Ambrisentan is an endothelin receptor antagonist used for the treatment of pulmonary hypertension (PH). Based on research suggesting a role for endothelin-1 in the pathogenesis of idiopathic pulmonary fibrosis (IPF) and the poor prognosis for patients with IPF who are also diagnosed with PH, this study was designed to evaluate the effectiveness and safety of ambrisentan in that patient population.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
40
Ambrisentan (5 mg or 10 mg tablet) administered orally once daily.
Placebo to match ambrisentan administered orally once daily.
Change From Baseline in Six-minute Walk Distance (6MWD).
The change from baseline in 6MWD at Week 16 (end of blinded treatment) was evaluated.
Time frame: Baseline to Week 16
Long-term Survival
Long-term survival was assessed as a Kaplan-Meier (KM) estimate of the percent probability of survival, with censoring at Week 48.
Time frame: Week 48
Transition Dyspnea Index (TDI)
The change in TDI at Week 16 (end of blinded treatment) was evaluated. TDI measures the change from the baseline characteristic "Baseline Dyspnea Index." The TDI range is -9 to +9 (worst to best; 0 = no change).
Time frame: Baseline to Week 16
Change From Baseline in WHO Functional Class
WHO functional class rates severity of pulmonary hypertension, with 4 categories on a scale of 1 to 4 with the worst category being 4. Change is represented as an increase ("+1: Improved"), decrease ("-1: Deteriorated"), or no change ("0: No change") on the scale.
Time frame: Baseline to Week 16
Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted
FVC is a pulmonary function test, and is defined as the volume of air that can forcibly be blown out after taking a full breath. FVC% predicted is defined as FVC% of the patient divided by the average FVC% in the population for any person of similar age, sex and body composition.
Time frame: Baseline to Week 16
Change From Baseline in N-terminal Pro-B-type Natriuretic Peptide (NT-proBNP)
Assessment of the the level of the amino acid fragment NT-proBNP is used to establish prognosis in cardiovascular disease.
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...and 75 more locations
Time frame: Baseline to Week 16
Change From Baseline in the Borg Dyspnea Index (BORG) Immediately Following Exercise
Borg Dyspnea Index is a measure of perceived shortness of breath: 0 units on a scale (none) to 10 units on a scale (maximum breathlessness).
Time frame: Baseline to Week 16
Hemoglobin-corrected Diffusing Capacity for Carbon Monoxide (DLCO) Percent Predicted
DLCO is a pulmonary function test, and measures the partial pressure difference between inspired and expired carbon monoxide. DLCO% predicted is defined as DLCO% of the patient divided by the average DLCO% in the population for any person of similar age, sex and body composition.
Time frame: Baseline to Week 16
Change in Quality of Life (QOL) Score as Assessed by the Short-Form 36® (SF-36)
Each SF-36 score is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. An increase in score indicates an improvement in health state.
Time frame: Baseline to Week 16
Change in QOL Score as Assessed by the St. George's Respiratory Questionnaire (SRGQ)
The SRGQ is designed to measure impact on overall health, daily life, and perceived well-being in patients with obstructive airways disease. Patients respond to questions about symptoms (frequency \& severity) and impact components (social functioning and psychological disturbances resulting from airways disease). Scores range from 0 to 100, with higher scores indicating more limitations.
Time frame: Baseline to Week 16