Can a common drug boost brain function in kids with rare genetic disorder?
NCT ID NCT01619644
First seen May 16, 2026 · Last updated Jun 19, 2026 · Updated 8 times
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.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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University Hospital Bordeaux, France
Bordeaux, 33076, France
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.
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.