Generalized Myasthenia Gravis
Conditions
Keywords
Generalized Myasthenia Gravis, gMG, rozanolixizumab, pediatric
Brief summary
The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006.
Interventions
DRUGrozanolixizumab
rozanolixizumab solution for injection
Sponsors
UCB Biopharma SRL
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Study participant must meet one of the following: * Study participant completed MG0006 according to the protocol * Study participant completed the MG0006 Treatment Period and has a worsening of generalized myasthenia gravis (gMG) symptoms in the Observation Period of MG0006
Exclusion criteria
* Study participant met any mandatory withdrawal or mandatory permanent investigational medicinal product (IMP) discontinuation criteria in MG0006 or permanently discontinued IMP * Study participant has a known hypersensitivity to any components of the IMP or other neonatal Fc receptor (FcRn) drugs * Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant's ability to participate in this study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit | From Baseline up to the EOS Visit (up to 52 weeks) | Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in patient hospitalisation or prolongation of existing hospitalisation * Results in persistent disability/incapacity * Is a congenital anomaly or birth defect * Important medical events |
| Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit | From Baseline up to the EOS Visit (up to 52 weeks) | 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 Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit | From Baseline up to the EOS Visit (up to 52 weeks) | AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP) | From Baseline to the end of Week 6 of each TP (up to 52 weeks) | Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP. |
| Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP | From Baseline to the end of Week 6 of each TP (up to 52 weeks) | 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. |
| Absolute change in total IgG from Baseline to the end of Week 6 of each TP | From Baseline to the end of Week 6 of each TP (up to 52 weeks) | Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP. |
| Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP | From Baseline to the end of Week 6 of each TP (up to 52 weeks) | 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. |
Countries
Italy, Japan, Poland, Taiwan, Turkey (Türkiye)
Contacts
STUDY_DIRECTORUCB Cares
001 844 599 2273
Outcome results
None listed