A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
- Registration Number
- NCT04124965
- Lead Sponsor
- UCB Biopharma SRL
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 71
- 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
- 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)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Rozanolixizumab dosage regimen 1 Rozanolixizumab Study participants randomized to dosage regimen 1 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched based on investigator discretion. Rozanolixizumab dosage regimen 2 Rozanolixizumab Study participants randomized to dosage regimen 2 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched based on investigator discretion.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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.
Trial Locations
- Locations (43)
Mg0004 50088
🇺🇸Washington, District of Columbia, United States
Mg0004 50070
🇨🇦Québec, Canada
Mg0004 50096
🇺🇸Philadelphia, Pennsylvania, United States
Mg0004 40124
🇨🇿Praha 2, Czechia
Mg0004 40140
🇩🇪Göttingen, Germany
Mg0004 40148
🇮🇹Roma, Italy
Mg0004 50120
🇺🇸Miami, Florida, United States
Mg0004 20077
🇯🇵Miyagi, Japan
Mg0004 50069
🇨🇦Toronto, Canada
Mg0004 40078
🇩🇪Leipzig, Germany
Mg0004 40131
🇫🇷Strasbourg, France
Mg0004 20027
🇷🇺Moscow, Russian Federation
Mg0004 40177
🇩🇪Münster, Germany
Mg0004 20079
🇯🇵Hiroshima, Japan
Mg0004 20028
🇷🇺Saint Petersburg, Russian Federation
Mg0004 40155
🇵🇱Gdańsk, Poland
Mg0004 40150
🇮🇹Roma, Italy
Mg0004 20001
🇷🇺Saint Petersburg, Russian Federation
Mg0004 40128
🇩🇰Aalborg, Denmark
Mg0004 40132
🇫🇷Nice, France
Mg0004 40133
🇫🇷Paris, France
Mg0004 20032
🇯🇵Suita, Japan
Mg0004 20055
🇷🇺Saint Petersburg, Russian Federation
Mg0004 50072
🇺🇸Los Angeles, California, United States
Mg0004 50081
🇺🇸Phoenix, Arizona, United States
Mg0004 50077
🇺🇸New York, New York, United States
Mg0004 50114
🇺🇸Indianapolis, Indiana, United States
Mg0004 50121
🇺🇸Lexington, Kentucky, United States
Mg0004 50066
🇨🇦Montréal, Canada
Mg0004 50090
🇺🇸Winston-Salem, North Carolina, United States
Mg0004 40129
🇫🇷Bordeaux, France
Mg0004 40125
🇨🇿Ostrava-Poruba, Czechia
Mg0004 40146
🇮🇹Pavia, Italy
Mg0004 40144
🇮🇹Milano, Italy
Mg0004 20078
🇯🇵Hanamaki shi, Japan
Mg0004 20076
🇯🇵Shinjuku-Ku, Japan
Mg0004 40154
🇵🇱Łódź, Poland
Mg0004 40151
🇵🇱Lublin, Poland
Mg0004 40159
🇪🇸Barcelona, Spain
Mg0004 40157
🇪🇸Hospitalet de Llobregat, Spain
Mg0004 20081
🇨🇳Taipei, Taiwan
Mg0004 20080
🇨🇳Taichung, Taiwan
Mg0004 50073
🇺🇸Tampa, Florida, United States