xml: <clinical_study>
    <!-- This xml conforms to an XML Schema at:
      https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
    <required_header>
      <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
      <link_text>Link to the current ClinicalTrials.gov record.</link_text>
      <url>https://clinicaltrials.gov/ct2/show/NCT05991336</url>
    </required_header>
    <id_info>
      <org_study_id>GDH-001</org_study_id>
      <nct_id>NCT05991336</nct_id>
    </id_info>
    <brief_title>Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy</brief_title>
    <official_title>Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Institute of Hematology &amp; Blood Diseases Hospital, China</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Institute of Hematology &amp; Blood Diseases Hospital, China</source>
    <oversight_info>
      <has_dmc>No</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>
        The investigate will conduct a cohort study to compare the growth and development,&#xD;
        metabolism, lifestyle behavior, and health-related quality of life among three groups:&#xD;
        children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT&#xD;
        children with lifelong supportive therapy and healthy children.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">June 5, 2023</start_date>
    <completion_date type="Anticipated">December 31, 2029</completion_date>
    <primary_completion_date type="Anticipated">December 31, 2028</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Cohort</observational_model>
      <time_perspective>Prospective</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>Growth in pediatric subjects will be assessed by height.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>Height in meters.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Growth in pediatric subjects will be assessed by weight.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>Weight in kilograms.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Growth in pediatric subjects will be assessed by the head and chest circumference.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>Head and chest circumference in centimeters.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Growth in pediatric subjects will be assessed by interocular distance.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>Interocular distance in centimeters.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Growth in pediatric subjects will be assessed by bone density.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>Bone density measurement is measured by a procedure called densitometry, performed in the radiology medicine departments of hospitals or clinics. The result is the bone mineral density in grams per unit area (g/cm2).</description>
    </primary_outcome>
    <primary_outcome>
      <measure>The pubertal status will be measured using by Tanner scale.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>The Tanner scale, a well-known technique used to define pubertal stage, is based on physical measurements of development based on external primary and secondary sex characteristics (from Tanner 1: pre-pubertal to Tanner 5: mature adolescent).It was developed by Marshall and Tanner while conducting a longitudinal study during the 1940s to the 1960s in England. Based on observational data, they developed separate scales for the development of external genitalia: phallus, scrotum, and testes volume in males; breasts in females; and pubic hair in both males and females.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Hormonal levels will be measured by clinical examination.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>Hormonal levels included thyroid-stimulating hormone (TSH), serum triiodothyronine (T3), serum-free triiodothyronine (T4), growth hormone (GH, including IGF-1 and IGF-2), and sex hormone examination. Sex hormone examination is only for the following subjects: estradiol, FSH and LH for girls aged 9-18; testosterone, FSH and LH for boys aged 12-18.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study.</measure>
      <time_frame>Up to 5 years.</time_frame>
      <description>The Pediatric Quality of Life Inventory (PedsQL) is a valid, standardized, generic, and self-reporting assessment tool to measure health-related quality of life HRQOL for pediatrics and adolescents. PedsQL contain 23-items, and consists of the following: physical functioning, emotional functioning, social functioning, and school functioning. Each item is scored on a 5-point scale, scores are linearly transformed to a 0-100 scale in which high score means better condition.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Lifestyle behaviors will be assessed by general questioning.</measure>
      <time_frame>Up to 5 years.</time_frame>
    </primary_outcome>
    <number_of_groups>3</number_of_groups>
    <enrollment type="Anticipated">100</enrollment>
    <condition>Transfusion-dependent Beta-Thalassemia</condition>
    <condition>Gene Therapy</condition>
    <arm_group>
      <arm_group_label>Gene therapy group</arm_group_label>
      <description>TDT Children who have received gene therapy between 3~14 years old.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Supportive therapy group</arm_group_label>
      <description>TDT Children who received lifelong treatment with blood transfusions and iron chelation. They were selected based on the age and sex of the gene therapy group.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Healthy children group</arm_group_label>
      <description>Healthy children were selected based on the age and sex of the gene therapy group.</description>
    </arm_group>
    <intervention>
      <intervention_type>Genetic</intervention_type>
      <intervention_name>Gene therapy</intervention_name>
      <description>Autologous edited hematopoietic stem cell transplantation</description>
      <arm_group_label>Gene therapy group</arm_group_label>
    </intervention>
    <biospec_retention>Samples With DNA</biospec_retention>
    <biospec_descr>
      <textblock>
        The peripheral blood sample was collected and stored in the IHBDH biobank.&#xD;
      </textblock>
    </biospec_descr>
    <eligibility>
      <study_pop>
        <textblock>
          Participants are divided into three groups: TDT children who have received gene therapy,&#xD;
          TDT children who have received lifelong supportive therapy, and healthy children.&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
          Gene therapy group-inclusion&#xD;
  &#xD;
            -  Male or female age of 3-14 years&#xD;
  &#xD;
            -  TDT Children who have received gene therapy.&#xD;
  &#xD;
            -  Subjects who are willing and able to provide written informed consent.&#xD;
  &#xD;
          Supportive therapy group-inclusion&#xD;
  &#xD;
            -  Gender same as the matched case&#xD;
  &#xD;
            -  Age similar to the matched case&#xD;
  &#xD;
            -  Children with β-TDT&#xD;
  &#xD;
            -  No history of gene therapy or allogeneic hematopoietic stem cell transplantation&#xD;
  &#xD;
            -  Subjects who are willing and able to provide written informed consent.&#xD;
  &#xD;
          Healthy children group-inclusion&#xD;
  &#xD;
            -  Gender same as the matched case&#xD;
  &#xD;
            -  Age similar to the matched case&#xD;
  &#xD;
            -  Subjects who are willing and able to provide written informed consent.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Diagnosis of compound α-thalassemia&#xD;
  &#xD;
            -  Uncontrolled systemic fungal, bacterial, or viral infection&#xD;
  &#xD;
            -  History of malignant solid tumors, myeloproliferative or immunodeficiency diseases&#xD;
  &#xD;
            -  Diagnosed with mental illness&#xD;
  &#xD;
            -  Patients considered to be ineligible for the study by the investigator for reasons&#xD;
               other than the above&#xD;
  &#xD;
          Discontinuation of Study :&#xD;
  &#xD;
            -  Subjects who are unwilling or unable to continue participating in the study&#xD;
               (withdrawal of informed consent) may withdraw from the study&#xD;
  &#xD;
            -  Subjects who received gene therapy or allogeneic hematopoietic stem cell&#xD;
               transplantation during the study&#xD;
  &#xD;
            -  The subject is seriously non-compliant with the study requirements, such as missing 2&#xD;
               consecutive visits&#xD;
  &#xD;
            -  Subject lost to follow-up&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>3 Years</minimum_age>
      <maximum_age>14 Years</maximum_age>
      <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>Jun Shi, PhD</last_name>
      <phone>2223900913</phone>
      <email>shijun@ihcams.ac.cn</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Jingyu Zhao, MPH</last_name>
      <phone>13752253515</phone>
      <email>zhaojingyu@ihcams.ac.cn</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Regenerative Medicine Center</name>
        <address>
          <city>Tianjin</city>
          <state>Tianjin</state>
          <country>China</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Jun Shi, PhD</last_name>
        <phone>(86)2223900913</phone>
        <email>shijun@ihcams.ac.cn</email>
      </contact>
      <contact_backup>
        <last_name>Jingyu Zhao, MPH</last_name>
        <phone>(86)13752253515</phone>
        <email>zhaojingyu@ihcams.ac.cn</email>
      </contact_backup>
    </location>
    <location_countries>
      <country>China</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>July 27, 2023</study_first_submitted>
    <study_first_submitted_qc>August 6, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 14, 2023</study_first_posted>
    <last_update_submitted>August 6, 2023</last_update_submitted>
    <last_update_submitted_qc>August 6, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 14, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Principal Investigator</responsible_party_type>
      <investigator_affiliation>Institute of Hematology &amp; Blood Diseases Hospital, China</investigator_affiliation>
      <investigator_full_name>Jun Shi</investigator_full_name>
      <investigator_title>Director, Regenerative Medicine Clinic Center</investigator_title>
    </responsible_party>
    <keyword>Transfusion-dependent Beta-Thalassemia</keyword>
    <keyword>gene therapy</keyword>
    <keyword>cohort study</keyword>
    <keyword>case-control</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Thalassemia</mesh_term>
      <mesh_term>beta-Thalassemia</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: download date: ClinicalTrials.gov processed this data on September 20, 2023
  link text: Link to the current ClinicalTrials.gov record.
  url: https://clinicaltrials.gov/ct2/show/NCT05991336
  org study id: GDH-001
  nct id: NCT05991336
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  The investigate will conduct a cohort study to compare the growth and development,
        metabolism, lifestyle behavior, and health-related quality of life among three groups:
        children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT
        children with lifelong supportive therapy and healthy children.
  observational model: Cohort
  time perspective: Prospective
  measure: Growth in pediatric subjects will be assessed by height.
  time frame: Up to 5 years.
  description: Height in meters.
  measure: Growth in pediatric subjects will be assessed by weight.
  time frame: Up to 5 years.
  description: Weight in kilograms.
  measure: Growth in pediatric subjects will be assessed by the head and chest circumference.
  time frame: Up to 5 years.
  description: Head and chest circumference in centimeters.
  measure: Growth in pediatric subjects will be assessed by interocular distance.
  time frame: Up to 5 years.
  description: Interocular distance in centimeters.
  measure: Growth in pediatric subjects will be assessed by bone density.
  time frame: Up to 5 years.
  description: Bone density measurement is measured by a procedure called densitometry, performed in the radiology medicine departments of hospitals or clinics. The result is the bone mineral density in grams per unit area (g/cm2).
  measure: The pubertal status will be measured using by Tanner scale.
  time frame: Up to 5 years.
  description: The Tanner scale, a well-known technique used to define pubertal stage, is based on physical measurements of development based on external primary and secondary sex characteristics (from Tanner 1: pre-pubertal to Tanner 5: mature adolescent).It was developed by Marshall and Tanner while conducting a longitudinal study during the 1940s to the 1960s in England. Based on observational data, they developed separate scales for the development of external genitalia: phallus, scrotum, and testes volume in males; breasts in females; and pubic hair in both males and females.
  measure: Hormonal levels will be measured by clinical examination.
  time frame: Up to 5 years.
  description: Hormonal levels included thyroid-stimulating hormone (TSH), serum triiodothyronine (T3), serum-free triiodothyronine (T4), growth hormone (GH, including IGF-1 and IGF-2), and sex hormone examination. Sex hormone examination is only for the following subjects: estradiol, FSH and LH for girls aged 9-18; testosterone, FSH and LH for boys aged 12-18.
  measure: Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study.
  time frame: Up to 5 years.
  description: The Pediatric Quality of Life Inventory (PedsQL) is a valid, standardized, generic, and self-reporting assessment tool to measure health-related quality of life HRQOL for pediatrics and adolescents. PedsQL contain 23-items, and consists of the following: physical functioning, emotional functioning, social functioning, and school functioning. Each item is scored on a 5-point scale, scores are linearly transformed to a 0-100 scale in which high score means better condition.
  measure: Lifestyle behaviors will be assessed by general questioning.
  time frame: Up to 5 years.
  arm group label: Gene therapy group
  description: TDT Children who have received gene therapy between 3~14 years old.
  arm group label: Supportive therapy group
  description: TDT Children who received lifelong treatment with blood transfusions and iron chelation. They were selected based on the age and sex of the gene therapy group.
  arm group label: Healthy children group
  description: Healthy children were selected based on the age and sex of the gene therapy group.
  intervention type: Genetic
  intervention name: Gene therapy
  description: Autologous edited hematopoietic stem cell transplantation
  arm group label: Gene therapy group
  The peripheral blood sample was collected and stored in the IHBDH biobank.
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: 3 Years
  maximum age: 14 Years
  healthy volunteers: Accepts Healthy Volunteers
  last name: Jun Shi, PhD
  phone: 2223900913
  email: shijun@ihcams.ac.cn
  last name: Jingyu Zhao, MPH
  phone: 13752253515
  email: zhaojingyu@ihcams.ac.cn
  facility: 
  status: Recruiting
  contact: 
  contact backup: 
  country: China
  responsible party type: Principal Investigator
  investigator affiliation: Institute of Hematology & Blood Diseases Hospital, China
  investigator full name: Jun Shi
  investigator title: Director, Regenerative Medicine Clinic Center
  mesh term: Thalassemia
  mesh term: beta-Thalassemia