Cryopyrin Associated Periodic SyndromeCAPSNCT04856540
Adult Outcomes of Children With CAPS
An observational study of Cryopyrin Associated Periodic Syndrome, run by University Hospital, Lille at 1 site in France.
Trial summary
The patients included in this study were followed up in the Internal Medicine and Paediatrics Departments of the Lille CHU, the Paediatric Rheumatology and Immunology Department of the Necker Enfant Malade Hospital in Paris and the Paediatric Rheumatology Department and Internal Medicine of the Bicêtre Hospital in… Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Cryopyrin Associated Periodic Syndrome
- How many people
- 54 participants
- Where
- 1 siteFrance
- Study dates
- 16 Sep 2021to 25 Feb 2022
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients follow-up in a CEREMAIA reference or competence center.
Who may be able to join
- Diagnosis of FCAS, Muckle-Wells or NOMID/CINCA;
- age < 16 years at the apparition of the first symptoms of the disease;
- age ≥ 16 years old at inclusion
- follow-up in a CEREMAIA reference or competence center.
Who cannot join
- Age < 16 years at inclusion;
- non-consenting patient
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Characteristic and Quality of Life Assessment in Adulthood of Patients With Cryopyrin Associated Periodic Syndrome
The patients included in this study were followed up in the Internal Medicine and Paediatrics Departments of the Lille CHU, the Paediatric Rheumatology and Immunology Department of the Necker Enfant Malade Hospital in Paris and the Paediatric Rheumatology Department and Internal Medicine of the Bicêtre Hospital in Paris. All patients selected presented one of the 3 CAPS clinical phenotypes (CINCA/NOMID, Muckle-Wells or Cold Urticaria). The mutation and the determination of the variant had to be confirmed by genetic analysis.
Patient data were collected from their medical records, retrospectively. Data collected concern childhood period from appearance of symptoms, adulthood period, in the last year and patients' way of life and quality of life upon the assessment. In addition, we collected demographic data related to the patients' lifestyle (intoxications, living arrangements, level of education) and we conducted individual telephone interviews lasting 15 minutes to complete a quality of life questionnaire including the SF36 questionnaire.
The study aimed to describe the clinical symptoms of patients in adulthood and to assess quality of life. We also wanted to compare the clinical phenotypes of patients according to their genetic variant.
- Conditions
- Cryopyrin Associated Periodic Syndrome
- Keywords
- CAPS Cryopyrin associated periodic syndromes, MWS Muckle-Wells syndrome, CINCA Chronic infantile neurological cutaneous and articular syndrome ;, FCAS Familial cold auto-inflammatory syndrome, Phenotype ; Quality of life, Quality of life
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical characteristics of patients with CAPS in adulthood
Measured: Baseline
Secondary outcomes (3)
Assessment of the quality of life of patients with CAPS in adulthood
Measured: Baseline
Compare the clinical status between childhood and adulthood
Measured: Baseline
Compare the clinical status of patients according to their genetic variant.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2021
- Study start16 Sep 2021
- Primary completion25 Feb 2022
- Study completion25 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cryopyrin Associated Periodic SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital, LilleThe 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
- Case-control
- Time perspective
- Retrospective
- Enrolment
- 54 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Lille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Eric Hachulla, MD,PhD, Principal investigator, University Hospital, Lille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04856540
- Sponsor's protocol ID
- 2021/0105
- Other IDs
- 2021-A00585-36
- Acronym
- CAPS
- Status last verified
- Jan 2023
- First submitted
- 13 Apr 2021
- Last updated
- 18 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Characteristic and Quality of Life Assessment in Adulthood of Patients With Cryopyrin Associated Periodic Syndrome
- ClinicalTrials.gov ID
- NCT04856540
- Acronym / sponsor ID
- CAPS / 2021/0105
- Conditions
- Cryopyrin Associated Periodic Syndrome
- Keywords
- CAPS Cryopyrin associated periodic syndromes, MWS Muckle-Wells syndrome, CINCA Chronic infantile neurological cutaneous and articular syndrome ;, FCAS Familial cold auto-inflammatory syndrome, Phenotype ; Quality of life, Quality of life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 54 participants (actual)
- Lead sponsor
- University Hospital, LilleUniversity, hospital or other
- Study start
- 16 Sep 2021 (actual)
- Primary completion
- 25 Feb 2022 (actual)
- Study completion
- 25 Feb 2022 (actual)
- First posted
- 23 Apr 2021 (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.
The patients included in this study were followed up in the Internal Medicine and Paediatrics Departments of the Lille CHU, the Paediatric Rheumatology and Immunology Department of the Necker Enfant Malade Hospital in Paris and the Paediatric Rheumatology Department and Internal Medicine of the Bicêtre Hospital in Paris. All patients selected presented one of the 3 CAPS clinical phenotypes (CINCA/NOMID, Muckle-Wells or Cold Urticaria). The mutation and the determination of the variant had to be confirmed by genetic analysis.
Patient data were collected from their medical records, retrospectively. Data collected concern childhood period from appearance of symptoms, adulthood period, in the last year and patients' way of life and quality of life upon the assessment. In addition, we collected demographic data related to the patients' lifestyle (intoxications, living arrangements, level of education) and we conducted individual telephone interviews lasting 15 minutes to complete a quality of life questionnaire including the SF36 questionnaire.
The study aimed to describe the clinical symptoms of patients in adulthood and to assess quality of life. We also wanted to compare the clinical phenotypes of patients according to their genetic variant.
Study design
- Observational model
- Case-control
- Time perspective
- Retrospective
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients follow-up in a CEREMAIA reference or competence center.
Key inclusion criteria (4 listed)
- Diagnosis of FCAS, Muckle-Wells or NOMID/CINCA;
- age < 16 years at the apparition of the first symptoms of the disease;
- age ≥ 16 years old at inclusion
- follow-up in a CEREMAIA reference or competence center.
Key exclusion criteria (2 listed)
- Age < 16 years at inclusion;
- non-consenting patient
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
Clinical characteristics of patients with CAPS in adulthood
Time frame: Baseline
3 secondary outcomes
Assessment of the quality of life of patients with CAPS in adulthood
Time frame: Baseline
Compare the clinical status between childhood and adulthood
Time frame: Baseline
Compare the clinical status of patients according to their genetic variant.
Time frame: Baseline
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 18 Jan 2023.
Related trials for Cryopyrin Associated Periodic Syndrome
RecruitingObservational study
Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases (NOMID/CAPS, DIRA, CANDLE, SAVI, NLRC4-MAS, Still'S-like Diseases, and Other Undifferentiated Autoinflammatory Diseases)
NOMID, DIRA
RecruitingPhase 1
Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load
Cryopyrin-Associated Periodic Syndromes, Amyloidosis
RecruitingObservational study
Ilaris NIS in Korea
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Recruiting
Circadian Rhythm Deregulation in Patients With CAPS
Cryopyrin Associated Periodic Syndrome, Familial Cold Urticaria
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.
Eric Hachulla, MD,PhD principal investigator
University Hospital, Lille
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 Cryopyrin Associated Periodic Syndrome, run by University Hospital, Lille 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 2 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 18 Jan 2023. 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?
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?
Clinical characteristics of patients with CAPS in adulthood (time frame: Baseline). 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 18 Jan 2023.