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A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants with Progressive Pulmonary Fibrosis

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years and Over
Gender	All

More 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 compared to Screening Visit 1. 2. Relative decline in FVC ≥5 to <10% predicted within the previous 24 months compared to Screening Visit 1 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, 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06329401
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Avalyn Pharma Inc.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Avalyn Pharma, Inc.
Principal Investigator Affiliation	Avalyn Pharma Inc.
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	Australia, Canada, France, Germany, Italy, New Zealand, Poland, Spain, United Kingdom, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Progressive Pulmonary Fibrosis
Study Website:	View Trial Website

Additional Details

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.

Arms & Interventions

Arms

Experimental: AP01 High Dose BID

Pirfenidone Solution for Inhalation

Experimental: AP01 Low Dose BID

Pirfenidone Solution for Inhalation

Placebo Comparator: Placebo BID

Placebo solution for inhalation

Interventions

Drug: - AP01

Oral inhalation solution

Other: - Placebo

Placebo oral inhalation solution

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

University of Alabama at Birmingham, Birmingham, Alabama

Status

Recruiting

Address

University of Alabama at Birmingham

Birmingham, Alabama, 35205

Site Contact

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