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NCT00068107 · ClinicalTrials.gov registry record · Phase 2
Dosing Study of Replagal in Patients With Fabry Disease
A Phase 2 study of Fabry Disease, sponsored by Baylor Research Institute.
- Completed
- Registry status
- Phase 2
- Development phase
- 13
- Enrollment target
- 1
- Study location
NCT00068107 is a Phase 2 study of Fabry Disease that has completed, run by Baylor Research Institute. The registered enrollment target is 13 participants, below the 542-participant average among 32 other Fabry Disease trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state.
The verdict
NCT00068107, a Phase 2 study of Fabry Disease, has completed, sponsored by Baylor Research Institute.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 13 participants
- Enrollment target
- 1
- Study location
Study Summary
This study will determine the safety and effectiveness of increasing Replagal infusions in certain patients with Fabry disease. Replagal is a genetically engineered form of Alpha-galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb3). In patients with Fabry disease, Alpha-galactosidase A does not function properly and, therefore, Gb3 builds up, causing problems with the kidneys, heart, nerves, and blood vessels. Patients with Fabry disease who are participating in NIH protocol 00-N-0185 or 02-N-0220 may be eligible for this study. This includes patients who are currently taking Replagal but whose kidney function continues to worsen, or patients who have certain test results that are much improved after Replagal infusion. Participants will receive Replagal infusions (0.2 mg/kg body weight) through a vein once a week (as opposed to the previous dosage of once every 2 weeks) for up to 2 years. The first infusion, and some others, are given at the NIH Clinical Center, but most are administered by the patient's local doctor. Vital signs are measured before, immediately after, and 1 hour after each infusion. Baseline evaluations are done on an inpatient basis at the NIH Clinical Center over a 1-week period before and after the first Replagal infusion and at 6-month intervals during the study. Tests include a check of vital signs (temperature, respiratory rate, pulse rate, and blood pressure); weight measurement; physical and neurological examinations; routine blood and urine tests; 24-hour urine collection; electrocardiogram; and review of treatment side effects. In addition, the following tests are done: * Quantitative sensory testing: This is a non-invasive test to measure the ability to sense warm, cold and vibration in the hand and foot. * QSART: This test measures the amount of sweat in a particular area of skin that did not sweat enough. A small amount of a medicine called acetylcholine is put on the skin and ma
Conditions Studied
Interventions
- DRUG Replagal
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center, 9000 Rockville Pike - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2003-09 |
| Est. Completion | 2013-12 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00068107
The ClinicalTrials.gov registry entry for NCT00068107 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 13 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 542-participant average among 32 other Fabry Disease trials with a reported enrollment target (98% lower). The listed sponsor is Baylor Research Institute, which has 180 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 Replagal is the first listed.
NCT00068107 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT00068107 about?
NCT00068107 is a clinical study titled "Dosing Study of Replagal in Patients With Fabry Disease". This study will determine the safety and effectiveness of increasing Replagal infusions in certain patients with Fabry disease. Replagal is a genetically engineered form of Alpha-galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb3). In patients wi...
What is the current status of trial NCT00068107?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 13 participants. The study started on 2003-09. Estimated completion is 2013-12.
What conditions does trial NCT00068107 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT00068107?
The interventions under investigation include: Replagal (DRUG).
Who is sponsoring clinical trial NCT00068107?
This trial is sponsored by Baylor Research Institute, which has 180 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00068107 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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