Ignite Creation Date:
2024-05-06 @ 7:01 PM
Last Modification Date:
2024-10-26 @ 2:58 PM
Study NCT ID:
NCT05861986
Status:
RECRUITING
Last Update Posted:
2024-07-05
First Post:
2023-05-08
Brief Title:
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Sponsor:
Hoffmann-La Roche
Organization:
Hoffmann-La Roche
Study Overview
Official Title:
A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HINALEA 1
Brief Summary:
This is an open-label single-arm multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy SMA and 2 SMN2 copies who have previously received onasemnogene abeparvovec Participants are children 2 years of age genetically diagnosed with SMA
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2023-504508-26-00 | REGISTRY | EU CT number | None |