An interventional study of Simvastatin and Lactose in Smith-Lemli-Opitz Syndrome, sponsored by Oregon Health and Science University. Terminated at 1 site in United States. Open to participants aged 1 Year to 89 Years. Per ClinicalTrials.gov, last updated 2019-10-01.
Sponsored by Oregon Health and Science University · Not applicable, Interventional, and Treatment
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.
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.
Oregon Health and Science University is the lead sponsor of 694 studies on the registry; 133 are open to participants now.
Of its 51 completed or terminated interventional studies of FDA-regulated products, 39 (76%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
placebo
Dietary Supplement: Lactose
0.5 mg/kg body weight/day
Drug: Simvastatin
Simvastatin will be administered as a powder mixed with lactose at the dose of 0.5 mg/kg/day
Also known as: Zocor, Flolipid
Lactose will be administered in a capsule formula.
Development Quotient (DQ)
neurocognitive assessment measured with Mullen Scales of Learning
Time frame: through study completion, an average of 2 per year
Whole Body Cholesterol Pool Size, Synthesis & Absorption Using Stable Isotope Testing
administration of cholesterol and water labeled with stable isotope followed by measurement overtime in blood concentrations
Time frame: end of treatment, an average of 1 per year
Plasma Marker of Sterol Metabolism
Blood cholesterol to 7-dehydrocholesterol ratio
Time frame: through study completion, an average of 2 per year
ADC
Apparent diffusion coefficient measured by brain diffusion tensor imaging (DTI)
Time frame: end of treatment, an average of 1 per year
MVA
urinary mevalonate excretion
Time frame: through study completion, an average of 2 per year
MRS Lipids
Brain magnetic resonance spectroscopy
Time frame: end of treatment, an average of 1 per year
FA
Fractional anisotropy as measured by brain diffusion tensor imaging (DTI)
Time frame: end of treatment, an average of 1 per year
Only one participant enrolled
| Milestone | Placebo - Lactose | Simvastatin |
|---|---|---|
| Started | 0 | 0 |
| Completed | 0 | 0 |
| Not completed | 0 | 0 |
neurocognitive assessment measured with Mullen Scales of Learning
No measurements were reported for this outcome.
administration of cholesterol and water labeled with stable isotope followed by measurement overtime in blood concentrations
No measurements were reported for this outcome.
Blood cholesterol to 7-dehydrocholesterol ratio
No measurements were reported for this outcome.
Apparent diffusion coefficient measured by brain diffusion tensor imaging (DTI)
No measurements were reported for this outcome.
urinary mevalonate excretion
No measurements were reported for this outcome.
Brain magnetic resonance spectroscopy
No measurements were reported for this outcome.
Fractional anisotropy as measured by brain diffusion tensor imaging (DTI)
No measurements were reported for this outcome.
Collected over 3 years. Non-serious events are listed at a 1% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo - Lactose | — | 0/1 (0%) | 0/1 (0%) |
| Simvastatin | — | 0/1 (0%) | 0/1 (0%) |
Analyses were not completed and will never be completed due to insufficient funding
| Age, Categorical | Placebo - Lactose | Simvastatin | Total |
|---|---|---|---|
| <=18 years | — | — | — |
| Between 18 and 65 years | — | — | — |
| >=65 years | — | — | — |
| Age, Continuous | Placebo - Lactose | Simvastatin | Total |
|---|
| Sex: Female, Male | Placebo - Lactose | Simvastatin | Total |
|---|---|---|---|
| Female | — | — | — |
| Male | — | — | — |
| Ethnicity (NIH/OMB) | Placebo - Lactose | Simvastatin | Total |
|---|---|---|---|
| Hispanic or Latino | — | — | — |
| Not Hispanic or Latino | — | — | — |
| Unknown or Not Reported | — | — | — |
| Race (NIH/OMB) | Placebo - Lactose | Simvastatin | Total |
|---|---|---|---|
| American Indian or Alaska Native | — | — | — |
| Asian | — | — | — |
| Native Hawaiian or Other Pacific Islander | — | — | — |
| Black or African American | — | — | — |
| White | — | — | — |
| More than one race | — | — | — |
| Unknown or Not Reported | — | — | — |
| Region of Enrollment(participants) | Placebo - Lactose | Simvastatin | Total |
|---|
The record gives a start of Sep 2011, a primary completion of Oct 2014 and a completion of Oct 2014. It does not say whether these dates are actual or estimated.
Results were first posted in Oct 2019, 5 years after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Sep 2019. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Simvastatin
Smith-Lemli-Opitz Syndrome
Oregon Health and Science University →