An Open Study of the Safety and Pharmacokinetics of GNR-038 in Sequential Dose-increase Cohorts in Healthy Volunteers
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- AO GENERIUM
- 入组人数
- 36
- 试验地点
- 2
- 主要终点
- Number of participants with Adverse Events
研究概览
简要总结
It is an open-label dose-escalating study in sequential cohorts to assess safety and pharmacokinetics of GNR-038.
详细描述
Hereditary angioedema (HAE) is a rare potentially life-threatening genetically determined disease associated with a deficiency or impairment of C1 esterase inhibitor (C1 inhibitor) functional activity. Main clinical manifestations of HAE are recurrent mucous membranes edema and localization of the derma deep layers. Attack persist from several hours to several days and disappear without a trace in most cases, without additional therapy.
The prevalence of the disease in the world is from 1:10 000 to 1: 150 000. The plasma/recombinant C1 inhibitor use to compensate for its deficiency or insufficient functional activity in patients with HAE is recommended both for severe and for long-term and short-term (before surgical interventions and dental manipulations) prophylaxis.
GNR-038 is a recombinant C1 inhibitor (rhC1-inh), which is a complete structural and functional analogue of the plasma C1 inhibitor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Months 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Number of participants with Adverse Events
时间窗: 28 Days
Adverse events, Laboratory tests, Vital signs, Physical examination, 12-lead electrocardiogram, Allergic associated reaction, Infusion associated reaction, Antidrug antibody.
次要结局
- Mean retention time (MRT)(Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Day 8)
- Area under the plasma concentration versus time curve (AUC)(Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Day 8)
- Elimination rate constant (Kel)(Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Day 8)
- Peak Plasma Concentration (Cmax)(45, 30 and 15 minutes before GNR-038 infusion; 0, 15, 30 minutes, 1, 2, 4, 8,12,16, 24, 48, 72, 96, 120,144 and 168 hours after GNR-038 infusion)
- Half-life (T1/2)(Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Day 8)
- Overall clearance (Cl)(Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Day 8)
- Kinetic volume of distribution (Vz)(Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Day 8)