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

StatusRecruiting
PhasePhase 1/2
Started
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Record History

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  1. Site details revised at 3 of 4 sitesv7
  2. Study sites: 3 to 4
    • Collaborators expanded, about 6 words
    v6
  3. Study sites: 2 to 3v5
  4. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Jan 2026 to Dec 19, 2025
    • Site details revised at 2 of 2 sites
    v4
  5. Study sites: 1 to 2v3
  6. Study sites: 0 to 1v2
  7. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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
Trial Details
NCT Number NCT07135050
Lead Sponsor Mahzi Therapeutics
Collaborators: California Institute for Regenerative Medicine (CIRM)
Conditions
(from sponsor)		 Pitt Hopkins Syndrome
Related terms Pitt-Hopkins syndrome
Enrollment 12 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov