Generalized myasthenia gravis
Conditions
Sponsors
UCB Japan Co., Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Study participant must be >=18 years of age, at the time of signing the informed consent - Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at Visit 1, based on study participants history and supported by previous evaluations - Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) at Screening (Visit 1). The presence of autoantibodies may be confirmed with repeat testing at Visit 1 - Study participant has Myasthenia Gravis Foundation of America (MGFA) Class ll to IVa at Visit 1 - Study participant with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of at least 3 AND a quantitative myasthenia gravis (QMG) score of at least 11 at Visit 1 and at Baseline (Visit 2) - Study participant is considered for additional treatment such as intravenous immunoglobulin g(IVIg) or plasma exchange (PEX) by the Investigator
Exclusion criteria
Exclusion criteria: - Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP) - Study participant has experienced hypersensitivity reaction after exposure to other antineonatal Fc receptor (FcRn) drugs - Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Visit 1 or Visit 2
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score | — |
Secondary
| Measure | Time frame |
|---|---|
| safety efficacy 1. Percentage of participants achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) response at Visit 10 2. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis-Composite (MG-C) score 3. Change from Baseline to (Day 43) Visit 10 in Quantitative Myasthenia Gravis (QMG) score 4. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) [Muscle Weakness Fatigability] score 5. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) [Physical Fatigue] score 6. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) [Bulbar Symptoms] score 7. Occurrence of treatment-emergent adverse events (TEAEs) 8. Treatment-emergent adverse events (TEAEs) leading to withdrawal of investigational medicinal product (IMP) | — |
Countries
Asia except Japan, Europe, Japan, North America
Outcome results
None listed