<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/NCT03750994</url>
  </required_header>
  <id_info>
    <org_study_id>RuBIH2</org_study_id>
    <nct_id>NCT03750994</nct_id>
  </id_info>
  <brief_title>Economic Evaluation of Innovative Molecular Analyses in Onco-haematology</brief_title>
  <official_title>Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Assistance Publique - Hôpitaux de Paris</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Ministry of Health, France</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Assistance Publique - Hôpitaux de Paris</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      To evaluate the impact of innovative molecular diagnostics on the clinical management of&#xD;
      patients with haematological malignancies via updated Appropriate-Prescribing-Guides&#xD;
      including Next-Generation Sequencing (NGS) panels, facilitated therapeutic orientation, and&#xD;
      optimised use of costly novel therapeutics and risk-adapted treatment. A micro-costing&#xD;
      approach will be used to develop flat fee tarifs for NGS analyses.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The 12 somatic genetic cancer tests that have received temporary authorisation in France form&#xD;
      the basis of this study. These tests are not yet in the national biology reimbursement&#xD;
      nomenclature but are supported by the ministry of health in a temporary list &quot;Le référentiel&#xD;
      des actes innovants hors nomenclature de biologie et d'anatomocytopathologie&quot; (RIHN).&#xD;
&#xD;
      The PRME RuBIH2 will focus on 5 clinical situations in onco-haematology:&#xD;
&#xD;
        1. Myelodysplasia (MDS)&#xD;
&#xD;
        2. Acute lymphocytic leukemia (T) (ALL)&#xD;
&#xD;
        3. Lymphoproliferative disorders (LPD)&#xD;
&#xD;
        4. Acute myeloblastic leukemia (AML)&#xD;
&#xD;
        5. Myeloproliferative disorders (MPD)&#xD;
&#xD;
      The project is organised in 4 complementary work packages (WP): WP1 Cost evaluation, WP2&#xD;
      Prescription Guidelines, WP3 Clinical Validation and WP4 Budget Impact and Organisation.&#xD;
&#xD;
      WP1 will provide costing information on molecular tests and will build on previous studies&#xD;
      conducted in France.&#xD;
&#xD;
      WP2 will update existing prescription guidelines based on evidence from the literature and&#xD;
      evidence from the WP3. These prescription guidelines will in turn be valued and provide&#xD;
      recommendations for a flat fee bundle for pre-specified clinical situations.&#xD;
&#xD;
      WP3 will provide evidence on the clinical impact of molecular diagnosis (in particular NGS)&#xD;
      in the 5 pre-specified conditions. Changes in patient management will be measured using a&#xD;
      prospective questionnaire for an estimated 3960 molecular tests. The impact of the test on&#xD;
      the patient clinical pathway will be analysed. The impact of molecular tests on patient&#xD;
      outcome will not be measured.&#xD;
&#xD;
      WP4 will use information from WP1 and WP2 to estimate the budget impact and to provide&#xD;
      scenario analyses on the territorial organisation of molecular biology platforms. Based on&#xD;
      the estimation of the national activity of molecular onco-haematology platforms the annual&#xD;
      functioning budget required to implement molecular diagnosis in France will be estimated.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">October 18, 2018</start_date>
  <completion_date type="Anticipated">October 2020</completion_date>
  <primary_completion_date type="Anticipated">October 2020</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies.</measure>
    <time_frame>2 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of Next Generation Sequencing (NGS) that are from the oncologists internal to the platform versus external centres.</measure>
    <time_frame>2 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Average time in days between the Next Generation Sequencing (NGS) prescription being issued and the results being rendered to the clinician.</measure>
    <time_frame>2 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of prescriptions for diagnostics, prognostic, theranostics or treatment response</measure>
    <time_frame>2 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of the genetic targets that are analysed for research purposes versus immediate clinical utility for the patient.</measure>
    <time_frame>2 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of patients prescribed the Next Generation Sequencing (NGS) at the diagnostic stage or before second (or higher) line treatment.</measure>
    <time_frame>2 years</time_frame>
  </primary_outcome>
  <enrollment type="Anticipated">3960</enrollment>
  <condition>Haematological Malignancy</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Patients referred for molecular diagnosis at one of the certified molecular&#xD;
        onco-haematology platforms in France.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients with haematological malignancies referred for molecular diagnosis workup.&#xD;
             RuBIH2 will focus on 5 clinical situations in onco-haematology:&#xD;
&#xD;
               1. Myelodysplasia (MDS)&#xD;
&#xD;
               2. Acute lymphocytic leukemia (T) (ALL)&#xD;
&#xD;
               3. Lymphoproliferative disorders (LPD)&#xD;
&#xD;
               4. Acute myeloblastic leukemia (AML)&#xD;
&#xD;
               5. Myeloproliferative disorders (MPD)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Other haematological diseases not included in the list above.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Claude Preudhomme, Professor</last_name>
    <role>Principal Investigator</role>
    <affiliation>DRCI AP-HP</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Claude Preudhomme, Professor</last_name>
    <phone>+ 33 3 20 44 47 82</phone>
    <email>claude.preudhomme@chru-lille.fr</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Elisabeth Macintyre, Professor</last_name>
    <phone>+ 33 1 44 49 49 47</phone>
    <email>elizabeth.macintyre@aphp.fr</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Centre hospitalier régional universitaire de Lille</name>
      <address>
        <city>Lille</city>
        <state>Hauts De France</state>
        <zip>59000</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Claude PREUDHOMME</last_name>
      <email>Claude.PREUDHOMME@chru-lille.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Angers</name>
      <address>
        <city>Angers</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Odile Blanchet</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Avicenne AP-HP</name>
      <address>
        <city>Bobigny</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Fanny Baran-Marszak</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU de Bordeaux</name>
      <address>
        <city>Bordeaux</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Audrey Bidet</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHRU Brest</name>
      <address>
        <city>Brest</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Eric Lippert</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Estaing</name>
      <address>
        <city>Clermont Ferrand</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Marc Berger</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Henri Mondor AP-HP</name>
      <address>
        <city>Créteil</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Dominique Bories</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHRU Dijon Bourgogne</name>
      <address>
        <city>Dijon</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Mary Callanan</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Limoges</name>
      <address>
        <city>Limoges</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr David Rizzo</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Lyon Sud Pierre Bénite</name>
      <address>
        <city>Lyon</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Pierre Sujobert</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Montpellier</name>
      <address>
        <city>Montpellier</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Melissa Alamé</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Hôtel Dieu</name>
      <address>
        <city>Nantes</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Yannick LE BRIS</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Nice</name>
      <address>
        <city>Nice</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Sophie Raynaud</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Pitié-Salpêtrière AP-HP</name>
      <address>
        <city>Paris</city>
        <zip>75004</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Frédéric Davi</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Robert Debré</name>
      <address>
        <city>Paris</city>
        <zip>75004</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Hélène Cave</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital St Louis AP-HP</name>
      <address>
        <city>Paris</city>
        <zip>75004</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Jean-Michel Cayuela</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Cochin AP-HP</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Olivier Kosmider</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Necker AP-HP</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Elizabeth Macintyre</last_name>
      <email>elizabeth.macintyre@aphp.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Saint Antoine AP-HP</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor François Delhommeau</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Robert Debré Reims</name>
      <address>
        <city>Reims</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Pascale Cornillet-Lefebvre</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Pontchaillou</name>
      <address>
        <city>Rennes</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Thierry Fest</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Centre Henri-Becquerel</name>
      <address>
        <city>Rouen</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Martine Becker</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Centre hospitalier universitaire de Saint-Étienne</name>
      <address>
        <city>Saint-Étienne</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Pascale Flandrin Gresta</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpitaux Universitaires Strasbourg</name>
      <address>
        <city>Strasbourg</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Laurent Miguet</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Toulouse</name>
      <address>
        <city>Toulouse</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Professor Eric Delabesse</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Institut Gustave Roussy</name>
      <address>
        <city>Villejuif</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dr Christophe Marzac</last_name>
    </contact>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>November 2018</verification_date>
  <study_first_submitted>August 11, 2017</study_first_submitted>
  <study_first_submitted_qc>November 21, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 23, 2018</study_first_posted>
  <last_update_submitted>November 21, 2018</last_update_submitted>
  <last_update_submitted_qc>November 21, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">November 23, 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>Hematologic Neoplasms</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/NCT03750994
org study id: RuBIH2
nct id: NCT03750994
lead sponsor: 
collaborator: 
is fda regulated drug: No
is fda regulated device: No
To evaluate the impact of innovative molecular diagnostics on the clinical management of
      patients with haematological malignancies via updated Appropriate-Prescribing-Guides
      including Next-Generation Sequencing (NGS) panels, facilitated therapeutic orientation, and
      optimised use of costly novel therapeutics and risk-adapted treatment. A micro-costing
      approach will be used to develop flat fee tarifs for NGS analyses.
The 12 somatic genetic cancer tests that have received temporary authorisation in France form
      the basis of this study. These tests are not yet in the national biology reimbursement
      nomenclature but are supported by the ministry of health in a temporary list "Le référentiel
      des actes innovants hors nomenclature de biologie et d'anatomocytopathologie" (RIHN).

      The PRME RuBIH2 will focus on 5 clinical situations in onco-haematology:

        1. Myelodysplasia (MDS)

        2. Acute lymphocytic leukemia (T) (ALL)

        3. Lymphoproliferative disorders (LPD)

        4. Acute myeloblastic leukemia (AML)

        5. Myeloproliferative disorders (MPD)

      The project is organised in 4 complementary work packages (WP): WP1 Cost evaluation, WP2
      Prescription Guidelines, WP3 Clinical Validation and WP4 Budget Impact and Organisation.

      WP1 will provide costing information on molecular tests and will build on previous studies
      conducted in France.

      WP2 will update existing prescription guidelines based on evidence from the literature and
      evidence from the WP3. These prescription guidelines will in turn be valued and provide
      recommendations for a flat fee bundle for pre-specified clinical situations.

      WP3 will provide evidence on the clinical impact of molecular diagnosis (in particular NGS)
      in the 5 pre-specified conditions. Changes in patient management will be measured using a
      prospective questionnaire for an estimated 3960 molecular tests. The impact of the test on
      the patient clinical pathway will be analysed. The impact of molecular tests on patient
      outcome will not be measured.

      WP4 will use information from WP1 and WP2 to estimate the budget impact and to provide
      scenario analyses on the territorial organisation of molecular biology platforms. Based on
      the estimation of the national activity of molecular onco-haematology platforms the annual
      functioning budget required to implement molecular diagnosis in France will be estimated.
observational model: Case-Only
time perspective: Prospective
measure: Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies.
time frame: 2 years
measure: Percentage of Next Generation Sequencing (NGS) that are from the oncologists internal to the platform versus external centres.
time frame: 2 years
measure: Average time in days between the Next Generation Sequencing (NGS) prescription being issued and the results being rendered to the clinician.
time frame: 2 years
measure: Percentage of prescriptions for diagnostics, prognostic, theranostics or treatment response
time frame: 2 years
measure: Percentage of the genetic targets that are analysed for research purposes versus immediate clinical utility for the patient.
time frame: 2 years
measure: Percentage of patients prescribed the Next Generation Sequencing (NGS) at the diagnostic stage or before second (or higher) line treatment.
time frame: 2 years
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Claude Preudhomme, Professor
role: Principal Investigator
affiliation: DRCI AP-HP
last name: Claude Preudhomme, Professor
phone: + 33 3 20 44 47 82
email: claude.preudhomme@chru-lille.fr
last name: Elisabeth Macintyre, Professor
phone: + 33 1 44 49 49 47
email: elizabeth.macintyre@aphp.fr
facility: 
status: Recruiting
contact: 
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country: France
responsible party type: Sponsor
mesh term: Hematologic Neoplasms