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A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2

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Trial Profile

A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 15 Sep 2023

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At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Acronyms SPR1NT
  • Sponsors Novartis Gene Therapies; Novartis Pharmaceuticals

    • Most Recent Events

    • 01 Aug 2023 Results of a post-hoc analysis assessing bulbar function of infants at risk for spinal muscular atrophy who received one-time gene replacement therapy (onasemnogene abeparvovec) before symptom onset published in the Neuromuscular Disorders
    • 14 Jul 2022 This trial has been discontinued in Netherlands according to European Clinical Trials Database record.
    • 01 Jul 2022 Results published in the Nature Medicine

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