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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06329401




Registration number
NCT06329401
Ethics application status
Date submitted
12/03/2024
Date registered
25/03/2024
Date last updated
25/11/2024

Titles & IDs
Public title
A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants with Progressive Pulmonary Fibrosis
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Participants with PPF
Secondary ID [1] 0 0
AP01-007
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Progressive Pulmonary Fibrosis 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AP01
Other interventions - Placebo

Experimental: AP01 High Dose BID - Pirfenidone Solution for Inhalation

Experimental: AP01 Low Dose BID - Pirfenidone Solution for Inhalation

Placebo comparator: Placebo BID - Placebo solution for inhalation


Treatment: Drugs: AP01
Oral inhalation solution

Other interventions: Placebo
Placebo oral inhalation solution
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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)
Timepoint [1] 0 0
Week 52
Secondary outcome [1] 0 0
To evaluate the effect of AP01 high dose, AP01 low dose compared to placebo on quality of life (QoL)
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
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)
Timepoint [2] 0 0
52 weeks
Secondary outcome [3] 0 0
To evaluate the change from baseline in quantitative lung fibrosis score.
Timepoint [3] 0 0
52 weeks

Eligibility
Key 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 compared to Screening Visit 1
2. Relative decline in FVC =5 to <10% predicted within the previous 24 months compared to Screening Visit 1 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, 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/04/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2026
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,Perth West AustraliaVIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Nepean Lung & Sleep - Kingswood
Recruitment hospital [3] 0 0
John Hunter Hospital - New Lambton
Recruitment hospital [4] 0 0
Macquarie University Clinical Trials Unit - Sydney
Recruitment hospital [5] 0 0
Westmead Hospital - Westmead
Recruitment hospital [6] 0 0
Institute for Respiratory Health - Nedlands
Recruitment hospital [7] 0 0
Lung Research Victoria - Footscray
Recruitment hospital [8] 0 0
Alfred Health - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2745 - Kingswood
Recruitment postcode(s) [3] 0 0
2305 - New Lambton
Recruitment postcode(s) [4] 0 0
2109 - Sydney
Recruitment postcode(s) [5] 0 0
2145 - Westmead
Recruitment postcode(s) [6] 0 0
6009 - Nedlands
Recruitment postcode(s) [7] 0 0
3011 - Footscray
Recruitment postcode(s) [8] 0 0
3004 - Melbourne
Recruitment outside Australia
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United States of America
State/province [1] 0 0
Alabama
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United States of America
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Arizona
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California
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Colorado
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Connecticut
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Florida
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Georgia
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Illinois
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Kansas
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Maryland
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Michigan
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Minnesota
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Missouri
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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South Carolina
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Tennessee
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Texas
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Utah
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Virginia
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Washington
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Argentina
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Buenos Aires
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Canada
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British Columbia
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Canada
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Ontario
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Canada
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Quebec
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France
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Indre et Loire
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France
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Lorraine
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France
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Angers
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France
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Lyon
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France
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Montpellier
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France
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Pessac
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France
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Rennes
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Germany
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Bavaria
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Germany
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Hessia
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Germany
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Lower Saxony
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Germany
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Northrhine westphalia
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Germany
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Rhineland-Palatinate
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Italy
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Ancona
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New Zealand
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Canterbury
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New Zealand
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Dunedine
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New Zealand
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South Tauranga
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New Zealand
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Auckland
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Poland
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Bydgoszc
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Poland
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Lodz
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Spain
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Asturias
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Spain
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Barcelona
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Spain
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Cadiz
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Spain
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Madrid
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Spain
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Málaga
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United Kingdom
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South West

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Avalyn Pharma Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
DevPro Biopharma
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT06329401
Trial related presentations / publications
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.
Public notes

Contacts
Principal investigator
Name 0 0
Avalyn Pharma, Inc.
Address 0 0
Avalyn Pharma Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Craig S. Conoscenti, MD
Address 0 0
Country 0 0
Phone 0 0
206-707-0304
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06329401