Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers

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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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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

Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers

Official Title

Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers

Brief Summary

      The purpose of this study is to determine how female hemophilia A carriers respond to a
      medication called DDAVP (Desmopressin).

Detailed Description

      DDAVP (Desmopressin) is commonly used in the treatment of persons with bleeding disorders
      such as hemophilia, von Willebrand disease, or qualitative platelet disorders to help them
      clot better. The investigator wants to assess the increase in the subjects' clotting factors
      in response to intravenous DDAVP (Desmopressin) and the levels of these internal clotting
      factors will be measured at different times after the medication is given. The investigator
      will compare the response to DDAVP (Desmopressin) in adult hemophilia A carriers to women
      with a diagnosis of qualitative platelet dysfunction.

Study Phase

Phase 1

Study Type

Interventional

Primary Outcome

Percentage of subjects that achieve and sustain >50% increase in Factor VIII antigen levels

Secondary Outcome

 Change in the time-course response of Factor VIII antigen levels

Condition

Hemophilia A

Intervention

Desmopressin

Study Arms / Comparison Groups

 Hemophilia A carriers with mild mutation
Description:  Hemophilia A carriers with a mild type mutation will be given a single intravenous dose of 0.3mcg/kg of DDAVP (Desmopressin).

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

2

Start Date

July 2015

Completion Date

June 15, 2018

Primary Completion Date

June 15, 2018

Eligibility Criteria

        Inclusion Criteria:

        Inclusion criteria for hemophilia A carriers:

          -  Females 18-60 years of age at time of enrollment

          -  Genetically verified or obligate hemophilia A carrier (mother of 2 boys with
             hemophilia A, daughter of a father with hemophilia A or mother of a son and another
             male relative with hemophilia A)

          -  To stratify patients by carriage of mutation type 10 hemophilia carriers of mild
             mutations that are predicted to lead to reduced FVIII secretion, protein stability or
             thrombin cleavage site interference and 10 hemophilia carriers of severe mutations
             that lead to predicted negative cross reactive material will be selected. Predicted
             FVIII function of the mutation will be verified by EAHAD (European Association for
             Haemophilia and Allied Disorders) Coagulant Factor Variant Database at
             www.eahad-db.org)

          -  Weight >40kg to ensure volumes of blood to be drawn are within accepted safe range

        Inclusion criteria for non-hemophilia A carriers (Females with mild qualitative platelet
        dysfunction):

          -  Females 18-60 years of age at time of enrollment

          -  Whole blood or platelet rich plasma lumiaggregometry consistent with reduced
             aggregation to at least 1 agonist on at least one occasion (excluding evidence of
             Glanzmanns Thrombasthenia or Bernard Soulier Syndrome) or determined by primary
             hematologist as having a qualitative platelet disorder

          -  Age-matched by 10 years to carrier enrolled

          -  Weight >40kg to ensure volumes of blood to be drawn are within accepted safe range

        Exclusion Criteria:

          -  Personal history of concomitant bleeding or clotting disorder

          -  Cardiac condition that requires the daily use of Aspirin or Clopidogrel

          -  Inability to comply with fluid restriction protocol for 24 hours following
             Desmopressin (DDAVP)

          -  Personal history of a myocardial infarction, renal or hepatic insufficiency or
             epilepsy

Gender

Female

Ages

18 Years - 60 Years

Accepts Healthy Volunteers

No

Contacts

Robert Sidonio, Jr., MD, ,

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT02506023

Organization ID

IRB00080329

Responsible Party

Principal Investigator

Study Sponsor

Emory University

Study Sponsor

Robert Sidonio, Jr., MD, Principal Investigator, Emory University

Verification Date

July 2018