Myositis (dermatomyositis [DM] or polymyositis [PM]) MedDRA version: 20.0 Level: PT Classification code 10012503 Term: Dermatomyositis System Organ Class: 10040785 - Skin and subcutaneous tissue disorders MedDRA version: 20.0 Level: PT Classification code 10028653 Term: Myositis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders MedDRA version: 20.0 Level: PT Classification code 10036102 Term: Polymyositis System Organ Class: 10028395 - Musculoskeletal and connecti
Conditions
Interventions
Trade Name: RoActemra 162 mg soluzione iniettabile in siringa preriempita
Product Name: RoActemra 162 mg soluzione iniettabile in siringa preriempita
Product Code: NA
Pharmaceutical Form: Solution for
Sponsors
Azienda Unità Sanitaria Locale - IRCCS di Reggio Emilia
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. age 18 years or older 2. capacity to provide a written informed consent 3. fulfil the Bohan and Peter criteria and 1) have the typical rash of DM (heliotrope rash and/or Gottron sign and/or Gottron papules) or 2) have a myositis-specific autoantibody (MSA) or else 3) have the diagnosis confirmed by muscle biopsy 4. have significant muscle weakness (grade 4 or less of the Medical Research Council scale in at least 2 proximal muscle groups) plus have either an elevated muscle enzyme (CK or aldolase) at least 1.3 the upper limit of normal and/or an MRI of the thigh muscles showing edema in fat-suppressed sequences 5. physician’s global assessment of at least 2 (0-10 cm VAS) 6. a patient’s global assessment of at least 2 (0-10 cm VAS) 7. Health assessment questionnaire (HAQ) disability index of at least 0.25 8. signed informed consent form Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 14 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1. subjects younger than 18 years 2. inability to provide informed consent 3. history of alcohol or recreational drug use within 1 year prior to screening 4. infections, including serious infections requiring hospitalization or treatment with intravenous antibiotics within 4 weeks of screening; 5. positive HbsAg status or positive anti-HCV antibodies; 6. latent untreated TB 7. current malignancies or malignancies diagnosed within the previous 5 years (except basal and squamous cell carcinoma of the skin or carcinoma in situ of the cervix uteri that has been removed) 8. severe co-morbidities, including immunodeficiency; any comorbidity that in the opinion of the PI may not allow the patients to safely complete the trial 9. initiation of an exercise program for muscle strengthening within 4 weeks of the screening visit and for the whole duration of the trial 10. major surgery 4 weeks prior to study entry or planned major surgery during the study period 11. immunization with a live vaccines within a month before study entry 12. pregnancy and lactation, patients with reproductive potential not willing to use an effective method of contraception 13. previous treatment with TCZ 14. history of severe hypersensitivity reactions to monoclonal antibodies 15. contraindications to having an MRI of the thigh muscles done 16. abnormal laboratory values, including creatinine above the upper limit of normal unless creatinine clearance is above 30 ml/min; white cell count <3,000 mm3, absolute neutrophil count less than 2,000 mm3, absolute lymphocyte count less than 500/mm3, platelet count less than 100,000 and hemoglobin less than 8.5 g/dl.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the efficacy of TCZ in patients with refractory myositis;Secondary Objective: Safety assessment;Primary end point(s): Percentage of patients achieving an improvement according to IMACS (International Myositis Assessment and Clinical Studies Group);Timepoint(s) of evaluation of this end point: Weeks 0, 4, 8, 12, 16, 24, 38 and 52 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. time from first treatment to improvement based on IMACS criteria, percentage of patients achieving remission (defined as normal or stable muscle strength with normal levels of muscle enzymes, absence of muscular edema at MRI and absence of active extramuscular manifestations), reduction glucocorticoid dose, significant minimal improvement in cutaneous dermatomyositis, changes in edema at MRI, changes in fatty atrophy at MRI 2. Frequency and type of adverse events;Timepoint(s) of evaluation of this end point: 1. Weeks 0, 4, 8, 12, 16, 24, 38 and 52 2. Week 0, 4, 8, 12, 16, 24, 38 and 52; any adverse event occurring at any time during the whole study | — |
Countries
Italy
Contacts
Public ContactStruttura Complessa di Reumatologia
Presidio Ospedaliero Arcispedale Santa Maria Nuova – AUSL-IRCCS di Reggio Emilia
Outcome results
None listed