A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
Status:
Terminated
Trial end date:
2016-11-21
Target enrollment:
Participant gender:
Summary
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS
396443) administered intrathecally (IT) to participants with infantile-onset with
infantile-onset spinal muscular atrophy (SMA). The secondary objective of the study is to
examine the safety and tolerability of nusinersen administered intrathecally to participants
with infantile-onset SMA.