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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 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 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

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.

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)

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…

Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B

This study has been transitioned to CTIS with ID 2024-510738-42-00 check the CTIS register for the current data. The primary aim of the trial is to demonstrate the non-inferiority of CSL222 (formerly AMT-061) (2 × 1013 gc/kg) during the 52 weeks…

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…

An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

This study has been transitioned to CTIS with ID 2023-509201-77-00 check the CTIS register for the current data. Primary ObjectiveTo evaluate the safety of long-term dosing with donidalorsen in patients with HAE. Secondary ObjectivesTo evaluate the…

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.

A Phase 3, Multicenter, Single-arm, Open-label Study of the Efficacy and Safety of
B-Domain Deleted Recombinant Porcine Factor VIII (BAX 802) in Subjects with
Congenital Hemophilia A with Factor VIII Inhibitors Undergoing Surgical or Other
Invasive Procedures

To evaluate the perioperative hemostatic efficacy of BAX 802 in male subjects with CHA with inhibitors to human factor VIII (hFVIII) undergoing major or minor elective surgical, dental, or other invasive procedures as determined by the Global…

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…

A prospective, phase 3, open label, international multicenter study on efficacy and safety of prophylaxis with rVWF in severe von willebrand disease

The primary objective of this study is to prospectively evaluate the annualized bleeding rate (ABR) for spontaneous bleeding episodes while on prophylactic treatment with rVWF (vonicog alfa) and to compare it to the subject's historical ABR for…

A Phase 3b Continuation study of the Safety and Efficacy of PEGylated
Recombinant Factor VIII (PEG-rFVIII; BAX 855) in Prophylaxis of Bleeding in
Previously Treated Patients with Severe Hemophilia A

Study Purpose* To continue the evaluation of the safety and efficacy of prophylaxis with BAX 855 for the prevention and treatment of bleeding episodes in PTPs (children and adults from 0 to 75 years of age) with severe hemophilia A.Primary…

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…