Ignite Creation Date:
2024-05-06 @ 10:37 AM
Last Modification Date:
2024-10-26 @ 12:32 PM
Study NCT ID:
NCT03306277
Status:
COMPLETED
Last Update Posted:
2022-08-16
First Post:
2017-10-02
Brief Title:
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Sponsor:
Novartis Gene Therapies
Organization:
Novartis
Study Overview
Official Title:
Phase 3 Open-Label Single-Arm Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Status:
COMPLETED
Status Verified Date:
2022-08
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:
STR1VE
Brief Summary:
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy SMA Type 1 participants
Detailed Description:
Phase 3 open-label single-arm single-dose study of onasemnogene abeparvovec-xioi gene replacement therapy in participants with spinal muscular atrophy SMA Type 1 who meet enrollment criteria and are genetically defined by nonfunctional survival motor neuron 1 gene SMN1 with 1 or 2 copies of survival motor neuron 2 gene SMN2 Fifteen 15 participants 6 months 180 days of age at the time of gene replacement therapy Day 1 will be enrolled
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 |
|---|---|---|---|
| COAV101A12302 | OTHER | Novartis Pharmaceuticals | None |
| 2020-000095-38 | EUDRACT_NUMBER | None | None |