Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00344331 · ClinicalTrials.gov registry record
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
A clinical trial of Niemann-Pick Disease, Type C, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- Recruiting
- Registry status
- 900
- Enrollment target
- 1
- Study location
NCT00344331 is a study of Niemann-Pick Disease, Type C that is actively recruiting participants, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The registered enrollment target is 900 participants, above the 63-participant average among 3 other Niemann-Pick Disease, Type C trials with a reported enrollment target (1329% higher). The trial reports 1 study location across 1 state.
The verdict
NCT00344331, a study of Niemann-Pick Disease, Type C, is actively recruiting participants, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- RECRUITING
- Registry status
- 900 participants
- Enrollment target
- 1
- Study location
Study Summary
This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick Disease, Type C (NPC), a genetic disorder that results in progressive loss of nervous system function. The study will: 1) look for a clinical or biochemical marker that can be used as a measure of response to treatment, and 2) define the rate of progression of biochemical marker abnormalities in a group of NPC patients who will later be invited to enroll in a treatment trial. Patients of any age with NPC may be eligible for this study. Participants undergo the following procedures every 6 months during 4- to 5-day admissions at the NIH Clinical Center. * Medical evaluation, including medical history, physical exam, neurological exam, neuropsychometric evaluation, and blood and urine tests. * Lumbar puncture (spinal tap): A sample of cerebrospinal fluid (CSF), the fluid that bathes the brain and spinal cord, is obtained for study. After administration of a local anesthetic, a small needle is inserted in the space between the bones in the lower back where the CSF circulates below the spinal cord. A small amount of fluid is collected through the needle. * Eye exam and eye movement study: The pupils of the eye are dilated to examine the structures of the eyes. For the eye movement study a special contact lens is placed on the eye and the patient looks at a series of target light spots moving on a screen. * Hearing tests. * Electroretinography (in patients who can cooperate with the test) to measure the function of the retina. Before the test, the patient's pupils are dilated and an electrode (small silver disk) is taped to the forehead. The patient sits in a dark room for 30 minutes and then a special contact lens is placed on one eye after it has been numbed with drops. The contact lens senses small electrical signals generated by the retina when lights flash. During the ERG recording, the eye is stimulated wit
Conditions Studied
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 900 participants |
| Start Date | 2006-08-14 |
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)305 total trials
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00344331
The ClinicalTrials.gov registry entry for NCT00344331 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 900 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 63-participant average among 3 other Niemann-Pick Disease, Type C trials with a reported enrollment target (1329% higher). The listed sponsor is Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Niemann-Pick Disease, Type C appearing as the primary indexed condition, and to 0 interventions.
NCT00344331 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT00344331 about?
NCT00344331 is a clinical study titled "Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C". This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick Disease, Type C (NPC), a genetic disorder that results in progressive loss of nervous system function. The study will: 1) look for a clinical...
What is the current status of trial NCT00344331?
This trial is currently recruiting. The enrollment target is 900 participants. The study started on 2006-08-14.
What conditions does trial NCT00344331 study?
This clinical trial studies the following conditions: Niemann-Pick Disease, Type C.
Who is sponsoring clinical trial NCT00344331?
This trial is sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00344331 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Niemann-Pick Disease, Type C
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.