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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 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…

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…

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…

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…

Verification of preoperative screening for bleeding tendency in patients that report bleeding symptoms

The main object is to evaluate the validity of a diagnostic screening package for bleeding tendency consisting of point-of-care devices when compared with the gold standard for bleeding tendency diagnosis.Other objects which will be investigated are…

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

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.

The value of ultrasound compared to magnetic resonance imaging in haemophilic arthropathy

The primary objective of this study is to establish the diagnostic accuracy of ultrasound assessment of the synovium in haemophilic arthropathy compared to MRI. Secondary objectives are (2A) to determine whether or not synovial hypertrophy on MRI is…

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…

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.

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…

Clinical validation of Factor VIII allo-antibody assays
in patients with hemophilia A.
The correlation of factor VIII antibodies, measured with different assays, on factor VIII survival in patients with severe haemophilia A.

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thrombin generation in plasma, the predictive value of endogenous thrombin generation (ETG)in relation to hypercoagulability and bleedingrisk

Assessing hereditary and aquired determinants of ETP. Evaluating the use of ETP as predictive stand alone test for recurrent DVT and bleeding complications due to anticoagulation therapy in comparisson with the current prediction methods.

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…

Development of a finger stick based whole blood micro-assay for the rapid detection of Von Willebrand Disease

Performance is the primary objective of this study and is defined as the ability of the in vitro diagnostic micro-assay to measure von Willebrand factor induced platelet aggregation in accordance with the golden standard diagnostic method, risto-…

A Phase 3 Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease

Primary ObjectiveThe primary objective is to assess the effect of vexelotor compared to placebo on improvement in hemoglobinSecondary ObjectivesThe secondary objectives are t o evaluate the effects of voxelotor compared to placebo on : - Clinical…

Desmopressin and FVIII concentrate combination treatment in non-severe hemophilia A patients undergoing minor interventions

To show a reduction in FVIII-concentrate consumption with perioperative desmopressin and FVIII concentrate combination treatment compared to FVIII concentrate monotherapy, without decreasing the effectivity of treatment.

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics and
Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe
Hemolytic Disease of the Fetus and Newborn (HDFN)

Primary Objectives: - To evaluate the safety in mother and neonate/infant of nipocalimab administered to pregnant women at high risk for EOS-HDFN. - To evaluate the efficacy of nipocalimab as measured by proportion of patients with live birth at or…

The Effect of Voxelotor on Cerebral Perfusion and Oxygenation (ESR-C005)

To study the effect of voxelotor on the hemodynamics of the cerebral vasculature (CBF and CVR)