NCT01482143已完成1 期
Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)
研究概览
简要总结
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed diagnosis of FXS
- •At Screening and first baseline, vital signs, body weight and body mass index (BMI) must be age-specific within normal ranges.
排除标准
- •Use of any other investigational drug within 30 days or 5 half-lives (whichever is longer) of the investigational drug prior to screening until end of study visit.
- •History of hypersensitivity to AFQ056 or any mGluR antagonist.
- •Female patients who are confirmed or suspected to be sexually active.
- •History or presence of any clinically significant disease of any major system organ class, within the past 2 years prior to screening including but not limited to psychiatric, neurological, cardiovascular, endocrine, metabolic, renal, or gastrointestinal disorders (except for typical features of FXS).
- •Loss of ≥10% of total blood volume within 8 weeks (or less if required for this age group and/or by local regulation) prior to dosing or longer if required for this age group and/or by local regulation.
- •Significant illness that did not completely resolve at least four weeks prior to the first baseline visit.
- •Any abnormal laboratory values at screening or first baseline that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject.
- •Use of (or use within at least 5 half lives before dosing) concomitant medications that are strong/moderate inhibitors or inducers of CYP1A1/2, CYP2C9/19 or CYP3A4
- •History or presence of Hepatitis B/C or HIV at screening
研究组 & 干预措施
All Study subjects
Experimental
干预措施: AFQ056 (Drug)
结局指标
主要结局
The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)
时间窗: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
The area under the plasma (or serum or blood) concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUClast)
时间窗: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Maximum observed plasma concentration (Cmax)
时间窗: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
次要结局
- Electrocardiograms(Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7)
- Physical examination(Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7)
- Vital signs and body measurements(Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7)
- hematology(Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7)
- blood chemistry(Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7)
- neurological examination(Screening: once anytime between Day -30 and Day -1; once on Day 7)
- Adverse events (AE) monitoring(During the study (total of approximately 32 days) and 3 days after study completion)
- Serious adverse events (SAE) monitoring(During the study (total of approximately 32 days) and 30 days after study completion)
研究者
研究点 (1)
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