Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Phase of Trial: Phase III
Latest Information Update: 19 Jan 2018
At a glance
- Drugs AVXS 101 (Primary)
- Indications Spinal muscular atrophy
- Focus Registrational; Therapeutic Use
- Acronyms STR1VE
- Sponsors AveXis
- 16 Jan 2018 According to an AveXis media release, 3 patients have been dosed in this trial.
- 04 Oct 2017 Status changed from planning to recruiting.
- 29 Sep 2017 According to an AveXis media release, the trial is projected to be conducted at 16 sites in the United States.