A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
2013年12月17日 更新者:Amicus Therapeutics
GR181413A/AT1001 (migalastat hydrochloride) is a low molecular weight iminosugar, an analog of the terminal galactose group that is cleaved from the substrate GL-3.
This compound was researched and developed as a drug for treatment of Fabry disease.
This study, MGM115806, will be the first administration of GR181413A/AT1001 to Japanese subjects to investigate the safety, tolerability and pharmacokinetics of single oral doses in healthy Japanese adult subjects.
Approximately 12 subjects will receive three treatments of 50, 150 and 450 mg GR181413A/AT1001 under fasted conditions plus placebo in a dose ascending crossover design.
Serial pharmacokinetic samples will be collected and safety assessments will be performed following each dose.
The pharmacokinetics and dose proportionality of GR181413A/AT1001 after single oral doses of GR181413A/AT1001 at the dose levels of 50, 150 and 450 mg under fasted conditions will be assessed.
研究概览
地位
完全的
条件
研究类型
介入性
注册 (实际的)
14
阶段
- 阶段1
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习地点
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New South Wales
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Randwick、New South Wales、澳大利亚、2031
- GSK Investigational Site
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参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
20年 至 55年 (成人)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Healthy as determined by a responsible and experienced physician, based on a medical evaluation including medical history, physical examination, laboratory tests and cardiac monitoring. A subject with a clinical abnormality or laboratory parameters outside the reference range for the population being studied may be included only if the Investigator and the GSK Medical Monitor agree that the finding is unlikely to introduce additional risk factors and will not interfere with the study procedures.
- Male or female between 20 and 55 years of age inclusive, at the time of signing the informed consent.
- A female subject is eligible to participate if she is of: Non-childbearing potential defined as pre-menopausal female.
- Male subjects with female partners of child-bearing potential must agree to use one of the contraception methods. This criterion must be followed from the time of the first dose of study medication until 5 terminal half-lives post-last dose.
- Body weight >=50 kg and BMI within the range 18.5 - 29.0 kg/m2 (inclusive).
- Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form.
- AST, ALT, alkaline phosphatase and bilirubin > 1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
- Single QTcB or QTcF < 450 msec; or QTc < 480 msec in subjects with Bundle Branch Block.
- Japanese defined being born in Japan, having four ethnic Japanese grandparents, holding a Japanese passport or identity papers and being able to speak Japanese. Japanese subjects should be also have lived outside Japan for less than 10 years.
Exclusion Criteria:
- A positive pre-study Hepatitis B surface antigen or positive Hepatitis C antibody result within 3 months of screening.
- A positive pre-study drug/alcohol screen.
- A positive test for HIV antibody.
- History of regular alcohol consumption within 6 months of the study
- The subject has participated in a clinical trial and has received an investigational product within the following time period prior to the first dosing day in the current study: 30 days, 5 half-lives or twice the duration of the biological effect of the investigational product (whichever is longest).
- Exposure to more than four new chemical entities within 12 months prior to the first dosing day.
- Use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements (including St Johns Wort) within 7 days (or 14 days if the drug is a potential enzyme inducer) or 5 half-lives (whichever is longer) prior to the first dose of study medication, unless in the opinion of the Investigator and GSK Medical Monitor the medication will not interfere with the study procedures or compromise subject safety.
- History of sensitivity to any of the study medications, or components thereof or a history of drug or other allergy that, in the opinion of the investigator or GSK Medical Monitor, contraindicates their participation.
- Where participation in the study would result in donation of blood or blood products in excess of 500 mL within a 56 day period.
- History or regular use of tobacco- or nicotine-containing products within 6 months prior to screening.
- Pregnant females as determined by positive serum or urine hCG test at screening or prior to dosing.
- Lactating females.
- Unwillingness or inability to follow the procedures outlined in the protocol.
- Subject is mentally or legally incapacitated.
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 分配:随机化
- 介入模型:交叉作业
- 屏蔽:单身的
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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安慰剂比较:安慰剂
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Powder for reconstitution
Matched, Size 2, hard gelatin capsule, white opaque/blue opaque
Solution matched
Size 2, hard gelatin capsule, white opaque / blue opaque
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实验性的:50 mg
GR181413A/AT1001
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Powder for reconstitution
Matched, Size 2, hard gelatin capsule, white opaque/blue opaque
Solution matched
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实验性的:150 mg
GR181413A/AT1001
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Size 2, hard gelatin capsule, white opaque / blue opaque
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实验性的:450 mg
GR181413A/AT1001
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Size 2, hard gelatin capsule, white opaque / blue opaque
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Profile of plasma pharmacokinetics
大体时间:0, 0.5, 1, 1.5, 2, 3, 3.5, 4, 5, 6, 8, 10, 12h post dose
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AUC, Cmax, tmax, Tlast , t1/2, CL/F, Vz/F
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0, 0.5, 1, 1.5, 2, 3, 3.5, 4, 5, 6, 8, 10, 12h post dose
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|
Number of Participants with adverse events
大体时间:up to 24 hr
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up to 24 hr
|
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Change from baseline in clinical laboratory tests (hematology, chemistry and urinalysis)
大体时间:0, 24h post dose
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0, 24h post dose
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|
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Change from baseline in vital signs (blood pressure and heart rate)
大体时间:0 ,1 , 2, 3, 4, 5, 6, 24h post dose
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0 ,1 , 2, 3, 4, 5, 6, 24h post dose
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Change from baseline in 12-lead ECG
大体时间:0, i, 2, 3, 6, 24h post dose
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0, i, 2, 3, 6, 24h post dose
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Profile of urine pharmacokinetics
大体时间:0-4, 4-10, 10-12, 12-24h post dose
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Ae, CLr, %Fx
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0-4, 4-10, 10-12, 12-24h post dose
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
出版物和有用的链接
负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始
2011年9月1日
初级完成 (实际的)
2011年12月1日
研究完成 (实际的)
2011年12月1日
研究注册日期
首次提交
2011年11月10日
首先提交符合 QC 标准的
2013年5月9日
首次发布 (估计)
2013年5月15日
研究记录更新
最后更新发布 (估计)
2013年12月18日
上次提交的符合 QC 标准的更新
2013年12月17日
最后验证
2013年12月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.