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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: Recruiting Phase 1 study of Pitt Hopkins Syndrome, sponsored by Mahzi Therapeutics.
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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
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
Primary Outcome
Frequency and severity of adverse events of special interest
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 |
What NCT07135050 shows while recruiting
NCT07135050 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
Similar trials for Pitt Hopkins Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT07135050's enrollment target sits among peer trials
12 1853rd of 2000 higher than 90 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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