FcRn Antagonists (Efgartigimod) for Acute NMOSD Attack

Launched by TIANJIN MEDICAL UNIVERSITY GENERAL HOSPITAL · Jul 4, 2024

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment called efgartigimod for patients experiencing an acute attack of neuromyelitis optica spectrum disorders (NMOSD), a condition where the immune system mistakenly attacks the central nervous system, particularly affecting vision and movement. Researchers want to see if efgartigimod can safely reduce the levels of certain antibodies that contribute to these attacks, potentially helping patients recover more effectively.

To participate in this study, you must be between 18 and 75 years old and have been diagnosed with NMOSD, confirmed by specific tests. You should also be experiencing a recent acute attack that has worsened your symptoms. This study is not yet recruiting participants, but once it does, eligible patients can expect to be closely monitored throughout the treatment process, which will include evaluations of their symptoms and imaging tests like MRIs to track their condition. It's important to note that certain medical history factors, like recent treatments or other health issues, might prevent someone from joining the trial.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Male or female, ages 18 to 75.
• • 2. Meet the 2015 International Panel of Experts (IPND) diagnostic criteria for neuromyelitis optica spectrum disorders.
• • 3. Acute EDSS nadir of 2.5-7.5, and a change of at least 0.5 points from baseline due to an acute relapse event.
• • 4. Confirmation of serum AQP4-IgG antibody positivity using the CBA assay.
• • 5. Confirmation of an acute attack of neuromyelitis optica spectrum disorders either with an acute optic neuritis and/or acute myelitis, defined as a worsening in the patient's signs and symptoms of neurological/visual impairment, an increase in the EDSS score, and symptoms lasting more than 24 hours and occurring more than 1 month since the last attack. Combination of imaging and clinical evaluation will be used to assess relapse and rule out a pseudorelapse.
• • 6. New lesions or enhanced lesions need to be found in MRI.
• • 7. Subjects who were receiving immunosuppressive therapy prior to the screening period will be required to agree to discontinue immunosuppression.
• • 8. Treatment is stable at least 3 months.
• Exclusion Criteria:
• • 1. Other core clinical symptoms besides optic neuritis and myelitis.
• • 2. Severe neuromyelitis optica spectrum disorder attack, which in the judgment of the investigator is not appropriate for this study. Severe is defined as requiring assisted ventilation or likely to require assisted ventilation during the study based on the judgment of the investigator.
• • 3. Subjects with total IgG levels ≤ 6 g/L at screening.
• • 4. Subjects with a B-cell count ≤ 5% of the lower limit of normal at screening.
• • 5. Received high-dose intravenous methylprednisolone within 4 weeks prior to the screening period.
• • 6. Received intravenous immunoglobulin, plasma exchange, or immunoadsorption treatment within 4 weeks prior to the screening period.
• • 7. Received a vaccination within the first 4 weeks of the screening period or planned during the study.
• • 8. Using of a monoclonal antibody or investigational drug not mentioned above that has immunomodulatory effects within 3 months or 5 half-lives (whichever is longer) prior to the screening period.
• • 9. Subject is known to be allergic to any component of the study drug or any other FcRn drug or contrast medium for enhanced MRI.
• • 10. Subject is known to have contraindications to taking methylprednisolone (for subjects in A and B group).
• • 11. Subjects with clinically significant active infections (including unresolved or inadequately treated infections, including active tuberculosis) as assessed by the investigator.
• • 12. Subjects with positive screening tests for hepatitis B and C who have received live or live attenuated vaccine within 6 weeks prior to baseline.
• • 13. Subjects is known unable to taken MRI.
• • 14. Subjects is known to have other ophthalmic disease that affect vision as assessed by the investigator.