Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Octapharma
- 入组人数
- 7
- 试验地点
- 4
- 主要终点
- The incidence of thromboembolic adverse drug reactions (ADRs)
研究概览
简要总结
Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
详细描述
There is a need to increase the body of data on treatment effectiveness and safety in the ultra-rare setting of congenital fibrinogen deficiency. Real-world evidence (RWE) derived from non-interventional studies can describe product utilization, demonstrate value, and facilitate benefit-risk assessments; RWE can only be fully assessed once a product is launched and used in a real-life setting.
This post-marketing, observational study is designed to collect information concerning safety, efficacy, and outcomes of Fibryga administration in routine clinical use in patients of any age with congenital afibrinogenemia or hypofibrinogenemia. Documentation of the administration of Fibryga in clinical practice for the treatment of both minor and major bleeding events (BEs) will not only enhance the knowledge on the efficacy and safety profile of Fibryga, but will also gather information that cannot be obtained in the same way in controlled clinical studies. These observational data will support the safety and efficacy data generated with Fibryga in good clinical practice (GCP) clinical studies, providing benefit for both physicians and patients.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of any age with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia expected to require on-demand in-hospital treatment for BEs with Fibryga
排除标准
- •Bleeding disorder other than congenital fibrinogen deficiency
- •Patients with acquired fibrinogen deficiency or dysfibrinogenemia
- •Suspicion of an anti-fibrinogen inhibitor as indicated by previous in vivo recovery, if available, of <0.5 (mg/dL)/(mg/kg); there is currently no standard test for inhibitors
- •Participation in an interventional clinical study at the time of or within 4 weeks prior to enrolment
研究组 & 干预措施
Fibryga
Fibryga (human plasma-derived fibrinogen concentrate)
干预措施: Fibryga (Biological)
结局指标
主要结局
The incidence of thromboembolic adverse drug reactions (ADRs)
时间窗: Day 0-28
The incidence of thromboembolic ADRs in patients receiving Fibryga for on-demand treatment of bleeding, including major bleeding, will be documented
次要结局
- Duration of BEs(Within 2-24 hours following treatment of BEs)
- Incidence of treatment-emergent adverse events (safety)(Day 0-28)
- Hemostatic efficacy of Fibryga for all bleeding events (BEs) collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale(Within 2-24 hours following treatment of BEs)
- Dosage of Fibryga(Within 2-24 hours following treatment of BEs)