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Fabry Disease Registry

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ClinicalTrials.gov Identifier: NCT00196742
Recruitment Status : Recruiting
First Posted : September 20, 2005
Last Update Posted : March 12, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief Summary:

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®

Condition or disease
Fabry Disease

Detailed Description:

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
Study Design
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Study Type : Observational
Estimated Enrollment : 9000 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Fabry Disease Registry
Actual Study Start Date : July 31, 2001
Estimated Primary Completion Date : January 31, 2034
Estimated Study Completion Date : January 31, 2034

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. To evaluate the long-term safety and effectiveness of Fabrazyme® [ Time Frame: 15 years ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All patients with a confirmed diagnosis of Fabry disease are eligible for inclusion in the Registry.
Criteria

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 (alpha-galactosidase) 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).

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00196742


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com
Contact: Fabry Registry HelpLine 617-591-5500 help@fabryregistry.com

Locations
Show Show 219 study locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Medical Monitor Genzyme, a Sanofi Company
More Information
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Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00196742    
Other Study ID Numbers: FABRY REGISTRY
DIREGC07006 ( Other Identifier: Sanofi )
First Posted: September 20, 2005    Key Record Dates
Last Update Posted: March 12, 2020
Last Verified: March 2020
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
alpha Galactosidase A
aGAL (alpha-galactosidase)
Fabry
GL3 (globotriaosylceramide)
 Anderson-Fabry Disease
angiokeratomas
GLA deficiency (gene deficiency)
errors in metabolism
Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
 Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders