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NCT00074984 · ClinicalTrials.gov registry record · Phase 4
A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
A Phase 4 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 4
- Development phase
- 82
- Enrollment target
NCT00074984: Completed Phase 4 study, sponsored by Genzyme, a Sanofi Company.
NCT00074984 is a Phase 4 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 82 participants, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (84% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00074984, a Phase 4 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 82 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. Fabrazyme (agalsidase beta) is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In 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 ("globotriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
Primary Outcome
The primary efficacy endpoint was the time to the first occurrence of a clinically significant renal (33% increase in serum creatinine, dialysis or transplant), cardiac (myocardial infarction, significant change in cardiac status, i.e., angina, congestive heart failure or symptomatic arrhythmia requiring medication or surgery) or cerebrovascular (stroke or transient ischemic attack) event and/or death (due to any cause) in Fabrazyme (agalsidase beta) patients as compared to placebo patients.
Interventions
- BIOLOGICAL Placebo
- BIOLOGICAL Fabrazyme (agalsidase beta)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 82 participants |
| Start Date | 2001-02 |
| Est. Completion | 2004-01 |
| Phase | Phase 4 |
What the finished NCT00074984 record still lists
NCT00074984 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 82 participants, a relatively small participant target, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (84% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT00074984 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00074984 about?
NCT00074984 is a clinical study titled "A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease". People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally pres...
What is the current status of trial NCT00074984?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 82 participants. The study started on 2001-02. Estimated completion is 2004-01.
What interventions are being tested in trial NCT00074984?
The interventions under investigation include: Placebo (BIOLOGICAL), Fabrazyme (agalsidase beta) (BIOLOGICAL).
Who is sponsoring clinical trial NCT00074984?
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 NCT00074984's enrollment target sits among peer trials
82 939th of 2000 higher than 1,061 of 2,000 other Phase 4 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 4 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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