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A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy — ENDEAR  

Spinal Muscular Atrophy Clinical Trial

Official title:
A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy

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

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