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

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

Efgartigimod
Drug
Dose of 10 mg/kg for IV infusion on days 1, 4, 11 and 18

Treatment groups

20 Participants

are divided into 1 treatment group

Group A: EfgartigimodExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at day 11

The Quantitative Myasthenia Gravis (QMG) is a scoring system that quantifies disease severity by measuring ocular, bulbar, respiratory and limb strength. The total scores range from 0 to 39, with higher scores indicating greater disease severity.

Time frame

Baseline, Day 11

Secondary outcomes

1

Percentage of participants requiring rescue therapy with Plasma Exchange (PLEX) or Intravenous Immunoglobulin (IVIG)

Number of participants requiring rescue therapy with Plasma Exchange (PLEX) or Intravenous Immunoglobulin (IVIG) over number of total participants

Time frame

During hospitalization, approximately 7 days

2

Change from baseline in Myasthenia Gravis Activities of Daily Life (MG-ADL) scale at day 18

The Myasthenia Gravis Activities of Daily Life (MG-ADL) is a questionnaire administered verbally that measures MG symptoms and functional activities related to activities of daily living. The summative total scores range from 0 to 24, with higher scores indicating greater disease severity.

Time frame

Baseline, Day 18

3

Change from baseline in Myasthenia Gravis Activities of Daily Life (MG-ADL) scale at day 32

The Myasthenia Gravis Activities of Daily Life (MG-ADL) is a questionnaire administered verbally that measures MG symptoms and functional activities related to activities of daily living. The summative total scores range from 0 to 24, with higher scores indicating greater disease severity.

Time frame

Baseline, Day 32

4

Change from baseline in Myasthenia Gravis Manual Muscle Test (MG-MMT) at day 11

The Myasthenia Gravis Manual Muscle Test (MG-MMT) is an assessment that assigns numerical values to the presence of mild, moderate or severe weakness for specific muscle groups that may be affected by MG. The summative total scores range from 0 to 120, with higher scores indicating greater disease severity.

Time frame

Baseline, Day 11

Treatment of Myasthenia Gravis Exacerbation or Crisis With Efgartigimod

About this trial

Eligibility criteria

Qualifiers

Disqualifiers

Trial design

Treatments tested in this trial

Treatment groups

Trial outcomes

Primary outcomes

Secondary outcomes

Other outcomes

Sponsors and contacts