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).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Zilucoplan will be administered subcutaneously to pediatric study participants.
Mg0014 50168
Chicago, Illinois, United States
WITHDRAWNMg0014 50574
Denton, Texas, United States
WITHDRAWNMg0014 40144
Milan, Italy
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.
Time frame: Week 4 (Day 29)
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.
Time frame: Week 4 (Day 29)
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.
Time frame: Week 4 (Day 29)
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.
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
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Mg0014 40774
Katowice, Poland
RECRUITINGMg0014 40218
Warsaw, Poland
RECRUITINGMg0014 20104
Seoul, South Korea
RECRUITINGMg0014 20220
Seoul, South Korea
RECRUITINGMg0014 40735
Glasgow, United Kingdom
RECRUITINGMg0014 40736
London, United Kingdom
RECRUITINGAn 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.
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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.
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)
ADA and anti-PEG antibodies will be evaluated in serum samples.
Time frame: Week 4 (Day 29)
Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability.
Time frame: Week 4 (Day 29)
Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)
QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.
Time frame: Week 4 (Day 29)
Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)
The MGFA-PIS is a physician-determined assessment of clinical symptoms of MG after initiation of MG specific therapy. For the purpose of the current study, Minimal Manifestation will be determined at each scheduled time point after treatment initiation (rather than after 1 year). Change in status (improved, unchanged, worse, exacerbation, or died of MG) will also be determined.
Time frame: Week 4 (Day 29)
Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)
The PedsQoL generic core scale (Version 4) is a validated instrument that is suitable for use with pediatric populations. PedsQoL generic core scales assess Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. The scale has 23 items with a score range of 0 to 4. Following transformation, the score range of each domain as well as the total score is 0-100 with higher scores indicating higher HRQoL.
Time frame: Week 4 (Day 29)