An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
Completed · Phase 1
Conditions studied: Spinal Muscular Atrophy
In brief
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS1 (NCT02865109). The secondary objective was to examine the plasma pharmacokinetics of a single dose of ISIS 396443 administered intrathecally to participants with SMA who previously participated in ISIS 396443-CS1.
Key facts
- Study ID
- NCT01780246
- Run by
- Biogen
- People needed
- 18
- Starts
- 2013-01-31
- Expected to finish
- 2014-02-28
- Last updated by the study team
- 2021-02-16
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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