<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/NCT04430881</url>
  </required_header>
  <id_info>
    <org_study_id>DIREGL07298</org_study_id>
    <nct_id>NCT04430881</nct_id>
  </id_info>
  <brief_title>A National Study in Patients With Unexplained Splenomegaly</brief_title>
  <acronym>SMS</acronym>
  <official_title>The SMS Study, a National Study on Prevalence of Unexplained Splenomegaly Etiologies</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Sanofi</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Sanofi</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>
      Primary Objective:&#xD;
&#xD;
      To estimate the prevalence of Gaucher disease and of other etiologies, in patients of 15&#xD;
      years old or more presenting with unexplained splenomegaly after exclusion of first&#xD;
      intention-diagnoses (e.g. portal hypertension, diagnosis or suspicion of haematological&#xD;
      malignancy, haemolytic anemia) based on basic physical and biological exams (e.g. full blood&#xD;
      count, liver enzymes, reticulocytes)&#xD;
&#xD;
      Secondary Objective:&#xD;
&#xD;
      To describe the exams and tests conducted for diagnosis purpose and the more frequent&#xD;
      associations&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Study duration per participant is between 1 and 12 months&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">September 2015</start_date>
  <completion_date type="Actual">April 27, 2021</completion_date>
  <primary_completion_date type="Actual">April 27, 2021</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>Percentage of patients diagnosed with Gaucher disease in the included population of patients with unexplained splenomegaly</measure>
    <time_frame>between 1 and 12 months</time_frame>
    <description>The diagnosis of Gaucher Disease is based on a value of beta-glucosidase enzyme activity</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage of patients with other than Gaucher disease-etiologies in the included population</measure>
    <time_frame>between 1 and 12 months</time_frame>
    <description>These will be any etiology among all the diseases that can be considered in the differential diagnosis of unexplained splenomegaly, e.g.: infection, haematological, congestive, inflammatory, neoplastic, infiltrative, benign tumors, immune, iron deficiencies and other miscellaneous rare causes</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of participants by type of exams and tests conducted for diagnosis purpose</measure>
    <time_frame>between 1 and 12 months</time_frame>
    <description>Relevant exams and tests performed, in each site, for the participant diagnosis other than Gaucher disease will be reported , this may include dried blood spot, medullary biopsy, imaging exploration</description>
  </secondary_outcome>
  <enrollment type="Actual">506</enrollment>
  <condition>Gaucher Disease</condition>
  <condition>Splenomegaly</condition>
  <biospec_retention>None Retained</biospec_retention>
  <biospec_descr>
    <textblock>
      max 1000 characters&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Participants of 15 years old or more presenting with unexplained splenomegaly after&#xD;
        exclusion of first intention-diagnoses (e.g. portal hypertension, diagnosis or suspicion of&#xD;
        haematological malignancy, haemolytic anemia) based on basic physical and biological exams&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion criteria:&#xD;
&#xD;
          -  Participants referred for the first time for splenomegaly exploration defined as :&#xD;
&#xD;
               -  Either a palpable mass on left upper abdominal quadrant, further confirmed by a ≥&#xD;
                  13 cm craniocaudal length on abdominal Imaging&#xD;
&#xD;
               -  Or a non palpable splenomegaly discovered on abdominal imaging and with a&#xD;
                  craniocaudal length ≥ 13 cm&#xD;
&#xD;
          -  Participants with splenomegaly (as defined above) of unknown origin&#xD;
&#xD;
        Exclusion criteria:&#xD;
&#xD;
          -  Participants with obvious diagnostics based on clinical exam, patient's interview and&#xD;
             the previous initial routine biological tests :&#xD;
&#xD;
               -  Diagnosis of portal hypertension&#xD;
&#xD;
               -  Diagnosis of hemolytic anemia&#xD;
&#xD;
               -  Diagnosis of hematological malignancy&#xD;
&#xD;
               -  Known diagnosis of Gaucher Disease&#xD;
&#xD;
        The above information is not intended to contain all considerations relevant to a patient's&#xD;
        potential participation in a clinical trial.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>15 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Clinical Sciences &amp; Operations</last_name>
    <role>Study Director</role>
    <affiliation>Sanofi</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Investigational site France</name>
      <address>
        <city>France</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>April 2022</verification_date>
  <study_first_submitted>June 11, 2020</study_first_submitted>
  <study_first_submitted_qc>June 11, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 12, 2020</study_first_posted>
  <last_update_submitted>April 21, 2022</last_update_submitted>
  <last_update_submitted_qc>April 21, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">April 25, 2022</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>Gaucher Disease</mesh_term>
    <mesh_term>Splenomegaly</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org</ipd_description>
  </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/NCT04430881
org study id: DIREGL07298
nct id: NCT04430881
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Primary Objective:

      To estimate the prevalence of Gaucher disease and of other etiologies, in patients of 15
      years old or more presenting with unexplained splenomegaly after exclusion of first
      intention-diagnoses (e.g. portal hypertension, diagnosis or suspicion of haematological
      malignancy, haemolytic anemia) based on basic physical and biological exams (e.g. full blood
      count, liver enzymes, reticulocytes)

      Secondary Objective:

      To describe the exams and tests conducted for diagnosis purpose and the more frequent
      associations
Study duration per participant is between 1 and 12 months
observational model: Cohort
time perspective: Prospective
measure: Percentage of patients diagnosed with Gaucher disease in the included population of patients with unexplained splenomegaly
time frame: between 1 and 12 months
description: The diagnosis of Gaucher Disease is based on a value of beta-glucosidase enzyme activity
measure: Percentage of patients with other than Gaucher disease-etiologies in the included population
time frame: between 1 and 12 months
description: These will be any etiology among all the diseases that can be considered in the differential diagnosis of unexplained splenomegaly, e.g.: infection, haematological, congestive, inflammatory, neoplastic, infiltrative, benign tumors, immune, iron deficiencies and other miscellaneous rare causes
measure: Number of participants by type of exams and tests conducted for diagnosis purpose
time frame: between 1 and 12 months
description: Relevant exams and tests performed, in each site, for the participant diagnosis other than Gaucher disease will be reported , this may include dried blood spot, medullary biopsy, imaging exploration
max 1000 characters
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 15 Years
maximum age: N/A
healthy volunteers: No
last name: Clinical Sciences & Operations
role: Study Director
affiliation: Sanofi
facility: 
country: France
responsible party type: Sponsor
mesh term: Gaucher Disease
mesh term: Splenomegaly
sharing ipd: Yes
ipd description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org