1208MO - Phase III trial of perioperative pirfenidone therapy for lung cancer with idiopathic pulmonary fibrosis (IPF): NEJ034 study

Background

IPF is a refractory disease with a short survival and an increased risk of lung cancer development. Standard treatment for lung cancer complicated by IPF has not been established. Even in surgical cases, fatal acute exacerbations (AE) occur in 20% of patients during the acute postoperative period, and is considered to have a negative impact on long-term prognosis.

Methods

Based on the former results of phase II trial, we conducted this trial protocol for preventing AE of IPF perioperatively in the perioperative treatment group, in which pirfenidone 600 mg/day was administered for two weeks before surgery, and the dose was increased to 1200 mg/day for another two weeks before surgery. In a control group, the surgery was performed after perioperative prophylaxis (including no treatment), which was conventionally administered at each centre were allowed, excluding antifibrotic drugs. This randomised, multi-institutional phase III trial was conducted in 73 centres. Patient enrollment took place from October 2017 to June 2023, and the primary endpoint was the rate of AE occurring up to 30 days postoperatively.

Results

Two hundred thirty-two patients were enrolled, and 116 were allocated to the trial treatment and control, respectively. AE within 30 days postoperatively occurred in 20 patients (8.7%). One patient was excluded by central review, and seven patients (6.1%) experienced events in the trial treatment cohort; on the contrary, 12 patients (10.3%) in the control cohort did not have a significant difference (p=0.339) in the full analysis set.

Conclusions

The planned number of patients enrolled in this study was completed within the study period, although the primary endpoint was not met. Then, in addition to the secondary analysis, the efficacy of other endpoints or safety information in the per protocol set will be investigated based on the central review.

Clinical trial identification

UMIN000029411. 2017/10/15.

Editorial acknowledgement

Legal entity responsible for the study

Ichiro Yoshino.

Funding

Has not received any funding.

Disclosure

M. Tsuboi: Financial Interests, Personal, Invited Speaker, Lecture: Johnson & Johnson Japan; Financial Interests, Personal, Advisory Board, Lectures, Advisory boards: AstraZeneca KK, Chugai Pharmaceutical CO.,LTD, MSD; Financial Interests, Personal, Invited Speaker, Lectures: Eli Lilly Japan, Bristol Myers Squibb KK, Taiho Pharma, Medtronic Japan, ONO Pharmaceutical CO.,LTD, Daiichi Sankyo company limited; Financial Interests, Personal, Advisory Board, Advisory boards: Novartis, MiREXS; Financial Interests, Institutional, Research Grant: Boehringer Ingelheim Japan, MSD, AstraZeneca KK, Ono Pharmaceutical CO.,LTD, Bristol Myers Squibb KK, Novartis, MiRXES; Financial Interests, Personal, Steering Committee Member: MSD, AstraZeneca, Novartis; Financial Interests, Institutional, Steering Committee Member: Eli Lilly Japan. K. Kobayashi: Financial Interests, Personal, Advisory Role: Daiichi Sankyo/UCB Japan; Financial Interests, Personal, Speaker’s Bureau: AstraZeneca; Financial Interests, Institutional, Research Funding: Zeria Pharmaceutical Co.. All other authors have declared no conflicts of interest.