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Home Therapy With Replagal in Fabry Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Shire
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01355146
First received: May 16, 2011
Last updated: February 1, 2017
Last verified: February 2017
  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 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 ]

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

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   (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
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

Contacts
Contact: Shire Contact clinicaltransparency@shire.com

Locations
Austria
Landeskrankenhaus Bregenz Recruiting
Bregenz, Austria, A-6900
Contact: PD Dr. med. Dipl.-Psych. Martina Huemer    +43 (0)5574 401-6507    Martina.Huemer@lkhb.at   
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    florian.lagler@pmu.ac.at   
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    Gere.Sunder-Plassmann@meduniwien.ac.at   
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    vassiliki.konstantopoulou@meduniwien.ac.at   
Principal Investigator: Vassiliki Konstantopoulou, MD         
Germany
Charité - Universitätsmedizin Berlin Recruiting
Berlin, Germany, D-10117
Contact: Dr. med. Jens Gaedeke    +49-30-450-614137    jens.gaedeke@charite.de   
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    kontakt@cuxhaven-dialyse.de   
Principal Investigator: Annkatrin Korschanowski, MD         
Alfried-Krupp-Krankenhaus Rüttenscheid Recruiting
Essen, Germany, D-45131
Contact: Dr. med. Markus Krämer    +49-20143441418    markus.kraemer@krupp-krankenhaus.de   
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    ekkehart.lausch@uniklinik-freiburg.de   
Principal Investigator: PD Dr. Ekkehart Lausch         
Universitätsklinikum Gießen und Marburg Recruiting
Giessen, Germany, 35392
Contact: Prof. Dr. med. Andreas Hahn    0641-985-43493    andreas.hahn@paediat.med.uni-giessen.de   
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    albrecht.guenther@med.uni-jena.de   
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    christine.kurschat@uk-koeln.de   
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    julia.hennermann@unimedizin-mainz.de   
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    Reinhard.Kuhnt@kfh-dialyse.de   
Principal Investigator: Reinhard Kuhnt, MD         
Klinikum rechts der Isar Recruiting
München, Germany, D-81675
Contact: Dr. med. Claudia Regenbogen    +49-08941405644    ClaudiRegenbogen@mri.tum.de   
Principal Investigator: Claudia Regenbogen, MD         
Landesklinikum St. Pölten, Neurologie Recruiting
Pölten, Germany, A-3100
Contact: Dr. med. Marco Tolino    +43 2742-9004-72730    marco.tolino@stpoelten.lknor.at   
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    katharina.knauer@uni-ulm.de   
Principal Investigator: Dr. med. Katharina Knauer         
Dialysezentrum Wertheim Withdrawn
Wertheim, Germany, D-97877
Universitätsklinikum Würzburg Withdrawn
Würzburg, Germany, D-97080
Sponsors and Collaborators
Shire
Investigators
Study Director: Shire Study Physician Shire
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01355146     History of Changes
Other Study ID Numbers: Shire/CS02
Study First Received: May 16, 2011
Last Updated: February 1, 2017

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 April 28, 2017