Home Therapy With Replagal in Fabry Disease

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.

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Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

4 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Landeskrankenhaus Bregenz, Bregenz, Austria

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Time frame: baseline compared to 12 months

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Official study title

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

Important dates

Study start: 2011
Primary completion: 2017
Study completion: 2017
First posted: May 17, 2011
Registry last updated: Mar 17, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed