A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers With an Open-Label, Randomized, Two-Way Crossover Arm
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 31
- 试验地点
- 1
- 主要终点
- Plasma pharmacokinetics of migalastat
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat HCl as compared to IV administered migalastat HCl in healthy volunteers. The data from this study will help us understand how migalastat works in the body and will help us determine what would be an effective dose in future studies with migalastat hydrochloride.
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
- Double (Participant, Investigator)
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
- •Males and females between 18 and 45 years of age.
- •Body weight range ≥ 50 kg ≤ 100 kg and BMI within the range 18.5 - 29.9 kg/m
- •Healthy as determined by a responsible and experienced physician, based on a medial evaluation.
- •Male and female subjects of childbearing potential agree to adhere to the contraception requirements.
- •Capable of giving written informed consent.
排除标准
- •History of sensitivity to migalastat HCl or related iminosugars (eg, miglitol, miglustat), or other significant drug allergy.
- •Past medical history, or physical examination findings, of clinically significant abnormalities that may put the subject at risk or interfere with outcome variables.
- •Positive pre-study drug/alcohol screen.
- •Pregnant or lactating females.
- •The subject has participated in a clinical trial and has received an investigational product within 60 days prior to the first dosing day in the current study.
研究组 & 干预措施
1 mg/kg
IV infusion of migalastat HCl or placebo
干预措施: IV placebo (Drug)
10 mg/kg
IV infusion of migalastat HCl or placebo
干预措施: IV migalastat HCl (Drug)
0.3 mg/kg
IV infusion of migalastat HCl or placebo
干预措施: IV migalastat HCl (Drug)
0.3 mg/kg
IV infusion of migalastat HCl or placebo
干预措施: IV placebo (Drug)
1 mg/kg
IV infusion of migalastat HCl or placebo
干预措施: IV migalastat HCl (Drug)
10 mg/kg
IV infusion of migalastat HCl or placebo
干预措施: IV placebo (Drug)
150 mg IV
150 mg single IV infusion
干预措施: IV migalastat HCl (Drug)
150 mg IV
150 mg single IV infusion
干预措施: oral migalastat HCl (Drug)
150 mg oral
150 mg single oral dose
干预措施: IV migalastat HCl (Drug)
150 mg oral
150 mg single oral dose
干预措施: oral migalastat HCl (Drug)
结局指标
主要结局
Plasma pharmacokinetics of migalastat
时间窗: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48
To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects.
Safety and tolerability of migalastat
时间窗: 48 hours
Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations
次要结局
- Urinary pharmacokinetics(Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion)
- Plasma pharmacokinetics of migalastat(Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48)