A Non-interventional Post Authorization Study (PASS) to Evaluate Long-term Safety of Orfadin Treatment in Hypertyrosinemia Type 1 (HT-1) Patients in Standard Care
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 315
- 试验地点
- 2
- 主要终点
- Occurrence of Adverse events related to renal function
研究概览
简要总结
The purpose of this study is to look at the long term safety profile of Orfadin treatment in patients suffering from hereditary tyrosinemia type 1 (HT-1). Patients included in the study will use Orfadin according to normal clinical practice.
详细描述
The planned study is a non-interventional study that will look at the long-term safety of Orfadin treatment in patients suffering from hereditary tyrosinemia type 1. Orfadin will be used according to normal practice . There is an ongoing post-marketing surveillance (PMS) program to monitor hepatic, renal, hematological, neurological and ophthalmic status in all patients treated with Orfadin. The Committee for medicinal Products for Human Use (CHMP) has required this program and have looked at the data for approximately 400 patients and found the benefit-risk ratio to be positive. The present study (PASS) will replace the ongoing PMS. The transition of countries will be gradual; starting in 2013.The study will include HT-1 patients on Orfadin treatment in standard clinical care as well as newly diagnosed patients just starting with Orfadin treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All HT-1 patients receiving Orfadin treatment are eligible for entry.
排除标准
- •No exclusion criteria
研究组 & 干预措施
HT-1 patients on Orfadin treatment
HT-1 patients on Orfadin (nitisinone) treatment
干预措施: Nitisinone (Drug)
结局指标
主要结局
Occurrence of Adverse events related to renal function
时间窗: from 2005
Occurrence of Adverse events related to renal function
Occurrence of Adverse events related to hepatic function
时间窗: from 2005
Occurrence of Adverse events related to hepatic function
Occurrence of Adverse events related to Ophthalmological function
时间窗: from 2005
Occurrence of Adverse events related to Ophthalmological function
Occurrence of Adverse events related to hematological function
时间窗: from 2005
Occurrence of Adverse events related to hematological function
Occurrence of Adverse events related to cognitive developmental function
时间窗: from 2005
Occurrence of Adverse events related to cognitive developmental function
次要结局
- Occurrence of liver transplantation(from 2005)
- Occurrence of discontinuation of Orfadin treatment(from 2005)
- Occurrence of death(from 2005)
- Occurrence of other Adverse Events(from 2005)