NCT01434745

SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation

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

• Status: Terminated
• Phase: N/A
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Oregon Health and Science University · Academic / Other
• Sex: All
• Age: 1 Year – 89 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Smith-Lemli-Opitz Syndrome

Interventions

Type	Name	Description
DRUG	Simvastatin	Simvastatin will be administered as a powder mixed with lactose at the dose of 0.5 mg/kg/day
DIETARY_SUPPLEMENT	Lactose	Lactose will be administered in a capsule formula.

Timeline

• Start date: 2011-09-01
• Primary completion: 2014-10-01
• Completion: 2014-10-01
• First posted: 2011-09-15
• Last updated: 2019-10-01
• Results posted: 2019-10-01

Locations

1 site across 1 country: United States