NCT04246060Enrolling By Invitation不适用
Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis
Chiesi SA/NV7 个研究点 分布在 1 个国家目标入组 31 人开始时间: 2020年7月31日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 31
- 试验地点
- 7
- 主要终点
- to descriptively assess the change from baseline and percent change from baseline in the quality of life overall summary sores at 12 months in switch cohort
研究概览
简要总结
Most of the real world evidence data related to efficacy of cysteamine therapy is retrospective. This study is a ambispective study to investigate the impact of cystine depletion therapy on the quality of life of patients and their parents.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Cohort 1
Patients on extended release cysteamine treatment at study enrollment
干预措施: Cysteamine Bitartrate (Drug)
Cohort 2
Patients switching from immediate release cysteamine to extended release cysteamine during the study
干预措施: Cysteamine Bitartrate (Drug)
Cohort 3
Patients remaining on immediate release cysteamine treatment
干预措施: Cysteamine Bitartrate (Drug)
结局指标
主要结局
to descriptively assess the change from baseline and percent change from baseline in the quality of life overall summary sores at 12 months in switch cohort
时间窗: 12 months
次要结局
未报告次要终点
研究者
研究点 (7)
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