Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

Launched by ALEXION PHARMACEUTICALS, INC. · Dec 1, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a treatment called ravulizumab for children with a condition called Generalized Myasthenia Gravis (gMG). gMG is a disease that affects communication between nerves and muscles, leading to weakness in different parts of the body. The trial aims to understand how the medication works in children, how it affects their bodies, and how safe it is. Researchers are looking for participants who have been diagnosed with gMG and have specific test results showing they have certain antibodies. Eligible participants should be between certain ages, have a stable treatment plan, and be vaccinated against meningococcal infection.

If your child joins the trial, they will receive intravenous (IV) infusions of ravulizumab and will be closely monitored by the medical team throughout the study. This means that healthcare professionals will check on their health and how well the treatment is working. It’s important to know that participants should not have certain medical conditions, such as untreated thymic tumors or a history of specific infections. This study is currently recruiting participants, and it could provide valuable information about treating gMG in children.

Gender

ALL

Eligibility 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