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Home Therapy With Replagal in Fabry Disease

Sponsor
Shire (Industry)
Overall Status
Completed
CT.gov ID
NCT01355146
Collaborator
(none)
127
Enrollment
22
Locations
78.6
Actual Duration (Months)
5.8
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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 Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Actual Enrollment :
    127 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease
    Actual Study Start Date :
    Mar 15, 2011
    Actual Primary Completion Date :
    Sep 30, 2017
    Actual Study Completion Date :
    Sep 30, 2017

    Arms and Interventions

    Arm Intervention/Treatment
    Fabry's Disease under Replagal

    Outcome Measures

    Primary Outcome Measures

    1. Patient satisfaction estimated on a 10-ary Likert scale [comparison of baseline to 12 months value]

    Secondary Outcome Measures

    1. Number (per infusion) and severity of infusion-related side effects [baseline compared to 12 months]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    4 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Locations

    Site City State Country Postal Code
    1 Landeskrankenhaus Bregenz Bregenz Austria A-6900
    2 LKH-Universitätsklinikum Graz Graz Austria A-8036
    3 Paracelsus Medizinische Privatuniversität Salzburg Salzburg Austria A-5020
    4 Universitätsklinik für Innere Medizin III Wien Austria A-1090
    5 Universitätsklinik für Kinder- und Jugendheilkunde Wien Austria A-1090
    6 Charité - Universitätsmedizin Berlin Berlin Germany D-10117
    7 Med. Versorgungszentrum Dialyse-Centrum Cuxhaven Cuxhaven Germany D-27476
    8 Alfried-Krupp-Krankenhaus Rüttenscheid Essen Germany D-45131
    9 MVZ Immunologie am Krankenhaus Sachsenhausen Frankfurt Germany 60594
    10 Zentrum für Kinder und Jugendmedizin Freiburg Germany 79106
    11 Universitätsklinikum Gießen und Marburg Giessen Germany 35392
    12 Facharzt für Allgemeinmedizin Hagen Germany D-58089
    13 Facharzt für Innere Medizin/Kardiologie Höxter Germany 37671
    14 Hans-Berger-Klinik für Neurologie Jena Germany 07747
    15 Klinik II für Innere Medizin Köln Germany 50937
    16 Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin Mainz Germany 55131
    17 Private Practice, Dr. Glenn Sommer Marienberg Germany D-09496
    18 KfH Kuratorium für Dialyse und Nierentransplantation e.V. München Germany 80804
    19 Klinikum rechts der Isar München Germany D-81675
    20 Landesklinikum St. Pölten, Neurologie Pölten Germany A-3100
    21 Nephrologisches Zentrum Rendsburg Rendsburg Germany 24768
    22 Universitätsklinikum Ulm Ulm Germany D-89081

    Sponsors and Collaborators

    • Shire

    Investigators

    • Study Director: Study Director, Takeda

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    Shire
    ClinicalTrials.gov Identifier:
    NCT01355146
    Other Study ID Numbers:
    • Shire/CS02
    First Posted:
    May 17, 2011
    Last Update Posted:
    Mar 17, 2021
    Last Verified:
    Mar 1, 2021
    Keywords provided by Shire
    Patients with proven Fabry's Disease
    Additional relevant MeSH terms:
    Fabry Disease

    Study Results

    No Results Posted as of Mar 17, 2021