Unknown 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 Leicester Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Jan 2025 — Ongoing

In plain English

AI plain-English summary

Patients with progressive pulmonary fibrosis will inhale an experimental drug called AP01—a nebulised form of pirfenidone—to see if it slows lung scarring better than a placebo over 52 weeks. This matters because progressive pulmonary fibrosis is a serious, lifelong condition in which lung tissue scars and thickens, making breathing increasingly difficult. Current treatments are limited: oral pirfenidone capsules are approved for some types of interstitial lung disease, but they can cause side effects and may not deliver enough drug to the lungs. Inhaling the drug directly could improve efficacy while reducing systemic side effects. If this phase 2b trial shows AP01 is safe and effective, it could offer a new treatment option for patients whose disease continues to worsen despite existing therapies. A successful inhaled formulation would mean patients could add it to their current regimen without switching treatments, potentially slowing disease progression and preserving lung function for longer. The study also tracks how the drug behaves in the body by measuring drug levels in blood samples, providing crucial data for future development.

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

Charlotte Swales (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)

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