Human normal immunoglobulin for Idiopathic inflammatory myopathy|Dermatomyositis|Antisynthetase syndrome|Immune-mediated necrotizing myopathy|Polymyositis|Overlap/non-specific myositis

Inclusion criteria

• {"criterion_text":"- Adult patients (≥ 18 years)\n- IIM diagnosis accordiging to diagnostic criteria: - dermatomyositis, - antisynthetase syndrome, - Immune mediated necrotizing myopathy, - overlap/non-specific myositis, including polymyositis.\n- Disease duration <12 months\n- 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\n- Signed informed consent"}

Exclusion criteria

• {"criterion_text":"- Severe muscle weakness (i.e. bedridden, not able to walk, severe dysphagia requiring a nasogastric tube, or symptomatic respiratory muscle weakness (respiratory symptoms in combination with a forced vital capacity below 50% of predicted in upright position)) necessitating more intensive treatment than standard glucocorticoids.\n- A known malignancy, which is likely to interfere with outcome assessment.\n- Related to IVIg: o History of thrombotic episodes within 10 years prior to enrolment o Known allergic reactions or other severe reactions to any blood-derived product o Known IgA deficiency and IgA serum antibodies o Pregnancy or trying to conceive o Use of nephrotoxic medication\n- Conditions that are likely to interfere with: o Compliance (legally incompetent and/or incapacitated patients are excluded), or, o Evaluation of efficacy (e.g. due to severe pre-existing disability as a result of any disease other than myositis or due to a language barrier)\n- 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). Exceptions to abovementioned exclusion criteria: • Patients are eligible for inclusion 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 biologicals or other immunosuppressive or immunomodulatory treatment when meeting all of the following criteria: o Stable dose for the last 6 months o The biological or other immunosuppressive or immunomodulatory treatment has been approved for a non-muscular condition (e.g. hematological condition, eczema) o The biological or other immunosuppressive or immunomodulatory treatment is not known to induce inflammatory myopathy"}

Primary endpoints

• {"endpoint_text":"- treatment response at 12 weeks, measured by 'total improvement score'","definition_or_measurement_approach":"Measured by 'total improvement score' at 12 weeks"}

Secondary endpoints

• {"endpoint_text":"- Health related quality of life (HR-QoL) is measured by EQ-5D.\n- Physical functioning is measured by accelerometry.\n- Biomarker (imaging): assess the presence of muscle edema as a marker of disease activity by whole body muscle MRI\n- Biomarker (blood): interferon biomakers (CXCL-10, galectin-9 and Siglec-1)\n- IgG blood levels","definition_or_measurement_approach":"HR-QoL measured by EQ-5D questionnaire; physical activity measured by accelerometry; imaging biomarker assessed by whole body muscle MRI for muscle edema; blood biomarkers measured for interferon markers (CXCL-10, galectin-9, Siglec-1); IgG concentrations measured in blood."}

Netherlands

Earliest CTIS Part Ii Submission Date

07-10-2024

Latest Decision Or Authorization Date

10-10-2024

Processing Time Days

3

Number Of Sites

1

Number Of Participants

48

Primary sponsor

Full Name

Stichting Amsterdam UMC

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands