<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/NCT03830749</url>
  </required_header>
  <id_info>
    <org_study_id>HHRC_ITP</org_study_id>
    <nct_id>NCT03830749</nct_id>
  </id_info>
  <brief_title>Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone for Subjects With Idiopathic Thrombocytopenic Purpura</brief_title>
  <official_title>A Pilot Study to Evaluate the Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone as First Line Therapy for Subjects With Idiopathic Thrombocytopenic Purpura (ITP)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Humanity &amp; Health Medical Group Limited</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Humanity &amp; Health Medical Group Limited</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>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      Current first line treatments for immune thrombocytopenia (ITP) usually have transient&#xD;
      effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate&#xD;
      whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can&#xD;
      increase the proportion of patients with prolonged response. Diagnosis of ITP is established&#xD;
      according to the American Society of Hematology guidelines. Eligible ITP subjects have&#xD;
      platelet counts &lt;30×109/L or counts &lt;50×109/L and significant bleeding symptoms (WHO bleeding&#xD;
      scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions.&#xD;
      Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks&#xD;
      plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses.&#xD;
      The primary endpoint is prolonged response rate which was defined as the proportion of&#xD;
      enrolled subjects maintaining platelet counts &gt;50×109/L for more than 6 months without any&#xD;
      ITP therapy after completion of 12-week therapy.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Suspended</overall_status>
  <why_stopped>
    Due to the COVID-19 pandemic&#xD;
  </why_stopped>
  <start_date type="Actual">July 1, 2018</start_date>
  <completion_date type="Anticipated">March 31, 2023</completion_date>
  <primary_completion_date type="Anticipated">December 31, 2022</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>The prolonged response rate</measure>
    <time_frame>6 months after completion of therapy</time_frame>
    <description>Defined as proportion of patients with platelet counts &gt; 50,000/μl at 6 months after completion of therapy and free of ITP rescue therapy</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Time to relapse</measure>
    <time_frame>6 months after completion of therapy</time_frame>
    <description>Defined as the interval from completion of 3 months treatment to platelet count&lt;30,000/ul and restarting of ITP therapy including but not limited to platelet transfusion, IVIG, corticosteroids, immunosuppressive drugs, rituximab</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Early response rate</measure>
    <time_frame>6 months after completion of therapy</time_frame>
    <description>Defined as proportion of patients requiring less than 3 courses of pulsed dexamethasone to maintain platelet counts &gt; 150,000/ul</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Health related quality of life</measure>
    <time_frame>6 months after completion of therapy</time_frame>
    <description>Assessed by SV36 HRQoL questionnaires</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">60</enrollment>
  <condition>Immune Thrombocytopenia</condition>
  <arm_group>
    <arm_group_label>Single Arm</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Eltrombopag</intervention_name>
    <description>Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone</description>
    <arm_group_label>Single Arm</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Subject has signed and dated a written informed consent.&#xD;
&#xD;
          2. Adults (≥18 years) diagnosed with ITP according to the American Society for&#xD;
             Hematology/British Committee for Standards in Haematology (ASH/BCSH) Guidelines. In&#xD;
             addition, the peripheral blood smear should support the diagnosis of ITP with no&#xD;
             evidence of other disease causative of thrombocytopenia (e.g., pseudo&#xD;
             thrombocytopenia, myelofibrosis). The physical examination should be normal or at&#xD;
             least not show signs suggestive of any disease likely to be associated with&#xD;
             thrombocytopenia.&#xD;
&#xD;
          3. No prior ITP treatment except platelet transfusions&#xD;
&#xD;
          4. Subject has no intercurrent medical event, including evidence of any thrombosis.&#xD;
&#xD;
          5. Normal prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT), no&#xD;
             history of hypercoagulable state.&#xD;
&#xD;
          6. The following clinical chemistries must be within the normal reference range:&#xD;
&#xD;
             creatinine, ALT, AST, total bilirubin, total albumin and alkaline phosphatase.&#xD;
&#xD;
          7. Subject is practicing an acceptable method of contraception (documented in chart).&#xD;
&#xD;
          8. Subject is able to understand and comply with protocol requirements and instructions.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Any clinically relevant abnormality, other than ITP, identified on the screening&#xD;
             examination, or any other medical condition or circumstance, which in the opinion of&#xD;
             the investigator makes the subject unsuitable for participation in the study or&#xD;
             suggests another diagnosis.&#xD;
&#xD;
          2. History of active malignancy or on cancer therapy. Patients with a history of&#xD;
             malignancy in complete remission for longer than 5 years are eligible&#xD;
&#xD;
          3. History of arterial or venous thrombosis (stroke, transient ischemic attack,&#xD;
             myocardial infarction, deep vein thrombosis or pulmonary embolism).&#xD;
&#xD;
          4. ≥ two of the following risk factors: Factor V Leiden, hormone replacement therapy,&#xD;
             systemic contraception containing estrogen, smoking, diabetes, hypercholesterolemia,&#xD;
             medications for hypertension or cancer.&#xD;
&#xD;
          5. Pre-existing cardiac disease (including congestive heart failure, and arrhythmia&#xD;
             requiring treatment), or clinically significant findings on resting 12-lead ECG at&#xD;
             screening.&#xD;
&#xD;
          6. Female subjects who are nursing or pregnant (positive serum or urine β-human chorionic&#xD;
             gonadotrophin (β-hCG) pregnancy test) at screening or pre-dose on Day 1.&#xD;
&#xD;
          7. History of alcohol/drug abuse.&#xD;
&#xD;
          8. Treatment with an investigational drug within 30 days or five half-lives (whichever is&#xD;
             longer) preceding the first dose of study medication&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Gregory Cheng, PhD, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Humanity &amp; Health Research Centre</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Humanity &amp; Health Research Centre</name>
      <address>
        <city>Hong Kong</city>
        <state>Hong Kong SAR</state>
        <country>Hong Kong</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Hong Kong</country>
  </location_countries>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>February 1, 2019</study_first_submitted>
  <study_first_submitted_qc>February 3, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 5, 2019</study_first_posted>
  <last_update_submitted>March 16, 2022</last_update_submitted>
  <last_update_submitted_qc>March 16, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 17, 2022</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>Thrombocytopenia</mesh_term>
    <mesh_term>Purpura</mesh_term>
    <mesh_term>Purpura, Thrombocytopenic, Idiopathic</mesh_term>
    <mesh_term>Purpura, Thrombocytopenic</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/NCT03830749
org study id: HHRC_ITP
nct id: NCT03830749
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
is us export: No
Current first line treatments for immune thrombocytopenia (ITP) usually have transient
      effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate
      whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can
      increase the proportion of patients with prolonged response. Diagnosis of ITP is established
      according to the American Society of Hematology guidelines. Eligible ITP subjects have
      platelet counts <30×109/L or counts <50×109/L and significant bleeding symptoms (WHO bleeding
      scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions.
      Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks
      plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses.
      The primary endpoint is prolonged response rate which was defined as the proportion of
      enrolled subjects maintaining platelet counts >50×109/L for more than 6 months without any
      ITP therapy after completion of 12-week therapy.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The prolonged response rate
time frame: 6 months after completion of therapy
description: Defined as proportion of patients with platelet counts > 50,000/μl at 6 months after completion of therapy and free of ITP rescue therapy
measure: Time to relapse
time frame: 6 months after completion of therapy
description: Defined as the interval from completion of 3 months treatment to platelet count<30,000/ul and restarting of ITP therapy including but not limited to platelet transfusion, IVIG, corticosteroids, immunosuppressive drugs, rituximab
measure: Early response rate
time frame: 6 months after completion of therapy
description: Defined as proportion of patients requiring less than 3 courses of pulsed dexamethasone to maintain platelet counts > 150,000/ul
measure: Health related quality of life
time frame: 6 months after completion of therapy
description: Assessed by SV36 HRQoL questionnaires
arm group label: Single Arm
arm group type: Experimental
description: Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone
intervention type: Drug
intervention name: Eltrombopag
description: Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone
arm group label: Single Arm
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Gregory Cheng, PhD, MD
role: Principal Investigator
affiliation: Humanity & Health Research Centre
facility: 
country: Hong Kong
responsible party type: Sponsor
mesh term: Thrombocytopenia
mesh term: Purpura
mesh term: Purpura, Thrombocytopenic, Idiopathic
mesh term: Purpura, Thrombocytopenic