Home Therapy With Replagal in Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01355146
Recruitment Status : Recruiting
First Posted : May 17, 2011
Last Update Posted : February 3, 2017
Information provided by (Responsible Party):

Brief Summary:
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 or disease
Fabry Disease

Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease
Study Start Date : May 2011
Estimated Primary Completion Date : May 2019
Estimated Study Completion Date : May 2020

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Fabry's Disease under Replagal

Primary Outcome Measures :
  1. Patient satisfaction estimated on a 10-ary Likert scale [ Time Frame: comparison of baseline to 12 months value ]

Secondary Outcome Measures :
  1. Number (per infusion) and severity of infusion-related side effects [ Time Frame: baseline compared to 12 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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


  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01355146

Contact: Shire Contact

Landeskrankenhaus Bregenz Recruiting
Bregenz, Austria, A-6900
Contact: PD Dr. med. Dipl.-Psych. Martina Huemer    +43 (0)5574 401-6507   
Principal Investigator: Martina Huemer, MD         
LKH-Universitätsklinikum Graz Recruiting
Graz, Austria, A-8036
Contact: Dr. med. Michaela Brunner-Krainz    43-316-385-12813      
Principal Investigator: Michaela Brunner-Krainz, MD         
Paracelsus Medizinische Privatuniversität Salzburg Recruiting
Salzburg, Austria, A-5020
Contact: Florian B Lagler, MD    +43-6624420021224   
Principal Investigator: Florian B Lagler, MD         
Universitätsklinik für Innere Medizin III Recruiting
Wien, Austria, A-1090
Contact: Prof. Gere Sunder-Plassmann, MD    +43-1-4040042170   
Principal Investigator: Prof. Gere Sunder-Plassmann, MD         
Universitätsklinik für Kinder- und Jugendheilkunde Recruiting
Wien, Austria, A-1090
Contact: Vassiliki Konstantopoulou, MD    +43-1-40400-32320   
Principal Investigator: Vassiliki Konstantopoulou, MD         
Charité - Universitätsmedizin Berlin Recruiting
Berlin, Germany, D-10117
Contact: Dr. med. Jens Gaedeke    +49-30-450-614137   
Principal Investigator: Jens Gaedeke, MD         
Praxis Manfred Pannhausen Withdrawn
Bodenwerder, Germany, D-37619
Universitätsklinikum Bonn Withdrawn
Bonn, Germany, D-53113
Med. Versorgungszentrum Dialyse-Centrum Cuxhaven Recruiting
Cuxhaven, Germany, D-27476
Contact: Annkatrin Korschanowski, MD    +49-472142080   
Principal Investigator: Annkatrin Korschanowski, MD         
Alfried-Krupp-Krankenhaus Rüttenscheid Recruiting
Essen, Germany, D-45131
Contact: Dr. med. Markus Krämer    +49-20143441418   
Principal Investigator: Markus Krämer, MD         
Ilona-Maria Kapfberger Withdrawn
Fischbachau, Germany, D-83730
MVZ Immunologie am Krankenhaus Sachsenhausen Recruiting
Frankfurt, Germany, 60594
Contact: Prof. Roland Schäfer    +49-069/714478-29      
Principal Investigator: Prof. Roland Schäfer         
Zentrum für Kinder und Jugendmedizin Recruiting
Freiburg, Germany, 79106
Contact: PD Dr. Ekkehart Lausch    +49 761 270-43630   
Principal Investigator: PD Dr. Ekkehart Lausch         
Universitätsklinikum Gießen und Marburg Recruiting
Giessen, Germany, 35392
Contact: Prof. Dr. med. Andreas Hahn    0641-985-43493   
Principal Investigator: Prof. Dr. med. Andreas Hahn         
Drs Hans-Jürgen Guth, Ralf Dessel Berger and Nils Bordihn Group Practice Withdrawn
Greifswald, Germany, D-17489
Facharzt für Allgemeinmedizin Recruiting
Hagen, Germany, D-58089
Contact: Dr. med. Thomas Stücher, MD    +49-2331 305051      
Principal Investigator: Dr. med. Thomas Stücher         
Facharzt für Innere Medizin/Kardiologie Recruiting
Höxter, Germany, 37671
Contact: Dr. med. Martin Buerschaper, MD    +49 5271 - 96 66 50      
Principal Investigator: Dr. med. Martin Buerschaper         
Hans-Berger-Klinik für Neurologie Recruiting
Jena, Germany, 07747
Contact: OA Dr. Albrecht Günther, MD    +49 3641 - 9 323 417   
Principal Investigator: OA Dr. Albrecht Günther         
Medizinisches Versorgungs Zentrum Withdrawn
Kassel, Germany, 34121
General Medicine Kassel Withdrawn
Kassel, Germany, D-34132
Klinik II für Innere Medizin Recruiting
Köln, Germany, 50937
Contact: Prof. Dr. med. Christine Kurschat    +49-221-478-89032   
Principal Investigator: Prof. Dr. med. Christine Kurschat         
Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin Recruiting
Mainz, Germany, 55131
Contact: Prof. Dr. med. Julia Hennermann    +49-6131-175754   
Principal Investigator: Prof. Dr. med. Julia Hennermann         
Private Practice, Dr. Glenn Sommer Recruiting
Marienberg, Germany, D-09496
Contact: Glenn Sommer    +49-373523141      
Principal Investigator: Glenn Sommer, Dr., GP         
Dr. med. Stefan Streng, Arzt für Allgemeinmedizin Withdrawn
Mittenwald, Germany, D-82481
Fachinternistische Gemeinschaftspraxis Markgräflerland Withdrawn
Müllheim, Germany, 79379
KfH Kuratorium für Dialyse und Nierentransplantation e.V. Recruiting
München, Germany, 80804
Contact: Reinhard Kuhnt, MD    +49-893079900   
Principal Investigator: Reinhard Kuhnt, MD         
Klinikum rechts der Isar Recruiting
München, Germany, D-81675
Contact: Dr. med. Claudia Regenbogen    +49-08941405644   
Principal Investigator: Claudia Regenbogen, MD         
Landesklinikum St. Pölten, Neurologie Recruiting
Pölten, Germany, A-3100
Contact: Dr. med. Marco Tolino    +43 2742-9004-72730   
Principal Investigator: Marco Tolino, MD         
Nephrologisches Zentrum Rendsburg Recruiting
Rendsburg, Germany, 24768
Contact: Volkmar Lufft, MD    +49-4331/4595940      
Principal Investigator: Volkmar Lufft, MD         
Universität Rostock Withdrawn
Rostock, Germany, D-18147
Universitätsklinikum Ulm Recruiting
Ulm, Germany, D-89081
Contact: Dr. med. Katharina Althaus (geb. Knauer)    +49-73150063006   
Principal Investigator: Dr. med. Katharina Knauer         
Dialysezentrum Wertheim Withdrawn
Wertheim, Germany, D-97877
Universitätsklinikum Würzburg Withdrawn
Würzburg, Germany, D-97080
Sponsors and Collaborators
Study Director: Shire Study Physician Shire

Responsible Party: Shire Identifier: NCT01355146     History of Changes
Other Study ID Numbers: Shire/CS02
First Posted: May 17, 2011    Key Record Dates
Last Update Posted: February 3, 2017
Last Verified: February 2017

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

Additional relevant MeSH terms:
Fabry Disease
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders