Home Therapy With Replagal in Fabry Disease

Shire

Status: Completed

Conditions: Fabry Disease

Study type: Observational

Funder types: Industry

Identifiers

NCT01355146

Shire/CS02

Details and patient eligibility

About

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.

Enrollment: 127 patients

Sex: All

Ages: 4+ years old

Volunteers: No Healthy Volunteers

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

Trial design

127 participants in 1 patient group

Fabry's Disease under Replagal