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Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

NCT ID: NCT04933721

Last Updated: 2025-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

139 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-07-23

Study Completion Date

2031-08-31

Brief Summary

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This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

Detailed Description

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APeX-A, 7353-312, is a single-arm, open-label, multicenter study. The study will be conducted in countries where berotralstat is not available either commercially or via another mechanism and is planned in countries where pediatric participants have been enrolled in BCX7353-304 (Study 304). Adult and adolescent (≥12 years) participants will receive berotralstat 150 mg administered orally once daily (QD). Pediatric participants will receive a weight-appropriate dose. The study will assess the long term safety and tolerability of berotralstat.

Eligible subjects will be enrolled directly into Study BCX7353-312 (Study 312) from Studies 302, 204, and 304. Subjects will receive berotralstat administered orally once daily (QD). Subjects will return to the clinic every 24 weeks for drug dispensation and safety monitoring. Participants form Studies 302 and 204 may remain on study for up to 480 weeks (approximately 10 years). Participants from Study 304 may remain in the study until 16 years of age and have access to berotralstat via another mechanism; or up to 5 years, whichever comes first.

Safety and tolerability will be evaluated through assessment of serious adverse events (SAEs) and treatment-related, treatment-emergent adverse events (TEAEs). Up to 139 subjects are planned to enroll.

Conditions

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Hereditary Angioedema HAE

Keywords

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prophylaxis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open-label berotralstat (BCX7353) orally administered QD.
Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

No masking as this is open-label.

Study Groups

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BCX7353 capsules or granules once daily

Berotralstat (BCX7353) capsules or granules orally administered once daily.

Group Type EXPERIMENTAL

berotralstat

Intervention Type DRUG

BCX7353 capsules or granules administered orally once daily

Interventions

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berotralstat

BCX7353 capsules or granules administered orally once daily

Intervention Type DRUG

Other Intervention Names

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Orladeyo

Eligibility Criteria

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Inclusion Criteria

* Males and females currently enrolled in BioCryst-sponsored Study 302, 204, or 304
* Participant or parent/legally designated representative (for participants \<18 years of age) able to provide written informed consent.
* Would benefit from continued berotralstat treatment
* Acceptable effective contraception

Exclusion Criteria

* Pregnancy or breast-feeding
* Any condition or situation, including medical history that, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study
* Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study.
* Use of any other investigational medicinal product at the Baseline visit or any time during the study.
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioCryst Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Vesna Grivcheva-Panoska, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

PHI University Clinic of Dermatology

Locations

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Study Center

Ottawa, , Canada

Site Status

Study Center

Brno, , Czechia

Site Status

Study Center

Pilsen, , Czechia

Site Status

Study Center

Grenoble, , France

Site Status

Study Center

Marseille, , France

Site Status

Study Center

Paris, , France

Site Status

Study Center

Frankfurt, , Germany

Site Status

Study Center

Skopje, , North Macedonia

Site Status

Study Center

Krakow, , Poland

Site Status

Study Center

Martin, , Slovakia

Site Status

Study Center

Cape Town, , South Africa

Site Status

Study Center

Daegu, , South Korea

Site Status

Study Center

Gwangju, , South Korea

Site Status

Study Center

Seoul, , South Korea

Site Status

Study Center

Madrid, , Spain

Site Status

Study Center

Bristol, , United Kingdom

Site Status

Countries

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Canada Czechia France Germany North Macedonia Poland Slovakia South Africa South Korea Spain United Kingdom

Other Identifiers

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BCX7353-312

Identifier Type: -

Identifier Source: org_study_id