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Clinical Trial Summary

The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA). The secondary objective of the study is to examine the safety and tolerability of nusinersen administered intrathecally to participants with infantile-onset SMA.


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02193074
Study type Interventional
Source Biogen
Contact
Status Terminated
Phase Phase 3
Start date August 19, 2014
Completion date November 21, 2016

See also
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