Treatment With add-on IVIg in Myositis Early In the diSease Course May be sUperior to Steroids Alone for Reaching CLinical improvemEnt

NL74270.018.20

In patients with myositis early immunomodulation by intensive treatment ("hit-early/hit-hard" principle) may induce faster reduction of disease activity and prevent chronic disability. Intravenous immunoglobulin (IVIg) in addition to standard treatment with glucocorticoids may be beneficial for this purpose: add-on IVIg improved symptoms in steroid-resistant myositis, and first-line monotherapy IVIg led to a fast and clinically relevant response in a pilot study in nearly 50% of patients with myositis.

2021-09-13

2024-02-01

2024-09-01

Recruiting

Early phase I

48

Inclusion Criteria: * Adult patients (≥ 18 years) with IIM, according to diagnostic criteria: * Dermatomyositis * Polymyositis * Anti-synthetase syndrome * Immune mediated necrotizing myopathy * Overlap myositis * Disease duration \< 12 months * Minimal disability defined as at least 10% loss on Manual Muscle Testing (MMT) and abnormal scores on two other Core Set Measures (CSMs) of the international Myositis Assessment and Clinical Studies (IMACS) group (see 'Primary and secondary outcomes'). * Patients are eligible for inclusion if they are treatment-naive, or if there is no clinical evident response (as carefully judged by the treating physician at a screening visit) to prior treatment with: * High dosed glucocorticoids, such as dexamethasone (e.g. 40 mg per day up to 4 days) or intravenous methylprednisolone (e.g. 1000 mg daily for three days), within 1 week prior to screening visit. * Daily dosed prednisone 1 mg/kg, or equivalent, used for up to 2 weeks prior to screening visit. * Treatment with low-dosed prednisone (max 20 mg daily) up to three months prior to screening visit. * Treatment with biologicals or other immunosuppressive or immunomodulatory treatment when meeting all of the following criteria: * Stable dose for the last 6 months * The biological or other immunosuppressive or immunomodulatory treatment has been approved for a non-muscular condition (e.g. hematological condition, eczema) and is not known for its use in idiopathic inflammatory myopathy * The biological or other immunosuppressive or immunomodulatory treatment is not known to induce inflammatory myopathy * Signed informed consent Exclusion Criteria: A potentially eligible patient who meets any of the following criteria will be excluded from participation in this study: * Severe muscle weakness (i.e. bedridden, severe dysphagia requiring a feeding tube, or respiratory muscle weakness (forced vital capacity below 50% of predicted in upright position)) necessitating more intensive treatment than standard glucocorticoids from the start. * Related to IVIg: * History of thrombotic episodes within 10 years prior to enrolment * Known allergic reactions or other severe reactions to any blood-derived product * Known Immunoglobulin A (IgA) deficiency and IgA serum antibodies * Pregnancy or trying to conceive * Use of loop diuretics * Use of nephrotoxic medication * Conditions that are likely to interfere with: * Compliance (legally incompetent and/or incapacitated patients are excluded), or, * Evaluation of efficacy (e.g. due to severe pre-existing disability as a result of any other disease than myositis or due to language barrier) * Immunosuppressive medication or immunomodulatory treatment within the last 3 months (e.g. azathioprine, methotrexate, mycophenolate mofetil, tacrolimus, cyclophosphamide, cyclosporine, IVIg, biologicals, Janus kinase inhibitors, plasmapheresis).

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'maskingDescription': 'Home-care nurse preparing study medication will be unblinded. Home-care nurse is not involved in outcome assessment.', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 48, 'type': 'ESTIMATED'}}

2023-04-27