Krabbe Disease
13
3
6
3
Key Insights
Highlights
Success Rate
60% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 60/100
15.4%
2 terminated out of 13 trials
60.0%
-26.5% vs benchmark
0%
0 trials in Phase 3/4
100%
3 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (13)
Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
The Myelin Disorders Biorepository Project
Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
Early Check: Expanded Screening in Newborns
Krabbe Disease Global Patient Registry
Gene Transfer Clinical Trial for Krabbe Disease
Biomarker for Krabbe Disease (BioKrabbe)
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism