JPRN-jRCTs061180090已完成2 期
An Open-Label, Dose Escalation, Proof-of-Concept Clinical Trial of Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol - Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol
Maegaki Yoshihiro0 个研究点目标入组 25 人开始时间: 2019年3月22日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 25
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- ot applicable 至 ot applicable（—）
- 性别
- All
入选标准
- •1)Patients with neuronopathic Gaucher disease, defined by the presence of deficiency of glucocerebrosidase activities in lymphocytes/fibroblasts and 2 mutated GBA alleles.
- •2)Patients with known sequence variants associated with ABX effectiveness (such as F213I,N188S,R120W,G202R), or prior known positive result on in-vitro chaperone test using ABX.
- •3)Signed written informed consent
排除标准
- •1) Current participation in another investigational study, or using other investigational drug within 3 months prior to the participation to the trial.
- •2) Patients who are not appropriate to participate to the trial.
研究者
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