Open-label Comparative Cohort Non-interventional Epidemiological Study of the Efficacy and Safety of Enzyme Replacement Therapy in Russian Patients With Fabry Disease
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 100
- 试验地点
- 23
- 主要终点
- Change from Baseline in Lyso-Gb3 Level
研究概览
简要总结
The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is:
What medical problems do participants have?
Participants already on enzyme replacement therapy as part of their regular medical care for Fabry disease will be observed as usual. Their data will be collected by their doctor.
详细描述
All patients on enzyme replacement therapy could be enrolled in the study. Three medicines are currently available in Russia (Fabagal®, Fabrazyme®, Replagal®) so the patients form three cohorts.
For all patients retrospective data are collected for 12 months prior enrollment.
All patients are observed prospectively for 6 months. At the point of 6 months patients taking Fabrazyme or Replagal at the moment finish the study.
Patients on Fabagal are observed for 12 more months (up to 18 months after enrollment).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 8 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men and women with a confirmed diagnosis of Fabry disease;
- •Prescribed course of enzyme replacement therapy with any drug (Fabagal, Fabrazyme, Replagal);
- •Age 8 - 65 years;
- •Signed informed consent of the patient and/or patient's legal representative for participation in the study and processing of personal data.
排除标准
- •Renal replacement therapy or kidney transplantation;
- •Critical illness unrelated to Fabry disease;
- •Pregnancy or breastfeeding;
- •Refusal of the patient (or patient's legal representative) from further participation in the study or medical care.
结局指标
主要结局
Change from Baseline in Lyso-Gb3 Level
时间窗: From 12 months before enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation
Level of globotriaosylsphingosine (lyso-Gb3) is measured in dried blood spots.
次要结局
- Change from Baseline in α-GAL Activity(From 12 months before enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation)
- Change from Baseline in GFR(From 12 months before enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation)
- Change from Baseline in Neuropathic Pain Level(From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation)
- Incidence Rate of Significant Clinical Outcomes(From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation)