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

Recruiting now · Phase 2

Conditions studied: GM2 Gangliosidosis, Niemann-Pick Type C Disease

In brief

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).

Key facts

Study ID

NCT07399704

Run by

Azafaros B.V.

People needed

21

Starts

2026-02-04

Expected to finish

2030-08-07

Last updated by the study team

2026-06-18

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Associação Hospitalar de Prot à Infância Dr. Raul Carneiro — Água Verde, Curitiba, Brazil (enrolling)
• Hospital de Clinicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
• Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira — Rio de Janeiro, Brazil (enrolling)

Full record on ClinicalTrials.gov

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