First human trial tests potential drug for rare genetic disorder
NCT ID NCT07150026
Summary
This is a very early, small study to see if a drug called vorinostat is safe and might help people with Pitt Hopkins Syndrome, a rare genetic disorder. Up to 5 children and young adults will take the drug at two different doses, with periods of placebo for comparison, to check for side effects and early signs of benefit. The main goal is to gather information to design larger future studies.
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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)
Medellín, Colombia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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