Home Therapy With Replagal in Fabry Disease

March 15, 2021 updated by: Shire

Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

127

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Bregenz, Austria, A-6900
        • Landeskrankenhaus Bregenz
      • Graz, Austria, A-8036
        • LKH-Universitätsklinikum Graz
      • Salzburg, Austria, A-5020
        • Paracelsus Medizinische Privatuniversität Salzburg
      • Wien, Austria, A-1090
        • Universitätsklinik für Innere Medizin III
      • Wien, Austria, A-1090
        • Universitätsklinik für Kinder- und Jugendheilkunde
  • Germany
      • Berlin, Germany, D-10117
        • Charité - Universitätsmedizin Berlin
      • Cuxhaven, Germany, D-27476
        • Med. Versorgungszentrum Dialyse-Centrum Cuxhaven
      • Essen, Germany, D-45131
        • Alfried-Krupp-Krankenhaus Rüttenscheid
      • Frankfurt, Germany, 60594
        • MVZ Immunologie am Krankenhaus Sachsenhausen
      • Freiburg, Germany, 79106
        • Zentrum für Kinder und Jugendmedizin
      • Giessen, Germany, 35392
        • Universitätsklinikum Giessen und Marburg
      • Hagen, Germany, D-58089
        • Facharzt für Allgemeinmedizin
      • Höxter, Germany, 37671
        • Facharzt fur Innere Medizin/Kardiologie
      • Jena, Germany, 07747
        • Hans-Berger-Klinik für Neurologie
      • Köln, Germany, 50937
        • Klinik II für Innere Medizin
      • Mainz, Germany, 55131
        • Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
      • Marienberg, Germany, D-09496
        • Private Practice, Dr. Glenn Sommer
      • München, Germany, 80804
        • KfH Kuratorium für Dialyse und Nierentransplantation e.V.
      • München, Germany, D-81675
        • Klinikum Rechts der Isar
      • Pölten, Germany, A-3100
        • Landesklinikum St. Pölten, Neurologie
      • Rendsburg, Germany, 24768
        • Nephrologisches Zentrum Rendsburg
      • Ulm, Germany, D-89081
        • Universitätsklinikum Ulm

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Fabry's Disease under Replagal

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Patient satisfaction estimated on a 10-ary Likert scale
Time Frame: comparison of baseline to 12 months value
comparison of baseline to 12 months value

Secondary Outcome Measures

Outcome Measure
Time Frame
Number (per infusion) and severity of infusion-related side effects
Time Frame: baseline compared to 12 months
baseline compared to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 15, 2011

Primary Completion (Actual)

September 30, 2017

Study Completion (Actual)

September 30, 2017

Study Registration Dates

First Submitted

May 16, 2011

First Submitted That Met QC Criteria

May 16, 2011

First Posted (Estimate)

May 17, 2011

Study Record Updates

Last Update Posted (Actual)

March 17, 2021

Last Update Submitted That Met QC Criteria

March 15, 2021

Last Verified

March 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Shire/CS02

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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