Recruiting

Efgartigimod

Sponsor:

University of Colorado, Denver

Code:

NCT06860633

Conditions

Myasthenia Gravis Crisis

Myasthenia Gravis Exacerbations

AChR Myasthenia Gravis

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Efgartigimod

Study Details

Brief summary:

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.

Conditions

Myasthenia Gravis Crisis

Myasthenia Gravis Exacerbations

AChR Myasthenia Gravis

Study ID

NCT06860633

Start date

Mar 6, 2025

Status verified date

Jul, 2025

Completion date

Dec 31, 2026

Anticipated

Primary completion date

Dec 31, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Adults ≥ age 18 years with known generalized MG as identified by characteristic signs of generalized MG on clinical assessment and positive serology for AchR antibodies as well as one of the following:

1. Documented positive response to cholinesterase inhibitors such as pyridostigmine or edrophonium
2. Abnormal decrement on slow repetitive nerve stimulation testing
3. Abnormal single fiber EMG
  • Evidence of worsening weakness requiring hospital admission for stabilization and change in therapy as determined by a neuromuscular expert including:

1. Quantitative Myasthenia Gravis (QMG) scale ≥ 11
2. MG-ADL score ≥ 6
3. Worsening weakness that is unlikely to be ameliorated by adjustment of current medications including impaired respiratory status, dysarthria, dysphagia, difficulty chewing, limb weakness, diplopia, ptosis.
  • Ability to sign consent and be enrolled within 24 hours of hospital admission. For participants transferred to University of Colorado Hospital, the time of admission/presentation to the outside hospital is counted towards this 24-hour cap.

Exclusion Criteria:

  • 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).
  • Other medical conditions that, in the opinion of the investigator and treating clinicians, might interfere with the validity of assessment measures used in the study (e.g. steroid myopathy, CNS pathology, severe arthritis, fractures, etc.). This criterion is a standard exclusion in MG trials and relates solely to other conditions that reduce muscle power or range of motion and would thus worsen scores on assessment measures like the QMG due to non-MG conditions.
  • Known history of coagulopathy, blood clotting, recent severe bleeding (e.g. GI bleed).
  • Pregnancy or breastfeeding. Pregnancy must be excluded for all potential participants who are able to become pregnant prior to initiation of treatment.
  • IgG levels < 600mg/dL
  • Evidence of active or chronic Hepatitis B infection, untreated Hepatitis C infection, HIV with low CD4 (<200) count.

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Efgartigimod

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

Interventions

Efgartigimod

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

Primary outcome measure

  • Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at day 11 [ Time Frame: Baseline, Day 11 ]

Central Contacts and Locations

Central contacts

Alyssa Avilez, BS

303.724.3522alyssa.avilez@cuanschutz.edu

Locations

University of Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Alyssa Avilez, BS

303-724-3522alyssa.avilez@cuanschutz.edu

Principal Investigator:

Thomas Ragole, MD

More Information

Sponsor

University of Colorado, Denver

Last update posted

Jul 11, 2025

Last verified

Jul, 2025

Keywords

  • myasthenia exacerbation
  • efgartigimod

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Colorado, Denver on 2025-07-11.