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

Stephen Mathai, Omar Minai, Sean D. Sullivan, Debra Lerner, Deborah Levine

Research output: Contribution to journalArticle

Abstract

In clinical trials of treatments for pulmonary arterial hypertension (PAH), objective measures, such as 6-min walking 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).

Original languageEnglish (US)
JournalRespiratory Medicine
DOIs
StateAccepted/In press - Jun 8 2016

Fingerprint

Pulmonary Hypertension
Outcome Assessment (Health Care)
Walking
Quality of Life
Therapeutics
Health Surveys
Dyspnea
Maintenance
Clinical Trials
Safety
Patient Reported Outcome Measures
riociguat
Surveys and Questionnaires

Keywords

  • Health-related quality of life
  • Patient-reported outcomes
  • Pulmonary arterial hypertension
  • Riociguat

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

Cite this

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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 walking 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).",
keywords = "Health-related quality of life, Patient-reported outcomes, Pulmonary arterial hypertension, Riociguat",
author = "Stephen Mathai and Omar Minai and Sullivan, {Sean D.} and Debra Lerner and Deborah Levine",
year = "2016",
month = "6",
day = "8",
doi = "10.1016/j.rmed.2016.11.002",
language = "English (US)",
journal = "Respiratory Medicine",
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AU - Mathai, Stephen

AU - Minai, Omar

AU - Sullivan, Sean D.

AU - Lerner, Debra

AU - Levine, Deborah

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