Home Therapy With Replagal in Fabry Disease

NCT ID: NCT01355146

Last Updated: 2021-03-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

127 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-03-15

Study Completion Date

2017-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Fabry Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Fabry's Disease under Replagal

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

4 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Landeskrankenhaus Bregenz

Bregenz, , Austria

Site Status

LKH-Universitätsklinikum Graz

Graz, , Austria

Site Status

Paracelsus Medizinische Privatuniversität Salzburg

Salzburg, , Austria

Site Status

Universitätsklinik für Innere Medizin III

Vienna, , Austria

Site Status

Universitätsklinik für Kinder- und Jugendheilkunde

Vienna, , Austria

Site Status

Charité - Universitätsmedizin Berlin

Berlin, , Germany

Site Status

Klinik II für Innere Medizin

Cologne, , Germany

Site Status

Med. Versorgungszentrum Dialyse-Centrum Cuxhaven

Cuxhaven, , Germany

Site Status

Alfried-Krupp-Krankenhaus Rüttenscheid

Essen, , Germany

Site Status

MVZ Immunologie am Krankenhaus Sachsenhausen

Frankfurt, , Germany

Site Status

Zentrum für Kinder und Jugendmedizin

Freiburg im Breisgau, , Germany

Site Status

Universitätsklinikum Gießen und Marburg

Giessen, , Germany

Site Status

Facharzt für Allgemeinmedizin

Hagen, , Germany

Site Status

Facharzt für Innere Medizin/Kardiologie

Höxter, , Germany

Site Status

Hans-Berger-Klinik für Neurologie

Jena, , Germany

Site Status

Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin

Mainz, , Germany

Site Status

Private Practice, Dr. Glenn Sommer

Marienberg, , Germany

Site Status

KfH Kuratorium für Dialyse und Nierentransplantation e.V.

München, , Germany

Site Status

Klinikum rechts der Isar

München, , Germany

Site Status

Landesklinikum St. Pölten, Neurologie

Pölten, , Germany

Site Status

Nephrologisches Zentrum Rendsburg

Rendsburg, , Germany

Site Status

Universitätsklinikum Ulm

Ulm, , Germany

Site Status

Countries

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Austria Germany

Related Links

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https://clinicaltrials.takeda.com/study-detail/5f6b5fd24db2bf003ab46a7c

To obtain more information on the study, click here/on this link.

Other Identifiers

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Shire/CS02

Identifier Type: -

Identifier Source: org_study_id

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