Could a cancer drug ease rett syndrome? early trial launches

NCT ID NCT07150013

Recruiting now Disease control Sponsor: Unravel Biosciences, Inc. Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Rett syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

RETT SYNDROME

Contacts and locations

Locations

Grupo de Investigación Clínica PECET (GIC-PECET)
RECRUITING

Medellín, Colombia

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