Ignite Creation Date:
2025-12-24 @ 4:50 PM
Ignite Modification Date:
2026-04-09 @ 12:13 PM
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:
Organization:
Study Design
Study Type:
INTERVENTIONAL
Expanded Access Type Individual:
None
Expanded Access Type Intermediate:
None
Expanded Access Type Treatment:
None
Patient Registry:
None
Target Duration:
None
Design Allocation:
Design Intervention Model:
Design Intervention Model Description:
Design Masking Description:
Bio Spec Retention:
None
Bio Spec Description:
None
Enrollment Count:
Enrollment Type:
Design Primary Purpose:
Design Masking:
Time Perspective List:
Who Masked List: