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Clinical Trials associated with MZ-1866 / Not yet recruitingPhase 1/2 Phase 1/2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
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
100 Clinical Results associated with MZ-1866
100 Translational Medicine associated with MZ-1866
100 Patents (Medical) associated with MZ-1866
100 Deals associated with MZ-1866