Efgartigimod for Myasthenia Gravis

Recruiting in Palo Alto (17 mi)

Overseen byThomas Ragole, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Waitlist Available

Sponsor: University of Colorado, Denver

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This study plans to learn more about if the drug efgartigimod can be used in the hospital to treat exacerbations in participants with myasthenia gravis (MG). Efgartigimod has been approved by the FDA for ongoing (chronic) treatment of generalized MG in adult patients who are anti-acetylcholine receptor (AChR) antibody positive but has not been studied in the treatment of worsening weakness requiring hospital admission (known as "exacerbation"). This investigation aims to see if using efgartigimod in this way improves symptoms and recovery from exacerbation, and how it affects certain MG markers in the blood. The main questions it aims to answer are: * Is efgartigimod effective as a hospital-administered acute therapy for participants with worsening MG (MG exacerbation) who require hospitalization? * Will efgartigimod lead to clinical improvement with a similar reduction in validated research scales, such as the Quantitative MG (QMG) scale, as standard of care therapies? Participants will receive 4 doses of efgartigimod over the course of 4 weeks with an additional follow-up visit at the clinic.

Eligibility Criteria

This trial is for hospitalized adults with myasthenia gravis (MG) experiencing a worsening of symptoms, known as an exacerbation. Participants must have MG that's confirmed by specific antibodies. Details on who can't join are not provided.

Inclusion Criteria

Abnormal single fiber EMG

Abnormal decrement on slow repetitive nerve stimulation testing

Quantitative Myasthenia Gravis (QMG) scale ≥ 11

+6 more

Exclusion Criteria

I am currently using ravulizumab or eculizumab.

Intubation prior to ability to sign informed consent or intubation within 24 hours of hospitalization

I don't have health issues that could affect study assessments.

+6 more

Participant Groups

The study tests if efgartigimod, already approved for chronic MG treatment, is effective when given in the hospital to treat acute MG exacerbations. Patients will receive four doses over four weeks and be monitored using the QMG scale.

1Treatment groups

Experimental Treatment

Group I: EfgartigimodExperimental Treatment1 Intervention

Participants receive 4 doses of efgartigimod via intravenous (IV) infusion over the course of the study on days 1, 4, 11 and 18.