NCT00517153已完成2 期
A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease
Actelion0 个研究点目标入组 29 人开始时间: 2002年1月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 29
- 主要终点
- The primary endpoints will be two parameters - Horizontal Saccadic α and Horizontal Saccadic β - which are estimated for each patient from their saccadic eye movement data
研究概览
简要总结
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a non-treatment group. Both groups will follow an identical visit schedule.
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
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with Niemann-Pick Type C disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
- •Patients who can ingest a capsule.
- •Patients who are above the age of four (4) years of age.
排除标准
- •Patients younger than 18 years who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
- •Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
- •Sexually active and fertile patients who do not agree to use adequate contraception throughout the study and for three months after cessation of OGT 918 treatment.
- •Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
- •Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
- •Patients suffering from clinically significant diarrhea (>3 liquid stools per day for >7 days) without definable cause within 3 months of Screening Visit, or who have a history of significant gastrointestinal disorders.
- •Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection.
- •Patients who in the opinion of the Investigator (for whatever reason) are thought to be unsuitable for the study.
- •Patients with an adjusted Creatinine Clearance of less than 70 ml/min/1.73m2 (CrCl <70).
- •Patients younger than four (4) years of age.
研究组 & 干预措施
1
Experimental
OGT-918 - Zavesca (miglustat)
干预措施: miglustat (Drug)
结局指标
主要结局
The primary endpoints will be two parameters - Horizontal Saccadic α and Horizontal Saccadic β - which are estimated for each patient from their saccadic eye movement data
时间窗: Baseline to Month 12
次要结局
- Highest amplitude for which a velocity measurement is obtained(Baseline to Month 12)
研究者
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