Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients

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Investigation of the Pharmacokinetics of NNC172-2021, at Two Different Dose Levels, in Healthy Japanese Subjects Handheld Ultrasound (HHUS) for Home Use in Hemophilia A Post Marketing Surveillance Study for ADYNOVATE in South Korea Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes. Non-interventional Observation of Practical Implementation, Efficacy and Safety of Continuous Infusion With KOGENATE Bayer in Surgery Survey of Inhibitors in Plasma-Product Exposed Toddlers Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII Emicizumab PUPs and Nuwiq ITI Study Biomarkers Serum Collection Methodology Pilot Study INdividualized ITI Based on Fviii(ATE) Protection by VWF ADYNOVATE Drug Use-Results Survey Covalent Tolerance Induction to Factor VIII-Prediction of Inhibitors in Hemophilia Individualizing Hemophilia Prophylaxis Using Thromboelastography Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries German Pediatric Hemophilia Research Database Intra-subject Variability Following Administrations of Activated Recombinant Human Factor VII in Haemophilia Patients in a Non-bleeding State Investigating Safety and Pharmacokinetics of NNC 0128-0000-2011 Compared to NNC 0128-0000-2021 in Healthy Male Subjects Post-marketing Safety Surveillance of NovoSeven® in Patients With Haemophilia and Inhibitors by Means of the UK Haemophilia Database Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds Prospective Biomarkers of Bone Metabolism in Hemophilia A Effekt-2 – Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis Evaluation of a Standardized Protocol for Thrombin Generation Assay BAX 855 Pediatric Study Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ MOTHIF II : THERAPEUTIC MANAGEMENT AND USE OF CLOTTING FACTORS IN HEMOPHILIA A & B IN FRANCE II Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa Study Evaluating Safety Of Patients Switching To ReFacto AF In Usual Care Settings Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis Pharmacokinetic Study of ADVATE Reconstituted in 2 mL Sterile Water for Injection Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia BAX 855 PK-guided Dosing Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand’s Disease in Upper Egypt China ADVATE PTP Study ADVATE Post Authorization Safety Surveillance BAY94-9027 PK Study Comparing to Another Long Acting Product Efficacy and Safety of IL-11 in DDAVP Unresponsive Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia BAY14-2222 Continuous Infusion in Surgeries Study Evaluating Liver Transplantation in Haemophilia Patients in Spain Relative Bioavailability Of Two Formulations Of Moroctocog Alfa (AF-CC) A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health International PMS Study – KOGENATE Bayer Kogenate FS Regulatory Post-Marketing Surveillance Comparison of Ideal vs. Actual Weight Base Factor Dosing BAY14-2222 Prophylaxis and Joint Function Improvement (Adults) Impact on French Physician’s Haemophilia Treatment Management Decision Based on Systematic Joint Examination A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI EffeKt Taiwan- Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Taiwan Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ? Could Physical Activity Help to Counteract the Blood Haemophilia Disturbance? A Pilot Study. Epidemiology and Immunology of Hemophilia A Inhibitors Hemophilia Inhibitor Previously Untreated Patient Study Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay Non Neutralizing Antibodies: Prevalence and Characterization Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale Project to Update the Study of Congenital Haemophilia in Spain Trial to Evaluate the Pharmacokinetics and Safety Profile of BAY94-9027 Following Single and Multiple Dose Administration Global Hemostatic Methods in Hemophilia and Von Willebrand’s Disease Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs) BAX 826 Dose-Escalation Safety Study Prophylaxis Versus On-demand Therapy Through Economic Report BAY81-8973 Pediatric Safety and Efficacy Trial Pharmacokinetics of rFVIIIFc at Two Vial Strengths Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A Quality of Life Study of Helixate NexGen Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia World Bleeding Disorders Registry Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII BAX 802 in CHA With Inhibitors BAX 855 Dose-Escalation Safety Study Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980 A Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and Venipuncture Bleeding Symptoms of Carriers of Hemophilia A and B Comparative Pharmacokinetic Study of Two Different Strengths of BAY14-2222 Recombinant Factor VIIa (NovoSeven) on Restoring Coagulation Activation GAS-Hem Feasibility Study BAX 855 Continuation Colorado Adult Joint Assessment Scale (CAJAS) Validation Personalized Prediction of Tolerance and Immunogenicity in Hemophilia ATHN 2: Factor Switching Study Evaluation of Clot Stability Induced by Solulin: Evaluation of New Solulin Mutants Lacking Protein C Activation Capacity Study Evaluating Refacto For Pharmacovigilance Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A Russian Kogenate Pediatric Study Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews Cooperative Study of Factor VIII Inhibitors EFFEKT – Efficacy and Safety of Long-term Treatment With KOGENATE Bayer/FS A Study to Evaluate the Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection ATHN 8: PUPs Matter Study Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor – RES.I.S.T. Naive A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World Study of Biostate for Treatment of Children With Hemophilia A Complicated by Antibody Development Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A Single Dose Study in Subjects With Severe Hemophilia A Comparing Pharmacokinetic Parameters for BAY81-8973 and Advate Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A Safety and Dose Escalation Study of an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia A Participants Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial Virtual Reality for Hemophilia Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015 Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A Study Evaluating BDDRFVIII and ReFacto AF in Hemophilia A A Study to Investigate the Safety and Efficacy of Optivate® in Children Under 6 Years of Age With Haemophilia A. Pharmacokinetics Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A. Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects Study Evaluating ReFacto AF in Severe Hemophilia A A Multi-centre, Comparative, Double Blind, Randomised Cross-over Trial Investigating Single Dose Pharmacokinetics and Safety of Turoctocog Alfa Pegol From the Pivotal Process and Turoctocog Alfa Pegol From the Commercial Process in Patients With Severe Haemophilia A An Open Study to Investigate the Safety and Efficacy of Optivate® in Severe Haemophilia A Patients. A Trial Investigating the Pharmacokinetics and Pharmacodynamics of rFVIIa in Patients With Haemophilia A or B With or Without Inhibitors Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A Subjects Post-Marketing Surveillance (Use-results Surveillance) With Esperoct® A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects Study of Recombinant Porcine Factor VIII (FVIII) in Hemophilia and Inhibitors to FVIII Comparison of the Action of Drugs in the Body and Safety of N8 and Advate® in Haemophilia A Subjects To Investigate Safety and Efficacy of NovoEight® (rFVIII) During Long-term Treatment of Haemophilia A in Japan Evaluating the Pharmacokinetics of NovoEight® (Turoctocog Alfa) in Relation to BMI in Subjects With Haemophilia A Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A Weight-based Dosing in Hemophilia A A Single Dose Trial of Recombinant Factor VIII (N8) in Japanese Subjects With Haemophilia A: An Extension to Trial NN7008-3543 Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophila A (Post-marketing Investigation) Investigation of the Pharmacokinetics of Turoctocog Alfa in Subjects With Haemophilia A Study Investigating Novoeight®/NovoEight® (Turoctocog Alfa) in Mexican Haemophilia A Patients Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A A Single Dose Trial of NNC 0155-0000-0004 in Patients With Haemophilia A Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A. A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia The Effect of Patient Counseling on Adolescent Hemophilia Patient Compliance With Bleeding Logs Early Prophylaxis Immunologic Challenge (EPIC) Study Exit Interviews to Assess Impact of Infusion Frequency in Hemophilia A Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 PK Driven Prophylaxis for Hemophilia A Personalized Medicine for Canadians With Hemophilia Lentiviral FVIII Gene Therapy Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A Efficacy and Safety of Recombinant Factor VIII (GreenGene) in Patients With Hemophilia A Study to Compare How the Body Distributes and Excretes the Drugs Jivi (BAY 94-9027) and Adynovi in Patients With Severe Hemophilia A (Bleeding Disorder Resulting From a Lack of Blood Clotting Factor VIII) Individualized Prophylaxis for Severe Hemophilia A Children A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients. Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A. An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc – Von Willebrand Factor – XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A) Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults Pharmacokinetic Study of ADVATE 3000 IU in Previously Treated Patients With Severe Hemophilia A Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients Exercise Versus DDAVP in Patients With Mild Hemophilia A A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A Study of rFVIIIFc for ITI in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study) Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A. Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery Safety, Efficacy and Pharmacokinetics of GreenGene™ F to Previously Treated Patients With Severe Hemophilia A Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures The Hemophilia Ultrasound Project An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A Safety and Efficacy Extension Study of GreenGene™ F in Previously Treated Patients Diagnosed With Severe Hemophilia A Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A A Gene Transfer Study for Hemophilia A PHYSICAL ACTIVITY AND FVIII CLEARANCE: RELEVANCE FOR PERSONALIZED THERAPY IN SEVERE HAEMOPHILIA A (PHYSEMO) Prophylaxis Versus on Demand Treatment for Children With Hemophilia A Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A Long-Term Safety and Efficacy of SPK-8011 in Males With Hemophilia A An Open Study to Investigate the Safety and Efficacy of Optivate® in Haemophilia A Patients Undergoing Surgery. Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD) Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients Safety and Mode of Action of a Single Dose and Multiple Doses of Long Acting Activated Recombinant Human Factor VII in Patients With Haemophilia A and B Gene Therapy for Haemophilia A. Health Related Quality of Life of Youth and Young Adults With Hemophilia A Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment Web-based Application for the Population Pharmacokinetic Service – Phase 1 Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry & Jivi: A Nested Study Within an Existing Registry Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A Validation Study of a cOmputer Pharmacokinetic Tool to assIst in the Follow up Care of haeMophilia A Patients Joint Status in Subjects With Severe Hemophilia A in Relation to Different Treatment Regimens Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A Treatment of Hemophilia A Patients With FVIII Inhibitors Comparison of Prophylaxis and On-demand Treatment in Children With Moderate to Severe Hemophilia A An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A Drug Use Investigation of Kovaltry in Hemophilia A Patients Safety, Immunogenicity and Hemostatic Efficacy of PEGylated Recombinant FVIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years of Age With Severe Hemophilia A 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants Individually Tailored Prophylaxis in Patients With Severe Hemophilia A Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A Clot Formation and Clot Stability in Patients With Severe Haemophilia A Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A RIXUBIS PMS India (RIXUBIS PMS) Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A Gene Therapy Study in Severe Haemophilia A Patients Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients Study Evaluating Inhibitor Specificity in Hemophilia A A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors Study of Biostate® in Children With Hemophilia A Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A Extension at 10 Years of the: “Observational Study Evaluating Efficacy and Costs of 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Hemophilia A or B Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study Socialization of Adult Men With Congenital Hemophilia A or B Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment Inhibitor Development in Patients With Hemophilia A Undergoing Surgery Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Patients With Severe Hemophilia A or B Post-marketing Surveillance (Use Result Surveillance) With Refixia® Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency The Effectiveness of Recombinant Fusion Protein Linking Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients Switching From Previous Factor IX Treatment The Safety and Tolerability of SerpinPC in Healthy Men and in Men With Severe Blood Disorders (Haemophilia A and B) IMMUNINE Pre-Treatment Study Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B Study of Ataluren (PTC124®) in Hemophilia A and B Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S) Study Evaluating Approach to Treatment of Haemophilia A and B in Spain Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia Safety 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Mutation p.Ile112Thr : Discrepancy Between Factor IX Level and Bleeding Phenotype LTFU for Gene Transfer Subjects With Hemophilia B A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects Adverse Event Data Collection From External Registries on Nonacog Beta Pegol Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients Lentiviral FIX Gene Therapy An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B Understanding Hemophilia A and B Drug Dosage Administration Patterns Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B Phase I/IIa Study of FIXFc in Hemophilia B Patients Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B. 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Brief Title

Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients

Official Title

Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients

Brief Summary

      Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients
      Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does
      different factor VIII dosage effect outcome of hemophilia treatment in term of breakthrough
      bleeding, number of hospital stay and day-off from school? Type Research: Clinical research

      Study design: Single center clinical trials

      Concise methodology:

        1. Study Population: Children with hemophilia A who received treatment at KCMH from May
           2015 to March 2016 will be enrolled in this study. The consent will be obtained before
           the study.

        2. Observation and measurement:

             1. History, interesting clinical data and laboratory data will be recorded in Clinical
                record Form (CRF)

             2. Measurement:

           i. Complete blood count (CBC), Factor VIII level, Factor VIII inhibitor level ii. Number
           of breakthrough bleedings, number of hospital stay and day-off from school iii. Joint
           score from Hemophilia Joint Health Score 2.1 iv. EQ-5D-5L quality of life assessment
           score

        3. Data analysis: The p-value of less than 0.05 will be considered statistically
           significant. Mann-Whitney test will be used to test correlation of these variables (CBC,
           Factor VIII level, Factor VIII inhibitor level, Number of breakthrough bleedings, number
           of hospital stay, day-off from school, Hemophilia Joint Health Score 2.1, EQ-5D-5L
           quality of life assessment score) Sample size: 16 patients

      Potential impacts:

      The outcomes of different factor VIII concentrate dose between 15-20 U/kg/dose 2 times/week
      and 35-40 U/kg/dose 1 time/week will be revealed. These outcomes include number of
      breakthrough bleeding, number of hospital stay, day-off from school, joint health and quality
      of life. The result of this study will guide further study on optimal dose and duration of
      factor VIII treatment of hemophilia A patients in the future.

Study Phase

Phase 4

Study Type

Interventional

Primary Outcome

Annualized bleeding rate per year

Secondary Outcome

 Hemophilia joint health score

Condition

Hemophilia A

Intervention

FVIII

Study Arms / Comparison Groups

 Arm 1
Description:  FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

16

Start Date

May 2015

Completion Date

March 2016

Primary Completion Date

March 2016

Eligibility Criteria

        Inclusion Criteria:

          -  Hemophilia patients with moderate (factor level 1-3%) or severe (factor level <1%)
             severity

        Exclusion Criteria:

          -  Platelet count less than 100,000 /mm3 or other bleeding tendency

          -  Hemophilia patients who have FVIII inhibitor >0.6 BU (modified Nijmogen method)

          -  Hemophilia patients who have no bleeding symptoms

Gender

All

Ages

6 Months - 20 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Darintr Sosothikul, MD, ,

Location Countries

Thailand

Location Countries

Thailand

Administrative Informations

NCT ID

NCT02727647

Organization ID

3125056

Responsible Party

Principal Investigator

Study Sponsor

Chulalongkorn University

Study Sponsor

Darintr Sosothikul, MD, Principal Investigator, Chulalongkorn University

Verification Date

October 2015