The purpose of this study is to assess the safety and efficacy of Rodatristat Ethyl in pulmonary arterial hypertension (PAH) patients.
Rodatristat Ethyl is a peripherally restricted TPH inhibitor being studied as a potential treatment for PAH. This dose-ranging, randomized, double-blind, placebo-controlled, multicenter study will evaluate the effect of Rodatristat Ethyl from baseline on pulmonary vascular resistance as measured at right heart catheterization. Patients will be enrolled into a main study with an option to enroll into an open label extension. The study is expected to enroll patients in the USA, Canada and Europe.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
108
rodatristat ethyl 300 mg tablet + matching placebo tablet twice daily on top of standard of care
2 rodatristat ethyl 300 mg tablets twice daily on top of standard of care
2 matching placebo tablets on top of standard of care
Percent Change From Baseline of Pulmonary Vascular Resistance (PVR) at Week 24
Pulmonary vascular resistance (PVR) was measured by right heart catheterization (RHC)
Time frame: 24 Weeks
Change From Baseline in World Health Organization (WHO) Functional Class (FC)
PAH functional disease severity is classified in 4 classes (I to IV) according to World Health Organization (WHO). I:Patients with pulmonary hypertension (PH) but without resulting limitation of physical act.Ordinary physical act does not cause undue dyspnea,fatigue,chest pain,near syncope. II:Patients with PH with slight limitation of physical act.Ordinary physical act causes undue dyspnea or fatigue, chest pain,or near syncope. III:Patients with PH with marked limitation of physical act.Less than ordinary act causes undue dyspnea or fatigue,chest pain,or near syncope. IV:Patients with PH with inability to carry out any physical act without symptoms,manifest signs of right-heart failure.Dyspnea and/or fatigue may even be present at rest.Discomfort is increased by physical act -II, -I:Patients condition improved at WK24 compared with baseline II, I:Patients condition worsened at WK24 compared with baseline No change:Patients condition not changes at WK24 compare with baseline
Time frame: 24 Weeks
Change From Baseline in Six-minute Walk Distance (6MWD)
The six-minute walk distance (6MWD) is a simple, commonly used, standardized measure of functional exercise capacity and endurance. It is a commonly used measure of efficacy in PAH clinical studies.
Time frame: 24 Weeks
Change From Baseline in N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP) Levels
N-terminal prohormone of brain natriuretic peptide (NT-proBNP) is a strong predictor of disease progression and mortality in PAH patients. Current PAH treatment guidelines recommend measurement of NT-proBNP levels for both risk assessment and longitudinal follow up. NT-proBNP levels are also a good marker of response to treatment.
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Arizona Pulmonary Specialists
Phoenix, Arizona, United States
University of California San Diego Health Sciences
La Jolla, California, United States
VA Greater LA Healthcare System/UCLA
Los Angeles, California, United States
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
UC Davis Medical Center
Sacramento, California, United States
Jeffrey S. Sager, MD Medical Corporation
Santa Barbara, California, United States
University of Colorado
Aurora, Colorado, United States
George Washington University Medical Center
Washington D.C., District of Columbia, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
The University of Kansas Medical Center
Kansas City, Kansas, United States
...and 54 more locations
Time frame: 24 Weeks