A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Sponsor: UCB Biopharma SRL

UCB 
 
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Clinical Study Protocol Amendment 4 
Rozanolixizumab 
MG0003 
 
 
 
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A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-
CONTROLLED STUDY EVALUATING EFFICACY AND SAFETY 
OF ROZANOLIXIZUMAB IN ADULT PATIENTS WITH 
GENERALIZED MYASTHENIA GRAVIS 
PROTOCOL MG0003 AMENDMENT 4 
PHASE 3 
Short title: 
A Phase 3 study evaluating the safety and efficacy of rozanolixizumab in patients with 
myasthenia gravis. 
Sponsor: 
UCB Biopharma SRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Regulatory agency identifying number(s): 
Eudra CT Number: 
2019-000968-18 
IND Number: 
132407 
NCT Number: 
NCT03971422 
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 4 
23 Feb 2021 
Substantial 
Protocol Amendment 3 
29 Jul 2020 
Substantial 
Protocol Amendment 2a 
04 Mar 2020 
Substantial 
Protocol Amendment 1 
30 Oct 2019 
Substantial 
Original Protocol 
22 Feb 2019 
Not applicable 
a Protocol amendment 2 was an internally approved document, which was not implemented at the 
study sites or submitted to the regulatory authorities. Protocol amendment 3 included changes 
related to coronavirus disease 2019 (COVID-19) and incorporates protocol amendment 2 
(Section 10.11). 
Amendment 4 (23 Feb 2021) 
Overall Rationale for the Amendment 
The primary reasons for this protocol amendment are to 1) update the requirement for study 
participants receiving rescue therapy to be follow up through to the end of the Observation 
Period; 2) update the criteria for discontinuation due to other adverse events or medical 
conditions; and 3) remove the participant exit interview. Additional updates have been 
incorporated to provide further clarity on the protocol and/or to correct errors. 
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. 
Updated to provide clarity and 
remain consistent with remainder 
of protocol. 
1.1 Synopsis, Objectives 
and Endpoints 
3 Objectives and 
Endpoints 
An additional other efficacy 
endpoint specific to change from 
baseline in MD-ADL (excluding 
ocular items) has been included.  
Additional wording "any time up to 
and including" has been included 
for the other efficacy endpoint 
specific to minimal symptom 
expression.  
For other efficacy endpoints 
specific to MG Impairment Index, 
“Observation Period” has been 
removed.  
Updated as it was an omission 
from the previous protocol 
amendment. 
 
Updated as it was an omission 
from the previous protocol 
amendment. 
 
 
This PRO will not be assessed 
during the Observation Period.  
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Section # and Name 
Description of Change 
Brief Rationale 
1.1 Synopsis, Objectives 
and Endpoints 
3 Objectives and 
Endpoints 
Footnote a has been updated to 
remove "serious" as a level of 
severity for the triggered AEs. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
1.1 Synopsis  
4.1 Overall Design 
The following wording in reference 
to participants opting to receive 
rescue medication has been updated 
to include: 
"complete any remaining visits in 
the Observation Period and" 
Additionally, wording in reference 
to follow-up instructions after 
completion of rescue treatment has 
been removed.  
Updated to provide clarity and 
remain consistent with the study 
design. 
1.2 Schema  
Study schematic updated to remove 
the following wording: "+ 
additional bi-weekly visits up to 8 
weeks after last IVIg/PEX session." 
Updated to remain consistent with 
the study design. 
1.3 Schedule of activities 
For the following activity, "Call or 
enter IRT to register the visit", 
Visits 2 (Baseline), 3, 7, 11, 12, and 
13 have been removed.  
Footnote l has been updated to 
remove "serious" as a level of 
severity for the triggered AEs, and 
"at Visits 4, 5, 6, 8 and 9". 
Updated to correct an error from 
the previous protocol amendment. 
 
 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
2.3 Benefit/Risk 
Assessment 
Wording on potential risks 
associated with administration of 
rozanolixizumab has been updated. 
Updated to remove events that are 
no longer considered a potential 
risk across the Phase 3 
rozanolixizumab clinical program. 
5.1 Inclusion Criteria 
#5a: The MG-ADL score was 
updated to replace > with ≥. 
#8b (now 8c): Wording on female 
study participants of childbearing 
potential and use of a highly 
effective method of birth control 
has been removed. 
Updated to correct an error from 
the previous protocol amendment. 
Updated to for clarity and to 
remove repetitive information. 
5.2 Exclusion Criteria 
#13a (now 13b): Wording was 
slightly edited, and the following 
was removed: "history thereof in 
past 6 months prior to Visit 1." 
Table 5-1: A new biologic 
(inebulizumab) as well the 
treatment-free period were added. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
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Section # and Name 
Description of Change 
Brief Rationale 
#29 (now 29a): Update to replace 
family history with "current or 
medical." 
6.5.2 Prohibited 
concomitant treatments 
(medications and 
therapies) 
The following concomitant 
medication was added:  
Vinca alkaloids (vincristine, 
vinblastine) 
Updated for consistency and to 
correct an error from the previous 
protocol amendment. 
6.7 Treatment after the 
End of the Study 
The following wording in reference 
to participants opting to receive 
rescue medication has been updated 
to include: 
"complete any remaining visits in 
the Observation Period and" 
Additionally, wording in reference 
to participants considered for 
additional treatment with 
prophylactic antimicrobial therapy 
has been removed. 
Updated to provide clarity and 
remain consistent with the study 
design. 
7.1.3 Discontinuation due 
to other adverse events or 
medical conditions 
Criterion #5 was updated to include 
recurrence severe AE of headache.  
New criterion (#7) relating to 
potential drug induced liver injury 
has been included.  
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
8 Study Assessment and 
Procedures 
The following wording has been 
removed: "follow study participants 
who experience symptom 
worsening during the 8-week 
observation period and opt to 
receive IVIg or PEX as rescue 
therapy." 
A specific reason for an 
unscheduled visit is not required, 
as it can be conducted at the 
discretion of the Investigator.  
8.1.4.1 MG-Activities of 
Daily Living 
Additional wording specifically 
stating which items are considered 
ocular has been included.  
Updated to provide clarity in 
relation to inclusion criteria #5 
and Section 8.1.4.1. 
8.2.6 Assessment and 
management of TB and 
TB risk factors 
TB assessment by chest x-ray: 
Version year has been removed and 
replaced with "current." 
Updated to ensure information 
related to TB assessment by chest 
x-ray is aligned with the up-to-
date version of the WHO Global 
Tuberculosis Report. 
8.2.6 Assessment and 
management of TB and 
TB risk factors 
TB assessment by IGRA: 
New wording on indeterminate 
IGRA test results and an evaluation 
by a TB specialist relating to study 
participant eligibility have been 
included.  
Updated to provide clarity on the 
requirements for a study 
participant’s eligibility. 
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Section # and Name 
Description of Change 
Brief Rationale 
8.3.3 Follow-up of AEs 
and SAEs 
Adverse events of special 
monitoring have been added. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
8.3.6 Adverse events of 
special interest 
The definition for Hy’s Law has 
been updated. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
8.5 Treatment of 
Overdose 
In relation to #3, autoantibodies 
have been replaced with 
"antibodies." 
Updated to clarify all antibodies 
are measured.  
8.9 Biomarkers 
"Serious" has been removed as a 
level of severity for an AESM of 
headache.  
"Severe" has been added as a level 
of severity for an AESM of GI 
disorders. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
8.11 Participant exit 
interview 
This section has been removed. 
The optional participant exit 
interview will no longer be 
conducted.  
9.1 Definition of Analysis 
Sets 
The Randomization Set replaces the 
Full Analysis Set as the primary 
analysis set for efficacy analyses.  
Updated to be consistent with 
estimands table (Table 9-1). 
9.3 Estimands 
Wording for handling of 
intercurrent events due to treatment 
discontinuation has been updated. 
Updated to provide further 
clarification. 
9.3 Estimands, Table 9-1 
The PLS (analysis) for the MG-
ADL objective (secondary) was 
updated: difference in proportion 
has been replace by "odds ratio." 
Updated to clarify the population 
summary being estimated. 
10.2 Appendix 2: Clinical 
laboratory test 
The following parameter, glucose, 
has been updated to include 
"fasting state, preferred." 
High-density lipoprotein, total 
cholesterol and triglycerides have 
been added. 
Updated to clarify the specifics 
for fasting glucose. 
 
Updated to include lipid panel 
assessments. 
10.4 Appendix 4: 
Contraceptive Guidance 
and Collection of 
Pregnancy Information 
Contraception guidance for male 
participants: Wording specific to 
the agreed use of a male condom 
plus partner use of a contraceptive 
method with a failure rate of <1% 
per year has been removed. 
Table 10-1: Contraception guidance 
for female participants and 
Updated to provide further clarity 
on the use of contraception during 
the study.  
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Section # and Name 
Description of Change 
Brief Rationale 
vasectomized partner has been 
updated. 
10.11 Appendix 11: 
Protocol Amendment 
History 
Details of the previous amendment 
(protocol amendment 3) have been 
added.  
General update. 
10.20 Appendix 20: MG 
Impairment Index 
Current copy of MG Impairment 
Index has been updated to include a 
missing page (page containing 
questions 7 to 12).  
Updated to correct an error from 
the previous protocol amendment.  
10.23 Appendix 23: 
Management of Headache 
The following wording has been 
included: "The questionnaire 
should be administered via an 
interview with the study 
participant."  
Updated to provide further clarity 
on conducting the headache 
questionnaire. 
 
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SERIOUS ADVERSE EVENT REPORTING 
Serious adverse event reporting (24h) 
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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TABLE OF CONTENTS 
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE ...................................... 2 
SERIOUS ADVERSE EVENT REPORTING .......................................................................... 7 
1 
PROTOCOL SUMMARY ............................................................................................... 13 
1.1 Synopsis ........................................................................................................................... 13 
1.2 Schema ............................................................................................................................. 21 
1.3 Schedule of Activities ...................................................................................................... 22 
1.4 Schedule of Activities (sub-study) ................................................................................... 26 
2 
INTRODUCTION ........................................................................................................... 27 
2.1 Study Rationale ................................................................................................................ 27 
2.2 Background ...................................................................................................................... 28 
2.3 Benefit/Risk Assessment ................................................................................................. 29 
3 
OBJECTIVES AND ENDPOINTS ................................................................................. 30 
4 
STUDY DESIGN ............................................................................................................. 35 
4.1 Overall Design ................................................................................................................. 35 
4.2 Scientific Rationale for Study Design .............................................................................. 37 
4.3 Justification for Dose ....................................................................................................... 37 
4.4 End of Study definition .................................................................................................... 39 
5 
STUDY POPULATION .................................................................................................. 39 
5.1 Inclusion Criteria ............................................................................................................. 39 
5.2 Exclusion Criteria ............................................................................................................ 40 
5.3 Lifestyle Restrictions ....................................................................................................... 44 
5.4 Screen Failures ................................................................................................................. 44 
6 
STUDY TREATMENTS ................................................................................................. 45 
6.1 Treatments Administered ................................................................................................. 45 
6.2 Preparation, Handling, Storage, and Accountability requirements .................................. 45 
6.2.1 
Drug accountability ............................................................................................. 46 
6.3 Measures to Minimize Bias: Randomization and Blinding ............................................. 46 
6.3.1 
Procedures for maintaining and breaking the treatment blind ............................. 47 
6.3.1.1 
Maintenance of study treatment blind ....................................................... 47 
6.3.1.2 
Breaking the treatment blind in an emergency situation ........................... 48 
6.4 Treatment Compliance ..................................................................................................... 48 
6.5 Concomitant Medication(s)/Treatment(s) ........................................................................ 48 
6.5.1 
Permitted concomitant treatments (medications and therapies) .......................... 48 
6.5.2 
Prohibited concomitant treatments (medications and therapies) ......................... 49 
6.5.3 
Treatments specific to NMJ interference ............................................................. 49 
6.5.4 
Rescue therapy ..................................................................................................... 50 
6.6 Dose Modification ........................................................................................................... 50 
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6.7 Treatment after the End of the Study ............................................................................... 50 
7 
DISCONTINUATION OF STUDY MEDICATION AND PARTICIPANT 
DISCONTINUATION/WITHDRAWAL ........................................................................ 51 
7.1 Discontinuation of Study Medication .............................................................................. 51 
7.1.1 
Liver Chemistry Stopping Criteria ...................................................................... 51 
7.1.2 
QTc Stopping Criteria .......................................................................................... 52 
7.1.3 
Discontinuation due to other adverse events or medical conditions .................... 53 
7.1.4 
Temporary IMP discontinuation .......................................................................... 53 
7.2 Participant Discontinuation/Withdrawal from the study ................................................. 54 
7.3 Lost to Follow up ............................................................................................................. 55 
8 
STUDY ASSESSMENTS AND PROCEDURES ........................................................... 55 
8.1 Efficacy Assessments ....................................................................................................... 57 
8.1.1 
MGFA Classification ........................................................................................... 57 
8.1.2 
Quantitative Myasthenia Gravis scale ................................................................. 57 
8.1.3 
MG-Composite scale ........................................................................................... 57 
8.1.4 
Patient-reported outcomes ................................................................................... 57 
8.1.4.1 
MG-Activities of Daily Living .................................................................. 58 
8.1.4.2 
MG Symptoms PRO .................................................................................. 58 
8.1.4.3 
Patient Global Impression of Severity ....................................................... 59 
8.1.4.4 
Patient Global Impression of Change ........................................................ 59 
8.1.4.5 
EQ-5D-5L .................................................................................................. 59 
8.1.4.6 
MG Impairment Index ............................................................................... 59 
8.1.4.7 
MG-QOL15r .............................................................................................. 60 
8.2 Safety Assessments .......................................................................................................... 60 
8.2.1 
Physical examination ........................................................................................... 60 
Full physical examination .............................................................................................. 60 
Brief physical examination ............................................................................................ 60 
8.2.2 
Vital signs ............................................................................................................ 61 
8.2.3 
Electrocardiograms .............................................................................................. 61 
8.2.4 
Clinical safety laboratory assessments ................................................................ 61 
8.2.5 
Suicidal risk monitoring ...................................................................................... 61 
8.2.6 
Assessment and management of TB and TB risk factors .................................... 62 
8.3 Adverse Events ................................................................................................................ 65 
8.3.1 
Time period and frequency for collecting AE and SAE information .................. 65 
8.3.2 
Method of detecting AEs and SAEs .................................................................... 65 
8.3.3 
Follow-up of AEs and SAEs ................................................................................ 65 
8.3.4 
Regulatory reporting requirements for SAEs ...................................................... 66 
8.3.5 
Pregnancy ............................................................................................................ 66 
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8.3.6 
Adverse events of special interest ........................................................................ 66 
8.3.7 
Adverse events of special monitoring .................................................................. 67 
8.3.8 
Treatment-emergent adverse events .................................................................... 67 
8.4 Safety Signal Detection .................................................................................................... 67 
8.5 Treatment of Overdose..................................................................................................... 67 
8.6 Pharmacokinetics and Anti-drug Antibodies ................................................................... 68 
8.7 Pharmacodynamics .......................................................................................................... 68 
8.8 Genetics ............................................................................................................................ 69 
8.9 Biomarkers ....................................................................................................................... 69 
8.9.1 
Immunology ......................................................................................................... 69 
8.10 Medical Resource Utilization and Health Economics ..................................................... 70 
9 
STATISTICAL CONSIDERATIONS ............................................................................. 70 
9.1 Definition of Analysis Sets .............................................................................................. 70 
9.2 General Statistical Considerations ................................................................................... 70 
9.3 Estimands ......................................................................................................................... 71 
9.4 Planned Efficacy/Outcome Analyses ............................................................................... 80 
9.4.1 
Analysis of the primary efficacy endpoint ........................................................... 80 
9.4.2 
Analysis of secondary and other efficacy endpoints ........................................... 81 
9.5 Planned Safety and other Analyses .................................................................................. 82 
9.5.1 
Safety analyses ..................................................................................................... 82 
9.5.2 
Other analyses ...................................................................................................... 82 
9.5.2.1 
Pharmacokinetic analyses .......................................................................... 82 
9.5.2.2 
Anti-drug antibodies analyses ................................................................... 82 
9.6 Handling of protocol deviations ....................................................................................... 83 
9.7 Handling of dropouts or missing data .............................................................................. 83 
9.8 Planned interim analysis and data monitoring ................................................................. 83 
9.8.1 
Interim analysis .................................................................................................... 83 
9.8.2 
Periodic Data Reviews ......................................................................................... 84 
9.8.3 
Data monitoring ................................................................................................... 84 
9.9 Determination of sample size ........................................................................................... 85 
10 SUPPORTING DOCUMENTATION AND OPERATIONAL CONSIDERATIONS... 87 
10.1 Appendix 1: Regulatory, ethical, and study oversight considerations ............................. 87 
10.1.1 
Regulatory and ethical considerations ................................................................. 87 
10.1.2 
Financial disclosure ............................................................................................. 87 
10.1.3 
Informed consent process .................................................................................... 88 
10.1.4 
Data protection ..................................................................................................... 88 
10.1.5 
Committees structure ........................................................................................... 89 
10.1.6 
Data quality assurance ......................................................................................... 89 
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10.1.6.1 
Electronic Case Report Form completion ................................................. 89 
10.1.6.2 
Apps ........................................................................................................... 90 
10.1.7 
Source documents ................................................................................................ 90 
10.1.8 
Study and Site Closure ......................................................................................... 90 
10.1.9 
Publication Policy ................................................................................................ 91 
10.2 Appendix 2: Clinical Laboratory Tests ............................................................................ 92 
10.3 Appendix 3: Adverse Events – Definitions and Procedures for Recording, Evaluating, 
Follow-up, and Reporting ................................................................................................ 94 
10.3.1 
Definition of AE .................................................................................................. 94 
10.3.2 
Definition of SAE ................................................................................................ 95 
10.3.3 
Recording and Follow-Up of AE and/or SAE ..................................................... 96 
10.3.4 
Reporting of SAEs ............................................................................................... 98 
10.4 Appendix 4: Contraceptive Guidance and Collection of Pregnancy Information ......... 100 
Definitions 100 
Contraception Guidance .............................................................................................. 100 
Pregnancy testing ......................................................................................................... 102 
10.5 Appendix 5: Genetics ..................................................................................................... 104 
10.6 Appendix 6: Liver Safety – Suggested Actions and Follow-up Assessments ............... 105 
Table 10‒2:  Liver Chemistry Stopping Criteria and Follow-Up assessments ............... 105 
10.7 Appendix 7: Medical Device Incidents – Definition and Procedures for Recording, 
Evaluating, Follow-up, and reporting ............................................................................ 108 
10.8 Appendix 8: Rapid Alert Procedures ............................................................................. 109 
10.9 Appendix 9: Country-specific Requirements ................................................................. 110 
10.10 
Appendix 10: Abbreviations and Trademarks ..................................................... 111 
10.11 
Appendix 11: Protocol Amendment History ....................................................... 115 
10.12 
Appendix 12: MGFA Classification .................................................................... 142 
10.13 
Appendix 13: Quantitative Myasthenia Gravis Scale .......................................... 143 
10.14 
Appendix 14: MG Composite Scale .................................................................... 152 
10.15 
Appendix 15: MG-Activities of Daily Living ..................................................... 153 
10.16 
Appendix 16: MG Symptoms PRO ..................................................................... 154 
10.17 
Appendix 17: Patient Global Impression of Severity .......................................... 157 
10.18 
Appendix 18: Patient Global Impression of Change ........................................... 158 
10.19 
Appendix 19: EQ-5D-5L ..................................................................................... 159 
10.20 
Appendix 20: MG Impairment Index .................................................................. 161 
10.21 
Appendix 21: MG-QOL15r ................................................................................. 170 
10.22 
Appendix 22: Tuberculosis Questionnaire........................................................... 171 
10.23 
Appendix 23: Management of Headache ............................................................. 173 
10.24 
Appendix 24: Management of Diarrhea .............................................................. 174 
10.25 
Appendix 25: Management of Infections and Hypogammaglobulinemia ........... 175 
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10.26 
Appendix 26: Management of Infusion Reactions or Hypersensitivity Reactions176 
10.27 
Appendix 27: Headache Questionnaire ............................................................... 178 
10.28 
Appendix 28: Sampson Criteria Questionnaire ................................................... 182 
10.29 
Appendix 29: Columbia-Suicide Severity Rating Scale ...................................... 183 
11 REFERENCES .............................................................................................................. 187 
SPONSOR DECLARATION ................................................................................................ 188 
 
 LIST OF TABLES 
Table 3-1: 
Study Objectives and Endpoints ...................................................................... 30 
Table 5-1: 
Treatment-free Period for Exclusionary Immunosuppressants, Biologics, 
and Other Therapies Prior to Baseline (Visit 2)............................................... 41 
Table 6-1: 
Permitted Concomitant Treatments ................................................................. 48 
Table 9-1: 
Estimand Details and Attributes ...................................................................... 72 
Table 9-2: 
Simulation results from ADDPLAN MC (x100,000 simulations per 
scenario) with a capped maximum sample size of 80 per arm ........................ 86 
Table 10-1: 
Highly Effective Contraceptive Methodsa ..................................................... 101 
Table 10‒2:  Liver Chemistry Stopping Criteria and Follow-Up assessments ................... 105 
Table 10‒3: 
Liver chemistry increased monitoring criteria with continued study 
medication ...................................................................................................... 107 
Table 10-4: 
Suggested management guidelines for infusion reactions or anaphylaxis..... 176 
 
LIST OF FIGURES 
Figure 1-1: 
Study Schematic............................................................................................... 21 
Figure 7-1: 
Liver Chemistry Stopping Criteria and Increased Monitoring Algorithm ....... 51 
Figure 7-2: 
Liver Chemistry Increased Monitoring Algorithm with Continued Study 
Intervention for Participants with ALT ≥3xULN but ≤8ULN ......................... 52 
 
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1 
PROTOCOL SUMMARY 
1.1 
Synopsis  
Protocol Title:  
A Phase 3, randomized, double-blind, placebo-controlled study evaluating efficacy and safety of 
rozanolixizumab in adult patients with generalized myasthenia gravis. 
Short Title: 
A Phase 3 study evaluating the safety and efficacy of rozanolixizumab in patients with 
myasthenia gravis. 
Rationale: 
Myasthenia gravis (MG) is a serious, sometimes life threatening, debilitating condition 
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 of a variety of therapeutic agents including cholinesterase inhibitors, 
immunomodulators, corticosteroid, biologics, high dose intravenous immunoglobulin (IVIg), 
plasmapheresis or immunoadsorption, there remains a need for a safe and effective treatment 
devoid of significant side effects to conveniently treat patients with MG. 
By blocking the activity of neonatal Fc receptor (FcRn), rozanolixizumab accelerates the 
catabolism of antibodies and reduces the serum IgG concentration, including pathogenic IgG in 
MG patients, thus offering a potentially safe, effective, and convenient alternative to existing 
treatments. This Phase 3 study will provide the required data to establish the safety and efficacy 
of rozanolixizumab in anti-AChR or anti-MuSK autoantibody-positive patients with generalized 
MG who experience moderate to severe symptoms and are being considered for additional 
treatment with IVIg or plasma exchange (PEX). 
Objectives and Endpoints 
Objectives 
Endpoints 
Primary 
• To demonstrate the clinical efficacy of 
rozanolixizumab in patients with 
generalized MG 
The primary efficacy endpoint is:  
• Change from Baseline to Day 43 (Visit 
10) in MG-activities of daily living (ADL) 
score 
The secondary efficacy endpoints are:  
• MG-ADL responder (≥2.0 points 
improvement from Baseline) at Day 43 
(Visit 10) 
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• Change from Baseline to Day 43 (Visit 
10) in the MG-Composite (MG-C) score 
• Change from Baseline to Day 43 (Visit 
10) in quantitative myasthenia gravis 
(QMG) score 
• Change from Baseline to Day 43 (Visit 
10) in the MG Symptoms patient-reported 
outcome (PRO) ‘Muscle Weakness 
Fatigability’ score 
• Change from Baseline to Day 43 (Visit 
10) in the MG Symptoms PRO ‘Physical 
Fatigue’ score 
• Change from Baseline to Day 43 (Visit 
10) in the MG Symptoms PRO ‘Bulbar 
Symptoms’ score 
The other efficacy endpoints are:  
• Use of rescue therapy due to worsening 
(IVIg, PEX) 
• Time to first rescue therapy 
• Time to MG-ADL response (≥2.0 points 
improvement from Baseline) 
• Change from Baseline in QMG at each 
scheduled assessment during Treatment 
and Observation Periods 
• QMG responder (≥3.0 points 
improvement from Baseline) 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 
• MG-C responder (≥5.0 points 
improvement from Baseline) at each 
scheduled assessment during Treatment 
and Observation Periods 
• Change from Baseline in MG-ADL at 
each scheduled assessment during 
Treatment and Observation Periods 
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• MG-ADL responder (≥2.0 points 
improvement from Baseline) at each 
scheduled assessment during Treatment 
and Observation Periods  
• 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 Patient Global 
Impressions of Severity (PGI-S) at each 
scheduled assessment during the 
Treatment and Observation Periods 
• Patient Global Impressions of Change 
(PGI-C) at each scheduled assessment 
during Treatment and Observation Periods 
• Change from Baseline in MG-ADL 
(excluding ocular items) at each 
scheduled assessment during Treatment 
and Observation Periods 
• Change from Baseline in QMG 
(excluding ocular items) at each 
scheduled assessment during Treatment 
and Observation Periods 
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• Change from Baseline in MG-C score 
(excluding ocular items) at each 
scheduled assessment during Treatment 
and Observation Periods 
• Change from Baseline to Day 43 (Visit 
10) in Myasthenia Gravis Quality of Life 
(MG-QOL) 15r 
• Change from Baseline to Day 43 (Visit 
10) in EuroQol(EQ)-5D-5L  
• Minimal symptom expression (MG-ADL 
score of 0 or 1) at any time up to and 
including Day 43 (Visit 10) 
• Change from Baseline in MG Impairment 
Index (MGII) scores at each scheduled 
assessment during Treatment Period 
• Change from Baseline in MGII ocular 
sub-scores at each scheduled assessment 
during Treatment Period 
• Change from Baseline in MGII 
generalized domain sub-scores at each 
scheduled assessment during Treatment 
Period 
Secondary 
• To assess safety and tolerability of 
rozanolixizumab in MG patients 
The secondary safety endpoints are:  
• Occurrence of treatment-emergent 
adverse events (TEAEs) 
• TEAEs leading to withdrawal of 
investigational medicinal product (IMP) 
The other safety endpoints are: 
• Occurrence of adverse event of special 
monitoring (AESM) 
• Vital sign changes from Baseline (systolic 
and diastolic blood pressure [BP] and 
pulse rate at each scheduled assessment 
during Treatment and Observation 
Periods) 
• 12-lead electrocardiogram (ECG) change 
from Baseline at each scheduled 
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assessment during Treatment and 
Observation Periods 
• Laboratory changes from Baseline at each 
scheduled assessment during Treatment 
and Observation Periods (hematology, 
clinical chemistry, and urinalysis)  
• Suicidality as measured by the Columbia 
Suicide Severity Rating Scale (C-SSRS) 
Other 
• To assess pharmacokinetic (PK) 
characteristics of rozanolixizumab 
• Plasma concentration of rozanolixizumab 
at each scheduled assessment during the 
Treatment and Observation Periods 
• To assess the pharmacodynamics (PD) 
effects of rozanolixizumab on IgG, 
disease-specific autoantibodies 
• Change (absolute and percentage) from 
Baseline in MG-specific autoantibodies at 
each scheduled assessment during 
Treatment and Observation Periods  
• Change (absolute and percentage) from 
Baseline in serum total IgG and IgG 
subclasses concentrations at each 
scheduled assessment during Treatment 
and Observation Periods 
• To evaluate the emergence and incidence 
of anti-drug antibody (ADA) and impact 
on PK and PD 
• Anti-drug antibody at each scheduled 
assessment during the Treatment Period 
and Observation Periods 
• To evaluate the effects of 
rozanolixizumab on the concentration of 
total protein, 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 Perioda 
• Change from Baseline in serum 
immunoglobulin concentrations (total 
IgA, IgE, and IgM) at each scheduled 
assessment during Treatment and 
Observation Periods 
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• 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 
• To assess the effect of rozanolixizumab 
on exploratory biomarkers and protein 
expression, and explore the relationship 
between protein, and metabolite 
biomarkers and cause, progression, and 
appropriate treatment of MG 
• Exploratory biomarkers may be measured 
to evaluate the effect of rozanolixizumab. 
• Proteins, and metabolites changes that 
may be measured to understand the cause, 
progression, and appropriate treatment of 
MG 
a To be performed for study participants with severe headache and/or infusion reaction or hypersensitivity reaction. 
Other exploratory safety biomarkers may be assessed. 
Overall Design 
This is a Phase 3 study of rozanolixizumab in anti-AChR or anti-MuSK autoantibody-positive 
patients with generalized MG who experience moderate to severe symptoms and are being 
considered for additional treatment such as IVIg or PEX. The primary objective of the study is to 
demonstrate the clinical efficacy of rozanolixizumab in patients with generalized MG. 
Participants have the opportunity to rollover into an OLE study, as described below. The OLE 
study, MG0004 (52-week chronic treatment) will be replaced by MG0007, which consists of 
6-week treatment cycles 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. In the event a study participant has already started MG0004, a minimum 
treatment duration of 6 visits (if IMP treatment is held 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) must be completed prior to moving into 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 discontinued MG0004 and had the opportunity to 
rollover into MG0007. 
The study is composed of a Screening Period of up to 4 weeks, followed by a 6-week double-
blind Treatment Period and a blinded Observation Period of 8 weeks:  
1. All study participants who complete the 6-week Treatment Period will roll over into an 
8-week Observation Period. 
2. All eligible study participants who complete the Observation Period will be invited to be re-
randomized into an open-label extension (OLE) study. 
3. Study participants who experience disease worsening during the 6-week Treatment Period 
(eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between two 
consecutive visits) may be considered for rescue therapy (IVIg or PEX) at the discretion of 
the Investigator. 
4. Study participants who need rescue therapy during the 6-week Treatment Period will receive 
IVIg or PEX and complete any subsequent visit(s). No further infusions of IMP in MG0003 
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will be administered after initiation of rescue therapy. Once the participant completes the 
premature end of treatment (PEOT) Visit (Visit 10), he or she will move into the 8-week 
Observation Period.  
5. Study participants who complete the Treatment Period and require initiation of rescue 
therapy after they start the 8-week Observation Period, may either opt to receive IVIg or PEX 
or complete the End of Study (EOS) visit and immediately roll over into an OLE study where 
the participant will receive rozanolixizumab. Study participants who opt to receive IVIg or 
PEX will complete any remaining visits in the Observation Period and will not be invited to 
join an OLE study. 
6. Study participants who did not complete the 6-week Treatment Period because they needed 
rescue therapy and require initiation of a second course of rescue therapy while in the 8-week 
Observation Period can roll over to an OLE study if a minimum of 2 weeks have lapsed since 
completion of the last IVIg or PEX session. The EOS visit must be completed before 
enrolling into an OLE study. Alternatively, they can be treated with IVIg or PEX. Study 
participants who opt to receive IVIg or PEX will  complete any remaining visits in the 
Observation Period and will not be invited to join an OLE study.  
7. Study participants who discontinue study medication for any reason other than requiring 
rescue therapy will not be eligible for enrollment into an OLE study. 
 
Number of Participants 
A formal interim analysis will be conducted when approximately
 eligible study participants 
have been treated and are evaluable for the primary endpoint, ie, approximately 
 study 
participants per dose group. If the study is not stopped for futility after the interim analysis, then 
depending upon the selection of one, or two, of the doses after the interim analysis and the 
calculation of conditional power, a further 
 eligible study participants will be randomized 
into the study. Thus, the total sample size of the study could range between 
 and 
study 
participants if the study is not futile (for sample size determination, see Section 9.9). 
Treatment Groups and Duration 
The maximum duration of the study per study participant will be up to 18 weeks, consisting of a 
Screening Period (1 to 28 days to account for central laboratory turn-around time), a Treatment 
Period (6 weeks), and an Observation Period (8 weeks). 
Fixed unit doses across body weight tiers and study arms will be employed.  
The placebo arm will be 0.9% sodium chloride aqueous solution (physiological saline, 
preservative free) for subcutaneous (sc) administration. 
Two sc treatment arms will assume fixed unit doses stratified on weight tiers as described below:  
Treatment Arm 1 (rozanolixizumab) – equivalent to approximately 
 
• Bodyweight <50kg: dose to be administered 
 
• Bodyweight ≥50kg and <70kg: dose to be administered 
 
• Bodyweight ≥70kg and <100kg: dose to be administered 
 
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• Bodyweight ≥100kg; dose to be administered 
  
Treatment Arm 2 (rozanolixizumab) – equivalent to approximately 
  
• 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 
  
An independent Data Monitoring Committee (IDMC) will be formed to monitor the ongoing 
safety and efficacy of the study. Futility and samples size adjustment will also be reviewed and 
assessed by the IDMC during a formal interim analysis at the end of Stage 1, when
 enrolled 
study participants are evaluable for the primary endpoint. Approximately 3 periodic data reviews 
(in addition to the futility analysis mentioned above) will be performed: the first periodic data 
review will be performed when approximately 
study participants have completed the 6-week 
Treatment Period; the second periodic data review will be conducted after approximately
 
study participants have completed the 6-week Treatment Period (ad hoc as needed); and a third 
review will be conducted after approximately 
 study participants (dependent on the 
outcome of the interim analysis) have completed the 6-week Treatment Period. The timing of 
any further data reviews will be decided by the IDMC in conjunction with sponsor. Further 
details of the IDMC will be provided in a DMC charter. 
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1.2 
Schema 
A schematic diagram of the study is provided in Figure 1-1. 
Figure 1-1: Study Schematic  
 
 
IVIg=intravenous immunoglobulin G; OLE=open-label extension; PEX=plasma exchange; 
Rozimab=rozanolixizumab 
The sample size for Stage 1 of the study is
 (approximately 
study participants per arm). 
Provided the study is not stopped for futility at the end of Stage 1, a further 
 study 
participants will be enrolled (See Section 9.8 for details) and the total sample size for this study 
will range between 
 study participants.
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1.3 
Schedule of Activities 
Procedure 
Screening 
Treatment Period 
Observation Period  
Visit 
V1 
V2 
(BL) 
V3a 
V4 
V5 
V6 
V7a 
V8 
V9 
V10 
(EOT or 
PEOT) 
V11b 
V12 
V13b 
V14 
(EOS)  
Day (days)c  
-28 to -1 
1 
3(±1)  
8(±2)  
15(±2) 
22(±2) 
24(±1)  29(±2) 
36(±2) 
43(±2) 
57(±3) 
71(±4) 
85(±3) 
99(±4) 
Written informed consent  
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Optional PK sub-study consentd 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic and Baselinee 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Verification of 
inclusion/exclusion criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Prior and concomitant 
medications and medical 
procedures 
X 
X 
 
X 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
General medical history 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Psychiatric history/C-SSRS 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Query for suicidalityf  
 
X 
 
X 
X 
X 
 
X 
X 
X 
 
X 
 
X  
Chest X-rayg  
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
IGRA TB testh 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
TB Signs and Symptoms 
questionnaire 
X 
X 
 
 
 
 
 
 
 
X 
 
 
 
X 
12-lead ECG 
X 
X 
 
X 
 
 
 
 
 
X 
 
 
 
X 
Full physical examination 
X 
X 
 
 
 
 
 
 
 
X 
 
X 
 
X 
Brief physical examination 
 
 
 
X 
X 
X 
 
X 
X 
 
 
 
 
 
Pregnancy test (serum)  
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Pregnancy test (urine)i 
 
X 
 
  
  
  
 
X 
  
 
 
 
 
X 
Hematology, serum chemistry, 
urinalysis 
X 
X 
 
X 
 
X 
 
 
X 
X 
 
X 
 
X 
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Procedure 
Screening 
Treatment Period 
Observation Period  
Visit 
V1 
V2 
(BL) 
V3a 
V4 
V5 
V6 
V7a 
V8 
V9 
V10 
(EOT or 
PEOT) 
V11b 
V12 
V13b 
V14 
(EOS)  
Day (days)c  
-28 to -1 
1 
3(±1)  
8(±2)  
15(±2) 
22(±2) 
24(±1)  29(±2) 
36(±2) 
43(±2) 
57(±3) 
71(±4) 
85(±3) 
99(±4) 
PTT and INR measurements 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
X 
Serology testing for HIV, 
Hepatitis B, and Hepatitis C 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Anti-tetanus toxoid titer  
 
X 
 
 
 
 
 
 
 
X 
 
 
 
X 
Vital signs 
X  
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
 
X 
Call or enter IRT to register the 
visit 
X 
 
 
X 
X 
X 
 
X 
X 
X 
 
 
 
X 
Call or enter IRT/ Randomization 
 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Study participants identification 
card assigned 
 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Recording of AEs 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Study drug administrationj 
 
X 
 
X 
X 
X 
 
X 
X 
 
 
 
 
 
Blood sampling for PKk 
 
X 
X 
  
X 
 
X 
 
 
X 
  
 
 
 
Blood sampling for ADA 
X 
X 
 
 
X 
 
X 
 
 
X 
 
X  
 
X 
Blood sampling for exploratory 
safety biomarker analysisl 
 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Blood sampling for future 
exploratory biomarker analysis  
 
X 
 
X 
 
 
 
 
 
 
 
 
 
X 
Blood sampling for total IgG and 
IgG subclasses  
X 
X 
 
X 
X 
X 
 
X 
X 
X 
 
X 
 
X 
Blood sampling for IgA, IgM, 
IgE 
 
X 
 
 
 
 
 
 
 
X 
 
 
 
X 
MG-specific autoantibodiesm 
 
X 
 
 
X 
 
 
X 
 
X 
 
X 
 
X 
Serum complement levels 
(C3,C4) and cytokinesn 
 
X 
 
 
 
 
 
 
X 
 
 
 
 
 
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Procedure 
Screening 
Treatment Period 
Observation Period  
Visit 
V1 
V2 
(BL) 
V3a 
V4 
V5 
V6 
V7a 
V8 
V9 
V10 
(EOT or 
PEOT) 
V11b 
V12 
V13b 
V14 
(EOS)  
Day (days)c  
-28 to -1 
1 
3(±1)  
8(±2)  
15(±2) 
22(±2) 
24(±1)  29(±2) 
36(±2) 
43(±2) 
57(±3) 
71(±4) 
85(±3) 
99(±4) 
Plasma complement levels (C3a 
and C5a)n  
 
X 
 
 
 
 
 
 
X 
 
 
 
 
 
MGFA classification 
X 
X 
 
 
 
 
 
 
 
X 
 
 
 
X 
QMG scale  
X 
X 
 
X 
X 
X 
 
X 
X 
X 
 
X 
 
X 
MG-C scale  
X 
X 
 
X 
X 
X 
 
X 
X 
X 
 
X 
 
X 
MG-ADL 
X 
X 
 
X 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
MG Symptoms PRO 
X 
X 
 
X 
X 
 
 
X 
 
X 
 
X 
 
X 
PGI-S 
X 
X 
 
 
X 
 
 
X 
 
 
 
X 
 
X 
PGI-C 
 
 
 
 
X 
 
 
X 
 
 
 
X 
 
X 
EQ-5D-5L 
 
X 
 
 
 
 
 
 
 
X 
 
 
 
 
MGIIo 
 
X 
 
 
 
 
 
 
 
X 
 
 
 
 
MG-QOL15r 
 
X 
 
 
 
 
 
 
 
X 
 
 
 
 
Study drug discontinuation 
criteria 
 
 
 
X 
X 
X 
 
X 
X 
 
 
 
 
 
Study withdrawal criteria  
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
AChR=acetylcholine receptor; ADA=anti-drug antibody; AE=adverse event; AESM=adverse events of special monitoring; BL=Baseline; C-SSRS=Columbia 
Suicide Severity Rating Scale; ECG=electrocardiogram; EQ-5D-5L=EuroQol-5D-5L; EOS=End of Study; EOT=End of Treatment; GI=gastrointestinal; 
HIV=human immunodeficiency virus; IgA=immunoglobulin A; IgE=immunoglobulin E; IgG=immunoglobulin G; IgM= immunoglobulin M; 
IGRA=interferon-gamma release assay; IMP=investigational medicinal product; INR=international normalized ratio; IRT=interactive response technology; 
MG=myasthenia gravis; MG-ADL=myasthenia gravis-Activities of Daily Living; MG-C=Myasthenia Gravis Composite; MGFA=Myasthenia Gravis Foundation 
of America; MGII=Myasthenia Gravis Impairment Index; MG-QOL15r=Myasthenia Gravis Quality of Life; MuSK=muscle-specific kinase; PEOT=premature 
end of treatment; PGI-C=Patient Global Impression of Change; PGI-S=Patient Global Impression of Severity; PK=pharmacokinetics; PTT=partial 
thromboplastin time; PRO=patient-reported outcome; QMG=Quantitative Myasthenia Gravis; TB=tuberculosis; V=Visit 
a This visit can be scheduled as a home visit, unless the study participant prefers to attend the site. 
b This visit will be a virtual visit, unless the study participant prefers to attend the site. 
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c A visit window of ±1 day is allowed for V3 and V7. A visit window of ±2 day is allowed for all dosing visits. The visit window of ±2 days is relative to the 
first dosing visit date. However, there should be a minimum interval of 5 days and maximum interval of 9 days as compared to the previous dose. A visit 
window of ±3 days is allowed for V11 and V13. A visit window of ±4 days is allowed for V12 and V14. 
d Refer to Section 1.4. 
e Only weight will be repeated at Baseline (V2). 
f A full 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. 
g Chest X-ray should only be performed at sites in countries at high risk for TB (see Section 8.2.6). If chest X-ray is performed, the study participant cannot be 
dosed until the result is available, and negative. 
h The IGRA test will be performed in a central laboratory. Study participant should not be dosed in case of any positive IGRA or two indeterminate IGRAs until 
the study participants has been evaluated by a TB specialist. 
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 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.  
k At dosing visits, PK samples should be taken predose for all study participants. 
l Additional exploratory safety samples must be collected 4 hours postdose or as soon as possible before the next IMP in case of AESM of severe headache 
and/or AESM of severe GI disorders as described in Section 8.3.7. 
m MuSK and AChR antibodies will be tested at Baseline. All subsequent testing will be limited to the positive antibody. 
n To be collected predose at V2, and 4 hours postdose at V9. Additional samples should be collected 2 hours and 4 hours postevent or as soon as possible before 
the next IMP for study participants with severe headache and/or infusion reaction or hypersensitivity reaction. 
o MGII is optional for all study participants. 
 
 
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1.4 
Schedule of Activities (sub-study) 
Procedure 
Treatment Period 
Visit 
V3a 
V9a 
V9b 
Day (days) 
6(±1) 
38(±1) 
41(±1) 
Blood sampling for PKa 
X 
X 
X 
PK=pharmacokinetics; V=Visit 
The PK samples will be collected at selected sites for study participants who have consented to 
participate in the sub-study as described in Section 8.6. 
a Samples can be obtained at home, if appropriate. 
 
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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 pathogenic autoantibodies. The aim is to reduce the concentration of pathogenic 
IgG in patients 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, immune thrombocytopenia 
(ITP), Goodpasture’s syndrome, neuromyelitis optica, Guillain-Barré Syndrome, 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 other immunomodulatory 
therapy. These therapeutic approaches are not effective in all patients 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 diseases. 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, sometimes life-threatening, debilitating condition 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. Most available treatments act, at least in part, by reducing the quantity of such 
circulating IgG autoantibodies. While the standard of care for MG involves a variety of 
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therapeutic agents including cholinesterase inhibitors, immunomodulators, corticosteroid, 
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 patients with MG. 
By blocking the activity of FcRn, rozanolixizumab accelerates the catabolism of antibodies and 
reduces the concentration of pathogenic IgG in MG patients, thus offering a safe, effective, and 
convenient alternative to existing treatments. This Phase 3 study will provide the required data to 
establish the safety and efficacy of rozanolixizumab in anti-AChR or anti-MuSK autoantibody-
positive patients with generalized MG who experience moderate to severe symptoms and are 
being considered for additional treatment such as IVIg or PEX. 
2.2 
Background 
To date, rozanolixizumab has been administered to human study participants in the following 
clinical studies: UP0018, MG0002, TP0001, TP0003, TP0006, CIDP01, CIDP04 and UP0060. 
UP0018 is a completed first-in-human study (FIH), and 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. CIPD01 is a Phase 2a study in study participants with chronic 
inflammatory demyelinating polyradiculoneuropathy (CIDP), and UP0060 is a Phase 1 study in 
healthy volunteers. 
UP0018 is a completed, FIH study conducted in 49 healthy volunteers. The study evaluated the 
safety, tolerability, PK, and the PD effect on total IgG levels of single ascending doses of 
intravenous (iv) and sc rozanolixizumab. Doses of rozanolixizumab
 were 
administered by both iv and sc routes in 3 cohorts, respectively, as a 
 The 
information from the study is summarized below. 
There were no deaths or serious adverse events (SAEs) reported during UP0018. 
Rozanolixizumab was tolerated with an acceptable safety profile after the single administration 
of 
 iv and 
sc doses. Four TEAEs with a maximum intensity of severe were 
reported in this study: headache (3 study participants [50.0%]) and back pain (1 study participant 
[16.7%]); all of which were reported in the rozanolixizumab 
 iv group. For sc 
administration of rozanolixizumab, the most frequently reported TEAEs were headache (5 study 
participants [27.8%]), back pain and diarrhea (each reported by 3 study participants [16.7%]). No 
severe adverse events (AEs) were reported following sc administration. The peak and total 
exposure of rozanolixizumab showed nonlinear increases consistent with target-mediated drug 
disposition. Dose-dependent statistically significant reductions in levels of total IgG and dose-
dependent reductions in levels of IgG subclasses (IgG 1 to 4) were observed after 
rozanolixizumab was administered by iv or sc routes. 
MG0002 is a completed Phase 2a, multicenter, randomized, double-blind, placebo-controlled, 
treatment-sequence study evaluating the safety, tolerability, and efficacy of rozanolixizumab in 
study participants with moderate to severe MG. The study included 2 Dosing Periods:  
 
 
 
 
 
 
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The primary efficacy variable was the change from Baseline in QMG score to Visit 9 (Day 29).  
Clinically relevant improvements in day-to-day functioning, as measured by change from 
Baseline to Day 29 in MG-ADL (secondary endpoint), were observed following treatment with 
rozanolixizumab 
compared with placebo (p=0.036). Numerical differences in favor of 
rozanolixizumab 
vs placebo were observed in reductions from Baseline in QMG 
(p=0.221) and MG-C score (p=0.089).  
Overall, repeated administrations of rozanolixizumab at dose levels of 
 and 
sc 
have been generally 
and well tolerated, with an acceptable safety profile. No new safety 
concerns were identified. The TEAE profile was similar between rozanolixizumab and placebo, 
except for headaches where increased frequency and severity was observed in the 
rozanolixizumab treated study participants. 
TP0001 is a completed Phase 2, multicenter, open-label, multiple-arm study designed to evaluate 
the safety, tolerability, and efficacy of rozanolixizumab in study participants with primary 
persistent or chronic ITP. The following dose arms were used in the study: 
• Dose Arm 1 (
 study participants): rozanolixizumab 
 
 
• Dose Arm 2 (
 study participants): rozanolixizumab 
 
 
• Dose Arm 3 (
 study participants): rozanolixizumab 
 
 
• Dose Arm 4 (
study participants): rozanolixizumab 
 
• Dose Arm 5 (
study participants): rozanolixizumab 
 
CIDP01 is a Phase 2a, multicenter, randomized, subject-blind, investigator-blind, placebo-
controlled, parallel-group study with the primary objective of evaluating the clinical efficacy of 
rozanolixizumab as a treatment for subjects with CIDP. There are 2 treatment arms in this study:  
• Treatment Arm 1: 
 
 
• Treatment Arm 2: 
 
 
UP0060 is a randomized, participant-blind, investigator-blind, placebo-controlled, single dose-
ascending, cohort design to compare the safety, tolerability, and PK of rozanolixizumab, and to 
explore the PD of rozanolixizumab administered by sc infusion in Japanese, Chinese, and 
Caucasian healthy study participants. 
Further details on UP0018, MG0002, TP0001, TP0003, TP0006, CIDP01, CIDP04 and UP0060 
may be found in Section 5.1 of the current version of the IB. 
2.3 
Benefit/Risk Assessment 
Generalized myasthenia gravis (gMG) 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 
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autoantibody formation. 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 administration of immunoglobulins at a healthcare facility. 
Rozanolixizumab represents an innovative, subcutaneous anti-FcRn monoclonal antibody that 
may provide a novel and specific therapeutic approach for the treatment of patients with gMG. 
Data shows that rozanolixizumab markedly lowers serum IgG and IgG autoantibody levels in 
patients with gMG. 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 
and well tolerated, with an 
acceptable safety profile and in-line with subcutaneous dosing in the Phase 1 program, and the 
safety profile observed in the proof of concept ITP study.  
The most common adverse drug reactions observed after use of rozanolixizumab across 
indications are headaches and gastrointestinal (GI) disturbances (diarrhea, nausea, vomiting). 
Potential risks are serious infusion/hypersensitivity reactions, serious infections, and effects on 
vaccination response. The risks of serious infusion/hypersensitivity and serious infections 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 and/or serious headaches, and infusions is also provided as well as 
expedited reporting requirements of AESM.  
More detailed information about the known and expected benefits and risks and reasonably 
expected adverse events of rozanolixizumab may be found in the current version of the IB. 
3 
OBJECTIVES AND ENDPOINTS 
The objectives and corresponding endpoints for this study are presented in Table 3-1.  
Table 3-1: 
Study Objectives and Endpoints 
Objectives 
Endpoints 
Primary 
• To demonstrate the clinical efficacy of 
rozanolixizumab in patients with 
generalized MG 
The primary efficacy endpoint is:  
• Change from Baseline to Day 43 (Visit 
10) in MG-ADL score 
The secondary efficacy endpoints are:  
• MG-ADL responder (≥2.0 points 
improvement from Baseline) at Day 43 
(Visit 10) 
• Change from Baseline to Day 43 (Visit 
10) in the MG-C score 
• Change from Baseline to Day 43 (Visit 
10) in QMG score 
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• Change from Baseline to Day 43 (Visit 
10) in the MG Symptoms PRO ‘Muscle 
Weakness Fatigability’ score 
• Change from Baseline to Day 43 (Visit 
10) in the MG Symptoms PRO ‘Physical 
Fatigue’ score 
• Change from Baseline to Day 43 (Visit 
10) in the MG Symptoms PRO ‘Bulbar 
Symptoms’ score 
The other efficacy endpoints are:  
• Use of rescue therapy due to worsening 
(IVIg, PEX) 
• Time to first rescue therapy 
• Time to MG-ADL response (≥2.0 points 
improvement from Baseline) 
• Change from Baseline in QMG at each 
scheduled assessment during Treatment 
and Observation Periods 
• QMG responder (≥3.0 points 
improvement from Baseline) 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 
• MG-C responder (≥5.0 points 
improvement from Baseline) at each 
scheduled assessment during Treatment 
and Observation Periods 
• Change from Baseline in MG-ADL at 
each scheduled assessment during 
Treatment and Observation Periods 
• MG-ADL responder (≥2.0 points 
improvement from Baseline) at each 
scheduled assessment during Treatment 
and Observation Periods  
• Change from Baseline in the MG 
Symptoms PRO ‘Muscle Weakness 
Fatigability’ score at each scheduled 
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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 Patient Global 
Impressions of Severity (PGI-S) at each 
scheduled assessment during the 
Treatment and Observation Periods 
• Patient Global Impressions of Change 
(PGI-C) at each scheduled assessment 
during Treatment and Observation Periods 
• Change from Baseline in MG-ADL 
(excluding ocular items) at each 
scheduled assessment during Treatment 
and Observation Periods  
• Change from Baseline in QMG 
(excluding ocular items) at each 
scheduled assessment during Treatment 
and Observation Periods 
• Change from Baseline in MG-C score 
(excluding ocular items) at each 
scheduled assessment during Treatment 
and Observation Periods 
• Change from Baseline to Day 43 (Visit 
10) in MG-QOL 15r 
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• Change from Baseline to Day 43 (Visit 
10) in EuroQol(EQ)-5D-5L  
• Minimal symptom expression (MG-ADL 
score of 0 or 1) at any time up to and 
including Day 43 (Visit 10) 
• Change from Baseline in MG Impairment 
Index (MGII) scores at each scheduled 
assessment during Treatment Period 
• Change from Baseline in MGII ocular 
sub-scores at each scheduled assessment 
during Treatment Period 
• Change from Baseline in MGII 
generalized domain sub-scores at each 
scheduled assessment during Treatment 
Period 
Secondary 
• To assess safety and tolerability of 
rozanolixizumab in MG patients 
The secondary safety endpoints are:  
• Occurrence of TEAEs 
• TEAEs leading to withdrawal of IMP 
The other safety endpoints are: 
• Occurrence of AESM 
• Vital sign changes from Baseline (systolic 
and diastolic BP and pulse rate at each 
scheduled assessment during Treatment 
and Observation Periods) 
• 12-lead ECG change from Baseline at 
each scheduled assessment during 
Treatment and Observation Periods 
• Laboratory changes from Baseline at each 
scheduled assessment during Treatment 
and Observation Periods (hematology, 
clinical chemistry, and urinalysis)  
• Suicidality as measured by the Columbia 
Suicide Severity Rating Scale (C-SSRS) 
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Other 
• To assess pharmacokinetic (PK) 
characteristics of rozanolixizumab 
• Plasma concentration of rozanolixizumab 
at each scheduled assessment during the 
Treatment and Observation Periods 
• To assess the pharmacodynamics (PD) 
effects of rozanolixizumab on IgG, 
disease-specific autoantibodies 
• Change (absolute and percentage) from 
Baseline in MG-specific autoantibodies at 
each scheduled assessment during 
Treatment and Observation Periods  
• Change (absolute and percentage) from 
Baseline in serum total IgG and IgG 
subclasses concentrations at each 
scheduled assessment during Treatment 
and Observation Periods 
• To evaluate the emergence and incidence 
of anti-drug antibody (ADA) and impact 
on PK and PD 
• Anti-drug antibody at each scheduled 
assessment during the Treatment Period 
and Observation Periods 
• To evaluate the effects of 
rozanolixizumab on the concentration of 
total protein, 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 Perioda 
• 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 
• To assess the effect of rozanolixizumab 
on exploratory biomarkers and protein 
expression, and explore the relationship 
between protein, and metabolite 
• Exploratory biomarkers may be measured 
to evaluate the effect of rozanolixizumab. 
• Proteins, and metabolites changes that 
may be measured to understand the cause, 
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biomarkers and cause, progression, and 
appropriate treatment of MG 
progression, and appropriate treatment of 
MG 
a To be performed for study participants with severe headache and/or infusion reaction or hypersensitivity reaction. 
Other exploratory safety biomarkers may be assessed. 
The primary efficacy analysis is the change from Baseline in MG-ADL score to Day 43 
(Visit 10), as further detailed in Section 9.3. 
4 
STUDY DESIGN 
4.1 
Overall Design 
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 3-arm, repeat dose 
study evaluating the efficacy and safety of two doses of rozanolixizumab and matching placebo 
in patients with generalized MG who experience moderate to severe symptoms (Myasthenia 
Gravis Foundation of America [MGFA II-IVa]) and are being considered for additional 
treatment such as IVIg or PEX (study schematic is provided in Figure 1-1). 
Fixed unit doses across body weight tiers and study arms will be employed: 
Treatment Arm 1 (rozanolixizumab) – equivalent to approximately
  
• 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 
  
Treatment Arm 2 (rozanolixizumab) – equivalent to approximately 
  
• 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 
  
The placebo arm will be 0.9% sodium chloride aqueous solution (physiological saline, 
preservative free) for sc administration. 
Approximately up to 
study participants will be screened at about 135 sites from North 
America, Europe and Asia to achieve the minimum targeted number of 
 to a maximum of 
 evaluable study participants. 
Participants have the opportunity to rollover into an OLE study, as described below. The OLE 
study, MG0004 (52-week chronic treatment) will be replaced by MG0007, which consists of 
6-week treatment cycles 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. In the event a study participant has already started MG0004, a minimum 
treatment duration of 6 visits (if IMP treatment is held 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) must be completed prior to moving into MG0007. Once the site is 
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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 discontinued MG0004 and had the opportunity to 
rollover into MG0007. 
The maximum duration of the study per study participant will be up to 18 weeks, consisting of a 
Screening Period (1 to 28 days to account for central laboratory turn-around time), a Treatment 
Period (6 weeks), and a blinded Observation Period (8 weeks). 
1. All study participants who complete the 6-week Treatment Period will roll over into an 
8-week Observation Period. 
2. All eligible study participants who complete the Observation Period will be invited to be re-
randomized into an OLE study to either active dose arm 1 or 2.  
3. Study participants who experience disease worsening during the 6-week Treatment Period 
(eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between two 
consecutive visits) may be considered for rescue therapy (IVIg or PEX) at the discretion of 
the Investigator. 
4. Study participants who need rescue therapy during the 6-week Treatment Period will receive 
IVIg or PEX and complete any subsequent visit(s). No further infusions of rozanolixizumab 
will be administered after initiation of rescue therapy. Once the participant completes Visit 
10, he or she will move into the 8-week Observation Period.  
5. Study participants who complete the Treatment Period and require initiation of rescue 
therapy after they start the 8-week Observation Period, may either opt to receive IVIg or PEX 
or complete the EOS visit and immediately roll over into an OLE study where the participant 
will receive rozanolixizumab. Study participants who opt to receive IVIg or PEX will 
complete any remaining visits in the Observation Period and will not be invited to join an 
OLE study. 
6. Study participants who did not complete the 6-week Treatment Period because they needed 
rescue therapy and require initiation of a second course of rescue therapy while in the 8-week 
Observation Period can roll over to an OLE study if a minimum of 2 weeks have lapsed since 
completion of the last IVIg or PEX session. The EOS visit must be completed before 
enrolling into an OLE study. Alternatively, they can be treated with IVIg or PEX. Study 
participants who opt to receive IVIg or PEX will complete any remaining visits in the 
Observation Period and will not be invited to join an OLE study .  
7. Study participants who discontinue study medication for any reason other than requiring 
rescue therapy will not be eligible for enrollment into an OLE study. 
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). 
An IDMC will be formed to monitor the ongoing safety and efficacy of the study. Futility and 
samples size adjustment will also be reviewed and assessed by the IDMC during a formal interim 
analysis at the end of Stage 1, when
 enrolled study participants are evaluable for the primary 
endpoint. Approximately 3 periodic data reviews (in addition to the futility analysis mentioned 
above) will be performed: the first periodic data review will be performed when approximately 
study participants have completed the 6-week Treatment Period; the second periodic data 
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review will be conducted after approximately 
study participants have completed the 6-week 
Treatment Period (ad hoc as needed); and a third review will be conducted after approximately 
 study participants (dependent on the outcome of the interim analysis) have completed 
the 6-week Treatment Period. The timing of any further data reviews will be decided by the 
IDMC in conjunction with sponsor. Further details of the IDMC will be provided in a DMC 
charter. 
4.2 
Scientific Rationale for Study Design 
MG0003 is designed to evaluate the efficacy, safety, and tolerability of rozanolixizumab given in 
dose exposures at 
 or 
 in study participants with MG. The previously 
conducted FIH study, UP0018, provided the safety, tolerability, and PK information for multiple 
dose exposures of 
 administered as iv or sc infusion over 
 in healthy volunteers. 
The duration of the Treatment and Observation Periods were defined based on data from UP0018 
and MG0002. In UP0018, results showed that upon administration of rozanolixizumab, the mean 
percentage change in total IgG over time demonstrated dose-dependent reductions across both 
the iv and sc routes of administration. Similar maximal reductions were observed for both the 
rozanolixizumab iv and sc doses (approximately 48% and 43%, respectively). The lowest level 
of IgG at maximum dose (both iv and sc) was reached by Day 10 on average. The total IgG 
levels generally returned to Baseline values by Day 57 on average. 
Results from MG0002 showed that sc administration of rozanolixizumab resulted in a rapid 
reduction of serum IgG concentrations (Section 4.2.4.4), which also showed the greatest 
reductions from Baseline in QMG, MG-C, and MG-ADL after 6 infusions (Day 50). Therefore, a 
treatment duration of 6 weeks has been selected for MG0003 to ensure the maximum treatment 
effect of rozanolixizumab is captured. An 8-week Observation Period was defined in order to 
follow the recovery of IgG and AChR and MuSK antibodies, as well as monitoring the 
sustainability of the clinical effects after discontinuation. 
The population in this study has been defined to exclude participants with severe weakness 
affecting oropharyngeal or respiratory muscles, or who have myasthenic crisis at Screening, as 
these participants would require urgent care incompatible with participation in the clinical study. 
This keeps consistent with the intended indication.  
4.3 
Justification for Dose 
The selection of fixed unit dosing is introduced as part of future development plans to support 
patient self-injection with a medical device in a home setting. Dose stratified on weight tiers 
have been defined to replicate PD effects throughout the weight range that is similar to weight-
based (mg/kg) dosing. The proposed doses and regimen of rozanolixizumab were selected based 
on the results from FIH study UP0018, alongside the efficacy and safety data from the MG Phase 
2 study (MG0002). 
UP0018 was a randomized, Investigator- and study participant-blind, placebo-controlled, single 
dose-escalating study to evaluate the safety and PK and to explore the PD of rozanolixizumab 
doses of 
 administered as iv or sc infusion over 
 in 
 
healthy male and female volunteers. Data indicate that mean absolute decreases in IgG and mean 
percent change from Baseline IgG were greater in the active dose groups (
) compared to 
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the pooled iv and sc placebo group (
) with median maximum decreases of 
 (range: 
) observed on Day 9 postdose for a rozanolixizumab 
 sc dose. 
Rozanolixizumab was tolerated with an acceptable safety profile after the single administration 
of a 
sc dose, and all participant-reported TEAEs were mild or moderate in severity. 
MG0002 was a Phase 2, multicenter, randomized, Investigator- and study participant-blind, 
placebo-controlled, 2-arm repeat dose, treatment sequence study evaluating the safety and 
efficacy of rozanolixizumab sc (
) in 
study participants with generalized 
MG. Marked improvements in patient-reported MG symptoms and disability, as measured by 
change from Baseline to Day 29 in MG-ADL, were observed following treatment with 
rozanolixizumab 
 compared with placebo. The least squares (LS) mean (Standard Error 
[SE]) change from Baseline was 
 for the Rozanolixizumab 
 Group and 
 
for the Placebo Group. The difference between treatment groups was 
(p=
 [
upper 
confidence interval (CI): 
]). Smaller but numerical differences in favor of rozanolixizumab 
 vs placebo were observed in reductions from Baseline in QMG on Day 29 with the LS 
mean (SE) change from Baseline of 
 for the Rozanolixizumab 
Group and 
 
 for the Placebo Group. The LS mean (SE) change from Baseline in MG-C score to Day 29 
was 
 for the Rozanolixizumab 
 Group and 
 for the Placebo Group. In 
general, responder rates for QMG score, MG-C score, and MG-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 in 
Dosing Period 1 and continued to decline on further dosing with 
to a mean nadir of 
 for total IgG (
 reduction from Baseline). Overall, repeated administrations of 
rozanolixizumab at dose levels of 
 and 
sc have been generally 
and well 
tolerated, with an acceptable safety profile. No new safety concerns have been identified to date. 
The TEAE profile was similar between rozanolixizumab and placebo, except for headaches 
where increased frequency and severity was observed in the rozanolixizumab-treated study 
participants.  
Consistent with the mechanism of action of rozanolixizumab, an increased catabolism of IgG 
will reduce disease-specific autoantibodies with corresponding improvements in clinical signs 
and symptoms. Two sc treatment arms, equivalent to individual
 and 
 dosing, 
have been selected to provide maximal reduction in autoantibody concentration and result in 
clinically significant improvements in the primary endpoint. A dose of 
 has demonstrated 
clinical improvements in MG-ADL in study MG0002 (as described above). Additional data from 
the Phase 2 study in study participants with ITP (TP0001) demonstrated that rozanolixizumab 
was well tolerated at doses at 
 with further IgG reduction achieved. An individual 
equivalent dose of 
has been included to assess if further improvements in MG-ADL 
(magnitude of response and time to onset of symptom relief) can be achieved at a higher dose 
level with greater and more rapid IgG reduction. 
A population PKPD 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 
prediction interval) to the weight-based (mg/kg) 
dosing regimens studied previously. These models-based simulations demonstrate that the 
proposed
 doses of rozanolixizumab for 6 consecutive weeks are expected to produce 
mean maximum IgG reductions of 
. These reductions will be achieved rapidly and 
maintained consistently over the Treatment Period. 
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4.4 
End of Study definition 
A study participant is considered to have completed the study if he/she has completed all phases 
of the study including the Observation Period. Study participants will have an EOS Visit 
performed 8 weeks after the last dose of study medication, or upon discontinuation of the study. 
The EOS is defined as the date of the last visit of the last study participant in the study. 
5 
STUDY POPULATION 
5.1 
Inclusion Criteria 
Study participants are eligible to be included in the study only if all of the following criteria 
apply: 
Age 
1. Study participant must be ≥18 years of age, at the time of signing the informed consent. 
Type of participant and disease characteristics 
2. Study participant has documented diagnosis of gMG at Visit 1, based on study participant’s 
history and supported by previous evaluations.  
3a. Study participant has a confirmed positive record of autoantibodies against AChR or MuSK 
at Screening (Visit 1). The presence of autoantibodies may be confirmed with repeat testing 
at Visit 1.  
4. Study participant has MGFA Class II to IVa at Visit 1. 
5a. Study participant with a MG-ADL score of at least 3 (with ≥3 points from non-ocular 
symptom) AND a QMG score of at least 11 at Visit 1 and at Baseline (Visit 2).  
6a. Study participant is considered for additional treatment such as IVIg or PEX by the 
Investigator. 
Weight 
7a. Body weight ≥35kg at Visit 1.  
Sex 
8c. Study participants may be male or female 
− A male study 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. 
− A female participant is eligible to participate if she is not pregnant (see Section 10.4), not 
breastfeeding, and at least one of the following conditions applies: 
◦ 
Not a woman of childbearing potential (WOCBP) as defined in Section 10.4 
OR 
◦ 
A WOCBP who agrees to follow the contraceptive guidance in Section 10.4 during 
the Treatment Period and for at least 90 days after the last dose of study treatment. 
The study participant must have a negative serum pregnancy test at Visit 1, which is 
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confirmed to be negative by urine testing prior to the first dose of study medication at 
Visit 2. 
Informed consent 
9. Capable of giving signed informed consent as described in Appendix 1, Section 10.1.3 which 
includes compliance with the requirements and restrictions listed in the informed consent 
form (ICF) and in this protocol. 
5.2 
Exclusion Criteria 
Unless otherwise stated, each criterion is applicable to at Visit 1. Study participants are excluded 
from the study if any of the following criteria apply: 
Medical conditions 
1. Study 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. Study participant has a history of alcohol use disorder or other substance use disorder (as per 
Diagnostic and Statistical Manual of Mental Disorders-5) within 12 months prior to Visit 1. 
3. Study participant has a known hypersensitivity to any components of the study medication or 
comparative drugs as stated in this protocol. 
4a. Study participant has a known history of hyperprolinemia, since L-proline is a constituent of 
the rozanolixizumab formulation. 
5. Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) 
in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or 
requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP. 
6. Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB 
infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous 
mycobacterial infection (NTMBI) will be excluded. 
Prior/Concomitant therapy 
7. Study participant has previously received rozanolixizumab drug product. 
8. Study participant has received a live vaccination within 8 weeks prior to Visit 2; or intends to 
have a live vaccination during the course of the study or within 8 weeks following the final 
dose of IMP. 
9a. Study participant has been treated with prohibited immunosuppressants, biologics, and other 
therapies within timeframe shorter than no-treatment period detailed in Table 5-1. 
10a.Study participant has been treated with any biological agent other than those listed in 
Table 5-1 in the past 3 months or within 5 half-lives prior to Visit 2, whichever was longer. 
11.Study participant has prior treatment with rituximab in the 6 months prior to Visit 2 or study 
participant has prior treatment with rituximab in the 12 months prior to Visit 2 and B cells 
monitoring have shown they did not return to normal range. 
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12a.Study participant had a thymectomy in the past 6 months or a thymoma at any time that 
required chemotherapy and/or radiotherapy prior to Visit 1. 
13b.Study participant has any of the following active GI disorders: inflammatory bowel disease 
(IBD), or GI ulceration or diverticulitis . 
Table 5-1: 
Treatment-free Period for Exclusionary Immunosuppressants, 
Biologics, and Other Therapies Prior to Baseline (Visit 2) 
Generic name (commercial/trade names) 
Period relative to Baseline Visit 
(regardless of route) 
Immunosuppressants 
 
Cyclophosphamide (Cytoxan®) 
 
Pimecrolimus (Elidel®) 
 
Vinca alkaloids (vincristine, vinblastine) 
 
Biologics (Mabs and fusion proteins) 
 
Abatacept (CTLA 4-Ig) (Orencia®) 
 
Eculizumab (Soliris®) 
 
Belimumab (Benlysta®) 
 
Golimumab (Simponi®) 
 
Natalizumab (Tysabri®) 
 
Ofatumumab (Arzerra) 
 
Rituximab (Rituxan®) 
 if B-cells 
did not return to normal range 
Ocrelizumab (Ocrevus®) 
 if B-cells 
did not return to normal range 
TACI-Ig (Atacicept) 
 
Veltuzumab 
 
Other biologics 
, or within 
 
(whichever was longer) prior to 
the Baseline Visit 
Inebulizumab 
 (prior to Baseline Visit) 
and B-cells are within normal 
range 
Others 
 
Intravenous or subcutaneous immunoglobulin 
 
IPP-201101 (Lupuzor™) 
 
PEX 
 
Immunoadsorption 
 
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Table 5-1: 
Treatment-free Period for Exclusionary Immunosuppressants, 
Biologics, and Other Therapies Prior to Baseline (Visit 2) 
Generic name (commercial/trade names) 
Period relative to Baseline Visit 
(regardless of route) 
Mabs=monoclonal antibodies; PEX=plasma exchange 
 
Prior/Concurrent clinical study experience 
14. Study participant has participated in another study of an IMP (and/or an investigational 
device) within the previous 3 months or is currently participating in another study of an IMP 
and/or an investigational device. 
15a.Study participant has been previously randomized in this study (re-screening for screen-
failed participants is allowed with prior consultation and permission of the medical monitor). 
16. Study participant has experienced hypersensitivity reaction after exposure to other anti-FcRn 
drugs.  
Diagnostic assessments 
17a.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 at 
Visit 1 or Visit 2. 
18. Study participant has a serum total IgG level ≤5.5g/L. 
19. Study participant has absolute neutrophil count <1500 cells/mm3. 
20. Study 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. 
21.Study participant has 12-lead ECG with findings considered to be clinically significant upon 
medical review. The clinical significance of the findings needs to be assessed by the 
Investigator to determine eligibility, and any queries regarding continuation of the study 
participants will have to be addressed with the Medical Monitor. 
22b.Study participant has renal impairment, defined as glomerular filtration rate (GFR) less than 
45ml/min/1.73m2 at Visit 1. 
23a.Alanine transaminase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase 
(ALP) are >3x upper limit of normal (ULN), or bilirubin >1.5xULN (isolated bilirubin 
>1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%). 
Study participant has elevations only in total bilirubin that were >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 
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elevation will have to be understood and recorded in the electronic Case Report form 
(eCRF). 
If study participant has >ULN, ALT, AST, or ALP that does not meet the exclusion limit at 
Visit 1, the tests should be repeated, if possible, prior to dosing to ensure there was no further 
ongoing clinically relevant increase. In case of a clinically relevant increase, inclusion of the 
study participants will have to be discussed with the Medical Monitor. 
Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit (>3xULN) will 
have to be repeated once for confirmation. This includes rescreening. 
24. Presence of Hepatitis B surface antigen (HBsAg) at Visit 1.  
25. Positive Hepatitis C antibody test result at Visit 1 or within 3 months prior to starting study 
treatment. NOTE: Study participant with a positive Hepatitis C antibody due to prior 
resolved disease can be enrolled, only if a confirmatory negative Hepatitis C RNA test is 
obtained.  
26. Positive Hepatitis C RNA test result at Visit 1 or within 3 months prior to first dose of study 
treatment. NOTE: Test is optional and a study participant with negative Hepatitis C antibody 
test is not required to also undergo Hepatitis C RNA testing. 
27b.Current unstable liver or biliary disease per Investigator assessment defined by the presence 
of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, 
persistent jaundice, or cirrhosis. NOTE: with exception of stable hepatobiliary conditions 
(including Gilbert’s syndrome, asymptomatic gallstones). 
28. Study participant tests positive for human immunodeficiency virus (HIV). 
29a.Study participant has a current or medical history of primary immunodeficiency. 
30a.Study participant has active neoplastic disease or history of neoplastic disease within 5 years 
of study entry prior to Visit 1 (except for basal or squamous cell carcinoma of the skin or 
carcinoma in situ of the uterine cervix that have been definitively treated with standard of 
care approaches). 
31a.Study participant has a planned elective surgical procedure within 4 months after Visit 1. 
32. Study participant has a history of a solid organ transplant or hematopoietic stem cell/marrow 
transplant. 
33.Study participant has corrected QT interval (QTcF) >450 msec (for male participants) or 
QTc >470 msec (for female participants) or QTc >480 msec in participants with bundle 
branch block. 
Other exclusions 
34. The study participant is not considered capable of adhering to the protocol visit schedule, or 
medication intake according to the judgment of the Investigator. 
35. A female study participant, who tests positive for pregnancy, plans to get pregnant during the 
participation in the study, or who is breastfeeding. 
36. Study participant has a lifetime history of suicide attempt (including an active attempt, 
interrupted attempt, or aborted attempt), or had suicidal ideation in the past 6 months as 
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indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia 
Suicide Severity Rating Scale (C-SSRS). 
37. Criterion removed. 
38. Participant with current or medical history of IgA deficiency. 
39a.Participant with a medical history of splenectomy. 
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 medicinal cannabidiols and medicinal marijuana (prescribed by a physician) is 
permitted. 
5.4 
Screen Failures 
Screen failures are defined as study participants who consent to participate in the clinical study 
but are not subsequently randomly assigned to study treatment. A minimal set of screen failure 
information is required to ensure transparent reporting of screen failure study 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 SAE. 
Individuals who do not meet the criteria for participation in this study (screen failure) may be 
rescreened, following discussion with the sponsor’s medical monitor/study physician. 
Rescreened study participants should be assigned a new participant number for rescreening and 
repeat all Visit 1 assessments. 
If a study participant has isolated test results outside the specific range which is deemed 
clinically nonsignificant, the abnormal value may be rechecked at the discretion of the 
Investigator, following information of the sponsor’s medical monitor/study physician. If the 
normalization of the test result occurs within the Screening Period, then no other Screening 
procedures need to be repeated. 
Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit may be repeated 
once for confirmation. This includes rescreening.  
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6 
STUDY TREATMENTS  
6.1 
Treatments Administered 
Study Treatment Name: 
Rozanolixizumab 
(Treatment Arm 1) 
Rozanolixizumab 
(Treatment Arm 2) 
Placebo 
(Treatment Arm 3) 
Dosage formulation: 
Solution for infusion 
Solution for infusion 
Aqueous solution 
Unit dose 
strength(s)/Dosage 
level(s): 
 
 
 
 
 
0.9% sodium 
chloride aqueous 
solution 
(physiological 
saline, preservative 
free) 
Route of Administration  
Subcutaneous (sc) 
Dosing instructions: 
 
 
 
 
 
 
 
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 
 
 
 
 
 
 
 
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 
 
6 sc doses of 
placebo at 1-week 
intervals 
Packaging and Labeling 
Rozanolixizumab and matching placebo are manufactured, packaged, 
and labeled according to Good Manufacturing Practice guidelines and 
applicable laws or regulations. 
Details on the preparation of study treatment for infusion, rate of infusion, administration, 
appropriate records handling, and blinded and unblinded site personnel roles are provided in the 
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. 
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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, or delegate 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. 
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. 
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 (SOPs) 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 and Blinding 
An interactive response technology (IRT) will be used for assigning eligible study 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. Each study participant will receive a 
5-digit number assigned at Screening that serves as the study participant identifier throughout the 
study. The study participant number will be required in all communication between the 
Investigator or designee and the IRT regarding a particular study participant. Study participant 
numbers and kit numbers will be tracked via the IRT.  
To randomize a study participant, the Investigator or designee will contact the IRT and provide 
brief details about the study participant to be randomized. The IRT will automatically inform the 
Investigator or designee of the study participant’s randomization number. The IRT will allocate 
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kit numbers to the study participant based on the participant number during the course of the 
study. The randomization number must be incorporated into the eCRF. 
The randomization will be stratified by the following: 
• MG-specific autoantibody (MuSK+ or AChR+) 
6.3.1 
Procedures for maintaining and breaking the treatment blind 
6.3.1.1 
Maintenance of study treatment blind 
All study participant treatment details, rozanolixizumab treatment arm, planned dose, or placebo 
will be allocated and maintained by the IRT system. 
The following individuals will receive the randomization code at the start of the study: 
• Designated bioanalytical staff analyzing PK samples 
• IRT provider 
Study site pharmacists or other suitably qualified site personnel who are responsible for 
preparation of IMP treatments will have access to treatment allocations for individual study 
participants via the IRT. The unblinded pharmacy monitors from the Contract Research 
Organization (CRO), and the Clinical Supply Manager will also have access to the treatment 
allocations and to the drug accountability records, if applicable. 
The following individuals may, as necessary, have access to the randomization code as indicated: 
• Sponsor Patient Safety staff as needed for reporting SAEs to regulatory authorities. 
• Members of the IDMC who participate in unblinded sessions will be given information about 
the IMP allocation for those study participants for whom data are provided at these sessions. 
• CRO staff supporting preparation of the data outputs for the IDMC review and/or any interim 
analyses. 
• Independent Medical Monitor external to the sponsor for monitoring unblinded clinical 
laboratory data, such as total IgG levels. 
If IgG levels drop below 1g/L, the IMP must be temporarily discontinued. If IgG levels is  ≥1 
and <2g/L, and study participant is experiencing a nonserious infection which is persisting or 
recurrent, rozanolixizumab treatment may be temporarily discontinued (Section 7.1.4). 
Temporary treatment discontinuation due to low IgG levels may informally unblind the treatment 
assignment to the participant and site personnel. Therefore, infusions will be continued but given 
as mock infusions with only placebo irrespective of IMP designation. Allocation of mock kit 
numbers will be handled via the IRT. An unblinded Medical Monitor will inform the Investigator 
of the initiation of mock infusions and bring attention to any potential infection risk. The 
Medical Monitor will continue to review available safety information for these study participants 
(eg, AEs for infection and white cell count) while the IgG levels are at the protocol-defined low 
threshold values, and directly contact the Investigator should this review identify additional 
information that may be important in the care of the study participants. When the IgG levels have 
returned to the protocol-defined levels to re-initiate IMP (Section 7.1.4), the unblinded Medical 
Monitor will inform the Investigator of this decision. Based on the clinical situation, the 
Investigator has the option of holding the dose until deemed appropriate. 
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Further details are provided in the study manual and site blinding plan. 
6.3.1.2 
Breaking the treatment blind in an emergency situation 
In the event of an emergency, it will be possible to determine to which treatment arm and dose 
the study participant has been allocated by contacting the IRT. All sites will be provided with 
details of how to contact the system for code breaking at the start of the study. The Medical 
Monitor or equivalent should be consulted prior to unblinding, whenever possible. 
The Clinical Project Manager and Medical Monitor will be informed immediately via the IRT 
when a code is broken, but will remain blinded to specific treatment information. Any unblinding 
of the study medication performed by the Investigator must be recorded in the source documents 
and on the Study Termination eCRF page. 
Inadvertent unblinding has to be listed as a major protocol deviation. 
6.4 
Treatment Compliance 
Drug accountability must be recorded on the Drug Accountability form (see Section 6.2.1). 
6.5 
Concomitant Medication(s)/Treatment(s) 
6.5.1 
Permitted concomitant treatments (medications and therapies) 
All concomitant treatments permitted during the study are presented in Table 6-1.  
Any violation of the permitted treatment criteria would lead to prohibited treatment and should 
be discussed with the Investigator, sponsor and Medical Monitor. 
Table 6-1: 
Permitted Concomitant Treatments 
Permitted 
Medications 
Dose 
Comment 
Oral Corticosteroids 
(eg, prednisolone) 
No specific requirements 
Stable for 
 prior to 
Baselinea 
Methotrexate 
≤30mg/week 
Treated for previous 
 and 
on stable dose for 
 prior 
to Baselinea 
Mycophenolate mofetil  
≤3g/day 
Treated for previous 
 and 
on stable dose for
 prior 
to Baselinea 
Cyclosporineb 
≤5mg/kg/day for 
unmodified 
≤4mg/kg/day for modified 
(microemulsion) 
Treated for previous 
 and 
on stable dose for 
 prior 
to Baselinea 
Azathioprine 
≤3mg/kg/day 
Treated for previous 
 and 
on stable dose for
 prior 
to Baselinea 
Cholinesterase 
inhibitors 
≤600mg 
Pyridostigmine/day 
Stable dose not required – dose 
 of efficacy 
outcomes 
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Permitted 
Medications 
Dose 
Comment 
Tacrolimusc 
≤5mg/day 
Treated for previous
 and 
on stable dose for 
 prior 
to Baselinea 
a Must also be stable for duration of study. 
b Doses higher than listed are permissible if trough level is ≤300ng/mL. 
c If the total daily weight-based dose is >5mg, then a plasma trough level should be checked to 
ensure study participant is not above the recommended therapeutic range. 
The use of medicinal cannabidiols and medicinal marijuana (prescribed by a physician) is also 
permitted. When applicable, the study participant must be on a stable dose of cannabidiols and/or 
medicinal marijuana for
 prior to Screening, and remain stable for the duration of the 
study. 
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 
• Vinca alkaloids (vincristine, vinblastine) 
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 IMP, but should be encouraged to continue with Observation Period visits. 
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. 
6.5.3 
Treatments specific to NMJ interference 
Treatments could interfere with the function of the NMJ (and which therefore could impair study 
participants with MG), such as, but not limited to, include the following medications: 
− botulinum toxin 
− aminoglycoside antibiotics  
− tetracycline antibiotics  
− penicillamine 
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− magnesium 
For a more detailed list please refer to the MGFA 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. 
6.5.4 
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 two consecutive visits) may be considered for 
rescue therapy at the discretion of the Investigator. 
Study participants who need rescue therapy during the Treatment Period may receive IVIg or 
PEX at the discretion of the Investigator, will complete any subsequent visit(s) and will move 
into the Observation Period.  
For study participants receiving IVIg or PEX as rescue therapy, the date and time of 
administration as well as the name and dosage regimen of the rescue therapy must be recorded. 
6.6 
Dose Modification 
There is no dose modification allowed in this study. 
6.7 
Treatment after the End of the Study 
Study participants who need rescue therapy during the 8-week Observation Period, or complete 
the Observation Period without rescue therapy, will be invited to enroll into the OLE study. They 
will be randomized to one of two rozanolixizumab dose arms, equivalent to approximately 
or 
. Alternatively, if a study participant opts to receive IVIg or PEX during the 
Observation Period, he or she will complete the Observation Period and will not be eligible to 
enter the OLE study. 
In the case of prolonged hypogammaglobulinemia after treatment discontinuation, study 
participants must be followed up until IgG levels return to values within the normal range or to 
individual Baseline values. 
For study participants not enrolling into the OLE study, there are no plans for continued 
provision with rozanolixizumab after the end of the study. The participant should discuss any 
needed care after the study with their healthcare provider. 
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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 study participant meets one of the conditions outlined in Figure 7-1 and 
Figure 7-2, or if the Investigator believes that it is in the best interest of the study participant.  
The study participant should follow the visit schedule as described in the protocol and the eCRF 
be completed accordingly. 
Figure 7-1: Liver Chemistry Stopping Criteria and Increased Monitoring 
Algorithm  
 
ALT=alanine transaminase; AST=aspartate aminotransferase; INR=international normalized ratio; SAE=serious 
adverse event; ULN=upper limit of normal 
 
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Figure 7-2: Liver Chemistry Increased Monitoring Algorithm with Continued 
Study Intervention for Participants with ALT ≥3xULN but ≤8ULN 
 
ALT=alanine transaminase; 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 are provided 
in Appendix 6 (Section 10.6).  
7.1.2 
QTc Stopping Criteria 
If a clinically significant finding is identified (including, but not limited to changes from 
Baseline in QT interval corrected using Fridericia’s formula [QTcF]) after enrollment, the 
Investigator or qualified designee will determine if the study 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 the bulleted criteria based on the average of triplicate ECG 
readings will discontinue rozanolixizumab 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 >600 msec 
• Change from Baseline of QTc >60 msec 
For study participants with underlying bundle branch block, follow the discontinuation criteria 
listed below: 
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Baseline QTc with Bundle Branch Block 
Discontinuation QTc Threshold with Bundle 
Branch Block 
< 450 msec 
> 500 msec 
450 to 480 msec 
≥ 530 msec 
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 the eCRF 
be completed accordingly. 
7.1.3 
Discontinuation due to other adverse events or medical conditions 
Study participants must permanently discontinue rozanolixizumab and move into the 
Observation Period if any of the following events occur: 
1.  Study participant develops an illness that would interfere with his/her continued exposure to 
rozanolixizumab. 
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 (ie, bacteremia or sepsis, meningitis, osteomyelitis or septic arthritis, pneumonia, or 
visceral abscess) (see Section 10.25). 
4. Study participant has an AE of severe or serious infusion or anaphylactic reaction requiring 
corticosteroid and/or epinephrine therapy (see Section 10.28) (Sampson et al, 2006). 
5. Study participant experiences a serious or recurrent (ie, second occurrence) severe AE of 
headache, which is considered related to the study medication in the opinion of the 
investigator. 
6. Study participant has a TB test that is confirmed positive or any further evidence suggestive 
of potential TB infection (eg, exposure) and further examinations result in a diagnosis of 
active TB or LTBI (Section 8.2.6). 
7. Study participant meets potential drug-induced liver injury permanent discontinuation 
criteria.  
If a NTMBI is identified during a study, the same withdrawal procedures as those used for an 
active TB infection identified during the study should be followed. 
7.1.4 
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 25; 
Section 10.25). 
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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 25; Section 10.25). 
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. 
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 study 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 study 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. There is confirmation of a pregnancy during the study, as evidenced by a positive pregnancy 
test. 
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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 
participants should be referred immediately to a Mental Healthcare Professional. 
Investigators should contact the Medical Monitor, whenever possible, to discuss the withdrawal 
of a participant in advance. 
Study participants may permanently discontinue rozanolixizumab and move into the 
Observation Period at the discretion of the Investigator, Medical Monitor, and Study Physician if 
any of the following events occur: 
5. Study participant is noncompliant with the study procedures or medications in the opinion of 
the Investigator.  
6. Study participant takes prohibited concomitant medications during the Treatment Period as 
defined in this protocol (Section 6.5.2).  
Study participants who permanently discontinue IMP for reasons other than requiring rescue 
therapy will not be eligible for enrollment into the OLE study. 
Study participants who withdraw from the study during the Treatment Period should complete 
the assessments outlined PEOT Visit and enter the Observation Period. 
7.3 
Lost to Follow up 
A study 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 study participant wishes to and/or should continue 
in the study. 
• Before a study participant is deemed lost to follow up, the Investigator or designee must 
make every effort to regain contact with the study 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) for removing the study participant, must be recorded in the 
source documents. The eCRF must document the primary reason for withdrawal. 
Should the study 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).  
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If needed, the study participant will be allowed to rest before starting any study procedures. 
Quantitative Myasthenia Gravis scale, MG-C scale, MG-ADL and all other PROs should be 
administered by the investigator or qualified designee before any other invasive procedures are 
performed.  
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. Screening assessments can be performed on different days throughout 
the 28-day Screening Period, if required. The Investigator will maintain a Screening log to record 
details of all participants screened and to confirm eligibility or record reasons for Screening 
failure, as applicable.  
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 must be performed: 
• AE reporting 
• Concomitant medications  
• Review of withdrawal criteria 
• Brief physical examination 
• Vital signs 
Blood samples for PK, IgG, hematology, biochemistry, other laboratory testing and assessments 
may be performed as clinically indicated, at 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 
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 
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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 
8.1.1 
MGFA Classification  
The Investigator will classify the study participant’s MG using the MGFA Clinical Classification 
(Appendix 12, Section 10.12) (Jaretzki et al, 2000). This is a 5-stage classification (I to V), with 
a higher class indicating more severe disease. To be eligible for this study, a participant must be 
graded MGFA CLASS II to IVa at Visit 1, as per inclusion criteria (Section 5.1).  
8.1.2 
Quantitative Myasthenia Gravis scale 
For assessment of the QMG scale, investigators or qualified designee will follow the MGFA’s 
QMG Manual instructions (Appendix 13, Section 10.13). Clinical personnel must complete 
mandatory training to assess study participants’ QMG score (details are provided in the Study 
Procedures Manual). If not medically appropriate, then the treatment can be continued but the 
testing should be performed as best as possible at the same timeframe post last 
acetylcholinesterase AChE inhibitor dosing for each evaluation during the study. Study 
participants should not take pyridostigmine (or other AChE inhibitor medication) from midnight 
before testing when medically safe to do so to standardize testing (ie, if AChE inhibitors cannot 
be stopped). 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. 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. Where possible, the same person should carry out the 
assessment at each visit. 
8.1.3 
MG-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 other 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 (Appendix 14, Section 10.14) and a 3-point change being of 
clinical relevance (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 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. 
8.1.4 
Patient-reported outcomes 
Patient-reported outcomes must be completed as per time points 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, PGI-S, 
PGI-C, EQ-5D-5L, MGII (optional assessment), 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.  
8.1.4.1 
MG-Activities of Daily Living 
Independently of study visit type (site, home, or virtual), the MG-ADL must be completed by 
study participants in a quiet place by themselves without the help of a partner or caregiver, 
before any clinical examination takes place. Study participants should be informed of the 
importance of this questionnaire and instructed to read the items and instructions carefully. They 
should be informed that there are no correct or incorrect answers. 
Study personnel are not allowed to interpret the items for the participant. If a participant asks for 
guidance, study personnel should instruct him/her to respond according to their best 
understanding of the item. The MG-ADL should only be checked for completeness by study 
personnel. In the event a few questionnaire items have not been completed, study personnel 
should only query this with the study participant, if this results from an omission. Study 
personnel shall neither complete missing data nor suggest changes to participant responses. As 
with other study data, responses to the questionnaire should be treated as confidential 
information. Data privacy considerations apply. 
In the specific context of virtual and home visits, paper copies of the MG-ADL should be made 
available to study participants ahead of the visit. Site coordinators or study personnel (or an 
automatic alert mechanism) should remind study participants to complete the MG-ADL before 
visit start and to record the date upon completion of the questionnaire. Once they have completed 
the MG-ADL, study participants should transfer it electronically to site personnel. In the event 
study personnel finds out that the MG-ADL has not been completed before the visit, study 
participants should be allowed some additional time to complete the questionnaire before any 
study related assessment is initiated. 
The MG-ADL is an 8-item PRO instrument developed on the basis of the QMG (Wolfe et al, 
1999) (Appendix 15, Section 10.15). The MG-ADL targets symptoms and disability across 
ocular (items #7 [double vision] and #8 [eyelid droop]), 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; and it was 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.  
8.1.4.2 
MG Symptoms PRO 
The MG Symptoms PRO instrument (Appendix 16, Section 10.16) consists of 42 items across 5 
scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms 
(items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). 
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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 they experience 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. 
8.1.4.3 
Patient Global Impression of Severity 
Patient Global Impression of Severity is a single-state, self-report measure that rates a 
participant's severity of specific condition (Appendix 17, Section 10.17). The PGI-S is a 5 point 
scale depicting a participant's rating of overall symptoms (“none,” “mild,” “moderate,” “severe,” 
“very severe,”). 
8.1.4.4 
Patient Global Impression of Change 
Patient Global Impression of Change is a single-state, self-report measure that reflects a 
participant's belief about the efficacy of treatment for a specific condition (Appendix 18, 
Section 10.18). The PGI-C is a 7-point scale depicting a participant's rating of overall 
improvement (“very much improved,” “much improved,” “minimally improved,” “no change,” 
“minimally worse,” “much worse,” or “very much worse”). 
8.1.4.5 
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 19, Section 10.19).  
The EQ-5D-5L essentially consists of 2 pages: the EQ-5D descriptive system and the EuroQol 
visual analogue scale (EQ VAS). 
The descriptive system comprises five 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 patient is asked to 
indicate his/her health state by ticking the box next to the most appropriate statement in each of 
the five dimensions. This decision results in a 1-digit number that expresses the level selected for 
that dimension. The digits for the five dimensions can be combined into a 5-digit number that 
describes the patient’s health state. 
The EQ VAS records the patient’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 reflect the 
patient’s own judgement. 
8.1.4.6 
MG Impairment Index 
The MGII is a measure of disease severity based on the signs and symptoms of MG patients 
(Appendix 20, Section 10.20). It was developed using a patient-centered approach and following 
current guidelines for outcome measure development, incorporating patient input throughout the 
different development phases (Barnett et al, 2014, Barnett et al, 2016). The MGII has 22 
patient-reported and 6 examination items, and scores are presented as a sum of all items for a 
total score but also as an ocular and generalized sub-score. The MGII is an optional assessment 
(see Section 1.3). The Investigator or qualified designee will examine the study participant prior 
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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 patients 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). 
8.1.4.7 
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 MG-QOL15r was designed to assess the 
"patient perspective" in the everyday clinic setting or in a clinical study (Appendix 21, 
Section 10.21). 
When completing the 15-item MG-QOL15r, MG study participant 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. 
8.2.1 
Physical examination 
Full physical examination  
A full 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, 
and musculoskeletal systems. Height and weight will be measured and recorded at the Visit 1 
(Screening). Body weight will be measured at Visit 1 and Baseline (Visit 2) only (as defined in 
Section 1.3) with the study participant wearing light clothing and without wearing shoes. 
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 full 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. 
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8.2.2 
Vital signs 
Oral, tympanic, or axillary temperature, pulse rate, and blood pressure 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 ECG will be obtained as outlined in the Schedule of Activities (Section 1.3) 
using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, 
and QTc intervals. Refer to Section 7.1.2 for QTc withdrawal criteria and any additional QTc 
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 ECG 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 for the list of clinical laboratory tests to be performed and to 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 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 (Section 10.2), must be 
conducted in accordance with the laboratory manual and the Schedule of Activities (Section 1.3).  
8.2.5 
Suicidal risk monitoring 
Study participants being treated with rozanolixizumab should be monitored appropriately for 
suicidal ideation and behavior or any other unusual changes in behavior. Consideration should be 
given to discontinuing rozanolixizumab in study participants who experience signs of suicidal 
ideation or behavior.  
Families and caregivers of study 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. 
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Suicidality will be assessed by trained study personnel using the C-SSRS (Columbia University 
Medical Center, 2008 [Appendix 28, Section 10.28]). This scale will be used for Screening, 
Baseline and all subsequent visits to assess suicidal ideation and behavior that may occur during 
the study. The C-SSRS will be performed at the scheduled timepoints as described in the 
Schedule of Activities (Section 1.3).  
8.2.6 
Assessment and management of TB and TB 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, genito urinary 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, abdominal pain (mimicking inflammatory 
bowel disease), etc. Unusual presentations should always be considered. 
Note: If available, respiratory or other specimens must also be smear and culture negative for TB 
(Center for Disease Control and Prevention 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 “
 
 
should not be allowed into the study pending 
further assessments (including TB specialist consult) as outlined previously. 
TB assessment by IGRA 
The TB screening is mandatory both before study entry and during the conduct of the study. The 
preferred screening test is interferon-gamma release assay (IGRA) performed at a Central 
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Laboratory by QuantiFERON tube test. Additional IGRA test may be performed if indicated (eg, 
presence of signs and symptoms suggestive of TB, recent exposure).  
In high TB incidence countries, it is recommended that the IGRA be the first test performed at 
screening to reduce the number of unnecessary screening procedures on any IGRA positive study 
participants that may need to be treated for TB prophylaxis or potentially withdrawn from the 
study.  
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. 
The retest must be done during the protocol-defined screening window. If the test is positive or 
indeterminate on retest, the study participant must be evaluated by a TB specialist. 
Study participants who have two indeterminate or any positive IGRA test and are cleared by a 
TB specialist will be potentially eligible to be randomized into the study after discussion with 
and approval from study physician (medical monitor). 
In such circumstances, the study participant must be subsequently evaluated by a TB specialist at 
protocol defined TB monitoring visits (in lieu of IGRA testing as it is likely to be positive). 
TB assessment by chest X-ray  
Chest X-ray is not required at Screening in countries where TB incidence is low (≤20 per 
100,000, 2018 TB incidence estimate by the current version of the World Health Organization 
(WHO) Global Tuberculosis Report ANNEX 4). It can, however, be performed at the 
Investigator’s discretion in study participants with risk factors (ie, patient aged 60 or older, 
prolonged steroid use). 
In countries (eg, Russian Federation) where TB incidence is >20 per 100,000 by the current 
version of the WHO Global Tuberculosis Report ANNEX 4, a plain posterioanterior chest X-ray 
should be performed during the Screening and results must be available during Baseline before 
first drug administration unless a chest X-ray (or a computed tomography of the chest) has been 
done from 3 months prior to the Screening Visit. The official report of the imaging must 
specifically indicate the absence of findings suggestive of current active TB or prior inactive TB.  
A chest X-ray or other imaging test after Screening should be performed only if indicated (eg, 
presence of signs and symptoms suggestive of TB, close exposure to persons with TB), and 
interpreted by a qualified specialist (ie, radiologist or pulmonologist). 
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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 case of an IGRA test conversion, the study participant must be considered as having either a 
suspected new latent or an active TB infection and be promptly referred to an appropriate 
specialist (eg, pulmonologist, infectious disease specialist) for further evaluation. If 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 would need to be updated with final diagnosis 
once available. 
Latent TB 
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. 
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-tuberculosis 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 a PEOT Visit must be 
scheduled as soon as possible, but no later than the next scheduled visit. Treatment for active TB 
or NTMB should be started immediately based on local guidelines. 
Confirmed active TB is always considered Serious Adverse Event. UCB’s process requires that 
these 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. 
LTBI, 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.  
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Follow-up information of suspected and confirmed TB cases should be provided to UCB at least 
after 3, 9, and 12 months of 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 of 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 the study treatment or MG0003 (see Section 7). 
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 the 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 the 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 to 30 days from the end of the study for each participant, and to also inform 
study 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 
non-leading 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 non-serious AEs of special 
interest and adverse events of special monitoring [AESM], as defined in Section 8.3.6 and 
Section 8.3.7, respectively), 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). 
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8.3.4 
Regulatory reporting requirements for SAEs 
Prompt notification (24 hrs) by the Investigator to the sponsor of an 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. 
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 Investigator’s Brochure 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, female partners of male 
participants will be collected after the start of study treatment and until 3 months 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 study participant must be withdrawn from the study as soon as pregnancy is known (by 
positive pregnancy test), and the following should be completed: 
• The study 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 immediately reported within 
24 hrs to UCB. Potential Hy’s Law, defined as ALT or AST 3xULN with coexisting 2xULN 
total bilirubin in the absence of ≥2xULN ALP, with no alternative explanation for the 
biochemical abnormality (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 SAE recording and reporting procedures as indicated in Appendix 3 
(Section 10.3). 
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8.3.7 
Adverse events of special monitoring 
For rozanolixizumab, AESM that require immediate reporting (within 24 hrs regardless of 
seriousness) to UCB are: 
• Severe headache 
• Severe GI disorders (ie, abdominal pain, diarrhea, vomiting) 
• Opportunistic infection  
In case of severe headache or serious headache (regardless of severity), the headache 
questionnaire (Appendix 27 [Section 10.27]) must be completed. Additional procedures for 
management of headaches are provided in Appendix 23 (Section 10.23). 
Procedures for the management of diarrhea, and infections and hypogammaglobulinemia are 
provided in Appendix 24 (Section 10.24), and Appendix 25 (Section 10.25), respectively. 
Although infusion-related reactions as well as hypersensitivity reactions or anaphylaxis are not 
classified as AESM, these AEs will be monitored by the investigator. If such an event is 
suspected it should be managed according the guidance provided in Appendix 26, Section 10.26. 
In case of suspected anaphylaxis, the Sampson’s Criteria (Sampson et al, 2006) in Appendix 28 
(Section 10.28) should be completed. 
All AESM 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 unblinded IDMC will periodically review and monitor safety data from this study 
and advise UCB. Details are provided in the DMC 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 or 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 
per week should be considered an overdose, irrespective of the weight tier band. Overdose events 
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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 or laboratory abnormality for at least 5 days. 
3. Obtain a plasma sample for PK analysis and IgG (total and subclasses) antibodies and 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). 
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). Additional 
PK samples will be collected (Section 1.4) at selected sites for study participants who have 
consented to participate in the sub-study. Blood samples may be collected at additional time 
points 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 and 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. 
Drug concentration information that may unblind the study will not be reported to study sites or 
blinded personnel until the study has been unblinded. 
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. 
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 
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• Serum MG-specific autoantibodies (anti-MuSK/anti-AChR) levels (all subsequent testing 
will be limited to the positive antibody) 
For all immunological 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 
Genetics are not evaluated in this study. 
8.9 
Biomarkers 
Collection of samples for exploratory biomarker research is also part of this study. Blood 
samples for biomarker research are required and will be collected from all study participants in 
this study as specified in the Schedule of Activities (Section 1.3). Exploratory samples are 
collected predose. Additional exploratory safety samples must be collected 4 hours postdose or 
as soon as possible before the next IMP in case of AESM of severe headache and/or AESM of 
severe GI disorders, regardless of severity at Visits 4, 5, 6, 8, 9. 
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) and serum cytokines  
• Plasma complement (C3a, C5a) 
• Tetanus toxoid IgG 
Samples for IgA, IgE, IgM are collected predose.  
Samples for serum complement (C3, C4), plasma complement (C3a, C5a) and serum cytokines 
are collected predose at Visit 2 and 4 hours postdose at Visit 9. Additional samples should be 
collected 2 hours and 4 hours postevent for study participants with severe headache and/or 
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infusion reaction or hypersensitivity reaction as specified in the Schedule of Activities 
(Section 1.3).  
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 parameters 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 Statistical 
Analysis Plan (SAP). 
The statistical design of this study involves two stages, with an interim analysis at the end of 
Stage 1. 
9.1 
Definition of Analysis Sets 
• Enrolled Set: All study participants who have signed the informed consent. 
• Randomized Set (RS): All enrolled study participants who were randomized. 
• Safety Set (SS): All randomized study participants who received at least one dose of IMP. 
Analysis of this set will be according to the treatment the study participants actually received, 
and will be used for the demographic and safety analyses. 
• Pharmacokinetic Per-Protocol Set (PK-PPS): A subset of the Safety Set, consisting of those 
study participants who received 1 dose, had at least 1 valid PK measurement post first dose 
of study treatment, and no important protocol deviations affecting the PK variable, as 
confirmed during a pre-analysis review of the data prior to database lock. 
• Full Analysis Set (FAS): Consists of all study participants in the SS, who have a Baseline 
and least 1 post-Baseline MG-ADL measurement. 
• The RS is the primary analysis set for efficacy analyses. Analysis of the RS will be according 
to the treatment to which the study participants were randomized.  
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 as follows:  
• Summarized by dose levels of rozanolixizumab or placebo 
All analyses will be performed using Statistical Analysis System (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 (n), mean, standard 
deviation (SD), median, minimum, and maximum. Categorical variables will be summarized by 
visit (where applicable) with frequency counts and percentages. 
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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. Placebo-treated study participants will be summarized globally. 
9.3 
Estimands 
The primary efficacy analysis will evaluate the ‘hypothetical’ estimand in anti-AChR or anti-
MuSK autoantibody-positive participants with generalized MG who experience moderate to 
severe symptoms and are being considered for additional treatment such as IVIg or PEX, where 
the main intercurrent events are the use of rescue medication prior to Day 43 and treatment 
discontinuation (or withdrawal from study) due to TEAEs. The primary efficacy analysis is the 
change from Baseline in MG-ADL score to Day 43 (Visit 10).  
A hypothetical strategy will be used for rescue medication, ie, the intervention effect will be 
estimated under the hypothetical condition where rescue medication is not given.  In the case of 
TEAEs leading to treatment discontinuation, observed data after treatment discontinuation will 
be utilized where possible. 
The reason why a hypothetical estimand approach has been selected as the primary approach is 
because the use of rescue medication is likely to have a significant effect on subsequent efficacy 
data and for those study participants who discontinue treatment due to AEs, they are likely to 
also commence rescue medication in which case the hypothetical strategy will be applied once 
rescue medication has been initiated. 
Missing data will be imputed under a missing at random assumption. 
Data handling conventions for data affected by COVID-19 will be detailed fully in the SAP. 
To check the assumptions of the primary analysis and definition of the Estimand, sensitivity and 
supplemental analyses of the change from Baseline in MG-ADL score to Day 43 (Visit 10) will 
be provided and are detailed in Table 9-1.  
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Table 9-1: 
Estimand Details and Attributes 
 
 
Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
Primary Objective: To demonstrate the clinical efficacy of rozanolixizumab in patients with gMG 
MG-ADL 
Primary 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
MG-ADL 
score 
RS 
The main intercurrent 
events are the use of 
rescue therapy prior 
to Day 43 (Visit 10) 
and treatment 
discontinuation (or 
withdrawal from 
study) due to TEAEs. 
A hypothetical 
strategy will be used 
for rescue therapy, ie, 
the intervention effect 
will be estimated 
under the 
hypothetical 
condition where 
rescue therapy is not 
given. The same 
strategy will be used 
for TEAEs leading to 
treatment 
discontinuation. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be Missing at Random 
(MAR). 
MG-ADL 
Sensitivity 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
MG-ADL 
score 
FAS 
Hypothetical 
strategy, as for the 
primary analysis. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
MG-ADL 
Sensitivity  
Change from 
Baseline to 
Day 43 
(Visit 10) in 
MG-ADL 
score 
RS 
Hypothetical 
strategy, as for the 
primary analysis. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. A 
Jump-to-reference 
multiple imputation 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
approach will be 
utilised to assess the 
validity of the MAR 
assumption. Missing 
data across all 
treatment groups will 
be imputed using the 
placebo distribution. 
MG-ADL 
Supplemental 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
MG-ADL 
score 
 
RS 
The main intercurrent 
events are the use of 
rescue therapy prior 
to Day 43 (Visit 10) 
and treatment 
discontinuation (or 
withdrawal from 
study) due to due to 
TEAEs. The 
intercurrent events 
will be handled using 
a composite 
strategy, ie, the 
occurrence of an 
intercurrent event 
will be handled by 
evaluating the 
corresponding 
participants as 
treatment failures and 
imputed with a worst 
score. 
A trimmed mean 
approach will be used 
where all missing data 
(including the main 
intercurrent events) 
will be imputed with 
the worst score. A 
permutation-based test 
will be carried out, 
with the % trimming 
applied adaptively. The 
mean treatment 
difference of the 
trimmed population 
will be calculated on 
covariate-adjusted 
scores which are 
derived from an 
ANCOVA analysis of 
completers data. 
MG-ADL 
Sensitivity  
Change from 
Baseline to 
Day 43 
(Visit 10) in 
MG-ADL 
score 
Subgroup of 
the RS who 
received all 
6 (sc) doses. 
Only those 
participants 
who 
complete a 
full 
treatment 
course will 
be included 
in the 
analysis. 
Hypothetical 
strategy, as for the 
primary analysis. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
MG-ADL 
Supplemental 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
RS 
The main intercurrent 
events are the use of 
rescue therapy prior 
to Day 43 (Visit 10) 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
MG-ADL 
score 
 
and treatment 
discontinuation (or 
withdrawal from 
study) due to TEAEs. 
The intercurrent 
events will be 
handled using a 
treatment policy 
strategy, whereby 
the data from the 
treatment period to 
Day 43 is used 
regardless of whether 
the intercurrent event 
occurred. 
(Visit 10) in the MG-
ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
 
MG-ADL 
Secondary 
MG-ADL 
responder 
(≥2.0 points 
improvemen
t from 
Baseline) at 
Day 43 
(Visit 10) 
RS 
The main intercurrent 
events are the use of 
rescue therapy prior 
to Day 43 (Visit 10) 
and treatment 
discontinuation (or 
withdrawal from 
study) due to TEAEs. 
The intercurrent 
events will be 
handled using a 
composite strategy, 
ie, the occurrence of 
an intercurrent event 
will be handled by 
evaluating the 
corresponding 
participants as 
nonresponders. 
Odds ratio at Day 43 
(Visit 10) analyzed 
using logistic 
regression model 
adjusting for covariates 
for participants 
receiving 
rozanolixizumab versus 
placebo. 
MG-C 
Secondary 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
MG-C score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
MG-ADL endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the MG-C 
score for participants 
receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
MG-C 
Sensitivity 
Change from 
Baseline to 
RS 
Hypothetical 
strategy, as 
Difference in mean 
change from Baseline 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
Day 43 
(Visit 10) in 
MG-C score 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. A 
Jump-to-reference 
multiple imputation 
approach will be 
utilised to assess the 
validity of the MAR 
assumption. Missing 
data across all 
treatment groups will 
be imputed using the 
placebo distribution. 
MG-C 
Supplemental 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
MG-C score 
RS 
The main intercurrent 
events are the use of 
rescue therapy prior 
to Day 43 (Visit 10) 
and treatment 
discontinuation (or 
withdrawal from 
study) due to TEAEs. 
The intercurrent 
events will be 
handled using a 
composite strategy, 
ie, the occurrence of 
an intercurrent event 
will be handled by 
evaluating the 
corresponding 
participants as 
treatment failures and 
imputed with a worst 
score. For simplicity, 
other intercurrent 
events leading to 
missing data which 
are not necessarily 
related to treatment 
will be handled in the 
same way, since this 
will include all 
randomized 
participants and is 
A trimmed mean 
approach will be used 
where all missing data 
(including the main 
intercurrent events) 
will be imputed with 
the worst score. A 
permutation-based test 
will be carried out, 
with the % trimming 
applied adaptively. The 
mean treatment 
difference of the 
trimmed population 
will be calculated on 
covariate-adjusted 
scores which are 
derived from an 
ANCOVA analysis of 
completers data. 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
unlikely to introduce 
bias.  
QMG 
Secondary 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
QMG score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the QMG 
score for participants 
receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
QMG 
Sensitivity 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
QMG score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. A 
Jump-to-reference 
multiple imputation 
approach will be 
utilised to assess the 
validity of the MAR 
assumption. Missing 
data across all 
treatment groups will 
be imputed using the 
placebo distribution. 
QMG 
Supplemental 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
QMG score 
RS 
Composite strategy, 
as for the MG-C 
sensitivity analysis 
A trimmed mean 
approach will be used 
where all missing data 
(including the main 
intercurrent events) 
will be imputed with 
the worst score. A 
permutation-based test 
will be carried out, 
with the % trimming 
applied adaptively. The 
mean treatment 
difference of the 
trimmed population 
will be calculated on 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
covariate-adjusted 
scores which are 
derived from an 
ANCOVA analysis of 
completers data. 
MG 
Symptoms 
PRO 
 
Secondary 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO 
‘Muscle 
Weakness 
Fatigability’ 
score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the MG 
Symptoms PRO 
‘Muscle Weakness 
Fatigability’ score for 
participants receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
MG 
Symptoms 
PRO 
 
Sensitivity 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO 
‘Muscle 
Weakness 
Fatigability’ 
score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. A 
Jump-to-reference 
multiple imputation 
approach will be 
utilised to assess the 
validity of the MAR 
assumption. Missing 
data across all 
treatment groups will 
be imputed using the 
placebo distribution. 
MG 
Symptoms 
PRO 
 
Supplemental 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO 
‘Muscle 
Weakness 
RS 
Composite strategy, 
as for the MG-C 
sensitivity analysis 
A trimmed mean 
approach will be used 
where all missing data 
(including the main 
intercurrent events) 
will be imputed with 
the worst score. A 
permutation-based test 
will be carried out, 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
Fatigability’ 
score 
with the % trimming 
applied adaptively. The 
mean treatment 
difference of the 
trimmed population 
will be calculated on 
covariate-adjusted 
scores which are 
derived from an 
ANCOVA analysis of 
completers data. 
MG 
Symptoms 
PRO  
Secondary 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO 
‘Physical 
Fatigue’ 
score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the MG 
Symptoms PRO 
‘Physical Fatigue’ 
score for participants 
receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
MG 
Symptoms 
PRO 
 
Sensitivity 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO 
‘Physical 
Fatigue 
score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. A 
Jump-to-reference 
multiple imputation 
approach will be 
utilised to assess the 
validity of the MAR 
assumption. Missing 
data across all 
treatment groups will 
be imputed using the 
placebo distribution. 
MG 
Symptoms 
PRO 
Supplemental 
Change from 
Baseline to 
Day 43 
RS 
Composite strategy, 
as for the MG-C 
sensitivity analysis 
A trimmed mean 
approach will be used 
where all missing data 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
(Visit 10) in 
the MG 
Symptoms 
PRO 
‘Physical 
Fatigue’ 
score 
(including the main 
intercurrent events) 
will be imputed with 
the worst score. A 
permutation-based test 
will be carried out, 
with the % trimming 
applied adaptively. The 
mean treatment 
difference of the 
trimmed population 
will be calculated on 
covariate-adjusted 
scores which are 
derived from an 
ANCOVA analysis of 
completers data. 
MG 
Symptoms 
PRO  
Secondary 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO ‘Bulbar 
Symptoms’ 
score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the MG 
Symptoms PRO 
‘Bulbar Symptoms’ 
score for participants 
receiving 
rozanolixizumab versus 
placebo. Under 
MMRM, missing 
values are assumed to 
be MAR. 
MG 
Symptoms 
PRO 
 
Sensitivity 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO ‘Bulbar 
Symptoms’ 
score 
RS 
Hypothetical 
strategy, as 
described for the 
primary analysis of 
the MG-ADL 
endpoint. 
Difference in mean 
change from Baseline 
(LSMD from CfB 
MMRM) to Day 43 
(Visit 10) in the 
MG-ADL score for 
participants receiving 
rozanolixizumab versus 
placebo. A 
Jump-to-reference 
multiple imputation 
approach will be 
utilised to assess the 
validity of the MAR 
assumption. Missing 
data across all 
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Estimand 
Objective 
Clinical 
Category 
Statistical 
Category 
Variable/ 
Endpoint 
Population 
IES 
PLS (Analysis) 
treatment groups will 
be imputed using the 
placebo distribution. 
MG 
Symptoms 
PRO 
Supplemental 
Change from 
Baseline to 
Day 43 
(Visit 10) in 
the MG 
Symptoms 
PRO ‘Bulbar 
Symptoms’ 
score 
RS 
Composite strategy, 
as for the MG-C 
sensitivity analysis 
A trimmed mean 
approach will be used 
where all missing data 
(including the main 
intercurrent events) 
will be imputed with 
the worst score. A 
permutation-based test 
will be carried out, 
with the % trimming 
applied adaptively. The 
mean treatment 
difference of the 
trimmed population 
will be calculated on 
covariate-adjusted 
scores which are 
derived from an 
ANCOVA analysis of 
completers data. 
ANCOVA=analysis of covariance; CfB=Change from Baseline; FAS=Full Analysis Set; IES=intercurrent 
event(s) strategy; gMG=generalized myasthenia gravis; LSMD=Least Squares Mean Difference; MAR=Missing 
at Random; MG=Myasthenia Gravis; MG-ADL=Myasthenia Gravis-Activities of Daily Living; 
MG-C=Myasthenia Gravis Composite; MMRM=mixed model for repeated measures; PLS=Population-level 
summary; PRO=patient-reported outcome; QMG=quantitative myasthenia gravis; RS=Randomized Set; 
sc=subcutaneous; TEAE=treatment-emergent adverse event.  
9.4 
Planned Efficacy/Outcome Analyses  
9.4.1 
Analysis of the primary efficacy endpoint 
Analysis of the primary efficacy variable 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.  
The formal testing strategy proposed is a flexible combination test based on the inverse normal 
method (Lehmacher and Wassmer, 1999) with equal weighting of Stage 1 (up to the formal 
interim analysis) and Stage 2 (post-interim analysis) data. Confirmatory testing of single 
hypotheses is based on a closed-testing procedure. For the closed testing procedure the 
Bonferroni approach is used as the intersection test for testing the global and intersection 
hypotheses. If a dose other than that defined by the “Select the best dose (and any dose with a 
mean response not worse than 1 point than the selected “best dose”)" option (see Section 9.8 for 
details) is chosen for Stage 2 the adjusted p-value for the chosen dose(s) will be used in the 
combination test.  
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The primary efficacy analysis of the study will include the cohort of study participants from 
Stage 1 (n=
) and those who received the selected doses in Stage 2. The primary 
endpoint, Change from Baseline in MG-ADL, will be analyzed by stage-wise mixed model for 
repeated measures (MMRM) ANCOVA including a term for Baseline MG-ADL score, region, 
treatment group, the treatment group by week interaction term, and a term for the stratification 
factor (categorized as AChR or MuSK). The model will define patient as a random effect and 
utilize an unstructured covariance pattern. If the model does not converge using the unstructured 
pattern then an autoregressive covariance structure will be used. 
The t-test statistics for each dose group (at each stage) will be used to calculate unadjusted p-
values and these will be used to form the p-value combination test(s) as described above. The 
multiplicity introduced by having potentially 2 doses at the end of the study will be addressed by 
using a Bonferroni approach. The critical value for this test will be the upper 1.25% percentile of 
the standard normal distribution (because no alpha will be spent in Stage 1, the futility criterion 
is not binding and in Stage 2 the alpha is divided between the 2 doses). Similar presentations 
within each stratification factor may be provided.  
9.4.2 
Analysis of secondary and other efficacy endpoints 
All continuous secondary efficacy variables will be analyzed using the combination testing 
strategy and model defined for the primary analysis. The binary secondary efficacy variable of 
MG-ADL responder (≥2.0 points improvement from Baseline) at Day 43 (Visit 10) will be 
analyzed using logistic regression, including a term for Baseline MG-ADL score, treatment 
group, and a term for the stratification factor (categorized as AChR or MuSK). 
In addition, all continuous secondary efficacy variables will be listed and summarized 
descriptively by treatment group and week. Descriptive statistics will be generated for the 
observed values and the change from Baseline. For the binary variable, frequency counts and 
percentages will be produced.  
A sequential hierarchical test procedure will be applied to protect the overall significance level 
for the multiplicity of endpoints. The predefined order of formal hypotheses testing for the 
secondary endpoints will be performed in the following sequence: 
• Change from Baseline to Day 43 (Visit 10) in MG-C score. 
• Change from Baseline to Day 43 (Visit 10) in QMG score. 
• Change from Baseline to Day 43 (Visit 10) in the MG Symptoms PRO ‘Muscle Weakness 
Fatigability’ score. 
• Change from Baseline to Day 43 (Visit 10) in the MG Symptoms PRO ‘Physical Fatigue’ 
score. 
• Change from Baseline to Day 43 (Visit 10) in the MG Symptoms PRO ‘Bulbar Symptoms’ 
score. 
All other variables will be listed and summarized descriptively. Time to first MG-ADL response 
(defined as ≥2.0 point improvement from Baseline) will be analyzed using a Cox Proportional 
Hazards model including a fixed term for treatment, and a term for the stratification factor 
(categorized as AChR or MuSK). The statistical model will be applied to each stage and the 
results combined using in a similar fashion to the primary analysis. An overall study used to 
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calculate the Hazard Ratio (test/reference), 95% CI and p-values for each treatment comparison 
of interest will be presented. The time to first MG-ADL response will be presented graphically 
using a Kaplan-Meier curve. Study participants will be censored at time of initiation of rescue 
therapy or end of treatment.  
Further exploratory analysis may be performed using adequate statistical methods; details will be 
provided in the SAP. 
9.5 
Planned Safety and other Analyses 
9.5.1 
Safety analyses 
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 study participants experiencing 
the TEAE, percentage of study participants, and number of TEAEs. A TEAE is defined as any 
event that was not present prior to the first administration of IMP or any unresolved event 
already present before the first administration of IMP that worsens in intensity following 
exposure to treatment. 
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. 
9.5.2 
Other analyses 
9.5.2.1 
Pharmacokinetic analyses 
The PK-PPS dataset will be used. Pharmacokinetic variables of rozanolixizumab like AUC (area 
under the curve), Cmax (maximum concentration) cannot be derived, since blood sampling will be 
performed at 1 time point post-dosing per visit only. Thus, PK is restricted to concentration data. 
In addition to the general descriptive display, 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). The concentration data will be also summarized 
by treatment group and ADA. Individual concentrations of rozanolixizumab will also be 
displayed graphically. 
A population PK and population PKPD analysis will be performed to evaluate PK and PKPD of 
rozanolixizumab. Details of such analysis will be further described in a separate Data Analysis 
Plan. The results of the analysis would be reported in a separate report outside the CSR. If 
required, an unblinded and relevant data transfer may be planned in order to initiate the PKPD 
analysis.  
9.5.2.2 
Anti-drug antibodies analyses 
A tiered ADA approach will be used for the study. Anti-drug antibody assessment 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-
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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. 
The impact of ADA will be evaluated on PK, PD, efficacy and safety endpoints when relevant. 
9.6 
Handling of protocol deviations 
Important protocol deviations are identified as part of the data cleaning process in the Data 
Cleaning Plan (DCP). 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 DCP. Furthermore, overall trends in protocol deviations will be 
discussed at the Data Evaluation Meeting. Through this ongoing data cleaning and evaluation 
process, all decisions regarding important protocol deviations are made on an ongoing basis. 
9.7 
Handling of dropouts or missing data 
All imputation of missing or partial dates for safety assessments, as well as handling missing 
efficacy data (where applicable), will be detailed in the SAP. 
9.8 
Planned interim analysis and data monitoring 
9.8.1 
Interim analysis  
A formal interim analysis is planned for this study at the end of Stage 1, once approximately 
(see Section 9.9 for sample size details) enrolled study participants 
) are evaluable 
for the primary endpoint (having completed up to Day 43). Study enrollment will not be halted 
during planned IDMC review of the safety and efficacy data. The interim analysis is based on an 
adaptive design and is based on the primary efficacy analysis of Change from Baseline in MG-
ADL. Data will be presented to and reviewed by the IDMC and the following decisions will be 
made:  
1. Early stopping for futility will be allowed in the event that there is no evidence of either dose 
of rozanolixizumab being effective.  
2. If futility is not established, then the “best dose” of rozanolixizumab (and any dose with a 
mean response not worse than 1 point than the selected “best dose”) is selected for Stage 2 of 
the study. See dose selection section below for further details. 
3. Sample size re-estimation will be conducted to ensure that the sample size in Stage 2 is 
appropriate to ensure an overall conditional power of 
 or until a predefined maximum 
increase in sample size has been reached. See Section 9.9 for further details. 
Early stopping for Efficacy 
Early stopping for efficacy will not take place, ie, there is no alpha spending function 
implemented. 
Early Stopping for Futility  
The futility stopping rule (non-binding) utilized for the study requires that the nominal Stage 1 
p-value of each dose to be greater than 
 This corresponds to both estimated treatment effects 
being in the wrong direction, ie, inferiority. 
Dose Selection  
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The statistical selection procedure proposed is equivalent to the “Select arm compared to the best 
and not worse than epsilon” option of ADDPLAN MC, where epsilon is equal to 1. This 
approach selects the dose with largest effect defined in terms of the observed mean responses 
and any doses with a mean response no worse than the best dose by 1 point providing that they 
are not dropped due to futility, ie, to be retained nominal p-value must be less than
 However, 
this may not be the sole criterion for selecting the dose, since, additionally, the IDMC will take 
account of factors, including safety, which will be detailed in the IDMC charter. 
Sample Size Re-estimation  
The sample size re-estimation will be performed with the only option to increase the sample size. 
The default sample size of
 for the study 
study participants per arm in each of Stage 1 and 
Stage 2) is based on a set of assumptions concerning the effect sizes and variability (see 
Section 9.9 for details). However, it is considered prudent to conduct a mid-study sample size re-
estimation to assess these important assumptions. The approach to be used is to determine the 
Stage 2 sample size required to give a conditional power of 
 for the overall study, based on 
the observed effect sizes at the interim analysis. 
9.8.2 
Periodic Data Reviews 
Approximately 3 periodic data reviews (in addition to the futility analysis) will be performed for 
the IDMC to oversee the safety of the study: the first periodic data review will be performed 
when approximately
 study participants have completed the 6-week Treatment Period; the 
second periodic data review will be conducted after approximately 
study participants have 
completed the 6-week Treatment Period (ad hoc as needed); and a third periodic data review will 
be conducted after approximately 
 study participants (dependent on the outcome of the 
interim analysis) have completed the 6-week Treatment Period. The timing of any further data 
reviews will be decided by the IDMC in conjunction with sponsor. 
9.8.3 
Data monitoring 
Data from the interim analysis will be presented to and reviewed by the IDMC by an unblinded 
statistician who will receive data from an independent statistical center. All information 
including electronic data capture, randomization and drug kit numbers are contained in a single 
database. The randomization and drug kit numbers are stored in encrypted form on the database 
and access to the database is strictly controlled by function so that project team members do not 
have access to the encryption key. The unblinded, independent statistician will provide the 
IDMC with information and will update the randomization lists following the choice of dose by 
the IDMC. The IDMC will evaluate efficacy by reviewing pre-specified efficacy data during the 
interim evaluation and make recommendations on implementation of the adaptive design options 
as detailed in Section 9.8. 
The IDMC will also oversee the safety of the study by reviewing safety and efficacy data at 
periodic data reviews to assess the benefit risk of rozanolixizumab.  
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.  
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9.9 
Determination of sample size 
As detailed in in Section 9.8, this study may consist of 2 stages, with a formal interim analysis at 
the end of Stage 1.  
Based on historical data, the difference in adjusted changes from Baseline of MG-ADL at Day 
43, between rozanolixizumab and placebo, is assumed to be 
 to 
 and the standard deviation 
is assumed to be 
 to  A difference of 
 could be judged to be clinically meaningful. 
It is proposed that the interim analysis will be conducted when approximately
 eligible study 
participants have been treated and are evaluable for the primary endpoint, ie, approximately 
 
study participants per dose group in Stage 1. If the study is not stopped for futility after Stage 1, 
then depending upon the selection of one, or two, of the doses after Stage 1 and the calculation of 
conditional power, a further 
 to
 eligible study participants will be randomized in Stage 2 
of the study. Thus, the total sample size of the study could range between 
 and 
study 
participants if the study is not futile. 
The power of the study using the above adaptive design and expected sample sizes with a sample 
size cap of 
 and various scenarios of true treatment effect and standard deviation were 
determined from a stimulation study using the statistical software ADDPLAN MC, the results of 
which are summarized in Table 9-2.  
The simulation results also indicate that across the various scenarios the average sample size for 
the study ranges between 
 and 
 study participants. The power for scenarios
 
to 
 that is, when the high dose has an effect compared to placebo of 2 points, are at least
 
indicating that the sample size re-assessment process helps maintain reasonable power when only 
one dose has a clinically meaningful effect of 2 and the data variability is greater than anticipated 
  
The chances of stopping for futility, when at least one dose has a clinically meaningful effect of 
or more versus placebo, is 
  
These results support the strategy of entering 
eligible study participants per dose group in 
Stage 1 and between 
 to 
study participants per dose group in Stage 2. Note that ADDPLAN 
MC implements the sample size increase rule ‘per arm’ rather than ‘per stage’, so that if only one 
dose is selected for Stage 2, then the maximum sample size for this stage is
 whereas if two 
doses are selected than the maximum Stage 2 sample size is 
 Therefore, if an overall study 
sample size cap of
 (ie,
 in Stage 2) were implemented, irrespective of whether one or two 
doses are selected, then we would expect power for each scenario to be slightly increased 
compared to those contained in Table 9-2. 
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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, 
International Council on Harmonisation-Good Clinical Practice (ICH-GCP), and in accordance 
with the ethical principles that have their origin in the Declaration of Helsinki. 
UCB or designee 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, UCB or designee will forward copies of the protocol, Informed Consent 
form, Investigator’s Brochure, 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. 
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 CRO agreements, as applicable. 
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10.1.3 
Informed consent process 
Participant’s informed consent, as well as a separate informed consent for the sub-study, 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. A 
separate informed consent may be obtained requesting the participation in a market research 
interview. 
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 Informed Consent form 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 Informed Consent form. 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 Informed Consent form 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 Informed Consent form by the IRB/IEC and use of the amended form. 
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 as enrolled in the study when he/she has signed the Informed Consent 
form. A CRF 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 study participant’s confidentiality. 
Throughout this study, all data forwarded to UCB (or designee) will be identified only by the 
study 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. 
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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 to decide on whether to proceed with the study. 
The data review by the IDMC will be unblinded data.  
An IDMC will be set up in line with the Food and Drug Administration regulatory requirements 
and European Medicines Agency 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 CRO, and have no conflict of interest related to the conduct or the 
outcomes of the study. The voting members of the IDMC will include, at minimum, an expert in 
clinical pharmacology, and two neurologists. 
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. 
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. 
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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 
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 
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• Discontinuation of further study medication development 
10.1.9 
Publication Policy 
The results of this study may be published or presented at scientific meetings.  
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. 
• 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: 
Mean corpuscular 
volume 
Mean corpuscular 
hemoglobin 
%Reticulocytes 
White blood cell count 
with Differential: 
Neutrophils 
Lymphocytes 
Monocytes 
Eosinophils 
Basophils 
Red blood cell (RBC) 
count 
Hemoglobin 
Hematocrit 
Clinical 
Chemistrya 
Blood Urea 
Nitrogen 
(BUN) 
Potassium 
Aspartate Aminotransferase 
(AST)/ Serum Glutamic-
Oxaloacetic Transaminase 
(SGOT) 
Total and 
direct 
bilirubin 
 
Creatinine 
Sodium 
Alanine Aminotransferase 
 (ALT)/ Serum Glutamic-
Pyruvic Transaminase 
(SGPT) 
Total Protein 
Albumin 
 
Glucose 
(fasting state, 
preferred)  
Calcium 
Alkaline phosphatase 
C-reactive 
protein (CRP) 
Lactate 
dehydrogenase 
(LDH) 
High-
density 
lipoprotein 
(HDL) 
Total Cholesterol 
Triglycerides 
Routine 
Urinalysis 
• Specific gravity 
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Laboratory 
Assessments 
Parameters 
• pH, glucose, protein, blood, ketones, albumin, bilirubin, urobilinogen, 
nitrite, leukocyte esterase by dipstick 
• Microscopic examination (if blood or protein is abnormal) 
Other 
Screening 
Tests 
• Serum or urine human chorionic gonadotropin pregnancy test (as needed 
for women of childbearing potential)b 
• PTT and INR tests 
• Serology testing (for Hepatitis B, Hepatitis C, and HIV) 
• All study-required laboratory assessments will be performed by a central 
laboratory.  
• The results of each test must be entered into the CRF. 
NOTES : 
a Details of liver chemistry stopping criteria and required actions and follow-up assessments 
after liver stopping or monitoring event are given in Section 10.6 and Appendix 6. All events 
of ALT 3xULN and bilirubin 2xULN (>35% direct bilirubin) or ALT 3xULN and 
international normalized ratio (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. 
Laboratory/analyte results that could unblind the study will not be reported to investigative sites 
or other blinded personnel until the study has been unblinded. 
 
 
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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 AE 
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.  
 
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. 
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• 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 SAE 
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). 
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. 
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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. 
10.3.3 
Recording and Follow-Up of AE and/or SAE 
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. 
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• 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 (NCI-
CTCAE) 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.  
 
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 Investigator’s Brochure (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 an SAE follow-up report with the updated causality assessment. 
• The causality assessment is one of the criteria used when determining regulatory 
reporting requirements. 
 
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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 AEs of special 
interest. 
• 
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 CRF. 
• The Investigator will submit any updated SAE data to UCB within 24 hours of receipt 
of the information. 
10.3.4 
Reporting of SAEs 
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 it becomes 
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. 
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• 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 
Definitions 
Woman of Childbearing Potential (WOCBP) 
A woman is considered fertile following menarche and until becoming post-menopausal 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 non-estrogen 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. 
Contraception Guidance 
Male participants 
Male participants with female partners of childbearing potential are eligible to participate if they 
agree to ONE of the following during the 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 when having penile-vaginal intercourse with a woman of 
childbearing potential who is not currently pregnant. 
In addition, male participants must refrain from donating sperm for the duration of the study and 
for at least 90 days after the final dose of study medication. 
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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 
throughout the duration of the study and for at least 90 days after the final dose of study 
treatment. 
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 Dependentb 
Failure rate of <1% per year when used consistently and correctly.  
Combined (estrogen and progestogen containing) hormonal contraception associated with 
inhibition of ovulationc  
• Oral  
• Intravaginal  
• Transdermal  
Progestogen only hormonal contraception associated with inhibition of ovulation 
• Oral  
• Injectable  
Highly Effective Methods That Are User Independentb 
Implantable progestogen only hormonal contraception associated with inhibition of ovulation 
• Intrauterine device  
• Intrauterine hormone-releasing system  
• Bilateral tubal occlusion 
Vasectomized partner  
A vasectomy is a highly effective contraception method provided that the vasectomized 
partner is the sole male sexual partner of the WOCBP 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.  
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NOTES: 
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 instructions 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.  
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 
• WOCBP should only be included after a confirmed menstrual period and a negative highly 
sensitive urine or serum pregnancy test. 
• Additional pregnancy testing should be performed once during the treatment period and 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. A serum and urine 
pregnancy test will be completed at Screening and Baseline, respectively. 
 
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. If the study participant is later found 
to be on placebo, then pregnancy data collection can stop. 
• 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 once working day 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. 
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 one working day 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 
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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 IMP by the investigator will be 
reported to the sponsor as described in Section 8.3.5. While the 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 potential drug-induced liver injury 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 potential drug-induced liver injury 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 CRF 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 >5 ULN. The monitoring plan should include 
any necessary follow-up assessments (until resolution of the abnormal lab values).  
Table 10‒2:  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 
Bilirubina, b 
ALT 3xULN and bilirubin 2xULN (>35% direct bilirubin)  
INR2  
ALT 3xULN and international normalized ratio (INR) >1.5, if INR 
measured 
Cannot 
Monitor 
ALT 5xULN but <8xULN and cannot be monitored weekly for 2 weeks 
ALT 3xULN but <5xULN and cannot be monitored weekly for 4 weeks 
Symptomaticc 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 
• Immediately discontinue study medication. 
• Report the event to UCB within 24 hours. 
• Complete the liver event case report form 
(CRF), and complete a serious adverse 
event (SAE) data collection tool if the event 
also met the criteria for an SAE.b 
• Viral hepatitis serologyd 
• 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 hepatitis B surface antigen, 
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• 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, 
aspartate transaminase [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. 
quantitative hepatitis B deoxyribonucleic 
acid (DNA) and hepatitis delta 
antibodye) 
• 
Obtain blood sample for 
pharmacokinetic (PK) analysis as soon 
as feasible after the most recent dosef 
• Serum creatine phosphokinase and 
lactate dehydrogenase (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 adverse event 
(AE) report form 
• Record use of concomitant medications 
(including acetaminophen, herbal 
remedies, and other over-the-counter 
medications) on the concomitant 
medications CRF. 
• Record alcohol use on the liver event 
alcohol intake CRF 
• 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.  
• Liver imaging (ultrasound, magnetic 
resonance, or computerized tomography) 
and/or liver biopsy to evaluate liver 
disease; complete Liver Imaging and/or 
Liver Biopsy CRFs. 
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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 immunoglobulin M (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 (PCR) 
of hepatitis D RNA virus (where needed) [Le Gal et al, 2005].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 CRF. 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. 
Table 10‒3: 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 and <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 
AE 
adverse event 
AESM 
adverse event of special monitoring 
AESI 
adverse event of special interest 
ALP 
 
alkaline phosphatase 
ALT 
alanine aminotransferase 
AST 
aspartate aminotransferase 
BP 
blood pressure 
BUN 
blood urea nitrogen 
CI 
confidence interval 
CIDP 
chronic inflammatory demyelinating polyradiculoneuropathy 
COVID-19 
coronavirus disease 2019 
CPMP 
Committee for Proprietary Medicinal Products 
CRF 
Case Report form 
CRO 
contract research organization 
C-SSRS 
Columbia Suicide Severity Rating Scale 
CTCAE 
Common Terminology Criteria for Adverse Events 
DCP 
Data Cleaning Plan 
DMC 
Data Monitoring Committee 
DNA 
deoxyribonucleic acid 
ECG 
electrocardiogram 
EOS 
End of Study 
eCRF 
electronic Case Report form 
EQ VAS 
EuroQol visual analogue scale  
FAS 
Full Analysis Set 
FcRn 
neonatal Fc receptor 
FIH 
first in human 
FSH 
follicle stimulating hormone 
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FVC 
forced vital capacity 
GCP 
Good Clinical Practice 
GI 
gastrointestinal 
gMG 
generalized myasthenia gravis 
HBsAg 
Hepatitis B surface antigen 
HIV 
human immunodeficiency virus 
HPLC 
high performance liquid chromatography  
HRT 
hormonal replacement therapy 
IB 
Investigator’s Brochure 
IBD 
inflammatory bowel disease 
ICF 
Informed Consent form 
ICH 
International Council for Harmonisation 
ICH-GCP 
International Council for Harmonisation-Good Clinical Practice 
IDMC 
Independent Data Monitoring Committee 
IEC 
Independent Ethics Committee 
Ig 
immunoglobulin 
IGRA 
interferon-gamma release assay 
INR 
international normalized ratio 
IMP 
investigational medicinal product 
IRB 
Institutional Review Board 
IRT 
interactive response technology 
ITP 
immune thrombocytopenia purpura 
iv 
intravenous 
IVIg 
intravenous immunoglobulin 
LDH 
lactate dehydrogenase 
LLOQ 
lower limit of quantification 
LS 
least squares 
LTBI 
latent tuberculosis infection 
MedDRA 
Medical Dictionary for Regulatory Activities 
MG 
myasthenia gravis 
MG-ADL 
Myasthenia Gravis-Activities of Daily Living 
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MG-C 
Myasthenia Gravis Composite 
MGFA 
Myasthenia Gravis Foundation of America 
MG-QOL15r 
Myasthenia Gravis Quality of Life 
MGII 
Myasthenia Gravis Impairment Index 
MMRM 
mixed model for repeated measures 
MuSK 
muscle-specific kinase 
NCI-CTCAE 
National Cancer Institute Common Terminology Criteria for Adverse 
Events 
NMJ 
neuromuscular junction 
NTMBI 
nontuberculous mycobacterial infection 
OLE 
open-label extension 
PD 
pharmacodynamics(s) 
PEF 
peak expiratory flow 
PEOT 
premature end of treatment 
PEX 
plasma exchange 
PGI-C 
Patient Global Impression of Change 
PGI-S 
Patient Global Impression of Severity 
PK 
pharmacokinetic(s) 
PK-PPS 
Pharmacokinetic Per-Protocol Set 
PRO 
patient-reported outcome 
PTT 
partial thromboplastin time 
QMG 
quantitative myasthenia gravis 
RBC 
red blood cell 
RNA 
ribonucleic acid 
SAE 
serious adverse event 
SAP 
statistical analysis plan 
SAS 
Statistical Analysis System 
sc 
subcutaneous 
SD 
standard deviation 
SE 
standard error 
SOP 
Standard Operating Procedure 
SS 
Safety Set 
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TB 
tuberculosis 
TEAE 
treatment-emergent adverse event 
ULN 
upper limit of normal 
WHO 
World Health Organization 
WOCBP 
women of childbearing potential 
 
 
 
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10.11 
Appendix 11: Protocol Amendment History 
Amendment 3 (29 Jul 2020) 
Overall Rationale for the Amendment 
The primary reason for this protocol amendment is to introduce the MG0007 study as the 
open-label extension (OLE) study to MG0003 and closure of MG0004 once MG0007 study is 
available, decrease the complexity of assessments to be performed, clarify some operational 
aspects of the study, 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. 
Updated to provide clarity and be 
consistent with remainder of 
protocol. 
Global 
Where applicable, all reference to 
the rollover OLE study, MG0004, 
has been replaced to introduce the 
new OLE study, MG0007. 
The current ongoing OLE study, 
MG0004 (52-week chronic 
treatment) will be replaced by 
MG0007, which is a less complex 
study with 6-week treatment 
cycles based on myasthenia gravis 
(MG) worsening. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
An additional other efficacy 
endpoint has been added: “Minimal 
symptom expression Myasthenia 
Gravis-Activities of Daily Living 
(MG-ADL [score of 0 or 1]) at 
Day 43 (Visit 10)” 
This endpoint reflects the current 
scientific and medical interests. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
Other efficacy endpoints related to 
MG Impairment Index (MGII) 
scores have been moved to the end 
of the list. 
MGII has switched from 
mandatory to optional and will be 
performed at fewer study visits. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
An additional other safety endpoint 
has been added: “Suicidality as 
measured by the Columbia Suicide 
Severity Rating Scale (C-SSRS)” 
Updated to be consistent with the 
statistical analysis plan (SAP). 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
The pharmacodynamic endpoint for 
neurofilament-light (NF-L) levels 
has been removed. 
The endpoint was removed to 
decrease study complexity, as 
scientific value is limited.  
1.1 Objectives and 
Endpoints 
The objective relating to anti-drug 
antibody (ADA) was updated to 
include the impact it has on 
Updated for consistency with the 
clarifying text in Section 9.5.2.2. 
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Section # and Name 
Description of Change 
Brief Rationale 
3 Objectives and 
Endpoints 
pharmacokinetics (PK) and 
pharmacodynamics (PD). 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
Evaluation of the effects of 
rozanolixizumab on albumin and 
the α- and β-globulins was removed 
from the study objectives. 
The objective was amended to 
decrease study complexity, as 
scientific value limited. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
Evaluation of the effects of 
rozanolixizumab on serum 
cytokines was added to the study 
objectives. 
Updated for consistency within 
the protocol. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
‘Observation Period’ was removed 
from the endpoint relating to serum 
cytokines. 
Updated for consistency within 
the protocol. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
Assessment of the effect of 
rozanolixizumab on 
deoxyribonucleic acid (DNA) and 
ribonucleic acid (RNA) was 
removed from the study objectives 
and associated endpoints. 
The objective and endpoints were 
amended to decrease study 
complexity, as scientific value 
limited. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
Genetics was removed from the 
study objective and endpoint. 
The objective and endpoints were 
amended to decrease study 
complexity, as scientific value 
limited. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
The objective and endpoints for 
exploratory biomarkers was made 
more general. 
The objective and endpoints were 
amended to decrease study 
complexity, as scientific value 
limited. 
1.1 Objectives and 
Endpoints 
3 Objectives and 
Endpoints 
The table footnote was updated to 
include serious headache and to 
clarify that other safety biomarkers 
may be assessed. 
Updated for consistency within 
the protocol. 
1.1 Treatment groups and 
Duration 
4.1 Overall Design 
9.8.2 Periodic Data 
Reviews 
The timing of the second and third 
periodic data reviews has been 
amended. 
To ensure appropriate timing of 
periodic data reviews relative to 
the interim analysis. 
1.2 Schema 
The study schematic and footnote 
were updated. 
Updated to reflect changes and be 
consistent with the introduction of 
MG0007. 
1.3 Schedule of Activities 
Visits 11 and 13 have been changed 
to virtual visits. 
This change was made to decrease 
the complexity of the study. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
Optional pharmacogenomic 
sub-study consent has been 
removed. 
Decrease study complexity as 
scientific value. 
1.3 Schedule of Activities 
Optional PK and ADA sub-study 
consent updated to remove ADA. 
The sub-study does not include 
ADA. 
1.3 Schedule of Activities 
The hemoglobin A1c (HbA1c) test 
at screening has been removed from 
the study. 
Participants with diabetes are not 
excluded from the study and 
therefore this additional test is not 
required. 
1.3 Schedule of Activities 
Hematology, serum chemistry and 
urinalysis assessments have been 
removed at Visits 5 and 8. 
This change was made to decrease 
the complexity of the study and 
reduce the burden for the study 
participants. 
1.3 Schedule of Activities 
Measurement of partial 
thromboplastin time (PTT) and 
international normalized ratio 
(INR) was added for the Screening 
Visit (Visit 1). 
Updated to correct an error in the 
Schedule of Activities. 
1.3 Schedule of Activities 
Vital signs assessments have been 
removed at Visits 11 and 13. 
Removed because these visits 
have become virtual visits. 
1.3 Schedule of Activities 
Call or enter interactive response 
technology (IRT) was added for 
Visits 2, 3, 7, 11, 12 and 13. 
Updated to correct an error in 
protocol amendment 2. 
1.3 Schedule of Activities 
Blood sampling for PK has been 
removed at Visits 8 and 9. 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Blood sampling for exploratory 
safety biomarker analysis has been 
removed at Visits 4, 5, 6, 8 and 9. 
Updated to clarify that it is event 
driven only and not applicable to 
all study participants. 
1.3 Schedule of Activities 
The α‑ and β‑globulins have been 
removed from future exploratory 
biomarker analysis. 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Blood sampling for total 
immunoglobulin (Ig)G and IgG 
subclasses has been removed at 
Visit 3. 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Blood sampling for IgA, IgM and 
IgE has been removed at Visits 5 
and 8.  
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Blood sampling for DNA and RNA 
has been removed throughout the 
study. 
Updated to decrease the 
complexity of the study. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
Assessment of serum complement 
levels (C3, C4) and cytokines has 
been combined into one procedure. 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Assessment of serum complement 
levels (C3, C4), cytokines and 
plasma complement levels (C3a 
and C5a) has been removed from 
Visits 4, 5, 6 and 8. 
Updated to correct an error in the 
original protocol. 
1.3 Schedule of Activities 
Assessment of NF-L has been 
removed throughout the study. 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Assessment of MG Symptoms 
patient-reported outcome (PRO) 
has been removed at Visits 6 and 9. 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Assessment of EQ-5D-5L has been 
removed at Visit 14 (End of Study 
[EOS]). 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Assessment of MGII has been 
removed at Screening, Visit 4 and 
Visit 14 (EOS). In addition, it has 
become an optional assessment at 
the other visits.  
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Assessment of Myasthenia Gravis 
Quality of Life (MG-QOL)15r has 
been removed at Visit 6 and 
Visit 14 (EOS). 
Updated to decrease the 
complexity of the study. 
1.3 Schedule of Activities 
Abbreviations have been updated to 
reflect the changes made in the 
table. 
Updated for consistency within 
the protocol. 
1.3 Schedule of Activities 
The order of the footnotes has been 
updated to reflect the changes made 
in the table. 
Updated for consistency within 
the protocol. 
1.3 Schedule of Activities 
Footnote b: “This visit will be a 
virtual visit, unless the study 
participant prefers to attend the 
site.” has been added. 
Added to indicate that Visits 11 
and 13 are virtual visits. 
1.3 Schedule of Activities 
Footnote d: “Refer to Section 1.4.” 
has been added. 
Added to indicate the details can 
be found in that Section 1.4. 
1.3 Schedule of Activities 
Footnote k: (Assessment is optional 
for all study participants) has been 
removed. 
The instruction applied to blood 
sampling for DNA and RNA and 
no longer applies as the 
assessments were removed. 
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Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
Footnote l (previously footnote j): 
Updated to include severe headache 
and AESM of gastrointestinal (GI) 
disorders. 
Updated for consistency within 
the protocol. 
1.3 Schedule of Activities 
Footnote n: “postdose” was 
replaced with “postevent”. 
Updated to correct terminology 
error. 
1.3 Schedule of Activities 
Footnote o: “MGII is optional for 
all study participants.” has been 
added. 
MGII has switched from 
mandatory to optional for each 
study visit. 
1.4 Schedule of Activities 
(sub-study) 
Visit 3 has been corrected to 3a. 
Updated to correct an error in 
protocol amendment 2. 
1.4 Schedule of Activities 
(sub-study) 
Abbreviations have been updated to 
reflect the changes made in the 
table. 
Updated for consistency within 
the protocol. 
1.4 Schedule of Activities 
(sub-study) 
Details have been added specify 
that PK samples will only be 
collected for study participants who 
have consented to participate in the 
sub-study. 
Updated to clarify that the 
sub-study is optional. 
1.4 Schedule of Activities 
(sub-study) 
Footnote a: “Samples can be 
obtained at home, if appropriate.” 
has been added. 
To simplify and decrease 
complexity of the study. 
2.2 Background 
The number of rozanolixizumab 
clinical studies has been amended 
to include the 3 additional studies; 
TP0003, TP0006 and CIDP04. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
4.1 Overall Design 
The number of sites has been 
changed from 125 to 135. In 
addition, Asia has been added as a 
region for the study to be 
performed. 
Number of sites and countries 
have been expanded for the study 
to account for increased 
recruitment challenges 
during/post the COVID 19 
pandemic. 
4.1 Overall Design 
A new sentence has been added to 
explain that contingency measures 
during a pandemic and other 
exceptional circumstances has been 
put in place. 
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. 
5.1 Inclusion Criteria 
Inclusion criterion #5a: The 
MG-ADL score at screening was 
updated to clarify that participants 
had to have >3 points from 
non-ocular symptom. 
To ensure target population 
(generalized myasthenia gravis 
[gMG]) is fully captured. 
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Section # and Name 
Description of Change 
Brief Rationale 
5.2 Exclusion criteria 
Exclusion criterion #22b: 
glomerular filtration rate (GFR) 
entry criteria has been adjusted 
from less than 60ml/min/1.73m2 to 
less than 45ml/min/1.73m2 at 
Visit 1. 
To expand the population for a 
more generalized safety database 
and minimizes the risk of a 
potential effect unique to neonatal 
Fc receptor (FcRn) renal 
dynamics. 
5.2 Exclusion criteria 
Exclusion criterion #39a: Criteria 
updated to exclude all participants 
with a medical history of 
splenectomy. 
To minimize the potential risk of 
overwhelming post-splenectomy 
infection.  
5.4 Screen Failures 
Clarified when isolated test results 
outside the specific range at 
Screening need to be rechecked 
with the sponsor’s medical 
monitor/study physician. 
Clarification of procedure. 
6.3.1.1 Maintenance of 
study treatment blind 
New wording was included to 
clarify that treatment must be 
temporarily discontinued for any 
participant with IgG levels below 
1g/L and may be temporarily 
discontinued for any participant 
with IgG levels ≥1g/L and <2g/L 
with nonserious infection which is 
persisting or recurrent. 
Updated to clarify the procedure 
on when treatment discontinuation 
is required. 
6.5.1 Permitted 
concomitant treatments 
(medications and 
therapies) 
Footnote b updated to correct the 
units for cyclosporine from “ng/L” 
to “ng/mL”. 
Updated to correct an error in 
protocol amendment 2. 
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 
discontinuation is required to low 
IgG levels. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
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. 
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Section # and Name 
Description of Change 
Brief Rationale 
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. 
8.1.4 Patient-reported 
outcomes  
 
 
Text updated to clarify that MGII is 
an optional assessment. 
MGII has switched from 
mandatory to optional for each 
study visit. 
8.1.4.1 MG-Activities of 
Daily Living 
Details have been added explaining 
how the MG-ADL should be 
completed. 
This has been updated to ensure 
the avoidance of bias when 
completing the MG-ADL.  
8.1.4.6 MG Impairment 
Index 
Text updated to clarify that MGII is 
an optional assessment. 
MGII has switched from 
mandatory to optional for each 
study visit. 
8.2.6 Assessment and 
management of TB and 
TB risk factors  
A cross reference to the Schedule 
of Activities has been added. 
Updated for consistency within 
the protocol. 
8.2.6 Assessment and 
management of TB and 
TB risk factors 
The World Health Organization 
(WHO) Global Tuberculosis Report 
was updated to the 2019 version. 
Updated in line with the current 
version. 
 
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. 
8.7 Pharmacodynamics 
Assessment of serum NF-L levels 
have removed from the study. 
Updated due to limited scientific 
value and to decrease the 
complexity of the study. 
8.8.1 Pharmacogenomics 
This section has been deleted. 
Pharmacogenomics are not 
evaluated in this study. 
8.8.2 Immunology 
This section was moved from 
Section 8.8 and put into Section 8.9 
and has been renumbered as 8.9.1. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
8.9 Biomarkers 
Evaluation of the effects of 
rozanolixizumab on albumin, the α- 
and β-globulins, B-cell activating 
factor and Circulating Immune 
Complexes has been removed. 
Updated to decrease the 
complexity of the study. 
8.9 Biomarkers 
Updated to include serious or 
severe headache. 
Updated for consistency within 
the protocol. 
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Section # and Name 
Description of Change 
Brief Rationale 
8.9.1 Immunology 
Serum complement levels (C3, C4) 
and serum cytokines combined into 
one procedure. 
Updated to decrease the 
complexity of the study. 
8.9.1 Immunology 
For collection of additional 
samples, “postdose” was replaced 
with “postevent”. 
Updated to correct terminology 
error. 
8.11 Participant Exit 
Interview 
A new section has been added 
explaining that study participants 
may be asked to sign a separate 
informed consent for possible 
participation in a market research 
interview. 
Additional consent has been 
included with the aim to collect 
the study participant’s experience 
with MG and the perceived 
changes during the course of the 
study. 
9.3 Estimands 
A sentence was added to explain 
that data handling conventions for 
data affected by COVID-19 will be 
detailed fully in the SAP. 
Added to confirm that the 
potential effects of COVID-19 on 
the data analysis will be 
addressed. 
9.3 Estimands 
Table 9-1: Estimand Details and 
Attributes has been updated to be 
consistent with the SAP. 
Updated to be consistent with the 
SAP. 
9.3 Estimands 
Table 9-1: Estimand Details and 
Attributes: an additional sensitivity 
analysis has been added for 
MG-ADL.  
Added to assess the validity of the 
MAR assumption in the primary 
endpoint. 
9.3 Estimands 
Table 9-1: Estimand Details and 
Attributes: Details for secondary 
estimands have been added to the 
table. 
Updated to be consistent with the 
SAP. 
9.3 Estimands 
Abbreviations have been updated to 
reflect the changes made in the 
table. 
Updated for consistency within 
the protocol. 
9.5.2.2 Anti-drug 
antibodies analyses 
Text added to clarify that the 
impact of ADA will be evaluated 
on PK, PD, efficacy and safety 
endpoints when relevant. 
Updated to be consistent with the 
SAP. 
10.1.3 Informed consent 
process 
A sentence has been added 
explaining that study participants 
may be asked to sign a separate 
informed consent for possible 
participation in a market research 
interview. 
Additional consent has been 
included with the aim to collect 
the study participant’s experience 
with MG and the perceived 
changes during the course of the 
study. 
10.2 Appendix 2: Clinical 
Laboratory Tests 
The following parameter was 
added: “albumin”. 
Updated for consistency within 
the protocol. 
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Section # and Name 
Description of Change 
Brief Rationale 
10.2 Appendix 2: Clinical 
Laboratory Tests 
The HbA1c test at screening has 
been removed from the study. 
Participants with diabetes are not 
excluded from the study and 
therefore this additional test is not 
required. 
10.5 Appendix 5: 
Genetics 
All text related to genetic testing 
and analyzing have been removed. 
Genetics are not evaluated in this 
study. 
10.10 Appendix 10: 
Abbreviations and 
Trademarks 
Two additions (COVID-19 and 
PTT) and two deletions (NF-L and 
HbA1c) were made to the list of 
abbreviations. 
Updated to reflect the parameters 
measured in the study. 
10.11 Appendix 11: 
Protocol Amendment 
History 
Details of the previous amendment 
(protocol amendment 2) have been 
added. 
General update. 
10.23 Appendix 23: 
Management of Headache 
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. 
Updated for consistency within 
the protocol. 
10.24 Appendix 24: 
Management of Diarrhea 
Definition of severe (grade 3) 
diarrhea has been updated. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
10.25 Appendix 25: 
Management of Infections 
and 
Hypogammaglobulinemia 
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. 
10.25 Appendix 25: 
Management of Infections 
and 
Hypogammaglobulinemia 
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. 
11 References 
Order of references corrected. 
Updated to be consistent with the 
style guide. 
 
Amendment 2 (04 Mar 2020) 
Overall Rationale for the Amendment 
The primary reason for this protocol amendment is to incorporate the harmonization of inclusion 
criteria with studies performed across the rozanolixizumab development program, and include a 
new section to include temporary discontinuation of IMP in case of low IgG levels observed in 
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study participants. Furthermore, additional blood and pharmacokinetic samples, as well details 
pertaining to a sub-study have been incorporated to provide the opportunity to increase 
confidence in the PK exposure from the selected doses and help clinically validate the ADA 
assays drug tolerance through analysis of a drug onboard postdose sample as opposed to the 
pre-dose samples that are likely to be BLQ. 
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. 
Protocol amendment 2 was an internally approved document, which was not implemented at the 
study sites or submitted to the regulatory authorities. Protocol amendment 3 included changes 
related to COVID-19 and incorporates protocol amendment 2. 
Section # and Name 
Description of Change 
Brief Rationale 
Global 
Minor administrative, formatting, 
and typographical changes have been 
made. 
Updated to provide clarity and 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”. 
1.3 Schedule of 
Activities 
End of Treatment “EOT” was added 
to Visit 10.  
Updated to provide clarity that 
Visit 10 is not only a PEOT Visit.  
1.3 Schedule of 
Activities 
New activity was added: “Optional 
PK and ADA sub-study consent” 
Updated to due to the optional 
sub-study. 
1.3 Schedule of 
Activities 
The scheduled timepoint for blood 
sampling for ADA was changed from 
Visit 8 to Visit 7. 
Updated to correct an error in 
protocol amendment 1. 
1.3 Schedule of 
Activities 
Addition of “BL” and “EOT” to 
abbreviation list.  
Updated for consistency. 
1.3 Schedule of 
Activities 
Footnote c: Visit 14 (EOS) was 
removed as a scheduled visit for the 
collection of body weight.  
Updated to correct an error in 
protocol amendment 1. 
1.4 Schedule of 
Activities 
An additional SoA for the sub-study 
has been added to include additional 
PK samples.  
Updated to due to the optional 
sub-study. 
4.1 Overall Design 
The number of sites has changed 
from 106 to 125. 
The number of potential sites 
participating in the study has 
increased. 
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Section # and Name 
Description of Change 
Brief Rationale 
5.1 Inclusion Criteria 
Inclusion criterion #8a: All reference 
to 90 days should be preceded with 
“at least”. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
5.2 Exclusion Criteria 
Criterion #22b: Definition for renal 
impairment has changed with GFR 
less than “41”ml/min/1.73m. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
5.2 Exclusion Criteria 
Criterion #27a: chronic liver disease 
has been replaced with “hepatobiliary 
conditions”  
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
5.2 Exclusion Criteria 
Exclusion criterion #37: 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. 
5.4 Screen Failures 
The number of isolated tests result 
outside the specific range deemed 
clinically nonsignificant has changed. 
Updated to remove the limit and 
to be consistent across the Phase 3 
rozanolixizumab clinical program. 
6.3.1.1 Maintenance of 
study treatment blind 
Clarification that treatment will be 
discontinued for any participant with 
IgG levels below 1g/L or 2g/L with 
no infection on consectitive visits. 
Updated to clarify when treatment 
discontinuation is required. 
7.1.1 Liver Chemistry 
Stopping Criteria 
7.1.2 QTc Stopping 
Criteria 
A new sentence was added to 
emphasise study participants should 
follow the visit schedule as described 
in the protocol. 
New wording was added to 
provide clarity for each subsection 
under 7.1. 
7.1.4 Temporary IMP 
discontinuation 
New subsection has been added: 
Temporary IMP discontinuation, 
covering procedure for 
implementation of mock infusions. 
This section was added to include 
the management of temporary 
IMP discontinuation for low IgG 
values in any study participant 
8.2.1 Physical 
examination (full 
physical examination) 
New wording “Visit 1 and Baseline 
(Visit 2) only” has been added to 
replace at other timepoints.  
Updated to be consistent with the 
Schedule of Activities.  
8.6 Pharmacokinetics 
and Anti-drug 
Antibodies 
Additional wording has been 
provided to details on additional PK 
blood sampling: “Additional PK 
samples will be collected 
(Section1.4) at selected sites for 
study particpants who have 
consented to participate in the sub-
study” 
Updated to due to the optional 
sub-study. 
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Section # and Name 
Description of Change 
Brief Rationale 
8.8.2 Immunology 
“Tetanus toxoid IgG” was added to 
the list of measurements.  
Updated to be consistent with the 
Schedule of Activities. 
9.3 Estimands, Table 9-
1 
An additional sensitivity analysis of 
primary endpoint, MG-ADL was 
added.  
Included for study participants 
who need to miss doses due to 
low IgG levels. 
10.1.3 Informed consent 
process 
Additiotnal wording pertaining to the 
sub-study and required informed 
consent has been added.  
Updated to due to the optional 
sub-study. 
10.2 Appendix 2: 
Clinical Laboratory 
Tests 
The following tests have been 
removed at Screening: Serology 
testing (“for Hepatitis A”), and 
“serum or urine alcohol and drug 
screen (to include at minimum: 
amphetamines, barbiturates, cocaine, 
opiates, cannabinoids and 
benzodiazepines”. 
 
The following parameter was added: 
“C-reactive protein”. 
Updated to correct an error in 
protocol amendment 1. 
10.4 Appendix 4: 
Contraceptive Guidance 
and Collection of 
Pregnancy Information 
All reference to 3 months had been 
replaced by “90 days”. 
All reference to 90 days should be 
preceded with “at least”. 
Updated to be consistent across 
the Phase 3 rozanolixizumab 
clinical program. 
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. 
10.24 Appendix 24: 
Management of 
Diarrhea  
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. 
10.29 Appendix 29: 
Columbia-Suicide 
Severity Rating Scale 
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 added:  
5th ed. Arlington. Diagnostic and 
Statistical Manual of Mental 
Updated to maintain consistency. 
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Section # and Name 
Description of Change 
Brief Rationale 
Disorders. American Psychiatric 
Association. 2013. 
The following reference was 
removed: 
James LP, Letzig L, Simpson PM, 
Capparelli E, Roberts DW, Hinson 
JA, et al. Pharmacokinetics of 
acetaminophen-protein adducts in 
adults with acetaminophen overdose 
and acute liver failure. Drug Metab 
Dispos. 2009; 37:1779-84 
 
Amendment 1 (30 Oct 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 
has 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 
3 Objectives and 
Endpoints 
An additional other efficacy 
endpoint was added: “Time to first 
rescue therapy”. 
An additional exploratory objective 
and endpoint on tetanus IgG 
anibodies was added. 
The following secondary efficacy 
endpoint was amended to include 
specific text:  
Change from Baseline to Day 43 
(Visit 10) in the MG Symptoms 
patient-reported outcome (PRO) 
‘Muscle Weakness Fatigability’ 
score 
The following secondary efficacy 
endpoint was amended to remove 
specific text:  
Change from Baseline to Day 43 
(Visit 10) in the MG Symptoms 
“Time to first rescue therapy” 
added to further characterize 
rozanolixizumab clinical benefits. 
Tetanus IgG antibodies was added 
to assess the effect of 
rozanolixizumab on tetanus IgG 
antibodies. 
Other updates were implemented 
for consistency, as there were 
redundancies. 
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Section # and Name 
Description of Change 
Brief Rationale 
PRO ‘Physical Fatigue, Limb and 
Axial Weakness’ score 
The following other efficacy 
endpoints have been removed:  
Value and change from Baseline in 
the MG Symptoms PRO multi-
component total score, at each 
scheduled assessment during 
Treatment and Observation Periods 
Value and change from Baseline in 
the enhanced MG Symptoms PRO 
total score, at each scheduled 
assessment during Treatment and 
Observation Periods 
The term “Value” has been 
removed from all endpoints, where 
applicable. 
1.1 Synopsis  
4.1 Overall Design 
The description for the overall 
design has been amended.  
Additional text to provide follow-
up instructions for study 
participants who receive IVIg and 
discontinue the study. 
Updated wording has been included 
to provide further clarification of 
the overall study design. 
1.2 Schema 
The study schematic was updated. 
Update was made to reflect changes 
to study design. 
1.3 Schedule of Activities 
Additional visits (Visits 4, 5, 6, 8, 
9, 10, and 14) have been added for 
IRT registering.  
Visits have been added to the 
Schedule of Activities to be 
consistent with the study design. 
1.3 Schedule of Activities 
Blood sampling for 
pharmacokinetics at additional 
visits (Visits 4, 6, and 11) have 
been removed. 
Pharmacokinetic concentrations are 
expected to be Below the Limit of 
Quantitation at these visits. 
1.3 Schedule of Activities 
Additional visits (Visits 8 and 14 
[EOS]) have been added for blood 
sampling for ADA. 
For Visit 8, information on the 
onset of ADA will be obtained. For 
the EOS visit, information on the 
ADA persistency in the system 
once drug is not on board wil be 
obtained. 
1.3 Schedule of Activities 
Additional visits (Visits 4, 5, 6, 8, 
and 9) have been added for blood 
sampling for DNA and RNA. 
Visits have been added to the 
Schedule of Activities to be 
consistent with the study design. 
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 
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Section # and Name 
Description of Change 
Brief Rationale 
for the effect of rozanolixizumab on 
tetanus IgG antibodies. 
1.3 Schedule of Activities 
Urine drug screen procedure has 
been removed from the study. 
The exclusionary criterion 
pertaining to use of marijuana was 
removed. 
1.3 Schedule of Activities 
Myasthenia Gravis Impairment 
Index was added for the Baseline 
Visit (Visit 2). 
Missing activity for that visit.  
1.3 Schedule of Activities 
An additional visit was added for 
PGI-S. 
Visit 1 collection was added to 
capture Baseline data.  
1.3 Schedule of Activities 
Additional abbreviations previously 
missing have been added.  
General consistency. 
1.3 Schedule of Activities 
Footnote b: A visit window of ±4 
days has been added for Visits 12 
and 14. 
General consistency. 
1.3 Schedule of Activities 
Footnote c: Body weight will also 
be collected at Visit 14 (EOS). 
Updated to provide Baseline value 
for the open-label extension study. 
1.3 Schedule of Activities 
Footnote d: The addition of 
“Question 1” has been added to the 
existing wording.  
Updated for further clarity. 
1.3 Schedule of Activities 
Footnote g (any recreational or 
medicinal use of cannabis [ie, 
marijuana] or cannabidiols is not 
authorized in the study) has been 
removed.  
The use of cannabidiols and 
medicinal marijuana (prescribed by 
a physician) is now part of the 
permitted concomitant treatments 
list. 
1.3 Schedule of Activities 
Footnote h: The following was 
added: "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" 
The footnote was included to 
provide clear instructions for 
postdose observation. 
1.3 Schedule of Activities 
Footnote j: Current wording has 
been modified to allow the 
collection of additional exploratory 
safety samples 4 hours postdose or 
as soon as possible before the next 
IMP in case of AESM and serious 
headaches regardless of severity at 
Visits 4, 5, 6, 8, and 9. 
The footnotes were modified to 
provide clear instructions and to 
ensure consistency within Section 
8.9 of the protocol. 
1.3 Schedule of Activities 
Footnote l (now footnote m): 
wording has been modified: “To be 
collected predose at Visit 2, and 4 
hours postdose at Visit 9. 
The footnote was modified to 
provide clear instructions and to 
ensure consistency with other blood 
sampling assessments.  
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Section # and Name 
Description of Change 
Brief Rationale 
Additional samples must be 
collected 2 hours and 4 hours 
postdose or as soon as possible 
before the next IMP for study 
participants with severe headache 
and/or infusion reaction or 
hypersensitivity reaction.” 
1.3 Schedule of Activities 
Footnote m has been replaced with 
footnote l (now listed as m since the 
updates). 
Updated for consistency. 
1.3 Schedule of Activities 
Footnote n has been removed.  
The instruction applied to the study 
withdrawal assessment no longer 
applies. 
2.2 Background 
The number of rozanolixizumab 
clinical studies has been amended 
from 3 to 5 due to the two 
additional studies; CIDP01 and 
UP0060. 
General update. 
3 Objectives and 
Endpoints 
The primary efficacy analysis “the 
change from Baseline in MG-ADL 
score to Day 43 (Visit 10)” has 
been added to this section. 
General update. 
4.1 Overall Design 
This section was modified to update 
the approximate number of study 
participants screened from 
 
 In addition, China has been 
removed as a possible participant 
for this study, and Hong Kong and 
Taiwan have been added. 
An increase in the range of 
screened study participants has 
been made based on an anticipated 
percentage (up to 
) screening 
failures. With 
 participants 
screened and a 
 screening 
failure, the study will randomize 
 study participants. 
4.3 Justification for Dose 
This section was amended to 
include additional text for UP0018 
data. 
To provide accurate information on 
maximum mean decreases for 
Baseline IgG. 
5.1 Inclusion Criteria 
Criterion #3: For Visit 1, replaced 
“prior to” with “at” for the 
requirement to have confirmed 
positive record of autoantibodies 
against AChR or MuSK. New 
wording has been added to allow 
repeated testing at Visit 1 for 
confirmed presence of 
autoantibodies.  
This change is to allow 
confirmation from medical history 
information during the Screening 
Period, if needed.  
5.1 Inclusion Criteria 
Criterion #6: added “additional” 
and “such as” to broaden the 
Updated for consistency. 
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Section # and Name 
Description of Change 
Brief Rationale 
criteria for treatments considered 
by the Investigator.  
5.1 Inclusion Criteria 
Criterion #7: the body weight 
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. 
5.1 Inclusion Criteria 
Criterion #8: The following 
wording: “and prior to further 
dosing at each study visit 
thereafter” was removed.  
Updated for consistency, as this 
conflicted with part of the study 
design. 
5.2 Exclusion Criteria 
New wording has been included to 
provide clarity on timing of each 
exclusion criteria, where applicable. 
Updated to provide clarity for 
timing of each exclusion criterion. 
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 study participant 
must have “known” history of 
hyperprolinemia. 
Updated to clarify previously 
obtained medical records can be 
used. 
5.2 Exclusion Criteria 
Criterion #9 and #10: Associated 
table was amended. 
The change was implemented based 
on feedback of investigators, and to 
align the exclusionary period of 
biologics in the protocol. 
5.2 Exclusion Criteria 
Criterion #12, #13, #30: All three 
criterias have been amended to 
include “prior to Visit 1” 
Updated to provide clarity for 
timing of each exclusion criterion. 
5.2 Exclusion Criteria 
Criterion #13: Diverticular disease 
has been added as historical 
condition or disease in the past 6 
months. 
Updated to be consistent with 
adverse reactions across the 
rozanolixizumab clinical program. 
5.2 Exclusion Criteria 
Table 5-1:  
The treatment period prior to 
Baseline for the following were 
changed:  
Eculizumab (Solaris®): 6 to 3 
months 
Other biologics: 6 months to 3 
months, or within 5 half-lives 
(whichever was longer) prior to 
the Baseline Visit. 
The change was implemented based 
on feedback of investigators, and to 
align the exclusionary period of 
biologics in the protocol. 
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Section # and Name 
Description of Change 
Brief Rationale 
Intravenous and subcutaneous IgG 
and PEX: from 6 to 4 weeks 
Immunoadsorption: from 6 to 4 
weeks 
5.2 Exclusion Criteria 
Criterion #15: This criterion has 
been amended to provide additional 
information for study participants 
who have previously been 
randomized in this study. 
Clarification has been provided on 
the rules with regards to previous 
randomization in this study.  
  
5.2 Exclusion Criteria 
Criterion #17: Additional visit 
(Baseline [Visit 2]) has been added.  
Visit 2 was added to provide 
additional clarity on the applicable 
timeframe for Exclusion Criterion 
#17 
5.2 Exclusion Criteria 
Criterion #22: Amended definition 
(GFR less than 60ml/min/1.73m2) 
has been included for renal 
impairment. 
The inclusion of mild renal 
impairment is applicable to the 
Phase 3 clinical program with prior 
Phase 2 experience and no 
observations on renal impact. 
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. 
5.2 Exclusion Criteria 
Criterion #31: Amended to include 
“after Visit 1” 
Updated to provide clarity for 
timing the exclusion criterion. 
5.2 Exclusion Criteria 
Criterion #37: The description for 
this criterion has been removed and 
reworded to fit into Section 6.5.1 
(permitted concomitant treatments 
[medications and therapies]). 
Original criterion was restrictive, 
and could significantly limit the 
number of study participants, 
therefore has been reworded to fit 
into the permitted concomitant 
treatments. 
5.2 Exclusion Criteria 
New criterion has been added, #38 
Participant with current or medical 
history of IgA deficiency. 
Per EMA request to minimise the 
risk of infection 
5.2 Exclusion Criteria 
New criterion has been added, #39: 
Participant has undergone a 
splenectomy in the 2 years prior to 
the Baseline Visit. 
Per EMA request to reduce the 
potential overwhilming post-
splenectomy infection 
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 
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.  
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Section # and Name 
Description of Change 
Brief Rationale 
cannabidiols, as prescribed by a 
physician. 
5.4 Screen Failures 
Additonal wording was included: 
“repeat all Visit 1 assessments”. 
Updated to provide clarity for 
details of rescreened study 
participants. 
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.  
6.3 Measure to Minimize 
Bias 
Additional wording was included: 
“The randomization will be 
stratified by the following: MG-
specific autoantibody (MuSK+ or 
AChR+)”. 
Missing information. 
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 study 
participant must be on a stable dose 
of cannabidiols and/or medicinal 
marijuana for
 prior to 
Screening Visit, and remain stable 
for the duration of the study. 
 
Table 6-1 Permitted concomitant 
medications for the treatment of 
myasthenia gravis:  
The dose for oral corticosteroids 
has been amended to state “no 
specific requirements” and “stable 
for 
 prior to Baseline 
instead of 
  
Cholinesterase inhibitors: Dose 
hold was changed from 
 
  
An additional footnote was added 
to include “Must also be stable for 
duration of study”. 
Due to the lack of 
pharmacodynamic and 
pharmacokinetic interactions 
between marijuana/ cannabidiols 
and rozanolixizumab, this criterion 
was revised. 
 
 
 
 
The table was updated to reflect 
changes made to the study design. 
6.5.3 Treatments specific 
to NMJ interference 
Correct reference has been added 
for the MGFA classification list. 
General update. 
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Section # and Name 
Description of Change 
Brief Rationale 
6.5.4 Rescue therapy  
Additional wording on completion 
of remaining visits was added.  
Updated to provide clarity. 
6.7 Treatment after the 
End of the Study 
Additional wording to include 
instructions that additional 
unscheduled visits every other 
week for 8 weeks from the 
completion of last IVIg or PEX 
session are required for study 
participants who opt for IVIg or 
PEX.  
New language was added to allow 
study participants to complete the 
planned 6 weeks of treatment visits 
at the request of EMA. 
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.  
8.1 Efficacy assessments 
Efficacy assesments were 
reordered.  
Order of efficacy assessments were 
amended to align with study 
procedure manual. 
8.1.2 Patient-reported 
outcomes 
Has become Section 8.1.4 and order 
of PROs (PGI-S, PGI-C, EQ-5D-
5L, MGII, MG-QOL15r) were 
amended to align with Study 
Procedures manual. 
New wording has been added: 
“Study participants should not take 
pyridostigmine (or other AChE 
inhibitor medication) from 
midnight when efficacy 
assessments are performed when 
medically safe to do so to 
standardize testing” 
Order of efficacy assessments were 
amended to align with study 
procedure manual. 
8.1.2.3 Quantitative 
Myasthenia Gravis scale  
Moved to standalone efficacy 
assessment (Section 8.1.2), as 
QMG scale is not a PRO. 
New wording has been added:  
In addition, “qualified designee” 
has been added to the text as 
another individual aside from the 
The QMG scale is not a PRO. 
 
Added to provide clairification and 
harmonize how the assessment is 
performed at the site. 
 
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Section # and Name 
Description of Change 
Brief Rationale 
Investigator who can examine the 
study participant. 
“Study participants should not take 
pyridostigmine (or other AChE 
inhibitor medication) from 
midnight before testing when 
medically safe to do so to 
standardize testing” 
“Where possible, the same person 
should carry out the assessment at 
each visit.” 
8.1.2.4 MG-Composite 
scale 
Moved to standalone efficacy 
assessment (Section 8.1.3), as MG-
C scale is not a PRO. 
In addition, “qualified designee” 
has been added to the text as 
another individual aside from the 
Investigator who can examine the 
study participant. 
New wording has been added: 
“Study participants should not take 
pyridostigmine (or other AChE 
inhibitor medication) from 
midnight before testing when 
medically safe to do so to 
standardize testing”  
“Where possible, the same person 
should carry out the assessment at 
each visit.” 
The MG-C scale is not a PRO. 
 
Added to provide clairification and 
harmonize how the assessment is 
performed at the site. 
8.1.4.2 MG Symptoms 
PRO 
The minimum total score was 
corrected to “from 0 to 100” as per 
scale presented in Appendix 16.  
The original minimum total score 
was incorrect. 
8.1.4.6 MG Impairment 
Index 
There is an additional step to the 
assessment, where the Investigator 
or qualified designee will examine 
the study participant prior to 
scoring all items. 
New wording has been added: 
“Where possible, the same person 
should carry out the assessment at 
each visit.” 
Added to provide clairification and 
harmonize how the assessment is 
performed at the site. 
8.2 Safety Assessment 
Additional information on onsite 
observation timings postdose was 
included. 
Added to provide clairification for 
postdose observations. 
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Section # and Name 
Description of Change 
Brief Rationale 
8.2.2 Vital signs 
The measurement of respiratory 
rate is no longer required as part of 
vital signs assessment.  
To be consistent other 
Rozanolixizumab protocols. 
8.2.5 Suicidal risk 
monitoring 
Additional wording has been added 
to include C-SSRS at “Baseline and 
all subsequent visits”. 
To ensure consistency throughout 
the protocol. 
8.2.6 Assessment and 
management of TB and 
TB risk factors 
Latent TB: The following sentence 
has been deleted; “It is the 
Sponsor’s requirement that all 
study participants who are on LTBI 
treatment at Baseline must comply 
with the full therapy course”. 
Active TB or non-tuberculosis 
mycobacterium infection: The text 
was changed to replace WD with 
PEOT (premature end of 
treatment). 
The deleted sentence states that 
participants may continue in the 
study provided they remain on the 
LTBI treatment, however, 
exclusion critierion (#6) states 
participants with LTBI should be 
excluded. 
To ensure consistency throughout 
the protocol. 
 
8.2.6 Assessment and 
management of TB and 
TB risk factors 
TB assessment by chest X-ray: 
China has been removed as an 
example from the list of countries. 
The TB incidence rate of >20 per 
100,000 does not apply to China. 
8.2.6 Assessment and 
management of TB and 
TB risk factors 
For chest X-rays, additional 
wording was added to ensure the 
assessment was performed by a 
qualified specialist (ie, radiologist 
or pulmonologist). 
 
As a non-study specifc procedure, 
the designee responsile for 
assessing a chest x-ray or 
equivalent computer imaging 
needed to be defined. 
8.2.6 Assessment and 
management of TB and 
TB risk factors 
LTBI, active TB or other NTMB 
identified during study: The 
following sentence has been 
deleted; “If a TB specialist excludes 
an active TB infection, the subject 
can restart the study medication no 
earlier than 4 weeks after the start 
of an appropriate prophylactic 
therapy” 
There is no restart procedure in 
place for particpants with an active 
TB infection. 
8.3 Adverse Events 
The following sentence was 
amendment to include “study 
treatment in place of 
rozanolixizumab: “AEs that are 
serious, considered related to the 
study treatment or study 
procedures, or that caused the 
participant to discontinue the study 
treatment or MG0003. 
This change was made to include 
any AE cases that included study 
participants receivng placebo. 
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Section # and Name 
Description of Change 
Brief Rationale 
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 Section 10.2, 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 hypersenstitivy 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-emrgent adverse event. 
8.5 Treatment of 
Overdose 
Definition of overdose was 
amended: Any dose increase of 
10% or greater from the assigned 
dose for each administered dose of 
IMP 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 added. 
To be consistent with the ICF and 
other Rozanolixizumab protocols. 
Further, to remove detail 
redundancies covered in the 
laboratory manual. 
8.7 Pharmacodynamics 
Additional information has been 
added on limited testing to the 
positive antibody. 
To be consistent with the ICF and 
other rozanolixizumab protocols. 
Further, to remove detail 
redundancies covered in the 
laboratory manual. 
8.8.1 Pharmacogenomics  
The following text has been 
deleted: Details on the collection, 
storage, preparation, and shipping 
of samples will be presented in the 
laboratory manual provided 
separately. 
Repetitive text.  
8.8.2 Immunology 
The following text was added:  
“Tetatnus toxoid IgG” 
Anti-tetanus toxoid titer will be 
collected as part of the assessment 
for the effect of rozanolixizumab on 
tetanus IgG antibodies 
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Section # and Name 
Description of Change 
Brief Rationale 
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. 
9.1 Definition of Analysis 
Sets 
Amended to say that efficacy data 
should be analysed as randomized 
and safety data should be analysed 
as treated. In addition, Per-Protocol 
Set and Pharmacokinetic Per-
Protocol Set have been included as 
analysis sets.  
Original wording was not 
consistent with the routine analysis 
format for a Phase 3 study. 
9.3 Estimands 
Estimands section has been 
amended to include a new table 
(Table 9-1:), such that the primary 
estimand now uses a hypothetical 
strategy to deal with intercurrent 
events and missing data is imputed 
using a jump to reference approach.  
This section has been amended 
such that the primary estimand 
assumes rescue therapy to be a 
serious treatment failure. 
9.3.1 Hypothetical 
estimand 
9.3.2 Treatment policy 
estimand 
Both sections were removed. 
These sections were removed due 
to changes in the primary estimand. 
9.4.1 Analysis of primary 
efficacy endpoint 
The text has been amended to: 
Remove “other covariance 
structures will be investigated and 
used” and to provide an alternative 
approach: If the model does not 
converge using the unstructured 
pattern then an autoregressive 
covariance structure will be used. 
Amended the closed testing 
procedure from Dunnett’s test to 
Bonferroni approach. 
Using data from MG0002, it has 
been deemed that an autoregressive 
covariance structure is the most 
appropriate backup should the 
model not converge using an 
unstructured covariance structure. 
9.4.2 Analysis of 
secondary and other 
efficacy endpoints 
The text has been amended to 
include:  
term for the stratification factor 
(categorized as AChR or MuSK) 
A sequential hierarchical test 
procedure will be applied to protect 
the overall significance level for the 
multiplicity of endpoints 
The section was amended to 
provide further clarity. 
9.5.2.1 Pharmacokinetic 
analyses 
PK-PPS dataset has been included.  
The following new wording has 
been added: “A population PK and 
To provide clarity on which data 
analysis set would be used in the 
study.  
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Section # and Name 
Description of Change 
Brief Rationale 
population PKPD analysis will be 
performed to evaluate PK and 
PKPD of rozanolixizumab. Details 
of such analysis will be further 
described in a separate Data 
Analysis Plan. The results of the 
analysis would be reported in a 
separate report outside the CSR. If 
required an unblinded and relevant 
data transfer may be planned in 
order to initiate the PKPD analysis” 
9.8.1 Interim analysis 
Further clarity has been provided 
for the definition of “best dose”. 
“Best dose” and any higher dose 
has been amended to “best dose” 
and any dose with a mean response 
not worse than 1 point than the 
selected “best dose”). 
The additional criteria relating to 
the maximum difference of 1 point 
between the two doses in order to 
carry both forward has been added 
such that both doses are not carried 
forward if the higher dose is much 
worse than the “best dose”. 
9.8.1 Interim analysis 
Paragraph on Early Stopping for 
Futility was amended to give the 
second paragraph a separate sub 
heading “dose selection” 
The section was amended to 
provide further clarity. 
9.9 Determination of 
sample size 
The text was modified to update the 
average sample size for the study 
ranges: 
 have been 
updated to
 
respectively. 
As a result of the amended dose 
selection criteria at the interim 
analysis, the sample size range has 
changed slightly. 
9.9 Determination of 
sample size 
The text was modified to update 
chances of stopping for futility: 
when a clinically meaningful effect 
of
 more versus placebo, is less 
than 
. 
As a result of the amended dose 
selection criteria at the interim 
analysis, the chance of stopping for 
futility at the interim has changed 
slightly. 
9.9 Determination of 
sample size 
Table 9-2: The table was modified 
to include updated simulation 
results for all scenarios: Power, 
Proportion Futility, Proportion 
Selecting Low Dose, Proportion 
Selecting High Dose, and Average 
Sample Size.  
As a result of the amended dose 
selection criteria at the interim 
analysis, the simulation results have 
changed slightly. 
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 
document as defined above” was 
removed. 
The information was repetative. 
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Section # and Name 
Description of Change 
Brief Rationale 
10.2 Appendix 2: Clinical 
Laboratory Tests 
Additional tests have been 
included: Serology testing (for 
Hepatitis A, Hepatitis B, Hepatitis 
C, and HIV). 
The following parameter was 
removed: C-reactive protein. 
Consistency with study design. 
10.4 Appendix 4: 
Contraceptive Guidance 
and Collection of 
Pregnancy Information 
Additional text for contraception 
guidance for male study 
participants was updated.  
Table 10-1: additional footnote ‘a’, 
was added. 
The update was made to be 
consistent across the Phase 3 
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 10.21 Appendix 
12 to 21  
Order of appendices were changed 
to match the order shown in the 
body of the protocol. 
General consistency  
10.23 Appendix 23: 
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. 
10.24 Appendix 24: 
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 for study 
participants reporting severe 
diarrhea. 
The language updated to clarify the 
actions necessary by the PI in case 
of severe diarrhea. 
10.26 Appendix 26: 
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 harmoninize 
10.29 Appendix 29: 
Columbia-Suicide 
Severity Rating Scale 
New appendix for C-SSRS 
(screening and last visit versions) 
has been included. 
Missing appendix 
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Section # and Name 
Description of Change 
Brief Rationale 
11 References 
Additonal references (2) have been 
included. 
General update. 
 
 
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10.12 
Appendix 12: MGFA Classification  
 
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10.14 
Appendix 14: MG Composite Scale 
 
 
 
 
 
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10.17 
Appendix 17: Patient Global Impression of Severity  
 
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10.18 
Appendix 18: Patient Global Impression of Change  
 
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10.21 
Appendix 21: MG-QOL15r 
 
  
 
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10.23 
Appendix 23: 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 Common Terminology Criteria 
for Adverse Events (CTCAE) version 5.0 (Appendix 3, 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 10.27, Appendix 27) daily until resolving or resolution (ie, if headache 
becomes non-serious, moderate or mild, or completely resolved, whichever comes first). The 
questionnaire should be administered via an interview with the study participant. 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 may be temporarily 
put on hold if a study participant experiences a severe AE of headache that is considered related 
to the IMP 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 national 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. 
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.  
 
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10.24 
Appendix 24: 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 (see Appendix 3, 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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10.25 
Appendix 25: 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, meningitis, osteomyelitis or 
septic arthritis, pneumonia, or visceral abscess).  
To maintain the study integrity, IgG level will remain blinded to the study sites and the UCB 
study team. 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. Mock infusions will be administered to 
maintain the blind in case of low IgG levels (see Section 7.1.4). 
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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10.26 
Appendix 26: Management of Infusion Reactions or 
Hypersensitivity Reactions 
Study participants must be closely monitored for reactions during and after the study drug 
administration period. Standard precautions must be taken for the study participants with regards 
to potential infusion related reactions. Suggested management guidelines for infusion-related 
reactions or anaphylaxis are provided in Table 10-4. Definitions of severity of the relevant events 
should be consistent with CTCAE version 5.0 (Section 10.3). 
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). 
In case of suspected anaphylaxis, the Sampson’s Criteria (Sampson et al, 2006) should be 
accessed and Appendix 28 (Section 10.28, Sampson Criteria Questionnaire) should be 
completed. The infusion must be discontinued immediately and emergency resuscitation 
measures implemented. 
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In study participants experiencing an infusion-related reaction or anaphylaxis, blood samples will 
be collected 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), Plasma complement (C3a, C5a) and serum cytokines 
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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10.28 
Appendix 28: 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 
a. Respiratory compromise (eg, dyspnea, wheeze-bronchospasm, stridor, reduced peak 
expiratory flow [PEF], hypoxemia) 
b. Reduced blood pressure [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 blood pressure 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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11 
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