Apply to trial NCT07135050

A few quick questions so the study team can decide if you might be a fit.

RecruitingGenetic intervention

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

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

How this works

  1. Answer a few questions

    About 5 to 10 minutes. Skip-friendly where possible.

  2. We forward your profile to the study team

    They see only the answers needed to decide if you can be screened.

  3. The team reaches out to schedule screening

    Usually within a few business days, via the contact you give.

We save your progress under this email so you can come back later, and the study team uses it to reach you.

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