NCT01355146CompletedNot Applicable
Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease
Conditions
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Shire
- Enrollment
- 127
- Locations
- 22
- Primary Endpoint
- Patient satisfaction estimated on a 10-ary Likert scale
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 4 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Outcomes
Primary Outcomes
Patient satisfaction estimated on a 10-ary Likert scale
Time Frame: comparison of baseline to 12 months value
Secondary Outcomes
- Number (per infusion) and severity of infusion-related side effects(baseline compared to 12 months)
Investigators
Study Sites (22)
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