A Randomized, Open-label, Active Comparator, 2-arm, Prospective Study to Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 发起方
- Sanofi
- 主要终点
- Change in Plasma globotriaosylsphingosine (lyso-GL3) level
研究概览
简要总结
Primary Objective:
To assess reduction of plasma lyso-GL3 level after switch to agalsidase beta from agalsidase alfa
Secondary Objectives:
- To assess reduction of kidney podocyte GL3 content after switch to agalsidase beta from agalsidase alfa
- To assess reduction of GL3 content in endothelial skin cells after switch to agalsidase beta from agalsidase alfa
- To assess change in renal function after switch to agalsidase beta from agalsidase alfa
- To assess disease severity and clinical changes after switch to agalsidase beta from agalsidase alfa
- To assess improvement in symptoms of Fabry disease after switch to agalsidase beta from agalsidase alfa
详细描述
The study will have a screening period of up to 9 weeks. Eligible participants will be randomized to switch to agalsidase beta or to continue agalsidase alfa in a 1:1 ratio for a period of 12 months (52 weeks).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 45 Years（Child, Adult）
- 性别
- Male
- 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
agalsidase beta
Commercially available agalsidase beta treatment at approved dose and regimen;administered once every 2 weeks as an IV infusion
干预措施: agalsidase beta (GZ419828) (Drug)
agalsidase alfa
Commercially available agalsidase alfa treatment at approved dose and regimen; administered once every 2 weeks as an IV infusion
干预措施: agalsidase alfa (Drug)
结局指标
主要结局
Change in Plasma globotriaosylsphingosine (lyso-GL3) level
时间窗: Baseline, 12 months (week 52)
Change from baseline to 12 months (week 52) for plasma lyso-GL3 level
次要结局
- Change in GL3 content in podocytes(Baseline, 12 months (week 52))
- Change in GL3 content in endothelial skin cells(Baseline, 12 months (week 52))
- Change in measured glomerular filtration rate (mGFR)(Baseline, 12 months (week 52))
- Change in estimated glomerular filtration rate (eGFR) calculated(Baseline, 12 months (week 52))
- Change in Mainz Severity Score Index (MSSI) total score(Baseline, 12 months (week 52))
- Change in Fabry Disease Patient Reported Outcomes (FD-PRO) total symptom score(Baseline, 12 months (week 52))