Pitt-Hopkins or Pitt-Hopkins-like syndrome
MONDO:09807323 clinical trials for this condition and its sub-types, 0 tagged with Pitt-Hopkins or Pitt-Hopkins-like syndrome itself.
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Sub-types of Pitt-Hopkins or Pitt-Hopkins-like syndrome
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Pitt-Hopkins syndrome 3 trials
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Pitt-Hopkins-like syndrome 0 trials
2 sub-types
- Pitt-Hopkins-like syndrome 2 0 trials
- Cortical dysplasia-focal epilepsy syndrome 0 trials
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Could a cancer drug help kids with rare brain disorder?
Disease control Recruiting nowThis study is testing whether a drug called vorinostat is safe and helpful for people with Pitt Hopkins syndrome, a rare genetic condition that affects development. Five participants will each receive a placebo and two different doses of the drug in a special 'N-of-1' design, mea…
Phase 1 • Sponsor: Unravel Biosciences, Inc. • Aim: Disease control
Last updated Sep 19, 2026 00:00 UTC
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Gene therapy breakthrough? first human trial launches for rare brain disorder
Disease control Recruiting nowThis study tests a new gene therapy called MZ-1866 for children and adults with Pitt Hopkins Syndrome, a rare genetic condition that affects development and breathing. The therapy is given as a single injection into the fluid around the brain. The main goal is to see if it is saf…
Phase 1/2 • Sponsor: Mahzi Therapeutics • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC