<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/NCT01718899</url>
  </required_header>
  <id_info>
    <org_study_id>2010-001</org_study_id>
    <nct_id>NCT01718899</nct_id>
  </id_info>
  <brief_title>Phase 1/2a Study of Cancer Vaccine to Treat Smoldering Multiple Myeloma</brief_title>
  <official_title>A Phase 1/2a Dose Escalation Study of PVX-410, a Multi-Peptide Cancer Vaccine, in Patients With Smoldering Multiple Myeloma</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>OncoPep, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>OncoPep, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to determine the safety and tolerability of PVX-410, (a cancer&#xD;
      vaccine), treatment regimen for patients with smoldering multiple myeloma as a single agent&#xD;
      and in combination with lenalidomide.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a dose escalation, phase 1/2a study to assess the safety and tolerability of PVX-410,&#xD;
      (a multi-peptide cancer vaccine), treatment regimen in patients with smoldering multiple&#xD;
      myeloma as a single agent and in combination with lenalidomide.. Approximately 22 patients&#xD;
      will receive six (6) bi-weekly, subcutaneous injections of PVX-410 for a total of twelve (12)&#xD;
      weeks of treatment. Safety will be monitored throughout the study. Tolerability,&#xD;
      immunogenicity and clinical response will also be measured as described in the protocol.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>November 2012</start_date>
  <completion_date type="Actual">September 2016</completion_date>
  <primary_completion_date type="Actual">June 2016</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>All adverse events will be recorded.</measure>
    <time_frame>Throughout treatment phase (3 months) and follow up period (12 months)</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Immune response to the vaccine will be measured</measure>
    <time_frame>Designated timepoints during the treatment phase (3 months) and follow up phase (12 months)</time_frame>
    <description>Patient blood samples will be measured for immune response through ELISPOT and Pentamer assays.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Clinical Response will be measured.</measure>
    <time_frame>Designated timepoints during the treatment phase (3 months) and follow up phase (12 months)</time_frame>
    <description>Clinical response will be determined by the treating physician according to the International Myeloma Working Group Disease Response Criteria.</description>
  </other_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">22</enrollment>
  <condition>Smoldering Multiple Myeloma</condition>
  <arm_group>
    <arm_group_label>PVX-410, .4 mg dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Approximately 3 patients will receive 6, bi-weekly, subcutaneous injections of a .4 mg dose of PVX-410 in combination with an intramuscular injection of Hiltonol (poly ICLC). Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PVX-410, .8 mg dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Approximately 10 patients will receive 6, bi-weekly, subcutaneous injections of an .8 mg dose of PVX-410 in combination with an intramuscular injection of Hiltonol (Poly ICLC). Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PVX-410 plus lenalidomide</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Approximately 10 patients will receive 6, bi-weekly, subcutaneous injections of an .8 mg dose of PVX-410 in combination with an intramuscular injection of Hiltonol (Poly ICLC). Patients will also receive 3 cycles of lenalidomide. Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>PVX-410</intervention_name>
    <description>Approximately 10 patients will receive 6, bi-weekly, subcutaneous injections of a dose of PVX-410 in combination with an intramuscular injection of Hiltonol (Poly ICLC). Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months</description>
    <arm_group_label>PVX-410 plus lenalidomide</arm_group_label>
    <arm_group_label>PVX-410, .4 mg dose</arm_group_label>
    <arm_group_label>PVX-410, .8 mg dose</arm_group_label>
    <other_name>Revlimid</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        -  Patient has confirmed clinical diagnosis of SMM according to a definition derived from&#xD;
             the International Myeloma Working Group (IMWG) definition: serum M-protein ≥3 g/dL or&#xD;
             bone marrow clonal plasma cells (BMPC) greater than or equal to 10%, or both, along&#xD;
             with normal organ and marrow function (CRAB) within 4 weeks before baseline.&#xD;
&#xD;
          -  C: Absence of hypercalcemia, evidenced by a calcium &lt;10.5 mg/dL.&#xD;
&#xD;
          -  R: Absence of renal failure, evidenced by a creatinine &lt;2.0 mg/dL or calculated&#xD;
             creatinine clearance (using the Modification of Diet in Renal Disease [MDRD] formula)&#xD;
             &gt;50 mL/min.&#xD;
&#xD;
          -  A: Absence of anemia, evidenced by a hemoglobin &gt;10 g/dL.&#xD;
&#xD;
          -  B: Absence of lytic bone lesions on standard skeletal survey.&#xD;
&#xD;
          -  Patient is at higher than average risk of progression to active MM, defined as having&#xD;
             2 or more of the following features:&#xD;
&#xD;
          -  Serum monoclonal (M)-protein ≥3 g/dL.&#xD;
&#xD;
          -  BMPC greater than or equal to 10%.&#xD;
&#xD;
          -  Abnormal serum free light chain (FLC) ratio (0.26-1.65).&#xD;
&#xD;
          -  Patient has a life expectancy of greater than 6 months&#xD;
&#xD;
          -  Patient is human leukocyte antigen (HLA)-A2 positive.&#xD;
&#xD;
          -  Patient has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.&#xD;
&#xD;
          -  Patient has adequate bone marrow function, evidenced by a platelet count ≥75×109/L and&#xD;
             an absolute neutrophil count (ANC) ≥1.0×109/L within 2 weeks before baseline.&#xD;
&#xD;
          -  Patient has adequate hepatic function, evidenced by a bilirubin ≤2.0 mg/dL and an&#xD;
             alanine transaminase (ALT), and aspartate transaminase (AST) ≤2.5× the upper limit of&#xD;
             normal (ULN) within 2 weeks before baseline.&#xD;
&#xD;
          -  If of child-bearing potential, patient agrees to use adequate birth control measures&#xD;
             during study participation.&#xD;
&#xD;
          -  If a female of child-bearing potential, patient has negative urine pregnancy test&#xD;
             results within 2 weeks before baseline and is not lactating.&#xD;
&#xD;
          -  Patient (or his or her legally accepted representative) has provided written informed&#xD;
             consent to participate in the study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patient has symptomatic multiple myeloma, as defined by any of the following:&#xD;
&#xD;
          -  Lytic lesions or pathologic fractures.&#xD;
&#xD;
          -  Anemia (hemoglobin &lt;10 g/dL).&#xD;
&#xD;
          -  Hypercalcemia (corrected serum calcium &gt;11.5 mg/dL).&#xD;
&#xD;
          -  Renal insufficiency (creatinine &gt;2 mg/dL).&#xD;
&#xD;
          -  Other: symptomatic hyperviscosity, amyloidosis.&#xD;
&#xD;
          -  Patient has abnormal cardiac status, evidenced by any of the following:&#xD;
&#xD;
          -  New York Heart Association (NYHA) stage III or IV congestive heart failure (CHF).&#xD;
&#xD;
          -  Myocardial infarction within the previous 6 months.&#xD;
&#xD;
          -  Symptomatic cardiac arrhythmia requiring treatment or persisting despite treatment.&#xD;
&#xD;
          -  Patient is receiving any other investigational agent.&#xD;
&#xD;
          -  Patient has a current active infectious disease or positive serology for human&#xD;
             immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV).&#xD;
&#xD;
          -  Patient has a history of or current auto-immune disease.&#xD;
&#xD;
          -  Patient has been vaccinated with live attenuated vaccines within 4 weeks before study&#xD;
             vaccination.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>95 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Noopur Raje, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Massachusetts General Hospital</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Michael Wang, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>M.D. Anderson Cancer Center</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Ajay Nooka, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Emory University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Winship Cancer Institute, Emory University</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Illinois Cancer Specialists</name>
      <address>
        <city>Niles</city>
        <state>Illinois</state>
        <zip>60714</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Beth Israel Deaconess Medical Center</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Dana Farber Cancer Institute</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Texas, MD Anderson Cancer Center</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2016</verification_date>
  <study_first_submitted>October 29, 2012</study_first_submitted>
  <study_first_submitted_qc>October 29, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 31, 2012</study_first_posted>
  <last_update_submitted>September 26, 2016</last_update_submitted>
  <last_update_submitted_qc>September 26, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">September 28, 2016</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>Smoldering Multiple Myeloma</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Lenalidomide</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/NCT01718899
org study id: 2010-001
nct id: NCT01718899
lead sponsor: 
has dmc: Yes
The purpose of this study is to determine the safety and tolerability of PVX-410, (a cancer
      vaccine), treatment regimen for patients with smoldering multiple myeloma as a single agent
      and in combination with lenalidomide.
This is a dose escalation, phase 1/2a study to assess the safety and tolerability of PVX-410,
      (a multi-peptide cancer vaccine), treatment regimen in patients with smoldering multiple
      myeloma as a single agent and in combination with lenalidomide.. Approximately 22 patients
      will receive six (6) bi-weekly, subcutaneous injections of PVX-410 for a total of twelve (12)
      weeks of treatment. Safety will be monitored throughout the study. Tolerability,
      immunogenicity and clinical response will also be measured as described in the protocol.
allocation: Non-Randomized
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: All adverse events will be recorded.
time frame: Throughout treatment phase (3 months) and follow up period (12 months)
measure: Immune response to the vaccine will be measured
time frame: Designated timepoints during the treatment phase (3 months) and follow up phase (12 months)
description: Patient blood samples will be measured for immune response through ELISPOT and Pentamer assays.
measure: Clinical Response will be measured.
time frame: Designated timepoints during the treatment phase (3 months) and follow up phase (12 months)
description: Clinical response will be determined by the treating physician according to the International Myeloma Working Group Disease Response Criteria.
arm group label: PVX-410, .4 mg dose
arm group type: Experimental
description: Approximately 3 patients will receive 6, bi-weekly, subcutaneous injections of a .4 mg dose of PVX-410 in combination with an intramuscular injection of Hiltonol (poly ICLC). Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months.
arm group label: PVX-410, .8 mg dose
arm group type: Experimental
description: Approximately 10 patients will receive 6, bi-weekly, subcutaneous injections of an .8 mg dose of PVX-410 in combination with an intramuscular injection of Hiltonol (Poly ICLC). Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months.
arm group label: PVX-410 plus lenalidomide
arm group type: Experimental
description: Approximately 10 patients will receive 6, bi-weekly, subcutaneous injections of an .8 mg dose of PVX-410 in combination with an intramuscular injection of Hiltonol (Poly ICLC). Patients will also receive 3 cycles of lenalidomide. Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months
intervention type: Biological
intervention name: PVX-410
description: Approximately 10 patients will receive 6, bi-weekly, subcutaneous injections of a dose of PVX-410 in combination with an intramuscular injection of Hiltonol (Poly ICLC). Patients will complete a 12 week treatment phase and then will be followed for safety, immunogenicity and clinical response for 12 months
arm group label: PVX-410 plus lenalidomide
arm group label: PVX-410, .4 mg dose
arm group label: PVX-410, .8 mg dose
other name: Revlimid
criteria: 
gender: All
minimum age: 18 Years
maximum age: 95 Years
healthy volunteers: No
last name: Noopur Raje, MD
role: Principal Investigator
affiliation: Massachusetts General Hospital
last name: Michael Wang, MD
role: Principal Investigator
affiliation: M.D. Anderson Cancer Center
last name: Ajay Nooka, MD
role: Principal Investigator
affiliation: Emory University
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Multiple Myeloma
mesh term: Neoplasms, Plasma Cell
mesh term: Smoldering Multiple Myeloma
mesh term: Lenalidomide