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Home Therapy With Replagal in Fabry Disease

This study is currently recruiting participants.
Verified February 2017 by Shire
Sponsor:
ClinicalTrials.gov Identifier:
NCT01355146
First Posted: May 17, 2011
Last Update Posted: February 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Shire
May 16, 2011
May 17, 2011
February 3, 2017
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 ]
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 ]
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 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
Sexes Eligible for Study: All
4 Years and older   (Child, Adult, Senior)
No
Contact: Shire Contact clinicaltransparency@shire.com
Austria,   Germany
 
 
NCT01355146
Shire/CS02
No
Not Provided
Not Provided
Shire
Shire
Not Provided
Study Director: Shire Study Physician Shire
Shire
February 2017