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

Inflammatory Myositis Clinical Trial

— MASC2  

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05454527
• Other study ID #: APHP220491
• Status: Recruiting
• Start date: October 26, 2022
• Est. completion date: October 26, 2057

Study information

• Verified date: October 2023
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Joe-Elie SALEM, MD PhD
• Phone: 142178531
• Email: joe-elie.salem[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 4000
• Est. completion date: October 26, 2057
• Est. primary completion date: October 26, 2057
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• Patients under AME

• Patients under legal protection

Related Conditions & MeSH terms

• Drug-induced Inflammatory Myositis
• Idiopathic Inflammatory Myositis
• Inflammatory Myositis
• Myositis

Locations

Country	Name	City	State
France	Département de médecine interne et immunolgie clinique, Hôpital Pitié Salpêtrière	Paris
France	Département de pharmacologie clinique, Hôpital Pitié Salpêtrière	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	baseline: first 30 days after inclusion
Secondary	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	up to twenty years after inclusion
Secondary	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	baseline: first 30 days after inclusion
Secondary	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	up to twenty years after inclusion
Secondary	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))	up to twenty years after inclusion
Secondary	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)	up to twenty years after inclusion
Secondary	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	up to twenty years after inclusion
Secondary	Characterisation of a global activity scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements	Using MM8 testing (0-150)	up to twenty years after inclusion
Secondary	Incidence of major cardio-vascular events	Incidence of major cardio-vascular events	up to twenty years after inclusion
Secondary	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	up to twenty years after inclusion
Secondary	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	up to twenty years after inclusion
Secondary	Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry	Using spirometry (Vital capacity in the sitting position)	up to twenty years after inclusion
Secondary	Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Vital capacity in supine position)	up to twenty years after inclusion
Secondary	Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (FEV1/VC ratio)	up to twenty years after inclusion
Secondary	Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Total lung capacity by Plethysmographic)	up to twenty years after inclusion
Secondary	Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Inspiratory capacity)	up to twenty years after inclusion
Secondary	Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (maximum static inspiratory pressure as % of predicted value)	up to twenty years after inclusion
Secondary	Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)	up to twenty years after inclusion
Secondary	Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Vital capacity in the sitting position )	up to twenty years after inclusion
Secondary	Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry	Using Spirometry ( Vital capacity in supine position )	up to twenty years after inclusion
Secondary	Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry	Using Spirometry (FEV1/VC ratio)	up to twenty years after inclusion
Secondary	Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Total lung capacity by Plethysmographic)	up to twenty years after inclusion
Secondary	Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Inspiratory capacity)	up to twenty years after inclusion
Secondary	Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry	Using Spirometry (maximum static inspiratory pressure as % of predicted value)	up to twenty years after inclusion
Secondary	Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)	up to twenty years after inclusion
Secondary	Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Vital capacity in supine position)	up to twenty years after inclusion
Secondary	Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Vital capacity in the sitting position)	up to twenty years after inclusion
Secondary	Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (FEV1/VC ratio)	up to twenty years after inclusion
Secondary	Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Total lung capacity by Plethysmographic)	up to twenty years after inclusion
Secondary	Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Inspiratory capacity)	up to twenty years after inclusion
Secondary	Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (maximum static inspiratory pressure as % of predicted value)	up to twenty years after inclusion
Secondary	Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)	up to twenty years after inclusion
Secondary	Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Sniff nasal inspiratory pressure in % of predicted value)	up to twenty years after inclusion
Secondary	Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (maximum static inspiratory pressure as % of predicted value)	up to twenty years after inclusion
Secondary	Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Inspiratory capacity)	up to twenty years after inclusion
Secondary	Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Total lung capacity by Plethysmographic)	up to twenty years after inclusion
Secondary	Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (FEV1/VC ratio)	up to twenty years after inclusion
Secondary	Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Vital capacity in the sitting position)	up to twenty years after inclusion
Secondary	Follow up of diaphragmatic function with pulmonary function test and thoracic tomodensitometry	Using Spirometry (Vital capacity in supine position)	up to twenty years after inclusion