JPRN-jRCT1031200137已完成4 期
An Observational Study to Investigate the Relationship between Plasma Lyso-Gb1 Concentration and the Treatment Outcomes of Gaucher disease patients in Japan - Lyso-Gb1
Contact for Clinical Trial Information0 个研究点目标入组 20 人开始时间: 2020年10月5日最近更新:
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
研究概览
简要总结
Clinically appropriate thresholds were set for the number of achieved goals regarding the 6 treatment goals of Gaucher disease, and summary statistics of Lyso-Gb1 concentrations were calculated to compare the factors by group of threshold. In 14 subjects with 100% achievement as the threshold and 6 subjects with other values, t-test yielded p=0.117 and Wilcoxon rank sum test yielded p=0.0588 for the between-group comparison.
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- ot applicable 至 ot applicable（—）
- 性别
- All
入选标准
- •1. In the opinion of the investigator, the participant is capable of understanding and complying with protocol requirements.
- •2. The participant or, when applicable, the participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.
- •3. Participants who are diagnosed as Gaucher disease.
- •4. Patients who have been treated with velaglucerase alfa for at least 3 months.
排除标准
- •Any reason that, in the Investigator's opinion, makes the patient unsuitable to participate in this study.
研究者
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