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

Completed · Phase 1

Conditions studied: Spinal Muscular Atrophy

In brief

This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).

Key facts

Study ID

NCT01494701

Run by

Biogen

People needed

28

Starts

2011-11-30

Expected to finish

2013-01-31

Last updated by the study team

2021-02-18

Who can join

Age: 2 and older, up to 14. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Children's Hospital Boston — 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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