Could a cancer drug help kids with rare pitt hopkins syndrome? small trial launches
NCT ID NCT07150026
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 32 times
Summary
This study tests whether vorinostat, a drug already used for cancer, is safe and helpful for people with Pitt Hopkins syndrome, a rare genetic condition that affects development. Five participants aged 3 to 21 will each receive placebo, a low dose, and a higher dose in a personalized 'N-of-1' design. 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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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: •••••@•••••
Conditions
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