SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

NA

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-09-30

Study Completion Date

2014-10-31

Brief Summary

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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.

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Detailed Description

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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

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Smith-Lemli-Opitz Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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Placebo

placebo

Group Type PLACEBO_COMPARATOR

Lactose

Intervention Type DIETARY_SUPPLEMENT

Lactose will be administered in a capsule formula.

Simvastatin

0.5 mg/kg body weight/day

Group Type EXPERIMENTAL

Simvastatin

Intervention Type DRUG

Simvastatin will be administered as a powder mixed with lactose at the dose of 0.5 mg/kg/day

Interventions

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Simvastatin

Simvastatin will be administered as a powder mixed with lactose at the dose of 0.5 mg/kg/day

Intervention Type DRUG

Lactose

Lactose will be administered in a capsule formula.

Intervention Type DIETARY_SUPPLEMENT

Other Intervention Names

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Zocor, Flolipid

Eligibility Criteria

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Inclusion Criteria

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