PITT HOPKINS SYNDROME
Clinical trials for PITT HOPKINS SYNDROME explained in plain language.
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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…
Matched conditions: PITT HOPKINS SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Mahzi Therapeutics • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC
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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…
Matched conditions: PITT HOPKINS SYNDROME
Phase: PHASE1 • Sponsor: Unravel Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC