Ignite Creation Date:
2025-12-25 @ 4:17 AM
Ignite Modification Date:
2025-12-26 @ 3:18 AM
Study NCT ID:
NCT07014020
Status:
RECRUITING
Last Update Posted:
2025-07-30
First Post:
2025-05-21
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)
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:
Phases:
Phases
| Phase Brief | Phase Text | View |
|---|---|---|
| None | NA | View |
Observational Models:
Time Perspective List:
Who Masked List: