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Evaluating inhaled AP01 for progressive pulmonary fibrosis

A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Participants With PPF

Phase 2 Interventional Avalyn Pharma Inc. · NCT06329401

This study is testing if inhaled AP01 can help slow down the worsening of lung disease in people with progressive pulmonary fibrosis.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment375 (estimated)
Ages18 Years and up
SexAll
SponsorAvalyn Pharma Inc. Industry-sponsored
Locations152 sites (Birmingham, Alabama and 151 other locations)
Trial IDNCT06329401 on ClinicalTrials.gov

What this trial studies

This clinical trial is a randomized, double-blind, placebo-controlled study designed to assess the safety and efficacy of inhaled AP01 (pirfenidone solution) in patients with progressive pulmonary fibrosis (PPF). Over a period of 52 weeks, up to 300 participants will be assigned to receive either a high dose, low dose of AP01, or a placebo, in addition to their standard care. The study aims to determine if inhaled AP01 can effectively slow disease progression in individuals with chronic fibrosing interstitial lung disease.

Who should consider this trial

Good fit: Ideal candidates include individuals diagnosed with progressive pulmonary fibrosis who have shown evidence of disease progression despite current treatments.

Not a fit: Patients with idiopathic pulmonary fibrosis or those who do not meet the criteria for progressive pulmonary fibrosis may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from progressive pulmonary fibrosis.

How similar studies have performed: Other studies have explored inhaled therapies for pulmonary fibrosis, but the specific approach of inhaled AP01 is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participant meets criteria for PPF, as follows:
* In subjects with interstitial lung disease (ILD) of known or unknown etiology other than idiopathic pulmonary fibrosis (IPF) who have radiological evidence of pulmonary fibrosis, PPF is defined as:

Physiological evidence of disease progression with at least 1 of the following criteria despite treatment with approved or unapproved medications commonly used in practice (per Investigator):

1. Relative decline in FVC ≥10% predicted within the previous 24 months based on documented historical spirometry assessments
2. Relative decline in FVC ≥5% to \<10% predicted within the previous 24 months based on documented historical spirometry assessments with at least 1 of the 2 following criteria:

   * Worsening respiratory symptoms (Note: Changes attributable to comorbidities e.g., infection, heart failure must be excluded) OR
   * Radiological (HRCT) evidence of disease progression per a local or central radiologist (from historical HRCT taken up to 24 months prior to Screening Visit 1), for example:

     * Increased extent or severity of traction bronchiectasis and bronchiolectasis
     * New ground-glass opacity with traction bronchiectasis
     * New fine reticulation
     * Increased extent or increased coarseness of reticular abnormality
     * New or increased honeycombing
     * Increased lobar volume loss
3. Worsening of respiratory symptoms (Note: Changes attributable to comorbidities e.g., infection, heart failure must be excluded) AND radiological (HRCT) evidence of disease progression per a local or central radiologist

   * Meeting all of the following criteria during the Screening Period:

a. FVC ≥45% of predicted normal at Screening Visit 1, b. Forced expiratory volume at 1 second (FEV1)/FVC ≥0.7 or ≥age-adjusted lower limit of normal at Screening Visit 1, c. Diffusing capacity of lung for carbon monoxide (DLCO) ≥30% of predicted, corrected for hemoglobin at Screening Visit 1, d. Acceptability: Participants can perform acceptable spirometry (i.e., meet American Thoracic Society (ATS)/ European Respiratory Society (ERS) acceptability criteria at both Screening Visits).

• For subjects already on nintedanib (up to 30% of subjects): Must have been on nintedanib for at least 6 months prior to Screening with or without dose adjustments and/or drug interruptions during that period. For subjects who have discontinued nintedanib prior to Screening: Must have been off of nintedanib for a minimum of 12 weeks.

Exclusion Criteria:

* Current treatment with oral pirfenidone or treatment with oral pirfenidone within 3 months prior to Screening.
* Elevated liver enzymes and liver injury at Screening defined as:

  1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ˃ 3 times the upper limit of normal (ULN)
  2. Bilirubin \>2.0 x ULN
* Renal disease with a creatinine clearance \< 30 mL/min, calculated according to the Chronic Kidney Disease Epidemiology Collaboration formula. Retesting is allowed once.
* Diagnosis of idiopathic pulmonary fibrosis (IPF) based on the ATS diagnostic algorithm for IPF. UIP that is not idiopathic, for example related to rheumatoid arthritis (RA), familial interstitial lung disease (ILD), or other is not exclusionary.
* Greater extent of emphysema than of fibrotic ILD on HRCT. Note: CT results must be confirmed through the central over read process.
* Significant clinical worsening of PPF between Screening
* Participants who cannot meet protocol-specified Baseline stability criteria. FVC Baseline stability is defined as the FVC assessments at Visit 3 being within ±12% of the mean of the FVC assessments obtained at the 2 preceding visits. At Visit 3, if the pre-dose FVC is outside of ±12% range, the participant will not be randomized and will be considered a screen failure.

Where this trial is running

Birmingham, Alabama and 151 other locations

+102 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Pulmonary FibrosisProgressive Pulmonary FibrosisPulmonary Fibrosis Secondary to Systemic SclerosisPulmonary Fibrosis, Interstitial Lung DiseaseInterstitial Lung DiseaseInterstitial Lung Disease Due to Connective Tissue DiseaseInterstitial Lung Disease in Patients With Rheumatoid ArthritisInterstitial Lung Disease With Progressive Fibrotic Phenotype in Diseases Classified Elsewhere
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.