Validation of the Risk Stratification Score in Idiopathic Pulmonary Fibrosis

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Observational
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - 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) and confirmed by a panel of expert chest radiologists.

Exclusion Criteria:

- Interstitial lung disease other than IPF

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT02632123
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Lawson Health Research Institute
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Recruiting
Countries Canada
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Idiopathic Pulmonary Fibrosis
Additional Details

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 criteria 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) and based on multi-disciplinary discussion with Chest Radiologist and, when a biopsy is available, Lung Pathologist.
  • - a pattern consistent with UIP/IPF will be further confirmed by at least 2 expert radiologists, unless a surgical lung biopsy is available and MDD confirms a diagnosis of IPF.
Exclusion criteria:
  • - diagnosis of ILD other than IPF.
  • - not a new diagnosis of IPF.
Patients newly diagnosed with IPF will be included in the study. Patients will be reassessed at 4 months intervals and at each visit the MRC dyspnea score (MRCDS), pulmonary function tests (FEV1, FVC and DLCO), and 6-minute walk distance (6MWD) will be recorded. 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 (every 4 months). A 2nd HRCT will be repeated at 24 months from the time of diagnosis, or sooner if clinically indicated. All relevant comorbidities (cardio-vascular disease, diabetes, sleep apnea, chronic obstructive pulmonary disease) will be taken into consideration. Enrollment in pulmonary rehabilitation physiotherapy will be considered and recorded. The length of the enrollment will be considered. Hospital admission for respiratory reasons will be considered and recorded. All protocol violations will be recorded. Patients will be prospectively followed for a period of at least 3 years and mortality events will be recorded. Three-year lung transplant-free survival 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 a secondary endpoint. Progression of disease will be defined as either: >10% absolute reduction in forced vital capacity (FVC) % pred; >50 m decline in 6-minute walk distance (6MWD); admission to hospital for respiratory causes; lung transplantation assessment; or death Progression of disease will be a secondary endpoint. Patients who received a lung transplant will be censored. 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 will 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), radiographic fibrosis score on high resolution chest CT scan (HRCT) at the time of diagnosis; Gender-Age-Physiology (GAP) Index (Ann Intern Med 2012;156:684-691) and Composite Physiologic Index (Am J Respir Crit Care Med 2033;167:962-969). 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. Variance inflation factors of variables predicting endpoint will be calculated to rule out the possibility of multicollinearity and demonstrate that the variables are truly independent. 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. Areas under the curve (AUC) were compared according to the methods of DeLong et al. (Biometrics 188;44:837-845). Fine-Gray competing risk regression analysis will be used to account for the competing risks of lung transplant and death (J Am Stat Assoc 1999;94:496-509). p-values <0.05 will regarded as significant.

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International Sites

London Health Science Centre, London, Ontario, Canada

Status

Recruiting

Address

London Health Science Centre

London, Ontario, N6A 5W9

Site Contact

Marco Mura, MD, PhD

marco.mura@lhsc.on.ca

+15196676744

Terms of Service

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