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NCT00114634 · ClinicalTrials.gov registry record · Phase 2
Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome
A Phase 2 study, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- Completed
- Registry status
- Phase 2
- Development phase
- 13
- Enrollment target
NCT00114634: Completed Phase 2 study, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
NCT00114634 is a Phase 2 study that has completed, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The registered enrollment target is 13 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (90% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00114634, a Phase 2 study, has completed, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 13 participants
- Enrollment target
Study Summary
This 10-week study will evaluate and compare behavior changes in children with Smith-Lemli-Opitz syndrome (SLOS) who are taking cholesterol supplementation versus those who are not on cholesterol supplementation. SLOS is a genetic disorder that affects the development of children both before and after birth. An enzyme deficiency in these children results in low levels of cholesterol, which can cause a variety of birth defects and behavioral problems. Typical abnormal physical features of patients include a small head, drooping eyelids, small upturned nose, small chin, cleft palate, heart defects, and extra fingers or toes. Children between 5 and 17 with mild SLOS who do not have a history of egg allergy or intolerance may be eligible for this study. Candidates are screened with a questionnaire about the patient's age, genotype (if known), sterol levels, symptoms, current treatment and medical history. Children participate in two 2-week study phases. Between the study phases the children will take 150 mg/kg daily of a cholesterol preparation typically used to supplement cholesterol in patients in SLOS studies at NIH. In the study phases, the participants are randomly assigned to take either egg yolk or an egg yolk substitute, such as Egg Beaters, that does not contain cholesterol. The study is done at the participant's home, and the cholesterol supplementation and egg/egg substitute are sent to the home each day with instructions on how to take them. The caretakers can stop the study phases after four days if behavior problems occur. The children's caretakers fill out a standard behavioral questionnaire, the Aberrant Behavior Checklist. The questionnaire is designed to assess the effects of treatment in mentally impaired persons.
Primary Outcome
The Aberrant Behavior Checklist-Community (ABC-C) is a measure used to identify treatment efficacy among intellectually impaired individuals. ABC Subscale IV (Hyperactivity) has 16 items, each can be rated from 0 to 3, with 0 equal to not at all a problems, one the problem is the behavior but slight in degree, to the problem is moderately serious, 3 the problem is severe in degree. For this subscale, score can go from 0 - 48. The higher the score, the worse the hyperactivity. The comparison in t
Interventions
- DIETARY_SUPPLEMENT Egg yolk preparation with cholesterol
- DIETARY_SUPPLEMENT Egg substitute, without cholesterol
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2005-06 |
| Est. Completion | 2009-02 |
| Phase | Phase 2 |
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)305 total trials
What the finished NCT00114634 record still lists
NCT00114634 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 13 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (90% lower).
The record links to 0 conditions, and to 2 interventions - of which Egg yolk preparation with cholesterol is the first listed.
NCT00114634 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00114634 about?
NCT00114634 is a clinical study titled "Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome". This 10-week study will evaluate and compare behavior changes in children with Smith-Lemli-Opitz syndrome (SLOS) who are taking cholesterol supplementation versus those who are not on cholesterol supplementation. SLOS is a genetic disorder that affects the development of children both before and aft...
What is the current status of trial NCT00114634?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 13 participants. The study started on 2005-06. Estimated completion is 2009-02.
What interventions are being tested in trial NCT00114634?
The interventions under investigation include: Egg yolk preparation with cholesterol (DIETARY_SUPPLEMENT), Egg substitute, without cholesterol (DIETARY_SUPPLEMENT).
Who is sponsoring clinical trial NCT00114634?
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00114634's enrollment target sits among peer trials
13 1947th of 2000 higher than 53 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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