Proteomic Changes in Patients With Myasthenia Gravis and Ravulizumab

Официальное название

Identification of Proteomic Changes in Patients With Generalized Myasthenia Gravis Treated With Ravulizumab: Insights Into Neuromuscular Junction Regeneration

Обзор

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.

Первичные конечные точки

• To identify significant changes in plasma protein profiles in patients with generalized myasthenia gravis (gMG) treated with Ravulizumab. [Срок оценки: 26 weeks]

Вторичные конечные точки (2)

• To identify plasma proteins associated with a clinically meaningful improvement in patients with gMG. [Срок оценки: 26 weeks]
• To identify potential biomarkers of neuromuscular junction regeneration in patients with gMG. [Срок оценки: 24 months]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• Age <18 years;
• Concomitant autoimmune diseases;
• Insufficient availability of clinical information;
• Ongoing neoplasia or infection at the time of biological sample collection

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Италия · 1 центр

• Fondazione Policlinico Universitario A. Gemelli IRCCS — Roma

Идентификаторы

NCT: NCT07337395 · 8167

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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