Clinical trial • Phase II • Respiratory

PIRFENIDONE for Progressive pulmonary fibrosis | Idiopathic pulmonary fibrosis

Phase II trial of PIRFENIDONE for Progressive pulmonary fibrosis | Idiopathic pulmonary fibrosis. open-label. 220 participants.

Overview

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

Key dates

Initial CTIS Submission Date
07-02-2025
First CTIS Authorization Date
03-06-2025

Trial design

open-label Phase II trial in Italy, Poland, Czechia and others.

Open Label
Yes
Target Sample Size
220
Trial Duration For Participant
364

Eligibility

Recruits 220 No vulnerable population selected. Written informed consent per the Institutional Review Board/Ethics Committee (IRB/EC) is required. Participants must be at least 18 years of age. No assent procedures for minors are described..

Pregnancy Exclusion
Is pregnant, nursing, or who plans to become pregnant while in the study.
Vulnerable Population
No vulnerable population selected. Written informed consent per the Institutional Review Board/Ethics Committee (IRB/EC) is required. Participants must be at least 18 years of age. No assent procedures for minors are described.

Inclusion criteria

  • {"criterion_text":"- Provide written informed consent per the Institutional Review Board/Ethics Committee (IRB/EC)\n- Previously participated in an Avalyn-sponsored inhaled antifibrotic clinical study for subjects with either IPF or PPF and with the approval of the Investigator. IPF is defined as: A specific form of chronic fibrosing interstitial pneumonia limited to the lung and associated with the pathological pattern of usual interstitial pneumonia (American Thoracic Society 2000; Raghu et al, 2018).  PPF is defined as: At least 2 of 3 criteria (worsening symptoms, radiological progression, and physiological progression) occurring within the past year with no alternative explanation in a patient with an interstitial lung disease other than IPF (Raghu et al, 2022).  Previous participation is defined as: Having completed the final visit of the Treatment Period on the full dose of study drug (either active or placebo).\n- Male subjects and female subjects of childbearing potential (FOCBP) (defined as females who are fertile, following menarche and until becoming post-menopausal unless permanently sterile) agree to use highly effective contraception measures from the time of first dose of study drug (for the male subject) or the signing of the informed consent form (ICF) (for the female subject), during the study, and until 90 days after the last dose of study drug. Subjects agree not to donate eggs or sperm during the same period. Male subjects must use a condom and female partners of male subjects who are of childbearing potential must use a highly effective method of contraception, defined below: a. A highly effective method of contraception is one that results in a low failure rate (i.e., <1% per year) when used consistently and correctly. The acceptable methods of contraception include: i. sexual abstinence, defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the subject. ii. a vasectomized partner iii. bilateral tubal occlusion iv. any effective intrauterine device/hormone-releasing system, and progesterone-only (oral, injectable, or implantable) or combined (estrogen- and progesterone-containing; oral, intravaginal, or transdermal) hormonal contraception associated with inhibition of ovulation. Note: Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception. b. The efficacy of oral hormonal contraceptives may be compromised by vomiting and/or diarrhea or other conditions where the drug absorption may be reduced. Advise women taking oral hormonal contraceptives experiencing these conditions to use alternative highly effective contraception. Note: Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and/or bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. In the absence of 12 months of amenorrhea, a single folliclestimulating hormone measurement is insufficient to document menopause.\n- Willingness to comply with all study visits and requirements.\n- Male or female at least 18 years of age at Screening/Baseline (Day 1)."}

Exclusion criteria

  • {"criterion_text":"- Have not previously participated in an Avalyn-sponsored inhaled antifibrotic lead-in study or if the subject was permanently discontinued from the lead-in study for any reason. Subjects who discontinued study drug but continued to attend study visits are ineligible.\n- Subjects who experienced an exacerbation of asthma or of chronic obstructive pulmonary disease (COPD) requiring oral or systemic corticosteroids within 3 months of Day 1 (Screening/Baseline Visit).\n- Subjects who experienced an acute exacerbation of IPF or of PPF (as defined in Section 12.2.3) within 3 months of Day 1 (Screening/Baseline Visit).\n- Any conditions or abnormalities (including electrocardiogram [ECG] or laboratory abnormalities) which, in the opinion of the Investigator may compromise the safety of the subject or interfere with the subject participating in or completing the study.\n- History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.\n- Participation in a concurrent clinical study or in a clinical study in which any other investigational drug product aside from the Avalyn nebulized antifibrotic medication from their lead-in study was administered within the previous 30 days, or 5 half-lives of the previously administered investigational product, whichever is longer. Subjects may be enrolled in registries.\n- History of hypersensitivity and/or allergic reaction to pirfenidone or the excipients to be used in this study.\n- Is pregnant, nursing, or who plans to become pregnant while in the study."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Incidence of adverse events of special interest (AESIs)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Incidence of treatment-emergent all-cause deaths","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Incidence of treatment-emergent respiratory deaths","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Incidence of exacerbation of pulmonary fibrosis","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Changes from baseline in clinical laboratory tests and vital signs","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- Change from baseline in forced vital capacity (FVC) (mL) at intervals of 6 months","definition_or_measurement_approach":"Change from baseline in forced vital capacity (FVC) measured in mL at intervals of 6 months"}
  • {"endpoint_text":"- Annual rate of decline in FVC (mL)","definition_or_measurement_approach":"Annual rate of decline in forced vital capacity (FVC) measured in mL"}

Recruitment

Planned Sample Size
220
Recruitment Window Months
80
Consent Approach
Written informed consent per Institutional Review Board/Ethics Committee (IRB/EC) is required. Participants must be at least 18 years old and provide their own consent. Informed consent documents (SIS and ICF) are available in multiple languages (English, Italian, French, Spanish, German, Dutch, Polish, Czech) as indicated in the submission documents. No assent procedures for minors are described.

Geography

Total Number Of Sites
19
Total Number Of Participants
121

Italy

Earliest CTIS Part Ii Submission Date
29-08-2025
Latest Decision Or Authorization Date
23-02-2026
Processing Time Days
178
Number Of Sites
3
Number Of Participants
20

Sites

Site Name
Humanitas Mirasole S.p.A.
Department Name
Internal Medicine – Pulmonology 1
Principal Investigator Name
Francesco Amati
Principal Investigator Email
francesco.amati@hunimed.eu
Contact Person Name
Francesco Amati
Contact Person Email
francesco.amati@hunimed.eu
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
Pneumology Division
Principal Investigator Name
Angelo Guido Corsico
Principal Investigator Email
corsico@unipv.it
Contact Person Name
Angelo Guido Corsico
Contact Person Email
corsico@unipv.it
Site Name
Multimedica S.p.A.
Department Name
Division of Pulmonary disease, Intensive Respiratory Care U
Principal Investigator Name
Sergio Harari
Principal Investigator Email
sergio@sergioharari.it
Contact Person Name
Sergio Harari
Contact Person Email
sergio@sergioharari.it

Poland

Earliest CTIS Part Ii Submission Date
02-05-2025
Latest Decision Or Authorization Date
30-01-2026
Processing Time Days
273
Number Of Sites
4
Number Of Participants
30

Sites

Site Name
Gornoslaskie Centrum Medyczne Im Prof. Leszka Gieca Sląskiego Uniwersytetu Medycznego W Katowicach
Department Name
Oddział Pneumologii
Principal Investigator Name
Adam Barczyk
Principal Investigator Email
abarczyk@icloud.com
Contact Person Name
Adam Barczyk
Contact Person Email
abarczyk@icloud.com
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Alergologii
Principal Investigator Name
Amelia Szymanowska-Narloch
Principal Investigator Email
aszymanowska@gumed.edu.pl
Contact Person Name
Amelia Szymanowska-Narloch
Contact Person Email
aszymanowska@gumed.edu.pl
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 z Pododdziałem Chrób Wewnętrznych
Principal Investigator Name
Wojciech Piotrowski
Principal Investigator Email
wojciech.piotrowski@umed.lodz.pl
Contact Person Name
Wojciech Piotrowski
Site Name
Vitamed Galaj I Cichomski Sp. j.
Department Name
Poradnia Pulmonologiczna
Principal Investigator Name
Iwona Patyk
Principal Investigator Email
i.patyk@vitamed.info
Contact Person Name
Iwona Patyk
Contact Person Email
i.patyk@vitamed.info

Czechia

Earliest CTIS Part Ii Submission Date
02-05-2025
Latest Decision Or Authorization Date
23-01-2026
Processing Time Days
266
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Fakultni Thomayerova nemocnice
Department Name
Pneumologicka klinika
Principal Investigator Name
Martina Sterclova
Principal Investigator Email
martina.sterclova@ftn.cz
Contact Person Name
Martina Sterclova
Contact Person Email
martina.sterclova@ftn.cz

Netherlands

Earliest CTIS Part Ii Submission Date
02-05-2025
Latest Decision Or Authorization Date
22-01-2026
Processing Time Days
265
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
St. Antonius Ziekenhuis
Department Name
Department of Pulmonology
Principal Investigator Name
Marcel Veltkamp
Principal Investigator Email
m.veltkamp@antoniusziekenhuis.nl
Contact Person Name
Marcel Veltkamp

France

Earliest CTIS Part Ii Submission Date
08-09-2025
Latest Decision Or Authorization Date
23-01-2026
Processing Time Days
137
Number Of Sites
4
Number Of Participants
21

Sites

Site Name
Hospices Civils De Lyon
Department Name
Service de Pneumologie
Principal Investigator Name
Vincent Cottin
Principal Investigator Email
vincent.cottin@chu-lyon.fr
Contact Person Name
Vincent Cottin
Contact Person Email
vincent.cottin@chu-lyon.fr
Site Name
Centre Hospitalier Universitaire D'Angers
Department Name
Département de Pneumologie
Principal Investigator Name
Frederic Gagnadoux
Principal Investigator Email
fgagnadoux@chu-angers.fr
Contact Person Name
Frederic Gagnadoux
Contact Person Email
fgagnadoux@chu-angers.fr
Site Name
Centre Hospitalier Regional Universitaire De Tours
Department Name
Service de Pneumologie et Explorations Fonctionnelles Respiratoires
Principal Investigator Name
Laurent Plantier
Principal Investigator Email
laurent.plantier@univ-tours.fr
Contact Person Name
Laurent Plantier
Contact Person Email
laurent.plantier@univ-tours.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Service de Pneumologie et Maladies Respiratoires
Principal Investigator Name
Arnaud Bourdin
Principal Investigator Email
a-bourdin@chu-montpellier.fr
Contact Person Name
Arnaud Bourdin
Contact Person Email
a-bourdin@chu-montpellier.fr

Spain

Earliest CTIS Part Ii Submission Date
18-08-2025
Latest Decision Or Authorization Date
28-01-2026
Processing Time Days
163
Number Of Sites
3
Number Of Participants
11

Sites

Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Neumology
Principal Investigator Name
Luis Puente Maestu
Principal Investigator Email
luis.puente@salud.madrid.org
Contact Person Name
Luis Puente Maestu
Contact Person Email
luis.puente@salud.madrid.org
Site Name
Hospital Universitario Virgen De Las Nieves
Department Name
Neumology
Principal Investigator Name
Ana Dolores Romero Ortiz
Principal Investigator Email
anadoloresromeroortiz@gmail.com
Contact Person Name
Ana Dolores Romero Ortiz
Site Name
Hospital Universitario Virgen De La Victoria
Department Name
Neumology
Principal Investigator Name
José Luis Velasco Garrido
Principal Investigator Email
josel.velasco.sspa@juntadeandalucia.es
Contact Person Name
José Luis Velasco Garrido

Germany

Earliest CTIS Part Ii Submission Date
28-08-2025
Latest Decision Or Authorization Date
26-01-2026
Processing Time Days
151
Number Of Sites
3
Number Of Participants
34

Sites

Site Name
Universitaetsklinikum Essen AöR
Department Name
Interstitial and Rare Lung Disease Centre
Principal Investigator Name
Francesco Tommaso Bonella
Principal Investigator Email
francesco.bonella@rlk.uk-essen.de
Contact Person Name
Francesco Tommaso Bonella
Site Name
Studienzentrum Dr. Claus Keller
Department Name
Studienzentrum Dr. Klaus Keller
Principal Investigator Name
Klaus Keller
Principal Investigator Email
ckeller-studien@praxis-ckeller.de
Contact Person Name
Klaus Keller
Site Name
Medizinische Hochschule Hannover
Department Name
Klinik für Pneumologie und Infektiologie, Klinisches Studienzentrum
Principal Investigator Name
Jonas Schupp
Principal Investigator Email
Schupp.jonas@mh-hannover.de
Contact Person Name
Jonas Schupp
Contact Person Email
Schupp.jonas@mh-hannover.de

Sponsor

Primary sponsor

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

Investigational products

Investigational Product Name
Pirfenidone Solution for Inhalation
Active Substance
PIRFENIDONE
Modality
Small molecule
Routes Of Administration
INHALATION
Route
INHALATION
Maximum Dose
200 mg

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