Ignite Creation Date:
2025-12-24 @ 3:01 PM
Ignite Modification Date:
2026-01-21 @ 8:51 AM
Study NCT ID:
NCT02462759
Status:
TERMINATED
Last Update Posted:
2021-02-17
First Post:
2015-05-14
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
Sponsor:
Biogen
Organization:
Study Overview
Official Title:
A Phase 2, Randomized, Double-blind, Sham-procedure Controlled Study to Assess the Safety and Tolerability and Explore the Efficacy of ISIS 396443 (BIIB058) Administered Intrathecally in Subjects With Spinal Muscular Atrophy Who Are Not Eligible to Participate in the Clinical Studies ISIS 396443-CS3B or ISIS 396443-CS4
Status:
TERMINATED
Status Verified Date:
2021-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The study was terminated early to roll over participants to open label extension study NCT02594124.
Has Expanded Access:
True
If Expanded Access, NCT#:
NCT02865109
Has Expanded Access, NCT# Status:
NO_LONGER_AVAILABLE
Acronym:
EMBRACE
Brief Summary:
The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS 396443-CS4 (NCT02292537). The secondary objective of Part 1 of this study is to examine the pharmacokinetics (PK) of Nusinersen in participants with SMA. The primary objective of Part 2 of this study is to assess the long-term safety and tolerability of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments. The secondary objective of Part 2 of this study is to examine the PK of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments.
Detailed Description:
Part 2 is an Open Label extension phase.
Study Oversight
Has Oversight DMC:
True
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?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2014-003657-33 | EUDRACT_NUMBER | None | View |