- active not recruitingPHASE1, PHASE2
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
This study is testing a new gene therapy, PBGM01, for children with GM1 gangliosidosis (types 1 and 2a). It aims to see if the treatment is safe and effective in delivering a working gene to help the body fix the underlying problem.
United States · Brazil · Turkey (Türkiye) - 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