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NCT07135050 · ClinicalTrials.gov registry record · Phase 1
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
A Phase 1 study of Pitt Hopkins Syndrome, sponsored by Mahzi Therapeutics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 3
- Study locations
NCT07135050 is a Phase 1 study of Pitt Hopkins Syndrome that is actively recruiting participants, run by Mahzi Therapeutics. The registered enrollment target is 12 participants. The trial reports 3 study locations across 3 states.
The verdict
NCT07135050, a Phase 1 study of Pitt Hopkins Syndrome, is actively recruiting participants, sponsored by Mahzi Therapeutics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 3
- Study locations
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 Studied
Interventions
- GENETIC MZ-1866
Study Locations (3)
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Colorado
- Children's Hospital Colorado - Aurora
Illinois
- Rush University Medical Center - Chicago
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2025-12-19 |
| Est. Completion | 2029-07 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07135050
The ClinicalTrials.gov registry entry for NCT07135050 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Mahzi Therapeutics, which has 1 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Pitt Hopkins Syndrome appearing as the primary indexed condition, and to 1 intervention - of which MZ-1866 is the first listed.
NCT07135050 reports 3 study locations spanning 3 distinct geographic areas - top geographies include California, Colorado, Illinois.
Frequently Asked Questions
What is clinical trial NCT07135050 about?
NCT07135050 is a clinical study titled "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 electr...
What is the current status of trial NCT07135050?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2025-12-19. Estimated completion is 2029-07.
What conditions does trial NCT07135050 study?
This clinical trial studies the following conditions: Pitt Hopkins Syndrome.
What interventions are being tested in trial NCT07135050?
The interventions under investigation include: MZ-1866 (GENETIC).
Who is sponsoring clinical trial NCT07135050?
This trial is sponsored by Mahzi Therapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07135050 being conducted?
This trial has 3 study locations across California, Colorado, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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