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SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation

This study is currently recruiting participants.
Verified March 2012 by Oregon Health and Science University
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Robert Steiner, Oregon Health and Science University
ClinicalTrials.gov Identifier:
NCT01434745
First received: September 7, 2011
Last updated: March 21, 2012
Last verified: March 2012
History of Changes

September 7, 2011
March 21, 2012
September 2011
October 2014   (final data collection date for primary outcome measure)
  • Intellectual quotient (IQ) [ Time Frame: every 4 months - in person or over the phone assessment ] [ Designated as safety issue: Yes ]
  • Development Quotient (DQ) [ Time Frame: every 4 months - in person or over the phone assessment ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT01434745 on ClinicalTrials.gov Archive Site
  • Whole body cholesterol pool size, synthesis & absorption using stable isotope testing [ Time Frame: every 4 months, after blood draws ] [ Designated as safety issue: Yes ]
  • Plasma and urine markers of sterol metabolism [ Time Frame: every 4 months, after blood & urine collection ] [ Designated as safety issue: Yes ]
  • Apparent diffusion coefficient (ADC) and fractional anisotopy FA and MRS lipids (brain MRI/MRS) [ Time Frame: once per year, after MRI ] [ Designated as safety issue: Yes ]
  • Log of response amplitude (electroretinogram) [ Time Frame: once per year, after ERG ] [ Designated as safety issue: Yes ]
Same as current
Not Provided
Not Provided
 
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

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.
Not Provided
Interventional
Not Provided
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Smith-Lemli-Opitz Syndrome
  • Drug: Simvastatin
    Simvastatin will be administered as a powder mixed with lactose at the dose of 0.5mg/kg/day
  • Dietary Supplement: Lactose
    Lactose will be administered in a capsule formula.
  • Experimental: Simvastatin
    Intervention: Drug: Simvastatin
  • Placebo Comparator: Placebo - Lactose
    Intervention: Dietary Supplement: Lactose
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
16
October 2014
October 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male or female over 1 years old
  • Subject has confirmed diagnosis of Smith-Lemli-Opitz Syndrome
  • Subject is currently receiving cholesterol supplementation

Exclusion Criteria:

  • Subjects too ill to travel to the study site
  • Subjects who are unable to safely undergo study procedures
  • Pregnant women
Both
1 Year to 89 Years
No
Contact: SHARON BUTCHER, RN 503-494-6524 BUTCHER@OHSU.EDU
United States
 
NCT01434745
R01HL073980, R01HL073980
Yes
Robert Steiner, Oregon Health and Science University
Oregon Health and Science University
National Heart, Lung, and Blood Institute (NHLBI)
Principal Investigator: ROBERT STEINER, MD Oregon Health and Science University
Oregon Health and Science University
March 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP