<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/NCT04409691</url>
  </required_header>
  <id_info>
    <org_study_id>SCMC2020</org_study_id>
    <nct_id>NCT04409691</nct_id>
  </id_info>
  <brief_title>SCMC Trial on KHE With KMP (V.2020)</brief_title>
  <acronym>SCMC-KK2020</acronym>
  <official_title>A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Shanghai Children's Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Shanghai Children's Medical Center</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      a phase I trial focusing on safety and efficacy of prednison shock plus sirolimus maintenance&#xD;
      in treating Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP)&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Not yet recruiting</last_known_status>
  <start_date type="Anticipated">July 1, 2020</start_date>
  <completion_date type="Anticipated">June 30, 2023</completion_date>
  <primary_completion_date type="Anticipated">January 31, 2023</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>response to treatment</measure>
    <time_frame>6 months after taking the drug</time_frame>
    <description>Complete Response：&#xD;
platelets counts is greater than 100×10^9/L. significant volume reduction is greater than 80%. Fibrinogen levels at 2-4g/L. The surface skin of the tumor is lighter or the tumor is softer significantly.&#xD;
Partial Response:&#xD;
platelets counts is greater than 40×10^9/L. significant volume reduction is greater than 50%. Fibrinogen levels at less than 50% reduction from baseline. The surface skin of the tumor and palpation of the tumor have no change or less change.&#xD;
No Response:&#xD;
platelets counts is less than 40×10^9/L. significant volume reduction is less than 50% or the tumor is bigger. Fibrinogen levels at grater then 50% reduction from baseline. The surface skin of the tumor is darker or the tumor is harder.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>side effect rate</measure>
    <time_frame>6 months after taking the drug</time_frame>
    <description>Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">20</enrollment>
  <condition>Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)</condition>
  <arm_group>
    <arm_group_label>prednison group</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>prednison+sirolimus group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>prednison and Sirolimus</intervention_name>
    <description>If the intravenous prednisolone 4 mg / kg / d (one time in the morning and one time in the evening, 2mg / kg body weight each time) is effective, it will be gradually reduced to 2mg / kg / D (one time in the morning and one time in the evening, 1mg / kg body weight each time), gradually converted to oral prednisone of equal dose, and the hormone will be removed within 4-6 weeks. At the same time, the dosage of sirolimus oral liquid is 0.8 mg / m2 twice a day, with an interval of 12 hours, maintaining the blood concentration of 8-15 ng / ml. if there is no intolerable side effect, the treatment will last for 6 months</description>
    <arm_group_label>prednison+sirolimus group</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>prednison</intervention_name>
    <description>Prednison is taken at a dose of 4mg/kg/d. If the hormone treatment is effective, it will be gradually reduced to 2mg / kg / d (one time in the morning and one time in the evening, 1 mg / kg body weight each time), and repeated after 2 months of continuous treatment and 1 month of drug withdrawal.</description>
    <arm_group_label>prednison group</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Kaposiform Hemangioendotheliomas with Kasabach-Merritt Phenomenon&#xD;
&#xD;
          -  0 - 12 years of age at the time of study entry&#xD;
&#xD;
          -  Male or female&#xD;
&#xD;
          -  Consent of parents (or the person having parental authority in families)&#xD;
&#xD;
          -  Signed and dated written informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  with hematological diseases&#xD;
&#xD;
          -  with other solid tumors&#xD;
&#xD;
          -  with hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and&#xD;
             kidney&#xD;
&#xD;
          -  dysfunction, and cardiopulmonary insufficiency&#xD;
&#xD;
          -  with tuberculosis,cytomegalovirus and Epstein-Barr virus infection before the&#xD;
             treatment&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>12 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Song Gu</last_name>
    <phone>18930830716</phone>
    <email>gusong@shsmu.edu.cn</email>
  </overall_contact>
  <location>
    <facility>
      <name>Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine</name>
      <address>
        <city>Shanghai</city>
        <state>Shanghai</state>
        <zip>200127</zip>
        <country>China</country>
      </address>
    </facility>
    <contact>
      <last_name>Song Gu, Doctor</last_name>
      <phone>18930830716</phone>
      <email>gusong@shsmu.edu.cn</email>
    </contact>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>June 2020</verification_date>
  <study_first_submitted>May 27, 2020</study_first_submitted>
  <study_first_submitted_qc>May 27, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 1, 2020</study_first_posted>
  <last_update_submitted>June 5, 2020</last_update_submitted>
  <last_update_submitted_qc>June 5, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">June 9, 2020</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>Sarcoma, Kaposi</mesh_term>
    <mesh_term>Hemangioendothelioma</mesh_term>
    <mesh_term>Kasabach-Merritt Syndrome</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Sirolimus</mesh_term>
  </intervention_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/NCT04409691
org study id: SCMC2020
nct id: NCT04409691
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
is us export: No
a phase I trial focusing on safety and efficacy of prednison shock plus sirolimus maintenance
      in treating Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP)
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: response to treatment
time frame: 6 months after taking the drug
description: Complete Response：
platelets counts is greater than 100×10^9/L. significant volume reduction is greater than 80%. Fibrinogen levels at 2-4g/L. The surface skin of the tumor is lighter or the tumor is softer significantly.
Partial Response:
platelets counts is greater than 40×10^9/L. significant volume reduction is greater than 50%. Fibrinogen levels at less than 50% reduction from baseline. The surface skin of the tumor and palpation of the tumor have no change or less change.
No Response:
platelets counts is less than 40×10^9/L. significant volume reduction is less than 50% or the tumor is bigger. Fibrinogen levels at grater then 50% reduction from baseline. The surface skin of the tumor is darker or the tumor is harder.
measure: side effect rate
time frame: 6 months after taking the drug
description: Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms
arm group label: prednison group
arm group type: Active Comparator
arm group label: prednison+sirolimus group
arm group type: Experimental
intervention type: Drug
intervention name: prednison and Sirolimus
description: If the intravenous prednisolone 4 mg / kg / d (one time in the morning and one time in the evening, 2mg / kg body weight each time) is effective, it will be gradually reduced to 2mg / kg / D (one time in the morning and one time in the evening, 1mg / kg body weight each time), gradually converted to oral prednisone of equal dose, and the hormone will be removed within 4-6 weeks. At the same time, the dosage of sirolimus oral liquid is 0.8 mg / m2 twice a day, with an interval of 12 hours, maintaining the blood concentration of 8-15 ng / ml. if there is no intolerable side effect, the treatment will last for 6 months
arm group label: prednison+sirolimus group
intervention type: Drug
intervention name: prednison
description: Prednison is taken at a dose of 4mg/kg/d. If the hormone treatment is effective, it will be gradually reduced to 2mg / kg / d (one time in the morning and one time in the evening, 1 mg / kg body weight each time), and repeated after 2 months of continuous treatment and 1 month of drug withdrawal.
arm group label: prednison group
criteria: 
gender: All
minimum age: N/A
maximum age: 12 Years
healthy volunteers: No
last name: Song Gu
phone: 18930830716
email: gusong@shsmu.edu.cn
facility: 
contact: 
country: China
responsible party type: Sponsor
mesh term: Sarcoma, Kaposi
mesh term: Hemangioendothelioma
mesh term: Kasabach-Merritt Syndrome
mesh term: Sirolimus