Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
Status:
Recruiting
Trial end date:
2023-07-04
Target enrollment:
Participant gender:
Summary
The primary objectives of this study are to examine the clinical efficacy of nusinersen
administered intrathecally at higher doses to participants with spinal muscular atrophy
(SMA), as measured by change in Children's Hospital of Philadelphia Infant Test of
Neuromuscular Disorders (CHOP INTEND) total score (Part B); to examine the safety and
tolerability of nusinersen administered intrathecally at higher doses to participants with
SMA (Parts A and C).
The secondary objectives of this study are to examine the clinical efficacy of nusinersen
administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to
examine the effect of nusinersen administered intrathecally at higher doses to participants
with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered
intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen
administered intrathecally at higher doses compared to the currently approved dose in
participants with SMA (Part B).