Home Therapy With Replagal in Fabry Disease

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2011 by Shire Human Genetic Therapies, Inc..
Recruitment status was  Recruiting
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

May 16, 2011
August 4, 2011
May 2011
Not Provided
Patient satisfaction estimated on a 10-ary Likert scale [ Time Frame: comparison of baseline to 12 months value ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01355146 on ClinicalTrials.gov Archive Site
Number (per infusion) and severity of infusion-related side effects [ Time Frame: baseline compared to 12 months ] [ Designated as safety issue: Yes ]
Same as current
Not Provided
Not Provided
 
Home Therapy With Replagal in Fabry Disease
Non Interventional Study of Replagal® Home Therapy in Patients With 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.

Not Provided
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Probability Sample

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

Fabry's Disease
Not Provided
Fabry's Disease under Replagal
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
50
Not Provided
Not Provided

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
Both
4 Years and older
No
Not Provided
Germany
 
NCT01355146
Shire/CS02
No
Dr. Martina Kralewski, Shire Deutschland GmbH
Shire Human Genetic Therapies, Inc.
Not Provided
Principal Investigator: Michael Beck, Prof. Dr. Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
Shire Human Genetic Therapies, Inc.
August 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP