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NCT00357786 · ClinicalTrials.gov registry record · Phase 1
An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease
A Phase 1 study, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- Phase 1
- Development phase
- 3
- Enrollment target
NCT00357786 is a Phase 1 study that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 3 participants.
The verdict
NCT00357786, a Phase 1 study, has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 3 participants
- Enrollment target
Study Summary
This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. 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 ceramidetrihexoside, 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. Vascular, renal, and neurological problems are the results. It is not known exactly how lipid accumulation brings about such problems, studies of another lipid storage disorder. Two patients 7 to 17 years of age who have Fabry disease and have been receiving intravenous infusions of Replagal at a dose of 0.2 mg/kg of body weight every 2 weeks may be eligible for this study. Participants will undergo the following tests and procedures: * Physical examination. * Neurological examination. * Medical and medication history. * Vital signs. * Assessment of height and weight. * Blood tests to determine complete blood count and chemistries. * Electrocardiogram. * Doppler blood flow study. Participants will go through a baseline evaluation, over a period of about 1 day. They will receive an intravenous infusion of Replagal every other week, at the dose of 0.2 mg/kg of body weight. Vital signs will be measured before the infusion and immediately and after and 1 hour afterward. There will be careful monitoring for allergic reactions and side effects. The infusion time takes approximately 40 minutes. This study will last at least 1 year, or until the sponsor doing the investigating or the drug manufacturer decides to withdraw support of the study.
Interventions
- DRUG Replagal
- DRUG Replagal agalsidase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2003-10 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00357786
The ClinicalTrials.gov registry entry for NCT00357786 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 3 participants, a figure that helps gauge the scale of data the investigators plan to collect. 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 0 conditions, and to 2 interventions - of which Replagal is the first listed.
NCT00357786 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00357786 about?
NCT00357786 is a clinical study titled "An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease". This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. 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 f...
What is the current status of trial NCT00357786?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2003-10.
What interventions are being tested in trial NCT00357786?
The interventions under investigation include: Replagal (DRUG), Replagal agalsidase alfa (DRUG).
Who is sponsoring clinical trial NCT00357786?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
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