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

NCT ID: NCT00413595

Last Updated: 2021-04-12

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2007-07-31
• Study Completion Date: 2019-12-01

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.

Conditions

Fabry Disease; Cerebrovascular Accident

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

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

No intervention

Intervention Type: OTHER

Observational, epidemiological, prognosis study; no drug tested; only laboratory analysis and diagnostic interventions done.

Interventions

No intervention

Observational, epidemiological, prognosis study; no drug tested; only laboratory analysis and diagnostic interventions done.

Intervention Type: OTHER

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shire Human Genetic Therapies, Inc.

INDUSTRY

Sponsor Role: collaborator

CENTOGENE GmbH Rostock

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Arndt Rolfs, Prof., MD

Role: PRINCIPAL_INVESTIGATOR

University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration

Locations

Universitätsklinikum für Neurologie

Graz, , Austria

Department of Neurology, University Hospital Sestre Milosrdnice

Zagreb, , Croatia

Hopital Neurologique de Lyon, Service d'urgences Neurovasculaires

Lyon, , France

Department of Neurology, S. Khechinashvili University clinic of Tbilisi state medical university

Tbilisi, , Georgia

Department of Neurology, Klinikum Hohe Warte

Bayreuth, , Germany

Charite Campus Benjamin Franklin, Dept. of Neurology

Berlin, , Germany

Department of Neurology, Allgemeines Krankenhaus Celle

Celle, , Germany

Department of Neurology, Klinikum Chemnitz gGmbH

Chemnitz, , Germany

Department of Neurology, Universitaetsklinikum Carl Gustav Carus

Dresden, , Germany

Heinrich-Heine-University Duesseldorf, Dept. of Neurology

Düsseldorf, , Germany

University of Giessen-Marburg Dept. of Neurology

Giessen, , Germany

Department of Neurology, Universitaetsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Department of Neurology, Universitaetsklinikum Jena

Jena, , Germany

Department of Neurology, Universitaetsklinikum Leipzig

Leipzig, , Germany

Dept. of Neurology, Ökumenisches Hainich Klinikum gGmbH

Mühlhausen, , Germany

Ludwig-Maximilians-University of Munich, Klinikum München-Großhadern, Dept. of Neurology

München, , Germany

Department of Neurology, University Tuebingen

Tübingen, , Germany

University of Ulm, Department of Neurology

Ulm, , Germany

Institute of Psychiatry and Neurology, Dept. of Neurology

Warsaw, , Poland

Centro Hospitalar de Lisboa Central, Servico de Neurologia

Lisbon, , Portugal

Countries

Austria; Croatia; France; Georgia; Germany; Poland; Portugal

References

Rolfs A, Bottcher T, Zschiesche M, Morris P, Winchester B, Bauer P, Walter U, Mix E, Lohr M, Harzer K, Strauss U, Pahnke J, Grossmann A, Benecke R. Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study. Lancet. 2005 Nov 19;366(9499):1794-6. doi: 10.1016/S0140-6736(05)67635-0.

Reference Type: BACKGROUND

PMID: 16298216 (View on PubMed)

Other Identifiers

II PV04/2006

Identifier Type: -

Identifier Source: org_study_id