Clinical Trial: 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

U.S., July 25 -- ClinicalTrials.gov registry received information related to the study (NCT07082543) titled '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' on June 27.

Brief 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

Study Start Date: June 30

Study Type: INTERVENTIONAL

Condition: Gangliosidoses, GM2 Gangliosidosis, GM1

Intervention: DRUG: AZ-3102

Nizubaglustat

DRUG: Placebo

Placebo

Recruitment Status: RECRUITING

Sponsor: Az...