This is a Phase II randomized, double-blind, placebo-controlled trial of sildenafil in men and women with Scleroderma with mildly elevated pulmonary pressures (SSc-MEP) to determine whether sildenafil may be an effective treatment for SSc-MEP.
Data shows that sildenafil (SIL) is an effective therapy in SSc-PAH. SIL has been safely used in many patients with various vascular and cardiovascular diseases over the past three decades. Randomized controlled trial data for SIL shows improvement in 6MWD, hemodynamics, and even evidence of cardiac remodeling in PAH and SSc-PAH patients. Based upon these data, SIL may be an effective therapy in SSc-MEP. This study will help determine whether sildenafil could be a good treatment for patients with scleroderma that have mildly elevated pulmonary pressures.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
30
Sildenafil 20 mg three times a day. This is the approved dose for the treatment of pulmonary arterial hypertension. It is being studied in this trial with a population who has mildly elevated pulmonary pressures.
Oral pill placebo.
Louisiana State University
New Orleans, Louisiana, United States
NOT_YET_RECRUITINGJohns Hopkins
Baltimore, Maryland, United States
RECRUITINGDifference in change in distance walked in 6 minute walk test (6MWT) at 4 months
As assessed by change in 6 minute walk distance (6MWD) in feet (from baseline to 4 months) between the sildenafil group and the placebo group.
Time frame: Baseline and 4 months
Difference in change in distance walked in 6MWT at 12 months
As assessed by change in 6 minute walk test distance in feet (from baseline to 12 months) between the sildenafil group and the placebo group.
Time frame: Baseline and 12 months
Difference in change in right ventricular function as assessed by cardiac MRI
As assessed by change in right ventricular function (normal/abnormal) on cardiac MRI.
Time frame: Baseline and 4 months
Difference in change in right ventricular function as assessed by cardiac MRI
As assessed by change in right ventricular function (normal/abnormal) on cardiac MRI.
Time frame: Baseline and 12 months
Difference in change in right ventricular function as assessed by invasive hemodynamics
As assessed by change in right ventricular function (normal/abnormal) on invasive hemodynamics.
Time frame: Baseline and 4 months
Difference in change in right ventricular function as assessed by invasive hemodynamics
As assessed by change in right ventricular function (normal/abnormal) on invasive hemodynamics.
Time frame: Baseline and 12 months
Difference in change in right ventricular function as assessed by echocardiography
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As assessed by change in right ventricular function (normal/abnormal) on echocardiography.
Time frame: Baseline and 4 months
Difference in change in right ventricular function as assessed by echocardiography
As assessed by change in right ventricular function (normal/abnormal) on echocardiography.
Time frame: Baseline and 12 months
Difference in change in N-terminal pro b-type natriuretic peptide level
As assessed by changes in plasma N-terminal pro b-type natriuretic peptide (NT-proBNP) level (pg/mL) between sildenafil and placebo groups at four months and twelve months.
Time frame: Baseline, 4 months and 12 months
Difference in change in health related quality of life as assessed by the 36-Item Short Form Health Survey (SF36) questionnaire
As assessed by changes in SF36 questionnaire between sildenafil and placebo groups at four months and twelve months. The SF36 yields a set of scaled scores in 8 domains, with higher numbers representing a better quality of life. Each scale is scored on a 0 to 100 range so that the lowest and highest possible scores are 0 and 100, respectively. Scores represent the percentage of total possible score achieved. In step 2, items in the same scale are averaged together to create the 8 scale scores. Items that are left blank (missing data) are not taken into account when calculating the scale scores. Hence, scale scores represent the average for all items in the scale that the respondent answered.
Time frame: Baseline, 4 months and 12 months
Difference in change in health related quality of life as assessed by the emPHasis-10 health-related quality of life questionnaire (emPHasis-10).
As assessed by changes in emPHasis-10 questionnaire between sildenafil and placebo groups. The Emphasis 10 score consists of 10 questions scored in a semantic 6-point scale (from 0 to 5), for a total maximum score of 50 (the higher the score, the worse the quality of life).
Time frame: Baseline, 4 months and 12 months
Difference in safety profile as assessed by frequency of adverse events
As assessed by frequency of adverse events between sildenafil and placebo groups.
Time frame: Ongoing until study closes, up to 4 years
Difference in safety profile as assessed by severity of adverse events
As assessed by severity of adverse events between sildenafil and placebo groups.
Time frame: Ongoing until study closes, up to 4 years