<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/NCT03777410</url>
  </required_header>
  <id_info>
    <org_study_id>TRV-001</org_study_id>
    <nct_id>NCT03777410</nct_id>
  </id_info>
  <brief_title>Mass Accumulation Rate (MAR) as a Predictive Biomarker in Multiple Myeloma</brief_title>
  <official_title>Mass Accumulation Rate (MAR) as a Predictive Biomarker in Multiple Myeloma</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Travera Inc</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Dana-Farber Cancer Institute</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Massachusetts General Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Weill Medical College of Cornell University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Icahn School of Medicine at Mount Sinai</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>City of Hope Comprehensive Cancer Center</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Emory University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Travera Inc</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>
      This study will collect bone marrow (BM) aspirate samples from patients with relapsed&#xD;
      refractory multiple myeloma (RRMM) prior to the start of a new treatment regimen for the&#xD;
      purposes of prospectively measuring single-cell mass accumulation rate (MAR) as a biomarker&#xD;
      of patient response to that regimen.&#xD;
&#xD;
      The primary study objective is to explore whether the single-cell MAR biomarker can predict&#xD;
      patient response in RRMM patients. In order to enable this primary objective, two patient&#xD;
      cohorts will be required. First, a small vanguard cohort of patients with treatment naïve&#xD;
      disease to define drug concentrations used for testing, and second, the main RRMM patient&#xD;
      cohort. Data will be collected to estimate the biomarker's predictive properties (accuracy,&#xD;
      sensitivity, specificity), and to support improvement of the MAR biomarker through additional&#xD;
      research and discovery within the study dataset.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">February 11, 2019</start_date>
  <completion_date type="Anticipated">January 2025</completion_date>
  <primary_completion_date type="Anticipated">January 2024</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Best Response 4 months</measure>
    <time_frame>0-4 months</time_frame>
    <description>The best International Myeloma Working Group (IMWG) response of each patient over 4 months of therapy</description>
  </primary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Anticipated">130</enrollment>
  <condition>Multiple Myeloma in Relapse</condition>
  <arm_group>
    <arm_group_label>Vanguard</arm_group_label>
    <description>(CLOSED TO ENROLLMENT) Bone marrow (BM) from this cohort of up to 30 treatment naïve subjects with a diagnosis of multiple myeloma (MM) will first be used to define sample processing pipeline performance and optimal drug dosages before sites on the study proceed to mass accumulation rate (MAR) testing of BM from the relapsed/refractory MM (RRMM) subject cohort.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Relapsed/Refractory MM</arm_group_label>
    <description>BM from this cohort of 100 relapsed subjects with a diagnosis of MM will be used to test the MAR assay's accuracy of condition by matching conditions tested in vitro to the patient's planned course of therapy. This is the main study cohort described in the Eligibility section.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Patient's pursuing initial care for their multiple myeloma at one of the 6 study sites&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Written Informed Consent provided by patient&#xD;
&#xD;
          2. MM, with the following conditions:&#xD;
&#xD;
        (CLOSED) *For patients in the Vanguard cohort*&#xD;
&#xD;
        1. Treatment naïve disease with BM clinically indicated&#xD;
&#xD;
        *For patients in the RRMM cohort*&#xD;
&#xD;
          1. Relapsed/refractory disease with BM samples clinically indicated&#xD;
&#xD;
          2. Within 4-weeks prior to initiation of 2nd-line or later therapy&#xD;
&#xD;
          3. Patient's oncologist must be planning to change the patient's next line of treatment&#xD;
             to a monotherapy or combination therapy composed exclusively of drugs from the&#xD;
             following list: Bortezomib (Velcade), Carfilzomib (Kyprolis), Lenalidomide (Revlimid),&#xD;
             Pomalidomide (Pomalyst), Cyclophosphamide (Cytoxan), Dexamethasone, Ixazomib&#xD;
             (Ninlaro), Venetoclax (Venclexta), Selinexor (Xpovio)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Unable or unwilling to provide informed consent&#xD;
&#xD;
          2. Daratumumab/Elotuzumab or other antibody-based therapeutic regimens as immediately&#xD;
             planned treatment (as prior therapy is acceptable)&#xD;
&#xD;
          3. Patient enrolled/enrolling in a clinical trial where data or specimen sharing&#xD;
             provisions preclude use in this study&#xD;
&#xD;
          4. Prior exposure to CAR-T therapy&#xD;
&#xD;
          5. Prior allogeneic stem cell transplant&#xD;
&#xD;
          6. Has received any systemic chemotherapy or RT, including palliative, within 7 days&#xD;
             prior to BM biopsy&#xD;
&#xD;
          7. Has received any Ab therapy within 4 weeks prior to BM biopsy&#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>Nikhil C Munshi, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Dana-Farber Cancer Institute</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Cara Rosenbaum, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Weill Medical College of Cornell University</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Mark Stevens, Ph.D.</last_name>
    <phone>6172999784</phone>
    <email>RRMMStudy@travera.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>City of Hope Comprehensive Cancer Center</name>
      <address>
        <city>Duarte</city>
        <state>California</state>
        <zip>91010</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Laurin Khan</last_name>
      <phone>626-218-0621</phone>
    </contact>
  </location>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Alexandra Wright</last_name>
      <phone>617-724-5251</phone>
    </contact>
  </location>
  <location>
    <facility>
      <name>Dana-Farber Cancer Institute</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02215</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Brenton LaStofka</last_name>
      <phone>617-632-1950</phone>
    </contact>
  </location>
  <location>
    <facility>
      <name>Icahn School of Medicine At Mount Sinai</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Alex Lieberman-Cribbin</last_name>
      <phone>212-241-7112</phone>
    </contact>
  </location>
  <location>
    <facility>
      <name>Weill Cornell Medicine - New York Presbyterian</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10065</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Brielle Liotta</last_name>
      <phone>646-962-9336</phone>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>December 14, 2018</study_first_submitted>
  <study_first_submitted_qc>December 14, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 17, 2018</study_first_posted>
  <last_update_submitted>May 4, 2023</last_update_submitted>
  <last_update_submitted_qc>May 4, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 8, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>functional</keyword>
  <keyword>biomarker</keyword>
  <keyword>multiple myeloma</keyword>
  <keyword>phenotypic</keyword>
  <keyword>biophysical</keyword>
  <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>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</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/NCT03777410
org study id: TRV-001
nct id: NCT03777410
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This study will collect bone marrow (BM) aspirate samples from patients with relapsed
      refractory multiple myeloma (RRMM) prior to the start of a new treatment regimen for the
      purposes of prospectively measuring single-cell mass accumulation rate (MAR) as a biomarker
      of patient response to that regimen.

      The primary study objective is to explore whether the single-cell MAR biomarker can predict
      patient response in RRMM patients. In order to enable this primary objective, two patient
      cohorts will be required. First, a small vanguard cohort of patients with treatment naïve
      disease to define drug concentrations used for testing, and second, the main RRMM patient
      cohort. Data will be collected to estimate the biomarker's predictive properties (accuracy,
      sensitivity, specificity), and to support improvement of the MAR biomarker through additional
      research and discovery within the study dataset.
observational model: Cohort
time perspective: Prospective
measure: Best Response 4 months
time frame: 0-4 months
description: The best International Myeloma Working Group (IMWG) response of each patient over 4 months of therapy
arm group label: Vanguard
description: (CLOSED TO ENROLLMENT) Bone marrow (BM) from this cohort of up to 30 treatment naïve subjects with a diagnosis of multiple myeloma (MM) will first be used to define sample processing pipeline performance and optimal drug dosages before sites on the study proceed to mass accumulation rate (MAR) testing of BM from the relapsed/refractory MM (RRMM) subject cohort.
arm group label: Relapsed/Refractory MM
description: BM from this cohort of 100 relapsed subjects with a diagnosis of MM will be used to test the MAR assay's accuracy of condition by matching conditions tested in vitro to the patient's planned course of therapy. This is the main study cohort described in the Eligibility section.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Nikhil C Munshi, M.D.
role: Principal Investigator
affiliation: Dana-Farber Cancer Institute
last name: Cara Rosenbaum, M.D.
role: Principal Investigator
affiliation: Weill Medical College of Cornell University
last name: Mark Stevens, Ph.D.
phone: 6172999784
email: RRMMStudy@travera.com
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
country: United States
responsible party type: Sponsor
mesh term: Multiple Myeloma
mesh term: Neoplasms, Plasma Cell
sharing ipd: No