Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Detailed Description
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Observation Adult patients (18 - 55 years of age) with an acute cerebrovascular event of any etiology and the genetic diagnosis (a-galactosidase defect) of Fabry disease |
Other: No intervention
Observational, epidemiological, prognosis study; no drug tested; only laboratory analysis and diagnostic interventions done.
|
Outcome Measures
Primary Outcome Measures
- Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches [54 months study duration]
Secondary Outcome Measures
- Quality of Life measured with the SF-36 [54 months study duration]
- Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis [54 months study duration]
- Beck Depression Inventory II (BDI II) [54 months study duration]
- Brief Pain Inventory (BPI) [54 months study duration]
- Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany) [54 months study period]
- Habi test (only in Austrian and German centers) [54 months study duration]
- Trail Making Test (TMT) [54 months study duration]
- Functional neurological deficits measured by the Mini Mental State Examination (MMSE) [54 months study duration]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
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
Exclusion Criteria:
-
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Universitätsklinikum für Neurologie | Graz | Austria | A-8036 | |
| 2 | Department of Neurology, University Hospital Sestre Milosrdnice | Zagreb | Croatia | 10000 | |
| 3 | Hopital Neurologique de Lyon, Service d'urgences Neurovasculaires | Lyon | France | F-69003 | |
| 4 | Department of Neurology, S. Khechinashvili University clinic of Tbilisi state medical university | Tbilisi | Georgia | 0179 | |
| 5 | Department of Neurology, Klinikum Hohe Warte | Bayreuth | Germany | 95445 | |
| 6 | Charite Campus Benjamin Franklin, Dept. of Neurology | Berlin | Germany | D-12200 | |
| 7 | Department of Neurology, Allgemeines Krankenhaus Celle | Celle | Germany | 29223 | |
| 8 | Department of Neurology, Klinikum Chemnitz gGmbH | Chemnitz | Germany | 09131 | |
| 9 | Department of Neurology, Universitaetsklinikum Carl Gustav Carus | Dresden | Germany | 01307 | |
| 10 | Heinrich-Heine-University Duesseldorf, Dept. of Neurology | Duesseldorf | Germany | D-40225 | |
| 11 | University of Giessen-Marburg Dept. of Neurology | Giessen | Germany | D-35385 | |
| 12 | Department of Neurology, Universitaetsklinikum Hamburg-Eppendorf | Hamburg | Germany | 20246 | |
| 13 | Department of Neurology, Universitaetsklinikum Jena | Jena | Germany | 07740 | |
| 14 | Department of Neurology, Universitaetsklinikum Leipzig | Leipzig | Germany | 04103 | |
| 15 | Dept. of Neurology, Ökumenisches Hainich Klinikum gGmbH | Mühlhausen | Germany | 99974 | |
| 16 | Ludwig-Maximilians-University of Munich, Klinikum München-Großhadern, Dept. of Neurology | München | Germany | D-81377 | |
| 17 | Department of Neurology, University Tuebingen | Tuebingen | Germany | 72076 | |
| 18 | University of Ulm, Department of Neurology | Ulm | Germany | D-89081 | |
| 19 | Institute of Psychiatry and Neurology, Dept. of Neurology | Warsaw | Poland | 02-957 | |
| 20 | Centro Hospitalar de Lisboa Central, Servico de Neurologia | Lisboa | Portugal | 1150-199 |
Sponsors and Collaborators
- CENTOGENE GmbH Rostock
- Shire Human Genetic Therapies, Inc.
Investigators
- Principal Investigator: Arndt Rolfs, Prof., MD, University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
- II PV04/2006