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NCT00028262 · ClinicalTrials.gov registry record · Phase 4
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
A Phase 4 study of Infantile Neronal Ceroid Lipofuscinosis, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- Completed
- Registry status
- Phase 4
- Development phase
- 10
- Enrollment target
- 1
- Study location
NCT00028262: Completed Phase 4 study of Infantile Neronal Ceroid Lipofuscinosis, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
NCT00028262 is a Phase 4 study of Infantile Neronal Ceroid Lipofuscinosis that has completed, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The registered enrollment target is 10 participants, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00028262, a Phase 4 study of Infantile Neronal Ceroid Lipofuscinosis, has completed, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children. At around 11 to 13 months of age, patients develop slowed head growth, mild brain atrophy (wasting), electroencephalographic (EEG) changes and retinal deterioration, with symptoms worsening over time. The disease results from an enzyme deficiency that causes fatty compounds called ceroid to accumulate in cells. In laboratory experiments, Cystagon has helped remove ceroid from cells of patients with INCL. Children with INCL between 6 months and 3 years of age may be eligible for this study. Participants take Cystagon daily by mouth every 6 hours. They are admitted to the NIH Clinical Center for a 4- to 5-day period every 6 months for the following tests and evaluations: * Review of medical history, including a detailed record of seizures, physical examination, blood tests and clinical photographs. For the initial baseline studies, examinations may also be scheduled with pediatric neurology, ophthalmology and anesthesia services. * Magnetic resonance imaging (MRI) of the brain MRI uses a powerful magnet, radio waves, and computers to provide detailed images of the brain without the use of X-rays. The patient lies on a table that slides inside a donut-shaped machine containing a magnetic field. The child requires general anesthesia for the procedure. * Electroretinogram (ERG) measures the function of the retina, the light-sensitive tissue in the back of the eye. To record the flash ERG, a special contact lens is placed on the eye s surface and the eye is stimulated with flashes of light. Infants and very young children require general anesthesia for the procedure. * Visual evoked potential (VEP) measures the function of the visual pathway from the eye to the brain. To record the VEP, five electrodes are placed on the scalp and the eye is stimulated with flashes of light. Infants and ver
Primary Outcome
The GRODs in peripheral white blood cells from all patients before and during treatment were analyzed by transmission electron microscopy (TEM) at 30000xmagnification. Two investigators working independently of each other identified and counted the GRODs and the results were averaged.
Conditions Studied
Interventions
- DRUG Cystagon
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center, 9000 Rockville Pike - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2001-02 |
| Est. Completion | 2013-11 |
| Phase | Phase 4 |
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)305 total trials
What the finished NCT00028262 record still lists
NCT00028262 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Infantile Neronal Ceroid Lipofuscinosis appearing as the primary indexed condition, and to 1 intervention - of which Cystagon is the first listed.
NCT00028262 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT00028262 about?
NCT00028262 is a clinical study titled "Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis". This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children. At around 11 to 13 months of age, patients develop slowed head growth, mild brain atrophy (wasting), electroencephal...
What is the current status of trial NCT00028262?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 10 participants. The study started on 2001-02. Estimated completion is 2013-11.
What conditions does trial NCT00028262 study?
This clinical trial studies the following conditions: Infantile Neronal Ceroid Lipofuscinosis.
What interventions are being tested in trial NCT00028262?
The interventions under investigation include: Cystagon (DRUG).
Who is sponsoring clinical trial NCT00028262?
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 NCT00028262 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
How this trial's enrollment target compares
Where NCT00028262's enrollment target sits among peer trials
10 1941st of 2000 higher than 33 of 2,000 other Phase 4 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 4 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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