Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome
Smith-Lemli-Opitz Syndrome
About this trial
This is an interventional treatment trial for Smith-Lemli-Opitz Syndrome focused on measuring SLOS, RSH syndrome, ABC Checklist, Cholesterol supplementation, Egg yolk, Smith-Lemli-Opitz Syndrome
Eligibility Criteria
Inclusion This study will include pediatric patients, ages 4-17 years old with a biochemical diagnosis of Smith-Lemli-Opitz Syndrome (SLOS). Only mild and classical patients will be enrolled. This study will be open to include SLOS patients regardless of whether or not they are participating in another NIH protocol. Exclusion Patients with a history of egg allergy or intolerance will be excluded from this study. Subjects must be well enough to be in a home setting. Patients participating in our simvastatin protocol (03-CH-3225) will be excluded from this study.
Sites / Locations
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Egg yolk preparation with cholesterol
Egg yolk preparation without cholesterol
Dietary cholesterol in the form of liquid egg yolk
No dietary cholesterol supplementation (egg substitute) Papetti Foods "Better 'n Eggs" egg substitute