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

Hereditary Angioedema Clinical Trial

— APeX-A  

Official title:

An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04933721
• Other study ID #: BCX7353-312
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: July 23, 2021
• Est. completion date: August 2026

Study information

• Verified date: May 2023
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 110
• Est. completion date: August 2026
• Est. primary completion date: August 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• HAE
• Hereditary Angioedema

Intervention

Drug:
• berotralstat
BCX7353 capsules administered orally once daily

Locations

Country	Name	City	State
Czechia	Study Center	Brno
Czechia	Study Center	Plzen
France	Study Center	Grenoble
France	Study Center	Paris
Korea, Republic of	Study Center	Daegu
Korea, Republic of	Study Center	Gwangju
Korea, Republic of	Study Center	Seoul
North Macedonia	Study Center	Skopje
Poland	Study Center	Kraków
Slovakia	Study Center	Martin
South Africa	Study Center	Cape Town

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

Czechia,  France,  Korea, Republic of,  North Macedonia,  Poland,  Slovakia,  South Africa, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number and proportion of subjects with a treatment-related TEAE		240 weeks
Primary	Number and proportion of subjects who experience a serious adverse event (SAE)		240 weeks
Primary	Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE		240 weeks
Primary	Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality		240 weeks
Primary	Number and proportion of subjects who discontinue due to a TEAE		240 weeks