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Randomized, Double Blind, Placebo Controlled, Multicenter, 12 Weeks Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease

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Trial Profile

Randomized, Double Blind, Placebo Controlled, Multicenter, 12 Weeks Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 09 Feb 2026

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At a glance

  • Drugs Nizubaglustat (Primary)
  • Indications GM2 gangliosidoses; Niemann-Pick disease type C; Sandhoff disease; Tay-Sachs disease
  • Focus Adverse reactions; Pharmacokinetics
  • Acronyms RAINBOW
  • Sponsors Azafaros

Most Recent Events

  • 22 Jan 2026 According to an Azafaros media release, company will feature preliminary clinical efficacy and safety data of Phase II study with oral nizubaglustat at the WORLDSymposium™ 2026 taking place in San Diego, California, USA between February 2-6.
  • 20 Mar 2025 Planned End Date changed from 1 Dec 2024 to 1 Mar 2026.
  • 19 Nov 2024 According to an Azafaros media release, the company will deliver a poster presentation on 6 Feb 2025 at the WORLDSymposium™ 2025, showing an analysis compares data from the RAINBOW and PRONTO studies, illustrating the role of natural history studies in guiding drug development.

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