Myasthenia Gravis
Conditions
Keywords
Rozanolixizumabg, gMG
Brief summary
The purpose of the study is to assess the clinical efficacy of rozanolixizumab in adult Chinese participants with generalized myasthenia gravis (gMG) in the first Treatment Cycle.
Interventions
DRUGRozanolixizumab
Subcutaneous infusion
Sponsors
UCB Biopharma SRL
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Study participant must be ≥18 years of age at the time of signing the informed consent form (ICF) * Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at the Screening Visit based on study participant's history and supported by previous evaluation * Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) documented in the medical history at the Screening Visit * Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening Visit. * Study participant with a myasthenia gravis-activities of daily living (MG-ADL) score of at least 3 points from non-ocular symptoms and a quantitative myasthenia gravis (QMG) score of at least 11 at the Screening and Baseline visits in the first Treatment Cycle. * Study participant is considered for additional treatment by the investigator * Body weight ≥35kg at the Screening Visit
Exclusion criteria
* Study participant has a known hypersensitivity to any components of the study drug or any other anti-neonatal Fc receptor (anti-FcRn) medications * Study participant has a clinically important active infection including unresolved or not adequately treated infection in the opinion of the investigator * 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) * Study participant has previously received rozanolixizumab drug product * Study participant has received any vaccine in the 4 weeks prior to the initiation of rozanolixizumab treatment or intends to receive any vaccine within 2 weeks after the last infusion of rozanolixizumab. Note: For participants that are on rozanolixizumab treatment, vaccination with live or live-attenuated vaccines is not recommended. During the whole study period, all other vaccines should take place at least 2 weeks after the last infusion of a Treatment Cycle and 4 weeks before initiating the next cycle * Study participant has been treated with prohibited immunosuppressants, biologics, and other therapies within the timeframe shorter than the treatment-free period * Study participant with severe (defined as Grade 3 on the MG-ADL scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis. Note: Impending Crisis is defined as bulbar or respiratory symptoms of a patient who significantly worsens in a short time (≤2 weeks) and meet MGFA IVb or score 3 in one QMG bulbar muscle item, or score 2 in respiratory muscle item, or bulbar+respiratory items score ≥4
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score in the first Treatment Cycle | Baseline to Day 43 (in first treatment cycle) | The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| MG-ADL responder (≥2.0-point improvement from Baseline) at Day 43 in the first Treatment Cycle | Baseline to Day 43 (in first treatment cycle) | A MG-ADL responder is defined as achieving at least 2.0-point improvement in the MG-ADL total score from Baseline. |
| Change from Baseline to Day 43 in Myasthenia Gravis-Composite (MG-C) score within first 6 week treatment cycle | Baseline to Day 43 (in first treatment cycle) | The MG-C scale is a validated assessment, with a higher score indicating more severe disease and a 3-point change being of clinical relevance. The scale tests 10 items, with individual items being weighted differently. The overall score ranges from 0 to 50 (more severe disease). The items included ptosis/upward gaze (range: 0 \[\>45 second\] - 3 \[Immediate\]), double vision on lateral gaze (range: 0 \[\>45 second\] - 4 \[Immediate\]), eye closure (range: 0 \[Normal\] - 2 \[severe weakness\]), talking (range: 0 \[Normal\] - 6 \[difficult to understand speech\]), chewing (range: 0 \[Normal\] - 6 \[gastric tube\]), swallowing (range: 0 \[Normal\] - 6 \[gastric tube\]), breathing (range: 0 \[Normal\] - 9 \[ventilator dependence\]), neck flexion (range: 0 \[Normal\] - 4 \[severe weakness\]), shoulder abduction (range: 0 \[Normal\] - 5 \[severe weakness\]) and hip flexion (range: 0 \[Normal\] - 5 \[severe weakness\]), lower scores= lower disease activity. |
| Change from Baseline to Day 43 in Quantitative Myasthenia Gravis (QMG) score within first 6-week treatment cycle | Baseline to Day 43 (in first treatment cycle) | The total QMG score is obtained by summing the responses to each individual item (13 items \[ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity\]; Responses: None=0, Mild=1, Moderate=2, Severe=3). The QMG is a validated assessment, with a higher score indicating more severe disease. Scoring for each item ranges from no weakness (0) to severe weakness (3), with an overall score range from 0 to 39. A 3-point change in the total score is considered clinically relevant. |
| Change from Baseline to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within first 6-week treatment cycle | Baseline to Day 43 (in first treatment cycle) | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants will be asked to choose response option that best describes how frequently they experienced muscle weakness fatigability (items 34-42) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item. |
| Change from Baseline to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within first 6-week treatment cycle | Baseline to Day 43 (in first treatment cycle) | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants will be asked to choose the response option that best describes how frequently they experienced physical fatigue (items 19-33) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item. |
| Percentage of participants with treatment-emergent adverse events (TEAEs) | Up to End of study (40 weeks) | Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment. |
| Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP) | Up to End of study (40 weeks) | Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment. |
Countries
China
Contacts
CONTACTUCB Cares
STUDY_DIRECTORUCB Cares
001 844 599 22733 (UCB)
Outcome results
None listed