<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/NCT03032055</url>
  </required_header>
  <id_info>
    <org_study_id>Presev 2 IRB 00003835</org_study_id>
    <nct_id>NCT03032055</nct_id>
  </id_info>
  <brief_title>Validation of a Predictive Score of Acute Chest Syndrome</brief_title>
  <acronym>Presev2</acronym>
  <official_title>Validation of a Predictive Score of Acute Chest Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Soutien aux Actions contre les Maladies du Globule Rouge</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>ADDMEDICA SASA</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>Pierre Fabre Laboratories</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Soutien aux Actions contre les Maladies du Globule Rouge</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Vaso-Occlusive Crisis (VOC), the most common manifestation of sickle cell disease (SCD), is&#xD;
      the first cause of death, particularly when complicated by an acute chest syndrome (ACS).&#xD;
&#xD;
      The PRESEV score could help the physicians to better manage VOC and could be used for future&#xD;
      therapeutic trials. This predictive score of secondary ACS has to be validated in a&#xD;
      multicenter international study.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      International multicentre prospective observational study.&#xD;
&#xD;
      Prediction of an ACS within 15 days after admission by the PRESEV2 score at arrival.&#xD;
&#xD;
      Validation of a Predictive Score of Acute Chest Syndrome (Presev2) associates a categorical&#xD;
      pain score of the spine /or pelvis and 3 biological parameters: Reticulocytes, Leucocytes and&#xD;
      Hemoglobin.&#xD;
&#xD;
      ACS is defined by crepitant or bronchial breathing or the association of new radiologic&#xD;
      infiltrate and chest pain&#xD;
&#xD;
      Inclusion criteria:&#xD;
&#xD;
        -  Children (&gt;2 years) and adults&#xD;
&#xD;
        -  Male and Female&#xD;
&#xD;
        -  Homozygous SCD patients&#xD;
&#xD;
        -  The patient can only be included once&#xD;
&#xD;
        -  VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness&#xD;
           affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine&#xD;
           and/or pelvis) that required hospitalization and opioids (level 3), and is not&#xD;
           attributable to other causes.&#xD;
&#xD;
        -  Patient has health care insurance (in Europe)&#xD;
&#xD;
        -  Written consent given after being informed of the purpose, progress and potential risks&#xD;
&#xD;
      Exclusion criteria:&#xD;
&#xD;
        -  No inaugural Acute Chest Syndrome&#xD;
&#xD;
        -  Homeless patients&#xD;
&#xD;
        -  Deprived of their liberty by a court or administrative order or under guardianship&#xD;
&#xD;
        -  Unable to understand the purpose and conditions of carrying out the study, unable to&#xD;
           give consent&#xD;
&#xD;
      STUDY SCHEDULE Screening and inclusion once patients are admitted at the accident and&#xD;
      emergency department or medical day unit.&#xD;
&#xD;
      Inclusion visit (day 1) Once admitted at the accident and emergency department, the patient&#xD;
      will be informed about the protocol and asked to participate in the study. Informed consent&#xD;
      will be obtained according to local regulations by signing the informed consent form. The&#xD;
      inclusion and non-inclusion criteria will be verified.&#xD;
&#xD;
      Demographic data, current and previous treatments taken within one month, and medical history&#xD;
      will be recorded.&#xD;
&#xD;
      The following parameters of the score will be collected:&#xD;
&#xD;
      Reticulocytes and/or leucocytes counts, urea (mmol/L) and Categorical pain score.&#xD;
&#xD;
      The Score is adjusted with Hydroxyurea treatment (Yes/No) and Hb level (g/dL). Plasmodium&#xD;
      falciparum test will be performed only in Africa. Hemoculture if fever (&gt;38°C) is recorded&#xD;
      within the 2 first days after admission.&#xD;
&#xD;
      Temperature, Blood pressure, Oxygen saturation, Respiratory rate, Visual analogue score will&#xD;
      be recorded.&#xD;
&#xD;
      STUDY DURATION Inclusion period: 2 years Per patient: 15 days Total duration of the study: 2&#xD;
      years&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">January 1, 2016</start_date>
  <completion_date type="Anticipated">December 31, 2023</completion_date>
  <primary_completion_date type="Anticipated">December 31, 2023</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>Primary Outcome Measure of Validation of a Predictive Score of Acute Chest Syndrome</measure>
    <time_frame>2 years</time_frame>
    <description>The primary outcome mesure is the occurrence of an ACS defined by crepitant or bronchial breathing or the association of new radiologic infiltrats and chest pain during the first 15 days of hospitalization for Vaso Occlusive Crisis</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Secondary Outcome Measures: Validation of a Predictive Score of Acute Chest Syndrome</measure>
    <time_frame>2 years</time_frame>
    <description>Association of the PRESEV 2 score with the following parameters collected for each patient:&#xD;
The number of days of hospitalization&#xD;
Blood transfusion requirement&#xD;
The number of hospitalization in intensive care unit&#xD;
Death</description>
  </secondary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Anticipated">800</enrollment>
  <condition>Vaso Occlusive Crisis</condition>
  <condition>Acute Chest Syndrome</condition>
  <arm_group>
    <arm_group_label>VOC</arm_group_label>
    <description>Patients who won't develop a secondary Acute chest syndrome during a vaso occlusive crisis within 15 days after admission.&#xD;
Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate and chest pain or decreased breath sounds .&#xD;
A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults. It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>2°ACS</arm_group_label>
    <description>Patients who will develop a secondary acute chest syndrome during a vaso occlusive crisis within 15 days after admission.&#xD;
Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate AND chest pain or decreased breath sounds .&#xD;
A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults.It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries.</description>
  </arm_group>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      serum and DNA in Africa&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Children (&gt;2 years) and adults&#xD;
&#xD;
          -  Male and Female&#xD;
&#xD;
          -  Homozygous SCD patients&#xD;
&#xD;
          -  VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness&#xD;
             affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull),&#xD;
             spine and/or pelvis) that required hospitalization and opioids (level 3), and is not&#xD;
             attributable to other causes&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Inclusion criteria:&#xD;
&#xD;
          -  Children (&gt;2 years) and adults&#xD;
&#xD;
          -  Male and Female&#xD;
&#xD;
          -  Homozygous SCD patients&#xD;
&#xD;
          -  VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness&#xD;
             affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull),&#xD;
             spine and/or pelvis) that required hospitalization and opioids (level 3), and is not&#xD;
             attributable to other causes.&#xD;
&#xD;
          -  Patient has health care insurance (in Europe)&#xD;
&#xD;
          -  Written consent given after being informed of the purpose, progress and potential&#xD;
             risks&#xD;
&#xD;
        Exclusion criteria:&#xD;
&#xD;
          -  No inaugural Acute Chest Syndrome&#xD;
&#xD;
          -  Homeless patients&#xD;
&#xD;
          -  Deprived of their liberty by a court or administrative order or under guardianship&#xD;
&#xD;
          -  Unable to understand the purpose and conditions of carrying out the study, unable to&#xD;
             give consent&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>BARTOLUCCI Pablo, MD PhD, Pr</last_name>
    <role>Principal Investigator</role>
    <affiliation>APHP, UPEC, INSERM</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>DJOUMAD Sabrina, Master's</last_name>
    <phone>33 (1) 49812441</phone>
    <email>sabrina.djoumad@aphp.fr</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>BERKENOU Jugurtha, Master's</last_name>
    <phone>33 (1) 49814414</phone>
    <email>jugurtha.berkenou@aphp.fr</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Henri Mondor Hospital</name>
      <address>
        <city>Creteil</city>
        <zip>94000</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Pablo BARTOLUCCI, Professor</last_name>
      <phone>33 (1) 49817406</phone>
      <email>pablo.bartolucci@aphp.fr</email>
    </contact>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>December 2022</verification_date>
  <study_first_submitted>November 18, 2015</study_first_submitted>
  <study_first_submitted_qc>January 23, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 26, 2017</study_first_posted>
  <last_update_submitted>December 12, 2022</last_update_submitted>
  <last_update_submitted_qc>December 12, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">December 13, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>sickle cell desease</keyword>
  <keyword>vaso occlusive crisis</keyword>
  <keyword>acute chest syndrome</keyword>
  <keyword>Validation of predictif score of acute chest syndrome</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Acute Chest Syndrome</mesh_term>
    <mesh_term>Syndrome</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/NCT03032055
org study id: Presev 2 IRB 00003835
nct id: NCT03032055
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
Vaso-Occlusive Crisis (VOC), the most common manifestation of sickle cell disease (SCD), is
      the first cause of death, particularly when complicated by an acute chest syndrome (ACS).

      The PRESEV score could help the physicians to better manage VOC and could be used for future
      therapeutic trials. This predictive score of secondary ACS has to be validated in a
      multicenter international study.
International multicentre prospective observational study.

      Prediction of an ACS within 15 days after admission by the PRESEV2 score at arrival.

      Validation of a Predictive Score of Acute Chest Syndrome (Presev2) associates a categorical
      pain score of the spine /or pelvis and 3 biological parameters: Reticulocytes, Leucocytes and
      Hemoglobin.

      ACS is defined by crepitant or bronchial breathing or the association of new radiologic
      infiltrate and chest pain

      Inclusion criteria:

        -  Children (>2 years) and adults

        -  Male and Female

        -  Homozygous SCD patients

        -  The patient can only be included once

        -  VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness
           affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine
           and/or pelvis) that required hospitalization and opioids (level 3), and is not
           attributable to other causes.

        -  Patient has health care insurance (in Europe)

        -  Written consent given after being informed of the purpose, progress and potential risks

      Exclusion criteria:

        -  No inaugural Acute Chest Syndrome

        -  Homeless patients

        -  Deprived of their liberty by a court or administrative order or under guardianship

        -  Unable to understand the purpose and conditions of carrying out the study, unable to
           give consent

      STUDY SCHEDULE Screening and inclusion once patients are admitted at the accident and
      emergency department or medical day unit.

      Inclusion visit (day 1) Once admitted at the accident and emergency department, the patient
      will be informed about the protocol and asked to participate in the study. Informed consent
      will be obtained according to local regulations by signing the informed consent form. The
      inclusion and non-inclusion criteria will be verified.

      Demographic data, current and previous treatments taken within one month, and medical history
      will be recorded.

      The following parameters of the score will be collected:

      Reticulocytes and/or leucocytes counts, urea (mmol/L) and Categorical pain score.

      The Score is adjusted with Hydroxyurea treatment (Yes/No) and Hb level (g/dL). Plasmodium
      falciparum test will be performed only in Africa. Hemoculture if fever (>38°C) is recorded
      within the 2 first days after admission.

      Temperature, Blood pressure, Oxygen saturation, Respiratory rate, Visual analogue score will
      be recorded.

      STUDY DURATION Inclusion period: 2 years Per patient: 15 days Total duration of the study: 2
      years
observational model: Cohort
time perspective: Prospective
measure: Primary Outcome Measure of Validation of a Predictive Score of Acute Chest Syndrome
time frame: 2 years
description: The primary outcome mesure is the occurrence of an ACS defined by crepitant or bronchial breathing or the association of new radiologic infiltrats and chest pain during the first 15 days of hospitalization for Vaso Occlusive Crisis
measure: Secondary Outcome Measures: Validation of a Predictive Score of Acute Chest Syndrome
time frame: 2 years
description: Association of the PRESEV 2 score with the following parameters collected for each patient:
The number of days of hospitalization
Blood transfusion requirement
The number of hospitalization in intensive care unit
Death
arm group label: VOC
description: Patients who won't develop a secondary Acute chest syndrome during a vaso occlusive crisis within 15 days after admission.
Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate and chest pain or decreased breath sounds .
A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults. It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries.
arm group label: 2°ACS
description: Patients who will develop a secondary acute chest syndrome during a vaso occlusive crisis within 15 days after admission.
Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate AND chest pain or decreased breath sounds .
A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults.It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries.
serum and DNA in Africa
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 2 Years
maximum age: N/A
healthy volunteers: No
last name: BARTOLUCCI Pablo, MD PhD, Pr
role: Principal Investigator
affiliation: APHP, UPEC, INSERM
last name: DJOUMAD Sabrina, Master's
phone: 33 (1) 49812441
email: sabrina.djoumad@aphp.fr
last name: BERKENOU Jugurtha, Master's
phone: 33 (1) 49814414
email: jugurtha.berkenou@aphp.fr
facility: 
status: Recruiting
contact: 
country: France
responsible party type: Sponsor
mesh term: Acute Chest Syndrome
mesh term: Syndrome
sharing ipd: No