Ignite Creation Date:
2025-12-25 @ 2:34 AM
Ignite Modification Date:
2026-02-20 @ 6:05 PM
Study NCT ID:
NCT03779334
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-11-26
First Post:
2018-12-17
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
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 | PHASE2 | View |
Observational Models:
Time Perspective List:
Who Masked List: