Validation of the Risk Stratification Score in Idiopathic Pulmonary Fibrosis
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|ClinicalTrials.gov Identifier: NCT02632123|
Recruitment Status : Recruiting
First Posted : December 16, 2015
Last Update Posted : March 6, 2018
Idiopathic pulmonary fibrosis (IPF) is a progressing scarring disease of the lungs with an average survival of only 30-36 months since the time of diagnosis. The clinical course of IPF is highly variable, with some patients remaining stable for a prolonged period of time, even in the absence of medical treatment, while others experience rapid and relentless progression. In some cases, the clinical course consists of a stepwise rather than steady decline, with periods of stability alternating with acute respiratory worsening. The variability in clinical course makes it challenging to define prognosis in these patients and, importantly, to determine the right time window for lung transplantation (LTx) referral, listing and priority status on the waiting list.
The risk stratification score (RISE) is a new staging system for IPF based on the MRC dyspnea score, on physiology variables (pulmonary function tests) captured in the Composite Physiologic Index (CPI), and on the 6-minute walk distance. RISE showed to predict survival in newly diagnosed patients with IPF better than any other individual clinical, functional or radiographic variable. RISE also predicted survival on the waiting list in patients assessed and listed for LTx.
|Condition or disease|
|Idiopathic Pulmonary Fibrosis|
The objective of this study is to validate the RISE as a reliable tool to predict survival in patients newly diagnosed with IPF and prospectively followed for a period of 3 years, or until death or lung transplant.
This is an observational, prospective cohort study.
Inclusion criterion will be a new diagnosis of IPF based on the American Thoracic Society/European Respiratory Society criteria (Am J Respir Crit Care Med 2011;183:788-824). A pattern consistent with IPF will be confirmed by expert radiologists, unless a surgical lung biopsy is available.
Patients newly diagnosed with IPF will be included in the study. At each visit, typically every 4 months, the MRC dyspnea score (MRCDS), the pulmonary function tests (FEV1, FVC and DLCO), and the 6-minute walk distance (6MWD) will be recorded. The RISE will be calculated ad described in Eur Respir J 2012;40:101-109.
MRCDS, PFTs and 6MWD are part of the standard of care in IPF and are routinely obtained at each visit of IPF patients in the ILD clinic (typically every 4 months).
Patients will be prospectively followed for a period of 3 years and mortality events will be recorded. Mortality will be the primary endpoint. Acute exacerbations (AEs) of IPF, as defined in AJR Am J Roentgenol 1997;168:79-83, will also be recorded. AEs will be the secondary endpoint. At the end of the study period, both baseline RISE and longitudinal changes of RISE will be tested as predictors of mortality. Other individual variables wil also be tested as predictors of mortality including age at the time of diagnosis, time between onset of symptoms and diagnosis (months), gender, body mass index, smoking history (pack-years), PFTs, 6MWD (meters and % predicted), and radiographic fibrosis score on high resolution chest CT scan (HRCT) at the time of diagnosis.
Values will expressed as mean±SD. Comparisons between survivors and non-survivors will be made with unpaired t-test or with the Mann-Whitney U-test, where appropriate. The optimal cut-off value for different variables to detect mortality or AE will assessed using receiver operating characteristics (ROC) analysis. Survival will evaluated using Kaplan-Meier curves and the log-rank test. Cox proportional hazards regression analysis will used to identify significant variables predicting survival status. Results will be summarized as hazard ratios, representing the relative risk of dying as a result of a specific characteristic during the observation period. Variables selected via univariate analysis (p<0.05) will evaluated in the multivariate Cox regression analysis. p-values <0.05 will regarded as significant.
|Study Type :||Observational|
|Estimated Enrollment :||500 participants|
|Official Title:||Validation of the Risk Stratification Score in Idiopathic Pulmonary Fibrosis|
|Actual Study Start Date :||December 2015|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2022|
Idiopathic pulmonary fibrosis
Patients newly diagnosed with idiopathic pulmonary fibrosis
- Survival [ Time Frame: 3 years ]Primary outcome is 3-year survival since the time of diagnosis
- Acute exacerbations of IPF [ Time Frame: 3 years ]Secondary outcome is the incidence of acute exacerbations since the time of diagnosis
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02632123
|Contact: Marco Mura, MD, PhDemail@example.com|
|London Health Science Centre||Recruiting|
|London, Ontario, Canada, N6A 5W9|
|Contact: Marco Mura, MD, PhD +15196676744 firstname.lastname@example.org|
|Contact: Karishma Hosein +15196858500 ext 55749 email@example.com|
|Principal Investigator: Marco Mura, MD, PhD|