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Home Therapy With Replagal in Fabry Disease

This study is currently recruiting participants.
Verified August 2011 by Shire Human Genetic Therapies, Inc.
Sponsor:
Information provided by:
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT01355146
First received: May 16, 2011
Last updated: August 4, 2011
Last verified: August 2011
History of Changes
  Purpose

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.


Condition
Fabry's Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Shire Human Genetic Therapies, Inc.:

Primary Outcome Measures:
  • Patient satisfaction estimated on a 10-ary Likert scale [ Time Frame: comparison of baseline to 12 months value ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number (per infusion) and severity of infusion-related side effects [ Time Frame: baseline compared to 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 50
Study Start Date: May 2011
Groups/Cohorts
Fabry's Disease under Replagal

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Patients with proven Fabry's Disease foreseen for home treatment with Agalsidase alfa at German Fabry centers

Criteria

Inclusion Criteria:

  • Male or female patient with proven diagnosis of Fabry disease
  • Age > 4 years
  • Patient is since at least 6 weeks under Replagal®-therapy
  • Patient is compliant
  • Patient has well tolerated Replagal®-therapy
  • Patient has been selected for home therapy and has already accepted home therapy with Replagal® before inclusion in this study
  • Patient/legal representative has given written informed consent to participation 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01355146

Locations
Germany
Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin Recruiting
Mainz, Germany, 55131
Contact: Michael Beck, Prof. Dr.     +49 6131 175 ext 754     beck@kinder.klinik.uni-mainz.de    
Principal Investigator: Michael Beck, Prof. Dr.            
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
Principal Investigator: Michael Beck, Prof. Dr. Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
  More Information

No publications provided

Responsible Party: Dr. Martina Kralewski, Shire Deutschland GmbH
ClinicalTrials.gov Identifier: NCT01355146     History of Changes
Other Study ID Numbers: Shire/CS02
Study First Received: May 16, 2011
Last Updated: August 4, 2011
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
Germany: Ethik-Kommission der Landesärztekammer Rheinland-Pfalz

Keywords provided by Shire Human Genetic Therapies, Inc.:
Patients with proven Fabry's Disease

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on June 17, 2013