebo
n=10 participants at risk
This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.
Sildenafil: 20mg tablet three times daily
|
|---|---|---|
|
General disorders
Dizziness
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
|
Gastrointestinal disorders
gastroparesis
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
|
Nervous system disorders
Headache
|
5.6%
1/18 • Number of events 1
|
20.0%
2/10 • Number of events 2
|
|
Cardiac disorders
tachycardia
|
5.6%
1/18 • Number of events 1
|
0.00%
0/10
|
|
Cardiac disorders
low blood pressure
|
22.2%
4/18 • Number of events 4
|
0.00%
0/10
|
|
General disorders
flushing
|
5.6%
1/18 • Number of events 1
|
10.0%
1/10 • Number of events 1
|
|
Gastrointestinal disorders
gastroenteritis
|
11.1%
2/18 • Number of events 2
|
10.0%
1/10 • Number of events 1
|
|
General disorders
tingling sensation
|
11.1%
2/18 • Number of events 2
|
0.00%
0/10
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/18
|
20.0%
2/10 • Number of events 2
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/18
|
10.0%
1/10 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place