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

Inclusion Criteria

• •Cohort 1 (NPC and GM2 patients):
• •* Have been randomized into Phase 2 Study AZA-001-5A2-01.
• •OR
• •Cohort 2 (NPC patients):
• •Be male or female aged ≥12 years
• •Have a genetically-confirmed diagnosis of NPC disease
• •Have received full-dose Miglustat treatment for at least 12 months and experienced disease stabilization or worsening with treatment over the 2 previous clinic visits. Patients experiencing clinical improvement with Miglustat over the preceding 3 months should not be considered for this study.
• •Wish to change treatment to Nizubaglustat for their NPC disease.
• •Participants from Phase 2 Study AZA-001-5A2-01 (RAINBOW) who transitioned to Miglustat may be eligible for Cohort 2 if they meet all other criteria.
• •Participation is supported and deemed beneficial by the Principal Investigator. Be willing and able to be evaluated for all protocol assessments. The participant, parent, and/or legal guardian can read, understand, and sign the informed consent form. Where appropriate, assent will also be sought for participants who have not reached the age of majority.