Status:
COMPLETED
Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome
Lead Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Conditions:
Smith-Lemli-Opitz Syndrome
Eligibility:
All Genders
4-17 years
Phase:
PHASE2
Brief 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 supp...
Detailed Description
Smith-Lemli- Opitz syndrome (SLOS) is an autosomal recessive genetic condition caused by a deficiency of the enzyme 3beta-hydroxysterol delta(7)- reductase (DHCR7). DHCR7 is the final enzyme in the st...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
June 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2009
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT00114634
Start Date
June 1 2005
End Date
February 1 2009
Last Update
January 29 2016
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892