23 results
The aim of the project is to examine the association between clinical, environmental and genetic characteristics of the recipient of erythrocyte transfusions and the risk or resistance to immunization against erythrocyte alloantigens that he/she was…
Frequency of PNH in a selection of patients with DVT. In several publications PNH has been described especially in patients with DVT and younger than 30 years of age and/or with DVT on unusual places.
Objectives: The following trial objectives for adolescent patients (from 12 and up to 18 years of age) with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:* Assess the effect of eculizumab on TMA-Event Free status…
Objectives: The following trial objectives for adult patients with plasma therapy-resistantAtypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:• Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA) asindicated by…
Objectives: The following trial objectives for adult patients with plasma therapy-resistantAtypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:• Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA) asindicated by…
Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
Assess the efficacy of eculizumab in adult patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
Objectives: The following trial objectives for adult patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:* Assess the effect of eculizumab on TMA-Event Free status defined as the absence of [1] decrease in…
To study the role of GPI-deficiency in susceptibility of hematopoietic stem cells to cytotoxic T lymphocyte (CTL) and Natural Killer (NK) cell mediated lysis in the pathogenesis of PNH.
1.To create insight in current disease burden by creating a descriptive cohort of patients, diagnosed with rare congenital hemolytic anemia. Points of interest are:- Prevalence and incidence of disease- Quality of life- Prevalence and incidence of…
To determine the safety and efficacy of coversin in the treatment of patients with PNH resistant to eculizumab.
Primary Objective: To compare the extent of haemolysis associated with the use of the conventional versus the miniaturised heart lung machine in the Amphia hospital, with the aim to set up evidence-based guidelines for the use of a specific type of…
1. To assess the effect of increased hematocrit on cerebral perfusion as depicted by CBF and CVR.2. To determine the effect of GBT440 on oxygen saturation of the cerebral vasculature and the oxygen local consumption.3. Determine the dose response…
The current study aims to evaluate magnetic resonance imaging (MRI)-based CVR measurements with ACZ in SCD. It*s primary objective is to assess whether there is a correlation between CVR with silent cerebral infarcts (SCI) (i.e. the cerebrovascular…
The primary objective of this study is to investigate whether darbepoetin alfa is effective in reducing the incidence of late anemia in infants with HDN treated with IUT and therefore in decreasing the number of top-up transfusion required per…
Part APrimary Objective:• The primary objective of Part A is to determine whether BIVV009 administration results in a >= 2 g/dL increase in hemoglobin (Hgb) levels or increases Hgb to >= 12 g/dL and obviates the need for blood…
* The purpose of this study is to to determine the safety profile of long-termCoversin treatment.* To observe the long term safety and efficacy of Coversin over periods in excess of 6 months* To assess the long term patient acceptability of Coversin…
The primary objective of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, andefficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).
The aim of this study is to determine whether LNP023 is effective and safe for the treatment of PNH. LNP023 is compared to the Standard of Care (SOC) anti-C5 antibody treatment. The primary objectives are to:• Demonstrate superiority of LNP023…
Primary objective1. The primary aim of the study is to answer the question whether specification of a positive direct antiglobulin test and/or red blood cell autoantibody specification is correlated with the clinical course in patients with AIHA. We…