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

Trial Profile

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

Recruiting
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
  • Most Recent Events

    • 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.
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