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A Phase 1b, Randomized, Double-Blind (Sponsor Open), Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PF-04447943, Co-Administered with and Without Hydroxyurea, in Subjects with Stable Sickle Cell Disease

Primary ObjectiveTo determine the safety and tolerability of multipledoses of PF-04447943 Secondary ObjectiveTo characterize the PK of PF-04447943 in plasma following oral administrationExploratory ObjectivesTo evaluate biomarkers that may be…

Evaluation of the ex vivo effect of AG-348 treatment on pyruvate kinase-deficient cell metabolism and erythroid development

To extend and strengthen the initial pilot observations on PKD red blood cells, and thereby further establish the activity of AG-348 treatment for PKD, UMC Utrecht and Agios Pharmaceuticals will collaborate in this pre-clinical study. In particular…

Clinical sequelae and pathophysiology of rare congenital hemolytic anemias

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…

National study of inherited platelet function disorders in the Netherlands

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…

Predictors of Bleeding Evaluation in Adult Hematologic Patients with Bleeding Tendencies
Patients with established bleeding disorders: The BePa verification study

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.

Blood Outgrowth Endothelial Cells (BOECs) to study the pathophysiology of von Willebrand disease and the feasibility of in vitro correction of von Willebrand factor defects

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-…