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

Cerebrovascular Accident Clinical Trial

— sifap2  

Official title:

Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation in Young Patients With Fabry Disease: An Epidemiological, International, Multicenter Prognosis Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00413595
• Other study ID #: II PV04/2006
• Status: Completed
• Start date: July 2007
• Est. completion date: December 1, 2019

Study information

• Verified date: April 2020
• Source: CENTOGENE GmbH Rostock
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 100
• Est. completion date: December 1, 2019
• Est. primary completion date: December 1, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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

Related Conditions & MeSH terms

• Cerebrovascular Accident
• Fabry Disease
• Stroke

Intervention

Other:
• No intervention
Observational, epidemiological, prognosis study; no drug tested; only laboratory analysis and diagnostic interventions done.

Locations

Country	Name	City	State
Austria	Universitätsklinikum für Neurologie	Graz
Croatia	Department of Neurology, University Hospital Sestre Milosrdnice	Zagreb
France	Hopital Neurologique de Lyon, Service d'urgences Neurovasculaires	Lyon
Georgia	Department of Neurology, S. Khechinashvili University clinic of Tbilisi state medical university	Tbilisi
Germany	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	Duesseldorf
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	Tuebingen
Germany	University of Ulm, Department of Neurology	Ulm
Poland	Institute of Psychiatry and Neurology, Dept. of Neurology	Warsaw
Portugal	Centro Hospitalar de Lisboa Central, Servico de Neurologia	Lisboa

Sponsors (2)

Lead Sponsor	Collaborator
CENTOGENE GmbH Rostock	Shire Human Genetic Therapies, Inc.

Countries where clinical trial is conducted

Austria,  Croatia,  France,  Georgia,  Germany,  Poland,  Portugal, 

References & Publications (1)

Rolfs A, Böttcher T, Zschiesche M, Morris P, Winchester B, Bauer P, Walter U, Mix E, Löhr 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. Erratum in: Lancet. 2006 Dec 23;368(9554):2210. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches		54 months study duration
Secondary	Quality of Life measured with the SF-36		54 months study duration
Secondary	Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis		54 months study duration
Secondary	Beck Depression Inventory II (BDI II)		54 months study duration
Secondary	Brief Pain Inventory (BPI)		54 months study duration
Secondary	Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany)		54 months study period
Secondary	Habi test (only in Austrian and German centers)		54 months study duration
Secondary	Trail Making Test (TMT)		54 months study duration
Secondary	Functional neurological deficits measured by the Mini Mental State Examination (MMSE)		54 months study duration

External Links

•   Study homepage