NCT00004293Unknown2 期
Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
National Center for Research Resources (NCRR)2 个研究点 分布在 1 个国家目标入组 24 人开始时间: 1999年11月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 24
- 试验地点
- 2
研究概览
简要总结
OBJECTIVES:
I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.
详细描述
PROTOCOL OUTLINE:
Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.
研究设计
- 研究类型
- Interventional
- 主要目的
- Treatment
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •PROTOCOL ENTRY CRITERIA:
- •-Disease Characteristics-- Gaucher disease with glucocerebrosidase deficiency confirmed by enzymatic or molecular assay At least 3 organ systems affected, based on the following criteria: Anemia Thrombocytopenia Organomegaly Bone deterioration on radiograph Pulmonary compromise Symptoms compromise daily activities or risk longevity No neurologic disease
排除标准
- 未提供
研究者
研究点 (2)
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