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

NCT07150026 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-05-01

No results posted yet for this study

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

Conditions

  • Pitt Hopkins Syndrome

Interventions

DRUG

Vorinostat (SAHA)

oral suspension

DRUG

Placebo

placebo

Sponsors & Collaborators

  • Unravel Biosciences, Inc.

    lead INDUSTRY

Principal Investigators

  • Neal I Muni, M.D., MSPH · Unravel Biosciences, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
CROSSOVER

Eligibility

Min Age
3 Years
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-15
Primary Completion
2026-12-31
Completion
2027-03-15

Countries

  • Colombia

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07150026 on ClinicalTrials.gov