NCT06329401

Recruiting

Phase 2

A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Participants With PPF

Avalyn Pharma Inc.258 sites in 4 countries375 target enrollmentApril 3, 2024

ConditionsPulmonary Fibrosis Pulmonary Fibrosis Secondary to Systemic Sclerosis Pulmonary Fibrosis, Interstitial Lung Disease Interstitial Lung Disease Interstitial Lung Disease Due to Connective Tissue Disease (Disorder)Interstitial Lung Disease in Patients With Rheumatoid Arthritis Interstitial Lung Disease With Progressive Fibrotic Phenotype in Diseases Classified Elsewhere Hypersensitivity Pneumonitis Interstitial Lung Disease With Systemic Sclerosis

InterventionsPlacebo AP01

Overview

Phase: Phase 2
Intervention: Placebo
Conditions: Pulmonary Fibrosis
Sponsor: Avalyn Pharma Inc.
Enrollment: 375
Locations: 258
Primary Endpoint: 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)
Status: Recruiting
Last Updated: 26 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Detailed 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.

Registry

euclinicaltrials.eu

Start Date

April 3, 2024

End Date

June 1, 2027

Last Updated

26 days ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Avalyn Pharma Inc.

Responsible Party

Sponsor

Principal Investigator

Craig S. Conoscenti

Scientific

Avalyn Pharma Inc.

Eligibility 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):
•Relative decline in FVC ≥10% predicted within the previous 24 months based on documented historical spirometry assessments
•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

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.

Arms & Interventions

Placebo BID

Placebo solution for inhalation

Intervention: Placebo

AP01 High Dose BID

Pirfenidone Solution for Inhalation

Intervention: AP01

AP01 Low Dose BID

Pirfenidone Solution for Inhalation

Intervention: AP01

Outcomes

Primary Outcomes

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)

Time Frame: Week 52

Change from baseline in forced vital capacity (FVC) (mL)

Secondary Outcomes

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)(52 weeks)
To evaluate the effect of AP01 high dose, AP01 low dose compared to placebo on quality of life (QoL)(52 weeks)
To evaluate the change from baseline in quantitative lung fibrosis score.(52 weeks)