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Not yet recruiting NCT05292768

Are Mast Cells Involved in Autoinflammatory Diseases

Observational Autoinflammatory Disease FMF TRAPS MKD

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Autoinflammatory Disease, FMF, TRAPS, MKD. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
France
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Overview

Autoinflammatory diseases (AID) are caused by innate immunity dysregulation. AID pathophysiology is only partly understood, especially in the case of unclassified AID. Mast cells (MC) are innate immune cells associated with a spectrum of disease between systemic mastocytosis and mast cell activation syndrome. The implication of MC has been shown in cryopyrin associated periodic syndrome (CAPS).Our aim is to evaluate the involvement of MC in AID by assessing clinical and biological signs of MC activation and studying cutaneous and digestive biopsies.

Detailed description

Autoinflammatory diseases (AID) are caused by innate immunity dysregulation and characterized by recurrent bouts of fever, frequently associated with digestive, articular or cutaneous symptoms, and sometimes ocular, auricular or neurologic inflammation. The most frequent monogenic AID is Familial Mediterranean fever (FMF).

Despite recent genetic progress AID pathophysiology is only partly understood, especially in the case of unclassified AID.

Mast cells (MC) are innate immune cells associated with a spectrum of disease between systemic mastocytosis and mast cell activation syndrome (MCAS). In MCAS, patients have various symptoms including abdominal pain, bloating, pruritus, flush, anxiety, fatigue, among which some are similar to those seen in patients with AID. The implication of MC has been shown in cryopyrin associated periodic syndrome (CAPS).

Our hypothesis is that MC could be involved in AID pathophysiology,

In order to test this hypothesis, we plan to study :

* clinical MC activation symptoms via a standardized clinical score * biological MC mediators : by measuring total serum tryptase and histamine in total blood, plasma and urine * MC infiltration on gastro-intestinal (GI) tract and cutaneous biopsies We will compare clinical MC activation score in AID patients to patients with mastocytosis, with other inflammatory diseases, and with healthy controls.

Primary outcome measures

  • : presence of MCAS [Time frame: at inclusion]
Secondary outcome measures (4)
  • comparison of clinical symptoms of MC activation between groups [Time frame: at inclusion]
  • MC mediators associated to AID [Time frame: at inclusion]
  • MC infiltration in biopsies from AID patients [Time frame: at inclusion, retrospectively]
  • basophilic polynuclear activation in AID patients [Time frame: at inclusion, retrospectively]

Eligibility criteria

Inclusion criteria

  • Patients >18 years old with Auto-inflammatory diseases already followed up at the CeRéMAIA (french national reference center for autoinflamamtory diseases and AA amyloidosis) of Tenon hospital and included in the JIRcohorte
  • Healthy adult controls, age- and sex-matched with MAI patients, and controls with mastocytosis, an immuno-inflammatory disease.
  • Subject affiliated to or entitled to a social security scheme
  • Collection of the patient's or healthy control's non-opposition

Exclusion criteria

  • Subjects unable to answer questions or express themselves
  • Subjects who do not speak French
  • Subject deprived of liberty or under legal protection.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Study design

Observational model
Cohort

Study locations

France · 1 center
  • Service de Médecine Interne — Paris

Identifiers

NCT: NCT05292768 · APHP210126

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗