Actively Recruiting
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
Led by Unravel Biosciences, Inc. · Updated on 2026-05-01
5
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subjects aged 3 to 21 years at screening
- Clinical diagnosis of Pitt Hopkins syndrome with documented TCF4 gene mutation
- No worsening of ambulation, hand function, speech, or communication skills in the 4 months before screening
- Stable medication or non-drug treatment for at least 4 weeks before baseline visit
- Stable seizure activity pattern for 4 weeks before screening
- Ability to swallow medication or receive it by gastrostomy tube
- Ability to wear an actigraphy data logging device on wrist or ankle
- If able to have children, agrees to use highly effective contraception during the study and for 3 months after last study drug dose
- Subject or legally authorized representative able to provide informed consent and complete caregiver assessments in study language
You will not qualify if you...
- Having other significant medical conditions unrelated to TCF4 mutation (e.g., diabetes, cardiovascular, renal, respiratory, blood disorders, cancer)
- Planning major surgery during study period
- Pregnant or nursing women
- History of brain injury, stroke, cerebrovascular disease, or hypoxic-ischemic encephalopathy
- Clinically significant abnormal vital signs at screening or baseline
- Abnormal ECG at screening including significant QT prolongation
- Clinically significant abnormal lab values at screening
- Liver disease or liver enzymes above 1.5 times normal limit at screening
- History of cancer within past 5 years before screening
- Participation in any clinical trial within 30 days before screening
- Treatment with growth hormone, IGF-1, or insulin within 12 weeks before baseline
- Use of anticoagulant therapy or other HDAC inhibitors
- Medication or treatment changes within 4 weeks before baseline
- Life expectancy less than 12 months
- History of alcoholism or drug/chemical abuse within 2 years before screening
- Considered unsuitable for the study by the investigator for any reason
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Grupo de Investigación Clínica PECET (GIC-PECET)
Medellín, Colombia
Actively Recruiting
Research Team
N
Neal I Muni, M.D., MSPH
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
NON_RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
3
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