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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
71
Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.
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)
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)
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
Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
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Phoenix, Arizona, United States
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Los Angeles, California, United States
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Washington D.C., District of Columbia, United States
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Miami, Florida, United States
Mg0004 50073
Tampa, Florida, United States
Mg0004 50114
Indianapolis, Indiana, United States
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Lexington, Kentucky, United States
Mg0004 50077
New York, New York, United States
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Winston-Salem, North Carolina, United States
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Philadelphia, Pennsylvania, United States
...and 33 more locations
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
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
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)