Condition
Pitt Hopkins Syndrome clinical trials and research
As of November 12, 2026, Find a Trial lists 3 Pitt Hopkins Syndrome clinical trials that are recruiting, at sites in 3 countries (most in United States (2), Colombia (1), Israel (1)). By phase: Phase 1 (1), Phase 1/2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 3Countries
Newest Pitt Hopkins Syndrome clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Testing vorinostat for safety and potential benefit in Pitt Hopkins syndrome | Phase 1 | Medellín | Recruiting | Sep 2, 2025 |
| MZ-1866 AAV9 gene therapy delivered into the brain ventricles for Pitt Hopkins syndrome | Phase 1/2 | Oakland, California and 3 other locations | Recruiting | Aug 21, 2025 |
| Registry for patients with rare diseases | — | Sioux Falls, South Dakota | Recruiting | Feb 15, 2013 |
All recruiting trials (3)
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Testing vorinostat for safety and potential benefit in Pitt Hopkins syndrome
This trial will try daily oral vorinostat at two dose levels and placebo to see if it is safe and helps children and young adults (ages 3–21) with Pitt Hopkins syndrome.
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MZ-1866 AAV9 gene therapy delivered into the brain ventricles for Pitt Hopkins syndrome
This will test a single intracerebroventricular dose of MZ-1866 AAV9 gene therapy in children and adults with Pitt Hopkins syndrome to see if it is safe and tolerable.
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Registry for patients with rare diseases
This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.
Related topics
Quick answers
How many Pitt Hopkins Syndrome clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 3 countries. The most sites are in United States (2 trials), Colombia (1 trial), Israel (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 1: 1, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.