A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 58
- 试验地点
- 39
- 主要终点
- Safety and efficacy
研究概览
简要总结
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid ("globatriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
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
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have successfully completed the previous double-blind study (AGAL-1-002-98)
- •Patients must provide written informed consent prior to study participation
- •Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted method of contraception throughout the study
排除标准
- •Patient has undergone kidney transplant or is currently on dialysis
- •Patient is pregnant or lactating
- •Patient is unwilling to comply with the requirements of the protocol
- •Patient has a clinically significant organic disease (with the exception of symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would preclude participation in the study
结局指标
主要结局
Safety and efficacy
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
次要结局
- Changes in McGill Pain Questionnaire
- Autonomic status
- Glomerular filtration
- Functional assessment of urinary protein excretion Ophthalmic changes
- SF-36 Health Survey
- Physician's assessment of Fabry Symptoms and pain medication