NCT03746587终止2 期
Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
ZevraDenmark8 个研究点 分布在 1 个国家目标入组 39 人开始时间: 2018年6月6日最近更新:
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 39
- 试验地点
- 8
- 主要终点
- Primary Endpoint
研究概览
简要总结
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
详细描述
Evaluating the response of 3 dose levels of arimoclomol on various pharmacodynamic biomarkers in blood and cerebrospinal fluid as indicators of enhanced GBA in Gaucher Disease Type 1 or 3.
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
- Triple (Participant, Care Provider, Investigator)
盲法说明
Double-blinded, randomized, placebo controlled
入排标准
- 年龄范围
- 4 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Be able to understand and voluntarily sign informed consent
- •A diagnosis GD, either Type 1 or Type 3
- •For GD3 at least 1 neurological symptom
- •Age ≥ 4 years and ≤ 60 years at the time of enrolment
- •Plasma or serum chitotriosidase activity greater than 3 times the upper limit of normal.
排除标准
- •Recipient of a liver transplant or planned liver transplantation during the course of the study.
- •Splenectomy within 4 months of study entry or planned splenectomy during the course of the study.
- •Severe liver damage.
- •Severe renal insufficiency.
- •Body weight < 10 kg.
- •Other inclusion and exclusion criteria may apply
研究组 & 干预措施
Arimoclomol I
Experimental
Arimoclomol, oral capsule
干预措施: Arimoclomol (Drug)
Arimoclomol II
Experimental
Arimoclomol, oral capsule
干预措施: Arimoclomol (Drug)
Arimoclomol III
Experimental
Arimoclomol, oral capsule
干预措施: Arimoclomol (Drug)
Placebo
Placebo Comparator
Placebo oral capsule matching experimental arm
干预措施: Placebo oral capsule (Drug)
结局指标
主要结局
Primary Endpoint
时间窗: 6 months
The percentage change in serum chitotriosidase levels from baseline to 6 months
次要结局
- Growth Endpoint(6 months)
- Imaging Endpoint(6 months)
- Maturation Endpoint(6 - 12 months)
研究者
研究点 (8)
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