TY - JOUR
T1 - Rationale and study design of MOTION
T2 - A phase 4, prospective, single-arm, open-label study to measure outcomes in patients with pulmonary arterial hypertension not on active treatment
AU - Mathai, Stephen C.
AU - Minai, Omar
AU - Sullivan, Sean D.
AU - Lerner, Debra
AU - Levine, Deborah
N1 - Publisher Copyright:
© 2016
PY - 2017/1/1
Y1 - 2017/1/1
N2 - 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).
AB - 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).
KW - Health-related quality of life
KW - Patient-reported outcomes
KW - Pulmonary arterial hypertension
KW - Riociguat
UR - http://www.scopus.com/inward/record.url?scp=85007162486&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85007162486&partnerID=8YFLogxK
U2 - 10.1016/j.rmed.2016.11.002
DO - 10.1016/j.rmed.2016.11.002
M3 - Article
C2 - 27890471
AN - SCOPUS:85007162486
SN - 0954-6111
VL - 122
SP - S23-S27
JO - Respiratory Medicine
JF - Respiratory Medicine
ER -