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NCT00001416 · ClinicalTrials.gov registry record · Phase 2
Bone Response to Enzyme Replacement in Gaucher's Disease
A Phase 2 study of Gaucher's Disease, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- Phase 2
- Development phase
- 100
- Enrollment target
- 1
- Study location
NCT00001416: Completed Phase 2 study of Gaucher's Disease, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
NCT00001416 is a Phase 2 study of Gaucher's Disease that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 100 participants, above the 65-participant average among 3 other Gaucher's Disease trials with a reported enrollment target (54% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00001416, a Phase 2 study of Gaucher's Disease, has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 100 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this study is to examine how the skeleton responds to repeated doses of enzyme replacement therapy in patients with type I Gaucher's disease who have had their spleens removed. Gaucher disease is a lysosomal storage disease resulting from glycocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in adults but occurs most severely in infants, in whom cerebroside also accumulates in neurons. Patients with Gaucher's disease experience enlargement of the liver and spleen and bone destruction. The condition is passed from generation to generation through autosomal recessive inheritance. Type I is the most common form. It is a chronic non-neuronopathic form, meaning the disease does not affect nerve cells. The symptoms of type I can appear at any age. In this study patients will be divided into three groups. Each group will receive different doses of enzyme replacement (Ceredase). In addition, two of the three groups will also receive doses of a form of vitamin D (calcitriol). Researchers believe the groups receiving vitamin D will have an improved response as compared to those patients only receiving enzyme replacement. Patients in each group who respond to enzyme replacement with increases in bone density will be compared to the other treatment groups.
Conditions Studied
Interventions
- DRUG CEREDASE™
Study Locations (1)
Maryland
- National Institute of Neurological Disorders and Stroke (NINDS) - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 100 participants |
| Start Date | 1993-12 |
| Est. Completion | 2000-11 |
| Phase | Phase 2 |
What the finished NCT00001416 record still lists
NCT00001416 is an interventional study that assigns participants to a tested intervention. The registered 100 participants enrollment target is mid-sized for trials with a published cap, above the 65-participant average among 3 other Gaucher's Disease trials with a reported enrollment target (54% higher).
The record links to 1 condition, with Gaucher's Disease appearing as the primary indexed condition, and to 1 intervention - of which CEREDASE™ is the first listed.
NCT00001416 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT00001416 about?
NCT00001416 is a clinical study titled "Bone Response to Enzyme Replacement in Gaucher's Disease". The purpose of this study is to examine how the skeleton responds to repeated doses of enzyme replacement therapy in patients with type I Gaucher's disease who have had their spleens removed. Gaucher disease is a lysosomal storage disease resulting from glycocerebroside accumulation in macrophages ...
What is the current status of trial NCT00001416?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 100 participants. The study started on 1993-12. Estimated completion is 2000-11.
What conditions does trial NCT00001416 study?
This clinical trial studies the following conditions: Gaucher's Disease.
What interventions are being tested in trial NCT00001416?
The interventions under investigation include: CEREDASE™ (DRUG).
Who is sponsoring clinical trial NCT00001416?
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 NCT00001416 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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