Actively Recruiting
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Led by Mahzi Therapeutics · Updated on 2026-05-11
12
Participants Needed
4
Research Sites
184 weeks
Total Duration
On this page
Sponsors
M
Mahzi Therapeutics
Lead Sponsor
C
California Institute for Regenerative Medicine (CIRM)
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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
You will not qualify if you...
- 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Rush University Medical Center
Chicago, Illinois, United States, 60612
Not Yet Recruiting
4
Sheba Medical Center
Tel Litwinsky, Israel
Actively Recruiting
Research Team
E
Emily Radomile
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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