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: 05 Mar 2018
At a glance
- Drugs AVXS 101 (Primary)
- Indications Spinal muscular atrophy
- Focus Registrational; Therapeutic Use
- Acronyms STR1VE
- Sponsors AveXis
- 27 Feb 2018 According to an AveXis media release, five patients have been dosed till date.
- 30 Jan 2018 According to an AveXis media release, following review of safety data and early signals of efficacy from the first three patients dosed in this trial, company will initiate screening for the remaining patients in the trial as per the protocol.There was at least a four-week interval scheduled between dosing the first three patients to allow review of the safety analysis as well as early signals of efficacy.
- 16 Jan 2018 According to an AveXis media release, 3 patients have been dosed in this trial.