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

Spinal Muscular Atrophy Clinical Trial

Official title:
An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy

Clinical Trial Summary

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.

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 NCT01703988
Study type Interventional
Source Biogen
Contact
Status Completed
Phase Phase 1/Phase 2
Start date October 31, 2012
Completion date January 31, 2015
View Details
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