Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies (APeX-A)

BioCryst

Status and phase: Active, not recruiting; Phase 3

Conditions: HAE; Hereditary Angioedema

Treatments: Drug: berotralstat

Study type: Interventional

Funder types: Industry

Identifiers

NCT04933721

BCX7353-312

Details and patient eligibility

About

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

Full description

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. All enrolled subjects will receive the planned marketed dose of berotralstat, 150 mg QD. 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 and 204. Subjects will receive berotralstat 150 mg administered orally once daily (QD). Subjects will return to the clinic every 24 weeks for drug dispensation and safety monitoring for up to 240 weeks (approximately 5 years).

Safety and tolerability will be evaluated through assessment of serious adverse events (SAEs) and treatment-related, treatment-emergent adverse events (TEAEs). Up to 110 subjects are planned to enroll.

Enrollment: 110 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Males and females currently enrolled in BioCryst-sponsored Study 302 or 204
• Able to provide written informed consent.
• Would benefit from continued berotralstat treatment
• Acceptable effective contraception

Key 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.

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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110 participants in 1 patient group

BCX7353 Capsules 150 mg once daily

Experimental group

Description:

Berotralstat (BCX7353) 150 mg capsule orally administered once daily.

Treatment:

Drug: berotralstat