MedTrace Logo

Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial

NCT01619644 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2026-05-14

No results posted yet for this study

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).

Conditions

  • Rubinstein-Taybi Syndrome

Interventions

DRUG

sodium valproate

sodium valproate (HDAC inhibitor) with an oral dosage of 30 mg/kg/jour

DRUG

Placebo

Placebo with an oral dosage of 30 mg/kg/jour

Sponsors & Collaborators

  • Fondation Syndrome de Rubinstein-Taybi

    collaborator UNKNOWN

  • University Hospital, Bordeaux

    lead OTHER

Principal Investigators

  • Didier LACOMBE, PU-PH · University Hospital Bordeaux, France

  • Paul PEREZ, PH · University Hospital Bordeaux, France

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-04-30
Primary Completion
2014-09-30
Completion
2014-09-30

Countries

  • France

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01619644 on ClinicalTrials.gov