Dermatomyositis Musculoskeletal Diseases Dermatomyositis
Conditions
Interventions
All subjects qualified to participate in the study at baseline will be block randomized to one of the two treatment arms by an electronic IRT tool. The randomization will apply a randomization ratio o
Sponsors
Octapharma Pharmazeutika Produktionsges.m.b.H.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Diagnosis of definite or probable DM according to the Bohan and Peter criteria 2. Responded to IGIV (intravenous immunoglobulin) treatment as assessed by the treating physician and on a stable dose for at least 3 months on 2 g/kg bodyweight (+/- 10%). 3. If on other medication(s) for the treatment of DM (immunosuppressants, corticosteroids): 3.1. Receiving these medication(s) at the start of IGIV treatment in the first place 3.2. Receiving these medication(s) for at least 3 months prior to study enrolment and at a stable dose for at least 4 weeks prior to study enrolment 4. MMT-8 score =144, with at least 3 of the 5 other core set measures to be normal or near normal as per the following criteria: 4.1. Visual Analogue Scale (VAS) of patient global disease activity =2 cm 4.2. Physician’s global disease activity =2 cm, 4.3. Extra-muscular disease activity =2 cm 4.4. No muscle enzyme >4x upper limit of normal due to myositis 4.5. Health Assessment Questionnaire (HAQ) =0.25. 5. Aged =18 to <80 years 6. Voluntarily given, fully informed written consent obtained from subject before any study-related procedures are conducted 7. Capable and willing to understand and comply with the relevant aspects of the study protocol
Exclusion criteria
Exclusion criteria: 1. Cancer-associated myositis, defined as the diagnosis of myositis within 2 years of the diagnosis of cancer (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured and at least 1 or 5 years, respectively, have passed since excision) 2. Evidence of active malignant disease or malignancies diagnosed within the previous 5 years (including hematological malignancies and solid tumors) or breast cancer diagnosed within the previous 10 years. Subjects >5 years (>10 years for breast cancer) of cancer diagnosis who have been treated and are in remission are allowed 3. Overlap myositis (except for overlap with Sjögren’s syndrome), connective tissue disease associated DM, inclusion body myositis, polymyositis or drug-induced myopathy 4. Immune-mediated necrotizing myopathy with absence of typical DM rash 5. Generalized, severe musculoskeletal conditions other than DM that prevent a sufficient assessment of the subject by the physician 6. Received blood or plasma-derived products (other than IGIV) or plasma exchange within the last 3 months before enrolment 7. Receiving permitted concomitant medications exceeding the maximally allowed conditions as above 8. Received non-permitted concomitant medications within the washout periods 9. Starting or planning to start a physical therapy–directed exercise regimen during the trial. Subjects on stable physical therapy for >4 weeks are allowed but the regimen should remain the same throughout the trial 10. Cardiac insufficiency (New York Heart Association III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease 11. Severe liver disease, with signs of ascites and hepatic encephalopathy 12. Severe kidney disease (as defined by estimated glomerular filtration rate (eGFR) 40 kg/m2 and/or body weight >120 kg 16. Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome) 17. Known IgA deficiency with antibodies to IgA 18. History of hypersensitivity, anaphylaxis or severe systemic response to immunoglobulin, blood or plasma derived products or any component of Octanorm 16.5% such as polysorbate 80 or to sodium chloride 19. Known blood hyperviscosity, or other hypercoagulable states 20. Subjects with a history of drug abuse within the past 5 years prior to study enrolment 21. Participating in another interventional clinical study with investigational treatment within 3 months prior to study enrolment. Subjects who participated in the Octagam 10% Dermatomyositis Study (GAM10-08) can be included 22. Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to apply an effective birth control method up to 4 weeks after the last Octagam infusion received
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Muscle strength assessed using MMT-8 (manual muscle testing of 8 designated muscles tested bilaterally [potential score 0-150]) at week 32 2. Cutaneous disease activity assessed using Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) at week 32 3. Disease activity at week 32 assessed using Physician's Global Disease Activity on a visual analog scale (VAS) assessing global disease activity from “No evidence of disease activity” to “Extremely active or severe disease activity”, with disease activity being defined as potentially reversible pathology or physiology resulting from the myositis) | — |
Secondary
| Measure | Time frame |
|---|---|
| 4. Extra-muscular disease activity at baseline and week 32 assessed using Myositis Disease Activity Assessment Tool (MDAAT), a combined tool that captures the physician’s assessment of disease activity of various organ systems using a scale from 0 = “Not present in the last 4 weeks” to 4 = “New - in the last 4 weeks (compared to the previous 4 weeks)” and a VAS. 5. Muscle damage assessed using blood aldolase, creatine kinase, alanine aminotransferase, aspartate aminotransferase and lactate dehydrogenase levels at baseline and week 32 6. Disability assessed using Health Assessment Questionnaire (HAQ) at baseline and week 32. HAQ is a generic rather than a disease-specific instrument; consisting of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 [without any difficulty] to 3 [unable to do]. For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8. 7. Patient-reported health assessed using SF-36 v2 Health Survey at baseline and week 32. The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index. 8. Improvement in patient-reported health assessed using Total Improvement Score (TIS) at week 32. 9. Time to clinically important deterioration defined as worsening of Core Set Measures and/or CDASI during the treatment period (Weeks 0, 4, 8, 12,16, 20, 24 and 28) 10. List of deviations from protocol requirements relating to home treatment: (e.g. dosing, timing) documented at weeks 4, 8,12, 16, 20, 24 and 28 11. Occurrence of all adverse events wit | — |
Countries
Czech Republic, Germany, Hungary, Netherlands, Poland, Romania, Russian Federation, United States of America
Outcome results
None listed