Inflammatory MyositisMASCNCT04637672
Clinical Database and Biobank of Patients With Inflammatory Myopathies: the MASC Project (Myositis, DNA, Serum, Cells) (MASC)
An observational study of Inflammatory Myositis and Idiopathic Inflammatory Myositis, run by Groupe Hospitalier Pitie-Salpetriere at 1 site in France.
Trial summary
Myositis are rare diseases for which the development of a cohort associated with a bank of biological samples (biobank) will allow for the conduct of researches to better delineate the underlying pathophysiology and find cures. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Inflammatory Myositis, Idiopathic Inflammatory Myositis, Drug-induced Inflammatory Myositis
- How many people
- 1,273 participants
- Where
- 1 siteFrance
- Study dates
- 19 Dec 2013to 14 Dec 2023
- How long will I take part?
- 100 Years
- 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
- No healthy volunteers
- Adult, Older adult
Study population: All patients who had a confirmed or suspected myositis
Who may be able to join
- All patients who had a confirmed (muscular biopsy, electromyogram, magnetic resonance imaging) or suspected clinically myositis. Myositis criteria are as follow:
- Dermatomyositis or polymyositis according to Bohan and Peter criteria (1975)
- Body inclusion myositis according to Griggs et al. criteria (1995)
- Necrotizing autoimmune myopathy according to Hoogendijk et al. criteria (2004)
- Drug-induced myositis
- Signature of the informed consent form for the study and for the biobank
- Age over 18 years old
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
France1 site
AP-HP, Pitié-Salpêtrière Hospital, Department of Internal Medicine and clinical immunology
Paris, 75013
About this study
Myositis are rare diseases for which the development of a cohort associated with a bank of biological samples (biobank) will allow for the conduct of researches to better delineate the underlying pathophysiology and find cures. This prospective cohort of patients with myositis will allow for identification of factors favouring the occurrence of myositis, whether they are constitutional (genetic) or acquired (environmental or drug). Different subgroups of myositis used for prognostication will be identified based on clinico-demographical variables, the nature of the organs involved beyond peripheral muscles (cardiac, diaphragm) and biomarkers abnormalities.
Read the detailed description
Myositis is a rare autoimmune disease in which the immune system mistakenly attacks the patient's own peripheral muscles. This aggression manifests by muscle inflammation and necrosis responsible for a motor deficit of varying severity.
The treatments available today are insufficient and are non-specific. Biological criteria, issued from simple blood or muscle tests are missing, and they will help to define the activity of the disease and the efficacy of treatments.
The MASC protocol will include patients with myositis, and investigators will collect clinical, radiological, electrophysiological, histological and biological data to be used for researches aiming at better understanding this entity. A biobank (muscle biopsy, DNA, serum, plasma, PBMCs) will be acquired on this prospective cohort.
The study itself will be composed of a baseline visit and monthly to yearly follow-up visits which will assess:
- Clinical examination with an evaluation of the muscle strength and function impairment/handicap, including but not limited to:
- Manual testing of proximal axial and distal muscles on the five points Medical Research Council (MRC) scale
- Barré tests and Mingazzini tests, number of stand-up / sitting, leg crossing
- Biometry, lab and radiological measurements: muscle enzymes (creatine phosphokinase CPK, troponin, C-reactive protein, quantification of autoantibodies, muscle MRI, muscle biopsy, thorax tomodensitometry, pulmonary test function
- Extra-muscular evaluation: cardiac examination and work-up (echocardiography, cardiac MRI and Positron Emission Tomography (PET) scanner, cardiac biopsies), pulmonary evaluation, rheumatological and dermatological assessment, history of thromboembolic disease and cancer
Patient activity assessment: evaluation of daily life activity by both patient and physician using a Visual Analogue Scale
- Quality of life questionnaires
- Evaluation of the efficacy and toxicity of specific treatments
For each patient, the date of last visit or contact will be collected as well as outcomes, particularly for the cause of death if relevant.
Data from the biobank MASC " Muscles DNA/RNA Serum and Cells " will be added to other data. The biobank has been fully registered with local authorities and ethical committees ("Committee for Personal Protection (CPP)" CPP agreement). It contains peripheral blood mononuclear cells (PBMC), serum, DNA and RNA from blood and muscular biopsies collected at the diagnosis stage. The database contains immunological and genetical data.
This prospective study will also aim at:
- Identify the differential pathophysiological processes between the different subgroups of myositis
- Identify prognostic factors, including the different treatment modalities used
- Improve physiopathological knowledge (clinico-anatomobiological characteristics and identification of other biomarkers through the biobank)
- Improve the evaluation of the clinical outcomes/endpoints for future trials
- Develop clinical trials for homogeneous subgroups of patients, based on their pathophysiology and evaluated on the appropriate endpoints.
- Conditions
- Inflammatory Myositis, Idiopathic Inflammatory Myositis, Drug-induced Inflammatory Myositis
- Medical subject headings
- Myositis
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations
Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations, including but not limited to: sexe, age, profession, a history of infection, cancer or other autoimmune and inflammatory diseases, diagnosis criteria, creatine phosphokinase, autoantibodies, immune systeme evaluation based on peripheral blood mononuclear cells, DNA sequencing muscular biopsies
Measured: baseline: first 30 days after inclusion
Secondary outcomes (14)
Characterisation of the natural history of myositis subgroups :responses to treatments, prognosis factors, evolution
Characterisation of the natural history of myositis subgroups :responses to treatments, prognosis factors, evolution
Measured: up to twenty years after inclusion
Characterisation of an immune system signature, using peripheral blood mononuclear cells and muscular biopsies, DNA and RNA sequencing, and autoantibodies
Characterisation of an immune system signature, using peripheral blood mononuclear cells and muscular biopsies, DNA and RNA sequencing, and autoantibodies
Measured: baseline: first 30 days after inclusion
Risk factors for All-cause mortality depending on patient's and disease characteristics
Risk factors for All-cause mortality depending on patient's and disease characteristics including clinical, radiological electrophysiological, histo-biological and immunological as well as treatment received stratified by each subgroup of myositis
Measured: up to twenty years after inclusion
Change of the quality of life, using quality of life questionnaires, depending of patients and disease characteristics
Change of the quality of life, using quality of life questionnaires, depending of patients and disease characteristics
Measured: up to twenty years after inclusion
Change of activity impairment using an evaluation of daily life activity by both patient and physician using a Visual Analogue Scale depending of patients and disease characteristics
Change of activity impairment using an evaluation of daily life activity by both patient and physician using a Visual Analogue Scale depending of patients and disease characteristics
Measured: up to twenty years after inclusion
Characterisation of a quality-of-life scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements
Characterisation of a quality-of-life scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements
Measured: up to twenty years after inclusion
Characterisation of a global activity scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements
Characterisation of a global activity scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements
Measured: up to twenty years after inclusion
Incidence of major cardio-vascular events
Incidence of major cardio-vascular events
Measured: up to twenty years after inclusion
Consequences on outcomes of major cardio-vascular events
Consequences on outcomes of major cardio-vascular events
Measured: up to twenty years after inclusion
Correlation of myositis with the development of extra-muscular diseases including but not limited to dermatological, rheumatological, cardiological and pneumological associated diseases
Correlation of myositis with the development of extra-muscular diseases including but not limited to dermatological, rheumatological, cardiological and pneumological associated diseases
Measured: up to twenty years after inclusion
Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry
Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry
Measured: up to twenty years after inclusion
Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry
Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry
Measured: up to twenty years after inclusion
Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry
Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry
Measured: up to twenty years after inclusion
Follow up of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry
Follow up of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry
Measured: up to twenty years after inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2020
- Study start19 Dec 2013
- Primary completion14 Dec 2023
- Study completion14 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Inflammatory MyositisStudies of the same condition, open and under way.
- 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
- 1,273 participants (actual)
- Target duration
- 100 Years
Sponsor and investigators
- Lead sponsor
- Groupe Hospitalier Pitie-Salpetriere
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Joe Elie Salem, Groupe Hospitalier Pitie-Salpetriere
- Investigators
- Olivier Benveniste, PU PH, Study director, Groupe Hospitalier Pitie-SalpetriereYves Allenbach, Study director, Groupe Hospitalier Pitie-SalpetriereJoe Elie SALEM, Study director, Groupe Hospitalier Pitie-Salpetriere
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04637672
- Sponsor's protocol ID
- CIC-1421-20-04
- Acronym
- MASC
- Status last verified
- Sep 2023
- First submitted
- 8 Nov 2020
- Last updated
- 22 May 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Teboul A, Allenbach Y, Tubach F, Belin L, Cassius C, Demortier J, Dossier A, Faucon C, Kasser C, Mekinian A, Monseau G, Fouchard M, Chambrelan E, Viguier M, Kluger N, Mahevas T, Bergeret B, Bachmeyer C, Lenormand C, Hotz C, Diaz E, Cordel N, Benveniste O, Bessis D, Bouaziz JD, Chasset F. Prognostic factors for patients with cancer-associated dermatomyositis: a retrospective, multicentre cohort study of 73 patients. Rheumatology (Oxford). 2025 May 1;64(5):2970-2978. doi: 10.1093/rheumatology/keae629.
PubMed 39589730Demortier J, Vautier M, Chosidow O, Gallay L, Bessis D, Berezne A, Cordel N, Schmidt J, Smail A, Duffau P, Jachiet M, Begon E, Gottlieb J, Chasset F, Graveleau J, Marque M, Cesbron E, Forestier A, Josse S, Kluger N, Beauchene C, Le Corre Y, Pagis V, Rigolet A, Guillaume-Jugnot P, Authier FJ, Guilain N, Streichenberger N, Leonard-Louis S, Boussouar S, Landon-Cardinal O, Benveniste O, Allenbach Y. Anti-SAE autoantibody in dermatomyositis: original comparative study and review of the literature. Rheumatology (Oxford). 2023 Dec 1;62(12):3932-3939. doi: 10.1093/rheumatology/kead154.
PubMed 37010495
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Database and Biobank of Patients With Inflammatory Myopathies: the MASC Project (Myositis, DNA, Serum, Cells) (MASC)
- ClinicalTrials.gov ID
- NCT04637672
- Acronym / sponsor ID
- MASC / CIC-1421-20-04
- Conditions
- Inflammatory Myositis, Idiopathic Inflammatory Myositis, Drug-induced Inflammatory Myositis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,273 participants (actual)
- Lead sponsor
- Groupe Hospitalier Pitie-SalpetriereUniversity, hospital or other
- Study start
- 19 Dec 2013 (actual)
- Primary completion
- 14 Dec 2023 (actual)
- Study completion
- 14 Dec 2023 (actual)
- First posted
- 20 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.
Myositis are rare diseases for which the development of a cohort associated with a bank of biological samples (biobank) will allow for the conduct of researches to better delineate the underlying pathophysiology and find cures. This prospective cohort of patients with myositis will allow for identification of factors favouring the occurrence of myositis, whether they are constitutional (genetic) or acquired (environmental or drug). Different subgroups of myositis used for prognostication will be identified based on clinico-demographical variables, the nature of the organs involved beyond peripheral muscles (cardiac, diaphragm) and biomarkers abnormalities.
Detailed description
Myositis is a rare autoimmune disease in which the immune system mistakenly attacks the patient's own peripheral muscles. This aggression manifests by muscle inflammation and necrosis responsible for a motor deficit of varying severity.
The treatments available today are insufficient and are non-specific. Biological criteria, issued from simple blood or muscle tests are missing, and they will help to define the activity of the disease and the efficacy of treatments.
The MASC protocol will include patients with myositis, and investigators will collect clinical, radiological, electrophysiological, histological and biological data to be used for researches aiming at better understanding this entity. A biobank (muscle biopsy, DNA, serum, plasma, PBMCs) will be acquired on this prospective cohort.
The study itself will be composed of a baseline visit and monthly to yearly follow-up visits which will assess:
- Clinical examination with an evaluation of the muscle strength and function impairment/handicap, including but not limited to:
- Manual testing of proximal axial and distal muscles on the five points Medical Research Council (MRC) scale
- Barré tests and Mingazzini tests, number of stand-up / sitting, leg crossing
- Biometry, lab and radiological measurements: muscle enzymes (creatine phosphokinase CPK, troponin, C-reactive protein, quantification of autoantibodies, muscle MRI, muscle biopsy, thorax tomodensitometry, pulmonary test function
- Extra-muscular evaluation: cardiac examination and work-up (echocardiography, cardiac MRI and Positron Emission Tomography (PET) scanner, cardiac biopsies), pulmonary evaluation, rheumatological and dermatological assessment, history of thromboembolic disease and cancer
Patient activity assessment: evaluation of daily life activity by both patient and physician using a Visual Analogue Scale
- Quality of life questionnaires
- Evaluation of the efficacy and toxicity of specific treatments
For each patient, the date of last visit or contact will be collected as well as outcomes, particularly for the cause of death if relevant.
Data from the biobank MASC " Muscles DNA/RNA Serum and Cells " will be added to other data. The biobank has been fully registered with local authorities and ethical committees ("Committee for Personal Protection (CPP)" CPP agreement). It contains peripheral blood mononuclear cells (PBMC), serum, DNA and RNA from blood and muscular biopsies collected at the diagnosis stage. The database contains immunological and genetical data.
This prospective study will also aim at:
- Identify the differential pathophysiological processes between the different subgroups of myositis
- Identify prognostic factors, including the different treatment modalities used
- Improve physiopathological knowledge (clinico-anatomobiological characteristics and identification of other biomarkers through the biobank)
- Improve the evaluation of the clinical outcomes/endpoints for future trials
- Develop clinical trials for homogeneous subgroups of patients, based on their pathophysiology and evaluated on the appropriate endpoints.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 100 Years
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All patients who had a confirmed or suspected myositis
Key inclusion criteria (7 listed)
- All patients who had a confirmed (muscular biopsy, electromyogram, magnetic resonance imaging) or suspected clinically myositis. Myositis criteria are as follow:
- Signature of the informed consent form for the study and for the biobank
- Age over 18 years old
Key exclusion criteria (1 listed)
- None
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
Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations
Time frame: baseline: first 30 days after inclusion
Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations, including but not limited to: sexe, age, profession, a history of infection, cancer or other autoimmune and inflammatory diseases, diagnosis criteria, creatine phosphokinase, autoantibodies, immune systeme evaluation based on peripheral blood mononuclear cells, DNA sequencing muscular biopsies
14 secondary outcomes
Characterisation of the natural history of myositis subgroups :responses to treatments, prognosis factors, evolution
Time frame: up to twenty years after inclusion
Characterisation of an immune system signature, using peripheral blood mononuclear cells and muscular biopsies, DNA and RNA sequencing, and autoantibodies
Time frame: baseline: first 30 days after inclusion
Risk factors for All-cause mortality depending on patient's and disease characteristics
Time frame: up to twenty years after inclusion
Change of the quality of life, using quality of life questionnaires, depending of patients and disease characteristics
Time frame: up to twenty years after inclusion
Change of activity impairment using an evaluation of daily life activity by both patient and physician using a Visual Analogue Scale depending of patients and disease characteristics
Time frame: up to twenty years after inclusion
Characterisation of a quality-of-life scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements
Time frame: up to twenty years after inclusion
Characterisation of a global activity scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements
Time frame: up to twenty years after inclusion
Incidence of major cardio-vascular events
Time frame: up to twenty years after inclusion
Consequences on outcomes of major cardio-vascular events
Time frame: up to twenty years after inclusion
Correlation of myositis with the development of extra-muscular diseases including but not limited to dermatological, rheumatological, cardiological and pneumological associated diseases
Time frame: up to twenty years after inclusion
Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry
Time frame: up to twenty years after inclusion
Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry
Time frame: up to twenty years after inclusion
Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry
Time frame: up to twenty years after inclusion
Follow up of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry
Time frame: up to twenty years after inclusion
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 22 May 2024.
- Derived from this study: PubMed 39589730Teboul A, Allenbach Y, Tubach F, Belin L, Cassius C, Demortier J, Dossier A, Faucon C, Kasser C, Mekinian A, Monseau G, Fouchard M, Chambrelan E, Viguier M, Kluger N, Mahevas T, Bergeret B, Bachmeyer C, Lenormand C, Hotz C, Diaz E, Cordel N, Benveniste O, Bessis D, Bouaziz JD, Chasset F. Prognostic factors for patients with cancer-associated dermatomyositis: a retrospective, multicentre cohort study of 73 patients. Rheumatology (Oxford). 2025 May 1;64(5):2970-2978. doi: 10.1093/rheumatology/keae629.
- Derived from this study: PubMed 37010495Demortier J, Vautier M, Chosidow O, Gallay L, Bessis D, Berezne A, Cordel N, Schmidt J, Smail A, Duffau P, Jachiet M, Begon E, Gottlieb J, Chasset F, Graveleau J, Marque M, Cesbron E, Forestier A, Josse S, Kluger N, Beauchene C, Le Corre Y, Pagis V, Rigolet A, Guillaume-Jugnot P, Authier FJ, Guilain N, Streichenberger N, Leonard-Louis S, Boussouar S, Landon-Cardinal O, Benveniste O, Allenbach Y. Anti-SAE autoantibody in dermatomyositis: original comparative study and review of the literature. Rheumatology (Oxford). 2023 Dec 1;62(12):3932-3939. doi: 10.1093/rheumatology/kead154.
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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.
Olivier Benveniste, PU PH study director
Groupe Hospitalier Pitie-Salpetriere
Yves Allenbach study director
Groupe Hospitalier Pitie-Salpetriere
Joe Elie SALEM study director
Groupe Hospitalier Pitie-Salpetriere
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 Inflammatory Myositis and Idiopathic Inflammatory Myositis, run by Groupe Hospitalier Pitie-Salpetriere 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 7 inclusion and 1 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 22 May 2024. 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?
Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations (time frame: baseline: first 30 days after inclusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04637672, which also lists 2 linked publications.
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 22 May 2024.