Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens

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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 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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

Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens

Official Title

Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens

Brief Summary

      This trial is designed to assess if there is evidence of subclinical joint bleeding on
      MRI/X-Ray in adults with severe Haemophilia A while on standard and/or pharmacokinetically
      tailored prophylaxis regimens. Participants with severe Haemophilia A will have longitudinal
      MRI and XRay imaging of their elbows, ankles and knees at 0, 6 and 18 months while on
      standard ( 0-6 months) and then pharmacokinetically tailored (7-18 months) recombinant Factor
      VIII prophylaxis.

Detailed Description

      Subclinical joint bleeding (SJB) in Haemophilia may cause early and progressive joint damage.
      Clinical haemarthrosis is a traditional outcome measure in Haemophilia trials but may not
      always correlate with the degree of arthropathy. Even in the absence of haemarthrosis,
      abnormalities may be detected on MRI. MRI offers greater sensitivity than physical
      examination for early joint damage and use of the International Prophylaxis Study Group
      (IPSG) score allows standardisation across clinical trials. Early awareness of haemophiliac
      arthropathy can prompt intervention with physiotherapy, specific exercise programmes,
      optimization of prophylaxis and orthotics to improve overall joint outcomes.

      The time spent with Factor VIII (FVIII) levels <0.01 IU/mL is a known risk for bleeding.
      Conventional prophylaxis schedules follow a weight based regimen and are titrated according
      to clinical bleeds. FVIII pharmacokinetics (PK) may be used to optimise FVIII prophylactic
      regimens, maintaining adequate FVIII trough levels. This offers the possibility to not only
      tailor individual regimens but also may potentially reduce the rate of clinical and
      subclinical joint bleeding.

      This is a national, investigator led clinical trial investigating the feasibility of PK
      tailored prophylaxis in adults with severe Haemophilia A. This trial will prospectively and
      longitudinally assess SJB and joint health in Irish adults with severe Haemophilia A.

      SJB will be compared while on standard (weight based, 20-40 IU/kg) and PK tailored
      prophylaxis(maintaining trough FVIII > 0.015 IU/mL). This is a crossover study will
      participants spending months 0-6 on standard prophylaxis and then changing over to PK
      tailored dosing for months 7-18. A comprehensive joint assessment involving bleed history,
      clinical examination, physical activity, specialist physiotherapy review, X-rays and MRI
      scanning of bilateral ankles, knees and elbow will be performed at months 0,6 and 18.
      Haemophilia Joint Health Score (HJHS), International Physical Activity (IPAQ) and EuroQoL
      5-Dimensions (EQ5D) Questionnaires will also be performed at these three timepoints.

      Clinical bleeds and FVIII usage will be recorded throughout the trial using the investigators
      Home Scan system, a smart phone application that allows patients to log factor VIII usage.

      Results will be compared between both arms and between participants on primary and secondary
      prophylaxis. Information on those with naïve joints versus established arthropathy will be
      compared.

      Due to the relative rarity of severe Haemophilia A the investigators plan to recruit 20
      patients in total. All patients will act as their own control, crossing over from standard to
      PK tailored prophylaxis with joint assessments prior to crossover to allow comparison of the
      two regimes.

Study Phase

Phase 4

Study Type

Interventional

Primary Outcome

Number of subclinical haemarthroses

Secondary Outcome

 EQ5D QoL score

Condition

Severe Haemophilia A

Intervention

ADVATE [Antihemophilic Factor (Recombinant)]

Study Arms / Comparison Groups

 Standard prophylaxis
Description:  Advate [Antihemophilic Factor(Recombinant)] 20-40 IU/kg 5-7 infusions per 14days

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

20

Start Date

April 2014

Completion Date

October 2016

Primary Completion Date

May 2016

Eligibility Criteria

        Inclusion Criteria:

          -  Male patients with severe Haemophilia A (baseline Factor VIII level of <0.01 IU/mL)

          -  Age 18 years and above

          -  Patients taking any regular prophylactic regimen (defined as regular factor VIII
             infusions, at least 5 times a fortnight, with the aim of minimising haemarthroses and
             other clinically significant bleeds).

          -  Low titre inhibitors, past history of an inhibitor, abnormal liver function, drugs
             that interfere with haemostasis and low Cluster of Differentiation 4 (CD4) counts are
             allowed.

        Exclusion Criteria:

          -  Presence of a target joint on prophylaxis (defined as 3 bleeds into one joint, during
             a 6 month period, during the last year).

          -  The occurrence of more than 3 haemarthroses in the last year that required more than 2
             infusions to resolve.

          -  Patients with a learning disability or dementia

          -  Prisoners

          -  Adults who are unconscious/unable to give informed consent

          -  Participants with a pacemaker or implanted medical devices which are unsuitable to
             have a MRI will be excluded from the MRI scans during the trial but may proceed with
             other components.

Gender

Male

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Niamh M O'Connell, PhD, FRCPath, +35314162142, [email protected]

Location Countries

Ireland

Location Countries

Ireland

Administrative Informations

NCT ID

NCT02314325

Organization ID

2013-003240-23

Responsible Party

Principal Investigator

Study Sponsor

St. James's Hospital, Ireland

Collaborators

 Baxter BioScience

Study Sponsor

Niamh M O'Connell, PhD, FRCPath, Principal Investigator, St. James's Hospital, Dublin

Verification Date

December 2014