Gangliosidosis

  • AZA-001-301: 18-month double-blind, randomized, placebocontrolled, 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

 

Acronym

AZA-001-301
Intervention Nizubaglustat- AZ-3102
Principal investigator Bénédicte Héron
Sponsor Azafaros
Study status In preparation
Recruitment status Not started
Population Child