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A Multicenter, Randomized, Open-Label, Standard-of-Care-Controlled, Phase III Clinical Trial to Evaluate the Safety and Efficacy of Intrathecal (IT) Injection of GC101 Adeno-Associated Virus Injection in the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA)

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Trial Profile

A Multicenter, Randomized, Open-Label, Standard-of-Care-Controlled, Phase III Clinical Trial to Evaluate the Safety and Efficacy of Intrathecal (IT) Injection of GC101 Adeno-Associated Virus Injection in the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA)

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 12 Jun 2025

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At a glance

  • Drugs GC 101 (Primary) ; Nusinersen
  • Indications Spinal muscular atrophy
  • Focus Registrational; Therapeutic Use
  • Sponsors GeneCradle Therapeutics

Most Recent Events

  • 22 May 2025 New trial record

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