Proteomic Changes in Patients With Myasthenia Gravis and Ravulizumab (PROMPT)

Institute of Hospitalization and Scientific Care (IRCCS)

Status: Not yet enrolling

Conditions: Myasthenia Gravis Generalised

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT07337395

8167 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 24 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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