Home Therapy With Replagal in Fabry Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Shire
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01355146
First received: May 16, 2011
Last updated: July 21, 2014
Last verified: July 2014
  Purpose

The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Morbus Fabry disease receiving their Enzyme Replacement Therapy with Replagal (Agalsidase alfa) at home compared to receiving the infusions at the clinic or at doctor's practice.


Condition
Fabry's Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Patient satisfaction estimated on a 10-ary Likert scale [ Time Frame: comparison of baseline to 12 months value ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number (per infusion) and severity of infusion-related side effects [ Time Frame: baseline compared to 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 50
Study Start Date: May 2011
Estimated Study Completion Date: May 2020
Estimated Primary Completion Date: May 2019 (Final data collection date for primary outcome measure)
Groups/Cohorts
Fabry's Disease under Replagal

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Patients with proven Fabry's Disease foreseen for home treatment with Agalsidase alfa at German Fabry centers

Criteria

INCLUSION CRITERIA:

  • Male or female patient with a confirmed diagnosis of Fabry disease
  • Age> 4 years
  • Patient is under Replagal since at least 12 weeks ® therapy
  • The patient is compliant, the previous Replagal ® infusions were performed approximately every 2 weeks in the center close to home or at the hospital / general practitioner at
  • Patient has been well tolerated Replagal ® therapy, and there were in the last 12 weeks before inclusion in this study no significant infusion reactions to
  • The patient has been selected before inclusion in this study for a home infusion therapy and has consented to (or their legal representative)
  • The patient / be lawful. Representative has consented in writing to participate in this study.

Exclusion Criteria:

  • Patient/legal representative does not give consent to participation in this study
  • Patient/legal representative declines Replagal® home therapy
  • The patient is participating in a clinical trial with a medicinal product
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01355146

Locations
Austria
Barmherzige Brüder Krankenhaus Graz Not yet recruiting
Graz, Austria, A-8020
Contact: Martin Schumacher, MD         
Principal Investigator: Martin Schumacher, MD         
Paracelsus Medical University Salzburg Recruiting
Salzburg, Austria, A-5020
Contact: Florian B. Lagler, MD    +43-6624420021224    florian.lagler@pmu.ac.at   
Principal Investigator: Florian B. Lagler, MD         
General Hospital, General Hospital of Vienna Recruiting
Wien, Austria, A-1090
Contact: Vassiliki Konstantopoulou, MD    +43-1-40400-3232    vassiliki.konstantopoulou@meduniwien.ac.at   
Principal Investigator: Vassiliki Konstantopoulou, MD         
General Hospital, General Hospital of Vienna Recruiting
Wien, Austria, A-1090
Contact: Prof. Gere Sunder-Plassmann, MD    +43-1-404004217    Gere.Sunder-Plassmann@meduniwien.ac.at   
Principal Investigator: Prof. Gere Sunder-Plassmann, MD         
Germany
Group Practice, Drs. Dieter Schweingel and Anne Stingl Terminated
Bayreuth, Germany, D-95444
Charité - Universitätsmedizin Berlin - Medizinische Klinik mit Schwerpunkt Nephrologie CCM Not yet recruiting
Berlin, Germany, 10117
Contact: Jens Gaedeke, MD    +49-30-450-614137    jens.gaedeke@charite.de   
Principal Investigator: Jens Gaedeke, MD         
GGmbH Catholic Hospital Bochum, Hospital of the Ruhr-University Bochum Not yet recruiting
Bochum, Germany, D-44791
Contact: Prof. Thomas Lücke, MD    +49-2345090    info@klinikum-bochum.de   
Principal Investigator: Prof. Thomas Lücke, MD         
Manfred Pannhausen practice, Specialist in general medicine Recruiting
Bodenwerder, Germany, D-37619
Contact: Manfred Pannhausen, MD    +49-5533/4919      
Principal Investigator: Manfred Pannhausen, MD         
University of Bonn Center for Pediatrics, Pediatric Nephrology, Immunology and Hypertension Recruiting
Bonn, Germany, D-53113
Contact: Prof. Bernd Hoppe, MD    +49-22828733333      
Principal Investigator: Prof. Bernd Hoppe, MD         
Med Care Center dialysis center Cuxhaven Recruiting
Cuxhaven, Germany, D-27476
Contact: Annkatrin Korschanowski, MD    +49-472142080    kontakt@cuxhaven-dialyse.de   
Principal Investigator: Annkatrin Korschanowski, MD         
KfH Kidney Center Erfurt Recruiting
Erfurt, Germany, D-99089
Contact: Christoph C. Haufe, MD    +49-3617815250      
Principal Investigator: Christoph C. Haufe, MD         
Alfried Krupp Hospital Ruettenscheid Recruiting
Essen, Germany, D-45131
Contact: Markus Krämer, MD    +49-20143441418    markus.kraemer@krupp-krankenhaus.de   
Principal Investigator: Markus Krämer, MD         
Ilona-Maria Kapfberger, Specialist in Internal Medicine Recruiting
Fischbachau, Germany, D-83730
Contact: Ilona-Maria Kapfberger, MD    +49-8028809      
Principal Investigator: Ilona-Maria Kapfberger, MD         
University of Münster Recruiting
Frankfurt, Germany, 60323
Contact: Prof. Roland Schäfer    +49-1732844387      
Principal Investigator: Prof. Roland Schäfer         
Zentrum für Kinder und Jugendmedizin Universitätsklinikum Freiburg Not yet recruiting
Freiburg, Germany, 79106
Contact: Ekkehart Lausch, MD    +49 761 270-43630    ekkehart.lausch@uniklinik-freiburg.de   
Principal Investigator: Ekkehart Lausch, MD         
Drs Hans-Jürgen Guth, Ralf Dessel Berger and Nils Bordihn Group Practice Recruiting
Greifswald, Germany, 17489
Contact: Nils Bordihn, MD    +49-3834585180      
Principal Investigator: Nils Bordihn, MD         
Dr. Thomas Stücher and Frauke Stücher, Specialist in general medicine Recruiting
Hagen, Germany, D-58089
Contact: Thomas Stücher, MD    +49-2331305051      
Principal Investigator: Thomas Stücher, MD         
Group Practice, Drs. Christina Beyer and Wilfried Aust Not yet recruiting
Hamburg, Germany, 22767
Contact: Wilfried Aust    +49-40385966      
Principal Investigator: Wilfried Aust         
Center Pediatrics and Adolescent Medicine, Hannover Medical School Recruiting
Hannover, Germany, D-30625
Contact: Prof. A.M. Das    +49-51153232203273    das.anibh@mh-hannover.de   
Principal Investigator: Prof. A.M. Das         
Group Practice, Drs. Manuela Hiller and Ulrich Hiller Recruiting
Hildburghausen, Germany, D-98646
Contact: Manuela Hiller, MD    +49-36854049224      
Principal Investigator: Manuela Hiller, MD         
Facharzt für Innere Medizin/Kardiologie Not yet recruiting
Höxter, Germany, 37671
Contact: Martin Buerschaper, MD    +49 5271 - 96 66 50      
Principal Investigator: Martin Buerschaper, MD         
Hans-Berger-Klinik für Neurologie Universitätsklinikum Jena Not yet recruiting
Jena, Germany, 07747
Contact: Albrecht Günther, MD    +49 3641 - 9 323 417    albrecht.guenther@med.uni-jena.de   
Principal Investigator: Albrecht Günther, MD         
General Medicine Kassel Recruiting
Kassel, Germany, D-34132
Contact: Josef Hesse, MD    05 61 / 40 55 45      
Principal Investigator: Josef Hesse, MD         
Medizinisches Versorgungs Zentrum Rotes Kreuz Krankenhaus Kassel GmbH Not yet recruiting
Kassel, Germany, 34121
Contact: Andreas Böger, MD    +49 561 - 30 86 57 90    schmerzzentrum@rkh-kassel.de   
Principal Investigator: Andreas Böger, MD         
University Hospital Schleswig-Holstein, Campus Kiel Not yet recruiting
Kiel, Germany, 24105
Contact: Annette Girardet    +494315971342    girardet@nephro.uni-kiel.de   
Principal Investigator: Annette Girardet         
Klinik II für Innere Medizin: Nephrologie, Rheumatologie, Diabetologie Not yet recruiting
Köln, Germany, 50937
Contact: Christine Kurschat, MD    +49-221-478-89032    christine.kurschat@uk-koeln.de   
Principal Investigator: Christine Kurschat, MD         
Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin Recruiting
Mainz, Germany, 55131
Contact: Prof. Julia Hennermann, MD    +49-6131-175754    julia.hennermann@unimedizin-mainz.de   
Principal Investigator: Prof. Julia Hennermann, MD         
Private Practice, Dr. Glenn Sommer Recruiting
Marienberg, Germany, D-09496
Contact: Glenn Sommer, Dr., GP    +49-373523141      
Principal Investigator: Glenn Sommer, Dr., GP         
General Practitioner, Upper Market 2 Recruiting
Mittenwald, Germany, D-82481
Contact: Stefan Streng, MD    +49-88233620      
Principal Investigator: Stefan Streng, MD         
Klinikum rechts der Isar, Department of Nephrology of the Second Department of Internal Medicine Recruiting
München, Germany, D-81675
Contact: Claudia Regenbogen, München    +49-08941405644    ClaudiRegenbogen@mri.tum.de   
Principal Investigator: Claudia Regenbogen, MD         
KfH Kidney Center Recruiting
München, Germany, 80804
Contact: Reinhard Kuhnt, MD    +49-893079900    Reinhard.Kuhnt@kfh-dialyse.de   
Principal Investigator: Reinhard Kuhnt, MD         
Thomas Schwerdtfeger and Silke Schwerdtfeger Recruiting
Naumburg, Germany, 06618
Contact: Thomas Schwerdtfeger    +49-3445266863      
Principal Investigator: Thomas Schwerdtfeger         
Practice Ramak Haghir specialist general practitioner Recruiting
Nordheim, Germany, 74226
Contact: Ramek Hagir, MD    +49-71335949      
Principal Investigator: Ramek Hagir, MD         
KfH Kidney Center Recruiting
Ochsenfurt, Germany, D-97199
Contact: Frank Breunig, MD    +49-9331 981980      
Principal Investigator: Frank Breunig, MD         
Regional Hospital St. Pölten, Neurology Recruiting
Polten, Germany, A-3100
Contact: Andreas Zidar, MD    +43-274230072730    andreas.zidar@yahoo.de   
Principal Investigator: Andreas Zidar, MD         
Practice for Haematology Winnfried Freund Recruiting
Rendsburg, Germany, 24768
Contact: Winnfried Freund, MD    +49-4331434800      
Principal Investigator: Winnfried Freund, MD         
Universität Rostock Albrecht-Kossel-Institut für Neuroregeneration (AKos) Not yet recruiting
Rostock, Germany, D-18147
Contact: Arndt Rolfs, MD    +49 381 4949540    arndt.rolfs@med.uni-rostock.de   
Principal Investigator: Arndt Rolfs, MD         
Helios Klinikum Schwerin, Department of Neurology Not yet recruiting
Schwerin, Germany, D-19055
Contact: Prof. Frank Block, MD    +49 (0) 385 5205031    frank.block@helios-kliniken.de   
Principal Investigator: Prof. Frank Block, MD         
University of Ulm, Department of Neurology in RKU Recruiting
Ulm, Germany, D-89081
Contact: Katharina Knauer, MD    +49-73150063006    katharina.knauer@uni-ulm.de   
Principal Investigator: Katharina Knauer, MD         
Universitätsklinikum Würzburg Zentrum Innere Medizin ZIM, Medizinische Klinik und Poliklinik I Not yet recruiting
Würzburg, Germany, D-97080
Contact: Christoph Wanner, MD    +49 931/201-39030    wanner_c@medizin.uni-wuerzburg.de   
Contact: Frank Weidemann    +49 931 20139012    Weidemann_F@ukw.de   
Principal Investigator: Christoph Wanner, MD         
Sponsors and Collaborators
Shire
Investigators
Principal Investigator: Nesrin Karabul, Dr. Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01355146     History of Changes
Other Study ID Numbers: Shire/CS02
Study First Received: May 16, 2011
Last Updated: July 21, 2014
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
Germany: Ethics Commission

Keywords provided by Shire:
Patients with proven Fabry's Disease

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

ClinicalTrials.gov processed this record on October 01, 2014