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

NCT06329401 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: AP01-007
• Study Type: interventional
• Target: 375
• Locations: 14 countries
• Sites: 152

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

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 (152)

University of Alabama at Birmingham

Birmingham, Alabama, 35205, United States

RECRUITING

Mayo Clinic- Scottsdale

Scottsdale, Arizona, 85259, United States

RECRUITING

University of Southern California

Los Angeles, California, 90033, United States

RECRUITING

Cedars-Sinai

Los Angeles, California, 90048, United States

RECRUITING

UCLA

Los Angeles, California, 90095, United States

RECRUITING

Newport Native MD, Inc.

Newport Beach, California, 92663, United States

RECRUITING

Paradigm Clinical Research - Redding

Redding, California, 96001, United States

RECRUITING

University of California - San Francisco

San Francisco, California, 94143, United States

RECRUITING

University of Colorado, Anschutz Medical Campus

Aurora, Colorado, 80045, United States

RECRUITING

National Jewish Health

Denver, Colorado, 80206, United States

RECRUITING

UCONN Health

Farmington, Connecticut, 06030, United States

RECRUITING

Yale University

New Haven, Connecticut, 06519, United States

RECRUITING

Clinical Site Partners, LCC

Leesburg, Florida, 34748, United States

RECRUITING

Renstar Medical Research

Ocala, Florida, 34470, United States

RECRUITING

Clinical Site Partners

Winter Park, Florida, 32789, United States

RECRUITING

Piedmont Healthcare, Inc.

Atlanta, Georgia, 30309, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

Endeavor Health

Evanston, Illinois, 60201, United States

RECRUITING

Loyola University Medical Center

Maywood, Illinois, 60153, United States

RECRUITING

The University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

University of Maryland

Baltimore, Maryland, 21201, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21224, United States

RECRUITING

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Hannibal Regional Healthcare System

Hannibal, Missouri, 63401, United States

RECRUITING

Northwell Health - Mount Kisco

Mount Kisco, New York, 10549, United States

RECRUITING

NYU Langone Health

New York, New York, 10017, United States

RECRUITING

Weill Cornell

New York, New York, 10021, United States

RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

Columbia University

New York, New York, 10032, United States

RECRUITING

Montefiore Medical Center

The Bronx, New York, 10467, United States

RECRUITING

Duke University

Durham, North Carolina, 27710, United States

RECRUITING

Piedmont HealthCare, PA

Statesville, North Carolina, 28625, United States

RECRUITING

Accellacare

Wilmington, North Carolina, 28401, United States

RECRUITING

Southeastern Research Center

Winston-Salem, North Carolina, 27103, United States

RECRUITING

University of Cincinnati

Cincinnati, Ohio, 45267, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Summit Health

Bend, Oregon, 97701, United States

RECRUITING

The Oregon Clinic Pulmonary East

Portland, Oregon, 97220, United States

RECRUITING

The Oregon Clinic Pulmonary West

Portland, Oregon, 97225, United States

RECRUITING

The Pennsylvania State University

Hershey, Pennsylvania, 17033, United States

RECRUITING

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Temple University

Philadelphia, Pennsylvania, 19140, United States

RECRUITING

Lowcountry Lung and Critical Care

Charleston, South Carolina, 29406, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Clinical Trials Center of Middle Tennessee

Franklin, Tennessee, 37067, United States

RECRUITING

Vanderbilt Lung Institute

Nashville, Tennessee, 37204, United States

RECRUITING

Baylor Scott & White Research Institute, Baylor University Medical Center

Dallas, Texas, 75246, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

El Paso Pulmonary Association

El Paso, Texas, 79902, United States

RECRUITING

Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

The University of Texas Health Science Center

Houston, Texas, 77030, United States

RECRUITING

Metroplex Pulmonary and Sleep Center, PA

McKinney, Texas, 75069, United States

RECRUITING

Intermountain Medical Center

Murray, Utah, 84157, United States

RECRUITING

University of Utah Health

Salt Lake City, Utah, 84108, United States

RECRUITING

Inova Healthcare

Falls Church, Virginia, 22042, United States

RECRUITING

University of Washington

Seattle, Washington, 98195, United States

RECRUITING

Fundacion Respirar

Buenos Aires, Buenos Aires, 1427, Argentina

RECRUITING

CINME

Buenos Aires, Buenos Aires, C1056AB, Argentina

RECRUITING

Hospital Italiano de Buenos Aires

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

RECRUITING

Instituto Ave Pulmo, Fundacion Enfisema

Mar del Plata, Buenos Aires, 7600, Argentina

RECRUITING

Clinica Monte Grande

Monte Grande, Buenos Aires, 1842, Argentina

RECRUITING

Centro Respiratorio Quilmes

Quilmes, Buenos Aires, B1878, Argentina

RECRUITING

Instituto de Medicina Respiratoria

Córdoba, Córdoba Province, 5003, Argentina

RECRUITING

CIMER Centro Integral de Medicina Respiratoria

San Miguel de Tucumán, Tucumán Province, 4000, Argentina

RECRUITING

Investigaciones En Patologias Respiratorias

San Miguel de Tucumán, Tucumán Province, 4000, Argentina

RECRUITING

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

RECRUITING

Nepean Lung & Sleep

Kingswood, New South Wales, 2745, Australia

RECRUITING

John Hunter Hospital

New Lambton, New South Wales, 2305, Australia

RECRUITING

Macquarie University Clinical Trials Unit

Sydney, New South Wales, 2109, Australia

RECRUITING

Westmead Hospital

Westmead, New South Wales, 2145, Australia

RECRUITING

Institute for Respiratory Health

Nedlands, Perth West Australia, 6009, Australia

RECRUITING

Wallace Street Specialist Centre/Lung Research QLD Pty Ltd

Brisbane, Queensland, 4032, Australia

RECRUITING

Lung Research Victoria

Footscray, Victoria, 3011, Australia

RECRUITING

Alfred Health

Melbourne, Victoria, 3004, Australia

RECRUITING

Canberra Hospital

Canberra, 2605, Australia

RECRUITING

UZ Leuven

Leuven, 3000, Belgium

RECRUITING

CHU de Liège

Liège, 4000, Belgium

RECRUITING

Vancouver General Hospital

Vancouver, British Columbia, V5Z1M9, Canada

RECRUITING

Dynamic Drug Advancement

Ajax, Ontario, L1S2J5, Canada

RECRUITING

St. Joseph's Healthcare Hamilton

Hamilton, Ontario, L8N 4A6, Canada

RECRUITING

Research Institute McGill University Health Center

Montreal, Quebec, H4A 3J1, Canada

RECRUITING

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

Québec, Quebec, G1V4G5, Canada

RECRUITING

CIC Mauricie

Trois-Rivières, Quebec, G8T7A1, Canada

RECRUITING

CHU de Rennes

Rennes, Cedex NA, 35033, France

RECRUITING

CHU Tours

Tours, Indre et Loire, 37000, France

RECRUITING

Robert Schuman Hospital UNEOS

Metz, Lorraine, 57000, France

RECRUITING

Louis Pradel Hospital

Lyon, Lyon, 69677, France

RECRUITING

University of Montpellier

Montpellier, Montpellier, 34295, France

RECRUITING

CHU Angers

Angers, 49933, France

RECRUITING

APHP - Hopital Bicetre

Le Kremlin-Bicêtre, 94270, France

RECRUITING

Hôpital Paris St Jozeph

Paris, 75014, France

RECRUITING

Hôpital Haut-Lévêque

Pessac, 33604, France

RECRUITING

Pneumologisches Studienzentrum München-West

Munich, Bavaria, 81241, Germany

RECRUITING

Muenchen Klinik Bogenhausen - Klinik für Pneumologie und Pneumologische Onkologie

München, Bavaria, 81925, Germany

RECRUITING

RoMed Klinikum Rosenheim

Rosenheim, Bavaria, 83022, Germany

RECRUITING

Medizinische Hochschule Hannover

Hanover, Lower Saxony, 30625, Germany

RECRUITING

Studienzentrum Dr. Claus Keller

Frankfurt, Main Hessia, 60389, Germany

RECRUITING

Ruhrlandklinik Essen, Klinik fuer Pneumologie

Essen, North Rhine-Westphalia, 45239, Germany

RECRUITING

Universitätsmedizin Mainz - Zentrum für Thoraxerkrankungen

Mainz, Rhineland-Palatinate, 55131, Germany

RECRUITING

Uniklinikum Leipzig Medizinische Klinik II - Bereich

Leipzig, Saxony, 04103, Germany

RECRUITING

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

Berlin, State of Berlin, 12351, Germany

RECRUITING

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

Torrette, Ancona, 60126, Italy

RECRUITING

Azienda Ospedaliera dei Colli - Ospedale Monaldi

Naples, Campania, 80131, Italy

RECRUITING

Università degli Studi di Napoli Federico II - Ospedale Monaldi

Naples, Campania, 80131, Italy

RECRUITING

IRCCS AOU di Bologna - Policlinico Sant' Orsola

Bologna, Emilia-Romagna, 40138, Italy

RECRUITING

Azienda Sanitaria Universitaria Giuliano Isontina

Venezia, Giulia, 34149, Italy

RECRUITING

Fondazione IRCCS Policlinico San Matteo - UOC Pneumologia

Pavia, Lombardy, 27100, Italy

RECRUITING

Ospedale San Giuseppe

Milan, Milano, 20123, Italy

RECRUITING

HUMANITAS Research Hospital

Rozzano, Milano, 20089, Italy

RECRUITING

A.O.U. Policlinico G. Rodolico

Catania, Sicily, 95123, Italy

RECRUITING

St Antoniusziekenhuis

Nieuwegein, Utrecht, 3435, Netherlands

RECRUITING

Erasmus University Medical Center

Rotterdam, 3015 GD, Netherlands

RECRUITING

Greenlane Clinical Centre

Auckland, Auckland, 1051, New Zealand

RECRUITING

Health New Zealand

Christchurch, Canterbury, 8011, New Zealand

RECRUITING

Dunedin Hospital

Dunedin, Dunedine, 9016, New Zealand

RECRUITING

Bay of Plenty Clinical School

Tauranga, South Tauranga, 3112, New Zealand

RECRUITING

Vitamed Galaj I Cichomski sp.j.

Bydgoszcz, Bydgoszc, 85-079, Poland

RECRUITING

University Hospital in Krakow

Krakow, Krakow, 30-688, Poland

RECRUITING

Department of Pneumology of the University Hospital No 1

Lodz, 90-153, Poland

RECRUITING

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

Lodz, 90-602, Poland

RECRUITING

AppleTreeClinics Network

Lodz, Łódź Voivodeship, 90-349, Poland

RECRUITING

Hospital Universitario Virgen de las Nieves

Granada, Andalusia, 18014, Spain

RECRUITING

Hospital de Sant Pau y la Santa Creu

Barcelona, Barcelona, 08025, Spain

RECRUITING

Hospital Universitario Marqués de Valdecilla

Santander, Cantabria, 39008, Spain

RECRUITING

Hospital Gregorio Marañon

Madrid, Madrid, 28007, Spain

RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, Madrid, 28034, Spain

RECRUITING

Hospital Universitario Virgen de la Victoria

Málaga, Málaga, 29010, Spain

RECRUITING

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33011, Spain

RECRUITING

Hospital Universitario Virgen del Rocío

Seville, Sevilla, 41013, Spain

RECRUITING

Hospital Universitary de Bellvitge

Barcelona, 08908, Spain

RECRUITING

Hospital Universitario Puerta del Mar - Leon-Jimenez

Cadiz, 11009, Spain

RECRUITING

Ege University Medical Faculty Hospital

Bornova, İzmir, 35100, Turkey (Türkiye)

RECRUITING

Cukurova University Medical Faculty

Adana, 01240, Turkey (Türkiye)

RECRUITING

Health Sciences University, Gulhane Faculty of Medicine

Ankara, 06010, Turkey (Türkiye)

RECRUITING

Hull University Teaching Hospital NHS Trust

Cottingham, East Yorkshire, HU16 5JQ, United Kingdom

RECRUITING

Birmingham Heartlands Hospital

Birmingham, Heartlands, B95SS, United Kingdom

RECRUITING

Leicester Biomedical Research Centre - Respiratory Theme

Glenfield, Leicester, LE3 9QP, United Kingdom

RECRUITING

St George's University Hospitals NHS Foundation Trust

London, London, sw17 0QT, United Kingdom

RECRUITING

ILD Unit, University Hospitals Birmingham NHS Foundation Trust Theme

Birmingham, Midlands, B15 2GW, United Kingdom

RECRUITING

C-TRIC Altnagelvin Hospital

Londonderry, Northern Ireland, BT46, United Kingdom

RECRUITING

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxon, OX3 7LE, United Kingdom

RECRUITING

Royal Devon

Exeter, South West, EX2 5DW, United Kingdom

RECRUITING

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

Wolverhampton, West Midlands, WV10 0QP, United Kingdom

RECRUITING

St James University Hospital - LTHT

Leeds, West Yorkshire, LS9 7TF, United Kingdom

RECRUITING

Royal Papworth Hospital

Cambridge, CB2 0BB, United Kingdom

RECRUITING

Royal Brompton Hospital

London, SW3 6HP, United Kingdom

RECRUITING

Manchester University NHS Foundation Trust

Manchester, M23 9LT, United Kingdom

RECRUITING

Nottingham University NHS Trust

Nottingham, NG5 1PB, United Kingdom

RECRUITING

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

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

RECRUITING

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

Study Officials

• Avalyn Pharma, Inc.

  Avalyn Pharma Inc.

  STUDY DIRECTOR

Central Study Contacts

Craig S. Conoscenti, MD

CONTACT

206-707-0304; cconoscenti@avalynpharma.com

Daniele Tompkins

CONTACT

973-983-3700; dtompkins@devprobiopharma.com

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: March 18, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

FR (9); ES (10); BE (2); NL (2); PL (5); GB (15); DE (9); CA (6); AU (10); AR (9); NZ (4); IT (9); US (59); TU (3)