Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

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Hemophilia B
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Experience in Ivory Coast. 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Joint Outcome Study A Single Dose Trial Investigating Safety and Local Tolerability of Ascending Doses of Long Acting Activated Recombinant Human Factor VII in Healthy Male Volunteers Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced) Study Evaluating The Safety Of Xyntha In Usual Care Settings Impact of Pain on Functional Impairment and Quality of Life in Adults With Hemophilia Outcomes of Prophylaxis and On-demand Treatment for Severe Hemophiliacs and Collection of Baseline Data in Hemophiliacs in Taiwan Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age Biomechanical Determinants and Patterns Associated to the Pathophysiological Cascade of Ankle Arthropathy in Children With Haemophilia: Non-invasive In-vivo Measurement of Foot Joints in Children With Haemophilia During Gait. 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Epidemiology and Immunology of Hemophilia A Inhibitors Hemophilia Inhibitor Previously Untreated Patient Study Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay Non Neutralizing Antibodies: Prevalence and Characterization Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale Project to Update the Study of Congenital Haemophilia in Spain Trial to Evaluate the Pharmacokinetics and Safety Profile of BAY94-9027 Following Single and Multiple Dose Administration Global Hemostatic Methods in Hemophilia and Von Willebrand’s Disease Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs) BAX 826 Dose-Escalation Safety Study Prophylaxis Versus On-demand Therapy Through Economic Report BAY81-8973 Pediatric Safety and Efficacy Trial Pharmacokinetics of rFVIIIFc at Two Vial Strengths Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A Quality of Life Study of Helixate NexGen Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia World Bleeding Disorders Registry Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII BAX 802 in CHA With Inhibitors BAX 855 Dose-Escalation Safety Study Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980 A Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and Venipuncture Bleeding Symptoms of Carriers of Hemophilia A and B Comparative Pharmacokinetic Study of Two Different Strengths of BAY14-2222 Recombinant Factor VIIa (NovoSeven) on Restoring Coagulation Activation GAS-Hem Feasibility Study BAX 855 Continuation Colorado Adult Joint Assessment Scale (CAJAS) Validation Personalized Prediction of Tolerance and Immunogenicity in Hemophilia ATHN 2: Factor Switching Study Evaluation of Clot Stability Induced by Solulin: Evaluation of New Solulin Mutants Lacking Protein C Activation Capacity Study Evaluating Refacto For Pharmacovigilance Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A Russian Kogenate Pediatric Study Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews Cooperative Study of Factor VIII Inhibitors EFFEKT – Efficacy and Safety of Long-term Treatment With KOGENATE Bayer/FS A Study to Evaluate the Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection ATHN 8: PUPs Matter Study Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor – RES.I.S.T. Naive A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World Study of Biostate for Treatment of Children With Hemophilia A Complicated by Antibody Development Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A Single Dose Study in Subjects With Severe Hemophilia A Comparing Pharmacokinetic Parameters for BAY81-8973 and Advate Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A Safety and Dose Escalation Study of an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia A Participants Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial Virtual Reality for Hemophilia Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015 Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A Study Evaluating BDDRFVIII and ReFacto AF in Hemophilia A A Study to Investigate the Safety and Efficacy of Optivate® in Children Under 6 Years of Age With Haemophilia A. Pharmacokinetics Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A. Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects Study Evaluating ReFacto AF in Severe Hemophilia A A Multi-centre, Comparative, Double Blind, Randomised Cross-over Trial Investigating Single Dose Pharmacokinetics and Safety of Turoctocog Alfa Pegol From the Pivotal Process and Turoctocog Alfa Pegol From the Commercial Process in Patients With Severe Haemophilia A An Open Study to Investigate the Safety and Efficacy of Optivate® in Severe Haemophilia A Patients. A Trial Investigating the Pharmacokinetics and Pharmacodynamics of rFVIIa in Patients With Haemophilia A or B With or Without Inhibitors Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A Subjects Post-Marketing Surveillance (Use-results Surveillance) With Esperoct® A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects Study of Recombinant Porcine Factor VIII (FVIII) in Hemophilia and Inhibitors to FVIII Comparison of the Action of Drugs in the Body and Safety of N8 and Advate® in Haemophilia A Subjects To Investigate Safety and Efficacy of NovoEight® (rFVIII) During Long-term Treatment of Haemophilia A in Japan Evaluating the Pharmacokinetics of NovoEight® (Turoctocog Alfa) in Relation to BMI in Subjects With Haemophilia A Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A Weight-based Dosing in Hemophilia A A Single Dose Trial of Recombinant Factor VIII (N8) in Japanese Subjects With Haemophilia A: An Extension to Trial NN7008-3543 Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophila A (Post-marketing Investigation) Investigation of the Pharmacokinetics of Turoctocog Alfa in Subjects With Haemophilia A Study Investigating Novoeight®/NovoEight® (Turoctocog Alfa) in Mexican Haemophilia A Patients Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A A Single Dose Trial of NNC 0155-0000-0004 in Patients With Haemophilia A Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A. A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia The Effect of Patient Counseling on Adolescent Hemophilia Patient Compliance With Bleeding Logs Early Prophylaxis Immunologic Challenge (EPIC) Study Exit Interviews to Assess Impact of Infusion Frequency in Hemophilia A Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 PK Driven Prophylaxis for Hemophilia A Personalized Medicine for Canadians With Hemophilia Lentiviral FVIII Gene Therapy Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A Efficacy and Safety of Recombinant Factor VIII (GreenGene) in Patients With Hemophilia A Study to Compare How the Body Distributes and Excretes the Drugs Jivi (BAY 94-9027) and Adynovi in Patients With Severe Hemophilia A (Bleeding Disorder Resulting From a Lack of Blood Clotting Factor VIII) Individualized Prophylaxis for Severe Hemophilia A Children A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients. Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A. An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc – Von Willebrand Factor – XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A) Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults Pharmacokinetic Study of ADVATE 3000 IU in Previously Treated Patients With Severe Hemophilia A Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients Exercise Versus DDAVP in Patients With Mild Hemophilia A A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A Study of rFVIIIFc for ITI in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study) Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A. Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery Safety, Efficacy and Pharmacokinetics of GreenGene™ F to Previously Treated Patients With Severe Hemophilia A Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures The Hemophilia Ultrasound Project An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A Safety and Efficacy Extension Study of GreenGene™ F in Previously Treated Patients Diagnosed With Severe Hemophilia A Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A A Gene Transfer Study for Hemophilia A PHYSICAL ACTIVITY AND FVIII CLEARANCE: RELEVANCE FOR PERSONALIZED THERAPY IN SEVERE HAEMOPHILIA A (PHYSEMO) Prophylaxis Versus on Demand Treatment for Children With Hemophilia A Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A Long-Term Safety and Efficacy of SPK-8011 in Males With Hemophilia A An Open Study to Investigate the Safety and Efficacy of Optivate® in Haemophilia A Patients Undergoing Surgery. Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD) Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients Safety and Mode of Action of a Single Dose and Multiple Doses of Long Acting Activated Recombinant Human Factor VII in Patients With Haemophilia A and B Gene Therapy for Haemophilia A. Health Related Quality of Life of Youth and Young Adults With Hemophilia A Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment Web-based Application for the Population Pharmacokinetic Service – Phase 1 Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry & Jivi: A Nested Study Within an Existing Registry Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A Validation Study of a cOmputer Pharmacokinetic Tool to assIst in the Follow up Care of haeMophilia A Patients Joint Status in Subjects With Severe Hemophilia A in Relation to Different Treatment Regimens Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A Treatment of Hemophilia A Patients With FVIII Inhibitors Comparison of Prophylaxis and On-demand Treatment in Children With Moderate to Severe Hemophilia A An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A Drug Use Investigation of Kovaltry in Hemophilia A Patients Safety, Immunogenicity and Hemostatic Efficacy of PEGylated Recombinant FVIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years of Age With Severe Hemophilia A 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants Individually Tailored Prophylaxis in Patients With Severe Hemophilia A Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A Clot Formation and Clot Stability in Patients With Severe Haemophilia A Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A RIXUBIS PMS India (RIXUBIS PMS) Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A Gene Therapy Study in Severe Haemophilia A Patients Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients Study Evaluating Inhibitor Specificity in Hemophilia A A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors Study of Biostate® in Children With Hemophilia A Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A Extension at 10 Years of the: “Observational Study Evaluating Efficacy and Costs of 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

Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

Official Title

A Dose Confirmation Study of FLT180a (Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene) in Adult Subjects With Hemophilia B

Brief Summary

      Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled
      to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the
      dose for a future Phase 3 study.

Study Phase

Phase 1/Phase 2

Study Type

Interventional

Primary Outcome

Safety and tolerability of FLT180a as assessed by incidence and severity of AEs and SAEs

Secondary Outcome

 Assessment of change in annualized bleeding rate (ABR)

Condition

Hemophilia B

Intervention

verbrinacogene setparvovec

Study Arms / Comparison Groups

 FLT180a
Description:  A single dose of FLT180a will be administered. Dose will be determined by enrollment cohort. The first 3 patients will receive 7.7 x 10e11 vg/kg. The dose in subsequent cohorts will be determined by the DMC based on review of data from the prior cohort(s).

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

Genetic

Estimated Enrollment

9

Start Date

December 6, 2021

Completion Date

November 2023

Primary Completion Date

November 2023

Eligibility Criteria

        Key Inclusion Criteria:

          -  Diagnosis of Hemophilia B with known severe or moderately severe FIX deficiency (≤2%
             normal circulating FIX activity) for which the subject is on continuous, stable and
             adequate FIX prophylaxis

          -  Have acceptable laboratory values of a) Hemoglobin ≥11g/dL; b) Platelets ≥100,000
             cells/µL; c) AST, ALT and alkaline phosphatase (ALP) ≤ upper limit of normal (ULN); d)
             Serum albumin > lower limit of normal (LLN); e) Total bilirubin ≤1.5 x ULN (except if
             caused by Gilbert's disease); f) Serum creatinine ≤2.0mg/dL.

          -  Level of neutralizing anti-AAV-S3 antibodies below the limit of the pre-established
             clinical cutoff using an in vitro transduction inhibition assay within the 4 weeks
             prior to FLT180a administration

          -  Has demonstrated ability to accurately, independently and in a timely manner enter
             bleed diary data during the lead-in study, as judged by the investigator

          -  At least 150 exposure days to FIX concentrates

          -  At least 6 months of satisfactory controlled prospective baseline data for bleeding
             events and FIX consumption data from the FLT-01 lead-in study (ECLIPSE)

        Key Exclusion Criteria:

          -  Any history of alcohol or drug dependence

          -  Presence of neutralizing anti human FIX antibodies (inhibitor; determined by the
             Nijmegen modified Bethesda inhibitor assay) at the time of enrolment or a previous
             history of FIX inhibitor

          -  Subjects at high risk of thromboembolic events

          -  Evidence of advanced liver fibrosis

          -  Prior treatment with a gene transfer medicinal product

          -  Subjects with active hepatitis B or C

          -  Serological evidence of HIV-1, not controlled with anti-viral therapy and as evidenced
             by cluster of differentiation 4 (CD4)+ counts ≤200 μL

          -  Cytomegalovirus (CMV) immunoglobulin G positive subjects who are CMV polymerase chain
             reaction (PCR) positive at screening

          -  Known coagulation disorder other than hemophilia B

          -  High sensitivity (hs) troponin-T ≥14 pg/mL during screening

          -  History of uncontrolled cardiac failure, unstable angina, or myocardial infarction or
             other acute cardiac conditions requiring clinical management in the past 6 months

          -  Planned surgical procedure within the next 12 months requiring prophylactic FIX
             treatment

          -  Known active severe infection (including documented coronavirus (COVID)-19 infection),
             or any other significant concurrent, uncontrolled medical condition

Gender

Male

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

, ,

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations

NCT ID

NCT05164471

Organization ID

FLT180a-06

Responsible Party

Sponsor

Study Sponsor

Freeline Therapeutics

Study Sponsor

, ,

Verification Date

October 2022