37 results
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 objective:The primary objective of this study is to assess the time to painful VOC resolution, measured from the first dose of sevuparine given to achievement of crises resolution, as compared to placebo. Secondary objectives: The secondary…
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…
CORE PERIODPrimary:* Evaluate the safety and tolerability of up to 24 weeks of AG-348 administration in patients with pyruvate kinase deficiency (PK deficiency).Secondary:* Evaluate the pharmacokinetics (PK) of AG-348 and the metabolite AGI-8702.*…
Currently, caplacizumab is being developed for treatment of acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare and potentially life-threatening thrombotic microangiopathy, in which accumulation of ULvWF multimers leads to an increased…
The primary objective is:- to characterize the pharmacokinetic/pharmacodynamic profile of a 7-day treatment with oral rivaroxabanThe secondary objectives are:- to assess the incidence of major bleeding and clinically relevant non-major bleeding- to…
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…
Primary Objective• To compare the annualised total bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII from study GENA-01Secondary…
In our laboratory we are able to characterize and culture MSCs. In order to study more extensively the possible alterations in MSC biology in AA we will investigate bone marrow cells of patients with newly diagnosed AA and at the time of response…
Primary objective: To evaluate the effect of intravenous vitamin K therapy on the vitamin K dependent coagulopathy in patients with peroxisomal biogenesis disorders measured with APTT, PT, FV, FVII, fibrinogen, d-dimer, thrombocytes and PIVKA.…
Determination of the coefficient of variation of platelet aggregation test with Multiplate. Determining the reliability of the test results after direct assay, when compared to the conventrional (recommended) 30 minutes waiting time.
The primary objective is to determine whether APC is elevated in thrombophilia carriers compared to non-carriers. In parallel we will address. Secondary objectives include assessing whether biomarkers related to angiogenic pathways downstream of APC…
To determine the safety and efficacy of coversin in the treatment of patients with PNH resistant to eculizumab.
Primary objective:- To assess the safety of concizumab given as multiple s.c. doses to subjects with haemophilia ASecondary objectives:- To assess pharmacokinetics of concizumab in subjects with haemophilia A after multiple s.c. doses- To assess…
The purpose of the study is to investigate the effect on the body of enoxaparin manufactured by the Sponsor (this is called pharmacodynamics) and to compare it with the pharmacodynamic effect of Clexane®. In addition, it will be investigated to what…
The main purpose of the study is to assess immune cell dynamics in Tecfidera-treated MS patients and untreated healthy subjects, and to explore whether differences in immune cell dynamics exist between both populations. The data generated in this…
To evaluate the efficacy, safety and feasibility of combined immune modulation with rituximab, ITI and MSCs in terms of eradication of FVIII inhibitory activity in hemophilia A patients.
The primary objective of the study is to determine whether the use of pre-and intraoperative warming with a forced air technique leads to a reduction of the incidence of hypothermia compared to patients with only intra-operative forced air warming.…
The primary efficacy objective of the study is:* to evaluate whether rivaroxaban added to ASA is superior to ASA alone in reducingthe risk of major thrombotic vascular events (defined as MI, ischemic stroke, CVdeath, ALI, and major amputation of a…
To explore the effects of acute fear on the coagulation system while sitting still.