Can a common drug boost brain function in kids with rare genetic disorder?
NCT ID NCT01619644
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tested sodium valproate, a drug used for seizures, in 41 children with Rubinstein-Taybi syndrome (RTS), a rare genetic condition causing intellectual disability. Participants aged 6 to 21 received either the drug or a placebo for one year. Researchers measured memory and motor skills using tests and brain scans to see if the drug could improve learning and coordination.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- sodium valproate
- What this could lead to
- If successful, this could point toward a treatment to improve memory and motor skills in children with Rubinstein-Taybi syndrome.
- What could go wrong
- This is a small, early-phase trial with only 41 participants, so results may not apply to everyone. Sodium valproate can cause side effects like drowsiness or liver issues.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
41 people
The number who actually took part.
- Start date
-
Apr 2012
- Finished
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Sep 2014
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
6 to 21 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Children over 6 and under 21 * RTS confirmed by a genetic study with a CBP gene or EP300 gene mutation * Sufficient cognitive capacities for neuropsychological evaluation * Free and informed consent of the parents or guardians * Children affiliated to or benefiting of the French social welfare system Exclusion Criteria: * Contraindication to sodium valproate * Women of reproductive age without effective contraception means * Case history of sodium valproate treatment * Monotherapy treatment for epilepsy with Lamictal with a dosage superior to 5 mg/kg/j * Family history of severe hepatitis including drug * Acute or chronic hepatitis * Pregnancy
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
University Hospital Bordeaux, France
Bordeaux, 33076, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.