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NCT00048906 · ClinicalTrials.gov registry record · Phase 2
Alpha-Galactosidase A Replacement Therapy for Fabry Disease
A Phase 2 study of Fabry Disease, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- Phase 2
- Development phase
- 3
- Enrollment target
- 1
- Study location
NCT00048906 is a Phase 2 study of Fabry Disease that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 3 participants, below the 543-participant average among 32 other Fabry Disease trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state.
The verdict
NCT00048906, a Phase 2 study of Fabry Disease, has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 3 participants
- Enrollment target
- 1
- Study location
Study Summary
This study will determine the safety of the drug Replagal or treating patients with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called Alpha-galactosidase A, which normally breaks down a fatty substance called globotriaosylceramide (Gb3), is missing or does not function properly. The resulting accumulation of Gb3 causes problems with the kidneys, heart, nerves, and blood vessels. Replagal is a genetically engineered form of Alpha-galactosidase A. Previous studies have shown that patients with Fabry disease who had not progressed to end-stage kidney failure tolerated Replagal replacement therapy well. This study will examine the effects of the drug in patients with kidney problems associated with Fabry disease. Patients with Fabry disease who are on kidney dialysis, or have had a kidney transplant, may be eligible for this study. During this 6 to 12-month study, participants will receive a 40-minute intravenous (IV) infusion of Replagal every other week, with close monitoring during and after the infusions. Before the first infusion, patients will be evaluated with a medical history, physical and neurological examinations, electrocardiogram (ECG), routine blood and urine tests, kidney test, and measurements of height, weight, and vital signs (blood pressure, pulse, breathing rate, temperature). In addition, they will have pharmacokinetic studies immediately before and following the first infusion of Replagal. For these studies, blood samples of less than a teaspoon each will be drawn to measure the level of Replagal enzyme activity. The samples will be collected at the following time points: immediately before the infusion; 20 minutes into the infusion; at the end of the infusion; after the infusion at 50, 60, and 90 minutes, and 2, 3, 4, and 8 hours. Safety evaluations will be done once a week for the first month and then once a month for the rest of the study period. These evaluations include a physical examination, measurement of
Conditions Studied
Interventions
- DRUG DRX005B
Study Locations (1)
Maryland
- National Institute of Neurological Disorders and Stroke (NINDS) - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2002-11 |
| Est. Completion | 2003-11 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00048906
The ClinicalTrials.gov registry entry for NCT00048906 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 3 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 543-participant average among 32 other Fabry Disease trials with a reported enrollment target (99% lower). 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 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 1 intervention - of which DRX005B is the first listed.
NCT00048906 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT00048906 about?
NCT00048906 is a clinical study titled "Alpha-Galactosidase A Replacement Therapy for Fabry Disease". This study will determine the safety of the drug Replagal or treating patients with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called Alpha-galactosidase A, which normally breaks down a fatty substance called globotriaosylceramide (Gb3), is missing or does not functio...
What is the current status of trial NCT00048906?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 3 participants. The study started on 2002-11. Estimated completion is 2003-11.
What conditions does trial NCT00048906 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT00048906?
The interventions under investigation include: DRX005B (DRUG).
Who is sponsoring clinical trial NCT00048906?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00048906 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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