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

Sponsor
Unravel Biosciences, Inc.
Study ID
NCT07150026
Phase
PHASE1
Status
Recruiting
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Conditions

  • Pitt Hopkins Syndrome

Eligibility Criteria

Sex
ALL
Age
3 Years - 21 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

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

Key Dates

Start date
Mar 15, 2026
Status verified
Apr 2026
Primary completion
Dec 31, 2026
Completion
Mar 15, 2027

Study Design

Enrollment
5 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: Low dose interventional arm
    vorinostat low dose 80mg/m2/day
  • Experimental: High dose interventional arm
    vorinostat 160mg/m2/day dose
  • Placebo Comparator: Placebo
    Placebo

Primary Outcome Measure

Frequency of treatment-related adverse events [ Time Frame: 20 weeks ]

Central Contacts

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