xml: <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/NCT05961826</url>
    </required_header>
    <id_info>
      <org_study_id>C16-14</org_study_id>
      <nct_id>NCT05961826</nct_id>
    </id_info>
    <brief_title>COLPAC (RaDiCo Cohort) (RaDiCo-COLPAC)</brief_title>
    <acronym>COLPAC</acronym>
    <official_title>National Cohort About Epidemiology, Clinical and Genetic Heterogeneity of the &quot;Low-Phospholipid-Associated Cholelithiasis&quot; (LPAC) Syndrome</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Institut National de la Santé Et de la Recherche Médicale, France</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Institut National de la Santé Et de la Recherche Médicale, France</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>
        The goal of this observational study is to describe the various clinical, biological and&#xD;
        radiological manifestations of LPAC syndrome, defined according to standard diagnostic&#xD;
        criteria, or according to criteria extended to all symptomatic recurrent biliary lithiasis,&#xD;
        and delineate the various possible evolutions.&#xD;
  &#xD;
        Participants will be followed for 5 years and the inclusion sites will collect the necessary&#xD;
        data at least once a year as part of routine patient care. A quality of life&#xD;
        self-questionnaire will be completed by participants during these visits.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Active, not recruiting</overall_status>
    <start_date type="Actual">November 6, 2017</start_date>
    <completion_date type="Anticipated">June 30, 2028</completion_date>
    <primary_completion_date type="Anticipated">June 30, 2028</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>Other</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>Descriptive analysis of the clinical manifestations of LPAC syndrome like personal and family medical history of the disease, clinical features, medical complications, co-morbidities, death (age of onset and cause).</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Descriptive analysis of the biological manifestations of LPAC syndrome like hepatic biochemical tests and glucido-lipid tests</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Descriptive analysis of the radiological manifestations of LPAC syndrome like presence of stones or signs of intrahepatic, vesicular or main bile duct micro-lithiasis</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <secondary_outcome>
      <measure>Description of diagnostic practices by a descriptive analysis of sequences of medical procedures used to diagnose LPAC syndrome.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Description of therapeutic practices by a descriptive analysis of the different curative and symptomatic treatments offered depending on the stage of the disease and the type of extra-hepatic complications.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Description of patient management practices.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Identification of new diagnostics by refining the collection of semiological fields of 1st degree relatives and including patients with recurrent symptomatic biliary lithiasis who do not fully meet the current diagnostic criteria for LPAC syndrome</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
      <description>The extension of the criteria will be proposed on the basis of the collection of all the semiological fields of patients diagnosed with an LPAC syndrome defined according to the usual or extended criteria and their 1st degree relatives.&#xD;
  The extended diagnostic criteria are :&#xD;
  Age of onset of the disease over 40 years of age&#xD;
  The presence of recurrent biliary lithiasis without radiological signs characteristic of LPAC syndrome (comet tails or intrahepatic lithiasis).&#xD;
  Patients with symptomatic gallstones who have only one of the 3 criteria for LPAC syndrome (onset before the age of 40, intrahepatic microlithiasis, recurrence after cholecystectomy).</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Evaluation of response to medical and interventional treatments.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
      <description>This evaluation criterion will be the search for correlation between response or non-response to medical and interventional treatments. This will be analysed by looking at the presence or absence of certain clinical, biochemical, molecular and radiological features of the disease such as :&#xD;
  No reduction in the frequency or intensity of painful attacks.&#xD;
  No reduction in the number of complications.&#xD;
  No improvement or normalisation of liver tests.&#xD;
  No regression or disappearance of radiological signs of intrahepatic lithiasis&#xD;
  No improvement in the criteria for assessing quality of life and impact on social life</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Identification of prognostic factors (clinical, biochemical, radiological) associated with a poor therapeutic response by assessing the occurrence of events such as : - Fatal or non-fatal complications - Death due to hepatic or other causes</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Assessing the impact of LPAC syndrome on quality of life.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
      <description>Impact of disease on quality of life will be evaluated through scores of quality of life questionnaires (SF-36)</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Evaluation of the proportion of patients referred to LPAC within the population referred to the hepatology and/or digestive surgery departments for biliary lithiasis during the same study period</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
      <description>The relative prevalence of LPAC syndrome (number of cases of LPAC syndrome compared with the number of cases of gallbladder disease) will be measured from 1 March 2016 to 28 February 2017, based on cases recorded in the centres participating in the study.&#xD;
  The relative incidence (number of new cases of LPAC syndrome compared with the number of new cases of gallstones) will be measured over the same period, in the same centres.&#xD;
  The total number of patients seen in hospital or in consultation during the same period for gallstones will be collected in each participating hospital using the pmsi code.</description>
    </secondary_outcome>
    <other_outcome>
      <measure>Deepening the study of the genotype/phenotype correlations of the ABCB4 gene with assessment of the number of painful attacks and complications, presence of radiological signs, measurement of biological assays, good or poor response to treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </other_outcome>
    <other_outcome>
      <measure>Documenting the genetic transmission profile and heritability (penetrance) of the LPAC syndrome by analysing the phenotypes and genotypes of patients and their 1st degree relatives (whether or not they have declared illness).</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
      <description>The evaluation criteria will be :&#xD;
  the collection of common genetic mutations in the patient and 1st degree relatives (carried out as part of the diagnostic study of the index case)&#xD;
  the search for common phenotypes in the patient and 1st degree relatives (carried out as part of the diagnostic study of the index case)</description>
    </other_outcome>
    <other_outcome>
      <measure>Search for new susceptibility genes in patients without gene alterations of ABCB4</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </other_outcome>
    <other_outcome>
      <measure>Search for modulating genes in mutated and non-mutated ABCB4 patients.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </other_outcome>
    <enrollment type="Anticipated">650</enrollment>
    <condition>Low Phospholipid Associated Cholelithiasis</condition>
    <eligibility>
      <study_pop>
        <textblock>
          This study concerns all prevalent and incident patients who meet the standard or extended&#xD;
          diagnostic criteria for LPAC syndrome, whatever the existing genetic data concerning ABCB4,&#xD;
          or who have been referred as having a mutation in the ABCB4 gene in a clinical context of&#xD;
          biliary lithiasis.&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Children or adults meeting the standard* or extended** diagnostic criteria for LPAC&#xD;
               syndrome:&#xD;
  &#xD;
                 1. First symptoms before the age of 40 years&#xD;
  &#xD;
                 2. Radiological images compatible with the existence of intrahepatic lithiasis&#xD;
  &#xD;
                 3. Recurrence of symptoms after cholecystectomy&#xD;
  &#xD;
                    (*) standard criteria: symptomatic biliary lithiasis with at least 2 out of 3&#xD;
                    criteria&#xD;
  &#xD;
                    (**) extended criteria: symptomatic biliary lithiasis with 1 out of 3 criteria&#xD;
  &#xD;
                    Exclusion Criteria:&#xD;
  &#xD;
            -  Patients who have undergone liver transplantation&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>13 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Christophe CORPECHOT</last_name>
      <role>Principal Investigator</role>
      <affiliation>INSERM UMR_S938</affiliation>
    </overall_official>
    <location>
      <facility>
        <name>Centre Hospitalier Intercommunal Aix-Pertuis</name>
        <address>
          <city>Aix-en-Provence</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Centre Hospitalier Annecy Genevois</name>
        <address>
          <city>Annecy</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Avicenne</name>
        <address>
          <city>Bobigny</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Haut-Lévêque</name>
        <address>
          <city>Bordeaux</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Saint Camille</name>
        <address>
          <city>Bry-sur-Marne</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Côte de Nacre</name>
        <address>
          <city>Caen</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Beaujon</name>
        <address>
          <city>Clichy</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Centre Hospitalier Sud Francilien</name>
        <address>
          <city>Corbeil-Essonnes</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Centre Hospitalier Intercommunal de Créteil</name>
        <address>
          <city>Créteil</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital François Mitterrand</name>
        <address>
          <city>Dijon</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Michallon</name>
        <address>
          <city>Grenoble</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Claude Huriez</name>
        <address>
          <city>Lille</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital de la Croix-Rousse</name>
        <address>
          <city>Lyon</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Centre hospitalier régional d'Orléans</name>
        <address>
          <city>Orléans</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Saint-Antoine</name>
        <address>
          <city>Paris</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Jean Bernard</name>
        <address>
          <city>Poitiers</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Robert Debré</name>
        <address>
          <city>Reims</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Charles Nicolle</name>
        <address>
          <city>Rouen</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Civil</name>
        <address>
          <city>Strasbourg</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Rangueil</name>
        <address>
          <city>Toulouse</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Hôpital Paul Brousse</name>
        <address>
          <city>Villejuif</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location_countries>
      <country>France</country>
    </location_countries>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>July 6, 2023</study_first_submitted>
    <study_first_submitted_qc>July 18, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">July 27, 2023</study_first_posted>
    <last_update_submitted>July 18, 2023</last_update_submitted>
    <last_update_submitted_qc>July 18, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">July 27, 2023</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>Cholelithiasis</mesh_term>
      <mesh_term>Cholecystolithiasis</mesh_term>
      <mesh_term>Gallstones</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05961826
  org study id: C16-14
  nct id: NCT05961826
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  The goal of this observational study is to describe the various clinical, biological and
        radiological manifestations of LPAC syndrome, defined according to standard diagnostic
        criteria, or according to criteria extended to all symptomatic recurrent biliary lithiasis,
        and delineate the various possible evolutions.
  
        Participants will be followed for 5 years and the inclusion sites will collect the necessary
        data at least once a year as part of routine patient care. A quality of life
        self-questionnaire will be completed by participants during these visits.
  observational model: Cohort
  time perspective: Other
  measure: Descriptive analysis of the clinical manifestations of LPAC syndrome like personal and family medical history of the disease, clinical features, medical complications, co-morbidities, death (age of onset and cause).
  time frame: Through study completion, an average of 5 years
  measure: Descriptive analysis of the biological manifestations of LPAC syndrome like hepatic biochemical tests and glucido-lipid tests
  time frame: Through study completion, an average of 5 years
  measure: Descriptive analysis of the radiological manifestations of LPAC syndrome like presence of stones or signs of intrahepatic, vesicular or main bile duct micro-lithiasis
  time frame: Through study completion, an average of 5 years
  measure: Description of diagnostic practices by a descriptive analysis of sequences of medical procedures used to diagnose LPAC syndrome.
  time frame: Through study completion, an average of 5 years
  measure: Description of therapeutic practices by a descriptive analysis of the different curative and symptomatic treatments offered depending on the stage of the disease and the type of extra-hepatic complications.
  time frame: Through study completion, an average of 5 years
  measure: Description of patient management practices.
  time frame: Through study completion, an average of 5 years
  measure: Identification of new diagnostics by refining the collection of semiological fields of 1st degree relatives and including patients with recurrent symptomatic biliary lithiasis who do not fully meet the current diagnostic criteria for LPAC syndrome
  time frame: Through study completion, an average of 5 years
  description: The extension of the criteria will be proposed on the basis of the collection of all the semiological fields of patients diagnosed with an LPAC syndrome defined according to the usual or extended criteria and their 1st degree relatives.
  The extended diagnostic criteria are :
  Age of onset of the disease over 40 years of age
  The presence of recurrent biliary lithiasis without radiological signs characteristic of LPAC syndrome (comet tails or intrahepatic lithiasis).
  Patients with symptomatic gallstones who have only one of the 3 criteria for LPAC syndrome (onset before the age of 40, intrahepatic microlithiasis, recurrence after cholecystectomy).
  measure: Evaluation of response to medical and interventional treatments.
  time frame: Through study completion, an average of 5 years
  description: This evaluation criterion will be the search for correlation between response or non-response to medical and interventional treatments. This will be analysed by looking at the presence or absence of certain clinical, biochemical, molecular and radiological features of the disease such as :
  No reduction in the frequency or intensity of painful attacks.
  No reduction in the number of complications.
  No improvement or normalisation of liver tests.
  No regression or disappearance of radiological signs of intrahepatic lithiasis
  No improvement in the criteria for assessing quality of life and impact on social life
  measure: Identification of prognostic factors (clinical, biochemical, radiological) associated with a poor therapeutic response by assessing the occurrence of events such as : - Fatal or non-fatal complications - Death due to hepatic or other causes
  time frame: Through study completion, an average of 5 years
  measure: Assessing the impact of LPAC syndrome on quality of life.
  time frame: Through study completion, an average of 5 years
  description: Impact of disease on quality of life will be evaluated through scores of quality of life questionnaires (SF-36)
  measure: Evaluation of the proportion of patients referred to LPAC within the population referred to the hepatology and/or digestive surgery departments for biliary lithiasis during the same study period
  time frame: Through study completion, an average of 5 years
  description: The relative prevalence of LPAC syndrome (number of cases of LPAC syndrome compared with the number of cases of gallbladder disease) will be measured from 1 March 2016 to 28 February 2017, based on cases recorded in the centres participating in the study.
  The relative incidence (number of new cases of LPAC syndrome compared with the number of new cases of gallstones) will be measured over the same period, in the same centres.
  The total number of patients seen in hospital or in consultation during the same period for gallstones will be collected in each participating hospital using the pmsi code.
  measure: Deepening the study of the genotype/phenotype correlations of the ABCB4 gene with assessment of the number of painful attacks and complications, presence of radiological signs, measurement of biological assays, good or poor response to treatment
  time frame: Through study completion, an average of 5 years
  measure: Documenting the genetic transmission profile and heritability (penetrance) of the LPAC syndrome by analysing the phenotypes and genotypes of patients and their 1st degree relatives (whether or not they have declared illness).
  time frame: Through study completion, an average of 5 years
  description: The evaluation criteria will be :
  the collection of common genetic mutations in the patient and 1st degree relatives (carried out as part of the diagnostic study of the index case)
  the search for common phenotypes in the patient and 1st degree relatives (carried out as part of the diagnostic study of the index case)
  measure: Search for new susceptibility genes in patients without gene alterations of ABCB4
  time frame: Through study completion, an average of 5 years
  measure: Search for modulating genes in mutated and non-mutated ABCB4 patients.
  time frame: Through study completion, an average of 5 years
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: 13 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Christophe CORPECHOT
  role: Principal Investigator
  affiliation: INSERM UMR_S938
  facility: 
  facility: 
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  country: France
  responsible party type: Sponsor
  mesh term: Cholelithiasis
  mesh term: Cholecystolithiasis
  mesh term: Gallstones