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18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

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Trial Profile

18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 11 Feb 2026

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At a glance

  • Drugs Nizubaglustat (Primary)
  • Indications GM1 gangliosidosis; GM2 gangliosidoses; Niemann-Pick disease type C
  • Focus Registrational; Therapeutic Use
  • Acronyms NAVIGATE
  • Sponsors Azafaros

Most Recent Events

  • 22 Jan 2026 According to an Azafaros media release, According to an Azafaros media release, company will host a satellite symposium at the WORLDSymposium™ 2026 taking place in San Diego, California, USA between February 2-6.
  • 08 Jan 2026 According to an Azafaros media release, Phase 3 studies are financed following the company's successful completion of Euro 132 million series B round completed in 1H 2025.
  • 08 Jan 2026 According to an Azafaros media release, data expected in 2027.

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