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Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)

Trial Profile

Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 09 Jun 2025

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At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Registrational
  • Acronyms STRENGTH
  • Sponsors Novartis Pharmaceuticals

Most Recent Events

  • 19 Mar 2025 According to Novartis Media release, the data from this study presented during the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference held in Dallas, Texas, from March 16 to 19, 2025.
  • 19 Mar 2025 Results published in the Novartis Media Release
  • 10 Jan 2025 Status changed from active, no longer recruiting to completed.

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