Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 67
- 试验地点
- 48
- 主要终点
- Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods
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 alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes 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 analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.
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-008-00 (NCT00074984)
- •Patients must provide written informed consent prior to study participation
- •Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study
排除标准
- •The patient was unable to complete AGAL-008-00 (NCT00074984)
- •The patient has undergone kidney transplantation or is currently on dialysis
- •The patient has diabetes mellitus or presence of confounding renal disease
- •The patient has a clinically significant organic disease or an unstable condition that precludes participation
- •The patient is unwilling to comply with the protocol requirements
结局指标
主要结局
Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods
时间窗: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)
The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope.
次要结局
- Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months(Pre-Fabrazyme, 6, 12, and 18 months)
- Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months(Pre-Fabrazyme, 6, 12, and 18 months)
- Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months(Pre-Fabrazyme and 6, 12, and 18 months)
- Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months(Pre-Fabrazyme and 6, 12, and 18 months)