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NCT00071877 · ClinicalTrials.gov registry record · Phase 2
An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With 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
NCT00071877 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
NCT00071877, 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
This study will evaluate the safety of multiple biweekly intravenous doses of Replagal over 26 weeks in 25 children with Fabry disease and the way in which that agent can improve the health of this patient population. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It causes a deficiency in the enzyme alpha galactosidase, which normally breaks down a lipid, or fatty substance, called ceramidetrihexosidase, a building block in all cells of the body. The deficiency in breaking down the lipid eventually causes that lipid to accumulate and injure cells. Problems in the blood vessels, kidneys, heart, and nerves are the result. The disease typically occurs in childhood or adolescence, with repeated episodes of severe pain in the extremities and other symptoms. There is no definitive treatment, but pain management is important in caring for patients with Fabry disease. Although it is not known exactly how lipid accumulation brings about such problems, studies of another lipid storage disorder, Gaucher's disease, have shown that the illness can be reversed if the lipid is removed when an appropriate enzyme, Replagal, is given intravenously. In this study, the gene response of the body's cells to Fabry disease will be described, as will any gene responses that change when the enzyme is used. Patients 7 to 17 years of age who have Fabry disease may be eligible for this study. They will undergo the following tests and procedures: * Physical examination. * Neurological examination. * Vital signs. * Urinalysis. * Blood tests to determine complete blood count and chemistries. * Questionnaire on pain. * Tests pertaining to sweating. * Electrocardiogram. * Doppler blood flow study. * Diary for recording symptoms and the use of pain medications. Participants will go through the evaluation, over a period of about 5 days, either as an inpatient or outpatient. Participants will receive an intravenous infusion of Replagal every other week, at the dose o
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 | 2003-10 |
| Est. Completion | 2005-06 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00071877
The ClinicalTrials.gov registry entry for NCT00071877 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.
NCT00071877 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT00071877 about?
NCT00071877 is a clinical study titled "An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease". This study will evaluate the safety of multiple biweekly intravenous doses of Replagal over 26 weeks in 25 children with Fabry disease and the way in which that agent can improve the health of this patient population. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It c...
What is the current status of trial NCT00071877?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 25 participants. The study started on 2003-10. Estimated completion is 2005-06.
What conditions does trial NCT00071877 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT00071877?
The interventions under investigation include: Replagal (DRUG).
Who is sponsoring clinical trial NCT00071877?
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 NCT00071877 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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