Evaluating inhaled AP01 for progressive pulmonary fibrosis
Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Participants With PPF
Run by Avalyn Pharma Inc. · NCT06329401 · Data updated 2026-05-14
This study is no longer in our active list. It may have stopped recruiting or finished. The details below are as of 2026-05-14 and are kept for reference. Check ClinicalTrials.gov for its current status, or see similar studies below.
This study is testing if inhaled AP01 can help slow down the worsening of lung disease in people with progressive pulmonary fibrosis.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 375 (estimated) |
| Sponsor | Avalyn Pharma Inc. Industry-sponsored |
| Locations | 152 sites (Birmingham, Alabama and 151 other locations) |
| Trial ID | NCT06329401 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include individuals diagnosed with progressive pulmonary fibrosis who have shown evidence of disease progression despite current treatments.
✕Probably 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.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
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.
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 gone: Other studies have explored inhaled therapies for pulmonary fibrosis, but the specific approach of inhaled AP01 is relatively novel.
Who can join: the full rules
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 it's running
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University of Alabama at Birmingham
Birmingham, Alabama, United States Recruiting -
Mayo Clinic- Scottsdale
Scottsdale, Arizona, United States Recruiting -
University of Southern California
Los Angeles, California, United States Recruiting -
Cedars-Sinai
Los Angeles, California, United States Recruiting -
UCLA
Los Angeles, California, United States Recruiting -
Newport Native MD, Inc.
Newport Beach, California, United States Recruiting
Show 44 more locations
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Paradigm Clinical Research - Redding
Redding, California, United States Recruiting -
University of California - San Francisco
San Francisco, California, United States Recruiting -
University of Colorado, Anschutz Medical Campus
Aurora, Colorado, United States Recruiting -
National Jewish Health
Denver, Colorado, United States Recruiting -
UCONN Health
Farmington, Connecticut, United States Recruiting -
Yale University
New Haven, Connecticut, United States Recruiting -
Clinical Site Partners, LCC
Leesburg, Florida, United States Recruiting -
Renstar Medical Research
Ocala, Florida, United States Recruiting -
Clinical Site Partners
Winter Park, Florida, United States Recruiting -
Piedmont Healthcare, Inc.
Atlanta, Georgia, United States Recruiting -
Northwestern University
Chicago, Illinois, United States Recruiting -
Endeavor Health
Evanston, Illinois, United States Recruiting -
Loyola University Medical Center
Maywood, Illinois, United States Recruiting -
The University of Kansas Medical Center
Kansas City, Kansas, United States Recruiting -
University of Maryland
Baltimore, Maryland, United States Recruiting -
Johns Hopkins University
Baltimore, Maryland, United States Recruiting -
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States Recruiting -
University of Michigan
Ann Arbor, Michigan, United States Recruiting -
University of Minnesota
Minneapolis, Minnesota, United States Recruiting -
Mayo Clinic Rochester
Rochester, Minnesota, United States Recruiting -
Hannibal Regional Healthcare System
Hannibal, Missouri, United States Recruiting -
Northwell Health - Mount Kisco
Mount Kisco, New York, United States Recruiting -
NYU Langone Health
New York, New York, United States Recruiting -
Weill Cornell
New York, New York, United States Recruiting -
Icahn School of Medicine at Mount Sinai
New York, New York, United States Recruiting -
Columbia University
New York, New York, United States Recruiting -
Montefiore Medical Center
The Bronx, New York, United States Recruiting -
Duke University
Durham, North Carolina, United States Recruiting -
Piedmont HealthCare, PA
Statesville, North Carolina, United States Recruiting -
Accellacare
Wilmington, North Carolina, United States Recruiting -
Southeastern Research Center
Winston-Salem, North Carolina, United States Recruiting -
University of Cincinnati
Cincinnati, Ohio, United States Recruiting -
Cleveland Clinic
Cleveland, Ohio, United States Recruiting -
Summit Health
Bend, Oregon, United States Recruiting -
The Oregon Clinic Pulmonary East
Portland, Oregon, United States Recruiting -
The Oregon Clinic Pulmonary West
Portland, Oregon, United States Recruiting -
The Pennsylvania State University
Hershey, Pennsylvania, United States Recruiting -
Thomas Jefferson University
Philadelphia, Pennsylvania, United States Recruiting -
Temple University
Philadelphia, Pennsylvania, United States Recruiting -
Lowcountry Lung and Critical Care
Charleston, South Carolina, United States Recruiting -
Medical University of South Carolina
Charleston, South Carolina, United States Recruiting -
Clinical Trials Center of Middle Tennessee
Franklin, Tennessee, United States Recruiting -
Vanderbilt Lung Institute
Nashville, Tennessee, United States Recruiting -
Baylor Scott & White Research Institute, Baylor University Medical Center
Dallas, Texas, United States Recruiting
+102 more sites. See ClinicalTrials.gov for the full list.
Study contacts
- Study coordinator: Craig S. Conoscenti, MD
- Email: cconoscenti@avalynpharma.com
- Phone: 206-707-0304
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.