AP02 (inhaled nintedanib) for idiopathic pulmonary fibrosis

A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Proof of Concept (POC) Study Evaluating the Safety, Tolerability, and Efficacy of Nintedanib Solution for Inhalation (AP02) in Participants With Idiopathic Pulmonary Fibrosis (IPF) (AURA-IPF)

This trial will test whether inhaled nintedanib (AP02), given twice daily at two different doses, improves lung function and fibrosis measures in adults 40 and older with idiopathic pulmonary fibrosis compared with placebo.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 160 (estimated)
• Ages: 40 Years and up
• Sex: All
• Sponsor: Avalyn Pharma Inc. (industry)
• Locations: 3 sites (Kingswood, New South Wales and 2 other locations)
• Trial ID: NCT07194382 on ClinicalTrials.gov

What this trial studies

This is a randomized, double-blind Phase 2 trial comparing two doses of inhaled nintedanib (AP02) against placebo in adults with IPF. Participants who are 40 years or older and meet lung function criteria will be randomized to one of three groups and will inhale study medication or placebo twice daily via nebulizer for 12 weeks. The study will measure changes in lung function and key fibrosis-related endpoints and will monitor safety and tolerability. Subjects will attend six in-person visits and receive one phone call over a 16-week period.

Who should consider this trial

Good fit: Adults aged 40 or older with a confirmed diagnosis of IPF (within 5 years), FVC ≥45% predicted, DLCO corrected for hemoglobin 30–80% predicted, and who are not currently on IPF therapy or have stopped oral nintedanib or pirfenidone for at least 3 months are ideal candidates.

Not a fit: Patients with very advanced disease (for example FVC <45% or DLCO <30%), recent use of antifibrotic therapy without required washout, or significant comorbidities are less likely to benefit from participation.

Why it matters

Potential benefit: If successful, inhaled AP02 could deliver antifibrotic therapy directly to the lungs and may improve lung outcomes with fewer systemic side effects than oral treatment.

How similar studies have performed: Oral nintedanib is an approved treatment that slows IPF progression, but inhaled AP02 is a novel formulation and has limited prior clinical data.

Eligibility criteria

Inclusion Criteria:

* Male or female patients aged ≥40 years at the time of signing the written informed consent form
* Diagnosis of idiopathic pulmonary fibrosis (IPF) as defined by American Thoracic Society/European Respiratory Society/Japanese Respiratory Society/ Asociación Latinoamericana de Tórax IPF guidelines within 5 years
* Combination of High-Resolution Computerized Tomography (HRCT) pattern, and if available, surgical lung biopsy pattern consistent with diagnosis of IPF
* In a stable condition and suitable for study participation based on the results of medical history, physical examination, vital signs, 12-lead ECG, and laboratory evaluation
* Forced vital capacity (FVC) ≥45% predicted of normal
* Lung diffusion test (DLCO) corrected for hemoglobin (Hgb) ≥30% and ≤80% predicted of normal.
* Women and men of childbearing potential must use highly effective contraception measures until 90 days after the last dose of study drug.

Exclusion Criteria:

* Current treatment with oral nintedanib, oral pirfenidone, or previous treatment with oral nintedanib or oral pirfenidone within 3 months prior to screening. Participants who have taken both oral nintedanib and pirfenidone together as a treatment will not be able to join the study.
* Forced expiratory volume (FEV) in the first second/FVC (FEV1/FVC) ratio ≥0.7 based on pre-bronchodilator value
* Participants with a history of serious cardiovascular disease, bleeding problems or significant liver or kidney disease (as defined in the study protocol)
* History of diverticular disease or abdominal surgery within 4 weeks prior to screening visit
* History of cancer within the past 5 years (except for certain types explained in the protocol)
* Participants who have smoked within the past 3 months prior to screening, who are not willing to stop smoking during the study, or who currently use illegal drugs or drugs of abuse
* Female participants who are pregnant or nursing
* Use of any investigational drugs including those for IPF within the past 30 days prior to screening

Further inclusion/exclusion criteria apply

Where this trial is running

Kingswood, New South Wales and 2 other locations

• Nepean Lung and Sleep — Kingswood, New South Wales, Australia (RECRUITING)
• Dynamic Drug Advancement — Ajax, Ontario, Canada (RECRUITING)
• CIC Mauricie — Trois-Rivières, Quebec, Canada (ACTIVE_NOT_RECRUITING)

Study contacts

• Study coordinator: Craig S Conoscenti, MD, FCCP, ATSF

Conditions: Idiopathic Pulmonary Fibrosis, IPF, AP02