CTRI/2020/09/028170尚未招募未知
yso-Gb1 as long-term prognostic biomarker in Gaucher disease - an international multicenter epidemiological study to determine the prognostic value of lyso-Gb1 for monitoring the progress of Gaucher disease - LYSO-PROOF
Prasad Research Foundation0 个研究点目标入组 0 人开始时间: 待定最近更新:
试验速览
- 阶段
- 未知
- 状态
- 尚未招募
- 发起方
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
入选标准
- •1. Patients with genetically confirmed diagnosis of Gaucher disease type 1
- •2. No prior treatment with enzyme replacement therapy or substrate reduction therapy no treatment for more than 24 months
- •3. Signed informed consent by parents/legal guardian and patient
排除标准
- •1. Male or female patients being younger than 6 months old
- •2. Patients without genetically confirmed diagnosis of Gaucher disease type 1
- •3. Gaucher disease type 2 or 3
- •4. The patient is currently undergoing enzyme replacement therapy or substrate reduction therapy
- •5. Missing signed informed consent
研究者
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