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).
Have been randomized into Phase 2 Study AZA-001-5A2-01.
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.
A positive serum pregnancy test (only tested for women of childbearing potential).
Female planning to breastfeed during the study.
Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.
Participation in another interventional or non-interventional study or early access program.