A Prospective, Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in Hereditary Tyrosinemia Type 1 (HT-1) Patients in Routine Clinical Care in China
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Occurrence of hepatic, renal or hematological adverse events (AEs) or death
研究概览
简要总结
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
详细描述
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study, though the expectation is that most of the tests and examinations listed in the protocol will be performed in the context of routine clinical care and relevant data will be captured. At enrollment, data on patient treatment, medical and surgical history together with other patient characteristics will be captured.Patients enrolled in the study will be followed for at least 1 year and for a maximum of 3.5 years.
The study aims to enroll at least 15 HT-1 patients aged 0-18 years. If adult patients are enrolled the study population will be larger as all eligible patients will be invited to participate. However, the enrollment will close when the target of 15 patients aged 0-18 years has been reached.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with a confirmed diagnosis of HT-1 treated with, or at enrollment prescribed, Nitisinone treatment (product manufactured by Sobi) in a routine clinical care setting. The decision to initiate treatment shall be made by the treating physician independently from the decision to include the patient in the study.
- •Signed and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age, should be obtained before any study-related activities are undertaken. Assent should be obtained from pediatric patients according to local regulations
排除标准
- •1. Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
研究组 & 干预措施
Full Analysis Set (FAS)
The Full Analysis Set (FAS) will include all patients with a confirmed HT-1 diagnosis on Nitisinone treatment in routine clinical care, who provide signed informed consent. Patients must be either on treatment with Nitisinone at study entry or they must have been prescribed Nitisinone at enrollment. No specific exclusion criteria from the analysis set will be applied. The FAS will be used for all analyses.
干预措施: Nitisinone (Drug)
结局指标
主要结局
Occurrence of hepatic, renal or hematological adverse events (AEs) or death
时间窗: Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.
Number and percent of patients with occurrence and number of occurrences per 100 patient years.
次要结局
- Occurrence of death(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Occurrence of hepatic malignancy(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Occurrence of other (non-hepatic) malignancies(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Occurrence of cognitive, developmental function AEs(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Laboratory investigations - Blood Chemistry (2)(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Laboratory investigations - Blood Chemistry (4)(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Occurrence of liver transplantation(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Occurrence of any reportable AEs(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Extent of Exposure(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Laboratory investigations - Blood Chemistry (1)(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Neurocognitive/developmental status as assessed by the investigator(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Occurrence of ophthalmic events(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Extent of exposure(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Laboratory investigations - Blood Chemistry (3)(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Occurrence of neurological events(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Treatment and diet compliance(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Laboratory investigations - Blood Coagulation (1)(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Laboratory investigations - Blood Coagulation (2)(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Laboratory investigations - Blood Coagulation (3)(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Overall clinical condition as assessed by the investigator(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)
- Ophthalmic status as assessed by the investigator(Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.)