A Randomized Open-Label, Phase 1b Study of the Safety of Pirfenidone Solution for Inhalation (AP01) in Patients with Idiopathic Pulmonary Fibrosis (ATLAS Study)
- Conditions
- build-up of scar tissue in the lungsIdiopathic pulmonary fibrosis (IPF)10038716
- Registration Number
- NL-OMON50041
- Lead Sponsor
- Avalyn Pharma, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 10
1. Male and female patients, at least 40 years of age at Screening
2. Not eligible for oral pirfenidone and nintedanib due to national formulary;
restrictions OR intolerant to or unwilling to start oral pirfenidone and
nintedanib, if previously offered;
3. Clinical symptoms consistent with IPF of * 12 months duration (with or
without IPF diagnosis)
4. Diagnosis of IPF, defined as the first instance in which a patient was
informed of having IPF, no more than 60 months before randomization;
Patients that have had an IPF diagnosis * 1 year, the following criteria must
be met:
- HRCT and/or Surgical Lung Biopsy findings consistent with UIP. If
honeycombing is not present on the HRCT, then one or both of the following
criteria must be present:
* Disease progression since diagnosis by HRCT and/or
* An absolute loss of FVC * 5% percent predicted over the past 12 months,
Patients that have had IPF diagnosis within the last year, the following
criteria must be met:
- Diagnosis of Usual Interstitial Pneumonia (UIP) or IPF by HRCT (HRCT must be
performed within 12 months prior to Screening) and/or Surgical Lung Biopsy
5. Extent of fibrotic changes (honeycombing, reticular changes) greater than
the extent of emphysema on HRCT scan, confirmed by central review;
6. No features supporting an alternative diagnosis on transbronchial biopsy,
BAL, or surgical lung biopsy, if performed;
7. 40% * FVC * 90 % predicted at Screening based on Global Lung Initiative12
equations. The first 20 patients randomized must have FVC * 50% predicted.
After the first 20 patients have randomized, patients with FVC 40% - 50%
predicted will be allowed to be randomized in the
study but randomization for these patients will be capped at 20;
8. Change in FVC (measured in liters) between Screening and Day 1 (pre-dose
measurement) must be a < 10% relative difference;
9. 30 * % DLCO * 90% at Screening;
10. In the investigator's opinion, no evidence of improvement in measure of IPF
disease severity over the preceding year;
11. FEV1/FVC * 70%;
12. Able to understand and sign a written informed consent form;
13. 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;
1. Significant clinical worsening of IPF 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 last3
months;
4. History of clinically significant environmental exposure known to cause
pulmonary fibrosis, including but not limited to drugs (such as amiodarone),
asbestos, beryllium, radiation, and domestic birds;
5. Known explanation for interstitial lung disease, including but not limited
to radiation, drug toxicity, sarcoidosis, hypersensitivity pneumonitis,
bronchiolitis obliterans organizing pneumonia, human
immunodeficiency virus, viral hepatitis, and cancer;
6. Clinical diagnosis of any connective tissue disease, including but not
limited to scleroderma, polymyositis/dermatomyositis, systemic lupus
erythematosus, and rheumatoid arthritis;
7. Current diagnosis of asthma or chronic obstructive pulmonary disease;
8. Clinical evidence of active infection, including but not limited to
bronchitis, pneumonia, sinusitis, urinary tract infection, or cellulitis;
9. Females with a positive pregnancy test at Screening or are currently
breastfeeding
10. 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);
11. 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;
12. History of severe hepatic impairment or end-stage liver disease or ALT or
AST greater than 5 times the upper limit of normal at Screening;
13. History of end-stage renal disease requiring dialysis
14. 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>* Treatment-emergent AEs<br /><br>* Change from pre-dose to post-dose FEV1 after initial dose<br /><br>* Treatment-emergent deaths<br /><br>* Treatment-emergent changes in clinical laboratory findings<br /><br>* Changes in vital signs</p><br>
- Secondary Outcome Measures
Name Time Method <p>* Change from Baseline in FVC % predicted<br /><br>* Change from Baseline in DLCO<br /><br>* Change from Baseline in Patient Reported Outcomes (PRO)<br /><br>* Change from Baseline in cough frequency and intensity<br /><br>* Change from Baseline in extent of fibrosis and lung volumes</p><br>