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

NCT ID: NCT06329401

Last Updated: 2025-11-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

375 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-03

Study Completion Date

2027-06-30

Brief Summary

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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

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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.

Conditions

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Progressive Pulmonary Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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AP01 High Dose BID

Pirfenidone Solution for Inhalation

Group Type EXPERIMENTAL

AP01

Intervention Type DRUG

Oral inhalation solution

AP01 Low Dose BID

Pirfenidone Solution for Inhalation

Group Type EXPERIMENTAL

AP01

Intervention Type DRUG

Oral inhalation solution

Placebo BID

Placebo solution for inhalation

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo oral inhalation solution

Interventions

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AP01

Oral inhalation solution

Intervention Type DRUG

Placebo

Placebo oral inhalation solution

Intervention Type OTHER

Other Intervention Names

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Pirfenidone Solution

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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DevPro Biopharma

INDUSTRY

Sponsor Role collaborator

Avalyn Pharma Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Avalyn Pharma, Inc.

Role: STUDY_DIRECTOR

Avalyn Pharma Inc.

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status RECRUITING

Pulmonary Associates, PA

Phoenix, Arizona, United States

Site Status RECRUITING

University of Southern California

Los Angeles, California, United States

Site Status RECRUITING

Cedars-Sinai

Los Angeles, California, United States

Site Status RECRUITING

UCLA

Los Angeles, California, United States

Site Status RECRUITING

Newport Native MD, Inc.

Newport Beach, California, United States

Site Status RECRUITING

Paradigm Clinical Research - Redding

Redding, California, United States

Site Status RECRUITING

University of Colorado, Anschutz Medical Campus

Aurora, Colorado, United States

Site Status RECRUITING

National Jewish Health

Denver, Colorado, United States

Site Status RECRUITING

UCONN Health

Farmington, Connecticut, United States

Site Status RECRUITING

Yale University

New Haven, Connecticut, United States

Site Status RECRUITING

Clinical Site Partners, LCC

Leesburg, Florida, United States

Site Status RECRUITING

Renstar Medical Research

Ocala, Florida, United States

Site Status RECRUITING

Central Florida Pulmonary Group

Orlando, Florida, United States

Site Status RECRUITING

SEC Clinical Research

Pensacola, Florida, United States

Site Status RECRUITING

Clinical Site Partners

Winter Park, Florida, United States

Site Status RECRUITING

Piedmont Healthcare, Inc.

Atlanta, Georgia, United States

Site Status RECRUITING

Northwestern University

Chicago, Illinois, United States

Site Status RECRUITING

Endeavor Health

Evanston, Illinois, United States

Site Status RECRUITING

Loyola University Medical Center

Maywood, Illinois, United States

Site Status RECRUITING

The University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status RECRUITING

University of Maryland

Baltimore, Maryland, United States

Site Status RECRUITING

Johns Hopkins University

Baltimore, Maryland, United States

Site Status RECRUITING

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status RECRUITING

Hannibal Regional Healthcare System

Hannibal, Missouri, United States

Site Status RECRUITING

Northwell Health - Mount Kisco

Mount Kisco, New York, United States

Site Status RECRUITING

NYU Langone Health

New York, New York, United States

Site Status RECRUITING

Weill Cornell

New York, New York, United States

Site Status RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status RECRUITING

Columbia University

New York, New York, United States

Site Status RECRUITING

Montefiore Medical Center

The Bronx, New York, United States

Site Status RECRUITING

Duke University

Durham, North Carolina, United States

Site Status RECRUITING

Pulmonix

Greensboro, North Carolina, United States

Site Status RECRUITING

Piedmont HealthCare, PA

Statesville, North Carolina, United States

Site Status RECRUITING

Accellacare

Wilmington, North Carolina, United States

Site Status RECRUITING

Southeastern Research Center

Winston-Salem, North Carolina, United States

Site Status RECRUITING

University of Cincinnati

Cincinnati, Ohio, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Summit Health

Bend, Oregon, United States

Site Status RECRUITING

The Oregon Clinic Pulmonary East

Portland, Oregon, United States

Site Status RECRUITING

The Oregon Clinic Pulmonary West

Portland, Oregon, United States

Site Status RECRUITING

The Pennsylvania State University

Hershey, Pennsylvania, United States

Site Status RECRUITING

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Temple University

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Lowcountry Lung and Critical Care

Charleston, South Carolina, United States

Site Status RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status RECRUITING

Clinical Trials Center of Middle Tennessee

Franklin, Tennessee, United States

Site Status RECRUITING

Vanderbilt Lung Institute

Nashville, Tennessee, United States

Site Status RECRUITING

Baylor Scott & White Research Institute, Baylor University Medical Center

Dallas, Texas, United States

Site Status RECRUITING

El Paso Pulmonary Association

El Paso, Texas, United States

Site Status RECRUITING

Baylor College of Medicine

Houston, Texas, United States

Site Status RECRUITING

The University of Texas Health Science Center

Houston, Texas, United States

Site Status RECRUITING

Metroplex Pulmonary and Sleep Center, PA

McKinney, Texas, United States

Site Status RECRUITING

Intermountain Medical Center

Murray, Utah, United States

Site Status RECRUITING

University of Utah Health

Salt Lake City, Utah, United States

Site Status RECRUITING

Inova Healthcare

Falls Church, Virginia, United States

Site Status RECRUITING

University of Washington

Seattle, Washington, United States

Site Status RECRUITING

Fundacion Respirar

Buenos Aires, Buenos Aires, Argentina

Site Status RECRUITING

CINME

Buenos Aires, Buenos Aires, Argentina

Site Status RECRUITING

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

Site Status RECRUITING

Instituto Ave Pulmo, Fundacion Enfisema

Mar del Plata, Buenos Aires, Argentina

Site Status RECRUITING

Centro Respiratorio Quilmes

Quilmes, Buenos Aires, Argentina

Site Status RECRUITING

Instituto de Medicina Respiratoria

Córdoba, Córdoba Province, Argentina

Site Status RECRUITING

CIMER Centro Integral de Medicina Respiratoria

San Miguel de Tucumán, Tucumán Province, Argentina

Site Status RECRUITING

Investigaciones En Patologias Respiratorias

San Miguel de Tucumán, Tucumán Province, Argentina

Site Status RECRUITING

Royal Prince Alfred Hospital

Camperdown, New South Wales, Australia

Site Status RECRUITING

Nepean Lung & Sleep

Kingswood, New South Wales, Australia

Site Status RECRUITING

John Hunter Hospital

New Lambton, New South Wales, Australia

Site Status RECRUITING

Macquarie University Clinical Trials Unit

Sydney, New South Wales, Australia

Site Status RECRUITING

Westmead Hospital

Westmead, New South Wales, Australia

Site Status RECRUITING

Institute for Respiratory Health

Nedlands, Perth West Australia, Australia

Site Status RECRUITING

Wallace Street Specialist Centre/Lung Research QLD Pty Ltd

Brisbane, Queensland, Australia

Site Status RECRUITING

Lung Research Victoria

Footscray, Victoria, Australia

Site Status RECRUITING

Alfred Health

Melbourne, Victoria, Australia

Site Status RECRUITING

Canberra Hospital

Canberra, , Australia

Site Status RECRUITING

Vancouver General Hospital

Vancouver, British Columbia, Canada

Site Status RECRUITING

Dynamic Drug Advancement

Ajax, Ontario, Canada

Site Status RECRUITING

St. Joseph's Healthcare Hamilton

Hamilton, Ontario, Canada

Site Status RECRUITING

Research Institute McGill University Health Center

Montreal, Quebec, Canada

Site Status RECRUITING

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

Québec, Quebec, Canada

Site Status RECRUITING

CIC Mauricie

Trois-Rivières, Quebec, Canada

Site Status RECRUITING

CHU de Rennes

Rennes, Cedex NA, France

Site Status RECRUITING

CHU Tours

Tours, Indre et Loire, France

Site Status RECRUITING

Robert Schuman Hospital UNEOS

Metz, Lorraine, France

Site Status RECRUITING

Louis Pradel Hospital

Lyon, Lyon, France

Site Status RECRUITING

University of Montpellier

Montpellier, Montpellier, France

Site Status RECRUITING

CHU Angers

Angers, , France

Site Status RECRUITING

APHP - Hopital Bicetre

Le Kremlin-Bicêtre, , France

Site Status RECRUITING

Hôpital Paris St Jozeph

Paris, , France

Site Status RECRUITING

Hôpital Haut-Lévêque

Pessac, , France

Site Status RECRUITING

Pneumologisches Studienzentrum München-West

Munich, Bavaria, Germany

Site Status RECRUITING

RoMed Klinikum Rosenheim

Rosenheim, Bavaria, Germany

Site Status RECRUITING

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Site Status RECRUITING

Studienzentrum Dr. Claus Keller

Frankfurt, Main Hessia, Germany

Site Status RECRUITING

Ruhrlandklinik Essen, Klinik fuer Pneumologie

Essen, North Rhine-Westphalia, Germany

Site Status RECRUITING

Universitätsmedizin Mainz - Zentrum für Thoraxerkrankungen

Mainz, Rhineland-Palatinate, Germany

Site Status RECRUITING

Uniklinikum Leipzig Medizinische Klinik II - Bereich

Leipzig, Saxony, Germany

Site Status RECRUITING

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

Berlin, State of Berlin, Germany

Site Status RECRUITING

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

Torrette, Ancona, Italy

Site Status RECRUITING

Azienda Ospedaliera dei Colli - Ospedale Monaldi

Naples, Campania, Italy

Site Status RECRUITING

Università degli Studi di Napoli Federico II - Ospedale Monaldi

Napoli, Campania, Italy

Site Status RECRUITING

IRCCS AOU di Bologna - Policlinico Sant' Orsola

Bologna, Emilia-Romagna, Italy

Site Status RECRUITING

Azienda Sanitaria Universitaria Giuliano Isontina

Venezia, Giulia, Italy

Site Status RECRUITING

Fondazione IRCCS Policlinico San Matteo - UOC Pneumologia

Pavia, Lombardy, Italy

Site Status RECRUITING

Ospedale San Giuseppe

Milan, Milano, Italy

Site Status RECRUITING

HUMANITAS Research Hospital

Rozzano, Milano, Italy

Site Status RECRUITING

A.O.U. Policlinico G. Rodolico

Catania, Sicily, Italy

Site Status RECRUITING

Erasmus University Medical Center

Rotterdam, , Netherlands

Site Status RECRUITING

Greenlane Clinical Centre

Auckland, Auckland, New Zealand

Site Status RECRUITING

Health New Zealand

Christchurch, Canterbury, New Zealand

Site Status RECRUITING

Dunedin Hospital

Dunedin, Dunedine, New Zealand

Site Status RECRUITING

Bay of Plenty Clinical School

Tauranga, South Tauranga, New Zealand

Site Status RECRUITING

Vitamed Galaj I Cichomski sp.j.

Bydgoszcz, Bydgoszc, Poland

Site Status RECRUITING

University Hospital in Krakow

Krakow, Krakow, Poland

Site Status RECRUITING

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

Lodz, , Poland

Site Status RECRUITING

AppleTreeClinics Network

Lodz, Łódź Voivodeship, Poland

Site Status RECRUITING

Hospital Universitario Virgen de las Nieves

Granada, Andalusia, Spain

Site Status RECRUITING

Hospital de Sant Pau y la Santa Creu

Barcelona, Barcelona, Spain

Site Status RECRUITING

Hospital Universitario Marqués de Valdecilla

Santander, Cantabria, Spain

Site Status RECRUITING

Hospital Gregorio Marañon

Madrid, Madrid, Spain

Site Status RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, Madrid, Spain

Site Status RECRUITING

Hospital Universitario Virgen de la Victoria

Málaga, Málaga, Spain

Site Status RECRUITING

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, Spain

Site Status RECRUITING

Hospital Universitario Virgen del Rocío

Seville, Sevilla, Spain

Site Status RECRUITING

Hospital Universitary de Bellvitge

Barcelona, , Spain

Site Status RECRUITING

Hospital Universitario Puerta del Mar - Leon-Jimenez

Cadiz, , Spain

Site Status RECRUITING

Ege University Medical Faculty Hospital

Bornova, İzmir, Turkey (Türkiye)

Site Status RECRUITING

Cukurova University Medical Faculty

Adana, , Turkey (Türkiye)

Site Status RECRUITING

Health Sciences University, Gulhane Faculty of Medicine

Ankara, , Turkey (Türkiye)

Site Status RECRUITING

Hull University Teaching Hospital NHS Trust

Cottingham, East Yorkshire, United Kingdom

Site Status RECRUITING

Birmingham Heartlands Hospital

Birmingham, Heartlands, United Kingdom

Site Status RECRUITING

Leicester Biomedical Research Centre - Respiratory Theme

Glenfield, Leicester, United Kingdom

Site Status RECRUITING

St George's University Hospitals NHS Foundation Trust

London, London, United Kingdom

Site Status RECRUITING

ILD Unit, University Hospitals Birmingham NHS Foundation Trust Theme

Birmingham, Midlands, United Kingdom

Site Status RECRUITING

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxon, United Kingdom

Site Status RECRUITING

Royal Devon

Exeter, South West, United Kingdom

Site Status RECRUITING

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

Wolverhampton, West Midlands, United Kingdom

Site Status RECRUITING

St James University Hospital - LTHT

Leeds, West Yorkshire, United Kingdom

Site Status RECRUITING

Royal Papworth Hospital

Cambridge, , United Kingdom

Site Status RECRUITING

Royal Brompton Hospital

London, , United Kingdom

Site Status RECRUITING

Manchester University NHS Foundation Trust

Manchester, , United Kingdom

Site Status RECRUITING

Nottingham University NHS Trust

Nottingham, , United Kingdom

Site Status RECRUITING

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

Stoke-on-Trent, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Australia Canada France Germany Italy Netherlands New Zealand Poland Spain Turkey (Türkiye) United Kingdom

Central Contacts

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Craig S. Conoscenti, MD

Role: CONTACT

206-707-0304

Daniele Tompkins

Role: CONTACT

973-983-3700 ext. 205

References

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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.

Reference Type RESULT
PMID: 36948586 (View on PubMed)

Related Links

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Other Identifiers

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AP01-007

Identifier Type: -

Identifier Source: org_study_id

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