The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Zilucoplan will be administered subcutaneously to pediatric study participants
Mg0015 50168
Chicago, Illinois, United States
Mg0015 40144
Milan, Italy
Mg0015 40774
Katowice, Poland
Mg0015 40218
Warsaw, Poland
Occurence of treatment emergent adverse events during the course of the study
An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant 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: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Occurence 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 Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.
Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product
An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation 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: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Occurence 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: Baseline (Day 1) to Safety Follow-up (up to Week 60)
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Mg0015 20104
Seoul, South Korea
Mg0015 20220
Seoul, South Korea
Mg0015 40735
Glasgow, United Kingdom
Mg0015 40736
London, United Kingdom
Plasma concentration of Zilucoplan at Week 52
Blood samples for the measurement of plasma concentrations of Zilucoplan will be collected at Week 52.
Time frame: Week 52
Sheep red blood cell (sRBC) lysis activity at Week 52
Blood samples for measurement of sRBC lysis will be collected at Week 52.
Time frame: Week 52
Blood complement component 5 (C5) levels at Week 52
Blood samples for measurement of C5 will be collected at Week 52.
Time frame: Week 52
Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52
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 52
Quantitative Myasthenia Gravis (QMG) score at Week 52
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 52