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Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

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Trial Profile

Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 20 Feb 2026

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At a glance

  • Drugs Nizubaglustat (Primary)
  • Indications GM2 gangliosidoses; Niemann-Pick disease type C
  • Focus Adverse reactions; Pharmacokinetics; Therapeutic Use
  • Acronyms PRISMA
  • Sponsors Azafaros

Most Recent Events

  • 17 Feb 2026 Status changed from not yet recruiting to recruiting.
  • 16 Feb 2026 New trial record

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