Proteomic Changes in Patients With Myasthenia Gravis and Ravulizumab

NCT07337395 | Not Yet Recruiting DMC

• 1 other identifier: 8167
• Study Type: observational
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Myasthenia Gravis Generalised

Keywords

Myasthenia Gravis; Ravulizumab; Proteomic changes

Outcome Measures

Primary Outcomes (1)

• To identify significant changes in plasma protein profiles in patients with generalized myasthenia gravis (gMG) treated with Ravulizumab.

  To identify changes in plasma proteins after 10 and 26 weeks of treatment with Ravulizumab compared with baseline, using proteomic technology.

  26 weeks

Secondary Outcomes (2)

• To identify plasma proteins associated with a clinically meaningful improvement in patients with gMG.

  26 weeks

• To identify potential biomarkers of neuromuscular junction regeneration in patients with gMG.

  24 months

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with generalized Myasthenia Gravis positive for anti-AChR antibodies undergoing therapy with Ravulizumab

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS: lead
• Alexion Pharmaceuticals, Inc.: collaborator

Study Sites (1)

Fondazione Policlinico Universitario A. Gemelli IRCCS

Roma, 00168, Italy

Location

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Raffaele Iorio, Prof

  Fondazione Policlinico Universitario A. Gemelli, IRCCS

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Raffaele Iorio, MD, PhD

CONTACT

+390630154807; raffaele.iorio@policlinicogemelli.it

Sofia Marini, MD

CONTACT

+390630154807; sofiamarini97@gmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 19, 2025
• First Posted: January 13, 2026
• Study Start: January 1, 2026
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): January 1, 2028
• Last Updated: January 13, 2026

Record last verified: 2025-12

Locations

IT (1)