- RecruitingPHASE3
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
This study is looking at a new medicine called AZ-3102 for children and young people with rare conditions like Niemann-Pick Type C, GM1 Gangliosidosis, or GM2 Gangliosidosis. It aims to see if the medicine is safe and if it helps improve their health over 18 months.
For: Niemann-Pick Type C Disease · GM1 Gangliosidosis · GM2 GangliosidosisUnited States · Argentina · Australia - RecruitingPHASE3
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
This study is looking at a new medicine called AZ-3102 for children and young people with GM1 or GM2 gangliosidosis. Researchers want to see if it's safe and helps improve balance and movement problems. Some people will get the medicine, others will get a dummy pill.
For: Gangliosidoses, GM2 · Gangliosidosis, GM1United States · Argentina · Australia