Metachromatic Leukodystrophy (MLD)
11
3
3
5
Key Insights
Highlights
Success Rate
71% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 47/100
18.2%
2 terminated out of 11 trials
71.4%
-15.1% vs benchmark
0%
0 trials in Phase 3/4
80%
4 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 5 completed trials
Clinical Trials (11)
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
Direct Lentiviral Injection Gene Therapy for MLD
An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
Natural History Study of Children With Metachromatic Leukodystrophy
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders