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

Recruiting

Phase 3

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

Sponsor:

Azafaros B.V.

Code:

NCT07082543

Conditions

Gangliosidoses, GM2

Gangliosidosis, GM1

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

Interventions

AZ-3102

Placebo

Study Details

Eligibility Criteria

Study Design

Interventions and Outcome Measures

Central Contacts and Locations

More Information

Trial information was received from ClinicalTrials.gov and was last updated on 2026-06-24. This information was provided to ClinicalTrials.gov by Azafaros B.V. on 2026-06-18.