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A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 147

Last updated 2026-05-05

No results posted yet for this study

Summary

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Conditions

  • Niemann-Pick Type C Disease
  • GM1 Gangliosidosis
  • GM2 Gangliosidosis

Interventions

DRUG

AZ-3102

Oral dispersible tablets

DRUG

Placebo

A matching placebo will be administered in the same regimen as the intervention

Sponsors & Collaborators

  • Azafaros A.G.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-30
Primary Completion
2027-11-04
Completion
2027-11-04
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Brazil
  • Canada
  • France
  • Germany
  • India
  • Italy
  • Mexico
  • Portugal
  • Spain
  • Sweden
  • Switzerland
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

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 NCT07054515 on ClinicalTrials.gov