RecruitingPhase 2NCT07399704

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

Sponsor

Azafaros B.V.

Enrollment

21 participants

Start Date

Feb 4, 2026

Study Type

INTERVENTIONAL

Conditions

GM2 Gangliosidosis Niemann-Pick Type C Disease

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).

Eligibility

Min Age: 12 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called AZ-3102 for people with gm2 gangliosidosis and niemann-pick type c disease. The study is currently recruiting participants at 3 locations. People eligible for this study include aged 12 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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