Phase 2 N=253 Randomized Double-blind Treatment

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

Lung Diseases, Interstitial

Bottom Line

View on ClinicalTrials.gov: NCT03099187 ↗

Enrolled (actual)

253

Serious AEs

20.0%

Results posted

Jan 2020

Primary outcome: Primary: Rate of Decline in Forced Vital Capacity (FVC) Over the 24-week Double-blind Treatment Period — -17.9; 116.6; -90.3; 125.6 milliliter (mL) — p=0.6777

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Pirfenidone (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Hoffmann-La Roche
Primary completion: Nov 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Rate of Decline in Forced Vital Capacity (FVC) Over the 24-week Double-blind Treatment Period	-17.9; 116.6; -90.3; 125.6	0.6777
SECONDARY Change in Percent Predicted FVC	73.95; 73.95; 74.04; 74.55; 73.98; 73.50	0.0383 sig
SECONDARY Change in FVC	2.36; 2.38; 2.37; 2.37; 2.37; 2.36	0.0018 sig
SECONDARY Categorical Change in FVC of >5%	47; 74; 47; 73	0.0006 sig
SECONDARY Categorical Change in FVC of >10%	18; 34; 18; 33	0.0114 sig
SECONDARY Change in Percent Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLco)	46.19; 49.57; -0.52; -0.56; -0.65; -2.47	0.0874
SECONDARY Change in 6-minute Walk Distance (6MWD)	391.6; 394.0; -14.8; -7.7; -2.0; -26.7	0.0395 sig
SECONDARY Change in University of California, San Diego-Shortness of Breath Questionnaire Score	44.17; 48.89; 1.47; 2.24; 5.21; 5.30	0.7788
SECONDARY Change in Score in Leicester Cough Questionnaire Score	16.13; 15.15; 0.35; -0.23; 0.36; 0.04	0.1872
SECONDARY Change in Cough Visual Analog Scale (VAS) Score	35.60; 37.18; -4.33; 3.32; -2.52; 0.78	0.2995
SECONDARY Change in Total and Sub-scores of the Saint George's Respiratory Questionnaire (SGRQ)	49.28; 53.10; -2.60; 0.86; -1.69; -0.66	0.1630
SECONDARY Number of Participants With Non-elective Hospitalization, Both Respiratory and All Cause	16; 13; 5; 5	0.5922
SECONDARY Percentage of Participants With Investigator-reported Acute Exacerbations	3.9; 5.6	—
SECONDARY Time to First Investigator-reported Acute Exacerbations	NA; NA	0.7871
SECONDARY Progression-free Survival (PFS)	NA; NA	0.2726
SECONDARY Progression-free Survival (PFS)	NA; NA	0.2726
SECONDARY Time to Death From Any Cause	NA; NA	0.9969
SECONDARY Time to Death From Respiratory Diseases	NA; NA	0.3231
SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs)	120; 101	—
SECONDARY Number of Participants With Dose Reductions and Treatment Interruptions During the Double-Blind Period	51; 34; 40; 12	—
SECONDARY Number of Participants With Dose Reductions and Treatment Interruptions During the 12-month Safety Follow-up	41; 60; 24; 34	—
SECONDARY Number of Participants Withdrawn From Trial Treatment or Trial Discontinuations During the Double-Blind Period	25; 12	—
SECONDARY Number of Participants Withdrawn From Trial Treatment or Trial Discontinuations During the 12-month Safety Follow-up	19; 26	—

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).

Eligibility Criteria

Inclusion Criteria

Age >= 18-85 years
Confirmed fibrosing ILD which, following multidisciplinary team review, cannot be classified with either high or moderate confidence as a specific idiopathic interstitial pneumonia or other defined ILD
Progressive disease as considered by the investigator as participants deterioration within the last 6 months, which is defined as a rate of decline in forced vital capacity (FVC) >5% or a significant symptomatic worsening not due to cardiac, pulmonary vascular or other causes
Extent of fibrosis >10% on high-resolution computed tomography
Forced vital capacity >= 45% of predicted value
Diffusing capacity of the lung for carbon monoxide (DLco) >= 30% of predicted value
Forced expiratory volume in 1 second/FVC ratio >= 0.7
Able to do 6-minute walk distance (6MWD) >= 150 meters
For women of childbearing potential: agreement to remain abstinent or use a non-hormonal or hormonal contraceptive method with a failure rate of 1.5 × ULN, and Alkaline phosphatase >2.0 × ULN
Creatinine clearance = 500 milliseconds at screening, or a family or personal history of long QT syndrome

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03099187). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.