This local Phase 3 study is planned to confirm the efficacy and safety in Japanese PAH participants. The primary population of this study is Japanese PAH participants with World Health Organization Functional Class (WHO FC) II or III while the study includes PAH participants with WHO FC I or IV as other populations. There are no hypotheses for this study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
46
SC injection at a starting dose of 0.3 mg/kg with a target dose of 0.7 mg/kg every 21 days plus background PAH therapy.
Nagoya University Hospital ( Site 2010)
Nagoya, Aichi-ken, Japan
Chiba Saiseikai Narashino hospital ( Site 2004)
Narashino, Chiba, Japan
Change From Pulmonary Vascular Resistance (PVR) From Baseline at Week 24
PVR was the resistance against blood flow from the pulmonary artery to the left atrium. PVR was measured in dyn\*sec/cm\^5 by right heart catheterization (RHC). RHC was performed during the screening period (baseline) and Week 24. Per protocol, the change in PVR from baseline at Week 24 was reported for the primary treatment period.
Time frame: Baseline and Week 24
Number of Participants Who Experienced an Adverse Event (AE)
An AE was any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Per protocol, the number of participants who experienced an AE were reported for the primary treatment period.
Time frame: Up to ~24 weeks
Number of Participants Who Discontinued Study Intervention Due to AEs
An AE was any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Per protocol, the number of participants who discontinued study treatment due to AEs were reported for the primary treatment period.
Time frame: Up to ~24 weeks
Change From Baseline in Six-Minute Walk Distance (6MWD) at Week 24
The 6MWD was the distance walked in 6 minutes as a measure of functional capacity was measured during the screening period (baseline) and at Week 24. This was assessed using the 6-minute walk test (6MWT). Per protocol, the change from baseline in 6MWD at Week 24 was reported for the primary treatment period.
Time frame: Baseline and Week 24
Percentage of Participants With Improvement in World Health Organization Functional Class (WHO FC) at Week 24
The severity of participant's pulmonary arterial hypertension (PAH) symptoms will be graded using the WHO FC system. WHO functional classification for PAH ranges from Class I (no limitation in physical activity, no dyspnea with normal activity), Class II (slight limitation of physical activity), Class III (marked limitation of physical activity) and Class IV (cannot perform a physical activity without any symptoms, dyspnea at rest). Participants who improve in WHO FC were classified into "Improved", "No change" and "Worsened". Improvement = reduction in FC, worsened = increase in FC and no change = no change in FC. Per protocol, the percentage of participants with improvement in WHO FC at Week 24 were presented for the primary treatment period.
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Kurume University Hospital ( Site 2014)
Kurume, Fukuoka, Japan
Kure Kyosai Hospital ( Site 2017)
Kure, Hiroshima, Japan
Sapporo Medical University Hospital ( Site 2018)
Sapporo, Hokkaido, Japan
Hokkaido University Hospital ( Site 2001)
Sapporo, Hokkaido, Japan
Kobe University Hospital ( Site 2012)
Kobe, Hyōgo, Japan
Tohoku University Hospital ( Site 2002)
Sendai, Miyagi, Japan
National Cerebral and Cardiovascular Center ( Site 2011)
Suita, Osaka, Japan
Hamamatsu University Hospital ( Site 2016)
Hamamatsu, Shizuoka, Japan
...and 7 more locations
Time frame: Baseline and Week 24
Change From Baseline in N-terminal proB-type Natriuretic Peptide (NT-proBNP) at Week 24
NT-proBNP is an established marker of ventricular dysfunction in participants with PAH. NT-proBNP was measured at Day 1 (baseline) and at Week 24. The change from baseline in NT-proBNP at Week 24 was reported for the primary treatment period.
Time frame: Baseline and Week 24