RECRUITINGPhase 2INTERVENTIONAL
A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With 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
About This Trial
A randomized, double-blind, placebo-controlled clinical study to evaluate the safety and efficacy of 2 doses of inhaled pirfenidone (AP01) versus placebo on top of standard of care in participants with PPF over 52 weeks.
Who May Be Eligible (Plain English)
Who May Qualify:
- 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:
...See full criteria on ClinicalTrials.gov
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.
Treatments Being Tested
DRUG
AP01
Oral inhalation solution
OTHER
Placebo
Placebo oral inhalation solution
Locations (20)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Mayo Clinic- Scottsdale
Scottsdale, Arizona, United States
University of Southern California
Los Angeles, California, United States
Cedars-Sinai
Los Angeles, California, United States
UCLA
Los Angeles, California, United States
Newport Native MD, Inc.
Newport Beach, California, United States
Paradigm Clinical Research - Redding
Redding, California, United States
University of California - San Francisco
San Francisco, California, United States
University of Colorado, Anschutz Medical Campus
Aurora, Colorado, United States
National Jewish Health
Denver, Colorado, United States
UCONN Health
Farmington, Connecticut, United States
Yale University
New Haven, Connecticut, United States
Clinical Site Partners, LCC
Leesburg, Florida, United States
Renstar Medical Research
Ocala, Florida, United States
Clinical Site Partners
Winter Park, Florida, United States
Piedmont Healthcare, Inc.
Atlanta, Georgia, United States
Northwestern University
Chicago, Illinois, United States
Endeavor Health
Evanston, Illinois, United States
Loyola University Medical Center
Maywood, Illinois, United States
The University of Kansas Medical Center
Kansas City, Kansas, United States