Safety of SEVENFACT® for the Treatment of Bleeding Events in Patients With Hemophilia A or B With Inhibitors
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 55
- 试验地点
- 40
- 主要终点
- Number of participants and percentage of Safety Events (AEs)
研究概览
简要总结
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk of experiencing a breakthrough bleeding event (BE), or who are not on prophylactic treatment who may need to control a BE.
详细描述
Primary Objective:
To evaluate the safety of SEVENFACT® when used to treat bleeding episodes in participants with Hemophilia A or B with inhibitors either with or without prophylactic treatment
Study Design:
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A and B with inhibitors12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk of experiencing a breakthrough bleeding event (BE), or who are not on prophylactic treatment who may need to control a BE.
Study Duration:
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a diagnosis of hemophilia A or B with inhibitors.
- •Be 12 years of age and older
- •Be capable of understanding and willing to comply with the conditions of the protocol or have a legal guardian who is capable of understanding and complying with the conditions of the protocol
- •Have read, understood, and documented written informed consent/assent
- •Be able to provide medical evidence through prior medical history of previous inhibitor levels
- •Be willing and able to use the ATHN mobile application or a paper diary to document BEs and medication usage
排除标准
- •Have a disorder of hemostasis in addition to Hemophilia A or B
- •Have a known or suspected intolerance or hypersensitivity to SEVENFACT® or its ingredients
- •Have a known allergy or hypersensitivity to rabbits or rabbit proteins
- •Are receiving prophylactic treatment for bleeding with a drug or biologic that is not approved for this use by the FDA
- •Have had implantation of an investigational medical device within the prior 6 months
- •Have received an investigational drug within 30 days of the baseline visit
- •Have an elective surgical procedure planned during the duration of their participation in the study*
- •Have any life-threatening disease, or other disease or condition which, in the investigator's judgment, could pose a potential hazard to the patient or interfere with study participation or study outcome (e.g., a history of non responsiveness to bypassing products or thromboembolic disease)
- •Should a participant require an unplanned surgery, the participant will not be withdrawn from the study unless the investigator deems it necessary. Instead, the participant will receive standard of care treatment as determined by the attending physician. If the participant is not withdrawn from the study, the participant's participation in the study will be paused until the investigator feels it is safe for them to continue.
研究组 & 干预措施
Hemophilia A and B Cases
SEVENFACT® has been approved for the treatment of bleeding events in individuals with hemophilia A or B with inhibitors. This study is intended to further investigate the safety and tolerability of SEVENFACT in participants with hemophilia A or B with inhibitors in the presence or absence of prophylactic therapies. Dosing will be at the discretion of the attending physician, and each participant will be supplied with the equivalent of nine 75 µg/kg doses, which aligns with the recommended dosing schedule as provided in SEVENFACT's United States Prescribing Information (USPI). In the event of a bleeding episode (BE), the participant will either self-administer the correct dose under the guidance of the treating investigator or the dose will be administered at a treatment facility.
干预措施: coagulation factor VIIa [recombinant]-jncw (Drug)
结局指标
主要结局
Number of participants and percentage of Safety Events (AEs)
时间窗: From time of consent through BE onset until 3 days after last dose of SEVENFACT®.
Adverse Events and SAEs defined by the European Haemophilia Safety Surveillance System 2018 (EUHASS) and Serious Adverse Events (SAEs) as defined by the US Food and Drug Association.
次要结局
未报告次要终点