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Evaluating ravulizumab for pediatric patients with generalized myasthenia gravis

A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG)

Phase 3 Interventional Alexion Pharmaceuticals, Inc. · NCT05644561

This study is testing a new drug called ravulizumab to see if it can help children with generalized myasthenia gravis feel better and stay safe while using it.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment12 (estimated)
SexAll
SponsorAlexion Pharmaceuticals, Inc. Industry-sponsored
Drugs / interventionsEculizumab, ravulizumab
Locations20 sites (Los Angeles, California and 19 other locations)
Trial IDNCT05644561 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the pharmacokinetics and pharmacodynamics of ravulizumab administered intravenously to pediatric participants diagnosed with generalized myasthenia gravis (gMG). The study will involve participants who have a confirmed diagnosis of gMG and are classified as Class II to Class IV according to the Myasthenia Gravis Foundation of America. Participants must be on a stable treatment regimen prior to screening and have received prior treatment with eculizumab if applicable. The trial will monitor the efficacy, safety, and immunogenicity of the treatment over the course of the study.

Who should consider this trial

Good fit: Ideal candidates include pediatric patients diagnosed with gMG who meet specific clinical classification criteria and have been on stable treatment regimens.

Not a fit: Patients with untreated thymic malignancies or those who have undergone recent thymic surgery may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of generalized myasthenia gravis in pediatric patients.

How similar studies have performed: Other studies involving similar treatments for gMG have shown promise, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period
* Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening
* Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.
* Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.
* All participants must be vaccinated against meningococcal infection

Exclusion Criteria:

Medical Conditions

* Any untreated thymic malignancy, carcinoma, or thymoma.
* Participants with a history of treated benign thymoma
* History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening
* History of N meningitidis infection
* Known to be human immunodeficiency virus (HIV) positive
* History of unexplained infections
* Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period

Where this trial is running

Los Angeles, California and 19 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Generalized Myasthenia GravisgMG
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.