Access to Pirfenidone Solution for Inhalation (AP01) for Treatment of Progressive, Fibrosing Interstitial Lung Diseases, including Idiopathic Pulmonary Fibrosis
Safety of Pirfenidone Solution for Inhalation (AP01) for Treatment of Progressive, Fibrosing Interstitial Lung Diseases, including Idiopathic Pulmonary Fibrosis in Adults with no Treatment Alternatives
Avalyn Pharma Pty Ltd
100 participants
Jun 11, 2021
Interventional
Conditions
Summary
The purpose of this protocol is to allow for patients on current AP01 studies to continue therapy and for patients with progressive, fibrosing ILD, including IPF without treatment options and who are unable to enroll in an AP01 study to be treated with AP01. The primary objective is to allow patients to continue or start AP01 therapy. 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 AP01 study, a modified screening visit will occur. If the patient stops treatment for any reason an end of treatment visit will take place. Patients will have an in clinic visit every 12 weeks to collect any safety and lung function tests that are performed as standard care. AP01 treatment within the realm of this study will continue if patient can tolerate the drug or regulatory approval is granted or the study is terminated. Study may be terminated by the sponsor at any time for any reason. The data in this study will be used to assess the safety of patients on AP01 therapy.
Eligibility
Inclusion Criteria11
- Prior participant in an Avalyn AP01 study, excluding normal volunteers
- Or
- Patients with no other treatment options with Interstitial Lung Diseases, including but not limited to Idiopathic Pulmonary Fibrosis, fibrosing phenotype Interstitial Lung Disease, pulmonary involvement of scleroderma, rheumatoid lung and silicosis. Prior Sponsor approval of non-Avalyn study rollover patients is required.
- Diagnosed chronic progressive fibrotic Interstitial Lung Disease, including Idiopathic Pulmonary Fibrosis, without treatment alternatives such as:
- a. Not eligible for oral pirfenidone and nintedanib due to national formulary restrictions or lack of 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
- Age greater than 18 years
- Able to understand and sign a written informed consent form (ICF)
- 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
- Females of childbearing potential (FOCBP) must use a contraceptive method during the clinical study and 30 days after the last dose of pirfenidone solution for inhalation (AP01)
Exclusion Criteria12
- Disease-Related Exclusions
- Significant clinical worsening of Interstitial Lung Disease/Idiopathic Pulmonary Fibrosis for AP01 naïve patients between Screening and Day 1, in the opinion of the investigator
- Not a suitable candidate for enrollment or unlikely to comply with the requirements of this study, in the opinion of the investigator
- History of acute Idiopathic Pulmonary Fibrosis exacerbation requiring hospitalization in the last 30 days
- Clinical evidence of active infection, including but not limited to bronchitis, pneumonia, sinusitis, urinary tract infection, or cellulitis
- Medical Exclusions
- Females with a positive pregnancy test at Screening (AP01 naïve patients) or Baseline (Rollover patients from previous study) or are currently breastfeeding
- 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)
- Any condition other than Interstitial Lung Disease/Idiopathic Pulmonary Fibrosis that, in the opinion of the investigator, is likely to result in the death of the patient within the next 6 months
- 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 (AP01 naïve patients)
- History of end-stage renal disease requiring dialysis
- 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 (IP) (AP01 naïve patients only)
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Interventions
Drug: Pirfenidone solution for inhalation (AP01): each patient will receive 100 mg inhaled pirfenidone taken twice daily for up to 48 weeks, using the eFlow nebuliser for inhalation. The only barriers to completing the full 48 weeks is tolerability to AP01 and patient consent. If patients cannot tolerate 100 mg AP01 twice daily, the dose may be reduced to 50 mg twice daily and if not tolerable, additional reduction to 50 mg once daily is available. Drug salbutamol: Patients who experience cough that limits their ability to complete dosing will administer 1 - 2 puffs (90-100 micrograms) of salbutamol using a metered dose inhaler in order to complete the in-clinic dose (new patients only). These patients, as well as patients with a history of asthma or smoking history of 20 pack years or greater will be required to administer 1 - 2 puffs (90-100 micrograms) of salbutamol using a metered dose inhaler prior to dosing throughout the study unless these patients are currently taking a long-acting beta-2-agonist therapy. 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. Eligible patients from the AP01-002 study will have the last AP01-002 study visit and their Baseline AP01-005 visit on the same day; there is no washout period for these patients. All patients rolling over from AP01-002 will be on the 100 mg twice daily dose unless the patient has tolerability issues. Patients will tolerability issues will stay on the dose being administered in the AP01-002 study. Compliance and tolerability will be assessed at clinic visits every 3 months. Overall study drug compliance will be assessed at clinic visits every 3 months for a total of 48 months or as long as the patient is on the study by calculating the amount of AP01 vials used versus the number of AP01 vials that were expected to be used during that study period. 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 AP01 study, a modified screening visit will occur. If the patient stops treatment for any reason an end of treatment visit will take place. Patients will have an in clinic visit every 12 weeks to collect any safety and lung function tests that are performed as standard care. AP01 treatment within the realm of this study will continue if patient can tolerate the drug or regulatory approval is granted or the study is terminated. Study may be terminated by the sponsor at any time for any reason.
Locations(13)
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ACTRN12621000170820