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

This study is currently recruiting participants.
Verified March 2013 by University of Rostock
Sponsor:
Collaborator:
Shire Human Genetic Therapies, Inc.
Information provided by (Responsible Party):
Prof. Dr. Arndt Rolfs, University of Rostock
ClinicalTrials.gov Identifier:
NCT00413595
First received: December 19, 2006
Last updated: March 15, 2013
Last verified: March 2013
History of Changes
  Purpose

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 Intervention
Fabry Disease
Cerebrovascular Accident
 Other: No intervention

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
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

Resource links provided by NLM:

MedlinePlus related topics: Rehabilitation
U.S. FDA Resources

Further study details as provided by University of Rostock:

Primary Outcome Measures:
  • Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]
  • Quality of Life measured with the SF-36 [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]
  • Beck Depression Inventory II (BDI II) [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]
  • Brief Pain Inventory (BPI) [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]
  • Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany) [ Time Frame: 54 months study period ] [ Designated as safety issue: No ]
  • Habi test (only in Austrian and German centers) [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]
  • Trail Making Test (TMT) [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]
  • Functional neurological deficits measured by the Mini Mental State Examination (MMSE) [ Time Frame: 54 months study duration ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA

EDTA-blood and urine sample for central laboratory analysis of agalsidase antibodies and Gb3 for safety issues.

There will be a proteomic analysis in blood to check whether there will be the possibility to characterize a biomarker for Fabry disease.


Estimated Enrollment: 100
Study Start Date: April 2007
Estimated Study Completion Date: November 2013
Estimated Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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.

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.

  Eligibility

Ages Eligible for Study:   18 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

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 and genetic diagnosis (a-galactosidase defect) of Fabry disease.

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00413595

Contacts
Contact: Arndt Rolfs, Prof., MD 49-381-494 ext -9540 arndt.rolfs@med.uni-rostock.de
Contact: Sabine Rösner 49-381-494 ext -4797 sabine.roesner@med.uni-rostock.de

Locations
Austria
Universitätsklinikum für Neurologie Recruiting
Graz, Austria, A-8036
Contact: Franz Fazekas, Prof., MD     +43-316-385- ext -2981     franz.fazekas@meduni-graz.at    
Contact: Gabriele Schrotter, MD     +43-316-385- ext -80424     gabriele.schrotter@meduni-graz.at    
Principal Investigator: Franz Fazekas, Prof., MD            
Sub-Investigator: Gabriele Schrotter, MD            
Sub-Investigator: Barbara Ebenbauer, MD            
Croatia
Department of Neurology, University Hospital Sestre Milosrdnice Recruiting
Zagreb, Croatia, 10000
Contact: Vida Demarin, Prof. MD     +385-13-787- ext -740     vida.demarin@zg.t-com.hr    
Contact: Marijana Bosnar-Puretic, MD     +385-91-554- ext -740     mbos-puretic@inet.hr    
Principal Investigator: Vida Demarin, Prof. MD            
Sub-Investigator: Marijana Bosnar-Puretic, MD            
France
Hopital Neurologique de Lyon, Service d'urgences Neurovasculaires Recruiting
Lyon, France, F-69003
Contact: Laurent Derex, MD     +33-472-357- ext -809     laurent.derex@chu-lyon.fr    
Principal Investigator: Laurent Derex, MD            
Georgia
Department of Neurology, S. Khechinashvili University clinic of Tbilisi state medical university Recruiting
Tblisi, Georgia, 0179
Contact: Marina Janelidze, Prof. MD     +995-322-268- ext -04     marjaneli@yahoo.com    
Contact: Maia Beridze, Prof. MD     +995-322-268- ext -04     postdoc2002@yahoo.com    
Principal Investigator: Marina Janelidze, Prof. MD            
Sub-Investigator: Maia Beridze, Prof. MD            
Germany
Department of Neurology, Klinikum Hohe Warte Recruiting
Bayreuth, Germany, 95445
Contact: Patrick Oschmann, Prof., MD     +49-921-400- ext -4602     neurologie@klinikum-bayreuth.de    
Contact: Michael Pott, MD     +49-921-400- ext -4602     neurologie@klinikum-bayreuth.de    
Principal Investigator: Patrick Oschmann, Prof., MD            
Sub-Investigator: Michael Pott, MD            
Charite Campus Benjamin Franklin, Dept. of Neurology Recruiting
Berlin, Germany, D-12200
Contact: Gerhard J. Jungehülsing, MD     +49-30-8445-64- ext -4088     jan.jungehuelsing@charite.de    
Contact: Wolf Schmidt, MD     +49-30-8445- ext -4101     wolf.schmidt@charite.de    
Principal Investigator: Gerhard J. Jungehülsing, MD            
Sub-Investigator: Wolf Schmidt, MD            
Department of Neurology, Allgemeines Krankenhaus Celle Recruiting
Celle, Germany, 29223
Contact: Wolfgang Heide, Prof. MD     +49-5141-72- ext -1400     wolfgang.heide@ak-h-celle.de    
Contact: Oliver Pape, MD     +49-5141-72- ext -6489     oliver.pape@akh-celle.de    
Principal Investigator: Wolfgang Heide, Prof. MD            
Sub-Investigator: Oliver Pape, MD            
Department of Neurology, Klinikum Chemnitz gGmbH Recruiting
Chemnitz, Germany, 09131
Contact: Juergen Klingelhoefer, Prof. MD     +49-371-333- ext -10530     neurologie@skc.de    
Contact: Sebastian Schwarz, MD     49-371-333- ext -12344     neurologie@skc.de    
Principal Investigator: Juergen Klingelhoefer, Prof. MD            
Sub-Investigator: Sebastian Schwarz, MD            
Department of Neurology, Universitaetsklinikum Carl Gustav Carus Recruiting
Dresden, Germany, 01307
Contact: Heinz Reichmann, Prof. MD     +49-351-458- ext -3565     heinz.reichmann@uniklinikum-dresden.de    
Principal Investigator: Heinz Reichmann, Prof. MD            
Sub-Investigator: Hjördis Hentschel, MD            
Heinrich-Heine-University Duesseldorf, Dept. of Neurology Recruiting
Duesseldorf, Germany, D-40225
Contact: Sebastian Jander, Prof., MD     +49-211-811- ext -8978     jander@uni-duesseldorf.de    
Contact: Christina Boettcher     +49-211-811- ext -18465     christina.boettcher@uni-duesseldorf.de    
Principal Investigator: Sebastian Jander, Prof., MD            
Sub-Investigator: Christina Boettcher            
University of Giessen-Marburg Dept. of Neurology Recruiting
Giessen, Germany, D-35385
Contact: Manfred Kaps, Prof., MD     +49-641-994- ext -5300     manfred.kaps@neuro.med.uni-giessen.de    
Contact: Christian Tanislav, MD     +49-641-994- ext -5316     christian.tanislav@uniklinikum-giessen.de    
Principal Investigator: Manfred Kaps, Prof., MD            
Sub-Investigator: Christian Tanislav, MD            
Sub-Investigator: Jens Allendoerfer, MD            
Department of Neurology, Universitaetsklinikum Hamburg-Eppendorf Recruiting
Hamburg, Germany, 20246
Contact: Michael Rosenkranz, MD     +49-40-42803- ext -4730     rosenkranz@uke-uni-hamburg.de    
Contact: Anna C Krützelmann, MD     +49-40-42803- ext -9824     a.kruetzelmann@uke-uni-hamburg.de    
Principal Investigator: Michael Rosenkranz, MD            
Sub-Investigator: Anna C. Krützelmann, MD            
Department of Neurology, Universitaetsklinikum Jena Recruiting
Jena, Germany, 07740
Contact: Otto W. Witte, Prof. MD     +49-3641-932- ext -3401     otto.witte@med.uni-jena.de    
Contact: Dirk Brämer, MD     +49-3641-935- ext -266     dirk.braemer@med.uni-jena.de    
Principal Investigator: Otto Witte, Prof. MD            
Sub-Investigator: Dirk Brämer, MD            
Sub-Investigator: Albrecht Günther, MD            
Department of Neurology, Universitaetsklinikum Leipzig Recruiting
Leipzig, Germany, 04103
Contact: Dietmar Schneider, Prof. MD     +49-341-9724- ext -221     dietmar.schneider@medizin.uni-leipzig.de    
Contact: Dominik Michalski, MD     +49-341-9724- ext -206     dominik.michalski@medizin.uni-leipzig.de    
Principal Investigator: Dietmar Schneider, Prof. MD            
Sub-Investigator: Dominik Michalski, MD            
Sub-Investigator: Wolfgang Kloppig, MD            
Dept. of Neurology, Ökumenisches Hainich Klinikum gGmbH Recruiting
Mühlhausen / Thürigen, Germany, 99974
Contact: Marek Jauß, PD MD     +49-3601-803- ext -456     m.jauss@oehk.de    
Principal Investigator: Marek Jauß, PD MD            
Sub-Investigator: Felicita Sojka, MD            
Sub-Investigator: Lars Adam, MD            
Ludwig-Maximilians-University of Munich, Klinikum München-Großhadern, Dept. of Neurology Recruiting
München, Germany, D-81377
Contact: Martin Dichgans, Prof., MD     +49-89-7095- ext -6670     martin.dichgans@med.uni-muenchen.de    
Contact: Anna Karpinska, MD     +49-89-7095- ext -6672     anna.karpinska@med.uni-muenchen.de    
Principal Investigator: Martin Dichgans, Prof., MD            
Sub-Investigator: Anna Karpinska, MD            
Department of Neurology, University Tuebingen Recruiting
Tuebingen, Germany, 72076
Contact: Arthur Melms, Prof. MD     +49-7071-29- ext -82057     arthur.melms@uni-tuebingen.de    
Contact: Karl Horber     +49-7071-29- ext -82051     karl.horber@uni-tuebingen.de    
Principal Investigator: Arthur Melms, Prof., MD            
Sub-Investigator: Karl Horber            
University of Ulm, Department of Neurology Recruiting
Ulm, Germany, D-89081
Contact: Albert Ludolph, Prof., MD     +49-731-177- ext -1201     albert.ludolph@uni-ulm.de    
Contact: Roman Huber, MD     +49-731-177- ext -5222     r.huber@uni-ulm.de    
Principal Investigator: Albert Ludolph, Prof., MD            
Sub-Investigator: Roman Huber, MD            
Poland
Institute of Psychiatry and Neurology, Dept. of Neurology Recruiting
Warsaw, Poland, 02-957
Contact: Anna Czlonkowska, Prof. MD     +48-22-842- ext -7683     czlonkow@ipin.edu.pl    
Contact: Beata Hyzorek-Bazejewska, MD     +48-22-842- ext -7683     hyzorek@ipin.edu.pl    
Principal Investigator: Anna Czlonkowska, Prof. MD            
Sub-Investigator: Beata Hyzorek-Bazejewska, MD            
Sub-Investigator: Anna Baranowska, MD            
Portugal
Centro Hospitalar de Lisboa Central, Servico de Neurologia Recruiting
Lisboa, Portugal, 1150-199
Contact: Alexandre Amaral e Silva, MD     +351-21-8841- ext -303     zefiros@netcabo.pt    
Contact: Joacquim Machado Candido, MD     +351-21-8841- ext -855     secunid.ucv@chlisboa-zc.min-saude.pt    
Sub-Investigator: Alexandre Amaral e Silva, MD            
Principal Investigator: Joacquim Machado Candido, MD            
Sponsors and Collaborators
University of Rostock
Shire Human Genetic Therapies, Inc.
Investigators
Principal Investigator: Arndt Rolfs, Prof., MD University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration
  More Information

Additional Information:
Study homepage  This link exits the ClinicalTrials.gov site

Publications:

Responsible Party: Prof. Dr. Arndt Rolfs, Prof. Dr. med., University of Rostock
ClinicalTrials.gov Identifier: NCT00413595     History of Changes
Other Study ID Numbers: II PV04/2006
Study First Received: December 19, 2006
Last Updated: March 15, 2013
Health Authority: Germany: Ethics Commission
France: Institutional Ethical Committee

Keywords provided by University of Rostock:
Cerebrovascular Accident
Cerebrovascular Accident, Acute
Fabry Disease
Fabry's Disease
 Anderson-Fabry Disease
CVA (Cerebrovascular Accident)
Stroke, acute
Cerebral Stroke

Additional relevant MeSH terms:
Fabry Disease
Cerebral Infarction
Stroke
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
 Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Brain Infarction
Brain Ischemia
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on May 21, 2013