46 results
This is a follow-up (FU) study for subjects who completed Study ALX0681-C301 (HERCULES) according to the protocol (i.e., completed the Final [28 day] FU visit). Subjects who completed Study ALX0681-C301 will be given the option to participate in…
(i) identification and cellular characteristics of red blood cells from patients with altered ion homeostasis and delivery of novel and easy-to-use diagnostics for rare anemia*s, for patients with: a) a known primary defect in the ion channels of…
Primary objectives:(i) To study the pathophysiology, in particular the role of disturbed ion channel function in hereditary haemolytic anaemia(ii) To define novel intra- and extracellular (bio)markers in hereditary haemolytic anaemia. Secondary…
Preparation for in vivo correction of SCD and X-SCID causing mutations by CRISPR-Cas9 by in vitro studies in cell lines
To determine the effect of a single red blood cell transfusion on the microcirculation.
The primary objective in this observational study is to determine microcirculatory profile in preterm and term neonates. We would like to determine how the microcirculation changes in time during the phase of adaptation and correlate inter- and…
Our principal aim is to assess the effects of phlebotomy on aortic augmentation (AIx) and central BP in supine position and after standing; second, to assess the effects of counter maneuvers (leg crossing and thigh compression) on aortic…
The main, primary objective of this study is identification of the molecular mechanisms that regulate structure and function of red blood cells in patients with neurodegeneration accompanied by the presence of abnormal red blood cells (…
To sess the relationship between cerebral perfusion and clinical findings (neurological and neuropsychological) in patients with sickle cell disease.To compare iamging fincdings in children with and without sickle cell disease.
To determine the role of VWF and ADAMTS13 in the adherence of sickled erythrocytes to vascular endothelium.
The main purpose of this project is to investigate the conditions under which blood transfusions are successful in promoting microcirculatory circulation and oxygenation in anemic patients. In this project we wish to apply our state of the art…
To study whether microcirculatory perfusion is improved by ECMO. Perfusion will be studied before & after ECMO start, during ECMO weaning and before & after ECMO stop. To study the precictive value of perfusion and its correlation…
Aim of the proposed study will be to investigate whether microvascular blood flow at baseline is impaired in Eisenmenger patients who experience symptoms of the *hyperviscosity syndrome*. Furthermore, in view of the controversy whether to perform…
To determine the rate of transmission of prilocaine across the placentaTo determine the levels of methemoglobinemia in newborns after perineal block with prilocaine during labor (following the regimen used in our hospital)To determine the levels of…
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis (ie, proportion ofpatients achieving lactate dehydrogenase (LDH) * 1.5× upper limit of normal (ULN) over 26 weeks) of…
The primary objective of this study is to assess the noninferiority of ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor. Noninferiority will be claimed if after 26 weeks of treatment…
The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patientswith active PNH who are treatment-naive to complement inhibitor therapy or have not recently received…
Primary Objectives: - To evaluate the safety in mother and neonate/infant of nipocalimab administered to pregnant women at high risk for EOS-HDFN. - To evaluate the efficacy of nipocalimab as measured by proportion of patients with live birth at or…
To study the effect of voxelotor on the hemodynamics of the cerebral vasculature (CBF and CVR)
Primary:• To evaluate PK noninferiority of ravulizumab SC versus ravulizumab IV in adult patients with PNH Secondary• To characterize PK of ravulizumab SC• To characterize PD of ravulizumab SC• To characterize immunogenicity of ravulizumab SC• To…