Metachromatic Leukodystrophy
24
2
4
12
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 24 trials
100.0%
+13.5% vs benchmark
4%
1 trials in Phase 3/4
25%
3 of 12 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 12 completed trials
Clinical Trials (24)
Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Gene Therapy for Metachromatic Leukodystrophy (MLD)
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
The Myelin Disorders Biorepository Project
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
Baby Detect : Genomic Newborn Screening
The Natural History of Metachromatic Leukodystrophy Study (HOME Study)
Early Check: Expanded Screening in Newborns
The Natural History of Metachromatic Leukodystrophy
Human Placental-Derived Stem Cell Transplantation
Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Single Patient Expanded Access Protocol: Metabolic Boost
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
HSCT for High Risk Inherited Inborn Errors