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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Pulmonary Fibrosis; Interstitial Lung Disease; Hypersensitivity Pneumonitis; Pulmonary Fibrosis, Interstitial Lung Disease; Interstitial Lung Disease in Patients With Rheumatoid Arthritis; Progressive Pulmonary Fibrosis; Pulmonary Fibrosis Secondary to Systemic Sclerosis; Interstitial Lung Disease With Systemic Sclerosis; Interstitial Lung Disease Due to Connective Tissue Disease (Disorder); Interstitial Lung Disease With Progressive Fibrotic Phenotype in Diseases Classified Elsewhere
• Intervention / Treatment: Drug: AP01; Other: Placebo

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.

• Study Type: Interventional
• Enrollment (Estimated): 375
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Craig S. Conoscenti, MD; Phone Number: 206-707-0304; Email: cconoscenti@avalynpharma.com
• Study Contact Backup: Name: Daniele Tompkins; Phone Number: 205 973-983-3700; Email: dtompkins@devprobiopharma.com

Study Locations

• Argentina
  • Buenos Aires
    • Buenos Aires, Buenos Aires, Argentina, 1427
      • Recruiting
      • Fundación Respirar
    • Buenos Aires, Buenos Aires, Argentina, C1056AB
      • Recruiting
      • CINME
    • Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina, C1199ABH
      • Recruiting
      • Hospital Italiano de Buenos Aires
    • Mar del Plata, Buenos Aires, Argentina, 7600
      • Recruiting
      • Instituto Ave Pulmo, Fundacion Enfisema
    • Monte Grande, Buenos Aires, Argentina, 1842
      • Recruiting
      • Clinica Monte Grande
    • Quilmes, Buenos Aires, Argentina, B1878
      • Recruiting
      • Centro Respiratorio Quilmes
  • Córdoba Province
    • Córdoba, Córdoba Province, Argentina, 5003
      • Recruiting
      • Instituto de Medicina Respiratoria
  • Tucumán Province
    • San Miguel de Tucumán, Tucumán Province, Argentina, 4000
      • Recruiting
      • Investigaciones en Patologias Respiratorias
    • San Miguel de Tucumán, Tucumán Province, Argentina, 4000
      • Recruiting
      • CIMER Centro Integral de Medicina Respiratoria
• Australia
  • Canberra, Australia, 2605
    • Recruiting
    • Canberra Hospital
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Recruiting
      • Royal Prince Alfred Hospital
    • Kingswood, New South Wales, Australia, 2745
      • Recruiting
      • Nepean Lung & Sleep
    • New Lambton, New South Wales, Australia, 2305
      • Recruiting
      • John Hunter Hospital
    • Sydney, New South Wales, Australia, 2109
      • Recruiting
      • Macquarie University Clinical Trials Unit
    • Westmead, New South Wales, Australia, 2145
      • Recruiting
      • Westmead Hospital
  • Perth West Australia
    • Nedlands, Perth West Australia, Australia, 6009
      • Recruiting
      • Institute for Respiratory Health
  • Queensland
    • Brisbane, Queensland, Australia, 4032
      • Recruiting
      • Wallace Street Specialist Centre/Lung Research QLD Pty Ltd
  • Victoria
    • Footscray, Victoria, Australia, 3011
      • Recruiting
      • Lung Research Victoria
    • Melbourne, Victoria, Australia, 3004
      • Recruiting
      • Alfred Health
• Belgium
  • Leuven, Belgium, 3000
    • Recruiting
    • UZ Leuven
  • Liège, Belgium, 4000
    • Recruiting
    • CHU de Liège
• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Z1M9
      • Recruiting
      • Vancouver General Hospital
  • Ontario
    • Ajax, Ontario, Canada, L1S2J5
      • Recruiting
      • Dynamic Drug Advancement
    • Hamilton, Ontario, Canada, L8N 4A6
      • Recruiting
      • St. Joseph's Healthcare Hamilton
  • Quebec
    • Montreal, Quebec, Canada, H4A 3J1
      • Recruiting
      • Research Institute McGill University Health Center
    • Québec, Quebec, Canada, G1V4G5
      • Recruiting
      • Institut Universitaire de Cardiologie et de Pneumologie de Québec-Université Laval
    • Trois-Rivières, Quebec, Canada, G8T7A1
      • Recruiting
      • CIC Mauricie
• France
  • Angers, France, 49933
    • Recruiting
    • CHU Angers
  • Le Kremlin-Bicêtre, France, 94270
    • Recruiting
    • APHP - Hôpital Bicêtre
  • Paris, France, 75014
    • Recruiting
    • Hôpital Paris St Jozeph
  • Pessac, France, 33604
    • Recruiting
    • Hôpital Haut-Lévêque
  • Cedex NA
    • Rennes, Cedex NA, France, 35033
      • Recruiting
      • CHU de Rennes
  • Indre et Loire
    • Tours, Indre et Loire, France, 37000
      • Recruiting
      • CHU Tours
  • Lorraine
    • Metz, Lorraine, France, 57000
      • Recruiting
      • Robert Schuman Hospital UNEOS
  • Lyon
    • Lyon, Lyon, France, 69677
      • Recruiting
      • Louis Pradel Hospital
  • Montpellier
    • Montpellier, Montpellier, France, 34295
      • Recruiting
      • University of Montpellier
• Germany
  • Bavaria
    • Munich, Bavaria, Germany, 81241
      • Recruiting
      • Pneumologisches Studienzentrum München-West
    • München, Bavaria, Germany, 81925
      • Recruiting
      • Muenchen Klinik Bogenhausen - Klinik für Pneumologie und Pneumologische Onkologie
    • Rosenheim, Bavaria, Germany, 83022
      • Recruiting
      • RoMed Klinikum Rosenheim
  • Lower Saxony
    • Hanover, Lower Saxony, Germany, 30625
      • Recruiting
      • Medizinische Hochschule Hannover
  • Main Hessia
    • Frankfurt, Main Hessia, Germany, 60389
      • Recruiting
      • Studienzentrum Dr. Claus Keller
  • North Rhine-Westphalia
    • Essen, North Rhine-Westphalia, Germany, 45239
      • Recruiting
      • Ruhrlandklinik Essen, Klinik fuer Pneumologie
  • Rhineland-Palatinate
    • Mainz, Rhineland-Palatinate, Germany, 55131
      • Recruiting
      • Universitätsmedizin Mainz - Zentrum für Thoraxerkrankungen
  • Saxony
    • Leipzig, Saxony, Germany, 04103
      • Recruiting
      • Uniklinikum Leipzig Medizinische Klinik II - Bereich
  • State of Berlin
    • Berlin, State of Berlin, Germany, 12351
      • Recruiting
      • Vivantes Klinikum Neukölln - Klinik für Innere Medizin - Pneumologie und Infektiologie
• Italy
  • Ancona
    • Torrette, Ancona, Italy, 60126
      • Recruiting
      • A.O.U. delle Marche - Ospedale di Torrette
  • Campania
    • Naples, Campania, Italy, 80131
      • Recruiting
      • Azienda Ospedaliera dei Colli - Ospedale Monaldi
    • Naples, Campania, Italy, 80131
      • Recruiting
      • Università degli Studi di Napoli Federico II - Ospedale Monaldi
  • Emilia-Romagna
    • Bologna, Emilia-Romagna, Italy, 40138
      • Recruiting
      • IRCCS AOU di Bologna - Policlinico Sant' Orsola
  • Giulia
    • Venezia, Giulia, Italy, 34149
      • Recruiting
      • Azienda Sanitaria Universitaria Giuliano Isontina
  • Lombardy
    • Pavia, Lombardy, Italy, 27100
      • Recruiting
      • Fondazione IRCCS Policlinico San Matteo - UOC Pneumologia
  • Milano
    • Milan, Milano, Italy, 20123
      • Recruiting
      • Ospedale San Giuseppe
    • Rozzano, Milano, Italy, 20089
      • Recruiting
      • Humanitas Research Hospital
  • Sicily
    • Catania, Sicily, Italy, 95123
      • Recruiting
      • A.O.U. Policlinico G. Rodolico
• Netherlands
  • Rotterdam, Netherlands, 3015 GD
    • Recruiting
    • Erasmus University Medical Center
  • Utrecht
    • Nieuwegein, Utrecht, Netherlands, 3435
      • Recruiting
      • St Antoniusziekenhuis
• New Zealand
  • Auckland
    • Auckland, Auckland, New Zealand, 1051
      • Recruiting
      • Greenlane Clinical Centre
  • Canterbury
    • Christchurch, Canterbury, New Zealand, 8011
      • Recruiting
      • Health New Zealand
  • Dunedine
    • Dunedin, Dunedine, New Zealand, 9016
      • Recruiting
      • Dunedin Hospital
  • South Tauranga
    • Tauranga, South Tauranga, New Zealand, 3112
      • Recruiting
      • Bay of Plenty Clinical School
• Poland
  • Lodz, Poland, 90-602
    • Recruiting
    • PRYWATNY GABINET LEKARSKI GINEKOLOGIA I POŁOZNICTWO ULTRASONOGRAFIA Prof. Dr hab. Med. Jacek Suzin
  • Lodz, Poland, 90-153
    • Recruiting
    • Department of Pneumology of the University Hospital No 1
  • Bydgoszc
    • Bydgoszcz, Bydgoszc, Poland, 85-079
      • Recruiting
      • Vitamed Galaj i Cichomski sp.j.
  • Krakow
    • Krakow, Krakow, Poland, 30-688
      • Recruiting
      • University Hospital in Krakow
  • Łódź Voivodeship
    • Lodz, Łódź Voivodeship, Poland, 90-349
      • Recruiting
      • AppleTreeClinics Network
• Spain
  • Barcelona, Spain, 08908
    • Recruiting
    • Hospital Universitary de Bellvitge
  • Cadiz, Spain, 11009
    • Recruiting
    • Hospital Universitario Puerta del Mar - Leon-Jimenez
  • Andalusia
    • Granada, Andalusia, Spain, 18014
      • Recruiting
      • Hospital Universitario Virgen de Las Nieves
  • Barcelona
    • Barcelona, Barcelona, Spain, 08025
      • Recruiting
      • Hospital de Sant Pau y la Santa Creu
  • Cantabria
    • Santander, Cantabria, Spain, 39008
      • Recruiting
      • Hospital Universitario Marques de Valdecilla
  • Madrid
    • Madrid, Madrid, Spain, 28034
      • Recruiting
      • Hospital Universitario Ramon y Cajal
    • Madrid, Madrid, Spain, 28007
      • Recruiting
      • Hospital Gregorio Marañon
  • Málaga
    • Málaga, Málaga, Spain, 29010
      • Recruiting
      • Hospital Universitario Virgen de la Victoria
  • Principality of Asturias
    • Oviedo, Principality of Asturias, Spain, 33011
      • Recruiting
      • Hospital Universitario Central de Asturias
  • Sevilla
    • Seville, Sevilla, Spain, 41013
      • Recruiting
      • Hospital Universitario Virgen del Rocio
• Turkey (Türkiye)
  • Adana, Turkey (Türkiye), 01240
    • Recruiting
    • Cukurova University Medical Faculty
  • Ankara, Turkey (Türkiye), 06010
    • Recruiting
    • Health Sciences University, Gulhane Faculty of Medicine
  • İzmir
    • Bornova, İzmir, Turkey (Türkiye), 35100
      • Recruiting
      • Ege University Medical Faculty Hospital
• United Kingdom
  • Cambridge, United Kingdom, CB2 0BB
    • Recruiting
    • Royal Papworth Hospital
  • London, United Kingdom, SW3 6HP
    • Recruiting
    • Royal Brompton Hospital
  • Manchester, United Kingdom, M23 9LT
    • Recruiting
    • Manchester University NHS Foundation Trust
  • Nottingham, United Kingdom, NG5 1PB
    • Recruiting
    • Nottingham University NHS Trust
  • Stoke-on-Trent, United Kingdom, ST4 6QG
    • Recruiting
    • University Hospitals of North Midlands NHS Trust, Royal Stoke University Hospital
  • East Yorkshire
    • Cottingham, East Yorkshire, United Kingdom, HU16 5JQ
      • Recruiting
      • Hull University Teaching Hospital NHS Trust
  • Heartlands
    • Birmingham, Heartlands, United Kingdom, B95SS
      • Recruiting
      • Birmingham Heartlands Hospital
  • Leicester
    • Glenfield, Leicester, United Kingdom, LE3 9QP
      • Recruiting
      • Leicester Biomedical Research Centre - Respiratory Theme
  • London
    • London, London, United Kingdom, sw17 0QT
      • Recruiting
      • St George's University Hospitals NHS Foundation Trust
  • Midlands
    • Birmingham, Midlands, United Kingdom, B15 2GW
      • Recruiting
      • ILD Unit, University Hospitals Birmingham NHS Foundation Trust Theme
  • Northern Ireland
    • Londonderry, Northern Ireland, United Kingdom, BT46
      • Recruiting
      • C-TRIC Altnagelvin Hospital
  • Oxon
    • Oxford, Oxon, United Kingdom, OX3 7LE
      • Recruiting
      • Oxford University Hospitals NHS Foundation Trust
  • South West
    • Exeter, South West, United Kingdom, EX2 5DW
      • Recruiting
      • Royal Devon
  • West Midlands
    • Wolverhampton, West Midlands, United Kingdom, WV10 0QP
      • Recruiting
      • The Royal Wolverhampton NHS Trust, New Cross Hospital, Research and Development
  • West Yorkshire
    • Leeds, West Yorkshire, United Kingdom, LS9 7TF
      • Recruiting
      • St James University Hospital - LTHT
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35205
      • Recruiting
      • University of Alabama at Birmingham
  • Arizona
    • Scottsdale, Arizona, United States, 85259
      • Recruiting
      • Mayo Clinic- Scottsdale
      • Contact: Vivek Nagaraja; Phone Number: 000-000-000
      • Contact: Vivek Nagaraja
  • California
    • Los Angeles, California, United States, 90048
      • Recruiting
      • Cedars-Sinai
    • Los Angeles, California, United States, 90033
      • Recruiting
      • University of Southern California
    • Los Angeles, California, United States, 90095
      • Recruiting
      • UCLA
    • Newport Beach, California, United States, 92663
      • Recruiting
      • Newport Native Md, Inc.
    • Redding, California, United States, 96001
      • Recruiting
      • Paradigm Clinical Research - Redding
    • San Francisco, California, United States, 94143
      • Recruiting
      • University of California - San Francisco
      • Contact: Golden Jeffrey; Phone Number: 000-000-000
      • Contact: Golden Jeffrey
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • University of Colorado, Anschutz Medical Campus
    • Denver, Colorado, United States, 80206
      • Recruiting
      • National Jewish Health
  • Connecticut
    • Farmington, Connecticut, United States, 06030
      • Recruiting
      • UConn Health
    • New Haven, Connecticut, United States, 06519
      • Recruiting
      • Yale University
  • Florida
    • Leesburg, Florida, United States, 34748
      • Recruiting
      • Clinical Site Partners, LCC
    • Ocala, Florida, United States, 34470
      • Recruiting
      • Renstar Medical Research
    • Winter Park, Florida, United States, 32789
      • Recruiting
      • Clinical Site Partners
  • Georgia
    • Atlanta, Georgia, United States, 30309
      • Recruiting
      • Piedmont Healthcare, Inc.
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Recruiting
      • Northwestern University
    • Evanston, Illinois, United States, 60201
      • Recruiting
      • Endeavor Health
    • Maywood, Illinois, United States, 60153
      • Recruiting
      • Loyola University Medical Center
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • Recruiting
      • The University of Kansas Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • Recruiting
      • University of Maryland
    • Baltimore, Maryland, United States, 21224
      • Recruiting
      • Johns Hopkins University
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Recruiting
      • Beth Israel Deaconess Medical Center
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Recruiting
      • University of Michigan
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Recruiting
      • University of Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic Rochester
  • Missouri
    • Hannibal, Missouri, United States, 63401
      • Recruiting
      • Hannibal Regional Healthcare System
  • New York
    • Mount Kisco, New York, United States, 10549
      • Recruiting
      • Northwell Health - Mount Kisco
    • New York, New York, United States, 10029
      • Recruiting
      • ICAHN School of Medicine at Mount Sinai
    • New York, New York, United States, 10032
      • Recruiting
      • Columbia University
    • New York, New York, United States, 10017
      • Recruiting
      • NYU Langone Health
    • New York, New York, United States, 10021
      • Recruiting
      • Weill Cornell
    • The Bronx, New York, United States, 10467
      • Recruiting
      • Montefiore Medical Center
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Recruiting
      • Duke University
    • Statesville, North Carolina, United States, 28625
      • Recruiting
      • Piedmont HealthCare, PA
    • Wilmington, North Carolina, United States, 28401
      • Recruiting
      • Accellacare
    • Winston-Salem, North Carolina, United States, 27103
      • Recruiting
      • Southeastern Research Center
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • Recruiting
      • University of Cincinnati
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Cleveland Clinic
  • Oregon
    • Bend, Oregon, United States, 97701
      • Recruiting
      • Summit Health
    • Portland, Oregon, United States, 97220
      • Recruiting
      • The Oregon Clinic Pulmonary East
    • Portland, Oregon, United States, 97225
      • Recruiting
      • The Oregon Clinic Pulmonary West
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Recruiting
      • The Pennsylvania State University
    • Philadelphia, Pennsylvania, United States, 19107
      • Recruiting
      • Thomas Jefferson University
    • Philadelphia, Pennsylvania, United States, 19140
      • Recruiting
      • Temple University
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina
    • Charleston, South Carolina, United States, 29406
      • Recruiting
      • Lowcountry Lung and Critical Care
  • Tennessee
    • Franklin, Tennessee, United States, 37067
      • Recruiting
      • Clinical Trials Center of Middle Tennessee
    • Nashville, Tennessee, United States, 37204
      • Recruiting
      • Vanderbilt Lung Institute
  • Texas
    • Dallas, Texas, United States, 75390
      • Recruiting
      • UT Southwestern Medical Center
      • Contact: Corey Kershaw; Phone Number: 000-000-000
      • Contact: Corey Kershaw
    • Dallas, Texas, United States, 75246
      • Recruiting
      • Baylor Scott & White Research Institute, Baylor University Medical Center
    • El Paso, Texas, United States, 79902
      • Recruiting
      • El Paso Pulmonary Association
    • Houston, Texas, United States, 77030
      • Recruiting
      • Baylor College of Medicine
    • Houston, Texas, United States, 77030
      • Recruiting
      • The University of Texas Health Science Center
    • McKinney, Texas, United States, 75069
      • Recruiting
      • Metroplex Pulmonary and Sleep Center, PA
  • Utah
    • Murray, Utah, United States, 84157
      • Recruiting
      • Intermountain Medical Center
    • Salt Lake City, Utah, United States, 84108
      • Recruiting
      • University of Utah Health
  • Virginia
    • Falls Church, Virginia, United States, 22042
      • Recruiting
      • Inova Healthcare
  • Washington
    • Seattle, Washington, United States, 98195
      • Recruiting
      • University of Washington

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 based on documented historical spirometry assessments

2. 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
     • 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AP01 High Dose BID; Pirfenidone Solution for Inhalation	Drug: AP01; Oral inhalation solution; Other Names: Pirfenidone Solution
Experimental: AP01 Low Dose BID; Pirfenidone Solution for Inhalation	Drug: AP01; Oral inhalation solution; Other Names: Pirfenidone Solution
Placebo Comparator: Placebo BID; Placebo solution for inhalation	Other: Placebo; Placebo oral inhalation solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	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 Measures

Outcome Measure	Measure Description	Time Frame
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 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 change from baseline in quantitative lung fibrosis score.	Change in lung fibrosis score.	52 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the safety of AP01 high dose and AP01 low dose compared to placebo	Incidents of adverse events (AEs), serious adverse events (SAEs), and treatment-emergent deaths	52 weeks

Collaborators and Investigators

• Sponsor: Avalyn Pharma Inc.
• Collaborators: DevPro Biopharma
• Investigators: Study Director: Avalyn Pharma, Inc., Avalyn Pharma Inc.

Publications and helpful links

General Publications

• West A, Chaudhuri N, Barczyk A, Wilsher ML, Hopkins P, Glaspole I, Corte TJ, Sterclova M, Veale A, Jassem E, Wijsenbeek MS, Grainge C, Piotrowski W, Raghu G, Shaffer ML, Nair D, Freeman L, Otto K, Montgomery AB. Inhaled pirfenidone solution (AP01) for IPF: a randomised, open-label, dose-response trial. Thorax. 2023 Sep;78(9):882-889. doi: 10.1136/thorax-2022-219391. Epub 2023 Mar 22.
• Kolb M, Corte TJ, Feldman J, Nathan SD, Reisner C, Nair D, Woodhead F, Lazarus H, Conoscenti C. Design of the MIST study: a double-blind, randomised, placebo-controlled phase 2b trial of pirfenidone solution for inhalation in patients with progressive pulmonary fibrosis. BMJ Open Respir Res. 2025 Dec 25;12(1):e003059. doi: 10.1136/bmjresp-2024-003059.

Helpful Links

• Content

Study record dates

Study Major Dates

• Study Start (Actual): April 3, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: March 12, 2024
• First Submitted That Met QC Criteria: March 19, 2024
• First Posted (Actual): March 25, 2024

Study Record Updates

• Last Update Posted (Actual): April 6, 2026
• Last Update Submitted That Met QC Criteria: April 1, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Chronic-Fibrosing-ILD with progressive phenotype, PF-ILD
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Respiratory Tract Diseases; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Fibrosis; Pathological Conditions, Signs and Symptoms; Pulmonary Fibrosis; Lung Diseases, Interstitial; Alveolitis, Extrinsic Allergic; Antineoplastic Agents; Physiological Effects of Drugs; Anti-Inflammatory Agents; Peripheral Nervous System Agents; Antirheumatic Agents; Sensory System Agents; Analgesics, Non-Narcotic; Analgesics; Anti-Inflammatory Agents, Non-Steroidal; Pirfenidone
• Other Study ID Numbers: AP01-007

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No