Home Therapy With Replagal in Fabry Disease

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01355146
• Other study ID #: Shire/CS02
• Status: Completed
• Start date: March 15, 2011
• Est. completion date: September 30, 2017

Study information

• Verified date: March 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 127
• Est. completion date: September 30, 2017
• Est. primary completion date: September 30, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Fabry Disease

Locations

Country	Name	City	State
Austria	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
Germany	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	Universitätsklinikum Gießen und Marburg	Giessen
Germany	Facharzt für Allgemeinmedizin	Hagen
Germany	Facharzt für Innere Medizin/Kardiologie	Höxter
Germany	Hans-Berger-Klinik für Neurologie	Jena
Germany	Klinik II für Innere Medizin	Köln
Germany	Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin	Mainz
Germany	Private Practice, Dr. Glenn Sommer	Marienberg
Germany	KfH Kuratorium für Dialyse und Nierentransplantation e.V.	München
Germany	Klinikum rechts der Isar	München
Germany	Landesklinikum St. Pölten, Neurologie	Pölten
Germany	Nephrologisches Zentrum Rendsburg	Rendsburg
Germany	Universitätsklinikum Ulm	Ulm

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

Austria,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient satisfaction estimated on a 10-ary Likert scale		comparison of baseline to 12 months value
Secondary	Number (per infusion) and severity of infusion-related side effects		baseline compared to 12 months

External Links

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