Research.Patient.info
Niemann-Pick Type C Disease research hub
2 indexed studies · 2 currently recruiting
- 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.
United States · Argentina · Australia - 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 (NPC)
This study looks at a new drug, Nizubaglustat, for Niemann-Pick Type C (NPC) disease in children and young people. It aims to see if the drug can help with movement problems and if it's safe, compared to a dummy pill.
United States · Argentina · Australia