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Tests treatment safety and results for Pitt Hopkins Syndrome
Official title An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
ClinicalTrials.gov ID: NCT07150026
What this study is testing
What is Vorinostat (SAHA)?
Vorinostat (SAHA) is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for pitt hopkins syndrome.
Also referred to as suberoylanilide hydroxamic acid.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- You might receive a placebo (an inactive treatment) instead of the study drug. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 21
You may be able to join if
- people ≥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...
- Has been on a stable regimen of medication or non-pharmacological treatment for at least 4 weeks prior to the baseline visit
- Has had a stable pattern of seizure activity for 4 weeks before screening
You likely can't join if
- Has another clinically significant medical condition other than those related to their TCF4 mutation (e.g. diabetes mellitus, cardiovascular disease...
- 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
- Has clinically significant abnormal vital signs at screening or baseline
- Has an abnormal ECG at screening, including clinically significant QT prolongation
See the full eligibility criteria
- people ≥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
- Has had a stable pattern of seizure activity for 4 weeks before screening
- Can swallow medication or can take it by gastrostomy tube
- Can wear actigraphy data logging device on wrist or ankle
- If of childbearing potential, must agree to use a highly effective method of contraception during the study and for 3 months after the last study drug administration (i.e., abstinence from sexual activity, hormonal...
- people or their legally authorized representative must be able to provide an informed consent and have sufficient language skill to complete caregiver assessments in the language in which the study assessments are...
- 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...
- 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
- Has clinically significant abnormal vital signs at screening or baseline
- Has an abnormal ECG at screening, including clinically significant QT prolongation
- Has a clinically significant abnormal laboratory value at screening
- Liver disease or transaminase levels \> 1.5 times the upper limit of the normal range as determined during screening
- Has a history of malignancy of any organ system within the past 5 years before screening
- Is participating in or has participated in another clinical trial within 30 days prior to the screening visit
- Has been treated with growth hormone, IGF-1, or insulin within 12 weeks of baseline
- Is taking anticoagulant therapy or other HDAC inhibitors
- Has had any change to their medication or non-pharmacological treatment within 4 weeks prior to the baseline visit
- Life expectancy of less than 12 months.
- Has a history of alcoholism or drug/chemical abuse within 2 years before screening.
- In the investigator's opinion, is inappropriate for this study for any reason
The study team makes the final eligibility decision.
Where it's taking place
- Medellín, Colombia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Medellín, Colombia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.