Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 90
- 试验地点
- 4
- 主要终点
- Clinical outcome specific to the prospective part of the study
研究概览
简要总结
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
详细描述
A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.
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
- 1 Year 至 90 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged 1 year to 90 years of age.
- •Physician established diagnosis of Wilson disease based on a Ferenci score >
- •Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent.
- •Able/willing to provide written informed consent.
- •For enrolment in the prospective part, enrolment in the retrospective part of the study is required.
排除标准
- •Incomplete history of medication use for trientine from initial diagnosis to latest follow up.
- •Unavailable outcome data for hepatic and neurological course of disease at assessment time points.
- •Patients with acute liver failure and fulminant hepatic disease with fatal outcome.
- •Hypersensitivity to trientine and severe anaemia.
研究组 & 干预措施
Prospective
干预措施: trientine dihydrochloride (Drug)
结局指标
主要结局
Clinical outcome specific to the prospective part of the study
时间窗: 12 months
The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit.
Clinical outcome specific to the retrospective part of the study
时间窗: 48 months
The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened.
次要结局
- Quality of Life Endpoints for the Prospective Part of the Study(12 months)
- Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study(Up to 60 months)