Ignite Creation Date:
2024-05-06 @ 12:45 PM
Last Modification Date:
2024-10-26 @ 1:03 PM
Study NCT ID:
NCT03837184
Status:
COMPLETED
Last Update Posted:
2022-11-22
First Post:
2019-02-08
Brief Title:
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Sponsor:
Organization:
Novartis
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:
2
Enrollment Type:
ACTUAL
Design Primary Purpose:
Design Masking:
Phases:
| Name |
|---|
| PHASE3 |
Observational Models:
Time Perspective List:
Who Masked List: