2024-511285-37-00招募中4 期
An open-label study to provide berotralstat access to subjects with type I and II hereditary angioedema who were previously enrolled in berotralstat studies
适应症
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 33
- 试验地点
- 4
- 主要终点
- Number and proportion of subjects with a treatment-related TEAE
研究概览
简要总结
To monitor the long-term safety of berotralstat
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Males and non-pregnant, non-lactating females who are currently enrolled as a subject or were previously enrolled and did not discontinue due to an adverse event or due to noncompliance in BioCryst-sponsored Study 302, 204, or
- •Participant or parent/ legally designated representative (for participants < 18 years of age) able to provide written informed consent. For participants < 18 years of age, based on the child’s age and local regulatory requirements, participant assent will be collected as appropriate.
- •Would benefit from continued berotralstat treatment in the opinion of the investigator
- •Female subjects must meet at least 1 of the following requirements: a. Be a WOCBP (defined as a female following menarche and prior to becoming post-menopausal who has not had a hysterectomy or bilateral salpingectomy and bilateral oophorectomy) who agrees to use at least an acceptable effective contraceptive method during the study. b. Be a woman of nonchildbearing potential (defined as postmenopausal [no menses for at least 12 months without an alternative medical cause] or has had a hysterectomy or a bilateral salpingectomy and bilateral oophorectomy).
- •In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures
排除标准
- •Any condition or situation, including medical history or changes in medical history, which, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study
- •Dementia, altered mental status, or any psychiatric condition that would prohibit the understanding or rendering of informed consent.
- •Hypersensitivity to the active substance or to any of the excipients in the IMP
- •Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study. These include: C1-INH, tranexamic acid, androgens, or lanadelumab
- •Use of any other investigational medicinal product at the Baseline visit or any time during the study.
- •Pregnancy or breastfeeding
- •Subjects with an immediate family relationship to either sponsor employees, the investigator, or employees of the study site who are named on the delegation log.
- •Subjects who are held in an institution by a governmental or judicial order.
结局指标
主要结局
Number and proportion of subjects with a treatment-related TEAE
Number and proportion of subjects with a treatment-related TEAE
Number and proportion of subjects who experience a serious adverse event (SAE)
Number and proportion of subjects who experience a serious adverse event (SAE)
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
Number and proportion of subjects who discontinue due to a TEAE
Number and proportion of subjects who discontinue due to a TEAE
次要结局
- Another mechanism is available to provide drug to the subject(eg, market access), or until the sponsor discontinues all global commercialization and development of berotralstat for the prevention of angioedema attacks, whichever comes first
研究者
Biocryst Pharmaceuticals Inc.
Scientific
Biocryst Pharmaceuticals Inc.
研究点 (4)
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