Pitt-Hopkins Syndrome clinical trials at University of California Health

The goal of this clinical trial is to learn if MZ-1866 is a safe and tolerable treatment for children and adults with Pitt Hopkins Syndrome. To evaluate the safety of MZ-1866, the following will be evaluated: - frequency and severity of adverse events - physical exam, laboratory results and electrocardiogram findings Participants will: - receive a single dose of MZ-1866 by intracerebroventricular injection - be seen by the study physician and site staff periodically to assess changes to their health status - be periodically evaluated using neurodevelopmental tools Caregivers will: - be interviewed periodically about the health status and development of the participant - keep diaries and complete periodic questionnaires regarding participant symptoms