Status:
NOT_YET_RECRUITING
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Lead Sponsor:
Mahzi Therapeutics
Conditions:
Pitt Hopkins Syndrome
Eligibility:
All Genders
2-25 years
Phase:
PHASE1
PHASE2
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 e...
Eligibility Criteria
Inclusion
- The participant has a TCF4 gene mutation confirmed as "pathogenic" or "likely pathogenic" by whole exome sequencing, whole genome sequencing, gene panel, single gene testing, or microarray, performed at an accredited laboratory
- Clinical phenotype consistent with Pitt Hopkins Syndrome, in the opinion of the investigator
- The participant, or the participant's parent or legal guardian, is willing to provide access to prior medical records for the collection of demographics and diagnostic and treatment history
Exclusion
- A deletion that includes the TCF4 gene that is over 12 Mbp in size
- Another genetic mutation or clinical comorbidity not associated with Pitt Hopkins Syndrome that could potentially confound interpretation of the study data
- A central nervous system structural or vascular abnormality that is a contraindication to the ICV administration procedure, including but not limited to: signs or symptoms of increased intracranial pressure, history of a space-occupying lesion, or presence of a ventricular shunt that would preclude ICV procedures or safety assessments, or increase risk to the participant
- Not able to receive prophylactic corticosteroids due to a medical contraindication or participant has a history of a condition that could worsen with corticosteroid therapy as assessed and determined by the Investigator
- Not able to undergo MRI procedures
- Cannot be anesthetized for the ICV injection
Key Trial Info
Start Date :
January 1 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 1 2029
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT07135050
Start Date
January 1 2026
End Date
July 1 2029
Last Update
December 12 2025
Active Locations (2)
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1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
2
Rush University Medical Center
Chicago, Illinois, United States, 60612