<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/NCT03255694</url>
  </required_header>
  <id_info>
    <org_study_id>GenSci 033 CT-Extension Period</org_study_id>
    <nct_id>NCT03255694</nct_id>
  </id_info>
  <brief_title>A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature</brief_title>
  <official_title>A Phase 2 Study Extension Period of Pegylated Somatropin (PEG-somatropin) in the Treatment of Children With Idiopathic Short Stature: An Open, Non-controlled Observational Study.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Changchun GeneScience Pharmaceutical Co., Ltd.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Tongji Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>The First Hospital of Jilin University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Affiliated Hospital of Jiangnan University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>The First Affiliated Hospital with Nanjing Medical University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Shanghai Children's Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>The Children's Hospital of Zhejiang University School of Medicine</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospital of Fudan University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Changchun GeneScience Pharmaceutical Co., Ltd.</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>
      After the first stage (52 weeks) of Phase II clinical trial, Pegylated recombinant human&#xD;
      growth hormone (PEG-rhGH) injection of appropriate dose in compliance with ISS clinical&#xD;
      treatment strategy is used to treat children with ISS (Idiopathic Short Stature). The&#xD;
      long-term efficacy and safety of the investigational product are evaluated, which can provide&#xD;
      more scientific and reliable medication guidance information for clinical diagnosis and&#xD;
      treatment.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">May 12, 2017</start_date>
  <completion_date type="Anticipated">June 2030</completion_date>
  <primary_completion_date type="Anticipated">June 2030</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change of yearly height velocity (ΔHV)</measure>
    <time_frame>Baseline，the end of 3-year addendum</time_frame>
    <description>Change of yearly height velocity before and after treatment. Yearly Height Velocity=12×(Height Yx - Height at Baseline)/(Date of Yx - Date of Baseline)(Yx refers to the height value at particular timepoint x)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Standard deviation score of height at the actual age (ΔHT SDS)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
    <description>Standard deviation score of height at the actual age.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change fo Bone maturation</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
    <description>Change fo Bone maturation before and after treatement (bone age/chronological age)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change of IGF-1 SDS (ΔIGF-1 SDS)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
    <description>Change of IGF-1 SDS before and after treatement</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes of standard deviation scores of body mass index (ΔBMI SDS)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
    <description>Changes of standard deviation scores of body mass index</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The yearly average dose of PEG-rhGH injection</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Final height (FH)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
    <description>Final height</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The improvement of FH compared with the baseline predicted adult height (PAH)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Improvement of NAH (near adult height)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
    <description>For subjects who reach NAH with treatment but fail to follow-ups before reaching FH, the improvement of NAH in comparison with the baseline PAH (predicted adult height) shall be evaluated</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>the improvement of PAH</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
    <description>For subjects who fail to reach NAH with treatment and fail to follow-ups before reaching FH, the improvement of PAH in comparison with the baseline PAH shall be evaluated</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The changes of the scores evaluated by the Quality of Life Scale</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The changes of lean body mass (LBM) (optional)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The changes of fat mass (torso) (FM) (optional)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The changes of the percentage of body fat (optional)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The changes of bone mineral density (BMD) (optional)</measure>
    <time_frame>Baseline，every 3 months，the end of 3-year addendum</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">360</enrollment>
  <condition>Dwarfism</condition>
  <arm_group>
    <arm_group_label>PEG-somatropin</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for the high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>PEG-somatropin</intervention_name>
    <description>After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.</description>
    <arm_group_label>PEG-somatropin</arm_group_label>
    <other_name>Polyethylene Glycol Recombinant Human Somatropin Injection</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  All subjects who have completed the first stage (52 weeks) of Phase II clinical trial&#xD;
             (including negative controls) with completed follow-up records may be enrolled in the&#xD;
             extension period study.&#xD;
&#xD;
          -  Before the extension period study, the investigator shall fully inform the subjects&#xD;
             and their guardians of all the information about the extension period study, including&#xD;
             detailed follow-up procedure, treatment plan, laboratory examination items during&#xD;
             follow-ups and possible benefits and risks. The extension period study shall only be&#xD;
             initiated after the subjects and their guardians are well informed, and agree to&#xD;
             cooperate and complete the treatment, follow-ups and examinations during the study,&#xD;
             and sign the written informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Subjects who have taken the following medications within 2 months before entering the&#xD;
             extension period study:&#xD;
&#xD;
               1. Aromatase inhibitors (which include but are not limited to Lelrozol and&#xD;
                  Anastrozole), with continuous medication ≥1 month;&#xD;
&#xD;
               2. Gonadotropin releasing hormone analogues (which include but are not limited to&#xD;
                  Triptorelin, Leuprorelin and Goserelin),, with continuous medication ≥1 month;&#xD;
&#xD;
               3. Sex steroids (which include but are not limited to any type of estrogen,&#xD;
                  progestin and androgen) , with continuous medication ≥1 month;&#xD;
&#xD;
               4. Protein anabolic drugs (which include but are not limited to Oxandrolone, Danazol&#xD;
                  and Strombafort), with continuous medication ≥1 month;&#xD;
&#xD;
               5. Glucocorticoids via oral/intravenous administration for more than 1 month..&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>4 Years</minimum_age>
    <maximum_age>9 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Xiaoping Luo</last_name>
    <role>Principal Investigator</role>
    <affiliation>Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan</name>
      <address>
        <city>Wuhan</city>
        <state>Hubei</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The First Affiated Hospital of Nanjing Medical Universit</name>
      <address>
        <city>Nanjing</city>
        <state>Jiangsu</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Affiliated Hospital of Jiangnan University</name>
      <address>
        <city>Wuxi</city>
        <state>Jiangsu</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The First Hospital of Jilin University</name>
      <address>
        <city>Changchun</city>
        <state>Jilin</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Children's Hospital of Zhejiang University School of Medicine</name>
      <address>
        <city>Hangzhou</city>
        <state>Zhejiang</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shanghai Children's Hospital of Fudan University</name>
      <address>
        <city>Shanghai</city>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shanghai Children's Hospital</name>
      <address>
        <city>Shanghai</city>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>February 2023</verification_date>
  <study_first_submitted>July 14, 2017</study_first_submitted>
  <study_first_submitted_qc>August 16, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 21, 2017</study_first_posted>
  <last_update_submitted>June 21, 2023</last_update_submitted>
  <last_update_submitted_qc>June 21, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 22, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Dwarfism</mesh_term>
  </condition_browse>
  <!-- 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/NCT03255694
org study id: GenSci 033 CT-Extension Period
nct id: NCT03255694
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
After the first stage (52 weeks) of Phase II clinical trial, Pegylated recombinant human
      growth hormone (PEG-rhGH) injection of appropriate dose in compliance with ISS clinical
      treatment strategy is used to treat children with ISS (Idiopathic Short Stature). The
      long-term efficacy and safety of the investigational product are evaluated, which can provide
      more scientific and reliable medication guidance information for clinical diagnosis and
      treatment.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change of yearly height velocity (ΔHV)
time frame: Baseline，the end of 3-year addendum
description: Change of yearly height velocity before and after treatment. Yearly Height Velocity=12×(Height Yx - Height at Baseline)/(Date of Yx - Date of Baseline)(Yx refers to the height value at particular timepoint x)
measure: Standard deviation score of height at the actual age (ΔHT SDS)
time frame: Baseline，every 3 months，the end of 3-year addendum
description: Standard deviation score of height at the actual age.
measure: Change fo Bone maturation
time frame: Baseline，every 3 months，the end of 3-year addendum
description: Change fo Bone maturation before and after treatement (bone age/chronological age)
measure: Change of IGF-1 SDS (ΔIGF-1 SDS)
time frame: Baseline，every 3 months，the end of 3-year addendum
description: Change of IGF-1 SDS before and after treatement
measure: Changes of standard deviation scores of body mass index (ΔBMI SDS)
time frame: Baseline，every 3 months，the end of 3-year addendum
description: Changes of standard deviation scores of body mass index
measure: The yearly average dose of PEG-rhGH injection
time frame: Baseline，every 3 months，the end of 3-year addendum
measure: Final height (FH)
time frame: Baseline，every 3 months，the end of 3-year addendum
description: Final height
measure: The improvement of FH compared with the baseline predicted adult height (PAH)
time frame: Baseline，every 3 months，the end of 3-year addendum
measure: Improvement of NAH (near adult height)
time frame: Baseline，every 3 months，the end of 3-year addendum
description: For subjects who reach NAH with treatment but fail to follow-ups before reaching FH, the improvement of NAH in comparison with the baseline PAH (predicted adult height) shall be evaluated
measure: the improvement of PAH
time frame: Baseline，every 3 months，the end of 3-year addendum
description: For subjects who fail to reach NAH with treatment and fail to follow-ups before reaching FH, the improvement of PAH in comparison with the baseline PAH shall be evaluated
measure: The changes of the scores evaluated by the Quality of Life Scale
time frame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of lean body mass (LBM) (optional)
time frame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of fat mass (torso) (FM) (optional)
time frame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of the percentage of body fat (optional)
time frame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of bone mineral density (BMD) (optional)
time frame: Baseline，every 3 months，the end of 3-year addendum
arm group label: PEG-somatropin
arm group type: Experimental
description: After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for the high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.
intervention type: Drug
intervention name: PEG-somatropin
description: After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.
arm group label: PEG-somatropin
other name: Polyethylene Glycol Recombinant Human Somatropin Injection
criteria: 
gender: All
minimum age: 4 Years
maximum age: 9 Years
healthy volunteers: No
last name: Xiaoping Luo
role: Principal Investigator
affiliation: Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: China
responsible party type: Sponsor
mesh term: Dwarfism