A Phase 1, Randomised, Double-Blind, Placebo Controlled, Single and Multiple Ascending Dose Study to Determine the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Food Effect of Inzomelid in Healthy Adult Participants as Well as an Open Label Cohort to Confirm the Safety, Pharmacokinetics, and Pharmacodynamics of Inzomelid in Adult Patients With Cryopyrin-Associated Periodic Syndromes
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- Incidence of treatment emergent advert events [Safety and Tolerability]
研究概览
简要总结
This is a first in human (FIH), single-centre, double blind, randomised, cross-over, SAD followed by a MAD study of Inzomelid. The study is designed to evaluate the safety, tolerability, PK, PD, and food effect of Inzomelid in healthy adult participants and to evaluate the safety, tolerability, PK, PD, and preliminary clinical efficacy of Inzomelid in adult patients with CAPS. Oversight will be provided by a safety monitoring committee (SMC) comprised of the Investigator, the Sponsor's Medical Monitor and an Independent Medical Monitor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older 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
研究组 & 干预措施
Single Ascending Dose
Inzomelid or Placebo
干预措施: Inzomelid (Drug)
Single Ascending Dose
Inzomelid or Placebo
干预措施: Placebo (Drug)
Multiple Ascending Dose
Inzomelid or Placebo
干预措施: Inzomelid (Drug)
Multiple Ascending Dose
Inzomelid or Placebo
干预措施: Placebo (Drug)
Patients with CAPS
Inzomelid Open Label
干预措施: Inzomelid (Drug)
结局指标
主要结局
Incidence of treatment emergent advert events [Safety and Tolerability]
时间窗: Day 1-8 for SAD, Day 1-16 for MAD
Incidence, frequency and severity of treatment emergent advert events
Area under the plasma concentration versus time curve (AUC)- single dose
时间窗: Day 1-3
AUC following single dose administration
Peak Plasma Concentration (Cmax)-single dose
时间窗: Day 1-3
Peak plasma concentration following single dose administration
Peak Plasma Concentration (Cmax)-multiple dose
时间窗: Days 1-16
Peak plasma concentration following multiple dose administration
Area under the plasma concentration versus time curve (AUC)- multiple dose
时间窗: Days 1-16
AUC following multiple dose administration
次要结局
- Pharmacodynamic activity(Day 1-3 for SAD and Day 1-9 for MAD)
- Reduction in CAPS symptom scores(Days 1-15)