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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Subjects with diagnosis of definite or probable DM according to the Bohan and Peter
criteria.
2. Subjects who have responded to IGIV treatment as assessed by the treating physician
and being on a stable dose for at least 3 months on 2 g/kg bodyweight (+/- 10%).
3. For subjects being on other medication(s) for the treatment of DM (immunosuppressants,
corticosteroids): a) subject was on such medication(s) at the start of IGIV treatment
in the first place, and b) received such 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 at
the maximally allowed conditions as per Table 2 (see section 4.2.1).
4. MMT-8 score =144, with at least 3 other CSM to be normal or near normal as per the
following criteria: Visual Analogue Scale [VAS] of patient global disease activity =2
cm, physician's global disease activity =2 cm, extra-muscular disease activity =2 cm;
no muscle enzyme >4 times upper limit of normal due to myositis, Health Assessment
Questionnaire [HAQ] =0.25.
5. Males or females = 18 to <80 years of age.
6. Voluntarily given, fully informed written consent obtained from subject before any
study-related procedures are conducted.
7. Subject must be capable and willing to understand and comply with the relevant aspects
of the study protocol.
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. Subjects with overlap myositis (except for overlap with Sjögren's syndrome),
connective tissue disease associated DM, inclusion body myositis, polymyositis or
drug-induced myopathy.
4. Subjects with immune-mediated necrotizing myopathy with absence of typical DM rash.
5. Subjects with generalized, severe musculoskeletal conditions other than DM that
prevent a sufficient assessment of the subject by the physician.
6. Subjects who received blood or plasma-derived products (other than IGIV) or plasma
exchange within the last 3 months before enrolment.
7. Subjects with administration of permitted concomitant medications exceeding the
maximally allowed conditions as per section 4.2.1.
8. Subjects with administration of forbidden concomitant medications within the washout
periods as defined in Table 3: see section 4.2.2.
9. Subjects 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) < 30
mL/min/1.73 m2).
13. Known hepatitis B, hepatitis C or HIV infection.
14. Subjects with a history of deep vein thrombosis within the last year prior to study
enrolment or pulmonary embolism ever.
15. Body mass index >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 (as per protocol section 7.3.9 b)
up to four weeks after the last IMP infusion received.
Age minimum:
18 Years
Age maximum:
80 Years
Gender:
All
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Secondary Outcome(s)
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Adverse Events
[Time Frame: 32 weeks]
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Body Temperature
[Time Frame: 32 weeks]
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D-dimers
[Time Frame: 32 weeks]
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Hematocrit
[Time Frame: 32 weeks]
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Injection Site Reactions
[Time Frame: 32 weeks]
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Muscle Enzymes - Aspartate Aminotransferase
[Time Frame: 32 weeks]
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Serum Haptoglobin
[Time Frame: 32 weeks]
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Blood Urea Nitrogen
[Time Frame: 32 weeks]
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Hepatitis C
[Time Frame: 32 weeks]
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Muscle Enzymes - Aldolase
[Time Frame: 32 weeks]
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Albumin
[Time Frame: 32 weeks]
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Hemoglobin
[Time Frame: 32 weeks]
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Muscle Enzymes - Lactate Dehydrogenase
[Time Frame: 32 weeks]
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Muscle Enzymes - Creatine Kinase
[Time Frame: 32 weeks]
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Total Bilirubin
[Time Frame: 32 weeks]
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Platelets
[Time Frame: 32 weeks]
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Urine Hemoglobin
[Time Frame: 32 weeks]
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Urine Leukocytes
[Time Frame: 32 weeks]
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Aldolase
[Time Frame: 32 weeks]
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Blood Pressure
[Time Frame: 32 weeks]
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Direct Coombs' Test
[Time Frame: 32 weeks]
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Extra-Muscular Disease Activity
[Time Frame: 32 weeks]
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LDH
[Time Frame: 32 weeks]
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Health Assessment Questionnaire
[Time Frame: 32 weeks]
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Heart Rate
[Time Frame: 32 weeks]
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Urine Urobilinogen
[Time Frame: 32 weeks]
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Urea
[Time Frame: 32 weeks]
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ALAT
[Time Frame: 32 weeks]
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ASAT
[Time Frame: 32 weeks]
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Creatinine
[Time Frame: 32 weeks]
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Serum IgG
[Time Frame: 32 weeks]
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SF-36v2 Health Survey
[Time Frame: 32 weeks]
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HTRs
[Time Frame: 32 weeks]
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Muscle Enzymes - Alanine Aminotransferase
[Time Frame: 32 weeks]
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Physical Examination
[Time Frame: 32 Weeks]
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Potassium
[Time Frame: 32 weeks]
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Respiratory Rate
[Time Frame: 32 weeks]
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Creatine Kinase
[Time Frame: 32 weeks]
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Glucose
[Time Frame: 32 weeks]
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HIV
[Time Frame: 32 weeks]
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Mean Change in TIS
[Time Frame: 32 weeks]
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Plasma-Free Hemoglobin
[Time Frame: 32 weeks]
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TEEs
[Time Frame: 32 weeks]
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Urine Bilirubin
[Time Frame: 32 weeks]
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Urine pH
[Time Frame: 32 weeks]
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Sodium
[Time Frame: 32 weeks]
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Urine Ketones
[Time Frame: 32 weeks]
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Pregnancy Test
[Time Frame: 32 weeks]
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White Blood Cell Count
[Time Frame: 32 weeks]
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Hepatitis B
[Time Frame: 32 weeks]
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Red Blood Cell Count
[Time Frame: 32 weeks]
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Time to Clinically Important Deterioration
[Time Frame: 32 weeks]
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Urine Glucose
[Time Frame: 32 weeks]
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Urine Hemosiderin
[Time Frame: 32 weeks]
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Urine Nitrite
[Time Frame: 32 weeks]
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Urine Protein
[Time Frame: 32 weeks]
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