Fabry Disease Registry
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Purpose
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 |
|---|
| Fabry Disease |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Other |
| Official Title: | Fabry Disease Registry |
- To evaluate the long-term safety and effectiveness of Fabrazyme® [ Time Frame: 15 years ]
| Estimated Enrollment: | 9000 |
| Actual Study Start Date: | July 31, 2001 |
| Estimated Study Completion Date: | September 2022 |
| Estimated Primary Completion Date: | September 2022 (Final data collection date for primary outcome measure) |
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
Eligibility
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Senior |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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
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
| 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 |
Show 64 Study Locations
| Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
More Information
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Genzyme, a Sanofi Company |
| ClinicalTrials.gov Identifier: | NCT00196742 History of Changes |
| Other Study ID Numbers: |
FABRY REGISTRY DIREGC07006 ( Other Identifier: Sanofi ) |
| First Submitted: | September 13, 2005 |
| First Posted: | September 20, 2005 |
| Last Update Posted: | September 21, 2017 |
| Last Verified: | September 2017 |
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:
|
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 |