Treatment of Myasthenia Gravis Exacerbation or Crisis With Efgartigimod
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity of Colorado, Denver
About 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
Qualifiers
Documented positive response to cholinesterase inhibitors such as pyridostigmine or edrophonium
Abnormal decrement on slow repetitive nerve stimulation testing
Abnormal single fiber EMG
Quantitative Myasthenia Gravis (QMG) scale ≥ 11
Disqualifiers
MG worsening thought to be related to active infection or due to medications (e.g. fluoroquinolone or aminoglycoside antibiotics, magnesium, chloroquine derivatives)
Intubation prior to ability to sign informed consent or intubation within 24 hours of hospitalization
Use of IVIG within 2 weeks, or having undergone plasma exchange or received efgartigimod in the 4 weeks prior to admission
Current ongoing use of ravulizumab or eculizumab (monoclonal antibody C5-complement inhibitors).
Trial design
Treatments tested in this trial
- Efgartigimod
Treatment groups
20 Participants
are divided into 1 treatment groupSponsors and collaborators
University of Colorado, Denver
Lead sponsor
argenx
Collaborator
Source ClinicalTrials.gov