Post-marketing Investigation (PMI) to Assess Safety and Efficacy of Jivi (BAY 94-9027) Treatment in Participants With Hemophilia A
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 32
- 试验地点
- 13
- 主要终点
- FVIII Inhibitor Development by the Nijmegen Bethesda Assay
研究概览
简要总结
The goal of this study is to give gather more information on how safe and well Jivi works in patients with severe hemophilia A. Jivi has been approved by various regulatory agencies, including the FDA, Health Canada, Japanese Health Authority and the European Medicinal Agency. 25 patients will be enrolled and will stay for 1 to 2 years in this study depending on their treatment frequency. Researcher will monitor during the course of the study whether patients are developing antibodies (a protein made by the body in response to the drug) affecting the effectiveness of Jivi. In addition information on bleedings and patient's wellbeing will be collected.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participants must ≥ 18 years of age inclusive, at the time of signing the informed consent.
- •Participants with severe hemophilia A (FVIII: C<1%)
- •PTPs (Previously treated patients) (≥150 ED (Exposure day)) on prophylaxis treatment before enrollment
- •Participants who are immunocompetent. If human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4)+ lymphocyte count >200/mm*3
- •Participants who are willing to complete an eDiary
- •Male participants
- •Capable of giving signed informed consent
排除标准
- •Any other inherited or acquired bleeding disorder in addition to Hemophilia A.
- •Platelet count < 100,000/mm*3
- •Creatinine > 2x upper limit of normal
- •AST or ALT > 5x upper limit of normal (AST: aspartate aminotransferase; ALT: alanine aminotransferase)
- •The participant has a planned major surgery.
- •The participant is currently participating in another investigational drug study, or has participated in a clinical study involving an investigational drug within 30 days of signing informed consent or previous treatment in a clinical phase III study with BAY 94-9027 (now marketed as Jivi).
- •Current evidence (by central laboratory) or history of inhibitor to FVIII with a titer ≥ 0.6 Bethesda unit (BU).
- •Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein.
研究组 & 干预措施
Severe hemophilia A patients
Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response.
干预措施: Damoctocog alfa pegol (Jivi, BAY94-9027) (Drug)
结局指标
主要结局
FVIII Inhibitor Development by the Nijmegen Bethesda Assay
时间窗: Observed for 100 exposure days (EDs), up to 2 years
FVIII inhibitor testing was performed using the Nijmegen-modified Bethesda assay. A positive inhibitor result was defined as a threshold of ≥0.6 BU/mL at the central laboratory and had to be confirmed with a second blood sample. After confirmation of the positive result, the inhibitor was to be reported as a serious adverse event (SAE).
次要结局
- Annualized Bleeding Rate (ABR)(Observed for 100 exposure days (EDs), up to 2 years)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(Observed for 100 exposure days (EDs), up to 2 years)
- Development of Treatment-emergent Anti-PEG Antibodies(Observed for 100 exposure days (EDs), up to 2 years)