NCT00258778已完成1 期
A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers
Protalix0 个研究点目标入组 6 人开始时间: 2005年11月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 6
- 主要终点
- Safety as measured by:
研究概览
简要总结
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD)leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer)in the cells of the monocyte-macrophage system.
This is the first trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD)which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Healthy male or female between 18 and 45 years of age.
- •Female subjects must agree to use a medically acceptable method of contraception at all times during the study and must have a negative serum pregnancy test at baseline and during the study period.
- •Females of child-bearing potential must be non-pregnant and not lactating and using adequate birth control such as oral contraceptives.
- •Negative laboratory tests for HIV, HBsAg or HCV.
- •Naive to any previous recombinant protein therapy.
- •Provide written informed consent.
- •Have the ability to understand the requirements of the study and to comply with the study protocol and dosing regimen.
排除标准
- •Have clinical evidence of any active significant disease that could potentially compromise the ability of the investigator to evaluate or interpret the effects of the study treatment on safety assessment and thus increase the risk to the subject to unacceptable levels.
- •Are pregnant or nursing.
- •Presence of any acute or chronic diseases.
- •Have a history of any allergies.
- •Have been exposed to long-term steroid treatment.
- •Had a minor operation in the last 6 months.
- •Have ever been exposed to any previous recombinant protein therapy.
- •Have received immuno-suppressive treatment.
- •Have a positive HIV, HBsAG and HCV laboratory result.
- •Use any medication other than vitamins or oral contraceptives (for female).
- •Have participated in another clinical trial during the previous 3 months
- •Have history of alcohol or drug abuse.
- •Are considered by the Investigator to be unsuitable candidate for this study.
结局指标
主要结局
Safety as measured by:
adverse events
change in vital signs
physical examination
laboratory test results
次要结局
- Pharmacokinetic parameters
- Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria
研究者
相似试验
已完成
3 期
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher DiseaseGaucher DiseaseNCT00376168Pfizer32
已完成
3 期
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension TrialGaucher DiseaseNCT00705939Pfizer45
已完成
2 期
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With ImigluceraseGaucher DiseaseNCT00478647Shire40
招募中
1 期
Phase 1/2 Study of CAN103 in Subjects With Gaucher DiseaseGaucher Disease, Type 1Gaucher Disease, Type 3NCT05447494CANbridge (Suzhou) Bio-pharma Co., Ltd.40
已完成
3 期
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher DiseaseGaucher Disease, Type 1NCT00553631Shire34