RecruitingPhase 1NCT07150026

An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome

An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome Using an "N of 1" Study Design


Sponsor

Unravel Biosciences, Inc.

Enrollment

5 participants

Start Date

Mar 15, 2026

Study Type

INTERVENTIONAL

Conditions

Summary

The study is an exploratory evaluation of the safety and efficacy of vorinostat in Pitt Hopkins syndrome. Each patient will be self-controlled in an adapted N-of-1 study design methodology with three treatment arms, including a 4-week placebo phase and two vorinostat dose arms, including every 8 weeks of daily dosing at a low dose of 80mg/m2/day and 8 weeks of a higher dose at 160mg/m2/day. Key objectives of the study include: * To confirm the safety and tolerability of oral vorinostat 80mg/m2/day and 160mg/ m2/day dose levels when administered to PTHS patients * To identify the nature and magnitude of treatment response to vorinostat, as measured by changes in clinical and laboratory parameters indicative of trend towards benefit, as well as changes in mRNA expression (transcriptome response) * Provide a data-driven justification for future study design and statistical analysis plan for subsequent clinical studies assessing safety and efficacy of vorinostat in PTHS


Eligibility

Min Age: 3 YearsMax Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called Vorinostat (SAHA) for people with pitt hopkins syndrome. The study is currently recruiting participants at 1 location.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGVorinostat (SAHA)

oral suspension

DRUGPlacebo

placebo


Locations(1)

Grupo de Investigación Clínica PECET (GIC-PECET)

Medellín, Colombia

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NCT07150026


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