Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis (Idiopathic Inflammatory Myopathy)
Condition: Dermatomyositis
MeSH terms: Dermatomyositis
Intervention: Octagam 10%; Placebo
Intervention description: Patients to be treated with Octagam 10%; Patients to be treated with a Placebo
Intervention MeSH terms: gamma-Globulins; Immunoglobulins, Intravenous; Rho(D) Immune Globulin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 95.0
Sex: All
Minimum age: 18 Years
Maximum age: 79 Years
Healthy volunteers: No
Keywords: DM

Summary:

      Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy
      and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subjects with diagnosis of definite or probable DM according to the Bohan and Peter
             criteria.

          2. Subjects under treatment with corticosteroids and/or maximally 2 immune-suppressants
             and being on stable therapy for at least 4 weeks (see Section 4.2.1) OR Subjects with
             previous failure of response or previous intolerance to corticosteroid and at least 1
             additional immunosuppressive drug, and with steroid/immunosuppressive drugs washed out
             as per Section 4.2.1 (Table 2).

          3. Subjects with active disease, assessed and agreed upon by an independent adjudication
             committee.

          4. Manual Muscle Testing-8 (MMT-8) score <142, with at least 2 other abnormal Core Set
             Measures (CSM) (Visual Analogue Scale [VAS] of patient global activity ≥2 cm,
             physician's global disease activity ≥2 cm, extra-muscular activity ≥2 cm; at least one
             muscle enzyme >1.5 times upper limit of normal, Health Assessment Questionnaire
             ≥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 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.

          3. Subjects with overlap myositis (except for overlap with Sjögren's syndrome),
             connective tissue disease associated DM, inclusion body myositis, polymyositis,
             juvenile dermatomyositis 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 have received IgG treatment within the last 6 months before enrolment.

          7. Subjects who received blood or plasma-derived products (other than IgG) or plasma
             exchange within the last 3 months before enrolment.

          8. Subjects starting or planning to start a physical therapy-directed exercise regimen
             during the trial.

          9. Cardiac insufficiency (New York Heart Association III/IV), cardiomyopathy, significant
             cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease.

         10. Severe liver disease, with signs of ascites and hepatic encephalopathy.

         11. Severe kidney disease (as defined by estimated glomerular filtration rate (eGFR) < 30
             mL/min/1.73 m2).

         12. Known hepatitis B, hepatitis C or HIV infection.

         13. Subjects with a history of TEE such as deep vein thrombosis, pulmonary embolism,
             myocardial infarction, ischemic stroke, transient ischemic attack, peripheral artery
             disease (Fontaine IV) ever.

         14. Body mass index ≥40 kg/m2.

         15. Medical conditions whose symptoms and effects could alter protein catabolism and/or
             IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).

         16. Known IgA deficiency with antibodies to IgA.

         17. History of hypersensitivity, anaphylaxis or severe systemic response to
             immuno-globulin, blood or plasma derived products or any component of Octagam 10%.

         18. Known blood hyperviscosity, or other hypercoagulable states.

         19. Subjects with a history of drug abuse within the past 5 years prior to study
             enrollment.

         20. Subjects unable or unwilling to understand or comply with the study protocol.

         21. Participating in another interventional clinical study with investigational treatment
             within 3 months prior to study enrollment.

         22. Women who are breast feeding, pregnant, or planning to become pregnant, or are
             unwilling to apply an effective birth control method (such as implants, injectables,
             combined oral contraceptives, some intrauterine devices [IUDs], sexual abstinence or
             vasectomized partner) up to four weeks after the last IMP infusion.

         23. Subjects who are accommodated in an institution or care facility based on an official
             directive or court order.

         24. Subjects who are in any way dependent on the Sponsor, Investigator or Study Site.

         25. Subjects who received forbidden medication within the washout period as defined in
             Section 4.2.2 (Table 3).