Randomised controlled trial of simvastatin treatment for autism in young children with neurofibromatosis type 1 (SANTA).

ABSTRACT:

Background

Neurofibromatosis 1 (NF1) is a monogenic model for syndromic autism. Statins rescue the social and cognitive phenotype in animal knockout models, but translational trials with subjects > 8 years using cognition/behaviour outcomes have shown mixed results. This trial breaks new ground by studying statin effects for the first time in younger children with NF1 and co-morbid autism and by using multiparametric imaging outcomes.

Methods

A single-site triple-blind RCT of simvastatin vs. placebo was done. Assessment (baseline and 12-week endpoint) included peripheral MAPK assay, awake magnetic resonance imaging spectroscopy (MRS; GABA and glutamate+glutamine (Glx)), arterial spin labelling (ASL), apparent diffusion coefficient (ADC), resting state functional MRI, and aut

SUBMITTER: Stivaros S 

PROVIDER: S-EPMC5824534 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature