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

  • Grupo de Investigación Clínica PECET (GIC-PECET)

    RECRUITING

    Medellín, Colombia

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