Conditions: Generalized Myasthenia Gravis
Keywords: generalized Myasthenia Gravis gMG rozanolixizumab
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 62
Healthy volunteers: no

Brief summary:
The purpose of this study is to evaluate the ability of study participants with generalized Myasthenia Gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods.

Detailed description:

Arms:
arm 1: Study participants will receive predefined weekly doses of rozanolixizumab for 18 weeks. arm 2: Study participants will receive predefined weekly doses of rozanolixizumab for 18 weeks.

Interventions: intervention 1: Rozanolixizumab
Intervention descriptions: intervention 1: Rozanolixizumab self-administration via Syringe Driver or Manual Push.

Protocol document:
Prot_000.pdf: 
UCB 
30 Nov 2023 
Clinical Study Protocol Amendment 5 
Rozanolixizumab 
MG0020 
Confidential 
Page 1 of 124 
AN OPEN-LABEL, CROSSOVER STUDY TO EVALUATE 
ROZANOLIXIZUMAB SELF-ADMINISTRATION BY STUDY 
PARTICIPANTS WITH GENERALIZED MYASTHENIA GRAVIS 
PROTOCOL MG0020 AMENDMENT 5 
PHASE 3 
SHORT TITLE: 
A Phase 3, open-label, crossover study to evaluate self-administration of rozanolixizumab by 
study participants with gMG 
Sponsor: 
UCB Biopharma SRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Regulatory agency identifying number(s): 
EudraCT Number: 
2022-003870-21 
IND Number: 
132407 
Confidential Material 
Confidential 
This document is the property of UCB and may not – in full or in part – be passed on, 
reproduced, published, or otherwise used without the express permission of UCB. 
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Clinical Study Protocol Amendment 5 
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MG0020 
 
 
 
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PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE 
Document History 
Document 
Date 
Type of amendment 
Protocol Amendment 5 
30 Nov 2023 
Substantial 
Protocol Amendment 4 
07 Sep 2023 
Substantial 
Protocol Amendment 3 
09 Dec 2022 
Non-substantial 
Protocol Amendment 2 
04 Nov 2022 
Substantial a 
Protocol Amendment 1 
31 Aug 2022 
Non-substantial a 
Original Protocol 
04 Aug 2022 
Not applicable 
a The Original Protocol and Protocol Amendment 1 were not submitted to any regulatory authorities prior to 
issuance of Protocol Amendment 2. 
Amendment 5 (30 Nov 2023) 
Overall rationale for the amendment 
The primary reason for this protocol amendment is to update the text to reflect the total number 
of participants screened, and to update the End of Study definition. 
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 
1.1 Synopsis  
4.1 Overall design 
9.9 Determination of sample 
size 
Text updated to reflect the total 
number of participants screened 
(from approximately 40 to a total of 
75) and to note that a total of 30 
randomized participants (15 per 
sequence) is the minimum 
evaluable number of participants. 
To update the actual number of 
screened participants. 
1.1 Synopsis  
1.2 Schema 
1.3 Schedule of activities 
4.1 Overall design 
4.4 End of study definition 
6.8 Treatment after the end 
of the study 
In addition to permitting 
participants access to post-trial 
rozanolixizumab (if available 
according to local guidance), the 
text has been updated to also permit 
participants access to commercially 
available rozanolixizumab, at the 
discretion of the investigator.  
Text also updated to note that 
participants moving to 
rozanolixizumab via either of these 
methods must have completed all 
Treatment Periods including the 
End of Treatment Visit prior to this 
move. 
To offer study participants the 
option of re-starting treatment 
with rozanolixizumab at an 
earlier timepoint following the 
completion of all Treatment 
Periods (including the End of 
Treatment Visit), and at the 
investigator's discretion. 
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MG0020 
 
 
 
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Section # and name 
Description of change 
Brief rationale 
A new footnote (footnote “s”) has 
been added to the Schedule of 
Activities and a new footnote 
(footnote “a”) has been added to the 
Schema to reflect this update. 
4.4 End of Study definition 
1.1 Synopsis 
3 Objectives and Endpoints 
The definition of study completers 
has been updated to allow 
participants who completed the End 
of Treatment Visit and then 
subsequently moved on to a post-
trial access program or 
commercially available product to 
be considered to have completed 
the study. 
The Week 26 timepoint for the End 
of Study Visit was removed from 
the respective endpoint definitions 
for consistency with the changes in 
the other sections. 
To offer study participants the 
option of re-starting treatment 
with rozanolixizumab at an 
earlier timepoint following the 
completion of all Treatment 
Periods (including the End of 
Treatment Visit), and at the 
investigator's discretion. 
9.3.3 Other safety analyses 
Paragraph #3 has been updated to 
remove the description of the 
statistical methods for estimating 
success. 
The confidence interval will be 
calculated for each 
self-administration method only 
for the primary endpoint. 
 
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) 
For details, see Appendix 3 (Section 10.3). 
 
REPORTING OF SELF-ADMINISTRATION MEDICATION ERRORS 
Safety reporting of self-administration medication errors (within 7 days)  
Email 
Complete paper Medication Error form and email to Global: 
DS_ICT@ucb.com 
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Fax 
Europe and Rest of the World: +32 2 386 24 21 
US and Canada: +1 800 880 6949 or +1 866 890 3175 
Medication errors associated with adverse reaction(s) will be recorded and reported within 24 hours 
following the SAE recording and reporting procedures and using the Medication Error form. For 
details, see Section 8.3.7.  
 
REPORTING OF ADVERSE DEVICE EFFECTS (SERIOUS AND 
NONSERIOUS) AND DEVICE DEFICIENCIES 
Reporting of adverse device effects (serious and nonserious) and device 
deficiencies for non-UCB devices used in the study (24h) 
Email 
Complete paper Adverse Event and Device Deficiency Form and 
email to MedicalDeviceSupport@parexel.com 
Note: Device deficiencies for non-UCB devices should not be 
captured in the electronic data collection tool. 
 
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TABLE OF CONTENTS 
PROTOCOL MG0020 AMENDMENT 5 .......................................................................................1 
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE ............................................2 
SERIOUS ADVERSE EVENT REPORTING ................................................................................3 
REPORTING OF SELF-ADMINISTRATION MEDICATION ERRORS ....................................3 
REPORTING OF ADVERSE DEVICE EFFECTS (SERIOUS AND NONSERIOUS) AND 
DEVICE DEFICIENCIES .......................................................................................................4 
TABLE OF CONTENTS .................................................................................................................5 
LIST OF TABLES .........................................................................................................................10 
LIST OF FIGURES .......................................................................................................................10 
1 
PROTOCOL SUMMARY .....................................................................................................11 
1.1 
Synopsis .............................................................................................................................11 
1.2 
Schema ...............................................................................................................................17 
1.3 
Schedule of activities .........................................................................................................18 
1.3.1 
Additional study assessments ....................................................................................24 
2 
INTRODUCTION .................................................................................................................25 
2.1 
Study rationale ...................................................................................................................26 
2.2 
Background ........................................................................................................................26 
2.3 
Benefit/risk assessment ......................................................................................................27 
3 
OBJECTIVES AND ENDPOINTS .......................................................................................29 
4 
STUDY DESIGN ...................................................................................................................31 
4.1 
Overall design ....................................................................................................................31 
4.1.1 
Unscheduled visit .......................................................................................................32 
4.2 
Scientific rationale for study design ..................................................................................32 
4.3 
Justification for dose ..........................................................................................................33 
4.4 
End of Study definition ......................................................................................................33 
5 
STUDY POPULATION ........................................................................................................33 
5.1 
Inclusion criteria ................................................................................................................33 
5.2 
Exclusion criteria ...............................................................................................................34 
5.3 
Lifestyle restrictions...........................................................................................................36 
5.4 
Screen failures ....................................................................................................................36 
6 
STUDY TREATMENTS .......................................................................................................37 
6.1 
Treatments administered ....................................................................................................37 
6.1.1 
Medical devices .........................................................................................................38 
6.2 
Preparation, handling, storage, and accountability requirements ......................................38 
6.2.1 
Drug/Device accountability .......................................................................................39 
6.3 
Measures to minimize bias: randomization and blinding ..................................................39 
6.4 
Treatment compliance ........................................................................................................40 
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6.5 
Concomitant medication(s)/treatment(s) ...........................................................................40 
6.5.1 
Permitted concomitant treatments (medications and therapies) ................................40 
6.5.2 
Prohibited concomitant treatments (medications and therapies) ...............................41 
6.5.3 
Treatments specific to NMJ interference ...................................................................41 
6.5.4 
Rescue medication .....................................................................................................41 
6.6 
Dose modification ..............................................................................................................42 
6.7 
Criteria for study hold or dosing stoppage .........................................................................42 
6.8 
Treatment after the end of the study ..................................................................................42 
7 
DISCONTINUATION OF STUDY MEDICATION AND PARTICIPANT 
DISCONTINUATION/WITHDRAWAL ..............................................................................42 
7.1 
Discontinuation of study medication .................................................................................42 
7.1.1 
Liver chemistry stopping criteria ...............................................................................42 
7.1.2 
Temporary IMP discontinuation ................................................................................43 
7.1.3 
Study medication permanent discontinuation criteria ...............................................44 
7.2 
Participant discontinuation/withdrawal from the study .....................................................45 
7.3 
Lost to follow-up................................................................................................................46 
8 
STUDY ASSESSMENTS AND PROCEDURES .................................................................46 
8.1 
Efficacy assessments ..........................................................................................................47 
8.2 
Safety assessments .............................................................................................................47 
8.2.1 
Safe and successful self-administration .....................................................................47 
8.2.2 
Participant’s preferred method of rozanolixizumab administration questions ..........48 
8.2.3 
Self-Injection Assessment Questionnaire (Infusion version) ....................................48 
8.2.4 
Myasthenia Gravis Activities of Daily Living ...........................................................48 
8.2.5 
Physical examination .................................................................................................49 
8.2.6 
Vital signs ..................................................................................................................49 
8.2.7 
Electrocardiograms ....................................................................................................50 
8.2.8 
Clinical safety laboratory assessments ......................................................................50 
8.2.9 
Suicidal risk monitoring ............................................................................................50 
8.2.10 
Assessment and management of TB and TB risk factors ..........................................50 
8.2.10.1 
Tuberculosis assessment ..................................................................................51 
8.2.11 
COVID-19 precautions ..............................................................................................51 
8.2.12 
MGFA classification ..................................................................................................52 
8.3 
Adverse events and serious adverse events .......................................................................52 
8.3.1 
Time period and frequency for collecting AE and SAE information ........................52 
8.3.2 
Method of detecting AEs and SAEs ..........................................................................53 
8.3.3 
Follow-up of AEs and SAEs ......................................................................................53 
8.3.4 
Regulatory reporting requirements for SAEs ............................................................53 
8.3.5 
Pregnancy ..................................................................................................................54 
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8.3.6 
Adverse events of special interest ..............................................................................54 
8.3.7 
Medication errors associated with self-administration ..............................................54 
8.3.8 
Adverse events of special monitoring ........................................................................55 
8.3.9 
Medical device – adverse events (ADEs, unanticipated ADEs, SAEs, SADEs, 
and unanticipated SADEs) and device deficiencies ..................................................55 
8.3.9.1 
Time period for detecting ADEs and device deficiencies ...............................55 
8.3.9.2 
Follow-up of ADEs and device deficiencies ...................................................56 
8.3.9.3 
Prompt reporting of ADEs and device deficiencies to sponsor .......................56 
8.3.9.4 
Regulatory reporting requirements for ADEs and device deficiencies ...........56 
8.4 
Safety signal detection .......................................................................................................56 
8.5 
Treatment of overdose .......................................................................................................57 
8.6 
Pharmacokinetics ...............................................................................................................57 
8.7 
Pharmacodynamics ............................................................................................................57 
8.8 
Biomarkers .........................................................................................................................57 
8.9 
Immunogenicity assessments .............................................................................................58 
8.10 
Medical resource utilization and health economics ...........................................................58 
9 
STATISTICAL CONSIDERATIONS ...................................................................................58 
9.1 
Definition of analysis sets ..................................................................................................58 
9.2 
General statistical considerations .......................................................................................59 
9.3 
Planned safety analyses......................................................................................................59 
9.3.1 
Analysis of the primary safety endpoint ....................................................................59 
9.3.2 
Analysis of the secondary safety endpoint ................................................................59 
9.3.3 
Other safety analyses .................................................................................................59 
9.4 
Planned efficacy/outcome analyses ...................................................................................61 
9.5 
Other planned analyses ......................................................................................................61 
9.6 
Handling of protocol deviations.........................................................................................61 
9.7 
Handling of dropouts or missing data ................................................................................61 
9.8 
Planned interim analysis and data monitoring ...................................................................61 
9.9 
Determination of sample size .............................................................................................61 
10 SUPPORTING DOCUMENTATION AND OPERATIONAL CONSIDERATIONS.........62 
10.1 
Appendix 1: Regulatory, ethical, and study oversight considerations ...............................62 
10.1.1 
Regulatory and ethical considerations .......................................................................62 
10.1.2 
Financial disclosure ...................................................................................................63 
10.1.3 
Informed consent process ..........................................................................................63 
10.1.4 
Data protection ...........................................................................................................63 
10.1.5 
Committees structure .................................................................................................64 
10.1.6 
Dissemination of clinical study data ..........................................................................64 
10.1.7 
Data quality assurance ...............................................................................................65 
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10.1.7.1 
Case report form completion ...........................................................................65 
10.1.7.2 
Apps .................................................................................................................66 
10.1.8 
Source documents ......................................................................................................66 
10.1.9 
Study and site start and closure .................................................................................67 
10.1.10 
Publication policy ......................................................................................................67 
10.2 
Appendix 2: Clinical laboratory tests ................................................................................68 
10.3 
Appendix 3: Adverse events – Definitions and procedures for recording, evaluating, 
follow-up, and reporting ....................................................................................................70 
10.4 
Appendix 4: Contraceptive guidance and collection of pregnancy information ...............75 
10.5 
Appendix 5: Genetics .........................................................................................................78 
10.6 
Appendix 6: Liver safety – suggested actions and follow-up assessments .......................79 
10.7 
Appendix 7: Medical device adverse events (AEs), adverse device effects (ADEs), 
serious adverse events (SAEs), and device deficiencies: Definition and procedures for 
recording, evaluating, follow-up, and reporting ................................................................83 
10.7.1 
Definition of AE and ADE ........................................................................................83 
10.7.2 
Definition of SAE, SADE, and unanticipated SADE ................................................83 
10.7.3 
Definition of device deficiency .................................................................................84 
10.8 
Appendix 8: Rapid alert procedures ..................................................................................85 
10.9 
Appendix 9: Country-specific requirements ......................................................................86 
10.10 Appendix 10: Abbreviations and trademarks ....................................................................87 
10.11 Appendix 11: Protocol amendment history .......................................................................89 
10.12 Appendix 12: Medication errors associated with self-administration .............................103 
10.13 Appendix 13: Management of infusion reactions or hypersensitivity reactions and 
infections and hypogammaglobulinemia .........................................................................104 
10.13.1 
Management of infusion reactions or hypersensitivity reactions ............................104 
10.13.2 
Management of infections and hypogammaglobulinemia .......................................105 
10.14 Appendix 14: Management of adverse event of special monitoring ...............................106 
Aseptic meningitis (suspected) ................................................................................................106 
Severe and/or serious headache ...............................................................................................107 
Medication error associated with adverse reaction(s) ..............................................................108 
10.15 Appendix 15: Evaluation by HCP at site of safe and successful self-administration (to 
collect data for primary endpoints) ..................................................................................109 
10.15(a): HCP evaluation checklist following self-administration at site at Visit 13 
(
) and Visit 19 (
) ..........................................................................109 
10.15(b): HCP conclusions following evaluation of self-administration at site at Visit 13 
(
) and Visit 19 (
) ..........................................................................110 
10.16 Appendix 16: Participant’s preferred method of rozanolixizumab administration 
questions ..........................................................................................................................111 
10.17 Appendix 17: Myasthenia Gravis Activities of Daily Living ..........................................113 
10.18 Appendix 18: Self-Injection Assessment Questionnaire .................................................114 
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10.19 Appendix 19: Tuberculosis Questionnaire.......................................................................121 
11 REFERENCES ....................................................................................................................122 
SPONSOR DECLARATION ......................................................................................................124 
 
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LIST OF TABLES 
Table 1-1: 
MG0020 crossover design .....................................................................................14 
Table 1-2: 
Rozanolixizumab weight tiers................................................................................14 
Table 1-3: 
Schedule of Activities ............................................................................................18 
Table 1-4: 
Additional study assessments ................................................................................24 
Table 6-1: 
Permitted concomitant treatments ..........................................................................40 
Table 9-1: 
Expected 90% confidence intervals considering different success rates 
and 30 study participants completing both Treatment Periods ..............................62 
Table 10-1: 
Protocol-required safety laboratory assessments ...................................................68 
Table 10-2: 
Phase 3-4 liver chemistry stopping criteria and follow-up assessments ................79 
Table 10-3: 
Phase 3-4 liver chemistry increased monitoring criteria with continued 
study medication ....................................................................................................82 
Table 10-4: 
Suggested management guidelines at the study site for infusion reactions .........104 
 
LIST OF FIGURES 
Figure 7-1: 
Liver Chemistry Stopping Criteria and Increased Monitoring Algorithm .............42 
Figure 7-2: 
Liver Chemistry Increased Monitoring Algorithm with Continued Study 
Intervention for Participants with ALT ≥3xULN but <8xULN.............................43 
 
 
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1 
PROTOCOL SUMMARY 
1.1 
Synopsis 
Protocol title: An open-label, crossover study to evaluate rozanolixizumab self-administration 
by study participants with generalized myasthenia gravis. 
Short title: A Phase 3, open-label, crossover study to evaluate self-administration of 
rozanolixizumab by study participants with gMG. 
Rationale: Generalized myasthenia gravis (gMG) is a chronic, autoimmune disease requiring 
long-term therapy. Successful self-administration of subcutaneous (SC) treatment has been 
demonstrated in gMG patients using immunoglobulin (Ig) (Alcantara et al, 2021). Currently, 
rozanolixizumab is administered SC by health care professionals (HCPs) using programmable 
syringe drivers at a 
. There is considerable clinical interest in 
providing an alternative administration method to programmable syringe drivers as well as 
providing patients the option to self-administer rozanolixizumab. Given the relatively low 
volumes involved in rozanolixizumab therapy, the manual push technique is being explored as an 
alternative method, which offers the advantages of independence from constant use of a syringe 
driver, and reduced infusion times (
 for abdominal sites). The 
manual push technique is also a well-established mode of self-administration of SC Ig (Bienvenu 
et al, 2018). 
Rozanolixizumab has been administered using manual push in an ongoing single-dose healthy 
volunteer study (UP0106) and the manual push administration by HCP is planned to be included 
in gMG study MG0007; other indications may offer alternative administration options for 
rozanolixizumab in Phase 3 open-label extension studies. 
Following a comprehensive training (consisting of 
 with the study participant 
practicing both methods at the study site), rozanolixizumab will be self-administered by both 
manual push and syringe driver in the clinic and unsupervised by an HCP at home. In addition to 
the evaluation of successful self-administration and safety, the participant’s preference for HCP 
administration versus self-administration and preference for the manual push versus the syringe 
driver method of administration will be compared. 
If required, only minimal assistance is allowed to support the study participant for a successful 
self-administration. However, if an informal caregiver is required, he/she must be fully trained 
(ie, attending all training sessions at the site) to perform the administration in this study on behalf 
of the study participant. In this case, the caregiver must perform all investigational medicinal 
product (IMP) administration activities described for the study participant throughout the Self-
administration Periods. 
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Objectives and endpoints: 
Objectives 
Endpoints 
Primary 
Primary objective: 
To evaluate the ability of study 
participants with gMG to successfully 
self-administer rozanolixizumab after 
training in the self-administration 
technique using the syringe driver 
and manual push methods 
Primary endpoint: 
• Successful self-administration of rozanolixizumab 
(with correct use of syringe driver and manual push, 
respectively) during the Self-administration Period 
at Visit 13 (
 last dose of Self-
administration Period 1) and Visit 19 (
; last 
dose of Self-administration Period 2). 
 
Successful self-administration is defined by the 
participant (i) choosing the correct infusion site, (ii) 
administering SC, and (iii) delivering the intended 
dose. 
Secondary 
Secondary objective: 
• To evaluate the safety of SC self-
administration of 
rozanolixizumab 
Secondary endpoints: 
• Occurrence of treatment-emergent adverse events 
(TEAEs) after syringe driver or manual push self-
administration from Visit 2 (
 up to the End 
of Study Visit (Visit 21). 
• Occurrence of local site reactions up to 24 hours 
after each administration during the Training Period 
and Self-administration Periods. 
• Occurrence of medication errors associated with 
adverse reactions during the 2 Self-administration 
Periods of the study. 
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Other 
Other objectives: 
• To evaluate the study 
participant’s preferred method of 
rozanolixizumab administration 
Other endpoints: 
• Participant’s relative preference for: 
− Subcutaneous infusions performed by an HCP 
versus self-administration 
− The manual push method versus the use of a 
syringe driver for self-administration 
using the participant’s preferred method of 
administration questions at Visit 19 (
). 
• To evaluate symptom changes in 
study participants with gMG 
• Myasthenia Gravis Activities of Daily Living 
(MG-ADL) score change from Baseline during the 
study. 
• To assess the pharmacodynamics 
of rozanolixizumab 
• Total IgG level over time during the Training Period 
and Self-administration Periods. 
• To evaluate the immunogenicity 
of rozanolixizumab following SC 
self-administration 
• Anti-rozanolixizumab antibodies (status and titer at 
trough during both Self-administration Periods). 
• To assess the study participant’s 
experience with self-
administration of SC infusions at 
home 
• 
• To assess the ability of study 
participants with gMG to 
successfully self-administer 
rozanolixizumab at home after 
training in the self-administration 
technique using the syringe driver 
and manual push methods 
• Successful self-administration of rozanolixizumab 
via manual push method or using a syringe driver at 
each of the 
 visits during 
Self-administration Period 1 or 2, respectively. 
Successful self-administration via manual push or 
syringe driver is defined by the participant (i) 
choosing the correct infusion site, (ii) administering 
SC, and (iii) delivering the intended dose, as 
reported by the study participant to the site staff 
following each of the 
. 
• To assess the safety and 
tolerability of rozanolixizumab in 
study participants with gMG 
• Occurrence of TEAEs leading to permanent 
withdrawal of rozanolixizumab during the study. 
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Overall design: 
MG0020 is a Phase 3, open-label, randomized, 2-period, 2-sequence crossover study planned to 
be conducted at multiple sites in Europe, Japan, and North America. In MG0020, the safe and 
effective self-administration of rozanolixizumab using the syringe driver and manual push 
administration methods by study participants with gMG will be evaluated using a crossover 
design (Table 1-1). Study participants are proposed to receive 
 weight tiered doses of 
rozanolixizumab for 
, as summarized in Table 1-2.  
The weight tiered dosing will only be applicable to the study participants starting the Treatment 
Period after the approval of Protocol Amendment 4 in their respective countries. The study 
participants who are already in the Treatment Period when Protocol Amendment 4 is approved in 
their respective countries will continue with the 
 fixed doses as per Protocol 
Amendment 3 until study completion. 
Rozanolixizumab-naïve study participants and study participants previously exposed to 
rozanolixizumab will be included in the study. 
Table 1-1: 
MG0020 crossover design 
 
Administration method 
Sequence 1 
Syringe driver 
Manual push 
Sequence 2 
Manual push 
Syringe driver 
 
Table 1-2: 
Rozanolixizumab weight tiers 
Body weight of study participant 
 Dose  
Volume to be 
infused 
Study participants in Europe and Canada 
≥35kg to <50kg 
≥50kg to <70kg 
≥70kg to <100kg 
≥100kg 
Study participants in the US 
<50kg 
≥50kg to <100kg 
≥100kg 
 
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Number of participants: 
A total of 75 participants have been screened to achieve a minimum of 30 randomly assigned and 
evaluable participants for an estimated minimum total of 15 evaluable participants per sequence. 
See Section 9.9 for the determination of sample size. 
Treatment groups and duration: 
The total duration for each study participant is up to 29 weeks, including a Screening Period of 
up to 4 weeks, an 
 Treatment Period, and an up to 7-week Safety Follow-up Period (ie, 
End of Study Visit is 8 weeks after the last dose of rozanolixizumab in the Treatment Period, or 
earlier, if the study participant moves on to a post-trial access program or to commercially 
available rozanolixizumab during the Safety Follow-up Period). 
The 
 Treatment Period of MG0020 starts with standardized 
 trainings over the 
course of a 
 Training Period when the study participant is trained on both the manual push 
and the syringe driver administration methods. During the Training Period, the study participant 
will receive 
 doses of rozanolixizumab SC and should practice both methods of 
administration at the study site. To act as a reference for the preference question (HCP vs self-
administration) and to demonstrate subcutaneous infusions of rozanolixizumab, the first 
administration will be imperatively performed by the HCP. For all remaining training visits, the 
study participant should perform as many self-administrations as possible (under HCP 
supervision) depending on their training status. Self-administration under HCP supervision or 
administration by HCP will be documented in the case report form (CRF). At Visit 8 (
), 
after the completion of the Training Period and following the investigator’s confirmation of the 
eligibility to perform self-administration, the study participant will be randomized 1:1 to either 
the syringe driver or to the manual push administration method. After completing Self-
administration Period 1 (at Visit 13 [
]), the study participant will crossover to the 
alternative administration method, entering Self-administration Period 2 (Visit 14 [
]). A 
final release of the study participant for home self-administration will be based on a successful 
demonstration of self-administration using the assigned mode of administration (manual push or 
syringe driver use) at Visit 9 (
 and Visit 15 (
), respectively. Study participants 
who cannot be confirmed eligible for self-administration will not perform self-administration and 
can continue to be treated on-site per protocol based on investigator decision. Additionally, if a 
study participant becomes no longer eligible for self-administration during the 
Self-administration Periods, he/she can continue to be treated by HCP on-site per protocol based 
on investigator decision. The study medication administration can be performed either via 
syringe driver or manual push. Adverse events (AEs), concomitant medication, vital signs, ECG, 
suicidality, blood and urine samples for safety laboratory tests, pregnancy tests, and IgG will 
continue to be collected from these study participants until the End of Study Visit/Early 
Withdrawal Visit. 
During both Self-administration Period 1 and Self-administration Period 2, 
 
 (ie, unsupervised/not in the presence of an 
HCP) for 
 
After the Self-administration Periods, study participants will enter the 7-week Safety Follow-up 
Period. 
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Study participants who have completed all Treatment Periods (including the End of Treatment 
Visit) will have the option to subsequently move, at the discretion of the investigator, into either 
a post-trial access program for rozanolixizumab (if available according to local guidance) or on 
to commercially available rozanolixizumab.  
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1.2 
Schema 
 
HCP=health care professional; MP=manual push; SD=syringe driver; SFU=safety follow-up 
a Participants completing all Treatment Periods, including the End of Treatment Visit, and moving on to either a post-trial access program or commercially 
available rozanolixizumab during the SFU Period must undergo an earlier End of Study Visit prior to this move (see Section 4.4 and Section 6.8). 
 
 
 
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1.3 
Schedule of activities 
The Schedule of Activities is provided in Table 1-3.  
Table 1-3: 
Schedule of Activities 
Procedure 
Scr 
Period 
Training Period 
Self-administration Period 1 
Self-administration Period 2 
SFU 
Period 
Visit 
V1 Scr 
V2 
BL 
V3 
V4 
V5 
V6 
V7 
V8 
V9 
V10 
V11 
V12 
V13 
V14 
V15 
V16 
V17 
V18 
V19 
EOT 
V20 
/EW 
EOS 
V21s 
Visit type 
Day (visit 
window)a 
Week 
Written 
informed 
consent 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic 
and Baseline 
characteristics 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Verification of 
inclusion/ 
exclusion 
criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
General 
medical 
history 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior and 
concomitant 
medications 
and medical 
procedures 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Height and 
body weight 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
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Table 1-3: 
Schedule of Activities 
Procedure 
Scr 
Period 
Training Period 
Self-administration Period 1 
Self-administration Period 2 
SFU 
Period 
Visit 
V1 Scr 
V2 
BL 
V3 
V4 
V5 
V6 
V7 
V8 
V9 
V10 
V11 
V12 
V13 
V14 
V15 
V16 
V17 
V18 
V19 
EOT 
V20 
/EW 
EOS 
V21s 
Visit type 
Day (visit 
window)a 
Week 
Psychiatric 
history/ 
C-SSRSb 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Query for 
suicidalityb 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
X 
X 
X 
 
 
 
X 
X 
 
Tuberculosis 
Signs and 
Symptoms 
questionnaire 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Complete 
physical 
examination 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Brief physical 
examinationm 
 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
Vital signsc 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
X 
X 
X 
 
 
 
X 
X 
X 
12-lead ECGn 
X 
 
 
 
 
 
X 
 
 
 
 
 
X 
 
 
 
 
 
 
X 
 
Recording of 
AEs 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Pregnancy 
testd 
X 
X 
 
 
 
 
 
X 
 
 
 
 
 
X 
 
 
 
 
 
 
 
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Table 1-3: 
Schedule of Activities 
Procedure 
Scr 
Period 
Training Period 
Self-administration Period 1 
Self-administration Period 2 
SFU 
Period 
Visit 
V1 Scr 
V2 
BL 
V3 
V4 
V5 
V6 
V7 
V8 
V9 
V10 
V11 
V12 
V13 
V14 
V15 
V16 
V17 
V18 
V19 
EOT 
V20 
/EW 
EOS 
V21s 
Visit type 
Day (visit 
window)a 
Week 
Hematology, 
clinical 
chemistry 
X 
X 
X 
 
 
 
 
X 
 
 
 
 
 
X 
 
 
 
 
 
X 
X 
Serology 
(HIV, 
Hepatitis B, 
and 
Hepatitis C) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Urinalysis 
X 
X 
X 
 
 
 
 
X 
 
 
 
 
 
X 
 
 
 
 
 
X 
X 
Blood 
sampling for 
ADAe 
 
X 
 
 
 
 
 
Xq 
Xq 
 
 
 
Xq 
Xq 
Xq 
 
 
 
Xq 
Xq 
Xq 
Blood 
sampling for 
total IgG 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
X 
X 
X 
 
 
 
X 
X 
X 
Blood 
sampling for 
exploratory 
safety 
biomarker 
analysis 
 
X 
Xr 
MGFA 
classification 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
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Table 1-3: 
Schedule of Activities 
Procedure 
Scr 
Period 
Training Period 
Self-administration Period 1 
Self-administration Period 2 
SFU 
Period 
Visit 
V1 Scr 
V2 
BL 
V3 
V4 
V5 
V6 
V7 
V8 
V9 
V10 
V11 
V12 
V13 
V14 
V15 
V16 
V17 
V18 
V19 
EOT 
V20 
/EW 
EOS 
V21s 
Visit type 
Day (visit 
window)a 
Week 
Randomization 
 
 
 
 
 
 
 
Xl 
 
 
 
 
 
 
 
 
 
 
 
 
 
Call or enter 
IRT to register 
the visit 
X 
X 
X 
X 
X 
X 
X 
Xl 
X 
 
 
 
X 
X 
X 
 
 
 
X 
X 
X 
Study drug 
adminh 
Return of vials 
from home 
self-admino 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
 
 
 
X 
 
 
MG-ADL 
X 
Xf 
 
 
 
 
 
Xf,q 
 
Xf,q 
Xf,q 
Xf,q 
Xf,q 
Xf,q 
 
Xf,q 
Xf,q 
Xf,q 
Xf,q 
 
 
SIAQ 
(Infusion 
version)g 
Site staff to 
evaluate study 
participant’s 
safe and 
successful 
self-admin 
 
 
 
 
 
 
Xi 
 
Xi,q 
Xj,q 
Xj,q 
Xj,q 
Xk,
q 
 
Xi,q 
Xj,q 
Xj,q 
Xj,q 
Xk,
q 
 
 
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Table 1-3: 
Schedule of Activities 
Procedure 
Scr 
Period 
Training Period 
Self-administration Period 1 
Self-administration Period 2 
SFU 
Period 
Visit 
V1 Scr 
V2 
BL 
V3 
V4 
V5 
V6 
V7 
V8 
V9 
V10 
V11 
V12 
V13 
V14 
V15 
V16 
V17 
V18 
V19 
EOT 
V20 
/EW 
EOS 
V21s 
Visit type 
Day (visit 
window)a 
Week 
Participant 
self-
assessment of 
home self-
admin 
 
 
 
 
 
 
 
 
 
Xj,q 
Xj,q 
Xj,q 
 
 
 
Xj,q 
Xj,q 
Xj,q 
 
 
 
Questions on 
study 
participant’s 
preferred 
method of 
admin 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Xq 
 
 
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ADA=antidrug antibody; admin=administration; AE=adverse event; BL=Baseline; C-SSRS=Columbia-Suicide Severity Rating Scale; ECG=electrocardiogram; EOS=end of 
study; EOT=end of treatment; EW=early withdrawal; HC/SP=health care professional administration or self-administration under healthcare professional supervision; 
HIV=human immunodeficiency virus; HSA=home self-administration; IgG=immunoglobulin G; IRT=interactive response technology; MG-ADL=Myasthenia Gravis 
Activities of Daily Living; MGFA=Myasthenia Gravis Foundation of America; S=site visit; SC=subcutaneous; Scr=screening; SFU=safety follow-up; SIAQ=Self-Injection 
Assessment Questionnaire; SP=self-administration by the study participant; V=visit 
Note: HSA=home self-administration, ie, unsupervised self-administration/not in the presence of an HCP. 
a The visit windows are relative to the first dosing visit date. A visit window of ±2 days is allowed. However, there should be a minimum interval of 5 days and a maximum 
interval of 9 days between 2 doses of IMP. 
b A full C-SSRS assessment will be performed for Screening and Baseline. For subsequent visits, a full C-SSRS assessment will only be performed if a study participant has a 
positive response to the query for suicidality. If a study participant has 
 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. 
c Vital signs comprise systolic and diastolic blood pressure, pulse rate, temperature, and respiratory rate. Vital signs will be taken at scheduled visits at the following 
timepoints. On Day 1 and Day 8, vital signs will be measured before rozanolixizumab administration (-5 mins), at the end of the infusion (+5 mins), and 2 hours (±15 mins) 
after the end of infusion. On Day 15, Day 22, Day 29, and Day 36, vital signs will be measured before rozanolixizumab administration (-5 mins) and 1 hour (±5 mins) after the 
end of infusion. For site visits from Day 43 onwards, vital signs will be measured before rozanolixizumab administration (-5 mins), at the end of the infusion (+5 mins), and 
15 minutes (±5 mins) after the end of infusion. Additional vital sign measurements can be performed at the investigator’s discretion.  
d Serum pregnancy test at Screening and urine pregnancy test at all other indicated visits. 
e Antidrug antibody sample collection predose. 
f The MG-ADL will be completed predose. 
h Study participants must be observed at site postdose for at least 2 hours following the first 
. Vital signs will be assessed as described above. 
i An HCP will conduct a mandatory evaluation of self-administration at site at the last visit of the Training Period and following the study participant’s demonstration of self-
administration using the assigned mode of administration (syringe driver or manual push) at Visit 9 
 and Visit 15 
 
j An HCP is required to have a direct contact with the study participant via telephone or video call as soon as possible after each home self-administration to confirm their 
health status. The direct telephone or video contact with the study participant will also be used to assess successful self-administration criteria. 
k An HCP will conduct a mandatory evaluation of self-administration at site Visit 13 (
) and Visit 19 (
) to confirm (i) correct infusion site, (ii) administered 
SC, and (iii) delivered the intended dose. Refer to Appendix 15 (Section 10.15) for documents to be used. 
l Participants will be randomized to a treatment sequence at Visit 8 (
 
m In addition to a brief physical examination, a full neurological examination should be performed for any study participant who experiences severe and/or serious headache 
and for any study participant who experiences features suggestive of aseptic meningitis (see Appendix 14 [Section 10.14]). For details of the assessments included in these 
examinations, see Section 8.2.5. For additional assessments that may be required in case of these AESM, see Section 1.3.1 (Table 1-4). 
n Single 12-lead ECGs, which will be read locally. 
o Return of used and unused vials as well as all equipment and ancillary materials used for IMP administration. 
p The timing between Screening and the Baseline Visit needs to be a minimum of 14 days to confirm eligibility and to ensure all prerequisites are met. 
q Not applicable for study participants not considered eligible for self-administration, who will continue receiving IMP administered by the HCP at the study site (for 
additional details, see Section 4.1). 
r Exploratory biomarker samples will be taken predose at Baseline (Day 1) for all study participants. In study participants who experience severe and/or serious headaches or 
features suggestive of aseptic meningitis, samples should also be collected 4 hours after the onset of the event, or otherwise as soon as possible within 72 hours after the onset 
of the event. For additional assessments that may be required in case of these AESM, see Section 1.3.1 (Table 1-4). 
s Participants completing all Treatment Periods, including the EOT Visit, and moving on to either a post-trial access program or commercially available 
rozanolixizumab during the SFU Period must undergo an earlier EOS Visit prior to this move (see Section 4.4 and Section 6.8). 
 
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1.3.1 
Additional study assessments  
In addition to those detailed in Table 1-3, the assessments in Table 1-4 may be required in case 
of adverse events of special monitoring (AESM) of severe and/or serious headache, or suspected 
aseptic meningitis (see Section 8.3.8). Note that additional vital sign measurements and/or 
additional investigations may be performed at the discretion of the investigator. 
Table 1-4: 
Additional study assessments 
Assessment 
When applicable 
For study participants who experience severe and/or serious headache and for study participants 
with suspected aseptic meningitis 
Headache or 
suspected aseptic 
meningitis 
follow-up 
questionnaire 
Headache follow-up questionnaire which sites will receive after reporting AESM 
of severe and/or serious headache should be completed promptly and returned to 
the sponsor via the SAE reporting process. 
Suspected aseptic meningitis follow-up questionnaire which sites will receive after 
reporting AESM of suspected aseptic meningitis should be completed promptly 
and returned to the sponsor via the SAE reporting process. 
Full neurological 
examination 
Assessments required for all study participants are detailed in the Schedule of 
Activities (Table 1-3).  
In study participants who report/are diagnosed with severe and/or serious headache 
or with a suspected aseptic meningitis at the clinic visit, a full neurological 
examination (including fundoscopy) should be performed (see Appendix 14 
[Section 10.14]).  
In study participants who report a severe and/or serious headache while at home or 
features suggestive of aseptic meningitis, a visit to the site for the full neurological 
examination should be arranged for as soon as is practically possible. 
Blood analysis 
Blood sample collection for exploratory analysis. 
Other 
In study participants who report severe and/or serious headache, other diagnostic 
procedures including but not limited to CT scan, MRI (Gadolinium-enhanced 
preferred) and/or lumbar puncture for CSF collection are to be performed if 
indicated at the discretion of the investigator. 
For study participants who experience suspected aseptic meningitis 
Lumbar puncture 
In study participants who reported signs and/or symptoms of meningitis which 
required a lumbar puncture, results of the CSF analysis should be recorded in the 
eCRF, and preliminary data should be included on the SAE form used for 
reporting the event as an AESM within 24 hours (ie, preliminary data reported on 
the first reporting may not have CSF results yet, but the reporting should occur as 
soon as there is a suspected diagnosis. Full results should be communicated in 
subsequent exchanges with UCB).  
Additional 
analysis 
Results of all investigations should be recorded in the eCRF, and preliminary data 
should be included on the SAE form used for reporting the event as an AESM. 
Please include details on all investigations results including but not limited to 
blood or CSF cultures and analysis/ PCR test (including list of microorganisms 
tested) / MRI scans +/- gadolinium. 
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Table 1-4: 
Additional study assessments 
Assessment 
When applicable 
AESM=adverse event of special monitoring; CSF=cerebrospinal fluid; CT=computed tomography; 
eCRF=electronic case report form; MRI=magnetic resonance imaging; PCR=polymerase chain reaction; 
SAE=serious adverse event 
The frequency of the collection of samples for exploratory biomarkers after severe and/or serious 
headache or suspected aseptic meningitis are described in Table 1-3 (footnote r). 
2 
INTRODUCTION 
Rozanolixizumab is a humanized monoclonal antibody being developed as an inhibitor of 
neonatal Fc receptor (FcRn; the major histocompatibility complex-class-I-like FcRn) activity 
with the aim to reduce the concentration of (pathogenic) IgG in patients with IgG autoantibody-
mediated diseases, including gMG. 
The FcRn recycles IgG and albumin and transports it bidirectionally across epithelial barriers. 
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). Neonatal Fc receptor for IgG 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. 
As individual disease entities, IgG autoantibody-mediated conditions are relatively rare. 
Treatment of these disorders remains a difficult clinical problem, requiring in many of these 
conditions the long-term use of corticosteroids alone or combined with cytotoxic agents. These 
therapeutic approaches are not effective in all 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 intravenous immunoglobulin (IVIg), are being 
used as primary and secondary therapy of autoimmune diseases, particularly where corticosteroid 
based immune suppression is not or no longer effective. 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, removal of the IgG autoantibodies by FcRn blockade may provide an effective 
therapeutic option for IgG autoantibody-mediated autoimmune disorders. 
To date, rozanolixizumab has been administered to human study participants in several clinical 
studies. 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. 
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2.1 
Study rationale 
Studies in patients with gMG have demonstrated improvements across a range of efficacy 
endpoints with rozanolixizumab treatment. These studies also indicated that rozanolixizumab has 
an acceptable safety profile and is generally well tolerated. 
Generalized myasthenia gravis is a chronic, autoimmune disease requiring long-term therapy. 
Successful self-administration of SC treatment has been demonstrated in gMG patients using Ig 
(Alcantara et al, 2021). Currently, rozanolixizumab is administered SC by HCPs using 
programmable syringe drivers at a 
. There is considerable 
clinical interest in providing an alternative administration method to programmable syringe 
drivers as well as providing patients the option to self-administer rozanolixizumab. Given the 
relatively low volumes involved in rozanolixizumab therapy, the manual push technique is being 
explored as an alternative method, which offers the advantages of independence from constant 
use of a syringe driver, and reduced infusion times (
 for 
abdominal sites). The manual push technique is also a well-established mode of self-
administration of SC Ig (Bienvenu et al, 2018). 
Rozanolixizumab has been administered using manual push in an ongoing 
 healthy 
volunteer study (UP0106) and the manual push administration by HCP is planned to be included 
in gMG study MG0007; other indications may offer alternative administration options for 
rozanolixizumab in Phase 3 open-label extension studies. 
Following a comprehensive training (consisting of 
 with the study participant 
practicing both methods at the study site), rozanolixizumab will be self-administered by both 
manual push and syringe driver in the clinic and unsupervised by an HCP at home. In addition to 
the evaluation of successful self-administration and safety, the participant’s preference for HCP 
administration versus self-administration and preference for the manual push versus the syringe 
driver method of administration will be compared. 
2.2 
Background 
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 production of IgG autoantibodies directed toward nicotinic 
acetylcholine receptor, or muscle-specific kinase 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, corticosteroids, biologics, 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. 
In a randomized, participant- and investigator-blind, placebo-controlled, Phase 2 study 
(MG0002), 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 
 compared with placebo were observed in reductions from Baseline 
in Myasthenia Gravis Composite (MG-C) score (p=0.089) and Quantitative Myasthenia Gravis 
(QMG) score (p=0.221). Overall, repeated administrations of rozanolixizumab at dose levels of 
 SC have been generally well tolerated, with an acceptable safety profile. 
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In the Phase 3 gMG program, clinically relevant improvements in day-to-day functioning were 
observed following treatment with rozanolixizumab compared with placebo. 
Data suggest that repeated administrations of rozanolixizumab at a dose approximating 
 
and 
 SC is generally well tolerated, with an acceptable safety profile. 
Treatment-emergent AEs were most frequently reported in the system organ class of nervous 
system disorders. Rapid, substantial, and sustained reductions in levels of total IgG and IgG 
subclasses (IgG 1 to 4) were observed after rozanolixizumab was administered.  
In the randomized, double-blind, placebo-controlled, Phase 3 study (MG0003), the 
 
clinical efficacy of rozanolixizumab was demonstrated by statistically significant results for the 
primary endpoint. At Day 43 (Visit 10), the differences in least square mean change from 
Baseline in MG-ADL score between groups (rozanolixizumab minus placebo) were: 
 
(95% confidence interval [CI]: 
; p<0.001) in the rozanolixizumab 
 group 
(in favor of rozanolixizumab) and 
 (95% CI: 
; p<0.001) in the 
rozanolixizumab 
 group (in favor of rozanolixizumab). Clinically relevant and 
statistically significant reductions (improvements) from Baseline in MG-C score and QMG 
scores were observed for each rozanolixizumab treatment group versus placebo. Statistically 
significant improvements in MG Symptoms patient reported outcomes (PROs) “Muscle 
Weakness Fatigability”, “Physical Fatigue”, and “Bulbar Muscle Weakness” scores were 
observed for both rozanolixizumab treatment groups. In addition, results for all other efficacy 
endpoints were consistent and supported the primary endpoint. The most frequently reported 
TEAEs in rozanolixizumab-treated study participants were headache, diarrhoea, pyrexia, nausea, 
and arthralgia. 
In a randomized, open-label extension study of MG0003 (MG0004), clinically relevant 
improvements from Baseline in the secondary efficacy endpoints MG-ADL, MG-C, and QMG 
mean total scores over time were observed for both the rozanolixizumab 
 
treatment groups. The most frequently reported TEAEs were headache, diarrhea, blood 
immunoglobulin G decreased, nausea, pyrexia, and urinary tract infection. 
MG0007 is a randomized, open-label extension study of MG0003 and MG0004 having as 
primary objective to assess the safety and tolerability of additional 
 treatment cycles with 
rozanolixizumab in study participants with gMG. Other study objectives are to assess the 
efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of rozanolixizumab. Study 
participants are randomized to receive an initial fixed 
 treatment cycle of a SC dose of 
rozanolixizumab equivalent to 
. At the end of the Treatment 
Period, study participants will enter an Observation Period. In case of symptom worsening, 
resulting in a need for additional treatment, study participants will undergo another 
 
treatment cycle followed by an Observation Period, based on the investigator’s discretion.  
2.3 
Benefit/risk assessment 
Generalized MG is a rare, debilitating, chronic autoimmune disease driven by, in large part, IgG 
autoantibodies that target neuromuscular junctions (NMJs). Most current treatment approaches 
are not targeted to the specific underlying pathology of IgG autoantibody formation, but rather 
they produce a broad cascade of immune suppression, which results in undesirable side effects 
such as those seen with high-dose chronic steroid use. Many treatments of choice often require 
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invasive, expensive, and time-consuming inpatient procedures such as plasma exchange (PEX), 
or intravenous (IV) administration of Ig at a healthcare facility. 
Rozanolixizumab represents an innovative, SC anti-FcRn monoclonal antibody that may provide 
a novel and specific targeted therapeutic approach for the treatment of patients with gMG. Data 
show that rozanolixizumab markedly lowers serum IgG and IgG autoantibody levels in patients 
with gMG. The completed Phase 3 studies have established evidence of efficacy for the 
treatment of gMG. Repeated administrations of rozanolixizumab were well tolerated with an 
acceptable safety profile. 
In MG0003, a Phase 3 study evaluating efficacy and safety of SC rozanolixizumab in adult study 
participants with gMG, the
 clinical efficacy of rozanolixizumab was demonstrated by 
improvements versus placebo in all efficacy endpoints tested in the study. There were clinically 
meaningful and statistically significant reductions from Baseline in the primary endpoint, 
MG-ADL score, at Day 43 for both rozanolixizumab dose groups versus placebo. 
The identified adverse drug reactions (ADRs) associated with SC administration of 
rozanolixizumab are headaches, diarrhoea, pyrexia, nausea, upper respiratory tract infections, 
arthralgia, rash, injections site reactions, vomiting, myalgia, and herpes simplex infections. 
Headache is the most commonly reported ADR. Headaches were mostly mild to moderate and 
easily managed with over-the-counter medications. Serious infections and hypersensitivity 
reactions are safety concerns with rozanolixizumab. Other safety topics of interest include effects 
on vaccination response, effects on the kidney, reductions in albumin and plasma proteins, 
drug-induced aseptic meningitis (DIAM), and 
 These risks can be 
mitigated by careful monitoring, exclusion of at-risk study participants, and appropriate protocol 
withdrawal and stopping criteria. Additionally, protocol guidance for management of severe 
and/or serious headaches (AESM), suspected aseptic meningitis (AESM), medication error 
associated with adverse reaction(s) (AESM), hypersensitivity reactions, and infections is also 
provided as well as expedited reporting requirements of AESM to UCB. 
Restrictions on the use of live vaccines have been defined in exclusion criterion 7a. If 
vaccination with non-live vaccines (including coronavirus disease 2019 [COVID-19] vaccines) is 
considered necessary once a study participant has started treatment with rozanolixizumab, the 
degree of protection afforded with a vaccine may be compromised while the participant receives 
treatment. Based on its mechanism of action, rozanolixizumab will reduce total IgG levels 
including vaccine-specific IgG. Immunization with vaccines during rozanolixizumab treatment 
has not been studied and the response to immunization with any vaccine is unknown. Given the 
study population characteristics (eg, status of the underlying disease, concomitant 
immunosuppressive therapies), it is recommended to perform individualized benefit-risk 
assessment for vaccination and specifically vaccination against COVID-19 infection. If 
COVID-19 vaccination is planned, information regarding the vaccine should be recorded 
(Section 6.5.1). A COVID-19 vaccination should be scheduled, if at all possible, to allow 
differentiation of safety profiles of rozanolixizumab and vaccine (eg, a minimum of 72 hours 
between COVID-19 vaccination and rozanolixizumab administration). If any AEs occur, they 
should be handled as described in Section 8.4 with causality assessment provided for both 
rozanolixizumab and vaccine. Additionally, to further characterize the effect of rozanolixizumab 
on COVID-19 vaccination response, measurements of vaccine titers are being tested in 
rozanolixizumab development programs. 
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Considering the potential benefits, risks, and mitigation measures in place, UCB considers the 
overall benefit/risk to be favorable for participants in this study. 
More detailed information about the known and expected benefits and risks and reasonably 
expected AEs of rozanolixizumab may be found in the Investigator’s Brochure. 
3 
OBJECTIVES AND ENDPOINTS 
Objectives 
Endpoints 
Primary 
Primary objective: 
To evaluate the ability of study 
participants with gMG to successfully 
self-administer rozanolixizumab after 
training in the self-administration 
technique using the syringe driver 
and manual push methods 
Primary endpoint: 
• Successful self-administration of rozanolixizumab 
(with correct use of syringe driver and manual push, 
respectively) during the Self-administration Period 
at Visit 13 (
; last dose of Self-
administration Period 1) and Visit 19 (
; last 
dose of Self-administration Period 2). 
 
Successful self-administration is defined by the 
participant (i) choosing the correct infusion site, (ii) 
administering SC, and (iii) delivering the intended 
dose. 
Secondary 
Secondary objective: 
• To evaluate the safety of SC self-
administration of 
rozanolixizumab 
Secondary endpoints: 
• Occurrence of TEAEs after syringe driver or manual 
push self-administration from Visit 2 (
 up to 
the End of Study Visit (Visit 21). 
• Occurrence of local site reactions up to 24 hours 
after each administration during the Training Period 
and Self-administration Periods. 
• Occurrence of medication errors associated with 
adverse reactions during the 2 Self-administration 
Periods of the study. 
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Other 
Other objectives: 
• To evaluate the study 
participant’s preferred method of 
rozanolixizumab administration 
Other endpoints: 
• Participant’s relative preference for: 
− Subcutaneous infusions performed by an HCP 
versus self-administration 
− The manual push method versus the use of a 
syringe driver for self-administration 
using the participant’s preferred method of 
administration questions at Visit 19 (
 
• To evaluate symptom changes in 
study participants with gMG 
• Myasthenia Gravis Activities of Daily Living score 
change from Baseline during the study. 
• To assess the pharmacodynamics 
of rozanolixizumab 
• Total IgG level over time during the Training Period 
and Self-administration Periods. 
• To evaluate the immunogenicity 
of rozanolixizumab following SC 
self-administration 
• Anti-rozanolixizumab antibodies (status and titer at 
trough during both Self-administration Periods). 
• To assess the study participant’s 
experience with self-
administration of SC infusions at 
home 
• 
• To assess the ability of study 
participants with gMG to 
successfully self-administer 
rozanolixizumab at home after 
training in the self-administration 
technique using the syringe driver 
and manual push methods 
• Successful self-administration of rozanolixizumab 
via manual push method or using a syringe driver at 
each of the 3 home self-administration visits during 
Self-administration Period 1 or 2, respectively. 
Successful self-administration via manual push or 
syringe driver is defined by the participant (i) 
choosing the correct infusion site, (ii) administering 
SC, and (iii) delivering the intended dose, as 
reported by the study participant to the site staff 
following each of the 3 home self-administrations. 
• To assess the safety and 
tolerability of rozanolixizumab in 
study participants with gMG 
• Occurrence of TEAEs leading to permanent 
withdrawal of rozanolixizumab during the study. 
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4 
STUDY DESIGN 
4.1 
Overall design 
MG0020 is a Phase 3, open-label, randomized, 2-period, 2-sequence crossover study planned to 
be conducted at multiple sites in Europe, Japan, and North America. In MG0020, the safe and 
effective self-administration of rozanolixizumab using the syringe driver and manual push 
administration methods by study participants with gMG will be evaluated using a crossover 
design (Table 1-1). Study participants are proposed to receive 
 weight tiered doses of 
rozanolixizumab for 
, as summarized in Table 1-2.  
The weight tiered dosing will only be applicable to the study participants starting the Treatment 
Period after the approval of Protocol Amendment 4 in their respective countries. The study 
participants who are already in the Treatment Period when Protocol Amendment 4 is approved in 
their respective countries will continue with the 
 fixed doses as per Protocol 
Amendment 3 until study completion. 
Rozanolixizumab-naïve study participants and study participants previously exposed to 
rozanolixizumab will be included in the study.  
A total of 75 participants have been screened to achieve a minimum of 30 randomly assigned and 
evaluable participants for an estimated minimum total of 15 evaluable participants per sequence. 
See Section 9.9 for the determination of sample size. 
The total duration for each study participant is up to 29 weeks, including a Screening Period of 
up to 4 weeks, an 
 Treatment Period, and an up to 7-week Safety Follow-up Period (ie, 
End of Study Visit is 8 weeks after the last dose of rozanolixizumab in the Treatment Period, or 
earlier, if the study participant moves on to a post-trial access program or to commercially 
available rozanolixizumab during the Safety Follow-up Period). 
The 
 Treatment Period of MG0020 starts with standardized 
 trainings over the 
course of a 
 Training Period when the study participant is trained on both the manual push 
and the syringe driver administration methods. During the Training Period, the study participant 
will receive 
 doses of rozanolixizumab SC and should practice both methods of 
administration at the study site. To act as a reference for the preference question (HCP vs self-
administration) and to demonstrate subcutaneous infusions of rozanolixizumab, the first 
administration will be imperatively performed by the HCP. For all remaining training visits, the 
study participant should perform as many self-administrations as possible (under HCP 
supervision) depending on their training status. Self-administration under HCP supervision or 
administration by HCP will be documented in the CRF. At Visit 8 (
), after the completion 
of the Training Period and following the investigator’s confirmation of the eligibility to perform 
self-administration, the study participant will be randomized 1:1 to either the syringe driver or to 
the manual push administration method. After completing Self-administration Period 1 (at 
Visit 13 [
]), the study participant will crossover to the alternative administration 
method, entering Self-administration Period 2 (Visit 14 [
]; see the study schema in 
Section 1.2). A final release of the study participant for home self-administration will be based 
on a successful demonstration of self-administration using the assigned mode of administration 
(manual push or syringe driver use) at Visit 9 (
 and Visit 15 (
), respectively. 
Study participants who cannot be confirmed eligible for self-administration will not perform 
self-administration and can continue to be treated on-site per protocol based on investigator 
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decision. Additionally, if a study participant becomes no longer eligible for self-administration 
during the Self-administration Periods, he/she can continue to be treated by HCP on-site per 
protocol based on investigator decision. The study medication administration can be performed 
either via syringe driver or manual push. Adverse events, concomitant medication, vital signs, 
ECG, suicidality, blood and urine samples for safety laboratory tests, pregnancy tests, and IgG 
will continue to be collected from these study participants until End of Study Visit/Early 
Withdrawal Visit. Handling of replacement participants is described in Section 7.2. 
During both Self-administration Period 1 and Self-administration Period 2, 
 
 (ie, unsupervised/not in the presence of an 
HCP) for 
 
After the Self-administration Periods, study participants will enter the 7-week Safety Follow-up 
Period. 
Study participants who have completed all Treatment Periods (including the End of Treatment 
Visit) will have the option to subsequently move, at the discretion of the investigator, into either 
a post-trial access program for rozanolixizumab (if available according to local guidance) or on 
to commercially available rozanolixizumab. 
If required, only minimal assistance is allowed to support the study participant for a successful 
self-administration. However, if an informal caregiver is required, he/she must be fully trained 
(ie, attending all training sessions at the site) to perform the administration in this study on behalf 
of the study participant. In this case, the caregiver must perform all investigational medicinal 
product (IMP) administration activities described for the study participant throughout the 
Self-administration Periods. 
4.1.1 
Unscheduled visit 
An Unscheduled Visit can be conducted at the discretion of the investigator (eg, due to an AE; 
see Section 8). 
4.2 
Scientific rationale for study design 
Manual push administration of SC Ig treatments has been demonstrated to be a safe and viable 
technique in primary immunodeficiency patients that allows individualization of treatment 
(Cowan et al, 2021, Bienvenu et al, 2018, Shapiro 2013). 
Manual push administration of a 
 rozanolixizumab SC dose has been performed (
 
) in an ongoing 
 healthy volunteer study 
(UP0106). Preliminary pharmacokinetic and local tolerability data suggest that administration 
via manual push is comparable to administration using a syringe driver. 
MG0020 will be conducted in study participants with gMG to evaluate the relative preference for 
HCP versus self-administration and the manual push method versus the syringe driver mode of 
administration in this patient population. 
An open-label design is required as the study will evaluate self-administration of 
rozanolixizumab using the syringe driver and manual push methods. 
A crossover design is used to allow each study participant to evaluate both methods of 
self-administration. 
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4.3 
Justification for dose 
The proposed weight-tiered doses of rozanolixizumab in Europe and Canada, 
 
(see Table 1-2), are equivalent to the weight-tiered dose approximating 
 evaluated in the 
Phase 3 studies in gMG. Based on the positive benefit-risk profile of the studied weight-tiered 
doses of 
 rozanolixizumab in the MG0003 Phase 3 pivotal study in gMG, 
a dose of 
 is proposed for this study as no consistent incremental benefit of the 
 dose compared with the 
 dose was observed. Pharmacodynamic data showed 
a desired reduction in IgG levels of 
 from Baseline for both weight-tiered doses.  
The proposed weight-tiered doses of rozanolixizumab in the US, 
 (see 
Table 1-2), are consistent with the recommended doses in the FDA-approved US Prescribing 
Information. 
4.4 
End of Study definition 
A participant is considered to have completed the study if he/she has: 
• Completed all periods of the study including the End of Study Visit (Visit 21)  
or 
• Completed all the Treatment Periods, including the End of Treatment Visit, and has 
subsequently moved on to a post-trial access program 
or 
• Completed all the Treatment Periods, including the End of Treatment Visit, and has 
subsequently moved on to commercially available rozanolixizumab 
Participants moving to either a post-trial access program or commercially available 
rozanolixizumab during the Safety Follow-up Period must undergo an earlier End of Study Visit 
prior to this move. 
The global end of the study is defined as the date of the last visit of the last participant in the 
study. 
5 
STUDY POPULATION 
Prospective approval of protocol deviations to recruitment and enrollment criteria, also known as 
protocol waivers or exemptions, is not permitted. 
5.1 
Inclusion criteria 
Participants are eligible to be included in the study only if all of the following criteria apply: 
Age 
1. Participant must be ≥18 years of age inclusive at the time of signing the informed consent 
form (ICF). 
Type of participant and disease characteristics 
2. Study participant must have a documented diagnosis of gMG, based on study participant’s 
history and supported by previous evaluations. 
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3. Study participant is willing to perform and capable of performing (according to the 
investigator’s judgment) home self-administration by using both the syringe driver method 
and the manual push method (or caregiver is willing to perform and capable of performing 
the administration). 
4. Study participant is considered by the investigator for additional rozanolixizumab treatment 
with the posology proposed in this study. 
5a. Study participant has a serum total IgG level ≤16g/L and ≥5.5g/L at Screening (Visit 1) 
(except for study participants receiving an anti-FcRn treatment within 8 weeks prior to the 
Screening Visit, who must have a serum total IgG level at the Screening Visit ≥2g/L).  
Weight 
6. Body weight ≥35kg at Screening (Visit 1). 
Sex 
7. Study participants may be male or female: 
a) A female participant is eligible to participate if she is not pregnant (see Appendix 4 
[Section 10.4]), not breastfeeding, and at least one of the following conditions applies: 
i) Not a woman of childbearing potential (WOCBP) as defined in Appendix 4 
(Section 10.4) 
OR 
ii) A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 
(Section 10.4) during the Treatment Period and for at least 90 days after the last dose 
of study medication. The study participant must have a negative serum pregnancy test 
at Screening (Visit 1) and a negative urine pregnancy test before the first dose of 
study medication during the Training Period and each of the Self-administration 
Periods. 
Informed consent 
8. Capable of giving signed informed consent, which includes compliance with the 
requirements and restrictions listed in the ICF. 
9. Study participant is considered reliable and capable of adhering to the protocol visit schedule, 
or medication intake according to the judgment of the investigator. 
5.2 
Exclusion criteria 
Participants are excluded from the study if any of the following criteria apply: 
Medical conditions 
1. Study participant has any medical or psychiatric condition or significant laboratory 
abnormality that, in the opinion of the investigator, could jeopardize or would compromise 
the study participant’s ability to participate in this study. 
2. Study participant has a history of alcohol use disorder or other substance use disorder (as per 
Diagnostic and Statistical Manual of Mental Disorders-5 [American Psychiatric Association, 
2013]) within the previous 12 months before Screening (Visit 1). 
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3a. Study participant has a known hypersensitivity to other anti-FcRn medications, to any 
components of the study medication, to any of the excipients (including 
), or 
has a known history of 
, since both 
 and 
are 
constituents of the rozanolixizumab formulation. 
4.  Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB 
infection, or latent tuberculosis infection (LTBI), or current or history of nontuberculous 
mycobacterial infection (NTMBI).  
5. Study participant has a clinically relevant active infection or a history of serious infection 
(resulting in hospitalization or requiring IV antibiotic treatment) within 6 weeks before the 
Baseline Visit (Visit 2 [
 
6a. The study participant previously participated in any rozanolixizumab MG study and met any 
mandatory withdrawal criteria (unless the reason is directly related to MG0020 participation) 
or mandatory study drug discontinuation criteria. 
Prior or concomitant therapy 
7a. Study participant has received a live vaccination within 4 weeks before starting treatment, or 
a BCG vaccine within 1 year before starting treatment; or intends to have a live vaccination 
during the course of the study or within 8 weeks following the last dose of rozanolixizumab. 
8. Study participant received treatment with rituximab or other anti-CD20 or anti-CD19 
medication within 
 before the Baseline Visit (Visit 2 [
 
9a. Study participant has received treatment with IVIg, SC Ig, or PEX within 
 before the 
Baseline Visit (Visit 2 
 treatment with efgartigimod within 
 before the 
Baseline Visit; treatment with eculizumab 
 before the Baseline Visit; treatment with 
ravulizumab within 
 before the Baseline Visit or prior treatment with 
cyclophosphamide. 
Diagnostic assessments 
10. Study participant has absolute neutrophil count <1500 cells/mm3 at Screening (Visit 1). 
11. 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. 
12. Study participant has renal impairment, defined as glomerular filtration rate less than 
30mL/min/1.73m2 at Screening (Visit 1). 
13. Study participant has 12-lead electrocardiogram (ECG) at Screening (Visit 1) 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. 
14. Study participant has >3.0xULN of any of the following: alanine aminotransferase (ALT), 
aspartate aminotransferase (AST), or alkaline phosphatase (ALP) at Screening (Visit 1). 
− If participant has >upper limit of normal (ULN) for ALT, AST, or ALP that does not 
meet the exclusion limit at Screening (Visit 1), repeat the tests, if possible, before dosing 
to ensure there is no further ongoing clinically relevant increase. 
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− For participants with a Baseline result >ULN for ALT, AST, ALP, or total bilirubin, a 
Baseline diagnosis and/or the cause of any clinically meaningful elevation must be 
recorded in the CRF. 
15. Study participant has bilirubin >1.5xULN at Screening (Visit 1) (unless confirmed Gilbert’s 
syndrome). If participant has elevations only in total bilirubin, the fractionate bilirubin needs 
to be checked, to identify possible undiagnosed Gilbert’s syndrome (ie, direct bilirubin 
<35%). 
16. Current unstable liver or biliary disease, at Screening (Visit 1), 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). 
17. Presence of Hepatitis B surface antigen (HBsAg) or positive Hepatitis C antibody test result 
at Screening (Visit 1). Note: a study participant with a positive hepatitis C antibody test due 
to prior resolved disease can be enrolled only if a confirmatory negative Hepatitis C RNA 
test is obtained. 
18. Study participant tests positive for human immunodeficiency virus at Screening (Visit 1). 
19a. Study participant has active malignant neoplastic disease or history of malignant neoplastic 
disease within 5 years of study entry before Screening (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 or thymoma that did not require 
chemotherapy and/or radiotherapy after removal). 
Other 
20. Study participant has a lifetime history of 
 
 as indicated by a positive 
response (Yes) to either Question 4 or Question 5 of the Columbia-Suicide Severity Rating 
Scale (C-SSRS) at Screening (Visit 1). 
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 participants who consent to participate in the clinical study but are 
not subsequently allocated to treatment during the Training Period. A minimal set of screen 
failure information is required to ensure transparent reporting of screen failure participants to 
meet the Consolidated Standards of Reporting Trials publishing requirements and to respond to 
queries from regulatory authorities. Minimal information includes demography, screen failure 
details, eligibility criteria, and any serious adverse event (SAE). 
Individuals who do not meet the criteria for participation in this study (screen failure) may be 
rescreened once. Additional rescreening of these individuals following upfront discussion with 
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the medical monitor or study physician might be allowed. Rescreened study participants should 
be assigned a new participant number for rescreening. 
If a study participant has 1 isolated test result outside the specific range which is deemed 
clinically nonsignificant, the abnormal value may be rechecked at the discretion of the 
investigator, following consultation with the sponsor’s medical monitor or study physician. If the 
normalization of the test result occurs within the Screening Period, then no other Screening 
procedures need to be repeated. 
6 
STUDY TREATMENTS 
Study treatment is defined as any investigational treatment(s), marketed product(s), placebo, or 
medical device(s) intended to be administered to a study participant according to the study 
protocol. Treatments in this study vary based on either an approved label in a region (US) or a 
proposed dosing under review in a region (Europe and Canada).  
6.1 
Treatments administered 
Study treatment name 
Treatment 
Treatment 
Intervention name 
Rozanolixizumab 
Rozanolixizumab 
Type 
Biologic 
Biologic 
Dose formulation 
 
 
Unit dose strength(s) 
Dosage level(s) US 
 fixed doses of 
 for 
study participants <50kg, 
 for 
study participants ≥50kg to <100kg 
or 
 for study participants 
≥100kg. 
 fixed doses of 
 for 
study participants <50kg, 
 for 
study participants ≥50kg to <100kg 
or 
 for study participants 
≥100kg. 
Dosage level(s) Europe 
and Canada 
 fixed doses of 
 for 
participants ≥35kg to <50kg, 
 
for study participants ≥50kg to 
<70kg, 
 for study participants 
≥70kg to <100kg or 
 for 
study participants ≥100kg. 
 fixed doses of 
 for 
participants ≥35kg to <50kg, 
 
for study participants ≥50kg to 
<70kg, 
 for study participants 
≥70kg to <100kg or 
 for 
study participants ≥100kg. 
Route of 
administration 
Subcutaneous infusion via syringe 
driver 
Subcutaneous infusion via manual 
push 
Use 
Experimental 
Experimental 
IMP and NIMP 
IMP 
IMP 
Sourcing 
Provided centrally by the sponsor 
Provided centrally by the sponsor 
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Packaging and labeling 
Packaging will be described in the 
IMP Handling Manual. Packaging 
will be labeled as required per 
country requirement. 
Packaging will be described in the 
IMP Handling Manual. Packaging 
will be labeled as required per 
country requirement. 
Current/Former 
name(s) or alias(es) 
UCB7665 
UCB7665 
Abbreviations: IMP=investigational medicinal product; NIMP=noninvestigational medicinal product 
 
6.1.1 
Medical devices 
Other medical devices (not manufactured by or for UCB) are provided for SC infusion. Medical 
devices used as part of this clinical study will be used within their approved intended use. Proof 
of registration will be kept on file and available for inspection. 
Instructions for use are provided in the IMP Handling Manual. 
All adverse device effects (ADEs), serious adverse device effects (SADEs), and device 
deficiencies (including malfunctions, use errors, and inadequate labeling) shall be documented 
and reported by the investigator throughout the study (see Section 8.3.9) and appropriately 
managed by the sponsor. 
6.2 
Preparation, handling, storage, and accountability requirements 
Details on the preparation of study medication for infusion, methods of administration, rate of 
infusion, as well as supply of kits for self-administration are provided in the IMP Handling 
Manual. 
The investigator or designee and the study participant (at home self-administration visits) must 
confirm that appropriate temperature conditions have been maintained during transit for all study 
medication received, and any discrepancies are reported and resolved before use of the study 
medication. 
Only participants enrolled in the study may receive study medication. All study medication 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 or 
authorized site staff and to the study participant or caregiver during home self-administration 
visits. 
The investigator, institution, or the head of the medical institution (where applicable) is 
responsible for study medication accountability, reconciliation, and record maintenance 
(ie, receipt, reconciliation, and final disposition records). 
In case an out-of-range temperature is noted, it must be immediately reported as per instructions 
contained in the IMP Handling Manual. 
The investigator (or designee) will instruct the participant to store the study medication 
following the instructions on the label and in the IMP Handling Manual. 
Further guidance and information for the final disposal of unused study medication are provided 
in the Study Reference Manual. 
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6.2.1 
Drug/Device accountability 
A Drug/Device 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 (or designee) is responsible for retaining all used, unused, and partially used 
containers of study medication until returned or destroyed. 
The investigator may assign some of the investigator’s duties for drug accountability at the study 
site to an appropriate pharmacist or 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 
or partially used containers), unused, damaged, and/or expired study medication must be 
reconciled and either destroyed at the site according to local laws, regulations, and UCB standard 
operating procedures or returned to UCB (or designee). Investigational medicinal product 
intended for the study cannot be used for any other purpose than that described in this protocol. 
6.3 
Measures to minimize bias: randomization and blinding 
This is an open-label study without blinding; however, the order that a specific intervention is to 
be taken by a participant will be assigned using an interactive response technology (IRT). 
Potential bias will be reduced using central randomization. The IRT will be used for assigning 
eligible participants to a treatment sequence (see Table 1-1) 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 participant will receive a 5-digit 
number assigned at Screening that serves as the participant identifier throughout the study. The 
participant number will be required in all communication between the investigator or designee 
and the IRT regarding a particular participant. Participant numbers and kit numbers will be 
tracked via the IRT.  
At Visit 2 (Baseline), the investigator or designee will contact the IRT to confirm participant’s 
eligibility (including the participant’s body weight as recorded at Screening). The IRT will 
allocate kit numbers to the participant. 
At Visit 8, in order to randomize a participant to a treatment sequence (see Table 1-1), the 
investigator or designee will contact the IRT and provide brief details about the participant to be 
randomized. The IRT will automatically inform the investigator or designee of the participant’s 
randomization number. The IRT will allocate kit numbers to the participant based on the 
participant number during the course of the study. The randomization number must be 
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incorporated into the CRF. The site will record the intervention assignment on the applicable 
CRF, if required. 
6.4 
Treatment compliance 
Participants must return all used and unused vials as well as all equipment and ancillary 
materials used for IMP administration at Visit 13 (
) and Visit 19 (
). Drug 
accountability must be done in the participant’s presence in order to obtain explanations 
regarding discrepancies in compliance with the dosing regimen. Drug accountability must be 
recorded on the Drug Accountability form. 
The intended dose is based on Table 1-2 (the acceptable administered range is ≥80% of the total 
volume used based on visual inspection of the residual IMP at site).  
For home self-administrations, the study participant should provide a respective photographic 
documentation to allow the HCP to assess the volume of IMP administered, using the mobile 
phone provided by UCB. Further details are provided in the IMP Handling Manual.  
If a participant is found to be persistently noncompliant (eg, does not comply with visit schedule 
or protocol procedures), the sponsor, in conjunction with the investigator, will make a decision 
as to whether the participant should be withdrawn from the study. 
6.5 
Concomitant medication(s)/treatment(s) 
6.5.1 
Permitted concomitant treatments (medications and therapies) 
Concomitant medications listed in Table 6-1 are permitted during the study for the treatment of 
gMG. With the exception of corticosteroids and acetylcholinesterase inhibitors, the dose of 
permitted concomitant medications should be maintained, if possible. 
If a study participant receives a COVID-19 vaccine, the product name and date of 
administration(s) should be captured in the CRF as a concomitant medication. 
Table 6-1: 
Permitted concomitant treatments 
Permitted medications 
Dose 
Oral corticosteroids (eg, prednisolone) 
No specific requirements 
Methotrexate 
≤30mg/week 
Mycophenolate mofetil  
≤3g/day 
Cyclosporinea 
≤5mg/kg/day for unmodified 
≤4mg/kg/day for modified (microemulsion) 
Azathioprine 
≤3mg/kg/day 
Cholinesterase inhibitors 
≤600mg pyridostigmine/day 
Tacrolimusb 
≤5mg/day 
a Doses higher than listed are permissible if trough level is ≤300ng/mL. 
b If the total daily weight-based dose is >5mg, then a plasma trough level should be checked to ensure study 
participant is not above the recommended therapeutic range. 
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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) 
6.5.3 
Treatments specific to NMJ interference 
Treatments that could interfere with the function of the NMJ (and which therefore could impair 
study participants with gMG) include, but are not limited to, the following medications: 
• Botulinum toxin 
• Aminoglycoside antibiotics  
• Tetracycline antibiotics  
• Penicillamine 
• Magnesium 
For a more detailed list please refer to the Myasthenia Gravis Foundation of America (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 before initiating therapy that can affect the NMJ. 
6.5.4 
Rescue medication 
The study site will supply rescue therapy that will be obtained locally. Rescue therapy will be 
given as per standard of care and at the discretion of the investigator. Study participants who 
continue to experience moderate to severe symptoms despite treatment with rozanolixizumab 
may receive treatment with the following as rescue therapy: 
• Intravenous Ig (ATC code: 
 [Immunoglobulins, normal human]) 
• Subcutaneous Ig (ATC code: 
 [Immunoglobulins, normal human]) 
• Plasma exchange or plasmapheresis 
• Intravenous corticosteroids at a higher dose than previous oral dose (ATC code: 
 
[Corticosteroids for systemic use, plain]) 
Study participants who received treatment with rescue therapy will be withdrawn from the study.  
The date and time of rescue medication administration as well as the name and dosage regimen 
of the rescue medication must be recorded.  
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6.6 
Dose modification 
There is no dose modification allowed in this study, ie, study participants must maintain their 
dose (based on body weight as recorded at Screening) throughout. 
6.7 
Criteria for study hold or dosing stoppage 
Not applicable. 
6.8 
Treatment after the end of the study 
Study participants who have completed all Treatment Periods (including the End of Treatment 
Visit) will have the option to subsequently move, at the discretion of the investigator, into either 
a post-trial access program for rozanolixizumab (if available according to local guidance) or on 
to commercially available rozanolixizumab. 
7 
DISCONTINUATION OF STUDY MEDICATION AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL 
7.1 
Discontinuation of study medication 
7.1.1 
Liver chemistry stopping criteria 
Discontinuation of study treatment for abnormal liver function should be considered by the 
investigator when a participant meets one of the conditions outlined Figure 7-1 and Figure 7-2 or 
if the investigator believes that it is in best interest of the participant. 
Figure 7-1: Liver Chemistry Stopping Criteria and Increased Monitoring 
Algorithm 
 
ALT=alanine aminotransferase; AST=aspartate aminotransferase; INR=international normalized ratio; SAE=serious 
adverse event; ULN=upper limit of normal 
 
Continue Study Treatment 
Discontinue Study Treatment 
Plus 
Bilirubin≥2x
ULN (>35% 
direct) or plus
INR>1.5, if 
measured*
Possible  
Hy’s Law
ALT≥3xULN
ALT
≥8xULN
Plus
Symptoms of 
liver injury
or 
hypersensitivity
No
Yes
Yes
Yes
No
No
No
See algorithm 
for continued 
therapy with 
increased liver 
chemistry 
monitoring
Yes
ALT 
≥3xULN 
but 
<8xULN
Yes
Refer to the Liver Safety Required Actions and Follow up Assessments section in 
Appendix 6. 
Report as an SAE if possible Hy’s Law case: AST/ALT ≥3xULN and Bilirubin 
≥2xULN (>35% direct) or INR>1.5, if measured 
(Note: INR value not applicable to patients on anticoagulants) 
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Treatment with study medication may be continued with increased monitoring if a study 
participant meets one of the criteria outlined in Figure 7-2. 
Figure 7-2: Liver Chemistry Increased Monitoring Algorithm with Continued 
Study Intervention for Participants with ALT ≥3xULN but <8xULN 
 
ALT=alanine aminotransferase; AST=aspartate aminotransferase; bili=bilirubin; INR=international normalized 
ratio; SAE=serious adverse event; ULN=upper limit of normal 
Specific assessments and follow-up actions for potential drug-induced liver injury (PDILI) are 
provided in Appendix 6 (Section 10.6). 
7.1.2 
Temporary IMP discontinuation 
Study participants must be TEMPORARILY discontinued from the IMP if any of the following 
events occur: 
1. Symptomatic cases of confirmed COVID-19 infection. The IMP may be restarted if clinically 
appropriate when signs and symptoms have resolved and in agreement with local guidelines. 
2. Study participant has a first diagnosis of suspected aseptic meningitis. The IMP may be 
restarted if clinically appropriate when signs and symptoms have resolved. Refer to 
Section 7.1.3 for permanent discontinuation criteria (criterion #9). 
Study participants may be TEMPORARILY discontinued from the IMP if 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 13 [Section 10.13.2]). 
 
Continue Study Treatment   and   Monitor Liver Chemistry
Discontinue Study Treatment 
ALT≥5xULN 
but <8xULN
+ bili <2xULN +
no symptoms
No
➢Must refer to Liver Safety Required Actions and Follow up Assessments section in the Appendix
Yes
ALT ≥3xULN 
but <5xULN
+ bili <2xULN  +
no symptoms
Able to 
monitor  
weekly 
for  ≥2 
weeks
Persists for 
≥2 weeks  
or other 
stopping 
criteria  
met
No
Yes
Yes
Yes
No
No
Able to 
monitor  
weekly 
for  ≥4 
weeks
Persists for 
≥4 weeks  
or other 
stopping 
criteria  
met
No
Yes
Yes
Yes
Yes
ALT ≥5xULN 
ALT <5xULN 
Yes
Yes
Refer to the Liver Safety Required Actions and Follow up Assessments section in 
Appendix 6. 
Report as an SAE if possible Hy’s Law case: AST/ALT ≥3xULN and Bilirubin 
≥2xULN (>35% direct) or INR>1.5, if measured 
(Note: INR value not applicable to patients on anticoagulants) 
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2. The study participant develops a nonserious 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 13 [Section 10.13.2]). 
3. Asymptomatic or suspected cases of COVID-19 infection. The IMP may be restarted when 
clinically appropriate as deemed by the investigator and in agreement with local guidelines.  
4. Study participant may be temporarily discontinued from study medication at the discretion of 
the investigator in cases deemed strictly necessary for the participant’s medical care. Efforts 
should be made to keep the length of temporary discontinuation to a minimum. 
The investigator should discuss with the medical monitor and/or sponsor’s study physician 
before reinitiating the IMP. As appropriate, virtual assessments could continue (eg, AE 
collection, patient-reported outcome [PRO] assessments as per contingency measures described 
in Section 8). 
If IMP treatment is resumed, the study participant should continue with the next dose as 
previously scheduled and subsequently follow the visit schedule as described in the protocol. The 
CRF should be completed accordingly, including the information if the visit was impacted by 
COVID-19 infection.  
The date and reason for dose hold of rozanolixizumab is to be recorded on each study 
participant’s CRF. 
7.1.3 
Study medication permanent discontinuation criteria 
Study participants must permanently discontinue study medication if any of the following events 
occur: 
1. Study participant develops an illness that would interfere with his or her continued 
participation. 
2a.  Study participant has new onset or reoccurrence of malignant neoplastic disease (except for 
superficial basal or squamous cell carcinoma of the skin not requiring targeted biological 
therapy, chemotherapy or radiation).3.  Study participant experiences an infective episode 
including but not limited to bacteremia or sepsis, infectious meningitis, septic arthritis, 
osteomyelitis, complicated pneumonia, or visceral abscess, which may or may not result in 
hospitalization during a Treatment Period (Appendix 13 [Section 10.13.2]). This list is not 
intended to be all inclusive, and the investigator is expected to apply their judgment on 
continuing IMP based on the clinical situation. 
4.  Study participant meets PDILI permanent discontinuation criteria. 
5.  Study participant has a TB test that is confirmed positive or any further evidence suggestive 
of potential TB infection (ie, exposure) and further examinations result in a diagnosis of 
active TB or LTBI. 
6.  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. 
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7.  Study participant has an AE of severe or serious infusion-related reaction, hypersensitivity, 
or anaphylactic reaction requiring corticosteroid and/or epinephrine therapy (Sampson et al, 
2006; Appendix 13 [Section 10.13.1]). 
8.  Study participant takes prohibited concomitant medications as defined in this protocol.  
9.  Study participant has a recurrence of aseptic meningitis (see also Appendix 14 
[Section 10.14]).  
10. Study participant has 
 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. 
Study participants may permanently discontinue rozanolixizumab at the discretion of the 
investigator (following consultation with the sponsor’s medical monitor or study physician if 
necessary) if the study participant is noncompliant or becomes unable to comply with the study 
procedures or medications in the opinion of the investigator.  
Investigators should contact the medical monitor, whenever possible, to discuss the withdrawal 
of a participant in advance. Investigators should attempt to obtain information on study 
participants in the event of withdrawal (eg, reason for withdrawal, any safety information). 
Study participants who permanently discontinue study medication should undergo an Early 
Withdrawal Visit and move into the Safety Follow-up Period. Handling of replacement 
participants is described in Section 7.2. 
7.2 
Participant discontinuation/withdrawal from the study 
Participants are free to withdraw from the study at any time, without prejudice to their continued 
care. 
Study participants must be withdrawn from the study if any of the following events occur: 
1. Study participant withdraws his/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. 
A participant may withdraw from the study at any time at his/her own request, or may be 
withdrawn at any time at the discretion of the investigator for safety, behavioral, compliance, or 
administrative reasons. 
If study participants discontinue before the end of the Self-administration Period 2, replacement 
participants may be considered at the discretion of the sponsor, depending on how the 
discontinuation affects the study’s ability to collect sufficient data to support the study 
objectives. For each case of participant discontinuation, the decision on whether to recruit a 
replacement and the reason behind the decision will be documented. 
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. 
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See the Schedule of Activities in Section 1.3 for data to be collected at the Early Withdrawal 
Visit and for any further evaluations that need to be completed. In all cases except for 
withdrawal of consent, the study participant should also return to the site for an End of Study 
Visit. 
Investigators should contact the medical monitor, whenever possible, to discuss the withdrawal 
of a study participant in advance. 
7.3 
Lost to follow-up 
A participant will be considered lost to follow-up if he or she repeatedly fails to return for 
scheduled visits and is unable to be contacted by the study site.  
The following actions must be taken if a participant fails to return to the clinic for a required 
study visit: 
• The site must attempt to contact the participant and reschedule the missed visit as soon as 
possible and counsel the participant on the importance of maintaining the assigned visit 
schedule and ascertain whether or not the participant wishes to and/or should continue in the 
study. 
• Before a participant is deemed lost to follow-up, the investigator or designee must make 
every effort to regain contact with the participant (at least 1 phone call and 1 written message 
to the participant) and document his/her effort (date and summary of the phone call and copy 
of the written message in the source documents), to complete the final evaluation. All results 
of these evaluations and observations, together with a narrative description of the reason(s) 
for removing the participant, must be recorded in the source documents. The CRF must 
document the primary reason for withdrawal. 
Should the participant continue to be unreachable, he or she will be considered to have 
withdrawn from the study with a primary reason of lost to follow-up documented in the CRF.  
8 
STUDY ASSESSMENTS AND PROCEDURES 
Study procedures and their timing are summarized in the Schedule of Activities (Section 1.3). 
Protocol waivers or exemptions are not allowed. 
Immediate safety concerns should be discussed with the sponsor 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, is essential and required for study conduct. 
All screening evaluations must be completed and reviewed to confirm that potential participants 
meet all eligibility criteria. 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 4 weeks 
of the assessment timepoint in the Schedule of Activities. 
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An Unscheduled Visit can be conducted at the discretion of the investigator (eg, due to an AE) 
(see Section 4.1.1). 
During the Unscheduled Visit, the following assessments will be performed: 
• AE reporting 
• Concomitant medications  
• Review of withdrawal criteria 
• Physical examination 
• Vital signs 
Blood samples for PK, IgG, hematology, biochemistry, and 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. 
The maximum amount of blood collected from each participant over the duration of the study, 
including any extra assessments that may be required, will not exceed 500mL.  
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 
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 
Not applicable. 
8.2 
Safety assessments 
Planned time points for all safety assessments are provided in the Schedule of Activities 
(Section 1.3). 
8.2.1 
Safe and successful self-administration 
Safe and successful self-administration as defined in the endpoints (Section 3) will be evaluated 
at the site by the HCP supervising the self-administration process and via a mandatory direct 
contact with the study participant via telephone or video call as soon as possible after each home 
self-administration. The direct telephone or video contact with the study participant will also be 
used to document the study participant’s self-assessment on the process of home self-
administration, eg, on potential challenges and deficiencies. 
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The HCP evaluation materials supporting the collection of data for Visit 13 (
) and 
Visit 19 (
) relating to primary endpoints are provided in Appendix 15 (Section 10.15). 
8.2.2 
Participant’s preferred method of rozanolixizumab administration 
questions 
The study participants will be asked to evaluate their relative preference for SC infusions 
performed by an HCP versus self-administration and the preference for the manual push method 
versus the use of a syringe driver for self-administration. 
Materials supporting the collection of data relating to the participant’s preferred method of 
rozanolixizumab administration are provided in Appendix 16 (Section 10.16). 
8.2.3 
Self-Injection Assessment Questionnaire (Infusion version) 
8.2.4 
Myasthenia Gravis Activities of Daily Living 
The MG-ADL is an 8-item PRO instrument developed on the basis of the QMG (Wolfe et al, 
1999). The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and 
axial symptoms. In a recent study, reliability, validity, and responsiveness of the MG-ADL were 
further assessed. The questionnaire showed strong construct validity when evaluated against the 
MG-C as well as against the MG-QOL15r; high test-retest reliability in a 1-week interval; 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 (Appendix 17 [Section 10.17]). 
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The MG-ADL is being utilized in MG0020 as a parameter of symptom monitoring rather than an 
assessment of treatment benefit. 
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 or 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. 
8.2.5 
Physical examination 
A complete physical examination will include, at a minimum, general appearance; ear, nose, and 
throat; eyes, hair, and skin; and assessments of the cardiovascular, respiratory, gastrointestinal, 
neurological, and musculoskeletal systems. Height and weight will also be measured and 
recorded. Body weight will be measured with the study participant wearing light clothing and 
without wearing shoes. 
A brief physical examination will include, at a minimum, assessments of the skin, respiratory 
system, cardiovascular system, and abdomen (liver and spleen).  
A full neurological examination should be performed for any study participant who experiences 
severe and/or serious headache and for any study participant who experiences features suggestive 
of aseptic meningitis (Appendix 14 [Section 10.14]). A full neurological assessment will include: 
(1) General appearance, including posture, motor activity and meningeal signs and, if indicated, 
the following assessments will be performed; (2) Cranial nerves examination; (3) Motor system 
examination, including muscle tone and power and sensory system examination – light touch; 
(4) Reflexes, including deep tendon reflexes; (5) Coordination, gait (if possible); and (6) 
Fundoscopy. 
Investigators should pay special attention to clinical signs related to previous serious illnesses. 
Clinically relevant findings or worsening of previous findings will be recorded as AEs.  
8.2.6 
Vital signs 
Oral, tympanic, or axillary temperature, pulse rate, respiratory rate, and blood pressure will be 
assessed. 
Blood pressure and pulse 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. 
Blood pressure and pulse measurements should be preceded by at least 5 minutes of rest for the 
participant in a quiet setting without distractions (eg, television, cell phones). 
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Vital signs (to be taken before blood collection for laboratory tests) will consist of 1 pulse and 
1 blood pressure measurement. The blood pressure reading will be recorded on the CRF. 
8.2.7 
Electrocardiograms 
Twelve-lead ECGs will be obtained as outlined in the Schedule of Activities using an ECG 
machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc 
intervals. 
All ECG recordings should be taken with the study participant resting in the supine position for 
at least 5 minutes before the recording. 
The ECGs will be read locally. All ECG readings from an individual study participant should be 
read by the same reader, if possible. Findings will be recorded in the CRF. 
8.2.8 
Clinical safety laboratory assessments 
See Appendix 2 (Section 10.2) for the list of clinical laboratory tests to be performed and the 
Schedule of Activities (Section 1.3) for the timing and frequency.  
The investigator must review the laboratory report, document this review, and record any 
clinically relevant changes occurring during the study in the AE section of the CRF. The 
laboratory reports must be filed with the source documents. Clinically significant abnormal 
laboratory findings are those which are not associated with the underlying disease, unless judged 
by the investigator to be more severe than expected for the participant's condition. 
All laboratory tests with values considered clinically significantly abnormal during participation 
in the study or within 8 weeks after the last dose of study medication should be repeated until the 
values return to normal or baseline or are no longer considered clinically significant by the 
investigator or medical monitor. 
If such values do not return to normal or baseline within a period of time judged reasonable by 
the investigator, 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. 
If laboratory values from nonprotocol-specified laboratory assessments performed at the 
institution’s local laboratory require a change in participant management or are considered 
clinically significant by the investigator (eg, SAE or AE), then the results must be recorded in the 
CRF. 
8.2.9 
Suicidal risk monitoring 
A full C-SSRS assessment (Columbia University Medical Center, 2008) will be performed by 
trained study personnel for Screening (Visit 1) and Baseline (Visit 2 [
]). For subsequent 
visits, a full C-SSRS assessment will only be performed if a study participant has a positive 
response to the query for suicidality. See Section 7.1.3 for related criteria for study medication 
permanent discontinuation. 
8.2.10 
Assessment and management of TB and TB risk factors 
Precautions are being taken within this protocol to monitor the risk of TB infection in this study 
(see Exclusion Criterion 4). Any presumptive diagnosis or diagnosis of a TB infection is a 
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reportable event. Assessment and management of TB and TB risk factors should follow local or 
national guidelines. 
8.2.10.1 
Tuberculosis assessment 
Monitoring for TB during the study 
Study participants will be monitored for signs and symptoms of TB using routine 
pharmacovigilance measures for AEs. Study participants reporting AEs related to signs and 
symptoms of TB will be evaluated for LTB and active TB according to the local medical practice 
guidelines. 
Confirmed LTBI, active TB, and NTMBI must be reported to UCB immediately regardless of 
seriousness using the SAE Report Form and the study participants must be immediately 
withdrawn from the study treatment. Additional information received by the investigator should 
be provided within 24 hours of awareness. 
Once withdrawn from study treatment, study participants should return for the Early Withdrawal 
Visit. 
Tuberculosis signs and symptoms questionnaire 
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 (Appendix 19 
[Section 10.19]), 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 may trigger further assessment 
to determine if the participant has either LTBI and must receive prophylactic LTBI therapy or 
treatment for active TB infection and screen failed/not randomized in the study. As an example, a 
participant who answers “Yes” at Screening to the question “Has the subject been in close (eg, 
sleeping in the same room) contact with an individual with active TB, or an individual who has 
recently been treated for TB?” should not be allowed into the study pending further assessments 
per local and national guidelines. 
8.2.11 
COVID-19 precautions 
This clinical study is likely to run during the ongoing COVID-19 pandemic and will be done in 
accordance with the clinical study center COVID-19 risk mitigation policy, which documents the 
clinical study center’s COVID-19 virus testing strategy for study participants and staff, social 
distancing measures, and management of COVID-19-like symptoms. The risk to study 
participants will be re-evaluated by the sponsor throughout the COVID-19 pandemic if deemed 
necessary by emerging events. 
Study participants will be closely monitored for any signs and symptoms of COVID-19 (ie, 
fever, dysgeusia [taste loss or change], dysosmia [loss or distortion or change of smell], or 
persistent cough) throughout the study. If symptoms and/or clinical signs of infection are 
identified, the investigator will decide whether these findings will be handled as suspected or 
confirmed COVID-19 (see Section 7.1.2 for COVID-19-related temporary IMP discontinuation 
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criteria). The use of testing for severe acute respiratory syndrome coronavirus 2 will be 
determined by reference to the regulatory and healthcare guidance in place locally at the time. 
8.2.12 
MGFA classification 
The investigator will classify the study participant’s MG using the MGFA Clinical Classification 
(Jaretzki et al, 2000). This is a 5-stage classification (I to V), with a higher class indicating more 
severe disease. 
8.3 
Adverse events and serious adverse events 
The definitions of AE and SAE can be found in Appendix 3 (Section 10.3). Adverse events will 
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 study (see Section 7). 
For results disclosure on public registries (eg, ClinicalTrials.gov), treatment-emergent AEs and 
treatment-emergent SAEs will be published. 
The definitions of device-related safety events, ADEs and SADEs can be found in Appendix 7 
(Section 10.7). Device deficiencies are addressed in Appendix 7. 
8.3.1 
Time period and frequency for collecting AE and SAE information 
All AEs and SAEs will be collected from the signing of the ICF until the End of Study Visit 
(Visit 21) at the time points specified in the Schedule of Activities (Section 1.3). 
All TEAEs occurring from the first administration of rozanolixizumab during the Training 
Period up to the first administration of rozanolixizumab in Self-administration Period 1 will be 
considered treatment-emergent in the Training Period. 
All TEAEs occurring during Self-administration Period 1 up to the first administration of 
rozanolixizumab in Self-administration Period 2 will be considered treatment-emergent in Self-
administration Period 1. 
All TEAEs occurring during Self-administration Period 2 up to 8 weeks after the last 
administration of rozanolixizumab will be considered treatment-emergent in Self-administration 
Period 2. 
Local site reaction AEs will be considered treatment-emergent up to 24 hours after each 
administration during the Training Period and Self-administration Periods. 
In order to ensure complete safety data collection, all AEs occurring during the study (ie, after 
the signing of the ICF), including any pretreatment and posttreatment periods required by the 
protocol, must be reported in the CRF even if no study medication was taken but specific study 
procedures were conducted. This includes all AEs not present before the initial visit and all AEs 
that recurred or worsened after the initial visit. 
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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 
participants of the need to inform the investigator of any SAE within this period. Serious AEs 
that the investigator thinks may be associated with the study medication must be reported to 
UCB regardless of the time between the event and the end of the study. 
The method of recording, evaluating, and assessing causality of AE and SAE and the procedures 
for completing and transmitting SAE reports are provided in Appendix 3 (Section 10.3). 
8.3.2 
Method of detecting AEs and SAEs 
Care will be taken not to introduce bias when detecting AEs and/or SAEs. Open-ended and 
nonleading verbal questioning of the participant is the preferred method to inquire about 
AE occurrences. 
8.3.3 
Follow-up of AEs and SAEs 
After the initial AE/SAE report, the investigator is required to proactively follow each participant 
at subsequent visits or contacts. All SAEs, nonserious AEs of special interest (as defined in 
Section 8.3.6), and AESMs (as defined in Section 8.3.8) will be followed until resolution, 
stabilization, the investigator determines that it is no longer clinically significant, the event is 
otherwise explained, or the participant is lost to follow-up (as defined in Section 7.3). Further 
information on follow-up procedures is given in Appendix 3 (Section 10.3). 
8.3.4 
Regulatory reporting requirements for SAEs 
Prompt notification of an SAE by the investigator to the sponsor 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 (IRBs)/Independent Ethics Committees 
(IECs), and investigators. 
Investigator safety reports must be prepared for suspected unexpected serious adverse reactions 
(SUSARs) according to local regulatory requirements and sponsor policy and forwarded to 
investigators as necessary. SUSAR reporting will be in adherence to requirements of EU 
pharmacovigilance legislation, clinical trial legislation and guidance, Clinical Trial Regulation 
EU 536/2014; CT-3, and all other applicable local regulations. 
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. 
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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 90 days after the last 
dose. 
If a pregnancy is reported, the investigator must immediately inform the sponsor within 24 hours 
of learning of the pregnancy and should follow the procedures outlined in Appendix 4 
(Section 10.4). 
The participant should be withdrawn from the study as soon as pregnancy is known (by positive 
pregnancy test), and the following should be completed: 
• The participant should immediately stop the intake of the study medication. 
• The participant should return for an Early Withdrawal Visit. 
• Safety follow-up should be scheduled after the participant has discontinued her study 
medication. 
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 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 or compound.For rozanolixizumab, the following events 
require immediate reporting (within 24 hours regardless of seriousness) to UCB: 
• Hy’s Law 
−  Potential Hy’s Law, defined as ≥3xULN ALT or AST 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 be reported if an alternative 
etiology is identified during investigation and monitoring of the participant. 
8.3.7 
Medication errors associated with self-administration 
The definitions of medication errors can be found in Appendix 12 (Section 10.12). The 
medication errors reported by the study participant (or, when appropriate, by a caregiver, or 
observed by the investigator) shall be recorded in the paper Medication Error form by the 
investigator and emailed to the Global mailbox: DS_ICT@ucb.com. The fax and mobile number 
are also provided in case of urgent necessity. All medication errors except those considered as 
AESM (see Section 8.3.8) will be recorded and reported to the sponsor or designee within 7 days 
of awareness. Medication errors that are considered AESM (see Section 8.3.8) will be recorded 
and reported within 24 hours following the SAE recording and reporting procedures as indicated 
in Appendix 3 (Section 10.3) and using the Medication Error form. If the medication error is 
observed from other periods of the study (apart from Self-administration Periods), the medical 
monitor should be informed for further advice.  
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The investigator and any designees are responsible for detecting, documenting, and recording 
events that meet the definition of a medication error and remain responsible for following up 
medication errors that are considered AESM (see Section 8.3.8). 
8.3.8 
Adverse events of special monitoring 
An AESM is a product-specific AE, adverse reaction, or safety topic requiring special 
monitoring by UCB. 
For rozanolixizumab, the following events require immediate reporting (within 24 hours 
regardless of seriousness) to UCB: 
• Severe and/or serious headache 
• Suspected aseptic meningitis 
• Medication error associated with adverse reaction(s) 
Procedures for the management of AESM are provided in Appendix 14 (Section 10.14).  
Although infections and hypersensitivity reactions including infusion-related reactions and 
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 to the guidance provided in Appendix 13 
(Section 10.13). Suspected anaphylaxis reactions should be diagnosed using Sampson’s Criteria 
(Sampson et al, 2006).  
All AESM will follow the SAE recording and reporting procedures as indicated in Appendix 3 
(Section 10.3). Medication error associated with adverse reaction(s) should also be assessed, 
recorded, and reported using the Medication Error form as indicated in Section 8.3.7. 
8.3.9 
Medical device – adverse events (ADEs, unanticipated ADEs, SAEs, 
SADEs, and unanticipated SADEs) and device deficiencies 
Medical devices are being provided for administration of IMP in this study. In order to fulfill 
regulatory reporting obligations worldwide, the investigator is responsible for the detection and 
documentation of events meeting the definitions of ADEs, SADEs, and device deficiencies that 
occur during the study with such devices. 
Adverse events will be reported according to the ISO 14155:2020, while recognizing and 
following requirements including reporting timelines specified in other specific laws, 
regulations, directives, standards and/or guidelines as appropriate and as required by the 
countries in which the clinical investigation is conducted.  
The definition of an ADE, SADE, and device deficiency can be found in Appendix 7 
(Section 10.7).  
NOTE: Events fulfilling the definition of an AE or SAE will also follow the processes outlined 
in Section 8.3.3 and Appendix 3 (Section 10.3) of the protocol. 
8.3.9.1 
Time period for detecting ADEs and device deficiencies 
Any ADEs or device deficiencies of the device will be detected, documented, and reported 
during all periods of the study in which the medical device is used.  
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If the investigator learns of any deficiency at any time after a participant has been discharged 
from the study, and such event(s) is considered reasonably related to a medical device provided 
for the study, the investigator will promptly notify the sponsor. 
The method of documenting medical device deficiency is provided in Appendix 7 (Section 10.7). 
8.3.9.2 
Follow-up of ADEs and device deficiencies 
Follow-up applies to all study participants, including those who discontinue study medication 
and/or the study. 
The investigator is responsible for ensuring that follow-up includes any supplemental 
investigations as indicated to elucidate the nature and/or causality of the deficiency. 
New or updated information will be recorded on the originally completed form with all changes 
signed and dated by the investigator. 
8.3.9.3 
Prompt reporting of ADEs and device deficiencies to sponsor 
Any ADEs or device deficiencies will be reported to the sponsor within 24 hours after the 
investigator determines that the event meets the protocol definition of an ADE or device 
deficiency. 
The Adverse Event and Device Deficiency Report Form will be sent to the sponsor using the 
paper form. 
The sponsor will then notify the ADE and device deficiency reports to the corresponding device 
manufacturer. The device manufacturer is responsible for the subsequent vigilance evaluation 
and reporting, if applicable. 
8.3.9.4 
Regulatory reporting requirements for ADEs and device deficiencies 
The investigator will promptly report all ADEs and device deficiencies occurring with any 
medical device provided for use to the sponsor. The sponsor will then notify the device 
manufacturer in order for the device manufacturer to fulfill vigilance responsibilities. 
The investigator, or responsible person according to local requirements (eg, the head of the 
medical institution), will comply with the applicable local regulatory requirements relating to the 
reporting of device deficiencies to the IRB or IEC. 
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. 
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. 
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8.5 
Treatment of overdose 
Unintentional overdose events are considered Medication Errors and should be reported as 
described in Section 8.3.7 and Section 8.3.8. 
For the purpose of this study an overdose will be defined as a dose that is 10% over the 
acceptable variance of 20% of the intended dose. For study participants assigned a 
 dose, 
a rozanolixizumab dose of 
 at any time will be considered an overdose. For study 
participants assigned a 
 dose, a rozanolixizumab dose of 
 at any time will be 
considered an overdose. For study participants assigned a dose of 
, a rozanolixizumab 
dose of 
 at any time will be considered an overdose. For study participants assigned a 
 dose, a rozanolixizumab dose of 
 at any time will be considered an overdose.  
Intentional overdose events (eg, suicide attempt) are not considered Medication Errors and must 
be reported as an SAE as described in Section 8.3.1.  
UCB does not recommend specific treatment for an overdose. Single subcutaneous dose of up to 
 and 
 subcutaneous doses of around 
 for up to 
 weeks have been administered per protocol in clinical studies without dose limiting toxicity. 
However, in the event of an overdose, the investigator or treating physician should: 
1. Contact the medical monitor immediately. 
2. Closely monitor the participant for any AE/SAE and laboratory abnormalities for at least 
5 days. 
3. Obtain a plasma sample for pharmacokinetic analysis within 72 hours from the date of the 
last dose of study medication 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 CRF. 
Decisions regarding dose interruptions will be made by the investigator in consultation with the 
medical monitor based on the clinical evaluation of the participant. 
8.6 
Pharmacokinetics 
Pharmacokinetic parameters are not evaluated in this study. Pharmacokinetic blood sample in the 
event of PDILI could be collected as described in Appendix 6 (Section 10.6). 
8.7 
Pharmacodynamics 
Venous blood samples will be collected at time points specified in the Schedule of Activities 
(Section 1.3) for measurement of serum total IgG concentrations. 
Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
8.8 
Biomarkers 
Blood samples for exploratory safety biomarker analysis will be collected at Baseline for all 
study participants. In study participants who experience severe and/or serious headaches or 
features suggestive of aseptic meningitis, samples should also be collected 4 hours after the onset 
of the event, or otherwise as soon as possible within 72 hours after the onset of the event.  
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These samples will only ever be used to further explore if biomarkers can advance the 
understanding of the pathological drivers of the AESM of severe and/or serious headache or 
suspected aseptic meningitis.  
If not used immediately, these samples will be stored at -80°C for up to 20 years for later 
exploratory analyses.  
Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study.  
8.9 
Immunogenicity assessments 
Antibodies to rozanolixizumab will be evaluated in serum samples collected from all participants 
according to the Schedule of Activities (Section 1.3). Additionally, serum samples should also be 
collected at the final visit from participants who discontinued study medication or were 
withdrawn from the study. These samples will be tested by the sponsor or sponsor’s designee. 
Serum samples will be screened for antibodies binding to rozanolixizumab and the titer of 
confirmed positive samples will be reported. Other analyses may be performed to verify the 
stability of antibodies to rozanolixizumab and/or further characterize the immunogenicity of 
rozanolixizumab. 
The detection and characterization of antibodies to rozanolixizumab will be performed using a 
validated assay method by or under the supervision of the sponsor. Antibodies may be further 
characterized and/or evaluated for their ability to neutralize the activity of the study 
medication(s). Samples may be stored for a maximum of 20 years (or according to local 
regulations) following the last participant’s last visit for the study at a facility selected by the 
sponsor to enable further analysis of immune responses to rozanolixizumab. In addition, surplus 
immunogenicity assessment samples may be stored and used for potential future biomarker 
research (including assay development and optimization), but not for future genetic biomarker 
research. 
8.10 
Medical resource utilization and health economics 
Medical resource utilization and health economics parameters are not evaluated in this study. 
9 
STATISTICAL CONSIDERATIONS 
A description of statistical methods follows and will be described in more detail in the Statistical 
Analysis Plan. 
9.1 
Definition of analysis sets 
• Enrolled Set: All study participants who have signed the informed consent form. 
• Safety Set: All study participants who received at least 1 dose of IMP (partial or full). 
Analysis of this set will be according to the administration method the participants actually 
used and will be used for analyses of safety and demography outcomes as required. 
• Randomized Safety Set: All randomized study participants who received at least 1 dose of 
IMP (partial or full). Analysis of this set will be according to the administration method the 
participants actually used and will be used for analyses of safety, demography, and 
efficacy/outcomes. 
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• Full Analysis Set: All randomized study participants who received at least 1 dose of IMP 
(partial or full) and completed both Self-administration Periods.  
9.2 
General statistical considerations 
All analyses will be performed using Statistical Analysis System (SAS®) version 9.3 or later 
(SAS Institute, Cary, NC, USA). 
This is an estimation study design with no formal statistical hypothesis testing. The study will 
estimate the true population proportion and/or mean of self‑administration–related endpoints for 
each self-administration method separately. Summary statistics for continuous variables will 
include number of available observations, mean, standard deviation, minimum, median, and 
maximum. For categorical variables, the number and proportion of participants will be presented. 
If not otherwise specified, summary statistics will be displayed by sequence and overall for 
demographics and by period and by self-administration method for period-specific assessments. 
The Baseline value is defined as the last nonmissing measurement before the first administration 
at Visit 2 (Baseline) if not otherwise specified. All data recorded in the CRF and questionnaires 
will be listed. 
9.3 
Planned safety analyses 
9.3.1 
Analysis of the primary safety endpoint 
The primary outcome variable is the proportion of study participants able to successfully self-
administer rozanolixizumab using both syringe driver and the manual push method, respectively, 
during the Self-administration Periods. Successful self-administration includes a correct SC 
infusion with the intended dose at the correct infusion site. Treatment success will be defined as 
a successful self-administration at the last dose of self-administration using the syringe driver 
(Visit 13 [
 for Sequence 1 and Visit 19 [
 for Sequence 2) or via manual push 
(Visit 19 [(
 for Sequence 1 and Visit 13 [
] for Sequence 2). 
The number and proportion of participants deemed a treatment success will be tabulated 
separately for each self-administration method considering the Full Analysis Set. The proportion 
of success together with its 90% CI will be estimated for each self-administration with syringe 
driver and manual push. 
9.3.2 
Analysis of the secondary safety endpoint 
The secondary endpoints of the occurrence of TEAEs after syringe driver or manual push self-
administration, the occurrence of local site reactions up to 24 hours after each administration, 
and the occurrence of medication errors associated with adverse reactions will be summarized 
during the Self-administration Periods by the number and proportion of participants separately 
by self-administration method. This analysis will be based on the Randomized Safety Set. 
Occurrence of TEAEs and occurrence of local site reactions during the Training Period will also 
be summarized based on the Safety Set. 
9.3.3 
Other safety analyses 
All other safety analyses will be based on the Randomized Safety Set or Safety Set as 
appropriate. 
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To assess the participant’s experience with self-administration of SC infusions, a within-
participant assessment of treatment success or nonsuccess with self-administration with the 
syringe drive or manual push will be tabulated through the use of a 2x2 table presenting the 
number and proportion of participants in each category. The last dose in each of the Self-
administration Periods will be considered. The tabulation will be presented by sequence and 
overall. 
The number and proportion of participants who achieved a successful self-administration at each 
of the 3 home self-administration visits will be tabulated separately for each self-administration 
method.  
The participant’s relative preference for SC infusions performed by an HCP versus 
self-administration as well as the preference for self-administration using the manual push 
method versus the use of a syringe driver will be summarized by sequence and overall. 
The PRE-SIAQ (Infusion version) domain scores will be summarized at Baseline by sequence 
and overall. The POST-SIAQ (Infusion version) will be summarized for each self-administration 
method. 
The change from Baseline MG-ADL score will be summarized by visit and period. Worsening in 
MG-ADL score will be defined by a >2 points increase from Baseline. Baseline value will be 
defined for each period separately. The number and proportion of study participants experiencing 
a worsening of their MG-ADL symptoms during the Self-administration Period will be 
summarized for each self-administration method. The proportion of worsening together with its 
90% CI for each self-administration with syringe driver and manual push will be estimated 
considering the period and sequence as fixed effects and participant as a random effect to 
encompass the within-participant correlation.  
All AE data will be listed, and no statistical testing will be performed. The frequency and 
severity of all TEAEs during the Self-administration Period will be presented separately for self-
administration by syringe driver and via manual push separately by system organ class, high 
level term, and preferred term (Medical Dictionary for Regulatory Activities). The data will be 
displayed as number of participants experiencing the TEAE, percentage of participants, and 
number of TEAEs. The frequency and severity of all TEAEs during the Training Period will also 
be summarized. 
The occurrence of TEAEs leading to permanent withdrawal of rozanolixizumab will be 
summarized during the Self-administration Periods by the number and proportion of participants 
separately by self-administration method. The occurrence of these TEAEs together with its 90% 
CI for each self-administration with syringe driver and manual push will be estimated 
considering the period and sequence as fixed effects and participant as a random effect to 
encompass the within-participant correlation. Occurrence of TEAEs leading to permanent 
withdrawal of rozanolixizumab during the Training Period will also be summarized. 
Laboratory evaluations and vital signs as well as ECG data will be analyzed over time. All safety 
analyses will be based on the Safety Set and Randomized Safety Set as required. 
Further analyses will be described in the Statistical Analysis Plan. 
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9.4 
Planned efficacy/outcome analyses 
Not applicable. 
9.5 
Other planned analyses 
The IgG levels at each visit will be summarized. Spaghetti plots of the IgG levels over time will 
be presented. 
Anti-rozanolixizumab antibodies (status and titer at trough) will be summarized. 
9.6 
Handling of protocol deviations 
Important protocol deviations are identified as part of the data cleaning process in the Data 
Cleaning Plan. Ongoing data cleaning meetings will be held throughout the duration of the study. 
Objectives of these meetings include the review of important protocol deviations and the update 
(if necessary) of the important protocol deviation specification. Furthermore, overall trends in 
protocol deviations will be discussed at the Data Evaluation Meetings. 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 will be detailed in the Statistical 
Analysis Plan. 
A sensitivity analysis will be performed to assess the primary endpoint by imputing a self-
administration failure for any Visit 13 (
) or Visit 19 (
) missed by a randomized 
participant considering the Randomized Safety Set. 
9.8 
Planned interim analysis and data monitoring 
No interim analysis is planned. 
A program Independent Data Monitoring Committee (pIDMC) has been established to provide a 
periodic review of safety data from studies across the rozanolixizumab program. Data emerging 
from this study will be included in the pIDMC periodic data reviews to assess the benefit-risk of 
rozanolixizumab. 
9.9 
Determination of sample size 
This study will not be powered with respect to any endpoint and sample size is based on practical 
considerations. Confidence intervals expected to be observed under assumptions of the true 
success rate are provided in Table 9-1. It is planned to have a minimum of 30 participants in total 
(minimum 15 per sequence) who will perform self-administration using both the syringe driver 
and the manual push administration methods. 
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Table 9-1: 
Expected 90% confidence intervals considering different success 
rates and 30 study participants completing both Treatment Periods 
Success rate 
Expected 90% confidence interval a 
80% 
64.3 to 90.9 
90% 
76.1 to 97.2 
97% 
85.1 to 99.8 
a Exact Clopper Pearson confidence intervals 
 
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 for Harmonisation Good Clinical Practice (ICH-GCP), and in accordance 
with the ethical principles that have their origin in the Declaration of Helsinki. 
The investigator/UCB will ensure that an appropriately constituted IRB/IEC that complies with 
the requirements of the current ICH-GCP version or applicable country-specific regulations will 
be responsible for the initial and continuing review and approval of the clinical study. Prior to 
initiation of the study, the investigator/UCB will forward copies of the protocol, ICF, 
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. 
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UCB (or its representative) will communicate safety information to the appropriate regulatory 
authorities and all active investigators in accordance with applicable regulatory requirements. 
The appropriate IRB/IEC will also be informed by the investigator or the sponsor, as specified by 
the applicable regulatory requirements in each concerned country. Where applicable, 
investigators are to provide the sponsor (or its representative) with evidence of such IRB/IEC 
notification. 
10.1.2 
Financial disclosure 
Insurance coverage will be handled according to local requirements. 
Finance and insurance are addressed in the investigator and/or contract research organization 
agreements, as applicable. 
10.1.3 
Informed consent process 
Participant’s informed consent and caregiver’s informed consent (if applicable) must be obtained 
and documented in accordance with local regulations, ICH-GCP requirements, and the ethical 
principles that have their origin in the principles of the Declaration of Helsinki. 
Prior to obtaining informed consent, information should be given in a language and at a level of 
complexity understandable to the participant in both oral and written form by the investigator (or 
designee). Each participant will have the opportunity to discuss the study and its alternatives 
with the investigator. 
Prior to participation in the study, the ICF should be signed and personally dated by the 
participant, or his/her legal representative, the caregiver (if applicable [latest at the Baseline 
visit]), and by the person who conducted the informed consent discussion (investigator or 
designee). The participant, or his/her legal representative, and the caregiver (if applicable) must 
receive a copy of the signed and dated ICF. As part of the consent process, each participant must 
consent to direct access to his/her medical records for study-related monitoring, auditing, 
IRB/IEC review, and regulatory inspection. 
If the ICF is amended during the study, the investigator (or the sponsor, if applicable) must 
follow all applicable regulatory requirements pertaining to the approval of the amended ICF by 
the IRB/IEC and use of the amended form. 
All studies conducted at centers in the US must include the use of a Health Insurance Portability 
and Accountability Act authorization form. 
The participant or the caregiver (if applicable) may withdraw his/her consent to participate in the 
study at any time. A participant is considered to be enrolled in the study when he/she has signed 
the ICF. 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. 
If the caregiver withdraws consent, the study participant can be treated on-site per protocol based 
on investigator decision. 
10.1.4 
Data protection 
UCB staff (or designee) will affirm and uphold the participant’s confidentiality. Throughout this 
study, all data forwarded to UCB (or designee) will be identified only by the participant number 
assigned at Screening. 
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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 and 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. 
The contract between UCB and study sites specifies responsibilities of the parties related data 
protection, including handling of data security breaches and respective communication and 
cooperation of the parties. 
Information technology systems used to collect, process, and store study-related data are secured 
by technical and organizational security measures designed to protect such data against 
accidental or unlawful loss, alteration, or unauthorized disclosure or access. 
10.1.5 
Committees structure 
Given this is an open-label study, oversight of individual study data will be maintained on an 
ongoing basis by regular reviews of the safety data performed by the medical monitors in 
collaboration with the UCB study physician, patient safety representative, and program safety 
review team. In addition, selected safety data across all studies, including MG0020, will be 
reviewed at a program level by a pIDMC. Details of the pIDMC composition, processes, and 
responsibilities will be documented in the pIDMC Charter. 
10.1.6 
Dissemination of clinical study data 
All Phase 1 to 4 clinical studies in patients will be registered on ClinicalTrials.gov with results 
posted after completion of the study. 
A plain language summary of the results of all Phase 1 to 4 clinical studies will be developed and 
shared on UCB’s website. 
UCB is committed to submitting all Phase 2 to 4 clinical study results, irrespective of outcome, 
for publication in a credible, peer-reviewed journal. While there are some exceptions owing to 
intellectual property considerations in early clinical development, our policy is also to submit 
Phase 1 study results for publication in a peer-reviewed journal wherever possible. 
Data from this study may be requested by qualified researchers 6 months after product approval 
in the US and/or Europe, or global development is discontinued, and 18 months after study 
completion. Investigators may request access to anonymized individual patient-level data and 
redacted study documents which may include analysis-ready datasets, study protocol, annotated 
CRFs, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the 
data, proposals need to be approved by an independent review panel at www.Vivli.org and a 
signed data sharing agreement will need to be executed. All documents are available in English 
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only, for a prespecified time, typically 12 months, on a password protected portal. This plan may 
change if the risk of re-identifying study participants is determined to be too high after the study 
is completed; in this case and to protect participants, individual patient-level data would not be 
made available. 
10.1.7 
Data quality assurance 
All participant data relating to the study will be recorded on printed or electronic CRF 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 CRF.  
The investigator must maintain accurate documentation (source data) that supports the 
information entered in the CRF.  
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 CRF by authorized site personnel are accurate, legible, contemporaneous, original, and 
attributable 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 must be retained by the investigator for the minimum retention period 
mandatory under the applicable local laws and regulations. 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. 
Quality tolerance limits will be established for the study using parameters related to patient 
safety reporting and reliability of study results. The parameters will be monitored throughout the 
study to identify systematic issues. Parameters used, parameter values, important deviations from 
the quality tolerance limits, and actions taken will be summarized in the clinical study report. 
10.1.7.1 
Case report form completion 
The investigator is responsible for prompt reporting of accurate, complete, and legible data in the 
electronic CRFs and in all required reports. 
Any change or correction to the CRF 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 electronic 
CRF. 
Detailed instructions will be provided in the CRF Completion Guidelines. 
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10.1.7.2 
Apps 
Study participants will be provided with a mobile phone with a preinstalled application (app) that 
will collect data from each participant throughout the study. The app will support the participant 
to complete study-specific activities, as follows: 
• Receiving reminders and notifications such as for the Visits, Patient Preference, MG-ADL, 
and the SIAQ questionnaires 
• Scheduling visits with the investigator and/or authorized site representative(s) 
The investigator and authorized site representative(s) will have access to a secure website portal 
via unique access credentials to access the study data from the completed activities noted above. 
Data will be reviewed by the investigator via the website portal view of participant-entered data 
results. 
Furthermore, this app is designed to only record data to support study participation, and therefore 
it is neither designed nor intended to be used to collect or report safety-related information about 
the participant. The primary functionality of the app consists of data storage and communication 
of unmodified data. This stored data will not be used to make clinical patient management 
decisions. Therefore, the aggregated app data will not provide benefit to any single study 
participant. 
Based on the current functionality, the app falls outside medical device regulations in the EU, 
US, and Japan. The app’s medical device status must be re-assessed upon future modification of 
the patient facing app functionality. Furthermore, risks to the patients related to cybersecurity 
and patients’ privacy must be addressed prior to app utilization in the clinical trial.  
10.1.8 
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 CRFs 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). 
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10.1.9 
Study and site start and closure 
The start of recruitment 
The start of recruitment is the first participant’s first visit and is also the start date of the clinical 
study. 
Study/site termination 
The sponsor designee reserves the right to close the study site or terminate the study at any time 
for any reason at the sole discretion of the sponsor. Study sites will be closed upon study 
completion. A study site is considered closed when all required documents and study supplies 
have been collected and a study site closure visit has been performed. 
The investigator may initiate study site closure at any time, provided there is reasonable cause 
and sufficient notice is given in advance of the intended termination. 
Reasons for the early closure of a study site by the sponsor or investigator may include but are 
not limited to: 
• Failure of the investigator to comply with the protocol, the requirements of the IRB/IEC or 
local health authorities, the sponsor's procedures, or GCP guidelines 
• Inadequate recruitment of participants by the investigator 
• Discontinuation of further study medication development 
10.1.10 
Publication policy 
The results of this study may be published or presented at scientific meetings. If this is foreseen, 
the investigator agrees to submit all manuscripts or abstracts to the sponsor before submission. 
This allows the sponsor to protect proprietary information and to provide comments.  
The sponsor will comply with the requirements for publication of study results. In accordance 
with standard editorial and ethical practice, the sponsor will generally support publication of 
multicenter studies only in their entirety and not as individual site data. In this case, a 
coordinating investigator will be designated by mutual agreement. 
Authorship will be determined by mutual agreement and in line with International Committee of 
Medical Journal Editors authorship requirements. 
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10.2 
Appendix 2: Clinical laboratory tests 
• The tests detailed in the table below will be performed by the central laboratory.  
• Local laboratory results are only required in the event that the central laboratory results are 
not available in time for either study medication 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 CRF. 
• 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. 
Table 10-1: Protocol-required safety laboratory assessments 
Laboratory 
Assessments 
Parameters 
Hematology 
Platelet count  
White blood cell (WBC) 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) c 
Calcium 
Alkaline phosphatase 
C-reactive 
protein (CRP) 
Lactate 
dehydrogenase 
(LDH) 
Triglycerides  Low-density lipoprotein (LDL) 
High-density lipoprotein 
(HDL) 
Total 
Cholesterol 
Routine 
Urinalysis 
• 
Specific gravity 
• 
pH, glucose, protein, blood, ketones, albumin, bilirubin, urobilinogen, 
nitrite, leukocyte esterase by dipstick 
• 
Microscopic examination (if blood or protein is abnormal) 
Other Screening 
Tests 
• 
Follicle-stimulating hormone and estradiol (as needed in women of 
non-childbearing potential only) 
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Table 10-1: Protocol-required safety laboratory assessments 
Laboratory 
Assessments 
Parameters 
• 
Serum or urine human chorionic gonadotropin (hCG) pregnancy test (as 
needed for women of childbearing potential)b 
• 
Serology testing (for human immunodeficiency virus, Hepatitis B, and 
Hepatitis C) 
All study-required laboratory assessments will be performed by a central laboratory. 
The results of each test must be entered into the CRF. 
NOTES : 
For additional assessments that may be required in case of AESM of severe and/or serious headache or 
suspected aseptic meningitis, see Table 1-4. 
a Details of liver chemistry stopping criteria and required actions and follow-up assessments after liver 
stopping or monitoring event are given in Section 7.1.1 and Appendix 6 (Section 10.6). All events of 
ALT 3 × upper limit of normal (ULN) and bilirubin 2 × ULN (>35% direct bilirubin) or ALT 3 × 
ULN 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. 
c To be done at Screening only 
Investigators must document their review of each laboratory safety report. 
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10.3 
Appendix 3: Adverse events – Definitions and procedures for 
recording, evaluating, follow-up, and reporting 
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.  
• 
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. Also, "lack of efficacy" or "failure 
of expected pharmacological action" also constitutes 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. 
• 
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. 
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• 
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. 
Definition of SAE 
If an event is not an AE per definition above, then it cannot be a SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease). 
A SAE is defined as any untoward medical occurrence that, at any dose: 
a. Results in death 
b. Is life-threatening 
The term ‘life-threatening’ in the definition of ‘serious’' refers to an event in which the participant was 
at risk of death at the time of the event. It does not refer to an event, which hypothetically might have 
caused death, if it were more severe. 
c. Requires inpatient hospitalization or prolongation of existing hospitalization 
In general, hospitalization signifies that the participant has been detained (usually involving at least an 
overnight stay) at the hospital or emergency ward for observation and/or treatment that would not have 
been appropriate in the physician’s office or outpatient setting. Complications that occur during 
hospitalization are AEs. If a complication prolongs hospitalization or fulfills any other serious criteria, 
the event is serious. When in doubt as to whether “hospitalization” occurred or was necessary, the AE 
should be considered serious. 
Hospitalization for elective treatment of a pre-existing condition that did not worsen from Baseline is 
not considered an AE. 
d. Results in persistent disability/incapacity 
• 
The term disability means a substantial disruption of a person’s ability to conduct normal life 
functions. 
• 
This definition is not intended to include experiences of relatively minor medical significance, 
such as uncomplicated headache, nausea, vomiting, diarrhea, influenza, and accidental trauma 
(eg, sprained ankle) which may interfere with or prevent everyday life functions but do not 
constitute a substantial disruption. 
e. Is a congenital anomaly/birth defect 
f. Important medical events: 
• 
Medical or scientific judgment should be exercised in deciding whether SAE reporting is 
appropriate in other situations, such as important medical events that may not be immediately 
life-threatening or result in death or hospitalization but may jeopardize the participant or may 
require medical or surgical intervention to prevent one of the other outcomes listed in the 
above definition. These events should usually be considered serious. 
• 
Examples of such events include, but are not limited to, potential Hy’s law, invasive or 
malignant cancers, intensive treatment in an emergency room or at home for allergic 
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bronchospasm, blood dyscrasias or convulsions that do not result in hospitalization, or 
development of drug dependency or drug abuse. 
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 CRF. 
• 
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 CRF page. 
• 
There may be instances when copies of medical records for certain cases are requested by 
UCB. In this case, all participant identifiers, with the exception of the participant number, will 
be redacted on the copies of the medical records before submission to UCB. 
• 
The investigator will attempt to establish a diagnosis of the event based on signs, symptoms, 
and/or other clinical information. Whenever possible, the diagnosis (not the individual 
signs/symptoms) will be documented as the AE/SAE. 
Assessment of intensity 
The investigator will make an assessment of intensity for each AE and SAE reported during the study 
and assign it to 1 of the following categories:  
• 
Mild: An event that is easily tolerated by the participant, causing minimal discomfort and not 
interfering with everyday activities. 
• 
Moderate: An event that causes sufficient discomfort and interferes with normal everyday 
activities. 
• 
Severe: An event that prevents normal everyday activities. An AE that is assessed as severe 
should not be confused with a 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 a 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. 
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• 
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) and/or Product Information, 
for marketed products, 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 a SAE has occurred and the investigator has minimal 
information to include in the initial report to UCB. However, it is very important that the 
investigator always make an assessment of causality for every event before the initial 
transmission of the SAE data to UCB. 
• 
The investigator may change his/her opinion of causality in light of follow-up information and 
send a SAE follow-up report with the updated causality assessment. 
• 
The causality assessment is one of the criteria used when determining regulatory reporting 
requirements. 
 
Follow-up of AEs and SAEs 
• 
The investigator is obligated to perform or arrange for the conduct of supplemental 
measurements and/or evaluations as medically indicated or as requested by UCB to elucidate 
the nature and/or causality of the AE or SAE as fully as possible. This may include additional 
laboratory tests or investigations, histopathological examinations, or consultation with other 
health care professionals. 
• 
An AE should be followed until it has resolved, has a stable sequelae, the investigator 
determines that it is no longer clinically significant, or the participant is lost to follow-up. This 
follow-up requirement applies to AEs, SAEs, and 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 the sponsor within 24 hours of receipt of 
the information. 
Reporting of SAEs 
SAE reporting to UCB via an electronic data collection tool 
• 
The primary mechanism for reporting a 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). 
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• 
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. 
• 
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 postmenopausal unless 
permanently sterile (see below). 
Women in the following categories are not considered WOCBP: 
1. Premenarchal 
2. Premenopausal female with 1 of the following: 
− Documented hysterectomy 
− Documented bilateral salpingectomy 
− Documented bilateral oophorectomy 
Note: Documentation can come from the site personnel’s review of the participant’s medical 
records, medical examination, or medical history interview. 
3. Postmenopausal female 
− A postmenopausal state is defined as no menses for 12 months without an alternative 
medical cause. A high follicle-stimulating hormone (FSH) level in the postmenopausal 
range may be used to confirm a postmenopausal state in women not using hormonal 
contraception or hormonal replacement therapy (HRT). However, in the absence of 
12 months of amenorrhea, a single FSH measurement is insufficient. 
− Females on HRT and whose menopausal status is in doubt will be required to use one of 
the nonestrogen hormonal highly effective contraception methods if they wish to 
continue their HRT during the study. Otherwise, they must discontinue HRT to allow 
confirmation of postmenopausal status before study enrollment. 
Contraception guidance 
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 the table 
below. 
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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 
ovulation 
• 
Oral  
• 
Intravaginal  
• 
Transdermal  
Progestogen only hormonal contraception associated with inhibition of ovulation 
• 
Oral  
• 
Injectable  
Highly effective methods that are user independent 
Implantable progestogen only hormonal contraception associated with inhibition of ovulation 
• 
Intrauterine device (IUD) 
• 
Intrauterine hormone-releasing system (IUS) 
• 
Bilateral tubal occlusion 
Vasectomized partner  
Vasectomy is a highly effective contraception method provided that the 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.  
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 instruction as to 
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.  
Pregnancy testing 
• WOCBP should only be included after a confirmed menstrual period and a negative highly 
sensitive serum pregnancy test. 
• Additional pregnancy testing should be performed as specified in the Schedule of Activities 
(Section 1.3) corresponding to protocol-defined time frame in Appendix 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. 
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• Pregnancy testing, with a sensitivity of ≥25mIU/mL will be performed. A serum pregnancy 
test will be completed at Screening. Urine pregnancy tests will be performed at all other 
timepoints. 
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 one 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 
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 a SAE and will be reported as such. Any post-study 
pregnancy-related SAE considered reasonably related to the study medication by the 
investigator will be reported to the sponsor as described in Section 8.3.5. While the 
investigator is not obligated to actively seek this information in former study participants, he 
or she may learn of a SAE through spontaneous reporting.  
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10.5 
Appendix 5: Genetics 
Not applicable. 
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10.6 
Appendix 6: Liver safety – suggested actions and follow-up 
assessments 
Participants with PDILI must be assessed to determine if study medication must be discontinued. 
In addition, all concomitant medications and herbal supplements that are not medically necessary 
should also be discontinued. 
Investigators should attempt to obtain information on study participants in the case of study 
medication discontinuation to complete the final evaluation.  
Study participants with PDILI should not be withdrawn from the study until investigation and 
monitoring are complete. All results of these evaluations and observations, as well as the 
reason(s) for study medication discontinuation and/or participant withdrawal (if applicable), 
must be recorded in the source documents. The 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 laboratory values). 
Phase 3-4 liver chemistry stopping criteria are designed to assure participant safety and to 
evaluate liver event etiology. 
Table 10-2: Phase 3-4 liver chemistry stopping criteria and follow-up 
assessments 
Liver chemistry stopping criteria 
ALT-absolute 
ALT 8x ULN 
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)  
INRb 
ALT 3xULN and 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 
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Table 10-2: Phase 3-4 liver chemistry stopping criteria and follow-up 
assessments 
Liver chemistry stopping criteria 
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 CRF, and complete a 
SAE data collection tool if the event also met the 
criteria for a SAE.b  
• 
Perform liver chemistry follow-up assessments.  
• 
Monitor the participant until liver chemistry test 
abnormalities resolve, stabilize, or return to 
Baseline (see MONITORING). 
• 
Do not restart/rechallenge participant with study 
medication unless allowed per protocol and UCB 
approval is granted. 
• 
If restart/rechallenge is not allowed per protocol 
or not granted, permanently discontinue study 
medication, and continue participant in the study 
for any protocol-specified follow-up 
assessments.Consider the need for a toxicology 
screening. 
MONITORING: 
For bilirubin or INR criteria  
• 
Repeat liver chemistry tests (include ALT, AST, 
ALP, 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, 
ALP, bilirubin) and perform liver chemistry 
follow-up assessments within 24 to 72 hours. 
• 
Monitor participants weekly until liver chemistry 
abnormalities resolve, stabilize, or return to 
Baseline. 
• 
Viral hepatitis 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 HBsAg): 
quantitative hepatitis B DNA and hepatitis delta 
antibodye 
• 
Obtain blood sample for PK analysis after the most 
recent dosef 
• 
Serum CPK and LDH 
• 
Fractionate bilirubin, if total bilirubin 2xULN 
• 
Obtain complete blood count with differential to 
assess eosinophilia 
• 
Record the appearance or worsening of clinical 
symptoms of liver injury (eg, fatigue, nausea, 
vomiting, right upper quadrant pain), or 
hypersensitivity, on the AE CRF 
• 
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 
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. 
• 
Serum acetaminophen adduct HPLC assay 
(quantifies potential acetaminophen contribution to 
liver injury in participants with definite or likely 
acetaminophen use in the preceding week [James 
et al, 2009]). 
NOTE: Not required in China. 
• 
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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Table 10-2: Phase 3-4 liver chemistry stopping criteria and follow-up 
assessments 
Liver chemistry stopping criteria 
AE=adverse event; ALP=alkaline phosphatase; ALT=alanine aminotransferase; AST=aspartate aminotransferase; 
CPK=creatine phosphokinase; CRF=case report form; HBsAg=hepatitis B surface antigen; HBcAb=hepatitis B 
core antibody; HPLC=high performance liquid chromatography; IgG=immunoglobulin G; IgM=immunoglobulin 
M; INR=international normalized ratio; LDH=lactate dehydrogenase; PCR=polymerase chain reaction; 
PK=pharmacokinetic; SAE=serious adverse event; ULN=upper limit of normal 
a Serum bilirubin fractionation should be performed if testing is available. If serum bilirubin fractionation is not 
immediately available, discontinue study medication if ALT ≥3xULN and bilirubin ≥2xULN. Additionally, if 
serum bilirubin fractionation testing is unavailable, record the absence/presence of detectable urinary bilirubin 
on dipstick which is indicative of direct bilirubin elevations suggesting liver injury.  
b All events of ALT ≥3xULN and bilirubin ≥2xULN (>35% direct bilirubin) or ALT ≥3xULN and INR >1.5 may 
indicate severe liver injury (possible ‘Hy’s Law’) and must be reported as a SAE (excluding studies of hepatic 
impairment or cirrhosis). The INR measurement is not required and the stated threshold value will not apply to 
participants receiving anticoagulants.  
c New or worsening symptoms believed to be related to liver injury (such as fatigue, nausea, vomiting, right upper 
quadrant pain or tenderness, or jaundice) or hypersensitivity (such as fever, rash or eosinophilia). 
d Includes: Hepatitis A IgM antibody; HBsAg and HBcAb; hepatitis C RNA; cytomegalovirus IgM antibody; 
Epstein-Barr viral capsid antigen IgM antibody (or if unavailable, heterophile antibody or monospot testing); and 
hepatitis E IgM antibody. 
e If hepatitis delta antibody assay cannot be performed, it can be replaced with a PCR of hepatitis delta RNA virus 
(where needed) (Le Gal et al, 2005). 
f PK sample may not be required for participants known to be receiving placebo or noncomparator interventions. 
Record the date/time of the PK blood sample draw and the date/time of the 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 
(Study Reference Manual). 
 
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Table 10-3: Phase 3-4 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 UCB 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, ALP, bilirubin) until the abnormalities 
resolve, stabilize, or return to Baseline.  
• 
If at any time, the participant meets liver chemistry 
stopping criteria, proceed as described in Section 7.1.1. 
• 
If ALT decreases from ALT 5xULN and <8xULN to 
≥3xULN but <5xULN, continue to monitor liver 
chemistries weekly.  
• 
If, after 4 weeks of monitoring, ALT <3xULN and bilirubin 
<2xULN, monitor participants twice monthly until liver 
chemistry tests resolve, stabilize, or return to baseline. 
ALP=alkaline phosphatase; ALT=alanine aminotransferase; AST=aspartate aminotransferase; ULN=upper limit 
of normal 
 
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10.7 
Appendix 7: Medical device adverse events (AEs), adverse 
device effects (ADEs), serious adverse events (SAEs), and 
device deficiencies: Definition and procedures for recording, 
evaluating, follow-up, and reporting 
The definitions and procedures detailed in this appendix are in accordance with ISO 14155.  
The detection and documentation procedures described in this protocol also apply to medical 
devices provided for use in the study. 
The Adverse Event and Device Deficiency Report Form will be sent to the sponsor using the 
paper form. The sponsor will then forward the ADE and device deficiency reports to the 
corresponding device manufacturer. The device manufacturer is responsible for the subsequent 
vigilance evaluation and reporting, if applicable. 
10.7.1 
Definition of AE and ADE 
AE and ADE Definition  
• 
An AE is defined as any untoward medical occurrence, unintended disease or injury, or 
untoward clinical signs (including abnormal laboratory finding) in study participants, users, or 
other persons, whether or not related to the investigational medical device. This definition 
includes events related to the investigational medical device or comparator and events related 
to the procedures involved except for events in users or other persons, which only include 
events related to investigational devices. 
• 
An adverse device effect (ADE) is defined as an adverse event related to the use of an 
investigational medical device. This definition includes any adverse events resulting from 
insufficient or inadequate instructions for use, deployment, implantation, installation, or 
operation, or any malfunction of the investigational medical device as well as any event 
resulting from use error or from intentional misuse of the investigational medical device. 
10.7.2 
Definition of SAE, SADE, and unanticipated SADE 
If an event is not an AE per definition above, then it cannot be a SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease). 
A SAE is an AE that: 
a. Led to death  
b. Led to serious deterioration in the health of the participant, that either resulted in: 
1. A life-threatening illness or injury. 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 
2. A permanent impairment of a body structure or a body function 
3. Inpatient or prolonged hospitalization. Planned hospitalization for a pre-existing condition, or a 
procedure required by the protocol, without serious deterioration in health, is not considered a SAE 
4. Medical or surgical intervention to prevent life-threatening illness or injury or permanent 
impairment to a body structure or a body function 
c. Led to fetal distress, fetal death, or a congenital abnormality or birth defect 
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SADE definition 
• 
A serious adverse device effect (SADE) is defined as an adverse device effect that has resulted 
in any of the consequences characteristic of a serious adverse event. 
USADE definition 
• 
An unanticipated serious adverse device effect (USADE) is a serious adverse device effect 
which by its nature, incidence, severity, or outcome has not been identified in the current 
version of the risk analysis report (see Section 8.3.9). 
10.7.3 
Definition of device deficiency 
Device deficiency definition 
• 
A device deficiency is an inadequacy of a medical device with respect to its identity, quality, 
durability, reliability, safety, or performance. Device deficiencies include malfunctions, use 
errors, and inadequate labeling. 
 
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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 
ADE 
adverse device effect 
ADL 
activities of daily living 
ADR 
adverse drug reaction 
AE 
adverse event 
AESM 
adverse event(s) of special monitoring 
ALP 
alkaline phosphatase 
ALT 
alanine aminotransferase 
AST 
aspartate aminotransferase 
C-SSRS 
Columbia-Suicide Severity Rating Scale 
CI 
confidence interval 
CNS 
central nervous system 
COVID-19 
coronavirus disease 2019 
CRF 
case report form 
CSF 
cerebrospinal fluid 
DIAM 
drug-induced aseptic meningitis 
ECG 
electrocardiogram 
FcRn 
neonatal Fc receptor 
FSH 
follicle-stimulating hormone 
gMG 
generalized myasthenia gravis 
GCP 
Good Clinical Practice 
HCP 
health care professional 
HRT 
hormonal replacement therapy 
ICF 
informed consent form 
ICH 
International Council for Harmonisation 
IEC 
Independent Ethics Committee 
Ig 
immunoglobulin 
IMP 
investigational medicinal product 
IRB 
Institutional Review Board 
IRT 
interactive response technology 
IV 
intravenous 
IVIg 
intravenous immunoglobulin 
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LTBI 
latent tuberculosis infection 
MG 
Myasthenia Gravis 
MG-ADL 
Myasthenia Gravis Activities of Daily Living 
MG-C 
Myasthenia Gravis Composite 
MGFA 
Myasthenia Gravis Foundation of America 
NMJ 
neuromuscular junction 
NCI-CTCAE 
National Cancer Institute Common Terminology Criteria for Adverse 
Events  
NTMBI 
nontuberculous mycobacterial infection 
pIDMC 
program Independent Data Monitoring Committee 
PRO 
patient reported outcome 
PDILI 
potential drug-induced liver injury 
PEX 
plasma exchange 
QMG 
Quantitative Myasthenia Gravis 
QTc 
corrected QT interval 
QTcF 
QT interval corrected for heart rate using Fridericia’s formula 
SADE 
serious adverse device effect 
SAE 
serious adverse event 
SAS 
Statistical Analysis System 
SC 
subcutaneous 
SIAQ 
Self-Injection Assessment Questionnaire 
SUSAR 
suspected unexpected serious adverse reaction 
TB 
tuberculosis 
TEAE 
treatment-emergent adverse event 
ULN 
upper limit of normal 
WOCBP 
woman of childbearing potential 
 
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10.11 
Appendix 11: Protocol amendment history 
The Protocol Amendment Summary of Changes Table for the current amendment is located 
directly before the Table of Contents. 
Amendment 4 (07 Sep 2023) 
Overall rationale for the amendment 
The primary reason for this protocol amendment is to align with the approved label in the US 
and with the proposed dosing regimen under review in Europe. 
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 
1.1 Synopsis (Overall 
design) 
Text amended to explain study 
participants will receive 
 
fixed weight tiered doses of 
rozanolixizumab. 
A paragraph was added to explain 
dosing for existing and new study 
participants starting the Treatment 
Period  
To align with the approved label 
in the US and with the proposed 
dosing regimen under review in 
Europe. 
1.1 Synopsis (Overall 
design) 
A table has been added to include 
the doses to be used in the US, 
Europe, and Canada. 
To align with the approved label 
in the US and with the proposed 
dosing regimen under review in 
Europe. 
1.1 Synopsis (Treatment 
groups and duration) 
A sentence was added to paragraph 
#2 to clarify the method of 
administration for participants who 
are no longer eligible for 
self-administration. 
Updated to provide clarity. 
1.3 Schedule of activities 
Footnote “c”: Updated to clarify 
that vital signs on Day 15 will be 
measured before rozanolixizumab 
administration, at the end of the 
infusion, and 1 hour after the end of 
infusion. In addition, the 
requirement to measure vital signs 
4 hours after the end of the infusion 
was removed for all days. 
To be consistent with remainder 
of protocol. 
1.3 Schedule of activities 
Footnote “c”: time windows added 
for vital signs measurements. An 
additional sentence was added that 
additional vital sign measurements 
can be performed at the 
investigator’s discretion.  
For logistical reasons. 
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Section # and name 
Description of change 
Brief rationale 
1.3 Schedule of activities 
Footnote “g”: Updated to clarify 
that the 
 
 
Updated to provide clarity. 
1.3 Schedule of activities 
Footnote “h”: postdose observation 
was updated from 4 to 2 hours. 
To align with the last postdose 
vital sign measurement. 
4.1 Overall design 
Text amended to explain study 
participants will receive 
 
weight-tiered doses of 
rozanolixizumab. 
A paragraph was added to explain 
dosing for existing and new study 
participants starting the Treatment 
Period. 
To align with the approved label 
in the US and with the proposed 
dosing regimen under review in 
Europe. 
4.1 Overall design 
A sentence was added to paragraph 
#6 to clarify the method of 
administration for participants who 
are no longer eligible for 
self-administration. 
Updated to provide clarity. 
4.1.1 Unscheduled visit 
New section added for unscheduled 
visits. 
Updated to provide clarity. 
4.3 Justification for dose 
The doses used in the US, Europe, 
and Canada have been updated. 
To align with the approved label 
in the US and with the proposed 
dosing regimen under review in 
Europe. 
5.1 Inclusion criteria 
Criterion #5 (now 5a) was updated 
to add an exception for study 
participants receiving an anti-FcRn 
treatment within 8 weeks prior to 
the Screening Visit, who must have 
a serum total IgG level at the 
Screening Visit ≥2g/L. 
To facilitate the enrollment of 
potential study participants 
recently treated with an 
anti-FcRn (rozanolixizumab in 
another MG study, or 
efgartgimod), considering that 
low levels of IgG are to be 
expected and explained by the 
mode of action of anti-FcRn 
treatments.  
5.2 Exclusion criteria 
Criterion #3 (now 3a) has been 
updated to “Study participant has a 
known hypersensitivity to other 
anti-FcRn medications, to any 
components of the study 
medication, to any of the excipients 
(including 
), or has a 
known history of 
, 
since both 
 and 
 are constituents of the 
rozanolixizumab formulation.” 
To align with the 
contraindications in the 
Investigator’s Brochure. 
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Section # and name 
Description of change 
Brief rationale 
5.2 Exclusion criteria 
Criterion #6 (now 6a) has been 
updated to include “(unless the 
reason is directly related to 
MG0020 participation).” 
Updated to provide clarity. 
5.2 Exclusion criteria 
Criterion #9 (now 9a) has been 
updated to include “treatment with 
ravulizumab within 
 
before the Baseline Visit or prior 
treatment with cyclophosphamide.” 
Updated to include the second 
intravenous C5 inhibitor 
approved for the treatment of 
gMG (ravulizumab), and to be 
consistent across the 
rozanolixizumab clinical 
program. 
5.2 Exclusion criteria 
Criterion #19 (now 19a) has been 
updated to add “malignant” before 
neoplastic disease. An additional 
exception was added for thymoma 
that did not require chemotherapy 
and/or radiotherapy after removal. 
To align with previous 
rozanolixizumab MG studies. 
5.3 Lifestyle restrictions 
Reference to Japan-specific 
regulations in Appendix 9 
(Section 10.9) was removed. 
Japan-specific changes are 
covered in a separate protocol 
amendment. 
6 STUDY TREATMENTS  
An additional sentence was added 
to state that treatments in this study 
vary based on either an approved 
label in a region (US) or a proposed 
dosing under review in a region 
(Europe and Canada). 
Updated to provide further 
clarity. 
6.1 Treatments administered 
Dosage level(s) have been updated. 
To align with the approved label 
in the US and with the proposed 
dosing regimen under review in 
Europe. 
6.4 Treatment compliance  
A cross reference was added to 
Table 1-2 in reference to the 
intended dose.  
Updated to provide clarity. 
6.6 Dose modification 
The following text has been 
updated “ie, study participants must 
maintain their dose (based on body 
weight as recorded at Screening) 
throughout.” 
Updated to provide clarity. 
7.1.2 Temporary IMP 
discontinuation 
Change to the temporary IMP 
discontinuation criterion with IgG 
threshold of 
hypogammaglobulinemia from a 
“must be” to “may be” category. 
To provide increased treatment 
flexibility considering that data 
from the rozanolixizumab 
completed clinical studies did 
not show an increased infection 
rate in study participants treated 
with similar dose regimens of 
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Section # and name 
Description of change 
Brief rationale 
rozanolixizumab  as proposed 
for MG0020. 
7.1.3 Study medication 
permanent discontinuation 
criteria 
Criterion #2 (now 2a) has been 
updated to add “reoccurrence of 
malignant” before neoplastic 
disease. 
To be consistent with the 
exclusion criteria. 
8 STUDY ASSESSMENTS 
AND PROCEDURES 
Details regarding unscheduled 
visits have been added. 
Updated to provide clarity. 
8.2.3 Self-Injection 
Assessment Questionnaire 
(Infusion version) 
Updated to clarify that the 
 
 
 
Updated to provide clarity. 
8.3 Adverse events and 
serious adverse events  
A sentence was added: For results 
disclosure on public registries 
(eg, ClinicalTrials.gov), 
treatment-emergent AEs and 
treatment-emergent SAEs will be 
published. 
Added for additional clarity in 
regard to results posting. 
8.5 Treatment of overdose 
Clarification of intentional and 
nonintentional overdose, and 
overdose thresholds for each dose 
provided. 
Updated to provide clarity and 
to provide overdose values for 
all doses. 
9.3.1 Analysis of the primary 
safety endpoint 
The last sentence was updated to 
remove the explanation on fixed 
and random effects.  
The confidence interval will be 
calculated for each 
self-administration method 
therefore the last part of the 
sentence is not applicable. 
10.9 Appendix 9: 
Country-specific 
requirements 
Country-specific requirements for 
Japan have been removed. 
There is a local protocol for 
Japan. 
10.11 Appendix 11: Protocol 
amendment history 
Details of the previous amendment 
(Protocol Amendment 3) have been 
added. 
General update. 
10.1.3 Informed consent 
process 
Updated to clarify that the caregiver 
must sign the informed consent 
form by the Baseline visit, if 
applicable. 
Updated to provide clarity. 
10.13.2: Management of 
infections and 
hypogammaglobulinemia 
Change to the temporary IMP 
discontinuation criterion with IgG 
threshold of 
hypogammaglobulinemia from a 
“must be” to “may be” category. 
Adapted as per the updated 
discontinuation criteria. 
10.15 Appendix 15: 
Evaluation by HCP at site of 
The intended doses were removed 
from the form  
Updated for clarity. 
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Section # and name 
Description of change 
Brief rationale 
safe and successful 
self-administration (to collect 
data for primary endpoints) 
 
Amendment 3 (09 Dec 2022) 
Overall rationale for the amendment 
The reason for this protocol amendment is to add the EudraCT number to the regulatory agency 
identifying numbers listed in the title page of the protocol. No other changes have been made. 
Amendment 2 (04 Nov 2022) 
Overall rationale for the amendment 
The primary reason for this protocol amendment is to provide an update on the safety 
information in line with the updated Investigator's Brochure (IB) dated Sep 2022, updates on the 
adverse events of special monitoring (AESM), and an update on the planned fixed dose of 
rozanolixizumab based on study participants’ body weight. The secondary endpoint related to the 
occurrence of medication errors, the Schedule of Activities, the criteria for study medication 
discontinuation and for participant discontinuation from the study have also been updated. 
Additional updates have been incorporated to provide further clarity on the protocol. 
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 editorial and formatting 
changes have been made. 
To provide clarity and remain 
consistent with remainder of 
protocol. 
Safety reporting contact page 
The contact details for the 
reporting of AEs (serious and 
nonserious) have been deleted 
and the contact details for the 
reporting of SAEs and self-
administration medication errors 
have been added. 
To update the reporting (24h) of 
AEs and add the reporting of 
self-administration medication 
errors. 
1.1 Synopsis (rationale) 
2.1 Study rationale 
The text has been updated to 
replace “IgG” with “Ig” when 
applicable and to delete “(also 
referred to as “rapid push”)” in 
reference to the manual push 
technique. 
Updated to provide clarity. 
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Section # and name 
Description of change 
Brief rationale 
1.1 Synopsis (objectives and 
endpoints) 
3 Objectives and endpoints 
The secondary objective has 
been updated to add “of 
rozanolixizumab.” 
Updated to provide clarity. 
The secondary endpoint related 
to the occurrence of medication 
errors has been updated to 
“Occurrence of medication 
errors associated with adverse 
reactions during the 2 Self-
administration Periods of the 
study.” 
To update the type of 
medication errors informing the 
secondary endpoint and the time 
frame for the collection of 
medication errors in the study. 
1.1 Synopsis (overall design) 
4.1 Overall design 
4.3 Justification for dose 
6.1 Treatments administered  
6.4 Treatment compliance 
8.5 Treatment of overdose 
10.15 Appendix 15: Evaluation 
by HCP at site of safe and 
successful self-administration 
(to collect data for primary 
endpoints) 
For study participants weighing 
≥35kg to <50kg, 
 fixed 
doses of rozanolixizumab 
 will be administered. 
To add a lower fixed dose of 
rozanolixizumab for study 
participants weighing ≥35kg to 
<50kg. 
1.1 Synopsis (overall design) 
4.1 Overall design 
The overall design wording has 
been updated to add that 
rozanolixizumab-naïve study 
participants and study 
participants previously exposed 
to rozanolixizumab will be 
included in the study. 
Updated to provide clarity. 
1.1 Synopsis (treatment groups 
and duration) 
4.1 Overall design 
The text in reference to the 
randomization at Visit 8 has 
been updated to add “after the 
completion of the Training 
Period.” 
Updated to provide clarity and 
remain consistent with the study 
design. 
1.1 Synopsis (treatment groups 
and duration) 
4.1 Overall design 
The text in reference to 
participants who cannot be 
confirmed eligible for self-
administration has been updated 
to delete “at Visit 8, 9, or 15.” 
Updated to simplify the protocol 
text. 
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Section # and name 
Description of change 
Brief rationale 
1.1 Synopsis (treatment groups 
and duration) 
4.1 Overall design 
The following wording has been 
added “Additionally, if a study 
participant becomes no longer 
eligible for self-administration 
during the Self-administration 
Periods, he/she can continue to 
be treated on-site per protocol 
based on investigator decision.” 
To provide further clarification. 
1.1 Synopsis (treatment groups 
and duration) 
4.1 Overall design 
6.8 Treatment after the end of 
the study 
The managed access program 
has been replaced with a post-
trial access to rozanolixizumab. 
To provide clarity on the access 
to rozanolixizumab at the time 
of completion of the study. 
1.3 Schedule of activities 
Added table caption and 
reference to the Schedule of 
Activities table. 
To be consistent with remainder 
of protocol. 
1.3 Schedule of activities 
Footnote “m”: Updated to 
clarify that a full neurological 
examination should be 
performed in the event of severe 
and/or serious headache or 
suspected aseptic meningitis. 
Added cross-reference to 
Section 8.2.5 and to Section 
1.3.1 (Table 1-3). 
Added row for blood sampling 
for exploratory safety biomarker 
analysis and related footnote “r”. 
The accumulated safety data on 
rozanolixizumab led to an 
update of the adverse events 
requiring special monitoring. 
As of the cut-off date of the IB 
(13 Jul 2022), the following 
serious adverse events (SAEs) 
 
 (PT terms) suggest a 
possible causal relationship to 
rozanolixizumab; based on both 
their temporal association with 
investigational medicinal 
product (IMP) infusion 
(primarily initial infusion) and 
given the events have occurred 
more than once in the 
rozanolixizumab clinical 
development program. 
1.3.1 Additional study 
assessments 
New section added to detail 
additional study assessments 
that may be required in case of 
AESM of severe and/or serious 
headache or the new AESM of 
suspected aseptic meningitis. 
2.3 Benefit/risk assessment 
Revised text on most common 
adverse drug reactions, safety 
concerns, and other safety topics 
of interest. 
8.3.8 Adverse events of special 
monitoring 
Added suspected aseptic 
meningitis to the list of AESM. 
Wording on infections and 
hypersensitivity reactions 
including infusion-related 
reactions and anaphylaxis and 
their guidance has been added. 
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Section # and name 
Description of change 
Brief rationale 
8.8 Biomarkers 
Added the details of biomarkers 
evaluation. 
10.2 Appendix 2: Clinical 
Laboratory Tests, 
Table 10-1 
Added cross-reference to 
Table 1-3. 
1.3 Schedule of activities 
Added row for brief physical 
examination. Footnote “m” and 
scheduled visits after V1 have 
been removed from the complete 
physical examination and added 
to the brief physical 
examination. 
To further clarify the physical 
examination assessment after 
Screening (V1). 
1.3 Schedule of activities 
Added row for randomization. 
To provide clarity and remain 
consistent with the study design. 
1.3 Schedule of activities 
The following note has been 
added “HSA=home self-
administration, ie, unsupervised 
self-administration/not in the 
presence of an HCP.” 
To provide clarity on HSA. 
1.3 Schedule of activities 
Footnote “q”: Updated to delete 
“at the Visit 7 (
 
evaluation” and to add a cross-
reference to Section 4.1.  
Updated to provide clarity and 
remain consistent with the study 
design. 
2 Introduction 
The text has been updated to add 
that rozanolixizumab has been 
administered to human study 
participants in several clinical 
studies. 
Updated in line with studies 
status at the time of this 
amendment and to provide 
clarity.  
2.2 Background 
A summary of the results of the 
Phase 3 gMG program has been 
added. The text in reference to 
the Phase 2 and Phase 3 gMG 
studies has been updated. 
2.3 Benefit/risk assessment 
Results of the Phase 3 study, 
MG0003, have been added.  
To provide data from the 
Phase 3 study, MG0003. 
6.1 Treatments administered 
References to vial and carton 
have been removed from the 
packaging and labeling row. 
Packaging will be described in 
the IMP Handling Manual. 
6.2 Preparation, handling, 
storage, and accountability 
requirements 
The text in reference to the 
instructions for storing study 
medication has been updated to 
add “and in the IMP Handling 
Manual.” 
Updated to provide clarity. 
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Section # and name 
Description of change 
Brief rationale 
6.2.1 Drug/Device 
accountability 
The text in reference to the 
recording of study medication 
dispensing has been updated to 
replace “case report form" with 
“Drug/Device Accountability 
form.” 
Updated to provide clarity.  
6.3 Measures to minimize bias: 
randomization and blinding 
Cross-references to Table 1-1 
and details pertaining to the IRT 
at Visit 2 have been added. 
Updated to provide further 
clarity. 
6.4 Treatment compliance 
The text in reference to the 
photographic documentation has 
been updated. 
To clarify that a mobile phone 
will be used for the 
photographic documentation and 
that further details are provided 
in the IMP Handling Manual.  
6.6 Dose modification 
The following text has been 
added “ie, study participants 
must maintain either the 
 
dose or the 
 dose (based 
on body weight as recorded at 
Screening) throughout.” 
To provide clarity. 
7.1.2 Temporary IMP 
discontinuation 
The temporary IMP 
discontinuation criteria in 
relation to COVID-19 infection 
have been updated. 
To adapt the withdrawal criteria 
to the evolution of the medical 
practices and local guidelines 
with regards to COVID-19 
management. 
7.1.2 Temporary IMP 
discontinuation 
Added a new criterion (#3) on 
the temporary discontinuation of 
IMP (must be) in the event of a 
first diagnosis of suspected 
aseptic meningitis. 
The accumulated safety data on 
rozanolixizumab led to an 
update of the discontinuation 
criteria which is in line with the 
revisions to adverse events 
requiring special monitoring. 
7.1.3 Study medication 
permanent discontinuation 
criteria 
Added a new criterion (#9) on 
the permanent discontinuation of 
study medication in the event of 
recurrence of aseptic meningitis.  
Deleted criterion #7 on the 
permanent discontinuation of 
study medication in the event of 
serious or recurrent (ie, >2 
occurrences) severe AE of 
headache which is considered 
related to the study medication. 
Subsequent criteria have been 
renumbered. 
7.1.2 Temporary IMP 
discontinuation 
Added a new criterion (#3) on 
the temporary discontinuation of 
Updated to be consistent with 
the rozanolixizumab clinical 
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Section # and name 
Description of change 
Brief rationale 
IMP (may be) at the discretion 
of the investigator in cases 
deemed strictly necessary for the 
participant’s medical care. 
program and to allow for 
investigator discretion. 
7.1.2 Temporary IMP 
discontinuation 
A cross-reference to Section 8 
has been added to the text. The 
text in reference to IMP 
treatment resumption has been 
updated. 
Updated to provide clarity.  
7.1.3 Study medication 
permanent discontinuation 
criteria 
The text in reference to 
rozanolixizumab discontinuation 
at the discretion of the 
investigator has been updated to 
add “or becomes unable to 
comply.” 
Updated to provide clarity. 
7.1.3 Study medication 
permanent discontinuation 
criteria 
The text has been updated to add 
that the handling of replacement 
participants is described in 
Section 7.2. 
Updated to provide clarity. 
7.2 Participant 
discontinuation/withdrawal from 
the study 
Criterion #4 on the withdrawal 
from the study in the event of 
active suicidal ideation has been 
moved to Section 7.1.3 (study 
medication permanent 
discontinuation criteria, 
criterion #10). 
Updated in order to increase the 
consistency and clarity of the 
IMP discontinuation criteria 
throughout the protocol. 
8.2.9 Suicidal risk monitoring 
The cross-reference to Section 
7.2 has been replaced with a 
cross-reference to Section 7.1.3. 
 
7.2 Participant 
discontinuation/withdrawal from 
the study 
The wording in reference to the 
replacement of study 
participants has been updated to 
replace “before the end of the 
second Treatment Period” with 
“before the end of the Self-
administration Period 2.” 
Updated to be consistent with 
the study design.  
8 Study assessments and 
procedures 
Added wording on contingency 
measures in case of exceptional 
circumstances.  
Updated to be consistent with 
the rozanolixizumab clinical 
program.  
8.2.5 Physical examination 
The details of the brief physical 
examination have been added. 
Added to provide clear guidance 
to investigators on the brief 
physical examination. 
8.2.5 Physical examination 
The details of the full 
neurological assessment in the 
event of severe and/or serious 
Added to provide clear guidance 
to investigators on the 
management of AESM of severe 
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Section # and name 
Description of change 
Brief rationale 
headache or suspected aseptic 
meningitis have been added. 
and/or serious headache or 
suspected aseptic meningitis. 
8.2.6 Vital signs 
Blood pressure measurements 
have been updated to 1 reading. 
Updated to be consistent with 
the rozanolixizumab clinical 
program. 
8.2.10.1 Tuberculosis 
assessment 
Added cross-reference to new 
Appendix 19. 
To provide clarity and remain 
consistent with remainder of 
protocol. 
8.2.11 COVID-19 precautions 
Added cross-reference to 
Section 7.1.2. 
To provide clarity and remain 
consistent with remainder of 
protocol. 
8.3.7 Medication errors 
associated with self-
administration 
Added new section on 
medication errors associated 
with self-administration. 
Subsequent subsections have 
been renumbered. 
To clarify medication errors, 
their classification and reporting 
procedures, and medication 
errors identified as AESM. 
8.3.8 Adverse events of special 
monitoring 
The AESM related to 
medication error has been 
updated to “medication error 
associated with adverse 
reaction(s).” 
10.12 Appendix 12: Medication 
errors associated with self-
administration 
Added new appendix on the 
definition of medication errors. 
Subsequent appendices have 
been renumbered. 
8.5 Treatment of overdose 
In the numbered point #3, 3 days 
has been replaced with 72 hours.  
Updated to be consistent with 
remainder of protocol. 
8.6 Pharmacokinetics 
Text updated to add that PK 
blood sample in the event of 
PDILI could be collected as 
described in Appendix 6. 
Updated to provide further 
clarification. 
9.1 Definition of analysis sets 
The Safety Set has been 
renamed Randomized Safety 
Set, the Training Safety Set has 
been renamed Safety Set, and 
their definition has been 
updated. A Full Analysis Set has 
been added. 
To update the analysis sets and 
their definition. 
9.2 General statistical 
considerations 
The text in reference to 
summary statistics by sequence 
and overall has been updated to 
delete “AEs.”  
Details will be provided in the 
SAP. 
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Section # and name 
Description of change 
Brief rationale 
9.3.1 Analysis of the primary 
safety endpoint 
9.3.2 Analysis of the secondary 
safety endpoint 
9.3.3 Other safety analyses 
9.7 Handling of dropouts or 
missing data 
The text has been updated to add 
or update the analysis set(s) on 
which the analyses will be 
based. 
To align with the updates in the 
analysis sets. 
9.3.2 Analysis of the secondary 
safety endpoint 
The text in reference to 
medication errors has been 
updated to add “associated with 
adverse reactions.” The analysis 
of medication errors during the 
Training Period has been 
deleted. 
To align with the updates in the 
secondary safety endpoint. 
9.3.2 Analysis of the secondary 
safety endpoint 
9.3.3 Other safety analyses 
The analysis of TEAEs by 
rozanolixizumab-naïve or non-
naïve participants has been 
deleted. 
To update the analysis of 
TEAEs. 
10.1.3 Informed consent process 
Information on caregiver’s 
informed consent (if applicable) 
has been added. 
To provide clarity on the 
informed consent process in 
case of a caregiver.  
10.1.5 Committees structure 
Further details pertaining to the 
review process of safety data 
have been added. 
To provide clarity on the review 
process of safety data. 
10.1.7.2 Apps 
The text has been updated to add 
Japan to the listed countries. 
To add Japan to the listed 
countries where the app falls 
outside medical device 
regulations. 
10.1.7.2 Apps 
The following has been deleted 
“Finally, the screenshots of the 
app should be made available 
during ethics committee review 
of the clinical trial protocol.” 
To simplify the protocol text. 
10.3 Appendix 3: Adverse 
events – Definitions and 
procedures for recording, 
evaluating, follow-up, and 
reporting 
The cross-reference to the 
contacts for SAE reporting has 
been updated. 
To align with the updates in the 
safety reporting contact page of 
the protocol. 
10.9 Appendix 9: Country-
specific requirements, 
Japan 
Clarified that country-specific 
requirements for Japan aligned 
with Japan-GCP will be 
provided separately in Protocol 
Exhibit. 
Clarification. 
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Section # and name 
Description of change 
Brief rationale 
10.10 Appendix 10: 
Abbreviations and trademarks 
Several additions and one 
deletion have been made to the 
list of abbreviations. 
General update. 
10.11 Appendix 11: Protocol 
amendment history 
Details of the previous 
amendment (protocol 
amendment 1) have been added. 
General update. 
10.12 Appendix 12: 
Management of headaches, 
infusion reactions or 
hypersensitivity reactions, and 
infections and 
hypogammaglobulinemia 
Has become Section 10.13 and 
the title has been updated to 
“Appendix 13: Management of 
infusion reactions or 
hypersensitivity reactions and 
infections and 
hypogammaglobulinemia.” 
To align with the updates in the 
adverse events requiring special 
monitoring and their 
management. 
10.12.1 Management of 
headache 
The protocol guidance for the 
management of headache has 
been updated and moved to 
Section 10.14 (Appendix 14). 
Subsequent subsections have 
been renumbered. 
10.14 Appendix 14: 
Management of adverse event of 
special monitoring 
New appendix: Added to 
provide guidance on the 
management of AESM. 
Subsequent appendices have 
been renumbered.  
10.19 Appendix 19: 
Tuberculosis Questionnaire 
New appendix has been added. 
To provide the tuberculosis 
questionnaire.  
11 References 
One reference has been added to 
the list of references. 
General update. 
 
Amendment 1 (31 Aug 2022) 
Overall rationale for the amendment 
The primary reason for this protocol amendment is to clarify that there is no formal rollover from 
study MG0007 to MG0020. 
One exclusion criterion was also updated to align with the current approach agreed for the 
rozanolixizumab clinical program. 
Section # and name 
Description of change 
Brief rationale 
1.1 Synopsis (overall design) 
4.1 Overall design 
Overall design wording updated 
to remove “Study participants 
from study MG0007 and 
rozanolixizumab-naïve 
Updated to provide clarity to the 
sites that there is no formal 
rollover from study MG0007 to 
MG0020. 
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participants will be included in 
the study.” 
5.2 Exclusion criteria 
Criterion #7 (now 7a) has been 
updated to include “or a BCG 
vaccine within 1 year before 
starting treatment;” 
Updated to align with the 
current approach agreed for the 
rozanolixizumab clinical 
program. 
 
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10.12 
Appendix 12: Medication errors associated with 
self-administration 
The definitions and procedures detailed in this appendix are in accordance with the 
pharmacovigilance obligations detailed in Title IX of Directive 2001/83/EC and Regulation (EC) 
726/2004, Chapter 3, Article 28 with regard to the recording, reporting, and assessment of 
suspected adverse reactions (serious and nonserious) associated with an error in prescribing, 
storing, dispensing, preparing for administration, or administering a medicinal product for human 
use authorized in the EU, which have been adapted as per MG0020 protocol specifications. All 
medication errors will be recorded and reported to the sponsor or designee as indicated in 
Section 8.3.7. 
Medication Error Definition 
Medication error: A medication error is an unintended failure in the drug treatment process that leads 
to, or has the potential to lead to, harm to the patient. A failure in the drug treatment process does not 
refer to lack of efficacy of the drug, rather to human- or process-mediated failures. 
The medication error can be further classified based on the factual information on the case, into the 
following categories: 
• 
Medication errors associated with adverse reaction(s): result from an error (which has already 
occurred or accomplished) in the medication use associated with harm to the study participant-this 
is an adverse event of special monitoring (AESM)-please see Section 10.14.  
• 
Medication errors without harm: result from an error (which has already occurred or 
accomplished) in the medication use associated with no harm to the study participant. 
• 
Intercepted medication errors (or near miss): in which an intervention (eg, the timely check of 
the incorrect preparation and administration of the study medication) has prevented an actual harm 
being caused to the study participant. 
• 
Potential medication errors: are the recognition of circumstances that could lead to a medication 
error, which may or may not involve a study participant. 
The term potential medication error refers to all possible mistakes in the storing, preparation for 
administration, or administration of the investigational medicinal product (IMP) by the study 
participant and may lead to: 
− 
A medication error with harm, but without knowing the actual cause  
− 
A medication error without harm and without knowing the actual cause 
− 
A medication error without harm, but with the awareness of the actual cause  
 
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10.13 
Appendix 13: Management of infusion reactions or 
hypersensitivity reactions and infections and 
hypogammaglobulinemia 
10.13.1 
Management of infusion reactions or hypersensitivity reactions 
Study participants must be closely monitored for reactions during and after rozanolixizumab 
administration. Standard precautions must be taken for the study participants with regard to 
potential infusion-related reactions. Suggested management guidelines for infusion-related 
reactions and anaphylaxis at the study site are provided in Table 10-4. Definitions of the severity 
of the relevant events will be consistent with NCI-CTCAE version 5.0 (Appendix 3 
[Section 10.3]). It is recommended that for study participants who self-administer at home, 
consideration is given to readily-available emergency medical services in case of an infusion 
reaction or hypersensitivity reaction. During study drug administration, the participant’s home or 
alternative location must not be so remote that a reasonable arrival time of an ambulance could 
not be predicted. 
Table 10-4: Suggested management guidelines at the study site for infusion 
reactions 
Type of reaction 
Suggested action 
Acute – Mild 
Grade 1 
Monitor vital signs every 10 minutes. 
If the reaction worsens to Grade 2, follow the instructions 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 or IM. 
Consider administering paracetamol or nonsteroidal anti-inflammatory 
drugs. 
Monitor vital signs initially every 5 minutes. 
If the reaction improves and upon further assessment it is clear that the 
event is not an anaphylaxis, restart the infusion cautiously. Continue to 
monitor vital signs every 5 minutes. 
If reaction recurs or worsens to Grade 3, discontinue infusion. 
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Table 10-4: Suggested management guidelines at the study site for infusion 
reactions 
Acute – Severe 
Grade 3 or 
anaphylaxis 
Discontinue rozanolixizumab 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 minutes. 
− Hospitalize, if condition not improving or worsens. 
− Monitor study participant until symptoms resolve. 
CTCAE=Common Terminology Criteria for Adverse Events; IM=intramuscular; IV=intravenous 
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 the Sampson Criteria Questionnaire should be completed. The infusion must be 
discontinued immediately, and emergency resuscitation measures implemented. 
10.13.2 
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. 
If a study participant has a significant infective episode including but not limited to bacteremia 
or sepsis, infectious meningitis, septic arthritis, osteomyelitis, complicated pneumonia, or 
visceral abscess which may or may not result in hospitalization, they MUST discontinue IMP, 
perform the Early Withdrawal Visit and may enter the Safety Follow-up Period. This list is not 
intended to be all inclusive and the investigator is expected to apply his/her judgment on 
continuing IMP based on the clinical situation at hand. 
In the event of a nonserious 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 nonserious persisting or 
recurrent infection with a serum total IgG level between ≥1g/L and <2g/L. Upon resolution of 
infection and the IgG returning to the level of ≥2g/L, the study participant may be allowed to 
resume treatment with the IMP. Ad hoc assessments of IgG levels may be performed to monitor 
their recovery. The blood samples collected for the ad hoc IgG assessments should be sent to 
both the local and the central laboratories. Decisions on whether the study participant can resume 
treatment may be taken based on the local laboratory results (IgG ≥2g/L as per local laboratory). 
In such cases, the local laboratory results must be entered in the eCRF. 
Treatment may 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. 
Ad hoc assessment may be performed to monitor the recovery of IgG levels. The blood samples 
collected for the ad hoc IgG assessments should be sent to both the local and the central 
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laboratories. Decisions on whether the study participant can resume treatment may be taken 
based on the local laboratory results (IgG ≥2g/L as per local laboratory). In such cases, the local 
laboratory results must be entered in the eCRF. 
10.14 
Appendix 14: Management of adverse event of special 
monitoring 
Adverse events of special monitoring are defined as product-specific AEs, adverse reactions, or 
safety topics requiring special monitoring by one or more regulatory authorities or by UCB. 
For rozanolixizumab, AESM (defined by UCB) are: 
• Severe and/or serious headache 
• Suspected aseptic meningitis  
• Medication error associated with adverse reaction(s) 
Occurrence of AESM require immediate reporting (within 24 hours regardless of seriousness) to 
UCB. Upon reception of AESM by UCB, a medical follow-up questionnaire will be sent to the 
site to gather extensive medical information about the AESM. See Table 1-4 for additional 
assessments that may be required in case of AESM of severe and/or serious headache or 
suspected aseptic meningitis. 
Aseptic meningitis (suspected) 
Drug-induced aseptic meningitis is a diagnosis of exclusion after ruling out infectious causes 
(Jolles et al, 2000). A few cases of aseptic meningitis (drug-induced) have been reported in the 
rozanolixizumab program. Consequently, aseptic meningitis (suspected) is being managed as an 
AESM (Section 8.3.8).  
Participants should be monitored for signs and symptoms suggestive of central nervous system 
(CNS) involvement and evaluated immediately if meningitis is suspected. A full neurological 
workup should be strongly considered including, but not limited to imaging eg, computed 
tomography (CT) scan, or preferably gadolinium-enhanced magnetic resonance imaging (MRI), 
a lumbar puncture with cerebrospinal fluid (CSF) analysis inclusive of glucose, protein, 
differential complete blood count (CBC), cultures, gram stain, and/or viral polymerase chain 
reactions (PCRs) as appropriate. Whenever possible, CSF should be stored for assessment of 
rozanolixizumab pharmacokinetic, pharmacodynamic, specific antibody titers, or other 
biomarkers. A concurrent blood sample should be collected as per local practice. The ultimate 
investigative procedures are at the discretion of the investigator or the treating physician. For 
studies where a neurologist is not the investigator, a neurological consultation is also 
recommended to aid in decision making and patient management. In addition, blood samples for 
exploratory safety biomarkers (see Section 8.8) should be collected for participants with a 
diagnosis of DIAM preferably within 72 hours after onset of symptoms. These investigations 
will be performed to further understand the potential mechanisms of DIAM in the participants. 
All procedures related to the diagnosis, treatment, and investigation of meningitis should be 
recorded in the eCRF, and preliminary data should be included on the SAE form used for 
reporting the event as an AESM within 24 hours (ie, preliminary data reported on the first 
reporting may not have CSF results yet, but the reporting should occur as soon as there is a 
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suspected diagnosis. Full results should be communicated in subsequent exchanges with the 
sponsor).  
Treatment must be temporarily held if a participant has a diagnosis of suspected meningitis of 
any cause until the diagnostic workup is complete. Based on CSF findings, negative cultures, 
absence of other disease causes, and relationship with IMP, a diagnosis of DIAM can be made. If 
deemed appropriate by the investigator and agreed upon by the participant and the sponsor, the 
study treatment can resume upon the complete resolution of symptoms. The benefit and risk of 
the treatment should be carefully considered prior to reinitiating the IMP. If a participant 
experiences a second episode of similar symptoms suggestive of DIAM, then the participant 
must permanently discontinue the IMP. 
Participants experiencing an event of DIAM should be strongly encouraged to remain in the 
study regardless of IMP discontinuation. This will allow for monitoring and follow-up of the 
participant including a complete neurological exam on subsequent physical examinations. 
Longer term follow-up on any AEs related to DIAM that are ongoing may be warranted until 
resolution. 
Associated symptoms with aseptic meningitis should be managed at the investigator’s discretion.  
Severe and/or serious headache  
Based on current available clinical data, headache is the most commonly reported ADR in study 
participants treated with rozanolixizumab. Study participants should be well informed of this 
potential ADR and should be instructed on how to manage it. 
Determination of the severity of headache will be consistent with NCI-CTCAE version 5.0. 
Severe headache is defined as severe pain limiting self-care activities of daily living (ADL). 
Self-care ADL refers to bathing, dressing and undressing, feeding self, using the toilet, and 
taking medications. 
In the event of a headache, the investigators should take the medical history of previous 
headaches, concomitant medication, and comorbidities (eg, asthma) into consideration. 
If the severe and/or serious headache is initially reported at a home visit or during a telephone 
call, the study participant should be evaluated by HCP as soon as possible for further 
investigations. Study participants should be monitored for signs and symptoms suggestive of 
CNS involvement and evaluated immediately if other causes (eg, meningitis, intracranial 
bleeding) are suspected (please see Section 1.3.1 [Table 1-4]). 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. 
Should the investigator elect to hold IMP for severe and/or serious headache, if deemed 
appropriate by the investigator and agreed upon by the study participant and the sponsor, the 
IMP can resume upon the resolution of symptoms. The benefit and risk of the treatment should 
be carefully considered before reinitiating the IMP.  
Headaches will be treated as clinically indicated according to national guidelines. It is 
recommended that the study participant has an analgesic available in case of headache with the 
instruction for frequency and dosage provided by an HCP. The analgesic can be started at the 
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early onset of headache. 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 headache after discussion with the medical 
monitor. The benefit and risk of continuing treatment with IMP and chronic prophylaxis with 
analgesics must be carefully evaluated by the investigator. 
Medication error associated with adverse reaction(s) 
A medication error associated with a suspected (serious and nonserious) adverse reaction(s) is an 
unintended failure in the self-administration of IMP that leads to harm to the study participant, 
and should be assessed, recorded, and reported within 24 hours of awareness following the SAE 
recording and reporting procedures as indicated in Appendix 3 (Section 10.3) and using the 
Medication Error form as indicated in Section 8.3.7.  
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10.15 
Appendix 15: Evaluation by HCP at site of safe and successful 
self-administration (to collect data for primary endpoints) 
10.15(a): HCP evaluation checklist following self-administration at site at Visit 13 
(
) and Visit 19 (
) 
 
Study participant/caregiver is able 
to perform following steps 
Visit 13 
Yes/No  
Comments  
Visit 19 
Yes/No  
Comments  
1  
Identify and prepare all supplies 
required  
  
  
  
  
2  
Confirm that the vial is safe to use  
  
  
  
  
3  
Prepare and plan sufficient time for 
the infusion as well as make sure the 
medication reaches room temperature 
gradually before the infusion starts 
(without the use of heating devices)  
  
  
  
  
4  
Prepare and clean all work surfaces 
thoroughly 
  
  
  
  
5  
Ensure hands are washed thoroughly 
before the infusion 
  
  
  
  
6  
Position themselves comfortably next 
to the work surface 
  
  
  
  
7  
Ensure rozanolixizumab supplies are 
ready for assembly and infusion 
  
  
  
  
8  
Fill the syringe/ infusion system with 
prescribed dose of rozanolixizumab 
and check for/remove any air bubbles 
  
  
  
  
9a  Syringe driver set up as per 
manufacturer’s instructions/IMP 
manual 
  
  
  
  
9b  Manual Push: Set up as per IMP 
manual 
  
  
  
  
10  Prepare the infusion site on the 
abdomen 
  
  
  
  
11  Insert the needle into the 
subcutaneous tissue  
  
  
  
  
12  Secure the needle to the skin 
  
  
  
  
13  Start the infusion and record start 
time 
  
  
  
  
14  End the infusion and record end time 
  
  
  
  
15  Apply a dressing 
  
  
  
  
16  Safely and correctly dispose of the 
syringe, transfer device, needle, vials 
including those containing any 
unused liquid and the infusion line 
  
  
  
 
 
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10.15(b): HCP conclusions following evaluation of self-administration at site at 
Visit 13 (
 and Visit 19 (
) 
 
Visit 
13  
Yes/No  
Comments  
Visit 19  
Yes/No  
Comments  
Did the study participant administer 
rozanolixizumab at the correct infusion 
site?  
  
  
  
  
Did the study participant successfully 
administer rozanolixizumab 
subcutaneously?  
  
  
  
  
Did the study participant administer the 
intended dose of rozanolixizumab (based 
on the study participant’s body weight at 
Screening)? 
  
  
  
 
 
10.15 HCP evaluation checklist (a) and HCP conclusions (b) are also applicable following self-
administration at home at Visits 10 to 12 (
) and Visits 16 to 18 (
). 
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10.16 
Appendix 16: Participant’s preferred method of rozanolixizumab 
administration questions 
You have tried two methods of subcutaneous infusions (i.e., infusions under the skin) to 
administer the study drug: 
• Manual push: the drug is delivered by pressing the syringe plunger by hand. 
• Pump: the drug is delivered through a pump that is connected to the syringe which presses on 
the syringe plunger. 
You have also received the subcutaneous infusions both at the hospital and at your home. 
We would now like to ask you some questions about your preferences regarding the 
subcutaneous infusions. 
1. Which method of administering the subcutaneous infusions did you prefer? (Please select one 
answer) 
Manual push  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Pump  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
No preference  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
2. In general, who did you prefer administering the infusions? (Please select one answer) 
Myself  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
A nurse or other healthcare professional  
 
 
 
 
 
 
 
 
 
 
 
 
No preference  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
3. Where did you prefer to receive the subcutaneous infusions? (Please select one answer) 
At my home  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
At the hospital 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
No preference  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
4. During the Training Period when you were receiving the subcutaneous infusions at the 
hospital, who did you prefer administering the infusions? (Please select one answer) 
Myself  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
A nurse or other healthcare professional  
 
 
 
 
 
 
 
 
 
 
 
 
No preference  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Please record any other feedback you have about how the infusions were administered: 
 
 
 
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I have answered the questions alone  
 
 
 
 
 
 
 
 
 
 
 
 
 
I have answered the questions together with a family member (or other persons 
 
 
who have accompanied me during the subcutaneous infusions of the study drug) 
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10.17 
Appendix 17: Myasthenia Gravis Activities of Daily Living 
 
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10.18 
Appendix 18: Self-Injection Assessment Questionnaire 
 
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10.19 
Appendix 19: Tuberculosis Questionnaire 
 
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11 
REFERENCES 
Clinical Trial Regulation EU 536/2014. April 2014. 
Communication from the Commission - Detailed guidance on the collection, verification and 
presentation of adverse event/reaction reports arising from clinical trials on medicinal products 
for human use (‘CT-3’). 2011/C172/01. 
ISO 14155:2020 Clinical investigations of medical devices for human subjects – Good Clinical 
Practice. 
American Psychiatric Association (US). Diagnostic and Statistical Manual of Mental Disorders. 
5th ed. Arlington: American Psychiatric Association; 2013. 
Alcantara M, Sarpong E, Barnett C, Katzberg H, Bril V. Chronic immunoglobulin maintenance 
therapy in myasthenia gravis. Eur J Neurol. 2021;28(2):639-646. 
Bienvenu B, Cozon G, Mataix Y, et al. Rapid Push vs Pump-Infused Subcutaneous 
Immunoglobulin Treatment: a Randomized Crossover Study of Quality of Life in Primary 
Immunodeficiency Patients. J Clin Immunol. 2018;38(4):503-12. 
Cowan J, Bonagura VR, Lugar PL, et al. Safety and Tolerability of Manual Push Administration 
of Subcutaneous IgPro20 at High Infusion Rates in Patients with Primary Immunodeficiency: 
Findings from the Manual Push Administration Cohort of the HILO Study. J Clin Immunol. 
2021;41(1):66-75. 
James LP, Letzig L, Simpson PM, et al. Pharmacokinetics of Acetaminophen-Protein Adducts in 
Adults with Acetaminophen Overdose and Acute Liver Failure. Drug Metab Dispos. 
2009;37:1779-84. 
Jaretzki A 3rd, Barohn RJ, Ernstoff RM, Kaminski HJ, Keesey JC, Penn AS, et al. Myasthenia 
gravis: Recommendations for clinical research standards. Task Force of the Medical Scientific 
Advisory Board of the Myasthenia Gravis Foundation of America. Neurol. 2000;55:16-23. 
Jolles S, Sewell WA, Leighton C. Drug-induced aseptic meningitis: diagnosis and management. 
Drug Saf. 2000;22(3):215-26. 
Keininger D, Coteur G. Assessment of self-injection experience in patients with rheumatoid 
arthritis: psychometric validation of the Self-Injection Assessment Questionnaire (SIAQ). Health 
Qual Life Outcomes. 2011;13:9:2. 
Le Gal F, Gordien E, Affolabi D, et al. Quantification of Hepatitis Delta Virus RNA in Serum by 
Consensus Real-Time PCR Indicates Different Patterns of Virological Response to Interferon 
Therapy in Chronically Infected Patients. J Clin Microbiol. 2005;43(5):2363-9.  
Muppidi S. The myasthenia gravis-specific activities of daily living profile. Ann N Y Acad Sci. 
2012;1274:114-9. 
Muppidi S, Wolfe GI, Conaway M, Burns TM. MG composite and MG-QOL15 study group. 
MG-ADL: still a relevant outcome measure. Muscle Nerve. 2011;44(5):727-31. 
Roopenian DC, Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol. 
2007;7(9):715-25. 
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Sampson HA, Muñoz-Furlong A, Campbell RL, Adkinson NF Jr, Bock SA, Branum A, et al. 
Second symposium on the definition and management of anaphylaxis: summary report – Second 
National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network 
symposium. J Allergy Clin Immunol. 2006;117(2):391-7. 
Shapiro RS. Subcutaneous immunoglobulin: rapid push vs. infusion pump in pediatrics. Pediatr 
Allergy Immunol. 2013;24(1):49-53. 
Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis 
activities of daily living profile. Neurology. 1999 Apr 22;52(7):1487-9. 
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SPONSOR DECLARATION 
I confirm that I have carefully read and understand this protocol and agree to conduct this 
clinical study as outlined in this protocol, according to Clinical Trial Regulation EU 536/2014, 
and according to current Good Clinical Practice. 
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Approval Signatures
Name:
mg0020-protocol-amend-5
Version:
1. 0
Document Number:
CLIN-000243670
Title:
mg0020 Protocol Amendment 5
Approved Date:
01 Dec 2023
Document Approvals
Approval
Verdict: Approved
Name: 
Capacity: Clinical
Date of Signature: 01-Dec-2023 04:41:34 GMT+0000
Approval
Verdict: Approved
Name:
Capacity: Clinical
Date of Signature: 01-Dec-2023 08:24:38 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Subject Matter Expert
Date of Signature: 01-Dec-2023 09:56:56 GMT+0000
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SAP_001.pdf: 
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Statistical Analysis Plan 
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Page 1 of 63 
STATISTICAL ANALYSIS PLAN 
Study: MG0020 
Product: Rozanolixizumab 
AN OPEN-LABEL, CROSSOVER STUDY TO EVALUATE 
ROZANOLIXIZUMAB SELF-ADMINISTRATION BY STUDY 
PARTICIPANTS WITH GENERALIZED MYASTHENIA GRAVIS 
A Phase 3, open-label, crossover study to evaluate self-administration of rozanolixizumab by 
study participants with gMG 
Sponsor Name: UCB Biopharma SRL 
Legal Registered Address: Allée de la Recherche 60, 1070 Brussels, BELGIUM 
Regulatory Agency Identifier Number(s): 
Registry 
ID 
Eudra CT Number 
2022-003870-21 
IND Number 
132407 
Confidentiality Statement 
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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TABLE OF CONTENTS 
VERSION HISTORY ......................................................................................................................4 
Amendment 1.0 ................................................................................................................................4 
1 
INTRODUCTION .................................................................................................................16 
1.1 
Objectives and endpoints ...................................................................................................16 
1.2 
Study design .......................................................................................................................18 
2 
STATISTICAL HYPOTHESES ............................................................................................20 
2.1 
Multiplicity adjustment ......................................................................................................20 
3 
POPULATIONS FOR ANALYSIS .......................................................................................20 
4 
STATISTICAL ANALYSES ................................................................................................21 
4.1 
General considerations .......................................................................................................21 
4.1.1 
Analysis time points ..................................................................................................21 
4.1.1.1 
Relative day for listings ...................................................................................21 
4.1.1.2 
Analysis periods ..............................................................................................22 
4.1.2 
Definition of baseline values .....................................................................................22 
4.1.3 
Mapping of assessments performed at Early Withdrawal Visit ................................22 
4.1.4 
Treatment assignment and treatment groups .............................................................22 
4.1.5 
Multicenter studies .....................................................................................................23 
4.1.6 
Center pooling strategy ..............................................................................................23 
4.1.7 
Intercurrent event handling ........................................................................................23 
4.1.8 
Handling of missing or partially missing dates .........................................................23 
4.2 
Primary endpoint analysis ..................................................................................................25 
4.2.1 
Definition of endpoint(s) ...........................................................................................25 
4.2.2 
Main analytical approach ...........................................................................................25 
4.2.3 
Sensitivity analyses ....................................................................................................26 
4.2.4 
Supplementary analysis .............................................................................................26 
4.3 
Secondary endpoints analysis ............................................................................................26 
4.3.1 
Secondary endpoints ..................................................................................................26 
4.3.1.1 
Definition of endpoint(s) .................................................................................26 
4.3.2 
Main analytical approach ...........................................................................................27 
4.4 
Exploratory endpoints analysis ..........................................................................................27 
4.4.1 
Participant’s relative preference ................................................................................27 
4.4.2 
MG-ADL ...................................................................................................................28 
4.4.3 
Pharmacodynamic ......................................................................................................28 
4.4.4 
Immunogenicity .........................................................................................................29 
4.4.5 
SIAQ ..........................................................................................................................31 
4.4.6 
Self-administration at home .......................................................................................33 
4.4.7 
TEAEs leading to permanent withdrawal ..................................................................33 
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4.5 
Other safety analysis ..........................................................................................................33 
4.5.1 
Extent of exposure .....................................................................................................33 
4.5.2 
Adverse events ...........................................................................................................34 
4.5.3 
Additional safety assessments ...................................................................................35 
4.5.3.1 
Clinical laboratory evaluations ........................................................................35 
4.5.3.2 
Vital signs ........................................................................................................38 
4.5.3.3 
Electrocardiograms ..........................................................................................39 
4.5.3.4 
Other safety endpoint(s) ..................................................................................40 
4.6 
Other analyses ....................................................................................................................40 
4.6.1 
Subgroup analyses .....................................................................................................40 
4.6.2 
Specific analyses for PMDA .....................................................................................41 
4.7 
Interim analyses .................................................................................................................42 
4.8 
Changes to protocol-planned analyses ...............................................................................42 
4.9 
Data Monitoring Committee (DMC) or other review board ..............................................42 
5 
SAMPLE SIZE DETERMINATION ....................................................................................43 
6 
APPENDIX: SUPPORTING DOCUMENTATION .............................................................43 
6.1 
Appendix 1: List of Abbreviations ....................................................................................43 
6.2 
Appendix 2: Coding dictionaries .......................................................................................45 
6.3 
Appendix 3: Participant disposition ...................................................................................45 
6.4 
Appendix 4: Baseline characteristics and demographics ...................................................46 
6.5 
Appendix 5: Protocol deviations ........................................................................................47 
6.6 
Appendix 6: Medical history .............................................................................................48 
6.7 
Appendix 7: Prior / concomitant medications ...................................................................48 
6.7.1 
Categorization ............................................................................................................48 
6.7.2 
Prior and concomitant medications summaries .........................................................48 
6.8 
Appendix 8: Data derivation rules .....................................................................................49 
6.8.1 
Handling of repeated and unscheduled measurements ..............................................49 
6.9 
Appendix 9: Markedly abnormal criteria for Rozanolixizumab program .........................49 
6.10 
Appendix 10: Compliance .................................................................................................51 
6.11 
Appendix 11: Myasthenia Gravis-Activities of Daily Living ............................................51 
6.12 
Appendix 12: Impact of the coronavirus disease 2019 (COVID-19) on study data ..........52 
6.13 
Appendix 13: AEs of focus for Rozanolixizumab program ..............................................52 
6.14 
Appendix 14: Self-Injection Assessment Questionnaire ...................................................55 
6.15 
Appendix 15: Tuberculosis Questionnaire.........................................................................62 
7 
REFERENCES ......................................................................................................................63 
 
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VERSION HISTORY 
SAP  
Version 
Date 
Change 
Rationale 
1.0 
03 Apr 2023 
Not Applicable 
Original version 
Amendment 1.0 03 May 2024 
Described below 
 
 
Amendment 1.0 
Section # and Name 
Description of Change 
Brief Rationale 
Global 
Minor administrative, 
consistency, formatting and 
typographical changes have 
been made. 
Updated to provide clarity and 
be consistent with the remainder 
of the SAP. 
1 Introduction 
Added Protocol Amendment 4, 
07 Sep 2023 and Protocol 
Amendment 5, 30 Nov 2023. 
New protocol amendments were 
developed after the first version 
of the SAP. 
1.2 Study Design 
Defined that the Safety Follow-
up Period may be up to 7 weeks, 
as participants may move to 
commercially available 
rozanolixizumab at the 
discretion of the investigator,  
shortening the duration of this 
period. 
The option for participants to 
access commercially available 
rozanolixizumab was introduced 
in Protocol Amendment 5. 
Moving to a post-trial access 
program or commercially 
available rozanolixizumab may 
reduce the length of the Safety 
Follow-up Period. 
3 Populations for Analysis 
Full Analysis Set (FAS): All 
participants who are included in 
SS, were randomized and 
completed both Self-
administration Periods. Both 
Self-administration Periods will 
be considered as complete if 
self-administration occurred on 
Visits 13 and 19 in accordance 
with the randomization scheme. 
Specified the definition of FAS 
accepting only treatment 
assignments in accordance with 
randomization scheme. 
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Section # and Name 
Description of Change 
Brief Rationale 
4.1 General considerations 
Removed: The primary analysis 
will be performed at database 
lock after Last Participant Last 
Visit. 
In the case of missing visit 
information for MG-ADL or 
SIAQ, visit windowing will be 
applied based on the Protocol 
SOA. Else, visit windows will 
not be used. 
All analysis, not limited to 
primary analysis, will be 
performed at database lock after 
Last Participant Last Visit. 
 
Ensures that data performed 
within the window of a visit will 
be assigned to the related visit 
appropriately. 
4.1.1.2 Analysis Periods 
Self-administration Period 2: 
Starts one day after the end of 
Self-administration Period 1 and 
ends at the end of Visit 20 
assessments (EOT date). 
By the Protocol, all end of 
treatment (EOT) assessments at 
Visit 20 (Week 19) belong to 
treatment phase. 
4.1.3 
Mapping of assessments 
performed at Early Withdrawal 
Visit 
 
Added: If the mapped visit is the 
first visit in the next period, the 
visit will be assigned to the 
previous period. 
To ensure that mapped data is 
assigned to the correct period. 
4.1.4 
Treatment assignment 
and treatment groups 
 
Added: In the case that all  
 in each period were 
performed using the alternate 
method to the one randomized, 
the actual treatment sequence 
will reflect this. 
To ensure that data is assigned 
to the correct self-administration 
method. 
Table 1‒1: Calculation rules for 
duration of AEs 
Added footnote: Note: ‘--' 
represents a missing value 
 
Footnote added to clarify 
missing onset/outcome dates. 
4.2.2 Main analytical approach 
 
Updated the analyses to specify 
the mean proportion of success 
would be presented with its 90% 
CI calculated using the Wilson 
method (without continuity 
correction), rather than a 
generalized linear mixed model. 
 
Specified that the Wilson 
method would be used rather 
than Clopper-Pearson exact 
method. 
Specified the method of the 
calculation of CI for the mean 
proportion of success. 
 
Wilson method was selected 
rather than Clopper-Pearson 
exact method due to a larger 
number of participants able to 
perform self-administration 
using both methods, than 
planned in Sample Size Section 
5. Therefore, a method 
considering Normal 
approximation was deemed 
sufficient. 
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Section # and Name 
Description of Change 
Brief Rationale 
4.2.2 Main analytical approach 
 
In case at least one of the three 
questions related to the 
administration is missing 
(correct infusion site, 
subcutaneous, intended dose), 
the self-administration is 
considered unsuccessful. 
Imputation will not be 
performed. 
Specified that a missing 
response will not be imputed. 
4.2.3 Sensitivity analyses 
Sensitivity analyses by imputing 
a self-administration failure at 
Visit 13 or Visit 19 will not be 
performed. Any references to 
model fitting have been 
removed. 
Specified that a missing 
response will not be imputed 
4.2.4 Supplementary analyses 
 
Specified supplementary 
analysis will be performed on 
the FAS only. 
As a 2x2 contingency table is 
produced, the analysis should be 
performed on the FAS as these 
participants completed both self-
administration periods and 
successful self-administration 
was assessed at Visit 13 and 
Visit 19. 
4.3.1 Secondary endpoints 
 
Defined a TEAE. 
Updated the definitions of 
assignment of TEAEs to a Self-
administration period. 
 
Assignment of TEAEs to a Self-
administration period updated 
since the 8 weeks from final 
dose are now covered in the 
TEAE main definition text. 
Assignment to Self-
administration period 2 updated 
to match definition of analysis 
periods. 
 
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Section # and Name 
Description of Change 
Brief Rationale 
4.4.2 
MG-ADL 
Specified worsening in MG-
ADL score will be a >2 point 
increase from Baseline at any 
timepoint during the treatment 
period. Added that MG-ADL 
worsening at the last available 
measurement of MG-ADL 
before the EOS Visit (Visit 21) 
would be presented in this table 
and a 90% CI estimated using 
the Wilson method. 
 
Stated that if time of collection 
is missing for MG-ADL at Visit 
2, this will be considered as the 
Baseline assessment. 
 
Specified MG-ADL will be 
summarized at each visit by 
sequence only, not also split by 
self-administration method. 
 
Line plots of mean MG-ADL 
total score and mean change 
from baseline over time added. 
Spaghetti plots of MG-ADL 
total score and change from 
baseline over time also 
presented. 
 
Aligns with the definition of 
MG-ADL worsening in the 
Protocol. MG-ADL worsening 
at the last available timepoint 
before EOS will be presented 
and a 90% CI calculated as 
specified in the Protocol.  
 
If time of collection for MG-
ADL at Visit 2 is missing, this 
can be assumed as the Baseline 
assessment as MG-ADL is 
collected predose per Protocol. 
 
As the MG-ADL summary is 
split by sequence and visit, 
additionally splitting by self-
administration method is not 
required. 
 
Plots added to visualize MG-
ADL changes over time. 
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Section # and Name 
Description of Change 
Brief Rationale 
4.4.3 
Pharmacodynamic 
 
For the RSS, observed values 
and percentage change from 
baseline will be summarized by 
sequence. 
Specified that if rescue therapy 
is taken, IgG values collected 8 
weeks after the start of rescue 
medication will not be included 
in summary analysis. 
Line plots of median IgG 
concentrations and percentage 
change from baseline over time 
added. Spaghetti plots of IgG 
concentrations and percentage 
change from baseline over time 
will also be presented. 
A line plot presenting mean 
change from baseline in MG-
ADL and median percentage 
change in IgG by sequence will 
be produced. 
Plots of individual percentage 
change from baseline in IgG 
concentrations and change from 
baseline in MG-ADL total score 
added. 
Specified IgG laboratory 
assessments are performed 
predose. 
 
To provide a clearer picture of 
participants’ IgG levels 
throughout the study. 
 
IgG values may be affected by 
rescue therapy use and influence 
analyses.  
  
Plots added to visualize IgG 
changes over time. 
 
 
4.4.4 
Immunogenicity 
 
ADA summaries will be 
presented by sequence and 
overall 
To provide a clearer picture of 
participants’ ADA status 
throughout the study. 
 
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Section # and Name 
Description of Change 
Brief Rationale 
4.4.5 SIAQ 
Specified PRE-SIAQ is obtained 
predose at the Baseline Visit per 
Protocol. 
An additional table for POST-
SIAQ individual item scores 
will be produced. A horizontal 
bar chart displaying individual 
domain scores for PRE-SIAQ 
and POST-SIAQ will be 
produced. 
Added that in the case of 
repeated measurements of PRE-
SIAQ at the Baseline Visit, the 
earliest measurement will be 
considered. 
 
Selecting the earliest PRE-SIAQ 
assessment ensures that the 
questionnaire was performed 
prior to administration of IMP at 
the Baseline Visit. 
4.4.6 Self-administration at 
home 
 
The number and proportion of 
participants deemed a treatment 
success at each of the self-
administration visits will be 
tabulated. 
Proportion of success with 90% 
CI for self-administration of 
syringe driver and manual push 
at home will not be estimated 
using a generalized linear 
model. 
Including all self-administration 
visits in the treatment success 
summary table. 
 
For 3 assessments of self-
administration at home only 
descriptive statistics will be 
used. 
4.4.7 TEAEs leading to 
permanent withdrawal 
 
Summary table will be presented 
for SS and RSS, by SOC, HLT 
and PT. 
Removed the estimation of 90% 
CI with a generalized linear 
mixed model. 
 
90% CI for occurrence of these 
TEAEs will not be calculated as 
explained in Section 4.8. 
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Section # and Name 
Description of Change 
Brief Rationale 
4.5.1 
Extent of exposure 
 
Duration of infusion will be 
summarized for the RSS by 
method and timepoint. 
Defined the equation to 
calculate duration of infusion. 
The study medication duration 
will also be described by 
category: ≥1 day, ≥ 36 days, ≥ 
78 days, and ≥ 120 days. 
Updated study medication 
duration category definitions 
from weeks to days. 
Included an analysis to assess 
study medication duration of 
participant in the RSS. 
 
Specified how duration of 
infusion should be calculated. 
 
Duration categories updated to 
days since duration is calculated 
in days 
4.5.2 Adverse events 
 
Safety analysis will present 
TEAEs using SOC, HLT and 
PT. 
 
The following summaries were 
added: 
• 
Incidence of TEAEs by 
Intensity 
• 
Incidence of TEAEs by 
Relationship 
• 
Incidence of drug-related 
TEAEs 
• 
Incidence of severe TEAEs 
 
The following summary was 
removed: 
• 
Incidence of Treatment-
Emergent Serious AEs – 
Participant Numbers 
 
Specified drug-related is based 
on the Investigator assessment. 
 
Incidence of TEAEs of Focus 
will also be presented for the 
RSS, in addition to the SS. 
Added coding of AEs with HLT 
term. All MG studies use coding 
of AEs with SOC, HLT and PT 
terms. 
 
Included additional AE 
summaries. 
 
Participant Numbers summaries 
removed as individual 
participant AE data can be 
found in listings. 
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Section # and Name 
Description of Change 
Brief Rationale 
4.5.3.1.1 
Laboratory values 
over time 
 
Updated the definitions of 
assignment of TE laboratory 
data to a period. 
 
Added: Central laboratory data 
will be used for the summary 
tables and figures. Local 
laboratory data will be listed 
only. 
 
Specified raw calcium values 
will be used in shift tables and 
corrected calcium values used 
for markedly abnormal 
assessments. 
 
Updated assignment to a period 
to align with the Analysis 
Periods definition. 
 
Central and local laboratory data 
have differing reference ranges 
thus local laboratory data is to 
be listed only. 
 
4.5.3.2 Vital signs 
 
Added: In case of early 
withdrawal, the visit will be 
mapped to the pre-dose 
timepoint of the next scheduled 
visit. 
Added the assignment of TEMA 
assessments to a period. 
Updated in order to present a 
timepoint for early withdrawal 
data. 
 
TEMA vital signs will be 
assigned to a period in outputs. 
4.5.3.3 Electrocardiograms 
 
Defined Treatment-emergent. 
Abnormal ECG findings will be 
listed. 
Defined treatment-emergent and 
specified ECG abnormalities 
will be listed. 
4.5.3.4.2 
Suicidal risk 
monitoring 
 
Actual attempts will be listed. 
Specified that a listing will be 
provided for actual attempts. 
 
4.6 Other analyses 
 
Deleted "No other analysis is 
planned”. 
Updated since PMDA specific 
analyses have been introduced. 
4.6.1 
Subgroup analyses 
No subgroup analyses are 
planned. 
The proposed subgroup analyses 
were deemed inappropriate for 
the study design and would be 
challenging to interpret, due to 
the exposure (amount and time 
frame) not being comparable 
between the 2 subgroups. 
4.6.2 Specific analyses for 
PMDA 
 
Section added, including a list of 
summary tables to include 
Japanese participants only. 
Specific analyses for the 
Japanese population are required 
by the PMDA. 
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Section # and Name 
Description of Change 
Brief Rationale 
4.8 Changes to protocol-
planned analyses 
 
Removed: Demographics will 
be displayed overall for the SS 
as per Section 6.4 although in 
the Clinical Study Protocol 
(CSP) Section 9.2, it was 
specified by sequence and 
overall. Indeed, demographics 
will be reported for the Safety 
Set and not for the Randomized 
Safety Set. 
Added: The Full Analysis Set 
(FAS) is specified in the SAP 
Section 3 as all participants who 
are included in Safety Set, were 
randomized, and completed both 
self-administration periods. 
Both self-administration periods 
will be considered as complete 
if self-administration occurred 
on Visits 13 and 19 in 
accordance with the 
randomization scheme. In the 
CSP it was defined as all 
randomized study participants 
who received at least 1 dose of 
IMP (partial or full) and 
completed both self-
administration periods. 
 
Added: Confidence intervals for 
the occurrence of TEAEs 
leading to permanent 
withdrawal of rozanolixizumab 
will not be estimated as 
presented in the Protocol. 
Text updated to specify 
demographics will be presented 
by sequence and overall, as per 
Protocol Section 9.2 for the 
RSS, however, will only be 
presented for overall for the SS.  
 
Rationale for changes added to 
this section are presented in 
Section 4.8. 
Table 1‒1: Expected 90% 
confidence intervals considering 
different success rates and 30 
study participants completing 
both Treatment Periods 
Success rate table added. 
 
For consistency with the section 
in the Protocol. 
6.1 Appendix 1: List of 
Abbreviations 
 
HLT,INR and TEMA added to 
list of abbreviations. 
Abbreviations used throughout 
the SAP. 
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Section # and Name 
Description of Change 
Brief Rationale 
Appendix 3: Participant 
disposition 
 
Reasons for screen failures will 
not be summarized by COVID-
19 timepoint. 
 
Disposition of analysis sets will 
be summarized using the SS 
rather than ES. 
 
Added: Completed Study (A 
study participant is considered 
to have 'Completed Study' if 
'Completed Study Participant' 
has been recorded in CRF form 
'Study Termination Enrolled') 
 
Added listings for study 
participant disposition including 
participant analysis sets, re-
screened participants, visit 
dates, self-administration 
training visits, and self-
administration eligibility 
criteria. 
 
Since no participant had a 
Baseline visit prior to the 
COVID-19 pandemic cut-off 
date of 05-May-2023, 
summaries split by timepoint of 
COVID-19 pandemic will not be 
presented. 
 
Disposition of analysis sets will 
be summarized using the SS as 
disposition of screen failures is 
presented in another table, and 
so presenting for the ES is not 
necessary. 
 
Specified the definition of 
Completed Study in accordance 
with Protocol Amendment 5. 
 
Added additional listings to 
display study participant 
disposition data. 
6.4 Appendix 4: Baseline 
characteristics and 
demographics 
 
COVID-19 pandemic cut-off 
end date of 05-May-2023 added. 
Myasthenic crisis in the past 
added to list of baseline 
characteristics. 
Specified demographics and 
baseline characteristics will also 
be presented by sequence and 
overall, for the RSS. 
 
Update in COVID-19 pandemic 
end date since last SAP. 
 
 
6.5 Appendix 5: Protocol 
deviations 
 
Summary tables for IPDs by 
relationship to COVID-19 and 
IPDs by COVID-19 pandemic 
timepoint will not be presented. 
Since no participant had a 
Baseline visit prior to the 
COVID-19 pandemic cut-off 
date of 05-May-2023, 
summaries split by relationship 
or timepoint of COVID-19 
pandemic will not be presented. 
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Section # and Name 
Description of Change 
Brief Rationale 
6.7.1 
Categorization 
 
Updated the definitions of 
assignment of prior/concomitant 
medications. 
Removed: The medications 
allocated to Self-administration 
Period 1 or Self-administration 
Period 2 will be included in the 
summary output. Other 
medications will only be listed. 
Updated to align with the 
Analysis Periods definition. 
A summary table will also be 
produced for the SS included 
Training and Follow-up period. 
6.7.2 
Prior and concomitant 
medications summaries 
Specified the list of rescue 
medications can be found in the 
Protocol and are identified in the 
CRF. A summary table will be 
presented for the SS. 
Rescue medication will be 
analyzed for RLZ Total. 
6.8.1 Handling of repeated and 
unscheduled measurements 
The following general rules will 
apply to all repeated and 
unscheduled measurements, 
unless otherwise specified: 
Repeated PRE-SIAQ 
measurements deviate from the 
general rules in this section. 
Handling of this data has been 
specified elsewhere in the SAP. 
Table 6-2: Chemistry: 
 
Creatinine: >3.0 x ULN or >3.0 
x baseline 
Potassium Low: <3.0 mmol/L 
Updated creatinine and 
potassium abnormality criteria. 
Table 6-3: Vital Signs: 
Removed “Body weight” from 
table. 
Body weight is captured only at 
screening 
Table 6-4: Electrocardiogram: 
Removed “Treatment emergent 
value” from table. 
The table specifies abnormality 
criteria, and all values are 
considered as treatment 
emergent. 
6.12 
Appendix 12: Impact of 
the coronavirus disease 2019 
(COVID-19) on study data 
 
Additional summary analyses 
based on COVID-19 timepoint 
will not be presented. 
Since no participant had a 
Baseline visit prior to the 
COVID-19 pandemic cut-off 
date of 05-May-2023, 
summaries split by timepoint of 
COVID-19 pandemic will not be 
presented. 
6.13 
Appendix 13: AEs of 
focus for Rozanolixizumab 
program 
Aseptic updated to Possible 
Aseptic, and removal of (Note: 
not applicable for MG0020) 
Aligns with rozanolixizumab 
PSAP Amendment 2. 
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Section # and Name 
Description of Change 
Brief Rationale 
7 References 
 
[2] Brown, Lawrence D.; Cai, 
T. Tony; DasGupta, 
Anirban. Interval Estimation 
for a Binomial Proportion. 
Statist. Sci. 16 (2001), no. 2, 
101–133. 
doi:10.1214/ss/1009213286. 
Reference added for calculating 
of the Wilson confidence 
interval. 
 
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1 
INTRODUCTION 
The purpose of this SAP is to provide the information that is necessary to perform statistical 
analyses for the final analysis of study MG0020. It also defines the summary tables, figures and 
listings (TFLs) to be included in the final Clinical Study Report (CSR) according to the protocol. 
TFLs specifications are contained in a separate document.  
This SAP is based upon, and assumes familiarity with the following documents: 
• Protocol Amendment 3, 09 Dec 2022 
• Protocol Amendment 4, 07 Sep 2023 
• Protocol Amendment 5, 30 Nov 2023 
If a future protocol amendment necessitates a substantial change to the statistical analysis of the 
study data, this SAP may be amended accordingly. Changes to the analysis from the protocol are 
documented in Section 4.8. The content of this SAP is compatible with the International Council 
for Harmonisation (ICH)/Food and Drug Administration (FDA) E9 Guidance documents. 
 
1.1 
Objectives and endpoints 
Table 1‒1: 
Objectives and Endpoints 
Objectives 
Endpoints 
Primary 
Primary objective:  
• To evaluate the ability of study 
participants with gMG to 
successfully self-administer 
rozanolixizumab after training in 
the self-administration technique 
using the syringe driver and 
manual push methods 
Primary endpoint: 
• Successful self-administration of rozanolixizumab 
(with correct use of syringe driver and manual push, 
respectively) during the Self-administration Period 
at Visit 13 (
; last dose of Self-
administration Period 1) and Visit 19 (
; 
last dose of Self-administration Period 2). 
Successful self-administration is defined by the 
participant (i) choosing the correct infusion site, (ii) 
administering subcutaneous (SC), and (iii) delivering 
the intended dose. 
Secondary 
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Objectives 
Endpoints 
Secondary objective:  
• To evaluate the safety of SC self-
administration of 
rozanolixizumab. 
Secondary endpoints: 
• Occurrence of TEAEs after syringe driver or 
manual push self-administration from Visit 2 
(
 up to the End of Study Visit (Visit 21 
[Week 26]). 
• Occurrence of local site reactions up to 24 hours 
after each administration during the Training Period 
and Self-administration Periods. 
• Occurrence of medication errors associated with 
adverse reactions during the 2 Self-administration 
Periods of the study. 
Other 
Other objectives:  
• To evaluate the study participant’s 
preferred method of 
rozanolixizumab administration 
 
Other endpoints: 
• Participant’s relative preference for: 
− Subcutaneous infusions performed by an health 
care provider (HCP) versus self-administration 
− The manual push method versus the use of a 
syringe driver for self-administration 
using the participant’s preferred method of 
administration questions at Visit 19 (
). 
• To evaluate symptom changes in 
study participants with gMG 
• Myasthenia Gravis Activities of Daily Living score 
change from Baseline during the study. 
• To assess the pharmacodynamics 
of rozanolixizumab 
• Total IgG level over time during the Training 
Period and Self-administration Periods. 
• To evaluate the immunogenicity 
of rozanolixizumab following SC 
self-administration 
• Anti-rozanolixizumab antibodies (status and titer at 
trough during both Self-administration Periods).  
• To assess the study participant’s 
experience with self-
administration of SC infusions at 
home 
• 
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Objectives 
Endpoints 
• To assess the ability of study 
participants with gMG to 
successfully self-administer 
rozanolixizumab at home after 
training in the self-administration 
technique using the syringe driver 
and manual push methods 
• Successful self-administration of rozanolixizumab 
via manual push method or using a syringe driver at 
each of the 3 home self-administration visits during 
Self-administration Period 1 or 2, respectively. 
Successful self-administration via manual push or 
syringe driver is defined by the participant (i) 
choosing the correct infusion site, (ii) administering 
SC, and (iii) delivering the intended dose, as 
reported by the study participant to the site staff 
following each of the 3 home self-administrations. 
• To assess the safety and 
tolerability of rozanolixizumab in 
study participants with gMG 
• Occurrence of TEAEs leading to permanent 
withdrawal of rozanolixizumab during the study. 
 
1.2 
Study design 
MG0020 is a Phase 3, open-label, randomized, 2-period, 2-sequence crossover study planned to 
be conducted at multiple sites in Europe, Japan, and North America. In MG0020, the safe and 
effective self-administration of rozanolixizumab using the syringe driver and manual push 
administration methods by study participants with gMG will be evaluated using a crossover 
design over 
 (Figure 1‒1). 
Study participants in Europe and North America are proposed to receive 
 fixed doses of 
rozanolixizumab for 
 as described below: 
• Body weight ≥35kg to <50kg: dose to be administered 
 
• Body weight ≥50kg: dose to be administered 
 
Study participants in Japan are proposed to receive 
 fixed unit doses of 
 
rozanolixizumab across body weight tiers for 
 as described below: 
• Body weight ≥35kg to <50kg: dose to be administered 
 
• Body weight ≥50kg to <70kg: dose to be administered 
 
• Body weight ≥70kg to <100kg: dose to be administered 
 
• Body weight ≥100kg: dose to be administered 
 
Rozanolixizumab-naïve study participants and study participants previously exposed to 
rozanolixizumab will be included in the study. 
Approximately 40 participants will be screened to achieve approximately 30 randomly assigned 
and evaluable participants for an estimated total of 15 evaluable participants per sequence. See 
Section 5 for the determination of sample size. 
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The total duration for each study participant is up to 29 weeks, including a Screening Period of 
up to 4 weeks, an 
 Treatment Period, and an up to 7-week Safety Follow-up Period (ie, 
End of Study Visit is 8 weeks after the last dose of rozanolixizumab in the Treatment Period, or 
earlier, if the study participant moves on to a post-trial access program or to commercially 
available rozanolixizumab during the Safety Follow-up Period). 
The 
 Treatment Period of MG0020 starts with standardized 
 trainings over the 
course of a 
 Training Period when the study participant is trained on both the manual push 
and the syringe driver administration methods. During the Training Period, the study participant 
will receive 
 doses of rozanolixizumab SC and should practice both methods of 
administration at the study site. To act as a reference for the preference question (HCP vs self-
administration) and to demonstrate subcutaneous infusions of rozanolixizumab, the first 
administration will be imperatively performed by the HCP. For all remaining training visits, the 
study participant should perform as many self-administrations as possible (under HCP 
supervision) depending on their training status. Self-administration under HCP supervision or 
administration by HCP will be documented in the case report form (CRF). At Visit 8 (
), 
after the completion of the Training Period and following the investigator’s confirmation of the 
eligibility to perform self-administration, the study participant will be randomized 1:1 to either 
the syringe driver or to the manual push administration method. After completing Self-
administration Period 1 (at Visit 13 [
]), the study participant will crossover to the 
alternative administration method, entering Self-administration Period 2 (Visit 14 [
]). A 
final release of the study participant for home self-administration will be based on a successful 
demonstration of self-administration using the assigned mode of administration (manual push or 
syringe driver use) at Visit 9 (
 and Visit 15 (
), respectively. Study participants 
who cannot be confirmed eligible for self-administration will not perform self-administration and 
can continue to be treated on-site per protocol based on investigator decision. Additionally, if a 
study participant becomes no longer eligible for self-administration during the Self-
administration Periods, he/she can continue to be treated on-site per protocol based on 
investigator decision. Adverse events, concomitant medication, vital signs, ECG, suicidality, 
blood and urine samples for safety laboratory tests, pregnancy tests and IgG will continue to be 
collected from these study participants until End of Study Visit/Early Withdrawal Visit. 
During both Self-administration Period 1 and Self-administration Period 2, self-administration 
will be performed at the site for 3 visits and at home (ie, unsupervised/not in the presence of an 
HCP) for 3 consecutive visits. 
After the Self-administration Periods, study participants will enter the 7-week Safety Follow-up 
Period. 
Following participation in MG0020, study participants who have completed the study (End of 
Study Visit performed) will have the option of a post-trial access to rozanolixizumab, if available 
according to local guidance, or to commercially available rozanolixizumab, at the discretion of 
the investigator. 
The schedule of activities can be found in the protocol. The infusions are planned 
 from 
Visit 2 (
 to Visit 7 (
 for the Training Period, from Visit 8 (
 to Visit 13 
(
) for the Self-administration Period 1 and from Visit 14 
 to Visit 19 (
 
) for the Self-administration Period 2. 
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Figure 1‒1: Study Schema 
 
HCP=health care professional; MP=manual push; SD=syringe driver; SFU=safety follow-up 
 
2 
STATISTICAL HYPOTHESES 
This is an estimation study design with no formal statistical hypothesis testing. 
2.1 
Multiplicity adjustment 
As there is no statistical hypothesis testing in this study, there will be no account for multiplicity. 
3 
POPULATIONS FOR ANALYSIS 
• Enrolled Set (ES): All study participants who have signed the informed consent form. 
• Safety Set (SS): All study participants who received at least 1 dose of investigational 
medicinal product (IMP) (partial or full). Analysis of this set will be according to the 
administration method the participants actually used and will be used for analyses of safety 
and demography outcomes as required. 
• Randomized Safety Set (RSS): All participants who are included in SS and were randomized. 
Analysis of this set will be according to the administration method the participants actually 
used and will be used for analyses of safety, demography, and efficacy/outcomes. 
• Full Analysis Set (FAS): All participants who are included in SS, were randomized and 
completed both Self-administration Periods. Both Self-administration Periods will be 
considered as complete if self-administration occurred on Visits 13 and 19 in accordance 
with the randomization scheme. 
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4 
STATISTICAL ANALYSES 
4.1 
General considerations 
Statistical analysis and generation of tables, figures, participant data listings, and statistical 
output will be performed using SAS Version 9.4 or higher. All tables and listings will use 
Courier New font size 9. 
Descriptive statistics will be displayed to provide an overview of the study results. For 
categorical parameters, the number and percentage of participants in each category will be 
presented. The denominator for percentages will be based on the number of participants 
appropriate for the purpose of analysis. Unless otherwise noted, all percentages will be displayed 
to 1 decimal place. No percentage will be displayed for zero counts, and no decimal will be 
presented when the percentage is 100%. For continuous parameters, descriptive statistics will 
include number of participants with available measurements (n), mean, standard deviation (SD), 
median, minimum, and maximum. If not otherwise specified, summary statistics will be 
displayed by sequence and overall for demographics, and by period and by self-administration 
method for period-specific assessments. 
Decimal places for descriptive statistics will always apply the following rules: 
• “n” will be an integer. 
• Mean, SD, and median will use 1 additional decimal place compared to the original data. 
• Minimum and maximum will have the same number of decimal places as the original value. 
The Baseline value is defined as the last nonmissing measurement before the first administration 
(prior to the Training Visits) at Visit 2 (
 if not otherwise specified. Scheduled or 
unscheduled measurements can be used as the Baseline value. 
In case of missing visit information for Myasthenia Gravis Activities of Daily Living (MG-ADL) 
and Self-Injection Assessment Questionnaire (SIAQ) data, visit windowing will be applied based 
on the Protocol Schedule of Activities (Section 1.3). Otherwise, visit windows will not be used. 
A complete set of data listings containing all documented data and all calculated data (eg, change 
from Baseline) will be generated. They will be ordered by sequence, participant number, period, 
and timepoints as applicable. 
4.1.1 
Analysis time points 
4.1.1.1 
Relative day for listings 
Relative day will be calculated depending on the position of the day compared to first and last 
doses of study drug: 
• For days on or after the day of first dose of study drug and prior to or on the day of last study 
drug dose: 
Relative Day = [(Event Date – Date of First Dose)+1] 
The day of first dose will be Day 1. 
• For days prior to the first dose of study drug: 
Relative Day = [(Event Date – Date of First Dose)] 
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The day prior to first dose will be Day -1. 
• For days after the last dose of study drug: 
Relative Day = + [(Event Date – Date of Last Infusion)] 
The day after the last dose will be Day +1. 
Relative day will not be calculated for partial dates. 
4.1.1.2 
Analysis periods 
The following study periods are defined for the classification by study period: 
• Screening Phase: Prior to the date of first dose of study drug 
• Treatment Phase: 
− Training Period: From the first dose (Visit 2) of study drug to the first dose in Self-
administration Period 1 (Visit 8) minus one day 
− Self-administration Period 1: Starts one day after the end of Training Period and ends 
with the first dose in Self-administration Period 2 (Visit 14) minus one day 
− Self-administration Period 2: Starts one day after the end of Self-administration Period 1 
and ends at the end of Visit 20 assessments (EOT date). 
• Follow-up Phase: Starts one day after the end of Self-administration Period 2 and ends after 
the safety follow up assessments of Visit 21. 
4.1.2 
Definition of baseline values 
The Baseline value is defined as the last nonmissing measurement before the first administration 
of IMP (prior to the Training Visits) at Visit 2 (
 if not otherwise specified. 
4.1.3 
Mapping of assessments performed at Early Withdrawal Visit 
Efficacy and safety assessments at Early Withdrawal (EW) will be assigned to the next 
scheduled visit (following the last scheduled visit that the study participant completed prior to 
withdrawal) where each assessment is evaluated as per protocol. This approach means that there 
is a chance that EW data will be mapped to different visits according to the schedule of 
assessments. If the mapped visit is the first visit in the next period, the visit will be assigned to 
the previous period. 
4.1.4 
Treatment assignment and treatment groups 
For the analyses using SS, Rozanolixizumab (RLZ) Total will be displayed.  
For some analyses using RSS, the actual sequence of treatment will be reported. Two sequences 
will be displayed: RLZ syringe driver – RLZ manual push and RLZ manual push – RLZ syringe 
driver as well as the column RLZ Total. In the case that all 
 in each period were 
performed using the alternate method to the one randomized, the actual treatment sequence will 
reflect this.  
For other analyses using RSS and focusing on period specific assessments, each self-
administration period and overall (Self-administration Period 1 and Period 2) will be split into 
two columns: RLZ syringe driver and RLZ manual push. For the AEs reporting, an RLZ Total 
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column will be added within the overall column to report the AEs occurring at least once in the 
self-administration period. 
4.1.5 
Multicenter studies 
Individual center results will not be displayed. 
4.1.6 
Center pooling strategy 
The data from all sites will be pooled for analyses purposes. 
4.1.7 
Intercurrent event handling 
As per protocol, if a study participant becomes no longer eligible for self-administration during 
the Self-administration Periods, he/she can continue to be treated on-site per protocol based on 
investigator decision. Adverse events, concomitant medication, vital signs, ECG, suicidality, 
blood and urine samples for safety laboratory tests, pregnancy tests, and IgG will continue to be 
collected from these study participants until End of Study Visit/Early Withdrawal Visit. 
Stopping self-administration during the Self-administration Periods will be considered as an 
intercurrent event in the analysis. 
For the primary and secondary endpoints focusing on the self-administration, all data collected 
after stopping the self-administration will not be included in the analysis of the primary and 
secondary endpoint. 
For the safety analysis, all data collected after stopping the self-administration will be included 
in the analysis (treatment policy strategy). It will be assumed that the planned use of manual 
push or syringe driver will follow the initial schedule. The following endpoints will also be 
analysed including the data collected after stopping Self-administration: 
• Total IgG level over time during the Training Period and Self-administration Periods. 
• Occurrence of TEAEs leading to permanent withdrawal of rozanolixizumab during the study. 
 
4.1.8 
Handling of missing or partially missing dates 
Partially or completely missing dates may be imputed for the following reasons: 
• Classification of AEs as TEAEs; 
• Classification of TEAEs into the study periods. 
• Classification of medications as prior, or concomitant medications;  
• Durations of AEs. 
Imputed dates will not be shown in listings.  All dates will be displayed as reported in the 
database. 
The following rules will be applied for partially or completely missing start dates: 
• If year, month and day are all missing then assign the date of first dose of IMP. If an imputed 
start date is after the specified end date, then assign January 01 of the year of the end date, or 
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the date of screening if this is later (if the latter imputation results in an end date that is 
earlier than the start date, then use January 01). 
• If month and day are missing, and year is: 
− the same as the year of the first dose of IMP then assign the month-day of first dose of 
IMP. If the imputed start date is after the specified end date, then assign January 01, or 
the month-day of screening date if this is later (if the latter imputation results in an end 
date that is earlier than the start date, then assign January 01); 
− not the same as the year of the first dose of IMP then assign January 01. 
• If only day is missing, and month-year is: 
− the same as the month-year of the first dose of IMP then assign the day of first dose of 
IMP. If the imputed start date is after the specified end date, then assign first day of the 
month, or the day of screening date if this is later (if the latter imputation results in an end 
date that is earlier than the start date, then assign first day of the month); 
− not the same as the month-year of the first dose of IMP then assign the first day of the 
month. 
The following rules will be applied for partially or completely missing stop dates: 
• If only the month and year are specified, then use the last day of the month. If an imputed 
stop date is after last contact date and last contact date is before the data cut-off date, then 
assign last contact date as the stop date. If an imputed stop date is after last contact date and 
last contact date is after the data cutoff date, then assign data cutoff date as the stop date. 
• If only the year is specified, then use December 31 of the known year. If an imputed stop 
date is after last contact date and last contact date is before the data cutoff date, then assign 
last contact date as the stop date. If an imputed stop date is after last contact date and last 
contact date is after the data cutoff date, then assign data cut-off date as the stop date. 
• If the stop date is completely unknown, then use discharge date or data cut-off date. 
Discharge date refers to the date of the end of study visit for completed participants or the 
date of discontinuation for participants that were withdrawn. For any AEs with known start 
date after the date of discontinuation, the date of last contact will be used as the discharge 
date. For participants still ongoing in the study at the time of the data cut-off, and for whom 
no discharge date is available, the date of the data cut-off will be used instead of the 
discharge date. 
Any medication with a start date on the first dosing date will be assumed to be concomitant. 
Each TEAE should be classified into exactly one study period for the classification of TEAEs 
into study periods except if doubt remains after imputing the dates. In that case, an AE will be 
considered as TEAE in each study period. 
Imputed AE dates will be used for the calculation of duration of AEs as described in Table 4‒1. 
 
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Table 4‒1: 
Calculation rules for duration of AEs 
Data 
availability 
Onset date Outcome 
date 
Calculation rules 
Complete data 
D1 
D2 
Duration = D2 – D1 + 1  d 
Start date 
partially or 
completely 
missing 
-- 
D2 
Duration ≤ D2 – D0 + 1 d 
Notes: D0 is imputed start date per above rules.  
End date 
partially or 
completely 
missing 
D1 
-- 
For ongoing AE: 
Duration ≥ D3 – D1 d 
For resolved AE: 
Duration ≤ D3 – D1 d 
Notes: D3 is imputed end date per above rules.  
Start and end 
date partially or 
completely 
missing 
-- 
-- 
For ongoing AE: 
Duration ≥ D3 – D0 d 
For resolved AE: 
Duration ≤ D3 – D0 d 
Notes: D0 is imputed start date and D3 is imputed end 
date per above rules. 
Note: ‘--' represents a missing value 
 
4.2 
Primary endpoint analysis 
4.2.1 
Definition of endpoint(s) 
The primary outcome variable is the proportion of study participants able to successfully self-
administer rozanolixizumab using both syringe driver and the manual push method, respectively, 
during the Self-administration Periods. Successful self-administration includes a correct SC 
infusion with the intended dose at the correct infusion site by the participant. Treatment success 
will be defined as a successful self-administration at the last dose of self-administration using the 
syringe driver (Visit 13 [
] for Sequence 1 and Visit 19 [
] for Sequence 2) or via 
manual push (Visit 19 [(
] for Sequence 1 and Visit 13 [
] for Sequence 2). 
4.2.2 
Main analytical approach 
The number and proportion of participants deemed a treatment success will be tabulated 
separately by period and overall for each self-administration method for the FAS.  
The proportion of success together with its 90% CI will be estimated for each self-administration 
with syringe driver and manual push overall using the Wilson method (without continuity 
correction) [2]. The Wilson method was selected rather than the Clopper Pearson exact method 
due to a larger number of participants able to perform self-administration using both methods 
than planned in Section 5. 
In case at least one of the three questions related to the administration is missing (correct 
infusion site, subcutaneous, intended dose), the self-administration is considered unsuccessful. 
The self-administration data will be listed for the SS including the training information. 
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4.2.3 
Sensitivity analyses 
A sensitivity analysis will be performed using the same analytical approach as for the primary 
endpoint, but reporting the data collected for RSS. 
4.2.4 
Supplementary analysis 
A within-participant assessment of treatment success (Y/N) with self-administration with the 
syringe driver or manual push will be tabulated through the use of a 2x2 contingency table 
presenting the number and proportion of participants in each category for the FAS. Successful 
self-administration includes a correct SC infusion with the intended dose at the correct infusion 
site. The last administered dose in each of the Self-administration Periods will be considered. 
The tabulation will be presented by sequence and  overall. 
4.3 
Secondary endpoints analysis 
4.3.1 
Secondary endpoints 
4.3.1.1 
Definition of endpoint(s) 
The secondary endpoints are: 
• Occurrence of TEAEs after syringe driver or manual push self-administration from Visit 2 
(
 up to the End of Study Visit (Visit 21 [Week 26]). 
• Occurrence of local site reactions up to 24 hours after each administration during the 
Training Period and Self-administration Periods. 
• Occurrence of medication errors associated with adverse reactions during the 2 Self-
administration Periods of the study. 
A TEAE is defined as an AE starting on or after the date of first administration of 
rozanolixizumab in the study, up to and including 8 weeks (56 days) after the final dose.  
A TEAE will be assigned to the Training period if the start date of the event is on or after the 
date of the first administration of rozanolixizumab during the Training Period and before the date 
of first administration of rozanolixizumab in Self-administration Period 1. 
A TEAE will be assigned to the Self-administration Period 1 if the start date of the event is on or 
after the date of the first administration of rozanolixizumab in Self-administration Period 1 and 
before the date of the first administration of rozanolixizumab in Self-administration Period 2. 
A TEAE will be assigned to the Self-administration Period 2 if the start date of the event is on or 
after the date of the first administration of rozanolixizumab in Self-administration Period 2 and 
up to the end of Visit 20 assessments (EOT date). 
Where dates are missing or partially missing, AEs will be assumed to be treatment-emergent 
unless evidence exists that does not allow the AE to be treatment-emergent. Handling of missing 
dates for classification of AEs as TEAEs is described in Section 4.1.8.  
The local site reactions up to 24 hours after each administration are defined as AEs reported as 
local site reactions as per CRF within one day after rozanolixizumab administration. 
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Medication errors associated with adverse reactions are defined as the AEs whose preferred 
terms belong to the Standardised MedDRA Queries (SMQ) “Medication errors” and leading to 
adverse reactions. 
4.3.2 
Main analytical approach 
The number and proportion of participants experiencing TEAEs during the Training Period will 
be summarized overall for the SS. The number and proportion of participants experiencing 
TEAEs during the Self-administration Periods will be summarized by period and overall for each 
self-administration method for the RSS. The number and proportion of participants experiencing 
TEAEs during the Self-administration Periods whatever the self-administration method will also 
be reported. 
The number and proportion of participants experiencing local site reactions up to 24 hours after 
IMP administration will be summarized: 
• overall for the SS 
• for the Training period for the SS 
• during the Self-administration Periods by period and overall for each self-administration - 
method for the RSS  
• during the Self-administration Periods whatever the self-administration method. 
The number and proportion of participants experiencing medication errors associated with 
adverse reactions during the Self-administration Periods will be summarized by period and 
overall for each self-administration method for the RSS. The number and proportion of 
participants experiencing medication errors associated with adverse reactions during the Self-
administration Periods whatever the self-administration method will also be reported. 
A by-participant listing will also be produced including the medications errors together with the 
related adverse reactions for the RSS. As per the guidance on medication related adverse events 
(Pharmacovigilance Risk Assessment Committee. Good practice guide on recording, coding, 
reporting and assessment of medication errors (EMA/762563/2014). 
http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline
/2015/11/WC500196979.pdf (2015). Accessed 30 Jan 2023.), a medication error is an 
unintended failure in the drug treatment process that leads to, or has the potential to lead to, harm 
to the patient. An adverse reaction is a response to a medicinal product which is noxious and 
unintended (Directive 2001/83/EC, Article 1 (11)). An adverse event which is reported as a 
consequence of a medication error is considered as an adverse reaction. 
4.4 
Exploratory endpoints analysis 
4.4.1 
Participant’s relative preference 
The participants’ relative preference for subcutaneous infusions performed by an HCP versus 
self-administration and the manual push method versus the use of a syringe driver for self-
administration will be evaluated at Visit 19 (
) by sequence and overall for the RSS. 
Participants’ preference will also be listed for the RSS. 
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4.4.2 
MG-ADL 
The symptom changes in study participants with gMG will be evaluated. The complete list of 
MG-ADL items and scores are provided in Table 6‒5. The total score will be calculated 
according to the rules set down in Section 6.11. 
The MG-ADL total score and change from Baseline will be summarized at each visit for the RSS 
by sequence using descriptive statistics.  
Worsening in MG-ADL score will be defined by a >2 points increase from Baseline at any 
timepoint during the treatment period. The number and proportion of study participants 
experiencing a worsening of their MG-ADL symptoms during the study will be summarized 
overall for the RSS. This table will also include the number, proportion and 90% CI of study 
participants experiencing MG-ADL worsening, considering the last available measurement in the 
study before the End of Study Visit (Visit 21). The 90% CI will be estimated using the Wilson 
method (without continuity correction) [2].  
Since MG-ADL is completed predose, if time of collection is missing for Visit 2, this will be 
considered as the Baseline assessment.  
Line plots of mean MG-ADL total score and mean change from baseline over time by sequence 
will be presented for the RSS. Spaghetti plots of MG-ADL total score and change from baseline 
over time will be presented for the RSS.  
By-participant listings of MG-ADL values will be provided for the SS. 
4.4.3 
Pharmacodynamic 
The pharmacodynamics of rozanolixizumab will be assessed considering the total IgG level over 
time. Total serum IgG concentrations including observed values and percentage change from 
baseline will be summarized overall for the Training period for the SS and by sequence for the 
RSS. For the analysis of the IgG data, in case rescue therapies are taken, the data up to (not 
including) the start date of rescue therapy and the data 8 weeks after the start date of rescue 
therapy will be utilized for the summary tables, ie the data from (including) start date of rescue 
therapy, to 8 weeks after start date of rescue therapy will be excluded from summary analysis. In 
cases where a study participant drops out, no missing value imputation will be performed for the 
IgG. 
Line plots of median IgG concentrations and percentage change from baseline over time by 
sequence will be presented for the RSS. Spaghetti plots of IgG concentrations and percentage 
change from baseline over time will also be presented for the RSS.  
A line plot presenting mean change from baseline in MG-ADL total score and median 
percentage change from baseline in IgG concentrations will be produced by sequence for the 
RSS. 
In addition, a plot displaying individual percentage change from baseline in IgG concentrations, 
change from baseline in MG-ADL total score and duration of infusion will be produced for the 
RSS. 
Total serum IgG concentrations will be listed by participant for the SS. 
IgG laboratory assessments are performed predose. 
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4.4.4 
Immunogenicity 
The immunogenicity of rozanolixizumab will be assessed following subcutaneous self-
administration. 
Evaluation of rozanolixizumab immunogenicity will be performed using data from all evaluable 
study participants in the SS, defined as all study participants who have an evaluable pretreatment 
(baseline) sample (negative or positive ADA sample status), and at least 1 evaluable post-
baseline value. Study participants with an evaluable pretreatment (baseline) sample but without a 
single evaluable sample taken post-baseline will be included in the reporting of pre-existing 
ADA but excluded from all other immunogenicity analyses. 
ADA Sample Status 
• The ADA sample status will be determined for the pre-treatment (Baseline) and post-
treatment (post-Baseline) visits where samples are taken for ADA analysis. The 
classifications below are made under the assumption that the rozanolixizumab concentrations 
(which are not measured in this study) are equal or below the validated drug tolerance limit 
of the ADA assay (200µg/mL rozanolixizumab) allowing detection of 100ng/mL ADA 
• Sample values that are either ‘negative screen’ or the combination of ‘positive screen’ and 
‘negative immunodepletion’ will be defined as ADA negative  
• Sample values that are ‘positive screen’ and ‘positive immunodepletion’ will be defined as 
ADA positive 
• Samples that could not be tested for ADA status due to inadequate sample volume, 
mishandling, or errors in sample collection, processing, storage, etc, will be defined as 
Missing. 
ADA Participant Status 
The ADA participant status will be classified on study participant and group level as outlined 
below (Shankar et al. 2014; Rup et al, 2015). A description of how study participants will be 
categorized for the immunogenicity assessment is provided in Table 4‒2  
Individual study participants will be assessed for ADA participant status, composed of 6 
categories: ADA negative, and ADA positive, whereby a positive participant’s status is 
determined as originating from a treatment-induced, boosted, reduced or unaffected ADA 
response.  
Study participants who are identified as being treatment-induced or treatment-boosted ADA-
positive will be grouped as treatment-emergent (TE)-ADA positive participants. Study 
participants who are identified as being treatment-reduced or treatment-unaffected ADA-positive 
will be grouped as non-TE-ADA positive participants.  
The individual and combined ADA participant categories will be derived and summarized 
through each scheduled assessment visit unless specified otherwise. Post-baseline time points 
where no ADA sample was collected, will be ignored for the categorization. 
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Table 4‒2: 
Terms and Definitions for ADA Status Evaluation in Study Participant 
Classification 
Classification Label 
Definition 
Individual participant categories 
1 
Pre-ADA negative – treatment 
induced ADA negative 
(ADA-NEG) 
Study participants who have an ADA negative 
sample at Baseline and at all sampling points 
post-Baseline up to the timepoint of interest. 
2 
Inconclusive 
Study participants who have an ADA positive or 
negative Baseline sample and some post-Baseline 
samples are missing, while other post-Baseline 
samples are ADA negative up to the timepoint of 
interest. 
3 
Pre-ADA negative – treatment 
induced ADA positive (TI-
POS) 
Study participants who have an ADA negative 
sample at Baseline and have at least one ADA 
positive sample at any sampling point post-
Baseline up to the timepoint of interest. 
4 
Pre-ADA positive – treatment 
boosted ADA positive (TB-
POS) 
Study participants who have an ADA positive 
sample at Baseline and at least one ADA positive 
sample at any sampling point post-Baseline up to 
the timepoint of interest, with increased titer 
values compared to Baseline (greater than a 
predefined fold difference increase from Baseline 
value which will be defined within the validation 
of the assay i.e. MSR of the assay). 
5 
Pre-ADA positive – treatment 
reduced ADA positive (TR-
POS) 
Study participants with an ADA positive sample 
at Baseline, and ADA negative samples at all 
sampling points post-Baseline up to the timepoint 
of interest. 
6 
Pre-ADA positive – treatment 
unaffected ADA positive 
(TU-POS) 
Study participants with an ADA positive sample 
at Baseline and an ADA positive sample at any 
sampling point post-Baseline up to the timepoint 
of interest, with titer values of the same 
magnitude as Baseline (less than a predefined fold 
difference from the Baseline value which will be 
defined within the validation of the assay, i.e. 
MSR of the assay1). 
Combined participant categories 
7 
Treatment emergent ADA 
positive (TE-POS) 
Includes study participants who are treatment 
induced ADA positive (category 3) or treatment 
boosted ADA positive (category 4). 
8 
Non-treatment emergent ADA 
positive (Non-TE-POS) 
Includes study participants who are treatment 
reduced ADA positive (category 5) or treatment 
unaffected ADA positive (category 6). 
Note: if a participant has no baseline ADA, then she/he can’t be treated as "Inconclusive". 
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Classification 
Classification Label 
Definition 
1 The fold difference increase from baseline value, i.e. the minimum significant ratio (MSR=1.36) determined 
during assay validation, will be reported in the relevant tables, listings and figures. It reflects the fold difference 
in titer level that considered higher than the assay variation in titer determination. 
 
The following tables will be produced: 
• Number and percentage of study participants with ADA (positive, negative, missing sample) 
status at the time of each visit will be summarized by self-administration sequence and 
overall for the RSS. Denominator is the number of study participants having a non-missing 
result at that visit. 
• Number and percentage of study participants in each of the individual and combined ADA 
participant status categories presented in Table 4‒2 will be summarized by self-
administration sequence and overall for the RSS. 
• Total prevalence of pre-existing ADA, defined as number and percentage of participants 
having an ADA positive sample status at baseline, with the denominator being the total 
number of study participants having an evaluable sample result at baseline will be 
summarized by self-administration sequence and overall for the RSS. Missing samples will 
not be included in the denominator.  
The following figures will be produced and will be based on the RSS: 
• Individual time course plots for ADA positive study participants with at least one ADA 
positive sample, representing ADA titers (on log-scale), and the percentage CFB for total 
IgG. The sub-title of the graph will include the study participant number, bodyweight 
category, and individual ADA participant category (3, 4, 5, 6). The dosing will be 
represented in the x-axis with bars/arrows at the time of dose. 
A by-participant listing by period, self-administration method and timepoint, of ADA sample 
status and titer will be produced. 
4.4.5 
SIAQ 
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4.4.6 
Self-administration at home 
The ability of study participants with gMG to successfully self-administer rozanolixizumab at 
home after training in the self-administration technique using the syringe driver and manual push 
methods will be assessed after each administration at home (Visits 10, 11, 12 [
] 
for Self-administration Period 1 and Visits 16, 17, 18 [
] for Self-administration 
Period 2). Successful self-administration includes a correct SC infusion with the intended dose at 
the correct infusion site by the participant and will be assessed by the site via a mandatory direct 
contact with the study participant via telephone or video call as soon as possible after each home 
self-administration. 
The number and proportion of participants deemed a treatment success at each of the 3 home 
self-administration visits will be tabulated by period and overall for each self-administration 
method for the RSS.  
The missing data will not be imputed. 
The self-administration data will be listed for the SS. 
4.4.7 
TEAEs leading to permanent withdrawal 
The safety and tolerability of rozanolixizumab in study participants with gMG will be assessed 
considering the occurrence of TEAEs leading to permanent withdrawal of rozanolixizumab 
during the study in the SS. 
The occurrence of TEAEs leading to permanent withdrawal of rozanolixizumab will be 
summarized during the Self-administration Periods by the number and proportion of participants 
separately by period and overall for each self-administration method. This will be included in the 
AEs overview table. TEAEs leading to permanent withdrawal of rozanolixizumab will also be 
summarized for SS and RSS by System Organ Class (SOC) , High Level Term (HLT) and 
Preferred Term (PT). 
Occurrence of TEAEs leading to permanent withdrawal of rozanolixizumab during the Training 
Period will also be summarized overall. 
4.5 
Other safety analysis 
4.5.1 
Extent of exposure 
Study medication duration (days) will be summarized overall for the SS for Training Period and 
overall. For the RSS, study medication duration will be summarized also by Treatment Periods 
and overall. It will be calculated as: 
• Date of last study medication infusion - Date of first study medication dosing + 1 
The study medication duration will also be described by category: ≥1 day, ≥ 36 days, ≥ 78 days, 
and ≥ 120 days. 
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The number of administrations done by an HCP will be summarized overall from the date of first 
infusion to the last infusion. 
Study medication duration (days) will also be reported overall for each period separately 
(Training period/Self-administration Period 1/ Self-administration Period 2). It will be calculated 
as: 
• Date of last study medication infusion within the period - Date of first study medication 
dosing within the period + 1 
The duration of infusion (in minutes) using self-administration treatment will be summarized for 
the RSS by method and timepoint. It will be derived as: 
Duration of infusion (minutes) = Infusion stop time – Infusion start time. 
All study medication administration details will be listed.  
4.5.2 
Adverse events 
All AEs and SAEs will be collected from the signing of the informed consent form until the End 
of Study Visit (Visit 21) and will be coded (Section 6.2). TEAE definition is available in 
Section 4.3.1.1.  
The following summaries will be provided overall for the SS and by period and overall for each 
self-administration method and overall for the RSS. Adverse events will be presented by SOC, 
HLT and PT: 
• Incidence of AEs overview including any TEAEs, serious TEAEs, participant study 
discontinuations due to TEAEs, drug-related TEAEs, severe TEAEs, AEs leading to death, 
TEAEs leading to death. AEs leading to death will be reported for all screened participants in 
the table produced for SS. 
• Incidence of TEAEs  
• Incidence of TEAEs by Maximum Intensity  
• Incidence of TEAEs by Intensity  
• Incidence of TEAEs by Maximum Relationship  
• Incidence of TEAEs by Relationship  
• Incidence of Treatment-Emergent Serious AEs  
• Incidence of TEAEs Leading to Study Discontinuation  
• Incidence of non-serious TEAEs above reporting threshold of 5% of study participants  
• Incidence of drug-related TEAEs  
• Incidence of severe TEAEs  
• Incidence of TEAEs leading to death  
• Incidence of treatment emergent adverse event of special interest (AESI)  
• Incidence of treatment emergent adverse event of special monitoring (AESM) by type of 
AESM 
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Drug-related is based on the Investigator assessment. 
AESIs are the cases of potential Hy’s Law (cf. Section 4.5.3.1.2).  
AESMs include severe and/or serious headache, suspected aseptic meningitis and medication 
error associated with adverse reaction(s). 
AESMs and AESIs will be identified based on the assessment by the Investigator as recorded in 
the CRF. An AE will be counted as an AESM if there is a ‘yes’ response to the question 
“Adverse event of Special Monitoring?” and ‘no’ otherwise. An AE will be counted as an AESI 
if there is a ‘yes’ response to the question “Adverse Event of Special Interest?” and ‘no’ 
otherwise. 
When applicable adverse event summaries will be ordered by alphabetical SOC, HLT and 
decreasing frequency of PT within SOC and HLT in the RLZ total column for tables.  
Listings of all AEs, all non-serious TEAEs, serious TEAEs, permanent withdrawal of IMP due to 
TEAEs, study participant discontinuation from study due to AEs, AEs leading to death, AESIs, 
and AESMs will be presented by sequence (if applicable), period (if applicable) and participant 
for the SS. AE duration will be included in the listings. Hospitalization data will be listed. 
Rozanolixizumab treatment-emergent adverse events of focus (TEAEOF) will also be analysed 
for the SS. TEAEOF are defined in Section 6.13. The number and percentage of study 
participants who experience each category of the TEAEOF will be summarized. The following 
summaries will be presented by SOC, HLT and PT: 
• TEAEOF (presented also for RSS) 
• Serious TEAEOF 
• TEAEOF by maximum intensity (mild, moderate and severe) 
A by-subject listing of all TEAEOF by category (as listed above) will be provided. Further 
details related to the statistical analysis of the above mentioned treatment-emergent AEOFs are 
provided in Section 6.13. 
4.5.3 
Additional safety assessments  
4.5.3.1 
Clinical laboratory evaluations 
The following table (Table 4‒4) lists safety laboratory assessments that are collected throughout 
the study. Laboratory assessments are performed predose.  
Table 4‒4: 
Protocol-required safety laboratory assessments 
Laboratory 
Assessments 
Parameters 
Hematology 
Platelet count  
White blood cell (WBC) Count with Differential: 
Neutrophils 
Lymphocytes 
Monocytes 
Eosinophils 
Basophils 
Red blood cell (RBC) 
count 
Hemoglobin 
Hematocrit 
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Laboratory 
Assessments 
Parameters 
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)b 
Calcium 
Alkaline phosphatase 
C-reactive 
protein (CRP) 
Lactate 
dehydrogenase 
(LDH) 
Triglycerides  Low-density lipoprotein (LDL) 
High-density lipoprotein 
(HDL) 
Total 
Cholesterol 
Routine 
Urinalysis 
• 
Specific gravity 
• 
pH, glucose, protein, blood, ketones, albumin, bilirubin, urobilinogen, 
nitrite, leukocyte esterase by dipstick 
• 
Microscopic examination (if blood or protein is abnormal) 
Other Screening 
Tests 
• 
Follicle-stimulating hormone and estradiol (as needed in women of 
non-childbearing potential only) 
• 
Serum or urine human chorionic gonadotropin (hCG) pregnancy test (as 
needed for women of childbearing potential)c 
• 
Serology testing (for human immunodeficiency virus, Hepatitis B, and 
Hepatitis C) 
All study-required laboratory assessments will be performed by a central laboratory. 
The results of each test must be entered into the CRF. 
NOTES : 
For additional assessments that may be required in case of AESM of severe and/or serious headache or 
suspected aseptic meningitis, see protocol Table 1-3. 
a Details of liver chemistry stopping criteria and required actions and follow-up assessments after liver 
stopping or monitoring event are given in the protocol Section 7.1.1 and Appendix 6 (Section 10.6). All 
events of ALT 3 × upper limit of normal (ULN) and bilirubin 2 × ULN (>35% direct bilirubin) or 
ALT 3 × ULN 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 To be done at Screening only  
c Local urine testing will be standard for the protocol unless serum testing is required by local 
regulation or IRB/IEC. 
4.5.3.1.1 
Laboratory values over time 
Hematology, clinical chemistry and quantitative urinalysis (observed value, absolute change 
from Baseline) will be summarized in standard units using descriptive statistics for the Training 
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period overall for the SS, for the Self-administration period by period and overall for each self-
administration method for the RSS and for the Follow-up period overall for the RSS. 
Central laboratory data will be used for the summary tables and figures. Local laboratory data 
will be listed only.  
Measurements below the lower limit of quantification (BLQ) will be imputed with half of the 
lower limit of quantification (LLOQ), and measurements above the upper limit of quantification 
(ALQ) will be imputed to the upper quantification limit for the purpose of quantitative 
summaries. Handling of repeated measurements is described in Section 6.8.1. 
An assessment is markedly abnormal (MA) if it meets the MA criteria outlined in Section 6.9. 
Treatment-emergent (TE) is defined as meeting the criteria at any post-Baseline visit after the 
first infusion of study medication and within 56-days of the last infusion and not meeting the 
same criteria at Baseline (see SAP section 4.1.2). 
A TE assessment will be assigned to the Training period if the start date of the assessment is on 
or after the date of the first administration of rozanolixizumab during the Training Period and 
before the first administration of rozanolixizumab in Self-administration Period 1. 
A TE assessment will be assigned to the Self-administration Period 1 if the start date of the 
assessment is on or after the date of the first administration of rozanolixizumab in Self-
administration Period 1 and before the first administration of rozanolixizumab in Self-
administration Period 2. 
A TE assessment will be assigned to the Self-administration Period 2 if the start date of the 
assessment is on or after the date of the first administration of rozanolixizumab in Self-
administration Period 2 and up to the end of Visit 20 assessments (EOT date). 
The number and percentage of participants who have TEMA clinical chemistry and hematology 
assessments at any visit (including unscheduled visit) will be summarized for the Training Period 
using the SS and by Treatment Period and follow-up for each self-administration method for the 
RSS. 
The laboratory variables that are categorized as normal, high or low based on the reference range 
supplied by the analytical laboratory will be presented in shift tables from Baseline to any post-
Baseline visit (including unscheduled visit) overall for the SS. Values of raw calcium will be 
used in shift tables, whereas corrected calcium is used for markedly abnormal assessments as 
outlined in Section 6.9. 
Mean values in serum albumin, C-reactive protein, White Blood Cell Count with absolute count 
and differential (neutrophils, lymphocytes, monocytes, eosinophils and basophils) and platelets 
will be plotted overall for the SS. 
Unscheduled visits assessment will not be included in the summary statistics. All laboratory test 
results will be listed, including Baseline, scheduled and unscheduled visits with results in 
standard unit. Values outside the reference range for the continuous variables will be flagged in 
the listings. The reference ranges will also be reported in the listings. Additional laboratory test, 
including pregnancy testing, will also be listed. 
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4.5.3.1.2 
Potential drug-induced liver injury 
The number and percentage of study participants who meet one or more of the following 
potential drug-induced liver injury (pDILI) criteria will be summarized overall for the SS: 
• Participants with at least one post-Baseline liver laboratory assessment 
• Incidence of Potential hepatotoxicity with symptoms potentially associated with hepatitis or 
hypersensitivity according to the Investigator. 
• Incidence of Potential hepatotoxicity with no symptoms potentially associated with hepatitis 
or hypersensitivity according to the Investigator. 
• Laboratory criteria for pDILI: 
− (AST or ALT ≥ 3 x ULN) and TBL ≥ 1.5 x ULN 
− (AST or ALT ≥ 3 x ULN) and TBL ≥ 2 x ULN 
− (AST or ALT ≥ 3 x ULN) and TBL ≥ 2 x ULN and ALP < 2 x ULN (potential Hy’s Law) 
In order to meet the above criteria, a study participant must experience the elevation in bilirubin 
and ALT or AST (and the absence of the ALP elevation) at the same visit. For example, a study 
participant who experiences a ≥2xULN elevation of bilirubin at one visit and a ≥3xULN 
elevation in ALT (or AST) at a subsequent visit has not fulfilled the Hy’s law criteria. If 
participant meets part of one criterion but at least one parameter is unknown, then he/she should 
not be considered for meeting the criterion. 
Additional analyses for liver function tests (LFTs) will be performed to assess the potential for 
liver toxicities in accordance with the United States Food and Drug Administration guidelines. 
Per guidelines, the following criteria will be used to define levels of LFT elevation: 
• Aspartate aminotransferase (AST): >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x 
ULN 
• Alanine aminotransferase (ALT): >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x ULN 
• AST or ALT: >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x ULN 
• Total bilirubin (TBL): >1.5 x ULN, >2 x ULN 
• Alkaline phosphatase (ALP) >1.5 x ULN 
The number and percentage of study participants who meet one or more of the above LFT 
elevation criteria will be summarized overall for the SS. 
A listing will also be provided for study participants who meet at least one of the above criteria. 
All results obtained at that visit for the specified parameters will be displayed. 
Lifestyle , family medical history, symptoms of hepatitis and hypersensitivity, potential hepato-
toxic medications inquiry and hepatic event supplemental medical history data, PK blood 
sampling will be listed for pDILI. 
4.5.3.2 
Vital signs 
Vital signs will be taken at scheduled visits at the following timepoints: 
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• On Visit 2 (
), Visit 3 (
 Visit 4 (
 vital signs will be measured before 
rozanolixizumab administration, at the end of the infusion, 2 hours after the end of infusion, 
and 4 hours after the end of the infusion. 
• On Visit 5 (
 Visit 6 (
 and Visit 7 (
 vital signs will be measured 
before rozanolixizumab administration, at the end of the infusion, and 1 hour after the end of 
infusion. 
• For site visits from Visit 8 (
 vital signs will be measured before rozanolixizumab 
administration, at the end of the infusion, and 15 minutes after the end of infusion. 
Observed values and changes from Baseline of vital signs variable (pulse rate, respiratory rate, 
systolic and diastolic blood pressure, and temperature) will be summarized at each timepoint for 
the Training period overall for the SS, for the Self-administration Period by period and overall 
for each self-administration method for the RSS and for the Follow-up period overall for the 
RSS. In case of early withdrawal, the visit will be mapped to the pre-dose timepoint of the next 
scheduled visit.  
Treatment-emergent (TE) is defined as meeting the criteria at any post-Baseline visit after the 
first infusion of study medication and within 56-days of the last infusion and not meeting the 
same criteria at Baseline (see SAP section 4.1.2).  
The number and percentage of participants who meet each of the TE markedly abnormal (MA) 
criteria outlined in Section 6.9 at any visit (including unscheduled visit) will be summarized for 
the Training Period using the SS and by Treatment Period and follow-up for each self-
administration method for the RSS. 
A TEMA assessment is assigned to the Treatment Period if the start date of the assessment is on 
or after the date of the first administration of RLZ during the Training Period and before the first 
administration of RLZ in Self-administration Period 1.  
A TEMA assessment is assigned to the Self-administration Period 1 if the start date of the 
assessment is on or after the date of the first administration of RLZ in Self-administration Period 
1 and before the first administration of RLZ in Self-administration Period 2.  
A TEMA assessment is assigned to the Self-administration Period 2 if the start date of the 
assessment is on or after the date of the first administration of RLZ in Self-administration Period 
2 and up to the end of Visit 20 assessments (EOT date). 
All vital signs measurements and change from Baseline will be listed by participant. The listing 
will include a flag for values identified as MA. Unscheduled measurements will be presented in 
the listings. 
4.5.3.3 
Electrocardiograms 
The following ECG variables will be reported: 
• Heart rate 
• PR interval 
• RR interval 
• QRS duration 
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• QT interval 
• QT corrected for heart rate using Fridericia’s formula (QTcF = QT/RR1/3) 
Observed values and changes from Baseline will be summarized overall for the SS, at scheduled 
visit and by ECG variable. The number and percentage of participants with normal, abnormal not 
clinically significant and abnormal clinically significant ECG results will be provided in a shift 
table from Baseline to worst post-Baseline interpretation overall for the SS. 
Treatment-emergent (TE) is defined as meeting the criteria at any post-Baseline visit after the 
first infusion of study medication and within 56-days of the last infusion and not meeting the 
same criteria at Baseline (see SAP section 4.1.2).  
The number and percentage of study participants who meet each of the TEMA criteria outlined 
in Section 6.9 will be summarized overall for the SS at any visit (including unscheduled visit). 
A listing of electrocardiogram data will be presented including unscheduled measurements. 
Abnormal ECG findings will be presented in a separate listing.  
4.5.3.4 
Other safety endpoint(s) 
4.5.3.4.1 
Physical examination 
Physical and neurological examination abnormal results will be listed for the SS. Any clinically 
significant abnormal results will be recorded as AEs. 
4.5.3.4.2 
Suicidal risk monitoring 
A by-participant listing of the C-SSRS questionnaire data will be provided for the SS. Any 
clinically significant abnormal results will be recorded as AEs. Actual attempts will be presented 
in a separate listing. 
4.5.3.4.3 
Assessment and management of Tuberculosis (TB) 
By-participant listings of TB signs and symptoms questionnaire will be provided for the SS. Any 
clinically significant abnormal results will be recorded as AEs. 
TB questionnaire is available in Section 6.15.  
4.5.3.4.4 
Hospitalization 
Hospitalization information will be listed for the SS. Hospitalization will be systematically 
related to an SAE. 
4.5.3.4.5 
History of headache 
The history of headache will be listed for study participants for whom data were collected using 
SS. 
4.6 
Other analyses 
4.6.1 
Subgroup analyses 
No subgroup analyses are planned. 
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4.6.2 
Specific analyses for PMDA 
The following endpoints will be summarized for study participants in Japan only using SS unless 
otherwise specified: 
• Study participant characteristics, including important PDs related to COVID-19 (if 
applicable): 
− Disposition and discontinuation reasons 
− Discontinuation due to AEs  
− Important PDs  
• Demographics and other baseline characteristics: 
− Demographics  
− Baseline characteristics  
• Extent of exposure: 
− Duration of study medication  
• Evaluation of self-administration of study medication: 
− Study participants with successful self-administration of rozanolixizumab (using FAS) 
− Study participants experiencing any TEAEs and any local site reactions up to 24 hours 
after administration during the training period 
− Study participants experiencing any TEAEs and any local site reactions up to 24 hours 
after administration 
− Study participants experiencing any TEAEs, any local site reactions up to 24 hours after 
administration and any medication errors associated with adverse reactions (using RSS) 
− MG-ADL total score observed results and changes from baseline (using RSS) 
− Pre-SIAQ (infusion version) score obtained predose at baseline (using RSS) 
− Post-SIAQ (infusion version) score (using RSS) 
− Post-SIAQ (infusion version) individual item score (using RSS) 
− Participants’ relative preference for subcutaneous infusions (using RSS) 
− Study participants with successful self-administration of rozanolixizumab by visit (using 
RSS) 
− Study participants with successful self-administration of rozanolixizumab at home (using 
RSS) 
• TEAE summaries: 
− Incidence of TEAE – overview  
− Incidence of TEAEs by SOC, HLT and PT 
− Incidence of TEAEs by maximum intensity 
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− Incidence of TEAEs by intensity 
− Incidence of TEAEs by maximum relationship 
− Incidence of TEAEs by relationship 
− Incidence of serious TEAEs by SOC, HLT and PT 
− Incidence of TEAEs leading to study discontinuation by SOC, HLT and PT 
− Incidence of TEAEs leading to permanent withdrawal of study medication by SOC, HLT 
and PT 
− Proportion of study participants with TEAEs leading to permanent withdrawal of study 
medication during the training period. 
4.7 
Interim analyses 
No interim analysis is planned. 
4.8 
Changes to protocol-planned analyses 
Demographics will be presented by sequence and overall as per Clinical Study Protocol (CSP) 
Section 9.2 for the RSS, however will only be presented for overall for the SS.  
The Randomized Safety Set (RSS) is defined in the SAP Section 3 as all participants who are 
included in Safety Set and were randomized but in the CSP it was defined as all randomized 
study participants who received at least 1 dose of IMP (partial or full). 
The Full Analysis Set (FAS) is specified in the SAP Section 3 as all participants who are 
included in Safety Set, were randomized, and completed both self-administration periods. Both 
self-administration periods will be considered as complete if self-administration occurred on 
Visits 13 and 19 in accordance with the randomization scheme. In the CSP it was defined as all 
randomized study participants who received at least 1 dose of IMP (partial or full) and completed 
both self-administration periods. 
In this SAP, baseline assessment for the MG-ADL score follows the same definition as for other 
parameters and consists in the last non-missing measurement before the first administration 
(prior to the Training Visits) at Visit 2 although it was stated differently in the protocol Section 
9.3.3. The reason is that there is no wash-out in this study and thus the first visit in Period 2 
cannot be considered as baseline. Consequently, MG-ADL will be reported by sequence 
although it was mentioned by period and self-administration method in the protocol. 
Confidence intervals for the occurrence of TEAEs leading to permanent withdrawal of 
rozanolixizumab will not be estimated as presented in the Protocol. A simpler summary table 
was deemed appropriate for the team interpretation of the other safety endpoint. 
4.9 
Data Monitoring Committee (DMC) or other review board 
A program Independent Data Monitoring Committee (pIDMC) has been established to provide a 
periodic review of safety data from studies across the rozanolixizumab program. Data emerging 
from this study will be included in the pIDMC periodic data reviews to assess the benefit-risk of 
rozanolixizumab. Analysis performed for the pIDMC is described in separate SAP and shells. 
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5 
SAMPLE SIZE DETERMINATION 
This study is not powered with respect to any endpoint and sample size is based on practical 
considerations. Confidence intervals expected to be observed under assumptions of the true 
success rate are provided in Table 5‒1. It is planned to have approximately 30 participants in 
total (15 per sequence) who will perform self-administration using both the syringe driver and 
the manual push administration methods. In the protocol, it is specified that if study participants 
discontinue before the end of the Self-administration Period 2, replacement participants may be 
considered at the discretion of the sponsor, depending on how the discontinuation affects the 
study’s ability to collect sufficient data to support the study objectives. 
Table 5‒1: 
Expected 90% confidence intervals considering different success 
rates and 30 study participants completing both Treatment Periods 
Success Rate 
Expected 90% confidence interval a 
80% 
64.3 to 90.9 
90% 
76.1 to 97.2 
97% 
85.1 to 99.8 
 a Exact Clopper Pearson confidence intervals 
 
6 
APPENDIX: SUPPORTING DOCUMENTATION 
6.1 
Appendix 1: List of Abbreviations 
List of Abbreviations 
AChR 
Acetylcholine Receptor 
ADA 
Antidrug Antibody 
AE 
Adverse Event 
AESI 
Adverse Event of Special Interest 
AESM 
Adverse Event of Special Monitoring 
ALQ 
Upper Limit of Quantification 
ALT 
Alanine Aminotransferase 
AST 
Aspartate Aminotransferase 
ATC 
Anatomical Therapeutic Chemical 
BLQ 
Below Lower Limit of Quantification 
BMI 
Body Mass Index 
CI 
Confidence Interval 
CO 
Self-confidence 
CRF 
Case Report Form 
CSR 
Clinical Study Report 
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List of Abbreviations 
C-SSRS 
Columbia-Suicide Severity Rating Scale 
CTCAE 
Common Terminology Criteria for Adverse Events 
DMC 
Data Monitoring Committee 
DRM 
Data Review Meeting 
ECG 
Electrocardiogram 
EDV 
Early Discontinuation Visit 
ES 
Enrolled set 
EW 
Early Withdrawal 
FAS 
Full Analysis Set 
FDA 
US Food and Drug Administration 
FL 
Feelings about injection 
gMG 
Generalized Myasthenia Gravis 
HCP 
Health Care Provider 
HLT 
High Level Term 
ICH 
The International Council for Harmonisation of Technical Requirements for 
Pharmaceuticals for Human Use 
IEC 
Independent Ethics Committee 
IgG 
Immunoglobulin 
IMP 
Investigational Medicinal Product 
INR 
International Normalized Ratio 
LLOQ 
Lower Limit of Quantification 
LRP-4 
Low-density lipoprotein Receptor-related Protein 4 
MA 
Markedly Abnormal 
MedDRA 
Medical Dictionary for Regulatory Activities 
MG 
Myasthenia Gravis 
MGFA 
Myasthenia Gravis Foundation of America 
MSR 
Minimum Significant Ratio 
MuSK 
Muscle-specific Kinase  
pDILI 
Potential Drug-Induced Liver Injury 
pIDMC 
Program Independent Data Monitoring Committee 
PK 
Pharmacokinetic 
PPS 
Per Protocol Set 
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List of Abbreviations 
PT 
Preferred Term 
RE 
Injection site Reactions 
RLZ 
Rozanolixizumab 
RSS 
Randomized Safety set 
SA 
Satisfaction with self-injection 
SAP 
Statistical Analysis Plan 
SC 
Subcutaneous 
SD 
Standard Deviation 
SIAQ 
Self-Injection Assessment Questionnaire 
SMQ 
Standardized MedDRA Query 
SOC 
System Organ Class 
SS 
Safety Set 
TB 
Tuberculosis 
TE 
Treatment Emergent 
TEAE 
Treatment-Emergent Adverse Event 
TEAEOF 
Treatment-Emergent Adverse Events Of Focus 
TEMA 
Treatment-Emergent Markedly Abnormal 
TFL 
Table Figure Listing 
ULN 
Upper Limit of Normal 
6.2 
Appendix 2: Coding dictionaries 
Adverse events (AEs) and medical histories will be coded using version 24.0 or later of the 
Medical Dictionary for Regulatory Activities (MedDRA®).  
Medications will be coded according to B3 version Sep 2020 or later of the World Health 
Organization Drug Dictionary (WHODD). 
6.3 
Appendix 3: Participant disposition 
The following outputs will be created: 
• Reasons for screen failures (as collected on the Study Termination Screen Failure CRF 
page) will be tabulated using the ES for overall.  
• Disposition of study participants screened will be tabulated using the ES for overall, by 
site. In this summary, the site number, principal investigator name, first participant in date, 
last participant out date will be reported together with the overall number of participants in 
SS, RSS and FAS and the number of participants by sequence for RSS and FAS. 
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• Disposition of analysis sets will be summarized by the randomized treatment groups, RLZ 
total and analysis sets (SS, RSS and FAS) using the SS. Participants included in the Training 
period will be displayed. 
• Disposition and discontinuation reasons will be tabulated using the SS and RSS, and will 
contain the number and percentage of study participants by treatment group for the RSS, 
overall for the SS who: 
− Started Study 
− Completed Study (A study participant is considered to have 'Completed Study' if 
'Completed Study Participant' has been recorded in CRF form 'Study Termination 
Enrolled')  
− Discontinued Study with primary reason for discontinuation (primary reason for 
premature study termination as collected in the Study Termination Enrolled CRF) 
• Discontinuation due to AEs will be presented using SS to display the total number of study 
participants who discontinued the study due to AEs overall. The categories: AE serious fatal, 
AE non-fatal and other (AE non-serious fatal) will be detailed. 
• Impact of COVID-19 will be presented using SS to display the number and percentage of 
participants in each impact category overall and by visit. 
Listings of study participant disposition including participant analysis sets, re-screened 
participants, visit dates, self-administration training visits, study discontinuation, impact of 
COVID-19 (if applicable) and study participants who did not meet study eligibility and self-
administration eligibility criteria will be provided. 
6.4 
Appendix 4: Baseline characteristics and demographics 
The following baseline variables will be summarized overall for the SS, and by sequence and 
overall for the RSS. Additional subgroup summary will be presented by, during, and post- the 
COVID-19 pandemic based on the enrolled date relative to the pandemic cut-off end date 05-
May-2023, if applicable. 
• Baseline MG-ADL (cf. Section 6.11) 
• Baseline MGFA classification 
• Myasthenic crisis in the past 
• Age at MG diagnosis 
Notes: Age at MG diagnosis will be calculated as Year of Initial MG Diagnosis – Year of 
birth 
• MG duration  
Notes: MG duration will be calculated as (Date of informed consent signed - Date of Initial 
MG Diagnosis+1)/365.25 
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If the date of initial MG diagnosis is partial, it should be imputed to the most recent feasible 
date (i.e., last day of the month if only day is missing, or the last day of the year if day and 
month are missing). 
• Historical AChR antibody status (positive, negative) 
• Historical MuSK antibody status (positive, negative) 
• Historical LRP-4 antibody status (positive, negative) 
• Expected dose level (
) 
The following demographics variables will be summarized overall for the SS, and by sequence 
and overall for the RSS. 
Categories for continuous variables (including n, mean, SD, Median, Min and Max): 
• Age (years) 
Notes: Missing age will be calculated as year of informed consent signed – year of birth. 
• Height (cm) 
• Weight (kg) 
• Body mass index (BMI, kg/m2), to be calculated as: BMI = Weight (kg) / (Height (m))2 
Categorical variables (using frequency counts and percentages): 
• Age (18 - <65, 65 - < 85, >=85 years) 
• Age (≤18,19-<65, ≥65 years) 
• BMI (<30 kg/ m2, ≥30 kg/m2) 
• Weight (35 - <50, 50-<70, 70-<100, ≥100kg) 
• Gender (Male, Female, Undifferentiated, Unknown) 
• Race (American Indian or Alaska Native, Asian, Black or African American, Native 
Hawaiian or Other Pacific Islander, White, Other/Mixed) 
• Ethnicity (Hispanic or Latino, Not Hispanic or Latino) and ethnic subgroup 
• Country 
• Rozanolixizumab status at study entry (Naïve, Non-Naïve). A participant will be considered 
as rozanolixizumab naïve if he did not receive it prior to study entry. 
A by-participant listings of baseline characteristics and demographics will be provided using the 
SS. Childbearing potential data will be listed using the ES separately. 
6.5 
Appendix 5: Protocol deviations 
Important protocol deviations are deviations from the protocol which potentially could have a 
meaningful impact on study conduct or on the primary endpoint, or key safety, for an individual 
participant. The criteria for identifying important protocol deviations will be defined within the 
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appropriate protocol-specific document. Important protocol deviations will be reviewed as part 
of the ongoing data cleaning process and data evaluation. 
A listing of important protocol deviations will be provided based on the SS. 
6.6 
Appendix 6: Medical history 
Previous and ongoing medical conditions will be summarized by primary system organ class and 
preferred term and listed for the SS. 
6.7 
Appendix 7: Prior / concomitant medications 
6.7.1 
Categorization 
Medications will be classified as follows based on imputed start and stop dates & times as 
outlined in Section 4.1.8. 
• Prior medications will include any medications that started before the first administration of 
IMP. 
• Concomitant medications will include any medications that have been taken at least once 
after the first administration of IMP during the Training/Self-administration/Follow-up 
Periods.  
Among concomitant medications, medications will also be classified as part of Self-
administration Period 1 or Period 2. 
The following rules will be used to assign a concomitant medication to Self-administration 
Period 1 or Period 2: 
• Self-administration Period 1: a medication will be assigned to Self-administration Period 1 if 
it has been taken at least once between the first administration of IMP in Self-administration 
Period 1 (Visit 8) and 1 day before the first administration of IMP in Self-administration 
Period 2 (Visit 14). This includes medications that started prior to Self-administration Period 
1 and those that continued into Self-administration Period 2. 
• Self-administration Period 2: a medication will be assigned to Self-administration Period 2 if 
it has been taken at least once between the first administration of IMP in Self-administration 
Period 2 (Visit 14) and up to the end of Visit 20 assessments (EOT date). 
6.7.2 
Prior and concomitant medications summaries 
The number and percentage of participants taking Prior or Concomitant medications will be 
summarized overall using the SS by Anatomical Therapeutic Chemical (ATC) class, presenting 
as Anatomical Main Group (ATC Level 1), Pharmacological Subgroup (ATC level 3), Preferred 
Term (PT). 
Additionally, rescue medications are those that are mentioned in Protocol section 6.5.4 and 
identified if rescue medication is ticked as yes on the CRF Concomitant Medication page. The 
start date of rescue therapy should be on or after Baseline. All rescue therapies and procedures 
will be summarized using the SS. 
The number and percentage of participants taking Concomitant medications will also be 
summarized using the RSS by Anatomical Therapeutic Chemical (ATC) class, presenting as 
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Anatomical Main Group (ATC Level 1), Pharmacological Subgroup (ATC level 3), Preferred 
Term (PT) by period and overall for each self-administration method. 
Medications classified as Prior or Concomitant will be listed using the SS. The rescue therapy 
flag will be included. A by-participant listing of concomitant procedures will also be listed using 
the SS. Originally reported dates will be used for listings. 
6.8 
Appendix 8: Data derivation rules 
6.8.1 
Handling of repeated and unscheduled measurements 
All repeated and unscheduled measurements will be presented in the listings, where applicable. 
The following general rules will apply to all repeated and unscheduled measurements, unless 
otherwise specified: 
• For repeated or unscheduled measurements obtained prior to the first dose of IMP the latest 
non-missing value (scheduled or unscheduled) will be used in the calculation of descriptive 
statistics (i.e. Baseline); 
• For repeated or unscheduled measurements obtained at any time point after the first dose of 
IMP, the scheduled values (if non-missing) will always be used in the calculation of changes 
from Baseline and for the descriptive statistics (ie, in summaries by time point). If repeated 
scheduled values are obtained at any time point, the latest non-missing values will be used. 
6.9 
Appendix 9: Markedly abnormal criteria for Rozanolixizumab 
program 
The following criteria will be applied in the determination of marked abnormalities for 
laboratory assessment values. They are based on Version 5 of the Common Terminology Criteria 
for Adverse Events (CTCAE) grade 3 or higher criteria unless otherwise noted. If both high and 
low criteria are shown for a parameter, the criteria should be summarized separately in tabular or 
graphical data summaries.   
Table 6‒1: 
Hematology 
Parameter 
Unit (conventional) 
Unit 
(standard) 
Marked Abnormality Criteria 
Hemoglobin  
g/dL 
 
g/L 
<8.0 g/dL; <80 g/L  
WBC (Leukocytes)1 
109/L 
109/L  
Low: <2.0 x 109/L  
High:  >30 x 109/L  
Lymphocytes Absolute 
109/L 
109/L  
Low:  <0.5 x 109/L  
High: >20 x 109/L 
Neutrophils Absolute  
109/L 
109/L  
 <1.0 x 109/L   
Platelets 
109/L 
109/L  
<50.0 x 109/L  
1WBC (Leukocytes) markedly abnormal high criterion is not based on Version 5 CTCAE Grade 3 or higher criteria. 
Due to the mechanism of action of RLZ, the safety alert is related to infection risk which would be identified by a 
lower cut-point than the standard which is related to acute leukemias. A markedly abnormal high cut-point >30 x 
109/L is applied to flag leukocytosis (George 2012). 
 
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Table 6‒2: 
Chemistry 
Parameter 
Unit 
(conventional) 
Unit 
(standard) 
Marked Abnormality Criteria 
AST (SGOT) 
U/L 
U/L 
>5.0 x ULN  
ALT (SGPT) 
U/L 
U/L 
>5.0 x ULN  
ALP (Alkaline 
Phosphatase) 
U/L 
U/L 
>5.0 x ULN  
GGT (Gamma 
Glutamyl 
Transferase) 
U/L 
U/L 
>5.0 x ULN  
Bilirubin (Total) 
mg/dL 
umol/L 
>3.0 x ULN if Baseline value is normal; 
>3.0 x Baseline value if Baseline is 
abnormal  
Albumin 
g/dL 
g/L 
<2 g/dL; <20 g/L  
Creatinine 
mg/dL 
umol/L 
>3.0 x ULN or >3.0 x baseline 
Estimate 
glomerular filtrate 
rate (eGFR)1 
mL/min/1.73 m2 
mL/min/1.73 
m2 
eGFR <29 mL/min/1.73 m2 
C reactive protein 
(CRP)2 
mg/L 
mg/L 
>100 mg/L 
Calcium3 
mg/dL 
mmol/L 
Low: Corrected serum calcium of <7.0 
mg/dL; <1.75 mmol/L 
High: Corrected serum calcium of >12.5 
mg/dL; >3.1 mmol/L 
Immunoglobulin 
G4  
(g/L) 
(g/L) 
≤1 g/L 
Potassium 
mmol/L 
mmol/L 
Low: <3.0 mmol/L 
High: >6.0 mmol/L 
Sodium 
mmol/L 
mmol/L 
Low: <125 mmol/L  
High: >155 mmol/L 
Glucose5 
mg/dL 
mmol/L 
Low: <40 mg/dL; <2.2 mmol/L  
High: > 250 mg/dL; >13.9 mmol/L 
Total Cholesterol 
mg/dL 
mmol/L 
>400 mg/dL; >10.34 mmol/L  
Triglycerides 
mg/dL 
mmol/L 
>500 mg/dL; >5.7 mmol/L 
Abbreviations: ALT= alanine aminotransferase; AST = aspartate aminotransferase; dL = deciliter; GGT: gamma 
glutamyltransferase; L = liter; mg = milligram; mmol = millimoles; μg = microgram; U = unit; ULN = upper limit of 
normal 
Note: Marked abnormality criteria are defined by Grade 3 or higher events according to the Common Terminology 
for Adverse Events (CTCAE), Version 5.0, unless otherwise noted.  
1The value of eGFR is calculated using the Chronic Kidney Disease Epidemiology Collaboration or CKD-EPI 
formula (https://qxmd.com/calculate/calculator_251/egfr-using-ckd-epi) which is eGFR = 141 * min(Scr/κ,1)α * 
max(Scr/κ, 1)-1.209 * 0.993Age * 1.018 [if female];  where Scr is serum creatinine (mg/dL), κ is 0.7 for females and 
0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/κ or 1, and max 
indicates the maximum of Scr/κ or 1. For derivation from values in standard units (umol/L) the κ values are 61.88 
for females and 79.56 for males. 
2Includes CRP and High Sensitivity (HS) CRP. Reference for marked abnormality criteria: Nehring, S.M.; Goyal, 
A.; Patel, B.C. (2020). StatPearls Publishing, web link: https://www.ncbi.nlm.nih.gov/books/NBK441843/. A 
moderate elevation of CRP level per referred reference is used for the marked abnormality criteria for RLZ to ensure 
a change suggestive of inflammatory process is captured. Standard CRP test should be used. In case high sensitivity 
CRP (hs-CRP) test have been used in any ongoing studies apply same value (>100mg/L) as marked abnormality 
criteria. 
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3Corrected Calcium (mmol/L) =0.02 * (40 – Albumin (g/L)) + Calcium (mmol/L). 
4Immunoglobulin G criterion based on immunodeficiency literature and noted in RLZ study protocols. 
5Glucose high criterion defined by Grade 3 and higher events according to CTCAE, Version 4.03, June 14, 2010. 
 
Table 6‒3: 
Vital Signs 
Parameter 
Abnormality Criteria 
Pulse Rate (beats/minute) 
 
<50 and a decrease from Baseline of ≥15 
>120 and an increase from Baseline of ≥15 
Systolic Blood Pressure (mmHg) 
≤ 90 and a decrease from Baseline of ≥20 
≥ 180 and an increase from Baseline of ≥20 
Diastolic Blood Pressure (mmHg) 
<50 and a decrease from Baseline of ≥15 
>105 and an increase from Baseline of ≥ 15 
Temperature 
>101 0F (38.3 0C) 
Table 6‒4: 
Electrocardiogram 
Parameter 
Abnormality Criteria 
QT interval (ms) 
 
>500ms 
≥60ms increase from Baseline 
QTc(F) (ms) 
>500ms 
≥60ms increase from Baseline 
PR interval (ms) 
>200ms 
QRS interval (ms) 
>100ms 
Heart rate (bpm) 
<50bpm 
>120bpm 
Abbreviations: bpm = beats per minute; ms = milliseconds; QTc(F) = Fridericia corrected QT interval;  
Note: Treatment-emergent is defined as meeting the criteria at any post-Baseline visit after the 
first infusion of study medication and within 56-days of the last infusion and not meeting the 
same criteria during Baseline. 
6.10 
Appendix 10: Compliance 
Not applicable.  The number of infusions will be recorded as detailed in Section 4.5.1. 
6.11 
Appendix 11: Myasthenia Gravis-Activities of Daily Living 
The MG-ADL score comprises 8 items, each with a score of 0 to 3. The total score is obtained by 
summing the responses to each individual item. Thus, the score ranges from 0 to 24 with a higher 
score indicating more disability. The MG-ADL testing form is provided in Table 6‒5. 
In the event of missing data, the following rules will be applied: 
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• If 1 or 2 items are not answered, the overall score will be obtained by imputing the missing 
items with the average score across the remaining items at the specific visit. The imputed 
value will be rounded to one decimal place 
• If more than 2 items are missing the overall score will not be calculated. 
Table 6‒5: 
MG-Activities of Daily Living 
 
6.12 
Appendix 12: Impact of the coronavirus disease 2019 (COVID-
19) on study data 
Missing data is expected to be one of the major implications of the COVID-19 pandemic. The 
following approaches/strategies will be applied to assess the impact of COVID-19 in this study.  
• Added an electronic Case Report Form (eCRF) page “COVID-19 Impact”, including 
impacted visits, impact categories and relationship to COVID-19;  
• Additional fields were added in protocol deviation specification documents to record 
protocol deviations relationship to COVID-19. 
6.13 
Appendix 13: AEs of focus for Rozanolixizumab program 
The AEOF selection criteria is specified in the Rozimab Safety AEs of Focus document 
developed by UCB. The purpose of this document is to detail the approach to identifying TEAEs 
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meeting criteria for AEOF for the Rozanolixizumab (also called RLZ) program.  
 
Following Events are AEOFs for Rozimab for MG studies: 
No 
Event (also included in 
Title of TFL output) 
Selection criteria 
1 
Headache 
(Note: also included in 
AESM if severe and/or 
serious) 
TEAE with HLGT=’Headaches’ 
2 
Possible aseptic meningitis 
(Note: also included in 
AESM) 
SMQ=’ Noninfectious meningitis’ narrow search 
3 
Gastrointestinal disturbances 
TEAE with  
HLT=‘Gastrointestinal and abdominal pains (excl oral and 
throat)’ or  
 HLT=‘Nausea and vomiting symptoms’ or 
HLT=‘Diarrhoea (excl infective)‘ or 
HLT=‘Gastritis (excl infective)’ or 
PT = ‘Abdominal discomfort’ 
4 
Hypersensitivity reactions  
 
SMQ=’Hypersensitivity’ narrow search 
5 
Anaphylactic reactions 
SMQ=‘Anaphylactic reaction’ and  
TEAEs that either emerged on the same day as when a study 
medication injection reaction was received, or that emerged 
one day after a study medication injection was received, and 
which fulfill any of the following 3 criteria should be included 
in the summary table: 
 
1. If a subject reports any TEAE which codes to a PT 
included in Category A, then the event will be flagged as 
an anaphylactic reaction. 
 
2. If a subject reports any TEAE which codes to a PT 
included in Category B AND reports any TEAE which 
codes to a PT included in Category C, and both TEAEs 
have the same start date, then both events will be 
flagged as anaphylactic reactions.  
 
3. If a subject reports any TEAE which codes to a PT 
included in Category D AND reports (either a TEAE 
which codes to a PT included in Category B OR a TEAE 
which codes to a PT included in Category C), and both 
TEAEs have the same start date, then both events will 
be flagged as anaphylactic reactions. 
 
6 
Injection site reactions 
TEAE with 
HLT=’Injection site reactions’ or 
HLT=’Infusion site reactions’ or 
HLT=’Administration site reactions NEC’ 
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No 
Event (also included in 
Title of TFL output) 
Selection criteria 
7 
Infections 
TEAE with SOC =”Infections and infestations”  
Note: This was added as a reminder for safety that infections 
are considered as AE of focus and require assessment. No 
programming of this topic is required as TEAEs can be found 
in general AE Tables.  
8 
Opportunistic infections 
 
TEAEs in MedDRA SMQ = ‘Opportunistic infections’ narrow 
search 
 
9 
Reductions in albumin and 
plasma proteins 
TEAEs with 
PT=’Blood albumin decreased’ or 
PT=’Protein albumin ratio’ or 
LLT=’Plasma protein abnormal’ or 
LLT=’Proteins serum plasma low’ 
10 
Effects on the kidney 
TEAEs in  
SMQ= ‘Acute renal failure’ narrow search 
11 
Drug related hepatic 
disorders  
TEAEs in  
SMQ=’Drug related hepatic disorders - comprehensive search’ 
narrow and broad search 
12 
Effect on lipids 
TEAEs with  
PT= ‘Blood cholesterol increased’ or  
PT= ‘Low density lipoprotein increased’ or  
PT= ‘Blood triglycerides increased’ or  
PT= ‘Hypercholesterolaemia’ or  
PT= ‘Hypertriglyceridaemia’ or  
PT= ‘Hyperlipidaemia’ or  
PT= ‘Dyslipidaemia’ or  
PT= ‘Lipids increased’ 
 
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6.14 
Appendix 14: Self-Injection Assessment Questionnaire 
 
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6.15 
Appendix 15: Tuberculosis Questionnaire 
 
 
 
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7 
REFERENCES 
[1] SIAQ©v2 user manual Feb 2015 
[2] Brown, Lawrence D.; Cai, T. Tony; DasGupta, Anirban. Interval Estimation for a Binomial 
Proportion. Statist. Sci. 16 (2001), no. 2, 101–133. doi:10.1214/ss/1009213286. 
 
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Approval Signatures
Name:
mg0020-sap-amend-1
Version:
1. 0
Document Number:
CLIN-000249431
Title:
MG0020 Statistical Analysis Plan - Amendment 1
Approved Date:
03 May 2024
Document Approvals
Approval
Verdict: Approved
Name: 
Capacity: Clinical
Date of Signature: 03-May-2024 13:00:42 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Subject Matter Expert
Date of Signature: 03-May-2024 13:14:50 GMT+0000
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