Early ravulizumab for adults with myasthenic crisis in the ICU

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

This project will test whether giving ravulizumab early to adults with AChR-positive generalized myasthenia gravis who are in the ICU for a myasthenic crisis helps speed recovery.

Quick facts

• Study type: Observational
• Enrollment: 30 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Centre Hospitalier Universitaire de Nice (Academic / other)
• Drugs / interventions: Ravulizumab
• Locations: 1 site (Nice, Alpes-maritimes)
• Trial ID: NCT07411963 on ClinicalTrials.gov

What this trial studies

This is an observational program of adults with AChR-antibody–positive generalized myasthenia gravis admitted to the intensive care unit for a myasthenic crisis who receive ravulizumab following plasma exchange or IVIg according to local practice. Clinical data during the ICU stay—such as duration of mechanical ventilation, length of ICU stay, complications, and clinical recovery measures—are collected. Treatment decisions follow local labeling and validation by the French FILNEMUS network, with the protocol documenting early use of the long-acting complement C5 inhibitor ravulizumab. The design is non-randomized and observational, aiming to describe outcomes and safety in this specific, high-acuity setting.

Who should consider this trial

Good fit: Adults (≥18 years) with generalized AChR-antibody–positive myasthenia gravis admitted to the ICU for myasthenic crisis who have received PLEX or IVIg and are prescribed ravulizumab under local FILNEMUS guidance are eligible.

Not a fit: Patients who are AChR-seronegative, not in ICU for a myasthenic crisis, or who do not receive ravulizumab as part of their care are unlikely to benefit from this observational program.

Why it matters

Potential benefit: If successful, early ravulizumab could shorten time on ventilators and reduce ICU length of stay for patients in myasthenic crisis.

How similar studies have performed: Complement inhibitors such as eculizumab and ravulizumab have shown benefit in AChR-positive gMG, but evidence for early use specifically during ICU-managed myasthenic crisis is limited.

Eligibility criteria

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.

Where this trial is running

Nice, Alpes-maritimes

• CHU de NICE — Nice, Alpes-maritimes, France (Recruiting)

Study contacts

• Study coordinator: Sabrina SACCONI

Conditions Myasthenia Gravis