Status:
COMPLETED
Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
Lead Sponsor:
Boston Children's Hospital
Conditions:
Smith-Lemli-Opitz Syndrome
Eligibility:
All Genders
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to determine whether supplementation with an oil-based cholesterol suspension will correct the biochemical abnormalities in cholesterol and its precursors in individuals w...
Detailed Description
This study involves treating individuals with the Smith-Lemli-Opitz syndrome, a rare inborn error of cholesterol metabolism, with supplemental cholesterol to determine it effects on biochemical sterol...
Eligibility Criteria
Inclusion
- Biochemical confirmation of sterol defect associated with Smith-Lemli-Opitz syndrome
Exclusion
- Inability to tolerate crystalline cholesterol
- Inability to travel to Boston 3-4 times/year based on age
Key Trial Info
Start Date :
January 1 1998
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2011
Estimated Enrollment :
23 Patients enrolled
Trial Details
Trial ID
NCT00272844
Start Date
January 1 1998
End Date
July 1 2011
Last Update
October 20 2017
Active Locations (1)
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1
Children's Hospital Boston
Boston, Massachusetts, United States, 02115