Actively Recruiting

Age: 18Years +
All Genders
ID06455709

Identification of Clinical, Biological, Cellular and Genetic Markers of Favorable Response to Complement Inhibitors Therapy in Patients With Generalized Myasthenia Gravis

Led by Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Updated on 2025-08-01

50

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Myasthenia gravis is an autoimmune neurological disease caused by antibodies attacking parts of the neuromuscular junction, especially the acetylcholine receptor (anti-AChR). This condition can lead to muscle weakness and other symptoms. Researchers are studying how different patients respond to complement inhibitor therapies, which target the immune system component contributing to this disease. The goal is to identify clinical, biological, cellular, and genetic markers that predict which patients will benefit most from these treatments. The study focuses on patients with generalized myasthenia gravis who are positive for anti-AChR antibodies and require treatment with complement inhibitors, as approved by regulatory authorities. No new drugs or treatments are administered in this observational study. Instead, patients will be followed while receiving complement inhibitor therapies prescribed according to standard medical guidelines. Participants in the study will undergo assessments over a 24-month period to collect clinical, biological, cellular, and genetic data. Researchers will analyze these data to find markers associated with favorable responses to complement inhibitors. The study also aims to develop a predictive algorithm to personalize treatment choices. Participants will be regularly monitored at the reference center, and their progress and response to therapy will be carefully documented throughout the study.

CONDITIONS

Brief Title

Markers of Favorable Response to Complement Inhibitors Therapy

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 years or older
  • Diagnosis of generalized anti-AChR positive myasthenia gravis
  • Need for therapy with complement inhibitor drugs as approved by AIFA
  • Ability to perform follow-up at the reference center
  • Signed informed consent to the study
Not Eligible

You will not qualify if you...

  • Age under 18 years
  • Poor compliance with drug therapy
  • Presence of other autoimmune diseases
  • Insufficient availability of clinical information
  • Ongoing cancer or infection at the time of biological sample collection
  • Refusal to sign informed consent to the study

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Monitoring

Duration - 24 months

Participants who undergo routine care with complement inhibitor therapy are observed for clinical, biological, cellular, and genetic markers related to therapy response.

Regular visits for clinical and biological assessments as part of routine care

Trial Site Locations

Total: 1 location

1

Fondazione Policlinico Universitario A. Gemelli IRCCS

Roma, Italy, 00168

Actively Recruiting

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Research Team

R

Raffaele Iorio, MD, PhD

S

Sofia Marini

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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