A Single/Multiple Ascending Dose Phase 1 Study of the Safety, Tolerability and Pharmacologic Activity of BT200 in Normal Human Volunteers
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 112
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
研究概览
简要总结
Study BT200-01 is a first in human (FIH) study in male and female normal human volunteers (NHVs) that uses an Integrated Protocol Design. This Phase 1 study will comprise 4 sub-parts: Part A, a single ascending dose (SAD) study; Part B, a multiple ascending dose (MAD) study; Part C, a desmopressin challenge study to explore (i) whether desmopressin could be used as an antidote, and/or (ii) whether desmopressin stimulated vonWillebrand Factor (VWF) release is overcome with increasing BT200 doses; and Part D, a relative bioavailability (BA) study.
The primary objective of this study is to assess the safety and tolerability profile of BT200 in NHVs.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Healthy male or female volunteers, age ≥ 18 years old at screening
- •If female, must be post-menopausal or status post hysterectomy
- •Able to comprehend and to give informed consent
- •Able to cooperate with the Investigator, to comply with the requirements of the study, and to complete the full sequence of protocol-related procedures
排除标准
- •Clinically significant medical history (including von Willebrand Disease, thrombocytopathy, or any type of bleeding diathesis) or ongoing chronic illness that would jeopardize the safety of the subject or compromise the quality of the data derived from his/her participation in this study
- •Clinically relevant abnormal findings on physical examination or clinically relevant laboratory abnormalities
- •History of infusion hypersensitivity reactions, significant drug allergy, or anaphylactic reactions
- •Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the subject to be able to comply fully with study procedures
- •Use of medication during 2 weeks before the start of the study, which in the judgment of the Investigator may adversely affect the subject's welfare or the integrity of the study's results
- •Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 elimination half-lives (whichever is longer) prior to treatment start
研究组 & 干预措施
BT200 0.18mg
Subjects will receive a single subcutaneous dose of BT200 0.18mg
干预措施: BT200 (Drug)
BT200 0.6mg
Subjects will receive a single subcutaneous dose of BT200 0.6mg
干预措施: BT200 (Drug)
BT200 1.8mg
Subjects will receive a single subcutaneous dose of BT200 1.8mg
干预措施: BT200 (Drug)
BT200 6.0mg
Subjects will receive a single subcutaneous dose of BT200 6.0mg
干预措施: BT200 (Drug)
BT200 12.0mg
Subjects will receive a single subcutaneous dose of BT200 12.0mg
干预措施: BT200 (Drug)
BT200 24.0mg
Subjects will receive a single subcutaneous dose of BT200 24.0mg
干预措施: BT200 (Drug)
BT200 24.0mg rep
Subjects will receive a single subcutaneous (SC) dose of BT200 24.0mg by gradual SC infusion
干预措施: BT200 (Drug)
Placebo SAD
Subjects will receive a single subcutaneous dose of placebo
干预措施: Placebo (Drug)
BT200 loading dose 12.0mg, maintenance doses of 12.0 mg
Subjects will receive an initial subcutaneous loading doses of BT200 12.0mg followed by 4 weekly (every 7 days) maintenance doses of BT200 12.0mg
干预措施: BT200 (Drug)
BT200 loading doses 24.0mg, maintenance doses of 24.0 mg
Subjects will receive an initial subcutaneous loading dose of BT200 24mg followed by 4 weekly (every 7 days) maintenance doses of BT200 24mg
干预措施: BT200 (Drug)
Placebo MAD
Subjects will receive an initial subcutaneous loading dose of Placebo followed by 4 weekly (every 7 days) maintenance doses of placebo
干预措施: Placebo (Drug)
BT200 48.0mg + desmopressin challenge
Subjects will receive a single subcutaneous dose of BT200 48.0mg followed by IV infusion (over 30 min) of 0.3µg/kg desmopressin administered 24 hours after single dose of BT200
干预措施: BT200 (Drug)
BT200 48.0mg + desmopressin challenge
Subjects will receive a single subcutaneous dose of BT200 48.0mg followed by IV infusion (over 30 min) of 0.3µg/kg desmopressin administered 24 hours after single dose of BT200
干预措施: Desmopressin (Drug)
Placebo + desmopressin challenge dose
Subjects will receive a single subcutaneous dose of placebo followed by IV infusion (over 30 min of 0.3µg/kg desmopressin administered 24 hours after single dose of placebo
干预措施: BT200 (Drug)
Placebo + desmopressin challenge dose
Subjects will receive a single subcutaneous dose of placebo followed by IV infusion (over 30 min of 0.3µg/kg desmopressin administered 24 hours after single dose of placebo
干预措施: Desmopressin (Drug)
Placebo infusion
Subjects will receive a single IV dose of placebo administered over 24 hours
干预措施: Placebo (Drug)
BT200 36.0mg
Subjects will receive a single subcutaneous (SC) dose of BT200 36.0mg by gradual SC infusion
干预措施: BT200 (Drug)
BT200 48.0 mg
Subjects will receive a single subcutaneous dose (SC) of BT200 48.0mg by gradual SC infusion
干预措施: BT200 (Drug)
BT200 18.0 mg
Subjects will receive a single subcutaneous (SC) dose of BT200 18.0mg by gradual SC infusion
干预措施: BT200 (Drug)
BT200 24mg IV infusion
Subjects will receive a single IV dose of BT200 24mg administered over 24 hours
干预措施: BT200 (Drug)
结局指标
主要结局
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
时间窗: Baseline through 8 weeks after dosing up to 56 days
Any AE or bleeding event related to study treatment
次要结局
- Measured Area Under the Curve (AUC)(Baseline through 8 weeks after dosing up to 56 days)
- Maximum Plasma Concentration (Cmax)(Baseline through 8 weeks after dosing up to 56 days)
- Time to Maximum Plasma Concentration (Tmax)(Baseline through 8 weeks after dosing up to 56 days)