This study has been transitioned to CTIS with ID 2024-515964-30-00 check the CTIS register for the current data. • Allow patients to continue or start AP01 therapy for the treatment of ILD and IPF prior to regulatory approval or until the study is…
ID
Source
Brief title
Condition
- Respiratory disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Safety Outcome Measures
• Treatment-emergent adverse events (AEs)
• Treatment-emergent deaths
Efficacy Outcome Measures
• No efficacy outcome measures
Secondary outcome
N/A
Background summary
The term Interstitial Lung Disease (ILD) encompasses a large group of over 200
pulmonary disorders, most of which are classified as rare. While idiopathic
pulmonary fibrosis (IPF) is the classic fibrosing ILD, clinical data suggest
that there is a larger group of patients with differing clinical ILD diagnoses
who develop a progressive fibrosing phenotype during the course of their
disease.
Momentarily the study drug (pirfenidone) is available as treatment for
idiopathic pulmonary fibrosis, which is given in a capsule for oral use.
However, studies have shown the use of oral pirfenidone leads to side effects
in many patients. In some cases these side effects prevent the use of
medication. A previous study has shown that a single dose up to 100 mg/mL given
as an inhaled formulation was safe and well tolerated. It has shown that more
pirfenidone was absorbed in the lungs. This may lead to better efficacy as well
as a reduction in the level of study drug which was absorbed by the blood. This
causes less side effects.
Study objective
This study has been transitioned to CTIS with ID 2024-515964-30-00 check the CTIS register for the current data.
• Allow patients to continue or start AP01 therapy for the treatment of ILD and
IPF prior to regulatory approval or until the study is terminated
• To evaluate safety outcomes of patients on AP01 therapy
Study design
This is a study for Pirfenidone Solution for Inhalation (AP01) 100 mg twice
daily. The primary objective is to allow patients to continue or start AP01
therapy for treatment of ILD, including IPF.
A screening visit will be performed if the patient has not previously been on
an Avalyn sponsored AP01 study. If a patient has been previously enrolled on
an Avalyn sponsored study with AP01, a modified screening visit will occur.
Patients will have an in-clinic visit every 12 weeks and AEs and lung function
will be captured. If the patient stops treatment for any reason, an end of
treatment visit will take place.
Intervention
Patients will receive 100 mg AP01 administered twice daily with the PARI eFlow®
nebulizer
Study burden and risks
Please refer to the study schedule in the protocol, table 5 (page 36).
This study will last about 48 months but could also end sooner once the study
drug obtains regulatory approval.
The subject will need to come to the hospital every 12 weeks while taking part
in this study. In addition the subject is asked to come to the hospital for an
end of study visit once they have completed the study drug treatment.
The screening visit (if required) and the first visit in the treatment period
will last approximately 3 to 4 hours. All other visits will last approximately
1 to 2.5 hours.
During these visits the following tests and procedures will take place:
- Demographic and medical history (1 time)
- Physical exam, vital signs (7 times)
- Urine tests (3 times)
- Pregnancy tests in wo men of childbearing potential (7 times)
- Blood test (7 times)
- Spirometry tests (7 times).
Pike Street, Suite 1500 701
Seattle, WA 98101
US
Pike Street, Suite 1500 701
Seattle, WA 98101
US
Listed location countries
Age
Inclusion criteria
A patient must meet the following inclusion criteria to be eligible for
enrollment in the clinical study:
1. Prior participant in an Avalyn AP01 study, excluding normal healthy
volunteers
OR
2. Patients with no other treatment options with ILDs including but not limited
to IPF, fibrosing phenotype ILD, pulmonary involvement of scleroderma,
rheumatoid lung and silicosis. Prior Sponsor (Avalyn Pharma, Inc.) approval of
non-Avalyn study rollover patients is required.
3. Diagnosed chronic progressive fibrotic lung disease, including IPF, without
treatment alternatives such as
a. Not eligible for oral pirfenidone and nintedanib due to national formulary
restrictions or lack of applicable regulatory approval
b. Intolerant to oral pirfenidone and nintedanib, if previously offered
c. Not eligible for an ongoing clinical study of AP01 other than this study
4. Age greater than 18 years at Screening
5. Able to understand and sign, prior to study entry, a written informed
consent form (ICF) consistent with International Council on Harmonisation
Guideline for Good Clinical Practice (ICH-GCP) and local laws
6. Able to understand the importance of adherence to study treatment and the
study protocol and willing to follow all study requirements, including the
concomitant medication restrictions, throughout the study
7. Females of childbearing potential must use an effective contraceptive method
during the clinical study and 30 days after the last dose of AP01
Exclusion criteria
The presence of any of the following exclusion criteria excludes a patient from
study enrollment:
Disease-Related Exclusions
1. Significant clinical worsening of IPF/ILD between Screening and Day 1, in
the opinion of the Investigator
2. Not a suitable candidate for enrollment or unlikely to comply with the
requirements of this study, in the opinion of the Investigator
3. History of acute IPF exacerbation requiring hospitalization in the last 30
days
4. Clinical evidence of active infection, including but not limited to
bronchitis, pneumonia, sinusitis, urinary tract infection, or cellulitis
Medical Exclusions
5. Females with a positive pregnancy test at Screening or are currently
breastfeeding
6. Any history of malignancy likely to result in significant disability or
likely to require significant medical or surgical intervention within the next
6 months. This does not include minor surgical procedures for localized cancer
(e.g., basal cell carcinoma)
7. Any condition other than IPF that, in the opinion of the investigator, is
likely to result in the death of the patient within the next 6 months
8. History of severe hepatic impairment or end-stage liver disease or AST or
ALT greater than 5 times the upper limit of normal at Screening
9. History of end-stage renal disease requiring dialysis
10. Participation in a clinical study with administration of an investigational
drug product within the previous 30 days, or five half-lives of the previously
administered investigational product
11. Hypersensitivity to the active substance or to any of the excipients of
pirfenidone
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | 380951 |
| EU-CTR | CTIS2024-515964-30-00 |
| EudraCT | EUCTR2020-005103-39-NL |
| CCMO | NL75957.100.20 |