<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/NCT03252548</url>
  </required_header>
  <id_info>
    <org_study_id>BCHlung006</org_study_id>
    <nct_id>NCT03252548</nct_id>
  </id_info>
  <brief_title>Pediatric Primary Immunodeficiency Disease (PID) in China</brief_title>
  <official_title>Clinical Characteristics and Prognosis of Primary Immunodeficiency Disease (PID) in Chinese Children</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Beijing Children's Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Capital Institute of Pediatrics, China</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Shenzhen Children's Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Beijing Children's Hospital</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study is a prospective cohort study of children diagnosed with primary immunodeficiency&#xD;
      disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in&#xD;
      Chinese children.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Primary immunodeficiency disease (PID) is a group of rare and fatal disease. However, the&#xD;
      research about clinical characteristics, treatment, management and prognosis of Chinese&#xD;
      children with PID is still not perfect and there is no basic and large database. Therefore,&#xD;
      this study is conducted, in order to set up a complete database and long term follow up of&#xD;
      Chinese children with PID, and establish foundations for basic research and precise medicine.&#xD;
&#xD;
      This study is divided into two parts. In part one, pediatric patients with PID in the past 10&#xD;
      years will be collected retrospectively by review medical records from these centers, and&#xD;
      investigate the clinical features and prognosis. In part two, all new cases with suspected&#xD;
      PID will be collected. Clinical symptoms, physical examination, blood tests, and humoral and&#xD;
      cellular immune functions will be detected. Gene tests would be done when necessary to&#xD;
      confirm the diagnosis. Then follow up at 6-moth, 1-year, 2-year, 3-year, 4-year and 5-year&#xD;
      respectively. The trial will be completed in 60 months.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Not yet recruiting</last_known_status>
  <start_date type="Anticipated">January 2018</start_date>
  <completion_date type="Anticipated">August 2022</completion_date>
  <primary_completion_date type="Anticipated">August 2022</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Frequencies of severe infection of children with PID</measure>
    <time_frame>60 months</time_frame>
    <description>Times of severe infection attack will be recorded every year from baseline to 60 months, assessed by questionnaire</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Life quality of children with PID</measure>
    <time_frame>60 months</time_frame>
    <description>Life Quality Questionnaire, once for every year</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Complications of children with PID</measure>
    <time_frame>60 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Long-term changes on lung function of children with PID</measure>
    <time_frame>60 months</time_frame>
  </secondary_outcome>
  <enrollment type="Anticipated">400</enrollment>
  <condition>Primary Immunodeficiency Disease</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Children who was confirmed diagnosis as PID at the certain hospitals&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Age: from neonates to 18 years old&#xD;
&#xD;
          2. Fulfilling the one of the followings:&#xD;
&#xD;
               1. Severe infection after vaccination (BCG, polio arthritis pill)&#xD;
&#xD;
               2. Chronic/recurrent ear infection, sinus infections and respiratory tract infection&#xD;
&#xD;
               3. Recurrent, deep skin or deep-seated infections&#xD;
&#xD;
               4. Boys with early period and refractory thrombocytopenia&#xD;
&#xD;
               5. Infants with obviously decreased absolute number of lymphocytes&#xD;
&#xD;
               6. Severe allergy with infection&#xD;
&#xD;
               7. Infant diabetes with severe diarrhea&#xD;
&#xD;
               8. Boys with severe Epstein-Barr virus infection&#xD;
&#xD;
               9. Infants with hepatosplenomegaly and generalized lymphadenopathy&#xD;
&#xD;
              10. Infantile hemophagocytic syndrome&#xD;
&#xD;
              11. Failure of an infant to gain weight or grow normally&#xD;
&#xD;
              12. Persistent thrush in mouth or fungalinfection on skin&#xD;
&#xD;
              13. Family history of primary immunodeficiency&#xD;
&#xD;
              14. Invasive pneumococcal infection&#xD;
&#xD;
              15. Erythrodermic psoriasis&#xD;
&#xD;
          3. Gene test confirmed PID pediatric patients&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Subject will be excluded if she or he would not enroll into this study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Day</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>kunling shen, MD,PhD</last_name>
    <role>Study Director</role>
    <affiliation>Beijing Children's Hospital, Capital Medical University, China</affiliation>
  </overall_official>
  <overall_official>
    <last_name>jianxin he, MD,PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Beijing Children's Hospital, Capital Medical University, China</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>baoping xu, MD,PhD</last_name>
    <phone>861059616308</phone>
    <email>xubaopingbch@163.com</email>
  </overall_contact>
  <verification_date>May 2017</verification_date>
  <study_first_submitted>August 15, 2017</study_first_submitted>
  <study_first_submitted_qc>August 15, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 17, 2017</study_first_posted>
  <last_update_submitted>January 1, 2018</last_update_submitted>
  <last_update_submitted_qc>January 1, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">January 3, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Beijing Children's Hospital</investigator_affiliation>
    <investigator_full_name>Kunling Shen</investigator_full_name>
    <investigator_title>Chief of China National Clinical Research Center for Respiratory Diseases</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Primary Immunodeficiency Diseases</mesh_term>
    <mesh_term>Immunologic Deficiency Syndromes</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</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/NCT03252548
org study id: BCHlung006
nct id: NCT03252548
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
This study is a prospective cohort study of children diagnosed with primary immunodeficiency
      disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in
      Chinese children.
Primary immunodeficiency disease (PID) is a group of rare and fatal disease. However, the
      research about clinical characteristics, treatment, management and prognosis of Chinese
      children with PID is still not perfect and there is no basic and large database. Therefore,
      this study is conducted, in order to set up a complete database and long term follow up of
      Chinese children with PID, and establish foundations for basic research and precise medicine.

      This study is divided into two parts. In part one, pediatric patients with PID in the past 10
      years will be collected retrospectively by review medical records from these centers, and
      investigate the clinical features and prognosis. In part two, all new cases with suspected
      PID will be collected. Clinical symptoms, physical examination, blood tests, and humoral and
      cellular immune functions will be detected. Gene tests would be done when necessary to
      confirm the diagnosis. Then follow up at 6-moth, 1-year, 2-year, 3-year, 4-year and 5-year
      respectively. The trial will be completed in 60 months.
observational model: Case-Only
time perspective: Prospective
measure: Frequencies of severe infection of children with PID
time frame: 60 months
description: Times of severe infection attack will be recorded every year from baseline to 60 months, assessed by questionnaire
measure: Life quality of children with PID
time frame: 60 months
description: Life Quality Questionnaire, once for every year
measure: Complications of children with PID
time frame: 60 months
measure: Long-term changes on lung function of children with PID
time frame: 60 months
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 1 Day
maximum age: 18 Years
healthy volunteers: No
last name: kunling shen, MD,PhD
role: Study Director
affiliation: Beijing Children's Hospital, Capital Medical University, China
last name: jianxin he, MD,PhD
role: Principal Investigator
affiliation: Beijing Children's Hospital, Capital Medical University, China
last name: baoping xu, MD,PhD
phone: 861059616308
email: xubaopingbch@163.com
responsible party type: Principal Investigator
investigator affiliation: Beijing Children's Hospital
investigator full name: Kunling Shen
investigator title: Chief of China National Clinical Research Center for Respiratory Diseases
mesh term: Primary Immunodeficiency Diseases
mesh term: Immunologic Deficiency Syndromes
sharing ipd: No