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

Study Overview
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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. 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.

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

Conditions
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Hereditary Angioedema, HAE

Intervention / Treatment
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* Drug: berotralstat


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label berotralstat (BCX7353) 150 mg orally administered QD.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BCX7353 Capsules 150 mg once daily<br>Berotralstat (BCX7353) 150 mg capsule orally administered once daily. | Drug: berotralstat<br>* BCX7353 capsules administered orally once daily<br>* Other names: Orladeyo;|

What is the study measuring?
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Primary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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prophylaxis