Active clinical trials for "Atypical Hemolytic Uremic Syndrome"

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

The objective of this retrospective trial is to assess safety and efficacy of eculizumab in aHUS patients treated outside of an Alexion-sponsored controlled clinical trial.

This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician. The purpose of this study was to assess disease manifestations of complement-mediated thrombotic microangiopathy (TMA) and evaluate potential clinical predictors of disease manifestations and progression in participants with aHUS with or without eculizumab treatment in the clinical setting.

There is growing but limited information on the long term clinical status of aHUS patients who have previously received or are continuing to receive treatment with eculizumab. This study is designed to collect clinical data that will provide insight into the long-term outcomes of patients with aHUS.

Atypical hemolytic uraemic syndrome is caused by defects in the regulating factors in the alternative pathway of the complement system. Triggering can cause an uncontrolled complement activation with endothelial damage and thrombotic micro-angiopathy, especially in the kidneys. This can result in endstage renal failure. Complement activation during hemodialysis has been described as a result of contact between blood and the dialysis membrane. Our hypothesis is that patients with atypical hemolytic uraemic syndrome have a stronger complement activation during hemodialysis than patients with another underlying kidney disease. This could be a reason to treat patients with endstage renal failure due to atypical hemolytic uraemic syndrome preferentially with peritoneal dialysis instead of hemodialysis.

This protocol is intended to formally collect data on the treatment of aHUS with eculizumab in Japanese patients.

The investigators propose to characterize MPs in aHUS and TTP both at the onset and throughout treatment. The investigators believe that the number, size, and cell origin of MPs will differ between these two diseases. The hypothesis is that endothelial derived MPs will be higher in number and comprise a larger portion of the MP population in aHUS and that platelet MPs will comprise a larger number and greater proportion of MPs in TTP. The investigators believe that MP identity and number can be used to reliably differentiate between aHUS and TTP at disease onset.

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.