Design of the MIST study: a double-blind, randomised, placebo-controlled phase 2b trial of pirfenidone solution for inhalation in patients with progressive pulmonary fibrosis.

ABSTRACT:

Introduction

Progressive pulmonary fibrosis (PPF) is a debilitating progression of pulmonary fibrosis in patients with interstitial lung diseases (ILDs) other than idiopathic pulmonary fibrosis (IPF). Oral pirfenidone has been studied in non-IPF ILDs, but in smaller academic studies, it did not achieve a statistically significant change in the primary endpoint. Increased systemic exposure with oral administration may lead to substantial adverse events and limit its utility. Clinical data support inhaled pirfenidone for the treatment of PPF with improved tolerability compared with orally administered pirfenidone.

Methods and analysis

Approximately 300 patients with PPF will be randomised 2:1:2 to one of three treatment arms: AP01 100 mg two times per day, AP01 50 mg two times

SUBMITTER: Kolb M 

PROVIDER: S-EPMC12742111 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature