Clinical Trials Logo

Clinical Trial Summary

Atypical Hemolytic Uremic Syndrome (aHUS) is a rare, lifethreatening, chronic disease of complement-mediated thrombotic microangiopathy (TMA) characterized by acute onset of renal impairment, thrombocytopenia, and microangiopathic hemolytic anemia. The estimated incidence of aHUS is approximately 0.5 per million per year. aHUS affects both adults and children, but is observed primarily in children and young adults. Atypical HUS commonly develops due to dysregulation of the alternative complement pathway and can be sporadic (80%) or familial(20%). The clinical course of aHUS is often unpredictable and can be dependent upon the specific genetic abnormality present within the complement system, if any, and/or triggering events associated with complement activation or inflammation, including autoimmune disease, transplant, pregnancy, infection, metabolic conditions, and drug use. In patients with dysregulated complement activity, such as those with complement mutations commonly observed in aHUS, the kidney vasculature is often the site of thrombosis stemming from endothelial injury. Cemdisiran has been designed to reduce the level of C5 mRNA in the liver, thereby reducing levels of circulating C5 protein, inhibiting terminal complement pathway activity, and preventing formation and deposition of the MAC (C5-b9) on endothelial cells in the kidney. As a result, complement-mediated endothelial cell damage in patients with aHUS and subsequent progression to End Stage Renal Disease (ESRD) may be reduced.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT03999840
Study type Interventional
Source Mario Negri Institute for Pharmacological Research
Contact
Status Withdrawn
Phase Phase 2
Start date January 2021
Completion date March 2024

See also
  Status Clinical Trial Phase
Recruiting NCT03605511 - TTP and aHUS in Complicated Pregnancies
Withdrawn NCT03303313 - A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome Phase 2
Recruiting NCT04861259 - A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Phase 3
Recruiting NCT06065852 - National Registry of Rare Kidney Diseases
Terminated NCT02614898 - Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
Completed NCT02574403 - Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS Phase 4
Recruiting NCT04958265 - A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Phase 3
Recruiting NCT05795140 - Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS Phase 3
Completed NCT00844545 - Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS Phase 2
Completed NCT00844844 - Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS Phase 2
Terminated NCT01522170 - aHUS Observational Long Term Follow-Up N/A
Terminated NCT02464891 - Complement Inhibition in aHUS Dialysis Patients Phase 2
Withdrawn NCT02626663 - The Role of Microparticles as a Biomarker
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Completed NCT00838513 - Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS) Phase 2
Completed NCT00844428 - Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS Phase 2
Recruiting NCT04889430 - Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy Phase 3
Recruiting NCT05935215 - Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Phase 3
Recruiting NCT03205995 - Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome Phase 3
Recruiting NCT05996731 - Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases N/A