A Phase 1a, Randomized, Double-blinded, Placebo-Controlled, Single Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ELX-02 in Healthy Adult Volunteers
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Adverse Events
研究概览
简要总结
Phase 1 Single Ascending Dose Study in Normal Healthy Volunteers
详细描述
This is a study in humans of ELX-02, an advanced synthetic aminoglycoside optimized as a translational read-through drug (TRID) for the treatment of genetic conditions caused by nonsense mutations. This is a classical Phase 1a study designed as a randomized, double-blinded, placebo-controlled, single dose escalation to evaluate the safety, tolerability and pharmacokinetics of ELX-02 in healthy adult volunteers.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 45 Years（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
Placebo Comparator Arm
Placebo
干预措施: Placebo (Drug)
Active treatment
ELX-02
干预措施: ELX-02 (Drug)
结局指标
主要结局
Adverse Events
时间窗: 0-10 days
Incidence and characteristics of adverse events occurring following single doses of ELX-02
Pharmacokinetics: Mean residence time (MRT)
时间窗: 0-10 days
MRT will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Area under the plasma concentration-time curve calculated from time of administration to time 48h (AUC48h)
时间窗: 0-10 days
AUC48h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)
时间窗: 0-10 days
AUCinf will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Volume of distribution (Vd/F)
时间窗: 0-10 days
Vd/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Elimination half-life (t1/2)
时间窗: 0-10 days
t1/2 will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Clearance (CL/F)
时间窗: 0-10 days
CL/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Area under the plasma concentration-time curve calculated from time of administration to time 24h (AUC24h)
时间窗: 0-10 days
AUC24h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Maximum plasma concentration (Cmax)
时间窗: 0-10 days
Cmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Time at which Cmax occurs (tmax)
时间窗: 0-10 days
tmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
次要结局
未报告次要终点