Clinical trial • Phase II • Respiratory

PIRFENIDONE for Progressive pulmonary fibrosis | Pulmonary fibrosis

Phase II trial of PIRFENIDONE for Progressive pulmonary fibrosis | Pulmonary fibrosis.

Overview

Trial Therapeutic Area
Respiratory
Trial Disease
Progressive pulmonary fibrosis | Pulmonary fibrosis
Trial Stage
Phase II
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
03-05-2024
First CTIS Authorization Date
20-08-2024

Trial design

Randomised, ap01 (pirfenidone solution for inhalation) 100 mg bid and ap01 50 mg bid compared to placebo (placebo for ap01). route: inhalation (eflow nebuliser system).-controlled Phase II trial across 45 sites in France, Poland, Belgium and others.

Randomised
Yes
Comparator
AP01 (Pirfenidone Solution for Inhalation) 100 mg BID and AP01 50 mg BID compared to placebo (Placebo for AP01). Route: inhalation (eFlow Nebuliser System).
Target Sample Size
197
Trial Duration For Participant
364

Eligibility

Recruits 197 Flagged as vulnerable population (isVulnerablePopulationSelected = true). Participants are adults (≥18 years) and informed consent documents (L1 SIS and ICF) are provided; no further details on assent or specific consent handling for vulnerable participants are available in the provided data..

Vulnerable Population
Flagged as vulnerable population (isVulnerablePopulationSelected = true). Participants are adults (≥18 years) and informed consent documents (L1 SIS and ICF) are provided; no further details on assent or specific consent handling for vulnerable participants are available in the provided data.

Inclusion criteria

  • {"criterion_text":"- Male or female at least 18 years of age at Screening.\n- Subject meets criteria for PPF (modified from Flaherty et al, 2019) (see full description in the protocol)\n- Meeting all of the following criteria during the Screening Period: a.\tForced vital capacity (FVC) ≥45% of predicted normal at Screening Visit 1, b.\tForced expiratory volume at 1 second (FEV1)/FVC ≥0.7 or ≥age-adjusted lower limit of normal Global Lung Function Initiative (GLI) values (Quanjer et al, 2012) at Screening Visit 1, c.\tDiffusing capacity of lung for carbon monoxide (DLCO) ≥30% of predicted, corrected for hemoglobin at Screening Visit 1, d.\tAcceptability: Participants can perform acceptable spirometry (i.e., meet American Thoracic Society (ATS)/ European Respiratory Society (ERS) acceptability criteria at both Screening Visits). Note: Spirometry (including DLCO) obtained at Visit 1 may be repeated to confirm eligibility once, if discussed and approved by the Medical Monitor (e.g., technical issue, subject fatigue).\n- For participants already on nintedanib (up to 30% of participants): Must have been on nintedanib for 12 weeks prior to Screening with or without dose adjustments and/or drug interruptions during that period. For participants who have discontinued nintedanib prior to Screening: Must have been off of nintedanib for a minimum of 12 weeks."}

Exclusion criteria

  • {"criterion_text":"- Current treatment with oral pirfenidone or treatment with oral pirfenidone within 3 months prior to Screening.\n- Elevated liver enzymes and liver injury at Screening defined as: a.\tAlanine aminotransferase (ALT) or aspartate aminotransferase (AST) ˃3 times the upper limit of normal (ULN), OR b. Bilirubin >2.0 x ULN\n- Renal disease with a creatinine clearance <30 mL/min, calculated according to the Chronic Kidney Disease Epidemiology Collaboration formula (Inker et al, 2021). Retesting is allowed once.\n- Diagnosis of idiopathic pulmonary fibrosis (IPF) based on the ATS diagnostic algorithm for IPF. Usual interstitial pneumonia (UIP) that is not idiopathic, for example related to rheumatoid arthritis (RA), familial interstitial lung disease (ILD), or other is not exclusionary.\n- Greater extent of emphysema than of fibrotic ILD on HRCT. Note: CT results must be confirmed through the central over read process.\n- Significant clinical worsening of PPF between Screening Visit 1 and Visit 3 (Week 0/Day 1/Randomization), as assessed by the Investigator.\n- 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."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- The main trial endpoint is the change from baseline in FVC, the maximum amount of air that can be exhaled when blowing out as fast as possible (measured in milliliters) at Week 52","definition_or_measurement_approach":"Change from baseline in forced vital capacity (FVC) measured in millilitres at Week 52 (spirometry per ATS/ERS acceptability criteria)."}

Secondary endpoints

  • {"endpoint_text":"- Change from Baseline in QoL measurements as assessed by Living with Pulmonary Fibrosis Symptoms and Impact Questionnaire (L-PF) total score at Week 52","definition_or_measurement_approach":"Change from baseline in L-PF total score at Week 52 (quality of life instrument specific to pulmonary fibrosis)."}
  • {"endpoint_text":"- Time to disease progression. Disease progression is defined as absolute FVC percent predicted decline of ≥10% through Week 52 or all-cause death","definition_or_measurement_approach":"Time-to-event analysis where disease progression = absolute decline in percent predicted FVC ≥10% through Week 52 or all-cause mortality."}
  • {"endpoint_text":"- Change in lung fibrosis score based on high-resolution computed tomography (HRCT) from Baseline to 52 weeks","definition_or_measurement_approach":"Change in radiologic lung fibrosis score on HRCT from baseline to Week 52; HRCT reads include central over-read (HRCT overread vendor listed)."}

Recruitment

Planned Sample Size
197
Recruitment Window Months
29
Consent Approach
Informed consent uses L1 subject information sheets and informed consent forms (multiple country/language versions present). Participants are adults (≥18) and consent is provided by the participant. Multiple language/country-specific ICFs are available (EN, ES, FR, PL, IT, NL, DE and country-specific versions as listed); pregnancy-specific information/ICF documents are also present. No further details on assent or additional consent handling are available in the provided data.

Geography

Total Number Of Sites
45
Total Number Of Participants
197

France

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
19-11-2025
Processing Time Days
490
Number Of Sites
9
Number Of Participants
45

Sites

Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
Pneumology
Contact Person Name
Stéphane JOUNEAU
Contact Person Email
Stephane.jouneau@chu-rennes.fr
Site Name
Hôpitaux Privés de Metz
Department Name
Pneumology
Contact Person Name
Magalie MERCY
Contact Person Email
Magalie.mercy@uneos.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Respiratory diseases
Contact Person Name
Elodie BLANCHARD
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Pneumology
Contact Person Name
Arnaud BOURDIN
Contact Person Email
a-bourdin@chu-montpellier.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Pulmology
Contact Person Name
David MONTANI
Contact Person Email
david.montani@aphp.fr
Site Name
GIE Groupe hospitalier Paris Saint-Joseph/Vinci
Department Name
Pneumology, allergology and thoracic oncology
Contact Person Name
Jean-Marc NACCACHE
Contact Person Email
jmnaccache@ghpsj.fr
Site Name
Centre Hospitalier Regional D'Angers
Department Name
Pneumology
Contact Person Name
Frédéric GAGNADOUX
Contact Person Email
fgagnadoux@chu-angers.fr
Site Name
Hospices Civils De Lyon
Department Name
Pulmology
Contact Person Name
Vincent COTTIN
Contact Person Email
Vincent.cottin@chu-lyon.fr
Site Name
Centre Hospitalier Regional Universitaire De Tours
Department Name
Pneumology
Contact Person Name
Laurent PLANTIER
Contact Person Email
Laurent.plantier@univ-tours.fr

Poland

Earliest CTIS Part Ii Submission Date
29-07-2024
Latest Decision Or Authorization Date
21-11-2025
Processing Time Days
480
Number Of Sites
4
Number Of Participants
18

Sites

Site Name
Vitamed Galaj I Cichomski Sp. j.
Contact Person Name
Iwona Patyk
Contact Person Email
i.patyk@vitamed.info
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Oddział Kliniczny Pulmonologii, Alergologii i Chorób Wewnętrznych
Contact Person Name
Krzysztof Sładek
Contact Person Email
mmsladek@cyf-kr.edu.pl
Site Name
PRYWATNY GABINET LEKARSKI GINEKOLOGIA I POŁOŻNICTWO ULTRASONOGRAFI Prof. Jacek Suzin
Contact Person Name
Izabela Grabska-Kobyłecka
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny Nr 1 Im. Norberta Barlickiego Uniwersytetu Medycznego W Lodzi
Department Name
Oddział Kliniczny Pulmonologii i Alergologii
Contact Person Name
Wojciech Piotrowski

Belgium

Earliest CTIS Part Ii Submission Date
17-06-2025
Latest Decision Or Authorization Date
19-11-2025
Processing Time Days
155
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Centre hospitalier universitaire de Liege
Department Name
Pneumology
Contact Person Name
Julien Guiot
Contact Person Email
j.guiot@chuliege.be
Site Name
UZ Leuven
Department Name
Pneumomogy
Contact Person Name
Wim Wuyts
Contact Person Email
wim.wuyts@uzleuven.be

Italy

Earliest CTIS Part Ii Submission Date
22-05-2024
Latest Decision Or Authorization Date
19-11-2025
Processing Time Days
546
Number Of Sites
9
Number Of Participants
20

Sites

Site Name
Azienda Sanitaria Universitaria Giuliano Isontina
Department Name
Pneumology
Contact Person Name
Marco Confalonieri
Site Name
Azienda Ospedaliera Dei Colli
Department Name
Pulmonology Unit
Contact Person Name
Marialuisa Bocchino
Contact Person Email
marialuisa.bocchino@unina.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
DEPARTMENT OF INTERNAL MEDICINE AND MEDICAL THERAPY
Contact Person Name
Angelo Guido Corsico
Contact Person Email
corsico@unipv.it
Site Name
Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco Di Catania
Department Name
Pulmonology Unit
Contact Person Name
Carlo Vancheri
Contact Person Email
vancheri@unict.it
Site Name
Multimedica S.p.A.
Department Name
Pneumology
Contact Person Name
Sergio Harari
Contact Person Email
sergio.harari@multimedica.it
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Pneumology
Contact Person Name
Stefano Nava
Contact Person Email
stefano.nava@aosp.bo.it
Site Name
Humanitas Mirasole S.p.A.
Department Name
Pneumologia I
Contact Person Name
Francesco Amati
Contact Person Email
francesco.amati@hunimed.eu
Site Name
Azienda Ospedaliero Universitaria Delle Marche
Department Name
Pneumology
Contact Person Name
Federico Mei
Site Name
Azienda Ospedaliera Dei Colli
Department Name
Pneumology
Contact Person Name
Anna Agnese Stanziola
Contact Person Email
annaagnese.stanziola@unina.it

Spain

Earliest CTIS Part Ii Submission Date
15-08-2024
Latest Decision Or Authorization Date
24-11-2025
Processing Time Days
466
Number Of Sites
10
Number Of Participants
40

Sites

Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Pneumology
Contact Person Name
Luis Puente Maestu
Contact Person Email
luis.puente@salud.madrid.org
Site Name
Hospital Universitario Virgen De La Victoria
Department Name
Pneumology
Contact Person Name
José Luis Velasco Garrido
Contact Person Email
jlvelascogarrido@hotmail.com
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Pneumology
Contact Person Name
Jose Antonio Rodriguez
Contact Person Email
josearportal@gmail.com
Site Name
Hospital Universitario Central De Asturias
Department Name
Pneumology
Contact Person Name
Miguel Arias Guillén
Contact Person Email
miguelariasguillen@gmail.com
Site Name
Bellvitge University Hospital
Principal Investigator Name
Wim Wuyts
Principal Investigator Email
wim.wuyts@uzleuven.be
Contact Person Name
Julien Guiot
Contact Person Email
j.guiot@chuliege.be
Site Name
Hospital Universitario Marques De Valdecilla
Department Name
Pneumology
Contact Person Name
Jose Manuel Cifrian Martinez
Contact Person Email
josemanuel.cifrian@scsalud.es
Site Name
Hospital Universitario Virgen De Las Nieves
Department Name
Pneumology
Contact Person Name
Ana Dolores Romero
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Pneumology
Contact Person Name
Diego Castillo Villegas
Contact Person Email
DCastillo@santpau.cat
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Pulmology
Contact Person Name
Juan Rigual
Contact Person Email
Jrigual1989@gmail.com
Site Name
Hospital Universitario Puerta Del Mar
Department Name
Pneumology
Contact Person Name
Antonio León Jiménez

Germany

Earliest CTIS Part Ii Submission Date
29-07-2024
Latest Decision Or Authorization Date
21-11-2025
Processing Time Days
480
Number Of Sites
9
Number Of Participants
34

Sites

Site Name
Vivantes Netzwerk fuer Gesundheit GmbH
Department Name
Klinik fuer Innere Medizin – Pneumologie & Infektiologie
Contact Person Name
Sven Glaeser
Contact Person Email
sven.glaeser@vivantes.de
Site Name
Pneumological Study Center Munich West
Contact Person Name
Rainald Fischer
Contact Person Email
rainald.fischer@gmail.com
Site Name
Studienzentrum Dr. Claus Keller
Contact Person Name
Claus Keller
Site Name
Ruhrlandklinik Westdeutsches Lungenzentrum Am Universitaetsklinikum Essen gGmbH
Department Name
Klinik fuer Pneumologie
Contact Person Name
Francesco Bonella
Site Name
Muenchen Klinik gGmbH
Department Name
Klinik fuer Pneumologie und Pneumologische Onkologie
Contact Person Name
Peter Schramm
Site Name
Romed Klinikum Rosenheim
Department Name
Studienzentrum Med 3
Contact Person Name
Stephan Budwiser
Contact Person Email
Stephan.Budweiser@ro-med.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
Zentrum fuer Thoraxerkrankungen (ZfT)
Contact Person Name
Michael Kreuter
Site Name
Medizinische Hochschule Hannover
Department Name
Klinik fuer Pneumologie und Infektiologie
Contact Person Name
Jonas Schupp
Contact Person Email
schupp.jonas@mh-hannover.de
Site Name
Universitaet Leipzig
Department Name
Klinik fuer Onkologie, Gastroenterologie, Hepatologie und Pneumologie
Contact Person Name
Hubert Wirtz

Netherlands

Earliest CTIS Part Ii Submission Date
04-07-2025
Latest Decision Or Authorization Date
22-01-2026
Processing Time Days
202
Number Of Sites
2
Number Of Participants
20

Sites

Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Pulmology
Contact Person Name
Thomas Koudstaal
Contact Person Email
t.koudstaal.1@erasmusmc.nl
Site Name
Sint Antonius Ziekenhuis Stichting
Department Name
Pulmonology
Contact Person Name
Marcel Veltkamp

Sponsor

Primary sponsor

Full Name
Avalyn Pharma Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Third parties

  • {"country":"United States","full_name":"Devpro Biopharma LLC","duties_or_roles":"[865399,865400,865401,865402,865403]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"[865409]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Acm Medical Laboratory Inc.","duties_or_roles":"[865391]","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Ireland","full_name":"Millmount Healthcare Limited","duties_or_roles":"[865404]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"[865393 - TMF]","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Primevigilance Limited","duties_or_roles":"[865410]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"[865392 - HRCT overread]","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Canada","full_name":"Everest Clinical Research Corporation","duties_or_roles":"[865405,865406,865407,865408]","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Vitalograph Limited","duties_or_roles":"[865398 - ECG, 6MWT, DLCO & Spirometry]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Trial By Fire Solutions LLC","duties_or_roles":"[865394 - CTMS]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"[865395,865396,865397]","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Pirfenidone Solution for Inhalation (AP01)
Active Substance
PIRFENIDONE
Modality
Small molecule
Routes Of Administration
INHALATION
Route
INHALATION
Starting Dose
50 mg BID and 100 mg BID (dose arms described in protocol)
Dose Levels
50 mg BID; 100 mg BID
Frequency
BID
Investigational Product Name
Pirfenidone Solution for Inhalation (AP01) (second product entry)
Active Substance
PIRFENIDONE
Modality
Small molecule
Routes Of Administration
INHALATION
Route
INHALATION
Starting Dose
50 mg BID and 100 mg BID (dose arms described in protocol)
Dose Levels
50 mg BID; 100 mg BID
Frequency
BID
Investigational Product Name
Placebo for AP01
Modality
Other
Combination Treatment
Yes

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