Clinical Trial: Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

U.S., Aug. 22 -- ClinicalTrials.gov registry received information related to the study (NCT07135050) titled 'Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome' on Aug. 04.

Brief Summary: 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 periodic...