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Bone Response to Enzyme Replacement in Gaucher's Disease
NCT00001416 · View on ClinicalTrials.gov ↗
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00001416
The ClinicalTrials.gov registry entry for NCT00001416 describes a study currently listed as completed. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 100 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 339 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT00001416 reports 1 study location spanning 1 distinct geographic area — top geographies include Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT00001416?
The interventions under investigation include: CEREDASE™ (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT00001416?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 339 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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