An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 years of age) with Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+) (PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients with AChR+ Generalized Myasthenia Gravis)

未提供0 个研究点目标入组 1 人开始时间: 待定

适应症

试验速览

暂无简介。

•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
•Must have QMG total score as outlined below:
•> Participants 12 to < 18 years of age must have QMG total score over or equal to 12 at Screening and on Day 1
•> 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

•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.

发起方

未提供