NCT04548791终止1 期
Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog Alfa (Activated) in Treatment of Episodic Bleeding in Subjects With Inherited Bleeding Disorders
Catalyst Biosciences27 个研究点 分布在 5 个国家目标入组 19 人开始时间: 2021年5月17日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 19
- 试验地点
- 27
- 主要终点
- Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
研究概览
简要总结
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of cohort: FVII deficiency, Glanzmann Thrombasthenia, or hemophilia A with inhibitors
- •Male or female, age 12 or older
- •History of frequent bleeding episodes
- •Affirmation of informed consent with signature confirmation and assent for children between ages 12 to 17 before any study related activities
- •Agreement to use highly effective birth control throughout the study if the subject has childbearing potential
排除标准
- •Genotype of FVIID subjects with identified mutations by central lab at screening
- •Previous participation in a clinical trial evaluating a modified rFVIIa agent
- •Received an investigational drug within 30 days or 5 half-lives or absence of clinical effect, whichever is longer
- •Known hypersensitivity to trial or related product
- •Known positive antibody to FVII or FVIIa detected by central lab at screening
- •Be immunosuppressed
- •Significant contraindication to participate
结局指标
主要结局
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
时间窗: Dosing period for each stage in a cohort will be approximately 5 to 11 days
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Bleeding episode treatment success
时间窗: 24 hours after the first administration of study drug
Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment
次要结局
未报告次要终点
研究者
研究点 (27)
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