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NCT00244582 · ClinicalTrials.gov registry record
Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease
A clinical trial, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- 1
- Enrollment target
NCT00244582 is a clinical trial that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 1 participants.
The verdict
NCT00244582 has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- 1 participants
- Enrollment target
Study Summary
This study will use an experimental method of delivering the enzyme glucocerebrosidase directly into the brain of a patient with Gaucher disease to treat disease symptoms. Patients with Gaucher disease have insufficient levels of glucocerebrosidase. As a result, substances normally broken down by this enzyme accumulate in the body, causing damage to the brain and other organs. Symptoms of Gaucher disease outside the brain can be treated by infusing the missing enzyme intravenously (IV, through a vein). However, IV infusions do not help the neurologic symptoms of the disease, because the enzyme cannot get into the brain. This study will use a new technique called convection-enhanced delivery to try to introduce glucocerebrosidase directly into the brain. This single-patient study includes an 8-month-old male with Type 2 Gaucher disease with progressive neurological decline. In preparation for the enzyme infusion, the patient will have a complete physical examination, including a detailed neurological examination, and blood and urine tests. On the day of surgery, the child will be placed under general anesthesia for magnetic resonance imaging (MRI) of the brain. MRI uses a magnetic field and radio waves to produce images of body tissues and organs. For this procedure, the child lies on a table that is moved into the scanner (a narrow cylinder), wearing earplugs to protect his hearing from the loud knocking and thumping sounds that occur during the scanning process. He will then be brought to the operating room for the infusion procedure, as follows: An incision will be made in the scalp and a small hole drilled through the skull. Then, a small tube (cannula) will be positioned through the hole into the target area in the brain. Once the tip of the cannula is in place, it will be connected with tubing to a syringe filled with glucocerebrosidase in saline (salt water). The child will then have a second MRI scan to make sure the cannula is placed correctly and to moni
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2005-10-22 |
| Est. Completion | 2006-11-03 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00244582
The ClinicalTrials.gov registry entry for NCT00244582 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 1 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 0 interventions.
NCT00244582 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00244582 about?
NCT00244582 is a clinical study titled "Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease". This study will use an experimental method of delivering the enzyme glucocerebrosidase directly into the brain of a patient with Gaucher disease to treat disease symptoms. Patients with Gaucher disease have insufficient levels of glucocerebrosidase. As a result, substances normally broken down by th...
What is the current status of trial NCT00244582?
This trial is currently completed. The enrollment target is 1 participants. The study started on 2005-10-22. Estimated completion is 2006-11-03.
Who is sponsoring clinical trial NCT00244582?
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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