This study evaluates the effect of ROC-101 in adults with either Pulmonary Arterial Hypertension (PAH) or Pulmonary Hypertension Associated with Interstitial Lung Disease (ILD-PH). Each eligible participant will receive standard of care (SOC) plus ROC-101 for a 24-week treatment period, followed by a long-term extension period of the study through the end of the program or marketing approval/authorization.
Must be age 18 or older at the time of signing the informed consent form (ICF). The participant must understand and voluntarily sign an ICF prior to any study-related procedures
Documented findings on a right heart catheterization (RHC) consistent with a diagnosis World Health Organization (WHO) Group 1 PAH or WHO GROUP 3 PAH
Symptomatic Pulmonary Hypertension (PH) classified as WHO Functional Class II or III symptoms
PAH participants: Pulmonary Vascular Resistance (PVR) of ≥ 5 Wood units (≥ 400 dynes/sec/cm-5) and, Pulmonary Capillary Wedge Pressure (PCWP) ≤ 15 mmHg and Mean Pulmonary Arterial Pressure (mPAP) > 20 mm Hg and ILD-PH participants: PVR of ≥ 3 Wood units (≥ 240 dynes/sec/cm-5) with a maximum enrollment of 5 participants with PVR between 3 and 4 Wood units inclusive (240-320 dynes/sec/cm-5 inclusive) and the remaining 5 participants must have PVR >4 Wood units (> 320 dynes/sec/cm-5) and, PCWP ≤ 15 mmHg and mPAP > 20 mm Hg
Diagnosis of PH WHO Groups 2, 4, or 5
Diagnosis of the following PAH Group 1 subtypes: human immunodeficiency virus (HIV)-associated PAH, PAH associated with portal hypertension, schistosomiasis-associated PAH and pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis
Positive blood test for hepatitis B virus surface antigen (HBsAg), hepatitis C virus (HCV) antibody (HCVAb) (unless participants have had treatment for HCV and have a negative HCV ribonucleic acid [RNA] polymerase chain reaction [PCR]) or HIV antibody
Participants with known hypersensitivity to ROC-101 or any components of its formulations
Single group
ROC-101
DrugOral, daily administered Rho kinase inhibitor
Change in Pulmonary Vascular Resistance (PVR) from Baseline versus at 24 weeks
Each participant's PVR, at resting supine, was measured by Right Heart Catheterization (RHC) at baseline and at 24 weeks.
Safety assessments: Number of Participants Who Experienced an Adverse Event (AE)
Number of Participants Who Experienced an Adverse Event (AE)
Change in 6-minute Walk Distance (6MWD) from Baseline versus at 24 weeks
The 6MWD was the distance walked in 6 minutes as a measure of functional capacity. This was assessed using the 6-minute walk test (6MWT). Per protocol, change from baseline in 6MWD at Week 24 will be measured
Change in NT-proBNP from Baseline versus at 24 weeks
NT-proBNP is a circulating biomarker that reflects myocardial stretch and is one of the biomarkers measured. The change from baseline in NT-proBNP level versus at Week 24 will be measured.
Change in World Health Organization (WHO) Functional Classification
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. Percentage of participants who improve in WHO FC at Week 24 from baseline will be reported.
Change in Right Atrial Pressure (RAP) measured by Right Heart Catheterization (RHC)
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