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Hemoglobin Trajectories: Genetic determinants in Donors (HETEROGENIC DONORS)

To investigate the association between genetic factors and Hb recovery after blood donation. If an association can be found, the added value of genetic factors in prediction models for Hb deferral will be assessed. Also, the association between iron…

A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to Recombinant Human Antihaemophilic Factor VIII (rFVIII; INN: octocog alfa) in Subjects with Hemophilia A, and a Repeat PK, Safety and Efficacy Study

The study objectives are to determine the incidence rate of FVIII inhibitors, frequency of adverse events (AEs), and serious adverse events (SAEs) associated with the use of CSL627, to evaluate the PK of 50 IU/kg CSL627, and to evaluate the efficacy…

A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients with Severe Hemophilia A

Primary ObjectiveThe primary objective is to compare the annualized rates of bleeding episodes (ABR)between subjects receiving a prophylactic regimen of BAX 855 with an on-demandtreatment regimen.Secondary ObjectivesThe key secondary objective is to…

A Phase 3 open-label, multicenter study of the safety, efficacy, and pharmacokinetics of intravenous recombinant coagulation Factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated pediatric patients <12 years of age with severe hemophilia A

To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A.

An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants with Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

The objective of this open-label extension (OLE) study is to assess the effect of long-term safety and treatment effect of Voxelotor in participants who have completed treatment in Study GBT440-031, using the following parameters:- Safety based upon…

Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients - The DosEmi study

This study has been transitioned to CTIS with ID 2024-515528-35-00 check the CTIS register for the current data. Primary objectiveTo determine whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is non-inferior to conventional…

Protocol EFC16293: A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc von Willebrand Factor XTEN Fusion Protein (rFVIIIFc VWF XTEN; BIVV001) in Previously Treated Patients *12 Years of Age With Severe Hemophilia A.

This study is being conducted to determine the safety and efficacy of the study drug BIVV001 when used as a once-a-week prophylaxis treatment or as an on-demand (as-needed) treatment for bleeding in patients 12 years and older with severe hemophilia…

A Phase 3 open-label, multicenter study of the long-term safety and efficacy of intravenous recombinant coagulation factor VIII Fc-von Willebrand factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients with severe hemophilia A

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…

A SINGLE-ARM, MULTICENTER PHASE IIIB CLINICAL TRIAL TO EVALUATE THE SAFETY AND TOLERABILITY OF PROPHYLACTIC EMICIZUMAB IN HEMOPHILIA A PATIENTS WITH INHIBITORS

The study will investigate the safety and efficacy of emicizumab in patients with hemophilia A with inhibitors against FVIII.

An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B

The purpose of this study is to investigate the safety and efficacy of rFIXFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) guideline on clinical…

An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A

The purpose of this study is to investigate the safety and efficacy of rFVIIIFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) guideline on the…