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 IZD334 in Healthy Adult Participants as Well as an Open-label Cohort to Confirm the Safety, Pharmacokinetics, and Pharmacodynamics 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
- 64
- 试验地点
- 1
- 主要终点
- Area under the plasma concentration versus time curve (AUC)- single dose
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 IZD334 conducted in healthy adult participants as well as an open-label cohort in adult patients with CAPS. The study is designed to evaluate the safety, tolerability, PK, PD, and food effect of IZD334 in healthy adult participants, and to evaluate the safety, tolerability, PK, PD, and preliminary clinical efficacy of IZD334 in adult patients with CAPS.
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)
盲法说明
Healthy volunteer section is double blind.
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •(Healthy Volunteers)
- •Healthy male or female volunteers, aged 18 to 65 years (inclusive at the time of informed consent)
- •Participants must be in good general health, with no significant medical history, have no clinically significant abnormalities on physical examination at Screening and/or before administration of the initial dose of study drug
- •Participants must have a Body Mass Index (BMI) between ≥18.0 and ≤32.0 kg/m2 at Screening
- •Participants must have clinical laboratory values within normal range as specified by the testing laboratory, unless deemed not clinically significant by the Investigator or delegate
- •Inclusion Criteria: (CAPS Patients)
- •*Patients with a confirmed diagnosis of CAPS (FCAS or MWS) aged 18 to 65 years (inclusive at the time of informed consent)
排除标准
- •(Healthy volunteer)
- •Pregnant or lactating at Screening or planning to become pregnant (self or partner) at any time during the study, including the follow-up period
- •Prior or ongoing medical conditions, medical history, physical findings, or laboratory abnormality that, in the Investigator's (or delegate's) opinion, could adversely affect the safety of the participant
- •Presence of any underlying physical or psychological medical condition that, in the opinion of the Investigator, would make it unlikely that the participant will comply with the protocol or complete the study per protocol
- •Blood donation or significant blood loss within 60 days prior to the first study drug administration
- •Exclusion Criteria: (CAPS Patients)
- •Live vaccinations within 3 months prior to Screening, for the duration of the study and for up to 3 months following the last dose of study drug;
- •Positive QuantiFERON test at the Screening visit or within 2 months prior to Screening.
研究组 & 干预措施
Single Ascending Dose
Once daily oral IZD334 or Placebo
干预措施: IZD334 (Drug)
Single Ascending Dose
Once daily oral IZD334 or Placebo
干预措施: Placebos (Drug)
Multiple Ascending Dose
Once or twice daily oral IZD334 or Placebo
干预措施: IZD334 (Drug)
Multiple Ascending Dose
Once or twice daily oral IZD334 or Placebo
干预措施: Placebos (Drug)
结局指标
主要结局
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)-multiple dose
时间窗: Days 1-9
Peak plasma concentration following multiple dose administration
Incidence of treatment emergent adverse events [Safety and Tolerability]
时间窗: Day 1-16 for MAD
Incidence, frequency and severity of treatment emergent adverse events.
Peak plasma concentration (Cmax) single dose
时间窗: Day 1-3
Peak plasma concentration following single dose administration
Area under the plasma concentration versus time curve (AUC)- multiple dose
时间窗: Days 1-9
AUC following multiple dose administration
次要结局
- Reduction in CAPS symptom scores(Day 1-15)
- Reduction of IL-1 production in stimulated whole blood(Day 1-3 for SAD and Day 1-9 for MAD])