Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625000246482
Ethics application status
Approved
Date submitted
9/01/2025
Date registered
4/04/2025
Date last updated
4/04/2025
Date data sharing statement initially provided
4/04/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
An Open-Label Extension Study of Subjects Who Received an Avalyn Inhaled Antifibrotic Agent (SAIL)
Scientific title
A Multinational, Long-Term, Safety and Tolerability, Open-Label Extension Study of Subjects Who Have Participated in Avalyn Pharma Studies of Inhaled Antifibrotic Agents (AP-LTE-008 [SAIL])
Secondary ID [1] 313557 0
None
Universal Trial Number (UTN)
Trial acronym
SAIL
Linked study record
ACTRN12621000170820 is a lead-in study for this open-label extension. All participants currently in ACTRN12621000170820 will be offered enrollment in this alternative open-label extension. Another ongoing lead-in study is NCT06329401. Participants will be offered to enroll in the open-label extension following successful completion in NCT06329401. Both studies involve administration of the same investigational drug product.

Health condition
Health condition(s) or problem(s) studied:
Idiopathic Pulmonary Fibrosis 336060 0
IPF 336061 0
Progressive Pulmonary Fibrosis 336062 0
PPF 336063 0
Condition category
Condition code
Respiratory 332613 332613 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is an open-label extension study for participants who were previously enrolled in and completed an Avalyn Pharma Sponsored study with an inhaled antifibrotic, such as AP01. Eligible participants will have their final dose of drug at the end of study visit from the lead-in study and first AP-LTE-008 study visit on the same day.

All participants will receive 100 mg inhaled pirfenidone inhalation solution (AP01) taken twice daily using the eFlow Nebulizer System for until such a time that the drug is approved, the participant withdraws from the study, or the study is terminated.

New patients will be trained to use the nebuliser at the first treatment visit. All patients will use the eFlow nebuliser to administer AP01. Hands on training for use and cleaning of the eFlow nebuliser will be performed by site personnel with patients using the study specific instructions for use and quick reference guide provided. The first treatment for new patients will be overseen by clinic personnel.

A paper dosing diary will be used to monitor adherence along with returns of any unused investigational product.
Intervention code [1] 330147 0
Treatment: Drugs
Comparator / control treatment
No control group.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 340128 0
To evaluate the long-term safety and tolerability outcomes of subjects receiving Avalyn nebulized antifibrotic medications (AP01).
Timepoint [1] 340128 0
End of treatment visit
Secondary outcome [1] 442857 0
To evaluate the long-term effect of Avalyn nebulized antifibrotic medications on lung function the change from baseline in forced vital capacity (FVC) will be evaluated.
Timepoint [1] 442857 0
End of treatment visit.
Secondary outcome [2] 442858 0
To evaluate the long-term effect of Avalyn nebulized antifibrotic medications on stabilization of disease.
Timepoint [2] 442858 0
End of treatment visit
Secondary outcome [3] 442859 0
To evaluate the long-term impact of Avalyn nebulized antifibrotic medications on subject-reported quality of life (QoL).
Timepoint [3] 442859 0
End of treatment visit.

Eligibility
Key inclusion criteria
• Previously participated in an Avalyn-sponsored inhaled antifibrotic clinical
study for subjects with either idiopathic pulmonary fibrosis (IPF) or
progressive pulmonary fibrosis (PPF) and with the approval of the Study
Physician. 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).

• Male subjects and female subjects of childbearing potential (FOCBP)
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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
•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.

• 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).

• Subjects who experienced an acute exacerbation of IPF or of PPF within 3
months of Day 1 (Screening/Baseline Visit).

• 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 shorter. Subjects may be enrolled in
registries.

• History of hypersensitivity and/or allergic reaction to pirfenidone or the
excipients to be used in this study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
7/04/2025
Actual
Date of last participant enrolment
Anticipated
31/12/2027
Actual
Date of last data collection
Anticipated
31/12/2031
Actual
Sample size
Target
340
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment outside Australia
Country [1] 26777 0
New Zealand
State/province [1] 26777 0

Funding & Sponsors
Funding source category [1] 318020 0
Commercial sector/Industry
Name [1] 318020 0
Avalyn Pharma Inc.
Country [1] 318020 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Avalyn Pharma Inc.
Address
Country
United States of America
Secondary sponsor category [1] 320362 0
None
Name [1] 320362 0
Address [1] 320362 0
Country [1] 320362 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316678 0
Western Sydney Local Health District Human Research Ethics Committee
Ethics committee address [1] 316678 0
Ethics committee country [1] 316678 0
Australia
Date submitted for ethics approval [1] 316678 0
02/12/2024
Approval date [1] 316678 0
09/01/2025
Ethics approval number [1] 316678 0
2024/ETH02371

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 138634 0
Dr John Wheatley
Address 138634 0
Westmead Hospital, Cnr Hawkesbury Road and, Darcy Rd, Westmead NSW 2145
Country 138634 0
Australia
Phone 138634 0
+61 2 8890 7785
Fax 138634 0
Email 138634 0
[email protected]
Contact person for public queries
Name 138635 0
John Wheatley
Address 138635 0
Westmead Hospital, Cnr Hawkesbury Road and, Darcy Rd, Westmead NSW 2145
Country 138635 0
Australia
Phone 138635 0
+61 2 8890 7785
Fax 138635 0
Email 138635 0
[email protected]
Contact person for scientific queries
Name 138636 0
Dr. Felix Woodhead, MA, MB, BChir, FRCP, PhD
Address 138636 0
Avalyn Pharma Inc., 245 First Street, 18th Floor, Cambridge, MA 01242
Country 138636 0
United States of America
Phone 138636 0
+44 7999 885973
Fax 138636 0
Email 138636 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.