A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. (SHINE)

The primary objective is to evaluate the long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.


Study Type: Interventional  (Clinical Trial)
Actual Enrollment: 292 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Masking Description:
During the blinded loading period, the following participants will be masked:
- Key site personnel (Investigator, Study Coordinator, and Outcomes Assessors)
- Participant
- The sponsor
After the loading period has been completed. Subsequent doses will be unblinded.
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443
Actual Study Start Date: November 4, 2015
Estimated Primary Completion Date: August 29, 2023
Estimated Study Completion Date: August 29, 2023

Arms:
- Experimental: Group 1
- Experimental: Group 2
- Experimental: Group 3
- Experimental: Group 4
- Experimental: Group 5