NCT02528617撤回4 期
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa.
研究概览
简要总结
The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Enzyme Replacement Therapy naive,
- •confirmed diagnosis of Gaucher disease type 1 or 3,
- •able to travel to Dallas, Texas 1x per year for baseline plus 3 consecutive years,
- •able to tolerate all study procedures,
- •skeleton not fully formed as confirmed by DXA and MRI),
- •and willing to receive velaglucerase alfa infusions every other week for the duration of the study.
排除标准
- •Clinically unstable,
- •taking or have taken bisphosphonates,
- •Gaucher type 2,
- •pregnant female,
- •or deemed inappropriate for participation by the principal investigator.
研究组 & 干预措施
Gaucher Type 1 or 3
Other
Velaglucerase alfa IV 60 units/kg every other week for duration of the study.
干预措施: Velaglucerase alfa (Drug)
结局指标
主要结局
Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa.
时间窗: Baseline pre-intervention and yearly thereafter for 3 years
次要结局
- Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa.(Baseline pre intervention and yearly thereafter for 3 years)
- Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa.(Baseline pre intervention and yearly thereafter for 3 years)
- Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa.(Baseline pre-intervention and yearly thereafter for 3 years.)
- Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa.(Baseline pre-intervention and yearly thereafter for 3 years)
研究者
研究点 (1)
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