Conditions: Generalized Myasthenia Gravis
Keywords: UCB7665 generalized myasthenia gravis rozanolixizumab gMG
Allocation: RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 71
Healthy volunteers: no

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:

Arms:
arm 1: 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. arm 2: 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.

Interventions: intervention 1: Rozanolixizumab
Intervention descriptions: intervention 1: Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.

Protocol document:
SAP_002.pdf: 
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MG0004 
 
 
 
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STATISTICAL ANALYSIS PLAN 
AMENDMENT 1 
Study: MG0004 
Product: Rozanolixizumab 
A RANDOMIZED, OPEN-LABEL EXTENSION STUDY TO 
INVESTIGATE THE LONG-TERM SAFETY, TOLERABILITY, 
AND EFFICACY OF ROZANOLIXIZUMAB IN ADULT PATIENTS 
WITH GENERALIZED MYASTHENIA GRAVIS 
Short Title: 
A Phase 3 extension study to evaluate the safety, tolerability, and efficacy of rozanolixizumab in 
adult patients with generalized myasthenia gravis 
Sponsor: 
UCB Biopharma SRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Regulatory Agency Identifier Number(s): 
Registry 
ID 
Eudra CT Number: 
2019-000969-21 
IND Number: 
132407 
Confidentiality Statement 
Confidential 
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This document is the property of UCB and may not – in full or in part – be passed on, 
reproduced, published, or otherwise used without the express permission of UCB. 
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MG0004 
 
 
 
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TABLE OF CONTENTS 
VERSION HISTORY ................................................................................................................ 7 
LIST OF ABBREVIATIONS .................................................................................................... 8 
1 
INTRODUCTION ........................................................................................................... 10 
2 
PROTOCOL SUMMARY ............................................................................................... 10 
2.1 
Study objectives ........................................................................................................... 10 
2.1.1 
Primary objective ................................................................................................. 10 
2.1.2 
Secondary objective ............................................................................................. 10 
2.1.3 
Other objectives ................................................................................................... 10 
2.2 
Study endpoints ............................................................................................................ 11 
2.2.1 
Safety endpoints ................................................................................................... 11 
2.2.1.1 
Primary safety endpoints ........................................................................... 11 
2.2.1.2 
Other safety endpoints ............................................................................... 11 
2.2.2 
Efficacy endpoints ............................................................................................... 11 
2.2.2.1 
Secondary efficacy endpoints .................................................................... 11 
2.2.2.2 
Other efficacy endpoints ........................................................................... 11 
2.2.3 
Pharmacokinetic, and pharmacodynamic ............................................................ 12 
2.2.3.1 
Pharmacokinetic endpoints ........................................................................ 12 
2.2.3.2 
Pharmacodynamic endpoints ..................................................................... 12 
2.2.4 
Anti-drug antibody endpoints .............................................................................. 12 
2.2.5 
Immunological endpoints .................................................................................... 12 
2.2.6 
Other endpoints .................................................................................................... 12 
2.3 
Study design and conduct ............................................................................................ 13 
2.4 
Determination of sample size ....................................................................................... 14 
3 
DATA ANALYSIS CONSIDERATIONS ...................................................................... 15 
3.1 
General presentation of summaries and analyses ........................................................ 15 
3.2 
General study level definitions .................................................................................... 16 
3.2.1 
Analysis time points ............................................................................................ 16 
3.2.1.1 
Relative day ............................................................................................... 16 
3.2.2 
Study periods ....................................................................................................... 16 
3.2.3 
Mapping of assessments performed at Premature End of Treatment Visit ......... 17 
3.3 
Definition of Baseline values ....................................................................................... 17 
3.4 
Protocol deviations....................................................................................................... 17 
3.5 
Analysis sets................................................................................................................. 17 
3.5.1 
Enrolled Set ......................................................................................................... 17 
3.5.2 
Randomized Set ................................................................................................... 17 
3.5.3 
Safety Set ............................................................................................................. 17 
3.6 
Treatment assignment and treatment groups ............................................................... 18 
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3.7 
Center pooling strategy ................................................................................................ 18 
3.8 
Coding dictionaries ...................................................................................................... 18 
3.9 
Changes to protocol-defined analyses ......................................................................... 18 
4 
STATISTICAL/ANALYTICAL ISSUES ....................................................................... 19 
4.1 
Adjustments for covariates .......................................................................................... 19 
4.2 
Handling of dropouts or missing data .......................................................................... 19 
4.2.1 
Efficacy data ........................................................................................................ 19 
4.2.2 
Dates and times .................................................................................................... 19 
4.2.3 
Impact of COVID-19 pandemic on study data .................................................... 21 
4.3 
Handling of repeated and unscheduled measurements ................................................ 21 
4.4 
Interim analyses and data monitoring .......................................................................... 21 
4.4.1 
Timing for periodic data reviews ......................................................................... 22 
4.4.2 
Data required for periodic data reviews ............................................................... 22 
4.5 
Multicenter studies ....................................................................................................... 22 
4.6 
Multiple comparisons/multiplicity ............................................................................... 22 
4.7 
Use of an efficacy subset of study participants ............................................................ 22 
4.8 
Active-control studies intended to show equivalence .................................................. 22 
4.9 
Examination of subgroups ........................................................................................... 23 
5 
STUDY POPULATION CHARACTERISTICS ............................................................. 23 
5.1 
Subject disposition ....................................................................................................... 23 
5.2 
Important Protocol deviations ...................................................................................... 24 
6 
DEMOGRAPHICS AND OTHER BASELINE CHARACTERISTICS ........................ 24 
6.1 
Demographics .............................................................................................................. 24 
6.2 
Other Baseline characteristics ...................................................................................... 25 
6.3 
Medical history and concomitant diseases ................................................................... 26 
6.4 
Prior and concomitant medications .............................................................................. 26 
6.4.1 
Categories of prior and concomitant medications ............................................... 26 
6.4.2 
Assignment of medications to study period ......................................................... 26 
7 
MEASUREMENTS OF TREATMENT COMPLIANCE ............................................... 27 
8 
SAFETY ANALYSES ..................................................................................................... 27 
8.1 
Extent of exposure ....................................................................................................... 27 
8.2 
Adverse events ............................................................................................................. 27 
8.2.1 
Data considerations .............................................................................................. 27 
8.2.2 
Adverse events summaries .................................................................................. 28 
8.2.3 
Adverse events of focus ....................................................................................... 30 
8.3 
Clinical safety laboratory assessments ......................................................................... 30 
8.3.1 
Potential drug-induced liver injury ...................................................................... 32 
8.4 
Vital signs, physical findings, and other observations related to safety ...................... 33 
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8.4.1 
Vital signs ............................................................................................................ 33 
8.4.2 
Electrocardiograms .............................................................................................. 34 
8.4.3 
Other safety endpoints ......................................................................................... 34 
8.4.3.1 
Physical examination ................................................................................. 34 
8.4.3.2 
Suicidal risk monitoring ............................................................................ 34 
8.4.3.3 
Assessment and management of Tuberculosis (TB) ................................. 35 
9 
EFFICACY ANALYSES ................................................................................................ 35 
9.1 
Analysis of the secondary efficacy endpoints .............................................................. 35 
9.1.1 
MG-ADL score .................................................................................................... 35 
9.1.2 
MG-C score ......................................................................................................... 36 
9.1.3 
QMG scale ........................................................................................................... 36 
9.1.4 
Use of rescue medication (IVIg, PEX) due to worsening ................................... 36 
9.2 
Analysis of other efficacy endpoints............................................................................ 36 
9.2.1 
MGII .................................................................................................................... 36 
9.2.2 
MG Symptoms PRO ............................................................................................ 36 
9.2.3 
MG-QOL15r ........................................................................................................ 37 
9.2.4 
EQ-5D-5L ............................................................................................................ 37 
9.2.5 
MG-ADL responder rate (≥2.0 points improvement from Baseline) .................. 37 
9.2.6 
QMG responder rate (≥3.0 points improvement from Baseline) ......................... 37 
9.2.7 
MG-C responder rate (≥5.0 points improvement from Baseline) ........................ 37 
9.2.8 
Minimum symptom expression ........................................................................... 38 
10 PHARMACOKINETICS AND PHARMACODYNAMICS .......................................... 38 
10.1 
Pharmacokinetics ......................................................................................................... 38 
10.2 
Pharmacodynamics ...................................................................................................... 39 
10.2.1 
Total serum IgG and IgG subclasses ................................................................... 39 
10.2.2 
MG-specific autoantibodies ................................................................................. 39 
10.3 
Anti-drug antibody and neutralizing antibody (NAb) status ....................................... 39 
10.3.1 
Data consideration ............................................................................................... 40 
10.3.2 
ADA summaries .................................................................................................. 42 
10.4 
Immunology ................................................................................................................. 44 
10.4.1 
Serum complement levels and plasma complement levels .................................. 44 
10.4.2 
Cytokines ............................................................................................................. 44 
10.4.3 
Serum immunoglobulin concentrations ............................................................... 45 
10.4.4 
Anti-tetanus toxoid serum titers ........................................................................... 45 
11 OTHER ANALYSES ...................................................................................................... 45 
11.1 
Dose change in use of concomitant medications over time ......................................... 45 
11.2 
Specific analyses for Pharmaceuticals and Medical Devices Agency (PMDA).......... 46 
11.3 
Headache questionnaire ............................................................................................... 46 
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11.4 
Myasthenia Gravis Foundation of America (MGFA) by Visit .................................... 46 
12 REFERENCES ................................................................................................................ 47 
13 APPENDICES ................................................................................................................. 48 
13.1 
Quantitative Myasthenia Gravis scale ......................................................................... 48 
13.2 
Myasthenia Gravis-Composite scale............................................................................ 49 
13.3 
Myasthenia Gravis-Activities of Daily Living ............................................................ 50 
13.4 
Myasthenia Gravis Symptoms PRO ............................................................................ 51 
13.5 
Myasthenia Gravis Impairment Index ......................................................................... 55 
13.6 
MG-QOL15r ................................................................................................................ 62 
13.7 
Markedly abnormal laboratory and diagnostic criteria for Rozanolixizumab program64 
13.8 
AEs of focus for Rozanolixizumab program ............................................................... 66 
14 AMENDMENT(S) TO THE STATISTICAL ANALYSIS PLAN ................................. 70 
14.1 
Amendment 1 ............................................................................................................... 70 
STATISTICAL ANALYSIS PLAN SIGNATURE PAGE ..................................................... 73 
 
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VERSION HISTORY 
SAP Version 
Approval Date 
Change 
Rationale 
1.0 
18 Nov 2020 
Not Applicable 
Original version 
Amendment 1 
23 Sep 2021 
See summary of 
changes in Section 
14.1 
Updated based on 
DEM2 and DEM3 
comments 
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LIST OF ABBREVIATIONS 
ADA 
Antidrug Antibody 
ADD 
Average Daily Dose 
AE 
Adverse Event 
AEOF 
Adverse Event of Focus 
AESI 
Adverse Event of Special Interest 
AESM 
Adverse Event of Special Monitoring 
ALP 
Alkaline Phosphatase 
ALQ 
Above the Upper Limit of Quantification 
ALT 
Alanine Aminotransferase 
AST 
Aspartate Aminotransferase 
ATC 
Anatomical Therapeutic Chemical 
AUC 
Area Under the Curve 
BLQ 
Below the Lower Limit of Quantification 
BUN 
Blood Urea Nitrogen 
CI 
Confidence Interval 
CKD-EPI 
Chronic Kidney Disease Epidemiology Collaboration 
CRF 
Case Report Form 
CSR 
Clinical Study Report 
C-SSRS 
Columbia-Suicide Severity Rating Scale 
CTCAE 
Common Terminology Criteria for Adverse Events 
CV 
Coefficient of Variation 
DILI 
Drug-Induced Liver Injury 
ECG 
Electrocardiogram 
eGFR 
estimated Glomerular Filtration Rate 
EOS 
End of Study 
EQ-5D-5L 
5-Level European Quality of Life 5 Dimensions 
ES 
Enrolled Set 
FDA 
Food and Drug Administration 
geoCV 
Geometric Coefficient of Variation 
GI 
Gastrointestinal 
HbA1c 
Hemoglobin A1C 
hCG 
Human Chorionic Gonadotropin 
HLGT 
High Level Group Term 
HLT 
High Level Term 
ICH 
International Council for Harmonisation 
IDMC 
Independent Data Monitoring Committee 
IEC 
Independent Ethics Committee 
IGRA 
Interferon Gamma Release Assay 
IMP 
Investigational Medicinal Product 
INR 
International Normalized Ratio 
IRB 
Institutional Review Board 
IVIg 
Intravenous Infusion of Immunoglobulin G 
LDH 
Lactate Dehydrogenase 
LLOQ 
Lower Limit of Quantification 
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MA 
Markedly Abnormal  
MedDRA 
Medical Dictionary for Regulatory Activities 
MG 
Myasthenia Gravis 
MG-ADL 
MG-Activities of Daily Living 
MG-C 
MG-Composite 
MGII 
MG Impairment Index 
MGQoL15r 
Revised 15-item MG Quality of Life  
MRD 
Minimum Required Dilution 
NAb 
Neutralizing Antibody 
PDILI 
Potential Drug-Induced Liver Injury 
PEOT 
Premature End of Treatment 
PEX 
Plasma Exchange 
PK 
Pharmacokinetic 
PRO 
Patient-Reported Outcome 
PT 
Preferred Term 
PTT 
Partial Prothrombin Time 
QMG 
Quantitative Myasthenia Gravis 
RBC 
Red Blood Cell 
RLZ 
Rozanolixizumab 
RS 
Randomized Set 
SAP 
Statistical Analysis Plan 
sc 
Subcutaneous 
SD 
Standard Deviation 
SDR 
Safety Data Review 
SGPT 
Serum Glutamic-Pyruvic Transaminase 
SOC 
System Organ Class 
SS 
Safety Set 
TB 
Tuberculosis 
TEAEs 
Treatment-Emergent Adverse Events 
TFLs 
Tables, Figures and Listings 
ULN 
Upper Limit of Normal 
VAS 
Visual Analogue Scale 
WBC 
White Blood Cell 
WHODD 
World Health Organization Drug Dictionary 
 
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1 
INTRODUCTION 
The purpose of this statistical analysis plan (SAP) is to provide all information that is necessary 
to perform the statistical analyses of study MG0004, including independent Data Monitoring 
Committee (IDMC) and final analyses. It also defines the summary tables, figures and listings 
(TFLs) to be included in the final Clinical Study Report (CSR) according to the protocol. 
This SAP is based upon, and assumes familiarity with, the following documents: 
• Protocol Amendment 2: 30 July 2020 
• IDMC Charter v1.0: 31 March 2020 
If a future protocol amendment necessitates a substantial change to the statistical analysis of the 
study data, this SAP may be amended accordingly. Changes to the analysis from the protocol are 
documented in Section 3.9. The content of this SAP is compatible with the International Council 
for Harmonisation (ICH)/Food and Drug Administration (FDA) E9 Guidance documents. 
 
2 
PROTOCOL SUMMARY 
2.1 
Study objectives 
2.1.1 
Primary objective 
To evaluate the long-term safety and tolerability of rozanolixizumab in study participants with 
generalized myasthenia gravis (MG) 
2.1.2 
Secondary objective 
To evaluate the long-term efficacy of rozanolixizumab in study participants with generalized MG 
2.1.3 
Other objectives 
• To assess the dose change in use of concomitant medications in study participants receiving 
rozanolixizumab 
• To assess the plasma concentrations of rozanolixizumab administered by subcutaneous (sc) 
infusion 
• To assess the pharmacodynamic effects of rozanolixizumab as measured by IgG levels, IgG 
subclasses and MG-specific autoantibodies levels 
• To evaluate the incidence and emergence of anti-drug antibodies (ADAs) with respect to 
immunogenicity, pharmacokinetic (PK), and pharmacodynamics 
• To assess the effect of rozanolixizumab on biomarkers including IgM, IgA, and IgE, serum 
and plasma complement levels, and serum cytokines 
• To assess the effect of rozanolixizumab on tetanus IgG antibodies 
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2.2 
Study endpoints 
2.2.1 
Safety endpoints 
2.2.1.1 
Primary safety endpoints 
• Occurrence of treatment-emergent adverse events (TEAEs) 
• Occurrence of TEAEs leading to permanent withdrawal of study medication 
2.2.1.2 
Other safety endpoints 
• Occurrence of adverse events of special monitoring (AESMs) 
• Vital sign values and changes from Baseline (systolic and diastolic blood pressure and pulse 
rate) 
• 12-lead electrocardiogram (ECG) values and change from Baseline at each scheduled 
assessment 
• Clinical laboratory findings (hematology, biochemistry, and urinalysis) 
2.2.2 
Efficacy endpoints 
2.2.2.1 
Secondary efficacy endpoints 
• Change from Baseline in MG-Activities of Daily Living (MG-ADL) at each scheduled 
assessment during Treatment and Observation Periods 
• Change from Baseline in MG-Composite (MG-C) score at each scheduled assessment 
during Treatment and Observation Periods 
• Change from Baseline in Quantitative Myasthenia Gravis test (QMG) at each scheduled 
assessment during Treatment and Observation Periods 
• Use of rescue medication (intravenous infusion of immunoglobulin G [IVIg] or plasma 
exchange [PEX]) (Y/N) 
2.2.2.2 
Other efficacy endpoints 
• Change from Baseline in MG Impairment Index (MGII) total scores at each scheduled 
assessment during Treatment and Observation Periods 
• Change from Baseline in MGII ocular subscores at each scheduled assessment during 
Treatment and Observation Periods 
• Change from Baseline in MGII generalized domain subscores at each scheduled assessment 
during Treatment and Observation Periods 
• Change from Baseline in the MG Symptoms Patient-Reported Outcome (PRO) ‘Muscle 
Weakness Fatigability’ score at each scheduled assessment during Treatment and 
Observation Periods 
• Change from Baseline in the MG Symptoms PRO ‘Physical Fatigue’ score at each 
scheduled assessment during Treatment and Observation Periods 
• Change from Baseline in the MG Symptoms PRO ‘Bulbar symptoms’ score at each 
scheduled assessment during Treatment and Observation Periods 
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• Change from Baseline in the MG Symptoms PRO ‘Respiratory symptoms’ score at each 
scheduled assessment during Treatment and Observation Periods 
• Change from Baseline in the MG Symptoms PRO ‘Ocular symptoms’ score at each 
scheduled assessment during Treatment and Observation Periods 
• Change from Baseline in revised 15-item MG Quality of Life (MG-QoL15r) questionnaire 
at each scheduled assessment during Treatment and Observation Periods 
• Change from Baseline in 5-Level European Quality of Life 5 Dimensions (EQ-5D-5L) 
scores at each scheduled assessment during Treatment and Observation Periods 
For study participants who require rescue treatment during the Observation Period of MG0003 or 
MGC0003 and subsequently enter MG0004, the following endpoints will be assessed: 
• QMG responder rate (≥3.0 point improvement from Baseline) at each scheduled assessment 
during the first 6 weeks of the Treatment Period 
• MG-C responder rate (≥5.0 point improvement from Baseline) at each scheduled assessment 
during the first 6 weeks of the Treatment Period 
• MG-ADL responder rate (≥2.0 point improvement from Baseline) at each scheduled 
assessment during the first 6 weeks of the Treatment Period 
2.2.3 
Pharmacokinetic, and pharmacodynamic 
2.2.3.1 
Pharmacokinetic endpoints 
• Plasma concentration of rozanolixizumab at each scheduled assessment during the 
Treatment and Observation Periods 
2.2.3.2 
Pharmacodynamic endpoints 
• Change from Baseline in total IgG and IgG subclasses at each scheduled visit 
• Change from Baseline in MG-specific autoantibodies at each scheduled visit 
2.2.4 
Anti-drug antibody endpoints 
• ADAs at each scheduled assessment during the Treatment Period and Observation Periods 
2.2.5 
Immunological endpoints 
• Change from Baseline in serum complement levels (C3 and C4) and plasma complement 
levels (C3a and C5a) at each scheduled assessment during Treatment Period 
• Change from Baseline in serum cytokines at each scheduled assessment during Treatment 
and Observation Periods 
• Change from Baseline in serum immunoglobulin concentrations (total IgA, IgE, and IgM) at 
each scheduled assessment during Treatment and Observation Periods 
• Change from Baseline in anti-tetanus toxoid serum titers at each scheduled assessment during 
Treatment and Observation Period 
2.2.6 
Other endpoints 
• Dose change in use of concomitant medications during Treatment and Observation Period 
(See Section 3.9) 
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• Change from Baseline in average daily dose (ADD) of concomitant medications (See Section 
3.9) 
2.3 
Study design and conduct 
This is a randomized, open-label Phase 3 extension study to evaluate the long-term safety, 
tolerability, and efficacy of rozanolixizumab (RLZ) in study participants with generalized MG.  
Study participants will enter MG0004 from lead-in study MG0003. Study participants in 
MG0003 who complete the Observation Period or who require rescue medication during the 
Observation Period will be invited to be rerandomized in MG0004 (except for study participants 
who opt to receive IVIg or PEX as rescue medication). Study participants who receive IVIg or 
PEX as rescue therapy during the Observation Period of MG0003 will not be eligible to enroll in 
MG0004. Study participants who discontinue study medication in MG0003 for any reason other 
than requiring rescue medication will not be eligible for MG0004. 
For any study participant who enrolls in MG0004, the final visit in MG0003 (Visit 14) will serve 
as the first visit in MG0004 (Visit 1). All activities should be completed at Visit 1 (±1 week). For 
criteria pertaining to laboratory measures, the last available value from MG0003 will be used for 
evaluation of study participant eligibility, as long as the measurement was taken within the last 4 
weeks (not older than 32 days) prior to MG0004 Screening. Up to approximately 276 study 
participants will be enrolled into the lead-in study MG0003. All eligible study participants from 
MG0003 will be invited to participate in MG0004. 
In MG0004, study participants will be randomized in a 1:1 ratio to receive 1 of 2 doses of 
rozanolixizumab (equivalent to approximately 
, respectively). Specifically, 
the following dose arms will be used in the study: 
Dose arm 1: equivalent to approximately 
 rozanolixizumab 
• Bodyweight <50kg: dose to be administered 
 
• Bodyweight ≥50kg and <70kg: dose to be administered 
 
• Bodyweight ≥70kg and <100kg: dose to be administered 
 
• Bodyweight ≥100kg: dose to be administered 
 
Dose arm 2: equivalent to approximately 
 rozanolixizumab 
• Bodyweight <50kg: dose to be administered 
 
• Bodyweight ≥50kg and <70kg: dose to be administered 
 
• Bodyweight ≥70kg and <100kg: dose to be administered 
 
• Bodyweight ≥100kg: dose to be administered 
 
If one of the doses described above is determined to be discontinued after the interim analysis in 
MG0003, then that dose arm will be dropped from MG0004 as well, and study participants in the 
affected dose arm will be transferred to the continuing dose arm. If both dose arms are used in 
MG0004, study participants will be allowed to switch dose arms for tolerability and efficacy 
reasons at the discretion of the Investigator. 
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In MG0004, rozanolixizumab will be administered sc on a 
 basis over a 52-week 
Treatment Period. At the end of the 52-week Treatment Period in MG0004, study participants 
will participate in an 8-week Observation Period. The maximum study duration per study 
participant is 60 weeks. 
This open-label extension (OLE) study will be replaced by MG0007, an OLE study with 
 
 based on MG worsening. Upon study site approval of MG0007, as well as 
fulfilment of regulatory requirements, study participants will have the opportunity to rollover 
directly into MG0007 and will no longer have the opportunity to rollover to MG0004. In the 
event a study participant has already started MG0004, a minimum treatment duration of 6 visits 
must be completed (if IMP treatment is withheld for low IgG, study participants missed dose(s) 
can be counted as part of the total 6 visits for completion of MG0004 and meet eligibility 
requirements for MG0007) and then the participant will discontinue MG0004 and have the 
opportunity to enter into MG0007. For study participants entering MG0007, he/she must 
complete the Premature End of Treatment (PEOT) Visit in MG0004 which will serve as the 
Screening Visit for MG0007 or as the End of Study (EOS) Visit for MG0004. If a study 
participant completes the Treatment Period and still in Observation Period of MG0004, he/she 
will then complete EOS Visit to serve as the Screening Visit for MG0007. Once the site is active 
for MG0007, no further participants will enroll in MG0004 and the study will be closed at the 
site once all eligible study participants have rolled over into MG0007. 
A schematic of the study design is provided in Figure 2-1. 
Figure 2-1: Study schematic 
 
2.4 
Determination of sample size 
No formal sample size calculation can be performed. Up to approximately 240 study participants 
will be enrolled into the lead-in study MG0003. All eligible study participants from MG0003 
will be invited to participate in MG0004 until study site approval of MG0007 as well as 
fulfilment of regulatory requirements. 
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3 
DATA ANALYSIS CONSIDERATIONS 
3.1 
General presentation of summaries and analyses 
Statistical analysis and generation of tables, figures, participant data listings, and statistical 
output will be performed using SAS® Version 9.3 or higher. All tables and listings will use 
Courier New font size 9. 
Descriptive statistics will be displayed to provide an overview of the study results. For 
continuous variables, descriptive statistics will include number of participants with available 
measurements (n), mean, standard deviation (SD), median, minimum, and maximum. 
For categorical variables, the number and percentage of participants in each category will be 
presented. Unless otherwise noted, the denominator for percentages will be based on the number 
of participants included in the respective analysis set. Participants with missing data can 
generally be accounted for using the following approaches: 
• For summaries of demographics and Baseline characteristics: summarize percentages based 
on all participants in the analysis set and include a “Missing” category (corresponding to 
participants with missing data for the variable being summarized) as the last row in the list of 
categories being summarized.  
• For summaries of efficacy and safety endpoints, unless otherwise specified: summarize 
percentages based only on those participants with observed data for the variable being 
summarized. As the denominator may be different from the number of participants in the 
analysis set being considered, the denominator should be displayed in the table. The general 
format for displaying this will be “n/Nsub (%).” 
Unless otherwise noted, all percentages will be displayed to one decimal place. No percentage 
will be displayed for zero counts, and no decimal will be presented when the percentage is 100%.  
For PK concentration data, summary statistics will include geometric mean, geometric 
coefficient of variation (geoCV), 95% confidence intervals (CIs) for geometric mean, arithmetic 
mean, SD, median, minimum, and maximum.  
Decimal places for descriptive statistics will always apply the following rules: 
• “n” will be an integer; 
• Mean (arithmetic, geometric), SD, and median will use one additional decimal place 
compared to the original data; 
• geometric CV [%] will be presented with one decimal place; 
• Minimum and maximum will have the same number of decimal places as the original value. 
If no participants have data at a given visit, for example, then only n=0 will be presented. 
However, if 0<n<3, present the n, minimum and maximum only. If n=3, n, mean, median, 
minimum and maximum will be presented only. The other descriptive statistics will be left blank. 
Derived variables in general will display the mean and median to 1 more decimal place, SD to 2 
more decimal places and same decimal place to minimum and maximum than the variables used 
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in the derivation. If the number of decimal places reported in the raw data is varied, then use 
either the maximum raw number of reported decimal places or 3, whichever is the lowest, as a 
guide for the descriptive statistics. 
3.2 
General study level definitions 
3.2.1 
Analysis time points 
All data will be analyzed based on the visits identified per the Schedule of Activities in protocol. 
Mapping to analysis visit windows is not applied, except for PEOT visit (see Section 3.2.3). 
3.2.1.1 
Relative day 
Relative day for an event will be derived with the date of the first sc infusion of Investigational 
Medicinal Product (IMP) in MG0004 as reference. 
Relative days for an event of measurement occurring before the date of first sc infusion will be 
prefixed with '-' and are calculated as follows: 
 
Relative Day = [(Event Date – Date of First Infusion)] 
Relative days for an event or measurement occurring on or after the date of first sc infusion are 
calculated as follows: 
 
Relative Day = [(Event Date - Date of First Infusion) + 1] 
For events or measurements occurring after the date of the last sc infusion, relative day will be 
prefixed with ‘+’ in the data listings and are calculated as follows: 
 
Relative Day = + [(Event Date – Date of Last Infusion)] 
There is no relative Day 0. Relative day is not calculated for partial dates in cases where relative 
day is shown in a participant data listing. In such cases, relative day should be presented as ‘- -’ 
in the participant data listings. 
3.2.2 
Study periods 
The maximum duration of the study per participant will be approximately 60 weeks, consisting 
of the following 2 periods: 
• Treatment Period: 52 weeks  
• Observation Period: 8 weeks 
The end of the study is defined as the date of the last visit of the last participant in the study. 
The following definitions for starting and entering the study periods will be applied: 
• Treatment Period starts with the first day of IMP in MG0004 and ends after Week 52 or 
PEOT assessments.  All participants in the Safety Set will be considered to have started the 
Treatment Period. A participant is considered to have completed the Treatment Period if 
assessments from Treatment Period Week 52 are completed.  
• Observation Period starts with one day after the end of the Treatment Period and ends after 
the final assessments at the End of Study (EOS) visit. Participants with assessment on any 
Observation Period day are considered to have started the Observation Period. Participants 
who complete the assessments at the EOS visit will be considered to have completed the 
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Observation Period. Participants who have a completed status in the study termination case 
report form (CRF) are considered to have completed the Treatment and Observation Period.  
3.2.3 
Mapping of assessments performed at Premature End of Treatment 
Visit 
PEOT assessments will be assigned to the next scheduled site visit (following the last scheduled 
visit that the participant completed prior to PEOT) where each assessment is evaluated as per 
protocol. This approach means that there is a chance that PEOT data will be mapped to different 
visits according to the schedule of assessments.   
3.3 
Definition of Baseline values 
If not otherwise stated, Baseline values will be the last available value prior to or on the same 
date (and same time if time is collected for the individual assessment) of first administration of 
IMP in MG0004. Scheduled or unscheduled measurements can be used as the Baseline value.  
Baseline values for serum or plasma complements and serum cytokines will be the Baseline 
values determined in MG0003. 
Baseline for anti-drug antibody (ADA) for MG0004 should be the last measurement prior to 
receiving the very first rozanolixizumab (RLZ) infusion across all MG studies. Specifically, 
baseline for ADA is define as: 
• MG0003 ADA baseline if participants randomized to RLZ groups in MG0003; 
• MG0004 ADA baseline if participants randomized to placebo in MG0003. 
 
3.4 
Protocol deviations 
Important protocol deviations (IPDs) are deviations from the protocol which potentially could 
have a meaningful impact on study conduct or on the key safety, or PK/PD outcomes (if 
applicable) for an individual participant. The criteria for identifying important protocol 
deviations will be defined within the appropriate protocol-specific document. Important protocol 
deviations will be reviewed as part of the ongoing data cleaning process and data evaluation. 
3.5 
Analysis sets 
3.5.1 
Enrolled Set 
The Enrolled Set (ES) will consist of all study participants who sign the informed consent. 
3.5.2 
Randomized Set 
The Randomized Set (RS) will consist of all study participants who are randomized, using the 
treatment assigned instead of the actual treatment received. 
3.5.3 
Safety Set 
The Safety Set (SS) will consist of all randomized study participants who received at least 1 dose 
of IMP in this study. Analysis of this set will be according to the treatment the study participants 
actually received in MG0004, and will be used for the efficacy, demographic, PK/PD and safety 
analyses. The treatment assignment using SS is described in Section 3.6. 
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3.6 
Treatment assignment and treatment groups 
For the analyses using SS by the first dose received, study participants will be grouped according 
to the dose levels of IMP participants first received 
 in this study (i.e. 
before any switching occurs) using the exposure data from CRF. For example, if study 
participants randomized to IMP 
 and first received IMP 
, then these study 
participants will be allocated to IMP 
. If a study participant first received IMP 
 
and then switched to 
, then the study participant will be allocated to 
.  
For the analyses using SS by the most recent dose received, study participants will be grouped 
according to the most recent dose level of IMP participants received prior to each visit or the 
onset of events using the exposure data from CRF. For example, if a study participant first 
received IMP 
 and then switched to 
 prior to Visit 5, then the participant will be 
allocated to 
 at Visit 5. 
For the efficacy analyses using SS by dose switch during the Treatment Period, study 
participants will be grouped into dose adjuster or dose non-adjuster. A study participant who 
receive different dose during the Treatment Period from the randomized dose level will be 
classified as a dose adjuster, otherwise dose non-adjuster. For example, if a study participant was 
randomized to and first received IMP 
 and then switched to 
, then the study 
participant will be allocated to dose adjuster. 
For the analyses using RS, study participants will be grouped according to the dose levels of IMP 
to which study participants were randomized. 
3.7 
Center pooling strategy 
The participants from lead-in study, MG0003, will be rolled over in this study. The data from all 
sites will be pooled for analyses purposes. 
3.8 
Coding dictionaries 
Adverse events (AEs) and medical histories will be coded using version 24.0 or later of the 
Medical Dictionary for Regulatory Activities (MedDRA®).  
Medications will be coded according to B3 version Mar 2021 or later of the World Health 
Organization Drug Dictionary (WHODD). 
3.9 
Changes to protocol-defined analyses 
• The following two exploratory endpoints will not be analyzed as they have been removed 
from the MG program as a whole and have been included in the protocol in error: 
− Change from Baseline in the MG Symptoms PRO multicomponent total score, at each 
scheduled assessment during Treatment and Observation Periods, 
− Change from Baseline in the enhanced MG Symptoms PRO total score, at each scheduled 
assessment during Treatment and Observation Periods. 
• The increase or reduction in use of concomitant medications will be analyzed in the SAP, so 
objective is changed to “To assess the dose change in use of concomitant medications in 
study participants receiving rozanolixizumab” in the SAP. 
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• The dose change in use of concomitant medications in study participants receiving 
rozanolixizumab will be analyzed using number and percentage of participants with dose 
change of concomitant medications and change from Baseline in ADD in the study. There is 
no concentration data of concomitant medications in the study to generate area under the 
curve (AUC), so it is not included in the SAP.  
• No study participants from MGC003 will rollover into MG0004. Thus, the maximum sample 
size of this study should be 240, not 276. 
4 
STATISTICAL/ANALYTICAL ISSUES 
4.1 
Adjustments for covariates 
Statistical testing is not planned for this study, hence adjustment for covariates will not be 
required. 
4.2 
Handling of dropouts or missing data 
4.2.1 
Efficacy data 
The rules for handling missing data of individual items in the calculation of the QMG, MG-C, 
MG-ADL, MG Symptom PRO, MGII and MG-QOL15r scores are described in Section 13.1, 
Section 13.2, Section 13.3, Section 13.4, Section 13.5, and Section 13.6,  respectively.  
For ordinal endpoints (e.g. EQ-5D-5L), the complete case method will be applied. No further 
imputation is used. 
4.2.2 
Dates and times 
Partially or completely missing dates may be imputed for the following reasons: 
• Classification of AEs as TEAEs; 
• Classification of medications as past, prior, or concomitant medications;  
• Durations of AEs. 
Imputed dates will not be shown in listings.  All dates will be displayed as reported in the 
database. 
The following rules will be applied for partially or completely missing start dates: 
• If year, month and day are all missing then assign the date of first dose of IMP. If an imputed 
start date is after the specified end date, then assign January 01 of the year of the end date, or 
the date of screening if this is later (if the latter imputation results in an end date that is 
earlier than the start date, then use January 01). 
• If month and day are missing, and year is: 
− the same as the year of the first dose of IMP then assign the month-day of first dose of 
IMP. If the imputed start date is after the specified end date, then assign January 01, or 
the month-day of screening date if this is later (if the latter imputation results in an end 
date that is earlier than the start date, then assign January 01); 
− not the same as the year of the first dose of IMP then assign January 01. 
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• If only day is missing, and month-year is: 
− the same as the month-year of the first dose of IMP then assign the day of first dose of 
IMP. If the imputed start date is after the specified end date, then assign first day of the 
month, or the day of screening date if this is later (if the latter imputation results in an end 
date that is earlier than the start date, then assign first day of the month); 
− not the same as the month-year of the first dose of IMP then assign the first day of the 
month. 
The following rules will be applied for partially or completely missing stop dates: 
• If only the month and year are specified, then use the last day of the month. If an imputed 
stop dates is after last contact date, then assign last contact date as the stop date; 
• If only the year is specified, then use December 31 of the known year. If an imputed stop 
dates is after last contact date, then assign last contact date as the stop date; 
• If the stop date is completely unknown, then use discharge date or data cut-off date. 
Discharge date refers to the date of the end of study visit for completed participants or the 
date of discontinuation for participants that were withdrawn. For any AEs with known start 
date after the date of discontinuation, the date of last contact will be used as the discharge 
date. For participants still ongoing in the study at the time of the data cut-off, and for whom 
no discharge date is available, the date of the data cut-off will be used instead of the 
discharge date. 
Any medication with a start date on the first dosing date and time unknown, will be assumed to 
be concomitant. 
Imputed AE dates will be used for the calculation of duration of AEs as described in Table 4‒1.  
Table 4‒1: 
Calculation rules for duration of AEs 
Data 
availability 
Onset date 
Outcome 
date 
Calculation rules 
Complete data 
D1 
D2 
Duration = D2 – D1 + 1  d 
Start date 
partially or 
completely 
missing 
-- 
D2 
Duration = < D2 – D0 + 1 d 
Notes: D0 is imputed start date per above rules.  
End date 
partially or 
completely 
missing 
D1 
-- 
For ongoing AE: 
Duration = > D3 – D1 d 
For resolved AE: 
Duration = < D3 – D1 d 
Notes: D3 is imputed end date per above rules.  
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Table 4‒1: 
Calculation rules for duration of AEs 
Data 
availability 
Onset date 
Outcome 
date 
Calculation rules 
Start and end 
date partially or 
completely 
missing 
-- 
-- 
For ongoing AE: 
Duration = >D3 – D0 d 
For resolved AE: 
Duration = < D3 – D0 d 
Notes: D0 is imputed start date and D3 is imputed end 
date per above rules. 
4.2.3 
Impact of COVID-19 pandemic on study data 
Missing data is expected to be one of the major implications of the COVID-19 pandemic. The 
following approaches/strategies will be applied to assess the impact of COVID-19 pandemic on 
study data.  
• Added an eCRF page “COVID-19 Impact”, including impacted visits, impact categories and 
relationship to COVID-19;  
• Additional fields were added in protocol deviation specification documents to record 
protocol deviations relationship to COVID-19 pandemic; 
• Included additional summary analyses based on the timing of COVID-19 impact 
(pre/during/post COVID-19 pandemic). 
4.3 
Handling of repeated and unscheduled measurements 
All repeated and unscheduled measurements will be presented in the listings, where applicable. 
The following general rules will apply to all repeated and unscheduled measurements: 
• For repeated or unscheduled measurements obtained prior to the first dose of IMP the latest 
non-missing value (scheduled or unscheduled) will be used in the calculation of descriptive 
statistics (e.g. repeated measurements during the Screening period); 
• For repeated or unscheduled measurements obtained at any time point after the first dose of 
IMP, the scheduled values (if non-missing) will always be used in the calculation of changes 
from Baseline and for the descriptive statistics (ie, in summaries by time point). If repeated 
scheduled values are obtained at any time point, the latest non-missing values will be used. 
See Sections 8.4.2 for the rules applied to ECG triplicate measurements. 
4.4 
Interim analyses and data monitoring 
There is no interim analysis planned for this study. 
An independent Data Monitoring Committee (IDMC) will oversee the safety of the study by 
reviewing safety data at periodic data reviews.  
The safety data of this study will also be pooled with other Rozanolixizumab studies and 
reviewed by the overarching DMC. The details of overarching DMC will be described in a 
separate document. 
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4.4.1 
Timing for periodic data reviews 
The safety data review (SDR) meetings will be held in collaboration with the MG0003 study 
team. The orientation SDR meeting of this study will be held at the first SDR meeting of 
MG0003 study.  
First SDR meeting will be initiated when approximately 10 study patients have been randomized 
over 26 weeks and held at the second SDR meeting of MG0003 study. Ad-hoc meeting will be 
held based on the agreement of the IDMC.  
4.4.2 
Data required for periodic data reviews 
All available data at the time of the data cut-off will be included at each SDR. The database will 
not be locked, but the data should be as clean as possible, and it is not required to have resolved 
all queries prior to each database snapshot. Study enrollment will not be halted during planned 
IDMC review of the safety and efficacy data. 
Required safety data to be used to support the SDR will include the following: 
• Adverse events 
• ECGs 
• Vital signs 
• Medical and procedure history 
• Prior and concomitant medications 
• Demographics 
• Safety labs (hematology, chemistry, urinalysis) 
• Total IgG 
• Pregnancy  
• Columbia-Suicide Severity Rating Scale (C-SSRS) 
These data will be summarized using descriptive statistics. Data will also be provided in listings. 
4.5 
Multicenter studies 
Individual center results will not be displayed. 
4.6 
Multiple comparisons/multiplicity 
Adjustment for multiplicity will not be required since all analyses will be descriptive in nature. 
4.7 
Use of an efficacy subset of study participants 
Efficacy data will be summarized only, and there is no sensitivity analysis planned. 
4.8 
Active-control studies intended to show equivalence 
Not applicable. 
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4.9 
Examination of subgroups 
The continuous secondary efficacy endpoints will be evaluated for subgroups of interest 
including:  
• Age (18 to <65 years, ≥65 years) 
• Age (18-<65, 65-<85, ≥85 years) 
• Sex (male, female) 
• Region (North America, Europe, and Asia [excluding Japan], Japan)  
• Stratification factor in MG0003 - MG-specific autoantibody (MuSK+ or AChR+) 
Notes: Region captured from MG0003 CRF will be used. The stratification factors MuSK(+/-
) and AChR(+/-) will be based on the derived values from MG0003 subgroup analysis.  
The MG-ADL scores and change from Baseline will be summarized in the five subgroups as 
above and additional subgroups as follow: 
• Duration of disease at Baseline (<median, ≥ median) 
• MGFA disease class at Baseline 
• Thymectomy at Baseline (yes, no) 
• Baseline MG-ADL category (<5, ≥5). 
These evaluations will be descriptive; no statistical testing of treatment-by-subgroup interactions 
nor statistical testing of treatment effects within subgroups will be carried out. 
Subgroup analyses will only be performed for cases where there are at least 5 participants in a 
particular category, otherwise it will not be performed. 
5 
STUDY POPULATION CHARACTERISTICS 
5.1 
Subject disposition 
The following outputs will be created. 
Summaries: 
− Reasons for screen failures (as collected on the Study Termination Screen Failure CRF 
page) will be summarized using the ES for overall. Additionally, the reasons for screen 
failures will be summarized by pre-, during and post- the COVID-19 pandemic based on 
the screen failure date relative to the pandemic cut-off date. 
− Disposition of study participants screened will be summarized using the ES for overall, 
by region and by site. In this summary, the site number, principal investigator name, first 
participant in date and last participant out date, will be captured by randomized treatment 
and by each analysis set (RS and SS). 
− Disposition of analysis sets will be summarized by treatment groups and analysis sets 
(RS and SS) using the RS. 
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− Disposition and discontinuation reasons using the RS and SS will contain the number 
and percentage of study participants by treatment group and RLZ total and also by pre-, 
during and post- the COVID-19 pandemic who: 
o Started Study, 
o Completed Study, 
o Discontinued Study with 
▪ Primary Reason for discontinuation, including transitioned to study 
MG0007 (primary reason for premature study termination as collected in 
the Study Termination Enrolled CRF). 
Note: The summary by pre-, during and post- the COVID-19 pandemic will be 
based on the start, completed and discontinuation date relative to the pandemic 
cut-off date will be presented in the same table. The discontinuation reason in 
each period will also be summarized. Discontinuation due to COVID-19 
pandemic will be listed as sub-category under “Other” reason. 
− Discontinuation due to AEs using the RS and SS will summarize the total number of 
study participants who discontinued the study due to AEs by treatment group and the 
categories: AE, serious fatal, AE non-fatal and other (AE non-serious fatal). 
− Count of participant by visit using SS will summarize the number of participants at 
each visit by treatment group. 
− Impact of COVID-19 pandemic using the RS will summarize number and percentage of 
participants in each impact category by visit. 
Listings of study participant disposition, study discontinuation and study participants who did 
not meet study eligibility criteria will be provided. 
5.2 
Important Protocol deviations 
A summary of number and percentage of participants with an important protocol deviation by 
relationship to COVID-19 pandemic and treatment group will be provided for the RS. 
Additionally, the summary will be repeated by pre-, during and post- the COVID-19 pandemic 
based on the deviations start date relative to the pandemic cut-off date.  
A listing of important protocol deviations will be provided based on the RS. 
6 
DEMOGRAPHICS AND OTHER BASELINE 
CHARACTERISTICS 
6.1 
Demographics 
Demographic variables will be summarized on the RS and SS, by categories mentioned below 
using descriptive statistics by treatment group and RLZ total. Additional subgroup summary will 
be presented by pre-, during and post- the COVID-19 pandemic based on the enrolled date 
relative to the pandemic cut-off date.  
Categories for continuous variables (including n, mean, SD, Median, Min and Max): 
• Age at the time of Study MG0003 entry (years)  
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Notes: Missing age will be calculated as year of informed consent signed – year of birth 
• Height (cm) 
• Weight (kg) 
• BMI (kg/m2), to be calculated as: BMI = Weight (kg) / (Height (m))2 
Categorical variables (using frequency counts and percentages): 
• Age (18-<65, 65-<85, ≥85 years) 
• Age (<=18, 19-<65, ≥65 years)  
• BMI (<30 kg/m2, ≥30 kg/m2) 
• Weight (<50kg, 50kg-<70 kg, 70-<100kg, ≥100kg) 
• Weight (<50kg, ≥50kg) 
• Weight (<70kg, ≥70kg) 
• Sex (Male, Female) 
• Race (American Indian or Alaskan native, Asian, Black, Native Hawaiian or Other Pacific 
Islander, White, Other/Mixed) 
• Ethnicity (Hispanic or Latino, Not Hispanic or Latino) 
• Region (North America, Europe, Asia [excluding Japan], Japan) 
• Country 
• Needed rescue therapies in the observation period of MG0003 and entered MG0004 
Note: Height captured at screening visit from MG0003 CRF will be summarized and used in 
BMI calculation. Other variables listed here are captured from MG0004 CRF. 
A by-participant listings of demographics will be provided using the RS. Childbearing potential 
and lifestyle will be listed using the ES separately. 
6.2 
Other Baseline characteristics 
The following variables will be summarized by treatment group and RLZ total for the RS. The 
baseline values are derived according to Section 3.3. 
• Baseline MuSK antibody status (positive, negative) 
• Baseline AChR antibody status (positive, negative) 
• Baseline MG-ADL score 
• Baseline MG-ADL category (<5, ≥5) 
• Baseline QMG score 
• Baseline MG-C score 
• Baseline total IgG value 
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6.3 
Medical history and concomitant diseases 
Any medical conditions that were not reported in MG0003 will be captured on CRF of this study 
and listed using the RS. Besides, procedure history will be provided in separate by-participant 
listings using the RS. 
6.4 
Prior and concomitant medications 
The number and percentage of participants taking Prior or Concomitant medications will be 
summarized using the RS by Anatomical Therapeutic Chemical (ATC) class, presenting as 
Anatomical Main Group (ATC Level 1), Pharmacological Subgroup (ATC level 3), Preferred 
Term (PT), treatment group and RLZ total. The number and percentage of participants taking 
steroids, Immunosuppressants and AChE Inhibitors will be summarized. 
Additionally, rescue medications are those that are mentioned in Protocol section 6.5.3 and 
identified: 
• if Rescue Medication is ticked as yes on CRF Concomitant Medication page 
• Or PEX as procedure entered on CRF Concomitant Medical Procedure page 
The start date of rescue medication should be on or after Baseline. All rescue medications will be 
summarized using the RS. 
Medications classified as prior or concomitant will be listed using the RS. A by-participant 
listing of participants taking steroids, immunosuppressants and AChE Inhibitors will be listed, 
including ADD at Baseline, ADD at last study visit and change from Baseline in ADD (See 
Section 11.1).  A by-participant listing of concomitant procedures will also be listed using the 
RS. Originally reported dates will be used for listings. 
6.4.1 
Categories of prior and concomitant medications 
Medications will be classified as follow based on imputed start and stop dates & times as 
outlined in Section 4.2.2. 
• Prior medications will include any medications that started before the first administration of 
IMP in MG0004. 
• Concomitant medications will include any medications that have been taken at least once 
after the first administration of IMP during the Treatment and/or Observation Period of 
MG0004.  
Medication Started 
Medication finished 
Classification 
Before 1st Dose IMP 
Any time 
Prior 
Any time 
After 1st Dose IMP 
Concomitant 
6.4.2 
Assignment of medications to study period 
The following rules will be used to assign a concomitant medication to a study period: 
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• Treatment Period: a medication will be assigned to the Treatment Period if it has been 
taken at least once between the first administration of IMP on Day 1 and 7 days after the last 
dose of IMP.  
• Observation Period: a medication will be assigned to the Observation Period if it has been 
taken at least once from the day after Treatment Period to the EOS visit.  
Thus, a medication taken from the time of the first drug administration in the Treatment Period 
to the end of the study will be assigned to the Treatment Period and the Observation Period. 
 
7 
MEASUREMENTS OF TREATMENT COMPLIANCE 
Not applicable.  The summaries of infusions will be detailed in Section 8.1. 
8 
SAFETY ANALYSES 
All safety analyses will be presented using the SS. Listings will be presented by treatment group 
and participant; tabulations will be presented by treatment group. Unless otherwise specified, 
safety analyses will be presented by the most recent dose the study participants received in 
MG0004 as described in Section 3.6. 
8.1 
Extent of exposure 
The following descriptive summaries will be generated using the SS by first dose received: 
a) Study IMP duration (weeks) by first dose received, calculated as follow: 
𝑆𝑡𝑢𝑑𝑦 𝐼𝑀𝑃 𝑑𝑢𝑟𝑎𝑡𝑖𝑜𝑛 (𝑤𝑒𝑒𝑘𝑠) = 𝐷𝑎𝑡𝑒 𝑜𝑓 𝑙𝑎𝑠𝑡 𝑑𝑜𝑠𝑒−𝐷𝑎𝑡𝑒 𝑜𝑓 𝑓𝑖𝑟𝑠𝑡 𝑑𝑜𝑠𝑒+ 1
7
 
b) Cumulative study IMP Duration using > 0 weeks, ≥ 6 weeks, ≥ 12 weeks, ≥ 18 weeks, ≥ 
24 weeks, ≥ 30 weeks, ≥ 36 weeks, ≥ 42 weeks and ≥ 48 weeks and 52 weeks; 
c) Number of Infusions Received as continuous and using the following categorical values: 
1-6, 7-12, 13-18, 19-24, 25-30, 31-36, 37-42, 43-48, and 49-52.  
The number of participants with dose change at each visit will be summarized using the SS by 
the most recent dose received. 
All IMP administration details will be listed. 
8.2 
Adverse events 
8.2.1 
Data considerations 
Adverse events will be recorded from the time of informed consent until study completion. All 
AEs will be coded as described in Section 3.8. 
In addition, AEs will be classified according to the Common Terminology Criteria for Adverse 
Events (CTCAE) Version 5 or later for severity. For any AEs where it is not possible to provide 
a CTCAE grading, the events will be assessed using a standard intensity classification (mild, 
moderate and severe). For the purpose of reporting severe AEs, all CTCAE severity 
classifications will be mapped to a standard intensity classification as described below: 
• Grade 1 - Mild 
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• Grade 2 - Moderate 
• Grade 3, 4, 5 - Severe 
These will be tabulated together with the AEs that were not classified according to CTCAE 
criteria (i.e., all Grade 1 AEs as per CTCAE criteria will be included in the ‘mild’ category 
together with those AEs classified as mild as per the standard intensity classification). In the case 
a mapped standard intensity classification per above rule is different from the standard intensity 
classification on CRF, the worst case will be used as the standard intensity classification (i.e. an 
AE with Grade 1 and moderate as intensity classification will be classified into moderate). 
A TEAE is defined as an AE starting on or after the time of first administration of IMP in 
MG0004 or any unresolved event already present before the first administration of IMP in 
MG0004 that worsens in intensity following exposure to IMP in MG0004, up to and including 8 
weeks (56 days) after the end of the Treatment Period or 8 weeks after the last of IMP in study 
participants who discontinue the study or IMP.  
A persistent AE is defined as an unresolved AE that extends continuously from MG0003 to 
MG0004 and does not worsen in intensity following exposure to IMP in MG0004. Persistent 
AEs will be recorded in MG0004 database and as other non-TEAEs will be listed only. 
Where dates are missing or partially missing, AEs will be assumed to be treatment-emergent 
unless evidence exists that does not allow the AE to be treatment-emergent. Handling of missing 
dates for classification of AEs as TEAEs is described in Section 4.2.2. 
The following rules will be used to assign a TEAE to a study period: 
• Treatment Period: a TEAE will be assigned to the Treatment Period if the start date of the 
event is on or after the date of the first administration of MG0004 IMP on Day 1, up to 7 
days after the last dose of IMP; 
• Observation Period: a TEAE will be assigned to the Observation Period if the start date of 
the event is greater than the day after Treatment Period until 8 weeks following the final 
dose; events starting later than 8 weeks following the final dose of IMP are not considered 
TEAEs. 
A TEAE will be counted as a TEAE related to IMP if the response to the question “Relationship 
to Study Medication” is “Related”.  
AEs will be presented as “number of participants (percentage of participants) [number of 
events]”.  In this style of output, “[number of events]” will include all cases of an AE including 
repeat occurrences in individual participants, while “number of participants” will count each 
participant only once. 
8.2.2 
Adverse events summaries 
TEAEs will be summarized by the most recent dose level prior to onset of TEAEs and RLZ total, 
including the number and percentage of study participants and frequency. Additional details are 
described below: 
1. Overview of TEAEs will include following categories: any TEAEs, serious TEAEs, 
participant discontinuation due to TEAEs, permanent withdrawal of IMP due to TEAEs, 
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temporary withdrawal of IMP due to TEAEs, TEAEs requiring dose change, treatment-related 
TEAEs, severe TEAEs, TEAEs leading to death, all deaths (AEs leading to death).  
2. Incidence of TEAEs will be summarized by SOC, HLT, PT for:  
− Any TEAEs 
− Any TEAEs during Treatment Period 
− Any TEAEs by maximum intensity (mild, moderate and severe) 
− Any TEAEs by relationship 
− Severe TEAEs 
− Non-serious TEAEs above reporting threshold of 5% of study participants 
− Fatal TEAEs 
− Fatal TEAEs by relationship 
− Serious TEAEs 
− Serious TEAEs by relationship 
− Participant discontinuation due to TEAEs 
− TEAEs leading to permanent withdrawal of IMP 
− TEAEs leading to temporary withdrawal of IMP 
− TEAEs requiring dose change 
− Treatment-emergent AESM 
− Treatment-emergent AESI 
3. Incidence of any TEAEs will be summarized by decreasing frequency of PT. 
AESMs include severe headache, severe GI disorders (i.e., abdominal pain, diarrhea, vomiting), 
and opportunistic infection. 
AESIs are the cases of potential Hy’s Law (see Section 8.3.1 ). 
AESMs and AESIs will be identified based on the assessment by the Investigator as recorded in 
the CRF. An AE will be counted as an AESM if there is a ‘yes’ response to the question 
“Adverse event of Special Monitoring?” and ‘no’ otherwise. An AE will be counted as an AESI 
if there is a ‘yes’ response to the question “Adverse Event of Special Interest?” and ‘no’ 
otherwise. 
When applicable adverse event summaries will be ordered by alphabetical SOC, alphabetical 
HLT within SOC and decreasing frequency of PT in the RLZ total column for tables.  
Listings of all TEAEs, all non-serious TEAEs, serious TEAEs, permanent withdrawal of IMP 
due to AEs, study participant discontinuation from study due to AEs, AEs leading to death, 
AESIs, AESMs will be presented by treatment group and participant. Persistent AEs and TEAE 
will be flagged in the listings. Most recent dose will be presented in listing even when the AE 
start date is partial. 
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8.2.3 
Adverse events of focus 
Rozanolixizumab adverse events of focus (AEOF) include the following categories: 
− Headaches 
− Gastrointestinal disturbances 
− Hypersensitivity reactions 
− Anaphylactic reactions 
− Injection site reaction 
− Infusion reaction 
− Infections 
− Opportunistic infections 
− Reductions in albumin and plasma proteins 
− Effects on the kidney 
− Drug related hepatic disorders 
− Effects on lipids  
1. The number and percentage of study participants who experience each category of the AEOF 
will be summarized by most recent dose level prior to onset of the AEOF and RLZ total 
treatment group. The following summaries will be presented by SOC, HLT, and PT: 
− AEOF  
− Serious AEOF 
− AEOF by relationship 
− AEOF by maximum intensity (mild, moderate and severe) 
2. Additionally, the summaries for the AEOF headache and gastrointestinal disturbances in 
Treatment Period by intensity will be presented separately by most recent dose level and RLZ 
total. 
3. Graphs for AEOF headache in Treatment Period by time of onset (relative to infusion), 
severity, causality and duration for each study participant will also be provided. 
A by-subject listing of all AEOF by category (as listed above) will be provided by treatment 
group and participant. Most recent dose will be presented in listing even when the AE start date 
is partial. Further details related to the statistical analysis of the above mentioned treatment-
emergent AEOFs are provided in Section 13.8. 
8.3 
Clinical safety laboratory assessments 
The following table (Table 8-1) lists safety laboratory assessments that are collected throughout 
the study: 
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Table 8-1: 
Clinical Laboratory Parameters 
Laboratory 
Assessments 
Parameters 
Hematology 
Platelet Count 
RBC Indices: 
Mean corpuscular 
volume 
Mean corpuscular 
hemoglobin 
%Reticulocytes 
White Blood Cell 
(WBC) Count with 
Absolute Count and 
Differential: 
Neutrophils 
Lymphocytes 
Monocytes 
Eosinophils 
Basophils 
Red Blood Cell (RBC) 
Count 
Hemoglobin 
Hematocrit 
Clinical 
Chemistry 
Blood Urea 
Nitrogen 
(BUN) 
Potassium 
Aspartate Aminotransferase 
(AST) / Serum Glutamic-
Oxaloacetic Transaminase 
(SGOT) 
Total and 
direct 
bilirubin 
Estimated 
Glomerular 
Filtration Rate 
(eGFR)a 
Sodium 
Alanine Aminotransferase 
(ALT) / Serum Glutamic-
Pyruvic Transaminase 
(SGPT) 
Total Protein 
Glucose 
(fasting) 
Calcium 
Alkaline phosphatase (ALP) Creatinine 
Lactate 
dehydrogenase 
(LDH) 
C-reactive 
protein 
Albumin 
 
Routine 
Urinalysis 
• Specific gravity 
• Albumin, albumin/creatinine, creatinine 
• pH, glucose, protein, ketones, bilirubin, urobilinogen, nitrite, leukocyte 
esterase, hemoglobin by dipstick 
• Microscopic examination (if blood or protein is abnormal) 
Other 
Screening 
Tests 
• Urine human chorionic gonadotropin (hCG) pregnancy test (as needed for 
women of childbearing potential)b 
• Serology testing for HIV, hepatitis B, and hepatitis C 
• Partial Prothrombin Time (PTT), and International Normalized Ratio 
(INR), and Hemoglobin A1C (HbA1c) tests 
NOTES: 
All study-required laboratory assessments will be performed by a central laboratory.  
The results of each test must be entered into the CRF. 
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Laboratory 
Assessments 
Parameters 
a eGFR is calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-
EPI) formula which is eGFR = 141 * min(Scr/κ,1)α * max(Scr/κ, 1)-1.209 * 0.993Age * 1.018 [if 
female] * 1.159 [if black];  where Scr is serum creatinine (mg/dL), κ is 0.7 for females and 0.9 
for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/κ or 
1, and max indicates the maximum of Scr/κ or 1. If race is not captured, participant will be 
assumed not Black in the calculation. 
b Local urine testing will be standard for the protocol unless serum testing is required by local 
regulation or Institutional Review Board (IRB)/ Independent Ethics Committee (IEC). 
 
Chemistry, hematology and quantitative urinalysis (observed value, absolute change from 
Baseline) will be summarized in standard unit using descriptive statistics by most recent dose 
received and RLZ total at each scheduled visit. 
The central lab data will be used for the summary tables. Repeated lab measurements will be 
handled per Section 4.3. 
Measurements below the lower limit of quantification (BLQ) will be imputed with half of the 
lower limit of quantification (LLOQ), and measurements above the upper limit of quantification 
(ALQ) will be imputed to the upper quantification limit for the purpose of quantitative 
summaries. 
The number and percentage of participants who meet each of the markedly abnormal (MA) 
criteria outlined in Section 13.7 will be summarized by most recent dose received at any visit 
(including unscheduled visit).  
The laboratory variables that are categorized as normal, high or low based on the reference range 
supplied by the analytical laboratory will be presented in shift tables from Baseline to any post-
Baseline visit (including unscheduled visit) by most recent dose received.  
All central or local laboratory test results will be listed, including Baseline, scheduled and 
unscheduled visits with results in standard unit. Values outside the reference range for the 
continuous variables will be flagged in the listings. The reference ranges will also be reported in 
the listings. In addition, the listings will include a flag for values identified as MA. Additional 
lab test, including pregnancy testing, will also be listed. 
8.3.1 
Potential drug-induced liver injury  
The number and percentage of study participants who meet one or more of the following 
potential drug-induced liver injury (pDILI) criteria will be summarized by treatment group and 
RLZ total: 
• Participants with at least one post-Baseline liver laboratory assessment 
• Incidence of potential hepatotoxicity with symptoms potentially associated with hepatitis or 
hypersensitivity 
• Incidence of potential hepatotoxicity with no symptoms potentially associated with hepatitis 
or hypersensitivity 
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• Laboratory criteria for pDILI: 
− (AST or ALT > 3 x ULN) and TBL > 1.5 x ULN 
− (AST or ALT > 3 x ULN) and TBL > 2 x ULN 
− (AST or ALT > 3 x ULN) and TBL > 2 x ULN and ALP < 2 x ULN (Hy’s Law) 
In order to meet the above criteria, a study participant must experience the elevation in bilirubin 
and ALT or AST (and the absence of the ALP elevation) at the same visit. For example, a study 
participant who experiences a >2xULN elevation of bilirubin at one visit and a >3xULN 
elevation in ALT (or AST) at a subsequent visit has not fulfilled the Hy’s law criteria. If 
participant has one value unknown, then he/she should not be considered for meeting the above 
criteria. 
Additional analyses for liver function tests (LFTs) will be performed to assess the potential for 
liver toxicities in accordance with the United States Food and Drug Administration guidelines. 
Per guidelines, the following criteria will be used to define levels of LFT elevation: 
• Aspartate aminotransferase (AST): >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x 
ULN 
• Alanine aminotransferase (ALT): >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x ULN 
• AST or ALT: >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x ULN 
• Total bilirubin (TBL): >1.5 x ULN, >2 x ULN 
• Alkaline phosphatase (ALP) >1.5 x ULN 
A listing will also be provided for study participants who meet at least one of the above criteria. 
All results obtained at that visit for the specified parameters will be displayed. 
8.4 
Vital signs, physical findings, and other observations related to 
safety 
8.4.1 
Vital signs 
Observed values and changes from Baseline will be summarized by vital signs variable (pulse 
rate, systolic and diastolic blood pressure, and temperature), scheduled visit, most recent dose 
received and RLZ total.   
The number and percentage of participants who meet each of the MA criteria outlined in 
Section 13.7 will be summarized by most recent dose received and RLZ total at any visit 
(including unscheduled visit).  
Additionally, the vital signs variables that are categorized as normal or MA based on the MA 
criteria will be presented in shift tables from Baseline to any post-Baseline visit (including 
unscheduled visit) by most recent dose received. 
A by-participant listing of all vital sign measurements and change from Baseline will be 
presented by treatment group and visit. The listing will include a flag for values identified as 
MA. Unscheduled measurements will be presented in the listings. 
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8.4.2 
Electrocardiograms 
The following ECG variables will be reported: 
• Heart rate 
• PR interval 
• RR interval 
• QRS duration 
• QT interval 
• QT corrected for heart rate using Fridericia’s formula (QTcF = QT/RR1/3) 
Observed values and changes from Baseline will be summarized by most recent dose received 
and RLZ total at scheduled visit and by ECG variable. The number and percentage of 
participants with normal, abnormal not clinically significant and abnormal clinically significant 
ECG results will be provided in a shift table from Baseline to worst post-Baseline interpretation 
during the study. 
For the ECG data, all calculations of changes from Baseline and descriptive statistics will be 
based on the mean of the triplicate assessments at each visit. In the event that there are not 
3 available measurements at a given visit, the mean will be calculated based on the number of 
measurements for which data are provided.  
The number and percentage of participants who meet each of the MA criteria outlined in 
Section 13.7 will be summarized by most recent dose received at any visit (including 
unscheduled visit). 
A listing of electrocardiogram data will be presented, including repeated and unscheduled 
measurements.  
8.4.3 
Other safety endpoints 
8.4.3.1 
Physical examination 
Results of abnormalities in physical examination will be listed. 
8.4.3.2 
Suicidal risk monitoring 
Suicidal ideation is defined as an event in any of the following 5 categories: 
• 
• 
• 
• 
• 
Suicidal behavior is defined as an event in any of the following 5 categories: 
• 
• 
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• 
• 
• 
Suicidal behavior or ideation is defined as an event in any of the above 10 categories. 
Self-injurious behavior without suicidal intent is corresponding to the response to “Has subject 
engaged in Non-Suicidal Self-Injurious Behavior?” in questionnaire. 
A by-participant listing of the C-SSRS questionnaire data will be provided by treatment group. 
8.4.3.3 
Assessment and management of Tuberculosis (TB) 
By-participant listings of interferon gamma release assay (IGRA) TB test, chest X-ray and TB 
signs and symptoms questionnaire for TB will be provided. 
9 
EFFICACY ANALYSES 
All efficacy analyses will be performed based on the SS by first dose received as described in 
Section 3.6, unless specified otherwise. No statistical testing will be performed for efficacy 
analyses.  
9.1 
Analysis of the secondary efficacy endpoints 
9.1.1 
MG-ADL score 
The complete list of MG-ADL items and scores are provided in Table 13‒3. The total score will 
be calculated according to the rules set down in Section 13.3. 
The MG-ADL total score with associated change from Baseline will be summarized by first dose 
received and scheduled visit using descriptive statistics.  
The above MG-ADL summaries will be repeated for COVID-19 free study participants, where 
COVID-19 free participants for this SAP exclude: 
• Study participants who discontinue treatment due to suspected/confirmed COVID-19 
infection (“Confirmed COVID-19” or “Suspected COVID-19” as the relationship to COVID-
19 in the COVID-19 Impact eCRF); 
• Study participants who discontinue treatment due to non-infection related COVID-19 issues 
(“General circumstances around COVID-19 without infection” or “other” as the relationship 
to COVID-19 in the COVID-19 Impact eCRF); 
• Study participants have visits affected in treatment period (e.g. visit performed by video call) 
due to COVID-19 pandemic. 
The subgroup summaries will also be performed as specified in Section 4.9.  
The MG-ADL total score (excluding ocular items) with associated change from Baseline will be 
summarized by first dose received and scheduled visit using descriptive statistics. 
By-participant listings of MG-ADL values will be provided. 
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9.1.2 
MG-C score  
The complete list of MG-C items and scores are provided in Table 13‒2. The MG-C total score 
and MG-C total score (excluding ocular items) will be calculated according to the rules set down 
in Section 13.2. 
The total scores and total score (excluding ocular items) with associated change from Baseline 
will be summarized by first dose received and scheduled visit using descriptive statistics.  
The subgroup summaries (by MuSK+ or AChR+ only) will also be performed as specified in 
Section 4.9. 
By-participant listings of MG-C scores will be provided. 
9.1.3 
QMG scale  
The complete list of QMG items and scores are provided in Table 13‒1. Partially missing total 
scores will be imputed according to the rules set down in Section 13.1. 
The QMG total score and QMG total score (excluding ocular items) with associated change from 
Baseline will be summarized by first dose received and scheduled visit using descriptive 
statistics.  
The subgroup summaries (by MuSK+ or AChR+ only) will also be performed as specified in 
Section 4.9. 
By-participant listings of QMG scores will be provided. 
9.1.4 
Use of rescue medication (IVIg, PEX) due to worsening 
The use of rescue therapy will be identified as a ‘yes’ response to the “Rescue Medication?” 
question on the Prior and Concomitant Medications CRF and the start date of the rescue therapy 
is on or after Baseline. 
The number of participants in each treatment group with the use of rescue therapy (IVIg, PEX) 
during the Treatment Period due to worsening will be summarized by first dose received.  
9.2 
Analysis of other efficacy endpoints 
9.2.1 
MGII  
The complete list of MGII items and scores are provided in Table 13-8. The total score and sub-
scores will be calculated according to the rules set down in Section 13.5. 
The MGII total score and sub-scores (Ocular and a Generalized domain) with associated change 
from Baseline values will be summarized separately by first dose received and each scheduled 
visit. 
A by-participant listing of MGII scores will be provided. 
9.2.2 
MG Symptoms PRO  
The MG Symptoms PRO scale scores (physical fatigue, muscle weakness fatigability, bulbar 
symptoms) will be calculated according to the rules set down in Section 13.4. The scale scores 
with associated change from Baseline values will be summarized by first dose received and each 
scheduled visit. 
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By-participant listings of MG Symptoms PRO scores will be provided. 
9.2.3 
MG-QOL15r 
The MG-QOL15r total score is calculated by summing all the individual items. See Section 13.6 
for scoring details.  
The MGQOL15r total score and change from Baseline values will be summarized by first dose 
received and each scheduled visit. 
A by-participant listings of MG-QOL15r will be provided. 
9.2.4 
EQ-5D-5L 
The EQ-5D-5L essentially consists of 2 pages: the EQ-5D descriptive system and the EQ visual 
analogue scale (EQ VAS). 
A frequency table will be produced to summarize answers provided to each of the 5 dimensions 
of the EQ-5D descriptive system at each scheduled visit by first dose received. The observed 
values of EQ VAS scores and change from Baseline will be summarized by first dose received 
and each scheduled visit. No imputation will be applied on missing item in EQ-5D descriptive 
system and EQ VAS.  
A by-participant listings of EQ-5D-5L will be provided. 
9.2.5 
MG-ADL responder rate (≥2.0 points improvement from Baseline) 
Participants will be classified as responders at a scheduled visit if the MG-ADL total score is at 
least a 2-point improvement (decrease) from Baseline. Missing data will be imputed using NRI 
per Section 4.2.1. 
The number and percentage of observed responders will be summarized by first dose received 
and scheduled visit for participants who require rescue treatment during the Observation Period 
of MG0003 and subsequently enter MG0004 only.  
9.2.6 
QMG responder rate (≥3.0 points improvement from Baseline)  
Participants will be classified as responders at a scheduled visit if the QMG total score is at least 
a 3-point improvement (decrease) from Baseline. Missing data will be imputed using NRI per 
Section 4.2.1. 
The number and percentage of observed responders will be summarized by first dose received 
and scheduled visit for participants who require rescue treatment during the Observation Period 
of MG0003 and subsequently enter MG0004 only.  
9.2.7 
MG-C responder rate (≥5.0 points improvement from Baseline)  
Participants will be classified as responders at a scheduled visit if the MG-C total score is at least 
a 5-point improvement (decrease) from Baseline. Missing data will be imputed using NRI per 
Section 4.2.1. 
The number and percentage of observed responders will be summarized by first dose received 
and scheduled visit for participants who require rescue treatment during the Observation Period 
of MG0003 and subsequently enter MG0004 only.  
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9.2.8 
Minimum symptom expression 
Minimum Symptom Expression (MSE) is designed to assess how many participants become free 
or virtually free of MG symptoms as measured by achieving an MG-ADL total score of 0 or 1 on 
therapy (Vissing et al. 2020). The total score will be calculated according to the rules set down in 
Section 13.3. 
The number and percentage of participants achieving MSE at scheduled visit will be summarized 
by first dose received.  
10 
PHARMACOKINETICS AND PHARMACODYNAMICS  
All PK and PD analyses described in this section will be performed by first dose received on the 
SS, as described in Section 3.6, unless specified otherwise. 
10.1 
Pharmacokinetics 
Individual plasma concentrations of rozanolixizumab will be summarized by most recent dose 
received and scheduled sampling day using n, arithmetic mean, median, SD, minimum, 
maximum, geometric mean (geomean) with associated 95% CI, and geometric coefficient of 
variation (geoCV) (assuming log-normally distributed data). 
The following rules will apply for PK data listings and summaries: 
• Values below the LLOQ will be reported as BLQ 
• Descriptive statistics of concentrations will be calculated if at most 1/3 of the individual data 
points at a timepoint are missing or are not quantifiable (<LLOQ). Values that are BLQ will 
be replaced by the numerical value of the LLOQ/2 in this instance. If more than 1/3 of the 
individual data points at a timepoint are missing or are not quantifiable, then only n, 
minimum, median and maximum will be presented. The other descriptive statistics will be 
left blank.  
• If n<3, then only the n, minimum and maximum will be presented. If no study participants 
have data at a given timepoint, then only n=0 will be presented. 
• The 95% CI lower and 95% CI upper should be left blank if the SD (or equivalently, the 
geoCV) is 0 
• The geoCV will be calculated using the following formula where SD is the standard 
deviation from the log-transformed data 
 geoCV(%) = √exp(SD2) −1 × 100. 
Individual concentrations of rozanolixizumab will be listed by first dose received for the SS and 
will include the actual sampling time in days relative to the previous dose, most recent dose, the 
IgG observed at the same visit, the ADA titer observed for the binding assay and the NAb titer 
for the same visit, IgG and IgG subclasses, and MG-ADL change from baseline for the 
corresponding visit.   
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10.2 
Pharmacodynamics 
10.2.1 
Total serum IgG and IgG subclasses 
Total serum IgG concentrations and IgG subclasses will be summarized by first dose received 
and time point for observed values, change from Baseline, and percentage change from Baseline.  
The maximum change from Baseline in total serum IgG (absolute and percentage change) will be 
reported in the listing and summarized by first dose received. In the event that a change from 
Baseline in total serum IgG is not observed in a given participant, the maximum change will be 
reported as the smallest increase from Baseline.  
Mean and mean percentage change from Baseline values in total serum IgG will be plotted over 
time by first dose received with dose groups overlaid on the same plot.  
Spaghetti plots will be provided for absolute IgG and percentage change from baseline in IgG 
over time stratified by first dose received and Baseline bodyweight group (<50kg, 50kg-<70 kg, 
70-<100kg, ≥100kg) where the Baseline bodyweight group is multipaneled or overlaying with 
different colors within each treatment group. 
Serum concentrations of total IgG and IgG subclasses will be listed together with concentrations 
of rozanolixizumab, ADA and NAb sample status and titer, MG-specific autoantibodies, and 
MG-ADL change from Baseline, as specified in Section 10.3.2.  
10.2.2 
MG-specific autoantibodies 
MG-specific autoantibodies (anti-MuSK/anti-AChR) will be summarized by first dose received 
and RLZ total at scheduled visits for observed values, absolute and percentage changes from 
Baseline. The maximum change from Baseline in MG-specific autoantibodies (absolute and 
percentage change) will be reported in the listing and summarized for each treatment. 
MG-specific autoantibodies will also be summarized at scheduled visit by the participants who 
are anti-MuSK or anti-AChR autoantibody-positive at the Baseline visit. 
Mean absolute and mean percentage change from Baseline values in MG-Specific autoantibodies 
will be plotted over time by treatment group and RLZ total. Additionally, mean percentage 
change from Baseline in anti-MuSK, total IgG, MG-ADL, QMG, and MG-C will be plotted by 
first dose received. The same plot will be repeated for percentage change from Baseline values in 
anti-AChR. 
MG-specific autoantibodies will be listed, together with total IgG, MG-ADL, QMG, MG-C 
change from Baseline. 
10.3 
Anti-drug antibody and neutralizing antibody (NAb) status 
For the immunogenicity assessment, two types of anti-drug antibody (ADA) assays will be 
performed, a binding anti-drug antibody assay and a neutralizing antibody assay.  
For the binding assay, the ADA of rozanolixizumab will be measured using a three-tiered assay 
approach: screening assay, confirmatory assay and titration assay. Any sample confirmed 
positive for the binding ADA assay will be tested in the neutralizing assay, which produces a 
positive or negative result.  
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Evaluation of rozanolixizumab immunogenicity will be performed using data from all evaluable 
study participants in the SS, defined as all study participants who have an evaluable pretreatment 
(baseline) sample (negative or positive ADA sample status), and at least 1 evaluable post-
baseline value.  
Study participants with an evaluable pretreatment (baseline) sample but without a single 
evaluable sample taken post-baseline will be included in the reporting of pre-existing ADA but 
excluded from all other immunogenicity analyses. 
10.3.1 
Data consideration 
ADA Sample Status 
The ADA sample status will be determined for each pre-treatment (Baseline) and post-treatment 
(post-Baseline) visit where samples are taken for ADA analysis. 
• Sample values that are either ‘negative screen’ or ‘positive screen’ and ‘negative 
immunodepletion’ will be defined as ADA negative if corresponding rozanolixizumab 
concentrations are equal or below the validated drug tolerance limit of the ADA assay 
(200µg/mL rozanolixizumab) allowing detection of 100ng/mL ADA 
• Sample values that are either ‘negative screen’ or the combination of ‘positive screen’ 
and ‘negative immuno-depletion’, but with corresponding rozanolixizumab 
concentrations above the validated drug tolerance limit of the ADA assay, will be defined 
as ADA inconclusive  
• Sample values that are ‘positive screen’ and ‘positive immunodepletion’ will be defined 
as ADA positive 
• Samples that could not be tested for ADA status due to inadequate sample volume, 
mishandling, or errors in sample collection, processing, storage, etc., will be defined as 
Missing. 
Note: if a participant has no baseline ADA, then she/he can’t be treated as "Inconclusive". 
Neutralizing antibody (NAb) sample status (positive/negative/missing) will be determined for 
ADA positive samples. Samples that are NAb positive will be evaluated in a titration assay to 
quantify the NAb level and will be reported as titer. 
Definition of ADA Baseline 
Baseline for ADA should be the last measurement prior to receiving the very first 
rozanolixizumab (RLZ) infusion in either MG0004 or the feeder study MG0003. Specifically, 
baseline for ADA is defined as: 
• MG0003 ADA baseline if participants randomized to RLZ groups in MG0003 (note: 
MG0003 post-baseline ADA samples will not be considered for the ADA participant status 
in MG0004); 
• MG0004 ADA baseline if participants randomized to placebo in MG0003. 
If the ADA sample status for the visit prior to the first dose of RLZ is missing, the last 
non-missing sample will be used. 
 
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ADA/NAb Participant Status 
The ADA participant status will be classified on study participant and group level as outlined 
below (Shankar et al. 2014; Rup et al, 2015). A description of how study participants will be 
categorized for the immunogenicity assessment is provided in  
Table 10-1.  
Individual study participants will be assessed for ADA participant status, composed of 6 
categories: ADA negative, inconclusive, and ADA positive, whereby a positive participant’s  
status is determined as originating from a treatment-induced, boosted, reduced or unaffected 
ADA response. 
Study participants who are identified as being treatment-induced or treatment-boosted ADA-
positive will be grouped as treatment-emergent (TE)-ADA positive participants. Study 
participants who are identified as being treatment-reduced or treatment-unaffected ADA-positive 
will be grouped as non-TE-ADA positive participants. Both TE-ADA positive and non-TE–ADA 
positive participants will be further classified as NAb negative or NAb positive.  
The individual and combined ADA participant categories will be derived and summarized 
through each of the scheduled sampling points (ie Week 5, 9, 21, 33, 45, 52 and 60) and EOS 
visit, unless specified otherwise.  
Table 10-1: Terms and Definitions for ADA Status Evaluation in Study Participant 
Classification Classification Label 
Definition 
Individual participant categories 
1 
Pre-ADA negative – treatment 
induced ADA negative (ADA-
NEG) 
Study participants who have an ADA negative 
sample at Baseline and at all sampling points post-
Baseline up to the timepoint of interest. 
2 
Inconclusive 
Study participants who have an ADA positive or 
negative Baseline sample and some post-Baseline 
samples are missing or inconclusive, while other 
post-Baseline samples are ADA negative up to the 
timepoint of interest. 
3 
Pre-ADA negative – treatment 
induced ADA positive (TI-
POS) 
Study participants who have an ADA negative 
sample at Baseline and have at least one ADA 
positive sample at any sampling point post-Baseline 
up to the timepoint of interest. 
4 
Pre-ADA positive – treatment 
boosted ADA positive (TB-
POS) 
Study participants who have an ADA positive 
sample at Baseline and at least one ADA positive 
sample at any sampling point post-Baseline up to the 
timepoint of interest, with increased titer values 
compared to Baseline (greater than a predefined fold 
difference increase from Baseline value which will 
be defined within the validation of the assay i.e. 
MSR of the assay). 
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5 
Pre-ADA positive – treatment 
reduced ADA positive (TR-
POS) 
Study participants with an ADA positive sample at 
Baseline, and ADA negative samples at all sampling 
points post-Baseline up to the timepoint of interest. 
6 
Pre-ADA positive – treatment 
unaffected ADA positive (TU-
POS) 
Study participants with an ADA positive sample at 
Baseline and an ADA positive sample at any 
sampling point post-Baseline up to the timepoint of 
interest, with titer values of the same magnitude as 
Baseline (less than a predefined fold difference from 
the Baseline value which will be defined within the 
validation of the assay, i.e. MSR of the assay1). 
Combined participant categories 
7 
Treatment emergent ADA 
positive (TE-POS) 
Includes study participants who are treatment 
induced ADA positive (category 3) or treatment 
boosted ADA positive (category 4). 
8 
Non-treatment emergent ADA 
positive (Non-TE-POS) 
Includes study participants who are treatment 
reduced ADA positive (category 5) or treatment 
unaffected ADA positive (category 6). 
9 
Treatment emergent ADA 
positive – NAb positive (TE-
POS, NAb-POS) 
Includes study participants who are treatment 
emergent positive (category 7) and have at least one 
NAb positive sample. 
10 
Treatment emergent ADA 
positive – NAb negative (TE-
POS, NAb-NEG) 
Includes study participants who are treatment 
emergent positive (category 7) and have no NAb 
positive samples. 
11 
Non-treatment emergent ADA 
positive - NAb positive (Non-
TE-POS, NAb-POS) 
Includes study participants who are non-treatment 
emergent positive (category 8) and have at least one 
NAb positive sample. 
12 
Non-treatment emergent ADA 
positive - NAb negative (Non-
TE-POS, NAb-NEG) 
Includes study participants who are non-treatment 
emergent positive (category 8) and have no NAb 
positive samples. 
1 The fold difference increase from baseline value, i.e. the minimum significant ratio (MSR=1.36) determined 
during assay validation, will be reported in the relevant tables, listings and figures. It reflects the fold difference in 
titer level that considered higher than the assay variation in titer determination. 
10.3.2 
ADA summaries 
The following outputs will be presented on the SS. 
Tables: 
• Number and percentage of participants with ADA (positive, negative, inconclusive, missing) 
and NAb (positive, negative, missing) sample status at the time of each visit will be 
summarized by first dose received and RLZ total. Denominator is the number of study 
participants having a non-missing result at that visit.  
• Number and percentage of participants in each of the individual and combined ADA 
participant status categories presented in Table 10-1 will be summarized by first dose 
received and RLZ total at each scheduled visit and EOS visit. The table will be repeated by 
prior treatment in MG0003 (RLZ total or placebo) up to Week 9. 
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• Total prevalence of pre-existing ADA and NAb, defined as number and percentage of 
participants having an ADA positive sample status at baseline, with the denominator being 
the total number of study participants having an evaluable sample result at baseline. Missing 
samples will not be included in the denominator. The table will be repeated by prior 
treatment in MG0003 (RLZ or placebo). The same will be repeated for NAb. 
• The first occurrence of treatment-emergent ADA positivity: cumulative number and 
percentage of TE-ADA positive participants (category 7) who are ADA positive for the first 
time at each visit. The table will be repeated by prior treatment in MG0003 (RLZ or 
placebo). 
• Summary table of mean maximum percentage CFB in total IgG and mean CFB in MG-ADL, 
summarized by ADA participant categories 1, 2, 9, 10, 11 and 12 for the corresponding time 
period of interest and by treatment group and RLZ total 
.  
• Overall summary table of TEAEs at Week 9 and EOS summarized by ADA participant 
categories 1, 2, 7 and 8 for the corresponding time period of interest. 
• Summary of incidence of TEAE at Week 9 and EOS by ADA participant category 1, 2, 7 and 
8 for the corresponding time period of interest. 
• Summary table of AEOF hypersensitivity reactions, anaphylactic reactions and infusion 
reactions at Week 9 and EOS by ADA participant category 1, 2, 7 and 8 for the 
corresponding time period of interest. 
Figures: 
• Individual time course plots for ADA positive study participants with at least one ADA 
positive sample, representing ADA and NAb titers (on log-scale), percentage CFB for total 
IgG and MG-specific autoantibodies, and CFB for MG-ADL total score. The sub-title of the 
graph will include the study participant number, bodyweight category, treatment group 
(including prior treatment group in MG0003), and individual ADA participant category (3, 4, 
5, 6) up to EOS. The dosing will be represented in the x-axis with bars/arrows at the time of 
dose. 
• Spaghetti plots of individual time course of ADA titer for ADA positive participants in 
categories 9, 10, 11 and 12 (determined through EOS), whereby all categories are overlaid on 
the same plot and visualized using different symbols and colors. The ADA titer results will 
be presented on a log-scale. Dosing time points should be indicated below the x-axis. The 
same plot will be repeated for NAb titer for NAb positive participants in categories 9 and 11. 
• A box-and-whisker plot of maximum postdose ADA titer (on log-scale) through Week 5, 
Week 9, Week 52 and EOS versus ADA participant category for categories 9, 10, 11 and 12 
(determined for the corresponding time period of interest). The same plot will be repeated for 
NAb titer for ADA participant categories 9 and 11. 
• Time course plot of mean CFB in MG-ADL total score, summarized by ADA participant 
category 1, 2, 9, 10, 11 and 12. Categories will be determined for the time period from 
baseline up to timepoint of interest (Week 5, 9, 52 and EOS). Separate plots for each 
treatment group and RLZ total 
.  
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• Spaghetti plots of individual time course of percentage CFB for total IgG, for each of the 
ADA participant categories 1, 2, 9, 10, 11 and 12 and time period of interest (Baseline up to 
Week 5, Week 9, and EOS) whereby the ADA participant categories are multipaneled. 
Separate plots for each treatment group and RLZ total 
. Individual 
samples that tested positive for ADA will be visualized using a symbol and/or color. Dosing 
time points should be indicated below the x-axis.  
• Scatter dot plot of individual CFB in MG-ADL total score categorized by ADA titer tertile 
(including category ADA not present) for each scheduled assessment (Week 5, 9, 21, 33, 45, 
52, and 60). The same plot will be repeated for NAb titer. 
• Scatter dot plot of individual percentage CFB for total IgG categorized by ADA titer tertile 
(including category ADA not present) for each scheduled assessment (Week 5, 9, 21, 33, 45, 
52, and 60). The same plot will be repeated for NAb titer. 
Listing: 
• By-subject listing by treatment group and timepoint, of ADA and Nab sample status, ADA 
titer, NAb titer, rozanolixizumab plasma concentration, percentage CFB for total IgG and 
IgG subclasses, CFB for MG-specific autoantibodies, CFB for MG-ADL total score, QMG 
and MG-C. In addition, the time since administration of IMP will be reported (in days). 
 
10.4 
Immunology 
All analyses described in this section will be based on the SS. 
10.4.1 
Serum complement levels and plasma complement levels 
Serum (C3 and C4) and plasma (C3a and C5a) complement variables will be listed by treatment 
group, visit and time point including changes from Baseline. Descriptive summaries will be 
presented for both absolute values and changes from Baseline for participants who experience a 
severe headache, infusion reaction or hypersensitivity reaction. The Baseline values are defined 
as Section 3.3. 
Measurements BLQ will be imputed with half of the LLOQ for the purpose of calculating 
change from Baseline. Measurements above the limit of quantification (ALQ), if applicable, will 
be imputed to the upper limit of quantification. 
Serum and plasma complement results will be listed. 
10.4.2 
Cytokines 
Cytokines will be summarized by treatment group, visit and time point for both absolute values 
and changes from Baseline for participants experiencing with severe headache, infusion reactions 
or hypersensitivity reaction. The Baseline values are defined as Section 3.3. 
Measurements BLQ will be imputed with half of the LLOQ for the purpose of calculating 
change from Baseline. Measurements ALQ, if applicable, will be imputed to the upper limit of 
quantification. 
Cytokines results will be listed. 
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10.4.3 
Serum immunoglobulin concentrations 
Immunoglobulins (IgE, IgA and IgM) will be summarized by treatment group and visit for 
absolute values, change from Baseline, and percentage change from Baseline. 
Measurements BLQ will be imputed with half of the LLOQ for the purpose of calculating 
change from Baseline. Measurements ALQ, if applicable, will be imputed to the upper limit of 
quantification. 
Immunoglobulins (IgE, IgA, and IgM) will be listed. 
10.4.4 
Anti-tetanus toxoid serum titers 
Anti-tetanus toxoid serum titers will be summarized by treatment group and visit for both 
absolute values, change from Baseline, and percentage change from Baseline. Same summary 
will be repeated excluding all participants with undetectable baseline titers. 
The mean change from Baseline in anti-tetanus toxoid serum titers versus time at each scheduled 
timepoint will be plotted by treatment group excluding all participants with undetectable baseline 
titers. Mean change from Baseline in IgG values will be overlaid on the same plot. 
Measurements BLQ will be imputed with half of the LLOQ for the purpose of calculating 
change from Baseline. Measurements ALQ, if applicable, will be imputed to the upper limit of 
quantification. 
Anti-tetanus toxoid serum titers will be listed. 
11 
OTHER ANALYSES  
11.1 
Dose change in use of concomitant medications over time 
The following summaries will be produced using SS in all participants: 
• The number and percentage of participants with dose change in use of steroids (ATC3 code: 
), immunosuppressants (ATC3 code: 
) or AChE Inhibitor (ATC3 code: 
) 
during the study;  
• Average daily dose (ADD) by PT of medications at Baseline and last study visit (i.e. 
PEOT/EOS visit); 
Note: ADD will be calculated for each PT according to the dose and dose frequency on Prior 
and Concomitant Medications CRF (e.g. ADD of a participant taking steroid 20 mg QOD is 
10 mg). Baseline ADD will follow the same Baseline definition in Section 3.3, which will be 
calculated using the steroid dose taken prior to first IMP in MG0004.  ADD at last study visit 
is calculated using the dose taken at EOS/PEOT visit. In the case last dose is stopped before 
EOS/PEOT visit, ADD at last study visit will be counted as zero.  
• The change from Baseline of average daily dose (ADD) at last study visit, calculated as 
ADD at last study visit – Baseline ADD; 
• A listing will be provided for participants with dose change of steroids, including MG-ADL 
total score at Baseline and last study visit (EOS/PEOT visit), last MG-ADL score prior to 
first steroid dose change, maintenance dose of RLZ, Baseline steroid ADD and steroid ADD 
at last study visit. 
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Note: Maintenance dose of RLZ is the most recent dose of RLZ when last steroid dose is 
given. 
11.2 
Specific analyses for Pharmaceuticals and Medical Devices 
Agency (PMDA) 
The following endpoints will be summarized for participants in Japan only: 
• Study participant characteristics, including important PDs (as specified in Section 5) 
• Demographics and other baseline characteristics, including medical history, prior and 
concomitant medications (as specified in Section 6) 
• Study IMP duration and number of infusions (as specified in Section 8.1) 
• Use of rescue medication (IVIg, PEX) due to worsening (as specified in Section 9.1.4) 
• MG-ADL total score and change from Baseline (as specified in Section 9.1.1) 
• TEAE Summaries (as specified in Section 8.2.2) 
− TEAE overview  
− The number, percentage of participants and frequency of the following TEAEs will be 
summarized by SOC, HLT, PT, and by most recent dose prior to onset of TEAEs and 
RLZ total 
◦ 
Any TEAEs 
◦ 
Any TEAEs by maximum intensity (mild, moderate and severe) 
◦ 
Any TEAEs by relationship 
◦ 
Serious TEAE 
◦ 
Participant discontinuation due to TEAEs  
◦ 
Permanent withdrawal of IMP due to TEAEs 
• ADA summaries (as specified in Section 10.3.2): 
− Number and percentage of participants with positive, negative, missing or inconclusive 
sample ADA status at the time of each visit and RLZ total will be summarized by 
treatment group and RLZ total. 
− Number and percentage of participants in each of the ADA classifications presented in 
Table 10-1 will be summarized by treatment group and RLZ total. 
11.3 
Headache questionnaire 
The results of the headache questionnaire will be listed for each study participant using ES. No 
summary tabulations will be provided for these assessments. 
11.4 
Myasthenia Gravis Foundation of America (MGFA) by Visit 
The results of MGFA by visit will be listed for each study participant using RS. No summary 
tabulations will be provided for these assessments. 
 
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12 
REFERENCES 
Myasthenia Gravis Symptoms PRO Instrument Scoring Manual. Modus Outcomes, 2019 
United States Department of Health and Human Services, National Cancer Institute. Common 
Terminology for Adverse Events (CTCAE), Version 5.0, November 17, 2017. 
Levey et al. A New Equation to Estimate Glomerular Filtration Rate. Ann Intern Med. 2009; 
150: 604-612. 
Nehring, S.M.; Goyal, A.; Patel, B.C. C Reactive Protein (CRP) (2020). StatPearls Publishing, 
web link: https://www.ncbi.nlm.nih.gov/books/NBK441843/.  
Rup B, Pallardy M, Sikkema D, et al. Standardizing terms, definitions and concepts for 
describing and interpreting unwanted immunogenicity of biopharmaceuticals: recommendations 
of the Innovative Medicines Initiative ABIRISK consortium. Clin Exp Immunol. 2015 
Sep;181(3):385-400.  
Shankar G, Arkin S, Cocea L, et al. Assessment and reporting of the clinical immunogenicity of 
therapeutic proteins and peptides-harmonized terminology and tactical recommendations. AAPS 
J. 2014 Jul;16(4):658-73.Vissing, J., Jacob, S., Fujita, K. P., O’Brien, F., & Howard, J. F. (2020). 
‘Minimal symptom expression’in patients with acetylcholine receptor antibody-positive 
refractory generalized myasthenia gravis treated with eculizumab. Journal of neurology, 1-11. 
 
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13 
APPENDICES 
13.1 
Quantitative Myasthenia Gravis scale 
The QMG scale comprises 13 items, including ocular and facial movement, swallowing, speech, 
limb strength and forced vital capacity. Scoring for each item ranges from no weakness (0) to 
severe weakness (3), with an overall score range from 0 to 39, ie, a higher score indicates more 
severe disease. A 3-point change in the overall score is considered to be clinically relevant. 
The QMG testing form is provided in Table 13‒1. The total score is obtained by summing the 
responses to each individual item. 
 
 The score ranges from 0 to 33. 
In the event of missing data, the following rules will be applied: 
• If 1 or 2 items are not answered, the overall score will be obtained by imputing the missing 
items with the average score across the remaining items at the specific visit. The imputed 
value will be rounded to one decimal place 
• If more than 2 items are missing the overall score will not be calculated at the specific visit 
Table 13‒1: Quantitative myasthenia gravis testing form 
Item 
None 
Mild 
Moderate 
Severe 
Grade 
0 
1 
2 
3 
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Item 
None 
Mild 
Moderate 
Severe 
Grade 
0 
1 
2 
3 
F=female; M=male. 
13.2 
Myasthenia Gravis-Composite scale 
The MG-C score items and associated scores are provided in Table 13‒2.  
The MG-C score comprises 10 items, each of which is weighted differently in the calculation of 
the overall score. The overall score ranges from 0 to 50, with a higher score indicating more 
severe disease. 
The total score is obtained by summing the responses to each individual item.  
 
 The score ranges from 0 to 41. 
In the event of missing data at a particular visit, the MG-C score will not be calculated. Due to 
the different weighting applied to each item it is not possible to impute the missing data with the 
average score across the remaining items. 
Table 13‒2: MG-C score items and scoring algorithm 
Item 
Result/Grade 
Result/Grade 
Result/Grade 
Result/Grade 
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a Moderate weakness for head and neck items should be construed as weakness that equals roughly 50%+/-15% of 
expected normal strength. Any weakness milder than that would be mild and any weakness more severe than that 
would be classified as severe. 
13.3 
Myasthenia Gravis-Activities of Daily Living 
The MG-ADL score comprises 8 items, each with a score of 0 to 3. The total score is obtained by 
summing the responses to each individual item. Thus, the score ranges from 0 to 24 with a higher 
score indicating more disability. The MG-ADL testing form is provided in Table 13‒3. The total 
score is obtained by summing the responses to each individual item. Thus, the score ranges from 
0 to 24.  
In the event of missing data, the following rules will be applied: 
• If 1 or 2 items are not answered, the overall score will be obtained by imputing the missing 
items with the average score across the remaining items at the specific visit. The imputed 
value will be rounded to one decimal place 
• If more than 2 items are missing the overall score will not be calculated. 
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Table 13‒3: MG-Activities of Daily Living 
13.4 
Myasthenia Gravis Symptoms PRO 
The MG Symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms 
(items 1 to 5); bulbar symptoms (items 6 to 15); respiratory symptoms (items 16 to 18); physical 
fatigability (items 19 to 33) and muscle weakness fatigability (items 34 to 42).  
The MG Symptoms PRO calculation includes two steps: 1) rescoring the item responses; and 2) 
calculation of the final scores.  Details of the scoring is found in the Myasthenia Gravis 
Symptoms PRO Instrument Scoring Manual dated 28 February 2019. 
Step 1: Rescoring the item responses. The item responses should be rescored so as the lowest 
item-level score is 0.  Rescoring rules are provided in Table 13-4, Table 13-5, and Table 13-6.  
Step 2: Calculation of the MG Symptoms PRO Scores 
• MG Symptom PRO scale scores 
The MG symptom PRO scale scores are calculated using the formula below, i.e. the sum of item 
scores is linearly transformed to have all domain scores ranging from 0 to 100: 
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𝑀𝐺 𝑆𝑦𝑚𝑝𝑡𝑜𝑚 𝑃𝑅𝑂 𝑠𝑐𝑎𝑙𝑒 𝑠𝑐𝑜𝑟𝑒
= 𝑆𝑢𝑚 𝑜𝑓 𝑖𝑡𝑒𝑚 𝑠𝑐𝑜𝑟𝑒𝑠 𝑤𝑖𝑡ℎ𝑖𝑛 𝑡ℎ𝑒 𝑠𝑐𝑎𝑙𝑒
𝑅𝑎𝑤 𝑠𝑐𝑜𝑟𝑒 𝑟𝑎𝑛𝑔𝑒
×
𝑇𝑜𝑡𝑎𝑙 𝑛𝑢𝑚𝑏𝑒𝑟 𝑜𝑓 𝑖𝑡𝑒𝑚𝑠 𝑖𝑛 𝑡ℎ𝑒 𝑠𝑐𝑎𝑙𝑒
𝑁𝑢𝑚𝑏𝑒𝑟 𝑜𝑓 𝑛𝑜𝑛 𝑚𝑖𝑠𝑠𝑖𝑛𝑔 𝑖𝑡𝑒𝑚𝑠 𝑖𝑛 𝑡ℎ𝑒 𝑠𝑐𝑎𝑙𝑒× 100 
The score for each scale is calculated only when at least 70% of the items are completed.  Details 
on the minimum number of items needed for score calculation and the range of raw score are 
provided in Table 13-7. 
 
Table 13-4: MG Symptoms: Ocular, Bulbar, and Respiratory Symptoms 
Rescoring 
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Table 13-4: MG Symptoms: Ocular, Bulbar, and Respiratory Symptoms 
Rescoring 
Table 13-5: MG Symptoms: Physical Fatigue Rescoring 
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 Table 13-6: MG Symptoms: Muscle Weakness Fatigability 
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Table 13-7: Description of the MG Symptoms PRO Scale Scores 
13.5 
Myasthenia Gravis Impairment Index 
The MG-II is provided in Table 13-8. The MGII can be summarized as a total score and also as 2 
sub-scores reflecting an Ocular and a Generalized domain. 
The total score is the raw sum of all the items, including the clinical examination and the patient-
reported questionnaire. 
The ocular score is calculated by summing 8 items reflecting ocular impairments. These items 
are: patient questionnaire items 1 to 6 and examination items 1 and 2. 
The generalized score is calculated by adding items 7 to 22 from the patient questionnaire and 
items 3 to 6 from the examination. 
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In the event of missing data, the following rules will be applied: 
• In the case where at least 7 of the 8 items from the Ocular subscale are answered, the Ocular 
subscale score will be generated after imputing the missing response by the average of non-
missing responses, by adding all item scores. If there are 2 or more missing data out of the 8 
Ocular subscale items, then the Ocular subscale score will be missing. 
• In the case where at least 18 of the 20 items from the Generalized subscale are answered, the 
Generalized subscale score will be generated after imputing the missing response(s) by the 
average of non-missing responses, by adding all item scores. If there are 3 or more missing 
data out of the 20 Generalized subscale items, then the Generalized subscale score will be 
missing. 
• In the case where both the Ocular and the Generalized subscale scores are non-missing, then 
the Total score will be calculated as the sum of the Ocular and the Generalized subscale 
scores. If one of the two subscale scores are missing, then the Total score will be missing. 
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Table 13-8: MG Impairment Index 
 
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Table 13-8: MG Impairment Index 
 
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Table 13-8: MG Impairment Index 
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Table 13-8: MG Impairment Index 
 
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Table 13-8: MG Impairment Index 
 
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13.6 
MG-QOL15r 
The MG-QOL15r is a brief survey, completed by the study participant, that is designed to assess 
some aspects of "quality of life" related to MG. The total score is calculated by summing all 15 
individual items.  
In the event of missing data, the following rules will be applied: 
• In the case where at least 70% of the items (i.e. 11 items out of the 15) are answered, the total 
score will be generated after imputing the missing responses by the average of available (ie 
non-missing) responses, by adding all item scores. 
• In the case where more than 30% (i.e. 5 items or more) are missing, the total score will not be 
generated.  
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Table 13-9: MG-QOL15r 
 
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13.7 
Markedly abnormal laboratory and diagnostic criteria for 
Rozanolixizumab program 
The following criteria will be applied in the determination of marked abnormalities for 
laboratory assessment values. They are based on Version 5 of the Common Terminology Criteria 
for Adverse Events (CTCAE) grade 3 or higher criteria unless otherwise noted. If both high and 
low criteria are shown for a parameter, the criteria should be summarized separately in tabular or 
graphical data summaries.   
Table 13-10: Hematology 
Parameter 
Unit 
(conventional) 
Unit 
(standard) 
Marked Abnormality 
Criteria 
Hemoglobin  
g/dL 
 
g/L 
<8.0 g/dL; <80 g/L  
WBC (Leukocytes)1 
109/L 
109/L  
Low: <2.0 x 109/L  
High:  >30 x 109/L  
Lymphocytes 
Absolute 
109/L 
109/L  
Low:  <0.5 x 109/L  
High: >20 x 109/L 
Neutrophils Absolute  
109/L 
109/L  
 <1.0 x 109/L   
Platelets 
109/L 
109/L  
<50.0 x 109/L  
1WBC (Leukocytes) markedly abnormal high criterion is not based on Version 5 CTCAE Grade 3 or higher criteria. 
Due to the mechanism of action of RLZ, the safety alert is related to infection risk which would be identified by a 
lower cut-point than the standard which is related to acute leukemias. A markedly abnormal high cut-point >30 x 
109/L is applied to flag leukocytosis (George 2012). 
 
Table 13-11: Chemistry 
Parameter 
Unit 
(conventional) 
Unit 
(standard) 
Marked Abnormality Criteria 
AST (SGOT) 
U/L 
U/L 
>5.0 x ULN  
ALT (SGPT) 
U/L 
U/L 
>5.0 x ULN  
ALP (Alkaline 
Phosphatase) 
U/L 
U/L 
>5.0 x ULN  
GGT (Gamma 
Glutamyl 
Transferase) 
U/L 
U/L 
>5.0 x ULN  
Bilirubin (Total) 
mg/dL 
umol/L 
>3.0 x ULN if Baseline value is normal; 
>3.0 x Baseline value if Baseline is 
abnormal  
Albumin 
g/dL 
g/L 
<2 g/dL; <20 g/L  
Creatinine 
mg/dL 
umol/L 
>3.0 x ULN 
Estimate 
glomerular 
filtrate rate 
(eGFR)1 
mL/min/1.73 
m2 
mL/min/1.73 
m2 
eGFR <29 mL/min/1.73 m2 
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C reactive 
protein (CRP)2 
mg/L 
mg/L 
>10 mg/L 
Calcium3 
mg/dL 
mmol/L 
Low: Corrected serum calcium of <7.0 
mg/dL; <1.75 mmol/L 
High: Corrected serum calcium of >12.5 
mg/dL; >3.1 mmol/L 
Immunoglobulin 
G4  
(g/L) 
(g/L) 
≤1 g/L 
Potassium 
mmol/L 
mmol/L 
Low: <2.5 mmol/L 
High: >6.0 mmol/L 
Sodium 
mmol/L 
mmol/L 
Low: <125 mmol/L  
High: >155 mmol/L 
Glucose5 
mg/dL 
mmol/L 
Low: <40 mg/dL; <2.2 mmol/L  
High: > 250 mg/dL; >13.9 mmol/L 
Total 
Cholesterol 
mg/dL 
mmol/L 
>400 /dl; >10.34 mmol/L  
Triglycerides 
mg/dL 
mmol/L 
>500 mg/dl; >5.7 mmol/L 
Abbreviations: ALT= alanine aminotransferase; AST = aspartate aminotransferase; dL = deciliter; GGT: gamma-
glutamyltransferase; L = liter; mg = milligram; mmol = millimoles; μg = microgram; U = unit; ULN = upper limit 
of normal 
Note: Marked abnormality criteria are defined by Grade 3 or higher events according to the Common Terminology 
for Adverse Events (CTCAE), Version 5.0, November 17, 2017 unless otherwise noted.  
1eGFR is calculated using the Chronic Kidney Disease Epidemiology Collaboration or CKD-EPI formula 
(https://qxmd.com/calculate/calculator_251/egfr-using-ckd-epi) which is eGFR = 141 * min(Scr/κ,1)α * 
max(Scr/κ, 1)-1.209 * 0.993Age * 1.018 [if female] * 1.159 [if black];  where Scr is serum creatinine (mg/dL), κ is 0.7 
for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/κ or 
1, and max indicates the maximum of Scr/κ or 1. For derivation from values in standard units (umol/L) the κ 
values are 61.88 for females and 79.56 for males. 
2Includes CRP and High Sensitivity (HS) CRP. Reference for marked abnormality criteria: Nehring, S.M.; Goyal, 
A.; Patel, B.C. (2020). StatPearls Publishing, web link: https://www.ncbi.nlm.nih.gov/books/NBK441843/. 
3Corrected Calcium (mmol/L) =0.02 * (40 – Albumin (g/L)) + Calcium (mmol/L). 
4Immunoglobulin G criterion based on immunodeficiency literature and noted in RLZ study protocols. 
5Glucose high criterion defined by Grade 3 and higher events according to CTCAE, Version 4.03, June 14, 2010. 
 
Table 13-12: Vital Signs 
Parameter 
Abnormality Criteria 
Pulse Rate (beats/minute) 
 
<50 and a decrease from Baseline of ≥15 
>120 and an increase from Baseline of ≥15 
Systolic Blood Pressure (mmHg) 
≤ 90 and a decrease from Baseline of ≥20 
≥ 180 and an increase from Baseline of ≥20 
Diastolic Blood Pressure (mmHg) 
<50 and a decrease from Baseline of ≥15 
>105 and an increase from Baseline of ≥ 15 
Temperature 
>101 0F (38.3 0C) 
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Parameter 
Abnormality Criteria 
Body Weight 
≥ 10% decrease from Baseline 
≥ 10% increase from Baseline 
 
Table 13-13: Electrocardiogram 
Parameter 
Abnormality Criteria 
QT interval (ms) 
 
>500ms 
≥60ms increase from Baseline 
QTc(F) (ms) 
>500ms 
≥60ms increase from Baseline 
PR interval (ms) 
Treatment-emergent value >200ms 
QRS interval (ms) 
Treatment-emergent value >100ms 
Heart rate (bpm) 
<50bpm 
>120bpm 
Abbreviations: bpm = beats per minute; ms = milliseconds; QTc(F) = Fridericia corrected QT interval;  
Note: Treatment-emergent is defined as meeting the criteria at any post-Baseline visit after the first infusion of study 
medication and within 56-days of the last infusion and not meeting the same criteria during Baseline 
13.8 
AEs of focus for Rozanolixizumab program 
The AEOF selection criteria is specified in the Rozanolixizumab Safety AEs of Focus document 
developed by UCB. The purpose of this document is to detail the approach to identifying TEAEs 
meeting criteria for AEOF for the Rozanolixizumab (also called RLZ) program.  
Following Events are AEOFs for Rozanolixizumab for MG studies: 
No 
Event (also included in 
Title of TFL output) 
Selection criteria 
1 
Headache 
(Note: also included in 
AESM if severe) 
TEAE with HLGT=’Headaches’ 
2 
Gastrointestinal 
disturbances 
(Note: also included in 
AESM if severe) 
TEAE with  
HLT=‘Gastrointestinal and abdominal pains (excl oral 
and throat)’ or  
HLT=‘Gastrointestinal signs and symptoms NEC’ or   
HLT=‘Nausea and vomiting symptoms’ or 
HLT=‘Diarrhoea (excl infective)‘ or 
HLT=‘Gastritis (excl infective)’ 
3 
Hypersensitivity reactions  
 
SMQ=’Hypersensitivity’ 
4 
Anaphylactic reactions 
SMQ=‘Anaphylactic reaction’ and  
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TEAEs that either emerged on the same day as when a 
study medication injection reaction was received, or that 
emerged one day after a study medication injection was 
received, and which fulfill any of the following 3 criteria 
should be included in the summary table: 
 
1. If a subject reports any TEAE which codes to a PT 
included in Category A, then the event will be 
flagged as an anaphylactic reaction. 
 
2. If a subject reports any TEAE which codes to a PT 
included in Category B AND reports any TEAE 
which codes to a PT included in Category C, and 
both TEAEs have the same start date, then both 
events will be flagged as anaphylactic reactions.  
 
3. If a subject reports any TEAE which codes to a PT 
included in Category D AND reports (either a TEAE 
which codes to a PT included in Category B OR a 
TEAE which codes to a PT included in Category C), 
and both TEAEs have the same start date, then 
both events will be flagged as anaphylactic reactions. 
 
5 
Injection site reactions 
TEAE with 
HLT=’Injection site reactions’ or 
HLT=’Infusion site reactions’ or 
HLT=’Administration site reactions NEC’ 
6 
Infusion Reactions  
Infusion reaction marked on AE CRF page 
(based on the assessment by the Investigator) 
7 
Infections 
TEAE with SOC =”Infections and infestations”  
Note: This was added as a reminder for safety that 
infections are considered as AE of focus and require 
assessment. No programming of this topic is required as 
TEAEs can be found in general AE Tables.  
8 
Opportunistic infections 
(Note: also included in 
AESM) 
Opportunistic infections (including tuberculosis) will be 
summarized in a stand-alone table using UCB-defined 
search criteria. Opportunistic infections are identified in 
two steps using the attached spreadsheet 
for MedDRA v24.0 in UCB Rozimab Safety AEs of 
Focus document: 
Step 1: Refer to column B of the spreadsheet which 
identifies the Preferred Terms (PTs) to be classified as 
opportunistic infections using either a single ‘x’ or a 
double ‘xx’. 
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• TEAEs which code to a PT flagged with a single 
‘x’ need to also be serious in order to be 
considered an opportunistic infection.  
• All TEAEs which code to a PT flagged with a 
double ‘xx’ are considered to be an opportunistic 
infection, regardless of seriousness.  
All serious TEAEs in the study database which code to a 
PT flagged with a single ‘x’ and all TEAEs in the study 
database which code to a PT flagged with a double ‘xx’ 
will be summarized as an opportunistic infection in the 
stand-alone table. [CQ97NAM= ’Opportunistic Infection 
- Automatic’] 
 
Step 2: Refer to column C of the spreadsheet which 
identifies the PTs that need to be evaluated on a case-by-
case basis by the study physician and safety physician in 
order to determine whether it is a true opportunistic 
infection or not. The process for physician review is as 
follows: 
1. Study programming team creates a spreadsheet 
which lists all of the subjects with a TEAE 
present in the database which codes to a PT 
identified as case-by-case. [CQ98NAM= 
Opportunistic Infection - Manual Review 
Candidate]  
Information from the AE dataset to be included in 
the spreadsheet: Subject ID, AE verbatim term, 
System Organ Class (SOC), High Level Term 
(HLT), Lower Level Term (LLT), PT, AE start 
date, AE end date, seriousness, severity, 
relationship to study medication, action taken. 
Additionally, a column will be included where the 
study physician/safety physician can document 
their decision on the case. 
2. Study physician/safety physician (SPs) reviews 
the cases in the spreadsheet separately and 
reconciles final decision, and indicates in the 
additional column which AEs are confirmed to be 
opportunistic infections via a single ‘x’. 
3. Study programming team incorporates these 
decisions into the AE dataset by merging the SPs 
decisions for individual subjects / PTs and 
flagging both the automatic and the confirmed 
opportunistic infections as such in the dataset. 
 [CQ99NAM= ‘Opportunistic Infection – 
Adjudicated’] 
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The SPs reviews the context of all of a subject’s data 
(AEs and possibly other) and concludes individually.   
Indicators of relevant cases may be e.g. repetitive 
occurrences, conjunction of other events or findings 
considered relevant.  
All subjects with a case-by-case PT reported that has 
been confirmed by the SPs to be an OI will be 
summarized as such in the stand-alone table, along with 
all of the events identified in Step 1 of this process.  
[CQ99NAM= ‘Opportunistic Infection – Adjudicated’]  
The timing and frequency of Step 2 should be outlined 
and agreed to by the study team at the beginning of the 
study. It is suggested that this process be executed 
multiple times throughout the course of the study, more 
frequently in the weeks leading up to database lock, and 
one final time immediately prior to database lock.   
 Following the initial physician review of case-by-case 
events, subsequent reviews will be based on the 
cumulative set of case-by-case events present in the 
database at each time point of spreadsheet creation.   
  
Physician decisions from previous runs should be 
retained in each subsequent run. The final run of the 
spreadsheet, with all SP decisions on the full set of case-
by-case events, will be archived at the conclusion of the 
study analysis prepared for agency submission. 
9 
Reductions in albumin 
and plasma proteins 
TEAEs with 
PT=’Blood albumin decreased’ or 
PT=’Protein albumin ratio’ or 
LLT=’Plasma protein abnormal’ or 
LLT=’Proteins serum plasma low’ 
10 
Effects on the kidney 
TEAEs in  
SMQ= ‘Acute renal failure’ 
11 
Drug related hepatic 
disorders  
TEAEs in  
SMQ=’Drug related hepatic disorders - comprehensive 
search’ 
12 
Effect on lipids 
TEAEs with  
PT= ‘Blood cholesterol increased’ or  
PT= ‘Low density lipoprotein increased’ or  
PT= ‘Blood triglycerides increased’ or  
PT= ‘Hypercholesterolaemia’ or  
PT= ‘Hypertriglyceridaemia’ or  
PT= ‘Hyperlipidaemia’ or  
PT= ‘Dyslipidaemia’ or  
PT= ‘Lipids increased’ 
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14 
AMENDMENT(S) TO THE STATISTICAL ANALYSIS PLAN  
14.1 
Amendment 1 
Rationale for the amendment 
This amendment is to apply following changes: 
• Adverse event summaries to be consistent with MG0003 
• Update definitions of ADA participant categories, and update related analyses 
• Minor cosmetic changes 
Modifications and changes 
Section # and Name 
Description of Change 
Brief Rationale 
General 
Added RLZ total to summary tables 
To be consistent with MG0003 
General 
Minor administrative, formatting, and 
typographical changes have been made. 
Updated to provide clarity and 
be consistent with remainder of 
SAP. 
3.3 Definition of Baseline 
values 
Modified general definition of Baseline 
values and added Baseline definition for 
anti-drug antibody. 
To consider actual assessment 
time and add more accurate 
definition for ADA considering 
MG0003 treatment effect  
3.8 Coding dictionaries 
Updated MedDRA and WHODD version 
Newer versions were released 
4.9 Examination of 
subgroups 
The stratification factors MuSK(+/-) and 
AChR(+/-) in the subgroup analysis were 
changed to be based on the values from 
MG-specific autoantibody assessment 
taken at Baseline in MG0003. 
To be consistent with MG0003 
5.1 Subject disposition  
Added a table to summarize the number of 
participants at each visit by treatment 
group. 
Based on DEM3 comment to 
understand the pattern of 
dropouts 
6.1 Demographics 
Age was changed to be the age at the time 
of MG0003 entry. 
To compare age population 
across MG studies 
6.1 Demographics 
Added race, ethnicity group and needed 
rescue therapies in the observation period 
of MG0003 and entered MG0004 
To be consistent with MG0003 
and MG0007 
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6.2 Other Baseline 
characteristics 
Defined MG-ADL cutoff as 5 
The cutoff was determined in 
MG0003 
6.4 Prior and concomitant 
medications 
Added definitions of rescue medications 
To provide clear definitions of 
rescue medications in 
programming 
8.2 Adverse events 
Reorganized adverse event summaries  
To be consistent with MG0003 
8.3.1 Potential drug-
induced liver injury  
Modified PDILI criteria and added new 
summaries 
To be consistent with PSAP 
8.4.2 Electrocardiograms 
Removed QTcB 
QTcB is not of interest 
8.4.3.2 Suicidal risk 
monitoring 
Suicidal risk monitoring summary 
analysis was removed 
Listing is enough to provide all 
information 
9.1.1 MG-ADL score 
Added COVID-19 free analysis for MG-
ADL summary. 
Added MG-ADL (excluding ocular items) 
summary. 
To evaluate impact of COVID-
19 pandemic. 
To be consistent with MG0003. 
9.1.2 MG-C score  
Added by MuSK+ or AChR+ summary of 
MG-C score 
Missing from last version 
9.1.3 QMG scale  
Changed subgroup analysis by MuSK+ or 
AChR+ only 
Other subgroups are not needed 
9.2.5 MG-ADL responder 
rate (≥2.0 points 
improvement from 
Baseline) 
Removed summary for imputed values 
and changed the summary for all visits 
instead of first 6 weeks 
NRI imputation is not 
applicable to MG0004. 
9.2.6 QMG responder rate 
(≥3.0 points improvement 
from Baseline) 
Removed summary for imputed values 
and changed the summary for all visits 
instead of first 6 weeks 
NRI imputation is not 
applicable to MG0004. 
9.2.7 MG-C responder rate 
(≥5.0 points improvement 
from Baseline) 
Removed summary for imputed values 
and changed the summary for all visits 
instead of first 6 weeks 
NRI imputation is not 
applicable to MG0004. 
10 
PHARMACOKINETICS 
AND 
PHARMACODYNAMICS 
Updated ADA participant categories and 
related summary analysis 
To be consistent with MG0003 
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11.2 Specific analyses for 
Pharmaceuticals and 
Medical Devices Agency 
(PMDA) 
Updated AE summaries based on changes 
in Section 8.2 
To be consistent with AE 
summaries on all participants 
11.3 Headache 
questionnaire 
Added text in SAP for headache listing in 
shells 
To be consistent with shells 
11.4 Myasthenia Gravis 
Foundation of America 
(MGFA) by Visit 
Added MGFA listing to SAP 
To include MGFA data 
13.7 Markedly abnormal 
laboratory and diagnostic 
criteria for 
Rozanolixizumab program 
Added corrected calcium algorithm and 
updated the marked abnormality criteria 
for corrected calcium and eGFR formula 
parameters. 
For clarity 
13.8 AEs of focus for 
Rozanolixizumab program 
Updated definitions of AEOF  
To be consistent across MG 
studies 
 
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STATISTICAL ANALYSIS PLAN SIGNATURE PAGE 
This document has been reviewed and approved per the Review and Approval of Clinical 
Documents Standard Operating Procedures. Signatures indicate that the final version of the 
Statistical Analysis Plan (SAP) or amended SAP is released for execution. 
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Prot_001.pdf: 
UCB 
 
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Clinical Study Protocol 
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A RANDOMIZED, OPEN-LABEL EXTENSION STUDY TO 
INVESTIGATE THE LONG-TERM SAFETY, TOLERABILITY, 
AND EFFICACY OF ROZANOLIXIZUMAB IN ADULT PATIENTS 
WITH GENERALIZED MYASTHENIA GRAVIS 
 
 
PROTOCOL MG0004 AMENDMENT 2 
PHASE 3 
 
 
Short title: 
A Phase 3 extension study to evaluate the safety, tolerability, and efficacy of rozanolixizumab in 
adult patients with generalized myasthenia gravis 
Sponsor: 
UCB Biopharma SRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Regulatory agency identifying numbers: 
Eudra CT Number: 
2019-000969-21 
IND Number: 
132407 
Confidential Material 
Confidential 
This document is the property of UCB and may not – in full or in part – be passed on, 
reproduced, published, or otherwise used without the express permission of UCB. 
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PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE 
Document History 
Document 
Date 
Type of amendment 
Protocol Amendment 2 
30 Jul 2020 
Substantial 
Protocol Amendment 1 
01 Nov 2019 
Substantial 
Original Protocol 
21 Mar 2019 
Not applicable 
Amendment 2 (30 Jul 2020) 
Overall Rationale for the Amendment 
The primary reasons for this protocol amendment are to introduce the transition of study 
participants to MG0007 and closure of MG0004, once MG0007 is available as the open-label 
study to MG0003 and MGC003. In addition, changes have been made to decrease the complexity 
of assessments to be performed; clarify some operational aspects of the study; incorporate the 
harmonization of inclusion criteria with studies performed across the rozanolixizumab clinical 
development program; and to include the management of study participant treatment during the 
coronavirus disease 2019 (COVID-19) pandemic including contingency measures. 
This amendment is considered to be substantial based on the criteria set forth in Article 10(a) of 
Directive 2001/20/EC of the European Parliament and the Council of the European Union. 
Section # and Name 
Description of Change 
Brief Rationale 
Global 
Minor administrative, formatting, 
and typographical changes have 
been made. 
Where applicable, the term 
“patient” has been replaced by 
“study participant.” 
Updated to provide clarity 
and to be consistent with 
remainder of protocol. 
Title page 
Sponsor name has been updated. 
Belgium has recently 
adopted a new Code of 
Companies and 
Associations, resulting in 
a mandatory change of the 
name of the legal form of 
the entity “société privée 
à responsabilité limitée”, 
abbreviated “SPRL,” to 
“société à responsabilité 
limitée”, abbreviated 
“SRL.” 
1.1 Objectives and 
Endpoints 
1.3 Schedule of Activities 
3 Objectives and 
Endpoints 
Other efficacy endpoints PGI-S and 
PGI-C have been removed.  
The endpoint was 
removed to decrease 
study complexity, as 
scientific value is limited. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.1 Objectives and 
Endpoints 
1.3 Schedule of Activities 
3 Objectives and 
Endpoints 
8.7 Pharmacodynamics 
The pharmacodynamic endpoint for 
neurofilament-light levels has been 
removed. 
The endpoint was 
removed to decrease 
study complexity, as 
scientific value is limited.  
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
Evaluation of the effects of 
rozanolixizumab on albumin, α- 
and β-globulins, and IgG subclasses 
was removed from the study 
objectives. 
The objective was 
amended to decrease 
study complexity, as 
scientific value is limited. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
The endpoints for exploratory 
biomarkers such as B-cell 
activating factor and Circulating 
Immune Complexes and proteins 
and metabolite changes have been 
removed. 
The endpoint was 
removed to decrease 
study complexity, as 
scientific value is limited. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
The following new sentence “Other 
exploratory safety biomarkers may 
be assessed” was added to footnote 
a under the Objectives and 
Endpoints table. 
This addition was 
included to maintain 
consistency within the 
protocol and schedule of 
activities. 
1.1 Overall design 
4.1 Overall design 
A new paragraph was inserted 
describing the rollover of 
participants from MG0004 to OLE 
study MG0007. 
The current ongoing OLE 
study, MG0004 (52-week 
chronic treatment) will be 
replaced by MG0007, 
which is a less complex 
with 
 
 based on 
myasthenia gravis (MG) 
worsening. 
1.3 Schedule of Activities 
A new sentence describing visit and 
screening information for entry into 
MG0007 has been added.  
The PEOT of MG0004 
serves as the introductory 
baseline and will have to 
be completed for entry 
into MG0007. 
1.3 Schedule of Activities 
Week 52/Visit 52 PEOT and Week 
60/Visit 53 EOS assessments have 
been harmonized to match 
MG0003. 
Updated to ensure all 
screening criteria 
assessments match with 
MG0007. The PEOT of 
MG0004 serves as the 
introductory baseline and 
will have to be completed 
for entry into MG0007. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
A new assessment, MGFA 
classification, was added at Visit 52 
or PEOT and Visit 53 EOS. 
Updated as screening 
criteria for entering into  
MG0007.  
1.3 Schedule of Activities 
MG Symptoms PRO assessment 
has been removed at Visit 7. 
Updated to simplify and 
decrease the complexity 
of the study. 
1.3 Schedule of Activities 
MGQoL15r assessment has been 
added at 52 or PEOT. 
Updated to simplify and 
decrease the complexity 
of the study. 
1.3 Schedule of Activities 
Full physical examination 
assessment has been added at Visit 
52 or PEOT. 
This update was included 
to maintain consistency 
with the study design and 
protocol 
1.3 Schedule of Activities 
Brief physical examination 
assessment has been removed at 
Visit 52 or PEOT. 
This update was included 
to maintain consistency 
with the study design and 
protocol 
1.3 Schedule of Activities 
Pregnancy test (urine) assessment 
has been removed at Visit 5. 
Updated to simplify and 
decrease the complexity 
of the study. 
1.3 Schedule of Activities 
Hematology, serum chemistry, 
urinalysis assessment has been 
removed at Visit 5.  
Updated to simplify and 
decrease the complexity 
of the study. 
1.3 Schedule of Activities 
Additional assessment for PTT and 
INR has been added at Visit 52 or 
PEOT. 
Updated as screening 
criteria for entering into  
MG0007. 
1.3 Schedule of Activities 
An additional footnote was added 
for the Anti-tetanus toxoid titer at 
1st visit for Weeks 9-48 (footnote 
j): The following new text “Every 6 
months” was added. 
Updated to be provide 
clarity for timings of this 
assessment. 
1.3 Schedule of Activities 
IGRA tuberculosis test assessment 
was added at Visit 52 or PEOT. 
Updated as screening 
criteria for entering into  
MG0007. 
1.3 Schedule of Activities 
Blood sampling for PK assessment 
has been added at Visit 5. 
Updated for consistency 
within the protocol. 
1.3 Schedule of Activities 
Blood sampling for ADA 
assessment has been removed at 
Visit 7 and Visit 49. 
Updated to simplify and 
decrease the complexity 
of the study. 
1.3 Schedule of Activities 
Blood sampling for ADA was 
added to footnote o for 1st Visit for 
Weeks 9-48. 
Updated for consistency 
within the protocol. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
Blood sampling IgA, IgM, IgE 
assessment has been added at 1st 
visit for Weeks 9-48 and Week 52 
or PEOT.  
Updated for consistency 
and to correct an error in 
protocol amendment 1. 
1.3 Schedule of Activities 
Blood sampling for exploratory 
biomarker analysis was removed. 
Updated to simplify and 
decrease the complexity 
of the study. 
1.3 Schedule of Activities 
Blood sampling for exploratory 
safety biomarker analysis 
assessment was removed at Visits 
1, 5, 7, 1st visit, and 49, and added 
at Visit 2-4. 
Updated to clarify that it 
is event driven only and 
not applicable to all study 
participants. 
1.3 Schedule of Activities 
MG-specific autoantibodies 
assessments were added at Visits 5 
and 52 or PEOT. 
This was included as part 
of the entry requirements 
for MG0007. 
1.3 Schedule of Activities 
Footnote o was added to MG-
specific autoantibodies assessment 
at 1st visit for Weeks 9-48. 
Updated to provide clarity 
and be consistent with 
remainder of protocol. 
1.3 Schedule of Activities 
Assessment of serum complement 
levels (C3, C4) and cytokines has 
been combined into one procedure. 
Updated to simplify and 
decrease the complexity 
of the study. 
1.3 Schedule of Activities 
Serum complement levels (C3,C4) 
and cytokines assessments was 
removed at Visits 1 and 49, and 
added at Visit 2-4 and at 1st visit for 
Weeks 9-48. 
Updated to correct an 
error in the original 
protocol. 
1.3 Schedule of Activities 
Plasma complement levels (C3a 
and C5a) assessments were 
removed at Visits 1 and 49, and 
added at Visit 2-4 and at 1st visit for 
Weeks 9-48. 
Updated to correct an 
error in the original 
protocol. 
1.3 Schedule of Activities 
Reference to the lead-in study, 
MGC003, has been added where 
applicable.  
Updated for clarity and 
consistency within the 
protocol. 
1.3 Schedule of Activities 
Footnote d: The following new text 
“not older than 32 days” was added. 
Updated for consistency 
and to provide clarity.  
1.3 Schedule of Activities 
An additional footnote was added 
for body weight (footnote e): “To 
be collected four times during the 
study: Screening/Baseline, Week 
25 (site visit), Week 52 (or PEOT), 
and EOS Visit.” 
To provide clarity for 
exact timings for 
collection of body weight 
during the study. 
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MG0004 
 
 
 
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Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
Footnote f: New wording was 
added regarding the first question 
of the C-SSRS to the suicidal 
ideation assessment query. 
Updated for consistency 
and to provide clarity. 
1.3 Schedule of Activities 
Footnote f: New wording was 
added: “Complete C-SSRS will 
observe the “since last visit” 
version recorded at Visit 14 in 
MG0003 or MGC003.” 
Updated to provide clarity 
for the rollover visit from 
MG0003 to MG0004. 
1.3 Schedule of Activities 
An additional footnote was added 
for MGII (footnote g:) “MGII is 
optional for all study participants.” 
MGII has switched from 
mandatory to optional for 
each study visit. 
1.3 Schedule of Activities 
Footnotes s and t (previously p, q, 
r): “Severe headache” was added to 
the list of events. 
Updated to correct an 
error in protocol 
amendment 1. 
1.3 Schedule of Activities 
Footnote s and t: “postdose” was 
replaced with “postevent.” 
Updated to correct 
terminology error. 
1.3 Schedule of Activities 
An additional footnote was added 
(footnote r). 
Updated for consistency 
and to provide clarity. 
1.3 Schedule of Activities 
Footnote s: New wording “Baseline 
from MG0003 and MGC003 will 
serve as Baseline in MG0004. 
Additional exploratory safety 
samples must be collected 4 hours 
postevent or as soon as possible 
before the next IMP in case of 
AESM of serious or severe 
headache or AESM of GI disorders 
as described in Section 8.9” was 
added. 
New text was added to 
provide clarity and 
consistency with the study 
design and procedures. 
1.3 Schedule of Activities 
Footnote t (previously footnote p): 
The first sentence “Serum 
complement levels” has been 
removed, and a new sentence 
“Baseline from MG0003 and 
MGC003 will serve as Baseline in 
MG0004” has been added. 
New text was added to 
provide clarity and 
consistency with the study 
design and procedures. 
4.1 Overall design 
5.2 Exclusion criteria 
New text “(not older than 32 days)” 
has been added to the laboratory 
measurement paragraphs. 
Updated for consistency 
and to provide clarity. 
4.1 Overall design 
A new sentence relating to the 
updated language in Section 8 
regarding contingency measures 
during a pandemic and other 
Contingency measures 
have been implemented to 
ensure study participant 
safety in response to the 
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MG0004 
 
 
 
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Section # and Name 
Description of Change 
Brief Rationale 
exceptional circumstances has been 
included. 
COVID-19 pandemic 
causing the inability for 
sites to adhere to protocol 
visits and assessments. 
5.1 Inclusion Criteria 
8.3.5 Pregnancy 
10.4.2 Contraception 
guidance 
All reference to 3 months have 
been replaced with “at least 90 
days.” 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
5.2 Exclusion Criteria 
Criterion #11: Amended definition 
(glomerular filtration rate less than 
45ml/min/1.73m2) was included for 
renal impairment. 
To expand the population 
for a more generalized 
safety database. 
5.2 Exclusion Criteria 
Criterion #17: The number was 
reinserted, but the criterion itself 
remains removed from the study.  
Updated to be consistent 
with UCB standards, and 
to correct an error in 
protocol amendment 1.  
5.3.1 Meals and dietary 
restrictions 
5.3.2 Caffeine, alcohol, 
and tobacco 
Both sections have been removed.  
These sections were 
deemed unnecessary as 
the wording in Section 5.3 
(Lifestyle Restrictions) 
cover the content. 
6.5.1 Permitted 
concomitant treatments 
(medications and 
therapies) 
Footnote a under Table 6-2 has 
been modified to read 
“≤300ng/mL” from “≤300ng/L.” 
Updated to correct an 
error and ensure clarity. 
7.1.1 Liver Chemistry 
Stopping Criteria 
7.1.2 QTc Stopping 
Criteria 
A new sentence was added to 
emphasize study participants should 
follow the visit schedule as 
described in the protocol and to 
complete the eCRF. 
New wording was added 
to provide clarity. 
7.1.3 Discontinuation of 
study medication due to 
other AEs or medical 
conditions 
Language related to the temporary 
discontinuation of study medication 
was removed as it pertained to IgG 
levels and rescue medication. 
Updated to simplify and 
decrease the complexity 
of the study. 
7.1.3 Discontinuation due 
to other adverse events or 
medical conditions 
Criterion #3: Details regarding 
serious infective episodes was 
updated. 
Updated for consistency 
within the protocol. 
7.1.4 Temporary IMP 
discontinuation 
New details have been included to 
replace text pertaining to 
procedures for temporary IMP 
discontinuation. Furthermore, 
additional clarity has been included 
for procedures undertaken when 
temporary treatment 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
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MG0004 
 
 
 
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Section # and Name 
Description of Change 
Brief Rationale 
discontinuation is required to low 
IgG levels. 
7.1.4 Temporary IMP 
discontinuation 
In addition, details have been added 
for participants that have suspected 
or confirmed COVID-19. 
Updated to clarify the 
procedures on when 
treatment discontinuation 
is required in response to 
the COVID-19 pandemic. 
8 Study Assessments and 
Procedures 
A new paragraph describing 
contingency measures during a 
pandemic and other exceptional 
circumstances has been included. 
Contingency measures 
have been implemented to 
ensure study participant 
safety in response to the 
COVID-19 pandemic 
causing the inability for 
sites to adhere to protocol 
visits and assessments. 
8.1.3 Patient-reported 
outcomes 
8.1.3.3 Patient Global 
Impression of Severity 
8.1.3.4 Patient Global 
Impression of Change 
References to PGI-S and PGI-C, 
including both sections, have been 
deleted. 
Updated to simplify and 
decrease the complexity 
of the study. 
8.2 Safety assessments 
The following wording was added: 
“until Week 7, where the 
participant can be observed for 1 
hour at the Investigator’s 
discretion.” 
Updated to be consistent 
with the Schedule of 
Activities. 
8.5 Treatment of 
Overdose 
Details related to monitoring of 
participants any adverse event 
(AE)/serious adverse event (SAE) 
or laboratory abnormality have 
been updated. 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
8.9 Biomarkers 
For collection of additional 
samples, “postdose” was replaced 
with “postevent.” 
Updated to correct 
terminology error. 
8.9 Biomarkers 
The last sentence of the first 
paragraph was modified to read 
“Additional exploratory safety 
samples must be collected 4 hours 
postevent or as soon as possible 
before the next IMP in case of 
AESM of serious or severe 
headache or AESM of GI 
disorders.” 
Updated to provide clarity 
and be consistent with 
remainder of protocol. 
8.9 Biomarkers 
Language related to albumin, α- 
and β-globulins, B-cell activating 
The endpoints were 
removed to decrease 
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Clinical Study Protocol 
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MG0004 
 
 
 
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Section # and Name 
Description of Change 
Brief Rationale 
factor, and Circulating Immune 
Complexes was removed. 
study complexity, as 
scientific value is limited. 
8.9.1 Immunology 
For collection of additional 
samples, “postdose” was replaced 
with “postevent.” 
Updated to correct 
terminology error. 
8.9.1 Immunology 
The following was added: “Tetanus 
toxoid IgG” 
Information for the collection of 
samples has been amended: 
“Additional samples may be 
collected 2 hours and 4 hours 
postevent in case of severe 
headache, infusion reaction, or 
hypersensitivity reaction at Visits 5, 
7, 1st visit (of the 4-week cycle 
from Weeks 9-48) and Visit 49.” 
To ensure consistency 
with the wording for 
Schedule of Activities 
footnotes specific to 
serum complement, serum 
cytokines, and plasma 
complement. 
9.3.3.2 Pharmacodynamic 
analyses and biomarkers 
Language related to albumin and α- 
and β-globulins was removed. 
The endpoints were 
removed to decrease 
study complexity, as 
scientific value is limited. 
10.2 Appendix 2: Clinical 
Laboratory Tests 
The following parameters were 
added: “C-reactive protein” and 
“Albumin.” 
Updated to correct an 
error in protocol 
amendment 1. 
10.6 Appendix 6: Liver 
Safety – Suggested 
Actions and Follow-up 
Assessments 
The choice of specific assay for the 
bilirubin or INR criteria. 
Updated to remove the 
description of a specific 
assay to give the 
Investigator a broader 
range of tests to choose 
from. 
10.16 Appendix 16: 
Patient Global Impression 
of Severity 
10.17 Appendix 17: 
Patient Global Impression 
of Change 
These appendices were removed. 
Updated to be consistent 
with the protocol. 
10.20.1 Management of 
headache 
This section was previously 
10.22.1. Clarified that treatment 
may be temporarily put on hold if a 
study participant experiences a 
severe AE of headache that is not 
resolved prior to the next scheduled 
study treatment. 
The language updated to 
be consistent across the 
Phase 3 rozanolixizumab 
clinical program. 
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Clinical Study Protocol 
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Section # and Name 
Description of Change 
Brief Rationale 
10.20.2 Management of 
Diarrhea 
This section was previously 
10.22.2. The phrase “over baseline” 
has been removed. 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
10.20.2 Management of 
Diarrhea  
This section was previously 
10.22.2. Stool sample collection has 
been amended: Stool samples may 
be collected for stool “analysis” to 
“rule out infection” for study 
participants reporting severe 
diarrhea. Stool sampling will be as 
clinically indicated in the opinion 
of the Investigator and assessed per 
local guidance. 
The language updated to 
be consistent across the 
Phase 3 rozanolixizumab 
clinical program. 
10.20.3 Management of 
infections and 
hypgammaglobulinemia 
This section was previously 
10.22.3. Language related to mock 
infusions has been removed. 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
10.20.3 Management of 
infections and 
hypgammaglobulinemia 
This section was previously 
10.22.3. The following sentence 
was modified to include “must”: 
Treatment must be temporarily 
discontinued for the study 
participant who develops an event 
of hypogammaglobulinemia with a 
serum total IgG of <1g/L 
irrespective of infection. 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
10.20.3 Management of 
infections and 
hypgammaglobulinemia 
This section was previously 
10.22.3. The following sentence 
“Ad hoc assessment can be 
performed to monitor the recovery 
of IgG levels” has been added. 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
10.20.4 Management of 
infusion reactions or 
hypersensitivity reactions 
This section was previously 
10.22.4. Additional guidance for 
nurses administering IMP at home 
has been added. 
Updated to be consistent 
across the Phase 3 
rozanolixizumab clinical 
program. 
10.23 Appendix 23: 
Columbia-Suicide 
Severity Rating Scale 
This section was previously 10.25. 
New text, “Screening” and “Since 
Last Visit” have been included to 
define each version provided in the 
appendix. 
Updated to provide clarity 
for each example. 
11 References 
The following reference was 
removed: 
James LP, Letzig L, Simpson PM, 
Capparelli E, Roberts DW, Hinson 
Updated to maintain 
consistency within the 
protocol. 
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MG0004 
 
 
 
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Section # and Name 
Description of Change 
Brief Rationale 
JA, et al. Pharmacokinetics of 
acetaminophen-protein adducts in 
adults with acetaminophen 
overdose and acute liver failure. 
Drug Metab Dispos. 2009; 
37:1779-84 
 
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MG0004 
 
 
 
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SERIOUS ADVERSE EVENT REPORTING 
 
Serious adverse event reporting (24-hour) 
Fax 
Europe and Rest of the World: +32 2 386 24 21 
US and Canada: +1 800 880 6949 or +1 866 890 3175 
 
Email 
Global: DS_ICT@ucb.com (for interventional clinical studies) 
 
 
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Page 13 of 147 
TABLE OF CONTENTS 
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE ...................................... 2 
SERIOUS ADVERSE EVENT REPORTING ........................................................................ 12 
1 
PROTOCOL SUMMARY ............................................................................................... 18 
1.1 Synopsis ........................................................................................................................... 18 
1.2 Schema ............................................................................................................................. 24 
1.3 Schedule of activities ....................................................................................................... 25 
2 
INTRODUCTION ........................................................................................................... 30 
2.1 Study rationale ................................................................................................................. 30 
2.2 Background ...................................................................................................................... 31 
2.3 Benefit/Risk assessment ................................................................................................... 32 
3 
OBJECTIVES AND ENDPOINTS ................................................................................. 33 
4 
STUDY DESIGN ............................................................................................................. 36 
4.1 Overall design .................................................................................................................. 36 
4.2 Scientific rationale for study design................................................................................. 38 
4.3 Justification for dose ........................................................................................................ 39 
4.4 End of study definition ..................................................................................................... 40 
5 
STUDY POPULATION .................................................................................................. 40 
5.1 Inclusion criteria .............................................................................................................. 40 
5.2 Exclusion criteria ............................................................................................................. 41 
5.3 Lifestyle restrictions ......................................................................................................... 42 
5.4 Screen failures .................................................................................................................. 43 
6 
STUDY TREATMENTS ................................................................................................. 43 
6.1 Treatments administered .................................................................................................. 43 
6.2 Preparation, handling, storage, and accountability requirements .................................... 44 
6.2.1 
Drug accountability ............................................................................................. 44 
6.3 Measures to minimize bias: randomization ..................................................................... 45 
6.3.1 
Procedures for maintaining and breaking the treatment blind ............................. 45 
6.3.1.1 
Maintenance of study treatment blind ....................................................... 45 
6.3.1.2 
Breaking the treatment blind in an emergency situation ........................... 45 
6.4 Treatment compliance ...................................................................................................... 45 
6.5 Concomitant medications/treatments ............................................................................... 46 
6.5.1 
Permitted concomitant treatments (medications and therapies) .......................... 46 
6.5.2 
Prohibited concomitant treatments (medications and therapies) ......................... 46 
6.5.2.1 
Treatments specific to NMJ interference .................................................. 47 
6.5.3 
Rescue therapy ..................................................................................................... 47 
6.6 Dose modification ............................................................................................................ 47 
6.7 Home visits ...................................................................................................................... 48 
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6.8 Treatment after the end of the study ................................................................................ 48 
7 
DISCONTINUATION OF STUDY MEDICATION AND PARTICIPANT 
DISCONTINUATION/WITHDRAWAL ........................................................................ 49 
7.1 Discontinuation of study medication ............................................................................... 49 
7.1.1 
Liver chemistry stopping criteria ......................................................................... 49 
7.1.2 
QTc stopping criteria ........................................................................................... 50 
7.1.3 
Discontinuation of study medication due to other AEs or medical conditions ... 51 
7.1.4 
Temporary IMP discontinuation .......................................................................... 52 
7.2 Participant discontinuation/withdrawal from the study ................................................... 53 
7.3 Lost to follow-up .............................................................................................................. 53 
8 
STUDY ASSESSMENTS AND PROCEDURES ........................................................... 54 
8.1 Efficacy assessments ........................................................................................................ 55 
8.1.1 
Quantitative Myasthenia Gravis scale ................................................................. 55 
8.1.2 
Myasthenia Gravis-Composite scale ................................................................... 55 
8.1.3 
Patient-reported outcomes ................................................................................... 55 
8.1.3.1 
MG-Activities of Daily Living scale ......................................................... 56 
8.1.3.2 
MG Symptoms PRO .................................................................................. 56 
8.1.3.3 
EQ-5D-5L .................................................................................................. 56 
8.1.3.4 
Myasthenia Gravis Impairment Index ....................................................... 57 
8.1.3.5 
Revised 15-item myasthenia gravis quality of life questionnaire ............. 57 
8.2 Safety assessments ........................................................................................................... 57 
8.2.1 
Physical examinations ......................................................................................... 57 
8.2.2 
Vital signs ............................................................................................................ 58 
8.2.3 
Electrocardiograms .............................................................................................. 58 
8.2.4 
Clinical safety laboratory assessments ................................................................ 58 
8.2.5 
Suicidal risk monitoring ...................................................................................... 58 
8.2.6 
Assessment and management of tuberculosis and tuberculosis risk factors ........ 59 
8.3 Adverse events ................................................................................................................. 61 
8.3.1 
Time period and frequency for collecting AE and SAE information .................. 62 
8.3.2 
Method of detecting AEs and SAEs .................................................................... 62 
8.3.3 
Follow-up of AEs and SAEs ................................................................................ 62 
8.3.4 
Regulatory reporting requirements for serious adverse events ............................ 62 
8.3.5 
Pregnancy ............................................................................................................ 63 
8.3.6 
Adverse events of special interest ........................................................................ 63 
8.3.7 
Adverse events of special monitoring .................................................................. 63 
8.3.8 
Treatment-emergent adverse events .................................................................... 64 
8.4 Safety signal detection ..................................................................................................... 64 
8.5 Treatment of overdose ..................................................................................................... 64 
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8.6 Pharmacokinetics and anti-drug antibodies ..................................................................... 65 
8.7 Pharmacodynamics .......................................................................................................... 65 
8.8 Genetics ............................................................................................................................ 65 
8.9 Biomarkers ....................................................................................................................... 65 
8.9.1 
Immunology ......................................................................................................... 66 
8.10 Medical resource utilization and health economics ......................................................... 66 
9 
STATISTICAL CONSIDERATIONS ............................................................................. 66 
9.1 Definition of analysis sets ................................................................................................ 67 
9.2 General statistical considerations ..................................................................................... 67 
9.3 Planned safety and other analyses .................................................................................... 67 
9.3.1 
Analysis of the primary safety endpoints ............................................................ 67 
9.3.2 
Analysis of the other safety endpoints ................................................................. 67 
9.3.3 
Other analyses ...................................................................................................... 68 
9.3.3.1 
Pharmacokinetic analyses .......................................................................... 68 
9.3.3.2 
Pharmacodynamic analyses and biomarkers ............................................. 68 
9.3.3.3 
Anti-drug antibody analyses ...................................................................... 68 
9.4 Planned efficacy analyses ................................................................................................ 68 
9.5 Handling of protocol deviations ....................................................................................... 68 
9.6 Handling of dropouts or missing data .............................................................................. 69 
9.7 Planned interim analysis and data monitoring ................................................................. 69 
9.7.1 
Interim analysis .................................................................................................... 69 
9.7.2 
Data monitoring ................................................................................................... 69 
9.8 Determination of sample size ........................................................................................... 69 
10 SUPPORTING DOCUMENTATION AND OPERATIONAL CONSIDERATIONS... 69 
10.1 Appendix 1: Regulatory, ethical, and study oversight considerations ............................. 69 
10.1.1 
Regulatory and ethical considerations ................................................................. 69 
10.1.2 
Financial disclosure ............................................................................................. 70 
10.1.3 
Informed consent process .................................................................................... 70 
10.1.4 
Data protection ..................................................................................................... 71 
10.1.5 
Committees structure ........................................................................................... 71 
10.1.6 
Data quality assurance ......................................................................................... 71 
10.1.6.1 
Electronic Case Report form completion .................................................. 72 
10.1.6.2 
Apps ........................................................................................................... 72 
10.1.7 
Source documents ................................................................................................ 72 
10.1.8 
Study and site closure .......................................................................................... 73 
10.1.9 
Publication policy ................................................................................................ 73 
10.2 Appendix 2: Clinical laboratory tests .............................................................................. 74 
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10.3 Appendix 3: Adverse events – definitions and procedures for recording, evaluating, 
follow-up, and reporting .................................................................................................. 76 
10.3.1 
Definition of adverse event .................................................................................. 76 
10.3.2 
Definition of serious adverse event ..................................................................... 77 
10.3.3 
Recording and follow-up of adverse events and/or serious adverse events ........ 79 
10.3.4 
Reporting of serious adverse events .................................................................... 81 
10.4 Appendix 4: Contraceptive guidance and collection of pregnancy information ............. 82 
10.4.1 
Definitions ........................................................................................................... 82 
10.4.1.1 
Woman of childbearing potential .............................................................. 82 
10.4.2 
Contraception guidance ....................................................................................... 82 
Male participants ............................................................................................................ 82 
Female participants ........................................................................................................ 83 
10.4.2.1 
Male participants with partners who become pregnant ............................. 84 
10.4.2.2 
Female participants who become pregnant ............................................... 84 
10.5 Appendix 5: Genetics ....................................................................................................... 86 
10.6 Appendix 6: Liver Safety – Suggested Actions and Follow-up Assessments ................. 87 
10.7 Appendix 7: Medical device incidents – definition and procedures for recording, 
evaluating, follow-up, and reporting ................................................................................ 92 
10.8 Appendix 8: Rapid alert procedures ................................................................................ 93 
10.9 Appendix 9: Country-specific requirements .................................................................... 94 
10.10 
Appendix 10: Abbreviations and trademarks ........................................................ 95 
10.11 
Appendix 11: Protocol amendment history ........................................................... 98 
10.12 
Appendix 12: Quantitative Myasthenia Gravis Scale .......................................... 107 
10.13 
Appendix 13: Myasthenia Gravis-Composite scale ............................................. 116 
10.14 
Appendix 14: Myasthenia Gravis-Activities of Daily Living .............................. 117 
10.15 
Appendix 15 Myasthenia Gravis Symptoms PRO............................................... 118 
10.16 
Appendix 16: 5-Level European quality of life 5 dimensions ............................. 121 
10.17 
Appendix 17: Myasthenia Gravis Impairment Index ........................................... 123 
10.18 
Appendix 18: Revised 15-item myasthenia gravis quality of life questionnaire . 129 
10.19 
Appendix 19: Tuberculosis questionnaire ........................................................... 130 
10.20 
Appendix 20: Management of headaches, diarrhea, and infections and 
hypogammaglobulinemia ............................................................................................... 132 
10.20.1 
Management of headache .................................................................................. 132 
10.20.2 
Management of diarrhea .................................................................................... 134 
10.20.3 
Management of infections and hypogammaglobulinemia ................................. 135 
10.20.4 
Management of infusion reactions or hypersensitivity reactions ...................... 136 
10.21 
Appendix 21: Headache questionnaire ................................................................ 138 
10.22 
Appendix 22: Sampson Criteria Questionnaire ................................................... 141 
10.23 
Appendix 23: Columbia-Suicide Severity Rating Scale ...................................... 142 
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11 REFERENCES .............................................................................................................. 146 
SPONSOR DECLARATION ................................................................................................ 147 
 
LIST OF TABLES 
Table 1-1: 
Schedule of activities ....................................................................................... 25 
Table 6-1: 
Study treatments administered ......................................................................... 43 
Table 6-2: 
Permitted concomitant medications for the treatment of myasthenia gravis ... 46 
Table 10-1: 
Highly Effective Contraceptive Methodsa ....................................................... 83 
Table 10-2: 
Phase 3 liver chemistry stopping criteria and follow-up assessments ............. 88 
Table 10-3: 
Phase 3 liver chemistry increased monitoring criteria with continued 
study medication .............................................................................................. 91 
Table 10-4: 
Suggested management guidelines for infusion reactions or anaphylaxis..... 136 
 
LIST OF FIGURES 
Figure 1-1: 
Study schematic ............................................................................................... 24 
Figure 7-1: 
Liver chemistry stopping criteria and increased monitoring algorithm ........... 49 
Figure 7-2: 
Liver chemistry increased monitoring algorithm with continued study 
intervention for participants with ALT at least 3xULN but less than 
8xULN ............................................................................................................. 50 
 
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1 
PROTOCOL SUMMARY 
1.1 
Synopsis 
Protocol title: 
A randomized, open-label extension study to investigate the long-term safety, tolerability, and 
efficacy of rozanolixizumab in adult patients with generalized myasthenia gravis 
Short title: 
A Phase 3 extension study to evaluate the safety, tolerability, and efficacy of rozanolixizumab in 
adult patients with generalized myasthenia gravis 
Rationale: 
Myasthenia gravis (MG) is a serious, debilitating, sometimes life-threatening condition that is 
associated with numerous symptoms, including muscular weakness and fatigue. The major 
pathophysiology leading to MG is the abnormal production of immunoglobulin (Ig) G 
autoantibodies directed toward nicotinic acetylcholine receptor (AChR), or muscle-specific 
kinase (MuSK) protein. Several commonly-prescribed treatments act, at least in part, by reducing 
the quantity of such circulating IgG autoantibodies. While the standard of care for MG involves 
the utilization a variety of therapeutic agents including cholinesterase inhibitors, 
immunomodulators, corticosteroid, biologics interfering with IgG turnover, high dose 
intravenous infusion of immunoglobulin (IVIg), plasmapheresis, or immunoadsorption, there 
remains a need for a safe and effective treatment devoid of significant side effects to 
conveniently treat study participants with MG. 
Rozanolixizumab is a humanized IgG4 monoclonal antibody that is being developed as an 
inhibitor of the activity of the neonatal Fc receptor (FcRn) for IgG. By blocking the activity of 
FcRn, rozanolixizumab accelerates the catabolism of antibodies and reduces the serum IgG 
concentration, including pathogenic IgG in MG study participants, thus offering a potentially 
safe, effective, and convenient alternative to existing treatments. A Phase 3 study (MG0003) is 
underway to provide the required data to establish the efficacy and safety of rozanolixizumab in 
anti-AChR or anti-MuSK autoantibody-positive study participants with generalized MG who 
experience moderate to severe symptoms and are being considered for additional treatment with 
IVIg or plasma exchange (PEX). 
MG0004 is a Phase 3 open-label extension study to evaluate the long-term safety, tolerability, 
and efficacy of rozanolixizumab with a 
 dosing regimen for 52 weeks in study 
participants with generalized MG. The MG0004 open-label extension study will provide the 
opportunity to assess the long-term safety of rozanolixizumab in study participants with MG who 
participated in MG0003 and will provide them with a chance to benefit from long-term 
treatment. 
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Objectives and endpoints: 
Objectives 
Endpoints 
Primary 
To evaluate the long-term safety and tolerability 
of rozanolixizumab in study participants with 
generalized MG 
The primary safety endpoints are: 
• 
Occurrence of TEAEs 
• 
Occurrence of TEAEs leading to permanent 
withdrawal of study medication 
The other safety endpoints are: 
• 
Occurrence of AESMs 
• 
Vital sign values and changes from Baseline 
(systolic and diastolic blood pressure and 
pulse rate) 
• 
12-lead ECG values and change from 
Baseline at each scheduled assessment 
• 
Clinical laboratory findings (hematology, 
biochemistry, and urinalysis) 
Secondary 
To evaluate the long-term efficacy of 
rozanolixizumab in study participants with 
generalized MG 
The secondary efficacy endpoints are: 
• 
Change from Baseline in MG-ADL at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in MG-Composite 
(MG-C) score at each scheduled assessment 
during Treatment and Observation Periods 
• 
Change from Baseline in QMG at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Use of rescue medication (IVIg or PEX) 
The other efficacy endpoints are: 
• 
Change from Baseline in MGII scores at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in MGII ocular 
subscores at each scheduled assessment 
during Treatment and Observation Periods 
• 
Change from Baseline in MGII generalized 
domain subscores at each scheduled 
assessment during Treatment and 
Observation Periods 
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• 
Change from Baseline in the MG Symptoms 
PRO ‘Muscle Weakness Fatigability’ score at 
each scheduled assessment during Treatment 
and Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Physical Fatigue’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Bulbar symptoms’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Respiratory symptoms’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Ocular symptoms’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO multicomponent total score, at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the enhanced MG 
Symptoms PRO total score, at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in MGQoL15r at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in EQ-5D-5L scores at 
each scheduled assessment during Treatment 
and Observation Periods 
For study participants who require rescue 
treatment during the Observation Period of 
MG0003 and subsequently enter MG0004, the 
following endpoints will be assessed: 
• 
QMG responder rate (≥3.0 point 
improvement from Baseline) at each 
scheduled assessment during the first 
6 weeks of the Treatment Period 
• 
MG-C responder rate (≥5.0 point 
improvement from Baseline) at each 
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scheduled assessment during the first 
6 weeks of the Treatment Period 
• 
MG-ADL responder rate (≥2.0 point 
improvement from Baseline) at each 
scheduled assessment during the first 
6 weeks of the Treatment Period 
Other 
To assess the reduction in use of steroids in study 
participants receiving rozanolixizumab 
• 
AUC of the oral steroid dose over time 
To assess the plasma concentrations of 
rozanolixizumab administered by SC infusion 
• 
Plasma concentration of rozanolixizumab at 
each scheduled assessment during the 
Treatment and Observation Periods 
To assess the pharmacodynamic effects of 
rozanolixizumab as measured by IgG levels, IgG 
subclasses, and MG-specific autoantibodies levels 
• 
Change from baseline in total IgG and IgG 
subclasses autoantibodies at each scheduled 
visit 
• 
Change from baseline in MG-specific 
autoantibodies at each scheduled visit 
To evaluate the incidence and emergence of 
ADAs with respect to immunogenicity, PK, and 
pharmacodynamics 
• 
ADAs at each scheduled assessment during 
the Treatment Period and Observation 
Periods 
To assess the effect of rozanolixizumab on 
biomarkers including IgM, IgA, and IgE, serum 
and plasma complement levels, and serum 
cytokines  
• 
Change from Baseline in serum complement 
levels (C3 and C4) and plasma complement 
levels (C3a and C5a) at each scheduled 
assessment during Treatment Perioda 
• 
Change from Baseline in serum cytokines at 
each scheduled assessment during Treatment 
and Observation Periodsa 
• 
Change from Baseline in serum 
immunoglobulin concentrations (total IgA, 
IgE, and IgM) at each scheduled assessment 
during Treatment and Observation Periods 
To assess the effect of rozanolixizumab on tetanus 
IgG antibodies 
• 
Change from Baseline in anti-tetanus toxoid 
serum titers at each scheduled assessment 
during Treatment and Observation Period 
ADA=anti-drug antibody; ADL=activities of daily living; AESM=adverse event of special monitoring; 
ECG=electrocardiogram; Ig=immunoglobulin; IVIg=intravenous infusion of immunoglobulin G; 
MG=myasthenia gravis; MG-ADL=myasthenia gravis-activities of daily living; MGII=myasthenia gravis 
impairment index; MGQoL15r=revised 15-item myasthenia gravis quality of life questionnaire; PEX=plasma 
exchange; PK=pharmacokinetic; PRO=patient-reported outcome; QMG=quantitative myasthenia gravis test; 
SC=subcutaneous(ly); TEAE=treatment-emergent adverse event 
a To be performed for study participants with severe and/or serious headache and/or infusion reaction or 
hypersensitivity reaction. Other exploratory safety biomarkers may be assessed. 
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Overall design: 
This is a randomized, open-label Phase 3 extension study to evaluate the long-term safety, 
tolerability, and efficacy of rozanolixizumab in study participants with generalized MG. Study 
participants will enter MG0004 from MG0003 or MGC003. Study participants in MG0003 or 
MGC003 who complete the Observation Period or who require rescue medication during the 
Observation Period will be invited to be rerandomized in MG0004 (except for study participants 
who opt to receive IVIg or PEX as rescue medication). Study participants who receive IVIg or 
PEX as rescue therapy during the Observation Period of MG0003 or MGC003 will not be 
eligible to enroll in MG0004. Study participants who discontinue study medication in MG0003 
or MGC003 for any reason other than requiring rescue medication will not be eligible for 
MG0004. 
In MG0004, study participants with generalized MG will be randomized in a 1:1 ratio to receive 
1 of 2 doses of rozanolixizumab (equivalent to approximately 
, 
respectively). Rozanolixizumab will be administered subcutaneously (SC) on a 
basis 
over a 52-week period. Study participants will be allowed to switch dose arms for tolerability 
and efficacy reasons at the discretion of the Investigator (see Section 6.6). At the end of the 
52-week Treatment Period in MG0004, study participants will participate in an 8-week 
Observation Period. 
This open-label extension (OLE) study will be replaced by MG0007, an OLE study with 
 
 based on MG worsening. Upon study site approval of MG0007, as well as 
fulfilment of regulatory requirements, MG0003 study participants will have the opportunity to 
rollover directly into MG0007 and will no longer have the opportunity to rollover to MG0004. In 
the event a study participant has already started MG0004, a minimum treatment duration of 6 
visits must be completed (if IMP treatment is withheld for low IgG, study participants missed 
dose(s) can be counted as part of the total 6 visits for completion of MG0004 and meet eligibility 
requirements for MG0007), then the participant will discontinue MG0004 and have the 
opportunity to enter into MG0007. For study participants entering MG0007 from the treatment 
period of MG0004, he/she must complete the PEOT Visit in MG0004 which will serve both as 
the Screening Visit for MG0007 and as the End of Study (EOS) for MG0004. If a study 
participant completes the Treatment Period and is in the Observation Period of MG0004, he/she 
will then complete the EOS visit to serve as the Screening Visit for MG0007. Once the site is 
active for MG0007, no further participants will enroll in MG0004 and the study will be closed at 
the site once all eligible study participants have rolled over into MG0007. 
Number of participants: 
No formal sample size calculation can be performed. Up to approximately 276 study participants 
will be enrolled into the lead-in study MG0003 and MGC003. All eligible study participants 
from lead-in study MG0003 will be invited to participate in MG0004. 
At selected sites, study participants may also be able to participate in a substudy. 
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Treatment groups and duration: 
Study participants will be randomized in a 1:1 ratio to receive 1 of 2 doses of rozanolixizumab 
(equivalent to approximately 
, respectively). Specifically, the following 
dose arms will be used in the study: 
Dose arm 1: equivalent to approximately 
 rozanolixizumab 
• Bodyweight <50kg: dose to be administered 
 
• Bodyweight ≥50kg and <70kg: dose to be administered 
 
• Bodyweight ≥70kg and <100kg: dose to be administered 
 
• Bodyweight ≥100kg; dose to be administered 
 
Dose arm 2: equivalent to approximately 
 rozanolixizumab 
• Bodyweight <50kg: dose to be administered 
 
• Bodyweight ≥50kg and <70kg: dose to be administered 
 
• Bodyweight ≥70kg and <100kg: dose to be administered 
 
• Bodyweight ≥100kg: dose to be administered 
 
Further dose adjustment may be made if the study participant’s bodyweight changes during the 
study. 
If a dose arm is determined to be futile and is discontinued after the interim analysis in MG0003 
or MGC003, then that dose arm will be dropped from MG0004 as well, and study participants in 
the affected dose arm will be transferred to the continuing dose arm. An independent Data 
Monitoring Committee (IDMC) will be formed to monitor the ongoing safety and efficacy of the 
study. Further details of the IDMC will be provided in an IDMC charter. 
Rozanolixizumab will be administered SC on a 
 basis over a 52-week Treatment Period, 
and study participants will then participate in an 8-week Observation Period. The maximum 
study duration per study participant is 60 weeks. 
 
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1.2 
Schema 
A schematic of the study design is provided in Figure 1-1. 
Figure 1-1: Study schematic 
 
 
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1.3 
Schedule of activities 
The schedule of activities is provided in Table 1-1. For study participants entering MG0007, he/she must complete at least 6 visits and 
then complete the PEOT visit in MG0004, which serves as the Screening Visit for MG0007 and as the EOS for MG0004. If a study 
participant has completed the Treatment Period and is in the Observation Period of MG0004, he/she will then complete the EOS visit 
to serve as the Screening Visit for MG0007. 
Table 1-1: 
Schedule of activities 
 
Week 
Weeks 1-4 
Weeks 5-8 
Weeks 9-48a 
Weeks 49-51 Week 52 Week 60 
Assessments 
Visitb 
1 
(Screening
/Baseline)c  2-4 
5 
6 
7 
8 
1st visit 
2nd, 
3rd, and  
4th 
visits 
49 
50 
51 
52  
or PEOT 
53 
EOS 
Visit 
type 
S 
S 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
Written informed consent 
X 
 
 
 
 
 
 
 
 
 
 
 
Verification of inclusion/exclusion criteria 
Xd 
 
 
 
 
 
 
 
 
 
 
 
Withdrawal criteria 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Medical history update 
X 
 
X 
 
 
 
X 
 
X 
 
X 
X 
Concomitant medications and medical 
procedures 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Body weighte 
X 
 
 
 
 
 
X 
 
 
 
X 
X 
Vital signs 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
C-SSRSf 
X 
 
 
 
 
 
 
 
 
 
 
 
Query for suicidalityf 
 
X 
X 
 
X 
 
X 
 
X 
 
X 
X 
MGFA classification 
 
 
 
 
 
 
 
 
 
 
X 
X 
QMG 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
MG-C Scale 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
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Table 1-1: 
Schedule of activities 
 
Week 
Weeks 1-4 
Weeks 5-8 
Weeks 9-48a 
Weeks 49-51 Week 52 Week 60 
Assessments 
Visitb 
1 
(Screening
/Baseline)c  2-4 
5 
6 
7 
8 
1st visit 
2nd, 
3rd, and  
4th 
visits 
49 
50 
51 
52  
or PEOT 
53 
EOS 
Visit 
type 
S 
S 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
MG-ADL 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
MG Symptoms PRO 
X 
 
X 
 
  
 
X 
 
X 
 
X 
X 
EQ-5D-5L 
X 
 
 
 
 
 
X 
 
 
 
X 
 
MGIIg 
X 
 
 
 
 
 
X 
 
 
 
X 
 
MGQoL15r 
X 
 
 
 
 
 
X 
 
X 
 
X 
X 
Full physical examination 
X 
 
 
 
 
 
 
 
 
 
X 
X 
Brief physical examination 
 
X 
X 
 
X 
 
X 
 
X 
 
 
 
12-lead ECG  
X 
 
X 
 
 
 
Xh 
 
 
 
X 
X 
Pregnancy test (urine)i 
X 
  
  
  
  
  
X 
 
X 
 
X 
X 
Hematology, serum chemistry, urinalysis 
X 
X 
 
 
X 
 
X 
 
X 
 
X 
X 
PTT and INR measurements 
X 
  
 
 
 
 
 
 
 
 
X 
X 
Serology testing for HIV, hepatitis B, and 
hepatitis C 
X 
 
 
 
 
 
 
 
 
 
 
 
Anti-tetanus toxoid titer  
 
 
 
 
 
 
Xj 
 
 
 
 
X 
IGRA tuberculosis test 
 
 
 
 
 
 
 
 
 
 
X 
X 
Tuberculosis signs and symptoms 
questionnaire 
X 
 
X 
 
 
 
Xk 
 
 
 
X 
X 
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Table 1-1: 
Schedule of activities 
 
Week 
Weeks 1-4 
Weeks 5-8 
Weeks 9-48a 
Weeks 49-51 Week 52 Week 60 
Assessments 
Visitb 
1 
(Screening
/Baseline)c  2-4 
5 
6 
7 
8 
1st visit 
2nd, 
3rd, and  
4th 
visits 
49 
50 
51 
52  
or PEOT 
53 
EOS 
Visit 
type 
S 
S 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
Contact IRT 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Study participants identification card 
assigned 
X 
 
 
 
 
 
 
 
 
 
 
 
Recording of AEs 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Blood sampling for PKn 
X 
 
X 
 
 
 
 
 
 
 
X 
 
Blood sampling for ADA  
X 
 
X 
 
 
 
Xo 
 
 
 
X 
X 
Blood sampling for IgG and IgG 
subclassesp 
X 
X 
X 
 
X 
 
X 
 
X 
 
X 
X 
Blood sampling for IgA, IgM, IgE 
X 
 
 
 
 
 
Xq 
  
 
 
Xr 
X 
Blood sampling for exploratory safety 
biomarker analysiss 
  
X 
 
 
 
 
 
 
 
 
 
 
MG-specific autoantibodies 
X 
 
X 
 
 
 
Xo 
 
 
 
X 
X 
Serum complement levels (C3,C4) and 
cytokinest 
 
X 
 
 
 
 
X 
 
 
 
 
 
Plasma complement levels (C3a and C5a) 
t  
 
X 
 
 
 
 
X 
 
 
 
 
 
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Table 1-1: 
Schedule of activities 
 
Week 
Weeks 1-4 
Weeks 5-8 
Weeks 9-48a 
Weeks 49-51 Week 52 Week 60 
Assessments 
Visitb 
1 
(Screening
/Baseline)c  2-4 
5 
6 
7 
8 
1st visit 
2nd, 
3rd, and  
4th 
visits 
49 
50 
51 
52  
or PEOT 
53 
EOS 
Visit 
type 
S 
S 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
ADA=anti-drug antibody; ADL=activities of daily living; AE=adverse event; AESM=adverse events of special monitoring; C-SSRS= Columbia Suicide 
Severity Rating Scale; ECG=electrocardiogram; EOS=End of Study; EQ-5D-5L=5-level European quality of life 5 dimension; GI=gastrointestinal; H=home 
visit; HIV=human immunodeficiency virus; Ig=immunoglobulin; IGRA=interferon gamma release assay; IMP=investigational medicinal product; 
INR=international normalized ratio; IRT=interactive response technology; MG=myasthenia gravis; MGII=myasthenia gravis impairment index; MG-
ADL=myasthenia gravis-activities of daily living; MGFA=Myasthenia Gravis Foundation of America; MGQoL15r=revised 15-item myasthenia gravis 
quality of life questionnaire; PEOT=premature end of treatment; PK=pharmacokinetic(s); PRO=patient-reported outcome; PTT=partial thromboplastin time; 
QMG=quantitative myasthenia gravis test; S=site visit 
a From Weeks 9 to 48, the schedule will operate on recurring 4-week cycles wherein the first visit of each 4 weeks will be site visits and the subsequent 3 visits 
(2nd, 3rd, and 4th visits) will be home visits. Accordingly, Visits 9, 13, 17, 21, 25, 29, 33, 37, 41, and 45 will be site visits and the remaining visits will be 
home visits. At the discretion of the Investigator, home visits may be changed to site visits. Study participants may also choose to complete the visits at the site 
on the scheduled home visits. 
b Visit windows of ±2 days are allowed for all visits. 
c For any study participant who enrolls in MG0004, the final visit in MG0003 (Visit 14) or MGC003 (Visit 14) will serve as Visit 1 in MG0004. All activities 
should be completed at Visit 1 (±1 week). 
d For criteria pertaining to laboratory measures, the last available value from MG0003 or MGC003 will be used for evaluation of study participant eligibility, as 
long as the measurement was taken within the last 4 weeks (not older than 32 days) prior to MG0004 screening. 
e To be collected four times during the study: Screening/Baseline,1 Week 25 (site visit), Week 52 (or PEOT), and EOS Visit. 
f A full Columbia-Suicide Severity Rating Scale (C-SSRS) assessment will be performed only when study participant has a positive response to Question 1 of 
the suicidal ideation query. If a study participant has active suicidal ideation as confirmed by the answer “Yes” to Question 4 or Question 5 of the C-SSRS 
assessments, the study participant will be excluded or withdrawn from the study and immediately referred to a mental healthcare professional. Complete C-
SSRS will observe the “since last visit” version recorded at Visit 14 in MG0003 or MGC003. 
g MGII is optional for all study participants. 
h From Weeks 9 to 48, an ECG will be performed every 3 months (ie, Weeks 9, 21, 33, and 45). 
i Urine pregnancy test will be performed using a dipstick test; a positive urine pregnancy test must be confirmed using a serum pregnancy test. 
j Every 6 months. 
k From Weeks 9 to 48, the tuberculosis questionnaire will be performed every 3 months (ie, Weeks 9, 21, 33, and 45). 
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l Study participants must be observed at site postdose for at least 4 hours following the first 2 infusions, and then 2 hours thereafter for subsequent infusions until 
Week 7, where the participant can be observed for 1 hour at the Investigator’s discretion. 
m No IMP administration will occur during the PEOT visit for study participants who withdraw from the study or discontinue IMP. 
n PK samples should be taken predose for all study participants. 
o From Weeks 9 to 48, ADA- and MG-specific autoantibodies will be measured every 3 months (ie, Weeks 9, 21, 33, and 45). 
p Total IgG will be assessed at all scheduled onsite visits; IgG subclasses will be assessed at Visit 1 and every 3 months thereafter. 
q From Weeks 9 to 48, blood sampling for IgA, IgE and IgM will be measured every 3 months (ie, Weeks 9, 21, 33, and 45). 
r To be performed only in the event of a PEOT Visit. 
s Baseline from MG0003 and MGC003 will serve as Baseline in MG0004. Additional exploratory safety samples must be collected 4 hours postevent or as soon 
as possible before the next IMP in case of AESM of serious or severe headache or AESM of GI disorders as described in Section 8.9. 
t Baseline from MG0003 and MGC003 will serve as Baseline in MG0004. Additional samples may be collected 2 hours and 4 hours postevent in case of severe 
headache, infusion reaction, or hypersensitivity reaction at Visits 5, 7, 1st visit (of the 4-week cycle from Weeks 9-48) and Visit 49. 
 
 
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2 
INTRODUCTION 
Rozanolixizumab is a humanized IgG4 monoclonal antibody that is being developed as an 
inhibitor of the activity of the FcRn for IgG. 
By blocking the activity of FcRn, rozanolixizumab accelerates the catabolism of IgG antibodies, 
including IgG autoantibodies. The aim is to reduce the concentration of pathogenic IgG in study 
participants with autoimmune diseases mediated by the action of IgG autoantibodies. 
The FcRn recycles IgG and albumin and transports it bidirectionally across epithelial barriers. 
Recent studies have shown that FcRn rescues both IgG and albumin from intracellular lysosomal 
degradation by recycling it from the sorting endosome to the cell surface (Roopenian and 
Akilesh, 2007). FcRn may also mediate transcytosis of IgG to facilitate its distribution within 
tissues. Rozanolixizumab has been specifically designed to block IgG binding to FcRn without 
blocking the binding and recycling of albumin. 
Rozanolixizumab binds with high affinity to FcRn at both neutral and acidic pH. 
Immunoglobulin G that is constitutively taken up by pinocytosis into cells fails to bind to FcRn, 
even at the acidic pH found in the endosome. It is therefore not recycled and is trafficked to the 
lysosomes for degradation. 
Production of pathogenic IgG autoantibodies is the major pathophysiology leading to a number 
of autoimmune diseases, which include MG, pemphigus vulgaris (PV), immune 
thrombocytopenia (ITP), Goodpasture’s syndrome, neuromyelitis optica, Guillain-Barré 
Syndrome (GBS), and chronic inflammatory demyelinating polyradiculoneuropathy. 
As individual disease entities, IgG autoantibody-mediated conditions are relatively rare. 
Treatment of these disorders remains a difficult clinical problem, requiring in many of these 
conditions the long-term use of corticosteroids alone or combined with cytotoxic agents. These 
therapeutic approaches are not effective in all study participants and conditions and have broad 
immunosuppressive effects causing considerable toxicity and treatment-related morbidity. 
Treatments aimed at reducing the quantity of circulating IgG autoantibodies, including 
plasmapheresis, immunoadsorption, or high dose IVIg, are being used for primary and secondary 
therapy of autoimmune disease, particularly where corticosteroid based immune suppression is 
not or no longer effective (eg, ITP, MG, GBS, PV). The therapeutic approach of these treatments 
is thought in part to be based on lowering levels of pathogenic autoantibodies, which represents 
rational and effective treatment modalities of autoimmune diseases. 
Therefore, specific removal of the IgG autoantibodies by FcRn blockade may provide an 
effective therapeutic option for IgG autoantibody-mediated autoimmune disorders. 
More detailed information regarding the nonclinical and clinical development programs for 
rozanolixizumab, including all completed and ongoing studies, can be found in the latest version 
of the Investigator’s Brochure (IB). 
2.1 
Study rationale 
Myasthenia gravis is a serious, debilitating, sometimes life-threatening condition that is 
associated with numerous symptoms, including muscular weakness and fatigue. The major 
pathophysiology leading to MG is the abnormal production of IgG autoantibodies directed 
toward nicotinic AChR, or MuSK protein. Several commonly prescribed treatments act, at least 
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in part, by reducing the quantity of such circulating IgG autoantibodies. While the standard of 
care for MG involves the utilization of a variety of therapeutic agents including cholinesterase 
inhibitors, immunomodulators, corticosteroids, biologics interfering with IgG turnover, 
high-dose IVIg, plasmapheresis, or immunoadsorption, there remains a need for a safe and 
effective treatment devoid of significant side effects to conveniently treat study participants with 
MG. 
By blocking the activity of FcRn, rozanolixizumab accelerates the catabolism of antibodies and 
reduces the concentration of pathogenic IgG in MG study participants, thus offering a safe, 
effective, and convenient alternative to existing treatments. A Phase 3 study (MG0003) is 
underway to establish the safety and efficacy of rozanolixizumab in study participants with 
generalized MG who experience moderate-to-severe symptoms. 
This open-label extension study (MG0004) will evaluate the long-term safety, tolerability, and 
efficacy of rozanolixizumab. 
2.2 
Background 
Rozanolixizumab is a humanized anti-FcRn monoclonal antibody that has been specifically 
designed to inhibit IgG binding to FcRn without inhibiting albumin binding to FcRn. 
Rozanolixizumab is being developed as an inhibitor of FcRn activity with the aim to reduce the 
concentration of pathogenic IgG in study participants with IgG autoantibody-mediated diseases. 
To date, rozanolixizumab has been administered to human study participants in 6 completed or 
ongoing clinical studies: UP0018, MG0002, MG0003, TP0001, CIDP01, and UP0060. UP0018 
is a completed first-in-human study, MG0002 is a completed Phase 2 study in study participants 
with generalized MG, and TP0001 is a completed Phase 2 study in study participants with 
primary ITP, and UP0060 is an ongoing Phase 1 study comparing the safety, tolerability, 
pharmacokinetics (PK), and pharmacodynamics (PD) of rozanolixizumab in Chinese, Japanese 
and Caucasian healthy volunteers. 
In MG0002, clinically relevant improvements in day-to-day functioning, as measured by change 
from Baseline to Day 29 in myasthenia gravis-activities of daily living (MG-ADL) (secondary 
endpoint), were observed following treatment with rozanolixizumab 
 compared with 
placebo (p=0.036). Numerical differences numerical in favor of rozanolixizumab 
 
compared with placebo were observed in reductions from Baseline in quantitative myasthenia 
gravis (QMG) (p=0.221) and MG-C score (p=0.089). Overall, repeated administrations of 
rozanolixizumab at dose levels of 
 SC have been generally well tolerated, 
with an acceptable safety profile. No new safety concerns were identified. The treatment-
emergent adverse event (TEAE) profile was similar between rozanolixizumab and placebo 
except for headaches where increased frequency and severity was observed in the 
rozanolixizumab study participants. 
Final data from the proof-of-concept ITP study TP0001 demonstrated that rozanolixizumab was 
tolerated with an acceptable safety profile after multiple 
 and single 
 
 doses. 
MG0003 is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the 
efficacy and safety of 2 doses of rozanolixizumab (equivalent to approximately 
 
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) in study participants with generalized MG who experience moderate to severe 
symptoms and are being considered for treatment with IVIg or PEX. 
This study (MG0004) is an open-label extension study for the global lead-in study MG0003 and 
China specific lead-in study, MGC003 (this study will mirror the study design of MG0003). 
MG0004 will evaluate the long-term safety, tolerability, and efficacy of rozanolixizumab in 
study participants with generalized MG. Study participants in MG0004 will be randomized to 
receive 1 of 2 doses of rozanolixizumab (equivalent to approximately 
, 
respectively) over a 52-week Treatment Period. 
2.3 
Benefit/Risk assessment 
Generalized MG is a rare, debilitating, chronic autoimmune disease driven by, in large part, IgG 
autoantibodies that target neuromuscular junctions (NMJs). Most current treatment approaches 
are not targeted treatments to the specific underlying pathology of IgG autoantibody formation, 
but rather they produce a broad cascade of immune suppression, which results in undesirable 
side effects such as those seen with high-dose chronic steroid use. Many treatments of choice 
often require invasive, expensive, and time-consuming inpatient procedures such as PEX, or 
intravenous (IV) administration of immunoglobulins at a healthcare facility. 
Rozanolixizumab represents an innovative, SC anti-FcRn monoclonal antibody that may provide 
a novel and specific therapeutic approach for the treatment of study participants with MG. Data 
show that rozanolixizumab markedly lowers serum IgG and IgG autoantibody levels in study 
participants with generalized MG. The completed Phase 2 study MG0002 established supportive 
evidence of efficacy for the treatment of MG, achieving significant and clinically meaningful 
improvements to Day 29 in MG-ADL with rozanolixizumab 
 compared with placebo 
(p=0.036). Repeated administrations of rozanolixizumab were generally well tolerated, with an 
acceptable safety profile and in line with SC dosing in the Phase 1 program and the safety profile 
observed in TP0001. 
The potential risks associated with SC administration of rozanolixizumab are gastrointestinal 
(GI) disturbances, headaches, infusion/hypersensitivity reactions, infections, potential effects on 
immunizations, effects on the kidney, and metabolic effect of FcRn on albumin. These risks can 
be mitigated by careful monitoring, exclusion of at-risk study participants, and appropriate 
protocol withdrawal and stopping criteria. Additionally, protocol guidance for management of GI 
disturbances, severe headaches, and infusions/hypersensitivity reactions is also provided as well 
as expedited reporting requirements of adverse events of special monitoring (AESMs). 
More detailed information about the known and expected benefits and risks and reasonably 
expected adverse events (AEs) of rozanolixizumab may be found in the IB. 
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3 
OBJECTIVES AND ENDPOINTS 
Objectives 
Endpoints 
Primary 
To evaluate the long-term safety and tolerability 
of rozanolixizumab in study participants with 
generalized MG 
The primary safety endpoints are: 
• 
Occurrence of TEAEs 
• 
Occurrence of TEAEs leading to permanent 
withdrawal of study medication 
The other safety endpoints are: 
• 
Occurrence of AESMs 
• 
Vital sign values and changes from Baseline 
(systolic and diastolic blood pressure and 
pulse rate) 
• 
12-lead ECG values and change from 
Baseline at each scheduled assessment  
• 
Clinical laboratory findings (hematology, 
biochemistry, and urinalysis) 
Secondary 
To evaluate the long-term efficacy of 
rozanolixizumab in study participants with 
generalized MG 
The secondary efficacy endpoints are: 
• 
Change from Baseline in MG-ADL at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in MG-C score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in QMG at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Use of rescue medication (IVIg or PEX) 
The other efficacy endpoints are: 
• 
Change from Baseline in MGII scores at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in MGII ocular 
subscores at each scheduled assessment 
during Treatment and Observation Periods 
• 
Change from Baseline in MGII generalized 
domain subscores at each scheduled 
assessment during Treatment and 
Observation Periods 
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• 
Change from Baseline in the MG Symptoms 
PRO ‘Muscle Weakness Fatigability’ score at 
each scheduled assessment during Treatment 
and Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Physical Fatigue’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Bulbar symptoms’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Respiratory symptoms’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO ‘Ocular symptoms’ score at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the MG Symptoms 
PRO multicomponent total score, at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in the enhanced MG 
Symptoms PRO total score, at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in MGQoL15r at each 
scheduled assessment during Treatment and 
Observation Periods 
• 
Change from Baseline in EQ-5D-5L scores at 
each scheduled assessment during Treatment 
and Observation Periods 
For study participants who require rescue 
treatment during the Observation Period of 
MG0003 and subsequently enter MG0004, the 
following endpoints will be assessed: 
• 
QMG responder rate (≥3.0 point 
improvement from Baseline) at each 
scheduled assessment during the first 
6 weeks of the Treatment Period 
• 
MG-C responder rate (≥5.0 point 
improvement from Baseline) at each 
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scheduled assessment during the first 
6 weeks of the Treatment Period 
• 
MG-ADL responder rate (≥2.0 point 
improvement from Baseline) at each 
scheduled assessment during the first 
6 weeks of the Treatment Period 
Other 
To assess the reduction in use of steroids in study 
participants receiving rozanolixizumab 
• 
AUC of the oral steroid dose over time 
To assess the plasma concentrations of 
rozanolixizumab administered by SC infusion 
• 
Plasma concentration of rozanolixizumab at 
each scheduled assessment during the 
Treatment and Observation Periods 
To assess the pharmacodynamic effects of 
rozanolixizumab as measured by IgG levels, IgG 
subclasses, and MG-specific autoantibodies levels 
• 
Change from baseline in total IgG and IgG 
subclasses autoantibodies at each scheduled 
visit  
• 
Change from baseline in MG-specific 
autoantibodies at each scheduled visit 
To evaluate the incidence and emergence of 
ADAs with respect to immunogenicity, PK, and 
pharmacodynamics 
• 
ADAs at each scheduled assessment during 
the Treatment Period and Observation 
Periods 
To assess the effect of rozanolixizumab on 
biomarkers including IgM, IgA, and IgE, serum 
and plasma complement levels, and serum 
cytokines 
• 
Change from Baseline in serum complement 
levels (C3 and C4) and plasma complement 
levels (C3a and C5a) at each scheduled 
assessment during Treatment Perioda 
• 
Change from Baseline in serum cytokines at 
each scheduled assessment during Treatment 
and Observation Periodsa 
• 
Change from Baseline in serum 
immunoglobulin concentrations (total IgA, 
IgE, and IgM) at each scheduled assessment 
during Treatment and Observation Periods 
To assess the effect of rozanolixizumab on tetanus 
IgG antibodies 
• 
Change from Baseline in anti-tetanus toxoid 
serum titers at each scheduled assessment 
during Treatment and Observation Period 
ADA=anti-drug antibody; ADL=activities of daily living; AESM=adverse event of special monitoring; 
ECG=electrocardiogram; Ig=immunoglobulin; IVIg=intravenous infusion of immunoglobulin G; 
MG=myasthenia gravis; MG-ADL=myasthenia gravis-activities of daily living; MGII=myasthenia gravis 
impairment index; MGQoL15r=revised 15-item myasthenia gravis quality of life questionnaire; PEX=plasma 
exchange; PK=pharmacokinetic; PRO=patient-reported outcome; QMG=quantitative myasthenia gravis test; 
SC=subcutaneous(ly); TEAE=treatment-emergent adverse event 
a To be performed for study participants with severe and/or serious headache and/or infusion reaction or 
hypersensitivity reaction. Other exploratory safety biomarkers may be assessed. 
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Estimands  
The aim of MG0004 is to compare safety (primarily) of long-term treatment of 2 dose levels of 
rozanolixizumab. Rescue medication is allowed and is expected to be temporary; dose switching 
(up or down) is also allowed. Using a treatment policy estimand is considered the only viable 
option to deal with this complexity. Indeed, a hypothetical estimand that appropriately accounted 
for treatment switching in this study, differing lengths of treatment in MG0004, and differing 
exposure to rescue medications in both studies would be very assumption-rich. This estimand 
will be based on the Safety Set (SS). 
For all variables, the population is the study participants meeting the protocol-specified 
inclusion/exclusion criteria. The main intercurrent events are the use of rescue medication during 
the course of the study, withdrawal due to TEAEs and treatment switching. 
All safety variables, biomarkers, and anti-drug antibody (ADA) results will be summarized for 
the SS classified by first treatment dose received 
 in this study (ie, before 
any switching occurs), both with and without subdivision by treatment dose received in MG0003 
(placebo or 
). 
Efficacy variables will be summarized using a ‘treatment policy’ approach as follows: 
• Participant-level outcome:  The given variable and time point being summarized (eg, value of 
MG-ADL at Week 5). 
• Population-level summary measure: For categorical variables, unadjusted proportions by first 
treatment received and, for continuous variables, unadjusted means by first treatment 
received. 
Missing values will not be imputed. 
Further details will be provided in the Statistical Analysis Plan (SAP). 
4 
STUDY DESIGN 
4.1 
Overall design 
This is a randomized, open-label Phase 3 extension study to evaluate the long-term safety, 
tolerability, and efficacy of rozanolixizumab in study participants with generalized MG. A study 
schema is provided in Figure 1-1 and a Schedule of Activities is provided in Table 1-1. 
Study participants will enter MG0004 from lead-in studies MG0003 or MGC003. Study 
participants in MG0003 or MGC003 who complete the Observation Period or who require rescue 
medication during the Observation Period will be invited to be rerandomized in MG0004 (except 
for study participants who opt to receive IVIg or PEX as rescue medication). Study participants 
who receive IVIg or PEX as rescue therapy during the Observation Period of MG0003 or 
MGC003 will not be eligible to enroll in MG0004. Study participants who discontinue study 
medication in MG0003 or MGC003 for any reason other than requiring rescue medication will 
not be eligible for MG0004. 
For any study participant who enrolls in MG0004, the final visit in MG0003 (Visit 14) or 
MGC003 will serve as the first visit in MG0004 (Visit 1). All activities should be completed at 
Visit 1 (±1 week). For criteria pertaining to laboratory measures, the last available value from 
MG0003 or MGC003 will be used for evaluation of study participant eligibility, as long as the 
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measurement was taken within the last 4 weeks (not older than 32 days) prior to MG0004 
Screening. Up to approximately 276 study participants will be enrolled into the lead-in study 
MG0003 and MGC003. All eligible study participants from MG0003 or MGC003 will be invited 
to participate in MG0004. 
In MG0004, study participants will be randomized in a 1:1 ratio to receive 1 of 2 doses of 
rozanolixizumab (equivalent to approximately 
, respectively). Specifically, 
the following dose arms will be used in the study: 
Dose arm 1: equivalent to approximately 
 rozanolixizumab 
• Bodyweight <50kg: dose to be administered 
 
• Bodyweight ≥50kg and <70kg: dose to be administered 
 
• Bodyweight ≥70kg and <100kg: dose to be administered 
 
• Bodyweight ≥100kg: dose to be administered 
 
Dose arm 2: equivalent to approximately 
 rozanolixizumab 
• Bodyweight <50kg: dose to be administered 
 
• Bodyweight ≥50kg and <70kg: dose to be administered 
 
• Bodyweight ≥70kg and <100kg: dose to be administered 
 
• Bodyweight ≥100kg: dose to be administered 
 
If one of the doses described above is determined to be futile and is discontinued after the 
interim analysis in MG0003 or MGC003, then that dose arm will be dropped from MG0004 as 
well, and study participants in the affected dose arm will be transferred to the continuing dose 
arm. If both dose arms are used in MG0004, study participants will be allowed to switch dose 
arms for tolerability and efficacy reasons at the discretion of the Investigator (see Section 6.6). 
In MG0004, rozanolixizumab will be administered SC on a 
 basis over a 52-week 
Treatment Period. Weekly visits will be either on-site or at home, as described in the Schedule of 
Activities (Section 1.3). During the first 4 weeks, the weekly visits will be on-site. From 
Weeks 5 to 8, on-site visits and at home visits will occur on an alternating basis. From Weeks 9 
to 48, study participants will have 1 site visit every 4 weeks, such that 1 site visit will be 
followed by 3 home visits, unless the study participant chooses to complete the scheduled 
at-home assessments at the site. Weeks 49 to 51 will then involve 1 site visit followed by 2 home 
visits. At the end of the 52-week Treatment Period in MG0004, study participants will participate 
in an 8-week Observation Period. 
In exceptional circumstances (eg, pandemic, hurricanes, etc) where study-specific investigations 
may not be conducted according to study protocol, contingency measures will be in place (see 
Section 8). 
This OLE study will be replaced by MG0007, an OLE study with 
 based 
on MG worsening. Upon study site approval of MG0007, as well as fulfilment of regulatory 
requirements, study participants will have the opportunity to rollover directly into MG0007 and 
will no longer have the opportunity to rollover to MG0004. In the event a study participant has 
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already started MG0004, a minimum treatment duration of 6 visits must be completed (if IMP 
treatment is withheld for low IgG, study participants missed dose(s) can be counted as part of the 
total 6 visits for completion of MG0004 and meet eligibility requirements for MG0007) and then 
the participant will discontinue MG0004 and have the opportunity to enter into MG0007. For 
study participants entering MG0007 from the treatment period of MG0004, he/she must 
complete the PEOT Visit in MG0004 which will serve both as the Screening Visit for MG0007 
and as the EOS for MG0004. If a study participant completes the Treatment Period and is in the 
Observation Period of MG0004, he/she will then complete the EOS visit to serve as the 
Screening Visit for MG0007. Once the site is active for MG0007, no further participants will 
enroll in MG0004 and the study will be closed at the site once all eligible study participants have 
rolled over into MG0007. 
An IDMC will be formed to monitor the ongoing safety of the study. The IDMC will review 
implications of individual and cumulative cases to continuance of the study in an ongoing 
fashion. 
4.2 
Scientific rationale for study design 
The lead-in studies, MG0003 and MGC003, will evaluate the efficacy, safety, and tolerability of 
rozanolixizumab compared with placebo in study participants with generalized MG who are 
treated with study medication over a 
 at 
. Study 
participants in the lead-in studies will have generalized MG, as evidenced by the presence of 
anti-AChR or anti-MuSK antibodies. Additionally, study participants in the lead-in studies will 
have moderate to severe symptoms of MG and should be under consideration for IVIg or PEX 
therapy, indicating that they are in need of additional therapeutic intervention. 
Study participants in MG0003 or MGC003 will be randomized to receive either placebo or 1 of 2 
doses of rozanolixizumab. Study participants in MG0003 or MGC003 who require rescue 
medication (other than IVIg or PEX) during the Observation Period will be invited to be 
rerandomized in MG0004: this will offer all study participants, even those previously treated 
with placebo, a chance to be treated with rozanolixizumab. Rerandomization in MG0004 is 
applied to account for the limited efficacy information on the 
 dose. Due to the open-
label design of MG0004, study participants are allowed to switch dose at the Investigator's 
discretion during the course of the study (dose modification recommendations for safety are 
provided in Section 6.6). Study participants developing TEAEs could be switched to a dose 
equivalent to 
 and study participants experiencing symptom worsening can be switched to 
a dose equivalent to 
. 
MG0004 will then evaluate the long-term safety, tolerability, and efficacy of rozanolixizumab 
over a 52-week Treatment Period. An open-label study with a treatment duration of 52 weeks is 
considered sufficient to adequately assess long-term safety with rozanolixizumab for the 
proposed indication. The dose regimen for rozanolixizumab in MG0004 will be the same one 
that is used in MG0003 and MGC003 (equivalent to approximately 
), 
although the dose may be modified based on interim analysis in MG0003 (Section 4.3). In 
MG0004, study participants will be rerandomized to 1 of 2 doses of rozanolixizumab, and will 
be allowed to switch between dose arms at the discretion of the Investigator. MG0004 will 
therefore allow study participants to switch their dose of study medication on an individual basis. 
MG0004 also offers study participants the chance to benefit from long-term treatment. MG0004 
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will conclude with an 8-week Observation Period, which is the anticipated duration of time 
required for antibodies to return to Baseline levels after rozanolixizumab treatment. 
4.3 
Justification for dose 
The dose regimen for rozanolixizumab in MG0004 will be the same one that is used in MG0003 
and MGC003 (see Section 4.1 for details of dosing according to weight). The fixed dosing units 
were introduced to support the potential for study participant self-injection in a home setting and 
doses have been defined to reach PD effect on IgG levels throughout a weight range that is 
similar to weight-based dosing. 
The proposed doses and regimen of rozanolixizumab in MG0003 were based on the results from 
first-in-human study UP0018, alongside the efficacy and safety data from the MG Phase 2 study 
(MG0002), and safety and tolerability data from TP0001. UP0018 evaluated the effect of a single 
dose of rozanolixizumab (administered at doses of 
 as IV or SC 
infusions) in healthy study participants. The mean absolute decreases in IgG and mean percent 
change from Baseline IgG were greater in the active dose groups compared with placebo-treated 
study participants, and rozanolixizumab treatment was tolerated with an acceptable safety 
profile. 
MG0002 evaluated the effect of repeated doses of rozanolixizumab (administered at doses of 
 as SC infusions) in study participants with generalized MG. Significant 
improvements in day-to-day functioning (measured by change from Baseline to Day 29) were 
observed. In general, responder rates for quantitative myasthenia gravis (QMG) score, MG-C 
score, and MG-activities of daily living (ADL) score were higher for the rozanolixizumab 
 group than the placebo group. Serum total IgG concentrations and AChR autoantibodies 
rapidly decreased from Baseline in the rozanolixizumab 
 group, with a mean nadir of 
3.3g/L for total IgG (~70% reduction from Baseline). Overall, repeated administrations of 
rozanolixizumab were generally well tolerated, with an acceptable safety profile. 
TP0001 indicated that rozanolixizumab dose higher than 
 was tolerated with an 
acceptable safety profile after multiple 
 and 
 
 doses. 
Based on these results, 2 SC rozanolixizumab treatment arms, equivalent to 
 
, were selected for MG0003. A population PK-PD model that characterizes the dose-
exposure-IgG relationship was used to guide, through simulation, the choice of fixed unit doses 
at each weight bracket that achieved equivalent IgG reductions (mean and 90% prediction 
interval) to the weight-based (mg/kg) dosing regimens studied previously. The proposed 
rozanolixizumab dosing regiments were selected to provide maximal reduction in autoantibody 
concentration and result in clinically significant improvements in MG-ADL score. 
Since MG0004 is the open-label extension study for MG0003 and MGC003, the same 
rozanolixizumab dose regimens will also be used in all 3 studies. MG0004. However, if any 
safety concerns are identified in the lead-in studies, or if one of the dose arms is determined to be 
futile and is discontinued in MG0003, corresponding changes will be made in the MG0004 
dosing scheme. If both dose arms are used in MG0004, study participants will be allowed to 
switch dose arms for tolerability and efficacy reasons at the discretion of the Investigator (see 
Section 6.6 for dose modification recommendations for safety). 
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4.4 
End of study definition 
A participant is considered to have completed the study if he/she has completed all phases of the 
study including the End of Study (EOS) Visit (Week 60; see Section 1.3). The end of the study is 
defined as the date of the last visit of the last participant in the study. The maximum study 
duration per study participant is 60 weeks. 
5 
STUDY POPULATION 
5.1 
Inclusion criteria 
Participants are eligible to be included in the study only if all of the following criteria apply: 
Age 
1. Participant must be ≥18 years of age at the time of signing the informed consent. 
Type of participant and disease characteristics 
2a. Participant was eligible for MG0003 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. 
Weight 
3a. Body weight ≥35kg at Visit 1. 
Sex 
4. Participants may be male or female. 
− A male participant must agree to use contraception as detailed in Appendix 4 
(Section 10.4) of this protocol during the Treatment Period and for at least 90 days 
after the last dose of study treatment and refrain from donating sperm during this 
period. 
− Female study participants of childbearing potential must agree to use a highly 
effective method of birth control during the study and for a period of at least 90 days 
after their final dose of study medication. According to the International Council for 
Harmonisation (ICH) M3 (R2), highly effective forms of birth control are methods 
that achieve a failure rate of less than 1% per year when used consistently and 
correctly. 
− A female participant is eligible to participate if she is not pregnant (see Appendix 4, 
Section 10.4), not breastfeeding, and at least one of the following conditions applies: 
o Not a woman of childbearing potential (WOCBP) as defined in Appendix 4 
(Section 10.4)  
OR 
o A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 
(Section 10.4) during the Treatment Period and for at least 90 days after the last 
dose of study treatment. 
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Informed consent 
5. Capable of giving signed informed consent as described in Appendix 1 (Section 10.1) which 
includes compliance with the requirements and restrictions listed in the informed consent 
form (ICF) and in this protocol. 
5.2 
Exclusion criteria 
For criteria pertaining to laboratory measures, the last value from MG0003 or MGC003 will be 
used for evaluation of study participant eligibility, as long as the measurement was taken within 
the last 4 weeks (not older than 32 days) prior to MG0004 screening. 
Unless otherwise stated, each criterion is applicable to the Screening visit (Visit 1). Participants 
are excluded from the study if any of the following criteria apply: 
Medical conditions 
1. Participant has any medical or psychiatric condition that, in the opinion of the Investigator, 
could jeopardize or would compromise the study participant’s ability to participate in this 
study. 
2a. Participant has a history of use disorder or other substance use disorder (as per Diagnostic 
and Statistical Manual of Mental Disorders-5) within the previous 12 months. 
3. Participant has a known hypersensitivity to any components of the study medication. 
4a. 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. 
Prior/Concomitant therapy 
5. 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. 
Prior/Concurrent clinical study experience 
6. Study participant has experienced hypersensitivity reaction after exposure to other anti-FcRn 
drugs. 
Diagnostic assessments 
7. Study participant with severe (defined as Grade 3 on the MG-ADL scale) weakness affecting 
oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis. 
8. Participant has absolute neutrophil count <1500 cells/mm3. 
9. 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. 
10. Participant has 12-lead electrocardiogram (ECG) with findings considered to be clinically 
significant upon medical review. The clinical significance of the findings needs to be 
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assessed by the Investigator to determine eligibility, and any queries regarding continuation 
of the study participant must be addressed with the Medical Monitor. 
11b.Study participant has renal impairment, defined as glomerular filtration rate less than 
45ml/min/1.73m2. 
12a.Study participant has >3x upper limit of normal (ULN) of any of the following at Visit 1: 
alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase 
(ALP), or bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is 
fractionated and direct bilirubin <35%). 
If study participant has elevations only in total bilirubin that are >ULN and <1.5xULN, 
fractionate bilirubin to identify possible undiagnosed Gilbert’s syndrome (ie, direct bilirubin 
<35%). 
For randomized study participants with a baseline result >ULN for ALT, AST, ALP, or total 
bilirubin but <1.5xULN, a baseline diagnosis and/or the cause of any clinically meaningful 
elevation must be understood and recorded in the electronic Case Report form (eCRF). 
Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit (>3xULN) may 
be repeated once for confirmation. 
13. Study participant has positive human immunodeficiency virus antibody test. 
Other exclusions 
14a.Study participant met any mandatory withdrawal or mandatory study drug discontinuation 
criteria MG0003 or MGC003, or discontinued study medication in either study, with the 
exception of discontinuation due to a need for rescue treatment 
15. Study participant is not considered capable of adhering to the protocol visit schedule, or 
medication intake according to the judgment of the Investigator. 
16. 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). 
17. Criterion removed.  
18. Study participant has corrected QT interval (QTc) >450 msec (for male participants) or 
QTc >470 msec (for female participants) or QTc >480 msec in participants with bundle 
branch block. 
5.3 
Lifestyle restrictions 
There are no lifestyle restrictions during the study unless deemed to interfere with compliance 
with the protocol as deemed by the Investigator. 
The use of cannabidiols and medicinal marijuana (prescribed by a physician) is permitted (see 
Section 6.5.1). 
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5.4 
Screen failures 
Screen failures are defined as participants who consent to participate in the clinical study but are 
not subsequently entered into the study. A minimal set of screen failure information is required 
to ensure transparent reporting of screen failure participants to meet the Consolidated Standards 
of Reporting Trials (CONSORT) publishing requirements and to respond to queries from 
regulatory authorities. Minimal information includes demography, screen failure details, 
eligibility criteria, and any serious adverse events (SAEs). 
If a study participant has 1 isolated test result outside the specific range which is deemed 
clinically nonsignificant, the abnormal value may be rechecked at the discretion of the 
Investigator, following discussion with the Sponsor’s medical monitor/study physician. 
Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit may be repeated 
once for confirmation. 
6 
STUDY TREATMENTS 
Study treatment is defined as any investigational treatment(s), intended to be administered to a 
study participant according to the study protocol. 
6.1 
Treatments administered 
A summary of study treatments is provided in Table 6-1. 
Table 6-1: 
Study treatments administered 
Study Treatment Name: 
Rozanolixizumab 
 
Rozanolixizumab 
 
Dosage formulation: 
Solution for infusion 
Solution for infusion 
Unit dose strength(s)/Dosage 
level(s): 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Route of administration 
SC 
SC 
Dosing instructions: 
 SC doses of 
rozanolixizumab for 52 weeks: 
Bodyweight <50kg: dose to be 
administered 
  
Bodyweight ≥50kg and <70kg: 
dose to be administered 
 
Bodyweight ≥70kg and <100kg: 
dose to be administered 
 
Bodyweight ≥100kg: dose to be 
administered 
 
 SC doses of 
rozanolixizumab for 52 weeks: 
Bodyweight <50kg: dose to be 
administered 
  
Bodyweight ≥50kg and <70kg: 
dose to be administered 
 
Bodyweight ≥70kg and <100kg: 
dose to be administered 
 
Bodyweight ≥100kg: dose to be 
administered 
 
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Table 6-1: 
Study treatments administered 
Study Treatment Name: 
Rozanolixizumab 
 
Rozanolixizumab 
 
Packaging and labeling 
Rozanolixizumab is manufactured, packaged, and labeled according 
to Good Manufacturing Practice guidelines and applicable laws or 
regulations. 
SC=subcutaneous(ly); w/v=weight/volume 
Note: 
 administration of rozanolixizumab will be either on-site or at home, as described in the Schedule of 
Activities (Section 1.3). During the first 4 weeks, the weekly visits will be on-site. From Weeks 5 to 8, on-site 
visits and at-home visits will occur on an alternating basis. From Weeks 9 to 48, study participants will have 
1 site visit every 4 weeks, such that 1 site visit will be followed by 3 home visits, unless the study participant 
chooses to complete the scheduled at-home visit at the site. Weeks 49 to 51 will then involve 1 site visit 
followed by 2 home visits. 
Details on the preparation of study treatment for infusion, rate of infusion, administration, 
appropriate records handling, and site personnel roles are provided in the investigational 
medicinal product (IMP) Handling Manual. All site personnel delegated to handle study 
treatment storage, preparation and administration must be trained to IMP Handling Manual.  
6.2 
Preparation, handling, storage, and accountability requirements 
The Investigator or designee must confirm appropriate temperature conditions have been 
maintained during transit for all study treatment received and any discrepancies are reported and 
resolved before use of the study treatment. 
Only participants enrolled in the study may receive study treatment and only authorized site staff 
may supply or administer study treatment. All study treatments must be stored in a secure, 
environmentally controlled, and monitored (manual or automated) area in accordance with the 
labeled storage conditions with access limited to the Investigator and authorized site staff. 
The Investigator, institution, or the head of the medical institution (where applicable) is 
responsible for study treatment accountability, reconciliation, and record maintenance 
(ie, receipt, reconciliation, and final disposition records). 
In case an out-of-range temperature is noted, it must be immediately reported as per instructions 
contained in the IMP Handling Manual. 
The Investigator (or designee) will instruct the participant to store the study medication 
following the instructions on the label. 
Further guidance and information for the final disposition of unused study treatment are provided 
in the IMP Handling Manual. 
6.2.1 
Drug accountability 
A Drug Accountability form will be used to record study medication dispensing and return 
information on a by-participant basis and will serve as source documentation during the course 
of the study. Details of any study medication lost, damaged (due to breakage or wastage), not 
used, partially used, disposed of at the study site, or returned to the Sponsor or designee must 
also be recorded on the appropriate forms. All supplies and pharmacy documentation must be 
made available throughout the study for UCB (or designee) to review. 
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The Investigator (or designee) is responsible for retaining all used, unused, and partially used 
containers of study medication until returned or destroyed. 
The Investigator may assign some of the Investigator’s duties for drug accountability at the study 
site to an appropriate pharmacist/designee. 
The Investigator must ensure that the study medication is used only in accordance with the 
protocol. 
Periodically, and/or after completion of the clinical phase of the study, all used (including empty 
containers)/partially used, unused, damaged, and/or expired study medication must be reconciled 
and either destroyed at the site according to local laws, regulations, and UCB Standard Operating 
Procedures or returned to UCB (or designee). Investigational medicinal product intended for the 
study cannot be used for any other purpose than that described in this protocol. 
6.3 
Measures to minimize bias: randomization 
An interactive response technology (IRT) will be used for assigning eligible participants to a 
treatment regimen (as applicable) based on a predetermined production randomization and/or 
packaging schedule provided by UCB (or designee). The randomization schedule will be 
produced by the IRT vendor. The IRT will generate individual assignments for kits of study 
medication, as appropriate, according to the visit schedule. 
To enroll a study participant (Visit 1), the Investigator or designee will contact the IRT and 
provide brief details about the participant to be enrolled. Study participants will retain the same 
5-digit number assigned in MG0003 or MGC003 that serves as the participant identifier 
throughout the study. The participant number will be required in all communication between the 
Investigator or designee and the IRT regarding a particular participant. Participant numbers and 
kit numbers will be tracked via the IRT. 
To randomize a participant, the Investigator or designee will contact the IRT and provide brief 
details about the participant to be randomized. The IRT will automatically inform the 
Investigator or designee of the participant’s randomization number. The IRT will allocate kit 
numbers to the participant based on the participant number during the course of the study. The 
randomization number must be incorporated into the eCRF. 
6.3.1 
Procedures for maintaining and breaking the treatment blind 
6.3.1.1 
Maintenance of study treatment blind 
This is an open-label study and treatment details (ie, dose arm) will not be blinded. 
6.3.1.2 
Breaking the treatment blind in an emergency situation 
Not applicable. 
6.4 
Treatment compliance 
Drug accountability must be recorded on the Drug Accountability form (Section 6.2.1). 
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6.5 
Concomitant medications/treatments 
6.5.1 
Permitted concomitant treatments (medications and therapies) 
The concomitant medications in Table 6-2 are permitted during the study for the treatment of 
MG, and dose adjustments are allowed during the study. 
Any violation of the permitted treatment criteria should be discussed with the Investigator, 
Sponsor, and Medical Monitor. 
Table 6-2: 
Permitted concomitant medications for the treatment of 
myasthenia gravis 
Permitted medications 
Dose 
Oral corticosteroids (ie, prednisolone) 
No specific requirements 
Methotrexate 
≤30mg/week 
Mycophenolate mofetil  
≤3g/day 
Cyclosporin a 
≤5mg/kg/day for unmodified 
≤4mg/kg/day for modified (microemulsion) 
Azathioprine 
≤3mg/kg/day 
Cholinesterase inhibitors 
≤600mg pyridostigmine/day 
Tacrolimus b 
≤5mg/day 
a Doses higher than those listed are permissible if the trough level is ≤300ng/mL. 
b If the total daily weight-based dose is >5mg, then a plasma trough level should be checked to ensure study 
participants are not above the recommended therapeutic range. 
The use of medicinal cannabidiols and medicinal marijuana (prescribed by a physician) is also 
permitted. 
6.5.2 
Prohibited concomitant treatments (medications and therapies) 
The following concomitant medications are prohibited during the study: 
• All biologics including rituximab 
• Cyclophosphamide 
• Pimecrolimus 
• IPP-201101 (Lupuzor™) 
• Immunoadsorption 
If a study participant needs or takes any prohibited medication or therapy, the Investigator will 
(where possible) discuss with the Medical Monitor and/or Sponsor’s Study Physician, and a 
decision will be made whether the study participant can continue in the study or must be 
withdrawn. If the study participant is treated with rituximab, the study participant must be 
withdrawn from the study medication, but should be encouraged to continue with Observation 
Period visits. 
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For study participants who require a medical or surgical procedure that requires the use of 
general anesthesia, discussion must occur prior to the procedure with the Medical Monitor or 
study physician. In an emergency situation, discussion should occur as soon as possible after the 
procedure, such that a decision on the study participant's continued participation in the study can 
be made. 
6.5.2.1 
Treatments specific to NMJ interference 
Treatments that could interfere with the function of the NMJ (and which therefore could impair 
study participants with MG) include, but are not limited to, the following medications: 
− botulinum toxin 
− aminoglycoside antibiotics 
− tetracycline antibiotics 
− penicillamine 
− magnesium 
For a more detailed list please refer to the Myasthenia Gravis Foundation of America medication 
list (https://myasthenia.org/What-is-MG/MG-Management/Cautionary-Drugs). The benefit-risk 
of starting these medications should be carefully considered by the Investigator, and where 
possible, the Investigator will discuss with the Medical Monitor and/or Sponsor’s Study 
Physician prior to initiating therapy that can affect the NMJ. 
6.5.3 
Rescue therapy 
Rescue therapy for the study will consist of IVIg or PEX. The study site will supply rescue 
therapy that will be obtained locally. 
Study participants who experience 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. 
If the study participant receives IVIg or PEX as rescue therapy during the study, treatment with 
rozanolixizumab should be discontinued or paused for a minimum of 2 weeks, but continue with 
visits as per Schedule of Activities, after which the participant may continue to receive 
rozanolixizumab at the discretion of the Investigator. This 2-week period may be extended at the 
discretion of the Investigator, but for no longer than 6 weeks. Following the temporary 
discontinuation of study medication, study participants should be restarted at the same dose of 
rozanolixizumab as previously. Study participants at a dose level of 
 rozanolixizumab can 
be restarted at 
 rozanolixizumab at the discretion of the Investigator. 
6.6 
Dose modification 
Study participants will be allowed to switch dose arms for tolerability and efficacy reasons, as 
well as bodyweight changes at the discretion of the Investigator after the first dose of 
rozanolixizumab. However, when possible, the dose assigned at randomization should be 
maintained for at least 6 weeks, unless a dose modification is required earlier for tolerability or 
efficacy reasons. If a dose adjustment is made, the Investigator should encourage study 
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participants to stay on the modified dose for at least 6 weeks, unless further dose adjustments or 
discontinuation of study medication is required for the safety of the study participant. 
Study participants who experience any AEs that meet the study medication discontinuation 
criteria (Section 7.1 and Section 0) should have rozanolixizumab discontinued. Study 
participants in the 
 cohort who experience a possibly related toxicity other than those 
that met the study drug discontinuation criteria as defined in Section 7.1 and Section 0 are 
allowed to switch to 
 at the Investigator’s discretion. Recommended dose modifications 
due to toxicities may include (but not exclusive): 
• Moderate to severe headaches that are considered to be related to rozanolixizumab 
(Section 10.20.1) 
• Moderate to severe GI disturbances that are considered to be related to rozanolixizumab 
• Moderate to severe toxicities (≥Grade 2 as defined by CTCAE, version 5) for which 
rozanolixizumab cannot be excluded as a cause 
• Recurrent hypogammaglobulinemia with a serum total IgG <2g/L 
The date and reason for dose change of rozanolixizumab is to be recorded on each study 
participant’s eCRF. 
If any safety concerns are identified in MG0003 (ie, as recommended by the IDMC), or if one of 
the dose arms is determined to be futile and is discontinued in MG0003, corresponding changes 
may be made in the MG0004 dosing scheme. A study participant on a dose that was discontinued 
will switch doses immediately. 
6.7 
Home visits 
During the 52-week Treatment Period, the at-home visits will be conducted by fully trained 
healthcare professional visiting the study participant at his or her home. Alternatively, these 
visits can be conducted at the site as deemed necessary by site personnel and/or the study 
participant. For home/self-dosing, the same safety monitoring schedule will be followed as per 
onsite dosing. The home nurse will be present during the full duration of the visit. Home visits 
can be conducted if the following conditions are met: 
• The study participant is willing to be dosed and monitored at home by a home nurse. 
• The study participant has shown good acute tolerability to previous administrations of IMP 
(namely, he or she must have had no moderate or severe infusion reactions or other AEs that 
the Investigator considers could increase the risk of home administration). 
The Investigator should complete a checklist to confirm that criteria for home self-administration 
have been fully evaluated. 
6.8 
Treatment after the end of the study 
Study participants who complete participation in MG0004 may have the possibility to continue 
receiving rozanolixizumab through a managed access program as per applicable local 
regulations. 
In the case of prolonged hypogammaglobulinemia after treatment discontinuation, study 
participants can be considered for treatment with prophylactic antimicrobial therapy per local 
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guidelines. Study participants must be followed up until IgG levels return to values within the 
normal range or to individual Baseline values. 
7 
DISCONTINUATION OF STUDY MEDICATION AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL 
7.1 
Discontinuation of study medication 
7.1.1 
Liver chemistry stopping criteria 
Discontinuation of study treatment for abnormal liver function should be considered by the 
Investigator when a participant meets one of the conditions outlined Figure 7-1 or if the 
Investigator believes that it is in best interest of the participant. 
The study participant should follow the visit schedule as described in the protocol and complete 
the eCRF accordingly. 
Figure 7-1: Liver chemistry stopping criteria and increased monitoring algorithm 
 
ALT=alanine aminotransferase; AST=aspartate aminotransferase; INR=international normalized ratio; SAE=serious 
adverse event; ULN=upper limit of normal 
* The INR value is not applicable to study participants on anticoagulants. 
Treatment with study medication may be continued with increased monitoring if a study 
participant meets one of the criteria outlined in Figure 7-2. 
 
 
 
 
 
 
Continue Study Treatment 
Discontinue Study Treatment 
Plus 
Bilirubin≥2x
ULN (>35% 
direct) or plus
INR>1.5, if 
measured*
Possible  
Hy’s Law
ALT≥3xULN
ALT
≥8xULN
Plus
Symptoms of 
liver injury
or 
hypersensitivity
No
Yes
Yes
Yes
No
No
No
See algorithm 
for continued 
therapy with 
increased liver 
chemistry 
monitoring
Yes
ALT 
≥3xULN 
but 
<8xULN
Yes
Refer to the Liver Safety Required Actions and Follow up Assessments section in 
Appendix 6. 
Report as an SAE if possible Hy’s Law case: AST/ALT ≥3xULN and Bilirubin 
≥2xULN (>35% direct) or INR>1.5, if measured 
(Note: INR value not applicable to patients on anticoagulants) 
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Figure 7-2: Liver chemistry increased monitoring algorithm with continued study 
intervention for participants with ALT at least 3xULN but less than 
8xULN 
 
ALT=alanine aminotransferase; AST=aspartate aminotransferase; bili=bilirubin; INR=international normalized 
ratio; SAE=serious adverse event; ULN=upper limit of normal 
Specific assessments and follow-up actions for potential drug induced liver injury (PDILI) are 
provided in Appendix 6 (Section 10.6). 
7.1.2 
QTc stopping criteria 
If a clinically significant finding is identified after enrollment (including, but not limited to 
changes from Baseline in QT interval corrected using QT interval corrected for heart rate using 
Fridericia’s formula [QTcF]), the Investigator or qualified designee will determine if the 
participant can continue in the study and if any change in participant management is needed. 
This review of the ECG printed at the time of collection must be documented. Any new 
clinically relevant finding should be reported as an AE. 
A study participant who meets either bulleted criterion based on the average of triplicate ECG 
readings will be withdrawn from study medication and move into the Observation Period. The 
study participant should be referred to a specialist (ie, cardiologist) and managed as per local 
guidance. 
• QTc >500 msec OR uncorrected QT >600msec 
• Change from baseline of QTc >60msec 
 
 
 
 
 
 
 
 
Continue Study Treatment   and   Monitor Liver Chemistry
Discontinue Study Treatment 
ALT≥5xULN 
but <8xULN
+ bili <2xULN +
no symptoms
No
➢Must refer to Liver Safety Required Actions and Follow up Assessments section in the Appendix
Yes
ALT ≥3xULN 
but <5xULN
+ bili <2xULN  +
no symptoms
Able to 
monitor  
weekly 
for  ≥2 
weeks
Persists for 
≥2 weeks  
or other 
stopping 
criteria  
met
No
Yes
Yes
Yes
No
No
Able to 
monitor  
weekly 
for  ≥4 
weeks
Persists for 
≥4 weeks  
or other 
stopping 
criteria  
met
No
Yes
Yes
Yes
Yes
ALT ≥5xULN 
ALT <5xULN 
Yes
Yes
Refer to the Liver Safety Required Actions and Follow up Assessments section in 
Appendix 6. 
Report as an SAE if possible Hy’s Law case: AST/ALT ≥3xULN and Bilirubin 
≥2xULN (>35% direct) or INR>1.5, if measured 
(Note: INR value not applicable to patients on anticoagulants) 
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For participants with underlying bundle branch block, follow the discontinuation criteria listed 
below: 
Baseline QTc with Bundle Branch Block 
Discontinuation QTc Threshold with Bundle 
Branch Block 
<450msec 
>500msec 
450 to 480msec 
≥530msec 
See the Schedule of Activities (Section 1.3) for data to be collected at the time of treatment 
discontinuation and follow-up and for any further evaluations that need to be completed. 
The study participant should follow the visit schedule as described in the protocol and complete 
the eCRF accordingly. 
7.1.3 
Discontinuation of study medication due to other AEs or medical 
conditions 
A study participant must permanently discontinue study medication if any of the following 
events occur: 
1. Study participant develops an illness that would interfere with his or her continued 
participation. 
2. Study participant has new onset or recurrent neoplastic disease (except for superficial basal 
or squamous cell carcinoma of the skin not requiring targeted biological therapy, 
chemotherapy or radiation) 
3. Study participant has a serious infective episode requiring hospitalization or IV antibiotic 
therapy (including but not limited to bacteremia or sepsis, meningitis, osteomyelitis or septic 
arthritis, pneumonia, or visceral abscess). 
4. Study participant meets potential drug-induced liver injury (PDILI) permanent 
discontinuation criteria. 
5. Study participant has a TB test that is confirmed positive or any further evidence suggestive 
of potential TB infection (ie, exposure) and further examinations result in a diagnosis of 
active TB or latent TB infection (LTBI). 
6. If a nontuberculosis mycobacterium infection (NTMB) is identified during a study, the same 
withdrawal procedures as those used for an active TB infection identified during the study 
should be followed. 
7. Study participant experiences a serious AE of headache which is considered related to the 
study medication in the opinion of the Investigator. 
8. Study participant has an AE of severe or serious infusion or anaphylactic reaction requiring 
corticosteroid and/or epinephrine therapy (see Section 10.22) (Sampson et al, 2006). 
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Study participants may permanently discontinue study medication at the discretion of the 
Investigator, Medical Monitor, and Study Physician if any of the following events occur: 
9. Study participant is noncompliant with the study procedures or medications in the opinion of 
the Investigator. 
10. Study participant takes prohibited concomitant medications as defined in this protocol. 
Investigators should contact the Medical Monitor, whenever possible, to discuss the withdrawal 
of a participant in advance. Investigators should attempt to obtain information on study 
participants in the event of withdrawal (eg, reason for withdrawal, any safety information). 
7.1.4 
Temporary IMP discontinuation 
Study participant must be TEMPORARILY discontinued from the IMP if any of the following 
events occur: 
1. The study participant develops an event of hypogammaglobulinemia with a serum total 
IgG of <1g/L irrespective of infection. When the IgG level reaches ≥2g/L, the study 
participant may be allowed to continue treatment with IMP (see Appendix 22; 
Section 10.22.3). 
2. In the event of confirmed COVID-19 infection. IMP may be restarted if clinically 
appropriate when signs and symptoms have resolved. 
Study participants may be TEMPORARILY discontinued from the IMP if the following events 
occur: 
1. The study participant develops a non-serious persisting or recurrent infection with serum 
total IgG level between ≥1 and <2g/L. Upon resolution of infection and the IgG returning to 
level of ≥2g/L, the study participant may be allowed to resume treatment with the IMP (see 
Appendix 22; Section 10.22.3). 
2. In the event of suspected (eg, signs/symptoms such as fever, cough, shortness of breath) 
COVID-19 infection, or known exposure sufficient to necessitate testing or self-imposed 
quarantine, IMP may be restarted when clinically appropriate if: 
a. COVID-19 test is negative, and signs and symptoms have resolved. 
b. If test is not available, resolution of signs and symptoms and 14 days have passed since 
initial presentation of the clinical signs/symptoms. 
c. If asymptomatic, 14 days have passed since known exposure.  
The investigator should discuss with sponsor’s study physician prior to re-initiating the IMP. As 
appropriate, virtual assessments could continue (eg, AE collection, PRO assessments). 
If IMP treatment is resumed, continue the next dose as previously scheduled. No “make up” dose 
is permitted. The participant should subsequently follow the visit schedule as described in the 
protocol and the eCRF should be completed accordingly. 
The study participant should follow the visit schedule as described in the protocol and the eCRF 
completed accordingly. 
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7.2 
Participant discontinuation/withdrawal from the study 
Study participants are free to withdraw from the study at any time, without prejudice to their 
continued care. 
A study participant may also be withdrawn at any time at the discretion of the Investigator for 
safety, behavioral, compliance, or administrative reasons. 
If the participant withdraws consent for disclosure of future information, the Sponsor may retain 
and continue to use any data collected before such a withdrawal of consent. 
If a participant withdraws from the study, he/she may request destruction of any samples taken 
and not tested, and the Investigator must document this in the site study records. 
See the Schedule of Activities (Section 1.3) for data to be collected at the time of study 
discontinuation and follow-up and for any further evaluations that need to be completed. 
Study participants must be withdrawn from the study if any of the following events occur: 
1. Study participant withdraws his or her consent. 
2. The Sponsor or a regulatory agency requests withdrawal of the study participant. 
3. Study participant becomes pregnant during the study, as confirmed by a positive pregnancy 
test. 
4. Study participant has active suicidal ideation as indicated by a positive response (Yes) to 
either Question 4 or Question 5 of the “Since Last Visit” version of the C-SSRS. The study 
participant should be referred immediately to a mental healthcare professional. 
Study participants who withdraw from the study or discontinue study medication during the 
Treatment Period should complete the assessments outlined for premature end of treatment 
(PEOT) visit and enter the Observation Period. 
Study participants who withdraw for any reason will not be eligible to participate in the managed 
access program. 
7.3 
Lost to follow-up 
A participant will be considered lost to follow-up if he or she repeatedly fails to return for 
scheduled visits and is unable to be contacted by the study site. 
The following actions must be taken if a participant fails to return to the clinic for a required 
study visit: 
• The site must attempt to contact the participant and reschedule the missed visit as soon as 
possible and counsel the participant on the importance of maintaining the assigned visit 
schedule and ascertain whether or not the participant wishes to and/or should continue in the 
study. 
• Before a participant is deemed lost to follow-up, the Investigator or designee must make 
every effort to regain contact with the participant (at least 1 phone call and 1 written message 
to the participant) and document his/her effort (date and summary of the phone call and copy 
of the written message in the source documents), to complete the final evaluation. All results 
of these evaluations and observations, together with a narrative description of the reason(s) 
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for removing the participant, must be recorded in the source documents. The eCRF must 
document the primary reason for withdrawal. 
Should the participant continue to be unreachable, he/she will be considered to have withdrawn 
from the study with a primary reason of lost to follow-up documented in the eCRF. 
Investigators should contact the Medical Monitor, whenever possible, to discuss the withdrawal 
of a study participant in advance. Study participants who are withdrawn will not be replaced. 
8 
STUDY ASSESSMENTS AND PROCEDURES 
Study procedures and their timing are summarized in the Schedule of Activities (Section 1.3). 
Protocol waivers or exemptions are not allowed. 
Immediate safety concerns should be discussed with the Sponsor or Medical Monitor 
immediately upon occurrence or awareness to determine if the participant should continue or 
discontinue study treatment. 
Adherence to the study design requirements, including those specified in the Schedule of 
Activities (Section 1.3), is essential and required for study conduct. 
All Screening evaluations must be completed and reviewed to confirm that potential participants 
meet all eligibility criteria. 
Procedures conducted as part of the participant’s routine clinical management (eg, blood count) 
and obtained before signing of the ICF may be utilized for Screening or Baseline purposes 
provided the procedures met the protocol-specified criteria and were performed within the time 
frame defined in the Schedule of Activities (Section 1.3). 
An Unscheduled Visit can be conducted at the discretion of the Investigator (eg, due to an AE). 
During the Unscheduled Visit, the following assessments will be performed: 
• AE reporting 
• Concomitant medications  
• Review of withdrawal criteria 
• Physical examination 
• Vital signs 
Blood samples for PK, IgG, hematology, biochemistry, other laboratory testing and assessments 
may be performed as clinically indicated the discretion of the Investigator. 
Repeat or unscheduled samples may be taken for safety reasons or for technical issues with the 
samples. 
Some study-specific investigations may not be conducted according to the study protocol during 
a pandemic or other exceptional circumstances (eg, hurricanes) due to the need to implement 
safety measures and guidance from regulatory authorities. In such a situation, which may be 
accompanied by local or global containment or other measures, sites may need to prohibit access 
to study participants and study-related personnel. Study participants visits to the study site may 
be replaced by contingency measures. These measures are primarily established to ensure the 
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safety of study participants during the course of the study and to maintain the study participants 
treatment schedule, if the Investigator considers it appropriate. These measures include but are 
not limited to virtual visits or home-nursing visits replacing site visits, eg, telemedicine contacts 
or home-nursing visits when treatment and/or blood sampling is scheduled. The contingency 
measures are described in a contingency plan and will be implemented as required. 
8.1 
Efficacy assessments 
Planned timepoints for all efficacy assessments are provided in the Schedule of Activities 
(Section 1.3). 
8.1.1 
Quantitative Myasthenia Gravis scale 
For assessment of the QMG scale, Investigators or qualified designee will follow the Myasthenia 
Gravis Foundation of America’s QMG Manual instructions (Section 10.12, Appendix 12). 
Clinical personnel must complete mandatory training and be certified to assess study 
participants’ QMG scores (details are provided in the Study Procedures Manual). Study 
participants should not take pyridostigmine (or any acetylcholinesterase [AChE] inhibitor 
medication) at any point after midnight before testing when medically safe to do so to 
standardize testing. If it is not medically appropriate to halt AChE inhibitor medications, then the 
treatment can be continued but the testing should be performed as best as possible at the same 
time relative to the most recent dose of AChE inhibitor during the study. 
The scale tests 13 items, including ocular and facial involvement, swallowing, speech, limb 
strength, and forced vital capacity (FVC). For the assessment of FVC, the same spirometer 
should be used each time a study participant is tested, and if possible, the same person should 
carry out the assessment. Parameters and normal values for FVC will be decided between the 
study sites, such that all sites are using the same information. The QMG is a validated 
assessment (Barnett et al, 2012), with a higher score indicating more severe disease. Scoring for 
each item ranges from no weakness (0) to severe weakness (3), with an overall score range from 
0 to 39. A 3-point change in the total score is considered clinically relevant.  
8.1.2 
Myasthenia Gravis-Composite scale 
For assessment of the MG-C scale, the Investigator or qualified designee will examine the study 
participant to score all items, except for talking, chewing, and swallowing, for which the study 
participant will self-assess. Study participants should not take pyridostigmine (or any other 
acetylcholinesterase [AChE] inhibitor medication) from midnight before testing when medically 
safe to do so to standardize testing. The MG-C scale is a validated assessment (Burns et al, 
2010), with a higher score indicating more severe disease (Section 10.13, Appendix 13), and a 3-
point change is considered clinically relevant (Muppidi et al, 2011). The scale tests 10 items, 
with individual items being weighted differently. The overall score ranges from 0 to 50. Clinical 
personnel must complete mandatory training and be certified to assess study participants’ MG-C 
score (details are provided in the Study Procedures Manual). Where possible, the same person 
should carry out the assessment at each visit. 
8.1.3 
Patient-reported outcomes 
Patient-reported outcomes (PROs) must be completed as per timepoints mentioned in the 
Schedule of Activities (Section 1.3). The PROs should be completed prior to any intrusive 
procedures in a quiet place. 
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The PROs should be completed in the following order: MG-ADL, MG Symptoms PRO, 
EQ-5D-5L, MGII, and MG-QOL15r. The PROs should only be checked for completeness. On 
dosing days, the PROs will be completed prior to dosing. Study participants should not take 
pyridostigmine (or other AChE inhibitor medication) from midnight before the days when 
efficacy assessments are performed, when medically safe to do so to standardize testing. 
8.1.3.1 
MG-Activities of Daily Living scale 
The MG-ADL is an 8-item PRO instrument developed on the basis of the QMG (Wolfe et al, 
1999) (Section 10.14, Appendix 14). The MG-ADL targets symptoms and disability across 
ocular, bulbar, respiratory, and axial symptoms. In a recent study, reliability, validity, and 
responsiveness of the MG-ADL were further assessed. The questionnaire showed strong 
construct validity when evaluated against the MG-C as well as against the MG-QoL15r; high test 
retest reliability in a 1-week interval. It was also demonstrated that a 2-point improvement 
indicates clinical improvement (Muppidi, 2012; Muppidi et al, 2011). The total MG-ADL score 
ranges from 0 to 24, with a higher score indicating more disability. 
8.1.3.2 
MG Symptoms PRO 
The MG Symptoms PRO instrument (Appendix 15, Section 10.15) consists of 42 items across 5 
scales: ocular symptoms (items 1 to 5); bulbar symptoms (items 6 to 15); respiratory symptoms 
(items 16 to 18); physical fatigability (items 19 to 33) and muscle weakness fatigability (items 34 
to 42). 
The study participant will be asked to choose the response option that best describes the severity 
of ocular, bulbar-, and respiratory symptoms over the past 7 days using a 4-point Likert scale 
(“none” to “severe”) and how frequently he or she experiences physical fatigue and muscle 
weakness fatigability over the past 7 days using a 5-point Likert scale (“none of the time” to “all 
of the time”), respectively. A score can be obtained for each scale. All scores range from 0 to 
100, with higher scores indicating more severe symptoms. 
8.1.3.3 
EQ-5D-5L 
The 5-level EQ-5D (EQ-5D-5L) is designed to improve the instrument’s sensitivity and to 
reduce ceiling effects (Appendix 16, Section 10.16). 
The EQ-5D-5L essentially consists of 2 pages: the EQ-5D descriptive system and the EQ visual 
analogue scale (EQ VAS). 
The descriptive system comprises 5 dimensions: mobility, self-care, usual activities, 
pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight 
problems, moderate problems, severe problems and extreme problems. The study participant is 
asked to indicate his/her health state by ticking the box next to the most appropriate statement in 
each of the 5 dimensions. This decision results in a 1-digit number that expresses the level 
selected for that dimension. The digits for the 5 dimensions can be combined into a 5-digit 
number that describes the study participant’s health state. 
The EQ VAS records the study participant’s self-rated health on a vertical visual analogue scale, 
where the endpoints are labelled ‘The best health you can imagine’ and ‘The worst health you 
can imagine’. The VAS can be used as a quantitative measure of health outcome that reflects the 
study participant’s own judgement. 
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8.1.3.4 
Myasthenia Gravis Impairment Index 
The MGII is a measure of disease severity based on the signs and symptoms of study participants 
with MG (Appendix 17, Section 10.17). It was developed using a study participant-centered 
approach and following current guidelines for outcome measure development, incorporating 
study participant input throughout the different development phases (Barnett et al, 2014; Barnett 
et al, 2016). The MGII has 22 study participant-reported and 6 examination items, and scores are 
presented as a sum of all items for a total score but also as ocular and generalized sub-scores. 
The Investigator or qualified designee will examine the study participant prior to scoring all 
items. Where possible, the same person should carry out the assessment at each visit. 
The MGII has shown construct validity and reliability in an outpatient setting. It has less floor 
effect compared to other commonly used outcome measures, and it can effectively discriminate 
among study participants with different degrees of severity (Barnett et al, 2016, Barnett et al, 
2017). The MGII is sensitive to detect clinical change after interventions. Additionally, the MGII 
showed more relative efficiency than the QMG scale, MG-C, and MG-ADL to detect change in 
short-term interventions for MG. Estimates for the minimal important difference were developed 
(5.5 for individuals). 
8.1.3.5 
Revised 15-item myasthenia gravis quality of life questionnaire 
The MG-QOL15r is a brief survey, completed by the study participant, which is designed to 
assess some aspects of "quality of life" related to MG. The MG-QOL15r was designed to assess 
the “patient perspective” in the everyday clinic setting or in a clinical study (Appendix 18, 
Section 10.18). 
When completing the 15-item MG-QOL15r, MG study participants should consider only how 
their MG affects these items. For example, if a study participant has no leg weakness but has a 
painful hip (unrelated to the MG) that causes walking trouble, the study participant should report 
“not at all” to the item of, “I have trouble walking.” This is because any hip-related walking 
trouble is unrelated to the MG. One other note of clarification: if the study participant is retired 
(unrelated to MG), he or she should report “not at all” to the item about whether the MG 
negatively impacts job/occupational status. 
8.2 
Safety assessments 
Planned time points for all safety assessments are provided in the Schedule of Activities 
(Section 1.3). 
Study participants must be observed at site postdose for at least 4 hours following the first 2 
infusions, and then 2 hours thereafter for subsequent infusions until Week 7, where the 
participant can be observed for 1 hour at the Investigator’s discretion. 
8.2.1 
Physical examinations 
Complete physical examination 
A complete physical examination will include, at a minimum, general appearance; ear, nose, and 
throat; eyes, hair, and skin; and assessments of the cardiovascular, respiratory, GI, neurological, 
musculoskeletal, and hepatic systems. Height and weight will also be measured and recorded. 
Body weight will be measured with the study participant wearing light clothing and without 
wearing shoes. 
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Brief physical examination 
A brief physical examination will include, at a minimum, assessments of the skin, lungs, 
cardiovascular system, and abdomen (liver and spleen). 
For complete and brief physical examinations, Investigators should pay special attention to 
clinical signs related to previous serious illnesses, as well as signs and symptoms of infections. 
Clinically relevant findings or worsening of previous findings will be recorded as AEs. 
8.2.2 
Vital signs 
Oral, tympanic, or axillary temperature, pulse rate, and blood pressure (BP) will be assessed. 
Blood pressure (systolic and diastolic), and pulse rate measurements should be preceded by at 
least 5 minutes of rest for the study participant in a quiet setting without distractions (eg, 
television, cell phones). All measurements will be assessed in a sitting position with a completely 
automated device. Manual techniques will be used only if an automated device is not available. 
All vital signs should be taken before any blood sampling. 
8.2.3 
Electrocardiograms 
Triplicate 12-lead ECGs will be obtained as outlined in the Schedule of Activities (see 
Section 1.3) using an ECG machine that automatically calculates the heart rate and measures 
pulse rate (PR), QRS, QT, and QTc intervals. Refer to Section 7.1.2 for QTc withdrawal criteria 
and any additional readings that may be necessary. 
All ECG recordings should be taken with the study participant resting in the supine position for 
at least 5 minutes before the recording. 
At each time point at which triplicate ECGs are required, 3 individual ECG tracings should be 
obtained as closely as possible in succession. 
8.2.4 
Clinical safety laboratory assessments 
See Appendix 2 (Section 10.2) for the list of clinical laboratory tests to be performed and the 
Schedule of Activities (Section 1.3) for the timing and frequency. 
The Investigator must review the laboratory report, document this review, and record any 
clinically relevant changes occurring during the study in the AE section of the eCRF. The 
laboratory reports must be filed with the source documents. 
All laboratory tests with values considered clinically significantly abnormal during participation 
in the study should be repeated until the values return to normal or Baseline or until they are no 
longer considered clinically significant by the Investigator or Medical Monitor. 
For clinically significant abnormal values, the etiology should be identified, and the Sponsor 
notified. 
All protocol-required laboratory assessments, as defined in Appendix 2, must be conducted in 
accordance with the laboratory manual and the Schedule of Activities. 
8.2.5 
Suicidal risk monitoring 
Participants being treated with rozanolixizumab should be monitored appropriately and observed 
closely for suicidal ideation and behavior or any other unusual changes in behavior. 
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Consideration should be given to discontinuing rozanolixizumab in participants who experience 
signs of suicidal ideation or behavior. 
Families and caregivers of participants being treated with rozanolixizumab should be instructed 
to monitor participants for the emergence of unusual changes in behavior, as well as the 
emergence of suicidal ideation and behavior, and to report such symptoms immediately to the 
study Investigator. 
Suicidality will be assessed by trained study personnel using the C-SSRS (Columbia University 
Medical Center, 2008 [Appendix 23, Section 10.23]). This scale will be used for screening as 
well as to assess suicidal ideation and behavior that may occur during the study. 
8.2.6 
Assessment and management of tuberculosis and tuberculosis risk 
factors 
Appropriate rigorous precautions are being taken within this protocol to monitor the risk of TB 
infection in this study (see Section 5.2). Any presumptive diagnosis or diagnosis of a TB 
infection is a reportable event. 
Physical examination 
The Investigator should consider all potential sites of infection when assessing for TB during the 
physical examination and other evaluations and based on the study participant’s medical or 
social history. Sites commonly infected by TB include: the lungs, larynx, lymph glands, pleura, 
GI system, genitourinary tract (including renal), bones and joints, meninges, peritoneum, 
pericardium, and skin. This is not an exhaustive list and unusual presentations and areas of 
involvement should always be considered. 
Some common symptoms that the study participant may present are dependent on the primary 
focus of infection and may include cough, blood in sputum, night sweats, lymphadenitis, joint 
pain/swelling, spinal deformity, headache/confusion, and abdominal pain (mimicking 
inflammatory bowel disease). Unusual presentations should always be considered. 
If available, respiratory or other specimens must also be smear- and culture-negative for TB 
(CDC diagnosis of LTB infection [http://www.cdc.gov/TB/topic/testing/default.htm]). 
TB signs and symptoms questionnaire 
In addition to a physical examination done intermittently throughout the study, study participants 
will be evaluated both for signs and symptoms of latent or active TB infection and for risk 
factors for exposure to TB using the TB questionnaire as indicated in the Schedule of Activities 
(Section 1.3). 
The TB questionnaire should be completed accurately and filed as a critical source document. 
The questionnaire will assist with the identification of study participants who may require 
therapy for TB. 
A “Yes” response to any of the questions in the TB questionnaire during the study may trigger 
further assessment to determine if the study participant has either LTBI and must receive 
prophylactic LTBI therapy or active TB infection and must be withdrawn from the study. As an 
example, a study participant who answers “Yes” at Screening to the question 
 
 
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 should not be allowed into the study 
pending further assessments (including TB specialist consult) as outlined previously. 
A detailed description of the TB questionnaire is provided in Appendix 19 (Section 10.19). 
TB assessment by interferon gamma release assay 
The TB screening test interferon gamma release assay (IGRA) performed at MG0003 entry will 
be used as the Baseline result. Additional IGRA tests may be performed if indicated (eg, 
presence of signs and symptoms suggestive of TB, recent exposure). 
The test results will be reported as positive, negative, or indeterminate. If an IGRA is positive or 
indeterminate, the study participant must be evaluated by a TB specialist. 
• Positive IGRA 
The positive IGRA may represent new LTBI or active TB infection. The positive IGRA 
result may also reflect positivity from a recently diagnosed and adequately treated (in 
progress or completed within the past 12 months) LTBI or from adequately treated past TB 
infection. In such cases, the study participants must be evaluated by a TB specialist. 
• Indeterminate IGRA 
If the IGRA test result is indeterminate, the IGRA previously performed may be repeated 
once. If the test is positive or indeterminate on retest, the study participant must not be 
randomized to study medication. 
TB assessment by chest x-ray 
A Screening chest x-ray is not required for this study. 
However, a chest x-ray or other imaging test can be performed if indicated (eg, presence of signs 
and symptoms suggestive of TB, close exposure to persons with TB). 
Test conversion 
Tuberculosis test conversion is defined as a positive or indeterminate (and confirmed 
indeterminate on repeat) IGRA result for the current test when previous IGRA test results were 
negative. The IGRA result must be negative for study participants to enroll in this study. During 
the study, all study participants with positive or indeterminate IGRA test results must 
immediately stop study drug administration. 
In the event of an IGRA test conversion, the study participant must be considered as having 
either a suspected new LTBI or an active TB infection and be promptly referred to an appropriate 
specialist (eg, pulmonologist, infectious disease specialist) for further evaluation. If the test 
conversion indicates LTBI, active TB, or NTMB, then TB test conversion (confirmed) should be 
classified adequately, either as due to LTBI, active TB infection, or NTMB, respectively. 
Additional assessments (eg, blood tests or IGRA, chest x-rays, or other imaging) should be 
performed where medically relevant and documented. Such conversions should be reported as 
AEs as described in the protocol. The AE term should be updated with a final diagnosis once it is 
available. 
Latent TB 
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Latent TB infection is defined as the absence of signs, symptoms (eg, evidence of organ-specific 
involvement), or physical findings suggestive of TB infection with a positive IGRA test (or 
2 indeterminate IGRA test results) and a chest x-ray (or other imaging) without evidence of TB 
infection. 
Any LTBI must be reported as an AE and graded appropriately as described in the protocol. 
Follow-up reports should be completed as per protocol requirement until such time as the LTBI 
resolves. 
Active TB or non-TB mycobacterium infection 
Study participants who develop active TB or NTMB infection during the study (conversion 
demonstrated by IGRA) must be withdrawn from the study. The study participant must be 
immediately permanently discontinued from study medication and an PEOT Visit must be 
scheduled as soon as possible, but no later than the next scheduled visit. The study participant 
should be encouraged to keep the EOS Visit as specified by the protocol. Treatment for active 
TB or NTMB should be started immediately based on local guidelines. 
Confirmed active TB is always considered an SAE. UCB’s process requires that these events 
must be captured on an SAE report form and provided to the Sponsor in accordance with SAE 
reporting requirements. Follow-up reports should be completed as per protocol requirement until 
such time as the TB infection resolves. 
Latent tuberculosis infection, active TB, or other NTMB identified during study 
During the study, study participants who develop evidence of LTBI, active TB, or NTMB 
infection must immediately stop further administration of study medication and will be referred 
to an appropriate TB specialist (pulmonologist or infectious disease specialist) for further 
evaluation. Study participants diagnosed with active TB or LTBI should receive appropriate TB 
or prophylaxis therapy. The study participant should be transferred to the care of their physician 
and managed according to the standard of care. 
If infection with NTMB is identified during the study, the same procedure as for active TB 
acquired during the study and compliant TB treatment shall be followed. 
Follow-up information of suspected and confirmed TB cases should be provided to UCB at least 
3, 9, and 12 months after the start date of anti-TB treatment, including hematological and 
biochemical safety parameters, x-ray evolution data, and TB diagnostic procedures used to 
follow-up and confirm recovery from TB. 
8.3 
Adverse events 
The definitions of an AE or SAE can be found in Appendix 3 (Section 10.3). 
Adverse events can be reported by the participant (or, when appropriate, by a caregiver, 
surrogate, or the participant's legally authorized representative). 
The Investigator and any designees are responsible for detecting, documenting, and recording 
events that meet the definition of an AE or SAE and remain responsible for following up AEs 
that are serious, considered related to the study treatment or study procedures, or that caused the 
participant to discontinue rozanolixizumab treatment or from MG0004 (see Section 7). 
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8.3.1 
Time period and frequency for collecting AE and SAE information 
All SAEs will be collected from the signing of the ICF until EOS Visit at the time points 
specified in the Schedule of Activities (Section 1.3). 
All AEs will be collected from the signing of the ICF until EOS Visit at the time points specified 
in the Schedule of Activities (Section 1.3). 
All SAEs will be recorded and reported to the Sponsor or designee within 24 hours, as indicated 
in Appendix 3 (Section 10.3). The Investigator will submit any updated SAE data to the Sponsor 
within 24 hours of it being available. 
The Investigator is specifically requested to collect and report to UCB (or its representative) any 
SAEs (even if the Investigator is certain that they are in no way associated with the study 
medication), up until the End of Study Visit for each participant, and to also inform participants 
of the need to inform the Investigator of any SAE within this period. Serious AEs that the 
Investigator thinks may be associated with the study medication must be reported to UCB 
regardless of the time between the event and the end of the study. 
The method of recording, evaluating, and assessing causality of AE and SAE and the procedures 
for completing and transmitting SAE reports are provided in Appendix 3 (Section 10.3). 
8.3.2 
Method of detecting AEs and SAEs 
Care will be taken not to introduce bias when detecting AEs and/or SAEs. Open-ended and 
nonleading verbal questioning of the participant is the preferred method to inquire about 
AE occurrences. 
8.3.3 
Follow-up of AEs and SAEs 
After the initial AE/SAE report, the Investigator is required to proactively follow each 
participant at subsequent visits/contacts. All AEs and SAEs, (and nonserious AESMs, as defined 
in Section 8.3.6), will be followed until resolution, stabilization, the Investigator determines that 
it is no longer clinically significant, the event is otherwise explained, or the participant is lost to 
follow-up (as defined in Section 7.3). Further information on follow-up procedures is given in 
Appendix 3 (Section 10.3). 
8.3.4 
Regulatory reporting requirements for serious adverse events 
Prompt notification (24 hours) by the Investigator to the Sponsor of a SAE is essential so that 
legal obligations and ethical responsibilities towards the safety of participants and the safety of a 
study treatment under clinical investigation are met. 
The Sponsor has a legal responsibility to notify both the local regulatory authority and other 
regulatory agencies about the safety of a study treatment under clinical investigation. The 
Sponsor will comply with country-specific regulatory requirements relating to safety reporting to 
the regulatory authority, Institutional Review Boards (IRB)/Independent Ethics Committees 
(IEC), and Investigators. 
Investigator safety reports must be prepared for suspected unexpected serious adverse reactions 
according to local regulatory requirements and Sponsor policy and forwarded to Investigators as 
necessary. 
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An Investigator who receives an Investigator safety report describing an SAE or other specific 
safety information (eg, summary or listing of SAEs) from the Sponsor will review and then file it 
along with the IB and will notify the IRB/IEC, if appropriate according to local requirements. 
8.3.5 
Pregnancy 
Details of all pregnancies in female participants and, if indicated, in female partners of male 
participants will be collected after the start of study treatment and until at least 90 days after the 
last dose. 
If a pregnancy is reported, the Investigator must immediately inform the Sponsor within 24 hours 
of learning of the pregnancy and should follow the procedures outlined in Appendix 4 
(Section 10.4). 
The participant should be withdrawn from the study as soon as pregnancy is known (by positive 
pregnancy test), and the following should be completed: 
• The participant should return for an early discontinuation (PEOT) visit. 
Abnormal pregnancy outcomes (eg, spontaneous abortion, fetal death, stillbirth, congenital 
anomalies, ectopic pregnancy) are considered SAEs. 
8.3.6 
Adverse events of special interest 
An AE of special interest (AESI) is any AE that a regulatory authority has mandated be reported 
on an expedited basis, regardless of the seriousness, expectedness, or relatedness of the AE to the 
administration of a UCB product/compound. An AESI should be reported within 24 hours. 
Potential Hy’s Law, defined as ≥3xULN ALT and ≥2xULN bilirubin (>35% direct bilirubin) or 
ALT 3xULN and international normalized ratio (INR) >1.5, if INR measured, with no 
alternative explanation for the biochemical abnormality, must ALWAYS be reported to UCB as 
an AESI (ie, without waiting for any additional etiologic investigations to have been concluded). 
Follow-up information should then be reported if an alternative etiology is identified during 
investigation and monitoring of the participant. 
All AESIs will follow the AE recording and reporting procedures as indicated in Appendix 3 
(Section 10.3). 
8.3.7 
Adverse events of special monitoring 
For rozanolixizumab, AESMs that require immediate reporting (within 24 hours regardless of 
seriousness) to UCB are: 
• Severe headache 
• Severe GI disturbances (ie, abdominal pain, diarrhea, vomiting) 
• Opportunistic infection 
In case of severe headache or serious headache (regardless of severity), the headache 
questionnaire (Appendix 21 [Section 10.21]) must be completed. Additional procedures for 
management of headaches are provided in Appendix 20 (Section 10.20.1). 
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Procedures for the management of diarrhea, and infections and hypogammaglobulinemia are 
provided in Appendix 20 (Section 10.20.2 and Section 10.20.3, respectively). 
Although hypersensitivity reactions including infused-related reactions and anaphylaxis are not 
classified as AESM, these AEs will be monitored by the Investigators. If such an event is 
suspected, it should be managed according the guidance provided in Appendix 20 
(Section 10.20.4). In case of suspected anaphylaxis, the Sampson’s Criteria Questionnaire 
(Sampson et al, 2006) in Appendix 22 (Section 10.22) should be completed. 
All AESMs will follow the SAE recording and reporting procedures as indicated in Appendix 3 
(Section 10.3). 
8.3.8 
Treatment-emergent adverse events 
Treatment-emergent AEs are defined as AEs starting after the time of first IMP administration up 
to and including 8 weeks after the final dose. 
8.4 
Safety signal detection 
Selected data from this study will be reviewed periodically to detect as early as possible any 
safety concern(s) related to the study medication so that Investigators, clinical study participants, 
regulatory authorities, and IRBs/IECs will be informed appropriately and as early as possible. 
In addition, an IDMC will periodically review and monitor safety data from this study and advise 
UCB. Details are provided in the IDMC Charter. 
The Study Physician or medically qualified designee/equivalent will conduct an ongoing review 
of SAEs and perform ongoing SAE reconciliations in collaboration with the Patient Safety 
representative. 
As appropriate for the stage of development and accumulated experience with the study 
medication, medically qualified personnel at UCB may identify additional safety measures 
(eg, AEs, vital signs, laboratory, ECG results) for which data will be periodically reviewed 
during the course of the study. 
8.5 
Treatment of overdose 
Any dose increase of ≥10% or greater from the assigned dose for each administered dose of IMP 
 should be considered an overdose, irrespective of the weight tier band. Overdose events 
are only considered AEs or SAEs if there are associated clinical signs and symptoms or if the act 
of taking the excess study medication itself is an AE or SAE (eg, suicide attempt). 
UCB does not recommend specific treatment for an overdose. 
In the event of an overdose, the Investigator or treating physician should: 
1. Contact the Medical Monitor immediately. 
2. Closely monitor the participant for any AE/SAE and laboratory abnormalities for at least 5 
days. 
3. Obtain a plasma sample for PK analysis and IgG (total and subclasses) autoantibodies within 
3 days from the date of the last dose of study treatment if requested by the Medical Monitor 
(determined on a case-by-case basis). 
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4. Document the quantity of the excess dose as well as the duration of the overdose in the 
eCRF. 
Decisions regarding dose interruptions or modifications will be made by the Investigator in 
consultation with the Medical Monitor based on the clinical evaluation of the participant. 
8.6 
Pharmacokinetics and anti-drug antibodies 
Whole blood samples will be collected for measurement of plasma concentrations of 
rozanolixizumab and ADA as specified in the Schedule of Activities (Section 1.3). Blood 
samples may be collected at additional timepoints during the study if warranted and agreed upon 
between the Investigator and the Sponsor. Instructions for the collection and handling of 
biological samples will be provided by the Sponsor. The actual date and time (24-hour clock 
time) of each sample will be recorded. 
Samples will be used to evaluate the PK of rozanolixizumab and ADA and may be used for 
establishing assay parameters (eg, ADA cut point setting PK selectivity assessment). Samples 
collected for analyses of rozanolixizumab concentration may also be used to evaluate safety or 
efficacy aspects related to concerns arising during or after the study. 
Participant confidentiality will be maintained. At visits during which plasma/serum samples for 
the determination of multiple aspects of rozanolixizumab will be taken, one sample of sufficient 
volume can be used. 
Any changes in the timing or addition of time points for any planned study assessments must be 
documented and approved by the relevant study team member and then archived in the Sponsor 
and site study files, but will not constitute a protocol amendment. The IRB/IEC will be informed 
of any safety issues that require alteration of the safety monitoring scheme or amendment of the 
ICF. 
Blood samples for the measurement of plasma concentrations of rozanolixizumab will be 
collected at additional timepoints in a local substudy. 
8.7 
Pharmacodynamics 
Venous blood samples will be collected at timepoints specified in the Schedule of Activities 
(Section 1.3), for measurement of: 
• Serum IgG and IgG sub-classes concentrations 
• Serum MG-specific autoantibodies (anti-MuSK/anti-AChR) levels 
For all PD assessments, blood samples will be collected predose. Instructions pertaining to 
sample collection, processing, storage, labeling, and shipping are provided in the laboratory 
manual. 
8.8 
Genetics 
Not applicable. 
8.9 
Biomarkers 
Collection of samples for other biomarker research is also part of this study. Blood samples for 
biomarker research are required and will be collected from all participants in this study as 
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specified in the Schedule of Activities (Section 1.3). Exploratory samples are collected predose. 
Additional exploratory safety samples must be collected 4 hours postevent or as soon as possible 
before the next IMP in case of AESM of serious or severe headache or AESM of GI disorders. 
Protein and metabolites biomarkers may be measured to assess the effect of rozanolixizumab on 
exploratory biomarkers, and explore the relationship between protein, and metabolite biomarkers 
and cause, progression, and appropriate treatment of MG. 
If not used immediately, these samples will be stored at -80°C for up to 20 years for later 
exploratory analyses. Any exploratory biomarker will only ever be related to the exploration of 
cause, progression, and appropriate treatment of MG. They may also be used to develop 
tests/assays including diagnostic tests related to rozanolixizumab and/or FcRn inhibitor and MG. 
The nature and format of these tentative additional analyses will be determined at a later time. 
Details on the collection, storage, preparation, and shipping of samples will be presented in the 
laboratory manual provided separately. Instructions pertaining to sample collection, processing, 
storage, labeling, and shipping are provided in the laboratory manual for this study. Detailed 
information on sample analyses will be provided in a bioanalytical report. 
8.9.1 
Immunology 
Blood samples for immunological testing are required and will be collected from all study 
participants in this study as specified in the Schedule of Activities (Section 1.3) for measurement 
of: 
• IgA, IgE, IgM 
• Serum complement (C3, C4) 
• Serum cytokines 
• Plasma complement (C3a, C5a) 
• Tetanus toxoid IgG 
Samples for IgA, IgE, IgM are collected predose. 
Additional samples may be collected 2 hours and 4 hours postevent in case of severe headache, 
infusion reaction, or hypersensitivity reaction at Visits 5, 7, 1st visit (of the 4-week cycle from 
Weeks 9-48) and Visit 49. 
Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
8.10 
Medical resource utilization and health economics 
Medical resource utilization and health economics will not be measured for this study. 
9 
STATISTICAL CONSIDERATIONS 
A description of statistical methods follows and will be described in more detail in the SAP. 
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9.1 
Definition of analysis sets 
The following analysis sets will be created in the study: 
• Enrolled Set: All study participants who sign the informed consent. 
• Randomized Set: All enrolled study participants who are randomized. 
• Safety Set: All randomized study participants who received at least 1 dose of study 
medication in this study. Analysis of this set will be according to the treatment the study 
participants first received, and will be used for the efficacy, demographic, and safety 
analyses. 
9.2 
General statistical considerations 
Statistical evaluation will be performed by the Sponsor or designee and supervised by the 
Exploratory Statistics Department of UCB. Data will be summarized by dose levels of 
rozanolixizumab first received in this study. 
All analyses will be performed using SAS® version 9.3 or later (SAS Institute, Cary, NC, USA). 
Continuous variables will be summarized by visit (where applicable) with the statistics including 
the number of study participants, mean, standard deviation (SD), median, minimum, and 
maximum. Categorical variables will be summarized by visit (where applicable) using frequency 
counts and percentages. 
If not otherwise stated, Baseline values will be the last available predose value and will be 
clearly defined in the SAP. All relevant data will be listed by treatment group and study 
participant. 
9.3 
Planned safety and other analyses 
9.3.1 
Analysis of the primary safety endpoints 
The frequency and severity of all TEAEs will be presented for each treatment group separately 
by System Organ Class, high level term, and preferred term (Medical Dictionary for Regulatory 
Activities [MedDRA®]). The data will be displayed as number of participants experiencing the 
TEAE, percentage of participants, and number of TEAEs. A TEAE is defined as any event that 
was not present prior to the first administration of study medication or any unresolved event 
already present before the first administration of study medication that worsens in intensity 
following exposure to treatment, up to and including 8 weeks after the end of the Treatment 
Period or 8 weeks after the last of study medication in study participants who discontinue the 
study or study medication. 
All safety analyses will be based on the SS (Section 9.1). 
9.3.2 
Analysis of the other safety endpoints 
Laboratory evaluations and vital signs as well as ECG data will be analyzed over time. All safety 
analyses will be listed and summarized for the SS. 
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9.3.3 
Other analyses 
9.3.3.1 
Pharmacokinetic analyses 
Pharmacokinetic variables such as AUC or Cmax cannot be derived, since blood sampling will be 
performed at 1 time point per visit only. Thus, PK analysis is restricted to rozanolixizumab 
concentration data. In addition to the general descriptive display, of concentration data will be 
summarized by treatment group and time point using the number of available observations, 
mean, median, SD, minimum, maximum, geometric mean (and associated 95% CI), and 
geometric coefficient of variation (assuming log-normally distributed data). Values below the 
lower limit of quantification (LLOQ) will be reported with a clear sign indicating that they were 
below the LLOQ. Descriptive statistics of concentrations will be calculated if at least two-thirds 
of the individual data points are quantifiable (≥LLOQ). Individual concentrations of 
rozanolixizumab will also be displayed graphically. 
All PK analyses will be based on the SS (Section 9.1). 
9.3.3.2 
Pharmacodynamic analyses and biomarkers 
The levels of anti-MuSK, anti-AChR, and biomarkers (potentially including but not limited to 
total IgG, IgG subclasses, IgA, IgE, IgM, serum complement, and plasma complement) will be 
presented as continuous variables (Section 9.2). 
All pharmacodynamic and biomarker analyses will be based on the SS (Section 9.1). 
9.3.3.3 
Anti-drug antibody analyses 
A tiered ADA approach will be used for the study. Anti-drug antibody assessments will involve 
screening (above or below the cutpoint) of all samples for ADA, followed by a confirmatory 
assessment (confirmed or not confirmed positive) leading to an anti-rozanolixizumab antibody 
positive or negative assessment for each sample and a subsequent titration of those 
anti-rozanolixizumab antibody-positive samples during treatment and observation periods. For 
anti-rozanolixizumab antibody-positive samples (or subset of) further characterization for 
neutralizing ADA potential in vitro will be performed. 
All immunogenicity analyses will be based on the SS (Section 9.1). 
9.4 
Planned efficacy analyses 
Data will be listed, and descriptive statistics will be generated for the observed values and 
changes from Baseline. Summary outputs will be presented by treatment group and visit. For 
categorical variables, frequency counts and percentages will be produced. 
All efficacy analyses will be based on the SS (Section 9.1). 
9.5 
Handling of protocol deviations 
Important protocol deviations will be identified as part of the data cleaning process in the Data 
Cleaning Plan. Ongoing data cleaning meetings will be held throughout the duration of the study. 
Objectives of these meetings include to review and update (if necessary) the important protocol 
deviations in the Data Cleaning Plan and discuss exclusion of study participants from analysis 
populations. Furthermore, overall trends in protocol deviations will be discussed at the Data 
Evaluation Meeting. Through this ongoing data cleaning and evaluation process, all decisions 
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regarding important protocol deviations and exclusions from analysis populations are made on an 
ongoing basis. 
9.6 
Handling of dropouts or missing data 
All imputation of missing or partial dates for safety assessments will be detailed in the SAP. 
Efficacy data will be summarized only and no imputation will be performed.. 
9.7 
Planned interim analysis and data monitoring 
9.7.1 
Interim analysis 
There are no plans for hypothesis testing in this study and therefore no formal interim analysis 
will be conducted.  Interim cuts of open label data may be conducted to provide information on 
the ongoing study. 
9.7.2 
Data monitoring 
The IDMC will oversee the safety of the study by reviewing safety data at periodic data reviews. 
The IDMC will consist of members independent from UCB. Study enrollment will not be halted 
during planned IDMC review of the safety and efficacy data. The objectives and procedures for 
the IDMC will be detailed in the IDMC Charter. 
9.8 
Determination of sample size 
No formal sample size calculation can be performed. Up to approximately 276 study participants 
will be enrolled into the lead-in studies, MG0003 and MGC003. All eligible study participants 
from MG0003 and MGC003 will be invited to participate in MG0004. 
10 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS 
10.1 
Appendix 1: Regulatory, ethical, and study oversight 
considerations 
10.1.1 
Regulatory and ethical considerations 
The study will be conducted under the auspices of an IRB/IEC, as defined in local regulations, 
ICH-Good Clinical Practice (GCP), and in accordance with the ethical principles that have their 
origin in the Declaration of Helsinki. 
The Investigator/UCB will ensure that an appropriately constituted IRB/IEC that complies with 
the requirements of the current ICH-GCP version or applicable country-specific regulations will 
be responsible for the initial and continuing review and approval of the clinical study. Prior to 
initiation of the study, the Investigator/UCB will forward copies of the protocol, ICF, IB, and 
Investigator’s curriculum vitae (if applicable), advertisement (if applicable), and all other 
participant-related documents to be used for the study to the IRB/IEC for its review and 
approval. 
Before initiating a study, the Investigator will have written and dated full approval from the 
responsible IRB/IEC for the protocol. 
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The Investigator will also promptly report to the IRB/IEC all changes in the study, all 
unanticipated problems involving risks to participants or others, and any protocol deviations, to 
eliminate immediate hazards to participants. 
The Investigator will not make any changes in the study or study conduct without IRB/IEC 
approval, except where necessary to eliminate apparent immediate hazards to the participants. 
For minor changes to a previously approved protocol during the period covered by the original 
approval, it may be possible for the Investigator to obtain an expedited review by the IRB/IEC as 
allowed. 
As part of the IRB/IEC requirements for continuing review of approved studies, the Investigator 
will be responsible for submitting periodic progress reports to the IRB/IEC (based on IRB/IEC 
requirements), at intervals appropriate to the degree of participant risk involved, but no less than 
once per year. The Investigator should provide a final report to the IRB/IEC following study 
completion. 
UCB (or its representative) will communicate safety information to the appropriate regulatory 
authorities and all active Investigators in accordance with applicable regulatory requirements. 
The appropriate IRB/IEC will also be informed by the Investigator or the Sponsor, as specified 
by the applicable regulatory requirements in each concerned country. Where applicable, 
Investigators are to provide the Sponsor (or its representative) with evidence of such IRB/IEC 
notification. 
10.1.2 
Financial disclosure 
Insurance coverage will be handled according to local requirements. 
Finance and insurance are addressed in the Investigator and/or contract research organization 
agreements, as applicable. 
10.1.3 
Informed consent process 
Participant’s informed consent must be obtained and documented in accordance with local 
regulations, ICH-GCP requirements, and the ethical principles that have their origin in the 
principles of the Declaration of Helsinki. 
Prior to obtaining informed consent, information should be given in a language and at a level of 
complexity understandable to the participant in both oral and written form by the Investigator (or 
designee). Each participant will have the opportunity to discuss the study and its alternatives 
with the Investigator. 
Prior to participation in the study, the ICF should be signed and personally dated by the 
participant, or his/her legal representative, and by the person who conducted the informed 
consent discussion (Investigator or designee). The participant or his/her legal representative must 
receive a copy of the signed and dated ICF. As part of the consent process, each participant must 
consent to direct access to his/her medical records for study-related monitoring, auditing, 
IRB/IEC review, and regulatory inspection. 
If the ICF is amended during the study, the Investigator (or the Sponsor, if applicable) must 
follow all applicable regulatory requirements pertaining to the approval of the amended ICF by 
the IRB/IEC and use of the amended form. 
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All studies conducted at centers in the United States must include the use of a Health Insurance 
Portability and Accountability Act Authorization form. 
The participant may withdraw his/her consent to participate in the study at any time. A 
participant is considered to be enrolled in the study when he/she has signed the ICF. An eCRF 
must not be started, nor may any study specific procedure be performed for a given participant, 
without having obtained his/her written consent to participate in the study. 
10.1.4 
Data protection 
UCB staff (or designee) will affirm and uphold the participant’s confidentiality. Throughout this 
study, all data forwarded to UCB (or designee) will be identified only by the participant number 
assigned at Screening. 
The Investigator agrees that representatives of UCB, its designee, representatives of the relevant 
IRB/IEC, or representatives of regulatory authorities will be allowed to review that portion of the 
participant’s primary medical records that directly concerns this study (including, but not limited 
to, laboratory test result reports, ECG reports, admission/discharge summaries for hospital 
admissions occurring during a participant’s study participation, and autopsy reports for deaths 
occurring during the study). 
The participant must be informed that his/her personal study-related data will be used by the 
Sponsor in accordance with local data protection law. The level of disclosure must also be 
explained to the participant. 
The participant must be informed that his/her medical records may be examined by Clinical 
Quality Assurance auditors or other authorized personnel appointed by the Sponsor, by 
appropriate IRB/IEC members, and by inspectors from regulatory authorities. 
10.1.5 
Committees structure 
An IDMC will review the safety and tolerability data in this study in order to make 
recommendations for the Sponsor. 
An IDMC will be set up in line with the FDA regulatory requirements and EMA Guideline on 
IDMCs (EMEA/CHMP/EWP/5872/03 Corr, adopted 27/05/2005). The IDMC will consist of 
external experts who are independent from UCB and the clinical operations contract research 
organization, and have no conflict of interest related to the conduct or the outcomes of the study.  
10.1.6 
Data quality assurance 
All participant data relating to the study will be recorded on printed or eCRF unless transmitted 
to the Sponsor or designee electronically (eg, laboratory data). The Investigator is responsible for 
verifying that data entries are accurate and correct by physically or electronically signing the 
eCRF. 
The Investigator must maintain accurate documentation (source data) that supports the 
information entered in the eCRF. 
The Investigator must permit study-related monitoring, audits, IRB/IEC review, and regulatory 
agency inspections and provide direct access to source data documents. 
The Sponsor or designee is responsible for the data management of this study including quality 
checking of the data. 
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Study monitors will perform ongoing source data verification to confirm that data entered into 
the eCRF by authorized site personnel are accurate, complete, and verifiable from source 
documents; that the safety and rights of participants are being protected; and that the study is 
being conducted in accordance with the currently approved protocol and any other study 
agreements, ICH-GCP, and all applicable regulatory requirements. 
All essential documents are to be retained by the Investigator until at least 2 years after the last 
approval of a marketing application in an ICH region and until there are no pending or 
contemplated marketing applications in an ICH region, or at least 2 years have elapsed since the 
formal discontinuation of clinical development of the study medication/investigational device. 
These documents should be retained for a longer period, however, if required by the applicable 
regulatory requirement(s) or by an agreement with UCB (Committee for Proprietary Medicinal 
Products [CPMP]/ICH/135/95, 2002 [Section 4.9.5]). The Investigator will contact UCB for 
authorization prior to the destruction of any study records or in the event of accidental loss or 
destruction of any study records. The Investigator will also notify UCB should he/she relocate or 
move the study-related files to a location other than that specified in the Sponsor’s trial master 
file. 
10.1.6.1 
Electronic Case Report form completion 
The Investigator is responsible for prompt reporting of accurate, complete, and legible data in the 
eCRFs and in all required reports. 
Any change or correction to the eCRF after saving must be accompanied by a reason for the 
change. 
Corrections made after the Investigator’s review and approval (by means of a 
password/electronic signature) will be reapproved by the Investigator. 
The Investigator should maintain a list of personnel authorized to enter data into the eCRF. 
Detailed instructions will be provided in the eCRF Completion Guidelines. 
10.1.6.2 
Apps 
Not applicable. 
10.1.7 
Source documents 
All source documents must be accurate, clear, unambiguous, permanent, and capable of being 
audited. They should be made using some permanent form of recording (ink, typing, printing, 
optical disc). They should not be obscured by correction fluid or have temporary attachments 
(such as removable self-stick notes). Photocopies and/or printouts of eCRFs are not considered 
acceptable source documents. 
Source documents are original records in which raw data are first recorded. These may include 
hospital/clinic/general practitioner records, charts, diaries, x-rays, laboratory results, printouts, 
pharmacy records, care records, ECG or other printouts, completed scales, quality of life 
questionnaires, or video, for example. Source documents should be kept in a secure, limited 
access area. 
Source documents that are computer generated and stored electronically must be printed for 
review by the monitor (eg, ECG reports). Once printed, these copies should be signed and dated 
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by the Investigator and become a permanent part of the participant’s source documents. The 
Investigator will facilitate the process for enabling the monitor to compare the content of the 
printout and the data stored in the computer to ensure all data are consistent. 
Electronic data records, such as Holter monitor records or electroencephalogram records, must 
be saved and stored as instructed by UCB (or designee). 
10.1.8 
Study and site closure 
The Sponsor/designee reserves the right to close the study site or terminate the study at any time 
for any reason at the sole discretion of the Sponsor. Study sites will be closed upon study 
completion. A study site is considered closed when all required documents and study supplies 
have been collected and a study-site closure visit has been performed. 
The Investigator may initiate study-site closure at any time, provided there is reasonable cause 
and sufficient notice is given in advance of the intended termination. 
Reasons for the early closure of a study site by the Sponsor or Investigator may include but are 
not limited to: 
• Failure of the Investigator to comply with the protocol, the requirements of the IRB/IEC or 
local health authorities, the Sponsor's procedures, or GCP guidelines 
• Inadequate recruitment of participants by the Investigator 
• Discontinuation of further study medication development 
10.1.9 
Publication policy 
The results of this study may be published or presented at scientific meetings. If this is foreseen, 
the Investigator agrees to submit all manuscripts or abstracts to the Sponsor before submission. 
This allows the Sponsor to protect proprietary information and to provide comments. 
The Sponsor will comply with the requirements for publication of study results. In accordance 
with standard editorial and ethical practice, the Sponsor will generally support publication of 
multicenter studies only in their entirety and not as individual site data. In this case, a 
coordinating Investigator will be designated by mutual agreement. 
Authorship will be determined by mutual agreement and in line with International Committee of 
Medical Journal Editors authorship requirements. 
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10.2 
Appendix 2: Clinical laboratory tests 
• The tests detailed in the table below will be performed by the central laboratory. 
• Local laboratory results are only required in the event that the central laboratory results 
are not available in time for either study treatment administration and/or response 
evaluation. If a local sample is required, it is important that the sample for central 
analysis is obtained at the same time. Additionally, if the local laboratory results are used 
to make either a study treatment decision or response evaluation, the results must be 
entered into the eCRF. 
• Protocol-specific requirements for inclusion or exclusion of participants are detailed in 
Section 5.1 and Section 5.2 of the protocol, respectively. 
• Additional tests may be performed at any time during the study as determined necessary 
by the Investigator or required by local regulations. 
Protocol-Required Safety Laboratory Assessments 
Laboratory 
Assessments 
Parameters 
Hematology 
Platelet Count  
RBC Indices: 
MCV 
MCH 
%Reticulocytes 
WBC Count with 
Differential: 
Neutrophils 
Lymphocytes 
Monocytes 
Eosinophils 
Basophils 
RBC Count 
Hemoglobin 
Hematocrit 
Clinical 
Chemistry a 
BUN 
Potassium 
AST/SGOT 
Total and 
direct bilirubin 
 
Creatinine 
Sodium 
ALT/SGPT 
Total Protein 
 
Glucose 
(fasting) 
Calcium 
Alkaline phosphatase 
Albumin 
 
Lactate 
dehydrogenase 
CRP 
 
 
Routine 
Urinalysis 
• Specific gravity 
• pH, glucose, protein, blood, ketones, albumin, bilirubin, urobilinogen, 
nitrite, leukocyte esterase by dipstick 
• Microscopic examination (if blood or protein is abnormal) 
Other 
Screening 
Tests 
• Urine human chorionic gonadotropin pregnancy test (as needed for 
women of childbearing potential) b 
• Serology testing for HIV, hepatitis B, and hepatitis C 
• All study-required laboratory assessments will be performed by a central 
laboratory. 
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Laboratory 
Assessments 
Parameters 
• The results of each test must be entered into the eCRF. 
ALT=alanine aminotransferase; AST=aspartate aminotransferase; BUN=blood urea nitrogen; CRP=C-reactive 
protein; eCRF=electronic Case Report form; HIV=human immunodeficiency virus; IEC=Independent Ethics 
Committee; IRB=Internal Review Board; MCH=mean corpuscular hemoglobin; MCV=mean corpuscular 
volume; RBC=red blood cell count; SAE=serious adverse event; SGOT=serum glutamic oxaloacetic 
transaminase; SGPT=serum glutamic pyruvic transaminase; ULN=upper limit of normal; WBC=white blood 
cell count 
a Details of liver chemistry stopping criteria and required actions and follow-up assessments after liver stopping 
or monitoring event are given in Section 7.1.1 and Appendix 6 (Section 10.6). All events of ALT ≥3 × ULN and 
bilirubin ≥2 × ULN (>35% direct bilirubin) or ALT ≥3 × ULN and INR >1.5, if INR measured, may indicate 
severe liver injury (possible Hy’s Law) and must be reported as an SAE (excluding studies of hepatic 
impairment or cirrhosis). 
b Local urine testing will be standard for the protocol unless serum testing is required by local regulation or 
IRB/IEC. 
Investigators must document their review of each laboratory safety report. 
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10.3 
Appendix 3: Adverse events – definitions and procedures for 
recording, evaluating, follow-up, and reporting 
10.3.1 
Definition of adverse event 
AE Definition 
• An AE is any untoward medical occurrence in a patient or clinical study participant, 
temporally associated with the use of study medication, whether or not considered related 
to the study medication. 
• NOTE: An AE can therefore be any unfavorable and unintended sign (including an 
abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally 
associated with the use of study medication. 
 
Events Meeting the AE Definition  
• Any abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or 
other safety assessments (eg, ECG, radiological scans, vital signs measurements), 
including those that worsen from Baseline, considered clinically significant in the medical 
and scientific judgment of the Investigator (ie, not related to progression of underlying 
disease). 
• Exacerbation of a chronic or intermittent pre-existing condition including either an 
increase in frequency and/or intensity of the condition. 
• New conditions detected or diagnosed after study medication administration even though it 
may have been present before the start of the study. 
• Signs, symptoms, or the clinical sequelae of a suspected drug-drug interaction. 
• Signs, symptoms, or the clinical sequelae of a suspected overdose of either study 
medication or a concomitant medication. Overdose per se will not be reported as an 
AE/SAE unless it is an intentional overdose taken with possible suicidal/self-harming 
intent. Such overdoses should be reported regardless of sequelae. 
• "Lack of efficacy" or "failure of expected pharmacological action" per se will not be 
reported as an AE or SAE. Such instances will be captured in the efficacy assessments. 
However, the signs, symptoms, and/or clinical sequelae resulting from lack of efficacy will 
be reported as AE or SAE if they fulfill the definition of an AE or SAE. 
 
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Events NOT Meeting the AE Definition  
• Any clinically significant abnormal laboratory findings or other abnormal safety 
assessments which are associated with the underlying disease, unless judged by the 
Investigator to be more severe than expected for the participant’s condition. 
• The disease/disorder being studied or expected progression, signs, or symptoms of the 
disease/disorder being studied, unless more severe than expected for the study participant’s 
condition. 
• Medical or surgical procedure (eg, endoscopy, appendectomy): the condition that leads to 
the procedure is the AE. 
• Situations in which an untoward medical occurrence did not occur (social and/or 
convenience admission to a hospital). 
• Anticipated day-to-day fluctuations of pre-existing disease(s) or condition(s) present or 
detected at the start of the study that do not worsen. 
10.3.2 
Definition of serious adverse event 
If an event is not an AE per definition above, then it cannot be an SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease). 
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An SAE is defined as any untoward medical occurrence that, at any dose: 
a. Results in death 
b. Is life-threatening 
The term 'life-threatening' in the definition of 'serious' refers to an event in which the 
participant was at risk of death at the time of the event. It does not refer to an event, which 
hypothetically might have caused death, if it were more severe. 
c. 
Requires inpatient hospitalization or prolongation of existing hospitalization 
In general, hospitalization signifies that the participant has been detained (usually involving at 
least an overnight stay) at the hospital or emergency ward for observation and/or treatment that 
would not have been appropriate in the physician’s office or outpatient setting. Complications 
that occur during hospitalization are AEs. If a complication prolongs hospitalization or fulfills 
any other serious criteria, the event is serious. When in doubt as to whether “hospitalization” 
occurred or was necessary, the AE should be considered serious. 
Hospitalization for elective treatment of a pre-existing condition that did not worsen from 
baseline is not considered an AE. 
d. Results in persistent disability/incapacity 
• The term disability means a substantial disruption of a person’s ability to conduct normal 
life functions. 
• 
This definition is not intended to include experiences of relatively minor medical 
significance such as uncomplicated headache, nausea, vomiting, diarrhea, influenza, and 
accidental trauma (eg, sprained ankle) which may interfere with or prevent everyday life 
functions but do not constitute a substantial disruption. 
e. 
Is a congenital anomaly/birth defect 
f. 
Important medical events: 
• Medical or scientific judgment should be exercised in deciding whether SAE reporting is 
appropriate in other situations such as important medical events that may not be 
immediately life-threatening or result in death or hospitalization but may jeopardize the 
participant or may require medical or surgical intervention to prevent one of the other 
outcomes listed in the above definition. These events should usually be considered serious. 
Examples of such events include, but are not limited to, potential Hy’s law, invasive or 
malignant cancers, intensive treatment in an emergency room or at home for allergic 
bronchospasm, blood dyscrasias or convulsions that do not result in hospitalization, or 
development of drug dependency or drug abuse. 
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10.3.3 
Recording and follow-up of adverse events and/or serious adverse 
events 
AE and SAE Recording 
• When an AE/SAE occurs, it is the responsibility of the Investigator to review all 
documentation (eg, hospital progress notes, laboratory reports, and diagnostics reports) 
related to the event. 
• The Investigator will then record all relevant AE/SAE information in the eCRF. 
• It is not acceptable for the Investigator to send photocopies of the participant’s medical 
records to UCB in lieu of completion of the UCB AE/SAE eCRF page. 
• There may be instances when copies of medical records for certain cases are requested by 
UCB. In this case, all participant identifiers, with the exception of the participant number, 
will be redacted on the copies of the medical records before submission to UCB. 
• The Investigator will attempt to establish a diagnosis of the event based on signs, 
symptoms, and/or other clinical information. Whenever possible, the diagnosis (not the 
individual signs/symptoms) will be documented as the AE/SAE. 
Assessment of Intensity 
The Investigator will make an assessment of intensity for each AE and SAE reported during 
the study and assign it to 1 of the following categories: 
• Mild: An event that is easily tolerated by the participant, causing minimal discomfort and 
not interfering with everyday activities. 
• Moderate: An event that causes sufficient discomfort and interferes with normal everyday 
activities. 
• Severe: An event that prevents normal everyday activities. An AE that is assessed as 
severe should not be confused with an SAE. Severe is a category utilized for rating the 
intensity of an event; and both AEs and SAEs can be assessed as severe. 
• An event is defined as ‘serious’ when it meets at least 1 of the predefined outcomes as 
described in the definition of an SAE, NOT when it is rated as severe (eg, a severe AE may 
be either serious or not serious, depending on whether these criteria are also met). 
The National Cancer Institute Common Terminology Criteria for Adverse Events should be 
used as a supportive standardization instrument to evaluate AEs and SAEs but the final 
intensity grading by the Investigator must be mild, moderate, or severe. 
 
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Assessment of Causality 
• The Investigator is obligated to assess the relationship between study medication and each 
occurrence of each AE/SAE. 
• A "reasonable possibility" of a relationship conveys that there are facts, evidence, and/or 
arguments to suggest a causal relationship, rather than a relationship cannot be ruled out. 
• The Investigator will use clinical judgment to determine the relationship. 
• Alternative causes, such as underlying disease(s), concomitant therapy, and other risk 
factors, as well as the temporal relationship of the event to study medication administration 
will be considered and investigated. 
• The Investigator will also consult the IB in his/her assessment. 
• For each AE/SAE, the Investigator must document in the medical notes that he/she has 
reviewed the AE/SAE and has provided an assessment of causality. 
• There may be situations in which an SAE has occurred and the Investigator has minimal 
information to include in the initial report to UCB. However, it is very important that the 
Investigator always make an assessment of causality for every event before the initial 
transmission of the SAE data to UCB. 
• The Investigator may change his/her opinion of causality in light of follow-up information 
and send a SAE follow-up report with the updated causality assessment. 
• The causality assessment is one of the criteria used when determining regulatory reporting 
requirements. 
 
Follow-up of AEs and SAEs 
• The Investigator is obligated to perform or arrange for the conduct of supplemental 
measurements and/or evaluations as medically indicated or as requested by UCB to 
elucidate the nature and/or causality of the AE or SAE as fully as possible. This may 
include additional laboratory tests or investigations, histopathological examinations, or 
consultation with other health care professionals. 
• An AE should be followed until it has resolved, has a stable sequelae, the Investigator 
determines that it is no longer clinically significant, or the participant is lost to follow-up. 
This follow-up requirement applies to AEs, SAEs, and AESIs. 
• If a participant dies during participation in the study or during a recognized follow-up 
period, the Investigator will provide UCB with a copy of any post-mortem findings 
including histopathology. 
• New or updated information will be recorded in the originally completed eCRF. 
• The Investigator will submit any updated SAE data to the Sponsor within 24 hours of 
receipt of the information. 
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10.3.4 
Reporting of serious adverse events 
SAE Reporting to UCB via an Electronic Data Collection Tool 
• The primary mechanism for reporting an SAE to UCB will be the electronic data collection 
tool. 
• If the electronic system is unavailable for more than 24 hours, then the site will use the 
paper SAE data collection tool (see next section). 
• The site will enter the SAE data into the electronic system as soon as they become 
available. 
• After the study is completed at a given site, the electronic data collection tool will be taken 
off-line to prevent the entry of new data or changes to existing data. 
• If a site receives a report of a new SAE from a study participant or receives updated data 
on a previously reported SAE after the electronic data collection tool has been taken off-
line, then the site can report this information on a paper SAE form (see next section). 
Contacts for SAE reporting can be found in SERIOUS ADVERSE EVENT REPORTING. 
 
SAE Reporting to UCB via Paper CRF 
• Facsimile transmission of the SAE paper CRF is the preferred method to transmit this 
information to UCB, see SERIOUS ADVERSE EVENT REPORTING. 
• In rare circumstances and in the absence of facsimile equipment, notification by telephone 
is acceptable with a copy of the SAE data collection tool sent by overnight mail or courier 
service. 
• Initial notification via telephone does not replace the need for the Investigator to complete 
and sign the SAE CRF pages within the designated reporting time frames. 
• Contacts for SAE reporting can be found in SERIOUS ADVERSE EVENT REPORTING. 
 
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10.4 
Appendix 4: Contraceptive guidance and collection of 
pregnancy information 
10.4.1 
Definitions 
10.4.1.1 
Woman of childbearing potential 
A woman is considered fertile following menarche and until becoming postmenopausal unless 
permanently sterile (see below). 
Women in the following categories are not considered WOCBP: 
1. Premenarchal 
2. Premenopausal female with 1 of the following: 
• Documented hysterectomy 
• Documented bilateral salpingectomy 
• Documented bilateral oophorectomy 
Note: Documentation can come from the site personnel’s review of the participant’s medical 
records, medical examination, or medical history interview. 
3. Postmenopausal female 
• A postmenopausal state is defined as no menses for 12 months without an alternative 
medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal 
range may be used to confirm a postmenopausal state in women not using hormonal 
contraception or hormonal replacement therapy (HRT). However, in the absence of 
12 months of amenorrhea, a single FSH measurement is insufficient. 
• Females on HRT and whose menopausal status is in doubt will be required to use one of 
the nonestrogen hormonal highly effective contraception methods if they wish to 
continue their HRT during the study. Otherwise, they must discontinue HRT to allow 
confirmation of postmenopausal status before study enrollment. 
10.4.2 
Contraception guidance 
Male participants 
Male participants with female partners of childbearing potential are eligible to participate if they 
agree to ONE of the following Treatment Period and for at least 90 days after the final dose of 
study treatment: 
• Are abstinent from penile-vaginal intercourse as their usual and preferred lifestyle 
(abstinent on a long term and persistent basis) and agree to remain abstinent. 
• Agree to use a male condom plus partner use of a contraceptive method with a failure rate 
of <1% per year as described in Table 10-1 when having penile-vaginal intercourse with 
a WOCBP who is not currently pregnant. 
• Agree to use a male condom when having penile-vaginal intercourse with a woman of 
childbearing potential who is not currently pregnant. 
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In addition, male participants must refrain from donating sperm for the duration of the study and 
for at least 90 days after the last dose of study medication. 
Male participants with a pregnant or breastfeeding partner must agree to remain abstinent from 
penile-vaginal intercourse or use a male condom during each episode of penile penetration for at 
least 90 days after the last dose of study medication. 
Female participants 
Female participants of childbearing potential are eligible to participate if they agree to use a 
highly effective method of contraception consistently and correctly as described in Table 10-1. 
Table 10-1: Highly Effective Contraceptive Methodsa 
Highly Effective Contraceptive Methods That Are User Dependent b 
Failure rate of <1% per year when used consistently and correctly. 
Combined (estrogen- and progestogen-containing) hormonal contraception associated with 
inhibition of ovulation c 
• Oral 
• Intravaginal 
• Transdermal 
Progestogen-only hormonal contraception associated with inhibition of ovulation 
• Oral 
• Injectable 
Highly Effective Methods That Are User Independent b 
Implantable progestogen only hormonal contraception associated with inhibition of ovulation 
• Intrauterine device 
• Intrauterine hormone-releasing system 
• Bilateral tubal occlusion 
Vasectomized partner 
A vasectomized partner is a highly effective contraception method provided that the partner is 
the sole male sexual partner of the woman of childbearing potential and the absence of sperm 
has been confirmed. If not, an additional highly effective method of contraception should be 
used. 
Sexual abstinence 
Sexual abstinence is considered a highly effective method only if defined as refraining from 
heterosexual intercourse during the entire period of risk associated with the study medication. 
The reliability of sexual abstinence needs to be evaluated in relation to the duration of the 
study and the preferred and usual lifestyle of the participant. 
a In case of newly started contraception pills/IUDs, PI should consider the correct timing of starting/applying such 
methods in relation to the menstrual cycle and the manufacturing instruction as when these newly started methods 
would become effective. 
b Typical use failure rates may differ from those when used consistently and correctly. Use should be consistent with 
local regulations regarding the use of contraceptive methods for participants participating in clinical studies.  
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c Hormonal contraception may be susceptible to interaction with the study medication, which may reduce the 
efficacy of the contraceptive method. In this case, two highly effective methods of contraception should be utilized 
during the treatment period and for at least 90 days after the last dose of study medication. Pregnancy testing 
• A WOCBP should only be included after a confirmed menstrual period and a negative highly 
sensitive urine pregnancy test. 
• Additional pregnancy testing should be performed during the Treatment Period (Section 1.3), 
at the EOS visit, corresponding to protocol-defined time frame in Section 10.4 after the last 
dose of study medication and as required locally. 
• Pregnancy testing will be performed whenever a menstrual cycle is missed or when 
pregnancy is otherwise suspected. 
• Pregnancy testing, with a sensitivity of ≥25mIU/mL will be performed. Urine pregnancy tests 
will be performed at all other visits. 
10.4.2.1 
Male participants with partners who become pregnant 
• The Investigator will attempt to collect pregnancy information on any male participant’s 
female partner who becomes pregnant while the male participant is in this study. This applies 
only to male participants who receive study medication. 
• After obtaining the necessary signed informed consent from the pregnant female partner 
directly, the Investigator will record pregnancy information on the appropriate form and 
submit it to the Sponsor within 24 hours of learning of the partner’s pregnancy. The female 
partner will also be followed to determine the outcome of the pregnancy. Information on the 
status of the mother and child will be forwarded to the Sponsor. Generally, the follow-up will 
be at least 12 months after the delivery date. Any termination of the pregnancy will be 
reported regardless of fetal status (presence or absence of anomalies) or indication for the 
procedure. 
10.4.2.2 
Female participants who become pregnant 
• Any female participant who becomes pregnant while participating in the study will be 
withdrawn from the study. 
• The Investigator will collect pregnancy information on any female participant who becomes 
pregnant while participating in this study. Information will be recorded on the appropriate 
form and submitted to the Sponsor within 24 hours of learning of a participant's pregnancy. 
The participant will be followed to determine the outcome of the pregnancy. The Investigator 
will collect follow-up information on the participant and the neonate and the information will 
be forwarded to the Sponsor. Generally, the follow-up will be at least 12 months after the 
delivery date. Any termination of pregnancy will be reported, regardless of fetal status 
(presence or absence of anomalies) or indication for the procedure. 
• While pregnancy itself is not considered to be an AE or SAE, any pregnancy complication or 
elective termination of a pregnancy will be reported as an AE or SAE. A spontaneous 
abortion is always considered to be an SAE and will be reported as such. Any post-study 
pregnancy-related SAE considered reasonably related to the study medication by the 
Investigator will be reported to the Sponsor as described in Section 8.3.5. While the 
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Investigator is not obligated to actively seek this information in former study participants, he 
or she may learn of an SAE through spontaneous reporting. 
 
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10.5 
Appendix 5: Genetics 
Not applicable. 
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10.6 
Appendix 6: Liver Safety – Suggested Actions and Follow-up 
Assessments 
Participants with PDILI must be assessed to determine if study medication must be discontinued. 
In addition, all concomitant medications and herbal supplements that are not medically necessary 
should also be discontinued. 
Investigators should attempt to obtain information on study participants in the case of study 
medication discontinuation to complete the final evaluation. 
Study participants with PDILI should not be withdrawn from the study until investigation and 
monitoring are complete. All results of these evaluations and observations, as well as the 
reason(s) for study medication discontinuation and/or participant withdrawal (if applicable), 
must be recorded in the source documents. The eCRF must document the primary reason for 
discontinuation of study medication. 
A specific monitoring plan must be agreed between the UCB study physician and the 
Investigator for study participants who have ALT >5xULN. The monitoring plan should include 
any necessary follow-up assessments (until resolution of the abnormal lab values). 
Phase 3 liver chemistry stopping criteria are designed to assure participant safety and to evaluate 
liver event etiology. Stopping criteria are presented in Table 10-2 and increased monitoring 
criteria are presented in Table 10-3. 
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Table 10-2: Phase 3 liver chemistry stopping criteria and follow-up 
assessments 
Liver Chemistry Stopping Criteria 
ALT-absolute 
ALT 8xULN 
ALT increase 
ALT 5xULN but <8xULN persists for 2 weeks 
ALT 3xULN but <5xULN persists for 4 weeks 
Bilirubin a, b 
ALT 3xULN and bilirubin 2xULN (>35% direct bilirubin)  
INR b  
ALT 3xULN and INR >1.5, if INR measured 
Cannot 
mMonitor 
ALT 5xULN but <8xULN and cannot be monitored weekly for 2 weeks 
ALT 3xULN but <5xULN and cannot be monitored weekly for 4 weeks 
Symptomatic c 
ALT 3xULN associated with symptoms (new or worsening) believed to be related 
to liver injury or hypersensitivity 
Suggested Actions and Follow-up Assessments 
Actions 
Follow-up Assessments 
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• Immediately discontinue study medication. 
• Report the event to the Sponsor within 
24 hours. 
• Complete the liver event eCRF, and 
complete as SAE data collection tool if the 
event also met the criteria for an SAE. b 
• Perform liver chemistry follow-up 
assessments. 
• Monitor the participant until liver chemistry 
test abnormalities resolve, stabilize, or 
return to baseline (see MONITORING). 
• Do not restart/rechallenge participant 
with study medication. 
• If restart/rechallenge not allowed per 
protocol or not granted, permanently 
discontinue study medication and continue 
participant in the study for any protocol 
specified follow-up assessments.Consider 
the need for a toxicology screening. 
MONITORING: 
For bilirubin or INR criteria  
• Repeat liver chemistry tests (include ALT, 
AST, alkaline phosphatase, bilirubin) and 
perform liver event follow-up assessments 
within 24 hours. 
• Monitor participant twice weekly until liver 
chemistry test abnormalities resolve, 
stabilize, or return to Baseline. 
• A specialist or hepatology consultation is 
recommended. 
For all other criteria  
• Repeat liver chemistry tests (include ALT, 
AST, alkaline phosphatase, bilirubin) and 
perform liver chemistry follow-up 
assessments within 24 to 72 hours. 
• Monitor participants weekly until liver 
chemistry abnormalities resolve, stabilize, 
or return to Baseline. 
• Viral hepatitis serology. d 
• Obtain INR and recheck with each liver 
chemistry assessment until the 
transaminases values show downward 
trend. 
• Only in those with underlying chronic 
hepatitis B at study entry (identified by 
positive HBsAg), quantitative hepatitis B 
DNA and hepatitis delta antibody. e 
• 
Obtain blood sample for PK analysis as 
soon as feasible after the most recent 
dose. f 
• Serum CPK and LDH. 
• Fractionate bilirubin, if total bilirubin 
 2xULN. 
• Obtain complete blood count with 
differential to assess eosinophilia. 
• Record the appearance or worsening of 
clinical symptoms of liver injury, or 
hypersensitivity, on the AE report form. 
• Record use of concomitant medications 
(including acetaminophen, herbal 
remedies, and other over-the-counter 
medications) on the concomitant 
medications eCRF. 
• Record alcohol use on the liver event 
alcohol intake eCRF. 
• Exclude pregnancy. 
• Urine drug screen. 
For bilirubin or INR criteria: 
• Anti-nuclear antibody, anti-smooth 
muscle antibody, Type 1 anti-liver 
kidney microsomal antibodies, and 
quantitative total IgG or gamma 
globulins. 
• A serum acetaminophen adduct assay for 
assessing the potential acetaminophen 
contribution to liver injury in 
participants with definite or likely 
acetaminophen use in the preceding 
week. 
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• Liver imaging (ultrasound, magnetic 
resonance, or computerized tomography) 
and/or liver biopsy to evaluate liver 
disease; complete Liver Imaging and/or 
Liver Biopsy eCRFs. 
AE=adverse event; ALT=alanine aminotransferase; AST=aspartate aminotransferase; CPK=creatine 
phosphokinase; DNA=deoxyribonucleic acid; eCRF=electronic case report form; HBsAg=hepatitis B virus 
surface antigen; HCsAg=hepatitis C virus surface antigen; HPLC=high performance liquid chromatography; 
IgM=immunoglobulin M; INR=international normalized ratio; LDH=lactate dehydrogenase; 
PK=pharmacokinetic; RNA=ribonucleic acid; SAE=serious adverse event; ULN=upper limit of normal 
a Serum bilirubin fractionation should be performed if testing is available. If serum bilirubin fractionation is not 
immediately available, discontinue study medication if ALT 3xULN and bilirubin 2xULN. Additionally, if 
serum bilirubin fractionation testing is unavailable, record the absence/presence of detectable urinary bilirubin 
on dipstick which is indicative of direct bilirubin elevations suggesting liver injury. 
b All events of ALT 3xULN and bilirubin 2xULN (>35% direct bilirubin) or ALT 3xULN and INR >1.5 may 
indicate severe liver injury (possible ‘Hy’s Law’) and must be reported as an SAE (excluding studies of 
hepatic impairment or cirrhosis). The INR measurement is not required and the stated threshold value will not 
apply to participants receiving anticoagulants. 
c New or worsening symptoms believed to be related to liver injury (such as fatigue, nausea, vomiting, right upper 
quadrant pain or tenderness, or jaundice) or hypersensitivity (such as fever, rash or eosinophilia). 
d Includes: Hepatitis A IgM antibody; HBsAg and HBcAb; hepatitis C RNA; cytomegalovirus IgM antibody; 
Epstein-Barr viral capsid antigen IgM antibody (or if unavailable, heterophile antibody or monospot testing); and 
hepatitis E IgM antibody. 
e If hepatitis delta antibody assay cannot be performed, it can be replaced with a polymerase chain reaction of 
hepatitis D RNA virus (where needed) (Le Gal et al, 2005). 
f Record the date/time of the PK blood sample draw and the date/time of the last dose of study medication prior to 
the PK blood sample draw on the eCRF. If the date or time of the last dose is unclear, provide the participant’s 
best approximation. If the date/time of the last dose cannot be approximated OR a PK sample cannot be collected 
in the time period indicated above, do not obtain a PK sample. Instructions for sample handling and shipping are in 
the IMP Handling Manual. 
 
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Table 10-3: Phase 3 liver chemistry increased monitoring criteria with 
continued study medication 
Liver Chemistry Increased Monitoring Criteria 
Criteria 
Actions 
ALT 5xULN and <8xULN and 
bilirubin <2xULN without symptoms 
believed to be related to liver injury or 
hypersensitivity, and who can be 
monitored weekly for 2 weeks. 
OR 
ALT 3xULN and <5xULN and 
bilirubin <2xULN without symptoms 
believed to be related to liver injury or 
hypersensitivity, and who can be 
monitored weekly for 4 weeks. 
• Notify the Sponsor Medical Monitor within 
24 hours of learning of the abnormality to discuss 
participant safety. 
• Participant can continue study medication. 
• Participant must return weekly for repeat liver 
chemistry tests (ALT, AST, alkaline phosphatase, 
bilirubin) until the abnormalities resolve, 
stabilize, or return to baseline. 
• If at any time, the participant meets liver 
chemistry stopping criteria, proceed as described 
in Section 7.1.1. 
• If ALT decreases from ALT 5xULN and 
<8xULN to ≥3xULN but <5xULN, continue to 
monitor liver chemistries weekly. 
• If, after 4 weeks of monitoring, ALT <3xULN 
and bilirubin <2xULN, monitor participants twice 
monthly until liver chemistry tests resolve, 
stabilize, or return to baseline. 
ALT=alanine aminotransferase; AST=aspartate aminotransferase; ULN=upper limit of normal 
 
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10.7 
Appendix 7: Medical device incidents – definition and 
procedures for recording, evaluating, follow-up, and reporting 
Not applicable. 
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10.8 
Appendix 8: Rapid alert procedures 
Not applicable. 
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10.9 
Appendix 9: Country-specific requirements 
Not applicable. 
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10.10 
Appendix 10: Abbreviations and trademarks 
AChE 
acetylcholinesterase  
AChR 
acetylcholine receptor 
ADA 
anti-drug antibody 
ADL 
activities of daily living 
AE 
adverse event 
AESI 
adverse event of special interest 
AESM 
adverse event of special monitoring 
ALP 
alkaline phosphatase 
ALT 
alanine aminotransferase 
AST 
aspartate aminotransferase 
BP 
blood pressure 
CPMP 
Committee for Proprietary Medicinal Products 
C-SSRS 
Columbia-Suicide Severity Rating Scale 
CTCAE 
Common Terminology Criteria for Adverse Events 
IDMC 
Independent Data Monitoring Committee 
ECG 
electrocardiogram 
eCRF 
electronic case report form 
EOS 
End of Study 
EQ-5D-5L 
5-level European quality of life 5 dimension 
EQ VAS 
EQ visual analogue scale 
FcRn 
neonatal Fc receptor 
FSH 
follicle stimulating hormone 
FVC 
forced vital capacity 
GBS 
Guillain-Barré Syndrome 
GCP 
Good Clinical Practice 
GI 
gastrointestinal 
HRT 
hormonal replacement therapy 
IB 
Investigator’s Brochure 
ICF 
informed consent form 
ICH 
International Council for Harmonisation 
IEC 
Independent Ethics Committee 
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Ig 
immunoglobulin 
IGRA 
interferon gamma release assay 
IMP 
investigational medicinal product 
INR 
international normalized ratio 
IRB 
Institutional Review Board 
IRT 
interactive response technology 
ITP 
immune thrombocytopenia 
IV 
intravenous 
IVIg 
intravenous infusion of immunoglobulin G 
LLOQ 
lower limit of quantification 
LTBI 
latent tuberculosis infection 
MedDRA 
Medical Dictionary for Regulatory Activities 
MG 
myasthenia gravis 
MG-ADL 
myasthenia gravis-activates of daily living 
MGII 
myasthenia gravis impairment index 
MGQoL15r 
revised 15-item myasthenia gravis quality of life questionnaire 
MuSK 
muscle-specific kinase 
NMJ 
neuromuscular junctions 
NTMB 
nontuberculosis mycobacterium infection 
OLE 
open-label extension 
PD 
pharmacodynamic(s) 
PDILI 
potential drug-induced liver injury 
PEF 
peak expiratory flow 
PEOT 
premature end of treatment 
PEX 
plasma exchange 
PK 
pharmacokinetic(s) 
PRO 
patient-reported outcome 
PV 
pemphigus vulgaris 
QMG 
quantitative myasthenia gravis test 
QTc 
QT interval corrected for heart rate 
SAE 
serious adverse event 
SAP 
Statistical Analysis Plan 
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SC 
subcutaneous(ly) 
SD 
standard deviation 
SS 
Safety Set 
TB 
tuberculosis 
TEAE 
treatment-emergent adverse event 
ULN 
upper limit of normal 
WOCBP 
woman of childbearing potential 
 
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10.11 
Appendix 11: Protocol amendment history 
Amendment 1 (01 Nov 2019) 
Overall Rationale for the Amendment 
This amendment is considered to be substantial based on the criteria set forth in Article 10(a) of 
Directive 2001/20/EC of the European Parliament and the Council of the European Union. 
Section # and Name 
Description of Change 
Brief Rationale 
Serious Adverse Event 
Reporting 
All reporting instructions for 
Japan have been removed. 
This study will be conducted 
in Japan as a part of global 
study.  
Global 
Where applicable, the term 
“subject” has been replaced by 
“study participant” 
The change has been made as 
per UCB template and to 
remain consistent with the 
Phase 3 rozanolixizumab 
clinical program. 
1.1 Synopsis 
2.2 Background 
4.1 Overall Design 
4.2 Scientific Rationale for 
Study Design 
4.3 Justification for Dose  
5.1 Inclusion Criteria 
5.2 Exclusion Criteria 
9.8 Determination of Sample 
Size 
Reference to another lead-in 
study, MGC003, has been made 
throughout. 
General update. 
1.1 Synopsis 
4.1 Overall Design 
9.8 Determination of Sample 
Size 
The number of study participants 
was changed.  
The new number of study 
participants includes an 
approximate total from the 
China-specific study, 
MCG003. 
1.1 Synopsis 
Number of participants: 
Additional wording has been 
included to allow study 
participants (at selected sites) to 
enroll into a substudy. 
This is to allow for study 
participants to be included in 
Bioequivalence or Human 
Factor substudies. 
1.1 Synopsis 
3 Objectives and Endpoints 
The term “Value” has been 
removed from all endpoints, 
where applicable. 
 
Updated for consistency, as 
there were redundancies. 
1.1 Synopsis 
3 Objectives and Endpoints 
An additional exploratory 
objective and endpoint was added 
This was added to assess the 
effect of rozanolixizumab on 
tetanus IgG antibodies. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.1 Synopsis 
3 Objectives and Endpoints 
An additional exploratory 
objective and endpoint was added.  
This was added to capture the 
reduction steroid use in study 
participants receiving 
rozanolixizumab. 
1.1 Synopsis 
 
Additional text has been included 
to highlight dose adjustments can 
be made if the study participant’s 
bodyweight changes during the 
study. 
Updated to provide further 
clarification. 
1.3 Schedule of Activities 
4.4 End of Study definition 
8.2.6 Assessment and 
management of TB and TB risk 
factors 
8.3.1 Time period and frequency 
for collecting AE and SAE 
information 
10.4.3 Pregnancy testing 
Change last study visit from Final 
Visit to End of Study. 
The update was made to 
harmonize across the 
rozanolixizumab Phase 3 
clinical program. 
1.3 Schedule of Activities 
The frequency of bodyweight 
measurements has been reduced 
to every 6 months.  
Updated to provide further 
clarification, as bodyweight 
does not frequently change. 
1.3 Schedule of Activities 
Urine drug screen procedure was 
removed from the study. 
The exclusionary criterion 
pertaining to use of marijuana 
was removed. 
1.3 Schedule of Activities 
Study participants identification 
card assigned has been added.  
Missing activity from original 
protocol. 
1.3 Schedule of Activities 
An additional activity “anti-
tetanus toxoid titer” was added.  
Anti-tetanus toxoid titer will 
be collected as part of the 
assessment for the effect of 
rozanolixizumab on tetanus 
IgG antibodies. 
1.3 Schedule of Activities 
Blood sampling for 
pharmacokinetics at Visits 5 and 
the first visit during Weeks 9 to 
48 have been removed. 
Pharmacokinetic 
concentrations are expected to 
be Below the Limit of 
Quantitation at these visits. 
1.3 Schedule of Activities 
Blood sampling for IgA, IgE and 
IgM was added.  
Missing activity from original 
protocol. 
1.3 Schedule of Activities 
Footnote f (any recreational or 
medicinal use of cannabis [ie, 
marijuana] or cannabidiols is not 
authorized in the study) was 
removed.  
The use of cannabidiols and 
medicinal marijuana 
(prescribed by a physician) is 
now part of the permitted 
concomitant treatments list. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
Footnote j changed to k: The 
following wording “At dosing 
visits” was removed. 
The text no longer applied to 
these assessments 
1.3 Schedule of Activities 
4.1 Overall design 
 
A visit window for the Screening 
Visit has been added. 
The addition of ±1 week has 
been added to provide 
flexibility for Screening 
activities and randomization. 
2.2 Background 
The number of rozanolixizumab 
clinical studies has been amended 
from 4 to 6 due to the additional 
studies; CIDP01 and MG0003. 
General update. 
2.2 Background 
An additional country has been 
added in reference to the study 
population in UP0060. 
General update. 
4.4 End of Study definition 
 
The word “lacking in” has been 
changed to “limited”  
This change was made to 
clarify some efficacy 
information on the 
 
dose is available. 
5.1 Inclusion criteria 
Criterion #3: the bodyweight 
parameter was changed from 
greater than (“>”) to (“≥”) equal 
to or greater than. 
The update was made to 
harmonize the inclusion 
criterion across the 
rozanolixizumab Phase 3 
clinical program. 
5.2 Exclusion criteria 
Criterion #2: Replaced “drug 
abuse” with “other substance 
disorder use” and clarified study 
participants history is based on 12 
months prior to Screening Visit. 
The criterion was updated to 
reflect the new language 
adopted in DSM-5. 
5.2 Exclusion criteria 
Criterion #4: The testing for 
documented active or latent 
tuberculosis has been updated to 
QuantiFERON®-TB Gold Plus  
QuantiFERON®-TB Gold test 
is no longer commercially 
available.  
5.2 Exclusion Criteria 
Criterion #11: Amended 
definition (GFR less than 
60ml/min/1.73m2) has been 
included for renal impairment. 
The update was made to 
harmonize the exclusion 
criterion across the 
rozanolixizumab Phase 3 
clinical program. 
5.2 Exclusion Criteria 
Criterion #23: Amended upper 
limit of normal for alanine 
transaminase, aspartate 
aminotransferase, or alkaline 
phosphatase was changed from 
>2x to >3x. 
The inclusion of mild renal 
impairment is applicable to the 
Phase 3 clinical program with 
prior Phase 2 experience and 
no observations on hepatics 
impact. 
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Section # and Name 
Description of Change 
Brief Rationale 
5.2 Exclusion criteria 
Criterion #17: The description for 
the use of prescribed cannabidiols 
and/or medicinal marijuana has 
been amended and is no longer 
part of the exclusion criteria list. 
The new text has been placed 
under Section 6.5.1 Permitted 
concomitant treatments 
(medications and therapies).  
Original criterion was 
restrictive and could 
significantly limit the number 
of study participants in US and 
potentially other countries. 
5.2 Exclusion criteria 
Criterion #12: Amended upper 
limit of normal for alanine 
transaminase, aspartate 
aminotransferase, or alkaline 
phosphatase was changed from 
>2x to >3x. 
The inclusion of mild renal 
impairment is applicable to the 
Phase 3 clinical program with 
prior Phase 2 experience and 
no observations on hepatics 
impact. 
5.2 Exclusion criteria 
New criterion has been added, 
#18: Study participant has 
corrected QT interval (QTc) >450 
msec (for male participants) or 
QTc >470 msec (for female 
participants) or QTc >480 msec in 
participants with bundle branch 
block. 
The addition was made to 
harmonize the exclusion 
criterion across the MG 
studies. 
5.3 Lifestyle Restrictions 
Study participants will abstain 
from recreational use of cannabis 
has now been removed. Study 
participants will be allowed to use 
medicinal marijuana or 
cannabidiols, as prescribed by a 
physician. 
Due to the change for cannabis 
(ie, medicinal marijuana) or 
cannabidiols use (as prescribed 
by a physician), the lifestyle 
restriction no longer applies to 
the study.  
6.1 Treatments administered 
Additional wording for the details 
(rate of infusion, administration, 
appropriate records handling, 
blinded and unblinded site 
personnel roles) available in the 
IMP Handling Manual have been 
included.    
Previous wording did not 
accurately provide information 
for what contents are available 
in the IMP Handling Manual.  
6.5.1 Permitted concomitant 
treatments (medications and 
therapies 
Additional wording has been 
added as part of permitted 
medication use: The use of 
cannabidiols and medicinal 
marijuana (prescribed by a 
physician) is also permitted. 
When applicable, the subject must 
be on a stable dose of 
cannabidiols and/or medicinal 
Due to the lack of 
pharmacodynamic and 
pharmacokinetic interactions 
between marijuana/ 
cannabidiols and 
rozanolixizumab, this criterion 
was revised. 
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Section # and Name 
Description of Change 
Brief Rationale 
marijuana for 4 weeks prior to 
Screening Visit and remain stable 
for the duration of the study. 
Table 6-2 Permitted concomitant 
medications for the treatment of 
myasthenia gravis: The dose for 
oral corticosteroids has been 
amended to state “no specific 
requirements”. 
6.5.3 Treatments specific to 
NMJ interference 
Correct reference has been added 
for the MGFA classification list. 
Additional wording to provide 
clarity that participants on rescue 
therapy continue to attend study 
visits. 
General update for 
clarification. 
6.6 Dose modification 
8.3.7 Adverse events of special 
monitoring 
Change in terminology: GI 
disorders was changed to GI 
disturbances 
The update was made to 
harmonize across the 
rozanolixizumab Phase 3 
clinical program. 
6.7 Home visits 
New section added on home 
visits.  
Included to provide clear 
information for home visits. 
6.7 Treatment after the end of 
the study 
Now Section 6.8, the word “will” 
was replaced with “may” in the 
following sentence: Study 
participants who complete 
participation in MG0004 will 
have the possibility to continue 
receiving rozanolixizumab 
through a managed access 
program as per applicable local 
regulations. 
There is no guarantee as 
suggested with the previous 
wording, as a managed access 
program will not be available 
in some countries. 
7.1.3 Discontinuation due to 
other adverse events or medical 
conditions 
An additional event “study 
participant has new onset or 
recurrent neoplastic disease 
(except for superficial basal or 
squamous cell carcinoma of the 
skin not requiring targeted 
biological therapy, chemotherapy 
or radiation” as added.  
New language was added to 
align withdraw criteria with 
exclusion criteria, and not 
expose study participants with 
neoplastic disease to 
rozanolixizumab. 
8 Study assessments and 
procedures 
Additional information on 
Unscheduled Visits have been 
added.  
There were no specific details 
and instructions for the 
assessments needed at an 
Unscheduled Visit.  
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Section # and Name 
Description of Change 
Brief Rationale 
8.1 Efficacy assessments 
Order of efficacy assessments 
were amended.  
Order of efficacy assessments 
were amended to align with 
study procedure manual. 
8.1.1 Patient-reported outcomes 
8.1.2 QMG scale 
8.1.3 MG-Composite scale 
New wording has been added: 
“Study participants should not 
take pyridostigmine (or any AChE 
inhibitor medication) from 
midnight before testing when 
medically safe to do so to 
standardize testing” 
Updated to provide clear 
guidance for each assessment. 
8.1.3 Patient-reported outcomes 
Order of PROs (PGI-S, PGI-C, 
EQ-5D-5L, MGII, MG-QOL15r) 
were amended. 
Order of PRO assessments 
were amended to align with 
study procedure manual. 
8.1.3.1 Myasthenia gravis-
activities of daily living scale 
Section number has been changed 
to 8.1.1.1 
To align with study procedure 
manual. 
8.1.3.2 MG Impairment Index 
Section number has been changed 
to 8.1.1.6.  
There is an additional step to the 
assessment, where the 
Investigator will examine the 
study participant prior to scoring 
all items. 
To align with study procedure 
manual. 
8.1.3.3 MG PRO Symptoms 
Section number has been changed 
to 8.1.1.2.  
In addition, a total score range 
was included to this PRO 
description, as per scale presented 
in Appendix 15.  
Order of efficacy assessments 
were amended to align with 
study procedure manual. 
8.1.3.4 Revised 15-item 
myasthenia gravis quality of life 
questionnaire 
Section number has been changed 
to 8.1.1.7 
To align with study procedure 
manual. 
8.1.3.5 Patient Global 
Impression of Severity 
Section number has been changed 
to 8.1.1.3 
To align with study procedure 
manual. 
8.1.3.6 Patient Global 
Impression of Change 
Section number has been changed 
to 8.1.1.4 
To align with study procedure 
manual. 
8.2 Safety Assessment 
Additional information on onsite 
observation timings postdose was 
included. 
Added to provide clarification 
for postdose observations. 
8.2.2 Vital signs 
The measurement of respiratory is 
no longer required as part of vital 
signs assessment.  
To be consistent with other 
Rozanolixizumab protocols. 
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Section # and Name 
Description of Change 
Brief Rationale 
8.2.6 Assessment and 
management of TB and TB risk 
factors 
Layout and structure of 
assessment and management of 
TB and TB risk factors has been 
modified. 
To be consistent with other 
Rozanolixizumab protocols. 
8.3.6 Adverse events of special 
interest  
Measurements for ALT, and 
bilirubin or ALT were amended 
to: “3xULN ALT and ≥2xULN 
bilirubin (>35% direct bilirubin) 
or ALT 3xULN and international 
normalized ratio (INR) >1.5, if 
INR measured.” 
Original wording was 
inconsistent with Appendix 2 
Clinical laboratory tests. 
8.3.7 Adverse events of interest 
This section has renamed to 
“Adverse events of special 
monitoring” 
New information to include that 
AEs of hypersensitivity reactions 
and infused-related reactions will 
be monitored by the Investigators.  
Terminology change for 
consistency. 
 
Harmonize the AEs of special 
monitoring across the 
rozanolixizumab Phase 3 
clinical program. 
8.3.8 Treatment-emergent 
adverse events 
A new subsection has been added.  To provide a clear definition of 
a treatment-emergent adverse 
event. 
8.5 Treatment of Overdose 
Definition of overdose was 
amended: Any increase of 10% or 
greater from the assigned dose per 
week should be considered, 
irrespective of the weight tier 
band. 
The change was made to 
provide further clarification on 
the definition of overdose. 
8.6 Pharmacokinetics and 
antidrug antibodies  
Use of samples for establishing 
assay parameters have been 
added. 
To be consistent with the ICF 
and other Rozanolixizumab 
protocols. Further, to remove 
detail redundancies covered in 
the laboratory manual. 
8.6 Pharmacokinetics and 
antidrug antibodies 
New text pertaining to PK blood 
sample collection at additional 
timepoints in a local substudy has 
been added.  
New wording introduced to 
align with the Phase 3 clinical 
program. 
8.9 Biomarkers 
The following sentence has been 
amended to in include AESM, 
and to ensure safety samples must 
rather than may be collected. 
New wording introduced to 
align on UCB safety 
procedures. 
10.1.7 Source documents 
The following sentence, “The 
following data will be recorded 
directly in the eCRF and will not 
appear in a separate source 
The information was 
repetitive. 
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Section # and Name 
Description of Change 
Brief Rationale 
document as defined above” was 
removed. 
10.2 Appendix 2: Clinical 
laboratory tests 
Serology testing (for hepatitis B, 
hepatitis C, and HIV) has been 
added. 
C-reactive protein was removed. 
Consistency with study design 
10.4 Appendix 4: Contraceptive 
Guidance and Collection of 
Pregnancy Information 
The guidance and procedures for 
contraception and collection of 
pregnancy information has been 
amended. 
The information was amended 
to provide additional 
information and to keep 
consistency across the 
rozanolixizumab clinical 
program. 
10.6 Appendix 6: Liver Safety – 
Suggested Actions and Follow-
up Assessments 
Serum acetaminophen adduct 
assay changed from HPLC to 
spectrophotometry.  
High performance liquid 
chromatography (HPLC) is 
more time-consuming and 
samples will have to be sent to 
a referred ICON laboratory, 
ARUP. 
10.10 Appendix 10: 
Abbreviations and Trademarks 
Several additions, deletions, and 
edits were made to the list of 
abbreviations. 
General updates and 
typographical corrections for 
consistency. 
10.12 to 10.20 Appendix 12 to 
20  
Order of appendices were 
changed to match the order shown 
in the body of the protocol. 
General consistency.  
10.22.1 Appendix 22: 
Management of headaches 
The procedure for the 
management of headaches was 
amended to provide additional 
information. 
The updates were made to 
enhance the management of 
headache and to harmonize the 
procedures across the 
rozanolixizumab clinical 
program. 
10.22.2 Appendix 22: 
Management of diarrhea  
Stool sample collection has been 
amended to remove biomarker 
analysis to include: At the 
discretion of the Investigator, 
stool samples may be collected 
for local safety analysis. 
The language updated to 
clarify the actions necessary 
by the PI in case of severe 
diarrhea. 
10.22.4 Appendix 22: 
Management of Infusion 
Reactions or Hypersensitivity 
Reactions 
New appendix was added for the 
management of infusion reactions 
or hypersensitivity reactions 
The addition of management 
guidance for infusion reaction 
was requested by FDA for ITP 
phase 3 studies.  This is also 
added to MG studies to 
harmonize. 
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Section # and Name 
Description of Change 
Brief Rationale 
10.25 Appendix 25: Columbia-
Suicide Severity Rating Scale 
New appendix for C-SSRS 
(screening and last visit versions) 
has been included. 
Missing appendix 
 
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10.12 
Appendix 12: Quantitative Myasthenia Gravis Scale 
 
 
 
 
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10.13 
Appendix 13: Myasthenia Gravis-Composite scale 
 
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10.14 
Appendix 14: Myasthenia Gravis-Activities of Daily Living  
 
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10.15 
Appendix 15 Myasthenia Gravis Symptoms PRO 
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10.16 
Appendix 16: 5-Level European quality of life 5 dimensions 
 
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10.17 
Appendix 17: Myasthenia Gravis Impairment Index 
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10.18 
Appendix 18: Revised 15-item myasthenia gravis quality of life 
questionnaire 
 
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10.19 
Appendix 19: Tuberculosis questionnaire 
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10.20 
Appendix 20: Management of headaches, diarrhea, and 
infections and hypogammaglobulinemia 
10.20.1 
Management of headache 
Based on current available clinical data, headache is the most commonly reported adverse drug 
reaction in study participants treated with rozanolixizumab. Study participants should be well 
informed of this potential adverse drug reaction and should be instructed on how to manage it. 
Determination of the severity of headache will be consistent with National Cancer Institute 
Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 (Section 10.3). Severe 
headache is defined as severe pain limiting self-care activities of daily living (ADL). Self-care 
activities of daily living (ADL) refers to bathing, dressing and undressing, feeding self, using the 
toilet, and taking medications. 
In the event of headache Investigators will take medical history of previous headaches, 
concomitant medication, and co-morbidities (eg, asthma) in consideration. 
Study participants experiencing severe and/or serious headache will complete the Headache 
Questionnaire (Section 10.21, Appendix 21) daily until resolving or resolution (ie, if headache 
becomes nonserious, moderate or mild or completely resolved, whichever comes first). If the 
severe or serious headache is initially reported at a home visit or during a telephone call, the 
study participant should be reviewed at the study site as soon as is practically possible for further 
investigations. Study participants should be monitored for signs and symptoms suggestive of 
central nervous system involvement and evaluated immediately if other causes (eg, meningitis, 
intracranial bleeding) are suspected. Further neurological workup may be performed (if 
indicated) at the discretion of the Investigator or the treating physician and may include, a 
computed tomography scan, magnetic resonance imaging and/or a lumbar puncture for cerebral 
spinal fluid collection. In addition, samples for exploratory safety biomarkers should be collected 
for study participants experiencing severe headache or serious headaches when possible. These 
investigations will be performed to further understand the mechanism of headaches in the study 
participants. 
Treatments must be permanently discontinued if a study participant has a serious headache that is 
considered related to the IMP in the opinion of the Investigator. Treatment should be temporarily 
put on hold if a study participant experiences a severe AE of headache that is considered related 
to the study medication in the opinion of the Investigator, and is not resolved prior to the next 
scheduled study treatment. If deemed appropriate by the Investigator and agreed upon by the 
study participant and the sponsor, the study treatment can resume upon the resolution of the 
severe headache event. The benefit and risk of the treatment should be carefully considered prior 
to reinitiating the IMP. However, if a treatment related severe headache reoccurs, the treatment 
must be permanently discontinued. 
Headaches will be treated as clinically indicated according to local guidelines. It is recommended 
to start the analgesic at early onset of headache. At the discretion of the Investigator, study 
participants may carry a recommended analgesic with the instruction for frequency and dosage 
provided. Study participants experiencing any treatment related headache will be followed until 
resolution of the event. 
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Prophylactic treatment of headaches may be permitted for study participants who have 
experienced previous episodes of treatment related moderate or severe headache after discussion 
with the medical monitor. The benefit risk of continuing treatment with IMP and chronic 
prophylactic with analgesics must be carefully evaluated by the Investigator. 
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10.20.2 
Management of diarrhea 
Severe (Grade 3) diarrhea is defined as an increase of ≥7 stools per day or hospitalization for 
management of diarrhea or limiting self-care ADL. Determination of the severity of diarrhea will 
be consistent with CTCAE version 5.0 (Section 10.3). 
Diarrhea will be treated as clinically indicated according to the local guidelines. 
Stool samples may be collected for stool analysis to rule out infection for study participants 
reporting severe diarrhea. Stool sampling will be as clinically indicated in the opinion of the 
Investigator and assessed per local guidance. In addition, collection of blood samples for 
assessment of exploratory safety biomarkers is required for study participants with severe GI 
disturbances including diarrhea. 
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10.20.3 
Management of infections and hypogammaglobulinemia 
Study participants who have signs or symptoms of any infection should be monitored closely and 
managed according to local guidelines. This may include tests for specific organisms if clinically 
indicated. 
Study participants MUST discontinue IMP AND move into the SFU Period if any of the 
following events occur: Study participant has a serious infective episode requiring 
hospitalization or IV antibiotic therapy (ie, bacteremia or sepsis, bacterial meningitis, 
osteomyelitis or septic arthritis, bacterial pneumonia, or visceral abscess).  
To maintain the study integrity, IgG level will remain blinded to the study sites and the UCB 
study team for the first 4 weeks of the study. To ensure patient safety, serum IgG level will be 
monitored by an independent medical monitor external to UCB including signs and symptoms of 
infection and associated laboratory parameters. The IMP may be temporarily discontinued as 
requested by the independent medical monitor when deemed appropriate. 
In the event of a non-serious infection, the Benefit-Risk of continuing treatment with IMP must 
be carefully evaluated by the Investigator in collaboration with the medical monitor. Treatment 
may be temporarily discontinued for the study participant who develops a non-serious persisting 
or recurrent infection with a serum total IgG level between ≥1g/L and <2g/L. Upon resolution of 
infection and the IgGs returning to the level of ≥2g/L, the study participant may be allowed to 
resume treatment with the IMP. Ad hoc assessment can be performed to monitor the recovery of 
IgG levels. 
Treatment MUST be temporarily discontinued for the study participant who develops an event of 
hypogammaglobulinemia with a serum total IgG of <1g/L irrespective of infection. When the 
IgG level reaches ≥2g/L, the study participant may be allowed to continue treatment with IMP. 
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10.20.4 
Management of infusion reactions or hypersensitivity reactions 
Study participants must be closely monitored for reactions during and after the study treatment 
administration period. Standard precautions must be taken for the study participants with regard 
to SC infusion complications. Suggested management guidelines for infusion-related reactions 
and anaphylaxis at the study site are provided in Table 10-4. Definitions of mild, moderate, and 
severe events will be consistent with CTCAE version 5.0 (Section 10.3). Nurses administering 
the IMP at home should follow their own management guidelines, which should be reviewed and 
endorsed by the Investigator prior to first home administration. 
Table 10-4: Suggested management guidelines for infusion reactions or 
anaphylaxis 
Type of reaction 
Suggested action 
Acute – Mild 
Grade 1 
Monitor vital signs every 10 min. 
If the reaction worsens to Grade 2, follow the instruction below.   
Acute – Moderate 
Grade 2 
Interrupt/hold infusion temporarily to further assess and initiate 
treatment if necessary.  
Consider the use of IV fluid and antihistamine IV/IM. 
Consider administering paracetamol or NSAIDs. 
Monitor vital signs initially every 5 min. 
If the reaction improves and upon further assessment it is clear that the 
event is not an anaphylaxis, restart the infusion cautiously.  Continue 
monitor vital signs every 5 minutes. 
If reaction recurs or worsens to Grade 3, discontinue infusion. 
Acute – Severe 
Grade 3 or 
anaphylaxis 
Discontinue IMP infusion permanently. 
Alert crash team. 
Maintain airway; ensure oxygen is available. 
Administer: 
− Antihistamine IV/IM, corticosteroids IV, epinephrine IM, and 
IV fluids as appropriate. 
− Monitor vital signs every 2 min. 
− Hospitalize, if condition not improving or worsens  
− Monitor patient until symptoms resolve. 
CTCAE=Common Terminology Criteria for Adverse Events; IM=intramuscular; IMP=investigational medicinal 
product; IV=intravenous(ly); NSAID=nonsteroidal anti-inflammatory drug 
Note: Management criteria were adapted from the CTCAE v5.0 (National Cancer Institute, 2017). 
Conditions in which anaphylaxis is likely should be diagnosed using Sampson’s Criteria 
(Sampson et al, 2006) as described in (Appendix 22, Section 10.22). The infusion must be 
discontinued immediately, and emergency resuscitation measures implemented. 
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If an infusion-related reaction or anaphylaxis occurs, a blood sample will be collected from the 
study participant as soon as possible, while the event is ongoing, to investigate the nature of the 
reaction as per Schedule of Activities (Section 1.3). 
Samples for serum complement (C3, C4), serum cytokines, and plasma complement (C3a, C5a) 
should be collected as specified in the Schedule of Activities (Section 1.3). Additional tests such 
as IgE levels, tryptase may be performed when there is a suspicion of Type I or III 
hypersensitivity reaction. The results of all monitoring, including laboratory testing, should be 
made available to the study site and Sponsor. 
 
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10.21 
Appendix 21: Headache questionnaire 
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10.22 
Appendix 22: Sampson Criteria Questionnaire 
Anaphylaxis is highly likely when any of the following 3 criteria is fulfilled (Sampson et al, 
2006): 
1. Acute onset of an illness (minutes to several hours) with involvement of the skin, mucosal 
tissue, or both (eg, generalized hives, pruritus or flushing, swollen lips-tongue-uvula) 
AND AT LEAST ONE OF THE FOLLOWING 
d. Respiratory compromise (eg, dyspnea, wheeze-bronchospasm, stridor, reduced peak 
expiratory flow [PEF], hypoxemia) 
e. Reduced BP or associated symptoms of end-organ dysfunction (eg, hypotonia [collapse], 
syncope, incontinence) 
2. Two or more of the following that occur rapidly after exposure to a likely allergen for that 
patient (minutes to several hours): 
a. Involvement of the skin-mucosal tissue (eg, generalized hives, itch-flush, swollen lips 
tongue-uvula) 
b. Respiratory compromise (eg, dyspnea, wheeze-bronchospasm, stridor, reduced PEF, 
hypoxemia) 
c. Reduced BP or associated symptoms (eg, hypotonia [collapse], syncope, incontinence) 
d. Persistent GI symptoms (eg, crampy abdominal pain, vomiting) 
Reduced BP after exposure to known allergen for that subject (minutes to several hours): 
Systolic BP of less than 90mmHg or greater than 30% decrease from the subject’s Baseline 
systolic BP value. 
 
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10.23 
Appendix 23: Columbia-Suicide Severity Rating Scale 
 
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11 
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SPONSOR DECLARATION 
I confirm that I have carefully read and understand this protocol and agree to conduct this 
clinical study as outlined in this protocol and according to current Good Clinical Practice. 
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Approval Signatures
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Document Number:
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Title:
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Approved Date:
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