29 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.
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…
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…
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…
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 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)
Objectives: Primary:• To evaluate the long-term safety and tolerability of AG-348Secondary:• To evaluate the long-term efficacy of AG-348• To evaluate the efficacy of AG-348 in increasing hemoglobin (Hb) concentrations in subjects who previously…
To examine the determinants of survival of donor RBCs in SCD patientsTo look into the effect of transfusion on the innate immune system of sickle cell patients, in particular, the phenotype of the neutrophils.
This study has been transitioned to CTIS with ID 2024-515569-32-00 check the CTIS register for the current data. To study the safety and efficacy of mitapivat in the treatment of adult subjects with sickle cell disease.
To study the effect of apotransferrin administration in patients with β thalassemia intermedia on erythropoiesis as reflected by hemoglobin level or transfusion dependency. Secondary objectives are the effect of apotransferrin on the…
Primary Objectives: • To evaluate the safety and tolerability of ascending doses of KER 047 in participants with iron-refractory iron deficiency anemia (IRIDA)Secondary Objectives:• To evaluate the pharmacodynamic (PD) effects of KER 047 on iron…
In this study we will investigate how quickly and to what extent GBT021601 is absorbed, transported, and eliminated from the body. For this study, GBT021601 is radioactively labelled with carbon-14 (14C). In this way, GBT021601 can be traced in…
The primary objective of the study is to evaluate the efficacy of treatment with AG-348, as assessed by the reduction in transfusion burden.Secondary: The secondary objective of this study is to evaluate the safety of treatment with AG 348.