RECRUITING

Treatment of Myasthenia Gravis Exacerbation or Crisis With Efgartigimod

Conditions

Myasthenia Gravis Crisis Myasthenia Gravis Exacerbations AChR Myasthenia Gravis myasthenia exacerbation efgartigimod

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Treatment of Myasthenia Gravis Exacerbation or Crisis With Efgartigimod: A Single Arm, Open Label Prospective Cohort Study

Quick Facts

Study Start:2025-03-06

Study Completion:2026-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06860633

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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.

Inclusion Criteria

Exclusion 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.

* 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.

Contacts and Locations

Study Contact

Alyssa Avilez, BS

CONTACT

303.724.3522

alyssa.avilez@cuanschutz.edu

Principal Investigator

Thomas Ragole, MD

PRINCIPAL_INVESTIGATOR

University of Colorado, Denver

Study Locations (Sites)

University of Colorado

Aurora, Colorado, 80045

United States

Collaborators and Investigators

Sponsor: University of Colorado, Denver

Thomas Ragole, MD, PRINCIPAL_INVESTIGATOR, University of Colorado, Denver

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-03-06

Study Completion Date2026-12-31

Study Record Updates

Study Start Date2025-03-06

Study Completion Date2026-12-31

Terms related to this study

Keywords Provided by Researchers

myasthenia exacerbation
efgartigimod

Additional Relevant MeSH Terms

Myasthenia Gravis Crisis
Myasthenia Gravis Exacerbations
AChR Myasthenia Gravis