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NCT00075244 · ClinicalTrials.gov registry record · Phase 2
Alternative Dosing and Regimen of Replagal to Treat 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
- 25
- Enrollment target
- 1
- Study location
NCT00075244 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 25 participants, below the 542-participant average among 32 other Fabry Disease trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state.
The verdict
NCT00075244, 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
- 25 participants
- Enrollment target
- 1
- Study location
Study Summary
The main goal of this study is to assess the pharmacodynamic effects of different or more frequent doses of Replagal compared to the standard dosing regimen. Replagal is a genetically engineered form of alpha-Galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb(3)). In patients with Fabry disease, GB(3) does not function properly and therefore builds up causing problems with the kidneys, heart, nerves, and blood vessels. Male patients 18 years of age or older with Fabry disease who are not on dialysis and have not received a kidney transplant may be eligible for this study. Participants are randomly assigned to receive one of the following five regimens of Replagal infusions, given through a vein over 20 to 80 minutes: 0.1 mg/kg body weight every week 0.2 mg/kg body weight every week 0.2 mg/kg body weight every other week 0.4 mg/kg body weight every week 0.4 mg/kg body wieght every other week In the US, the infusions are given at the NIH Clinical Center. Vital signs are measured before, immediately after, and 1 hour after each infusion. Baseline evaluations are done on an inpatient or outpatient basis. Baseline tests include a check of vital signs (temperature, respiratory rate, pulse rate, and blood pressure); physical examination; laboratory tests; and review of treatment side effects. Evaluations are also done at every infusion visit, and 1 week and 1 month after the last infusion. Safety evaluations are done periodically and include vital sign measurements, physical examination, blood and urine tests, review of drug side effects, electrocardiogram (ECG), Holder monitor (2 hour ECG), and QSART (NIH only). The QSART (quantitative sudomotor axon reflex test) measures the amount of sweat in a particular area of skin, mostly the forearm. For this test, a cup partly filled with a liquid is strapped on the arm. A weak electric current is turned on, stimulating the sweat glands, and the amount of sweat produce
Conditions Studied
Interventions
- DRUG Replagal
Study Locations (1)
Maryland
- National Institute of Neurological Disorders and Stroke (NINDS) - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2004-01 |
| Est. Completion | 2005-11 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00075244
The ClinicalTrials.gov registry entry for NCT00075244 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 25 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 (95% 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 Replagal is the first listed.
NCT00075244 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT00075244 about?
NCT00075244 is a clinical study titled "Alternative Dosing and Regimen of Replagal to Treat Fabry Disease". The main goal of this study is to assess the pharmacodynamic effects of different or more frequent doses of Replagal compared to the standard dosing regimen. Replagal is a genetically engineered form of alpha-Galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosyl...
What is the current status of trial NCT00075244?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 25 participants. The study started on 2004-01. Estimated completion is 2005-11.
What conditions does trial NCT00075244 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT00075244?
The interventions under investigation include: Replagal (DRUG).
Who is sponsoring clinical trial NCT00075244?
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 NCT00075244 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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