An analysis of pooled data from three Phase 3 clinical trials in idiopathic pulmonary fibrosis (IPF) patients shows that treatment with pirfenidone for one year significantly reduced disease progression. The study, titled “Pirfenidone for idiopathic pulmonary fibrosis: analysis of pooled data from three multinational phase 3 trials,” was published in the European Respiratory Journal.
Pirfenidone (Esbriet, developed by Genentech, Roche) is an oral antifibrotic agent tested as a treatment for IPF in three multinational Phase 3 clinical trials: two CAPACITY trials, investigating pirfenidone’s efficacy and safety as a therapy for at least 72 weeks, and the ASCEND trial, which evaluated treatment with pirfenidone for 52 weeks.
Authors analyzed the pooled data to precisely understand the magnitude of the effects of pirfenidone treatment in IPF disease progression. Researchers included all patients randomly administered pirfenidone 2,403 mg/day or a placebo in the CAPACITY (both studies) and ASCEND trial. The analysis followed pre-specified endpoints and analytic procedures of the ASCEND trial.
In total, 1,247 IPF patients were included in the analysis, with 623 patients randomized in the studies to receive pirfenidone treatment and 624 randomized to receive placebo.
The analysis showed that treatment with pirfenidone for one year decreased by 43.8% the number of patients who died or who exhibited a more than 10% decline in predicted forced vital capacity (FVC; a measurement of the lung function). Researchers also found that pirfenidone treatment improved progression-free survival by 38% when compared to placebo. Furthermore, fewer patients (28.7%) treated with pirfenidone experienced more than a 50-meter decline in six-minute walk distance (6MWD, a test of exercise tolerance).
The pooling strategy from multiple clinical studies not only allows extracting more precise estimates on the magnitude of a treatment, including when its effects occur in a low-frequency in individual studies, but also demonstrates the wide effects of a particular treatment in a broad general population.
In conclusion, the results of the analysis of the three clinical trials demonstrated that pirfenidone treatment during one year significantly reduced disease progression in patients with IPF.