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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.
Full description
This is a randomized, double-blind, placebo-controlled clinical study to evaluate the safety and efficacy of 2 doses of AP01 (pirfenidone solution for inhalation) versus placebo on top of standard of care in participants with PPF over 52 weeks. Up to 300 eligible participants will be randomized to 1 of 3 treatment arms: AP01 high dose, AP01 low dose, or placebo.
Enrollment
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Inclusion criteria
I. Physiological evidence of disease progression:
a. Absolute decline in FVC ≥5% predicted within the previous 6 to 12 months relative to Screening Visit 1
And at least 1 of the following 2 criteria occurring within the past year with no alternative explanation:
II. Worsening respiratory symptoms (Note: Changes attributable to comorbidities e.g., infection, heart failure must be excluded)
III. Radiological evidence of disease progression (one or more of the following):
a. Increased extent or severity of traction bronchiectasis and bronchiolectasis b. New ground-glass opacity with traction bronchiectasis c. New fine reticulation d. Increased extent or increased coarseness of reticular abnormality e. New or increased honeycombing f. Increased lobar volume loss
Meeting all of the following criteria during the Screening Period:
For participants already on nintedanib (up to 30% of participants): Must have been on nintedanib for 6 to 12 months prior to Screening and have met criteria for PPF while on nintedanib for the same period in which the ≥5% decline in FVC was observed. Must have had no change in nintedanib dose for at least 12 weeks prior to Screening. For participants 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:
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 purpose
Allocation
Interventional model
Masking
300 participants in 3 patient groups, including a placebo group
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Central trial contact
Craig S. Conoscenti, MD; Daniele Tompkins
Data sourced from clinicaltrials.gov
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