Hereditary Autoinflammatory DiseasesMAI-PATIENTNCT03569644
Quality of Life in Patients With Auto-inflammatory Diseases
A study testing qualitative study in people with Hereditary Autoinflammatory Diseases, run by Assistance Publique - Hôpitaux de Paris at 3 sites in France.
Trial summary
This qualitative interview-based study aim to build a tool to assess quality of life in patients (adults or children) suffering from 6 autoinflammatory diseases (FMF, TRAPS, CAPS, MKD, Still and PFAPA). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Autoinflammatory Diseases
- What is being tested?
- qualitative study
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 3 sitesFrance
- Study dates
- 6 Jun 2018to 1 Apr 2021
- Phase
- Interventional
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Any patient (child and adult) with one of the 6 AIDs (FMF, TRAPS, MKD or CAPS, PFAPA or Still's disease) regardless of the activity of the disease. The diagnosis will have been made by the referring physician according to criteria specific to each AIDs
- Volunteer to participate in the study
- With at least one parent present at the consultation if it is a child under 18 years old
- For minors, agreement of the parents or their legal representative or one of the present parents
- Information for children as far as their age and condition allow
- Affiliation to a national health insurance.
Who cannot join
- patient or relative (according to age)refuse to participate.
- Bad understanding of French.
- Other chronic inflammatory pathology associated (example: Crohn's, SPA, uveitis, psoriasis ...)
Trial locations and contacts
3 sites in 1 country.
France3 sites
CH André Mignot
Le Chesnay, France, 78150
Dr Maryam PIRAM
Le Kremlin-Bicêtre, 94270
TENON
Paris, Paris, 75020
About this study
Official title: Creation of a Tool to Assess Quality of Life in Patient With Auto-inflammatory Diseases
This qualitative interview-based study aim to build a tool to assess quality of life in patients (adults or children) suffering from 6 autoinflammatory diseases (FMF, TRAPS, CAPS, MKD, Still and PFAPA).
Read the detailed description
Recent understanding of the physiopathological mechanisms that underpin autoinflammatory diseases (AIDs) allowed a revolution in the therapeutic management of these patients, particularly through the use of biotherapies. However new treatments need to be evaluated by valid tools. AIDAI score created and validated by our team evaluates activity of 4 autoinflammatory diseases. One of the important components, not explored by the scores currently available, is the impact of these treatments on quality of life (QoL) of patients. The existing quality of life scales are not adapted to these recurrent chronic pathologies. Throught a qualitative study based on interviews of patients and/ or parents, the investigators aim to build a tool to evaluate QoL in patients suffering from 6 aAIDs :familial Mediterranean fever (FMF), mevalonate kinase deficiency (MKD), tumour necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS), Still's disease and PFAPA syndrome.
- Conditions
- Hereditary Autoinflammatory Diseases
- Being tested
- qualitative study
- Medical subject headings
- Hereditary Autoinflammatory Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
qualitative studyOther
Interviews
| Study group | Role | What they receive |
|---|---|---|
| qualitative study | Other | heterogeneous group of patients Receives: Other: qualitative study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Qualitative evaluation of quality of life by interviews of patients and/or parents
The interviews will be recorded in digital audio and then transcribed literally. The analysis will be phenomenological. Four age group will be interviewed : < 6 year-old (parents), 6 to 12 year-old (patients and parents) , 13 to 17 year-old (patients and parents) , > 18 year-old (patients)
Measured: 45 to 90 min
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2018
- Study start6 Jun 2018
- Primary completion6 Nov 2019
- Study completion1 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Autoinflammatory DiseasesStudies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The study is based on interviews that will be recorded in digital audio and then transcribed literally. The consultation for this research is 45 to 90 minutes long. This prolongs their presence in consultation with the doctor. For this reason, the study was classified as Interventional according to French regulations
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03569644
- Sponsor's protocol ID
- K160903J
- Other IDs
- 2017-A02673-50
- Acronym
- MAI-PATIENT
- Status last verified
- Sep 2025
- First submitted
- 15 Jun 2018
- Last updated
- 2 Oct 2025
- Results posted
- No
- Data monitoring committee
- 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 2 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Creation of a Tool to Assess Quality of Life in Patient With Auto-inflammatory Diseases
- ClinicalTrials.gov ID
- NCT03569644
- Acronym / sponsor ID
- MAI-PATIENT / K160903J
- Conditions
- Hereditary Autoinflammatory Diseases
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 6 Jun 2018 (actual)
- Primary completion
- 6 Nov 2019 (actual)
- Study completion
- 1 Apr 2021 (actual)
- First posted
- 26 Jun 2018 (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.
This qualitative interview-based study aim to build a tool to assess quality of life in patients (adults or children) suffering from 6 autoinflammatory diseases (FMF, TRAPS, CAPS, MKD, Still and PFAPA).
Detailed description
Recent understanding of the physiopathological mechanisms that underpin autoinflammatory diseases (AIDs) allowed a revolution in the therapeutic management of these patients, particularly through the use of biotherapies. However new treatments need to be evaluated by valid tools. AIDAI score created and validated by our team evaluates activity of 4 autoinflammatory diseases. One of the important components, not explored by the scores currently available, is the impact of these treatments on quality of life (QoL) of patients. The existing quality of life scales are not adapted to these recurrent chronic pathologies. Throught a qualitative study based on interviews of patients and/ or parents, the investigators aim to build a tool to evaluate QoL in patients suffering from 6 aAIDs :familial Mediterranean fever (FMF), mevalonate kinase deficiency (MKD), tumour necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS), Still's disease and PFAPA syndrome.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The study is based on interviews that will be recorded in digital audio and then transcribed literally. The consultation for this research is 45 to 90 minutes long. This prolongs their presence in consultation with the doctor. For this reason, the study was classified as Interventional according to French regulations
| Arm | Type | Receives |
|---|---|---|
| qualitative study | Other | Other: qualitative studyheterogeneous group of patients |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Any patient (child and adult) with one of the 6 AIDs (FMF, TRAPS, MKD or CAPS, PFAPA or Still's disease) regardless of the activity of the disease. The diagnosis will have been made by the referring physician according to criteria specific to each AIDs
- Volunteer to participate in the study
- With at least one parent present at the consultation if it is a child under 18 years old
- For minors, agreement of the parents or their legal representative or one of the present parents
- Information for children as far as their age and condition allow
Key exclusion criteria (3 listed)
- patient or relative (according to age)refuse to participate.
- Bad understanding of French.
- Other chronic inflammatory pathology associated (example: Crohn's, SPA, uveitis, psoriasis ...)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| qualitative studyInterviews | Other | qualitative study |
Primary outcome
Qualitative evaluation of quality of life by interviews of patients and/or parents
Time frame: 45 to 90 min
The interviews will be recorded in digital audio and then transcribed literally. The analysis will be phenomenological. Four age group will be interviewed : < 6 year-old (parents), 6 to 12 year-old (patients and parents) , 13 to 17 year-old (patients and parents) , > 18 year-old (patients)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2025.
- Individual participant dataSharing planned: Undecided
Related trials for Hereditary Autoinflammatory Diseases
RecruitingObservational study
Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
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Not yet recruitingPhase 3
Goflikicept for Resolution and Prevention of Attacks in Pediatric and Adolescent Patients With Colchicine-Resistant or Colchicine-Intolerant Familial Mediterranean Fever
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Genotype and Phenotype in Pediatric Familial Mediterranean Fever
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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.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing qualitative study in people with Hereditary Autoinflammatory Diseases, run by Assistance Publique - Hôpitaux de Paris at 3 sites 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 6 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: finished, as of the sponsor's last update on 2 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Qualitative evaluation of quality of life by interviews of patients and/or parents (time frame: 45 to 90 min). 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 2 Oct 2025.