G70.0
Conditions
Interventions
Group 1: Therapy-naive MG patients will be included (MG = Myasthenia gravis). Only symptomatic therapy and steroids are allowed at baseline. Afterwards, patients are followed up every 6 month over 24
Sponsors
Charité – Universitätsmedizin Berlin
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: - Patients capable of giving consent - Written consent for participation - Established diagnosis of generalized MG, confirmed by a positive serological test for AChR antibodies at screening (confirmed by ELISA or cell-based assay) and one of the following: o positive decrement in history demonstrated by repeated nerve stimulation, except for the orbicularis oculi muscle; or o history of positive anticholinesterase test (e.g. edrophonium chloride or neostigmine test); or o Improvement of myasthenic symptoms with oral acetylcholinesterase inhibitors, as assessed by the study physician - Newly diagnosed MG at study entry (first documented MG diagnosis no more than 12 months prior) - Immunotherapy-naïve MG patients at study entry (previous steroid and pyridostigmine administration allowed)
Exclusion criteria
Exclusion criteria: - Previous treatment with immunosuppressive therapies (azathioprine, mycophenolate mofetil, methotrexate, ciclosporin, tacrolimus) and/or modern additional therapies (e.g. C5 inhibitors, FcRn inhibitors) - Previous therapy with B-cell-depleting therapies (e.g. rituximab, daratumumab) - previous therapy with intravenous immunoglobulins for immunomodulation = 3 months prior to study inclusion - previous therapy with exacerbation therapies (plasma exchange, immunoadsorption, intravenous immunoglobulins) = 3 months prior to study inclusion - Non-thymoma-associated MG patients with a history of thymectomy - Thymoma-associated MG patients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Identification of potentially prognostic biomarkers or the combination of different biomarkers (biomarker signature) in early-diagnosed MG patients for the prediction of a highly active disease course during the observational period of 24 months. Blood samples are drawn at baseline and every six months together with clinical outcome parameters. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Level difference in the factors of the complement system; investigation and description of the role of the different factors of the classical (C3a, C5, C5a, sC5b9), alternative (factor D, factor H, Bb and Ba) and lectin complement pathway (C4a) 2) Difference in patient characteristics (e.g. age at diagnosis, gender, MGFA, QMG, MGC at diagnosis, first symptoms, clinical presentation, immunosuppressive MG therapy, concomitant medication, etc.); to investigate whether and how demographic and clinical characteristics are associated with highly active disease progression. 3) To assess the severity of newly diagnosed MG patients treated with baseline therapy and to monitor and document the occurrence of myasthenic crisis; to estimate the frequency of patients developing a highly active disease course (myasthenic exacerbation/crisis) at 6, 12, 18 and 24 months after study inclusion. | — |
Countries
Germany
Contacts
Public ContactFrauke Stascheit
Charité – Universitätsmedizin Berlin
Outcome results
None listed