<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/NCT04470947</url>
  </required_header>
  <id_info>
    <org_study_id>FA711C1050</org_study_id>
    <nct_id>NCT04470947</nct_id>
  </id_info>
  <brief_title>Comprehensive Genomic Profiling and Next Generation Functional Drug Screening for Patients With Aggressive Haematological Malignancies</brief_title>
  <acronym>EXALT-2</acronym>
  <official_title>Comprehensive Genomic Profiling and Next Generation Functional Drug Screening for Patients With Aggressive Haematological Malignancies: Next Generation Personal Hematology</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Medical University of Vienna</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Roche Pharma AG</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>Allcyte GmbH</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Medical University of Vienna</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>
      EXALT-2 is a prospective, randomized, three arm study for treatment decision guided either by&#xD;
      either comprehensive genomic profiling, next generation drug screening or physician's choice&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">June 10, 2020</start_date>
  <completion_date type="Anticipated">December 31, 2024</completion_date>
  <primary_completion_date type="Anticipated">December 31, 2024</primary_completion_date>
  <study_type>Observational [Patient Registry]</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>
  <target_duration>8 Months</target_duration>
  <primary_outcome>
    <measure>Percentage of patients with a ratio ≥1.3 of progression free survival (PFS) compared to most recent treatment</measure>
    <time_frame>Through study completion, an average of 8 month</time_frame>
    <description>The study aims to identify if next-generation functional drug screening (ngFDS) and/or comprehensive genomic profiling (CGP; FoundationOne®Heme) compared to physicians' choice guided treatment will have an increased percentage of patients with a ratio&#xD;
≥1.3 of progression free survival (PFS)/PFS of most recent treatment in patients with aggressive haematological malignancies</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Average Ratio of PFS/PFS of most prior treatment</measure>
    <time_frame>Through study completion, an average of 8 months</time_frame>
    <description>Average Ratio of PFS/PFS of most prior treatment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall response rate (ORR)</measure>
    <time_frame>Through study completion, an average of 8 months</time_frame>
    <description>Overall response rate (ORR)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of treatable targets identified</measure>
    <time_frame>Through study completion, an average of 8 months</time_frame>
    <description>Number of treatable targets identified</description>
  </secondary_outcome>
  <number_of_groups>3</number_of_groups>
  <enrollment type="Anticipated">150</enrollment>
  <condition>Advanced Lymphoma</condition>
  <condition>Refractory Lymphoma</condition>
  <condition>Refractory Leukemia</condition>
  <condition>Refractory Acute Myeloid Leukemia</condition>
  <condition>Refractory T-Cell Lymphoma</condition>
  <arm_group>
    <arm_group_label>Next generation functional drug screening</arm_group_label>
  </arm_group>
  <arm_group>
    <arm_group_label>Comprehensive genomic profiling</arm_group_label>
  </arm_group>
  <arm_group>
    <arm_group_label>Physician's choice</arm_group_label>
  </arm_group>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>Next generation functional drug screening</intervention_name>
    <description>High-throughput image based in-vitro drug screening on primary patient tumor cells</description>
    <arm_group_label>Comprehensive genomic profiling</arm_group_label>
    <arm_group_label>Next generation functional drug screening</arm_group_label>
    <arm_group_label>Physician's choice</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>Comprehensive genomic profiling</intervention_name>
    <description>Comprehensive targeted profiling of genetic aberrations on primary patient tumor material</description>
    <arm_group_label>Comprehensive genomic profiling</arm_group_label>
    <arm_group_label>Next generation functional drug screening</arm_group_label>
    <arm_group_label>Physician's choice</arm_group_label>
    <other_name>FoundationOne Heme</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        150 patients with relapsed/ refractory acute leukemia and relapsed/ refractory aggressive&#xD;
        lymphoma after standard treatment fulfilling inclusion criteria.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  patient is suffering from aggressive haematological disease AND has undergone at least&#xD;
             two lines of previous therapies AND/OR has undergone at least one previous therapy and&#xD;
             no standard treatment is available in the specific disease setting and disease&#xD;
             specific guidelines recommend treatment in studies.&#xD;
&#xD;
          -  duration of last response is less than 6 months defined as first day of last treatment&#xD;
             to date of relapse, the response duration has to be available with dates (dd/mm/yyyy)&#xD;
             for initiation of and relapse to previous treatment.&#xD;
&#xD;
          -  best response to previous treatment has to be available.&#xD;
&#xD;
          -  The patient is able to give written informed consent and wishes to undergo further&#xD;
             therapy&#xD;
&#xD;
          -  further therapy is medically feasible&#xD;
&#xD;
          -  tumor cell-containing samples can be obtained&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  current participation in another experimental clinical trial&#xD;
&#xD;
          -  performance status does not allow participation (ECOG ˃ 1)&#xD;
&#xD;
          -  pregnancy, tested at screening&#xD;
&#xD;
          -  patient suffers from classical or nodular, lymphocyte predominant Hodgkins lymphoma.&#xD;
&#xD;
          -  other malignoma, diagnosed &lt;1a before inclusion (except localized squamous cell&#xD;
             carcinoma of the skin, surgically curable melanoma of the skin, basal cell carcinoma&#xD;
             of the skin)&#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_contact>
    <last_name>Philipp B. Staber, MD, PhD</last_name>
    <phone>+43 140400</phone>
    <phone_ext>73782</phone_ext>
    <email>philipp.staber@meduniwien.ac.at</email>
  </overall_contact>
  <location>
    <facility>
      <name>Medical University of Vienna</name>
      <address>
        <city>Vienna</city>
        <zip>1090</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Philipp B. Staber, MD, PhD</last_name>
      <phone>+43 140400</phone>
      <phone_ext>73782</phone_ext>
      <email>philipp.staber@meduniwien.ac.at</email>
    </contact>
  </location>
  <location_countries>
    <country>Austria</country>
  </location_countries>
  <reference>
    <citation>Snijder B, Vladimer GI, Krall N, Miura K, Schmolke AS, Kornauth C, Lopez de la Fuente O, Choi HS, van der Kouwe E, Gultekin S, Kazianka L, Bigenzahn JW, Hoermann G, Prutsch N, Merkel O, Ringler A, Sabler M, Jeryczynski G, Mayerhoefer ME, Simonitsch-Klupp I, Ocko K, Felberbauer F, Mullauer L, Prager GW, Korkmaz B, Kenner L, Sperr WR, Kralovics R, Gisslinger H, Valent P, Kubicek S, Jager U, Staber PB, Superti-Furga G. Image-based ex-vivo drug screening for patients with aggressive haematological malignancies: interim results from a single-arm, open-label, pilot study. Lancet Haematol. 2017 Dec;4(12):e595-e606. doi: 10.1016/S2352-3026(17)30208-9. Epub 2017 Nov 15.</citation>
    <PMID>29153976</PMID>
  </reference>
  <verification_date>July 2020</verification_date>
  <study_first_submitted>July 7, 2020</study_first_submitted>
  <study_first_submitted_qc>July 10, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 14, 2020</study_first_posted>
  <last_update_submitted>July 10, 2020</last_update_submitted>
  <last_update_submitted_qc>July 10, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">July 14, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Medical University of Vienna</investigator_affiliation>
    <investigator_full_name>Philipp Staber, MD, PhD</investigator_full_name>
    <investigator_title>Assoc. Prof. Priv.-Doz. Dr.med.univ. Philipp Staber</investigator_title>
  </responsible_party>
  <keyword>Personalized medicine</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Lymphoma</mesh_term>
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Hematologic Neoplasms</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/NCT04470947
org study id: FA711C1050
nct id: NCT04470947
lead sponsor: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
is us export: No
EXALT-2 is a prospective, randomized, three arm study for treatment decision guided either by
      either comprehensive genomic profiling, next generation drug screening or physician's choice
observational model: Cohort
time perspective: Prospective
measure: Percentage of patients with a ratio ≥1.3 of progression free survival (PFS) compared to most recent treatment
time frame: Through study completion, an average of 8 month
description: The study aims to identify if next-generation functional drug screening (ngFDS) and/or comprehensive genomic profiling (CGP; FoundationOne®Heme) compared to physicians' choice guided treatment will have an increased percentage of patients with a ratio
≥1.3 of progression free survival (PFS)/PFS of most recent treatment in patients with aggressive haematological malignancies
measure: Average Ratio of PFS/PFS of most prior treatment
time frame: Through study completion, an average of 8 months
description: Average Ratio of PFS/PFS of most prior treatment
measure: Overall response rate (ORR)
time frame: Through study completion, an average of 8 months
description: Overall response rate (ORR)
measure: Number of treatable targets identified
time frame: Through study completion, an average of 8 months
description: Number of treatable targets identified
arm group label: Next generation functional drug screening
arm group label: Comprehensive genomic profiling
arm group label: Physician's choice
intervention type: Diagnostic Test
intervention name: Next generation functional drug screening
description: High-throughput image based in-vitro drug screening on primary patient tumor cells
arm group label: Comprehensive genomic profiling
arm group label: Next generation functional drug screening
arm group label: Physician's choice
intervention type: Diagnostic Test
intervention name: Comprehensive genomic profiling
description: Comprehensive targeted profiling of genetic aberrations on primary patient tumor material
arm group label: Comprehensive genomic profiling
arm group label: Next generation functional drug screening
arm group label: Physician's choice
other name: FoundationOne Heme
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Philipp B. Staber, MD, PhD
phone: +43 140400
phone ext: 73782
email: philipp.staber@meduniwien.ac.at
facility: 
status: Recruiting
contact: 
country: Austria
citation: Snijder B, Vladimer GI, Krall N, Miura K, Schmolke AS, Kornauth C, Lopez de la Fuente O, Choi HS, van der Kouwe E, Gultekin S, Kazianka L, Bigenzahn JW, Hoermann G, Prutsch N, Merkel O, Ringler A, Sabler M, Jeryczynski G, Mayerhoefer ME, Simonitsch-Klupp I, Ocko K, Felberbauer F, Mullauer L, Prager GW, Korkmaz B, Kenner L, Sperr WR, Kralovics R, Gisslinger H, Valent P, Kubicek S, Jager U, Staber PB, Superti-Furga G. Image-based ex-vivo drug screening for patients with aggressive haematological malignancies: interim results from a single-arm, open-label, pilot study. Lancet Haematol. 2017 Dec;4(12):e595-e606. doi: 10.1016/S2352-3026(17)30208-9. Epub 2017 Nov 15.
PMID: 29153976
responsible party type: Principal Investigator
investigator affiliation: Medical University of Vienna
investigator full name: Philipp Staber, MD, PhD
investigator title: Assoc. Prof. Priv.-Doz. Dr.med.univ. Philipp Staber
mesh term: Lymphoma
mesh term: Leukemia
mesh term: Hematologic Neoplasms
sharing ipd: No