Evaluation of the Condition of Patients Receiving EARLY Ravulizumab and Admitted in ICU for gMG Crisis (EARLY-MG)

Evaluation of the Condition of Patients Receiving EARLY Treatment With Ravulizumab and Admitted in ICU for Generalized Myasthenia Gravis (gMG) Crisis : an Observational Prospective Multicentric Study in France

Myasthenia Gravis (MG) is a rare autoimmune disease that causes muscle weakness and fatigue. It occurs when the immune system produces antibodies that block communication between nerves and muscles. In some patients, the disease can suddenly worsen and cause severe breathing problems. This life-threatening situation is called a myasthenic crisis and requires immediate treatment in an intensive care unit (ICU). During such crises, patients may need to receive respiratory assistance through a ventilator. These episodes are often long and can lead to complications such as infections or heart problems.

To manage a myasthenic crisis, doctors usually use treatments that remove or neutralize the harmful antibodies: plasma exchange (PLEX) or intravenous immunoglobulin (IVIg). Although both are effective, recovery can be slow, and many patients remain in the ICU for several weeks.

Ravulizumab (Ultomiris®) is a new medicine that targets a specific part of the immune system called the complement system, which contributes to muscle damage in MG. It is already approved for adults with generalized MG who have anti-acetylcholine receptor (AChR) antibodies. Ravulizumab is given by intravenous infusion every eight weeks. Clinical studies have shown that it can improve symptoms within one week of starting treatment.

Some doctors have started using ravulizumab early, after PLEX or IVIg, for patients hospitalized in the ICU for a myasthenic crisis. Early use of this treatment could help reduce the duration and severity of the crisis, leading to faster recovery and shorter hospital stays. However, there is currently no national study that systematically collects data on this approach.

The EARLY-MG study aims to describe the condition and recovery of patients who receive ravulizumab early during a myasthenic crisis requiring ICU admission. The study will not test an experimental treatment or change medical care. It is an observational study.

The main hypothesis of the study is that early administration of ravulizumab, after PLEX or IVIg, may help patients recover faster, improve muscle strength, and reduce complications and hospital stay.

Around 30 adult patients with generalized MG and anti-AChR antibodies will be enrolled in 10 centers across France.

Each patient will be followed for 26 weeks (about six months). Assessments will be performed at the start of the study and at weeks 2, 4, 10, 18, and 26. Investigators will collect information such as:

• Duration of stay in the ICU and in the hospital after receiving ravulizumab
• Duration of mechanical ventilation, if needed
• Clinical improvement using standard evaluation scales (Myasthenia Gravis Activities of Daily Living, MG Foundation of America classification, and Garches' score)
• Occurrence of any complications or additional treatments The study will last about 18 months in total, including one year for patient inclusion and six months of follow-up per patient. The results may help guide future recommendations and improve patient care in France and worldwide.

Study Overview

• Status: Recruiting
• Conditions: Myasthenia Gravis
• Intervention / Treatment: Other: early use of Ravulizumab

• Study Type: Observational
• Enrollment (Estimated): 30

Contacts and Locations

• Study Contact: Name: Sabrina SACCONI; Phone Number: 0492035757; Email: sacconi.s@chu-nice.fr

Study Locations

• France
  • Alpes-maritimes
    • Nice, Alpes-maritimes, France, 06000
      • Recruiting
      • CHU de Nice
      • Contact: Sabrina SACCONI; Phone Number: 0492035757; Email: sacconi.s@chu-nice.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with myasthenia gravis

Description

Inclusion Criteria:

• Male or female aged ≥18 years.
• Diagnosed with gMG with confirmed documentation and supported by a physical exam and confirmed seropositivity for AChR-Abs.
• Patients having received more than 1 cycle of PLEX or IVIg 1-2 g/kg (max 50g/day), according to clinical practice.
• Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MFGA) for generalized MG class V - IV, at ICU admission.
• Patients treated with at least one standard MG-targeted therapy.
• Patients receiving Ravulizumab following PLEX of IVIg during their stay in the ICU, as per local label and reimbursement conditions. The prescription must be validated by experts from the French Health Care Network for rare neuromuscular diseases FILNEMUS.
• Patients capable of understanding written informed consent and providing signed, dated, and witnessed written informed consent. If unable to sign the consent due to muscular weakness, a dedicated informed consent can be signed by a trusted witness.
• Patients willing and able to comply with scheduled visits, treatment plan, study restrictions and other study procedures.
• Patients affiliated to a European social security system.
• Patients agree to comply with the prevention of meningococcal infections by vaccination and/or antibiotic prophylaxis in accordance with the current local vaccination/antibiotic prophylaxis recommendations.
• Patients with no contraindication to anti-C5 treatment.
• Patients with no contraindication to antibiotic therapy.

Exclusion Criteria:

• Active infection or other disorders causing weakness, known immunoglobulin A deficiency, active renal or hepatic disease, clinically significant cardiac disease, known hyperviscosity, or hypercoagulable state.
• Any active malignancy.
• Presence of antibodies other than anti-AChR-Ab+ (anti-titin-Ab+ were permitted as these are considered complementary markers to anti-AChR-Ab+).
• Patient with a diagnosed thymoma.
• Patient with a pathology judged by the investigator to interfere with the proper conduct of the study.
• Positive pregnancy test. A urine pregnancy test will be carried out for women of childbearing age.
• Any current mental condition (psychiatric disorder, senility, or dementia) that, in the opinion of the investigator, may affect study compliance or prevent understanding of the aims, investigational procedures, or possible consequences of the study.
• Vaccination with live or live-attenuated vaccines within the 6 weeks.
• Refusal of the subject to participate in the study.
• Patient protected by law, under guardianship or curator ship, or not able to participate in a clinical study according to the article L.1121-16 of the French Public Health Code.
• Known hypersensitivity to the active substance or to any of the excipients.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Short-term change in clinical outcomes	The change from the inclusion to week 2, week 4, and week 10 in the Myasthenia Gravis Activities of Daily Living scale (score from 0 - normal to 3 - worse outcome)	Week 0, 2, 4 and 10
Short-term change in clinical outcomes	The change from the inclusion to week 2, week 4, and week 10 in the Myasthenia Gravis Foundation of America (from class I to class V - worse outcome)	Week 0, 2, 4 and 10
Short-term change in clinical outcomes	The change from the inclusion to week 2, week 4, and week 10 in the Garches' score (from 0 to 100 with lower score indicating a greater disease severity)	Week 0, 2, 4 and 10

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long term change in clinical outcomes 1	change from the inclusion at week 18 and week 26 in the Myasthenia Gravis Activities of Daily Living scale (score from 0 - normal to 3 - worse outcome)	Week 18 and 26
Long term change in clinical outcomes 2	change from the inclusion at week 18 and week 26 in the Myasthenia Gravis Foundation of America scale (from class I to class V - worse outcome)	Week 18 and 26
Long term change in clinical outcomes 3	change from the inclusion at week 18 and week 26 in the Garches' score (from 0 to 100 with lower score indicating a greater disease severity)	Week 18 and 26
Time to observe a clinical improvement	time in days to reach the Myasthenia Gravis Foundation of America III category on the Myasthenia Gravis Foundation of America scale (from class I to class V - worse outcome)	From the day the patient begin the study to the first day the patient reach the Myasthenia Gravis Foundation of America III category - in days, up to 180 days
Time in days from Ravulizumab administration to the day of ICU discharge	Time in days from Ravulizumab administration to the day of ICU discharge	From the day the patient begin the Ravulizumab administration to the day the patient leaves ICU - in days, up to 180 days
Total Length Of Stay at the hospital after Ravulizumab administration	The time in days from Ravulizumab administration to the day of hospital discharge	From the day the patient begin the Ravulizumab administration to the day the patient leaves the hospital - in days, up to 180
Duration of ventilation after ravulizumab administration	if applicable, measured in days, calculated from the beginning to the end of the ventilation.	From the day the patient begin the Ravulizumab administration to the day the patient leaves ICU - in days, up to 180 days
Patient's condition	Evaluated by the type of medical complications.	Ongoing from ICF signing to the end of the study
Patient's condition	Evaluated by the severity of medical complications.	Ongoing from ICF signing to the end of the study
Patient's condition	Evaluated by the frequency of medical complications.	Ongoing from ICF signing to the end of the study
Patient's condition	Evaluated by the seriousness of medical complications.	Ongoing from ICF signing to the end of the study

Collaborators and Investigators

• Sponsor: Centre Hospitalier Universitaire de Nice

Study record dates

Study Major Dates

• Study Start (Actual): November 26, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: December 4, 2025
• First Submitted That Met QC Criteria: February 9, 2026
• First Posted (Actual): February 17, 2026

Study Record Updates

• Last Update Posted (Actual): February 17, 2026
• Last Update Submitted That Met QC Criteria: February 9, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis
• Other Study ID Numbers: 25-PP-09

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No