Myasthenia GravisNCT07079020
Efgartigimod for Consolidation Therapy in gMG
An observational study of Myasthenia Gravis, run by Da, Yuwei, M.D..
Trial summary
The goal of this observational study is to evaluate the efficacy and safety of consolidation therapy with corticosteroids and/or immunosuppressants combined with efgartigimod in patients with generalized myasthenia gravis after the acute exacerbation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- How many people
- 130 participants
- Where
- No sites listed yet
- Study dates
- 30 Jul 2025to 30 Sep 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: AChR antibody-positive adults with generalized myasthenia gravis who responded to acute exacerbation treatment and underwent consolidation therapy
Who may be able to join
- Age ≥18 years, male or female.
- Diagnosis of myasthenia gravis (MG) based on the following:
- Typical clinical features of MG.
- At least one of the following:
- Seropositive for anti-AChR antibodies (confirmed by reliable assay). Abnormal neuromuscular transmission on electrophysiological testing (e.g., RNS or SFEMG).
- Positive neostigmine test or documented response to cholinesterase inhibitors.
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- MGFA classification II-IV at consolidation baseline.
- Seropositive for anti-AChR antibodies (required for enrollment).
- Hospitalized for myasthenic crisis (MC) in the acute exacerbation and completed one treatment cycle of efgartigimod: acute exacerbation definition: Presence of MC warning signs (new or worsening within 2 weeks): Bulbar symptoms: Dysphagia, choking, weak cough, dysarthria. Respiratory symptoms: Dyspnea, respiratory muscle weakness. Generalized weakness: Head drop, jaw weakness, facial weakness. One treatment cycle of efgartigimod: 10 mg/kg IV, once weekly for 4 doses.
- ≥2-point improvement in MG-ADL score from acute exacerbation baseline by Day 4 of efgartigimod treatment.
- Willing and able to provide written informed consent before study participation.
Who cannot join
- Treatment with IVIg, plasma exchange (PE), IV methylprednisolone (IVMP), or immunoadsorption (IA) within 1 month before consolidation baseline.
- Thymectomy within 6 months before exacerbation baseline.
- Use of immunomodulatory monoclonal antibodies or investigational drugs (not listed above) within 3 months before baseline or 5 half-lives (whichever is longer).
- Pregnancy or lactation (women of childbearing potential must have negative pregnancy test and use contraception).
- Known hypersensitivity to efgartigimod or any FcRn-targeting therapy.
- Clinically significant comorbidities, including severe cardiovascular, hepatic, renal, pulmonary, or hematologic disorders, active malignancy and uncontrolled systemic infections.
- Other autoimmune diseases that may interfere with efficacy assessment (e.g., uncontrolled thyroid disease, severe rheumatoid arthritis).
- Any other condition deemed unsuitable by the investigator.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Multicenter Cohort Study on Efgartigimod for Consolidation Therapy After the Acute Exacerbation of Generalized Myasthenia Gravis in Adults
The goal of this observational study is to evaluate the efficacy and safety of consolidation therapy with corticosteroids and/or immunosuppressants combined with efgartigimod in patients with generalized myasthenia gravis after the acute exacerbation.
- Conditions
- Myasthenia Gravis
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod+SOC | Initiate efgartigimod combined with standard of care (SOC) therapy according to the protocol within one month after consolidation baseline. | |
| SOC | No additional treatments are to be administered other than the standard of care (SOC) therapy. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients achieving and maintaining minimal symptom expression (MSE) for at least 4 months after first attaining MSE status at any time during the consolidation therapy period within 2 months.
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered achieving MSE if there was a 1 or 0 on the MG-ADL total score ≥4 consecutive months after first attaining MSE status at any time during the consolidation therapy period within 2 months.
Measured: Consolidation baseline up to Month 6
Secondary outcomes (8)
Proportion of patients achieving both minimal symptom expression (MSE) and a daily corticosteroid dose ≤5 mg at Month 12.
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered achieving MSE if there was a 1 or 0 on the MG-ADL total score.
Measured: Consolidation baseline up to Month 12
Proportion of patients with MGFA Post-Intervention Status (MGFA-PIS) at Month 6 and Month 12
Measured: Consolidation baseline up to Month 6 AND Month 12
Change from baseline in MG-ADL scores at Month 6 and Month 12
The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity.
Measured: Consolidation baseline up to Month 6 AND Month 12
Change from baseline in QMG scores at Month 6 and Month 12
The Quantitative Myasthenia Gravis (QMG) scale quantifies disease severity based on impairments of body functions and structures as defined by the International Classification of Disability and Health. The QMG scale consists of 13 items that measure endurance or fatigability, and accounts for fluctuations in disease state. The QMG total score range is 0-39, with higher scores indicative of greater disease severity.
Measured: Consolidation baseline up to Month 6 AND Month 12
Proportion of patients achieving minimal symptom expression (MSE) at Month 6
Measured: Consolidation baseline up to Month 6
Proportion of patients achieving minimal symptom expression (MSE) with daily corticosteroid dose ≤5 mg at Month 6
Measured: Consolidation baseline up to Month 6
Incidence of acute exacerbations, impending myasthenic crisis, or myasthenic crisis events during the study period
Measured: Consolidation baseline up to Month 12
Types and frequencies of adverse events (AEs) and serious adverse events (SAEs) during the study period
Measured: Consolidation baseline up to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2025
- Study start30 Jul 2025 (estimated)
- Primary completion30 Jun 2026 (estimated)
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 130 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Da, Yuwei, M.D.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07079020
- Sponsor's protocol ID
- LYS[2025]190-002
- Status last verified
- Jun 2025
- First submitted
- 29 Jun 2025
- Last updated
- 22 Jul 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Cohort Study on Efgartigimod for Consolidation Therapy After the Acute Exacerbation of Generalized Myasthenia Gravis in Adults
- ClinicalTrials.gov ID
- NCT07079020
- Acronym / sponsor ID
- LYS[2025]190-002
- Conditions
- Myasthenia Gravis
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 130 participants (estimated)
- Lead sponsor
- Da, Yuwei, M.D.
- Study start
- 30 Jul 2025 (estimated)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 22 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this observational study is to evaluate the efficacy and safety of consolidation therapy with corticosteroids and/or immunosuppressants combined with efgartigimod in patients with generalized myasthenia gravis after the acute exacerbation.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod+SOC | – | –Initiate efgartigimod combined with standard of care (SOC) therapy according to the protocol within one month after consolidation baseline. |
| SOC | – | –No additional treatments are to be administered other than the standard of care (SOC) therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- AChR antibody-positive adults with generalized myasthenia gravis who responded to acute exacerbation treatment and underwent consolidation therapy
Key inclusion criteria (11 listed)
- Age ≥18 years, male or female.
- Diagnosis of myasthenia gravis (MG) based on the following:
- Seropositive for anti-AChR antibodies (confirmed by reliable assay). Abnormal neuromuscular transmission on electrophysiological testing (e.g., RNS or SFEMG).
- Positive neostigmine test or documented response to cholinesterase inhibitors.
- MGFA classification II-IV at consolidation baseline.
Key exclusion criteria (8 listed)
- Treatment with IVIg, plasma exchange (PE), IV methylprednisolone (IVMP), or immunoadsorption (IA) within 1 month before consolidation baseline.
- Thymectomy within 6 months before exacerbation baseline.
- Use of immunomodulatory monoclonal antibodies or investigational drugs (not listed above) within 3 months before baseline or 5 half-lives (whichever is longer).
- Pregnancy or lactation (women of childbearing potential must have negative pregnancy test and use contraception).
- Known hypersensitivity to efgartigimod or any FcRn-targeting therapy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Proportion of patients achieving and maintaining minimal symptom expression (MSE) for at least 4 months after first attaining MSE status at any time during the consolidation therapy period within 2 months.
Time frame: Consolidation baseline up to Month 6
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered achieving MSE if there was a 1 or 0 on the MG-ADL total score ≥4 consecutive months after first attaining MSE status at any time during the consolidation therapy period within 2 months.
8 secondary outcomes
Proportion of patients achieving both minimal symptom expression (MSE) and a daily corticosteroid dose ≤5 mg at Month 12.
Time frame: Consolidation baseline up to Month 12
Proportion of patients with MGFA Post-Intervention Status (MGFA-PIS) at Month 6 and Month 12
Time frame: Consolidation baseline up to Month 6 AND Month 12
Change from baseline in MG-ADL scores at Month 6 and Month 12
Time frame: Consolidation baseline up to Month 6 AND Month 12
Change from baseline in QMG scores at Month 6 and Month 12
Time frame: Consolidation baseline up to Month 6 AND Month 12
Proportion of patients achieving minimal symptom expression (MSE) at Month 6
Time frame: Consolidation baseline up to Month 6
Proportion of patients achieving minimal symptom expression (MSE) with daily corticosteroid dose ≤5 mg at Month 6
Time frame: Consolidation baseline up to Month 6
Incidence of acute exacerbations, impending myasthenic crisis, or myasthenic crisis events during the study period
Time frame: Consolidation baseline up to Month 12
Types and frequencies of adverse events (AEs) and serious adverse events (SAEs) during the study period
Time frame: Consolidation baseline up to Month 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
An observational study of Myasthenia Gravis, run by Da, Yuwei, M.D..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 22 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yu wei Da, M.D. (00-86-010-83198493, dayuwei100@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of patients achieving and maintaining minimal symptom expression (MSE) for at least 4 months after first attaining MSE status at any time during the consolidation therapy period within 2 months. (time frame: Consolidation baseline up to Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2025.