title: Rationale and study design of MOTION: A phase 4, prospective, single-arm, open-label study to measure outcomes in patients with pulmonary arterial hypertension not on active treatment.
abstract: In clinical trials of treatments for pulmonary arterial hypertension (PAH), objective measures, such as 6-min walk distance (6MWD) are limited in their ability to characterize the impact of PAH therapy from a patient's perspective. Few clinical studies have evaluated the primary effects of pharmacologic treatment on patient-reported outcomes, such as symptoms, health-related quality of life (HRQoL), and productivity. MOTION (NCT02191137) is a prospective, multicenter, single-arm, open-label, phase 4 trial designed to assess whether riociguat monotherapy will improve patient-reported outcomes in patients with PAH in the United States who are not currently on treatment. Following a screening period of up to 14 days, eligible subjects will receive riociguat (0.5-2.5 mg TID) during a 10-week titration phase and a 14-week maintenance phase. The primary endpoint is change from baseline in the Living with Pulmonary Hypertension (LPH) questionnaire, a disease-specific HRQoL measure, after 24 weeks of riociguat treatment. The Short Form-12 Health Survey (SF-12) and the Work Limitations Questionnaire 8 (WLQ-8) will also be utilized to assess patient-reported outcomes. Other variables include change from baseline in World Health Organization functional class, 6MWD, and modified Borg Dyspnea Index. In addition, accelerator band activity will be validated against the 6MWD test. Safety will also be assessed. The MOTION trial will provide information on the effect of riociguat on patient-reported outcomes in PAH patients in the United States who are not currently on active treatment through the use of disease-specific and generic HRQoL measures (LPH and SF-12) and a measure of worker productivity (WLQ-8).
authors.affiliation: Assistant Professor of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA. Electronic address: smathai4@jhmi.edu.
authors.forename: Stephen C
authors.initials: SC
authors.lastname: Mathai
authors.affiliation: Southside Regional Medical Center, Petersburg, VA, USA.
authors.forename: Omar
authors.initials: O
authors.lastname: Minai
authors.affiliation: University of Washington, Seattle, WA, USA.
authors.forename: Sean D
authors.initials: SD
authors.lastname: Sullivan
authors.affiliation: Program on Health, Work, and Productivity, Tufts University School of Medicine, Boston, MA, USA.
authors.forename: Debra
authors.initials: D
authors.lastname: Lerner
authors.affiliation: Director of Pulmonary Hypertension Center, Professor of Medicine, University of Texas Health Science Center San Antonio, San Antonio, TX, USA.
authors.forename: Deborah
authors.initials: D
authors.lastname: Levine
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020536:Enzyme Activators / Q000008:administration & dosage; D005260:Female; D006801:Humans; D006976:Hypertension, Pulmonary / Q000188:drug therapy* / Q000201:enzymology; D008297:Male; D008875:Middle Aged; D000071066:Patient Reported Outcome Measures; D011446:Prospective Studies; D011720:Pyrazoles / Q000008:administration & dosage*; D011743:Pyrimidines / Q000008:administration & dosage*; D011788:Quality of Life; D012107:Research Design; D000071756:Soluble Guanylyl Cyclase / Q000378:metabolism; D016896:Treatment Outcome; D014481:United States / Q000453:epidemiology; D055815:Young Adult
chemical_list: D020536:Enzyme Activators; D011720:Pyrazoles; D011743:Pyrimidines; D000071756:Soluble Guanylyl Cyclase; C542595:riociguat
keywords: Health-related quality of life; Patient-reported outcomes; Pulmonary arterial hypertension; Riociguat