A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
Hemophilia AHemophilia BThe purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular medication. In addition, the study assessed safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).
Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin...
Congenital Bleeding DisorderHaemophilia AThe trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients...
Hemophilia BThis study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics and pharmacodynamics of ISU304/CB2679d in previously treated hemophilia B patients.
A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients...
HaemostasisHaemophilia AThis trial is conducted in Asia, Europe and the United States of America (USA). The aim of the trial is to assess the efficacy of concizumab administered s.c. (subcutaneously, under the skin) once daily in preventing bleeding episodes in patients with severe haemophilia A without inhibitors.
Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A
Severe Haemophilia APatients with severe Haemophilia A need prophylactic factor VIII to reduce their risk of joint and soft tissue bleeds and to prevent or reduce joint damage. It is common practice to give enough factor VIII to maintain the trough level above 1% of normal and this has been supported in retrospective studies. The amount of factor VIII required to maintain this trough level varies markedly between patients because their factor VIII half lives are different. This study will assess the role of regular pharmacokinetic (PK)monitoring and dose adjusted factor VIII to establish whether this is a more cost effective way of giving treatment and whether it is feasible in routine clinical practice. Patients will be treated for 6 months with their standard factor VIII regimen and followed up to establish their bleed frequency. They will then receive pharmacokinetic adjusted factor VIII to maintain a trough above 1.5% for a year and their bleed rate compared to standard treatment. If they have increased break through bleeds their factor VIII will be increased to maintain a trough of 3%.
An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor...
Hemophilia AThe primary objectives of the study are to evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in the Prevention and Treatment of Bleeding in patients with hemophilia A. The secondary objectives are to evaluate the efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in the prevention and treatment of bleeding episodes, to investigate the quality of life in patients who used the FRSW107.
Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With...
Hemophilia BNonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.
Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation...
Hemophilia AThe primary objectives of the study are to further evaluate the efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in adolescent and adult patients with hemophilia A.
Early Prophylaxis Immunologic Challenge (EPIC) Study
Hemophilia AThe purpose of the study was to assess if a once-weekly prophylactic regimen of 25 IU/kg ADVATE started at or before 1 year of age and before the onset of a severe bleeding phenotype (ie, joint bleeding), together with the minimization of immunological danger signals, can reduce the incidence rate of inhibitor formation in PUPs with severe and moderately severe hemophilia A.
Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in...
Severe Hemophilia BThe primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B. The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.