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Hemoglobin Trajectories: Genetic determinants in Donors (HETEROGENIC DONORS)

To investigate the association between genetic factors and Hb recovery after blood donation. If an association can be found, the added value of genetic factors in prediction models for Hb deferral will be assessed. Also, the association between iron…

Assessment of laser speckle contrast imaging (LSCI) and dynamic light scattering (mDLSTM) to detect differences in blood flow and coagulation between healthy volunteers and patients with coagulation disorders or hemoglobinopathies

Primary objective- Short- and mid-term repeatability of mDLS and LSCI measures;- Comparison of blood flow obtained by LSCI and blood flow obtained by mDLS;- Comparison of coagulation status obtained by mDLS and coagulation status assessed by…

Prevention of local thromboembolic complications during continuous infusion of coagulation factor concentrates: parallel infusion of saline versus heparin and parallel infusion of saline?

To demonstrate that parallel infusion of saline added to the continous infusion of clotting factor concentrates prevents local thromboembolic complications to the same extent as heparin together with a parallel infusion of saline does.

Blood outgrowth endothelial cell - mediated coagulation

The aim of this research is to improve the understanding of the coagulation system through a detailed characterization and quantification of biosynthesis and expression of several coagulation proteins in BOECs. The data obtained in the study will be…

Study of (increased) expression of red blood cell pyruvate kinase antigen and ineffective erythropoiesis in patients with red blood cell pyruvate kinase deficiency.

Primary Objective: The here described study aims to strengthen the recently obtained results regarding increased expression of PK antigen in patients with PK-deficiency. This will be achieved by increasing the number of investigated patients and…

The Effect of Gaucher Disease on Bone Cells

This will be a pilot study that aims to gain insight into the effect of Gaucher disease on bone metabolism. For this purpose we formulated the following research questions:What is the effect of inflammatory factors in the serum of patients with…

A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to Recombinant Human Antihaemophilic Factor VIII (rFVIII; INN: octocog alfa) in Subjects with Hemophilia A, and a Repeat PK, Safety and Efficacy Study

The study objectives are to determine the incidence rate of FVIII inhibitors, frequency of adverse events (AEs), and serious adverse events (SAEs) associated with the use of CSL627, to evaluate the PK of 50 IU/kg CSL627, and to evaluate the efficacy…

Assessment of blood flow velocity and oximetry using non-invasive retinal and cutaneous microcirculation imaging in sickle cell disease (SCD) patients and healthy volunteers.

Assessment of the cutaneous and retinal microcirculation in SCD patients and matched controls:- Long- and short-term repeatability of LSCI and NiRFI ;- Comparison of cutaneous and retinal microcirculation between SCD patients and matched healthy…

A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients with Severe Hemophilia A

Primary ObjectiveThe primary objective is to compare the annualized rates of bleeding episodes (ABR)between subjects receiving a prophylactic regimen of BAX 855 with an on-demandtreatment regimen.Secondary ObjectivesThe key secondary objective is to…

Leiden's IVIg trial in Rhesus disease of the Neonate

AimTo determine whether the prophylactic use of IVIg reduces the need for ET in neonates with Rh-D hemolytic disease.

Home-monitoring of disease activity in pediatric chronic disease.

General- To compare physical activity levels of pediatric patient subjects with healthy control subjects.- To compare mean heart rate and variation in heart rate during the day of patients and healthy controls.- To compare sleep duration of patients…

A SINGLE-ARM, MULTICENTER PHASE IIIB CLINICAL TRIAL TO EVALUATE THE SAFETY AND TOLERABILITY OF PROPHYLACTIC EMICIZUMAB IN HEMOPHILIA A PATIENTS WITH INHIBITORS

The study will investigate the safety and efficacy of emicizumab in patients with hemophilia A with inhibitors against FVIII.

Joint distraction in the treatment of haemophilic ankle arthropathy: clinical and structural efficacy, a pilot study

This study is designed to gather prospective data on a) the clinical effectiveness, b) the tissue structure changes, and c) the short term costs of ankle joint distraction (AJD) in 10 patients with HAA during 10 years follow-up.

An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B

The purpose of this study is to investigate the safety and efficacy of rFIXFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) guideline on clinical…

An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A

The purpose of this study is to investigate the safety and efficacy of rFVIIIFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) guideline on the…

He-move-philia, a combined lifestyle intervention program for patients with hemophilia and other congenital coagulation disorders

The aim of this exploratory study is to compare the effect of a lifestyle intervention program consisting of combined individual coaching and group sessions (CLI) with a lifestyle intervention program with individual coaching only (II).The program…

A Phase 3 open-label, multicenter study of the safety, efficacy, and pharmacokinetics of intravenous recombinant coagulation Factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated pediatric patients <12 years of age with severe hemophilia A

To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A.

A Phase 3 open-label, multicenter study of the long-term safety and efficacy of intravenous recombinant coagulation factor VIII Fc-von Willebrand factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients with severe hemophilia A

This study has been transitioned to CTIS with ID 2023-508929-27-00 check the CTIS register for the current data. -To evaluate the long-term safety of BIVV001 in previously treated patients with hemophilia A.-To evaluate the efficacy of BIVV001 as a…

An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants with Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

The objective of this open-label extension (OLE) study is to assess the effect of long-term safety and treatment effect of Voxelotor in participants who have completed treatment in Study GBT440-031, using the following parameters:- Safety based upon…

Blood Outgrowth Endothelial Cells (BOECs) and Megakaryocytes (MKs) for in vitro studies of hemostatic and secretory function.

1. To establish optimized approaches for the isolation and characterization of BOECs, CD34+-derived MKs, iPSC- ECs and iPSC-MKs.2. Dissect the cellular mechanisms that control endothelial and platelet (secretory) function, thereby understanding the…