An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 139
- 试验地点
- 1
- 主要终点
- Number and proportion of subjects with a treatment-related TEAE
研究概览
简要总结
This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
盲法说明
No masking as this is open-label.
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
BCX7353 capsules or granules once daily
Berotralstat (BCX7353) capsules or granules orally administered once daily.
干预措施: berotralstat (Drug)
结局指标
主要结局
Number and proportion of subjects with a treatment-related TEAE
时间窗: 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
时间窗: 240 weeks
Number and proportion of subjects who experience a serious adverse event (SAE)
时间窗: 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
时间窗: 240 weeks
Number and proportion of subjects who discontinue due to a TEAE
时间窗: 240 weeks
次要结局
未报告次要终点