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SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation

Smith-Lemli-Opitz Syndrome Clinical Trial

Official title:
Smith-Lemli Opitz Syndrome: A Clinical Investigation of the Effect of Simvastatin in Patients Receiving Cholesterol Supplementation

Clinical Trial Summary

The purpose of this study is to determine if simvastatin improves development and behavior in patients with Smith Lemli-Opitz syndrome (SLOS) receiving dietary cholesterol supplementation.

Clinical Trial Description

Patients with SLOS receiving dietary cholesterol supplementation are given simvastatin, a drug that decreases the activity/expression of HMG-CoA reductase, an enzyme that controls the first step of the cholesterol synthesis pathway, reduces the accumulation of toxic 7-dehydrocholesterol (immediate metabolic precursor of cholesterol) and improve neurocognitive and behavioral outcomes. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01434745
Study type Interventional
Source Oregon Health and Science University
Contact
Status Terminated
Phase N/A
Start date September 2011
Completion date October 2014
View Details
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