NCT07135050

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

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Mahzi Therapeutics · Industry
• Sex: All
• Age: 2 Years – 25 Years
• Healthy volunteers: Not accepted

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 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

Conditions

• Pitt Hopkins Syndrome

Interventions

Type	Name	Description
GENETIC	MZ-1866	AAV-9 gene therapy delivered by intracerebroventricular injection

Timeline

• Start date: 2025-12-19
• Primary completion: 2029-03-01
• Completion: 2029-07-01
• First posted: 2025-08-21
• Last updated: 2026-02-23

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study