Conditions: Spinal Muscular Atrophy
Keywords: Spinal Muscular Atrophy SMA SMN SMNRx ISIS-SMNRx ISIS-SMN Rx ISIS 396443 IONIS-SMNRx IONIS-SMN Rx Spinraza ENDEAR
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 122
Healthy volunteers: no

Brief 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.

Detailed description:
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc..

In August 2016, sponsorship of the trial was transferred to Biogen.

Arms:
arm 1: None arm 2: None

Interventions: intervention 1: nusinersen intervention 2: Sham procedure
Intervention descriptions: intervention 1: Administered by intrathecal (IT) injection as specified in the treatment arm. intervention 2: Small needle prick on the lower back at the location where the IT injection is normally made

Protocol document: