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Prospective Follow-up Study for Patients who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)

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…

Combined Molecular Microscopy for Therapy and Personalized Medication in Rare Anaemias Treatments

(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…

A Phase III double-blind, randomized, parallel group, multicenter placebo-controlled trial to study the efficacy and safety of caplacizumab in patients with acquired thrombotic thrombocytopenic purpura.

Currently, caplacizumab is being developed for treatment of acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare and potentially life-threatening thrombotic microangiopathy, in which accumulation of ULvWF multimers leads to an increased…

Disturbed ion homeostasis in hereditary hemolytic anaemia

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…

Correcting mutations in vitro using CRISPR-Cas9; towards autologous stem cell transplantation in sickle cell disease and X-linked severe combined immunodeficiency

Preparation for in vivo correction of SCD and X-SCID causing mutations by CRISPR-Cas9 by in vitro studies in cell lines

A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Long-term Safety and Efficacy of Darbepoetin Alfa Administered at 500 µg Once-Every-3-Weeks (Q3W) in Anemic Subjects With Advanced Stage Non-small Cell Lung Cancer Receiving Multi-cycle Chemotherapy.

The purpose of this study is to evaluate the safety of darbepoetin alfa, inclusive of the effects on survival and cancer progression, and the necessity of blood transfusions.

The effects of red blood cell transfusion on the microcirculation in chronic anemic preterm neonates.

To determine the effect of a single red blood cell transfusion on the microcirculation.

A randomized, 2 part, 4-treatment, 2-way cross-over study in healthy volunteers to compare the pharmacokinetic and pharmacodynamics profiles of 1 *g/kg of DA-3880 and EU sourced AND EU Aranesp® (AMGEN) after single intravenous or subcutaneous administration

The purpose of the study is to investigate to what extent DA-3880 is absorbed and eliminated from the body (this is called pharmacokinetics) as compared to the above mentioned Aranesp® formulation. In addition, the effect DA-3880 on the number of…

A Phase 2, Randomized, Double-blind, Placebo-controlled, Multicenter Study Comparing Siltuximab Plus Best Supportive Care to Placebo Plus Best Supportive Care in Anemic Subjects with International Prognostic Scoring System Low- or Intermediate-1-Risk Myelodysplastic Syndrome

The primary objective is to assess the clinical efficacy of siltuximab (a chimeric (murine-human) IgG1* mAb that specifically binds human IL-6 with high affinity and prevents its interaction with the IL-6 receptor, glycoprotein (GP) 80),…

The microcirculation in neonatal adaptation, an observational study.

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…

PHASE I, DOUBLE BLIND, PLACEBO-CONTROLLED, DOSE-ESCALATION STUDY TO ASSESS THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS OF FMX-8 IN HEALTHY MALE VOLUNTEERS

Primary:To determine the safety, tolerability, and pharmacokinetic (PK) profile of single ascending intravenous (iv) doses and of multiple ascending iv doses of FMX-8 in healthy subjectsSecondary:To evaluate the pharmacodynamics (PD) of FMX-8 in…

Central Hemodynamics, Augmentation index and Microcirculation after Phlebotomy

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 Red Blood Cell in Patients with Neuroacanthocytosis: an explorative study on regulation of morphology and function of red blood cells of patients with neurodegeneration of the basal ganglia.

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 (…

Cerebral Perfusion Imaging in Children with Sickle Cell Disease

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.

Determining the role of Von Willebrand factor and ADAMTS13 in the adhesion of sickled erythrocytes to endothelial cells

To determine the role of VWF and ADAMTS13 in the adherence of sickled erythrocytes to vascular endothelium.

The efficacy of blood transfusions to improve microcirculatory oxygen delivery in anemic patients.

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…

The microcirculation in children treated with extracorporeal membrane oxygenation;
an observational longitudinal study

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…

Protocol C: Microcirculatory response to phlebotomy in adult patients with Eisenmenger syndrome.

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…

Perineal block with prilocaine during labour: effect on the newborn infant. A pilot study.

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…

An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)

The purpose of Part 1A is to investigate how safe RO7112689 is and how well RO7112689 is tolerated. Part 1A will also investigate how quickly and to what extent RO7112689 is absorbed into, distributed in, and eliminated from the body (this is called…