Homozygous Familial Hypercholesterolemia
36
5
6
23
Key Insights
Highlights
Success Rate
96% trial completion (above average)
Published Results
13 trials with published results (36%)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
2.8%
1 terminated out of 36 trials
95.8%
+9.3% vs benchmark
39%
14 trials in Phase 3/4
57%
13 of 23 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 23 completed trials
Clinical Trials (36)
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
HoFH, the International Clinical Collaborators Registry
A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia
NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
LOWER: Lomitapide Observational Worldwide Evaluation Registry
Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C
A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia
Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH
A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Evaluation of the Safety, Tolerability and Efficacy of Ezetimibe on a Select Population of Filipinos With Hypercholesterolemia (Study P04748)(COMPLETED)
Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)