Efgartigimod for Consolidation Therapy in gMG

NCT07079020 | Not Yet Recruiting

• 1 other identifier: LYS[2025]190-002
• Study Type: observational
• Target: 130
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• 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.

  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.

  Consolidation baseline up to Month 12

• Proportion of patients with MGFA Post-Intervention Status (MGFA-PIS) at Month 6 and Month 12

  Consolidation baseline up to Month 6 AND Month 12

• Change from baseline in MG-ADL scores at Month 6 and Month 12

  Consolidation baseline up to Month 6 AND Month 12

• Change from baseline in QMG scores at Month 6 and Month 12

  Consolidation baseline up to Month 6 AND Month 12

• Proportion of patients achieving minimal symptom expression (MSE) at Month 6

  Consolidation baseline up to Month 6

Study Arms (2)

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.

Eligibility Criteria

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

Study Population

AChR antibody-positive adults with generalized myasthenia gravis who responded to acute exacerbation treatment and underwent consolidation therapy

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Da, Yuwei, M.D.: lead

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Yu wei Da, M.D.

CONTACT

00-86-010-83198493; dayuwei100@hotmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDIV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2025
• First Posted: July 22, 2025
• Study Start: July 30, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: July 22, 2025

Record last verified: 2025-06