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

NCT07082543 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 75

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 GM1 gangliosidosis or GM2 gangliosidosis

Conditions

  • Gangliosidoses, GM2
  • Gangliosidosis, GM1

Interventions

DRUG

AZ-3102

Nizubaglustat

DRUG

Placebo

Placebo

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 NCT07082543 on ClinicalTrials.gov