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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: Completed Phase 2 study, sponsored by Genzyme, a Sanofi Company.
NCT00074958 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 16 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (88% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
Interventions
- BIOLOGICAL Fabrazyme (agalsidase beta)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2002-10 |
| Est. Completion | 2005-07 |
| Phase | Phase 2 |
What the finished NCT00074958 record still lists
NCT00074958 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (88% lower).
The record links to 0 conditions, and to 1 intervention - of which Fabrazyme (agalsidase beta) is the first listed.
NCT00074958 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00074958's enrollment target sits among peer trials
16 1908th of 2000 higher than 82 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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