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NCT00055016 · ClinicalTrials.gov registry record
Registry of Fabry Disease - A Multicenter Observational Study
A clinical trial, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- 100
- Enrollment target
NCT00055016 is a clinical trial that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 100 participants.
The verdict
NCT00055016 has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- 100 participants
- Enrollment target
Study Summary
The purpose of this study is to compile a registry of patients with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called a-galactosidase A, which normally breaks down a lipid (fatty substance) called globotriaosylceramide (Gb3), is missing or does not function properly. As a result, Gb3 accumulates, causing problems with the kidneys, heart, nerves, and blood vessels. It is not known exactly how lipid accumulation causes these problems, but in another lipid storage disease called Gaucher disease the illness can be reversed if the accumulated lipid is removed by repeated intravenous (into a vein) infusions of the deficient enzyme. The Fabry disease registry is a voluntary and anonymous list of patients that includes information about their health and allows doctors to follow changes in their symptoms and test results over time. It also allows doctors to compare symptoms between patients who are receiving certain therapies with those who are not receiving therapy. The goals of the registry are to: * Better understand the natural history of Fabry disease, including disease variations within and between affected families; * Provide a basis for developing guidelines for disease management; * Evaluate how treatment affects the course of disease; * Provide high-quality data and analyses that will help to continuously develop better treatments. Patients of all ages with biochemical or genetic evidence of Fabry disease (i.e., individuals who have a deficiency of the enzyme a-galactosidase A or a mutation in the gene that encodes this enzyme, or both) are eligible for this study. This worldwide study will include 100 patients participating in Fabry disease studies at the NIH. These patients will come to the NIH Clinical Center only as required for participation their Fabry disease study. No additional procedures will be required for the current registry study. NIH patients will take part in the registry study for their lifetime, or as long as t
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 100 participants |
| Start Date | 2003-02-13 |
| Est. Completion | 2008-03-03 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00055016
The ClinicalTrials.gov registry entry for NCT00055016 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 100 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 0 interventions.
NCT00055016 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00055016 about?
NCT00055016 is a clinical study titled "Registry of Fabry Disease - A Multicenter Observational Study". The purpose of this study is to compile a registry of patients with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called a-galactosidase A, which normally breaks down a lipid (fatty substance) called globotriaosylceramide (Gb3), is missing or does not function properly. ...
What is the current status of trial NCT00055016?
This trial is currently completed. The enrollment target is 100 participants. The study started on 2003-02-13. Estimated completion is 2008-03-03.
Who is sponsoring clinical trial NCT00055016?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
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