The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

COLPAC (RaDiCo Cohort) (RaDiCo-COLPAC) (COLPAC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05961826
Recruitment Status : Active, not recruiting
First Posted : July 27, 2023
Last Update Posted : July 27, 2023
Sponsor:
Information provided by (Responsible Party):
Institut National de la Santé Et de la Recherche Médicale, France

Study Description
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:

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.


Condition or disease
Low Phospholipid Associated Cholelithiasis

Study Design
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Observational
Estimated Enrollment : 650 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: National Cohort About Epidemiology, Clinical and Genetic Heterogeneity of the "Low-Phospholipid-Associated Cholelithiasis" (LPAC) Syndrome
Actual Study Start Date : November 6, 2017
Estimated Primary Completion Date : June 30, 2028
Estimated Study Completion Date : June 30, 2028

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gallstones

Groups and Cohorts
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information


Outcome Measures
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. Description of patient management practices. [ Time Frame: Through study completion, an average of 5 years ]
  4. 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 ]

    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).


  5. Evaluation of response to medical and interventional treatments. [ Time Frame: Through study completion, an average of 5 years ]

    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

  6. 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 ]
  7. Assessing the impact of LPAC syndrome on quality of life. [ Time Frame: Through study completion, an average of 5 years ]
    Impact of disease on quality of life will be evaluated through scores of quality of life questionnaires (SF-36)

  8. 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 ]

    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.



Other Outcome Measures:
  1. 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 ]
  2. 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 ]

    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)

  3. Search for new susceptibility genes in patients without gene alterations of ABCB4 [ Time Frame: Through study completion, an average of 5 years ]
  4. Search for modulating genes in mutated and non-mutated ABCB4 patients. [ Time Frame: Through study completion, an average of 5 years ]

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This study concerns all prevalent and incident patients who meet the standard or extended diagnostic criteria for LPAC syndrome, whatever the existing genetic data concerning ABCB4, or who have been referred as having a mutation in the ABCB4 gene in a clinical context of biliary lithiasis.
Criteria

Inclusion Criteria:

  • Children or adults meeting the standard* or extended** diagnostic criteria for LPAC syndrome:

    1. First symptoms before the age of 40 years
    2. Radiological images compatible with the existence of intrahepatic lithiasis

    3. Recurrence of symptoms after cholecystectomy

      (*) standard criteria: symptomatic biliary lithiasis with at least 2 out of 3 criteria

      (**) extended criteria: symptomatic biliary lithiasis with 1 out of 3 criteria

      Exclusion Criteria:

  • Patients who have undergone liver transplantation
Contacts and Locations
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05961826


Locations
Show Show 21 study locations
Sponsors and Collaborators
Institut National de la Santé Et de la Recherche Médicale, France
Investigators
Layout table for investigator information
Principal Investigator: Christophe CORPECHOT INSERM UMR_S938
More Information
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
ClinicalTrials.gov Identifier: NCT05961826    
Other Study ID Numbers: C16-14
First Posted: July 27, 2023    Key Record Dates
Last Update Posted: July 27, 2023
Last Verified: July 2023

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Cholelithiasis
Cholecystolithiasis
Gallstones
Biliary Tract Diseases
 Digestive System Diseases
Gallbladder Diseases
Calculi
Pathological Conditions, Anatomical