Clinical Trial: SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation
Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional
Official Title: Smith-Lemli Opitz Syndrome: A Clinical Investigation of the Effect of Simvastatin in Patients Receiving Cholesterol Supplementation
Brief 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 Summary:
Sponsor: Oregon Health and Science University
Current Primary Outcome:
- Intellectual quotient (IQ) [ Time Frame: every 4 months - in person or over the phone assessment ]
- Development Quotient (DQ) [ Time Frame: every 4 months - in person or over the phone assessment ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Whole body cholesterol pool size, synthesis & absorption using stable isotope testing [ Time Frame: every 4 months, after blood draws ]
- Plasma and urine markers of sterol metabolism [ Time Frame: every 4 months, after blood & urine collection ]
- Apparent diffusion coefficient (ADC) and fractional anisotopy FA and MRS lipids (brain MRI/MRS) [ Time Frame: once per year, after MRI ]
- Log of response amplitude (electroretinogram) [ Time Frame: once per year, after ERG ]
Original Secondary Outcome: Same as current
Information By: Oregon Health and Science University
Dates:
Date Received: September 7, 2011
Date Started: September 2011
Date Completion: October 2014
Last Updated: March 21, 2012
Last Verified: March 2012