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.
Phase of Trial: Phase I
Latest Information Update: 14 Apr 2017
At a glance
- Drugs Nusinersen (Primary)
- Indications Spinal muscular atrophy
- Focus Adverse reactions; Pharmacokinetics
- Sponsors Ionis Pharmaceuticals
- 20 Mar 2013 Status changed from active, no longer recruiting to completed, based on results reported in an Isis Pharmaceuticals media release.
- 14 Mar 2013 Phase I data was presented at the 2013 American Academy of Neurology Annual Meeting (AAN), according to an Isis Pharmaceuticals media release.
- 01 Nov 2012 This trial was ongoing at the end of October 2012, according to an Isis Pharmaceuticals media release.