Fabry Disease Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2013 by Genzyme, a Sanofi Company
Sponsor:
Information provided by (Responsible Party):
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00196742
First received: September 13, 2005
Last updated: August 21, 2013
Last verified: August 2013

September 13, 2005
August 21, 2013
April 2001
July 2020   (final data collection date for primary outcome measure)
To evaluate the long-term safety and effectiveness of Fabrazyme® [ Time Frame: 15 years ] [ Designated as safety issue: No ]
Not Provided
Complete list of historical versions of study NCT00196742 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Fabry Disease Registry
Fabry Disease Registry

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;
  • To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
  • To characterize and describe the Fabry population as a whole; and
  • To evaluate the long-term safety and effectiveness of Fabrazyme®

The Fabry Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

  • In Asia-Pacific - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com
  • In Europe - +31-35-699-1232, europe@FabryRegistry.com
  • In Latin America - +617-591-5500, help@FabryRegistry.com
  • In North America - +617-591-5500, help@FabryRegistry.com
Observational
Observational Model: Cohort
Not Provided
Not Provided
Non-Probability Sample

All patients with a confirmed diagnosis of Fabry disease are eligible for inclusion in the Registry.

Fabry Disease
Not Provided
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
9000
July 2020
July 2020   (final data collection date for primary outcome measure)

Inclusion Criteria 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 αGAL enzyme activity and/or mutation(s) in the gene coding for αGAL.

Exclusion Criteria There are no exclusion criteria in this Registry. Patients are allowed to participate in other clinical studies and may be receiving different therapies to treat their disease; however, enrollment in other clinical studies should be noted on the Registry case report forms (CRFs).

Both
Not Provided
No
Contact: Fabry Registry HelpLine 800-745-4447 ext 15500 help@fabryregistry.com
Contact: Fabry Registry HelpLine 617-591-5500 help@fabryregistry.com
United States
 
NCT00196742
FABRY REGISTRY
No
Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
August 2013

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