A Phase I, Randomized, Double-blind, Placebo Controlled, Single Dose Escalation Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 16
- 主要终点
- Number of participants with adverse events as a measure of safety and tolerability
研究概览
简要总结
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors. This is a randomized, double-blind, placebo-controlled, single-dose, dose escalation study. It is designed to investigate the safety, tolerability, potential immunogenicity, pharmacokinetic and pharmacodynamic profile of BAY86-6150 (B0189) and to determine a dose or range of doses to be examined in subsequent studies.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Factorial
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •History of moderate or severe congenital hemophilia A or B with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX)
- •Male subjects 18-65 years of age inclusive
- •Able to dismiss factor replacement therapy during the course of the study unless required for the treatment of an acute bleeding episode
- •Written informed consent
- •Willing and able to comply with the requirements of the protocol
- •Have adequate venous access
- •Willing to use an effective method of contraception until Day 30 of their study participation
排除标准
- •Received factor replacement therapy or treatment with any other procoagulant therapeutics, or any antifibrinolytic agents, including blood products, at anytime within 5 days prior to administration of investigational medicinal product (IMP)
- •Planned administration of factor replacement therapy or treatment with any other procoagulant therapeutics or any antifibrinolytic agents, including blood products, at anytime during the study period
- •Acute bleeding episode or any ongoing bleeding episode at any time within 7 days prior to administration IMP
- •Clinically relevant coagulation disorder other than congenital hemophilia A or B
- •History of angina or receiving treatment for angina
- •History of coronary atherosclerotic disease, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) >/= 160 mmHg or diastolic blood pressure (DBP) >/= 90 mmHg
- •History of transient ischemic attack, stroke, myocardial infarction, coronary artery disease, congestive heart failure, or thromboembolic event
- •Active infection on day of IMP administration or septicemia at any time within 30 days prior to administration of IMP
研究组 & 干预措施
BAY Factor VII (6.5 µg/kg) / Placebo
n = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: BAY Factor VII (BAY86-6150) (Drug)
BAY Factor VII (6.5 µg/kg) / Placebo
n = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: Placebo (Drug)
BAY Factor VII (20 µg/kg) / Placebo
n = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: BAY Factor VII (BAY86-6150) (Drug)
BAY Factor VII (20 µg/kg) / Placebo
n = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: Placebo (Drug)
BAY Factor VII (50 µg/kg) / Placebo
n = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: BAY Factor VII (BAY86-6150) (Drug)
BAY Factor VII (50 µg/kg) / Placebo
n = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: Placebo (Drug)
BAY Factor VII (90 µg/kg) / Placebo
n = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: BAY Factor VII (BAY86-6150) (Drug)
BAY Factor VII (90 µg/kg) / Placebo
n = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Number of participants with adverse events as a measure of safety and tolerability
时间窗: Up to Day 50
次要结局
- Immunogenicity assessment, based on anti-BAY86-6150 binding antibody levels(3 time points from pre-dosing on Day 1 up to Day 50)
- Pharmacokinetic assessment, based on plasma concentration of BAY86-6150(9 time points from pre-dosing on Day 1 up to 48 hours post-dosing)
- Pharmacodynamic assessment, based on plasma hemostasis marker level(9 time points from pre-dosing on Day 1 up to 48 hours post-dosing)