Alpha-Mannosidosis
14
3
4
7
Key Insights
Highlights
Success Rate
88% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 30/100
7.1%
1 terminated out of 14 trials
87.5%
+1.0% vs benchmark
36%
5 trials in Phase 3/4
0%
0 of 7 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 7 completed trials
Clinical Trials (14)
Longitudinal Study of Neurodegenerative Disorders
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
Expanded Access to Velmanase Alfa
Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients
Lamazym Aftercare Study
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis
Evaluation of Long-term Efficacy of Treatment With Lamazym
A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis
Clinical Biomarkers in Alpha-mannosidosis
Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis