16 results
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…
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 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…
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…
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…
This study has been transitioned to CTIS with ID 2023-506498-36-00 check the CTIS register for the current data. To evaluate the efficacy of crovalimab compared to eculizumab
We aim to further explore the role of decreased activity and stability of PK in several types of hereditary hemolytic anemia and non-regenerative anemia. Moreover, we will study the effect of restoring this instability using second generation (…
Part 1 - To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in subjects with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to…
The primary objective of this trial is to study in a prospective randomized comparison whether a restrictive RBC transfusions strategy is non-inferior compared to a liberal strategy in patients on ECMO with respect to 90-day mortality.
The primary objective of the study is to evaluate the percentage of subjects with at least 35%spleen volume reduction in the fedratinib and the BAT arms.The secondary objectives are:• To evaluate the percentage of subjects with at least 50%…
The main aim is to study early neurocognitive functioning and development in very young children with SCD. The secondary aims are to identify determinants (risk and protective factors) and biomarkers of early neurocognitive deficits in SCD and to…
This study has been transitioned to CTIS with ID 2023-506526-37-00 check the CTIS register for the current data. To evaluate the safety and tolerability of crovalimab compared witheculizumab
Efficacy of ALXN1210Sub-study: to Evaluate Patient Preference for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective is feasibility of a prospective randomized trial, to investigate whether a more restrictive RBC transfusion strategy of 5.0 g/dL (3.1 mmol/L) is non-inferior compared to the current transfusion threshold of 7.0 g/dL (4.3mmol/L…
The primary objectives of this study are to establish the efficacy and safety of APL-2 compared to eculizumab in patients with PNH who continue to have Hb levels <10.5 g/dL despite treatment with eculizumab.