Condition
Sphingolipidoses
Total Trials
6
Recruiting
1
Active
2
Completed
2
Success Rate
50.0%-37% vs avg
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
Termination Rate
33.3%
2 terminated out of 6 trials
Success Rate
50.0%
-36.5% vs benchmark
Late-Stage Pipeline
0%
0 trials in Phase 3/4
Results Transparency
100%
2 of 2 completed with results
Key Signals
2 with results50% success
Data Visualizations
Phase Distribution
3Total
P 2 (3)
Trial Status
Completed2
Terminated2
Recruiting1
Active Not Recruiting1
Trial Success Rate
50.0%
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (6)
Showing 6 of 6 trials
NCT02171104Phase 2Active Not Recruiting
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT04885179RecruitingPrimary
SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)
NCT02416661Completed
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
NCT01963650Terminated
Natural History Study of Children With Metachromatic Leukodystrophy
NCT01043640Phase 2Completed
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
NCT00668564Phase 2Terminated
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Showing all 6 trials