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 ?

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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 ? 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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. 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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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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 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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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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

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 ?

Official Title

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 ?

Brief Summary

      In the context of hemophilia, it is well know that the level of factor VIII alone does not
      reflect the clinical phenotype of the patients in an accurate way. At equal factor VIII
      levels, certain patients will bleed more than others.

      The thrombin generation test (TGT) is a test that seems to provide a better prediction of the
      overall hemostatic status of an individual patient. In a previous study, the investigators
      have established normal reference values of the thrombin generation curve in children aged 6
      months to 16 years and adults. The goal was to evaluate the use of this test in different
      clinical contexts and in severe hemophilia patients in particular. A pilot study showed that
      the patients having a thrombin generation <150 had a severe phenotype, whether those who
      received an appropriate prophylaxy had a thrombin generation superior to 150.

      Moreover, the investigators now have access to a software tool that allows them to
      individually determine the pharmacokinetic profile of the factor VIII injected to each
      patient. The factor VIII concentration is measured at injection and 30 minutes, 1 hour, 2
      hours and 24 hours afterwards. The introduction of these concentrations in the software
      allows to obtain the half-life of factor for a given patient, the maximum peak, and the
      minimum factor level (though level). The injected dosis might be sufficient (disappearance of
      substantial diminution of the bleedings) or unsufficient (persisting bleeding) for a given
      patient.

      This study aims:

        -  to measure the pharmacokinetic profile of factor VIII by two different methods, the
           time-based method and the chromogenic method

        -  to correlate the results with the TGT results obtained at the same time points and
           determine which method gives the best correlation

        -  to link the clinical symptomatology (improved symptomatology or not) with the TGT
           results

        -  to determine which minimal TGT result is linked to a minimal bleeding rate

        -  to adapt the prophylactic dosis of the patient in a personalized way.

Study Type

Interventional

Primary Outcome

Factor VIII blood concentration - chronometric method

Condition

Hemophilia

Intervention

Chronometric method

Study Arms / Comparison Groups

 hemophilia
Description:  Patients with severe hemophilia (or moderate hemophilia if presence of hemorrhages) under prophylaxy and subjected to a pharmacokinetic profile of factor VIII

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

Device

Estimated Enrollment

50

Start Date

January 2017

Completion Date

July 2020

Primary Completion Date

July 2020

Eligibility Criteria

        Inclusion Criteria:

          -  Patients with severe or moderate hemophilia, on prophylaxis, and suffering from
             bleedings.

        Exclusion Criteria:

          -  Patients with difficult venous access

          -  Patients who have had surgery or trauma in the month before, patients with acute
             disease (infection, inflammation)

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Anne Demulder, MD, 024773990, [email protected]

Location Countries

Belgium

Location Countries

Belgium

Administrative Informations

NCT ID

NCT02803502

Organization ID

CHUB-PK TGT

Responsible Party

Principal Investigator

Study Sponsor

Brugmann University Hospital

Study Sponsor

Anne Demulder, MD, Principal Investigator, CHU Brugmann

Verification Date

November 2017