An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
ConditionPitt Hopkins Syndrome
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age3-21
SponsorUnravel Biosciences, Inc.
About this trial
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 criteria
Qualifiers
Subjects ≥3 years of age and ≤ 21 years of age at time of screening
Clinical diagnosis of PTHS with documented pathologic mutation in the TCF4 gene
At time of screening, is in a post-regression phase with no degradation of ambulation, hand function, speech or communication skills in the 4 months prior to screening
Has been on a stable regimen of medication or non-pharmacological treatment for at least 4 weeks prior to the baseline visit
Disqualifiers
Has another clinically significant medical condition other than those related to their TCF4 mutation (e.g. diabetes mellitus, cardiovascular disease, renal disease, respiratory disease, hematological abnormalities, malignancy)
Has major surgery planned during the study period
Pregnant or nursing women
Has a history of brain injury, stroke, other cerebrovascular disease or hypoxic-ischemic encephalopathy
Trial design
Treatments tested in this trial
- Vorinostat (SAHA)
- Placebo
Treatment groups
5 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov