Clinical trials
Pitt Hopkins Syndrome clinical trials
Below are recruiting pitt hopkins syndrome clinical trials, each written for real people, not researchers. We’re tracking 3 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07150026RecruitingPhase 1
Testing vorinostat for Pitt Hopkins syndrome
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.
MedellínAges 3–21 - NCT07135050RecruitingPhase 1/Phase 2
Gene therapy study for Pitt Hopkins syndrome
This trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins syndrome. The therapy is given as a single injection into the fluid around the brain to help replace the missing or faulty TCF4 gene.
Oakland, CaliforniaAges 2–25 - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age
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Common questions
- Are there clinical trials for pitt hopkins syndrome?
- Yes. Clin2 currently lists 3 recruiting pitt hopkins syndrome studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a pitt hopkins syndrome trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a pitt hopkins syndrome trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.