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Clinical trial

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

NCT07135050 is registered in ClinicalTrials.gov with Mahzi Therapeutics as lead sponsor.

NCT NCT07135050
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Mahzi Therapeutics
Phase
Phase 1/2
Overall status
Recruiting
Start date
December 19, 2025
Completion date
July 31, 2029
Enrollment
12 (estimated)
Sites
4

Conditions

As the registry lists them; a condition with its own page is linked.

pitt hopkins syndrome