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A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory

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Trial Profile

A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 11 Mar 2026

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At a glance

  • Drugs Onasemnogene abeparvovec (Primary) ; Prednisolone sodium phosphate
  • Indications Spinal muscular atrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms STEER
  • Sponsors Novartis Gene Therapies; Novartis Pharma A.G.; Novartis Pharmaceuticals

Most Recent Events

  • 01 Feb 2026 Results published in the Nature Medicine
  • 24 Nov 2025 According to Novartis Media release, based on data from the registrational Phase lll STEER study and supported by the open-label Phase lllb STRENGTH study, US Food and Drug Administration (FDA) has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy (SMA).
  • 04 Jul 2025 Status changed from active, no longer recruiting to completed.

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