A 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

11 Sep 2025 → ongoing

Decision date (initial)

2025-08-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2025-521660-35-00

WHO UTN

U1111-1318-4328

ClinicalTrials.gov

NCT06842823

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

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

1) For adults - To assess the superiority of navenibart compared to placebo in preventing HAE attacks in participants with Hereditary Angioedema (HAE).
2) For adolescents - To assess the efficacy of navenibart in preventing HAE attacks in participants with HAE
3) For adolescents - to assess the safety and tolerability of navenibart as a preventive treatment in participants with HAE.

Secondary objectives 3

1. For adults and adolescents - To assess the clinical efficacy of navenibart as a preventive treatment for HAE in participants with HAE
2. For adults and adolescents -To assess the quality of life associated with the use of navenibart in participants with HAE. 3) For adults only - To assess the safety and tolerability of navenibart relative to placebo as a preventive treatment in participa
3. For adults only - To assess the safety and tolerability of navenibart relative to placebo as a preventive treatment in participants with HAE

Conditions and MedDRA coding

Hereditary Angioedema

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-003510-PIP01-23

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Documented diagnosis of HAE (Type 1 or 2). The following must be met: a. Documented clinical history consistent with HAE b. Lab findings consistent with HAE Type 1 or 2
2. Experienced at least 2 HAE attacks during the Run-In period, as confirmed by an investigator based on meeting the protocol-specified definition of an HAE attack.

Exclusion criteria 3

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. Use of therapies prescribed for the prevention of HAE attacks may not be used during the trial or within the below time frames prior to the Run-In Period (adult participants may be on these medications at the time of the Screening Visit, but will need to washout prior to entering the Run-In Period): a. Lanadelumab within 70 days prior to Run-In b. Berotralstat within 21 days prior to Run-In c. Garadacimab within 90 days prior to Run-In d. Plasma-derived C1INH for LTP within 14 days prior to Run-In e. Tranexamic acid, oral danazol, oral stanazolol, and oral oxandrolone within 3 days prior to Run-In f. All other prophylactic therapies, including investigational drugs, require consultation with the Medical Monitor
3. Any exposure to angiotensin-converting enzyme inhibitors or any estrogen containing medications with systemic absorption (such as hormonal contraceptives or hormone replacement therapy) within 30 days before Screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of time-normalized investigator-confirmed HAE attacks during the 6-month Treatment Period.

Secondary endpoints 8

1. Number of moderate or severe investigator-confirmed HAE attacks during the 6-month Treatment Period.
2. Number of investigator-confirmed HAE attacks that require on-demand treatment during the 6-month Treatment Period.
3. Percent reduction in monthly investigator-confirmed HAE attacks in the 6-month Treatment Period versus the Run-In Period.
4. Time to first investigator-confirmed HAE attack after first and second dose
5. The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from the Run-In Period in investigator‑confirmed HAE attack rate (for adult participants: compared to placebo during the 6-month Treatment Period).
6. Number of participants with no investigator-confirmed HAE attacks during the 6-month Treatment Period
7. Change from baseline (Day 1) in the Angioedema Quality of Life questionnaire total score.
8. Other Pre-specified Outcome Measures: Incidence of treatment‑emergent adverse events.

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 271 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