Conditions: Spinal Muscular Atrophy
Keywords: Spinal Muscular Atrophy SMA SMN SMNRx ISIS-SMNRx ISIS 396443
Allocation: NON_RANDOMIZED | Intervention model: PARALLEL
Enrollment: 34
Healthy volunteers: no

Brief summary:
This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA).

Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of approximately 8 participants, where all participants will receive active drug.

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: 3 mg nusinersen on Days 1, 29, 85, intrathecal (IT) injection arm 2: 6 mg nusinersen on Days 1, 29, 85, IT injection arm 3: 9 mg nusinersen on Days 1 and 85, IT injection arm 4: 12 mg nusinersen on Days 1, 29, 85, IT injection

Interventions: intervention 1: Nusinersen
Intervention descriptions: intervention 1: Single IT injection for each dose

Protocol document: