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: Completed

Phase of Trial: Phase II

Latest Information Update: 19 Jun 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

05 Jun 2026 According to an Azafaros media release, data published in the Molecular Genetics and Metabolism Journal.
05 Jun 2026 Results presented in the Azafaros Media Release.
14 May 2026 Status changed from active, no longer recruiting to completed.

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