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NCT00001410 · ClinicalTrials.gov registry record · Phase 1
PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
A Phase 1 study of Gaucher's Disease, sponsored by National Institute of Mental Health (NIMH).
- Completed
- Registry status
- Phase 1
- Development phase
- 18
- Enrollment target
- 1
- Study location
NCT00001410: Completed Phase 1 study of Gaucher's Disease, sponsored by National Institute of Mental Health (NIMH).
NCT00001410 is a Phase 1 study of Gaucher's Disease that has completed, run by National Institute of Mental Health (NIMH). The registered enrollment target is 18 participants, below the 93-participant average among 3 other Gaucher's Disease trials with a reported enrollment target (81% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00001410, a Phase 1 study of Gaucher's Disease, has completed, sponsored by National Institute of Mental Health (NIMH).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 18 participants
- Enrollment target
- 1
- Study location
Study Summary
Gaucher disease is a lysosomal storage disease resulting from glucocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages. The condition is marked by enlargement of the liver and spleen (hepatosplenomegaly), low blood and platelet counts, and bone abnormalities. The condition is passed from generation to generation on via autosomal recessive inheritance. There are actually three types of Gaucher disease. Type I is the most common form. It is a chronic non-neuronopathic form, meaning the disease does not affect the nervous system. The symptoms of type I can appear at any age. Type 2 Gaucher disease presents prenatally or in infancy and usually results in death for the patient. Type 2 is an acute neuronopathic form and can affect the brain stem. It is the most severe form of the disease. Type 3 Gaucher disease is also neuronopathic, however it is subacute in nature. This means the course of the illness lies somewhere between long-term (chronic) and short-term (acute). Currently there is not a cure for Gaucher disease. Treatment for the disease has traditionally been supportive. In some severely affected patients, bone-marrow transplants have corrected the enzyme deficiency, but it is considered a high-risk procedure and recovery can be very slow. Enzyme replacement therapy is another therapy option and has been approved by the Food and Drug Administration (FDA) for use in type 1 patients. PEG-glucocerbrosidase is a drug designed to clear out the accumulation of lipid (glucocerebroside) from the blood stream. The drug is actually an enzyme attached to large molecules called polyethylene glycol (PEG). The large molecules of PEG allow the enzyme to remain in the blood stream for long periods of time. By modifying glucocerebrosidase with PEG, it is believed that smaller doses will be required, meaning a reduction in cost for the patient and more convenient administration of the drug.
Conditions Studied
Interventions
- DRUG Lysodase
Study Locations (1)
Maryland
- National Institute of Mental Health (NIMH) - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 18 participants |
| Start Date | 1993-10 |
| Est. Completion | 2001-12 |
| Phase | Phase 1 |
What the finished NCT00001410 record still lists
NCT00001410 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 18 participants, a relatively small participant target, below the 93-participant average among 3 other Gaucher's Disease trials with a reported enrollment target (81% lower).
The record links to 1 condition, with Gaucher's Disease appearing as the primary indexed condition, and to 1 intervention - of which Lysodase is the first listed.
NCT00001410 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT00001410 about?
NCT00001410 is a clinical study titled "PEG-Glucocerebrosidase for the Treatment of Gaucher Disease". Gaucher disease is a lysosomal storage disease resulting from glucocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages. The condition is marked by enlargement of the liver and spleen (hepatosplenomegaly), low bloo...
What is the current status of trial NCT00001410?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 18 participants. The study started on 1993-10. Estimated completion is 2001-12.
What conditions does trial NCT00001410 study?
This clinical trial studies the following conditions: Gaucher's Disease.
What interventions are being tested in trial NCT00001410?
The interventions under investigation include: Lysodase (DRUG).
Who is sponsoring clinical trial NCT00001410?
This trial is sponsored by National Institute of Mental Health (NIMH), which has 343 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00001410 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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