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

Spinal Muscular Atrophy Clinical Trial

Official title:
An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy

Clinical Trial Summary

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).

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

Clinical Trial Details
NCT number NCT01494701
Study type Interventional
Source Biogen
Contact
Status Completed
Phase Phase 1
Start date November 30, 2011
Completion date January 31, 2013
View Details
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