Web-based Application for the Population Pharmacokinetic Service – Phase 1

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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. 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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 Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A.” Observational Study Describing the Usual Clinical Practice Use of NovoSeven® in the Home Treatment of Joint Bleeds in Patients With Haemophilia A or B and Inhibitors Thrombin Generation Numerical Models Validation in Haemophilic Case Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A – A Continuation of Baxter Study 060101 Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients – A Continuation of Clinical Study 069901 Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a “Shuttle”. A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A Joint Health Study Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors UK – EHL Outcomes Registry Study to Establish Bioequivalence of ReFacto AF (BDDrFVIII) With Advate (FLrFVIII) in Hemophilia A Evaluation of Long-term Safety of ADYNOVI/ADYNOVATE (Antihaemophilic Factor [Recombinant] PEGylated, Rurioctocog Alfa Pegol) in Patients With Haemophilia A – An ADYNOVI/ADYNOVATE Post-Authorisation Safety Study (PASS) Platelet Function in Patients With Hemophilia A Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study) Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT® Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy Pharmacokinetics, Efficacy and Safety Study of IMMUNATE SD (Human Plasma-Derived Coagulation Factor VIII Concentrate) in Hemophilia A Patients Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII Dose Confirmation Trial of AAV5-hFIXco-Padua Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment Web Accessible Population Pharmacokinetics Service – Hemophilia: Sources of Variability A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6) Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B RIXUBIS Drug Use-Result Survey (Japan) A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A Extended Half Life Factor (EHF) Products For Heavy Menstrual Bleeding in Hemophilia Carriers Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery A Non-interventional Retrospective Study of the Current Treatment Practice in European Haemophilia Care BAX 326 Pediatric Study Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With 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 and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773 Study Evaluating rFIX; BeneFIX® in Hemophilia B A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B Tissue Factor Pathway Inhibitor (TFPI) and Haemorrhagic Manifestations in Haemophilia A and B Patients Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B BAX 326 (rFIX) Continuation Study Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophelia B A Factor IX Gene Therapy Study (FIX-GT) 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. A Study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Patients Undergoing Major Surgery. HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B Study Evaluating Allergic Reactions To Benefix In Hemophilia B Patients A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B Prospective Registry of European Hemophilia B Patients Receiving BeneFIX® for Usual Use A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX A Gene Therapy Study for Hemophilia B A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B Gene Therapy for Chinese Hemophilia B BeneFIX Drug Use Results Survey [All-Case Surveillance] Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF – A Swedish Cohort Study Hemophilia B Gene Therapy With AAV8 Vector Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B Post Marketing Observational Study of Reformulated BeneFIX Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B A Study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Patients Under the Age of 6 Years Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B An Open-study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Subjects Undergoing Surgery Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B BAX 326 Surgery Study in Hemophilia B Patients Registry For Patients Treated With BeneFix In Usual Care Setting In Germany Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%) Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects Study Evaluating rFIX; BeneFIX in Severe Hemophilia B Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients Post Marketing Study in Haemophilia B Patients Using Nonafact® (Human Coagulation Factor IX) A Study to Compare the Pharmacokinetics and Safety of Replenine®-VF, Replenine® or Other Factor IX in Haemophilia B Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B Study Evaluating BENEFIX in Previously Treated Patients With Hemophilia B Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China

Brief Title

Web-based Application for the Population Pharmacokinetic Service - Phase 1

Official Title

The Development of the Web-based Application for the Population Pharmacokinetic Service - Hemophilia (WAPPS-Hemo) - Phase 1

Brief Summary

      The aims of this trials are:

        1. to collect published and unpublished individual classic pharmacokinetic data (individual
           patient data from independent investigators and pharmaceutical companies)

        2. to make available population pharmacokinetic models for the concentrates derived from
           the data collected

        3. to develop a web based application intended to use the above models to calculate
           pharmacokinetic parameters for individual patients, and

        4. to test the system functionality via simulation of the use of the prototype by use of
           faked test data.
    

Detailed Description

      Study setting: All the development phases (a to c) will take place at McMaster University in
      Hamilton, Ontario, Canada. Phase d) will involve the centers selected for the subsequent
      twinned protocol, but will not use real patient data. The IT system will sit on a web
      accessible platform developed and run by the Health Information Research Unit (HiRU) at
      McMaster University.

      Participants: There are no patients taking part in this development phase. Companies and
      independent investigators will act as co-investigators and will provide already existing
      on-file individual pharmacokinetic (PK) data. Faked test data will be used for step d).

      Eligibility criteria: There are no patients taking part in this development phase, thus
      eligibility criteria will be applied to pre-existing datasets. Primary investigators or
      companies who own original pharmacokinetic data of factor VIII and factor IX concentrates
      will provide data for the creation of the model. No restriction as to country, language or
      factor concentrate will be applied.

      Interventions: There is no therapeutic intervention planned as part of this study. The study
      will result in a system intended to be used to produce patient level estimates potentially
      useful to modify the treatment regimens of the patients. The phase 1 "activities" portion the
      WAPPS-Hemo project include the following:

        1. a systematic review to identify all the existing publications reporting pharmacokinetic
           data on factor VIII and IX. Only articles reporting individual classic pharmacokinetic
           data will be included. The data extracted will be extracted and compiled to be used to
           create the population pharmacokinetic models. Investigators and companies, fulfilling
           the eligibility criteria, will be invited to provide data and to sign a bilateral Data
           Transfer Agreement Data will be subsequently transferred to HIRU, stored in a secure
           database and coded as needed for step b).

        2. establishment of a population pharmacokinetic model by splitting the data into two sets:
           a modeling set, from which the pharmacokinetic model will be built, and a validation
           set, to be used to test the model before implementation. Initially, the population
           pharmacokinetic model will be built using only the modeling data and validated using the
           validation set through goodness-of-fit plots, bootstrapping, and visual prediction
           checks (VPC). After the model validation, the entire data set will be used to derive the
           final model for prospective use.

        3. integration of the population pharmacokinetic models into the WAPPS-HEMO web service.
           The Information Technology Support Group at HIRU will perform extensive in-house
           beta-testing using fabricated data.

        4. field testing of the website by asking centers participating in the research network to
           test the website with fabricated patient data. After successful registration, the
           centers will be provided with this data, and asked to generate records for those
           patients. We will then run Population PK estimates, pilot the reporting process, gather
           feedback on the testing process and troubleshoot any issues that come up. As well, we
           will gather focus group feed-back and modify the service accordingly.

      Outcomes: The WAPPS-Hemo project will result in:

        1. a repository of all published and unpublished dataset of concentration-time point for
           selected population of hemophilia patients studied after administration of factor
           concentrates. Published data will be summarized in a systematic review and submitted for
           publication.

        2. population pharmacokinetic models for the factor concentrates for which
           concentration-time point for population of hemophilia patients. The model development
           will be described in a scientific report. The extended version will be available as part
           of the study documentation, while a synthetic version will be submitted for publication.

        3. availability of the web application to be used in the phase d) of the study.

        4. availability of the prototypal web application to be used in the subsequent phase 2 of
           the WAPPS-Hemo protocol.

      Participant timeline: We will require 12 months to gather the data, build the model, and
      create the prototypal web service.

      Sample size: We expect to be able to derive models for concentrates for which we have 50 or
      more pre-existing individual PK data. We expect to have data available for 4 or more
      different factor concentrates.

      Recruitment: No patients will be recruited for this phase of the overall WAPPS program. Only
      already published data or data on file will be used to derive the model and set up the
      system. All the investigator of the studies identified via the literature search will be
      contacted and invited to provide data. All the drug manufactures active in the hemophilia
      field will be invited to provide on-file pharmacokinetic data.

      Data collection methods: Data will be collected from the primary investigators and companies
      that have agreed to provide original pharmacokinetic data on factor VIII and factor IX
      concentrates.

      Website development A cluster of fully resilient HP servers (Windows web servers in Network
      load balancing configuration for the hosting the site, Microsoft SQL for the database, and
      Windows server for the NONMEM software), located in two different buildings, will support the
      system platform. The system will also incorporate fully mirrored hard disks, and redundant
      Https connection. The administrative website and backup database will be accessible only over
      a virtual private network (VPN). The system will be available 24/7 with a downtime of < 0.01
      of total up time. All WAPPS related url (.com, .org, .ca, etc have been blocked immediately
      at project funding notification - August 2013). All the needed licenses will be acquired,
      including a single site license for the Icon NONMEM population PK software. The website,
      database access interface, back-end NONMEM interface will be programmed in dot.net.

      The system will be built ready to transfer information in a format compatible with the
      hypertext languages used to embed knowledge into Electronic Medical Records (e.g., Arden
      syntax and SEBASTIAN framework, http://www.openclinical.org/gmmsummaries.html), to facilitate
      potential interface with the electronic medical record, database, and clinical trial case
      report form at a later stage.

      Data management: Data from the primary investigators and companies will be stored on a secure
      McMaster University server and only the research team will be able to access it. As the data
      provided to the research team will already be anonymized, there will be no need for further
      anonymization. Notwithstanding in this phase of the study, only already existing data from
      previous publications and on-file company-owned database will be used. We are providing a
      description of the data management for the subsequent phase of the study (WAPPS phase 2,
      protocol: The Implementation, User Testing and Validation of the Web-based Application for
      the Population Pharmacokinetic Service - Hemophilia (WAPPS-HEMO)). Users will be allowed to
      access and use the system only after a moderated registration process. The head of the
      partner research center will need to go through a registration process, and after validation
      by the McMaster team, she/he will receive credentials to access the website. She/he will
      subsequently be able to authorize other users from her/his center to access the system. Each
      individual user will have unique access credentials, and will be authorized to manage
      patients from the center she/he belong to. Only authorized users will be able to create and
      access patient record, and/or input the information required for the PK assessment. The
      identification of patients will happen via a combination of three different keys, a minimum
      of two of which should be provided in case of subsequent access to the record. The three
      identifiers will be:

        1. the WAPPS unique identifier, generated by the system at patient's record creation, and
           reported on each page and printout relative to that patient,

        2. the patient birthdate, in the format "YYYY-MM-DD", or

        3. a local patient identifier. As to c, wherever allowed by the local center privacy
           regulation, we will recommend to use a string composed by
           "LASTNAME,FIRSTNAME,LOCAL_CENTER_ID". For centers whose relevant regulation does not
           allow the use of this information, any combination of alphanumeric codes will be
           accepted. It will then be the responsibility of the local center to track the local
           identifier in the patient file.

      The data above will be provided by the user creating a record for the patient on the system.
      For subsequent re-access of any user from the same center to any specific patient record from
      the center, the system will display a list of the registered patient, using the identifier
      provided (i.e. a) and b) mandatorily, c) with the information provided by the center. The
      user will be then able to access a specific patient record for the allowed operation (as
      described in the study protocol for step 2).

      Statistical methods: The NONMEM 7 version 1.0 (ICON Development Solutions, Ellicott City, MD)
      will be used with an Intel Fortran compiler (version 12) for population pharmacokinetic
      modeling. The statistical program R (version 2.15.0, R Foundation for Statistical Computing,
      Vienna, Austria) will be used to compile the NONMEM sets and generate the graphical
      representations of the models. Perl Speaks NONMEM (PsN, version 3.5.3) and PsN and Xpose 4
      will be used, respectively, for the bootstrapping and the VPC validation tests.

      In order to estimate the population pharmacokinetic parameters, we will use a first-order
      conditional estimation with interaction method (FOCEI). Exponential function will be used to
      model inter-individual variability (IIV), and the inclusion of IIV terms on pharmacokinetic
      parameters will be tested in sequential order. IIV addition with the most significant
      objective function value (OFV) reduction will enter the model first. Inter-assessment
      variability (IAV) will also be evaluated with baseline pharmacokinetics at first available
      assessment as assessment 1 and repeat pharmacokinetic profiling at the subsequent available
      time as assessment 2. Any residual errors found in the data will be modeled as combined
      additive and proportional errors.

      Potential demographic or clinical factors potentially affecting the pharmacokinetics
      (including the type of concentrates for the basic overarching model to be used for products
      for which product-specific data will not be available and the characteristics of the
      laboratory technique used to measure the plasma factor level concentration) will be screened
      using plots of IIV versus the covariates. When the covariates are continuous, scatter plots
      of ETA (IIV code used in NONMEM) versus the covariates will be used to determine functional
      relationships. These scatter plots will be overlaid with a non-parametric locally weighted
      smoother LOESS line. When the covariates are categorical, potential differences between
      groups will be identified using box and whisker plots. Any possible influence by continuous
      covariates should be suggested by a clear trend of positive or negative slopes and
      outstanding correlation coefficients. Influence from categorical covariates will be suggested
      by pronounced changes between the mean values of each group. In the covariate modeling, a
      full stepwise forward addition (P<0.005) and backward elimination (P<0.001) procedure will be
      used.

      The model selection will also be validated with goodness-of-fit plots, including observation
      (OV) versus population prediction (PRED), OV versus individual prediction (IPRED),
      conditional weighted residual (CWRES) versus TIME and PRED plots. Other diagnostics, such as
      parameter precision, ETA, and CWRES distribution and shrinkage, will also be used to choose
      the proper population pharmacokinetic model.

      Methods: Monitoring

      Data monitoring: For step I of the WAPPS protocol, there is no need to plan a data monitoring
      process. Indeed, all data will be validated as part of model derivation, and used as such in
      the rest of the study. Congruency checks will be performed on user's input data and PK
      estimations in the subsequent phase 2 of the study and subsequently in routine use of the
      interface.

      Harms: During the development phases (a to c), we will not offer the web application beyond
      the boundaries of the developing research unit. The website will be made available to the
      centers in the research network during the testing phase d) (using only fabricated data) and
      the subsequent phase 2 of the protocol. We will clearly identify the prototypal and
      experimental use of the system in both the Research Agreements and the website page as a
      "Research tool not yet ready for clinical practice". A clear disclaimer will indicate that
      "Any use of the results of the population pharmacokinetic estimates in the care of individual
      patients should not be considered part of the project in this phase".
    


Study Type

Observational [Patient Registry]


Primary Outcome

Population pharmacokinetic model(s) performance measure

Secondary Outcome

 Usability testing of the web-interface

Condition

Hemophilia A

Intervention

Population pharmacokinetic estimation

Study Arms / Comparison Groups

 Population pharmacokinetic estimation
Description:  Patients with severe or moderate hemophilia A or B, providing sparse data for population PK estimation

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

Other

Estimated Enrollment

400

Start Date

January 2015

Completion Date

June 2019

Primary Completion Date

June 2019

Eligibility Criteria

        Inclusion Criteria:

          -  Patients with severe or moderate Hemophilia A or B

          -  Informed consent to data processing

        Exclusion Criteria:

          -  Non measurable plasma factor VIII due to high titre inhibitor
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Alfonso Iorio, MD,PhD, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT02061072

Organization ID

13-351-D


Responsible Party

Sponsor

Study Sponsor

McMaster University

Collaborators

 Canadian Hemophilia Society

Study Sponsor

Alfonso Iorio, MD,PhD, Principal Investigator, McMaster University


Verification Date

May 2018