Completed Lungs & Breathing NIHR-supported project Heart, Stroke & Blood

A Placebo-Controlled Study of Inhaled Pirfenidone in PPF

Funder NIHR (part of DHSC)
Recipient organisation NIHR Exeter Biomedical Research Centre
NIHR support Recorded as supported by this research centre
Period Feb 2025 — Oct 2025

In plain English

AI plain-English summary

Patients with progressive pulmonary fibrosis will inhale an experimental drug called AP01 through a nebuliser twice daily for 52 weeks, to see if it slows lung scarring better than a placebo. This matters because pulmonary fibrosis is a relentless, incurable disease where lung tissue progressively scars and thickens, making breathing increasingly difficult. The oral version of pirfenidone, the active ingredient in AP01, is already approved for some interstitial lung diseases, but it can cause side effects and may not reach the lungs efficiently. Delivering the drug directly to the lungs via inhalation could reduce systemic side effects while delivering a higher dose to the scarred tissue. If this phase 2b trial shows that inhaled pirfenidone is safe and effective, it could offer a new treatment option for patients with progressive pulmonary fibrosis who currently have few alternatives. The study will also track how the drug moves through and leaves the body by analysing blood samples, providing crucial data for future trials. Success would not change daily life for most people, but for those with this condition, it could mean slower disease progression and preserved lung function.

View original technical description
This is a phase 2b research study to evaluate the safety and efficacy of Pirfenidone solution for inhalation (AP01) in participants with Progressive Pulmonary Fibrosis (PPF).Pulmonary fibrosis is a serious, lifelong lung disease. It causes lung scarring (tissues scar and thicken over time), making it harder to breathe. Symptoms may come on quickly or take years to develop.AP01is an experimental drug that may slow PPF symptoms and participants will inhale the study drug using the eFlow® nebuliser.The active component of the investigational drug (pirfenidone) is approved as treatment for certain types of Interstitial Lung Disease (ILD) when it is given as a capsule for oral use, but in this study, it is investigational because it will be given as a solution for inhalation.The purpose of this study is to see how safe and effective multiple doses of AP01 compared with placebo (a dummy treatment), over 52 weeks in participants with PPF. During the study, AP01 will be added to participants current PPF treatment regimen.The study will also look at how the study drug behaves inside the body and how it leaves the body. For this reason, blood samples will be collected to measure study drug within participants body.

Researchers

Michael Gibbons (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Subjects with Progressive Pulmonary Fibrosis (PPF)
A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Subjects with Progressive Pulmonary Fibrosis (PPF) -MIST
A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Subjects with Progressive Pulmonary Fibrosis (PPF) (MIST Devpro)
Access to Pirfenidone Solution for Inhalation (AP01) for Treatment of Progressive, Fibrosing Interstitial Lung Diseases, including Idiopathic Pulmonary Fibrosis
A Randomized Open-Label, Phase 1b Study of the Safety of Pirfenidone Solution for Inhalation (AP01) in Patients with Idiopathic Pulmonary Fibrosis (ATLAS Study)

Original classification

Clinical Mycology

Plain English summaries and category classifications on this site are generated by AI and may not perfectly reflect the original research.