Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Bayer
- 入组人数
- 24
- 试验地点
- 17
- 主要终点
- Number of Participants With Drug-related Treatment-emergent Adverse Events
研究概览
简要总结
The purpose of this study was to assess the safety and tolerability of multiple doses of a human monoclonal antibody (BAY1093884) given under the skin in subjects with hemophilia A or B. This antibody was intended to protect from bleeds by inhibiting a substance (Tissue Factor Pathway Inhibitor, TFPI) that reduces the ability of the body to form blood clots.
详细描述
The primary objective of the study was to assess the safety and tolerability of multiple subcutaneous injections of BAY1093884 (anti-TFPI monoclonal antibody, immunoglobulin G2, IgG2) in patients with hemophilia A or B with or without inhibitors.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 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
- •Male severe hemophilic patients with undetectable FVIII activity <1% or FIX activity <2%, with or without inhibitors (any titer) are eligible.
- •Subjects with a past history of inhibitors (any inhibitor titer) are eligible.
- •Age ≥18 years.
- •Documentation of ≥4 bleeding episodes (any type or location of bleeds, treated or not) within the 6 months prior to screening.
- •For subjects on prophylaxis: Willingness to interrupt ongoing prophylaxis.
- •For subjects on immune tolerance induction (ITI): Willingness to interrupt ongoing ITI.
排除标准
- •History of any other coagulation disorder (particularly disseminated intravascular coagulopathy or combined FVIII/FV deficiency) or platelet disorder.
- •History of diseases related to venous thromboembolic events (e.g., pulmonary embolism, deep vein thrombosis, thrombophlebitis) or thrombotic microangiopathy.
- •Risk factors for venous or arterial diseases (e.g., uncontrolled hypertension, uncontrolled diabetes).
- •History of cardiac, coronary and/or arterial peripheral atherosclerotic disease
- •Platelet count <100,000/μL.
- •Human immunodeficiency virus (HIV) infection with a cluster of differentiation 4 (CD4+) lymphocyte count of <200/mm^3
研究组 & 干预措施
BAY1093884 100mg
Subjects received BAY1093884 100 mg once a week until premature termination of the study
干预措施: Befovacimab (BAY1093884) (Drug)
BAY1093884 225mg
Subjects received BAY1093884 225 mg once a week until premature termination of the study
干预措施: Befovacimab (BAY1093884) (Drug)
BAY1093884 400mg
Subjects received BAY1093884 400mg once a week until premature termination of the study
干预措施: Befovacimab (BAY1093884) (Drug)
结局指标
主要结局
Number of Participants With Drug-related Treatment-emergent Adverse Events
时间窗: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
An adverse event (AE) was any untoward medical occurrence (i.e., any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a participant in the study. Any bleeding event occurring during the study was not documented as an AE because this event was planned to be captured in the assessment of efficacy. AEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AEs (TEAEs). Drug-related TEAEs were TEAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Number of Participants With Clinically Relevant Abnormalities in Laboratory Values
时间窗: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
"Clinically relevant "implied the presence of a clinical sign or symptom that required medical action.
Number of Participants With Serious Treatment-emergent Adverse Events
时间窗: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
A serious adverse event (SAE) was any untoward medical occurrence that at any dose was resulting in death, was lifethreatening, requires hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity. SAEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as serious treatment-emergent AEs (TESAEs). Drug-related TESAEs were TESAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Number of Participants With Treatment-emergent Adverse Events of Special Interest
时间窗: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Any thromboembolic or thrombotic microangiopathic event or any hypersensitivity reaction was an adverse event of special interest (AESI). AESIs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AESIs.
次要结局
未报告次要终点