Generalized Myasthenia Gravis
Conditions
Interventions
Gefurulimab(Integral combination product: biological product and device)
Sponsors
Terada Michinori
Eligibility
Inclusion criteria
Inclusion criteria: Participants who have a documented diagnosis of gMG over or equal to 3 months (90 days) prior to Screening based on clinical disease features and at least 1 of the following confirmatory tests. Test results may be collected from participant records or obtained during Screening: > Positive response in an acetylcholinesterase inhibitor test, for example, the edrophonium chloride test > Abnormal neuromuscular transmission demonstrated by repetitive nerve stimulation or single fiber electromyography > Previous improvement of symptoms or signs related to gMG during treatment with on oral acetylcholinesterase inhibitor, as confirmed by the treating physician Positive serological test for AChR autoantibodies (previous test results, if applicable, must be confirmed at Screening by central laboratory). MGFA Class II to IV at Screening and on Day 1. Must have QMG total score as outlined below: > Participants 12 to Participants 6 to < 12 years of age will have no minimum QMG total score required for inclusion; however, participants must have documented limb weakness in at least 1 limb
Exclusion criteria
Exclusion criteria: Clinical features that, in the opinion of the Investigator, are consistent with a Clinical Deterioration including myasthenic crisis, under or equal to 28 days prior to Screening or during the Screening Period. Any medical condition (eg, cardiac, pulmonary, renal, oncologic, neurological or psychiatric disorder) or risk factor that, in the opinion of the Investigator or the Medical Monitor, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of safety or efficacy of the study intervention. History of thymectomy or any other thymic surgery within 6 months prior to screening. Any untreated thymic malignancy, carcinoma, or thymoma.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To characterize the PK and PD of treatment with gefurulimab in pediatric participants with AChR+ gMG | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate safety of gefurulimab in pediatric participants with AChR+ gMG To assess the efficacy of gefurulimab in pediatric participants with AChR+ gMG To assess immunogenicity of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study | — |
Countries
Brazil, Japan, Poland, Taiwan, United States
Contacts
Public ContactMichinori Terada
Alexion Pharma GK
Outcome results
None listed