Overview

An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

Status:
Completed
Trial end date:
2015-01-31
Target enrollment:
Participant gender:
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Biogen
Ionis Pharmaceuticals, Inc.