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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

NCT07135050 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-11

No results posted yet for this study

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 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

  • Pitt Hopkins Syndrome

Interventions

GENETIC

MZ-1866

AAV-9 gene therapy delivered by intracerebroventricular injection

Sponsors & Collaborators

  • California Institute for Regenerative Medicine (CIRM)

    collaborator OTHER

  • Mahzi Therapeutics

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-12-19
Primary Completion
2029-03-31
Completion
2029-07-31
FDA Drug
Yes

Countries

  • United States
  • Israel

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07135050 on ClinicalTrials.gov