AZ-3102

DRUG4 trials

Sponsors

Azafaros B.V.

Conditions

GM1 GangliosidosisGM2 GangliosidosisGangliosidoses, GM2Gangliosidosis, GM1Gaucher Disease Type 3Niemann-Pick Type C Disease

Phase 2

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

RecruitingNCT07399704

Azafaros B.V.GM2 Gangliosidosis, Niemann-Pick Type C Disease

Start: 2026-02-04End: 2030-08-07Target: 21Updated: 2026-06-18

Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)

Not yet recruitingNCT07715084

Azafaros B.V.Gaucher Disease Type 3

Start: 2026-09-28End: 2029-07-01Target: 20Updated: 2026-07-20

Phase 3

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

RecruitingNCT07054515

Azafaros B.V.GM1 Gangliosidosis, GM2 Gangliosidosis, Niemann-Pick Type C Disease

Start: 2025-06-30End: 2028-06-14Target: 147Updated: 2026-09-16

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

Active, not recruitingNCT07082543

Azafaros B.V.Gangliosidoses, GM2, Gangliosidosis, GM1

Start: 2025-06-30End: 2028-03-06Target: 75Updated: 2026-07-24