<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/NCT02999308</url>
  </required_header>
  <id_info>
    <org_study_id>CHIPS</org_study_id>
    <nct_id>NCT02999308</nct_id>
  </id_info>
  <brief_title>A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children</brief_title>
  <acronym>CHIPS</acronym>
  <official_title>Hematology Oncology Center</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Beijing Children's Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Beijing Children's Hospital</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      In the past, due to economic and medical resource constraints, the hemophilia comprehensive&#xD;
      care in China was suboptimal. The BCH data of both retrospective and prospective studies&#xD;
      reveals that for 4-6y and 6-9y patients with severe hemophilia respectively: 45% and 82% of&#xD;
      the patients have suffered from joint bleeding, with mean AJBR of 4.18 and 4.95; and 24.2%&#xD;
      and 33.3% of them with AJBR&gt;10 times, led to arthropathies and made their quality of life be&#xD;
      heavy affected. Now, with the development of economy and medical science in China,&#xD;
      prophylaxis regimens have been initiated in more and more children with hemophilia.&#xD;
      Considering the difference between prophylaxis regimens, the frequency of joint bleeding was&#xD;
      reduced significantly, the quality of life of hemophilia kids improved. An assessment scoring&#xD;
      system for the appropriate validation of individualized prophylaxis treatment regimens are&#xD;
      urgently needed.&#xD;
&#xD;
      Before, the most important assessed indication for hemophilia prophylaxis was the frequency&#xD;
      of joint bleeding. But increasing evidences are showing that there is a discrepancy between&#xD;
      real joint damage and joint bleeding frequency. The single indicator of joint bleeding&#xD;
      frequency is not sufficient to evaluate the joint status of hemophilia children.&#xD;
&#xD;
      Under the World Health Organization's ICF guidelines, the assessment scoring system for&#xD;
      selecting prophylaxis for children with hemophilia should include the tools currently&#xD;
      available for assessment of structure/function of the joint, patient activities and patient&#xD;
      participation in hemophilia healthcare. According to ICF of WHO, the most common bleeding&#xD;
      parts are elbows, knees and ankles, therefore the assessment of children with hemophilia&#xD;
      should include the evaluation of the structure, the function of these 6 Index joints, the&#xD;
      capacity of activities and the capacity of participation of children. These will constitute a&#xD;
      comprehensive hemophilia evaluation system.&#xD;
&#xD;
      In China, exploration of the optimal and individualized prophylaxis regimen is urgent, and&#xD;
      the comprehensive evaluation system should include joint structure and function, body's&#xD;
      activities and individual participation, thus may be more appropriate for the individualized&#xD;
      prophylaxis for Chinese children with hemophilia.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date>August 2016</start_date>
  <completion_date type="Anticipated">December 2017</completion_date>
  <primary_completion_date type="Anticipated">September 2017</primary_completion_date>
  <phase>Phase 4</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change of Ultrasound HEAD-US score of index joints</measure>
    <time_frame>baseline,3 months, 6 months,9 months and 12 months</time_frame>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">40</enrollment>
  <condition>Haemophilia</condition>
  <arm_group>
    <arm_group_label>single arm</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Observe the patient's condition and then change the regimen</intervention_name>
    <description>The patients will be assessed every 3 months and the treatment regimen will be modified by the result of assessment.</description>
    <arm_group_label>single arm</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Severe hemophilia A (FVIII: C&lt;2%),&#xD;
&#xD;
          2. Age 1-7y&#xD;
&#xD;
          3. Historical bleeding in any knee, elbow and ankle joint (s),&#xD;
&#xD;
          4. &gt;50 EDs irrespective of FVIII product, including human coagulate factor and&#xD;
             recombinant factor VIII&#xD;
&#xD;
          5. No inhibitor present or history/family history,&#xD;
&#xD;
          6. On-demand or low-dose prophylaxis (the dose per kg.week less or as Step 1 prophylaxis&#xD;
             regimens factor consumption)&#xD;
&#xD;
          7. Regular clinical visit with accessible data,&#xD;
&#xD;
          8. Informed consent will be obtained from patient legal guardians before the enrollment.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  1. Other bleeding disorders or systemic disorders, or don't fit for enrollment&#xD;
             according to the investigator, 2. FVIII inhibitor development:&gt;0.6 BU (confirmed by&#xD;
             two separate tests), 3. Unable to follow the protocol or refuse to continue the&#xD;
             participation.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>7 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Wu Runhui, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Beijing Children's Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Wu Runhui, MD, PhD</last_name>
    <phone>0086-010-59617621</phone>
    <email>runhuiwu@hotmail.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Beijing Children's Hospital, Capital Medical University</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <zip>100054</zip>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Wu Runhui, MD, PhD</last_name>
      <phone>0086-010-59617621</phone>
      <email>runhuiwu@hotmail.com</email>
    </contact>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <results_reference>
    <citation>Berntorp E, Boulyjenkov V, Brettler D, Chandy M, Jones P, Lee C, Lusher J, Mannucci P, Peak I, Rickard K, et al. Modern treatment of haemophilia. Bull World Health Organ. 1995;73(5):691-701.</citation>
    <PMID>8846496</PMID>
  </results_reference>
  <results_reference>
    <citation>Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992 Jul;232(1):25-32. doi: 10.1111/j.1365-2796.1992.tb00546.x.</citation>
    <PMID>1640190</PMID>
  </results_reference>
  <verification_date>December 2016</verification_date>
  <study_first_submitted>September 1, 2016</study_first_submitted>
  <study_first_submitted_qc>December 17, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 21, 2016</study_first_posted>
  <last_update_submitted>December 17, 2016</last_update_submitted>
  <last_update_submitted_qc>December 17, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">December 21, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Beijing Children's Hospital</investigator_affiliation>
    <investigator_full_name>Runhui WU</investigator_full_name>
    <investigator_title>Chief doctor</investigator_title>
  </responsible_party>
  <keyword>haemophilia</keyword>
  <keyword>prophylaxis</keyword>
  <keyword>children</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hemophilia A</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


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/NCT02999308
org study id: CHIPS
nct id: NCT02999308
lead sponsor: 
has dmc: Yes
In the past, due to economic and medical resource constraints, the hemophilia comprehensive
      care in China was suboptimal. The BCH data of both retrospective and prospective studies
      reveals that for 4-6y and 6-9y patients with severe hemophilia respectively: 45% and 82% of
      the patients have suffered from joint bleeding, with mean AJBR of 4.18 and 4.95; and 24.2%
      and 33.3% of them with AJBR>10 times, led to arthropathies and made their quality of life be
      heavy affected. Now, with the development of economy and medical science in China,
      prophylaxis regimens have been initiated in more and more children with hemophilia.
      Considering the difference between prophylaxis regimens, the frequency of joint bleeding was
      reduced significantly, the quality of life of hemophilia kids improved. An assessment scoring
      system for the appropriate validation of individualized prophylaxis treatment regimens are
      urgently needed.

      Before, the most important assessed indication for hemophilia prophylaxis was the frequency
      of joint bleeding. But increasing evidences are showing that there is a discrepancy between
      real joint damage and joint bleeding frequency. The single indicator of joint bleeding
      frequency is not sufficient to evaluate the joint status of hemophilia children.

      Under the World Health Organization's ICF guidelines, the assessment scoring system for
      selecting prophylaxis for children with hemophilia should include the tools currently
      available for assessment of structure/function of the joint, patient activities and patient
      participation in hemophilia healthcare. According to ICF of WHO, the most common bleeding
      parts are elbows, knees and ankles, therefore the assessment of children with hemophilia
      should include the evaluation of the structure, the function of these 6 Index joints, the
      capacity of activities and the capacity of participation of children. These will constitute a
      comprehensive hemophilia evaluation system.

      In China, exploration of the optimal and individualized prophylaxis regimen is urgent, and
      the comprehensive evaluation system should include joint structure and function, body's
      activities and individual participation, thus may be more appropriate for the individualized
      prophylaxis for Chinese children with hemophilia.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change of Ultrasound HEAD-US score of index joints
time frame: baseline,3 months, 6 months,9 months and 12 months
arm group label: single arm
arm group type: Experimental
intervention type: Other
intervention name: Observe the patient's condition and then change the regimen
description: The patients will be assessed every 3 months and the treatment regimen will be modified by the result of assessment.
arm group label: single arm
criteria: 
gender: All
minimum age: 1 Year
maximum age: 7 Years
healthy volunteers: No
last name: Wu Runhui, MD, PhD
role: Principal Investigator
affiliation: Beijing Children's Hospital
last name: Wu Runhui, MD, PhD
phone: 0086-010-59617621
email: runhuiwu@hotmail.com
facility: 
status: Recruiting
contact: 
country: China
citation: Berntorp E, Boulyjenkov V, Brettler D, Chandy M, Jones P, Lee C, Lusher J, Mannucci P, Peak I, Rickard K, et al. Modern treatment of haemophilia. Bull World Health Organ. 1995;73(5):691-701.
PMID: 8846496
citation: Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992 Jul;232(1):25-32. doi: 10.1111/j.1365-2796.1992.tb00546.x.
PMID: 1640190
responsible party type: Principal Investigator
investigator affiliation: Beijing Children's Hospital
investigator full name: Runhui WU
investigator title: Chief doctor
mesh term: Hemophilia A
sharing ipd: Undecided