Ignite Creation Date:
2025-12-24 @ 10:30 PM
Ignite Modification Date:
2025-12-25 @ 8:03 PM
Study NCT ID:
NCT04798235
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-05-09
First Post:
2021-02-22
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
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: