Generalized myasthenia gravis MedDRA version: 21.1 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
UCB Biopharma SRL
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 participant’s 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 II to IVa at Visit 1 - Study participant with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of at least 3 (with = 3 points from non-ocular symptom) 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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 216 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 24
Exclusion criteria
Exclusion criteria: - Study participant has a known history of hyperprolinemia - 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 with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI) will be excluded - 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 a Visit 1 or Visit 2 - Study participant has a history of a solid organ transplant or hematopoietic stem cell/marrow transplant
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Demonstrate the clinical efficacy of rozanolixizumab in patients with generalized myasthenia gravis (MG) ;Secondary Objective: Assess safety and tolerability of rozanolixizumab in myasthenia gravis (MG) patients ;Primary end point(s): Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score ;Timepoint(s) of evaluation of this end point: Baseline and Visit 10 (Day 43) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 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 score 3. Change from Baseline to (Day 43) Visit 10 in Quantitative Myasthenia Gravis (QMG) score to Visit 10 4. Change from Baseline to 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 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);Timepoint(s) of evaluation of this end point: 1. Visit 10 (Day 43) 2.- 6. Baseline and Visit 10 (Day 43) 7. and 8. From Baseline until End of Study Visit (up to Week 14) | — |
Countries
Belgium, Canada, Czechia, Czech Republic, Denmark, France, Germany, Hong Kong, Hungary, Italy, Poland, Russian Federation, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactClin Trial Reg & Results Disclosure
UCB BIOSCIENCES GmbH
Outcome results
None listed