Fabry Disease
Conditions
Keywords
Patients with proven Fabry's Disease
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.
Interventions
None listed
Sponsors
Shire
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
4 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Patient satisfaction estimated on a 10-ary Likert scale | comparison of baseline to 12 months value |
Secondary
| Measure | Time frame |
|---|---|
| Number (per infusion) and severity of infusion-related side effects | baseline compared to 12 months |
Countries
Austria, Germany
Outcome results
None listed