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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00196742 |
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:
Condition |
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Fabry Disease |
Study Type: | Observational |
Study Design: | Cohort |
Official Title: | Fabry Disease Registry |
Study Start Date: | April 2001 |
Groups/Cohorts |
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1
Male patients
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2
Female patients
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The Fabry Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
All patients with a confirmed diagnosis of Fabry disease are eligible for inclusion in the Registry.
Inclusion Criteria:
Confirmed diagnosis is defined as: a documented plasma or leukocyte α-galactosidase A (α-gal A) deficiency, or a documented α-gal A deficiency and/or mutation in the α-gal A gene in heterozygous females.
Exclusion Criteria:
Contact: Fabry Registry HelpLine | 800-745-4447 ext 15500 | help@fabryregistry.com |
Contact: Fabry Registry HelpLine | 617-591-5500 | help@fabryregistry.com |
United States, Massachusetts | |
Registry participation is worldwide and not limited to this facility; facilities not yet active may enroll upon identification of a patient. | Recruiting |
Cambridge, Massachusetts, United States, 02142 |
Study Director: | Medical Monitor | Genzyme |
Responsible Party: | Genzyme Coporation ( Medical Monitor ) |
Study ID Numbers: | Fabry Registry |
Study First Received: | September 13, 2005 |
Last Updated: | July 28, 2009 |
ClinicalTrials.gov Identifier: | NCT00196742 History of Changes |
Health Authority: | United States: Institutional Review Board |
alpha Galactosidase A aGAL Fabry GL3 |
Anderson-Fabry Disease angiokeratomas GLA deficiency errors in metabolism |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn |
Fabry Disease Genetic Diseases, X-Linked Ceramide Trihexosidosis Brain Diseases, Metabolic, Inborn Lipidoses Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Fabry Disease Genetic Diseases, X-Linked Brain Diseases, Metabolic, Inborn Lipidoses Lipid Metabolism Disorders Brain Diseases, Metabolic |