Condition
Lysosomal Storage Disorders
Total Trials
5
Recruiting
0
Active
0
Completed
0
Key Insights
Highlights
No notable highlights available yet
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
Termination Rate
20.0%
1 terminated out of 5 trials
Success Rate
0.0%
-86.5% vs benchmark
Late-Stage Pipeline
20%
1 trials in Phase 3/4
Results Transparency
0%
0 of 0 completed with results
Key Signals
0
Data Visualizations
Phase Distribution
2Total
P 2 (1)
P 3 (1)
Trial Status
Unknown2
Terminated1
Enrolling By Invitation1
Not Yet Recruiting1
Clinical Trials (5)
Showing 5 of 5 trials
NCT00001215Enrolling By InvitationPrimary
Genetic Studies of Lysosomal Storage Disorders
NCT07740512Phase 2Not Yet RecruitingPrimary
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
NCT02000310UnknownPrimary
Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
NCT02120235UnknownPrimary
Investigating Lysosomal Storage Diseases in Minority Groups
NCT00654433Phase 3Terminated
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Showing all 5 trials