Testing vorinostat for Pitt Hopkins syndrome
Treatments studied
Part of Genetic & congenital clinical trials.
This trial tests whether vorinostat, a medication that may affect gene activity, is safe and helpful for children and young adults with Pitt Hopkins syndrome. Participants take the drug by mouth or feeding tube.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 3 to 21 years old at the start of the study
- Diagnosed with Pitt Hopkins syndrome and have a confirmed change in the TCF4 gene
- No worsening of movement, hand use, speech, or communication in the past 4 months
- Stable medications and seizure pattern for at least 4 weeks before the study begins
- Able to swallow pills or take medicine through a feeding tube
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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