PITT HOPKINS SYNDROME

Clinical trials for PITT HOPKINS SYNDROME explained in plain language.

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Trials to join now! 1 Not yet recruiting 1

Brain-Injected gene therapy trial offers hope for rare genetic disorder

Disease control Recruiting now

This early-stage study is testing whether a new gene therapy called MZ-1866 is safe for people with Pitt Hopkins Syndrome, a rare genetic disorder that affects brain development. Twelve children and adults will receive a single injection of the therapy directly into the fluid aro…

Matched conditions: PITT HOPKINS SYNDROME

Phase: PHASE1, PHASE2 • Sponsor: Mahzi Therapeutics • Aim: Disease control

Last updated Mar 27, 2026 12:38 UTC

PITT HOPKINS SYNDROME

Get alerted when new PITT HOPKINS SYNDROME trials appear

Brain-Injected gene therapy trial offers hope for rare genetic disorder