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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
To evaluate the effect of AP01 high dose twice a day (BID) or AP01 low dose twice a day (BID) compared to placebo twice a day (BID) — Week 52 Change from baseline in forced vital capacity (FVC) (mL)
Trial sites (154)
Facility
City
Region
Status
University of Alabama at Birmingham
Birmingham
Alabama
Mayo Clinic- Scottsdale
Scottsdale
Arizona
University of Southern California
Los Angeles
California
Cedars-Sinai
Los Angeles
California
UCLA
Los Angeles
California
Newport Native MD, Inc.
Newport Beach
California
Paradigm Clinical Research - Redding
Redding
California
University of California - San Francisco
San Francisco
California
University of Colorado, Anschutz Medical Campus
Aurora
Colorado
National Jewish Health
Denver
Colorado
UCONN Health
Farmington
Connecticut
Yale University
New Haven
Connecticut
Clinical Site Partners, LCC
Leesburg
Florida
Renstar Medical Research
Ocala
Florida
Clinical Site Partners
Winter Park
Florida
Piedmont Healthcare, Inc.
Atlanta
Georgia
Northwestern University
Chicago
Illinois
Endeavor Health
Evanston
Illinois
Loyola University Medical Center
Maywood
Illinois
The University of Kansas Medical Center
Kansas City
Kansas
University of Maryland
Baltimore
Maryland
Johns Hopkins University
Baltimore
Maryland
Beth Israel Deaconess Medical Center
Boston
Massachusetts
University of Michigan
Ann Arbor
Michigan
University of Minnesota
Minneapolis
Minnesota
Mayo Clinic Rochester
Rochester
Minnesota
Hannibal Regional Healthcare System
Hannibal
Missouri
Northern Westchester Hospital
Mount Kisco
New York
Northwell Health - Mount Kisco
Mount Kisco
New York
NYU Langone Health
New York
New York
Weill Cornell
New York
New York
Icahn School of Medicine at Mount Sinai
New York
New York
Columbia University
New York
New York
Montefiore Medical Center
The Bronx
New York
Duke University
Durham
North Carolina
Piedmont HealthCare, PA
Statesville
North Carolina
Accellacare
Wilmington
North Carolina
Southeastern Research Center
Winston-Salem
North Carolina
University of Cincinnati
Cincinnati
Ohio
Cleveland Clinic
Cleveland
Ohio
+ 114 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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