Lipid Metabolism, Inborn Errors
14
0
0
9
Key Insights
Highlights
Success Rate
75% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 54/100
21.4%
3 terminated out of 14 trials
75.0%
-11.5% vs benchmark
50%
7 trials in Phase 3/4
56%
5 of 9 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 9 completed trials
Clinical Trials (14)
Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
Mendelian Reverse Cholesterol Transport Study
Sitosterolemia Extension Study (0653-003)(COMPLETED)
An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia (0653-062)(COMPLETED)
Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)
Sitosterolemia Extension Study (0653-004)(COMPLETED)
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
Dietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase Deficiency
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
Anti-oxLDL IgM Antibodies as a Novel Therapy for Metabolic Lipid Diseases
Genetic Study of Sitosterolemia