Trial Outcomes & Findings for Role of Sildenafil for Fontan Associated Liver Disease (SiFALD) Study (NCT NCT03154476)
NCT ID: NCT03154476
Last Updated: 2021-08-23
Results Overview
Measured using magnetic resonance imaging derived liver stiffness (MRE-LS) reported in kilopascal (kPa).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
20 participants
Primary outcome timeframe
Baseline, 12 months (52 weeks), 24 months (104 weeks)
Results posted on
2021-08-23
Participant Flow
Participant milestones
| Measure |
Sildenafil
Subjects randomized to this arm will receive sildenafil 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week, and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
Sildenafil: Sildenafil will be initiated at 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
|
Placebo
Subjects will receive placebo times per day for 12 months.
Placebo: Placebo capsules matching study drug
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
10
|
|
Overall Study
COMPLETED
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
4
|
4
|
Reasons for withdrawal
| Measure |
Sildenafil
Subjects randomized to this arm will receive sildenafil 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week, and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
Sildenafil: Sildenafil will be initiated at 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
|
Placebo
Subjects will receive placebo times per day for 12 months.
Placebo: Placebo capsules matching study drug
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
4
|
4
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Sildenafil
n=10 Participants
Subjects randomized to this arm will receive sildenafil 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week, and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
Sildenafil: Sildenafil will be initiated at 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
|
Placebo
n=10 Participants
Subjects will receive placebo times per day for 12 months.
Placebo: Placebo capsules matching study drug
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
28 years
STANDARD_DEVIATION 6 • n=10 Participants
|
29 years
STANDARD_DEVIATION 5 • n=10 Participants
|
29 years
STANDARD_DEVIATION 5 • n=20 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
6 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=10 Participants
|
7 Participants
n=10 Participants
|
14 Participants
n=20 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
United States
|
10 participants
n=10 Participants
|
10 participants
n=10 Participants
|
20 participants
n=20 Participants
|
PRIMARY outcome
Timeframe: Baseline, 12 months (52 weeks), 24 months (104 weeks)Measured using magnetic resonance imaging derived liver stiffness (MRE-LS) reported in kilopascal (kPa).
Outcome measures
| Measure |
Sildenafil
n=6 Participants
Subjects randomized to this arm will receive sildenafil 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week, and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
Sildenafil: Sildenafil will be initiated at 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week and 20 mg 3 times per day from the third week to the end of the study period, 12 months.
|
Placebo
n=6 Participants
Subjects will receive placebo times per day for 12 months.
Placebo: Placebo capsules matching study drug
|
|---|---|---|
|
Liver Stiffness as Measured by Magnetic Resonance Elastography (MRE)
Baseline
|
5.69 kPa
Standard Deviation 0.73
|
5.72 kPa
Standard Deviation 0.83
|
|
Liver Stiffness as Measured by Magnetic Resonance Elastography (MRE)
52 weeks
|
5.02 kPa
Standard Deviation 0.94
|
5.68 kPa
Standard Deviation 0.74
|
|
Liver Stiffness as Measured by Magnetic Resonance Elastography (MRE)
104 weeks
|
4.67 kPa
Standard Deviation 0.86
|
5.74 kPa
Standard Deviation 0.84
|
Adverse Events
Sildenafil
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Placebo
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