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NCT00074958 · ClinicalTrials.gov registry record · Phase 2
A Study of Fabrazyme in Pediatric Patients With Fabry Disease
A Phase 2 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 16
- Enrollment target
NCT00074958 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 16 participants.
The verdict
NCT00074958, a Phase 2 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 16 participants
- Enrollment target
Study Summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In people with Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This study explored the safety, efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.
Interventions
- BIOLOGICAL Fabrazyme (agalsidase beta)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2002-10 |
| Est. Completion | 2005-07 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00074958
The ClinicalTrials.gov registry entry for NCT00074958 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 16 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Fabrazyme (agalsidase beta) is the first listed.
NCT00074958 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00074958 about?
NCT00074958 is a clinical study titled "A Study of Fabrazyme in Pediatric Patients With Fabry Disease". People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most ce...
What is the current status of trial NCT00074958?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 16 participants. The study started on 2002-10. Estimated completion is 2005-07.
What interventions are being tested in trial NCT00074958?
The interventions under investigation include: Fabrazyme (agalsidase beta) (BIOLOGICAL).
Who is sponsoring clinical trial NCT00074958?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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