GM1 Gangliosidosis
13
3
4
6
Key Insights
Highlights
Success Rate
86% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
7.7%
1 terminated out of 13 trials
85.7%
-0.8% vs benchmark
8%
1 trials in Phase 3/4
33%
2 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 6 completed trials
Clinical Trials (13)
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
A Natural History Study of the Gangliosidoses
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis
Interviews and Video Capture in Patients With GM1 Gangliosidosis
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Registry Gangliosidoses
HSCT for High Risk Inherited Inborn Errors
Stem Cell Transplant for Inborn Errors of Metabolism