Proteomic Changes in Patients With Myasthenia Gravis and Ravulizumab
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Myasthenia Gravis Generalised. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Identification of Proteomic Changes in Patients With Generalized Myasthenia Gravis Treated With Ravulizumab: Insights Into Neuromuscular Junction Regeneration
Overview
Myasthenia gravis (MG) is an autoimmune neuromuscular disorder primarily caused by antibodies targeting postsynaptic components of the neuromuscular junction, most commonly the acetylcholine receptor (AChR). In AChR-positive generalized MG, IgG1 and IgG3 antibodies activate the classical complement pathway, leading to membrane attack complex-mediated damage of the postsynaptic membrane and impaired neuromuscular transmission. Complement inhibition has therefore emerged as an effective therapeutic strategy. Ravulizumab, a long-acting monoclonal antibody targeting complement component C5, has demonstrated clinical efficacy in reducing disease severity in patients with AChR-positive generalized MG. However, clinical responses to complement inhibition remain heterogeneous, and reliable biomarkers to monitor treatment response and neuromuscular junction recovery are currently lacking. Blood-based proteomics represents a powerful approach for identifying molecular changes associated with disease activity and treatment response. In particular, aptamer-based proteomic platforms such as the SomaScan® assay allow high-throughput, highly sensitive quantification of thousands of circulating proteins from small volumes of plasma or serum. The primary aim of this study is to identify proteomic changes in patients with generalized MG treated with Ravulizumab, with a specific focus on proteins involved in neuromuscular junction regeneration and repair. By leveraging advanced proteomic technologies in a real-world clinical setting, this study seeks to identify biomarkers that may help monitor treatment response, guide optimization of concomitant immunosuppressive therapies, and improve patient stratification. Ultimately, the identification of molecular pathways associated with neuromuscular junction regeneration may open new therapeutic perspectives for autoimmune neuromuscular disorders.
Primary outcome measures
- To identify significant changes in plasma protein profiles in patients with generalized myasthenia gravis (gMG) treated with Ravulizumab. [Time frame: 26 weeks]
Secondary outcome measures (2)
- To identify plasma proteins associated with a clinically meaningful improvement in patients with gMG. [Time frame: 26 weeks]
- To identify potential biomarkers of neuromuscular junction regeneration in patients with gMG. [Time frame: 24 months]
Eligibility criteria
Inclusion criteria
- Age ≥18 years;
- Diagnosis of generalized anti-AChR positive Myasthenia Gravis;
- Need for therapy with Ravulizumab drugs according to the therapeutic indications approved by AIFA;
- Signed informed consent to the study.
Exclusion criteria
- Age <18 years;
- Concomitant autoimmune diseases;
- Insufficient availability of clinical information;
- Ongoing neoplasia or infection at the time of biological sample collection
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Italy · 1 center
- Fondazione Policlinico Universitario A. Gemelli IRCCS — Roma
Identifiers
NCT: NCT07337395 · 8167