Atypical Hemolytic Uremic Syndrome
28
8
10
8
Key Insights
Highlights
Success Rate
67% trial completion
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
14.3%
4 terminated out of 28 trials
66.7%
-19.8% vs benchmark
29%
8 trials in Phase 3/4
88%
7 of 8 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 8 completed trials
Clinical Trials (28)
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
The Burden of Atypical Hemolytic Uremic Syndrome and The Clinical Characteristics of Patients in Egyptian Hospitals A Multicenter, Observational, Retrospective Cohort Study in Egypt
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases
Ravulizumab Outcomes in Polish Patients With aHUS
Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome
Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
National Registry of Rare Kidney Diseases
Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients
Observational Study of Elizaria® in aHUS Patients
Eculizumab to Cemdisiran Switch in aHUS
Eculizumab Use in the Postpartum Period for the Treatment of Pregnancy Associated aHUS: A Case Series
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS
The Role of Microparticles as a Biomarker