Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation

Completed

• Conditions: Cerebrovascular Accident; Fabry Disease
• Interventions: Other: No intervention

• Registration Number: NCT00413595
• Lead Sponsor: CENTOGENE GmbH Rostock

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.

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 Timeline

• Study First Submitted: 2006-12-19
• Study First Submitted QC: 2006-12-19
• Study First Posted: 2006-12-20
• Study Start: 2007-07
• Primary Completion: 2019-12-01
• Study Completion: 2019-12-01
• Status Verified: 2020-04
• Last Update Submitted: 2021-04-08
• Last Update Posted: 2021-04-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 100

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Observation	No intervention	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

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Brief Pain Inventory (BPI)	54 months study duration
Beck Depression Inventory II (BDI II)	54 months study duration
Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis	54 months study duration
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
Quality of Life measured with the SF-36	54 months study duration
Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany)	54 months study period

Trial Locations

Locations (20)

Universitätsklinikum für Neurologie; 🇦🇹 Graz, Austria

Department of Neurology, Klinikum Hohe Warte; 🇩🇪 Bayreuth, Germany

Heinrich-Heine-University Duesseldorf, Dept. of Neurology; 🇩🇪 Duesseldorf, Germany

Charite Campus Benjamin Franklin, Dept. of Neurology; 🇩🇪 Berlin, Germany

Department of Neurology, Klinikum Chemnitz gGmbH; 🇩🇪 Chemnitz, Germany

University of Ulm, Department of Neurology; 🇩🇪 Ulm, Germany

Department of Neurology, Universitaetsklinikum Jena; 🇩🇪 Jena, Germany

Department of Neurology, Universitaetsklinikum Leipzig; 🇩🇪 Leipzig, Germany

Institute of Psychiatry and Neurology, Dept. of Neurology; 🇵🇱 Warsaw, Poland

Department of Neurology, S. Khechinashvili University clinic of Tbilisi state medical university; 🇬🇪 Tbilisi, Georgia

Department of Neurology, Allgemeines Krankenhaus Celle; 🇩🇪 Celle, Germany

Dept. of Neurology, Ökumenisches Hainich Klinikum gGmbH; 🇩🇪 Mühlhausen, Germany

Centro Hospitalar de Lisboa Central, Servico de Neurologia; 🇵🇹 Lisboa, Portugal

Hopital Neurologique de Lyon, Service d'urgences Neurovasculaires; 🇫🇷 Lyon, France

Department of Neurology, University Hospital Sestre Milosrdnice; 🇭🇷 Zagreb, Croatia

Department of Neurology, Universitaetsklinikum Carl Gustav Carus; 🇩🇪 Dresden, Germany

University of Giessen-Marburg Dept. of Neurology; 🇩🇪 Giessen, Germany

Ludwig-Maximilians-University of Munich, Klinikum München-Großhadern, Dept. of Neurology; 🇩🇪 München, Germany

Department of Neurology, University Tuebingen; 🇩🇪 Tuebingen, Germany

Department of Neurology, Universitaetsklinikum Hamburg-Eppendorf; 🇩🇪 Hamburg, Germany