Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome

Completed · Phase 1/Phase 2

Conditions studied: Smith-Lemli-Opitz Syndrome

In brief

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 with the Smith-Lemli-Opitz syndrome.

Key facts

Study ID

NCT00272844

Run by

Boston Children's Hospital

People needed

23

Starts

1998-01-01

Expected to finish

2011-07-01

Last updated by the study team

2017-10-20

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Biochemical confirmation of sterol defect associated with Smith-Lemli-Opitz syndrome

You may not qualify if…

• Inability to tolerate crystalline cholesterol
• Inability to travel to Boston 3-4 times/year based on age

Where it is running

• Children's Hospital Boston — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

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