Treatment of Myasthenia Gravis Exacerbation or Crisis With Efgartigimod

NCT06860633 | Recruiting Phase 4 FDA Drug

• 1 other identifier: 24-0158
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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

Keywords

myasthenia exacerbation; efgartigimod

Outcome Measures

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.

  Baseline, Day 11

Secondary Outcomes (19)

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

  During hospitalization, approximately 7 days

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

  Baseline, Day 18

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

  Baseline, Day 32

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

  Baseline, Day 11

• Change from baseline in Myasthenia Gravis Manual Muscle Test (MG-MMT) at day 18

  Baseline, Day 18

Study Arms (1)

Efgartigimod

EXPERIMENTAL

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

Drug: Efgartigimod

Interventions

Efgartigimod DRUG

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

Also known as: Vyvgart

Efgartigimod

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Documented positive response to cholinesterase inhibitors such as pyridostigmine or edrophonium
• Abnormal decrement on slow repetitive nerve stimulation testing
• Abnormal single fiber EMG
• Evidence of worsening weakness requiring hospital admission for stabilization and change in therapy as determined by a neuromuscular expert including:
• Quantitative Myasthenia Gravis (QMG) scale ≥ 11
• MG-ADL score ≥ 6
• 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.

You may not qualify if:

• 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).
• 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.

Sponsors & Collaborators

• University of Colorado, Denver: lead
• argenx: collaborator

Study Sites (1)

University of Colorado

Aurora, Colorado, 80045, United States

RECRUITING

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MeSH Terms

Interventions

efgartigimod alfa

Study Officials

• Thomas Ragole, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alyssa Avilez, BS

CONTACT

303.724.3522; alyssa.avilez@cuanschutz.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 28, 2025
• First Posted: March 6, 2025
• Study Start: March 6, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: July 11, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: Beginning 9 months and ending 36 months following article publication
• Access Criteria: Researchers who provide a methodologically sound proposal. Proposals should be directed to NeurologyResearch@cuanschutz.edu. To gain access, data requestors will need to sign a data access agreement.

Locations

US (1)