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Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Sponsor: University Hospital, Bordeaux
Summary
An exploratory phase 2 therapeutic trial in children from 6 to 21, RTS carriers, randomized to be treated either with sodium valproate with the usual pediatric dosage (30 mg/kg/j), or by placebo for one year. The investigator would like to include children because they could best profit from it due to their neuronal plasticity as CBP and EP300 take effect through neuronal and synaptic plasticity. The therapeutic effect of sodium valproate in RTS patients will be assessed thanks to a clinical approach (learning and memory neuropsychological evaluation, fine motor skills assessment by pointing), to a biological approach (histone acetylation functional tests), and to imaging (morphological and functional MRI).
Key Details
Gender
All
Age Range
6 Years - 21 Years
Study Type
INTERVENTIONAL
Enrollment
41
Start Date
2012-04
Completion Date
2014-09
Last Updated
2026-05-14
Healthy Volunteers
No
Conditions
Interventions
sodium valproate
sodium valproate (HDAC inhibitor) with an oral dosage of 30 mg/kg/jour
Placebo
Placebo with an oral dosage of 30 mg/kg/jour
Locations (1)
University Hospital Bordeaux, France
Bordeaux, France