Trial Outcomes & Findings for Blood Flow and Vascular Function in Cystic Fibrosis (NCT NCT02057458)
NCT ID: NCT02057458
Last Updated: 2020-04-24
Results Overview
FMD determined one hour after ingestion of 50 mg Sildenafil or placebo
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
pre-treatment Baseline and 1 hour post-treatment
Results posted on
2020-04-24
Participant Flow
Participant milestones
| Measure |
Acute Study: Sildenafil First, Then Placebo
In randomized order, on two separate days, endothelial function and exercise capacity will be determined 1 hour following a single dose of sildenafil (50 mg) or placebo.
|
Acute Study: Placebo First, Then Sildenafil
In randomized order, on two separate days, endothelial function and exercise capacity will be determined 1 hour following a single dose of sildenafil (50 mg) or placebo.
|
Sub-Chronic Only
participants who were unable to participate in the acute study due to distance from lab but participated in sub-chronic
|
|---|---|---|---|
|
Recruited for Participation
STARTED
|
8
|
8
|
3
|
|
Recruited for Participation
COMPLETED
|
8
|
8
|
3
|
|
Recruited for Participation
NOT COMPLETED
|
0
|
0
|
0
|
|
Met Inclusion Criteria
STARTED
|
8
|
8
|
3
|
|
Met Inclusion Criteria
COMPLETED
|
8
|
7
|
3
|
|
Met Inclusion Criteria
NOT COMPLETED
|
0
|
1
|
0
|
|
Acute Study (1 Hour): First Intervention
STARTED
|
8
|
7
|
0
|
|
Acute Study (1 Hour): First Intervention
COMPLETED
|
6
|
7
|
0
|
|
Acute Study (1 Hour): First Intervention
NOT COMPLETED
|
2
|
0
|
0
|
|
Acute Study (1 Hour): Second Int.
STARTED
|
6
|
7
|
0
|
|
Acute Study (1 Hour): Second Int.
COMPLETED
|
6
|
7
|
0
|
|
Acute Study (1 Hour): Second Int.
NOT COMPLETED
|
0
|
0
|
0
|
|
Sub-Chronic Study (4 Weeks)
STARTED
|
6
|
7
|
3
|
|
Sub-Chronic Study (4 Weeks)
COMPLETED
|
5
|
7
|
3
|
|
Sub-Chronic Study (4 Weeks)
NOT COMPLETED
|
1
|
0
|
0
|
Reasons for withdrawal
| Measure |
Acute Study: Sildenafil First, Then Placebo
In randomized order, on two separate days, endothelial function and exercise capacity will be determined 1 hour following a single dose of sildenafil (50 mg) or placebo.
|
Acute Study: Placebo First, Then Sildenafil
In randomized order, on two separate days, endothelial function and exercise capacity will be determined 1 hour following a single dose of sildenafil (50 mg) or placebo.
|
Sub-Chronic Only
participants who were unable to participate in the acute study due to distance from lab but participated in sub-chronic
|
|---|---|---|---|
|
Met Inclusion Criteria
Prior history of migraine headaches
|
0
|
1
|
0
|
|
Acute Study (1 Hour): First Intervention
Lost to Follow-up
|
2
|
0
|
0
|
|
Sub-Chronic Study (4 Weeks)
Lost to Follow-up
|
1
|
0
|
0
|
Baseline Characteristics
Blood Flow and Vascular Function in Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Overall Study
n=15 Participants
Baseline characteristics reported for the overall study
|
|---|---|
|
Age, Continuous
|
23 years
STANDARD_DEVIATION 11 • n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
|
Height
|
160 cm
STANDARD_DEVIATION 13 • n=5 Participants
|
|
Weight
|
54 kg
STANDARD_DEVIATION 17 • n=5 Participants
|
|
Body mass index
|
20.4 kg/m^2
STANDARD_DEVIATION 4.0 • n=5 Participants
|
|
Body fat
|
28.5 %
STANDARD_DEVIATION 7.9 • n=5 Participants
|
|
O2 saturation
|
98 %
STANDARD_DEVIATION 1 • n=5 Participants
|
|
Systolic blood pressure
|
108 mmHg
STANDARD_DEVIATION 11 • n=5 Participants
|
|
Diastolic blood pressure
|
65 mmHg
STANDARD_DEVIATION 7 • n=5 Participants
|
PRIMARY outcome
Timeframe: pre-treatment Baseline and 1 hour post-treatmentFMD determined one hour after ingestion of 50 mg Sildenafil or placebo
Outcome measures
| Measure |
Acute Study: Sildenafil
n=13 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=13 Participants
FMD % after acute placebo
|
Acute Study: Placebo
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
Acute Study: Percentage Flow-Mediated Dilation (FMD)
Pre
|
7.8 percent flow mediated dilation
Standard Deviation 4.3
|
7.7 percent flow mediated dilation
Standard Deviation 4.3
|
—
|
—
|
|
Acute Study: Percentage Flow-Mediated Dilation (FMD)
Post
|
7.3 percent flow mediated dilation
Standard Deviation 4.8
|
6.6 percent flow mediated dilation
Standard Deviation 3.7
|
—
|
—
|
PRIMARY outcome
Timeframe: pre-treatment Baseline and following 4 weeks sub-chronic treatmentBrachial Artery Diameter during FMD (pre-occlusion or "baseline")
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=15 Participants
FMD % after acute placebo
|
Acute Study: Placebo
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
Baseline Diameter
|
3.00 mm
Standard Deviation 0.59
|
3.06 mm
Standard Deviation 0.60
|
—
|
—
|
PRIMARY outcome
Timeframe: pre-treatment Baseline and following 4 weeks sub-chronic treatmentPeak Brachial Artery Diameter during FMD (post-occlusion)
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=15 Participants
FMD % after acute placebo
|
Acute Study: Placebo
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
Peak Diameter
|
3.22 mm
Standard Deviation 0.60
|
3.30 mm
Standard Deviation 0.60
|
—
|
—
|
PRIMARY outcome
Timeframe: pre-treatment Baseline and following 4 weeks sub-chronic treatmentAbsolute change in brachial artery diameter taken from the FMD assessment
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=15 Participants
FMD % after acute placebo
|
Acute Study: Placebo
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
Absolute Change in Diameter
|
0.21 mm
Standard Deviation 0.12
|
0.27 mm
Standard Deviation 0.09
|
—
|
—
|
PRIMARY outcome
Timeframe: pre-treatment Baseline, 1 hour post-treatment, and following 4 weeks sub-chronic treatmentForced Expiratory Volume in the first second expressed as a percent predicted.
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=13 Participants
FMD % after acute placebo
|
Acute Study: Placebo
n=13 Participants
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
n=15 Participants
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
FEV1 (% Predicted)
|
81 percent predicted
Standard Deviation 16
|
75 percent predicted
Standard Deviation 12
|
75 percent predicted
Standard Deviation 16
|
82 percent predicted
Standard Deviation 15
|
PRIMARY outcome
Timeframe: pre-treatment Baseline, 1 hour post-treatment, and following 4 weeks sub-chronic treatmentabsolute (L/min) peak oxygen consumption during maximal exercise test
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=13 Participants
FMD % after acute placebo
|
Acute Study: Placebo
n=13 Participants
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
n=15 Participants
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
VO2 Peak (Absolute)
|
1.5 L/min
Standard Deviation 0.4
|
1.7 L/min
Standard Deviation 0.5
|
1.6 L/min
Standard Deviation 0.5
|
1.6 L/min
Standard Deviation 0.5
|
PRIMARY outcome
Timeframe: pre-treatment Baseline, 1 hour post-treatment, and following 4 weeks sub-chronic treatmentrelative (mL/kg/min) peak oxygen consumption during maximal exercise test
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=13 Participants
FMD % after acute placebo
|
Acute Study: Placebo
n=13 Participants
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
n=15 Participants
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
VO2 Peak (Relative)
|
28.5 mL/kg/min
Standard Deviation 5.6
|
29.3 mL/kg/min
Standard Deviation 6.1
|
26.8 mL/kg/min
Standard Deviation 6.4
|
29.5 mL/kg/min
Standard Deviation 6.4
|
PRIMARY outcome
Timeframe: pre-treatment Baseline and 1 hour post-treatment, and 4 weeks sub-chronic treatmentMaximal Oxygen consumption expressed as percent predicted taken from maximal exercise test.
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=13 Participants
FMD % after acute placebo
|
Acute Study: Placebo
n=13 Participants
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
n=15 Participants
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
VO2 Peak (Percent Predicted)
|
72 percent predicted
Standard Deviation 12
|
77 percent predicted
Standard Deviation 13
|
72 percent predicted
Standard Deviation 13
|
75 percent predicted
Standard Deviation 12
|
PRIMARY outcome
Timeframe: pre-treatment Baseline, 1 hour post-treatment, and following 4 weeks sub-chronic treatmentpeak ventilation (L/min) during maximal exercise test
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=13 Participants
FMD % after acute placebo
|
Acute Study: Placebo
n=13 Participants
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
n=15 Participants
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
VE Peak
|
65 L/min
Standard Deviation 19
|
81 L/min
Standard Deviation 23
|
77 L/min
Standard Deviation 24
|
72 L/min
Standard Deviation 21
|
PRIMARY outcome
Timeframe: pre-treatment Baseline, 1 hour post-treatment, and following 4 weeks sub-chronic treatmentpeak respiratory exchange ratio during maximal exercise test
Outcome measures
| Measure |
Acute Study: Sildenafil
n=15 Participants
FMD % after acute sildenafil treatment
|
Acute Study: Placebo
n=13 Participants
FMD % after acute placebo
|
Acute Study: Placebo
n=13 Participants
FEV1 %predicted after acute placebo
|
Sub-chronic Study: Sildenafil
n=15 Participants
FEV1 %predicted after sub-chronic (4 weeks) sildenafil treatment
|
|---|---|---|---|---|
|
RER Peak
|
1.31 ratio
Standard Deviation 0.15
|
1.17 ratio
Standard Deviation 0.12
|
1.22 ratio
Standard Deviation 0.13
|
1.18 ratio
Standard Deviation 0.14
|
Adverse Events
Overall Study
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place