Fabry Disease Registry Protocol

Fabry Registry: To tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status Fabry Pregnancy Sub-registry: To track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated To determine the incidence and type of severe clinical events following initiation of enzyme replacement therapy (ERT) with agalsidase beta, as well as risk factors associated with occurrence of these events.

CT.gov Identifier
NCT00196742
EudraCT Identifier
N/A
CTIS:
N/A
Sponsor
Sanofi {Sanofi-Aventis}/Sanofi Genzyme {Genzyme}
Collaborator
N/A
Study Contact Information
+ 33 1 53 77 40 00
Recruiting
Contact Us

Study Details

Diseases
Fabry Disease
Study Drug
Agalsidase beta, Genzyme
See More
Undisclosed - investigational drug
Genes
GLA
Study Dates
Jul 2001 - Jan 2034
Sex
Female & Male
Age
1 Days - 99 Years
Inclusion and Exclusion Criteria
Inclusion Criteria:
  • Fabry Registry: All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte aGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for aGAL.
  • Fabry Pregnancy Sub-registry:
  • Eligible women must:
  • be enrolled in the Fabry Registry.
  • be pregnant, or have been pregnant with appropriate medical documentation available.
  • provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.
Exclusion Criteria:
  • There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.

Protocol Summary

Fabry Registry: To tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status

Fabry Pregnancy Sub-registry: To track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated

To determine the incidence and type of severe clinical events following initiation of enzyme replacement therapy (ERT) with agalsidase beta, as well as risk factors associated with occurrence of these events.

Study Locations

To view all trial locations, click here

Contact Us About More Information for the Clinical Trial Concierge Service

Natera’s Clinical Trial Concierge Program is comprised of a dedicated team that helps connect providers and patients with appropriate clinical trials. To learn more about a trial offered through this service, please complete the form below.  

Fields marked with asterisk (*) are mandatory.

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.