A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
First received: April 22, 2013
Last updated: July 28, 2017
Last verified: July 2017
No Study Results Posted on ClinicalTrials.gov for this Study
|Study Status:||This study is ongoing, but not recruiting participants.|
|Estimated Study Completion Date:||August 31, 2017|
|Estimated Primary Completion Date:||August 31, 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.