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

Completed · Phase 1/Phase 2

Conditions studied: Spinal Muscular Atrophy

In brief

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.

Key facts

Study ID

NCT01703988

Run by

Biogen

People needed

34

Starts

2012-10-31

Expected to finish

2015-01-31

Last updated by the study team

2021-04-13

Who can join

Age: 2 and older, up to 15. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Boston Children's Hospital — Boston, Massachusetts, United States
• Columbia University Medical Center — New York, New York, United States
• UT Southwestern Medical Center - Children's Medical Center Dallas — Dallas, Texas, United States
• University of Utah School of Medicine — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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