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: 03 May 2018

At a glance

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

    • 31 Aug 2018 Biomarkers information updated
    • 03 May 2018 According to an AveXis media release, Fourth Patient have been dosed to date.Three patients less than 60 months of age will be enrolled in Cohort 2 and, if safety is established according to the Data Safety Monitoring Board, an additional 21 patients will be enrolled in Cohort 2 until there are a total of 12 patients less than 24 months, and 12 patients at least 24 months but less than 60 months of age.
    • 27 Feb 2018 According to an AveXis media release, first patient was dosed in this study on January 16, 2018. Two patients have been dosed to date, with a four-week interval between the dosing of the first three patients for each dose cohort per the trial protocol.
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