Clinical Trial: A Study of Pirfenidone in Patients With Unclassifiable Progressive Fibrosing Interstitial Lung Disease

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Multicenter, International, Double-blind, Two-Arm, Randomized, Placebo-controlled Phase II Trial of Pirfenidone in Patients With Unclassifiable Progressive Fibrosing ILD

Brief Summary: The purpose of this study is to evaluate the efficacy and safety of pirfenidone in participants with fibrosing interstitial lung disease (ILD) who cannot be classified with moderate or high confidence into any other category of fibrosing ILD by multidisciplinary team (MDT) review ("unclassifiable" ILD).

Detailed Summary:
Sponsor: Hoffmann-La Roche

Current Primary Outcome: Rate of Decline in Forced Vital Capacity (FVC) Over the 24-week Double-blind Treatment Period [ Time Frame: Up to Week 24 ]

Rate of decline in FVC will be measured in mL by daily handheld spirometer.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in Percent Predicted FVC [ Time Frame: Baseline (Day 1) to Week 24 ]
    FVC will be measured in mL by daily handheld spirometer as well as by spirometry.
  • Categorical Change in FVC of >5% During Clinic Visits [ Time Frame: Baseline (Day 1) to Week 24 ]
    Categorical change in FVC will be measured both by daily spirometry as well as by spirometry.
  • Categorical Change in FVC of >10% During Clinic Visits [ Time Frame: Baseline (Day 1) to Week 24 ]
    Categorical change in FVC will be measured both by daily spirometry as well as by spirometry.
  • Change in Percent Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) [ Time Frame: Baseline (Day 1) to Week 24 ]
    The DLco is a pulmonary function test that measures the capacity for the lung to carry out gas exchange between the inhaled breath and the pulmonary capillary blood vessels and the DLco %-predicted represents the DLco expressed as a percentage of the expected normal valued based on the participant's age, height, gender and ethnicity.
  • Change in 6-minute Walk Distance (6MWD) [ Time Frame: Baseline (Day 1) to Week 24 ]
    Comparison of 6-minute walk distance before beginning and after completing study therapy.
  • Change in University of California, San Diego-Shortness of Breath Questionnaire Score [ Time Frame: Baseline (Day 1) to Week 24 ]
    University of California, San Diego Shortness of Breath Questionnaire (SOBQ) consists of 24-item on a scale of 0 to 5 with 0=not at all and 5=maximal or unable to do because of breathlessness. The total scores is calculated by summation of the 24 items scores and transformed into 0-100, with 0= poor quality of life , and 100= excellent quality of life.
  • Change in Score in Leicester Cough Questionnaire Score [ Time Frame: Baseline (Day 1) to Week 24 ]
    The Leicester Cough Questionnaire is a patient-reported questionnaire evaluating the impact of cough on quality of life. The questionnaire comprises 19 items. Each item assesses symptoms, or the impact of symptoms, over the last 2 weeks on a seven-point Likert scale. Scores in three domains (physical, psychological and social) are calculated as a mean for each domain (range 1 to 7). A total score (range 3 to 21) is also calculated by adding the domain scores together. Higher scores indicate better quality of life.
  • Change in Cough Visual Analog Scale (VAS) Score [ Time Frame: Baseline (Day 1) to Week 24 ]
    Cough VAS are 100-mm linear scales on which participants indicate the severity of their cough; 0 mm represents no cough and 100 mm the worst cough ever.
  • Change in Total and Sub-scores of the Saint George's Respiratory Questionnaire (SGRQ) [ Time Frame: Baseline (Day 1) to Week 24 ]
    SGRQ is a health related quality of life questionnaire consisting of 40 items in three areas: symptoms (respiratory symptoms and severity), activity (activities that cause or are limited by breathlessness) and impacts (social functioning and psychological disturbances due to airway disease). The total score is 0 to 100 with a higher score indicating poorer health status.
  • Number of Participants with Non-elective Hospitalization, Both Respiratory and all Cause [ Time Frame: Up to Week 91 ]
    Participants with non-elective hospitalization will be reported.
  • Percentage of Participants with Investigator-reported Acute Exacerbations [ Time Frame: Baseline (Day 1) to Week 24 ]
    Percentage of participants with acute exacerbation will be reported.
  • Time to First Investigator-reported Acute Exacerbations [ Time Frame: Baseline (Day 1) to Week 24 ]
    Time to first investigator reported acute exacerbations from start of treatment will be reported.
  • Progression-free Survival (PFS) [ Time Frame: Baseline (Day 1) to Week 24 ]
    PFS is defined as the time to the first occurrence of a >10% absolute decline in percent predicted FVC, a >50 m decline of 6MWD, or death.
  • Progression-free Survival (PFS) [ Time Frame: Baseline (Day 1) to Week 24 ]
    PFS is defined as the time to the first occurrence of a >10% absolute decline in percent predicted FVC, non-elective respiratory hospitalization, or death.
  • Time to Death From any Cause [ Time Frame: Up to Week 91 ]
    Time to first documented death from start of treatment will be reported.
  • Time to Death from Respiratory Diseases [ Time Frame: Up to Week 91 ]
    Time to first documente

    Original Secondary Outcome: Same as current

    Information By: Hoffmann-La Roche

    Dates:
    Date Received: March 31, 2017
    Date Started: April 18, 2017
    Date Completion: January 11, 2020
    Last Updated: April 21, 2017
    Last Verified: April 2017