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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Avalyn Pharma Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

14 Jul 2021 → 19 Aug 2025

Decision date (initial)

2024-08-30

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Avalyn Pharma, Inc., United States

External identifiers

EU CT number

2024-515964-30-00

EudraCT number

2020-005103-39

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

• 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
• To evaluate safety outcomes of patients while on AP01 therapy

Secondary objectives 1

1. Not applicable

Conditions and MedDRA coding

Interstitial Lung Diseases (ILD), including but not limited to idiopathic pulmonary fibrosis (IPF)

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

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.

Exclusion criteria 11

1. 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
5. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Not applicable

Secondary endpoints 2

1. Treatment-emergent adverse events (AEs)
2. Treatment-emergent deaths

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Avalyn Pharma Inc.

Sponsor organisation

Avalyn Pharma Inc.

Address

701 Pike Street Suite 1500

City

Seattle

Postcode

98101-3926

Country

United States

Scientific contact point

Organisation

Avalyn Pharma Inc.

Contact name

Craig S. Conoscenti, MD

Public contact point

Organisation

Avalyn Pharma Inc.

Contact name

Craig S. Conoscenti, MD

Third parties 2

Locations

3 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications