A Phase 1, Randomized, Double-Blinded, Placebo-Controlled, Third Party Open, Multiple Dose Escalation, Single Center Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Subcutaneously Administered ELX-02 in Independent Consecutive Cohorts of Healthy Subjects
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 62
- 试验地点
- 1
- 主要终点
- Pharmacokinetic Parameter - Plasma AUC0-inf
研究概览
简要总结
Phase 1 Multiple 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 1b study designed as a randomized, double-blinded, placebo-controlled, multiple 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 55 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)
ELX-02
ELX-02
干预措施: ELX-02 (Drug)
结局指标
主要结局
Pharmacokinetic Parameter - Plasma AUC0-inf
时间窗: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post dose
Day 29 Area under the curve (AUC0-inf) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 29 extrapolated to infinity
Pharmacokinetic Parameter Plasma - Tmax
时间窗: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post-dose
Day 29 Time to maximum concentration (Tmax) of ELX-02 plasma concentrations following the subcutaneous (SC) dose on Day 29
Urine Pharmacokinetics Parameter - CLR24h
时间窗: 24 h
Renal clearance on Day 29 (CLR=Ae24h/plasmaAUC24h)
Urine Pharmacokinetics Parameter - Fe 12h on Day 29
时间窗: 12 h on Day 29
Percent of dose excreted (Fe) in urine on Day 29
Pharmacokinetic Parameter - Plasma Tmax
时间窗: Day 1: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post-dose
Day 1 Time to maximum concentration (Tmax) of ELX-02 plasma concentrations following the subcutaneous (SC) dose on Day 1
Pharmacokinetic Parameters - Plasma Cmax
时间窗: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post-dose
Day 29 Peak Plasma Concentration (Cmax) of ELX-02 following the subcutaneous (SC) dose on Day 29 to 72 hours post-dose
Pharmacokinetic Parameter - Plasma Rac(AUC24h)
时间窗: Day 1 and Day 24 hr
Accumulation ratio, calculated as AUC24h Day29/AUC24h Day 1
Pharmacokinetic Parameter - Plasma RAC(Cmax)
时间窗: Day 1 and Day 29
Accumulation ratio, calculated as Cmax Day29/Cmax Day 1
Urine Pharmacokinetics Parameter - Ae72h
时间窗: Day 29: pre-dose and during 0-12h, 12-24h, 24-48h, and 48-72h post-dose
Day 29 Cumulative amount of unchanged drug excreted into urine (Ae72h) of ELX-02 following the subcutaneous (SC) dose on Day 29
Urine Pharmacokinetic Parameter - Rmax
时间窗: Day 29: pre-dose and during 0-12h, 12-24h, 24-48h, and 48-72h post-dose
Day 29 Maximum rate of urinary extraction (Rmax) of ELX-02 in each collection time interval following the subcutaneous (SC) dose on Day 29
Urine Pharmacokinetics Parameter - CLR24h on Day 1
时间窗: 24 hours
Renal clearance on Day 1 (CLR=Ae24h/plasmaAUC24h)
Pharmacokinetic Parameters - Plasma AUC0-24
时间窗: Day 1: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, post-dose
Day 1 Area under the curve (AUC0-24) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 1 to 24 hours post-ose
Pharmacokinetic Parameters- Plasma AUC0-24
时间窗: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, post-dose
Day 29 Area under the curve (AUC0-24) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 29 to 24 hours post-dose
Urine Pharmacokinetics Parameter - Fe12h Day 1
时间窗: 12 hours
Percent of dose excreted (Fe) in urine on Day 1
次要结局
- Number of Patients Experiencing at Least One Treatment-Emergent Adverse Events (TEAEs)(Day 1-29)