Home Therapy With Replagal in Fabry Disease

Completed

• Conditions: Fabry Disease

• Registration Number: NCT01355146
• Lead Sponsor: Shire

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-03-15
• Study First Submitted: 2011-05-16
• Study First Submitted QC: 2011-05-16
• Study First Posted: 2011-05-17
• Primary Completion: 2017-09-30
• Study Completion: 2017-09-30
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-15
• Last Update Posted: 2021-03-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 127

Inclusion Criteria

Not provided

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Patient satisfaction estimated on a 10-ary Likert scale	comparison of baseline to 12 months value

Secondary Outcome Measures

Name	Time	Method
Number (per infusion) and severity of infusion-related side effects	baseline compared to 12 months

Trial Locations

Locations (22)

Landeskrankenhaus Bregenz; 🇦🇹 Bregenz, Austria

LKH-Universitätsklinikum Graz; 🇦🇹 Graz, Austria

Paracelsus Medizinische Privatuniversität Salzburg; 🇦🇹 Salzburg, Austria

Universitätsklinik für Innere Medizin III; 🇦🇹 Wien, Austria

Universitätsklinik für Kinder- und Jugendheilkunde; 🇦🇹 Wien, Austria

Charité - Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany

Med. Versorgungszentrum Dialyse-Centrum Cuxhaven; 🇩🇪 Cuxhaven, Germany

Alfried-Krupp-Krankenhaus Rüttenscheid; 🇩🇪 Essen, Germany

MVZ Immunologie am Krankenhaus Sachsenhausen; 🇩🇪 Frankfurt, Germany

Zentrum für Kinder und Jugendmedizin; 🇩🇪 Freiburg, Germany

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