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

Part of paid clinical trials in Oakland, California.

Sponsor
Mahzi Therapeutics
Study ID
NCT07135050
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Pitt Hopkins Syndrome

Eligibility Criteria

Sex
ALL
Age
2 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

  • MZ-1866 — GENETIC
    AAV-9 gene therapy delivered by intracerebroventricular injection

Study Details

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

Key Dates

Start date
Dec 19, 2025
Status verified
May 2026
Primary completion
Mar 31, 2029
Completion
Jul 31, 2029

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1 (MZ-1866)
    Participants ages 12-25 years
  • Experimental: Cohort 2 (MZ-1866)
    Participants aged 2-11 years

Primary Outcome Measure

To evaluate the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome [ Time Frame: Week 104 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609
Alex Fay, M.D., Ph.D. (PRINCIPAL_INVESTIGATOR)
Children's Hospital ColoradoAuroraColorado80045
Sky Gould
[email protected]
750-777-1583
Diana Walleigh, MD (PRINCIPAL_INVESTIGATOR)
Rush University Medical CenterChicagoIllinois60612
Tara Khandelwal
[email protected]
Elizabeth Berry-Kravis, M.D., Ph.D. (PRINCIPAL_INVESTIGATOR)

Find similar trials in Oakland, CA

By research site
UCSF Benioff Children's Hospital Oakland· Oakland, CAChildren's Hospital Colorado· Aurora, CORush University Medical Center· Chicago, IL

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