A Long-term Study of Navenibart in Participants with Hereditary Angioedema

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Astria Therapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

27 Mar 2026 → ongoing

Decision date (initial)

2026-02-09

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2025-521142-22-00

WHO UTN

U1111-1324-6718

ClinicalTrials.gov

NCT07204938

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacokinetic, Dose response, Pharmacogenomic, Pharmacodynamic, Pharmacogenetic, Safety

To assess the long-term safety and tolerability of navenibart in participants with hereditary angioedema (HAE)

Secondary objectives 2

1. 1) To assess the long-term clinical efficacy of navenibart as a preventive treatment for HAE in participants with HAE
2. 2) To assess the quality of life associated with the use of navenibart in participants with HAE

Conditions and MedDRA coding

Hereditary Angioedema

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Participants from STAR-0215-301 who met one of the following conditions: a. Completed STAR-0215-301 through the Day 181 visit b. Withdrew from STAR-0215-301 but met the following criteria: i. Received 2 doses of IP ii. completed ≥ 2 months of trial follow-up after the second dose of IP iii. Met other eligibility criteria as assessed by Investigator

Exclusion criteria 4

1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH (also known as HAE type 3) , idiopathic angioedema, or angioedema associated with urticaria.
2. Participation in an investigational clinical trial other than STAR-0215-301 in the 30 days or any exposure to an investigational drug (other than navenibart in STAR-0215-301) within 5 half-lives before informed consent/assent
3. Any exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption (such as hormonal contraceptives or hormone replacement therapy) within 30 days before Screening.
4. Known sensitivity to the ingredients in the formulation of IP

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence of treatment-emergent adverse events

Secondary endpoints 8

1. Number of time-normalized investigator-confirmed HAE attacks
2. Number of moderate or severe investigator-confirmed HAE attacks
3. Number of investigator-confirmed HAE attacks that require on-demand treatment
4. Percent reduction in monthly investigator-confirmed HAE attacks
5. Time to first investigator-confirmed HAE attack after first dose
6. The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction in investigator-confirmed HAE attack rate
7. Number of participants with no investigator-confirmed HAE attacks
8. Angioedema Quality of Life (AE-QoL) questionnaire total score

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Astria Therapeutics Inc.

Sponsor organisation

Astria Therapeutics Inc.

Address

22 Boston Wharf Road, Floor 10 Floor 10

City

Boston

Postcode

02210

Country

United States

Scientific contact point

Organisation

Astria Therapeutics Inc.

Contact name

Astria Therapeutics Inc

Public contact point

Organisation

Astria Therapeutics Inc.

Contact name

Astria Therapeutics Inc

Third parties 5

Locations

11 EU/EEA countries · 33 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 135 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