Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy

Trial Profile

Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy

Recruiting
Phase of Trial: Phase I

Latest Information Update: 29 Dec 2017

At a glance

  • Drugs AVXS 101 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Proof of concept; Therapeutic Use
  • Acronyms STRONG
  • Sponsors AveXis
  • Most Recent Events

    • 18 Dec 2017 Status changed from planning to recruiting.
    • 13 Dec 2017 According to an AveXis media release, the US FDA has notified the company that, based on review of data submitted, the company may initiate this trial of AVXS-101 for patients with SMA Type 2 via the intrathecal (IT) route of administration, using material produced by the company's Good Manufacturing Practice (GMP) commercial manufacturing process at the AveXis manufacturing facility.
    • 13 Dec 2017 According to an AveXis media release, this trial is projected to be conducted at 11 sites in the United States, including Stanford University Medical Center, University of Central Florida College of Medicine, University of Texas Southwestern Medical Center, University of Utah and Washington University School of Medicine.
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