<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/NCT01676961</url>
  </required_header>
  <id_info>
    <org_study_id>10-02429</org_study_id>
    <secondary_id>P30CA016087</secondary_id>
    <nct_id>NCT01676961</nct_id>
  </id_info>
  <brief_title>Romiplostim in Increasing Low Platelet Counts in Patients With Multiple Myeloma Receiving Chemotherapy</brief_title>
  <official_title>Phase II Trial Evaluating the Efficacy and Safety of Romiplostim (Nplate) Treatment of Chemotherapy Induced Thrombocytopenia in Patients With Multiple Myeloma</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>NYU Langone Health</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Cancer Institute (NCI)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
  </sponsors>
  <source>NYU Langone Health</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This phase II trial studies how well romiplostim works in increasing low platelet counts in&#xD;
      patients with multiple myeloma receiving chemotherapy. Romiplostim may cause the body to make&#xD;
      platelets after chemotherapy&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      PRIMARY OBJECTIVES:&#xD;
&#xD;
      I. To determine if Nplate (romiplostim) is capable of increasing platelet counts to &gt; 50 x&#xD;
      10^9/L for greater than 2 weeks in myeloma patients with chemotherapy induced&#xD;
      thrombocytopenia.&#xD;
&#xD;
      SECONDARY OBJECTIVES:&#xD;
&#xD;
      I. To evaluate the toxicity of romiplostim in this patient population by standard Common&#xD;
      Toxicity Criteria (CTC).&#xD;
&#xD;
      II. To determine any increase in thrombosis or marrow fibrosis.&#xD;
&#xD;
      OUTLINE:&#xD;
&#xD;
      Patients receive romiplostim subcutaneously (SC) once weekly for up to 6 weeks. Patients&#xD;
      achieving a platelet count &gt; 50 x 10^9 then receive romiplostim once weekly during 1 course&#xD;
      of chemotherapy and may continue for as long as benefit is seen.&#xD;
&#xD;
      After completion of study treatment, patients are followed up every 3 months for 1 year.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    PI left the institution&#xD;
  </why_stopped>
  <start_date>January 2013</start_date>
  <completion_date type="Actual">November 2015</completion_date>
  <primary_completion_date type="Actual">November 2015</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>Supportive Care</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Percentage of Patients Who Have Responded</measure>
    <time_frame>8 weeks</time_frame>
    <description>Response is defined as platelet increases to greater than 50 x 10^9/L for more than 2 weeks.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage of Patients Who Experienced Thrombosis or Marrow Fibrosis</measure>
    <time_frame>Up to 1.5 years</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">5</enrollment>
  <condition>Refractory Multiple Myeloma</condition>
  <condition>Stage I Multiple Myeloma</condition>
  <condition>Stage II Multiple Myeloma</condition>
  <condition>Stage III Multiple Myeloma</condition>
  <condition>Thrombocytopenia</condition>
  <arm_group>
    <arm_group_label>Supportive care (romiplostim)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Patients receive romiplostim SC once weekly for up to 6 weeks. Patients achieving a platelet count &gt; 50 x 10^9 then receive romiplostim once weekly during 1 course of chemotherapy and may continue for as long as benefit is seen..</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>romiplostim</intervention_name>
    <description>Given SC</description>
    <arm_group_label>Supportive care (romiplostim)</arm_group_label>
    <other_name>AMG 531</other_name>
    <other_name>Amgen megakaryopoiesis protein 2</other_name>
    <other_name>Nplate</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Voluntary written informed consent before performance of any study-related procedure&#xD;
             not part of normal medical care, with the understanding that consent may be withdrawn&#xD;
             by the subject at any time without prejudice to future medical care&#xD;
&#xD;
          -  Subject is not pregnant or breast feeding, and cannot become pregnant within 30 days&#xD;
             after the end of treatment&#xD;
&#xD;
          -  Female subject of child bearing potential must be willing to use, in combination with&#xD;
             her partner, 2 forms highly effective contraception during treatment and for 1 month&#xD;
             after the end of treatment&#xD;
&#xD;
          -  Diagnosis of any stage of multiple myeloma based on standard criteria as follows:&#xD;
&#xD;
               -  Major criteria&#xD;
&#xD;
                    1. Plasmacytomas on tissue biopsy&#xD;
&#xD;
                    2. Bone marrow plasmacytosis (&gt; 30% plasma cells)&#xD;
&#xD;
                    3. Monoclonal immunoglobulin spike on serum electrophoresis (immunoglobin G&#xD;
                       [IgG] &gt; 3.5 G/dL or immunoglobin A [IgA] &gt; 2.0 G/dL) or kappa or lambda&#xD;
                       light chain excretion &gt; 1 G/day on 24 hour urine protein electrophoresis&#xD;
&#xD;
               -  Minor criteria&#xD;
&#xD;
                    1. Bone marrow plasmacytosis (10 to 30% plasma cells)&#xD;
&#xD;
                    2. Monoclonal immunoglobulin present but of lesser magnitude than given under&#xD;
                       major criteria&#xD;
&#xD;
                    3. Lytic bone lesions&#xD;
&#xD;
                    4. Normal immunoglobin M (IgM) &lt; 50 mg/dL, IgA &lt; 100 mg/dL, or IgG &lt; 600 mg/dL&#xD;
&#xD;
               -  Any of the following sets of criteria will confirm the diagnosis of multiple&#xD;
                  myeloma:&#xD;
&#xD;
                    -  Any two of the major criteria&#xD;
&#xD;
                    -  Major criterion 1 plus minor criterion b, c, or d&#xD;
&#xD;
                    -  Major criterion 3 plus minor criterion a or c&#xD;
&#xD;
                    -  Minor criteria a, b and c or a, b and d&#xD;
&#xD;
          -  Karnofsky performance status &gt;= 50&#xD;
&#xD;
          -  Platelet count =&lt; 50 x 10^9/L untransfused of at least 2 weeks duration, secondary to&#xD;
             prior chemotherapy. If there is a platelet count of &gt; or = 50 x 10(9)/L after a&#xD;
             transfusion, that value will be discounted.&#xD;
&#xD;
        This may include a combination regimen including lenalidomide; these regimens will include&#xD;
        dexamethasone, cyclophosphamide, etoposide, cisplatin (DCEP), Velcade with Doxil, Cytoxan&#xD;
        and/or lenalidomide; patients who have thrombocytopenia (CIT) from lenalidomide or from&#xD;
        radiation therapy alone will not be allowed&#xD;
&#xD;
          -  Calculated or measured creatinine clearance &gt;= 30 mL/min&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly,&#xD;
             endocrinopathy, monoclonal protein [M-protein] and skin changes)&#xD;
&#xD;
          -  Plasma cell leukemia&#xD;
&#xD;
          -  Receiving steroids daily for other medical conditions, e.g., asthma, systemic lupus&#xD;
             erythematosus, rheumatoid arthritis&#xD;
&#xD;
          -  Infection not controlled by antibiotics&#xD;
&#xD;
          -  Human immunodeficiency virus (HIV) infection; patients should provide consent for HIV&#xD;
             testing according to the institution's standard practice&#xD;
&#xD;
          -  Known active hepatitis B or C&#xD;
&#xD;
          -  Patient had myocardial infarction within 6 months prior to enrollment, New York&#xD;
             Hospital Association (NYHA) class III or IV heart failure, uncontrolled angina, severe&#xD;
             uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute&#xD;
             ischemia or active conduction system abnormalities; prior to study entry, any&#xD;
             electrocardiogram (ECG) abnormality at screening must be documented by the&#xD;
             investigator as not medically relevant&#xD;
&#xD;
          -  Diagnosed or treated for another malignancy within 3 years of enrollment, with the&#xD;
             exception of complete resection of basal cell carcinoma or squamous cell carcinoma of&#xD;
             the skin, an in situ malignancy, or low-risk prostate cancer after curative therapy&#xD;
&#xD;
          -  Other serious medical or psychiatric illness that could potentially interfere with the&#xD;
             completion of treatment according to this protocol&#xD;
&#xD;
          -  Female subject is pregnant or lactating; confirmation that the subject is not pregnant&#xD;
             must be established by a negative serum beta-human chorionic gonadotropin (hCG)&#xD;
             pregnancy test result obtained during screening; pregnancy testing is not required for&#xD;
             postmenopausal or surgically sterilized women&#xD;
&#xD;
          -  Patient has &gt; 1.5 x upper limit of normal (ULN) total bilirubin&#xD;
&#xD;
          -  Patients with existing deep venous thrombosis will be excluded&#xD;
&#xD;
          -  Patients receiving maintenance therapy with myelosuppressive medications such as&#xD;
             lenalidomide will be excluded&#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>Amitabha Mazumder</last_name>
    <role>Principal Investigator</role>
    <affiliation>NYU Langone Health</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>NYU Cancer Institute</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2018</verification_date>
  <study_first_submitted>August 29, 2012</study_first_submitted>
  <study_first_submitted_qc>August 29, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 31, 2012</study_first_posted>
  <results_first_submitted>August 3, 2017</results_first_submitted>
  <results_first_submitted_qc>August 3, 2017</results_first_submitted_qc>
  <results_first_posted type="Actual">September 5, 2017</results_first_posted>
  <last_update_submitted>September 13, 2018</last_update_submitted>
  <last_update_submitted_qc>September 13, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">October 15, 2018</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>Multiple Myeloma</mesh_term>
    <mesh_term>Neoplasms, Plasma Cell</mesh_term>
    <mesh_term>Thrombocytopenia</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Supportive Care (Romiplostim)</title>
          <description>Patients receive romiplostim SC once weekly for up to 6 weeks. Patients achieving a platelet count &gt; 50 x 10^9 then receive romiplostim once weekly during 1 course of chemotherapy and may continue for as long as benefit is seen..&#xD;
romiplostim: Given SC</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="5"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Death</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Confirmed diagnosis of multiple myeloma.</population>
      <group_list>
        <group group_id="B1">
          <title>Supportive Care (Romiplostim)</title>
          <description>Patients receive romiplostim SC once weekly for up to 6 weeks. Patients achieving a platelet count &gt; 50 x 10^9 then receive romiplostim once weekly during 1 course of chemotherapy and may continue for as long as benefit is seen..&#xD;
romiplostim: Given SC</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="5"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Percentage of Patients Who Have Responded</title>
        <description>Response is defined as platelet increases to greater than 50 x 10^9/L for more than 2 weeks.</description>
        <time_frame>8 weeks</time_frame>
        <population>Data was not analyzed. Dr. Mazumder left institution and data was not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Supportive Care (Romiplostim)</title>
            <description>Patients receive romiplostim SC once weekly for up to 6 weeks. Patients achieving a platelet count &gt; 50 x 10^9 then receive romiplostim once weekly during 1 course of chemotherapy and may continue for as long as benefit is seen..&#xD;
romiplostim: Given SC</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage of Patients Who Have Responded</title>
          <description>Response is defined as platelet increases to greater than 50 x 10^9/L for more than 2 weeks.</description>
          <population>Data was not analyzed. Dr. Mazumder left institution and data was not analyzed.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage of Patients Who Experienced Thrombosis or Marrow Fibrosis</title>
        <time_frame>Up to 1.5 years</time_frame>
        <population>Patient data was not analyzefd</population>
        <group_list>
          <group group_id="O1">
            <title>Supportive Care (Romiplostim)</title>
            <description>Patients receive romiplostim SC once weekly for up to 6 weeks. Patients achieving a platelet count &gt; 50 x 10^9 then receive romiplostim once weekly during 1 course of chemotherapy and may continue for as long as benefit is seen..&#xD;
romiplostim: Given SC</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage of Patients Who Experienced Thrombosis or Marrow Fibrosis</title>
          <population>Patient data was not analyzefd</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>4 Months</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Supportive Care (Romiplostim)</title>
          <description>Patients receive romiplostim SC once weekly for up to 6 weeks. Patients achieving a platelet count &gt; 50 x 10^9 then receive romiplostim once weekly during 1 course of chemotherapy and may continue for as long as benefit is seen..&#xD;
romiplostim: Given SC</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Amitabha Mazumder, MD</name_or_title>
      <organization>NYU Clinical Cancer Center</organization>
      <phone>(212) 731-5757</phone>
      <email>Amitabha.Mazumder@nyumc.org</email>
    </point_of_contact>
  </clinical_results>
</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/NCT01676961
org study id: 10-02429
secondary id: P30CA016087
nct id: NCT01676961
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This phase II trial studies how well romiplostim works in increasing low platelet counts in
      patients with multiple myeloma receiving chemotherapy. Romiplostim may cause the body to make
      platelets after chemotherapy
PRIMARY OBJECTIVES:

      I. To determine if Nplate (romiplostim) is capable of increasing platelet counts to > 50 x
      10^9/L for greater than 2 weeks in myeloma patients with chemotherapy induced
      thrombocytopenia.

      SECONDARY OBJECTIVES:

      I. To evaluate the toxicity of romiplostim in this patient population by standard Common
      Toxicity Criteria (CTC).

      II. To determine any increase in thrombosis or marrow fibrosis.

      OUTLINE:

      Patients receive romiplostim subcutaneously (SC) once weekly for up to 6 weeks. Patients
      achieving a platelet count > 50 x 10^9 then receive romiplostim once weekly during 1 course
      of chemotherapy and may continue for as long as benefit is seen.

      After completion of study treatment, patients are followed up every 3 months for 1 year.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Supportive Care
masking: None (Open Label)
measure: Percentage of Patients Who Have Responded
time frame: 8 weeks
description: Response is defined as platelet increases to greater than 50 x 10^9/L for more than 2 weeks.
measure: Percentage of Patients Who Experienced Thrombosis or Marrow Fibrosis
time frame: Up to 1.5 years
arm group label: Supportive care (romiplostim)
arm group type: Experimental
description: Patients receive romiplostim SC once weekly for up to 6 weeks. Patients achieving a platelet count > 50 x 10^9 then receive romiplostim once weekly during 1 course of chemotherapy and may continue for as long as benefit is seen..
intervention type: Biological
intervention name: romiplostim
description: Given SC
arm group label: Supportive care (romiplostim)
other name: AMG 531
other name: Amgen megakaryopoiesis protein 2
other name: Nplate
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Amitabha Mazumder
role: Principal Investigator
affiliation: NYU Langone Health
facility: 
country: United States
responsible party type: Sponsor
mesh term: Multiple Myeloma
mesh term: Neoplasms, Plasma Cell
mesh term: Thrombocytopenia
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 