Clinical Trial Results:
An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants with Generalized Myasthenia Gravis
Summary
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EudraCT number |
2020-003230-20 |
Trial protocol |
GB DE CZ FR PL HU DK BE IT |
Global end of trial date |
25 Jan 2024
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Results information
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Results version number |
v1 |
This version publication date |
07 Feb 2025
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First version publication date |
07 Feb 2025
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Other versions |
v2 , v3 |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
MG0007
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT04650854 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
UCB Biopharma SRL
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Sponsor organisation address |
Allée de la Recherche 60, Brussels, Belgium, 1070
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Public contact |
Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
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Scientific contact |
Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
12 Apr 2024
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
25 Jan 2024
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Global end of trial reached? |
Yes
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Global end of trial date |
25 Jan 2024
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
Assess the safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG)
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Protection of trial subjects |
During the conduct of the study all participants were closely monitored.
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Background therapy |
Background therapy as permitted in the protocol. | ||
Evidence for comparator |
Not applicable | ||
Actual start date of recruitment |
03 Feb 2021
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Canada: 19
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Country: Number of subjects enrolled |
Czechia: 2
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Country: Number of subjects enrolled |
Denmark: 5
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Country: Number of subjects enrolled |
France: 13
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Country: Number of subjects enrolled |
Georgia: 13
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Country: Number of subjects enrolled |
Germany: 7
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Country: Number of subjects enrolled |
Italy: 11
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Country: Number of subjects enrolled |
Japan: 12
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Country: Number of subjects enrolled |
Poland: 26
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Country: Number of subjects enrolled |
Russian Federation: 12
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Country: Number of subjects enrolled |
Serbia: 5
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Country: Number of subjects enrolled |
Taiwan: 3
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Country: Number of subjects enrolled |
United States: 32
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Country: Number of subjects enrolled |
Spain: 5
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Worldwide total number of subjects |
165
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EEA total number of subjects |
69
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
123
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From 65 to 84 years |
40
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85 years and over |
2
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Recruitment
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Recruitment details |
Study started to enroll participants in February 2021 and concluded in January 2024. Participant Flow refers to the Full Analysis Set (FAS). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Pre-assignment
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Screening details |
Participants who completed MG0003 (NCT03971422) or required rescue therapy during observation period (OP) in MG0003 (except who received intravenous immunoglobulin/plasma exchange) or completed at least 6 treatment visits in MG0004 (NCT04124965) were enrolled in study. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Not blinded | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Enrolled, but not Treated | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Arm description |
Participants were enrolled but did not require a treatment cycle in MG0007 per the Investigator’s discretion. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Arm type |
No intervention | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product name |
No investigational medicinal product assigned in this arm
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Arm title
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Rozanolixizumab ~7 mg/kg | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Arm description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product name |
Rozanolixizumab
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Investigational medicinal product code |
UCB7665
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Other name |
RLZ
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Pharmaceutical forms |
Infusion
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Participants received rozanolixizumab at pre-specified time-points.
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Arm title
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Rozanolixizumab ~10 mg/kg | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Arm description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product name |
Rozanolixizumab
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Investigational medicinal product code |
UCB7665
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Other name |
RLZ
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Pharmaceutical forms |
Infusion
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Participants received rozanolixizumab at pre-specified time-points.
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Baseline characteristics reporting groups
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Reporting group title |
Enrolled, but not Treated
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Reporting group description |
Participants were enrolled but did not require a treatment cycle in MG0007 per the Investigator’s discretion. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Rozanolixizumab ~7 mg/kg
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Reporting group description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Rozanolixizumab ~10 mg/kg
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Reporting group description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Enrolled, but not Treated
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Reporting group description |
Participants were enrolled but did not require a treatment cycle in MG0007 per the Investigator’s discretion. | ||
Reporting group title |
Rozanolixizumab ~7 mg/kg
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Reporting group description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | ||
Reporting group title |
Rozanolixizumab ~10 mg/kg
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Reporting group description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | ||
Subject analysis set title |
Rozanolixizumab ~7 mg/kg
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.
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Subject analysis set title |
Rozanolixizumab ~10 mg/kg
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.
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End point title |
Percentage of participants with treatment-emergent adverse events (TEAEs) [1] | ||||||||||||
End point description |
An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which did not necessarily had a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAE was defined as any event that was not present prior to first dose of investigational medicinal product (IMP), or any unresolved event already present that worsened in intensity following treatment, up to 8 weeks after end of Treatment Period or after last dose of IMP in participants who discontinued study or IMP. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Participants who switched doses were counted in both Rozanolixizumab (RLZ) doses.
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End point type |
Primary
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End point timeframe |
From Baseline (Day 1) to End of Study (up to 34 months)
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only. |
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No statistical analyses for this end point |
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End point title |
Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP) [2] | ||||||||||||
End point description |
An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Participants who switched doses were counted in both RLZ doses.
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End point type |
Primary
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End point timeframe |
From Baseline (Day 1) to End of Study (up to 34 months)
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Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only. |
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No statistical analyses for this end point |
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End point title |
Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle (Cycle 1, 2, and 3) [3] | |||||||||||||||||||||
End point description |
The MG-ADL is an 8-item PRO instrument developed on basis of QMG. The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0= no symptoms or impaired performance and 3= most severe symptoms or impaired performance. The total score ranges from 0 to 24, with higher score indicating more disability. A positive change in score indicates worsening and negative change indicates improvement. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline [Day 1]) value for that cycle. Safety Set (SS): All study participants in FAS who received at least one dose of IMP. Number of Participants Analyzed included those participants who were evaluable for assessment. 'n' included those participants who were evaluable at specified timepoint.
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End point type |
Secondary
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End point timeframe |
From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
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Notes [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle (Cycle 1, 2, and 3) [4] | |||||||||||||||||||||
End point description |
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 in the score indicates worsening and a negative change indicates improvement. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed included those participants who were evaluable for the assessment. 'n' included those participants who were evaluable at specified timepoint.
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End point type |
Secondary
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End point timeframe |
From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
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Notes [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle (Cycle 1, 2, and 3) [5] | |||||||||||||||||||||
End point description |
MG-C scale consists of 10 items: ptosis (0 to 3), double vision on lateral gaze left/right/both (0 to 4), eye closure (0 to 2), talking (0 to 6), chewing (0 to 6), swallowing (0 to 6), breathing (0 to 9), neck flexion or extension (0 to 4), shoulder abduction (0 to 5) and hip flexion (0 to 5), lower scores indicates lower disease activity. Total MG-C score obtained by summing responses to each individual item and score ranges from 0 - 50, (lower scores= lower disease activity). A positive change indicates worsening and a negative change improvement. Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed included participants who were evaluable for the assessment. 'n' included participants who were evaluable at specified timepoint.
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End point type |
Secondary
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End point timeframe |
From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
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Notes [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within one treatment cycle (Cycle 1, 2, and 3) [6] | |||||||||||||||||||||
End point description |
The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular symptoms (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option that best described severity of ocular, bulbar, and respiratory symptoms over past 7 days using a 4-point Likert scale (“none” to “severe”) and frequency of experiencing physical fatigue and muscle weakness fatigability over past 7 days using a 5-point Likert scale (1=“none of the time” – 5= “all of the time”), for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicates more severe symptoms. Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed: participants who were evaluable for assessment. 'n' included participants who were evaluable at specified timepoint.
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End point type |
Secondary
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End point timeframe |
From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
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Notes [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within one treatment cycle (Cycle 1, 2, and 3) [7] | |||||||||||||||||||||
End point description |
The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option based on frequency of experiencing physical fatigue (19-33) over past 7 days using a 5-point Likert scale (1=“none of time” - 5=“all of time”) for each item. Sum of each item score is linearly transformed to have all domain scores and total score ranging from 0 to 100, higher scores indicated severe symptoms. Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. 'n' included participants who were evaluable at specified timepoint.
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End point type |
Secondary
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End point timeframe |
From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
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Notes [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar Muscle Weakness' score within one treatment cycle (Cycle 1, 2, and 3) [8] | |||||||||||||||||||||
End point description |
The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Bulbar symptoms are now recognised as bulbar muscle weakness. Study participants were asked to choose response option that best described severity of bulbar muscle weakness (6-15) symptoms over past 7 days using a 4-point Likert scale (1=“none” to 4=“severe”) for each item. Sum of each item score is linearly transformed to have all domain scores and total score ranging from 0 to 100, higher scores indicated severe symptoms. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. 'n' included those participants who were evaluable at specified timepoint.
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End point type |
Secondary
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End point timeframe |
From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
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Notes [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
MG-ADL responder rate (>=2.0-point improvement from Baseline [Day 1]) within one treatment cycle (Cycle 1, 2, and 3 [Day 43]) [9] | |||||||||||||||||||||
End point description |
The MG-ADL is an 8-item 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 MG-ADL responder was defined as achieving at least 2.0-point improvement (decrease) in the MG-ADL score from Baseline. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. 'n' included those participants who were evaluable at specified timepoint.
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End point type |
Secondary
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End point timeframe |
Day 43 of Cycle 1, 2, and 3
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Notes [9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
Time to MG-ADL response (>=2.0-point improvement from Baseline [Day 1]) within one treatment cycle (Cycle 1, 2, and 3) [10] | |||||||||||||||||||||
End point description |
Time to achieve MG-ADL response, defined as at least 2.0-point improvement from Baseline. Time to first MG-ADL response (in days) by study cycle was defined as date of first MG-ADL Response within study cycle − date of MG-ADL Baseline within study cycle + 1. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number Analyzed included those participants who were evaluable at specified timepoint.
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End point type |
Secondary
|
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End point timeframe |
From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
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Notes [10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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End point title |
Time between consecutive treatment cycles [11] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Time between consecutive treatment cycles: Study participants were assessed for MG worsening prior to repeated cycles. In case of symptom worsening (eg, an increase of 2.0 points on MG-ADL or 3.0 points on QMG scale) between assessments, resulted additional treatment, study participants undergone another 6-week treatment cycle followed by an Observation Period, based on Investigator's discretion. Time between treatment cycles was calculated as: date of first sc infusion in consecutive cycle - date of last sc infusion before new cycle + 1 (or date of censoring - date of last sc infusion before potential new cycle + 1). Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. 'n' included participants who were evaluable for specified category. 99999 indicates median and full range were not estimated as no participants were analysed at specific timepoint.
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End point type |
Secondary
|
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End point timeframe |
From end of the 6-week treatment cycle (Day 43) to the next 6-week treatment cycle (Day 1), assessed up to 2.5 years
|
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Notes [11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study. |
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
From Baseline (Day 1) until End of Study (up to 34 months)
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Adverse event reporting additional description |
TEAE was defined as any event that was not present prior to first dose of IMP or any unresolved event already present that worsened in intensity following treatment, up to 8 weeks after end of Treatment Period or after last dose of IMP in participants who discontinued study or IMP. Participants who switched doses were counted in both RLZ doses.
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Assessment type |
Non-systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
24.0
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Reporting groups
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Reporting group title |
Rozanolixizumab ~10 mg/kg
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Reporting group description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Rozanolixizumab ~7 mg/kg
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Reporting group description |
Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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03 Mar 2021 |
Protocol Amendment 1 (dated 03 Mar 2021) was primarily implemented to incorporate an additional study objective and endpoint on post vaccination biomarkers in study participants who had received a coronavirus disease 2019 (COVID-19) vaccine. Other required changes included aligning the protocol with the updates for the rozanolixizumab generalized myasthenia gravis (gMG) clinical program, and to incorporate specific local ethics committees and/or agency requirements into the global protocol. |
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30 Jun 2022 |
Protocol Amendment 2 (dated 30 Jun 2022) was primarily implemented to incorporate the option to administer investigational medicinal product (IMP) via manual push instead of using a syringe driver, implement changes specific to the Schedule of Activities, update the temporary investigational medicinal product (IMP) discontinuation criteria, and update the study medication permanent discontinuation criteria, as well as provide additional information on the benefit-risk for study participants who had received a full COVID-19 vaccination.
Protocol Amendment 2 was approved and submitted to Food and Drug Administration (FDA) but was not implemented at the study sites. |
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03 Oct 2022 |
Protocol Amendment 3 (dated 03 Oct 2022) was primarily implemented to provide an update on the safety information in line with the updated Investigator’s Brochure dated Sep 2022 and to update the list of adverse events of special monitoring (adverse events of special monitoring [AESM]; remove severe gastrointestinal [GI] disorders [ie, abdominal pain, diarrhea, vomiting] and opportunistic infections and include serious headache and suspected aseptic meningitis). The possibility for participants receiving rescue therapy during the Observation Period to continue in the study was also added. The criteria for study medication discontinuation and the requirements for male contraception were also updated. Additional study assessments were specified in case of AESM, and a full neurological examination was added as part of the physical examination. |
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Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |