Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation in Young Patients With Fabry Disease: An Epidemiological, International, Multicenter Prognosis Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 100
- 试验地点
- 20
- 主要终点
- Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches
研究概览
简要总结
New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease. In this case certain fat molecules are not digested and broken down by the body - but remain in the cells. These fat molecules build up to dangerous levels, which start to damage the body, because they accumulate e.g. in the walls of the blood vessels. This accumulation in the blood vessels of the whole body may cause life-threatening malfunctions in the brain, inducing a stroke.
The purpose of this study is to investigate the stroke rehabilitation of Fabry patients during different therapeutic standard approaches for stroke and for Fabry disease (if any). During this study, stroke patients with Fabry disease will be monitored in greater detail to determine whether the differences in treatment are significant for patient recovery and on what they depend.
详细描述
In a group of young stroke patients with diagnosed Fabry disease the stroke rehabilitation will be investigated during different prophylactic therapeutic approaches. In this study the investigator will not be given any instructions on stroke and Fabry therapy.
All patients with any etiology of stroke and a diagnosed Fabry disease submitted to the stroke unit of the participating centres which commit to work with the EUSI (European Stroke Initiative) recommendations for stroke management and diagnosis will be included into the study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult patients (18 - 55 years of age) with an acute cerebrovascular event (CVE) of any etiology defined as patients having an ischemic stroke or transient ischemic attack
- •Genetic diagnosis (a-galactosidase defect)of Fabry disease
- •Written informed consent from patient
排除标准
- •No proven Fabry disease
- •Participating in an other clinical trial with any investigational new drug or medical device
- •Contraindication to any of the diagnostic procedures like e.g. MRI investigation
- •Patient has been pretreated with Enzyme Replacement Therapy at the date of informed consent of sifap2
结局指标
主要结局
Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches
时间窗: 54 months study duration
次要结局
- Habi test (only in Austrian and German centers)(54 months study duration)
- Trail Making Test (TMT)(54 months study duration)
- Brief Pain Inventory (BPI)(54 months study duration)
- Functional neurological deficits measured by the Mini Mental State Examination (MMSE)(54 months study duration)
- Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis(54 months study duration)
- Quality of Life measured with the SF-36(54 months study duration)
- Beck Depression Inventory II (BDI II)(54 months study duration)
- Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany)(54 months study period)