A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis

Summary

This is a Phase 3, randomized, double-blind, placebo-controlled,efficacy and safety study of pirfenidone in patients with IPF. Approximately 500 patients will be randomized with equal probability to receive pirfenidone 2403 mg/d or placebo for 52 weeks. The primary efficacy endpoint is change in %FVC from Baseline to Week 52. Eligible patients aged 40 and #150;80 years (inclusive) with a confident clinical and radiographic diagnosis of IPF according to pre-specified criteria, FVC greater than or equal 50% and less than or equal 90% and percent predicted carbon monoxide diffusing capacity (%DLCO) greater than or equal to 30% and less than or equal to 90% will be considered for inclusion in study. Other than brief periods of corticosteroid use for acute IPF exacerbation, patients will not receive any other therapy for the treatment of IPF. Patients who, in the opinion of the investigator, are compliant with study treatment dosing (=80%) will be permitted to enter the rollover study (during which all patients will receive pirfenidone 2403 mg/d)at the completion of their Week 52 visit.

Participant Eligibility

1. Clinical symptoms consistent with IPF of ≥12 months duration
2. Diagnosis of IPF, defined as the first instance in which a patient was informed of having IPF, at least 6 months and no more than 48 months before randomization
3. Age 40 through 80 years, inclusive, at randomization
4. Diagnosis of UIP or IPF by HRCT and SLB as outlined in Table 3-1 (Note:HRCT scan performed within 1 month of the start of Screening may be used if it meets image acquisition guidelines)
5. Extent of fibrotic changes (honeycombing, reticular changes) greater than the extent of emphysema on HRCT scan, as determined by central review
6. No features supporting an alternative diagnosis on transbronchial biopsy, bronchoalveolar lavage (BAL), or SLB, if performed
7. %FVC ≥50% and ≤90% at Screening, confirmed by central review
8. Change in pre- and post-bronchodilator FVC (measured in liters) between Screening and Day 1 must be a <10% relative difference
9. %DLCO ≥30% and ≤90% at Screening, confirmed by central review
10. In the investigator’s opinion, no evidence of improvement in measures of IPF disease severity over the preceding year
11. Able to walk ≥150 m during the 6-minute walk test (6MWT) at Screening
12. Able to understand and sign a written informed consent form
13. Able to understand the importance of adherence to study treatment and the study protocol and willing to follow all study requirements, including the concomitant medication restrictions, throughout the study