GM2 Gangliosidoses
2
0
0
1
Key Insights
Highlights
Success Rate
50% trial completion
Treatment Research
2 treatments under study, led by Miglustat
Research Leaders
8 sponsors active, led by Children's National Research Institute
Related Conditions
Often studied with 4 related conditions
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 55/100
50.0%
1 terminated out of 2 trials
50.0%
-36.6% vs benchmark
100%
2 trials in Phase 3/4
100%
1 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 1 completed trials
Clinical Trials (2)
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
Research Network
Common Treatments
- Miglustat2
- Ketogenic Diet1
Active Sponsors
- Children's National Research Institute1
- National Center for Advancing Translational Sciences (NCATS)1
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)1
- Rare Diseases Clinical Research Network1
- Actelion1
Top Investigators
University of Minnesota Fairview Hospital
Children's National Research Institute
Related Conditions
- Sandhoff Disease2
- Taysachs Disease1
- Gm1 Gangliosidoses1
- Taysachs1