Clinical trial

Study Of Colchicine Resistance In Familial Mediterranean Fever

Name

APHP211442

Description

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.

Trial arms

Trial start

2022-12-09

Estimated PCD

2023-07-04

Trial end

2023-10-27

Status

Completed

Treatment

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

* Adherence to colchicine therapy will be assessed by the Girerd questionnaire (6 questions ) completed by the patient at the inclusion visit. * A blood sample (8ml) is taken, including a haemogram, CRP and creatinine and proteinuria. * Standard on-site stool analysis for C. difficile toxin. For research purposes, samples of: * Stool. If the patient is unable to produce stools on the day of the visit, a collection kit with 2 tubes containing a preservative will be given to him/her as well as a stamped envelope to take the sample at home and send it by post within 10 days according to a validated procedure. * A strand of hair with the diameter of a pencil and a minimum length of 2 cm, in the region of the posterior vertex. * In patients with baldness or bleached hair only: pubic and/or axillary hair, a pencil-sized strand. * Additional blood (5ml) and urine (2ml) samples for colchicine determination.

Arms:

Colchicine-resistant Familial Mediterranean Fever patients

Size

Primary endpoint

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

Through study completion, an average of 18 months

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['NA'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'DIAGNOSTIC', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 30, 'type': 'ACTUAL'}}

Updated at

2024-04-08

1 organization

1 product

2 indications

Organization

Assistance Publique - Hôpitaux de Paris

Product

Capillary colchicine

Indication

Familial Mediterranean Fever

Indication

Colchicine Resistance