Autoinflammatory DiseaseINFLAMASTNCT05292768
Are Mast Cells Involved in Autoinflammatory Diseases
An observational study of Autoinflammatory Disease and FMF, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoinflammatory Disease, FMF, TRAPS
- How many people
- 590 participants
- Where
- 1 siteFrance
- Study dates
- Mar 2022to Oct 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Study population: Inpatients and outpatients wil be recruited in tertiary university centres of the Assitance Publique des Hopitaux de Paris. Healthy controls will be recruited from volunteer healthcare workers in Assitance Publique des Hopitaux de Paris
Who may be able to join
- 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
Who cannot join
- Subjects unable to answer questions or express themselves
- Subjects who do not speak French
- Subject deprived of liberty or under legal protection.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Autoinflammatory Disease, FMF, TRAPS, MKD, Cryopyrin Associated Periodic Syndrome, Haploinsufficiency
- Keywords
- Autoinflammatory disease, Mast cells, Mast cell activation syndrome
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes, Mast Cell Activation Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with AID | Not described | |
| Control patients with mastocytosis | Not described | |
| Control patients with normal digestive biopsy | Not described | |
| Control patients with renal biopsy | Not described | |
| Control patients with inflammatory disease | Not described | |
| Healthy control from healthcare workers | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
: presence of MCAS
presence of clinical and biological markers of MCAS
Measured: at inclusion
Secondary outcomes (4)
comparison of clinical symptoms of MC activation between groups
we will compare clinical symptoms of MC activation between AID patients and other groups
Measured: at inclusion
MC mediators associated to AID
determine which MC mediators are elevated in AID and if they correlate with inflammation
Measured: at inclusion
MC infiltration in biopsies from AID patients
we will study MC infiltration in digestive, renale and cutaneous biopsies from patients with AID and compare them with biopsies from the other groups from subgroups of patients who had a biopsy performed.
Measured: at inclusion, retrospectively
basophilic polynuclear activation in AID patients
we will study basophilic polynuclear activation from blood samples of AID patients
Measured: at inclusion, retrospectively
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2022
- Study startMar 2022 (estimated)
- Primary completionOct 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoinflammatory DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 590 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Institut Imagine
- Responsible party
- Sponsor
- Investigators
- Sophie GEORGIN-LAVIALLE, PU-PH, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05292768
- Sponsor's protocol ID
- APHP210126
- Acronym
- INFLAMAST
- Status last verified
- Mar 2022
- First submitted
- 14 Mar 2022
- Last updated
- 23 Mar 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Are Mast Cells Involved in Autoinflammatory Diseases
- ClinicalTrials.gov ID
- NCT05292768
- Acronym / sponsor ID
- INFLAMAST / APHP210126
- Conditions
- Autoinflammatory Disease, FMF, TRAPS, MKD, Cryopyrin Associated Periodic Syndrome, Haploinsufficiency
- Keywords
- Autoinflammatory disease, Mast cells, Mast cell activation syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 590 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Institut Imagine
- Study start
- Mar 2022 (estimated)
- Primary completion
- Oct 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 23 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Patients with AID | – | – |
| Control patients with mastocytosis | – | – |
| Control patients with normal digestive biopsy | – | – |
| Control patients with renal biopsy | – | – |
| Control patients with inflammatory disease | – | – |
| Healthy control from healthcare workers | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Inpatients and outpatients wil be recruited in tertiary university centres of the Assitance Publique des Hopitaux de Paris. Healthy controls will be recruited from volunteer healthcare workers in Assitance Publique des Hopitaux de Paris
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- Subjects unable to answer questions or express themselves
- Subjects who do not speak French
- Subject deprived of liberty or under legal protection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
: presence of MCAS
Time frame: at inclusion
presence of clinical and biological markers of MCAS
4 secondary outcomes
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2022.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sophie GEORGIN-LAVIALLE, PU-PH principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Autoinflammatory Disease and FMF, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sophie GEORGIN-LAVIALLE, PU-PH (+33 (0)1 56 01 72 04 or 60 77, sophie.georgin-lavialle@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
: presence of MCAS (time frame: at inclusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2022.