Generalized Myasthenia Gravis
Conditions
Keywords
UCB7665, generalized myasthenia gravis, rozanolixizumab, gMG
Brief summary
The purpose of the MycarinGstudy is to evaluate the long-term safety, tolerability and long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG).
Interventions
DRUGRozanolixizumab
Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.
Sponsors
UCB Biopharma SRL
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant was eligible for MG0003 \[NCT03971422\] or MGC003 at the time of enrollment into either study and the participant either completed the observation Period of MG0003 or MGC003 or required rescue therapy during the Observation Period of the lead-in studies * Body weight ≥35 kg at Visit 1 * Study participants may be male or female
Exclusion criteria
* Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest X-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold Plus * Participant has received a live vaccination within 8 weeks prior to the Baseline visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of study medication * Study participant has experienced hypersensitivity reaction after exposure to other anti-neonatal Fc receptor (FcRn) drugs - Study participant with severe (defined as Grade 3 on the myasthenia gravis-activates of daily living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis * Participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the study participant's ability to participate in this study * Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria MG0003 \[NCT03971422\] or MGC003, or discontinued study medication in either study, with the exception of discontinuation due to a need for rescue treatment * Study participant is not considered capable of adhering to the protocol visit schedule, or medication intake according to the judgment of the Investigator * Study participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation since the last visit in MG0003 as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From Baseline until End of Study (up to Week 60) | A TEAE is defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP. |
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication | From Baseline until End of Study (up to Week 60) | A TEAE is defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported outcome (PRO) instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement. |
| Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | MG-C scale is a validated assessment and scale tests 10 items with individual items being weighted differently. 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. Total MG-C score was obtained by summing responses to each individual item and score ranges from 0 to 50, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement. |
| Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement. |
| Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX)) | From Baseline until End of Study (up to Week 60) | Rescue therapy consisted of IVIg or PEX. Study participants who experienced disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator. |
Countries
Canada, Czechia, Denmark, France, Germany, Italy, Japan, Poland, Russia, Spain, Taiwan, United States
Contacts
STUDY_DIRECTORUCB Cares
001 844 599 22733 (UCB)
Participant flow
Recruitment details
The study started to enroll study participants in Oct 2019 and concluded in Sep 2021.
Pre-assignment details
Participant Flow refers to the Randomized Set.
Participants by arm
| Arm | Count |
|---|---|
| Rozanolixizumab ~7 mg/kg Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60). | 35 |
| Rozanolixizumab ~10 mg/kg Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60). | 36 |
| Total | 71 |
Baseline characteristics
| Characteristic | Rozanolixizumab ~7 mg/kg | Rozanolixizumab ~10 mg/kg | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 6 Participants | 10 Participants | 16 Participants |
| Age, Categorical Between 18 and 65 years | 29 Participants | 26 Participants | 55 Participants |
| Age, Continuous | 50.6 years STANDARD_DEVIATION 14.2 | 53.7 years STANDARD_DEVIATION 17.2 | 52.2 years STANDARD_DEVIATION 15.8 |
| Race/Ethnicity, Customized Asian | 4 Participants | 4 Participants | 8 Participants |
| Race/Ethnicity, Customized Black | 2 Participants | 3 Participants | 5 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 4 Participants | 2 Participants | 6 Participants |
| Race/Ethnicity, Customized Missing | 12 Participants | 10 Participants | 21 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 19 Participants | 25 Participants | 44 Participants |
| Race/Ethnicity, Customized White | 17 Participants | 19 Participants | 36 Participants |
| Sex: Female, Male Female | 19 Participants | 19 Participants | 38 Participants |
| Sex: Female, Male Male | 16 Participants | 17 Participants | 33 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 50 | 0 / 42 |
| other Total, other adverse events | 27 / 50 | 25 / 42 |
| serious Total, serious adverse events | 7 / 50 | 2 / 42 |
Outcome results
Primary
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
A TEAE is defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.
Time frame: From Baseline until End of Study (up to Week 60)
Population: The Safety Set (SS) consisted of all randomized study participants who received at least 1 dose of IMP in this study. This endpoint was planned to be analyzed using the SS by most recent dose received i.e. the most recent dose received at or before the AE onset. Participants who switched doses were counted in both rozanolixizumab (RLZ) doses.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Rozanolixizumab ~7 mg/kg | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 76.0 percentage of participants |
| Rozanolixizumab ~10 mg/kg | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 78.6 percentage of participants |
Primary
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication
A TEAE is defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.
Time frame: From Baseline until End of Study (up to Week 60)
Population: The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. This endpoint was planned to be analyzed using the SS by most recent dose received i.e. the most recent dose received at or before the AE onset. Participants who switched doses were counted in both rozanolixizumab doses.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Rozanolixizumab ~7 mg/kg | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication | 6.0 percentage of participants |
| Rozanolixizumab ~10 mg/kg | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication | 0 percentage of participants |
Secondary
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported outcome (PRO) instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement.
Time frame: Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Population: The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed signifies those participants who were evaluable at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 5 | -2.7 units on a scale | Standard Deviation 3.3 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 9 | -2.8 units on a scale | Standard Deviation 3.4 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 17 | -2.8 units on a scale | Standard Deviation 3.3 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 25 | -2.7 units on a scale | Standard Deviation 3 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 29 | -2.8 units on a scale | Standard Deviation 2.1 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 33 | -3.0 units on a scale | Standard Deviation 2.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 37 | -3.9 units on a scale | Standard Deviation 2.5 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 45 | -3.8 units on a scale | Standard Deviation 2.4 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 49 | -2.4 units on a scale | Standard Deviation 1.1 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 7 | -2.7 units on a scale | Standard Deviation 3.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 13 | -3.1 units on a scale | Standard Deviation 3.4 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 21 | -3.0 units on a scale | Standard Deviation 3.4 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 41 | -2.8 units on a scale | Standard Deviation 2.1 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 52 | -2.6 units on a scale | Standard Deviation 1.3 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 60 | -0.3 units on a scale | Standard Deviation 2.1 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 45 | -2.1 units on a scale | Standard Deviation 1.1 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 7 | -3.7 units on a scale | Standard Deviation 3.4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 41 | -1.8 units on a scale | Standard Deviation 1 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 13 | -3.9 units on a scale | Standard Deviation 4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 49 | -0.5 units on a scale | Standard Deviation 3.7 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 17 | -4.0 units on a scale | Standard Deviation 3.9 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 21 | -4.1 units on a scale | Standard Deviation 4.3 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 60 | -1.3 units on a scale | Standard Deviation 3.9 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 25 | -3.7 units on a scale | Standard Deviation 4.7 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 5 | -3.2 units on a scale | Standard Deviation 3.8 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 29 | -3.6 units on a scale | Standard Deviation 4.3 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 37 | -3.6 units on a scale | Standard Deviation 3.6 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 33 | -3.5 units on a scale | Standard Deviation 4.5 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 9 | -3.4 units on a scale | Standard Deviation 3.7 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 52 | -2.0 units on a scale | — |
Secondary
Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
MG-C scale is a validated assessment and scale tests 10 items with individual items being weighted differently. 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. Total MG-C score was obtained by summing responses to each individual item and score ranges from 0 to 50, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.
Time frame: Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Population: The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed signifies those participants who were evaluable at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 60 | 1.7 units on a scale | Standard Deviation 3.7 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 17 | -4.4 units on a scale | Standard Deviation 5.7 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 29 | -5.1 units on a scale | Standard Deviation 5.5 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 41 | -6.0 units on a scale | Standard Deviation 7.1 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 21 | -5.3 units on a scale | Standard Deviation 6.9 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 45 | -4.0 units on a scale | Standard Deviation 6.2 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 33 | -4.6 units on a scale | Standard Deviation 5.1 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 9 | -5.0 units on a scale | Standard Deviation 5.3 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 5 | -4.7 units on a scale | Standard Deviation 5.5 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 7 | -5.0 units on a scale | Standard Deviation 5.7 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 37 | -6.3 units on a scale | Standard Deviation 6.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 49 | -1.4 units on a scale | Standard Deviation 3.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 13 | -4.8 units on a scale | Standard Deviation 5.5 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 52 | -3.8 units on a scale | Standard Deviation 3.1 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 25 | -6.1 units on a scale | Standard Deviation 5.8 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 17 | -5.5 units on a scale | Standard Deviation 8.6 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 21 | -7.3 units on a scale | Standard Deviation 8.1 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 25 | -8.8 units on a scale | Standard Deviation 7.7 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 29 | -9.1 units on a scale | Standard Deviation 9.2 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 33 | -8.4 units on a scale | Standard Deviation 8.6 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 52 | NA units on a scale | — |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 60 | -2.3 units on a scale | Standard Deviation 8.5 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 37 | -8.6 units on a scale | Standard Deviation 6.9 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 41 | -6.5 units on a scale | Standard Deviation 5.8 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 45 | -5.3 units on a scale | Standard Deviation 4.9 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 49 | -0.8 units on a scale | Standard Deviation 9.2 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 7 | -7.1 units on a scale | Standard Deviation 7.4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 9 | -6.0 units on a scale | Standard Deviation 7.4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 13 | -7.0 units on a scale | Standard Deviation 7.8 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 5 | -5.5 units on a scale | Standard Deviation 7.3 |
Secondary
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.
Time frame: Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Population: The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed signifies those participants who were evaluable at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 9 | -3.3 units on a scale | Standard Deviation 3.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 21 | -4.0 units on a scale | Standard Deviation 4.7 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 5 | -2.9 units on a scale | Standard Deviation 4.7 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 7 | -3.3 units on a scale | Standard Deviation 4.4 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 13 | -2.6 units on a scale | Standard Deviation 4.2 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 17 | -3.1 units on a scale | Standard Deviation 4.9 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 25 | -5.1 units on a scale | Standard Deviation 4.6 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 29 | -5.4 units on a scale | Standard Deviation 3.6 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 33 | -4.9 units on a scale | Standard Deviation 4.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 37 | -5.3 units on a scale | Standard Deviation 3.9 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 41 | -5.7 units on a scale | Standard Deviation 4.3 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 45 | -5.3 units on a scale | Standard Deviation 2.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 49 | -3.8 units on a scale | Standard Deviation 0.8 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 52 | -4.6 units on a scale | Standard Deviation 2.9 |
| Rozanolixizumab ~7 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 60 | -0.9 units on a scale | Standard Deviation 2.4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 25 | -5.7 units on a scale | Standard Deviation 5.5 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 9 | -4.7 units on a scale | Standard Deviation 4.3 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 45 | -4.0 units on a scale | Standard Deviation 3.5 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 49 | -1.8 units on a scale | Standard Deviation 1.8 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 60 | -1.8 units on a scale | Standard Deviation 5.1 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 29 | -5.5 units on a scale | Standard Deviation 6.3 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 5 | -4.5 units on a scale | Standard Deviation 4.4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 41 | -4.0 units on a scale | Standard Deviation 3.1 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 7 | -5.2 units on a scale | Standard Deviation 4.3 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 33 | -6.2 units on a scale | Standard Deviation 5.7 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 13 | -5.5 units on a scale | Standard Deviation 4.4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 52 | NA units on a scale | — |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 17 | -4.2 units on a scale | Standard Deviation 4.4 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 21 | -5.1 units on a scale | Standard Deviation 4.9 |
| Rozanolixizumab ~10 mg/kg | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Week 37 | -6.8 units on a scale | Standard Deviation 5.9 |
Secondary
Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX))
Rescue therapy consisted of IVIg or PEX. Study participants who experienced disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator.
Time frame: From Baseline until End of Study (up to Week 60)
Population: The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Rozanolixizumab ~7 mg/kg | Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX)) | 11.4 percentage of participants |
| Rozanolixizumab ~10 mg/kg | Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX)) | 0 percentage of participants |