Markers of Favorable Response to FcRn Inhibitors(INFORM)

NCT06685055 | Recruiting

• 1 other identifier: 6717
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

Myasthenia gravis is an autoimmune neurological disease caused by autoantibodies primarily directed against components of the postsynaptic membrane of the neuromuscular junction. Approximately 85% of patients have antibodies directed against the acetylcholine receptor (anti-AChR). Anti-AChR antibodies act through three distinct mechanisms:

1. 1.Activation of the classical complement pathway: Formation of membrane-attack complexes (MACs) results in the destruction of the postsynaptic membrane.
2. 2.Mechanical blockade: Anti-AChR antibodies block the acetylcholine binding site on its receptor.
3. 3.Internalization and lysosomal degradation: Bivalent IgG causes cross-linking of adjacent receptors leading to internalization and degradation of AChRs (antigenic modulation).

Conditions

Myasthenia Gravis Generalized

Outcome Measures

Primary Outcomes (6)

• Identification of Clinical Markers of Favorable Response to FcRn Inhibitors Therapy in Patients With Generalized Myasthenia Gravis

  Analyze the clinical status of patients comparing the results of MG-ADL clinical scale pre/post FcRn Inhibitors

  24 months

• Identification of Biological and Cellular Markers of Favorable Response to FcRn Inhibitors Therapy in Patients With Generalized Myasthenia Gravis

  Analyze the differences between IgG levels pre/post therapy with FcRn inhibitors

  24 months

• Identification of Biological and Cellular Markers of Favorable Response to FcRn Inhibitors Therapy in Patients With Generalized Myasthenia Gravis

  • Evaluate anti-AChR positivity in patients treated with FcRn inhibitors

  24 months

• Identification of Biological and Cellular Markers of Favorable Response to FcRn Inhibitors Therapy in Patients With Generalized Myasthenia Gravis

  • Dosage of proteins involved in pathogenesis

  24 months

• Identification of Genetic Markers of Favorable Response to FcRn Inhibitors Therapy in Patients With Generalized Myasthenia Gravis

  Investigate the presence of polymorphisms in the FCGRT gene (VTNRs) in patients refractory to therapy with FcRn inhibitors

  24 months

• Identification of Clinical Markers of Favorable Response to FcRn Inhibitors Therapy in Patients With Generalized Myasthenia Gravis

  Analyze the clinical status of patients comparing the results of QMG clinical scale pre/post FcRn Inhibitors

  24 months

Secondary Outcomes (1)

• Predictive algorithm of favorable response

  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 FcRn inhibitor drugs

You may qualify if:

• \- Age ≥18 years.
• Diagnosis of generalized anti-AChR positive Myasthenia Gravis.
• Need for therapy with neonatal Fc receptor inhibitors for immunoglobulins (FcRn) as per AIFA-approved therapeutic indications (14).
• Ability to follow up at the reference center.
• Signed informed consent for the study.

You may not qualify if:

• Age \<18 years.
• Poor compliance with drug therapy.
• Concurrent autoimmune diseases.
• Insufficient availability of clinical information.
• Ongoing neoplasm or infection at the time of biological sample collection.
• Refusal to sign the informed consent for the study.

Sponsors & Collaborators

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS: lead

Study Sites (1)

Fondazione Policlinico Universitario A. Gemelli IRCCS

Rome, 00168, Italy

RECRUITING

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

  Fondazione Policlinico Universitario A. Gemelli, IRCCS

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Raffaele Iorio, MD, PhD

CONTACT

+390630154807; raffaele.iorio@policlinicogemelli.it

Silvia Falso, MD

CONTACT

+390630154807; falsosilvia@gmail.com

Study Design

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

Study Record Dates

• First Submitted: July 10, 2024
• First Posted: November 12, 2024
• Study Start: July 9, 2024
• Primary Completion (Estimated): July 9, 2026
• Study Completion (Estimated): July 9, 2026
• Last Updated: November 12, 2024

Record last verified: 2024-07

Locations

IT (1)