xml: <clinical_study>
    <!-- This xml conforms to an XML Schema at:
      https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
    <required_header>
      <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
      <link_text>Link to the current ClinicalTrials.gov record.</link_text>
      <url>https://clinicaltrials.gov/ct2/show/NCT05980377</url>
    </required_header>
    <id_info>
      <org_study_id>Pattern of Hemophilia care</org_study_id>
      <nct_id>NCT05980377</nct_id>
    </id_info>
    <brief_title>Patterns of Hemophilia Care in Assiut Children Patients</brief_title>
    <official_title>Patterns of Hemophilia Care in Patients Admitted in Assiut Children University Hospital</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Assiut University</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Assiut University</source>
    <brief_summary>
      <textblock>
        Hemophilia A and B are congenital, recessive X-linked disorders caused by lack or deficiency&#xD;
        of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease&#xD;
        depends on the reduction of levels of FVIII or FIX, which are determined by the type of the&#xD;
        causative mutation in the genes encoding the factors (F8 and F9, respectively). The hallmark&#xD;
        clinical characteristic, especially in untreated severe forms, is bleeding (spontaneous or&#xD;
        after trauma) into major joints such as ankles, knees and elbows, which can result in the&#xD;
        development of arthropathy. Intracranial bleeds and bleeds into internal organs may be&#xD;
        life-threatening. The median life expectancy was ~30 years until the 1960s, but improved&#xD;
        understanding of the disorder and development of efficacious therapy based on prophylactic&#xD;
        replacement of the missing factor has caused a paradigm shift, and today individuals with&#xD;
        hemophilia can look forward to a virtually normal life expectancy and quality of life.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        Hemophilia A and B are congenital, recessive X-linked disorders caused by lack or deficiency&#xD;
        of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease&#xD;
        depends on the reduction of levels of FVIII or FIX, which are determined by the type of the&#xD;
        causative mutation in the genes encoding the factors (F8 and F9, respectively). The hallmark&#xD;
        clinical characteristic, especially in untreated severe forms, is bleeding (spontaneous or&#xD;
        after trauma) into major joints such as ankles, knees and elbows, which can result in the&#xD;
        development of arthropathy. Intracranial bleeds and bleeds into internal organs may be&#xD;
        life-threatening. The median life expectancy was ~30 years until the 1960s, but improved&#xD;
        understanding of the disorder and development of efficacious therapy based on prophylactic&#xD;
        replacement of the missing factor has caused a paradigm shift, and today individuals with&#xD;
        hemophilia can look forward to a virtually normal life expectancy and quality of life.&#xD;
  &#xD;
        Historically, hemophilia treatment has focused on replacement of the missing coagulation&#xD;
        factor to achieve hemostasis. Treatment progressed from use of cryoprecipitate (FVIII&#xD;
        replacement in HA) or fresh frozen plasma (FFP) to plasma-derived factor concentrates,&#xD;
        allowing for early control of hemorrhage, home therapy and accessibility for the introduction&#xD;
        of prophylaxis. Contamination of factor concentrate supply with human immunodeficiency virus&#xD;
        and hepatitis C virus led to the unfortunate infection of most of the severe hemophilia&#xD;
        population in the 1980s, necessitating development of improved methods to screen and&#xD;
        inactivate viruses. These mechanisms include dry-heat, pasteurisation, solvent-detergent&#xD;
        treatment, immunoaffinity purification and nanofiltration, although some risk from emerging&#xD;
        infections remains. Recombinant factor therapies were introduced to address the concern for&#xD;
        infection transmission but raised new challenges regarding the risk of inhibitor formation.&#xD;
        The extended half-life (EHL) factor products are a result of engineering proteins for longer&#xD;
        recovery times in persons with hemophilia (PwH), to reduce frequency of dosing for prophylax&#xD;
  &#xD;
        -Factor VIII mimetic Emicizumab (Hemlibra, Genentech/Roche) is a first in-class humanised&#xD;
        bispecific antibody substitution for HA designed to function as FVIIIa by binding one arm of&#xD;
        the antibody to FIXa and the other arm to FX, accelerating activation of Fxa and propagating&#xD;
        thrombin production. The application and incorporation of this novel approach using a SC&#xD;
        antibody to prevent bleeding was outlined in a series of clinical trials (HAVEN) including&#xD;
        PwHA with inhibitors, paediatric PwHA with inhibitors and PwHA without inhibitors. The&#xD;
        bispecific antibody mechanism and half-life (˜28 days) overcomes the inhibitory alloantibody,&#xD;
        limits the need for IV access and decreases infusion frequency. In HAVEN-1, males with severe&#xD;
        HA and inhibitors aged ≥12 years received weekly prophylaxis (1·5 mg/kg) with emicizumab and&#xD;
        had an 87% reduction in overall annualized bleeding rate (ABR)&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">September 1, 2023</start_date>
    <completion_date type="Anticipated">August 1, 2024</completion_date>
    <primary_completion_date type="Anticipated">June 1, 2024</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Other</observational_model>
      <time_perspective>Other</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>care in hemophilia patients</measure>
      <time_frame>Baseline</time_frame>
      <description>Describe different aspects of care in hemophilia patients in Assiut children university hospital</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Describe complications in availability of factors</measure>
      <time_frame>Baseline</time_frame>
      <description>describe different aspects of complications and challenges in availability of factors ,bypassing agents which are used to manage hemophilia patients with inhibitors in Assiut children university hospital</description>
    </primary_outcome>
    <enrollment type="Anticipated">60</enrollment>
    <condition>Hemophilia A</condition>
    <condition>Hemophilia B</condition>
    <eligibility>
      <study_pop>
        <textblock>
          All patients diagnosed with hemophilia attending Assiut university hospital of children in&#xD;
          2 years duration&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  All patients diagnosed with hemophilia attending Assiut university hospital of&#xD;
               children&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Any patient with other bleeding disorder&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>N/A</minimum_age>
      <maximum_age>18 Years</maximum_age>
    </eligibility>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>July 29, 2023</study_first_submitted>
    <study_first_submitted_qc>July 29, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 8, 2023</study_first_posted>
    <last_update_submitted>July 29, 2023</last_update_submitted>
    <last_update_submitted_qc>July 29, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 8, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Principal Investigator</responsible_party_type>
      <investigator_affiliation>Assiut University</investigator_affiliation>
      <investigator_full_name>Sara Ahmed Abd El-Bary</investigator_full_name>
      <investigator_title>Principal Investigator</investigator_title>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Hemophilia A</mesh_term>
      <mesh_term>Hemophilia B</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: download date: ClinicalTrials.gov processed this data on September 20, 2023
  link text: Link to the current ClinicalTrials.gov record.
  url: https://clinicaltrials.gov/ct2/show/NCT05980377
  org study id: Pattern of Hemophilia care
  nct id: NCT05980377
  lead sponsor: 
  Hemophilia A and B are congenital, recessive X-linked disorders caused by lack or deficiency
        of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease
        depends on the reduction of levels of FVIII or FIX, which are determined by the type of the
        causative mutation in the genes encoding the factors (F8 and F9, respectively). The hallmark
        clinical characteristic, especially in untreated severe forms, is bleeding (spontaneous or
        after trauma) into major joints such as ankles, knees and elbows, which can result in the
        development of arthropathy. Intracranial bleeds and bleeds into internal organs may be
        life-threatening. The median life expectancy was ~30 years until the 1960s, but improved
        understanding of the disorder and development of efficacious therapy based on prophylactic
        replacement of the missing factor has caused a paradigm shift, and today individuals with
        hemophilia can look forward to a virtually normal life expectancy and quality of life.
  Hemophilia A and B are congenital, recessive X-linked disorders caused by lack or deficiency
        of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease
        depends on the reduction of levels of FVIII or FIX, which are determined by the type of the
        causative mutation in the genes encoding the factors (F8 and F9, respectively). The hallmark
        clinical characteristic, especially in untreated severe forms, is bleeding (spontaneous or
        after trauma) into major joints such as ankles, knees and elbows, which can result in the
        development of arthropathy. Intracranial bleeds and bleeds into internal organs may be
        life-threatening. The median life expectancy was ~30 years until the 1960s, but improved
        understanding of the disorder and development of efficacious therapy based on prophylactic
        replacement of the missing factor has caused a paradigm shift, and today individuals with
        hemophilia can look forward to a virtually normal life expectancy and quality of life.
  
        Historically, hemophilia treatment has focused on replacement of the missing coagulation
        factor to achieve hemostasis. Treatment progressed from use of cryoprecipitate (FVIII
        replacement in HA) or fresh frozen plasma (FFP) to plasma-derived factor concentrates,
        allowing for early control of hemorrhage, home therapy and accessibility for the introduction
        of prophylaxis. Contamination of factor concentrate supply with human immunodeficiency virus
        and hepatitis C virus led to the unfortunate infection of most of the severe hemophilia
        population in the 1980s, necessitating development of improved methods to screen and
        inactivate viruses. These mechanisms include dry-heat, pasteurisation, solvent-detergent
        treatment, immunoaffinity purification and nanofiltration, although some risk from emerging
        infections remains. Recombinant factor therapies were introduced to address the concern for
        infection transmission but raised new challenges regarding the risk of inhibitor formation.
        The extended half-life (EHL) factor products are a result of engineering proteins for longer
        recovery times in persons with hemophilia (PwH), to reduce frequency of dosing for prophylax
  
        -Factor VIII mimetic Emicizumab (Hemlibra, Genentech/Roche) is a first in-class humanised
        bispecific antibody substitution for HA designed to function as FVIIIa by binding one arm of
        the antibody to FIXa and the other arm to FX, accelerating activation of Fxa and propagating
        thrombin production. The application and incorporation of this novel approach using a SC
        antibody to prevent bleeding was outlined in a series of clinical trials (HAVEN) including
        PwHA with inhibitors, paediatric PwHA with inhibitors and PwHA without inhibitors. The
        bispecific antibody mechanism and half-life (˜28 days) overcomes the inhibitory alloantibody,
        limits the need for IV access and decreases infusion frequency. In HAVEN-1, males with severe
        HA and inhibitors aged ≥12 years received weekly prophylaxis (1·5 mg/kg) with emicizumab and
        had an 87% reduction in overall annualized bleeding rate (ABR)
  observational model: Other
  time perspective: Other
  measure: care in hemophilia patients
  time frame: Baseline
  description: Describe different aspects of care in hemophilia patients in Assiut children university hospital
  measure: Describe complications in availability of factors
  time frame: Baseline
  description: describe different aspects of complications and challenges in availability of factors ,bypassing agents which are used to manage hemophilia patients with inhibitors in Assiut children university hospital
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: N/A
  maximum age: 18 Years
  responsible party type: Principal Investigator
  investigator affiliation: Assiut University
  investigator full name: Sara Ahmed Abd El-Bary
  investigator title: Principal Investigator
  mesh term: Hemophilia A
  mesh term: Hemophilia B