<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/NCT03835312</url>
  </required_header>
  <id_info>
    <org_study_id>SeqTranUCBC&amp;IC-001</org_study_id>
    <nct_id>NCT03835312</nct_id>
  </id_info>
  <brief_title>Sequential Transplantation of UCBSCs and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency T1DM</brief_title>
  <official_title>Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Children's Hospital of Fudan University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Children's Hospital of Fudan University</source>
  <oversight_info>
    <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 evaluates the efficacy of sequential transplantation of umbilical cord blood stem&#xD;
      cells and islet cells in children with monogenic immunodeficiency type 1 diabetes mellitus.&#xD;
      Umbilical cord blood stem cell transplantation will be performed first. Children with stable&#xD;
      immune reconstruction will than receive islet cell transplantation.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Monogenic immunodeficiency type 1 diabetes mellitus (T1DM) usually onsets in early age and&#xD;
      has a long course of treatment. Because of T cell deficiency, patients are prone to recurrent&#xD;
      infection, hemorrhage, sepsis, colitis or complications of diabetes mellitus, which lead to&#xD;
      early death.&#xD;
&#xD;
      New clinical treatment schemes have been explored and introduced around the world. Sequential&#xD;
      transplantation of umbilical cord blood stem cells and islet cells is the latest treatment&#xD;
      method for these children. Early treatment of monogenic immunodeficiency T1DM children can&#xD;
      avoid disease-related organ toxicity, infection risk associated with chronic&#xD;
      immunosuppression, and possible prevention of autoimmune endocrine organ damage. Thus,&#xD;
      sequential transplantation of umbilical cord blood stem cells and islet cells is the only&#xD;
      possible cure for those patients currently.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">February 20, 2019</start_date>
  <completion_date type="Anticipated">December 31, 2025</completion_date>
  <primary_completion_date type="Anticipated">December 31, 2025</primary_completion_date>
  <phase>N/A</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>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 3 years</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum C-peptide</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Islet function (concentration of serum C-peptide)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of serum insulin</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Concentration of serum insulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fast blood glucose level</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Fast blood glucose level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HbA1c level</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>HbA1c level</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>occurrence of infection (number of infections)</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>occurrence of infection (number of infections)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of blood immunoglobulin</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Concentration of blood immunoglobulin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentration of T lymphocyte subsets</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Concentration of T lymphocyte subsets</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concentraion of interleukin-2</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Cytokines (concentraion of interleukin-2)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body height</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Body height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Body weight</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Body weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tanner stage</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Puberty change (Tanner stage)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 3 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 6 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 9 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 12 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 15 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 18 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 21 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 24 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 27 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 30 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 33 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Occurrence of graft versus host disease</measure>
    <time_frame>from the completion of treatment to 36 months</time_frame>
    <description>Occurrence of graft versus host disease</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">50</enrollment>
  <condition>Diabetes Mellitus, Type 1</condition>
  <condition>Immunologic Deficiency Syndromes</condition>
  <arm_group>
    <arm_group_label>Interventional</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Sequential transplantation of umbilical cord blood stem cells and islet cells</description>
  </arm_group>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>Sequential transplantation</intervention_name>
    <description>After successful matching of umbilical cord blood stem cells, patients will receive pretreatment and chemotherapy under protective isolation, followed by thawing and reinfusion of umbilical cord blood stem cells. Immunosuppressive agents will be used for GVHD prevention and anti-infection support will be provided after reinfusion. The status of umbilical cord blood stem cell implantation, immune reconstruction and therapeutic effect will be evaluated. Islet transplantation will be performed in those who meet the conditions. The long-term prognosis will be observed by long-term follow-up.</description>
    <arm_group_label>Interventional</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        1.Type 1 diabetes mellitus children with genetic immunodeficiency&#xD;
&#xD;
          1. Meet the diagnostic criteria of type 1 diabetes mellitus: clinical manifestations of&#xD;
             typical diabetes mellitus include polyphagia, polyuria, weight loss, or diabetic&#xD;
             ketoacidosis, confirmed by blood sugar level, islet function and autoimmune antibody.&#xD;
&#xD;
          2. Existence of extrapancreatic organ damage: (1) inflammatory bowel disease, (2)&#xD;
             impairment of renal function, (3) repeated infection of mouth, skin, anus or whole&#xD;
             body, (4) immune hepatitis, (5) persistent chronic immune iridocyclitis, (6) immune&#xD;
             adrenalinitis leading to adrenocortical dysfunction, (7) pituitary inflammation&#xD;
             leading to hypophysis, (8) rheumatoid disease, (9) immune vasculitis, (10) systemic&#xD;
             lupus erythematosus, (11) other organs besides thyroid function damage. Suffering from&#xD;
             one or more of above diseases. Recurrence after receiving regular clinical treatment,&#xD;
             including symptomatic treatment of organ protective drugs.&#xD;
&#xD;
          3. Gene mutation was found according to gene diagnosis: gene mutation was found by gene&#xD;
             sequencing. Literature searches at home and abroad confirmed that the defect of the&#xD;
             gene resulted in autoimmune or immune dysfunction, resulting in multiple organ&#xD;
             dysfunction and poor prognosis.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Mature and effective treatment methods are available.&#xD;
&#xD;
          2. HIV, HBV and HCV were positive.&#xD;
&#xD;
          3. A the active period of infection.&#xD;
&#xD;
          4. At the active stage of malignant tumors.&#xD;
&#xD;
          5. Combination of other fatal diseases.&#xD;
&#xD;
          6. Existence of mental and psychological diseases.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Luo Feihong</last_name>
    <role>Study Chair</role>
    <affiliation>Children's Hospital of Fudan University</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Luo Feihong</last_name>
    <phone>+862164931226</phone>
    <phone_ext>+862164931226</phone_ext>
    <email>luofh@fudan.edu.cn</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Xu Zhenran</last_name>
    <phone>+862164931226</phone>
    <phone_ext>+862164931226</phone_ext>
    <email>xu_zhenran@163.com</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Children's Hospital of Fudan University</name>
      <address>
        <city>Shanghai</city>
        <state>Shanghai</state>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Luo Feihong</last_name>
      <phone>+862164931226</phone>
      <phone_ext>+862164931226</phone_ext>
      <email>luofh@fudan.edu.cn</email>
    </contact>
    <contact_backup>
      <last_name>Xu Zhenran</last_name>
      <phone>+862164931226</phone>
      <phone_ext>+862164931226</phone_ext>
      <email>xu_zhenran@fudan.edu.cn</email>
    </contact_backup>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <reference>
    <citation>Cho NH, Shaw JE, Karuranga S, Huang Y, da Rocha Fernandes JD, Ohlrogge AW, Malanda B. IDF Diabetes Atlas: Global estimates of diabetes prevalence for 2017 and projections for 2045. Diabetes Res Clin Pract. 2018 Apr;138:271-281. doi: 10.1016/j.diabres.2018.02.023. Epub 2018 Feb 26.</citation>
    <PMID>29496507</PMID>
  </reference>
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    <citation>Fu H, Shen SX, Chen ZW, Wang JJ, Ye TT, LaPorte RE, Tajima N. Shanghai, China, has the lowest confirmed incidence of childhood diabetes in the world. Diabetes Care. 1994 Oct;17(10):1206-8. doi: 10.2337/diacare.17.10.1206.</citation>
    <PMID>7821146</PMID>
  </reference>
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    <citation>Zhao Z, Sun C, Wang C, Li P, Wang W, Ye J, Gu X, Wang X, Shen S, Zhi D, Lu Z, Ye R, Cheng R, Xi L, Li X, Zheng Z, Zhang M, Luo F. Rapidly rising incidence of childhood type 1 diabetes in Chinese population: epidemiology in Shanghai during 1997-2011. Acta Diabetol. 2014 Dec;51(6):947-53. doi: 10.1007/s00592-014-0590-2. Epub 2014 Apr 29.</citation>
    <PMID>24777734</PMID>
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    <citation>Daniels M, DuBose SN, Maahs DM, Beck RW, Fox LA, Gubitosi-Klug R, Laffel LM, Miller KM, Speer H, Tamborlane WV, Tansey MJ; T1D Exchange Clinic Network. Factors associated with microalbuminuria in 7,549 children and adolescents with type 1 diabetes in the T1D Exchange clinic registry. Diabetes Care. 2013 Sep;36(9):2639-45. doi: 10.2337/dc12-2192. Epub 2013 Apr 22.</citation>
    <PMID>23610082</PMID>
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    <citation>Amin R, Widmer B, Prevost AT, Schwarze P, Cooper J, Edge J, Marcovecchio L, Neil A, Dalton RN, Dunger DB. Risk of microalbuminuria and progression to macroalbuminuria in a cohort with childhood onset type 1 diabetes: prospective observational study. BMJ. 2008 Mar 29;336(7646):697-701. doi: 10.1136/bmj.39478.378241.BE. Epub 2008 Mar 18.</citation>
    <PMID>18349042</PMID>
  </reference>
  <reference>
    <citation>Mollsten A, Svensson M, Waernbaum I, Berhan Y, Schon S, Nystrom L, Arnqvist HJ, Dahlquist G; Swedish Childhood Diabetes Study Group; Diabetes Incidence Study in Sweden; Swedish Renal Registry. Cumulative risk, age at onset, and sex-specific differences for developing end-stage renal disease in young patients with type 1 diabetes: a nationwide population-based cohort study. Diabetes. 2010 Jul;59(7):1803-8. doi: 10.2337/db09-1744. Epub 2010 Apr 27.</citation>
    <PMID>20424230</PMID>
  </reference>
  <reference>
    <citation>American Diabetes Association. (2) Classification and diagnosis of diabetes. Diabetes Care. 2015 Jan;38 Suppl:S8-S16. doi: 10.2337/dc15-S005. No abstract available.</citation>
    <PMID>25537714</PMID>
  </reference>
  <reference>
    <citation>Wildin RS, Smyk-Pearson S, Filipovich AH. Clinical and molecular features of the immunodysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) syndrome. J Med Genet. 2002 Aug;39(8):537-45. doi: 10.1136/jmg.39.8.537.</citation>
    <PMID>12161590</PMID>
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    <citation>Uzel G, Sampaio EP, Lawrence MG, Hsu AP, Hackett M, Dorsey MJ, Noel RJ, Verbsky JW, Freeman AF, Janssen E, Bonilla FA, Pechacek J, Chandrasekaran P, Browne SK, Agharahimi A, Gharib AM, Mannurita SC, Yim JJ, Gambineri E, Torgerson T, Tran DQ, Milner JD, Holland SM. Dominant gain-of-function STAT1 mutations in FOXP3 wild-type immune dysregulation-polyendocrinopathy-enteropathy-X-linked-like syndrome. J Allergy Clin Immunol. 2013 Jun;131(6):1611-23. doi: 10.1016/j.jaci.2012.11.054. Epub 2013 Mar 25.</citation>
    <PMID>23534974</PMID>
  </reference>
  <reference>
    <citation>Bennett CL, Christie J, Ramsdell F, Brunkow ME, Ferguson PJ, Whitesell L, Kelly TE, Saulsbury FT, Chance PF, Ochs HD. The immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) is caused by mutations of FOXP3. Nat Genet. 2001 Jan;27(1):20-1. doi: 10.1038/83713.</citation>
    <PMID>11137993</PMID>
  </reference>
  <reference>
    <citation>Rao A, Kamani N, Filipovich A, Lee SM, Davies SM, Dalal J, Shenoy S. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood. 2007 Jan 1;109(1):383-5. doi: 10.1182/blood-2006-05-025072. Epub 2006 Sep 21.</citation>
    <PMID>16990602</PMID>
  </reference>
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    <citation>van de Veerdonk FL, Plantinga TS, Hoischen A, Smeekens SP, Joosten LA, Gilissen C, Arts P, Rosentul DC, Carmichael AJ, Smits-van der Graaf CA, Kullberg BJ, van der Meer JW, Lilic D, Veltman JA, Netea MG. STAT1 mutations in autosomal dominant chronic mucocutaneous candidiasis. N Engl J Med. 2011 Jul 7;365(1):54-61. doi: 10.1056/NEJMoa1100102. Epub 2011 Jun 29.</citation>
    <PMID>21714643</PMID>
  </reference>
  <reference>
    <citation>Toubiana J, Okada S, Hiller J, Oleastro M, Lagos Gomez M, Aldave Becerra JC, Ouachee-Chardin M, Fouyssac F, Girisha KM, Etzioni A, Van Montfrans J, Camcioglu Y, Kerns LA, Belohradsky B, Blanche S, Bousfiha A, Rodriguez-Gallego C, Meyts I, Kisand K, Reichenbach J, Renner ED, Rosenzweig S, Grimbacher B, van de Veerdonk FL, Traidl-Hoffmann C, Picard C, Marodi L, Morio T, Kobayashi M, Lilic D, Milner JD, Holland S, Casanova JL, Puel A; International STAT1 Gain-of-Function Study Group. Heterozygous STAT1 gain-of-function mutations underlie an unexpectedly broad clinical phenotype. Blood. 2016 Jun 23;127(25):3154-64. doi: 10.1182/blood-2015-11-679902. Epub 2016 Apr 25.</citation>
    <PMID>27114460</PMID>
  </reference>
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    <citation>Leiding JW, Okada S, Hagin D, Abinun M, Shcherbina A, Balashov DN, Kim VHD, Ovadia A, Guthery SL, Pulsipher M, Lilic D, Devlin LA, Christie S, Depner M, Fuchs S, van Royen-Kerkhof A, Lindemans C, Petrovic A, Sullivan KE, Bunin N, Kilic SS, Arpaci F, Calle-Martin O, Martinez-Martinez L, Aldave JC, Kobayashi M, Ohkawa T, Imai K, Iguchi A, Roifman CM, Gennery AR, Slatter M, Ochs HD, Morio T, Torgerson TR; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the Primary Immune Deficiency Treatment Consortium. Hematopoietic stem cell transplantation in patients with gain-of-function signal transducer and activator of transcription 1 mutations. J Allergy Clin Immunol. 2018 Feb;141(2):704-717.e5. doi: 10.1016/j.jaci.2017.03.049. Epub 2017 Jun 7.</citation>
    <PMID>28601685</PMID>
  </reference>
  <reference>
    <citation>Niclauss N, Morel P, Berney T. Has the gap between pancreas and islet transplantation closed? Transplantation. 2014 Sep 27;98(6):593-9. doi: 10.1097/TP.0000000000000288.</citation>
    <PMID>25029387</PMID>
  </reference>
  <reference>
    <citation>Ahearn AJ, Parekh JR, Posselt AM. Islet transplantation for Type 1 diabetes: where are we now? Expert Rev Clin Immunol. 2015 Jan;11(1):59-68. doi: 10.1586/1744666X.2015.978291. Epub 2014 Dec 2.</citation>
    <PMID>25454816</PMID>
  </reference>
  <reference>
    <citation>Kopan C, Tucker T, Alexander M, Mohammadi MR, Pone EJ, Lakey JRT. Approaches in Immunotherapy, Regenerative Medicine, and Bioengineering for Type 1 Diabetes. Front Immunol. 2018 Jun 12;9:1354. doi: 10.3389/fimmu.2018.01354. eCollection 2018.</citation>
    <PMID>29963051</PMID>
  </reference>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>January 31, 2019</study_first_submitted>
  <study_first_submitted_qc>February 6, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 8, 2019</study_first_posted>
  <last_update_submitted>August 29, 2023</last_update_submitted>
  <last_update_submitted_qc>August 29, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 31, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Islets of Langerhans Transplantation</keyword>
  <keyword>Cord Blood Stem Cell Transplantation</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Diabetes Mellitus</mesh_term>
    <mesh_term>Diabetes Mellitus, Type 1</mesh_term>
    <mesh_term>Immunologic Deficiency Syndromes</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>October 16, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/12/NCT03835312/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>October 16, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/12/NCT03835312/SAP_002.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Informed Consent Form</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>Yes</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>October 16, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/12/NCT03835312/ICF_001.pdf</document_url>
    </provided_document>
  </provided_document_section>
  <!-- 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/NCT03835312
org study id: SeqTranUCBC&IC-001
nct id: NCT03835312
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
This study evaluates the efficacy of sequential transplantation of umbilical cord blood stem
      cells and islet cells in children with monogenic immunodeficiency type 1 diabetes mellitus.
      Umbilical cord blood stem cell transplantation will be performed first. Children with stable
      immune reconstruction will than receive islet cell transplantation.
Monogenic immunodeficiency type 1 diabetes mellitus (T1DM) usually onsets in early age and
      has a long course of treatment. Because of T cell deficiency, patients are prone to recurrent
      infection, hemorrhage, sepsis, colitis or complications of diabetes mellitus, which lead to
      early death.

      New clinical treatment schemes have been explored and introduced around the world. Sequential
      transplantation of umbilical cord blood stem cells and islet cells is the latest treatment
      method for these children. Early treatment of monogenic immunodeficiency T1DM children can
      avoid disease-related organ toxicity, infection risk associated with chronic
      immunosuppression, and possible prevention of autoimmune endocrine organ damage. Thus,
      sequential transplantation of umbilical cord blood stem cells and islet cells is the only
      possible cure for those patients currently.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 3 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 3 years
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 6 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 9 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 12 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 15 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 18 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 21 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 24 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 27 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 30 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum C-peptide
time frame: from the completion of treatment to 33 months
description: Islet function (concentration of serum C-peptide)
measure: Concentration of serum insulin
time frame: from the completion of treatment to 3 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 6 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 9 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 12 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 15 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 18 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 21 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 24 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 27 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 30 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 33 months
description: Concentration of serum insulin
measure: Concentration of serum insulin
time frame: from the completion of treatment to 36 months
description: Concentration of serum insulin
measure: Fast blood glucose level
time frame: from the completion of treatment to 3 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 6 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 9 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 12 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 15 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 18 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 21 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 24 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 27 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 30 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 33 months
description: Fast blood glucose level
measure: Fast blood glucose level
time frame: from the completion of treatment to 36 months
description: Fast blood glucose level
measure: HbA1c level
time frame: from the completion of treatment to 3 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 6 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 9 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 12 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 15 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 18 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 21 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 24 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 27 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 30 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 33 months
description: HbA1c level
measure: HbA1c level
time frame: from the completion of treatment to 36 months
description: HbA1c level
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 3 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 6 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 9 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 12 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 15 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 18 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 21 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 24 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 27 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 30 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 33 months
description: occurrence of infection (number of infections)
measure: occurrence of infection (number of infections)
time frame: from the completion of treatment to 36 months
description: occurrence of infection (number of infections)
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 3 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 6 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 9 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 12 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 15 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 18 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 21 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 24 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 27 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 30 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 33 months
description: Concentration of blood immunoglobulin
measure: Concentration of blood immunoglobulin
time frame: from the completion of treatment to 36 months
description: Concentration of blood immunoglobulin
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 3 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 6 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 9 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 12 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 15 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 18 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 21 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 24 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 27 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 30 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 33 months
description: Concentration of T lymphocyte subsets
measure: Concentration of T lymphocyte subsets
time frame: from the completion of treatment to 36 months
description: Concentration of T lymphocyte subsets
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 3 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 6 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 9 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 12 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 15 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 18 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 21 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 24 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 27 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 30 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 33 months
description: Cytokines (concentraion of interleukin-2)
measure: Concentraion of interleukin-2
time frame: from the completion of treatment to 36 months
description: Cytokines (concentraion of interleukin-2)
measure: Body height
time frame: from the completion of treatment to 3 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 6 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 9 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 12 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 15 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 18 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 21 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 24 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 27 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 30 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 33 months
description: Body height
measure: Body height
time frame: from the completion of treatment to 36 months
description: Body height
measure: Body weight
time frame: from the completion of treatment to 3 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 6 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 9 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 12 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 15 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 18 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 21 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 24 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 27 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 30 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 33 months
description: Body weight
measure: Body weight
time frame: from the completion of treatment to 36 months
description: Body weight
measure: Tanner stage
time frame: from the completion of treatment to 3 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 6 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 9 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 12 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 15 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 18 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 21 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 24 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 27 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 30 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 33 months
description: Puberty change (Tanner stage)
measure: Tanner stage
time frame: from the completion of treatment to 36 months
description: Puberty change (Tanner stage)
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 3 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 6 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 9 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 12 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 15 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 18 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 21 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 24 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 27 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 30 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 33 months
description: Occurrence of graft versus host disease
measure: Occurrence of graft versus host disease
time frame: from the completion of treatment to 36 months
description: Occurrence of graft versus host disease
arm group label: Interventional
arm group type: Experimental
description: Sequential transplantation of umbilical cord blood stem cells and islet cells
intervention type: Procedure
intervention name: Sequential transplantation
description: After successful matching of umbilical cord blood stem cells, patients will receive pretreatment and chemotherapy under protective isolation, followed by thawing and reinfusion of umbilical cord blood stem cells. Immunosuppressive agents will be used for GVHD prevention and anti-infection support will be provided after reinfusion. The status of umbilical cord blood stem cell implantation, immune reconstruction and therapeutic effect will be evaluated. Islet transplantation will be performed in those who meet the conditions. The long-term prognosis will be observed by long-term follow-up.
arm group label: Interventional
criteria: 
gender: All
minimum age: N/A
maximum age: 18 Years
healthy volunteers: No
last name: Luo Feihong
role: Study Chair
affiliation: Children's Hospital of Fudan University
last name: Luo Feihong
phone: +862164931226
phone ext: +862164931226
email: luofh@fudan.edu.cn
last name: Xu Zhenran
phone: +862164931226
phone ext: +862164931226
email: xu_zhenran@163.com
facility: 
status: Recruiting
contact: 
contact backup: 
country: China
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PMID: 25537714
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PMID: 29963051
responsible party type: Sponsor
mesh term: Diabetes Mellitus
mesh term: Diabetes Mellitus, Type 1
mesh term: Immunologic Deficiency Syndromes
sharing ipd: No
provided document: 
provided document: 
provided document: 