Pompe Disease
74
8
11
40
Key Insights
Highlights
Success Rate
85% trial completion
Published Results
16 trials with published results (22%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 39/100
9.5%
7 terminated out of 74 trials
85.1%
-1.4% vs benchmark
12%
9 trials in Phase 3/4
40%
16 of 40 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 40 completed trials
Clinical Trials (74)
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease Registry Protocol
AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
A Global Prospective Observational Registry of Patients With Pompe Disease
Cognitive and Neurological Pathologies in Pompe Disease
Avalglucosidase Alfa Pregnancy Study
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.
Baby Detect : Genomic Newborn Screening
Drug-drug Interaction Study
MSOT in Pompe Disease
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Pompe Lactation Sub-Registry