Study of Colchicine Resistance in Familial Mediterranean Fever

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Brief Title

Study of Colchicine Resistance in Familial Mediterranean Fever

Official Title

Study Of Colchicine Resistance In Familial Mediterranean Fever

Brief Summary

      Five to 10% of familial mediterranean patients are considered colchicine-resistant (i.e.
      patients with a persistent inflammatory syndrome, despite taking the maximum tolerated dose
      of colchicine daily). The recommended treatment in this case is a subcutaneous
      anti-interleukin 1 biotherapy (anakinra or canakinumab). These treatments are expensive
      (1,000 to 12,000 euros/month).

      However, for a patient to be considered colchicine-resistant, compliance with the treatment
      must be verified. Furthermore specific activation of the pyrin inflammasome by Clostrioides
      difficile toxin and the overrepresentation of these bacteria in the stools of our patients
      led us to systematically search for them in our resistant patients. The demonstration of the
      involvement of C. difficile in the imbalance of the disease has not yet been published.

      The colchiresist study aim to better characterize colchicine-resistance by confirming good
      compliance to treatment with colchicine hair measurement and by looking for clostrioides
      infection or intestinal dysbiosis.
    

Detailed Description

      Patients meeting the criteria for colchicine resistance will be included in a follow-up
      visit.

        -  Adherence to colchicine therapy will be assessed by the Girerd questionnaire completed
           by the patient at the inclusion visit.

        -  A blood test is performed including a haemogram, CRP and SAA, creatinine and
           proteinuria.

        -  a standard stool analysis on site to look for C. difficile toxin.

      For research purposes, samples of:

        -  Stool.

        -  A strand of hair with the diameter of a pencil and a minimum length of 2 cm, in the
           region of the posterior vertex, where the growth and integration are relatively constant
           to avoid variability.

        -  In patients with baldness or bleached hair only: pubic and/or axillary hair will be
           taken, The samples will then be processed without segmentation.

        -  Additional blood (5ml) and urine (2ml) samples for colchicine dosage. The patient's
           participation ends after this visit
    


Study Type

Interventional


Primary Outcome

Amount of colchicine measured in the first 2 cm of hair from the scalp of an FMF patient considered colchicine resistant or from pubic hair in patients with bald or colored hair, which corresponds to the average compliance of the last 2 months of intake

Secondary Outcome

 Correlation between the amount of colchicine measured in the hair and the blood and urine levels of colchicine attesting to the last days' intake.

Condition

Mediterranean Fever

Intervention

Capillary colchicine dosage in colchicine-resistant Familial Mediterranean Fever patients.

Study Arms / Comparison Groups

 Colchicine-resistant Familial Mediterranean Fever patients
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

30

Start Date

July 2022

Completion Date

January 2024

Primary Completion Date

January 2024

Eligibility Criteria

        Inclusion Criteria:

        Inclusion Criteria Age ≥ 12 years with no upper age limit -Patients with familial
        Mediterranean fever (FMF) meeting international criteria for the disease (Livneh criteria)
        and carrying 2 pathogenic mutations in the MEFV gene.

        Patients considered colchicine-resistant according to EULAR criteria reviewed by an expert
        consensus:

          -  Persistence of signs of activity (at least one attack per month for at least 3 months)
             despite taking the maximum tolerated dosage of colchicine.

          -  Persistence of a biological inflammatory syndrome after exclusion of other causes and
             despite taking a maximum tolerated dosage of colchicine.

          -  Signature of an informed consent by the patient (or his parents if under 18 years of
             age)

          -  Patients affiliated to a social security system

        Exclusion Criteria:

        - Patients participating in another interventional trial
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Léa SAVEY, MD, 0033156017431, [email protected]

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT05418686

Organization ID

APHP211442


Responsible Party

Sponsor

Study Sponsor

Assistance Publique - Hôpitaux de Paris


Study Sponsor

Léa SAVEY, MD, Principal Investigator, Assistance Publique - Hôpitaux de Paris


Verification Date

June 2022