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Bone density as outcome parameter in haemophilia treatment

• Compare bone density between patients with mild haemophilic and those with severe haemophilia*.• Compare the relationship between treatment regimen: prophylactic (high-dose vs. intermediate dose) and on-demand and bone density.• Examine the…

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…

Multi-center, Randomized, Open-label, Parallel-arm, Single-dose, Pharmacokinetic Study of rVIIa-FP (CSL689) in Subjects with Congenital Factor VII Deficiency

- to evaluate the FVIIa activity PK of 2 CSL689 dose levels in subjects with congenital FVII deficiency- to determine the PK characteristics of FVIIa activity of CSL689- to evaluate the safety and tolerability of intravenous administration of CSL689

A Phase 3, Multi-Center, Open Label Study of Efficacy and Safety of PEGylated rFVIII (BAX 855) in Previously Treated Patients With Severe Hemophilia A Undergoing Surgical or Other Invasive Procedures

The primary objective is to evaluate the peri-operative hemostatic efficacy of BAX 855 in male PTPs aged 18 - 75 years with severe hemophilia A (FVIII <1%) undergoing major or minor, elective or minor emergency surgical, dental or other…

Immune Tolerance Induction study in patients with severe type A haemophilia
with inhibitor after failure of a previous induction of immune tolerance with
FVIII concentrates without Von Willebrand factor. (RESIST-EXPERIENCED)

Evaluate whether FVIII/VWF concentrates successfully induce immune tolerance in patients who have already experienced and failed immune tolerance induction with VWF-free FVIII concentrates.

Cerebral imaging in adults with sickle cell disease using ultra high field magnetic resonance imaging

To determine whether pathological changes are visible in brain parenchyma, blood vessels or other intracranial structures on ultra high field MRI in SCD patients in normal anatomical areas on 3.0 Tesla MRI and to determine the nature of these…

Coronary artery calcification measured with multi detector-row computed tomography in patients with hemophilia A

To investigate whether patients with hemophilia A develop less coronary artery atherosclerosis as compared to non-hemophilic males.

A Phase Ib, Dose Escalation Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients

To assess the Pharmacokinetic and Pharmacodynamic properties of three dosages of Coagulation Factor VIIa (Recombinant) in congenital hemophilia A or B patientsTo assess the safety of three dosages of Coagulation factor VIIa (Recombinant) in…

A Clinical, Molecular Genetic and Coagulation study in patients with Klippel-Trenaunay Syndrome and related entities

Detection of mutations in AKT1 in patients with KTS

Finding early Indicators of Neurological Damage in Sickle Cell Disease

Main objective of the current study is to evaluatie clinical, laboratory and genetic risk factors associated with prograssion of neurological damage. The final objective is to make a prognostic model of these risk factors to predict early…

A phase 3 prospective, uncontrolled, multicenter study evaluating pharmacokinetics, efficacy, safety, and immunogenicity of BAX 855 (PEGylated full-length Recombinant FVIII) in previously treated pediatric patients with severe hemophilia A

7.2 Primary ObjectiveThe primary objective is to assess the incidence of FVIII inhibitory antibodies(>=0.6 Bethesda units [BU] using the Nijmegen modification of the Bethesda assay).7.3 Secondary Objectives1. To evaluate the PK parameters of…

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…

Blood outgrowth endothelial cells as a patient-derived ex vivo model system to study degranulation mechanisms in storage pool disease

1. To study the mechanisms that control endothelial and platelet secretion using blood outgrowth endothelial cells (BOECs) as an ex vivo model of endothelial and platelet secretion, in order to identify new regulators of and further unravel their…

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…

Genotype - phenotype correlation in congenital sideroblastic anemia

The family, described in a publication from 1987, is recently re-identified. It seems to be an asymptomatic condition: there seems to be no anemia or secondary iron overload. Again mapping this family has two purposes:1. Have these patients…

Variability in bleeding phenotype in patients with hemophilia A.

To investigate the relation of platelet responsiveness and systemic platelet activity to phenotype in patients with severe hemophilia A.

A Phase 3 clinical study to determine the pharmacokinetics, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in subjects diagnosed with von Willebrand disease

Primary objective:• Assess the pharmacokinetics (PK) of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary VWDSecundary objectives:• Compare the…

An Open-Label Study of the Safety and Efficacy of ReFacto AF in Previously Untreated Patients in Usual Care Settings

The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients (no prior exposure to factor products or any blood products) of less than 6 years of age.

A Phase III Open-label Pharmacokinetic, Efficacy and Safety Study of rVIII-SingleChain in a Pediatric Population with Severe Hemophilia A

To evaluate the efficacy of rVIII-SingleChain in the treatment of major andminor bleeding events based on the investigator*s 4-point assessment scale

Molecular basis of lymphatic dysplasia in children

Our first aim is to find the cause of lymphatic dysplasia in children. The subsequent aim is to study the pathogenesis of the entity. The final aim is to increase our understanding of regulation of lymph vessel formation in man.