Could a cancer drug help kids with rare brain disorder?
NCT ID NCT07150026
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing whether a drug called vorinostat is safe and helpful for people with Pitt Hopkins syndrome, a rare genetic condition that affects development. Five participants will each receive a placebo and two different doses of the drug in a special 'N-of-1' design, meaning each person acts as their own comparison. The goal is to see if the drug improves symptoms and to gather data for future larger studies.
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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
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Grupo de Investigación Clínica PECET (GIC-PECET)
RECRUITINGMedellín, Colombia
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