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Inflammatory MyositisMASC2NCT05454527

Follow-up of Patients With Inflammatory Myopathies Associated With a Biobank

An observational study of Inflammatory Myositis and Idiopathic Inflammatory Myositis, run by Assistance Publique - Hôpitaux de Paris at 2 sites in France.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

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
How many people
4,000 participants
Where
2 sitesFrance
Study dates
26 Oct 2022to 26 Oct 2057
Phase
Observational
Are there results?
Not yet: the study is under way

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: Major patients with myositis included during consultation visits or routine hospitalizations in the Internal Medicine Department, Reference Center for Neuromuscular Pathology, at the Pitié Salpêtrière Hospital

Who may be able to join

  • Age ≥ 18 years
  • Suspicion of Myositis defined according to reference classifications: Dermatomyositis (DM), polymyositis (PM) defined as early as 1975 by Bohan and Peter, inclusion myositis (IM) according to the criteria of Griggs et al, 1995 and autoimmune necrotizing myopathy (ANM) by Hoogendijk et al, in 2004 and iatrogenic (e.g. drug-induced) myositis.
  • No opposition from patients to the use of their data
  • Signature of consents for the constitution of the biobank and the genetic analyses

Who cannot join

  • Patients under AME
  • Patients under legal protection

Trial locations and contacts

2 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Joe-Elie SALEM, MD PhD contact

142178531 ext. +33

joe-elie.salem@aphp.fr

Olivier Benveniste, PU PH contact

olivier.benveniste@aphp.fr

France2 sites, 2 recruiting

  • Département de médecine interne et immunolgie clinique, Hôpital Pitié Salpêtrière

    Paris, 75013

    Olivier BENVENISTE, MD, PhD, 01 42 16 10 88, olivier.benveniste@aphp.fr

    Recruiting

  • Département de pharmacologie clinique, Hôpital Pitié Salpêtrière

    Paris, 75013

    Joe-Elie SALEM, MD, PhD, 01 42 17 85 35, joe-elie.salem@aphp.fr

    Recruiting

Other trials in France

About this study

Official title: Follow-up of a Cohort of Patients With Inflammatory Myopathies Associated With a Biobank: MASC 2 Project (Myositis, Muscles, DNA/RNA Serum Cells)

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.
Medical subject headings
Myositis

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histobiological evaluations

    Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histobiological evaluations

    Measured: baseline: first 30 days after inclusion

Secondary outcomes (38)

  1. 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

  2. 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

  3. 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

  4. 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 (HAQ (Health Assessment Questionnaire)global health status scale (0-100))

    Measured: up to twenty years after inclusion

  5. 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

    Using analogue Scale depending of patients and disease characteristics:

    Physician's assessment of disease activity in the muscle area (VAS 0-10) Physician's evaluation of the disease activity in the skin area (VAS 0-10) Physician's evaluation of general signs of disease activity (VAS 0-10) Physician's assessment of disease activity in rheumatology (VAS 0-10) Physician's assessment of disease activity in the digestive area (VAS 0-10) Physician's assessment of disease activity in the pulmonary area (VAS 0-10) Physician's assessment of disease activity in the cardiac area (VAS 0-10) Doctor's assessment of disease activity in the extra-muscular area (VAS 0-10) Overall evaluation of the disease activity (muscular and extra-muscular) by the PHYSICIAN (VAS 0-10) Global evaluation of the disease activity by the PATIENT (VAS 0-10)

    Measured: up to twenty years after inclusion

  6. Characterisation of a quality-of-life scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements

    Using MM8 testing (0-150) visceral involvements

    Measured: up to twenty years after inclusion

  7. Characterisation of a global activity scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements

    Using MM8 testing (0-150)

    Measured: up to twenty years after inclusion

  8. Incidence of major cardio-vascular events

    Incidence of major cardio-vascular events

    Measured: up to twenty years after inclusion

  9. Consequences on outcomes of major cardio-vascular events

    Major cardiovascular will include:

    • Heart failure
    • Myocardial infarction
    • Stroke
    • Sudden cardiac death
    • High-degree atrio-ventricular blocks
    • High-degree sinus dysfunction
    • Sustained ventricular tachycardia

    Measured: up to twenty years after inclusion

  10. 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

  11. Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using spirometry (Vital capacity in the sitting position)

    Measured: up to twenty years after inclusion

  12. Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Vital capacity in supine position)

    Measured: up to twenty years after inclusion

  13. Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (FEV1/VC ratio)

    Measured: up to twenty years after inclusion

  14. Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Total lung capacity by Plethysmographic)

    Measured: up to twenty years after inclusion

  15. Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Inspiratory capacity)

    Measured: up to twenty years after inclusion

  16. Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (maximum static inspiratory pressure as % of predicted value)

    Measured: up to twenty years after inclusion

  17. Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)

    Measured: up to twenty years after inclusion

  18. Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Vital capacity in the sitting position )

    Measured: up to twenty years after inclusion

  19. Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry

    Using Spirometry ( Vital capacity in supine position )

    Measured: up to twenty years after inclusion

  20. Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (FEV1/VC ratio)

    Measured: up to twenty years after inclusion

  21. Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Total lung capacity by Plethysmographic)

    Measured: up to twenty years after inclusion

  22. Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Inspiratory capacity)

    Measured: up to twenty years after inclusion

  23. Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (maximum static inspiratory pressure as % of predicted value)

    Measured: up to twenty years after inclusion

  24. Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)

    Measured: up to twenty years after inclusion

  25. Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Vital capacity in supine position)

    Measured: up to twenty years after inclusion

  26. Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Vital capacity in the sitting position)

    Measured: up to twenty years after inclusion

  27. Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (FEV1/VC ratio)

    Measured: up to twenty years after inclusion

  28. Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Total lung capacity by Plethysmographic)

    Measured: up to twenty years after inclusion

  29. Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Inspiratory capacity)

    Measured: up to twenty years after inclusion

  30. Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (maximum static inspiratory pressure as % of predicted value)

    Measured: up to twenty years after inclusion

  31. Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)

    Measured: up to twenty years after inclusion

  32. Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)

    Measured: up to twenty years after inclusion

  33. Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (maximum static inspiratory pressure as % of predicted value)

    Measured: up to twenty years after inclusion

  34. Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Inspiratory capacity)

    Measured: up to twenty years after inclusion

  35. Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Total lung capacity by Plethysmographic)

    Measured: up to twenty years after inclusion

  36. Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (FEV1/VC ratio)

    Measured: up to twenty years after inclusion

  37. Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Vital capacity in the sitting position)

    Measured: up to twenty years after inclusion

  38. Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry

    Using Spirometry (Vital capacity in supine position)

    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.

  1. First posted12 Jul 2022
  2. Study start26 Oct 2022
  3. Primary completion26 Oct 2057 (estimated)
  4. Study completion26 Oct 2057 (estimated)

Resources

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
Cross-sectional
Enrolment
4,000 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05454527
Sponsor's protocol ID
APHP220491
Acronym
MASC2
Status last verified
Apr 2025
First submitted
17 Jun 2022
Last updated
23 Apr 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Power JR, Dolladille C, Ozbay B, Procureur A, Ederhy S, Palaskas NL, Lehmann LH, Cautela J, Courand PY, Hayek SS, Zhu H, Zaha VG, Cheng RK, Alexandre J, Roubille F, Baldassarre LA, Chen YC, Baik AH, Laufer-Perl M, Tamura Y, Asnani A, Francis S, Gaughan EM, Rainer PP, Bailly G, Flint D, Arangalage D, Cariou E, Florido R, Narezkina A, Liu Y, Sandhu S, Leong D, Issa N, Piriou N, Heinzerling L, Peretto G, Crusz SM, Akhter N, Levenson JE, Turker I, Eslami A, Fenioux C, Moliner P, Obeid M, Chan WT, Ewer SM, Kassaian SE, Johnson DB, Nohria A, Ben Zadok OI, Moslehi JJ, Salem JE; International ICI-Myocarditis Registry. Immune checkpoint inhibitor-associated myocarditis: a novel risk score. Eur Heart J. 2026 Mar 5;47(9):1050-1062. doi: 10.1093/eurheartj/ehaf315.

    PubMed 40569849

  • Power JR, Dolladille C, Ozbay B, Procureur AM, Ederhy S, Palaskas NL, Lehmann LH, Cautela J, Courand PY, Hayek SS, Zhu H, Zaha VG, Cheng RK, Alexandre J, Roubille F, Baldassarre LA, Chen YC, Baik AH, Laufer-Perl M, Tamura Y, Asnani A, Francis S, Gaughan EM, Rainer PP, Bailly G, Flint D, Arangalage D, Cariou E, Florido R, Narezkina A, Liu Y, Sandhu S, Leong D, Issa N, Piriou N, Heinzerling L, Peretto G, Crusz SM, Akhter N, Levenson JE, Turker I, Eslami A, Fenioux C, Moliner P, Obeid M, Chan WT, Ewer SM, Kassaian SE; International ICI-Myocarditis Registry; Johnson DB, Nohria A, Zadok OIB, Moslehi JJ, Salem JE. Predictors and Risk Score for Immune Checkpoint-Inhibitor-Associated Myocarditis Severity. medRxiv [Preprint]. 2024 Jun 3:2024.06.02.24308336. doi: 10.1101/2024.06.02.24308336.

    PubMed 38883792

Questions about this trial

What is this clinical trial studying?

An observational study of Inflammatory Myositis and Idiopathic Inflammatory Myositis, run by Assistance Publique - Hôpitaux de Paris at 2 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 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: looking for participants now, as of the sponsor's last update on 23 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Joe-Elie SALEM, MD PhD (142178531 ext. +33, joe-elie.salem@aphp.fr), or a site near the patient. 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 histobiological 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 NCT05454527, 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 23 Apr 2025.

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