Studies blood protein changes in myasthenia gravis patients treated with ravulizumab
Looks at blood proteins in adults with generalized myasthenia gravis receiving ravulizumab.
Plain-language title generated from the registry record
Registry title: Proteomic Changes in Patients With Myasthenia Gravis and Ravulizumab
Key facts
- Where
- Roma, ItalySee the site
Where in Italy
- Fondazione Policlinico Universitario A. Gemelli IRCCSRoma, ItalyNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Trial code
- NCT07337395
- Record updated
- 13 January 2026
- Original description
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.Read full original descriptionShow less
Identification of Proteomic Changes in Patients With Generalized Myasthenia Gravis Treated With Ravulizumab: Insights Into Neuromuscular Junction Regeneration
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.
Inclusion: 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: Concomitant autoimmune diseases; Ongoing neoplasia or infection at the time of biological sample collection; Age <18 years; Insufficient availability of clinical information.