A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
This study has been completed.
First Posted: April 25, 2013
Last Update Posted: November 7, 2017
Information provided by (Responsible Party):
No Study Results Posted on ClinicalTrials.gov for this Study
|Study Status:||This study has been completed.|
|Study Completion Date:||August 21, 2017|
|Primary Completion Date:||August 21, 2017 (Final data collection date for primary outcome measure)|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E, Norris DA, Xia S, Bennett CF, Bishop KM. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016 Dec 17;388(10063):3017-3026. doi: 10.1016/S0140-6736(16)31408-8. Epub 2016 Dec 7.