Generalized Myasthenia Gravis
Conditions
Keywords
generalized Myasthenia Gravis, gMG, RA101495, Zilucoplan, pediatric, MG0015
Brief summary
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
Interventions
DRUGZilucoplan
Zilucoplan will be administered subcutaneously to pediatric study participants
Sponsors
UCB Biopharma SRL
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
United States of America (USA) specific inclusion criterion: \- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation. Rest of World (ROW) specific inclusion criterion: \- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation. Global specific inclusion criteria: * Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion * Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care
Exclusion criteria
* Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP * Participant has known positive serology for muscle-specific kinase * Participant has known hypersensitivity to any components of the IMP * Participant has a prior history of meningococcal disease
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Occurence of treatment emergent adverse events during the course of the study | Baseline (Day 1) to Safety Follow-up (up to Week 60) | 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. |
| Occurence of treatment-emergent serious adverse events (TESAEs) | Baseline (Day 1) to Safety Follow-up (up to Week 60) | 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. |
| Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product | Baseline (Day 1) to Safety Follow-up (up to Week 60) | 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. |
| Occurence of treatment-emergent infections | Baseline (Day 1) to Safety Follow-up (up to Week 60) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma concentration of Zilucoplan at Week 52 | Week 52 | Blood samples for the measurement of plasma concentrations of Zilucoplan will be collected at Week 52. |
| Sheep red blood cell (sRBC) lysis activity at Week 52 | Week 52 | Blood samples for measurement of sRBC lysis will be collected at Week 52. |
| Blood complement component 5 (C5) levels at Week 52 | Week 52 | Blood samples for measurement of C5 will be collected at Week 52. |
| Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52 | 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. |
| Quantitative Myasthenia Gravis (QMG) score at Week 52 | 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. |
Countries
Italy, Poland, South Korea, United Kingdom
Contacts
STUDY_DIRECTORUCB Cares
001 844 599 2273 (UCB)
Outcome results
None listed