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NCT Number: NCT06906692

Meningococcal Vaccination in Patients on Complement Inhibitors

Myasthenia gravis is an autoimmune neurological disorder caused by autoantibodies directed predominantly against components of the postsynaptic membrane of the neuromuscular junction. Anti-aquaporin-4 antibody-associated neuromyelitis optica (AQP4-IgG) is an autoimmune inflammatory disease of the central nervous system (CNS) characterized by three main clinical manifestations: transverse myelitis, optic neuritis, and postrema area syndrome. Over the past five years, the FDA and EMA have approved complement inhibitor drugs for the treatment of generalized myasthenia gravis with anti-AChR antibody positivity and for neuromyelitis optica spectrum disorders with anti-AQP4 antibody positivity. Eculizumab is a humanized monoclonal antibody that binds to the C5 fragment of the human complement specifically and with high affinity, inhibiting its cleavage in C5a and C5b and preventing the formation of the membrane attachment C5b-9 complex (MAC) of the terminal portion of the complement cascade. This monoclonal antibody maintains the early components of complement activation (C3b), which are essential for the opsonization of microorganisms and the clearance of immune complexes. Ravulizumab is a monoclonal antibody derived from eculizumab, through the substitution of four specific amino acids, which binds specifically to complement protein C5. These substitutions increase the dissociation of the monoclonal antibody from the C5 fragment within the endosome and promote the recycling of the neonatal Fc receptor-mediated unbound antibody, extending its half-life and duration of action up to an interval of 8 weeks. Zilucoplan is a synthetic macrocyclic peptide composed of 15 amino acids that specifically binds the complement protein C5, inhibiting its cleavage into C5a and C5b by C5 convertase, which results in an underregulation of MAC assembly and cytolytic activity. In addition, this drug binds the C5b fraction of C5, creating a steric encumbrance for the binding of C5b to C6 and preventing subsequent assembly of the MAC if any C5b is formed. Due to their mechanism of action, eculizumab, ravulizumab and zilucoplan result in an increased susceptibility of the patient to systemic infections by encapsulated bacteria (S. pneumoniae, H. influenzae, N. meningitidis). To reduce the risk of infection, all patients should be vaccinated against serogroups A, B, C, W, Y at least 2 weeks prior to treatment; patients who start treatment before 2 weeks after administration of meningococcal vaccines should be given appropriate antibiotic prophylaxis for up to 2 weeks after vaccination. Currently, there is no global agreement on the vaccination schedule to be followed after the first dose. According to Italian guidelines, a booster dose is recommended for the monovalent vaccine only at least 1 month after the first administration and a period of antibiotic prophylaxis beyond the first two weeks after the first doses is not indicated. The primary objective of the study is to evaluate the antibody titles after tetravalent meningococcal vaccination (serogroups A, C, W, Y) in patients candidate to complement inhibitors therapy.

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This study is active but is not currently recruiting participants.

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Fondazione Policlinico A. Gemelli IRCCS

Rome, RM, 00168, Italy

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Study Participant must be ≥ 18 years old;
  • Diagnosis of anti-AChR positive generalized Myasthenia Gravis or Positive anti-AQP4 NMOSD;
  • Need for therapy with complement inhibitor drugs according to the therapeutic indications approved by AIFA;
  • Possibility of follow-up in the reference centre;
  • Signing of the Informed Consent to the Study.

Exclusion criteria

  • Age < 18 years;
  • Poor Compliance with Drug Therapy
  • Insufficient availability of Clinical Information;
  • Current Neoplasm or Infection at the Time of Collection of Biological Samples;
  • Refusal to Sign the Informed Consent to the Study

Treatment and study plan

Serum Bactericidal Assay

Diagnostic Test

The Serum Bactericidal Assay (SBA) will be used for the analysis of the antibody titer, which represents a valid surrogate for the efficacy of the tetravalent vaccine. Subjects' sera will be serially diluted and incubated with a solution containing a certain strain of meningococcus. At this point, an exogenous source of rabbit complement will be added. Bactericidal activity will be assessed against controls. Neutralizing antibody titers will be defined as the highest dilution of sera at which at least 50% of bacteria death occurs. Antibody titers will be considered protective with a cut-off greater than or equal to 1:8.

Primary outcomes

  1. Evaluation of antibody titres after tetravalent meningococcal vaccine (serogroups A, C, W, Y) in patients candidate to complement inhibitors therapy

    Time frame: 24 months

    Antibody titers will be considered protective with a cut-off greater than or equal to 1:8. The primary endpoint of this study is to evaluate the number of patients that will develope a protective titer of neutralizing antibodies against meningococcus A, C, W, Y after vaccination

Secondary outcomes

  1. Evaluation of chronic intake of immunosuppressive therapies

    Time frame: 24 months

    Evaluation of correlations between antibody titles and chronic intake of immunosuppressive therapies

Sponsors and collaborators

Lead sponsor

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Other

Registry information

Official study title

Evaluation of Antibody Titles After Meningococcal Vaccination in Patients With Myasthenia Gravis and Neuromyelitis Optica Candidate to Complement Inhibitors Therapy

Acronym: Vax_MenACWY

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Apr 2, 2025
Registry last updated
Apr 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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