The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening
Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening
An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to <18 years of age at Screening
Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to <12 years of age at Screening (does not apply to US)
Participant has known positive serology for muscle-specific kinase
Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study
Participant has had a thymectomy within 6 months prior to Baseline
Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator
Single group
Zilucoplan
DrugZilucoplan will be administered subcutaneously to pediatric study participants.
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
Samples for measurement of C5 will be collected on Day 29 predose.
Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Occurrence of treatment-emergent serious adverse events (TESAEs)
A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death Is life-threatening Requires inpatient hospitalization or prolongation of existing hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical events
Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.
Occurrence of treatment-emergent infections
Percentage of participants who experienced treatment-emergent infections as adverse events. An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment.
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