Imagine-1

Phase 2, open-label trial of PBGM01 in GM1 gangliosidosis (GM1) (4 arms), sponsored by GEMMA Biotherapeutics. Primary endpoints: Number of Participants with Treatment Related Adverse Events and Serious Adverse Events at 5 years and Change from Baseline in Developmental Milestones as Assessed by the Bayley Scale of Infant and Toddler Development, Third Edition at 2 years. Started Mar 2021; status: active, not recruiting.