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

NCT06329401 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: AP01-007
• Study Type: interventional
• Target: 375
• Locations: 14 countries
• Sites: 154

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.

Conditions

Progressive Pulmonary Fibrosis; Pulmonary 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

Keywords

Chronic-Fibrosing-ILD with progressive phenotype, PF-ILD

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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

Other Outcomes (1)

• To evaluate the safety of AP01 high dose and AP01 low dose compared to placebo

  52 weeks

Study Arms (3)

AP01 High Dose BID

EXPERIMENTAL

Pirfenidone Solution for Inhalation

Drug: AP01

AP01 Low Dose BID

EXPERIMENTAL

Pirfenidone Solution for Inhalation

Drug: AP01

Placebo BID

PLACEBO COMPARATOR

Placebo solution for inhalation

Other: Placebo

Interventions

AP01 DRUG

Oral inhalation solution

Also known as: Pirfenidone Solution

AP01 High Dose BID; AP01 Low Dose BID

Placebo OTHER

Placebo oral inhalation solution

Placebo BID

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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:

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Avalyn Pharma Inc.: lead
• DevPro Biopharma: collaborator

Study Sites (154)

University of Alabama at Birmingham

Birmingham, Alabama, 35205, United States

Location

Mayo Clinic- Scottsdale

Scottsdale, Arizona, 85259, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

Cedars-Sinai

Los Angeles, California, 90048, United States

Location

UCLA

Los Angeles, California, 90095, United States

Location

Newport Native MD, Inc.

Newport Beach, California, 92663, United States

Location

Paradigm Clinical Research - Redding

Redding, California, 96001, United States

Location

University of California - San Francisco

San Francisco, California, 94143, United States

Location

University of Colorado, Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

UCONN Health

Farmington, Connecticut, 06030, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

Clinical Site Partners, LCC

Leesburg, Florida, 34748, United States

Location

Renstar Medical Research

Ocala, Florida, 34470, United States

Location

Clinical Site Partners

Winter Park, Florida, 32789, United States

Location

Piedmont Healthcare, Inc.

Atlanta, Georgia, 30309, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Endeavor Health

Evanston, Illinois, 60201, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

The University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21224, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

Location

Hannibal Regional Healthcare System

Hannibal, Missouri, 63401, United States

Location

Northern Westchester Hospital

Mount Kisco, New York, 10549, United States

Location

Northwell Health - Mount Kisco

Mount Kisco, New York, 10549, United States

Location

NYU Langone Health

New York, New York, 10017, United States

Location

Weill Cornell

New York, New York, 10021, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Piedmont HealthCare, PA

Statesville, North Carolina, 28625, United States

Location

Accellacare

Wilmington, North Carolina, 28401, United States

Location

Southeastern Research Center

Winston-Salem, North Carolina, 27103, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

The Oregon Clinic Pulmonary West

Beaverton, Oregon, 97008, United States

Location

Summit Health

Bend, Oregon, 97701, United States

Location

The Oregon Clinic Pulmonary East

Portland, Oregon, 97220, United States

Location

The Oregon Clinic Pulmonary West

Portland, Oregon, 97225, United States

Location

The Pennsylvania State University

Hershey, Pennsylvania, 17033, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Temple University

Philadelphia, Pennsylvania, 19140, United States

Location

Lowcountry Lung and Critical Care

Charleston, South Carolina, 29406, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Clinical Trials Center of Middle Tennessee

Franklin, Tennessee, 37067, United States

Location

Vanderbilt Lung Institute

Nashville, Tennessee, 37204, United States

Location

Baylor Scott & White Research Institute, Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

El Paso Pulmonary Association

El Paso, Texas, 79902, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

The University of Texas Health Science Center

Houston, Texas, 77030, United States

Location

Metroplex Pulmonary and Sleep Center, PA

McKinney, Texas, 75069, United States

Location

Intermountain Medical Center

Murray, Utah, 84157, United States

Location

University of Utah Health

Salt Lake City, Utah, 84108, United States

Location

Inova Healthcare

Falls Church, Virginia, 22042, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

Fundacion Respirar

Buenos Aires, Buenos Aires, 1427, Argentina

Location

CINME

Buenos Aires, Buenos Aires, C1056AB, Argentina

Location

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Buenos Aires, C1199ABH, Argentina

Location

Instituto Ave Pulmo, Fundacion Enfisema

Mar del Plata, Buenos Aires, 7600, Argentina

Location

Clinica Monte Grande

Monte Grande, Buenos Aires, 1842, Argentina

Location

Centro Respiratorio Quilmes

Quilmes, Buenos Aires, B1878, Argentina

Location

Instituto de Medicina Respiratoria

Córdoba, Córdoba Province, 5003, Argentina

Location

CIMER Centro Integral de Medicina Respiratoria

San Miguel de Tucumán, Tucumán Province, 4000, Argentina

Location

Investigaciones En Patologias Respiratorias

San Miguel de Tucumán, Tucumán Province, 4000, Argentina

Location

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

Location

Nepean Lung & Sleep

Kingswood, New South Wales, 2745, Australia

Location

John Hunter Hospital

New Lambton, New South Wales, 2305, Australia

Location

Macquarie University Clinical Trials Unit

Sydney, New South Wales, 2109, Australia

Location

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Institute for Respiratory Health

Nedlands, Perth West Australia, 6009, Australia

Location

Wallace Street Specialist Centre/Lung Research QLD Pty Ltd

Brisbane, Queensland, 4032, Australia

Location

Lung Research Victoria

Footscray, Victoria, 3011, Australia

Location

Alfred Health

Melbourne, Victoria, 3004, Australia

Location

Canberra Hospital

Canberra, 2605, Australia

Location

UZ Leuven

Leuven, 3000, Belgium

Location

CHU de Liège

Liège, 4000, Belgium

Location

Vancouver General Hospital

Vancouver, British Columbia, V5Z1M9, Canada

Location

Dynamic Drug Advancement

Ajax, Ontario, L1S2J5, Canada

Location

St. Joseph's Healthcare Hamilton

Hamilton, Ontario, L8N 4A6, Canada

Location

Research Institute McGill University Health Center

Montreal, Quebec, H4A 3J1, Canada

Location

Institut universitaire de cardiologie et de pneumologie de Québec-Université Laval

Québec, Quebec, G1V4G5, Canada

Location

CIC Mauricie

Trois-Rivières, Quebec, G8T7A1, Canada

Location

CHU de Rennes

Rennes, Cedex NA, 35033, France

Location

CHU Tours

Tours, Indre et Loire, 37000, France

Location

Robert Schuman Hospital UNEOS

Metz, Lorraine, 57000, France

Location

Louis Pradel Hospital

Lyon, Lyon, 69677, France

Location

University of Montpellier

Montpellier, Montpellier, 34295, France

Location

CHU Angers

Angers, 49933, France

Location

APHP - Hopital Bicetre

Le Kremlin-Bicêtre, 94270, France

Location

Hôpital Paris St Jozeph

Paris, 75014, France

Location

Hôpital Haut-Lévêque

Pessac, 33604, France

Location

Pneumologisches Studienzentrum München-West

Munich, Bavaria, 81241, Germany

Location

Muenchen Klinik Bogenhausen - Klinik für Pneumologie und Pneumologische Onkologie

München, Bavaria, 81925, Germany

Location

RoMed Klinikum Rosenheim

Rosenheim, Bavaria, 83022, Germany

Location

Medizinische Hochschule Hannover

Hanover, Lower Saxony, 30625, Germany

Location

Studienzentrum Dr. Claus Keller

Frankfurt, Main Hessia, 60389, Germany

Location

Ruhrlandklinik Essen, Klinik fuer Pneumologie

Essen, North Rhine-Westphalia, 45239, Germany

Location

Universitätsmedizin Mainz - Zentrum für Thoraxerkrankungen

Mainz, Rhineland-Palatinate, 55131, Germany

Location

Uniklinikum Leipzig Medizinische Klinik II - Bereich

Leipzig, Saxony, 04103, Germany

Location

Vivantes Klinikum Neukölln - Klinik für Innere Medizin - Pneumologie und Infektiologie

Berlin, State of Berlin, 12351, Germany

Location

A.O.U. delle Marche - Ospedale di Torrette

Torrette, Ancona, 60126, Italy

Location

Azienda Ospedaliera dei Colli - Ospedale Monaldi

Naples, Campania, 80131, Italy

Location

Università degli Studi di Napoli Federico II - Ospedale Monaldi

Naples, Campania, 80131, Italy

Location

IRCCS AOU di Bologna - Policlinico Sant' Orsola

Bologna, Emilia-Romagna, 40138, Italy

Location

Azienda Sanitaria Universitaria Giuliano Isontina

Venezia, Giulia, 34149, Italy

Location

Fondazione IRCCS Policlinico San Matteo - UOC Pneumologia

Pavia, Lombardy, 27100, Italy

Location

Ospedale San Giuseppe

Milan, Milano, 20123, Italy

Location

HUMANITAS Research Hospital

Rozzano, Milano, 20089, Italy

Location

A.O.U. Policlinico G. Rodolico

Catania, Sicily, 95123, Italy

Location

St Antoniusziekenhuis

Nieuwegein, Utrecht, 3435, Netherlands

Location

Erasmus University Medical Center

Rotterdam, 3015 GD, Netherlands

Location

Greenlane Clinical Centre

Auckland, Auckland, 1051, New Zealand

Location

Health New Zealand

Christchurch, Canterbury, 8011, New Zealand

Location

Dunedin Hospital

Dunedin, Dunedine, 9016, New Zealand

Location

Bay of Plenty Clinical School

Tauranga, South Tauranga, 3112, New Zealand

Location

Vitamed Galaj I Cichomski sp.j.

Bydgoszcz, Bydgoszc, 85-079, Poland

Location

University Hospital in Krakow

Krakow, Krakow, 30-688, Poland

Location

Department of Pneumology of the University Hospital No 1

Lodz, 90-153, Poland

Location

PRYWATNY GABINET LEKARSKI GINEKOLOGIA I POŁOZNICTWO ULTRASONOGRAFIA Prof. Dr hab. Med. Jacek Suzin

Lodz, 90-602, Poland

Location

AppleTreeClinics Network

Lodz, Łódź Voivodeship, 90-349, Poland

Location

Hospital Universitario Virgen de las Nieves

Granada, Andalusia, 18014, Spain

Location

Hospital de Sant Pau y la Santa Creu

Barcelona, Barcelona, 08025, Spain

Location

Hospital Universitario Marqués de Valdecilla

Santander, Cantabria, 39008, Spain

Location

Hospital Gregorio Marañon

Madrid, Madrid, 28007, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, Madrid, 28034, Spain

Location

Hospital Universitario Virgen de la Victoria

Málaga, Málaga, 29010, Spain

Location

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33011, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, Sevilla, 41013, Spain

Location

Hospital Universitary de Bellvitge

Barcelona, 08908, Spain

Location

Hospital Universitario Puerta del Mar - Leon-Jimenez

Cadiz, 11009, Spain

Location

Ege University Medical Faculty Hospital

Bornova, İzmir, 35100, Turkey (Türkiye)

Location

Cukurova University Medical Faculty

Adana, 01240, Turkey (Türkiye)

Location

Health Sciences University, Gulhane Faculty of Medicine

Ankara, 06010, Turkey (Türkiye)

Location

Hull University Teaching Hospital NHS Trust

Cottingham, East Yorkshire, HU16 5JQ, United Kingdom

Location

Birmingham Heartlands Hospital

Birmingham, Heartlands, B95SS, United Kingdom

Location

Leicester Biomedical Research Centre - Respiratory Theme

Glenfield, Leicester, LE3 9QP, United Kingdom

Location

St George's University Hospitals NHS Foundation Trust

London, London, sw17 0QT, United Kingdom

Location

ILD Unit, University Hospitals Birmingham NHS Foundation Trust Theme

Birmingham, Midlands, B15 2GW, United Kingdom

Location

C-TRIC Altnagelvin Hospital

Londonderry, Northern Ireland, BT46, United Kingdom

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxon, OX3 7LE, United Kingdom

Location

Royal Devon

Exeter, South West, EX2 5DW, United Kingdom

Location

The Royal Wolverhampton NHS Trust, New Cross Hospital, Research and Development

Wolverhampton, West Midlands, WV10 0QP, United Kingdom

Location

St James University Hospital - LTHT

Leeds, West Yorkshire, LS9 7TF, United Kingdom

Location

Royal Papworth Hospital

Cambridge, CB2 0BB, United Kingdom

Location

Royal Brompton Hospital

London, SW3 6HP, United Kingdom

Location

Manchester University NHS Foundation Trust

Manchester, M23 9LT, United Kingdom

Location

Nottingham University NHS Trust

Nottingham, NG5 1PB, United Kingdom

Location

University Hospitals of North Midlands NHS Trust, Royal Stoke University Hospital

Stoke-on-Trent, ST4 6QG, United Kingdom

Location

Related Publications (2)

• 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.

  PMID: 36948586 RESULT

• 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.

  PMID: 41448793 DERIVED

Related Links

• Content

MeSH Terms

Conditions

Pulmonary Fibrosis; Lung Diseases, Interstitial; Alveolitis, Extrinsic Allergic

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Study Officials

• Avalyn Pharma, Inc.

  Avalyn Pharma Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 12, 2024
• First Posted: March 25, 2024
• Study Start: April 3, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: July 2, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (9); ES (10); BE (2); NL (2); PL (5); GB (15); DE (9); AU (10); NZ (4); TU (3); US (61); IT (9); AR (9); CA (6)