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

Geneva, Aug. 29 -- International Clinical Trials 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. 4.

Study Type: Interventional

Study Design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).

Primary Sponsor: Mahzi Therapeutics

Condition: Pitt Hopkins Syndrome

Intervention: Genetic: MZ-1866

Recruitment Status: Not recruiting

Phase: Phase 1/Phase 2

Date of First Enrollment: January 2026

Target Sample Size: 12

To know more, visit https://clinicaltrials.gov/ct2/show/NCT07135050 ...