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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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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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Grupo de Investigación Clínica PECET (GIC-PECET)
RECRUITINGMedellín, Colombia
Contact Phone: •••-•••-•••• Email: •••••@•••••