Could a cancer drug ease rett syndrome? early trial launches
NCT ID NCT07150013
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial is testing the drug vorinostat in 15 girls and young women with Rett syndrome, a rare genetic disorder that affects brain development. Each participant will receive placebo and two different doses of vorinostat, acting as their own control. The study aims to check safety, find the best dose, and see if the drug changes symptoms or gene activity patterns linked to the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
vorinostat (also known as SAHA)
What this could lead to
If it works, this could point toward a treatment that improves symptoms or slows progression of Rett syndrome.
What could go wrong
This is a very early Phase 1 trial with only 15 participants. It is designed mainly to test safety and find the right dose, not to prove effectiveness. Many early-stage drugs do not succeed in later trials.
Disclaimer
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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.
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: •••••@•••••