MastocytosisMCD&BuDiNCT04615663
Assessment of Burden Disease in Patients With Mast Cell Disorders
An observational study of Mastocytosis and Cutaneous, run by University Hospital, Toulouse at 1 site in France.
Trial summary
The symptoms caused by mast cell disorders can have a significant impact on the state of health of individuals, constituting a real burden for them, and consequently altering their quality of life. Read more about this study
- Who is it for?
- Ages 18 to 65 months
- Conditions the study is for
- Mastocytosis, Cutaneous
- What is being tested?
- Score SMI
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 200 participants
- Where
- 1 siteFrance
- Study dates
- 28 Jan 2020to 15 Sep 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 18 to 65 months
- All sexes
- No healthy volunteers
- Child
Study population: Prospective, descriptive study, both transversal and longitudinal, open, monocentric, performed in 200 patients with mast cell disease and follow-up at the CEREMAST Toulouse
Who may be able to join
- Adult patient (> 18 years old) with confirmed mast cell disorder according to international criteria
- Patient affiliated to social security regime
Who cannot join
- Patient under legal protection (guardianship, curators or court order)
- Patient does not speak French
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Cristina BULAI LIVIDEANU
Toulouse
Cristina BULAI LIVIDEANU, MD, 567778135 ext. +33, livideanu.c@chut-oulouse.fr
Isabelle OLIVIER, PhD, 0561777051 ext. +33, olivier.i@chut-oulouse.fr
About this study
Official title: Assessment of Personal, Psychosocial, Work, and Economic Burden in Patients With Mast Cell Disorders,
The symptoms caused by mast cell disorders can have a significant impact on the state of health of individuals, constituting a real burden for them, and consequently altering their quality of life. It therefore seems important to clarify the impact on the quality of life, on the psycho-affective sphere, on professional life and on the direct and indirect costs caused by the disease, as well as on the "patient's remaining burden". It seems possible by a longitudinal study (patient follow-up over 1 year).
Primary objective is Assessment of quality of life in adult patient with mast cell diseases at M0.
Read the detailed description
Mast cell activation symptoms are due to the release of mast cell mediators after uncontrolled activation of mast cells. The release by mast cells of mast cell mediators (tryptase, histamine, prostaglandins, serotonin) results in mast cell activation symptoms, found in mast cell activation syndrome but also in mastocytosis. These symptoms interested the skin, gastrointestinal gut, pulmonary, cardiovascular and neuropsychiatric organs, etc.
In mastocytosis, along with these symptoms of mast cell activation, the infiltration of different organs by abnormal mast cells produces many clinical signs related to the excess of monoclonal mast cells present in the organs.
According to our clinical experience, the impact of mast cell disorder on various dimensions of life, including economic life, seems important, but it has never accurately evaluated.
- Conditions
- Mastocytosis, Cutaneous
- Being tested
- Score SMI
- Keywords
- Burden, quality of life, mast cell disease, work impairment
- Medical subject headings
- Mastocytosis, Mast Cell Activation Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Score SMIBehavioural
the investigator will complete the SMI score
| Study group | Role | What they receive |
|---|---|---|
| face-to-face patients | During this visit, the investigator will complete the SMI score. Receives: Behavioral: Score SMI | |
| Email patients | this visit at M0 + 7d will correspond to the emailing of the Mc_QoL and Burden_MCD questionnaires completed by the patient. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life of mast cell disorder patients
Quality of life is evaluated with the WHOQOL-bref questionary. This validated questionnaire in French assesses six dimensions of quality of life.
Measured: Baseline
Secondary outcomes (2)
Mast cell disorder patient's life quality
Quality of life is evaluated with the WHOQOL-bref.
Measured: 6 months
Mast cell disorder patient's quality of life
Quality of life is evaluated with the WHOQOL-bref.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2020
- Study start28 Jan 2020
- Primary completion28 Jan 2021 (estimated)
- Study completion15 Sep 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MastocytosisStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe 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
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- LIVIDEANU Cristina, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04615663
- Sponsor's protocol ID
- RC31/19/0354
- Acronym
- MCD&BuDi
- Status last verified
- Nov 2020
- First submitted
- 16 Jun 2020
- Last updated
- 4 Nov 2020
- 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 4 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Personal, Psychosocial, Work, and Economic Burden in Patients With Mast Cell Disorders,
- ClinicalTrials.gov ID
- NCT04615663
- Acronym / sponsor ID
- MCD&BuDi / RC31/19/0354
- Conditions
- Mastocytosis, Cutaneous
- Keywords
- Burden, quality of life, mast cell disease, work impairment
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 28 Jan 2020 (actual)
- Primary completion
- 28 Jan 2021 (estimated)
- Study completion
- 15 Sep 2022 (estimated)
- First posted
- 4 Nov 2020 (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 symptoms caused by mast cell disorders can have a significant impact on the state of health of individuals, constituting a real burden for them, and consequently altering their quality of life. It therefore seems important to clarify the impact on the quality of life, on the psycho-affective sphere, on professional life and on the direct and indirect costs caused by the disease, as well as on the "patient's remaining burden". It seems possible by a longitudinal study (patient follow-up over 1 year).
Primary objective is Assessment of quality of life in adult patient with mast cell diseases at M0.
Detailed description
Mast cell activation symptoms are due to the release of mast cell mediators after uncontrolled activation of mast cells. The release by mast cells of mast cell mediators (tryptase, histamine, prostaglandins, serotonin) results in mast cell activation symptoms, found in mast cell activation syndrome but also in mastocytosis. These symptoms interested the skin, gastrointestinal gut, pulmonary, cardiovascular and neuropsychiatric organs, etc.
In mastocytosis, along with these symptoms of mast cell activation, the infiltration of different organs by abnormal mast cells produces many clinical signs related to the excess of monoclonal mast cells present in the organs.
According to our clinical experience, the impact of mast cell disorder on various dimensions of life, including economic life, seems important, but it has never accurately evaluated.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| face-to-face patients | – | Behavioral: Score SMIDuring this visit, the investigator will complete the SMI score. |
| Email patients | – | –this visit at M0 + 7d will correspond to the emailing of the Mc_QoL and Burden_MCD questionnaires completed by the patient. |
Who may qualify
- Ages
- 18 to 65 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Prospective, descriptive study, both transversal and longitudinal, open, monocentric, performed in 200 patients with mast cell disease and follow-up at the CEREMAST Toulouse
Key inclusion criteria (2 listed)
- Adult patient (> 18 years old) with confirmed mast cell disorder according to international criteria
- Patient affiliated to social security regime
Key exclusion criteria (2 listed)
- Patient under legal protection (guardianship, curators or court order)
- Patient does not speak French
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 |
|---|---|---|
| Score SMIthe investigator will complete the SMI score | Behavioural | face-to-face patients |
Primary outcome
Quality of life of mast cell disorder patients
Time frame: Baseline
Quality of life is evaluated with the WHOQOL-bref questionary. This validated questionnaire in French assesses six dimensions of quality of life.
2 secondary outcomes
Mast cell disorder patient's life quality
Time frame: 6 months
Mast cell disorder patient's quality of life
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2020.
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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.
LIVIDEANU Cristina, MD principal investigator
University Hospital, Toulouse
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 Mastocytosis and Cutaneous, run by University Hospital, Toulouse 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 2 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: status not confirmed, as of the sponsor's last update on 4 Nov 2020. 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 LIVIDEANU Cristina, MD (0567778135 ext. +33, livideanu.c@chu-toulouse.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quality of life of mast cell disorder patients (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 4 Nov 2020.