Ignite Creation Date:
2025-12-24 @ 4:50 PM
Ignite Modification Date:
2026-01-02 @ 10:39 AM
Study NCT ID:
NCT07135050
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-12
First Post:
2025-08-04
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Sponsor:
Mahzi Therapeutics
Organization:
Study Overview
Official Title:
Phase 1/2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: