NCT07399704

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

Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (estimated)
• Sponsor: Azafaros A.G. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

This is a multicenter, open-label study to assess the safety, tolerability, PK, PD, and efficacy of Nizubaglustat in male or female patients with late-infantile or juvenile onset GM2 gangliosidosis or NPC disease in two cohorts: * Cohort 1: Patients who previously took part in Phase 2 Study AZA-001-5A2-01 (RAINBOW) and wish to continue in this open-label study * Cohort 2: Approximately 10 patients with NPC disease, aged ≥12 years who received full-dose Miglustat for more than 12 months, have stable or worsening disease over the 2 previous clinic visits, and who wish to stop Miglustat treatment and transition to Nizubaglustat.

Conditions

• GM2 Gangliosidosis
• Niemann-Pick Type C Disease

Interventions

Type	Name	Description
DRUG	AZ-3102	Daily oral intake of AZ-3102 dispersible tablets

Timeline

• Start date: 2026-02-04
• Primary completion: 2030-04-15
• Completion: 2030-08-07
• First posted: 2026-02-10
• Last updated: 2026-03-19

Locations

3 sites across 1 country: Brazil