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NCT00041535 · ClinicalTrials.gov registry record · Phase 2
OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
A Phase 2 study, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- Phase 2
- Development phase
- 30
- Enrollment target
NCT00041535 is a Phase 2 study that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 30 participants.
The verdict
NCT00041535, a Phase 2 study, has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 30 participants
- Enrollment target
Study Summary
Gaucher disease is an inherited functional deficiency of glucocerebrosidase. This enzyme breaks down a fatty substance (lipid) called glucocerebroside, which is present in all cells of the body. When cells renew themselves, the lipids must be broken down and discarded. Because the enzyme does not function well, the lipid builds up in certain tissues, such as the liver and spleen. The nervous system is involved as well; memory is impaired and it is difficult to move the eyes from side to side. It has been shown that repeated infusions of glucocerebrosidase help break down the stored lipid. However, this treatment does not improve any neurological symptoms. A medicine called OGT 918 has been shown to slow the production of the lipid that builds up in Gaucher disease. It also has been shown to enter the brain. It is hoped that taking OGT 918 will reduce the storage of glycolipids in cells and improve the neurological symptoms of the disease. This clinical trial seeks to evaluate OGT 918 as a treatment for neuronopathic Gaucher disease by assessing changes in eye movement velocity. A secondary goal is to assess the clinical safety and tolerability of OGT 918 therapy. Up to 30 patients from the National Institutes of Health and the Institute of Child Health (London) will be randomly assigned to OGT 918 or no treatment for 12 months. Study participants must be clinically diagnosed with neuronopathic Gaucher disease, 12 years of age or older, and able to swallow capsules. They must have been stable on ERT for at least 6 months before the study. Patients receiving OGT 918 will receive a dose of 200 mg OGT 918 three times daily. Data analysis will be done after 12 months. The study will be extended up to 12 months to collect safety and efficacy data. All patients who complete the main study and enter the extension study will receive OGT 918. During a 4-week screening period, eye movement velocity will be measured. These assessments will be repeated at months 12 and 24. A
Interventions
- DRUG OGT 918
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2002-07-05 |
| Est. Completion | 2007-03-21 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00041535
The ClinicalTrials.gov registry entry for NCT00041535 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 30 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 1 intervention - of which OGT 918 is the first listed.
NCT00041535 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00041535 about?
NCT00041535 is a clinical study titled "OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease". Gaucher disease is an inherited functional deficiency of glucocerebrosidase. This enzyme breaks down a fatty substance (lipid) called glucocerebroside, which is present in all cells of the body. When cells renew themselves, the lipids must be broken down and discarded. Because the enzyme does not fu...
What is the current status of trial NCT00041535?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 30 participants. The study started on 2002-07-05. Estimated completion is 2007-03-21.
What interventions are being tested in trial NCT00041535?
The interventions under investigation include: OGT 918 (DRUG).
Who is sponsoring clinical trial NCT00041535?
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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