NCT02520934Unknown不适用
Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Improve in Purdue Pegboard test speed
研究概览
简要总结
evaluate the combination therapy with Miglustat and enzyme replacement therapy (ERT) on Gaucher disease
详细描述
understand if Miglustat (glucosylceramide synthase inhibitor) could improve neuropathy in patients with Gaucher disease
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Confirmed diagnosis of Gaucher Disease: blood test shown lack of beta- glucocerebrosidase, and found L444P homozygous on GBA gene.
- •Aged 6 years old or above.
- •Already have regular ERT (30-120 IU/kg/ every 2 weeks) at least a year; dosage and frequency of ERT had not been changed in recent 3 months.
排除标准
- •History of tremor and abnormal extremities perception ( pain, numbness, tingle etc.)
- •Abnormal kidney function.
- •Pregnant or plan to have a baby ( potentially pregnant patient need to be transferred to gynecologist for the test and promise to have proper contraception measures).
- •Allergic to Miglustat.
- •Control_normal Inclusion Criteria
- •Age 6-18 years
- •No significant physical, mental, or psychiatric problems
- •Exclusion criteria
- •Children with eye disease (not include myopia, hyperopia, Astigmatism)
研究组 & 干预措施
Case_Miglustat
Experimental
Besides regular ERT, patients in this group also need to take Miglustat for 24 months.
干预措施: Miglustat (Drug)
Case_Miglustat
Experimental
Besides regular ERT, patients in this group also need to take Miglustat for 24 months.
干预措施: ERT (Drug)
结局指标
主要结局
Improve in Purdue Pegboard test speed
时间窗: 24 months
次要结局
未报告次要终点
研究者
研究点 (1)
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