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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: 07 Jul 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

  • 02 Jul 2026 According to a Novartis media release, based on data from STEER study and supportive Phase IIIb STRENGTH and Phase I/II STRONG studies, the European Commission (EC) approved onasemnogene abeparvovec (Itvisma) for the treatment of children two years and older, teens and adults living with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene.
  • 02 Jul 2026 Results presented in the Novartis media release.
  • 24 Apr 2026 Results presented in the Novartis media release.

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