Access to Pirfenidone Solution for Inhalation (AP01) for Treatment of Progressive, Fibrosing Interstitial Lung Diseases, including Idiopathic Pulmonary Fibrosis

Phase 1

Conditions: Interstitial Lung Diseases (ILD), including but not limited to idiopathic pulmonary fibrosis (IPF)
MedDRA version: 21.1Level: PTClassification code 10022611Term: Interstitial lung diseaseSystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Registration Number: EUCTR2020-005103-39-NL

Lead Sponsor: Avalyn Pharma, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 100

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 approval of non-Avalyn study rollover patients is required.
3. Diagnosed chronic progressive fibrotic ILD, 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 (FOCBP) must use an effective contraceptive method during the clinical study and 30 days after the last dose of AP01 as described in Section 7.3.6, Pregnancy Testing.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

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 ILD/IPF for AP01 naïve patients 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 (AP01 naïve patients) or Baseline (Rollover patients from previous study) 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 ILD/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, end-stage liver disease or AST or ALT greater than 5 times the upper limit of normal at Screening (AP01 naïve patients)
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 (IP) (AP01 naïve patients only)
11. Hypersensitivity to the active substance or to any of the excipients of pirfenidone

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Secondary Objective: Not Applicable;Primary end point(s): Not Applicable;Timepoint(s) of evaluation of this end point: Not Applicable;Main Objective: • Allow patients to continue or start AP01 therapy for the treatment of ILD and IPF until applicable regulatory approval or until the study is terminated<br>• To evaluate safety outcomes of patients while on AP01 therapy

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Treatment-emergent adverse events (AEs)<br>• Treatment-emergent deaths;Timepoint(s) of evaluation of this end point: Adverse events (AEs): AEs will be collected from the start of administration of study drug through the last administration of AP01

Related Trials