Identifying markers for effective treatment of Myasthenia Gravis with complement inhibitors
Identification of Clinical, Biological, Cellular and Genetic Markers of Favorable Response Complement Inhibitors Therapy in Patients With Generalized Myasthenia Gravis
This study is trying to find out which markers can help doctors know if complement inhibitor therapy will work well for people with generalized Myasthenia Gravis who have anti-AChR antibodies.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Fondazione Policlinico Universitario Agostino Gemelli IRCCS Academic / other |
| Drugs / interventions | Eculizumab |
| Locations | 1 site (Roma) |
| Trial ID | NCT06455709 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on patients with generalized Myasthenia Gravis who are positive for anti-AChR antibodies. It aims to identify specific markers that indicate a favorable response to complement inhibitor therapy, which has been recently approved for treatment. The study will involve monitoring patients who meet the eligibility criteria and require therapy with these drugs, assessing their clinical outcomes and biological responses. The findings could help tailor treatments for better patient outcomes.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with generalized anti-AChR positive Myasthenia Gravis who require complement inhibitor therapy.
Not a fit: Patients under 18 years old, those with poor compliance to therapy, or those with other autoimmune diseases may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to more personalized and effective treatment strategies for patients with Myasthenia Gravis.
How similar studies have performed: Other studies have shown promise in using complement inhibitors for Myasthenia Gravis, indicating a potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age ≥18 years; * Diagnosis of generalized anti-AChR positive Myasthenia Gravis; * Need for therapy with complement inhibitor drugs according to the therapeutic indications approved by AIFA (16); * Ability to perform follow-up at the reference center; * Signed informed consent to the study. Exclusion Criteria: * Age \<18 years; * Poor compliance with drug therapy; * Concomitant autoimmune diseases; * Insufficient availability of clinical information; * Ongoing neoplasia or infection at the time of biological sample collection; * Refusal to sign the informed consent to the study
Where this trial is running
Roma
- Fondazione Policlinico Universitario A. Gemelli IRCCS — Roma, Italy (Recruiting)
Study contacts
- Principal investigator: Raffaele Iorio — Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Study coordinator: Raffaele Iorio, MD, PhD
- Email: raffaele.iorio@policlinicogemelli.it
- Phone: +390630154807
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.