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: 16 Oct 2017

At a glance

  • Drugs AVXS 101 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Therapeutic Use
  • Acronyms STR1VE
  • Sponsors AveXis
  • Most Recent Events

    • 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.
    • 29 Sep 2017 According to an AveXis media release, U.S. Food and Drug Administration (FDA) has notified the company that based on review of data submitted, including the potency assay, it may initiate its planned pivotal trial of AVXS-101 for patients with spinal muscular atrophy (SMA) Type 1 using the intravenous (IV) formulation produced by the company's Good Manufacturing Practice (GMP) commercial manufacturing process. The company plans to initiate this trial immediately.
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