View clinical trials related to Azotemia.
Filter by:Eculizumab is an anti-C5 monoclonal antibody approved for rare diseases including atypical haemolytic-uraemic syndrome. The maintenance phase dosing regimen is identical for all adult patients but several studies have shown a high interindividual kinetics variability. A tailored administration of eculizumab based on therapeutic drug monitoring will be compared with real-life administration in adults suffering from an atypical haemolytic uraemic syndrome. The objective is to improve efficiency of eculizumab administration.
It is a multicenter observational non-comparative study of the efficacy and safety of long-term pathogenetic Elizaria® therapy in patients with atypical Hemolytic Uremic Syndrome
Thrombotic microangiopathy (TMA) is a severe and life-threatening condition, often affecting the kidneys and brain. It can occur on the background of various clinical conditions. Dysregulation of the alternative pathway of complement may be the etiological factor and this type of TMA is classified, according to the current nomenclature, as primary atypical hemolytic uremic syndrome (HUS). Half the patients with primary atypical HUS present with rare variants in complement genes, although coexisting conditions are often needed for the TMA to become manifest. In patients with secondary atypical HUS, certain coexisting conditions appear to drive the disease and treatment should target the underlying condition to remit the TMA. Recently, the investigators demonstrated, by using a novel in-house developed functional endothelial cell-based test, that complement dysregulation and overactivation is the dominant cause of disease and its sequelae in a subset of patients with secondary atypical HUS, having impact on treatment and prognosis. The investigators did first prove this concept in patients presenting with TMA and hypertensive emergency. A prospective study is needed to further corroborate these findings along the spectrum of TMA. The investigators hypothesize that their functional endothelial cell-based test, the so-called "HMEC" test, can better categorizes the TMA into different groups with potential therapeutic and prognostic implications. Thus, paving the road to the ultimate goal of precision medicine.
This study will evaluate the impact of early administration of erythropoietin in the number of red blood cell transfusions in children with Shiga toxin-producing Escherichia coli hemolytic uremic syndrome (STEC-HUS).
Diarrhea-associated hemolytic uremic syndrome (D+HUS) is defined as a prodrome of enteritis followed by thrombocytopenia (< 150,000/mm3), microangiopathic hemolytic anemia, and signs of variable degrees of renal damage (increase in serum Cr, proteinuria, and/or hematuria) . Our aim is to detect the most reliable early predictors of poor prognosis to identify children at major risk of bad outcome who could eventually benefit from early specific treatments.
A single site observational study aiming to: (i) Identify cases of previously undiagnosed thrombotic thrombocytopenic purpura (TTP) and atypical haemolytic syndrome (aHUS) in a cohort of women with complicated pregnancies (ii) Characterise the clinical features of these cases and (ii) Identify clinical features or biomarkers which may help distinguish TTP/aHUS from other complications of pregnancy such as preeclampsia
Eculizumab is a humanized monoclonal IgG antibody against protein C5 that works to inhibit the activation of the terminal complement cascade. The Eculizumab is currently FDA approved for the treatment of Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome (aHUS) and has been shown to improve the quality of life and overall survival in these patients. aHUS is a life-threatening disease of complement mediated thrombotic microangiopathy often triggered by an inciting event, such as an infection or immunocompromised state. Pregnancy has also been identified as an inciting event, with patients most often experiencing aHUS in the postpartum period. Due to its rare nature, pregnancy-associated aHUS is often mistaken for preeclampsia or hemolysis, elevated liver enzyme, low platelet (HELLP) syndrome. As standard treatment for preeclampsia and HELLP syndrome is completion of the pregnancy by expediting delivery of the baby. A missed diagnosis of aHUS can result in delays in treatment, including use of Eculizumab when appropriate; such delay can increase the risk of maternal morbidity and mortality. When aHUS is suspected in the postpartum period, Eculizumab could be initiated early; however, there is limited data on use of Eculizumab in this setting.
Hematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TMA) is an understudied complication of HCT that significantly affects transplant related morbidity and mortality. The investigators hypothesize that early intervention with complement blocker eculizumab will double survival in HCT recipients with high risk TMA, as compared to historical untreated controls. An optimal eculizumab dosing schedule can be determined for this population through eculizumab pharmacokinetic/pharmacodynamic (PK/PD) testing.
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
The purpose of this study is to evaluate the platelet count change from baseline and safety of OMS721 in adults and adolescents with atypical hemolytic uremic syndrome (aHUS). The study will also evaluate pharmacokinetics (PK), pharmacodynamics (PD), and anti-drug antibody response (ADA).