92 results
1. To establish optimized approaches for the isolation and characterization of BOECs and iPSC-ECs2. To further explore the biology of VWF, the pathophysiology of different subtypes of VWD and the role of VWF as a signalling protein in for example…
DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild hemophilia A.
Primary objectives:To assess the proportion of non-severe hemophilia A patients within FVIII target levels with the DDAVP and FVIII concentrate combination treatment in the first 72 hours after the start of combination treatment, without adding off-…
Primary objectives of the study are:• Arm 1 and Arm 3 (PTPs): to characterize the long-term safety profile of rVIII SingleChain with respect to inhibitor development in PTPs.• Arm 2 (PUPs): • To characterize the safety with respect to inhibitor…
To investigate whether peri-operative dosing using a population-based pharmacokinetic model (non-linear mixed effect modelling) in hemophilia patients leads to a significant reduction in clotting factor consumption in comparison to the standard…
- International network among European (125 cases) and Iranian (125 cases) centers- Prospective enrollment of the 250 VWD3 patients using a common database online- Detailed information about previous bleedings and exposure to VWF concentrates-…
Two main research questions:- improving the knowledge of the pathofysiologic mechanisms of immune tolerance induction (ITI)- determining what type of factor VIII product (based on pathophysiology and an in-vitro model of ITI) is the most effective…
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…
This study has been transitioned to CTIS with ID 2024-517249-15-00 check the CTIS register for the current data. Primary Objective:To evaluate the safety of extended dosing, and alternative dosing and/or dose frequency with ISIS 721744 in patients…
To improve the knowledge and understanding of the (patho)physiology of lymph disorders Main objective: the anatomy of the central lymph vessels, the velocity and direction of lymph flow and its influencers and thoracic duct outlet location and…
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…
This study has been transitioned to CTIS with ID 2024-512603-39-00 check the CTIS register for the current data. Primary: To assess the long-term safety (6-10 years after dosing) of a systemic administration of AAV5-hFIX, an AAV vector containing a…
HSCT cohort:* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the risk of graft failure* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the…
The primary objective of this study is to assess the feasibility and safety to image αvβ3-integrin expression in patients with AVM using Ga-68-DOTA-(RGD)2 PET/CT.
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…
Identification, segregation analysis, and functional characterization of genetic variants possibly causing rare bleeding disorders. The following questions will be answered:1) Can we identify (new) causal variants that explain the phenotype of…
This study has been transitioned to CTIS with ID 2023-509201-77-00 check the CTIS register for the current data. Primary ObjectiveTo evaluate the safety of long-term dosing with donidalorsen in patients with HAE. Secondary ObjectivesTo evaluate the…
To evaluate the diagnostic accuracy of the experimental tests to detect bleeding disorders and to evaluate whether or not they can be used for monitoring the effects of coagulant factor replacement therapy.
To investigate whether on demand dosing using population-based PK-models in VWD patients is reliable and feasible.
To determine the natural history of sickle cell disease and to identify modifying factors, including: (epi)genetic, biological, pathophysiological, social/ demographic, psychological and therapeutic determinants, which contribute to morbidity and…
Primary objective: to register and investigate Dutch patients suspect for an inherited platelet function disorder, to assess clinical presentation, bleeding score, treatment, burden of disease and quality of life. Secondary objectives: to…