A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, PK, and Food Effect of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Healthy Participants.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 129
- 试验地点
- 4
- 主要终点
- Safety, as assessed by frequency and severity of adverse events (AEs)
研究概览
简要总结
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
详细描述
This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.
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
- •Healthy males and females ≥ 18 to ≤ 55 years of age
- •Body mass index ≥ 18.0 to ≤ 30.0 kg/m2
- •Body weight ≥ 50 kg at screening and Day -1
排除标准
- •Positive pregnancy test or currently breastfeeding.
研究组 & 干预措施
Placebo
Placebo as a tablet or capsule with dose based off of preceding cohort's data.
干预措施: GBT021601 (Drug)
GBT021601
GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.
干预措施: GBT021601 (Drug)
结局指标
主要结局
Safety, as assessed by frequency and severity of adverse events (AEs)
时间窗: 119 days from screening Part A, 134 days from screening Part B
AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Safety, as assessed by changes in Heart Rate.
时间窗: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in heart rate (bpm) as compared to baseline.
Safety, as assessed by changes in eGFR
时间窗: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in eGFR from baseline
Safety, as assessed by changes in alanine aminotransferase (ALT)
时间窗: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in alanine aminotransferase (ALT)
Safety, as assessed by changes in Blood pressure
时间窗: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Plasma concentration
时间窗: 134 days from screening Part B
Cmax on D1-D15
次要结局
- Determine whole blood concentration of GBT021601(119 days from screening Part A)
- Safety, as assessed by changes in QTcF(119 days from screening Part A, 134 days from screening Part B)
- Determine plasma concentration of GBT021601.(134 days from screening Part B)