An Open-Label Extension Study of Subjects who Received an Avalyn Inhaled Antifibrotic Agent

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

4 Jul 2025 → ongoing

Decision date (initial)

2025-06-03

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Avalyn Pharma Inc. 245 First Street, 18th Floor, Cambridge, MA, 02142

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

To evaluate long-term safety and tolerability outcomes of subjects receiving Avalyn nebulized antifibrotic medications

Secondary objectives 1

1. To evaluate the long-term effect of Avalyn nebulized antifibrotic medications on lung function

Conditions and MedDRA coding

Progressive pulmonary fibrosis [PPF] Idiopathic pulmonary fibrosis [IPF]

Regulatory references

Plan to share IPD

No

IPD plan description

NA

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Provide written informed consent per the Institutional Review Board/Ethics Committee (IRB/EC)
2. Previously participated in an Avalyn-sponsored inhaled antifibrotic clinical study for subjects with either IPF or PPF and with the approval of the Investigator. IPF is defined as: A specific form of chronic fibrosing interstitial pneumonia limited to the lung and associated with the pathological pattern of usual interstitial pneumonia (American Thoracic Society 2000; Raghu et al, 2018).  PPF is defined as: At least 2 of 3 criteria (worsening symptoms, radiological progression, and physiological progression) occurring within the past year with no alternative explanation in a patient with an interstitial lung disease other than IPF (Raghu et al, 2022).  Previous participation is defined as: Having completed the final visit of the Treatment Period on the full dose of study drug (either active or placebo).
3. Male subjects and female subjects of childbearing potential (FOCBP) (defined as females who are fertile, following menarche and until becoming post-menopausal unless permanently sterile) agree to use highly effective contraception measures from the time of first dose of study drug (for the male subject) or the signing of the informed consent form (ICF) (for the female subject), during the study, and until 90 days after the last dose of study drug. Subjects agree not to donate eggs or sperm during the same period. Male subjects must use a condom and female partners of male subjects who are of childbearing potential must use a highly effective method of contraception, defined below: a. A highly effective method of contraception is one that results in a low failure rate (i.e., <1% per year) when used consistently and correctly. The acceptable methods of contraception include: i. sexual abstinence, defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the subject. ii. a vasectomized partner iii. bilateral tubal occlusion iv. any effective intrauterine device/hormone-releasing system, and progesterone-only (oral, injectable, or implantable) or combined (estrogen- and progesterone-containing; oral, intravaginal, or transdermal) hormonal contraception associated with inhibition of ovulation. Note: Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception. b. The efficacy of oral hormonal contraceptives may be compromised by vomiting and/or diarrhea or other conditions where the drug absorption may be reduced. Advise women taking oral hormonal contraceptives experiencing these conditions to use alternative highly effective contraception. Note: Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and/or bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. In the absence of 12 months of amenorrhea, a single folliclestimulating hormone measurement is insufficient to document menopause.
4. Willingness to comply with all study visits and requirements.
5. Male or female at least 18 years of age at Screening/Baseline (Day 1).

Exclusion criteria 8

1. Have not previously participated in an Avalyn-sponsored inhaled antifibrotic lead-in study or if the subject was permanently discontinued from the lead-in study for any reason. Subjects who discontinued study drug but continued to attend study visits are ineligible.
2. Subjects who experienced an exacerbation of asthma or of chronic obstructive pulmonary disease (COPD) requiring oral or systemic corticosteroids within 3 months of Day 1 (Screening/Baseline Visit).
3. Subjects who experienced an acute exacerbation of IPF or of PPF (as defined in Section 12.2.3) within 3 months of Day 1 (Screening/Baseline Visit).
4. Any conditions or abnormalities (including electrocardiogram [ECG] or laboratory abnormalities) which, in the opinion of the Investigator may compromise the safety of the subject or interfere with the subject participating in or completing the study.
5. History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.
6. Participation in a concurrent clinical study or in a clinical study in which any other investigational drug product aside from the Avalyn nebulized antifibrotic medication from their lead-in study was administered within the previous 30 days, or 5 half-lives of the previously administered investigational product, whichever is longer. Subjects may be enrolled in registries.
7. History of hypersensitivity and/or allergic reaction to pirfenidone or the excipients to be used in this study.
8. Is pregnant, nursing, or who plans to become pregnant while in the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 6

1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
2. Incidence of adverse events of special interest (AESIs)
3. Incidence of treatment-emergent all-cause deaths
4. Incidence of treatment-emergent respiratory deaths
5. Incidence of exacerbation of pulmonary fibrosis
6. Changes from baseline in clinical laboratory tests and vital signs

Secondary endpoints 2

1. Change from baseline in forced vital capacity (FVC) (mL) at intervals of 6 months
2. Annual rate of decline in FVC (mL)

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

Dr Felix Woodhead, MA MB BChir FRCP PhD Senior Medical Director, Avalyn Pharma Inc.

Public contact point

Organisation

Avalyn Pharma Inc.

Contact name

Dr Felix Woodhead, MA MB BChir FRCP PhD Senior Medical Director, Avalyn Pharma Inc.

Locations

7 EU/EEA countries · 19 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 79 files

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

Application history

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