<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/NCT04505995</url>
  </required_header>
  <id_info>
    <org_study_id>JMML-CAMS-2020</org_study_id>
    <nct_id>NCT04505995</nct_id>
  </id_info>
  <brief_title>Azacitidine and Homoharringtonine in JMML</brief_title>
  <official_title>Efficacy of Concomitant Use of Azacitidine and Homoharringtonine in Children With Juvenile Myelomonocytic Leukemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Air Force Military Medical University, China</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Qilu Hospital of Shandong University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Taian City Central Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>The Affiliated Hospital of Qingdao University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>The Second Hospital of Hebei Medical University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospital of Hebei Province</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Air Force Military Medical University, China</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>
      Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic&#xD;
      stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and&#xD;
      homoharringtonine, which exhibited a anticancer mechanism and has been widely used in&#xD;
      patients with relapse/refractory acute myeloid leukemia (AML). However, the efficacy of these&#xD;
      agents has not been elucidated in children with JMML. The primary objective is to assess the&#xD;
      treatment effect on response rate in this subject population.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">January 1, 2020</start_date>
  <completion_date type="Anticipated">January 1, 2025</completion_date>
  <primary_completion_date type="Anticipated">January 1, 2023</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>Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months</measure>
    <time_frame>Up to 4 Months</time_frame>
    <description>Defined as proportion of patients with sustained clinical complete remission [cCR] or clinical partial remission [cPR] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4 months).</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">30</enrollment>
  <condition>JMML</condition>
  <arm_group>
    <arm_group_label>Intervention group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Azacitidine and homoharringtonine</intervention_name>
    <description>Concommitant use of azacitidine and homoharringtonine</description>
    <arm_group_label>Intervention group</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Newly diagnosed JMML, with PB and BM confirming diagnosis prior to informed consent&#xD;
             signature, with one of the following:&#xD;
&#xD;
               1. somatic mutation in PTPN11&#xD;
&#xD;
               2. somatic mutation in KRAS&#xD;
&#xD;
               3. somatic mutation in NRAS and HbF % &gt; 5x normal value for age&#xD;
&#xD;
               4. clinical diagnosis of neurofibromatosis Type 1.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  1. Any condition that would prevent the subject from participating in the study.&#xD;
&#xD;
             2. Any condition including the presence of laboratory abnormalities, which places the&#xD;
             subject at unacceptable risk if participated in the study.&#xD;
&#xD;
             3. Any condition that confounds the ability to interpret data from the study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Month</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Wen-Yu Yang, M.D.</last_name>
    <phone>+86-22-23909138</phone>
    <email>yangwenyu@ihcams.ac.cn</email>
  </overall_contact>
  <location>
    <facility>
      <name>Institute of Hematology &amp; Blood Diseases Hospital, Chinese Academy of Medical Sciences &amp; Peking Union Medical College</name>
      <address>
        <city>Tianjin</city>
        <state>Tianjin</state>
        <zip>300020</zip>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Xiao-Fan Zhu, M.D.</last_name>
      <phone>+86-22-23909001</phone>
      <email>xfzhu@ihcams.ac.cn</email>
    </contact>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>August 2020</verification_date>
  <study_first_submitted>August 6, 2020</study_first_submitted>
  <study_first_submitted_qc>August 6, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">August 10, 2020</study_first_posted>
  <last_update_submitted>August 6, 2020</last_update_submitted>
  <last_update_submitted_qc>August 6, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">August 10, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Air Force Military Medical University, China</investigator_affiliation>
    <investigator_full_name>Lipeng Liu</investigator_full_name>
    <investigator_title>Dr</investigator_title>
  </responsible_party>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Azacitidine</mesh_term>
    <mesh_term>Homoharringtonine</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04505995
org study id: JMML-CAMS-2020
nct id: NCT04505995
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
is fda regulated drug: No
is fda regulated device: No
Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic
      stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and
      homoharringtonine, which exhibited a anticancer mechanism and has been widely used in
      patients with relapse/refractory acute myeloid leukemia (AML). However, the efficacy of these
      agents has not been elucidated in children with JMML. The primary objective is to assess the
      treatment effect on response rate in this subject population.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months
time frame: Up to 4 Months
description: Defined as proportion of patients with sustained clinical complete remission [cCR] or clinical partial remission [cPR] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4 months).
arm group label: Intervention group
arm group type: Experimental
intervention type: Drug
intervention name: Azacitidine and homoharringtonine
description: Concommitant use of azacitidine and homoharringtonine
arm group label: Intervention group
criteria: 
gender: All
minimum age: 1 Month
maximum age: 18 Years
healthy volunteers: No
last name: Wen-Yu Yang, M.D.
phone: +86-22-23909138
email: yangwenyu@ihcams.ac.cn
facility: 
status: Recruiting
contact: 
country: China
responsible party type: Principal Investigator
investigator affiliation: Air Force Military Medical University, China
investigator full name: Lipeng Liu
investigator title: Dr
mesh term: Azacitidine
mesh term: Homoharringtonine
sharing ipd: Undecided