Clinical trial NCT01355146: Home Therapy With Replagal in Fabry Disease
Official title: Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Fabry Disease
Keywords: Patients with proven Fabry's Disease
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2011-05-17
Enrollment: 127
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with proven Fabry's Disease foreseen for home treatment with Agalsidase alfa at German Fabry centers

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.


Eligibility criteria: 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

Primary outcomes:
measure: Patient satisfaction estimated on a 10-ary Likert scale
timeFrame: comparison of baseline to 12 months value
Secondary outcomes:
measure: Number (per infusion) and severity of infusion-related side effects
timeFrame: baseline compared to 12 months
Other outcomes:
Locations:
city: Bregenz
country: Austria
facility: Landeskrankenhaus Bregenz
city: Graz
country: Austria
facility: LKH-Universitätsklinikum Graz
city: Salzburg
country: Austria
facility: Paracelsus Medizinische Privatuniversität Salzburg
city: Wien
country: Austria
facility: Universitätsklinik für Innere Medizin III
city: Wien
country: Austria
facility: Universitätsklinik für Kinder- und Jugendheilkunde
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Cuxhaven
country: Germany
facility: Med. Versorgungszentrum Dialyse-Centrum Cuxhaven
city: Essen
country: Germany
facility: Alfried-Krupp-Krankenhaus Rüttenscheid
city: Frankfurt
country: Germany
facility: MVZ Immunologie am Krankenhaus Sachsenhausen
city: Freiburg
country: Germany
facility: Zentrum für Kinder und Jugendmedizin
city: Giessen
country: Germany
facility: Universitätsklinikum Gießen und Marburg
city: Hagen
country: Germany
facility: Facharzt für Allgemeinmedizin
city: Höxter
country: Germany
facility: Facharzt für Innere Medizin/Kardiologie
city: Jena
country: Germany
facility: Hans-Berger-Klinik für Neurologie
city: Köln
country: Germany
facility: Klinik II für Innere Medizin
city: Mainz
country: Germany
facility: Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
city: Marienberg
country: Germany
facility: Private Practice, Dr. Glenn Sommer
city: München
country: Germany
facility: KfH Kuratorium für Dialyse und Nierentransplantation e.V.
city: München
country: Germany
facility: Klinikum rechts der Isar
city: Pölten
country: Germany
facility: Landesklinikum St. Pölten, Neurologie
city: Rendsburg
country: Germany
facility: Nephrologisches Zentrum Rendsburg
city: Ulm
country: Germany
facility: Universitätsklinikum Ulm
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: