Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

The primary objective of the study is to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA).

The secondary objectives of the study are to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA; to examine the effect of nusinersen administered at higher doses compared to the currently approved dose in participants with SMA; and to examine the pharmacokinetic(s) (PK) of nusinersen [cerebrospinal fluid (CSF) and plasma] after intrathecal administration of nusinersen given at higher doses to participants with SMA.


Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 125 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Care Provider)
Primary Purpose: Treatment
Official Title: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
Estimated Study Start Date: February 26, 2020
Estimated Primary Completion Date: June 13, 2022
Estimated Study Completion Date: December 18, 2022

Arms:
- Experimental: 28/28 Milligram (mg) Safety Group
- Active Comparator: 12/12 mg Randomized Control Group
- Experimental: 50/28 mg Randomized Treatment Group
- Experimental: 12/50/28 mg Titration Group