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

PHASE2RECRUITING

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.

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Study 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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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):

Relative decline in FVC ≥10% predicted within the previous 24 months compared to Screening Visit 1

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

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:

FVC ≥45% of predicted normal at Screening Visit 1

Forced expiratory volume at 1 second (FEV1)/FVC ≥0.7 or ≥age-adjusted lower limit of normal at Screening Visit 1

Diffusing capacity of lung for carbon monoxide (DLCO) ≥30% of predicted, corrected for hemoglobin at Screening Visit 1

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:

Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times the upper limit of normal (ULN)

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.

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2024-04-03

Primary completion: 2026-04-01

Study completion finish: 2026-04-01

Study type

TREATMENT

Phase

PHASE2

Trial ID

NCT06329401

Intervention or treatment

DRUG: AP01

OTHER: Placebo

Conditions

• Progressive Pulmonary Fibrosis

Image related to Progressive Pulmonary Fibrosis

Condition: Progressive Pulmonary Fibrosis
DRUG: AP01 and other drugs
Westmead, New South Wales, Australia and more
Sponsor: Avalyn Pharma Inc.

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

Westmead Hospital

Research sites nearby

Select from list below to view details:

Westmead Hospital
Westmead, New South Wales, Australia
Macquarie University Clinical Trials Unit
Sydney, New South Wales, Australia
Institute for Respiratory Health
Nedlands, Perth West Australia, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: AP01 High Dose BID Pirfenidone Solution for Inhalation	DRUG: AP01 Oral inhalation solution
EXPERIMENTAL: AP01 Low Dose BID Pirfenidone Solution for Inhalation	DRUG: AP01 Oral inhalation solution
PLACEBO_COMPARATOR: Placebo BID Placebo solution for inhalation	OTHER: Placebo Placebo oral inhalation solution

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
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)	Change from baseline in forced vital capacity (FVC) (mL)	Week 52

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
To evaluate the effect of AP01 high dose, AP01 low dose compared to placebo on quality of life (QoL)	Absolute change from Baseline in QoL measurements as assessed by Living with Pulmonary Fibrosis Symptoms and Impact Questionnaire (L-PF) total score. The L-PF is a 44-item questionnaire to assess how impacted a participant is by disease symptoms on a scale from 0 (Not at all) to 4 (Extremely). The higher the summary score, the greater the impairment.	52 weeks
To evaluate the effect of AP01 high dose and AP01 low dose compared to placebo on disease progression (defined as absolute FVC percent predicted decline of ≥10% prior to Week 52)	Time to disease progression	52 weeks
To evaluate the change from baseline in quantitative lung fibrosis score.	Change in lung fibrosis score.	52 weeks

Frequently Asked Questions

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References

Clinical Trials Gov: A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis