This is a multicenter, randomized (2:1; oral treprostinil:placebo), double-blind, placebo-controlled event-driven (time to pulmonary hypertension \[PH\] clinical worsening) study in subjects with PH associated with sickle cell disease (SCD). Once enrolled, subjects will be evaluated at Weeks 6, 12, 24, and then every 12 weeks for the duration of the study. Subjects will be permitted to enter a 48-week open-label extension period if they experience a PH clinical worsening event.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Extended-release oral tablet for TID administration
Matching placebo for TID administration
Effect of oral treprostinil compared with placebo on time to first adjudicated PH clinical worsening (morbidity or mortality) event in subjects with PH associated with SCD
Clinical worsening is defined as the occurrence of any 1 of the following events: hospitalization related to PH and/or right heart failure, initiation of an infused prostacyclin to treat worsening PH, decrease in 6-Minute Walk Distance (6MWD) \>15% from Baseline directly related to disease under study.
Time frame: Baseline until the first adjudicated PH clinical worsening event, assessed up to approximately 4 years
Effect of oral treprostinil compared with placebo on exercise capacity as assessed by 6-Minute Walk Distance (6MWD)
Time frame: Baseline to Week 24
Effect of oral treprostinil compared with placebo on combined 6MWD/Borg dyspnea score
Time frame: Baseline to Week 24
Effect of oral treprostinil compared with placebo on Borg dyspnea score
Time frame: Baseline to Week 24
Effect of oral treprostinil compared with placebo on N-Terminal pro-brain natriuretic peptide (NT-proBNP) levels
Time frame: Baseline to Week 24
Effect of oral treprostinil compared with placebo on maximal tricuspid regurgitant velocity (TRV) assessed by transthoracic Doppler echocardiography
Time frame: Baseline to Week 24
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