A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with stable, full-dose Miglustat (Cohort 2).

NCT Number	NCT07399704
Lead Sponsor	Azafaros B.V.
Conditions	GM2 Gangliosidosis, Niemann-Pick Type C Disease
Enrollment	21 participants
Start Date	2026-02-04
Primary Completion	2030-04-15 (estimated)
Study Completion	2030-08-07 (estimated)
Updated on ClinicalTrials.gov	2026-06-18