Study of 'Vascular Competence' Profile and Endothelial Activation in the Hemolytic Uremic Syndrome...
Hemolytic Uremic SyndromeThe Hemolytic Uremic Syndrome (HUS) is a rare thrombotic microangiopathy (TMA), affecting both children and adults. HUS is characterized by the abnormal occurrence of diffuse thrombosis in the microcirculation resulting in the occurrence of ischemic events affecting especially the kidneys and is associated with hemolytic anemia. One of the major problems encountered in the management of HUS is the absence of reliable marker of treatment response or relapse; conventional hematological markers being too insensitive to judge therapeutic efficacy or identify early relapse. Data from the literature suggest that the endothelial cell is a major target of this syndrome. Our hypothesis is that an initial micro-endothelial activation plays a critical role in the initiation and / or relapse of the disease.The main objective of this study is to define a "vascular competence" profile in a population of patients with typical or atypical HUS; both in the acute phase and in remission of the disease.
Erythropoietin in Hemolytic Uremic Syndrome
Hemolytic-Uremic SyndromeAnemiaThis 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).
Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome
Thrombotic MicroangiopathiesAtypical Hemolytic Uremic SyndromeThe 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).
Rituximab in Patients With Relapsed or Refractory TTP-HUS
Thrombotic Thrombocytopenic PurpuraHemolytic Uremic SyndromeThe general objective of this study is to assess the efficacy and safety of Rituximab in the management of patients with refractory or relapsed thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). There have been several case reports and case series describing the use of Rituximab in patients with TTP-HUS; however its use has not been studied in a large trial. It is hypothesized that Rituximab may ameliorate the severity of certain cases of TTP-HUS by decreasing the number of activity of B-cells which may result in decreased production of the ADAMTS13 protease inhibitor. Patients with TTP-HUS not responding to standard therapy or patients with relapsed disease may have particular benefit. Treatments that decrease the frequency of relapse or shorten the time to remission of TTP-HUS will be of benefit by decreasing the need for blood product support.
Study of Repetitive Intestinal Lavage in Patients With EHEC Associated Hemorrhagic Colitis
Hemolytic Uremic SyndromeHemorrhagic Colitis2 moreThe investigators examined the outcome of patients with severe Enterohaemorrhagic E. Coli (EHEC) O104:H4 infection suffering from bloody diarrhoea that were at risk to develop hemolytic uremic syndrome and underwent repetitive whole bowl lavage during hospitalization.
Observational Study of Elizaria® in aHUS Patients
Atypical Hemolytic Uremic SyndromeaHUSIt 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
A Retrospective, Observational, Non-interventional Trial to Assess Eculizumab Treatment Effect in...
Atypical Hemolytic Uremic Syndrome (aHUS)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.
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic...
Atypical Hemolytic Uremic SyndromeThis 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.
International Registry and Biorepository for TMA(Thrombotic Microangiopathy)
Thrombotic MicroangiopathyHemolytic Uremic Syndrome1 moreThis registry will collect clinical data and store biosamples (seru, plasma, urine, and DNA) annually from pediatric patients with thrombotic mcroangiopathy
aHUS Observational Long Term Follow-Up
Atypical Hemolytic Uremic SyndromeThere 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.