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

NCT07399704 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2026-05-07

No results posted yet for this study

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).

Conditions

  • GM2 Gangliosidosis
  • Niemann-Pick Type C Disease

Interventions

DRUG

AZ-3102

Daily oral intake of AZ-3102 dispersible tablets

Sponsors & Collaborators

  • Azafaros A.G.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-02-04
Primary Completion
2030-04-15
Completion
2030-08-07

Countries

  • Brazil

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07399704 on ClinicalTrials.gov