Home Therapy With Replagal in Fabry Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Shire
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01355146
First received: May 16, 2011
Last updated: July 21, 2014
Last verified: July 2014

May 16, 2011
July 21, 2014
May 2011
May 2019   (final data collection date for primary outcome measure)
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
May 2020
May 2019   (final data collection date for primary outcome measure)

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
Both
4 Years and older
No
Austria,   Germany
 
NCT01355146
Shire/CS02
No
Shire
Shire
Not Provided
Principal Investigator: Nesrin Karabul, Dr. Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
Shire
July 2014

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