An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Phase: PHASE3
Conditions: Primary Immune Thrombocytopenia
Sponsor: UCB Biopharma SRL

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 1 of 199 
AN OPEN-LABEL EXTENSION STUDY TO INVESTIGATE THE 
LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF 
ROZANOLIXIZUMAB IN STUDY PARTICIPANTS WITH 
PERSISTENT OR CHRONIC PRIMARY IMMUNE 
THROMBOCYTOPENIA (ITP)  
PROTOCOL TP0004 AMENDMENT 4 
 
Short title: 
A Phase 3 study evaluating the long-term safety, tolerability, and efficacy of rozanolixizumab in 
study participants with ITP 
Sponsor: 
UCB Biopharma SRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
  
  
 
 
  
  
Regulatory agency identifying number: 
Eudra CT Number: 
2019-000883-40 
IND Number 
143499  
NCT Number 
NCT04596995 
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. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 2 of 199 
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE 
Document History 
Document 
Date 
Type of amendment 
Protocol Amendment 4 
04 Apr 2022 
Non-substantial 
Protocol Amendment 3 
09 Dec 2021 
Non-substantial 
Protocol Amendment 2b 
03 Dec 2021 
Substantial 
Protocol Amendment 1.1 
(China) 
01 Jul 2021 
Substantial 
Protocol Amendment 1a 
11 May 2021 
Substantial 
Protocol Amendment 0.3 
(China) 
04 Feb 2021 
Substantial 
Protocol Amendment 0.2 
(France) 
04 Feb 2021 
Substantial 
Protocol Addendum A 
(UK) 
26 Oct 2020 
Not applicable 
Protocol Amendment 0.1 
(Japan)  
03 Jun 2020 
Substantial 
Original Protocol 
21 Nov 2019 
Not applicable 
a Protocol Amendment 1 was not submitted to all regulatory authorities prior to submission of 
Protocol Amendment 2. Details of the changes between the original protocol and protocol 
amendment 1 are provided in Section 10.25. 
b Protocol Amendment 2 was not submitted to any regulatory authorities prior to issuance of 
Protocol Amendment 3.  
Amendment 4 (04 Apr 2022) 
Overall Rationale for the Amendment 
The primary reason for this protocol amendment is to correct errors and inconsistencies from 
Protocol Amendment 3.  
This amendment is considered to be non-substantial based on the criteria set forth in Article 
10(a) of Directive 2001/20/EC of the European Parliament and the Council of the European 
Union. 
Section # and Name 
Description of Change 
Brief Rationale 
Global 
Minor administrative, consistency, 
formatting and typographical 
changes have been made. 
Updated to provide clarity and be 
consistent with remainder of 
protocol. 
Global  
Reference to 
 when 
describing the safety of the study 
drug has been amended to “well 
To acknowledge the common 
industry codes, the description of 
the study drug has been updated 
from 
 and well tolerated” to 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 3 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
tolerated, with an acceptable safety 
profile” instead. 
“well tolerated with an acceptable 
safety profile.” 
1.1 Synopsis, Overall 
Design 
Paragraph #4 was updated to 
clarify that all study participants 
will receive rozanolixizumab at the 
assigned dose level from the parent 
study, if the platelet count was 
between ≥50×109/L and 
≤150×109/L. 
Updated for clarity. 
1.3 Schedule of activities  
“W13 and W25 only” was removed 
from the Visit column for 
laboratory parameters. A new 
footnote m was added to clarify 
that samples for total cholesterol, 
LDL, HDL, and triglycerides will 
be collected at Weeks 13 and 25 
only. Subsequent footnotes were 
reorganized.  
 
Footnote j was updated to state 
EOS Visit (Week 60). 
 
Footnote r was added to all dosing 
visits.  
Blood samples are taken every 
4 weeks for safety assessments. 
Analysis of total cholesterol, LDL, 
HDL, and triglycerides will be 
done at Weeks 13 and 25 only.  
 
 
 
 
 
Updated for consistency and to 
correct an error.  
 
Updated for clarity. 
2.1 Study rationale  
 was replaced with “well-
tolerated” in paragraph #5.  
To acknowledge the common 
industry codes, the description of 
the study drug has been updated 
from 
to “well tolerated.” 
3 Objectives and endpoints  One of the other safety endpoints, 
“Changes from Baselinea in 
concentrations of total protein, 
albumin, α-globulin, and β-
globulin”, was updated to remove 
α-globulin and β-globulin.  
 
One of the other efficacy endpoints, 
"Stable Response defined as 
platelet count ≥30×109/L and 
absence of bleedingb without rescue 
therapy at ≥70% of the visits over 
the planned 52-week Treatment 
Period at Week 4" was updated to 
“starting at Week 4.”  
 
One of the other secondary efficacy 
endpoints, “Change from Baselinea 
to Week 54 including all 
Updated for consistency to Section 
1.1. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 4 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
intermediate timepoints for ITP-
PAQ Symptoms domain score” was 
updated to “Change from Baselinea 
to Week 53 including all 
intermediate timepoints for ITP-
PAQ Symptoms domain score.” 
4.1 Overall design  
Paragraph #3 was updated to 
clarify that all study partcipants 
will receive rozanolixizumab at the 
assigned dose level from the parent 
study, if the platelet count was 
between ≥50×109/L and 
≤150×109/L. 
Updated for clarity.   
4.1 Overall design 
Table 4-2 and Figure 4-1 were 
updated to clarify the dose 
adjustments for platelet count 
results of ≥10×109/L to <30×109/L 
(on at least two consecutive visits) 
and <10×109/L. 
Updated for clarity. 
4.1 Overall design 
A sentence was added to the dose 
adjustments paragraph to state: If a 
study participant has been down-
titrated to a fixed-unit dose 
equivalent to 
 or the 
 
total dose (corresponding to an 
average
 dose in a 
participant weighing 70kg), and 
platelet count decreases to 
<50×109/L, then up-titration to a 
fixed-unit dose equivalent to 
 or 
 
respectively, would be allowed. 
Updated for clarity. 
6.4.3.1 Rescue therapy not 
leading to discontinuation  
Bullet point #2 was updated to 
remove reference to “intravenous” 
and “steroids” was updated to 
“corticosteroids.” 
Updated for consistency. 
6.4.3.2 Rescue therapy 
leading to discontinuation 
Bullet point #4: “steroids” was 
updated to “corticosteroids.” 
Updated for consistency. 
6.7 Home visits  
The requirement for stable platelet 
count “during the last two dosing 
visits” was updated to “during the 
last four dosing visits.”  
Platelet count result between 50 to 
150×109/L was corrected to 
between ≥50 to ≤150×109/L. 
Updated for consistency and to 
correct errors. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 5 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
8.1.5.1 Tuberculosis 
assessment 
Reference to the TB questionnaire, 
Appendix 18 was corrected.  
Updated for consistency and to 
correct an error. 
10.1.3 Informed consent 
process 
 
Appendix 10.7, Japan 
Japan-specific requirement for 
study participants <20 was 
removed.  
Since 01 Apr 2022, the revision to 
the Japan civil code has lowered 
the age of adulthood to 18 from 20. 
Appendix 10.7, France  
Anticipated timelines for 
completing enrollment of all 
participants in the parent studies 
were updated.  
To provide the updated projected 
dates.  
10.9 Appendix 9: 
Abbreviations and 
trademarks 
Additional abbreviations and 
definitions were added for: 
UADE and USADE 
General update.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 6 of 199 
SERIOUS ADVERSE EVENT REPORTING 
Serious adverse event reporting (24h) 
All serious adverse events (SAEs) will be reported and transmitted to Patient 
Safety through the electronic Case Report form (eCRF) system. The numbers 
below are to be used to send ancillary documentation only (eg, discharge 
summaries, death certificates) or in the event that the eCRF is not available. 
Fax 
Europe and Rest of the World: +32 2 386 24 21 
US: +1 800 880 6949  or +1 866 890 3175  
Email 
Global (for interventional clinical studies): DS_ICT@ucb.com 
 
Serious adverse event (investigational device) and  
device deficiency reporting (24h)  
Fax: 
Japan: +81 3 6864 7400 
Email: 
Japan: UCBJ-Safety@ucb.com 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 7 of 199 
TABLE OF CONTENTS 
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE ...................................... 2 
SERIOUS ADVERSE EVENT REPORTING .......................................................................... 6 
1 
PROTOCOL SUMMARY ............................................................................................... 12 
1.1 Synopsis ........................................................................................................................... 12 
1.2 Schema ............................................................................................................................. 17 
1.3 Schedule of Activities ...................................................................................................... 19 
2 
INTRODUCTION ........................................................................................................... 32 
2.1 Study rationale ................................................................................................................. 32 
2.2 Background ...................................................................................................................... 34 
2.3 Benefit/risk assessment .................................................................................................... 35 
3 
OBJECTIVES AND ENDPOINTS ................................................................................. 38 
4 
STUDY DESIGN ............................................................................................................. 42 
4.1 Overall design .................................................................................................................. 42 
4.2 Scientific rationale for study design................................................................................. 46 
4.3 Justification for dose ........................................................................................................ 46 
4.4 End of study definition ..................................................................................................... 47 
5 
STUDY POPULATION .................................................................................................. 47 
5.1 Inclusion criteria .............................................................................................................. 48 
5.2 Exclusion criteria ............................................................................................................. 48 
5.3 Lifestyle restrictions ......................................................................................................... 50 
6 
STUDY TREATMENTS ................................................................................................. 50 
6.1 Preparation, handling, storage, and accountability requirements .................................... 50 
6.1.1 
Drug accountability ............................................................................................. 51 
6.2 Measures to minimize bias: randomization ..................................................................... 51 
6.3 Treatment compliance ...................................................................................................... 51 
6.4 Concomitant medications/treatments ............................................................................... 52 
6.4.1 
Permitted concomitant treatments (medications and therapies) .......................... 52 
6.4.2 
Prohibited concomitant treatments (medications and therapies) ......................... 52 
6.4.3 
Rescue therapy ..................................................................................................... 53 
6.4.3.1 
Rescue therapy not leading to discontinuation .......................................... 53 
6.4.3.2 
Rescue therapy leading to discontinuation ................................................ 54 
6.5 Dose modification ............................................................................................................ 54 
6.6 Self-administration ........................................................................................................... 54 
6.7 Home visits ...................................................................................................................... 55 
6.8 Treatment after the end of the study ................................................................................ 55 
7 
DISCONTINUATION OF IMP AND PARTICIPANT 
DISCONTINUATION/WITHDRAWAL ........................................................................ 55 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 8 of 199 
7.1 Discontinuation of IMP .................................................................................................... 55 
7.1.1 
Liver chemistry stopping criteria ......................................................................... 55 
7.1.2 
QTc stopping criteria ........................................................................................... 57 
7.1.3 
Discontinuation of IMP due to other adverse events or medical condition ......... 58 
7.1.4 
Temporary IMP discontinuation .......................................................................... 59 
7.2 Participant discontinuation/withdrawal from the study ................................................... 59 
7.3 Lost to follow-up .............................................................................................................. 60 
8 
STUDY ASSESSMENTS AND PROCEDURES ........................................................... 60 
8.1 Safety assessments ........................................................................................................... 61 
8.1.1 
Physical examination ........................................................................................... 61 
8.1.2 
Vital signs ............................................................................................................ 62 
8.1.3 
Electrocardiograms .............................................................................................. 62 
8.1.4 
Clinical safety laboratory assessments ................................................................ 62 
8.1.5 
Assessment and management of TB and TB risk factors .................................... 63 
8.1.5.1 
Tuberculosis assessment ............................................................................ 63 
8.1.5.2 
Tuberculosis management ......................................................................... 63 
8.1.6 
Splenectomized study participants ...................................................................... 64 
8.2 Adverse events ................................................................................................................. 65 
8.2.1 
Time period and frequency for collecting AE and SAE information .................. 65 
8.2.2 
Method of detecting AEs and SAEs .................................................................... 65 
8.2.3 
Follow-up of AEs and SAEs ................................................................................ 65 
8.2.4 
Regulatory reporting requirements for SAEs ...................................................... 66 
8.2.5 
Pregnancy ............................................................................................................ 66 
8.2.6 
Anticipated serious adverse events ...................................................................... 66 
8.2.7 
Adverse events of special interest ........................................................................ 67 
8.2.8 
Adverse events of special monitoring .................................................................. 67 
8.2.9 
COVID-19 vaccination ........................................................................................ 67 
8.3 Safety signal detection ..................................................................................................... 68 
8.4 Treatment of overdose ..................................................................................................... 68 
8.5 Efficacy assessments ........................................................................................................ 68 
8.5.1 
Platelet counts ...................................................................................................... 68 
8.5.2 
ITP bleeding score ............................................................................................... 69 
8.5.3 
Patient Reported Outcomes ................................................................................. 70 
8.5.3.1 
ITP-PAQ .................................................................................................... 70 
8.5.3.2 
SF-36 ......................................................................................................... 70 
8.5.3.3 
FATIGUE-PRO Physical Fatigue Scale .................................................... 70 
8.5.3.4 
EQ-5D-5L .................................................................................................. 71 
8.5.3.5 
PGI-S ......................................................................................................... 71 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 9 of 199 
8.5.3.6 
PGI-C ......................................................................................................... 71 
8.5.3.7 
SIAQ (Infusion version) ............................................................................ 71 
8.6 Pharmacokinetics and antidrug antibodies ....................................................................... 72 
8.7 Pharmacodynamics .......................................................................................................... 72 
8.8 Exploratory biomarkers ................................................................................................... 73 
8.9 Immunology biomarkers .................................................................................................. 73 
8.10 Medical resource utilization and health economics ......................................................... 73 
9 
STATISTICAL CONSIDERATIONS ............................................................................. 74 
9.1 Definition of analysis sets ................................................................................................ 74 
9.2 General statistical considerations ..................................................................................... 74 
9.3 Estimands ......................................................................................................................... 74 
9.4 Planned safety analyses .................................................................................................... 75 
9.5 Planned efficacy/outcome analyses ................................................................................. 75 
9.6 Planned other analyses ..................................................................................................... 76 
9.6.1 
Analysis of pharmacodynamics endpoints .......................................................... 76 
9.6.2 
Analysis of pharmacokinetics endpoints ............................................................. 76 
9.6.3 
Antidrug antibodies analyses ............................................................................... 76 
9.7 Handling of protocol deviations ....................................................................................... 77 
9.8 Handling of dropouts or missing data .............................................................................. 77 
9.9 Planned interim analysis, data monitoring, and data snapshots ....................................... 77 
9.9.1 
Interim analysis .................................................................................................... 77 
9.9.2 
Data monitoring ................................................................................................... 77 
9.9.3 
Data snapshots ..................................................................................................... 77 
9.10 Determination of sample size ........................................................................................... 77 
10 SUPPORTING DOCUMENTATION AND OPERATIONAL CONSIDERATIONS... 78 
10.1 Appendix 1: Regulatory, ethical, and study oversight considerations ............................. 78 
10.1.1 
Regulatory and ethical considerations ................................................................. 78 
10.1.2 
Financial disclosure ............................................................................................. 78 
10.1.3 
Informed consent process .................................................................................... 79 
10.1.4 
Data protection ..................................................................................................... 79 
10.1.5 
Committees structure ........................................................................................... 80 
10.1.6 
Data quality assurance ......................................................................................... 80 
10.1.6.1 
Electronic Case Report form completion .................................................. 80 
10.1.6.2 
Apps ........................................................................................................... 81 
10.1.7 
Source documents ................................................................................................ 81 
10.1.8 
Study and site closure .......................................................................................... 81 
10.1.9 
Publication policy ................................................................................................ 82 
10.2 Appendix 2: Clinical laboratory tests .............................................................................. 83 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 10 of 199 
10.3 Appendix 3: Adverse events – definitions and procedures for recording, evaluating, 
follow-up, and reporting .................................................................................................. 85 
10.3.1 
Definition of AE .................................................................................................. 85 
10.3.2 
Definition of SAE ................................................................................................ 86 
10.3.3 
Recording and Follow-up of AE and/or SAE ...................................................... 87 
10.3.4 
Reporting of SAEs ............................................................................................... 89 
10.4 Appendix 4: Contraceptive guidance and collection of pregnancy information ............. 91 
10.5 Appendix 5: Liver safety – suggested actions and follow-up assessments ..................... 94 
10.6 Appendix 6: Medical device incidents – definition and procedures for recording, 
evaluating, follow-up, and reporting ................................................................................ 97 
10.7 Appendix 7: Country-specific requirements .................................................................... 98 
Poland 
98 
France 
98 
Japan 
98 
10.7.1 
Countries where self-administration will be offered as an option ..................... 104 
10.8 Appendix 8: Rapid alert procedures .............................................................................. 105 
10.9 Appendix 9: Abbreviations and trademarks .................................................................. 106 
10.10 
Appendix 10: ITP-BAT ....................................................................................... 109 
10.11 
Appendix 11: ITP-PAQ ....................................................................................... 119 
10.12 
Appendix 12: SF-36 Form ................................................................................... 130 
10.13 
Appendix 13: FATIGUE-PRO Physical Fatigue Scale ....................................... 136 
10.14 
Appendix 14: EQ-5D-5L ..................................................................................... 137 
10.15 
Appendix 15: PGI-S ............................................................................................. 139 
10.16 
Appendix 16: PGI-C ............................................................................................ 140 
10.17 
Appendix 17: Headache Questionnaire ............................................................... 141 
10.18 
Appendix 18: Tuberculosis assessment ............................................................... 145 
10.19 
Appendix 19: Sampson Criteria Questionnaire ................................................... 146 
10.20 
Appendix 20: Management of headache .............................................................. 147 
10.21 
Appendix 21: Management of diarrhea ............................................................... 149 
10.22 
Appendix 22: Management of infections and hypogammaglobulinemia ............ 150 
Dose adjustments/dose skipped ......................................................................................... 151 
Splenectomized Study Participants .................................................................................... 151 
10.23 
Appendix 23: Management of infusion-related reactions or hypersensitivity 
reactions ......................................................................................................................... 153 
10.24 
Appendix 24: SIAQ (infusion version) ................................................................ 155 
10.25 
Appendix 25: Protocol Amendment History ....................................................... 162 
11 REFERENCES .............................................................................................................. 197 
SPONSOR DECLARATION ................................................................................................ 199 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 11 of 199 
LIST OF TABLES 
Table 1-1: 
Schedule of activities ....................................................................................... 19 
Table 4-1: 
TP0004 dose levels and weight tiers of IMP ................................................... 43 
Table 4-2: 
Dose adjustments of rozanolixizumab ............................................................. 44 
Table 6-1: 
Treatments administered .................................................................................. 50 
Table 6-2: 
Permitted concomitant treatments (medications and therapies) ...................... 52 
Table 8-1: 
Anticipated serious adverse events for ITP population ................................... 66 
Table 10-1: 
Protocol-required safety laboratory assessments ............................................. 83 
Table 10-2: 
Highly effective contraceptive methodsa ......................................................... 92 
Table 10-3: 
Phase 3 or 4 liver chemistry stopping criteria and follow-up assessments ...... 94 
Table 10-4: 
Phase 3-4 liver chemistry increased monitoring criteria with continued 
IMP .................................................................................................................. 96 
Table 10-5: 
ITP-Bleeding Assessment Tool ..................................................................... 109 
Table 10-6: 
Headache Questionnaire ................................................................................ 141 
Table 10-7: 
Management of infections and hypogammaglobulinemia ............................. 151 
Table 10-8: 
Suggested management guidelines for infusion reactions ............................. 153 
 
LIST OF FIGURES 
Figure 1-1: 
TP0004 study schematic .................................................................................. 18 
Figure 4-1: 
Dose adjustments of rozanolixizumab ............................................................. 45 
Figure 7-1: 
Phase 3-4 liver chemistry stopping criteria and increased monitoring 
algorithm .......................................................................................................... 56 
Figure 7-2: 
Liver chemistry increased monitoring algorithm with continued study 
intervention for participants with ALT ≥3xULN but <8xULN ....................... 57 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 12 of 199 
1 
PROTOCOL SUMMARY 
1.1 
Synopsis 
Protocol title: 
An open-label extension study to investigate the long-term safety, tolerability, and efficacy of 
rozanolixizumab in study participants with persistent or chronic primary immune 
thrombocytopenia (ITP). 
Short Title: 
A Phase 3 study evaluating the long-term safety, tolerability, and efficacy of rozanolixizumab in 
study participants with primary ITP. 
Rationale: 
Immune thrombocytopenia is a clinical disorder in which thrombocytopenia manifests as a 
bleeding tendency, purpura, or petechiae. Autoantibodies against platelet antigens are considered 
to be a hallmark of ITP. Production of pathogenic immunoglobulin (Ig)G autoantibodies by 
plasma cells is accepted as the central underlying pathophysiological mechanism in a number of 
IgG-mediated autoimmune diseases, which includes ITP. In some patients, antibodies recognize 
antigens derived from a single glycoprotein, whereas in others, antibodies recognize multiple 
glycoproteins. The spleen is the key organ in the pathophysiology of ITP, not only because 
platelet autoantibodies are formed in the white pulp, but also because mononuclear macrophages 
in the red pulp destroy immunoglobulin-coated platelets. 
Treatments aimed at reducing the quantity of circulating IgG autoantibodies are being used for 
primary and secondary therapy of autoimmune diseases including ITP, particularly where 
corticosteroid-based immune suppression is not or no longer effective. The therapeutic approach 
of these treatments is based on lowering levels of pathogenic autoantibodies, which represents 
rational and effective treatment modalities of autoimmune diseases. The major goal for treatment 
of ITP is to achieve a platelet count that prevents major bleeding rather than correcting the 
platelet count to normal levels. 
Rozanolixizumab is a humanized IgG4P monoclonal antibody that is being developed as an 
inhibitor of the activity of neonatal Fc receptor (FcRn). The FcRn receptor recycles IgG and 
albumin and transports it bidirectionally across epithelial barriers. Recent studies have shown 
that FcRn rescues both IgG and albumin from intracellular lysosomal degradation by recycling it 
from the sorting endosome to the cell surface (Anderson et al, 2006). Rozanolixizumab has been 
specifically designed to block IgG binding to FcRn without blocking the binding and recycling 
of albumin. By blocking the activity of FcRn, rozanolixizumab accelerates the catabolism of IgG 
antibodies, including IgG autoantibodies. TP0003 and TP0006, the parent studies of the current 
open-label extension (OLE) study, TP0004, are designed to evaluate the efficacy, safety, and 
tolerability of rozanolixizumab given as doses equivalent to 
 or 
reduced doses equivalent to 
 or approximately 
 in 
adult study participants with ITP. This study, TP0004, is designed to assess whether repeat 
treatment over a period of 52 weeks with rozanolixizumab sc infusion is well tolerated with an 
acceptable safety profile and will result in platelet increases as a measure of efficacy. Data from 
this study will support the data from the parent studies (TP0003 and TP0006). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 13 of 199 
Objectives and Endpoints 
Objectives 
Endpoints 
Primary 
• To assess the long-term safety and 
tolerability of treatment with 
rozanolixizumab 
The primary safety endpoints are: 
• Occurrence of treatment-emergent adverse 
events (TEAEs) 
• Occurrence of TEAEs leading to permanent 
withdrawal of rozanolixizumab (ie, study 
discontinuation) 
The other safety endpoints are: 
• Occurrence of treatment-emergent adverse 
events of special monitoring (AESM) 
• Occurrence of serious TEAEs 
• Occurrence of treatment-related TEAEs 
• Vital sign change from Baselinea (blood 
pressure [BP], body temperature, and pulse 
rate) at each scheduled assessment during 
Treatment and Safety Follow-Up (SFU) 
Periods 
• 12-lead electrocardiogram (ECG) change from 
Baselinea at each scheduled ECG assessment 
visit 
• Laboratory change from Baselinea 
(hematology, including coagulation parameters, 
clinical chemistry, and urinalysis) at each 
scheduled assessment during Treatment and 
SFU Periods 
• Changes from Baselinea in concentrations of 
total protein and albumin 
• Changes from Baselinea in serum (C3 and C4) 
and plasma (C3a and C5a) complement levels 
at each scheduled assessment during study (for 
study participants experiencing infusion or 
hypersensitivity reactions) 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 14 of 199 
Objectives 
Endpoints 
Secondary 
• To assess the long-term clinical 
efficacy of treatment with 
rozanolixizumab 
The secondary efficacy endpoint is: 
• Stable Clinically Meaningful Response, defined 
as Clinically Meaningful Response (ie, platelet 
count ≥50×109/L) without rescue therapy at 
≥70% of the visits over the planned 52 week 
Treatment Period starting at Week 4 
The other efficacy endpoints are: 
• Stable Response defined as platelet count 
≥30×109/L and absence of bleedingb without 
rescue therapy at ≥70% of the visits over the 
planned 52-week Treatment Period starting at 
Week 4  
• Cumulative number of weeks with Clinically 
Meaningful Response over the 52-week 
Treatment Period 
• Cumulative number of weeks with platelet 
counts ≥30×109/L over the planned 52-week 
Treatment Period 
• Duration of first Clinically Meaningful 
Response starting at Week 4  
• Mean Change from Baselinea in platelet count 
at each visit 
• Use of rescue medication by visit 
• ITP specific Bleeding Assessment Tool (ITP-
BAT) bleeding events and severity by visit  
• To assess the effect of 
rozanolixizumab on study participant 
perceived symptoms 
• Change from Baselinea to Week 53 including 
all intermediate timepoints for ITP-PAQ 
Symptoms domain score 
• To assess the reduction in use of 
steroids and other concomitant ITP 
medications  
• AUC of the oral steroid dose over time 
• Change in dose and/or frequency of 
concomitant ITP medications (excluding 
corticosteroids) over time  
Exploratory 
• To assess the effect of 
rozanolixizumab on patient-reported 
outcomes (PROs) 
• Change from Baselinea in FATIGUE-PRO 
Physical Fatigue Score  
• Change from Baselinea in Patient Global 
Impression of Severity (PGI-S)  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 15 of 199 
Objectives 
Endpoints 
• Patient Global Impression of Change (PGI-C) 
at all available post-Baselinea assessments  
• Change from Baselinea in ITP-Patient 
Assessment Questionnaire (ITP-PAQ) domain 
scores  
• To assess the clinical efficacy of 
rozanolixizumab in study 
participants with first exposure to 
rozanolixizumab 
• In study participants with first exposure to 
rozanolixizumab: Durable Clinically 
Meaningful Platelet Response of ≥50×109/L, 
for at least 4 of 6 
 visits between 
Weeks 13 to 25 of the study 
• In study participants with first exposure to 
rozanolixizumab: Time to first Clinically 
Meaningful Platelet Response of ≥50×109/L: 
time from starting treatment to achievement of 
first response of ≥50×109/L 
• To assess effect of rozanolixizumab 
on health-related quality of life 
(HRQoL) and broader disease 
impact 
• Change from Baselinea in European Quality of 
Life (EuroQol)-5 dimension 5 Levels 
Assessment (EQ-5D-5L) item responses 
• Change from Baselinea in Short-Form 36-Item 
(SF-36) domain and composite scores 
• To assess resource utilization 
• Number and length of hospitalizations 
• Number of infusion center admissions 
• To assess the experience with the 
subcutaneous (sc) self-administration 
• PRE-Self Injection Assessment Questionnaire 
(SIAQ) (Infusion version) domains scores 
before the first sc self-administration in 
participants that self-administer  
• POST-SIAQ (Infusion version) domains scores 
at each available visit in participants that self-
administer 
• To assess the PD effect of 
rozanolixizumab 
• Total serum IgG (absolute value) and change 
from Baselinea (absolute value and percentage) 
in serum IgG and in serum IgG subclass 
concentration at each scheduled assessment 
• Absolute value and change from Baselinea 
(absolute value and percentage) in serum Ig 
concentrations (IgA, IgE, IgM) at each 
scheduled assessment 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 16 of 199 
Objectives 
Endpoints 
• To evaluate the incidence and 
emergence of antidrug antibody 
(ADA) of rozanolixizumab 
• ADA at each scheduled assessment 
• To assess the pharmacokinetics (PK) 
of rozanolixizumab administered by 
sc infusion 
• Plasma concentrations of rozanolixizumab at 
each scheduled assessment  
• To evaluate clinical response 
following end of dosing   
• Platelet count by visit after end of dosing 
• Time from end of rozanolixizumab dosing to 
loss of clinically relevant response 
• To assess the influence of 
rozanolixizumab treatment on 
vaccination titers 
• Percent change from Baselinea in vaccination 
titers against Streptococcus pneumoniae, 
Neisseria meningitidis, and Haemophilus 
influenzae in splenectomized study participants  
• To assess the effect of 
rozanolixizumab on post-vaccination 
biomarker(s) in study participants 
who received the COVID-19 vaccine 
• Change in post-vaccination biomarker(s) over 
time in study participants who received the 
COVID-19 vaccine  
a Baseline definitions will be detailed in the SAP. 
bAbsence of bleeding indicated by Grade 0 for all domains of the Skin-Visible Mucosa-Internal Organs (SMOG) 
tool, or a Skin Grade of 0 or 1. 
Overall Design 
This is a Phase 3, multicenter, 59-week OLE study of rozanolixizumab in study participants with 
persistent or chronic primary ITP. Study participants from TP0003 and TP0006 who have 
completed the 24-week Treatment Period (irrespective of rescue therapy) and continue to meet 
the eligibility criteria will be offered enrollment into TP0004.  
This study will assess whether continued dosing
 with rozanolixizumab is well tolerated 
with an acceptable safety profile. This study will also assess whether continued rozanolixizumab 
sc infusions 
 over a period of 52 weeks will maintain a durable Clinically Meaningful 
Platelet count of ≥50×109/L. 
The rollover from TP0003 or TP0006 to TP0004 needs to be completed within 3 days after 
Week 25 (Visit 27) of the parent studies at the latest. In case of a late rollover, platelet counts 
will need to be remeasured. 
On Day 1 (Baseline Visit), which corresponds to Week 25 of the parent studies, TP0003 or 
TP0006, all study participants will enter TP0004. All study participants will receive 
rozanolixizumab treatment 
at the assigned dose level at the end of TP0003 or TP0006, if the 
platelet count was between ≥50×109/L and ≤150×109/L. If outside of this range, a higher or 
lower dose level than the final sc infusion in TP0003 or TP0006 is permitted according to 
Table 4-2 and Figure 4-1. Rozanolixizumab will be administered 
 as a sc infusion to 
maintain platelet count ≥50×109/L. Dose levels equivalent to 
 and 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 17 of 199 
 total dose (corresponding to an average 
 dose in a participant weighing 70kg) 
 
can be utilized for maintenance treatment.  
If treatment with rozanolixizumab does not lead to an increase in platelet counts ≥30×109/L, 
study participants can be treated with rescue therapy if deemed needed by the investigator. If 
platelet counts are ≥10×109/L to <30×109/L or active bleeding, rescue therapy is recommended 
(eg, commercially available medication, such as intravenous (iv) immunoglobulin (IVIg), high 
dose corticosteroids and pulse steroids, platelet transfusions, or any other medication listed in 
Section 6.4.3). If platelet counts are <10×109/L, rescue therapy is highly recommended as per 
Section 6.4.3. 
An external Independent Data Monitoring Committee (IDMC) will be utilized to review the 
safety data at predefined intervals and ad hoc as needed, should any emerging safety concern 
arise during the study. Key safety data may be pooled at the discretion of the IDMC for 
protection of all study participants if warranted. 
Based on feedback from the IDMC, 
 dosing was implemented in protocol amendment 2. 
Study participants being treated with the 
 dosing regimen will switch to the 
 
dosing regimen once protocol amendment 2 is approved at the respective study site.  
Additionally, the sponsor’s Safety Signal Detection Team will perform aggregated safety data 
reviews at predefined intervals across the rozanolixizumab program. 
Number of Participants 
Based on the number of study participants planned for the parent studies, up to a maximum of 
180 study participants who complete TP0003 or TP0006 may be eligible to enroll in this OLE 
study. 
Treatment Groups and Duration 
Study participants will be treated for a maximum of 52 weeks. After the final dose, study 
participants will be followed in an 8-week SFU Period. The maximum total duration of the study 
for each study participant will be 59 weeks (For France-specific requirements, see Appendix 7, 
Section 10.7). 
1.2 
Schema 
A schematic diagram for the study is presented in Figure 1-1. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 18 of 199  
Figure 1-1: TP0004 study schematic 
 
 SFU=Safety Follow-Up 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 19 of 199  
1.3 
Schedule of Activities 
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
Written informed consent 
and written 
pharmacogenomics 
substudy informed 
consent (for participating 
study participants) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Verification of 
inclusion/exclusion 
criteria 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic data 
Xe 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 20 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
General 
medical/procedures 
history 
Xe 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
ITP history update  
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior and concomitant 
medication 
Xe 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant medical 
procedures 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Urine pregnancy testf 
X 
 
 
 
 
X 
 
 
X 
 
 
 
X 
 
X 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 21 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
Platelet count (local 
laboratory)g 
Xh 
X 
X 
X 
X 
X 
Xi 
X 
X 
  
X 
Xi 
X 
X 
X 
Withdrawal criteria 
X 
X 
X 
X 
X 
X 
X  
X 
X 
X 
X 
X 
X 
X 
 
12-lead ECGj 
Xk 
pre- and 
postdose 
 
 
 
X 
pre-
dose 
 
 
 
 
X pre-
dose 
(W25 
only) 
 
 
 
  
 
X 
Vital signsl 
Xe 
X 
X 
X 
X 
X 
X 
X 
X 
X  
X 
X  
X 
 
X 
Body weight  
X 
 
 
 
 
 
 
 
X 
(W25 
only) 
 
 
 
X 
 
X 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 22 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
Height 
Xe 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Full physical 
examination 
Xe 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
Short physical 
examination 
 
 
X 
 
 
X 
 
 
X 
 
 
 
 
 
X 
Recording of AEs 
(including 
hospitalizations) 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 23 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
Laboratory parameters 
(coagulation, 
hematology, chemistry, 
and urinalysis using 
central laboratory) 
Xe 
 
X 
X 
 
X 
 
 
Xm 
 
 
 
 
X 
 
X 
Vaccination titer (in 
splenectomized 
participants) 
Xe 
 
 
 
 
 
 
 
X  
(W25 
only) 
 
 
 
 
 
X 
COVID sample (for 
study participants 
vaccinated prior to entry 
into TP0004)n 
X 
 
 
 
 
 
 
 
X 
(W25 
only) 
 
 
 
 
 
X 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 24 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
COVID sample (for 
study participants 
vaccinated during 
enrollment in TP0004) 
Xo 
 
X 
TB signs and symptoms 
questionnairep 
Xe 
 
 
 
 
 
 
 
X 
(W13, 
W25, 
W37, 
W49 only) 
 
 
 
 
 
X 
Contact IRT 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
Administration of IMPq 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 25 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
Blood sampling for 
plasma concentration of 
rozanolixizumabs 
X 
X 
 
X 
 
X 
 
 
X 
(W17, 
W29, 
W41 only) 
 
 
 
 
 
X 
Anti-rozanolixizumab 
antibodiess 
X 
 
  
X 
 
X 
 
 
X 
(W17, 
W29, 
W41 only) 
 
 
 
 
 
X 
Serum complements (C3 
and C4) and plasma 
complements (C3a and 
C5a)t 
X  
X 
X 
X 
X 
X 
X 
X 
X  
(W33 
only) 
X 
X 
X 
X 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 26 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
Blood collection for 
DNA and RNAu 
Xe 
 
 
 
 
X 
 
 
 
 
 
 
 
 
 
Blood collection for 
exploratory biomarker 
analysisv 
Xe 
X 
X 
X 
X 
X 
X 
X 
X 
(W33 
only) 
X 
X 
X 
X 
 
 
Immunoglobulins (total 
IgG, IgG subclasses)w 
Xe 
 
X 
X 
X 
X 
 
X 
X 
 
X 
(W19, 
W23 
only)  
 
 
X 
X 
IgA, IgM, IgE 
Xe 
 
X 
 
 
X 
 
 
X (W17, 
W29, 
W41 only) 
 
 
 
X 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 27 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
ITP-specific 
autoantibodies 
Xe 
 
 
X 
 
 
 
 
X (W33 
only) 
 
 
 
 
 
X 
ITP bleeding scale 
Xe 
X 
X 
X 
X 
X 
 
X 
(W7 
and 11 
only) 
X 
 
X 
 
X 
X 
X 
ITP-PAQx 
Xe 
 
 
 
 
X 
 
 
X 
 
 
 
X 
 
 
SF-36x 
Xe 
 
 
 
 
X  
 
 
X (W17, 
W29, 
W41 only) 
 
 
 
X 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 28 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
EQ-5D-5Lx 
Xe 
 
 
 
 
X 
 
 
X (W17, 
W29, 
W41 only) 
 
 
 
X 
 
 
PGI-Cx 
Xe 
 
 
 
 
X 
 
 
X 
 
 
 
X 
 
 
PGI-Sx 
Xe 
 
 
 
 
X 
 
 
X 
 
 
 
X 
 
 
FATIGUE-PRO Physical 
Fatigue Scalex 
Xe 
 
 
 
 
X 
 
 
X 
 
 
 
X 
 
 
Pre-SIAQ (self-infusion)y 
 
 
 
 
 
 
 
X 
X 
X 
X 
X 
 
 
 
Post-SIAQ (self-
infusion)z 
 
 
 
 
 
 
 
X 
X 
X 
X 
X 
 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 29 of 199  
Table 1-1: 
Schedule of activities 
Assessmentsa 
 
 
Treatment Period 
SFU Period 
Visitb 
1/ 
BL 
2 
3 
4 
5 
6  
7, 9, 
11, 13,  
8, 12, 
16 
10, 14, 18, 
22, 26, 30, 
34, 38, 42, 
46, 50 
15, 17, 19, 21, 
23, 25, 27, 29, 
31, 33, 35, 37, 
39, 41, 43, 45, 
47, 49, 51, 53 
20, 
24, 
28, 52 
32, 36, 
40, 44, 
48 
54 
55 
56 
EOSc/
EW 
Week 
Day 
Visit 
window 
up to +3 
days 
from 
W25 of 
parent 
study 
+2
d 
±2
dd 
±2d
d 
±2d
d 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
±2dd 
Headache questionnaireaa 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
          Site visit 
        Optional home visit: Home IMP administration and assessments are optional and can be conducted (if approved by regulatory agencies). Alternatively, these 
visits can be conducted at the sites, as deemed necessary by site personnel and/or the study participant. 
ADA=antidrug antibody; AE=adverse event; BL=Baseline; BP=blood pressure; d=day(s); DNA=deoxyribonucleic acid; ECG=electrocardiogram; EOS=End of 
Study; EQ-5D-5L=European Quality of Life-5 Dimension 5 Levels; EW=Early Withdrawal; GI=gastrointestinal; HCP=healthcare professional; HDL=high-
density lipoprotein; ICF=Informed Consent form; Ig=immunoglobulin; IMP=investigational medicinal product; IRT=interactive response technology; 
ITP=immune thrombocytopenia; ITP-PAQ=ITP-Patient Assessment Questionnaire; LDL=low-density lipoprotein; PGI-C=Patient Global Impression of Change; 
PGI-S=Patient Global Impression of Severity; PK=pharmacokinetic; RNA=ribonucleic acid; SF-36=Short-Form 36-Item Health Survey; SFU=Safety Follow-up; 
TB=tuberculosis 
a  The allowed time window for assessments is ±5 mins for assessments <30 mins and ±15 mins for assessments >30 mins.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 30 of 199  
b  Home visits, if possible, are applicable for the individual site at the Weeks displayed in the grey columns. Home visits will be performed by a healthcare 
professional (HCP) visiting the study participant at his/her home. Alternately, the visits can be conducted at the site as deemed necessary by site and/or study 
participant. Feasibility of IMP dosing in a home setting will have to be confirmed before the visit is conducted (see Section 6.7).   
c  The EOS Visit is 8 weeks following the final dose of rozanolixizumab. 
d  A visit window of ±2 days is allowed for all visits starting at Visit 3. The visit window of ±2 days is relative to the first dosing visit date.  
e  Data will be transferred from the Screening or Baseline Visit, or Week 25 (Visit 27) of TP0003 or TP0006, as applicable. 
f  If urine pregnancy test is positive, a confirmatory serum pregnancy test is required. The study participant should not be dosed if pregnancy is confirmed. 
g  If required by site’s local procedure, the platelet count assessed by the local laboratory can be done -1 day for all visits. In exceptional cases for site dosing 
visits without a current platelet count, the last two platelet counts can be considered, provided the platelet counts were stable during the last two dosing visits 
(between 50 to 150×109/L and no dose change) and that the dose administered during the last two dosing visits was not changed and is not planned to be 
changed at the current dosing visit. Study participants should not have experienced any ITP signs and symptoms or AEs since the last dosing visit. 
h  If study participants rolled over from TP0003 or TP0006 using the +3 day window, then a new platelet count is needed prior to IMP dosing. 
i  Platelet counts can be taken at the indicated home visits; home visit are allowed if the previous 4 platelet counts were stable between 50 to 150×109/L and there 
was no dose change. If criteria are met, the visit may be conducted at home, administering the same dose of IMP as for the previous visit. 
j  The ECG should be performed prior to blood collection for assessment of laboratory parameters. The ECGs have to be performed pre-dose and 4 hours post-
dose at Day 1, and only pre-dose at Day 15, and Week 25. ECGs should also be performed at the EOS Visit (Week 60). 
k  Local reading for inclusion of study participants by qualified personnel is sufficient. 
l  Vital signs will include BP, pulse rate, and body temperature. On the dosing days, for the first 2 weeks (W1, W2), vital signs will be measured prior to IMP 
administration, at the end of the infusion, and 4 hours after the end of the infusion. For the next 3 visits (W3, W4, W5) vital signs will be measured prior to 
IMP administration, at the end of infusion, and 1 hour after the end of infusion. From W6, vital signs will be measured prior to IMP administration, at the end 
of the infusion, and 15 minutes after the end of infusion. At nondosing visits, vital signs need only to be taken once during the visit. In case of dose increase, 
vital signs will be measured prior to IMP administration, at the end of infusion and 1 hour after the end of infusion for the next 2 infusions. In case of an 
untoward event, additional vital signs (unscheduled assessment) should be taken at the discretion of the investigator and post-observation time can be extended. 
These recommendations are also applicable for dosing at site and at home. 
m  Samples for total cholesterol, LDL, HDL, and triglycerides will be collected at Weeks 13 and 25 only.  
n  For study participants who received COVID vaccine prior to entry into TP0004. 
o  For study participants who received COVID vaccine during enrollment into TP0004. The collection of COVID-19 Baseline samples should occur 2 to 3 weeks 
after the participant has received his/her last vaccination (ie, second dose of a 2-dose vaccine, or a booster vaccination). A second sample should be collected 3 
months after last vaccination and a third sample should be collected 6 months after last vaccination. 
p  The TB signs and symptoms questionnaire have to be performed every 12 weeks (90 days) starting with Week 13 and in Weeks 25, 37, 49, and at the EOS 
Visit (Week 60). 
q  Prior to dosing, management of infections and hypogammaglobulinemia must be considered as per Appendix 22, Section 10.22. 
r  On dosing days, for the first 2 weeks, a 4-hour post-dose observation will be in place. Assuming the first 2 doses were well tolerated, on dosing days in Week 
3, 4, and 5, a 1-hour post dose observation will be in place. Assuming the first 5 doses were well tolerated, on subsequent weeks, a 15-minute post-dose 
observation will be in place. However, if the dose needs to be decreased due to a TEAE(s) or dose needs to be increased, post dose observation will be extended 
to 1 hour after the end of infusion for the next 2 infusions. The post-dose observation time frame may be extended at the discretion of the investigator. These 
recommendations are applicable for dosing at site and at home. Approval from the investigator and sponsor is required prior to the start of home dosing. 
s  Unless specified, samples are collected predose when the IMP is administered at the same visit. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
 
Page 31 of 199  
t  Serum complements (C3, C4) and plasma complements (C3a, C5a) should be taken predose at Baseline (Day 1) and at Week 33 for all study participants. 
Additional samples should be collected 2 hours and 4 hours postevent or as soon as possible before the next IMP for study participants with infusion reaction or 
hypersensitivity reaction (see Section 8.2.8). 
u  Optional blood collection if study participant ticks “yes” on the pharmacogenomics substudy ICF. 
v  Exploratory biomarker samples should be taken predose at Baseline (Day 1) and predose at Week 33. Additional samples should be collected 4 hours postevent 
or as soon as possible before the next IMP for study participants with severe and/or serious headaches or severe and/or serious GI disorders (ie, abdominal pain, 
diarrhea, vomiting; see Section 8.2.8). 
w  If total IgG levels are <1g/L, ad hoc assessments (eg, additional IgG samples) may be performed to monitor recovery of IgG levels. See Section 10.22. 
x  PROs to be completed prior to IMP dose and prior to any other study procedures. 
y   For study participants who self-infuse, to be completed once, prior to first self-infusion. 
z   To be completed within 1 hour after the first and second consecutive self-infusion. 
aa  This assessment is applicable only to participants experiencing moderate, severe and/or serious headache (see Section 10.20), and will be performed daily until 
resolution (ie, if headache becomes mild, normal collection of AEs should apply). This assessment can also be done remotely eg, via phone interview. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 32 of 199 
2 
INTRODUCTION 
Rozanolixizumab (UCB7665) is a humanized IgG4P monoclonal antibody that is being 
developed as an inhibitor of the activity of the FcRn for IgG. 
By blocking the IgG binding site of FcRn, rozanolixizumab accelerates the catabolism of IgG 
antibodies, including IgG autoantibodies. The aim is to reduce the concentration of pathogenic 
IgG in patients with autoimmune diseases mediated by the action of IgG autoantibodies. 
The FcRn recycles IgG and albumin and transports it bidirectionally across epithelial barriers. 
Recent studies have shown that FcRn rescues both IgG and albumin from intracellular lysosomal 
degradation by recycling it from the sorting endosome to the cell surface (Roopenian and 
Akilesh, 2007). Neonatal Fc receptor may also mediate transcytosis of IgG to facilitate its 
distribution within tissues. Rozanolixizumab has been specifically designed to block IgG binding 
to FcRn without blocking the binding and recycling of albumin. 
Rozanolixizumab binds with high affinity to FcRn at both neutral and acidic pH. 
Immunoglobulin G that is constitutively taken up by pinocytosis into cells fails to bind to FcRn, 
even at the acidic pH found in the endosome. It is therefore not recycled and is trafficked to the 
lysosomes for degradation. 
Production of pathogenic IgG autoantibodies is the major pathophysiology leading to a number 
of autoimmune diseases, which include ITP, myasthenia gravis (MG), pemphigus vulgaris, 
Goodpasture’s syndrome, neuromyelitis optica, Guillain-Barré syndrome, and chronic 
inflammatory demyelinating polyradiculoneuropathy. 
As individual disease entities, IgG autoantibody-mediated conditions are relatively rare. 
Treatment of these disorders remains a difficult clinical problem, requiring in many of these 
conditions the long-term use of corticosteroids alone or combined with other immunomodulatory 
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 IVIg, are being used for primary 
and secondary therapy of autoimmune diseases. The therapeutic approach of these treatments is 
thought in part to be based on lowering levels of pathogenic autoantibodies, which represents 
rational and effective treatment modalities of autoimmune diseases. 
Therefore, specific and sustained removal of the IgG autoantibodies by FcRn blockade may 
provide an effective therapeutic option for IgG autoantibody-mediated autoimmune disorders. 
More detailed information regarding the nonclinical and clinical development programs for 
rozanolixizumab, including all completed and ongoing studies, can be found in the Investigator’s 
Brochure (IB). 
2.1 
Study rationale 
Immune thrombocytopenia, previously defined as immune thrombocytopenic purpura and 
idiopathic thrombocytopenic purpura (Rodeghiero et al, 2009), is a rare, orphan, hematological 
autoimmune disease characterized by an isolated low platelet count (thrombocytopenia) caused 
by specific antibodies directed against platelets and the absence of other causes of 
thrombocytopenia. In a pathological disease state these platelets are coated with autoantibodies 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 33 of 199 
to platelet membrane antigens, resulting in splenic sequestration and phagocytosis by 
mononuclear macrophages. The resulting shortened life span of platelets in the circulation, 
together with platelet underproduction by autoantibody-mediated megakaryocytes inhibition 
results in a decreased platelet count (Arnold et al, 2015; McMillan et al, 2004). 
The spleen is the key organ in the pathophysiology of ITP, as it is the site of autoantibody 
production (white pulp) and the site of phagocytosis of autoantibody-coated platelets (red pulp). 
The slow passage of platelets through splenic sinusoids with a high local concentration of 
antibodies, and Fc-gamma receptors on splenic macrophages supports the role of the spleen as a 
site of platelet destruction (Sandler, 2000). 
The major goal for treatment of ITP is to achieve a platelet count that prevents major bleeding 
rather than correcting the platelet count to normal levels. The management of ITP should be 
tailored to the individual patient and it is rarely indicated in those with platelet counts >50×109/L 
in the absence of bleeding, trauma, surgery, or high-risk factors (eg, patients on anticoagulation 
therapy) (EMA/CHMP/153191/2013, 2014). However, there is general agreement that adults 
with a count of <30×109/L with bleeding at diagnosis require treatment. 
The first line of treatment for newly-diagnosed ITP is generally agreed and is based on the use of 
corticosteroids and IVIg. Although corticosteroids are effective, their long-term use is not 
recommended due to concerns over their safety (eg, osteoporosis, hypertension, 
immunosuppression). Patients who fail to respond or who relapse face the options of treatment 
with second line drug therapy or splenectomy, but there is no clear evidence to support the best 
approach (Rodeghiero et al, 2009; Provan et al, 2010; Neunert et al, 2011). Splenectomy can 
provide long-term efficacy in approximately 60% of cases, and recent guidelines suggest 
considering a splenectomy after 12 months. Splenectomy is an invasive procedure associated 
with acute complications (due to thrombocytopenia-like bleeding events) and long-term 
complications from loss of splenic functions. Asplenic individuals are at an increased risk of life-
threatening infections, although minimized with vaccination prophylaxis. Splenectomy may 
increase morbidity from venous thromboembolism or atherosclerosis (Ghanima et al, 2012). 
Second‑line drug therapies include high-dose dexamethasone or methylprednisolone; high-dose 
IVIg or anti-Rho (D) Ig; vinca alkaloids; dapsone and danazol; the immunosuppressants 
cyclophosphamide, azathioprine, and cyclosporine; or mycophenolate mofetil and Helicobacter 
pylori eradication, if applicable. The anti-CD20 monoclonal antibody rituximab, even if not 
licensed for the treatment of ITP, and the thrombopoietin-receptor agonists (eltrombopag, 
romiplostim [both approved in the US and Europe] and avatrombopag [approved in the US in 
2019]) are considered as second-line (Ghanima et al, 2018) as well as third-line options. In 2018, 
in the US, fostamatinib, a spleen tyrosine kinase-inhibitor, was been approved for the treatment 
of chronic ITP. Each of these treatments discussed above, has unique benefits, limitations, 
tolerability considerations, and risks. Taking the side-effect profile and long‑term complications 
of existing treatments together, there remains a considerable unmet medical need for novel 
therapeutic options in the treatment of persistent and or chronic ITP. 
By blocking the activity of FcRn, rozanolixizumab accelerates the catabolism of antibodies and 
reduces the concentration of pathogenic IgG in primary ITP patients, thus potentially offering a 
well-tolerated, effective, and convenient alternative to existing treatments. This Phase 3 OLE 
study will evaluate the longer-term safety and tolerability of rozanolixizumab with repeat dosing 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 34 of 199 
in study participants with primary ITP who have completed the parent studies TP0003 or 
TP0006.  
2.2 
Background 
To date, rozanolixizumab has been administered to human study participants in 9 clinical studies: 
UP0018, UP0060, CIDP01, CIDP04, MG0002, MG0003, MG0004, and its replacement study, 
MG0007, and TP0001. UP0018 is a completed first-in-human (FIH) study. MG0002 is a 
completed Phase 2a study in participants with generalized MG, TP0001 is a completed Phase 2 
study in study participants with primary ITP and CIPD01 is a completed Phase 2a study in study 
participants with CIDP. UP0060 is a Phase 1 study in healthy volunteers, CIDP04 (OLE) is an 
ongoing Phase 2a study in study participants with CIDP, and MG0003 and MG0004 (OLE 
replaced by MG0007) are ongoing Phase 3 studies in study participants with generalized MG. 
CIDP01 is a completed Phase 2a, multicenter, randomized, participant-blind, investigator-blind, 
placebo-controlled, parallel-group study with the primary objective of evaluating the clinical 
efficacy of rozanolixizumab as a treatment for study participants with CIDP. There were 2 
treatment arms in this study:  
• Treatment Arm 1: rozanolixizumab 10mg/kg sc (12 doses administered at 12 weekly visits) 
• Treatment Arm 2: placebo sc (12 doses administered at 12 weekly visits) 
Overall, weekly administration of rozanolixizumab at a dose level of 10mg/kg SC was generally 
well tolerated, with an acceptable safety profile. No new safety concerns were identified. The 
incidence of TEAEs was similar between the rozanolixizumab and placebo groups, with the 
exception of the TEAE of injection/infusion site reactions which was reported at a higher 
frequency in study participants treated with rozanolixizumab. The majority of TEAEs were of 
mild to moderate intensity. The only severe TEAEs reported were in the context of underlying 
CIDP. During the Treatment Period, 10 (58.8%) study participants in the rozanolixizumab group 
and 5 (29.4%) in the placebo group experienced at least 1 drug-related TEAE. During the 
Observation Period, 1 (5.9%) study participant each in the rozanolixizumab and placebo groups 
experienced at least 1 drug-related TEAE. Two (11.8%) study participants in the 
rozanolixizumab group experienced serious TEAEs of CIDP relapse which were in the context 
of their underlying CIDP. No cases of severe headache, moderate or severe abdominal pain, 
moderate or severe vomiting, or severe diarrhea were reported. One (5.9%) study participant 
each in the rozanolixizumab and placebo groups experienced moderate diarrhea. No clinically 
meaningful trends were observed for clinical laboratory evaluations, vital signs measurements, or 
physical examinations.  
CIDP04 is an ongoing Phase 2a, multicenter, open-label extension study to investigate the long-
term safety, tolerability, and efficacy of rozanolixizumab in subjects with CIDP. The study 
includes 2 Treatment periods with 10mg/kg doses of rozanolixizumab by sc infusion at weekly 
intervals in two parts of the study (Treatment Period Part 1: 24 weeks, Treatment Period Part 2: 
52 weeks). 
TP0001 is a completed, Phase 2, multicenter, open-label, multiple-arm study to evaluate the 
safety and tolerability of rozanolixizumab in study participants with primary persistent and 
chronic ITP. The following dose arms were used in the study: 
• Dose Arm 1 (15 participants): rozanolixizumab 4mg/kg sc (5 doses at 1-week intervals) 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 35 of 199 
• Dose Arm 2 (15 participants): rozanolixizumab 7mg/kg sc (3 doses at 1-week intervals) 
• Dose Arm 3 (12 participants): rozanolixizumab 10mg/kg sc (2 doses at 1-week intervals) 
• Dose Arm 4 (12 participants): rozanolixizumab 15mg/kg sc (1 dose) 
• Dose Arm 5 (12 participants): rozanolixizumab 20mg/kg sc (1 dose) 
 
 
Data from TP0001 indicate that rozanolixizumab was tolerated with an acceptable safety profile 
after multiple doses (4mg/kg, 7mg/kg, and 10mg/kg) and single doses (15mg/kg and 20mg/kg). 
The most frequent adverse event (AE) was headache (mild to moderate in intensity) and no 
severe headache was reported. There were no TEAEs leading to IMP discontinuation. 
Four serious TEAEs were reported overall (none were considered to be treatment-related by the 
investigator and were related to the underlying disease). A dose-dependent decrease in total IgG 
was observed: 43.6% (range 21.9% to 68.6%) for the 4mg/kg group (~Day 29), 49.9% (range 
29.5% to 65.5%) for the 7mg/kg group (~Day 22), 63.8% (range 38.4% to 75.0%) for the 
10mg/kg group (~Day 15), and on Day 8, after a single dose, for the 15mg/kg and 20mg/kg 
groups 52.3% (range 30.1% to 68.0%) and 60.2% (range 51.8% to 65.4%), respectively. 
Responders were classified as having a clinically relevant platelet response (≥50×109/L). Five 
(35.7%) participants in the 4mg/kg and 7mg/kg groups, 5 (45.5%) of 11 participants in the 
10mg/kg group, 8 (66.7%) of 12 participants in the single administration 15mg/kg dose group, 
and 6 (54.5%) of 12 participants in the single administration 20mg/kg group were clinically 
relevant responders. 
Based on the above, UCB considered that TP0001 established supportive evidence of efficacy 
and safety, and proceeded with the development of rozanolixizumab for the treatment of adults 
with persistent or chronic primary ITP in two pivotal phase 3 studies prior to the start of this 
study, TP0004. 
TP0003 and TP0006 are Phase 3, multicenter, randomized, double-blind, placebo-controlled 
studies evaluating the efficacy, safety, and tolerability of rozanolixizumab initially administered 
as a single fixed dose equivalent to 
 sc infusion followed by repeated bi-weekly fixed 
doses of approximately
 sc infusion in adult study participants with primary ITP. In 
protocol amendment 3, the starting fixed dose equivalent to 
sc was removed due to the 
change to 
 weekly dosing.  
The current OLE study, TP0004, is a study that will assess the long-term safety, tolerability, and 
efficacy of rozanolixizumab in all study participants completing the 24-week Treatment Period 
in TP0003 or TP0006 (irrespective of rescue medication) and fulfilling the eligibility criteria for 
TP0004. 
2.3 
Benefit/risk assessment 
Although there were a number of treatment options approved for chronic ITP in recent years, 
there is still an unmet medical need as, for many of these medications, the effect diminishes over 
time. The current available therapies like thrombopoietin-receptor agonists (TPO-RAs) have 
dietary exclusions, require liver function test monitoring, or increase the risk of thrombosis or 
thromboembolic events. Corticosteroids are associated with significant long-term side effects. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 36 of 199 
Thus, there is an unmet medical need for new treatments that can reduce the burden of existing 
therapies to patients, both for chronic and intermittent treatment. 
The clinical efficacy data obtained in the Phase 2 study, TP0001, indicate that rozanolixizumab 
has a rapid effect on lowering IgG and rapid onset of platelet response (increase in platelet count 
>50×109/L) and an acceptable safety profile. The ability to administer via sc infusion results in 
low systemic fluid volumes with no impact on plasma viscosity, significant reduction in time for 
infusion, and reduced potential for infusion reactions.  
The clinical safety data to date indicate that rozanolixizumab is well tolerated, and the common 
side effects are generally mild to moderate in severity, can be monitored and are manageable. 
The most common adverse drug reactions observed after use of rozanolixizumab across 
indications are headache, and GI disturbances (diarrhea, nausea, vomiting). Potential risks 
include serious infusion/hypersensitivity reactions, serious infections, and effects on vaccination 
response. These risks of serious infusion/hypersensitivity and serious infections can be mitigated 
by careful monitoring, exclusion of at-risk study participants, and appropriate protocol 
withdrawal and stopping criteria. Additionally, protocol guidance for management of GI 
disturbances, severe headaches, and infection is also provided as well as expedited reporting 
requirements of AESM. 
As with any other monoclonal antibody therapy, the treatment with rozanolixizumab could be 
associated with the development of ADA against rozanolixizumab.  
Participants will be closely monitored for acute infusion reactions or hypersensitivity reactions in 
TP0004. In the event of a recorded infusion reaction or hypersensitivity reaction, further 
complements analyses will be performed alongside the planned ADA assessment to assist in 
immune complex disease determination. The 52-week treatment duration of TP0004 is regarded 
as adequate to assess the risk of immunogenicity after 
 treatment administration. 
Restrictions on use of live vaccines have been defined in the exclusion criterion #14. If 
vaccination with non-live vaccines (including COVID-19 vaccines) is considered necessary once 
a study participant has started therapy with IMP, the degree of protection afforded with a vaccine 
may be compromised while the participant is being treated with IMP. 
Based on its mechanism of action, rozanolixizumab will reduce total IgG levels including 
vaccine specific IgG. However, it is unlikely that the immunogenicity of the vaccine will be 
compromised by FcRn inhibition. Given the study population characteristics (eg, status of the 
underlying disease, concomitant immunosuppressive therapies, etc) 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 
vaccine should be recorded (Section 6.4.1). COVID-19 vaccination should be scheduled, if at all 
possible, to allow differentiation of safety profiles of IMP and vaccine (eg, a minimum window 
of 72 hours between COVID-19 vaccination and IMP administration). If any AEs were to occur, 
they should be handled as described in (Section 8.2.9) with causality assessment provided for 
both IMP and vaccine. Additionally, to further characterize the effect of rozanolixizumab on 
COVID-19 vaccination response, measurement of vaccine titers are being tested in our 
development programs. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 37 of 199 
More detailed information about the known and expected benefits and risks and reasonably 
expected AEs of TP0004 may be found in the current version of the IB.
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 38 of 199 
3 
OBJECTIVES AND ENDPOINTS 
Objectives 
Endpoints 
Primary 
• To assess the long-term safety and 
tolerability of treatment with 
rozanolixizumab 
The primary safety endpoints are: 
• Occurrence of treatment-emergent adverse 
events (TEAEs) 
• Occurrence of TEAEs leading to permanent 
withdrawal of rozanolixizumab (ie, study 
discontinuation) 
The other safety endpoints are: 
• Occurrence of treatment-emergent adverse 
events of special monitoring (AESM) 
• Occurrence of serious TEAEs 
• Occurrence of treatment-related TEAEs 
• Vital sign change from Baselinea (blood 
pressure [BP], body temperature, and pulse 
rate) at each scheduled assessment during 
Treatment and Safety Follow-Up (SFU) 
Periods 
• 12-lead electrocardiogram (ECG) change from 
Baselinea at each scheduled ECG assessment 
visit 
• Laboratory change from Baselinea 
(hematology, including coagulation parameters, 
clinical chemistry, and urinalysis) at each 
scheduled assessment during Treatment and 
SFU Periods 
• Changes from Baselinea in concentrations of 
total protein and albumin 
• Changes from Baselinea in serum (C3 and C4) 
and plasma (C3a and C5a) complement levels 
at each scheduled assessment during study (for 
study participants experiencing infusion or 
hypersensitivity reactions) 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 39 of 199 
Objectives 
Endpoints 
Secondary 
• To assess the long-term clinical 
efficacy of treatment with 
rozanolixizumab 
The secondary efficacy endpoint is: 
• Stable Clinically Meaningful Response, defined 
as Clinically Meaningful Response (ie, platelet 
count ≥50×109/L) without rescue therapy at 
≥70% of the visits over the planned 52 week 
Treatment Period starting at Week 4 
The other efficacy endpoints are: 
• Stable Response defined as platelet count 
≥30×109/L and absence of bleedingb without 
rescue therapy at ≥70% of the visits over the 
planned 52-week Treatment Period starting at 
Week 4  
• Cumulative number of weeks with Clinically 
Meaningful Response over the 52-week 
Treatment Period 
• Cumulative number of weeks with platelet 
counts ≥30×109/L over the planned 52-week 
Treatment Period 
• Duration of first Clinically Meaningful 
Response starting at Week 4 
• Mean Change from Baselinea in platelet count 
at each visit 
• Use of rescue medication by visit 
• ITP specific Bleeding Assessment Tool (ITP-
BAT) bleeding events and severity by visit  
• To assess the effect of 
rozanolixizumab on study participant 
perceived symptoms 
• Change from Baselinea to Week 53 including 
all intermediate timepoints for ITP-PAQ 
Symptoms domain score 
• To assess the reduction in use of 
steroids and other concomitant ITP 
medications  
• AUC of the oral steroid dose over time 
• Change in dose and/or frequency of 
concomitant ITP medications (excluding 
corticosteroids) over time  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 40 of 199 
Objectives 
Endpoints 
Exploratory 
• To assess the effect of 
rozanolixizumab on patient-reported 
outcomes (PROs) 
• Change from Baselinea in FATIGUE-PRO 
Physical Fatigue Score  
• Change from Baselinea in Patient Global 
Impression of Severity (PGI-S)  
• Patient Global Impression of Change (PGI-C) 
at all available post-Baselinea assessments  
• Change from Baselinea in ITP-Patient 
Assessment Questionnaire (ITP-PAQ) domain 
scores  
• To assess the clinical efficacy of 
rozanolixizumab in study 
participants with first exposure to 
rozanolixizumab 
• In study participants with first exposure to 
rozanolixizumab: Durable Clinically 
Meaningful Platelet Response of ≥50×109/L, 
for at least 4 of 6 
 visits between 
Weeks 13 to 25 of the study 
• In study participants with first exposure to 
rozanolixizumab: Time to first Clinically 
Meaningful Platelet Response of ≥50×109/L: 
time from starting treatment to achievement of 
first response of ≥50×109/L 
• To assess effect of rozanolixizumab 
on health-related quality of life 
(HRQoL) and broader disease 
impact 
• Change from Baselinea in European Quality of 
Life (EuroQol)-5 dimension 5 Levels 
Assessment (EQ-5D-5L) item responses 
• Change from Baselinea in Short-Form 36-Item 
(SF-36) domain and composite scores 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 41 of 199 
Objectives 
Endpoints 
• To assess resource utilization 
• Number and length of hospitalizations 
• Number of infusion center admissions 
• To assess the experience with the 
subcutaneous (sc) self-administration 
• PRE-Self Injection Assessment Questionnaire 
(SIAQ) (Infusion version) domains scores 
before the first sc self-administration in 
participants that self-administer 
• POST-SIAQ (Infusion version) domains scores 
at each available visit in participants that self-
administer 
• To assess the PD effect of 
rozanolixizumab 
• Total serum IgG (absolute value) and change 
from Baselinea (absolute value and percentage) 
in serum IgG and in serum IgG subclass 
concentration at each scheduled assessment 
• Absolute value and change from Baselinea 
(absolute value and percentage) in serum Ig 
concentrations (IgA, IgE, IgM) at each 
scheduled assessment 
• To evaluate the incidence and 
emergence of antidrug antibody 
(ADA) of rozanolixizumab 
• ADA at each scheduled assessment 
• To assess the pharmacokinetics (PK) 
of rozanolixizumab administered by 
sc infusion 
• Plasma concentrations of rozanolixizumab at 
each scheduled assessment  
• To evaluate clinical response 
following end of dosing  
• Platelet count by visit after end of dosing 
• Time from end of rozanolixizumab dosing to 
loss of clinically relevant response 
• To assess the influence of 
rozanolixizumab treatment on 
vaccination titers 
• Percent change from Baselinea in vaccination 
titers against Streptococcus pneumoniae, 
Neisseria meningitidis, and Haemophilus 
influenzae in splenectomized study participants  
• To assess the effect of 
rozanolixizumab on post-vaccination 
biomarker(s) in study participants 
who received the COVID-19 vaccine 
• Change in post-vaccination biomarker(s) over 
time in study participants who received the 
COVID-19 vaccine  
a Baseline definitions  will be detailed in the SAP. 
bAbsence of bleeding indicated by Grade 0 for all domains of the Skin-Visible Mucosa-Internal Organs (SMOG) 
tool, or a Skin Grade of 0 or 1. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 42 of 199 
4 
STUDY DESIGN 
4.1 
Overall design 
This is a Phase 3, multicenter, 1-year OLE study of TP0003 and TP0006 parent studies with 
rozanolixizumab in study participants with persistent or chronic primary ITP.  
Study participants from TP0003 or TP0006 who have completed the 24-week Treatment Period 
(irrespective of rescue therapy) and meet eligibility criteria for TP0004 will be offered 
enrollment. Splenectomized participants rolling over from parent studies will have a vaccination 
titer for Streptococcus pneumoniae (S. pneumoniae), Neisseria meningitidis (N. meningitidis), 
and Haemophilus influenzae (H. influenzae) collected per Schedule of Activities (Section 1.3). In 
addition, splenectomized participants should continue to carry a splenectomy card and a 
recommended antibiotic (if applicable per local guidance).  
All study participants rolling over into TP0004 will receive rozanolixizumab at the assigned dose 
level from Week 25 in the parent studies if the platelet count was between ≥50×109/L and 
≤150×109/L. If outside of this range, a higher or lower dose level than the final sc infusion in 
TP0003 or TP0006 is permitted according to Table 4-2 and Figure 4-1.   
During the first two dosing visits, all study participants will stay for 4 hours after the end of 
infusion for observation at the site. Assuming the first 2 doses were well tolerated, the 
observation time will be 1 hour after the end of infusion for another 3 infusions. Assuming the 
first 5 doses were well tolerated, on subsequent weeks, a 15-minute post-dose observation will be 
in place for the remaining visits until Week 52. If needed, observation time can be extended for 
each study participant. However, if the dose needs to be decreased due to a treatment-emergent 
adverse event(s) (TEAE[s]) or dose needs to be increased, the post-infusion observation period 
will be changed to 1 hour for the next 2 infusions. The post-dose observation time frame may be 
extended at the discretion of the investigator.  
This study will assess whether continued 
dosing with rozanolixizumab is well tolerated with 
an acceptable safety profile. This study will also assess whether continued rozanolixizumab sc 
infusions 
 over a period of 52 weeks will maintain a durable Clinically Meaningful Platelet 
count of ≥50×109/L. Starting at Week 2 and until Week 13, platelet counts will be measured 
every week at a local laboratory and every 2 weeks starting at Week 15. Site visits will occur 
every week starting at Week 2. If applicable, home visits and home dosing are permitted per the 
Schedule of Activities (Section 1.3). 
In exceptional circumstances (eg, pandemic, hurricanes, etc) where study-specific investigations 
may not be conducted according to study protocol, contingency measures will be in place (see 
Section 8). If enrollment into TP0004 is not possible at the time of Week 25 (+3 days) in 
TP0003/TP0006, the SFU assessments should be conducted. Once the circumstances during or 
after the pandemic allow, these study participants may be allowed to continue in the OLE study 
in consultation with the UCB study physician. 
The total maximum study duration per study participant is up to 59 weeks, including a Treatment 
Period (52 weeks) and a SFU Period (8 weeks after final dose). The eligibility to participate in 
the study will be determined at the Baseline Visit (Day 1). Participants will receive a dose of 
rozanolixizumab 
 through Week 52. After the final dose in TP0004, participants will be 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 43 of 199 
followed in an 8-week SFU Period. The End of Study (EOS) visit will be performed after the 
SFU Period. 
Fixed dose equivalents across body weight tiers will be employed in this study according to 
Table 4-1. 
Table 4-1: 
TP0004 dose levels and weight tiers of IMP 
Dose eqv 
 
Bodyweight 
 
Dose level 1 
 
Dose level 2 
 
Dose level 3 
>35 to <50kg 
 
 
No weight adjustment    
 
≥50 to <70kg 
 
 
≥70 to <100kg 
 
 
≥100kg 
 
 
eqv=equivalent 
In the case of a study participant of the lowest body weight tier (below 50kg) and in the lowest dose level with a 
platelet count between >150×109/L and <400×109/L, the investigator might decide to temporarily stop treatment 
according to medical judgment based on the observation of platelet variability. Otherwise the treatment must be 
stopped if platelets increase to above 400×109/L. 
An external IDMC will be utilized to review the safety data at predefined intervals and ad hoc as 
needed should any emerging safety concern arise during the study. The IDMC will review 
implications of individual and cumulative cases to continuance of the study in an ongoing 
fashion. Key safety data may be pooled at the discretion of the IDMC for protection of all study 
participants, if warranted. 
Based on feedback from the IDMC, 
 dosing was implemented in protocol amendment 2. 
Study participants being treated with the 
 dosing regimen will switch to the 
 
dosing regimen once protocol amendment 2 is approved at the respective study site.  
At Baseline (Day 1), which corresponds to Week 25 (Visit 27) of the parent studies, TP0003 or 
TP0006, all study participants (whether randomized to placebo or rozanolixizumab in the parent 
studies) will enter TP0004 and receive rozanolixizumab treatment 
 at the assigned dose level 
at the end of TP0003 or TP0006, if the platelet count was between ≥50×109/L and ≤150×109/L. 
Study participants previously randomized to placebo will also enter the study at the assigned 
dose level at the end of the parent studies. Doses can be adjusted if platelets are outside the 
specified range, as per dose adjustment in Table 4-2. After Baseline, the next administration of 
rozanolixizumab will be on Week 2 (Visit 3) and 
 thereafter, as an sc infusion to maintain 
platelet count ≥50×109/L. 
Rozanolixizumab dose levels based on body weight tiers being explored in TP0004 are as 
follows: 
• Equivalent to 
 
• Equivalent to
 
• Equivalent to approximately 
 (
 total dose) 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 44 of 199 
If platelet counts are >150×109/L or <50×109/L on 2 consecutive visits, the dose should be 
adjusted for safety reasons (ie, high platelet count or AE) or efficacy reasons as specified in 
Table 4-2 and Table 4-1. The same dose regimen should be maintained if possible throughout the 
study. 
Table 4-2: 
Dose adjustments of rozanolixizumab 
Platelet count resulta  
Dose adjustment (based on weekly platelet counts) 
<10×109/L  
Increase by one dose level, unless study participant is on 
maintenance dose of 
Additional rescue therapy is 
highly recommended as per Section 6.4.3  
≥10×109/L to <30×109/L (on at least two 
consecutive visits) 
Increase by one dose level, unless study participant is on 
maintenance dose of 
 Additional rescue therapy is 
recommended as per Section 6.4.3  
≥30×109/L to <50×109/L 
(on at least two consecutive visits) 
Increase by one dose level, unless study participant is on 
maintenance dose of 
 
≥50×109/L to ≤150×109/Lb  
Continue with current dose level 
>150×109/L to <400 x109/L (on at least 
two consecutive visits) 
Decrease by one dose level, unless study participant is on 
maintenance dose of 
 
≥400×109/L 
Stop IMP treatment. 
Once the platelet count is ≤150×109/L, reinitiate treatment 
decreased by one dose level  
a  All analyses of platelet counts will be based on local laboratory results. 
b  Due to the interindividual variable platelet response in some study participants, platelet count may abruptly fall 
below 50×109/L after dose reduction or treatment discontinuation. In these cases, if clinically appropriate, higher 
cut-off levels of platelet count for dose reduction (>150×109/L) may be considered according to medical 
judgment, but should not exceed 250×109/L. 
For the dose adjustments where a decrease is required, study participants receiving 
 
should continue at this dose level, as dose reductions below 
 are not applicable. In the case 
of a study participant of the lowest body weight tier (below 50kg) and in the lowest dose level, 
with a platelet count between >150×109/L and <400×109/L, the investigator might decide to 
temporarily stop treatment according to medical judgment based on the observation of platelet 
variability. If platelet count increases to above 400×109/L then IMP must be stopped. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 45 of 199 
Figure 4-1: Dose adjustments of rozanolixizumab 
 
eqv=equivalent; 
 
* eqv: fix unit dose with body weight tiers equivalent to the mg/kg dose in terms of IgG reductions and safety 
profile. 
If treatment as per Table 4-2 and Figure 4-1 does not lead to an increase in platelet count 
≥30×109/L, the study participant can be treated with rescue therapy, if deemed needed by the 
investigator. If platelet counts are ≥10×109/L to <30×109/L or active bleeding, rescue therapy is 
recommended (eg, commercially available medication, such as intravenous immunoglobulin 
(IVIg), high dose corticosteroids and pulse steroids, platelet transfusions, or any other 
medication listed in Section 6.4.3). If platelet counts are <10×109/L, rescue therapy is highly 
recommended as per Section 6.4.3. If a study participant has been down-titrated to a fixed-unit 
dose equivalent to 
 or the 
total dose (corresponding to an average 
dose in 
a participant weighing 70kg), and platelet count decreases to <50×109/L, then up-titration to a 
fixed-unit dose equivalent to 
 or 
 respectively, would be allowed. 
Study participants will be treated for a maximum of approximately 52 weeks. After the final dose 
in TP0004, participants will be followed for safety follow-up purposes in an 8-week SFU Period. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 46 of 199 
4.2 
Scientific rationale for study design 
The target population for rozanolixizumab is patients with IgG autoantibody-mediated 
autoimmune disorders. TP0003 and TP0006, the parent studies of the current OLE study 
TP0004, are designed to assess whether multiple sc infusions of rozanolixizumab will result in a 
durable clinically meaningful platelet increase to count of ≥50×109/L in adult study participants 
with ITP. TP0004 plans to assess participants from TP0003 and TP0006 for up to a year and 
primarily evaluate the safety and tolerability of rozanolixizumab over a longer duration of 
treatment. 
The possibility of chronic, regular, treatment with rozanolixizumab will be assessed in TP0004. 
Study participants who roll over from TP0003 or TP0006 will receive rozanolixizumab at the 
assigned dose level at the end of the parent studies as an sc infusion on Baseline (Day 1) which 
will be repeated on Week 2 (Visit 3), and then 
 thereafter. Study participants assigned to 
placebo through to Week 25 (in TP0003 or TP0006) will also receive rozanolixizumab at the 
assigned dose level at the end of the parent studies. As the study participants previously assigned 
to placebo in TP0003 and TP0006 are likely to have received rescue therapy in order to keep 
their platelet count >30×109/L, it is appropriate to start dosing with the previously assigned dose 
(equivalent to 
 or below). The platelet counts in all study participants are anticipated to 
be already above a critical level (>30×109/L) through previous therapy with rozanolixizumab 
itself or with rescue therapies. Thus, in order to maintain the blind of the pivotal studies, it is 
considered adequate to start with the final dose administered in TP0003 or TP0006. However. 
based on platelet counts, a higher or lower dose level is permitted. Dose levels being explored 
are equivalent to 
 and approximately 
 (
total dose) 
  
In case the enrollment into TP0004 is delayed, the rollover from TP0003 or TP0006 needs to be 
completed 3 days after the last visit, at the latest. In case of a late rollover, platelet counts will 
need to be remeasured. 
4.3 
Justification for dose 
Initially, a fixed-unit dosing regimen across body weight tiers equivalent to 
 (starting 
dose) followed by 
 (maintenance doses) 
 was proposed for use in the confirmatory 
TP0003 and TP0006 studies, based upon response rate (>50×109/L), time to onset of the platelet 
count increase, and safety profile observed in the Phase 2 study. Data from TP0001 showed that 
a 
 dose of 
 reached a 52% decrease in IgG from Baseline by Day 8, and a clinical 
response was observed for most study participants in the cohort before the IgG nadir on Day 8. 
The study also showed that the median (and range) of duration of platelet response (platelet 
count >50×109/L) following a dose of 
 and a 
 dose of 
 was 12 (6 
to 19) days and 11.96 (6 to 20) days, respectively. Hence, a starting dose of 
 equivalent 
followed by 
 equivalent 
 was expected to sustain IgG levels reduced from its 
Baseline level by >50% and expected to be sufficient to translate into a relevant platelet 
response. Dose adjustments to 
 or approximately 
 were allowed if medically 
indicated, eg, for thrombocytosis. The proposed dose regimens were stratified by weight tiers as 
presented in Table 4-1.  
Following the IDMC data review and the available OLE TP0004 data with the initially proposed 
dose regimen of
 the IgG reductions appear to be in concordance with the anticipated 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 47 of 199 
reductions of ~50%, however the observed platelet count data do not seem to be sustained in 
TP0004 during the 
 dosing interval. Protocol Amendment 2 followed the advice from 
the external IDMC to increase the frequency of dosing, as in the OLE study (TP0004), platelet 
counts are not sustained over a 
dosing interval (observed platelet counts <50×109/L). 
Following the advice, the dose regimen for studies TP0003, TP0006 and OLE TP0004 will be 
changed to 
 equivalent 
This dose regimen is anticipated to be well tolerated, with 
an acceptable safety profile and more likely to sustain the platelet counts across the dosing 
interval reducing the risks associated with low platelet counts. A dose of 
 equivalent 
 is predicted to sustain IgG levels with mean percentage change from Baseline reduction of 
70% and maintain FcRn receptor occupancy (RO%) levels above 50% throughout the dosing 
interval as opposed to the 
 that only sustained it above this level during the first week 
following each dose and not for the entire
 period between doses.  
A 
 equivalent 
 dose regimen is anticipated to achieve RO% from peak to trough in a 
range of 99 to 50% during the 
 dosing interval and predicted to achieve mean 
concentrations at steady state (Cmax,ss) of ~30μg/mL (2 days post dose) and Ctrough of 0.31μg/mL. 
In terms of safety, 2x10mg/kg weekly doses have been tested in TP0001 and showed a good 
safety and tolerability profile. In addition, this dose regimen of 10mg/kg of rozanolixizumab QW 
has been studied in other indications like CIDP01, where 34 study participants (1:1 
placebo:rozanolixizumab) followed 12 weekly doses of 10mg/kg administrations, from which at 
least 6 study participants enrolled in the follow up study CIDP04 with a total of 52 weeks 
10mg/kg QW doses. This dose regimen was generally well tolerated, with an acceptable safety 
profile, while no new safety concerns were identified. In addition, in order to avoid platelet count 
increases to ≥150×109/L, down-titration of the IMP dose level as described in Table 4-1 will be 
allowed. Weekly platelet assessments will be performed during the study, to support the dosing 
strategy and stabilize the dose regimen leading to platelet counts ≥50×109/L and minimize 
platelet counts ≥150×109/L in the participants. Up titration to fixed-unit doses of 
 
rozanolixizumab equivalent 
will also be allowed to maintain stable platelet counts of 
>50×109/L over the Treatment Period if a previous down titration has occurred and subsequently 
platelet counts dropped again to below 50×109/L. 
In summary, the new proposed dose regimen for the Phase 3 studies is a 
 equivalent 
dose on a 
basis, allowing to downtitrate to 
 and 
 total dose based on the 
efficacy and safety profile. This regimen is supported by the observed data in the program thus 
far. 
4.4 
End of study definition 
A study participant is considered to have completed the study if he/she has completed all phases 
of the study including the SFU Period. Study participants will have an EOS Visit performed 
8 weeks after the final dose of IMP or the Early Withdrawal (EW) Visit. 
The EOS 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. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 48 of 199 
5.1 
Inclusion criteria 
Study participants are eligible to be included in the study only if all of the following criteria 
apply: 
1b. Study participant completed TP0003 or TP0006 until Visit 27 (Week 25) and, in the opinion 
of the investigator, has been compliant with the TP0003 or TP0006 study assessments. 
2. The study participant is considered reliable and capable of adhering to the protocol, visit 
schedule, or medication intake according to the judgment of the investigator. 
Type of participant and disease characteristics: 
3. Criterion removed. 
4. Criterion removed. 
Sex 
5a. Study participants may be male or female:  
− A male participant must agree to use contraception as detailed in Appendix 4, 
Section 10.4 of this protocol during the Treatment Period and for at least 3 months after 
the final dose of study treatment and refrain from donating sperm during this period. 
− A female participant is eligible to participate if she is not pregnant (see Appendix 4, 
Section 10.4) as confirmed by a negative urine pregnancy test and not planning to get 
pregnant during the participation in the study, not breastfeeding, and at least one of the 
following conditions applies: 
◦ 
Not a woman of childbearing potential (WOCBP) as defined in Appendix 4 
(Section 10.4). 
OR 
◦ 
A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 
(Section 10.4) during the Treatment Period and for at least 3 months after the final 
dose of study treatment. 
Informed consent 
6. Study participant is capable of giving signed informed consent as described in Appendix 1, 
Section 10.1.3 which includes compliance with the requirements and restrictions listed in the 
Informed Consent form (ICF) and in this protocol. 
5.2 
Exclusion criteria 
Participants are excluded from the study if any of the following criteria specific to TP0004 
apply: 
Medical conditions 
1. Study participant has any ongoing IMP-related SAE or ongoing severe IMP-related TEAE 
experienced during TP0003 or TP0006.  
2. Criterion removed. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 49 of 199 
3. Any withdrawal or permanent IMP discontinuation criteria from TP0003 or TP0006 met, a 
change in medical condition from TP0003 or TP0006, or significant bleeding requiring 
immediate treatment which, in the judgment of the investigator, precludes the participant 
from inclusion in TP0004 (eg, clinically significant bleeding, ongoing active or chronic 
infection, acute myocardial infarction, thromboembolic event, liver disease, high risk of GI 
bleeding, severe psychiatric condition). 
4. Late detection of an inclusion criterion not met for TP0003 or TP0006 if it affects the health 
status/safety of the study participant. 
5. Late detection of an exclusion criterion met for TP0003 or TP0006 that is related to ITP 
diagnosis or to the health status/safety of the study participant. 
Diagnostic assessments 
6b. Study participant has, at last available assessment of TP0003 or TP0006, 3.0x upper limit of 
normal (ULN) of any of the following: alanine aminotransferase (ALT), aspartate 
aminotransferase (AST), or alkaline phosphatase (ALP). 
− If study participant has >ULN for ALT, AST, or ALP that does not meet the exclusion 
limit at enrollment (last available assessment of TP0003 or TP0006), repeat the tests, if 
possible, prior to dosing to ensure there is no further ongoing clinically relevant increase. 
− For study 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 electronic Case Report form (eCRF). 
7a. Study participant has bilirubin >1.5xULN (unless confirmed Gilbert’s syndrome) at the last 
available assessment in TP0003/TP0006. If participant has elevations only in total bilirubin, 
fractionate bilirubin to identify possible undiagnosed Gilbert’s syndrome (ie, direct bilirubin 
<35%). 
8. Female study participant who is pregnant or lactating. 
9a. Study participant has planned major elective surgical procedure for the duration of the study 
period. 
10a.Study participant has at the last available assessment in TP0003/TP0006, any laboratory 
abnormality that, in the opinion of the investigator, is clinically significant, has not resolved 
at Visit 1 (Day 1), and could jeopardize or compromise the study participant's ability to 
participate in this study. 
11a.Study participant has 12-lead ECG at Baseline with changes considered to be clinically 
significant upon medical review. The clinical significance of the findings needs to be 
assessed by the investigator to determine eligibility. 
12.Criterion removed. 
13. Study participant has at Baseline corrected QT interval for heart rate using Fridericia’s 
formula (QTcF) >450 msec (for male participants) or QTcF >470 msec (for female 
participants) or QTcF >480 msec in participants with bundle branch block. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 50 of 199 
14. Study participant has received a live vaccination within 8 weeks prior to Day 1 (Baseline 
Visit); or intends to have a live vaccination during the course of the study or within 7 weeks 
following the final dose of IMP. 
For France-specific exclusion criteria (criterions #15, #16 and #17), see Appendix 7 
(Section 10.7). 
5.3 
Lifestyle restrictions 
There are no lifestyle restrictions during the study unless deemed to interfere with the 
compliance with the protocol as deemed by the investigator. 
6 
STUDY TREATMENTS 
Table 6-1: 
Treatments administered 
Study treatment name: 
Rozanolixizumab 
Dosage formulation: 
Solution for infusion 
Unit dose 
strength(s)/Dosage 
level(s): 
 
 
 
Fixed dose equivalent to 
 and approximately 
 based on body weight tiers.  
Route of 
administration: 
sc infusion 
Dosing instructions: 
Rozanolixizumab will be administered as a sc infusion with an 
 
 
 The study participant’s 
body weight at Baseline (TP0004 [re-measured] Baseline and Week 25) 
(refer to Section 9.2 for the definition of Baseline) will be used for the dose 
calculation. In the case of any blockage of the infusion, a suitable flush (eg, 
) is allowed. 
If the participant is unable to tolerate a
 a slower rate may be 
used at the discretion of the clinical personnel administering the infusion. 
Packaging and 
labeling: 
Rozanolixizumab will be provided in glass vials and will be labeled as 
required per country requirement. 
hr=hour; 
 sc=subcutaneous; w/v=weight/volume 
Details on the rate of infusion are provided in the IMP Handling Manual. 
6.1 
Preparation, handling, storage, and accountability requirements 
The investigator or designee must confirm appropriate temperature conditions have been 
maintained during transit for all IMP received and that any discrepancies were reported and 
resolved before the use of the IMP. 
Only participants enrolled in the study may receive IMP and only authorized site staff may 
supply or administer IMP. All IMPs must be stored in a secure, environmentally controlled, and 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 51 of 199 
monitored (manual or automated) area in accordance with the labeled storage conditions with 
access limited to the investigator and authorized site staff. 
The investigator, institution, or the head of the medical institution (where applicable) is 
responsible for IMP accountability, reconciliation, and record maintenance (ie, receipt, 
reconciliation, and final disposition records). 
In case an out-of-range temperature is noted, it must be immediately reported as per instructions 
contained in the IMP Handling Manual. 
Further guidance and information for the final disposition of unused IMP are provided in the 
IMP Handling Manual. 
6.1.1 
Drug accountability 
A Drug Accountability form will be used to record IMP dispensing and return information on a 
by-participant basis and will serve as source documentation during the course of the study. 
Details of any IMP 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. 
UCB is responsible for retaining all used, unused, and partially used containers of IMP until 
returned or destroyed. 
The investigator may assign some of the investigator’s duties for drug accountability at the study 
site to an appropriate qualified person and/or pharmacist/designee. 
The investigator must ensure that the IMP is used only in accordance with the protocol. 
Periodically, and/or after completion of the clinical phase of the study, all used (including empty 
containers)/partially used, unused, damaged, and/or expired IMP must be reconciled and either 
destroyed at the site according to local laws, regulations, and UCB standard operating procedures 
(SOPs). Investigational medicinal product intended for the study cannot be used for any other 
purpose than that described in this protocol. 
6.2 
Measures to minimize bias: randomization 
An interactive response technology (IRT) will be used for assigning eligible participants to IMP 
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 IMP, as appropriate, according to the visit 
schedule. 
The participant number assigned to study participants enrolled in TP0003 and TP0006 will be the 
same identifier used in TP0004. 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. 
6.3 
Treatment compliance 
Drug accountability must be recorded on the Drug Accountability form (Section 6.1.1). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 52 of 199 
Details regarding treatment compliance are outlined in the Important Protocol Deviation 
Specification document. 
6.4 
Concomitant medications/treatments 
6.4.1 
Permitted concomitant treatments (medications and therapies) 
The concomitant medications and the highest dose permitted during the study are listed in 
Table 6-2. Dose tapering, especially for oral corticosteroids, is allowed. Any increase in 
concomitant ITP medication above Baseline (Visit 1, TP0004) dose is regarded as rescue 
therapy.  
For study participants who started rescue medication during TP0003/TP0006 and are still 
receiving rescue medication when rolling over to TP0004, Table 6-2 is applicable as soon as the 
respective rescue medication is stopped or the dose is decreased to less than or equal to the limit 
specified in Table 6-2. In such a situation, the rescue medication will not be considered as 
Baseline concomitant ITP medication and the rules for restarting IMP administration will apply, 
as per Section 6.4.3.  
If a study participant receives a COVID-19 vaccine, the product name and date of 
administration(s) should be captured in the eCRF as a concomitant medication. 
Table 6-2: 
Permitted concomitant treatments (medications and therapies) 
Permitted Medications 
Dose 
Oral Corticosteroids  
≤20mg/day (prednisone equivalent dose) 
Mycophenolate mofetil  
≤3g/day 
Cyclosporin 
≤5mg/kg/day for unmodified 
≤4mg/kg/day for modified 
Azathioprine 
≤3mg/kg/day 
Danazol 
≤15mg/kg/day 
Dapsone 
100mg/day 
Eltrombopag, Avatrombopag 
Any dose 
Fostamatinib 
Any dose 
The use of medicinal cannabidiols and medicinal marijuana (prescribed by a physician) is also 
permitted. For Japan-specific requirements, see Appendix 7, Section 10.7. 
6.4.2 
Prohibited concomitant treatments (medications and therapies) 
The following concomitant medications are prohibited during the study: 
• IVIg (unless it is given as rescue therapy) 
• All biologics including rituximab 
• Cyclophosphamide 
• Pimecrolimus  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 53 of 199 
• Plasma exchange 
• Immunoadsorption 
• Anti-D 
• Any systemically administered corticosteroids above the allowed background dose (unless 
required as a rescue therapy) 
• Romiplostim 
• Vinca alkaloids (vincristine, vinblastine) 
• Anticoagulants and anti-platelets (eg, warfarin, heparin, clopidogrel, aspirin [enteric coated 
up to 150mg daily allowed], salicylics, dipyridamole, prasugrel, ticagrelor, tirofiban, 
abciximab) 
For study participants who require a medical or surgical procedure that requires the use of 
general anesthesia, discussion must occur prior to the procedure with the Medical Monitor or 
study physician, such that a decision on the participant's continued participation in the study can 
be made. In an emergency situation, discussion should occur as soon as possible after the 
procedure. 
The use of prohibited concomitant treatment will lead to permanent discontinuation from IMP. 
Study participants should then complete the assessments outlined for the EW Visit and enter the 
SFU Period. 
6.4.3 
Rescue therapy 
Rescue therapy will be given per standard of care and at the discretion of the investigator. Any 
increase in concomitant ITP medication above Baseline (Visit 1, TP0004) dose is regarded as 
rescue therapy. Any systemically administered corticosteroids above the background doses (ie, 
pulse, oral and iv steroids) and any systemic corticosteroids used for the management of infusion 
reactions or other medical condition are also considered rescue therapy. Depending on the dose 
and/or type of rescue medication that is required a study participant may have to be discontinued 
as described in Section 6.4.3.1 and Section 6.4.3.2. 
6.4.3.1 
Rescue therapy not leading to discontinuation 
The following are defined as rescue therapies and can be administered without requiring study 
participants to be discontinued from the study (per investigator’s judgment): 
• Commercially available IVIg (once platelet count is <50×109/L, rozanolixizumab treatment 
can commence at the same dose as previously administered) 
• High dose corticosteroids (once platelet count is <50×109/L, rozanolixizumab treatment can 
commence at the same dose as previously administered)  
Note: A high dose of corticosteroids is defined as any iv administration of corticosteroids 
given for any reason or doubling of an oral dose resulting in a dose more than 20mg/day 
prednisolone equivalent dose. 
• An increase less than 25% from Baseline (Day 1 in TP0004), in permitted concomitant ITP 
therapies  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 54 of 199 
• An increase less than 25% from Baseline (Day 1 in TP0004), in permitted concomitant 
immunosuppressants 
If such rescue therapy is given, the participant should continue in the study and complete all 
scheduled visits, as per the Schedule of Activities (Section 1.3). 
The date and time of rescue therapy administration as well as the name and dosage regimen of 
the rescue therapy must be recorded. 
Although the intervention of rescue therapy is allowable at any time during the study, the use of 
rescue therapy should be delayed, if possible, for at least 2 days following the administration of 
IMP to allow for the study treatment to have an effect. 
6.4.3.2 
Rescue therapy leading to discontinuation  
The following rescue therapies, if administered, will lead to permanent discontinuation of the 
study participant from the study: 
• Any increase in dose above 25% in a Baseline-permitted concomitant ITP therapy and 
immunosuppressants (Baseline, Day 1 in TP0004)  
• Initiation of oral steroids above 20mg/day 
• Platelet transfusion 
• Initiation of any additional ITP therapy (apart from IVIg and high dose corticosteroids) 
6.5 
Dose modification 
Dose modifications of the IMP are permitted in order to maintain the platelet counts between 
≥50×109/L and ≤150×109/L (Table 4-2 and Figure 4-1). 
Dose modifications or temporary discontinuation of the IMP treatment are permitted if serum 
total IgG value is <1g/L (Section 10.22).  
In the event of drug-related AEs, other than those that meet the IMP discontinuation criteria (as 
defined in Section 7.1) a dose reduction is permitted. Drug-related adverse events due to which 
dose modifications are recommended, may include, but are not limited to: 
• Moderate to severe headaches (Appendix 20, Section 10.20) 
• Severe vomiting and diarrhea (Appendix 21, Section 10.21) 
• Moderate to severe toxicities (Grade 2 and above, as defined by Common Terminology 
Criteria for Adverse Events version 5.0) for which rozanolixizumab cannot be excluded 
as a cause 
The date and reason for dose change of rozanolixizumab is to be recorded on each study 
participant’s eCRF. 
6.6 
Self-administration 
Self-administration will be offered to the study participant, if desired by the participant and 
approved by the investigator. Self-administration can occur at home or onsite, provided the 
participant is willing to do so and has received appropriate training. Training consists of different 
sessions performed by the study nurses, in which the patient would be made familiar with the 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 55 of 199 
process. Only after the healthcare professional (HCP) confirms that the patient is considered 
adequately prepared to perform self-administration, a checklist will be completed and filed in the 
medical chart. Once all of the above is completed, the patient can perform the self-
administration. Self-administration will ONLY occur under the supervision of a fully trained 
HCP (to confirm that study participant is proceeding correctly).  
Note: self-administration will be offered as an option to countries listed in Section 10.7.1. 
6.7 
Home visits 
During the 52-week Treatment Period, certain study visits such as dosing visits (by HCP or self-
administration), may be conducted at a participant’s home or other locations (eg, rehabilitation or 
day care centers, etc), if desired by the participant and approved by the investigator.  
Healthcare professional home visits will be conducted by a fully trained HCP and will follow the 
schedule of assessments and safety monitoring schedule as an onsite visit. Eligibility for home 
visits includes the following: the study participant is deemed a suitable candidate by the 
investigator; the study participant is willing to be seen, dosed and monitored at home by a home 
nurse, and the study participant has shown good tolerability to previous 6 administrations of 
rozanolixizumab at the site (including, but not limited to, no moderate or severe infusion 
reactions, nor any AEs that the investigator considers unsuitable for home administration). A 
further prerequisite for dosing during a home visit is that the platelet counts were stable during 
the last four dosing visits (between ≥50 to ≤150×109/L) and that the dose administered during the 
last four dosing visits was not changed and is not planned to be changed at the home dosing visit. 
Study participants should not have experienced any ITP signs and symptoms or AEs since the 
last dosing visit. 
The investigator will be asked to complete a checklist confirming all criteria have been fully 
evaluated. This checklist will be shared with the UCB study physician or designee and reviewed 
before the first home visit for IMP administration can take place.  
6.8 
Treatment after the end of the study 
Study participants who complete participation in TP0004 may have the possibility to continue 
receiving rozanolixizumab through a Managed Access Program (MAP) if available, as indicated 
by the Sponsor and applicable per local regulations. 
7 
DISCONTINUATION OF IMP AND PARTICIPANT 
DISCONTINUATION/WITHDRAWAL 
7.1 
Discontinuation of IMP 
7.1.1 
Liver chemistry stopping criteria 
Discontinuation of IMP for abnormal liver function should be considered by the investigator 
when a study participant meets one of the conditions outlined in Figure 7-1 or if the investigator 
believes that it is in the best interest of the participant. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 56 of 199 
Figure 7-1: Phase 3-4 liver chemistry stopping criteria and increased monitoring 
algorithm 
 
ALT=alanine aminotransferase; INR=International Normalized Ratio; ULN=upper limit of normal 
Treatment with IMP may be continued with increased monitoring if a study participant meets 
one of the criteria outlined in Figure 7-2. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 57 of 199 
Figure 7-2: Liver chemistry increased monitoring algorithm with continued study 
intervention for participants with ALT ≥3xULN but <8xULN 
  
ALT=alanine aminotransferase; bili=bilirubin; ULN=upper limit of normal 
Specific assessments and follow-up actions for potential drug-induced liver injury (PDILI) are 
provided in Appendix 5, Section 10.5. 
7.1.2 
QTc stopping criteria 
A participant who meets either bulleted criterion based on the ECG readings will be withdrawn 
from IMP and move into the SFU Period. The study participant should be referred to a specialist 
(ie, cardiologist) and managed as per local guidance.   
• Corrected QT interval (QTc) >500ms OR uncorrected QT >600ms 
• Change from Baseline of QTc >60ms 
For participants with underlying bundle branch block, follow the discontinuation criteria listed 
below: 
Baseline QTc with bundle branch 
block 
Discontinuation QTc threshold with bundle branch 
block 
<450ms 
>500ms 
450 to 480ms 
≥530ms 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 58 of 199 
QTc=corrected QT interval 
If a clinically significant finding is identified (including, but not limited to changes from 
Baseline in QT interval corrected for heart rate according to Fridericia's formula) after 
enrollment, the investigator or qualified designee will determine if the participant can continue in 
the study and if any change in participant management is needed. This review of the ECG 
printed at the time of collection must be documented. Any new clinically relevant finding should 
be reported as an AE. 
See the Schedule of Activities (Section 1.3) for data to be collected at the time of treatment 
discontinuation and follow-up and for any further evaluations that need to be completed. 
7.1.3 
Discontinuation of IMP due to other adverse events or medical 
condition 
Study participants MUST permanently discontinue IMP if they develop an illness that would 
interfere with his/her continued exposure to IMP including, but not limited to:  
− Study participant has a significant infective episode including but not limited to 
bacteremia or sepsis, infectious meningitis, osteomyelitis, septic arthritis, complicated 
pneumonia or visceral abscess which may or may not result in hospitalization. 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 (see Appendix 22, 
Section 10.22). 
− Study participant has new onset or recurrent neoplastic disease (except for superficial 
basal or squamous cell carcinoma of the skin not requiring targeted biological therapy, 
chemotherapy or radiation). 
− Study participant has an AE of severe or serious hypersensitivity, infusion related 
reaction (see Appendix 23, Section 10.23) or anaphylaxis requiring corticosteroid and/or 
epinephrine therapy (see Appendix 19, Section 10.19) (Sampson et al, 2006). 
− Study participant has a serious AE of headache or GI disturbance that is considered 
related to the IMP in the opinion of the investigator; or recurrence of severe AE of GI 
disturbance (see Appendix 21, Section 10.21) or headache (see Appendix 20, 
Section 10.20) that is considered related to the IMP in the opinion of the investigator. 
− Study participant has a life-threatening bleeding event. 
− Study participant has an AE of arterial or venous thromboembolic event (eg, stroke, 
myocardial infarction, pulmonary embolism, deep vein thrombosis). 
− Study participant has a tuberculosis (TB) test that is confirmed positive or any further 
evidence suggestive of potential TB infection (eg, close exposure) and further 
examinations result in a diagnosis of active TB or latent TB (LTB), or nontuberculous 
mycobacterial infection (NTMBI) (see Section 8.1.5).  
− If a NTMBI is identified during the study, the same withdrawal procedures as those used 
for an active TB infection identified during the study should be followed. 
− Study participant requires rescue therapy (for exceptions, refer to Section 6.4.3). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 59 of 199 
Study participants who permanently discontinue IMP during the Treatment Period and hence are 
withdrawn from the study should complete the assessments outlined for the EW Visit and enter 
the SFU Period. 
7.1.4 
Temporary IMP discontinuation  
Study participant MUST temporarily discontinue IMP if any of the following events occur: 
1. Thrombocytosis (platelet count of ≥400×109/L). 
2. In the event of confirmed coronavirus disease (COVID-19) infection (eg, signs/symptoms 
such as fever, cough, shortness of breath), or known exposure sufficient to necessitate 
testing or self-imposed quarantine. The IMP may be restarted if: 
a. COVID-19 test is negative, and signs and symptoms have resolved 
b. If test is not available, resolution of signs and symptoms and 14 days have passed since 
initial presentation of the clinical signs/symptoms.  
c. If asymptomatic, 14 days have elapsed since known exposure.  
Study participant MAY temporarily discontinue IMP if any of the following events occur: 
1. A severe AE of headache that is considered related to the IMP in the opinion of the 
investigator (Section 10.20).  
2. A splenectomised study participant develops a (persistent or re-occurring) nonserious 
infection, as per investigator’s decision (Section 10.22). 
3. Total serum IgG values goes <1g/L as per investigator’s decision (Section 10.22). As IMP 
treatment is administered 
 the decision is based on the most recently available total 
IgG value. In order to maintain the blind from the parent studies, IgG, IgG subclasses, total 
protein, and albumin values will be unblinded starting with the blood samples taken at 
Week 4 (Visit 5). Additionally, guidance for investigators on management of infections can 
be found in Appendix 22, Section 10.22.  
7.2 
Participant discontinuation/withdrawal from the study 
Study participants are free to withdraw from the study at any time, without prejudice to their 
continued care. Participants who withdraw from the study should complete the EOS visit. 
A study participant may also be withdrawn from the study at any time at the discretion of the 
investigator for safety, behavioral, compliance, or administrative reasons. 
If the study participant withdraws consent for disclosure of future information, the sponsor may 
retain and continue to use any data collected before such a withdrawal of consent. 
If a study participant withdraws from the study, he/she may request destruction of any samples 
taken and not tested, and the investigator must document this in the site study records. 
See the Schedule of Activities (Section 1.3) for data to be collected at the time of study 
discontinuation and follow-up and for any further evaluations that need to be completed. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 60 of 199 
Study participants must be withdrawn from the study and permanently discontinued from IMP if 
any of the following events occur: 
1. Participant withdraws his/her consent. 
2. Study participant takes prohibited concomitant medications as defined in this protocol. 
3. Study participant meets the mandatory IMP discontinuation criteria as per Section 7.1.1, 
Section 7.1.2 or Section 7.1.3. 
4. There is confirmation of a pregnancy during the study, as evidenced by a positive 
pregnancy test. 
5. The sponsor or a regulatory agency requests withdrawal of the study participant. 
Study participants who withdraw from the study or permanently discontinue IMP during the 
Treatment Period should complete the assessments outlined for the EW Visit, enter the SFU 
Period and complete the EOS Visit.  
Investigators should contact the Medical Monitor, whenever possible, to discuss the withdrawal 
of a participant in advance. Study participants who are withdrawn will not be replaced. 
7.3 
Lost to follow-up 
A study participant will be considered lost to follow-up if he or she repeatedly fails to return for 
scheduled visits and is unable to be contacted by the study site. 
The following actions must be taken if a participant fails to return to the clinic for a required 
study visit: 
• The site must attempt to contact the participant and reschedule the missed visit as soon as 
possible and counsel the participant on the importance of maintaining the assigned visit 
schedule and ascertain whether or not the 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. 
Should the participant continue to be unreachable, he/she will be considered to have withdrawn 
from the study with a primary reason of lost to follow-up documented in the eCRF. 
8 
STUDY ASSESSMENTS AND PROCEDURES 
Study procedures and their timing are summarized in the Schedule of Activities (Section 1.3). 
Protocol waivers or exemptions are not allowed. 
Immediate safety concerns should be discussed with the sponsor (or designee - Medical Monitor 
at contract research organization [CRO]) immediately upon occurrence or awareness to 
determine if the participant should continue or discontinue IMP. 
Adherence to the study design requirements, including those specified in the Schedule of 
Activities, is essential and required for study conduct. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 61 of 199 
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 Baseline purposes provided the 
procedures met the protocol-specified criteria and were performed within the time frame defined 
in the Schedule of Activities (Section 1.3). 
An Unscheduled Visit can be conducted at the discretion of the investigator (eg, due to an AE). 
During the Unscheduled Visit, the following assessments will be performed: 
• AE reporting 
• Concomitant medications  
• Review of withdrawal criteria 
• Physical examination 
• Vital signs 
• Blood samples for PK, IgG, hematology, biochemistry, other testing such as for TB or 
C-reactive protein (CRP) as clinically indicated in the opinion of the investigator 
Other assessments may be performed at the discretion of the investigator.  
Repeat or unscheduled samples may be taken for safety reasons or for technical issues with the 
samples. 
Some study-specific investigations may not be conducted according to the study protocol during 
a pandemic or other exceptional circumstances (eg, hurricanes) due to the need to implement 
safety measures and guidance from regulatory authorities. In such a situation, which may be 
accompanied by local or global containment or other measures, sites may need to prohibit access 
to study participants and study-related personnel. Study participant 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 which will be maintained by UCB for the 
respective study. The contingency measures are shared with the investigator and the respective 
study-related personnel as soon as there are indications that it is necessary to implement any of 
the measures. 
8.1 
Safety assessments 
Planned time points for all safety assessments are provided in the Schedule of Activities 
(Section 1.3). 
8.1.1 
Physical examination 
A full physical examination will include, at a minimum, assessments of general appearance; ear, 
nose, and throat; eyes, hair, and skin; assessments of the cardiovascular, respiratory, GI, 
neurological, musculoskeletal, and hepatic systems; and mental status.  
A short physical examination will include, at a minimum, assessments of general appearance; 
ear, nose, and throat; skin, respiratory, GI, and neurological systems. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 62 of 199 
For full and short physical examinations, investigators should pay special attention to clinical 
signs related to previous serious illnesses as well as signs and symptoms of infections. 
Clinically relevant findings or worsening of previous findings will be recorded as AEs. 
8.1.2 
Vital signs 
Oral, tympanic, or axillary temperature, pulse rate, and BP 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). 
Vital signs (to be taken before blood collection for laboratory tests) will include 1 pulse, 1 BP 
and body temperature measurement. 
8.1.3 
Electrocardiograms 
12-lead ECGs will be obtained as outlined in the Schedule of Activities (Section 1.3) using an 
ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc 
intervals. Refer to Section 7.1.2 for QTc withdrawal criteria and any additional QTc readings 
that may be necessary. 
Central reading of ECGs will be performed, but for the assessment of the ECGs during the visit, 
the assessment of the investigator or designee will be used to determine eligibility and 
continuation in the study. 
All ECG recordings should be taken prior to blood collection for assessment of laboratory 
parameters, with the study participant resting in the supine position for at least 5 minutes before 
the recording. 
8.1.4 
Clinical safety laboratory assessments 
See Appendix 2 (Section 10.2) for the list of clinical laboratory tests to be performed and the 
Schedule of Activities (Section 1.3) for the timing and frequency. 
The investigator must review the laboratory report, document this review, and record any 
clinically relevant changes occurring during the study in the AE section of the eCRF. The 
laboratory reports must be filed with the source documents. Clinically significant abnormal 
laboratory findings are those that 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 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 (Section 1.3). 
If laboratory values from nonprotocol-specified laboratory assessments performed at the 
institution’s local laboratory require a change in participant management or are considered 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 63 of 199 
clinically significant by the investigator (eg, SAE, AE, or dose modification), then the results 
must be recorded in the eCRF. 
8.1.5 
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. 
Any presumptive diagnosis or diagnosis of a TB infection is a reportable event. Assessment and 
management of TB and TB risk factors should follow local/national guidelines. 
8.1.5.1 
Tuberculosis assessment 
Monitoring for TB during the study 
Study participants will be monitored for signs/symptoms of TB using routine pharmacovigilance 
measures for AEs. Study participants reporting AEs related to signs/symptoms of TB will be 
evaluated for LTB and active TB according to the local medical practice guidelines. 
Confirmed LTB, active TB, and NTMBI must be reported to UCB immediately regardless of 
seriousness using the SAE Report Form. 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 EW and complete 
all EOS assessments. 
TB 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 18, Section 10.18) 
as indicated in the Schedule of Activities (Section 1.3). 
The TB questionnaire at study entry of TP0004 will not be collected. The TP0003 or TP0006 
Week 25 (Visit 27) questionnaire will be used as the study entry questionnaire for TP0004. 
The TB questionnaire be completed accurately and filed as a critical source document. The 
questionnaire will assist with the identification of participants who may require therapy for TB. 
A “Yes” response to any of the questions in the TB questionnaire may trigger further 
assessments to determine if the participant has either latent TB infection (LTBI) or active TB 
infection and, if confirmed, must be withdrawn from the study. As an example, a participant who 
answered “Yes” to the question “
 
 
should not be allowed into the study pending further assessments per local/national 
guidelines. 
8.1.5.2 
Tuberculosis management 
In line with the World Health Organization (WHO) and the Centers for Disease Control and 
Prevention (CDC) guidelines, UCB has adopted the following definitions for latent TB, active 
TB and non-tuberculosis mycobacterium infection for the purposes of its clinical trials. 
Latent TB 
Latent TB infection is defined an infection by Mycobacterium tuberculosis with: 
• a positive IGRA test or 2 indeterminate IGRA tests AND 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 64 of 199 
• a chest x-ray [or other imaging]) negative for TB infection AND 
• the absence of signs, symptoms (eg, evidence of organ specific involvement, or physical 
findings) suggestive of TB infection. 
Active TB and non-tuberculosis mycobacterium infection 
A clinically verified case of TB meets the following criteria: 
• a positive Tuberculin Skin Test (TST) or positive IGRA for Mycobacterium tuberculosis  
• other signs and symptoms compatible with TB (eg, abnormal chest radiograph, abnormal 
chest computerized axial tomography scan or other chest imaging study, or other clinical 
evidence of active disease) 
Nontuberculous mycobacterium infection (NTMBI) is defined as a clinical infection caused by 
mycobacterial species other than those belonging to the Mycobacterium tuberculosis complex. 
Study participants who develop active TB or NTMBI during the study must be withdrawn from 
the study. The participant must be permanently discontinued from study medication and the EW 
Visit must be scheduled as soon as possible, but no later than the next scheduled visit.  
Participants should be encouraged to enter the SFU Period, and keep the EOS Visit as specified 
by the protocol. Treatment for active TB or NTMBI should be started immediately based on 
local guidelines. 
Confirmed active TB is always considered an SAE. UCB’s process requires that this must be 
captured on an SAE report form and provided to UCB in accordance with SAE reporting 
requirements. Follow-up reports should be completed as per protocol requirement until such time 
as the TB infection resolves. 
LTBI, active TB, or other NTMBI identified during study 
During the study, study participants who develop evidence of LTBI, active TB or NTMBI must 
immediately stop further administration of IMP and will be referred to an appropriate TB 
specialist (pulmonologist or infectious disease specialist) for further evaluation. Participants 
diagnosed with active TB treatment or LTBI should receive appropriate TB treatment or 
prophylaxis therapy. The participant should be transferred to the care of their physician and 
managed according to the standard of care. 
If an NTMBI is identified during the study, the same procedure as for active TB acquired during 
the study and compliant TB treatment shall be followed. 
Follow-up information of suspected and confirmed TB cases should be provided to UCB at least 
after 3, 9, and 12 months of the start date of anti-TB treatment, including hematological and 
biochemical safety parameters, x-ray evolution data, and TB diagnostic procedures used to 
follow-up and confirm recovery of TB. 
8.1.6 
Splenectomized study participants 
Asplenic study participants will be required to carry a splenectomy card for the whole duration 
of the study indicating they have undergone splenectomy and may be at increased risk of 
infection particularly with encapsulated organisms. Prophylactic antibiotic may be given to these 
participants, if required per local guidance. Antibody titers against S. pneumoniae, N. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 65 of 199 
meningitidis, and H. influenzae in splenectomized study participants will be measured at the 
defined time points as specified in the Schedule of Activities. 
Splenectomized study participants should have documented evidence of vaccinations from the 
parent studies against the following encapsulated organisms: S. pneumoniae, N. meningitidis, and 
H. influenzae (as per local/or a national guidance, as applicable) or as evidenced from personal 
immunization records. 
8.2 
Adverse events 
The definitions of an AE or SAE can be found in Appendix 3, Section 10.3. 
Adverse events can be reported by the participant (or, when appropriate, by a caregiver, 
surrogate, or the participant's legally authorized representative). 
The investigator and any designees are responsible for detecting, documenting, and recording 
events that meet the definition of an AE or SAE and remain responsible for following up AEs 
that are serious, considered related to the IMP or study procedures, or that caused the participant 
to discontinue rozanolixizumab or TP0004 (see Section 7). 
8.2.1 
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 EOS Visit at the time 
points specified in the Schedule of Activities (Section 1.3). 
All SAEs will be recorded and reported to the sponsor or designee within 24 hours, as indicated 
in Appendix 3, Section 10.3. The investigator will submit any updated SAE data to the sponsor 
within 24 hours of it being available. 
The investigator is specifically requested to collect and report to UCB (or its representative) any 
SAEs (even if the investigator is certain that they are in no way associated with the IMP), 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 IMP 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. 
8.2.2 
Method of detecting AEs and SAEs 
Care will be taken not to introduce bias when detecting AEs and/or SAEs. Open-ended and 
non-leading verbal questioning of the participant is the preferred method to inquire about 
AE occurrences. 
8.2.3 
Follow-up of AEs and SAEs 
After the initial AE/SAE report, the investigator is required to proactively follow each participant 
at subsequent visits/contacts. All SAEs and nonserious AEs of special interest (as defined in 
Section 8.2.7) 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. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 66 of 199 
8.2.4 
Regulatory reporting requirements for SAEs 
Prompt notification by the investigator to the sponsor of a SAE is essential so that legal 
obligations and ethical responsibilities towards the safety of participants and the IMP under 
clinical investigation are met. 
UCB has a legal responsibility to notify both the local regulatory authority and other regulatory 
agencies about the safety of the IMP under clinical investigation. UCB 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 
(SUSAR) according to local regulatory requirements and sponsor policy and forwarded to 
investigators as necessary. 
An investigator who receives an investigator safety report describing a SAE or other specific 
safety information (eg, summary or listing of SAEs) from the sponsor will review and then file it 
along with the IB and will notify the IRB/IEC, if appropriate according to local requirements. 
8.2.5 
Pregnancy 
Details of all pregnancies in female participants and, if indicated, female partners of male 
participants will be collected after the start of IMP and until 90 days after the final dose. 
If a pregnancy is reported, the investigator must immediately inform UCB 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 IMP. 
• The study participant should return for the EW Visit and complete the SFU Period. 
Abnormal pregnancy outcomes (eg, spontaneous abortion, fetal death, stillbirth, congenital 
anomalies, ectopic pregnancy) are considered SAEs. 
8.2.6 
Anticipated serious adverse events 
The following anticipated SAEs are anticipated to occur in the population studied in this protocol 
at some frequency that is independent of drug exposure (Table 8-1). 
This list does not change the investigator’s obligation to report all SAEs (including anticipated 
SAEs) as detailed in Section 8.2.1 and Appendix 3, Section 10.3. 
Table 8-1: 
Anticipated serious adverse events for ITP population 
Anemia 
Hemorrhagic events 
Fatigue 
Thrombocytopenia 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 67 of 199 
8.2.7 
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/compound. 
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, 
must ALWAYS be reported to UCB as an AE of special interest (ie, without waiting for any 
additional etiologic investigations to have been concluded). Follow-up information should then 
be reported if an alternative etiology is identified during investigation and monitoring of the 
participant. 
See Appendix 5, Section 10.5 for further information on liver safety monitoring. 
8.2.8 
Adverse events of special monitoring 
For rozanolixizumab, AESM that require immediate reporting (within 24 hours regardless of 
seriousness) to UCB are: 
• Severe headache 
• Severe GI disorders (ie, diarrhea, abdominal pain, vomiting) 
• Opportunistic infection 
• Arterial and venous thrombotic and thromboembolic events 
An AESM is not necessarily a serious adverse event unless one of the seriousness criteria 
defined in the Appendix 3 (Section 10.3) is met. All AESM will follow the SAE recording and 
reporting procedures as indicated in Appendix 3, Section 10.3. 
In case of moderate or severe headache or serious headache (regardless of severity), the 
headache questionnaire (Appendix 17, Section 10.17) must be completed, though moderate 
headache is not considered an AESM. Additional procedures for management of headaches are 
provided in Appendix 20, Section 10.20. 
Procedures for the management of diarrhea is provided in Appendix 21, Section 10.21. 
Although hypersensitivity reactions including infused-related reactions and anaphylaxis are not 
classified as AESM, these AEs will be monitored by the investigators. If such an event is 
suspected it should be managed according the guidance provided in Appendix 23, Section 10.23. 
In case of suspected anaphylaxis, the Sampson Criteria (Sampson et al, 2006) (Appendix 19, 
Section 10.19) should be completed. All hypersensitivity reactions will be recorded and reported 
as per the AE reporting procedure outlined in Appendix 3, Section 10.3. 
8.2.9 
COVID-19 vaccination 
COVID-19 vaccines will be recorded on the concomitant medication eCRF page (Section 6.4.1). 
If an AE is considered related to COVID-19 vaccine, causality assessment should be entered on 
the AE eCRF (in the AE eCRF page, there is the possibility to assess causality to the IMP or to 
any concomitant medication). Note that in this case, the national recommendation for reporting 
adverse event related to COVID-19 vaccines should be followed. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 68 of 199 
If an AE is the result of an interaction of a COVID-19 vaccine with the IMP in the clinical study 
then the causal association should be for both IMP and COVID-19 vaccine. In case of a 
seriousness criteria, the SUSAR process will be followed. 
8.3 
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 IMP so that investigators, clinical study participants, regulatory 
authorities, and IRBs/IECs will be informed appropriately and as early as possible. 
In addition, an IDMC will periodically review and monitor safety data from this study and advise 
UCB. Details are provided in the IDMC Charter. 
The Study Physician or medically qualified designee/equivalent will conduct an ongoing review 
of SAEs and perform ongoing SAE reconciliations in collaboration with the Patient Safety 
representative. 
As appropriate for the stage of development and accumulated experience with the IMP, 
medically qualified personnel at UCB may identify additional safety measures (eg, AEs, vital 
signs, laboratory, or ECG results) for which data will be periodically reviewed during the course 
of the study. 
8.4 
Treatment of overdose 
For this study, any dose increase of 10% or greater than the assigned dose for each administered 
dose of IMP will be considered an overdose, irrespective of the weight tier band. Overdose 
events are only considered AEs or SAEs if there are associated clinical signs and symptoms or if 
the act of taking the excess IMP itself is an AE or SAE (eg, suicide attempt). 
UCB does not recommend specific treatment for an overdose. 
In the event of an overdose, the investigator/treating physician should: 
1. Contact the Medical Monitor immediately. 
2. Closely monitor the participant for any AE/SAE and laboratory abnormalities for at least 
5 days. 
3. Obtain a plasma sample for PK analysis within 3 days from the date of the final dose of 
IMP if requested by the Medical Monitor (determined on a case-by-case basis). 
4. Document the quantity of the excess dose as well as the duration of the overdose in the 
eCRF. 
Decisions regarding dose interruptions or modifications will be made by the investigator in 
consultation with the Medical Monitor based on the clinical evaluation of the participant. 
8.5 
Efficacy assessments 
8.5.1 
Platelet counts 
As in the parent studies, TP0003 and TP0006, for assessment of platelet counts, blood samples 
will be collected by qualified site personnel at the same time that samples are collected for 
standard clinical laboratory assessments. The time and date of the blood draws will be recorded 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 69 of 199 
in medical source data and the eCRF. Assessment will be performed according to the Schedule 
of Activities (Section 1.3). 
Platelet counts will be determined at local laboratories and the following endpoints will be 
derived for the purpose of analysis. 
Platelet count-related endpoints are: 
• Stable Clinically Meaningful Response, defined as Clinically Meaningful Response (ie, 
platelet count ≥50×109/L) without rescue therapy at ≥70% of the visits over the planned 52-
week Treatment Period starting at Week 4 
• Stable Response, defined as platelet count ≥30×109/L and absence of bleeding without rescue 
therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4 
• Cumulative number of weeks with Clinically Meaningful Response over the planned 52-
week Treatment Period 
• Cumulative number of weeks with platelet counts ≥30×109/L over the planned 52-week 
Treatment Period 
• Mean Change from Baseline in platelet count at each visit 
• Use of rescue medication by visit 
• ITP-BAT bleeding events and severity by visit 
Platelet endpoints in study participants with first exposure to rozanolixizumab: 
• In study participants with first exposure to rozanolixizumab: Durable Clinically Meaningful 
Platelet Response of ≥50×109/L, for at least 4 of 6 
visits between Weeks 13 to 25 
of the study. 
• In study participants with first exposure to rozanolixizumab: Time to first Clinically 
Meaningful Platelet Response of ≥50×109/L: time from starting treatment to achievement of 
first response of ≥50×109/L. 
The clinical response variables will be assessed only for visits for which both platelet counts and 
the ITP bleeding score are assessed (with the exception of the confirmatory platelet assessments, 
which may be obtained at any visit [scheduled or unscheduled] provided that they meet the 
criteria below). In order to define a Response (platelet count ≥30×109/L), the platelet count must 
be confirmed on 2 consecutive occasions and in the absence of bleeding. The time to first 
Response will be taken as the time to the first platelet assessment (obtained at the same time as 
the corresponding ITP bleeding score assessment). If the second assessment does not fulfill the 
required criteria for a Clinical Response, the study participant will be considered as a 
nonresponder at the respective visits. Further details will be provided in the Statistical Analysis 
Plan (SAP). 
8.5.2 
ITP bleeding score 
The International Working Group on ITP proposes a consensus-based ITP-BAT, based on a 
precise definition of bleeding manifestations and on the grading of their severity (Rodeghiero et 
al, 2013). The ITP bleeding score will be assessed using the ITP-BAT tool Version 1.0. This 
assessment will be performed according to the Schedule of Activities (Section 1.3). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 70 of 199 
For the ITP-BAT, bleeding manifestations were grouped into 3 major domains: skin (S), visible 
mucosae (M), and organs (O), with gradation of severity (SMOG). Each bleeding manifestation 
is assessed at the time of examination. Severity is graded from 0 to 3 or 4, with Grade 5 for any 
fatal bleeding. Bleeding reported by the participant without medical documentation is Grade 1. 
Within each domain, the same grade is assigned to bleeding manifestations of similar clinical 
impact. The “worst” bleeding manifestation since the last visit is graded, and the highest grade 
within each domain is recorded. The SMOG system provides a consistent description of the 
bleeding phenotype in ITP. 
A standardized data collection form will be used to facilitate collection of information and 
communication among physicians and investigators. The grading of bleeding symptoms at 
presentation and at each subsequent evaluation is presented in Appendix 10, Section 10.10. 
8.5.3 
Patient Reported Outcomes 
Patient reported outcomes must be completed as per time points mentioned in the Schedule of 
Activities (Section 1.3). The PROs should be completed prior to any intrusive procedures in a 
quiet place. On dosing days, PROs will be completed prior to dosing. 
The PROs should be completed in the following order: ITP-PAQ, SF-36, the FATIGUE-PRO 
Physical Fatigue Scale, EQ-5D-5L, PGI-S, and PGI-C. 
8.5.3.1 
ITP-PAQ 
The ITP-PAQ is a 44-item disease-specific quality of life (QoL) questionnaire developed for use 
in adults with chronic ITP. It includes 11 scales: Symptoms, Fatigue, Physical Health – Bother, 
Physical Health - Activity, Emotional Health – Psychological, Emotional Health – Fear, Overall 
QoL, Social Activity, Women’s Reproductive Health – Fertility, Women’s Reproductive 
Health - Menstrual Symptoms, and Work. Each item is rated on a Likert-type scale containing 
4 to 7 responses. All item scores are transformed to a 0 to 100 continuum and are weighted 
equally to derive individual scale scores. Higher scores indicate better health status 
(Mathias et al, 2009; Mathias et al, 2007). 
The ITP-PAQ Symptoms score was selected as secondary efficacy endpoint for the study to 
capture the benefits of rozanolixizumab in terms of patient-perceived symptoms. It covers 6 core 
symptoms of ITP: bruising/petechiae, wounds/scars (from blood tests, injections or iv needles), 
blood blisters in mouth, bleeding episodes, muscle aches, and cramps in legs. 
The ITP-PAQ sample questionnaire is available in Appendix 11, Section 10.11. 
8.5.3.2 
SF-36 
Study participants will complete the SF-36 questionnaire according to the Schedule of Activities 
(Section 1.3). The SF-36 sample questionnaire is available in Appendix 12, Section 10.12. 
8.5.3.3 
FATIGUE-PRO Physical Fatigue Scale 
The FATIGUE-PRO Physical Fatigue Scale is one of the three scales of the broader FATIGUE-
PRO instrument developed by UCB. It consists of 9 items rated on a 5-point frequency response 
scale ranging from “none of the time” to “all of the time”, with a 7-day recall period. The 
FATIGUE-PRO Physical Fatigue score ranges 0 to 100, with a higher score meaning more 
physical fatigue.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 71 of 199 
The Physical Fatigue is available in Appendix 13, Section 10.13. 
8.5.3.4 
EQ-5D-5L 
The EQ-5D-5L is designed to improve the instrument’s sensitivity and to reduce ceiling effects. 
The EQ-5D-5L consists of 2 pages: the EQ-5D descriptive system and the EuroQoL visual 
analog scale (EQ VAS). 
The descriptive system comprises 5 dimensions: mobility, self-care, usual activities, 
pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight 
problems, moderate problems, severe problems, and extreme problems. The study participant is 
asked to indicate his/her health state by ticking the box next to the most appropriate statement in 
each of the 5 dimensions. This decision results in a 1-digit number that expresses the level 
selected for that dimension. The digits for the 5 dimensions can be combined into a 5-digit 
number that describes the study participant’s health state. 
The EQ VAS records the study participant’s self-rated health on a vertical visual analog scale 
(VAS), where the endpoints are labeled ‘The best health you can imagine’ and ‘The worst health 
you can imagine’. The VAS can be used as a quantitative measure of health outcome that reflect 
the study participant’s own judgment. 
Study participants will complete the EQ-5D-5L questionnaire according to the Schedule of 
Activities (Section 1.3). The EQ-5D-5L sample questionnaire is available in Appendix 14, 
Section 10.14. 
8.5.3.5 
PGI-S 
The PGI-S is a single-state, self-report measure that rates a study participant's severity of a 
specific condition. The PGI-S is a 5-point scale depicting a study participant's rating of overall 
symptoms (“none,” “mild,” “moderate,” “severe,” or “very severe”). The PGI-S sample 
questionnaire is available in Appendix 15, Section 10.15. 
8.5.3.6 
PGI-C 
The PGI-C is a single-state, self-report measure that reflects a study participant's belief about the 
efficacy of treatment for a specific condition. The PGI-C is a 7-point scale depicting a study 
participant's rating of overall improvement (“very much improved,” “much improved,” 
“minimally improved,” “no change,” “minimally worse,” “much worse,” or “very much worse”). 
The PGI-C sample questionnaire is available in Appendix 16, Section 10.16. 
8.5.3.7 
SIAQ (Infusion version) 
To assess the experience of study participants with the sc self-administration using an infusion 
device, an adaptation of the SIAQ will be used in the participants that self-administer.  
The SIAQ was developed by UCB to assess the perceived advantages and the potential 
limitations of self-administration of an sc medication (Keininger and Coteur, 2011). There are 2 
modules for the SIAQ (SIAQ PRE-Self-Injection and SIAQ POST-Self-Injection). The SIAQ 
PRE-Self-Injection is composed of 7 items grouped into 3 domains (feelings about injection, 
self-confidence, and satisfaction with the current mode of administration). The SIAQ POST-
Self-Injection is composed of 21 items grouped into 6 domains (feelings about injection, self-
image, self-confidence, injection site reactions, ease of use, and satisfaction with self-injection). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 72 of 199 
Each domain score of the SIAQ PRE-Self-Injection and SIAQ POST-Self-Injection ranges from 
0 to 10, 0 being the worst experience and 10 being the best experience.  
The adaptation of the SIAQ consists in replacing the terminology “injection” with the 
terminology “infusion” in both modules, and removing the “ease of use” domain which is not 
accurate for the self-administration method used in the study from the SIAQ POST-Self-
Injection module. This adapted version is named SIAQ (Infusion version). The domain scores of 
the SIAQ (Infusion version) are calculated the same way as the SIAQ domain scores; hence 
domain scores ranges from 0 to 10, 0 being the worst experience and 10 being the best 
experience. 
The PRE-SIAQ (Infusion version) will be completed once before the first self-administration, 
and the POST-SIAQ (Infusion version) will be completed within 1 hour after self-administration 
at consecutive visits. Paper versions will be used. The sample questionnaires are available in 
Appendix 24, Section 10.24.  
8.6 
Pharmacokinetics and antidrug antibodies 
Whole blood samples will be collected for measurement of plasma concentrations of 
rozanolixizumab and ADA as specified in the Schedule of Activities (Section 1.3). Blood 
samples may be collected at additional time points during the study if warranted and agreed upon 
between the investigator and the sponsor. Instructions for the collection and handling of 
biological samples will be provided in the laboratory manual for this study. The actual date and 
time (24-hour clock time) of each sample will be recorded. 
Samples will be used to evaluate the PK of rozanolixizumab and ADA and may be used for 
establishing assay parameters (eg, ADA cut point setting and PK selectivity assessment). 
Samples collected for analyses of rozanolixizumab concentration may also be used to evaluate 
safety or efficacy aspects related to concerns arising during or after the study. 
Participant confidentiality will be maintained. At visits during which whole blood samples for 
the determination of rozanolixizumab PK and ADA will be taken, one sample of sufficient 
volume can be used. 
Any changes in the timing or addition of time points for any planned study assessments must be 
documented and then archived in the sponsor and site study files, but will not constitute a 
protocol amendment. The IRB/IEC will be informed of any safety issues that require alteration 
of the safety monitoring scheme or amendment of the ICF. 
8.7 
Pharmacodynamics 
Venous blood samples will be collected at time points specified in the Schedule of Activities 
(Section 1.3) for measurement of: 
• Serum IgG and IgG subclasses concentrations 
• Serum IgA, IgE, and IgM concentrations 
• Serum ITP-specific autoantibodies 
For all PD assessments, blood samples will be collected predose. Instructions pertaining to 
sample collection, processing, storage, labeling, and shipping are provided in the laboratory 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 73 of 199 
manual for this study. Results of the ITP-specific autoantibodies analyses may not be outlined in 
the Clinical Study Report for this study, but in a separate report. 
8.8 
Exploratory biomarkers 
Collection of samples for exploratory biomarker research is also part of this study. Blood 
samples for biomarker research are required and will be collected from all study participants in 
this study as specified in the Schedule of Activities (Section 1.3). Exploratory samples will be 
collected predose. Additional exploratory biomarker samples may be collected 4 hours postdose 
in case of severe and/or serious headache or GI-related AESM as specified in the Schedule of 
Activities (Section 1.3). 
Protein and metabolites biomarkers such as but not limited to albumin, B-cell activating factor, 
and circulating immune complexes may be measured to assess the effect of rozanolixizumab on 
exploratory biomarkers, and explore the relationship between protein and metabolite biomarkers 
and cause, progression, and appropriate treatment of ITP. 
If not used immediately, these samples will be stored at -80°C for up to 20 years for later 
exploratory analyses. Any exploratory biomarker will only ever be related to the exploration of 
cause, progression, and appropriate treatment of ITP. They may also be used to develop 
tests/assays including diagnostic tests related to rozanolixizumab and/or FcRn inhibitor and ITP. 
The nature and format of these tentative additional analyses will be determined at a later time. 
Details on the collection, storage, preparation, and shipping of samples will be presented in the 
laboratory manual provided separately. Detailed information on sample analyses will be 
provided in a bioanalytical report. Results of the DNA and RNA biomarker analyses will not be 
outlined in the Clinical Study Report for this study. 
8.9 
Immunology biomarkers 
Blood samples for immunological testing are required and will be collected from all study 
participants in this study as specified in the Schedule of Activities (Section 1.3) for measurement 
of: 
• Serum complements (C3, C4) 
• Plasma complements (C3a, C5a)  
• Biomarkers of COVID-19 vaccine response (only for study participants who received 
COVID-19 vaccine) 
Samples for serum complements (C3, C4) and plasma complements (C3a, C5a) will be collected 
predose at Baseline (Day 1) for all participants. Additional samples may be collected 2 hours and 
4 hours postdose for study participants with an infusion reaction or hypersensitivity reaction as 
specified in the Schedule of Activities (Section 1.3). 
Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
8.10 
Medical resource utilization and health economics 
Medical resource utilization will be collected by capturing number and length of hospitalizations 
and by capturing number of infusion center admissions. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 74 of 199 
9 
STATISTICAL CONSIDERATIONS 
A description of statistical methods follows and will be described in more detail in the SAP. 
9.1 
Definition of analysis sets 
• Enrolled Set: All study participants who have signed the informed consent. 
• Safety Set (SS): All study participants who received at least 1 dose of IMP (partial or full). 
Analysis of this set will be according to the treatment the participants actually received, and 
will be used for the for the efficacy, demographic, PK and safety analyses. 
• Pharmacokinetic Per-Protocol Set (PK-PPS): A subset of the Safety Set, consisting of those 
study participants who received at least 1 dose, and had at least 1 valid PK measurement. 
9.2 
General statistical considerations 
All analyses will be performed using SAS® version 9.2 or later (SAS Institute, Cary, NC, USA). 
Continuous variables will be summarized with statistics including the number of participants (n), 
mean, standard deviation, median, minimum, and maximum. Categorical variables will be 
summarized with frequency counts and percentages. 
Data listings containing all documented data and all calculated data will be generated. 
Baseline will be the last non-missing data collected prior to the dose of IMP, and 
measurement-specific Baseline values will be defined in the SAP. 
Data handling conventions for data affected by COVID-19 will be detailed fully in the SAP. 
9.3 
Estimands 
The aim of TP0004 is to investigate the safety (primarily) of long term treatment of 
rozanolixizumab.  
The primary safety analysis is based on a “treatment policy” estimand to allow for the inclusion 
of study participants that receive rescue medication. This estimand will be based on the SS who 
started TP0004 on 
 dosing. 
There is no analysis strategy to protect the type I error as there is no significance testing amongst 
the primary and secondary endpoints. 
The primary safety estimand will be defined as follows: 
• Population: participants with primary ITP that meet the protocol-specified 
inclusion/exclusion criteria and who started TP0004 on 
 dosing. 
• Participant-level outcome: occurrence of TEAEs. 
• Population-level summary measure: descriptive summaries. 
• Intercurrent event handling: The main intercurrent events are the use of rescue medication 
during the course of the study and withdrawal due to TEAEs. 
A further secondary safety estimand, also based on “treatment policy”, will use the SS who 
started TP0004 on 
dosing. It is defined as follows: 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 75 of 199 
• Population: participants with primary ITP that meet the protocol-specified 
inclusion/exclusion criteria and who started TP0004 on 
dosing. 
• Participant-level outcome: occurrence of TEAEs. 
• Population-level summary measure: descriptive summaries. 
• Intercurrent event handling: The main intercurrent events are the use of rescue medication 
during the course of the study and withdrawal due to TEAEs. 
Missing safety values will not be imputed. 
Further details will be provided in the SAP. 
9.4 
Planned safety analyses 
The frequency and severity of all TEAEs will be presented by system organ class, high level 
term, and preferred term (Medical Dictionary for Regulatory Activities [MedDRA]®). The data 
will be displayed as number of participants experiencing the TEAE, percentage of participants, 
and number of TEAEs. A TEAE is defined as any event that was not present prior to the first 
administration of IMP or any unresolved event already present before the first administration of 
IMP that worsens in intensity following exposure to treatment, up to and including 8 weeks after 
the final dose of IMP. 
Laboratory evaluations, ECGs, and vital signs will be analyzed over time. 
All safety analyses will be based on the SS and be presented by both the treatment the 
participants received during the parent studies (TP0003/TP0006), and whether they started 
TP0004 on 
 dosing. 
9.5 
Planned efficacy/outcome analyses 
Data will be listed, and for continuous variables, descriptive statistics will be generated for the 
observed values and changes from Baseline. For categorical variables, frequency counts and 
percentages will be presented.  
All summary outputs will be presented by the treatment the participants received during the 
parent studies (TP0003/TP0006) and whether they started TP0004 on 
 
dosing. 
The key secondary endpoint in TP0004 for efficacy is stable clinically meaningful response 
defined as Clinically Meaningful Response (≥50×109/L platelet count) without rescue therapy at 
≥70% of the visits starting at Week 4, as it is anticipated that the effect of any rescue medication 
that might have been administered to the study participants in the placebo arm close to the end of 
the parent study would have dissipated at the time of assessing the stable clinically meaningful 
response in TP0004.  
The other platelet count-related efficacy endpoints are Stable Response (defined as platelet count 
≥30×109/L and absence of bleeding without rescue therapy) at ≥70% of the visits starting at 
Week 4; cumulative number of weeks with Clinically Meaningful Response; cumulative number 
of weeks with platelet counts ≥30×109/L and mean Change from Baseline in platelet count. 
These efficacy endpoints will be measured over time (52 weeks of treatment) in the same manner 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 76 of 199 
as the key secondary endpoint. The Baseline platelet count is defined as platelet count at Visit 1 
(TP0004). 
Cumulative number of weeks with Clinically Meaningful Response and cumulative number of 
weeks with platelet counts ≥30×109/L will be also be presented for the first 24 weeks of the 
study. 
Platelet count data will be presented both as collected, and with imputation as follows: Missing 
platelet count at any visit will be considered "worst case" and set to zero ("no platelet response"). 
The same will apply to missing platelet count at any visit after the initiation of rescue therapy. 
Baseline platelet count for each endpoint will be defined in the SAP. Rescue therapy given to 
participants who received placebo in the parent studies to ensure platelet count >30×109/L will 
be summarized.  
Subgroup analyses split by rescue medication use in TP0003 and TP0006 may be conducted. 
Further details will be provided in the SAP. 
9.6 
Planned other analyses 
9.6.1 
Analysis of pharmacodynamics endpoints 
The analyses (potentially including, but not limited to albumin, and total IgG, IgG subclasses, 
IgA, IgE, IgM, serum complement, plasma complement, and ITP specific autoantibodies) will be 
presented as continuous variables. All PD and biomarker analyses will be based on the SS and by 
dosing regimen.  
9.6.2 
Analysis of pharmacokinetics endpoints 
All PK analyses will be based on the PK dataset. 
Individual concentrations of rozanolixizumab will be listed and may be summarized at each 
scheduled time point and may be stratified by ADA status and dose regimen when applicable. 
Descriptive statistics of concentrations will be calculated only if at least two-thirds of the 
individual data points are quantifiable at a certain time point (≥ lower limit of quantification 
[LLOQ]). For summary statistics, below the LLOQ will be represented as LLOQ/2. Individual 
concentrations may be depicted graphically as individual plots and/or spaghetti plots. The impact 
of ADA on rozanolixizumab concentrations will be evaluated. 
Further details will be provided in the SAP. 
9.6.3 
Antidrug antibodies analyses 
A tiered ADA approach will be used for the study. Samples will first be evaluated in the 
screening assay using a false positivity rate of 5% (reported as negative screen or positive 
screen), followed by analysis of screened positive samples in the confirmatory assay (which is a 
drug depletion assay) to confirm the true positivity of the samples (reported as negative 
immunodepletion or positive immunodepletion). Samples that are confirmed as positive will be 
evaluated in a titration assay to quantify the ADA level and will be reported as titer (reciprocal 
dilution factor including MRD). For anti-rozanolixizumab antibody positive samples (or subset 
of), further characterization for neutralizing ADA potential in vitro will be performed, and 
results will be presented in a listing. Full details will be provided in the SAP. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 77 of 199 
9.7 
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. 
9.8 
Handling of dropouts or missing data 
All imputation of missing or partial dates for safety assessments, as well as handling missing 
efficacy data (where applicable), will be detailed in the SAP. 
9.9 
Planned interim analysis, data monitoring, and data snapshots 
9.9.1 
Interim analysis 
No formal interim analysis is planned for this study. 
In addition, based on data cutoffs, safety and efficacy data for submission purposes will be 
summarized. 
9.9.2 
Data monitoring 
An external IDMC will be established to review the safety data at predefined intervals and ad 
hoc as needed, should any emerging safety concern arise during the study. Details of the IDMC 
composition, processes, and responsibilities will be documented in the IDMC charter. Key safety 
data may be pooled at the discretion of the IDMC for protection of all study participants if 
warranted. 
The IDMC will consist of members independent from UCB. Study enrollment will not be halted 
during planned IDMC review of the safety and efficacy data. The objectives and procedures for 
the IDMC will be detailed in the IDMC Charter. 
9.9.3 
Data snapshots 
There will be periodic data snapshots for submission and market approval. 
9.10 
Determination of sample size 
No formal sample size calculation can be performed. All participants from the parent studies 
(TP0003 and TP0006) eligible for the OLE will be included. It is expected that up to a maximum 
of 180 participants will be included. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 78 of 199 
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 (ICH)-Good Clinical Practice (GCP), and in accordance 
with the ethical principles that have their origin in the Declaration of Helsinki. 
The investigator/UCB will ensure that an appropriately constituted IRB/IEC that complies with 
the requirements of the current ICH-GCP version or applicable country-specific regulations will 
be responsible for the initial and continuing review and approval of the clinical study. Prior to 
initiation of the study, the investigator/UCB will forward copies of the protocol, ICF, IB, 
investigator’s curriculum vitae (if applicable), advertisement (if applicable), and all other 
participant-related documents to be used for the study to the IRB/IEC for its review and 
approval. 
Before initiating a study, the investigator will have written and dated full approval from the 
responsible IRB/IEC for the protocol. 
The investigator will also promptly report to the IRB/IEC all changes in the study, all 
unanticipated problems involving risks to participants or others, and any protocol deviations, to 
eliminate immediate hazards to participants. 
The investigator will not make any changes in the study or study conduct without IRB/IEC 
approval, except where necessary to eliminate apparent immediate hazards to the participants. 
For minor changes to a previously approved protocol during the period covered by the original 
approval, it may be possible for the investigator to obtain an expedited review by the IRB/IEC as 
allowed. 
As part of the IRB/IEC requirements for continuing review of approved studies, the investigator 
will be responsible for submitting periodic progress reports to the IRB/IEC (based on IRB/IEC 
requirements), at intervals appropriate to the degree of participant risk involved, but no less than 
once per year. The investigator should provide a final report to the IRB/IEC following study 
completion. 
UCB (or its representative) will communicate safety information to the appropriate regulatory 
authorities and all active investigators in accordance with applicable regulatory requirements. 
The appropriate IRB/IEC will also be informed by the investigator or the sponsor, as specified by 
the applicable regulatory requirements in each concerned country. Where applicable, 
investigators are to provide the sponsor (or its representative) with evidence of such IRB/IEC 
notification. 
10.1.2 
Financial disclosure 
Insurance coverage will be handled according to local requirements. 
Finance and insurance are addressed in the investigator and/or CRO agreements, as applicable. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 79 of 199 
10.1.3 
Informed consent process 
Participant’s informed consent must be obtained and documented in accordance with local 
regulations, ICH-GCP requirements, and the ethical principles that have their origin in the 
principles of the Declaration of Helsinki. 
Prior to obtaining informed consent, information should be given in a language and at a level of 
complexity understandable to the participant in both oral and written form by the investigator (or 
designee). Each participant will have the opportunity to discuss the study and its alternatives 
with the investigator. 
Prior to participation in the study, the ICF should be signed and personally dated by the study 
participant and by the person who conducted the informed consent discussion (investigator or 
designee) at the site. The study participant 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 UCB, 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 United States must include the use of a Health Insurance 
Portability and Accountability Act Authorization form. 
The participant may withdraw his/her consent to participate in the study at any time. A 
participant is considered as enrolled in the study when he/she has signed the ICF. An eCRF must 
not be started, nor may any study specific procedure be performed for a given participant, 
without having obtained his/her written consent to participate in the study. 
10.1.4 
Data protection 
UCB staff (or designee) will affirm and uphold the participant’s confidentiality. Throughout this 
study, all data forwarded to UCB (or designee) will be identified only by the participant number 
assigned at Screening of the parent study (TP0003 or TP0006). 
The investigator agrees that representatives of UCB, its designee, representatives of the relevant 
IRB/IEC, or representatives of regulatory authorities will be allowed to review that portion of the 
participant’s primary medical records that directly concerns this study (including, but not limited 
to, laboratory test result reports, ECG reports, admission/discharge summaries for hospital 
admissions occurring during a participant’s study participation, and autopsy reports for deaths 
occurring during the study). 
The participant must be informed that his/her personal study-related data will be used by the 
sponsor in accordance with local data protection law. The level of disclosure must also be 
explained to the participant.  
The participant must be informed that his/her medical records may be examined by Clinical 
Quality Assurance auditors or other authorized personnel appointed by the sponsor, by 
appropriate IRB/IEC members, and by inspectors from regulatory authorities. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 80 of 199 
10.1.5 
Committees structure 
An external IDMC will be established to mainly review the safety data at predefined intervals 
and ad hoc as needed, should emerging safety concerns arise during the study. The IDMC will 
have the possibility to unblind the data. Details of the IDMC composition, processes, and 
responsibilities will be documented in the IDMC Charter. 
10.1.6 
Data quality assurance 
All participant data relating to the study will be recorded in the eCRF. The investigator is 
responsible for verifying that data entries are accurate and correct by electronically signing the 
eCRF. 
The investigator must maintain accurate documentation (source data) that supports the 
information entered in the eCRF. 
The investigator must permit study-related monitoring, audits, IRB/IEC review, and regulatory 
agency inspections and provide direct access to source data documents. 
The sponsor or designee is responsible for the data management of this study including quality 
checking of the data. 
Study monitors will perform ongoing source data verification to confirm that data entered into 
the eCRF by authorized site personnel are accurate, complete, and verifiable from source 
documents; that the safety and rights of participants are being protected; and that the study is 
being conducted in accordance with the currently approved protocol and any other study 
agreements, ICH-GCP, and all applicable regulatory requirements. 
All essential documents are to be retained by the investigator until at least 2 years after the last 
approval of a marketing application in an ICH region and until there are no pending or 
contemplated marketing applications in an ICH region, or at least 2 years have elapsed since the 
formal discontinuation of clinical development of the IMP. These documents should be retained 
for a longer period, however, if required by the applicable regulatory requirement(s) or by an 
agreement with UCB (CPMP/ICH/135/95, 2002 [Section 4.9.5]). The investigator will contact 
UCB for authorization prior to the destruction of any study records or in the event of accidental 
loss or destruction of any study records. The investigator will also notify UCB should he/she 
relocate or move the study-related files to a location other than that specified in the sponsor’s 
trial master file. 
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. 
10.1.6.1 
Electronic Case Report form completion 
The investigator is responsible for prompt reporting of accurate, complete, and legible data in the 
eCRFs and in all required reports. 
Any change or correction to the eCRF after saving must be accompanied by a reason for the 
change. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 81 of 199 
Corrections made after the investigator’s review and approval (by means of a 
password/electronic signature) will be reapproved by the investigator. 
The investigator should maintain a list of personnel authorized to enter data into the eCRF. 
Detailed instructions will be provided in the eCRF Completion Guidelines. 
10.1.6.2 
Apps 
Not applicable. 
10.1.7 
Source documents 
All source documents must be accurate, clear, unambiguous, permanent, and capable of being 
audited. They should be made using some permanent form of recording (ink, typing, printing, 
optical disc). They should not be obscured by correction fluid or have temporary attachments 
(such as removable self-stick notes). Photocopies and/or printouts of eCRFs are not considered 
acceptable source documents. 
Source documents are original records in which raw data are first recorded. These may include 
hospital/clinic/general practitioner records, charts, diaries, x-rays, laboratory results, printouts, 
pharmacy records, care records, ECG or other printouts, completed scales, quality of life 
questionnaires, or video, for example. Source documents should be kept in a secure, limited 
access area. 
Source documents that are computer generated and stored electronically must be printed for 
review by the monitor (eg, ECG reports). Once printed, these copies should be signed and dated 
by the investigator and become a permanent part of the participant’s source documents. The 
investigator will facilitate the process for enabling the monitor to compare the content of the 
printout and the data stored in the computer to ensure all data are consistent. 
Electronic data records, such as Holter monitor records or electroencephalogram records, must 
be saved and stored as instructed by UCB (or designee). 
10.1.8 
Study and site closure 
The UCB 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 IMP development 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 82 of 199 
10.1.9 
Publication policy 
The results of this study may be published or presented at scientific meetings. If this is foreseen, 
the investigator agrees to submit all manuscripts or abstracts to the sponsor before submission. 
This allows UCB to protect proprietary information and to provide comments. 
UCB will comply with the requirements for publication of study results. In accordance with 
standard editorial and ethical practice, UCB 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. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 83 of 199 
10.2 
Appendix 2: Clinical laboratory tests 
• The tests detailed in Table 10-1 will be performed by the central laboratory, with the 
exception of platelet count assessments. 
• With the exception of platelet counts, local laboratory results are only required in the event 
that the central laboratory results are not available in time for either IMP administration 
and/or response evaluation. If a local sample is required, it is important that the sample for 
central analysis is obtained at the same time. Additionally, if the local laboratory results are 
used to make either a study treatment decision or response evaluation, the results must be 
entered into the eCRF. 
• Protocol-specific requirements for inclusion or exclusion of participants are detailed in 
Section 5.1 and Section 5.2 of the protocol, respectively. 
• Additional tests may be performed at any time during the study as determined necessary by 
the investigator or required by local regulations. 
Table 10-1: Protocol-required safety laboratory assessments 
Laboratory 
Assessments Parameters 
Hematology 
Platelet Count (local 
laboratory) a 
Red blood cell (RBC) Indices: 
Mean corpuscular volume (MCV) 
Mean corpuscular hemoglobin (MCH) 
White blood cell 
(WBC) Count with 
Differential: 
Neutrophils 
Lymphocytes 
Monocytes 
Eosinophils 
Basophils 
RBC Count 
Hemoglobin 
Hematocrit 
Coagulation 
International  
Normalized Ratio 
(INR) b 
Activated partial thromboplastin time 
(aPTT) c 
Fibrinogen c 
Clinical 
Chemistryd 
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 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 84 of 199 
Laboratory 
Assessments Parameters 
Glucose (fasting 
state preferred) 
Calcium 
Alkaline phosphatase 
C-reactive protein 
(CRP) 
Immunoglobulinse 
Albumin 
Low-density 
lipoprotein (LDL) 
High-density 
lipoprotein (HDL) 
Total cholesterol 
Triglycerides 
 
Routine 
Urinalysis 
• 
Specific gravity 
• 
pH, glucose, protein, blood, ketones, bilirubin, urobilinogen, nitrite, leukocyte 
esterase by dipstick 
• 
Microscopic examination (if blood or protein is abnormal) 
Other 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) f 
• 
Serology (human immunodeficiency antibody [HIV], hepatitis B surface antigen 
[HBsAg], and hepatitis C virus antibody) 
• 
All study-required laboratory assessments will be performed by a central laboratory 
except the platelet count which is done by local laboratory 
• 
The results of each test must be entered into the electronic Case Report form  
(eCRF) 
a Platelet count testing will be performed by a local laboratory.  
b International Normalized Ratio (INR) to be done only at Baseline (Visit 1) and for PDILI. 
c Activated partial thromboplastin time (aPTT) and fibrinogen will be done at all visits which require safety 
laboratory assessments. 
d 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 Section 10.5, Appendix 5. All events of ALT 3x upper limit 
of normal (ULN) and bilirubin 2xULN (>35% direct bilirubin) or ALT 3xULN and INR >1.5, if INR 
measured, may indicate severe liver injury (possible Hy’s Law) and must be reported as a serious adverse event 
(SAE) (excluding studies of hepatic impairment or cirrhosis). 
e Includes total IgG, IgG subclasses, IgA, IgM, and IgE. Results of the IgG testing will be unblinded starting with 
the blood samples taken at Week 4 (Visit 5) in TP0004 in order maintain blind of the parent studies. 
f Local urine testing will be standard for the protocol unless serum testing is required by local regulation or 
Independent Review Board/Independent Ethics Committee. 
Investigators must document their review of each laboratory safety report. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 85 of 199 
10.3 
Appendix 3: Adverse events – definitions and procedures for 
recording, evaluating, follow-up, and reporting 
10.3.1 
Definition of AE 
Adverse Event (AE) Definition 
• An AE is any untoward medical occurrence in a patient or clinical study participant, 
temporally associated with the use of IMP, whether or not considered related to the IMP. 
• 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 IMP. 
 
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 IMP 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 IMP or a 
concomitant medication. Overdose per se will not be reported as an AE/serious 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 fulfil the definition of an AE or SAE. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 86 of 199 
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 study participant’s condition. 
• The disease/disorder being studied or expected progression, signs, or symptoms of the 
disease/disorder being studied, unless more severe than expected for the study participant’s 
condition. 
• Medical or surgical procedure (eg, endoscopy, appendectomy): the condition that leads to 
the procedure is the AE. 
• Situations in which an untoward medical occurrence did not occur (social and/or 
convenience admission to a hospital). 
• Anticipated day-to-day fluctuations of pre-existing disease(s) or condition(s) present or 
detected at the start of the study that do not worsen. 
10.3.2 
Definition of SAE 
If an event is not an AE per definition above, then it cannot be an SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease). 
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. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 87 of 199 
e. 
Is a congenital anomaly/birth defect 
f. 
Important medical events: 
• Medical or scientific judgment should be exercised in deciding whether SAE reporting is 
appropriate in other situations such as important medical events that may not be 
immediately life-threatening or result in death or hospitalization but may jeopardize the 
participant or may require medical or surgical intervention to prevent one of the other 
outcomes listed in the above definition. These events should usually be considered serious. 
• Examples of such events include, but are not limited to, potential Hy’s law, invasive or 
malignant cancers, intensive treatment in an emergency room or at home for allergic 
bronchospasm, blood dyscrasias or convulsions that do not result in hospitalization, or 
development of drug dependency or drug abuse. 
10.3.3 
Recording and Follow-up of AE and/or SAE 
AE and SAE Recording 
• When an AE/SAE occurs, it is the responsibility of the investigator to review all 
documentation (eg, hospital progress notes, laboratory reports, and diagnostics reports) 
related to the event. 
• The investigator will then record all relevant AE/SAE information in the eCRF. 
• It is not acceptable for the investigator to send photocopies of the participant’s medical 
records to UCB in lieu of completion of the UCB AE/SAE eCRF page. 
• There may be instances when copies of medical records for certain cases are requested by 
UCB. In this case, all participant identifiers, with the exception of the participant number, 
will be redacted on the copies of the medical records before submission to UCB. 
• The investigator will attempt to establish a diagnosis of the event based on signs, 
symptoms, and/or other clinical information. Whenever possible, the diagnosis (not the 
individual signs/symptoms) will be documented as the AE/SAE. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 88 of 199 
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 study 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 an SAE, NOT when it is rated as severe (eg, a severe AE may 
be either serious or not serious, depending on whether these criteria are also met). 
The National Cancer Institute Common Terminology Criteria for Adverse Events 
(NCI-CTCAE) should be used as a supportive standardization instrument to evaluate AEs and 
SAEs but the final intensity grading by the investigator must be mild, moderate, or severe. 
 
Assessment of Causality 
• The investigator is obligated to assess the relationship between IMP and each occurrence 
of each AE/SAE. 
• A "reasonable possibility" of a relationship conveys that there are facts, evidence, and/or 
arguments to suggest a causal relationship, rather than a relationship cannot be ruled out. 
• The investigator will use clinical judgment to determine the relationship. 
• Alternative causes, such as underlying disease(s), concomitant therapy, and other risk 
factors, as well as the temporal relationship of the event to IMP 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 an SAE has occurred and the investigator has minimal 
information to include in the initial report to UCB. However, it is very important that the 
investigator always make an assessment of causality for every event before the initial 
transmission of the SAE data to UCB. 
• The investigator may change his/her opinion of causality in light of follow-up information 
and send a SAE follow-up report with the updated causality assessment. 
• The causality assessment is one of the criteria used when determining regulatory reporting 
requirements. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 89 of 199 
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, the event is otherwise explained or the 
participant is lost to follow-up. This follow-up requirement applies to AEs, SAEs, and AEs 
of special interest. 
• If a study participant dies during participation in the study or during a recognized 
Follow-up Period, the investigator will provide UCB with a copy of any post-mortem 
findings including histopathology. 
• New or updated information will be recorded in the originally completed eCRF. 
• The investigator will submit any updated SAE data to UCB within 24 hours of receipt of 
the information. 
10.3.4 
Reporting of SAEs 
SAE Reporting to UCB via an Electronic Data Collection Tool 
• The primary mechanism for reporting an SAE to UCB will be the electronic data collection 
tool. 
• If the electronic system is unavailable for more than 24 hours, then the site will use the 
paper SAE data collection tool (see “SAE Reporting to UCB via Paper Case Report Form 
[CRF]” below). 
• The site will enter the SAE data into the electronic system as soon as it becomes available. 
• After the study is completed at a given site, the electronic data collection tool will be taken 
off-line to prevent the entry of new data or changes to existing data. 
• If a site receives a report of a new SAE from a study participant or receives updated data 
on a previously reported SAE after the electronic data collection tool has been taken off-
line, then the site can report this information on a paper SAE form (see next section). 
• Contacts for SAE reporting can be found in SERIOUS ADVERSE EVENT REPORTING. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 90 of 199 
SAE Reporting to UCB via Paper CRF 
• Facsimile transmission of the SAE paper CRF is the preferred method to transmit this 
information to UCB; see SERIOUS ADVERSE EVENT REPORTING. 
• In rare circumstances and in the absence of facsimile equipment, notification by telephone 
is acceptable with a copy of the SAE data collection tool sent by overnight mail or courier 
service. 
• Initial notification via telephone does not replace the need for the investigator to complete 
and sign the SAE CRF pages within the designated reporting time frames. 
• Contacts for SAE reporting can be found in SERIOUS ADVERSE EVENT REPORTING. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 91 of 199 
10.4 
Appendix 4: Contraceptive guidance and collection of 
pregnancy information 
Definitions 
Woman of Childbearing Potential (WOCBP) 
A woman is considered fertile following menarche and until becoming post-menopausal unless 
permanently sterile (see below). 
Women in the following categories are not considered WOCBP: 
1. Premenarchal 
2. Premenopausal female with 1 of the following: 
− Documented hysterectomy 
− Documented bilateral salpingectomy 
− Documented bilateral oophorectomy 
Note: Documentation can come from the site personnel’s review of the participant’s medical 
records, medical examination, or medical history interview. 
3. Postmenopausal female 
− A postmenopausal state is defined as no menses for 12 months without an alternative 
medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal 
range may be used to confirm a postmenopausal state in women not using hormonal 
contraception or hormone replacement therapy (HRT). However, in the absence of 12 
months of amenorrhea, a single FSH measurement is insufficient. 
− Females on HRT and whose menopausal status is in doubt will be required to use one of 
the non-estrogen hormonal highly effective contraception methods if they wish to 
continue their HRT during the study. Otherwise, they must discontinue HRT to allow 
confirmation of postmenopausal status before study enrollment. 
Contraception Guidance 
Male participants 
Male participants with female partners of childbearing potential are eligible to participate if they 
agree to ONE of the following during the Treatment Period and for at least 3 months after the 
final dose of study treatment: 
• Are abstinent from penile-vaginal intercourse as their usual and preferred lifestyle (abstinent 
on a long term and persistent basis) and agree to remain abstinent 
• Agree to use a male condom when having penile-vaginal intercourse with a woman of 
childbearing potential who is not currently pregnant 
In addition, male participants must refrain from donating sperm for the duration of the study and 
for 3 months after the final dose of study medication. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 92 of 199 
Male participants with a pregnant or breastfeeding partner must agree to remain abstinent from 
penile-vaginal intercourse or use a male condom during each episode of penile penetration 
during the Treatment Period and for at least 3 months after the final dose of study treatment. 
Female participants 
Female participants of childbearing potential are eligible to participate if they agree to use a 
highly effective method of contraception consistently and correctly as described in Table 10-2. 
Table 10-2: 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 progesterone-containing) hormonal contraception associated with 
inhibition of ovulation 
• Oral 
• Intravaginal 
• Transdermal 
Progesterone only hormonal contraception associated with inhibition of ovulation 
• Oral 
• Injectable 
Highly Effective Methods That Are User Independent 
Implantable progesterone 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 vasectomized partner is 
the sole male sexual partner of the WOCBP and the absence of sperm has been confirmed. If 
not, an additional highly effective method of contraception should be used. For Japan-specific 
requirements, see Appendix 7, Section 10.7. 
Sexual abstinence 
Sexual abstinence is considered a highly effective method only if defined as refraining from 
heterosexual intercourse during the entire period of risk associated with the study medication. 
The reliability of sexual abstinence needs to be evaluated in relation to the duration of the study 
and the preferred and usual lifestyle of the participant. 
a In case of newly started contraception pills/IUDs, primary investigator should consider the correct timing of 
starting/applying such methods in relation to the menstrual cycle and the manufacturing instruction as when these 
newly started methods would become effective. 
b Typical use failure rates may differ from those when used consistently and correctly. Use should be consistent 
with local regulations regarding the use of contraceptive methods for participants in clinical studies. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 93 of 199 
Pregnancy testing 
• WOCBP should only be included after a confirmed menstrual period and a negative highly 
sensitive urine pregnancy test. 
• Additional urine pregnancy testing should be performed at time points specified in the 
Schedule of Activities (Section 1.3) and as required locally. 
• Pregnancy testing will be performed whenever a menstrual cycle is missed or when 
pregnancy is otherwise suspected. 
• Pregnancy testing with a sensitivity of 25mIU/mL will be performed. A serum pregnancy test 
will be performed to confirm a positive urine pregnancy test. 
Male participants with partners who become pregnant 
• The investigator will attempt to collect pregnancy information on any male participant’s 
female partner who becomes pregnant while the male participant is in this study. This applies 
only to male participants who receive study medication.   
• After obtaining the necessary signed informed consent from the pregnant female partner 
directly, the investigator will record pregnancy information on the appropriate form and 
submit it to the sponsor within 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 
discontinue study medication or 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 an SAE and will be reported as such. Any post-study 
pregnancy-related SAE considered reasonably related to the study medication by the 
investigator will be reported to the sponsor as described in Section 8.2.5. While the 
investigator is not obligated to actively seek this information in former study participants, he 
or she may learn of an SAE through spontaneous reporting.
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 94 of 199 
10.5 
Appendix 5: Liver safety – suggested actions and follow-up 
assessments 
Study participants with PDILI must be assessed to determine if IMP 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 IMP 
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 IMP discontinuation and/or participant withdrawal (if applicable), must be recorded 
in the source documents. The eCRF must document the primary reason for discontinuation of 
IMP. 
A specific monitoring plan must be agreed between the UCB Study Physician and the 
investigator for study participants who have ALT >5xULN. The monitoring plan should include 
any necessary follow-up assessments (until resolution of the abnormal laboratory values). 
Phase 3 or 4 liver chemistry stopping criteria are designed to assure participant safety and to 
evaluate liver event etiology (Table 10-3). 
Table 10-3: Phase 3 or 4 liver chemistry stopping criteria and follow-up 
assessments 
Liver Chemistry Stopping Criteria 
ALT absolute 
ALT 8xULN 
ALT increase 
ALT 5xULN but <8xULN persists for 2 weeks 
ALT 3xULN but <5xULN persists for 4 weeks 
Bilirubin a b 
ALT 3xULN and bilirubin 2xULN (>35% direct bilirubin) 
INR b 
ALT 3xULN and International Normalized Ratio (INR) >1.5, if INR 
measured 
Cannot 
Monitor 
ALT 5xULN but <8xULN and cannot be monitored weekly for 2 weeks 
ALT 3xULN but <5xULN and cannot be monitored weekly for 4 weeks 
Symptomatic c 
ALT 3xULN associated with symptoms (new or worsening) believed to be 
related to liver injury or hypersensitivity 
Suggested Actions and Follow-up Assessments 
Actions 
Follow-up Assessments 
Immediately discontinue IMP. 
Report the event to UCB within 24 hours. 
Complete the liver event case report form 
(CRF), and complete a serious adverse event 
Viral hepatitis serology.d 
Obtain INR and recheck with each liver 
chemistry assessment until the transaminases 
values show downward trend. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 95 of 199 
(SAE) data collection tool if the event also 
met the criteria for an SAE. b 
Perform liver chemistry follow-up 
assessments. 
Monitor the participant until liver chemistry 
test abnormalities resolve, stabilize, or return 
to Baseline (see MONITORING). 
Do not restart/rechallenge participant with 
IMP unless allowed per protocol and UCB 
approval is granted. 
If restart/rechallenge not allowed per 
protocol or not granted, permanently 
discontinue IMP and continue participant in 
the study for any protocol specified follow-
up assessments. Consider the need for a 
toxicology screening. MONITORING: 
For bilirubin or INR criteria 
Repeat liver chemistry tests (include ALT, 
AST, alkaline phosphatase, bilirubin) and 
perform liver event follow-up assessments 
within 24 hours. 
Monitor participant twice weekly until liver 
chemistry test abnormalities resolve, 
stabilize, or return to Baseline. 
A specialist or hepatology consultation is 
recommended. 
For all other criteria 
Repeat liver chemistry tests (include ALT, 
AST, alkaline phosphatase, bilirubin) and 
perform liver chemistry follow-up 
assessments within 24 to 72 hours. 
Monitor participants weekly until liver 
chemistry abnormalities resolve, stabilize, or 
return to Baseline. 
Only in those with underlying chronic hepatitis 
B at study entry (identified by positive 
hepatitis B surface antigen), quantitative 
hepatitis B deoxyribonucleic acid (DNA), and 
hepatitis delta antibody.e 
Obtain blood sample for pharmacokinetic (PK) 
analysis as soon as feasible after the most 
recent dose.f 
Serum creatine phosphokinase (CPK) and 
lactate dehydrogenase (LDH). 
Fractionate bilirubin, if total bilirubin 
 2xULN. 
Obtain complete blood count with differential 
to assess eosinophilia. 
Record the appearance or worsening of clinical 
symptoms of liver injury, or hypersensitivity, 
on the adverse event (AE) report form. 
Record use of concomitant medications 
(including acetaminophen, herbal remedies, 
and other over-the-counter medications) on the 
concomitant medications CRF. 
Record alcohol use on the liver event alcohol 
intake CRF. 
Exclude pregnancy. 
For bilirubin or INR criteria 
Anti-nuclear antibody, anti-smooth muscle 
antibody, Type 1 anti-liver kidney microsomal 
antibodies, and quantitative total. 
immunoglobulin G (IgG) or gamma globulins. 
Serum acetaminophen concentration and 
serum acetaminophen adduct assay (where 
available) for assessing the potential 
acetaminophen contribution to liver injury. 
Liver imaging (ultrasound, magnetic 
resonance, or computerized tomography) and 
or liver biopsy to evaluate liver disease; 
complete Liver Imaging and/or Liver Biopsy 
CRFs. 
a Serum bilirubin fractionation should be performed if testing is available. If serum bilirubin fractionation is not 
immediately available, discontinue IMP if ALT ≥3xULN and bilirubin ≥2xULN. Additionally, if serum bilirubin 
fractionation testing is unavailable, record the absence/presence of detectable urinary bilirubin on dipstick, 
which is indicative of direct bilirubin elevations suggesting liver injury. 
b All events of ALT ≥3xULN and bilirubin ≥2xULN (>35% direct bilirubin) or ALT ≥3xULN and INR >1.5 may 
indicate severe liver injury (possible ‘Hy’s Law’) and must be reported as an SAE (excluding studies of 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 96 of 199 
hepatic impairment or cirrhosis). The INR measurement is not required and the stated threshold value will not 
apply to participants receiving anticoagulants. 
c New or worsening symptoms believed to be related to liver injury (such as fatigue, nausea, vomiting, right upper 
quadrant pain or tenderness, or jaundice) or hypersensitivity (such as fever, rash, or eosinophilia). 
d Includes: Hepatitis A immunoglobulin M (IgM) antibody; HBsAg and HBcAb; hepatitis C RNA; cytomegalovirus 
IgM antibody; Epstein-Barr viral capsid antigen IgM antibody (or if unavailable, heterophile antibody or monospot 
testing); and hepatitis E IgM antibody. 
e If hepatitis delta antibody assay cannot be performed, it can be replaced with a polymerase chain reaction (PCR) of 
hepatitis D RNA virus (where needed) (Le Gal et al, 2005). 
f Record the date/time of the PK blood sample draw and the date/time of the final dose of IMP prior to the PK blood 
sample draw on the eCRF. If the date or time of the final dose is unclear, provide the participant’s best 
approximation. If the date/time of the final 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 Laboratory Manual. 
Table 10-4: Phase 3-4 liver chemistry increased monitoring criteria with 
continued IMP 
Liver Chemistry Increased Monitoring Criteria 
Criteria 
Actions 
ALT 5xULN and <8xULN and 
bilirubin <2xULN without symptoms 
believed to be related to liver injury or 
hypersensitivity, and who can be 
monitored weekly for 2 weeks. 
OR 
ALT 3xULN and <5xULN and 
bilirubin <2xULN without symptoms 
believed to be related to liver injury or 
hypersensitivity, and who can be 
monitored weekly for 4 weeks. 
Notify the UCB medical monitor within 24 hours of 
learning of the abnormality to discuss participant 
safety. 
Participant can continue IMP. 
Participant must return weekly for repeat liver 
chemistry tests (ALT, AST, alkaline phosphatase, 
bilirubin) until the abnormalities resolve, stabilize, or 
return to Baseline. 
If at any time, the participant meets liver chemistry 
stopping criteria, proceed as described in 
Section 7.1.1. 
If ALT decreases from ALT 5xULN and <8xULN 
to ≥3xULN but <5xULN, continue to monitor liver 
chemistries weekly. 
If, after 4 weeks of monitoring, ALT <3xULN and 
bilirubin <2xULN, monitor participants twice 
monthly until liver chemistry tests resolve, stabilize, 
or return to Baseline. 
REFERENCES 
Le Gal F, Gordien E, Affolabi D, Hanslik T, Alloui C, Dény P, Gault E. 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):2363–2369. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 97 of 199 
10.6 
Appendix 6: Medical device incidents – definition and 
procedures for recording, evaluating, follow-up, and reporting 
Not applicable. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 98 of 199 
10.7 
Appendix 7: Country-specific requirements 
Poland 
The Polish Health Authority’s and Clinical Trial Facilitation Group have made recommendations 
related to contraception and pregnancy testing in a clinical study with IMP where the human data 
for exposure at pregnancy and nonclinical reproductive toxicology data are not available. As a 
mitigation measure, pregnancy testing is to be implemented monthly up to 2 months after the 
final dose of IMP. A urine pregnancy test is added at 4 weeks after final IMP administration, 
thus it meets the above requirement. Based on the short half-life of IMP and because 
genotoxicity is not a class effect of the monoclonal antibodies, it is considered safe to reduce the 
duration of post-IMP administration requirement on contraception for female study participants 
of childbearing potential from 3 months to 2 months, with implementation of monthly pregnancy 
testing during this period. 
France 
Specific requirements for study participants in the France include: 
• In reference to Section 1.1 (Synopsis - Treatment Groups and Duration), the expected 
duration of the enrollment phase for an individual participant is defined as a visit window of 
up to +3 days from Week 25 of the parent study (TP0003 or TP0006). As study participants 
will enter this OLE study after completing participation in one of the parent studies, the 
overall timeline for this study is dependent on the enrollment trajectory of the parent studies. 
Anticipated timelines for completing enrollment of all participants in the parent studies is as 
follows: the last patient first visit of parent study is expected Jan 2024 with the last patient 
last dosing visit of parent study expected in Jul 2024. Based on these projections, the last 
patient first visit of TP0004 study is anticipated in Aug 2024, with the last patient last visit 
expected in Sep 2025. 
• In reference to Section 5.2 (Exclusion Criteria), 3 additional exclusion criteria: 
15. Study participant is deprived of their liberty by a judicial or administrative decision, or is 
receiving psychiatric care, and is admitted to a health or social institution, as defined in the 
French Public Health Code. 
16. Study participant is subject to legal protection or is unable to express consent, as defined in 
the French Public Health Code. 
17. Study participant is <18 years of age at the time of the Baseline Visit. 
Japan 
Specific requirements for study participants in Japan include: 
Locally approved devices are to be used during the study. If a pump and infusion set are used 
that are regarded in Japan as investigational devices, then additional adherence to specific 
reporting obligations will be required. All adverse device effects (ADEs), serious adverse device 
effects (SADEs), and medical device deficiency (including malfunction use error, and inadequate 
labeling) of these investigational devices shall be documented and reported by the investigator 
throughout the study and appropriately managed by the sponsor. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 99 of 199 
This reporting requirement is not applicable for locally approved devices for the purpose of sc 
infusions or other purposes in the course of this study, regardless if provided by the sponsor or 
not. 
• In reference to Section 6.4.1, the use of medicinal cannabidiols and medicinal marijuana are 
prohibited by law. 
• In reference to Section 10.4, Table 10-2 the following method has been added: 
“(eg, proper use of condom in combination with spermicide)” 
Furthermore, specific rules for repetition of an ADE and device deficiency should be followed 
by all study sites in Japan; this requirement is not applicable for locally approved devices, 
regardless if provided by the sponsor or not.  
For ADEs and/or device deficiencies that are not related to the natural course of the disease 
under study, an increase in the intensity of the original ADE, and/or device deficiency should 
lead to the repetition of the original ADE and/or device deficiency with the following guidelines: 
• The outcome date of the original ADE and/or device deficiency must be the same as the start 
date of the repeated ADE and/or device deficiency. 
• The outcome of the original ADE and/or device deficiency must be recorded as “worsening.” 
• The verbatim term for the repeated ADE and/or device deficiency must be the same as the 
verbatim term for the original ADE and/or device deficiency so that the repeated ADE and/or 
device deficiency is obviously a worsening of the original. 
As per local requirements in Japan, SAEs associated to an investigational device, and device 
deficiencies (eg, infusion pump product provided from sponsor) should be reported in 
accordance with the following.  
This requirement is not applicable for approved devices used in the course of the study, 
regardless if provided by the sponsor or not. 
Medical Device – Adverse events (ADEs, SAEs, and SADEs) and device 
deficiencies 
Medical devices are being provided for use in this study for subcutaneous infusions. In order to 
fulfill regulatory reporting obligations worldwide, the investigator is responsible for the detection 
and documentation of events meeting the definitions of incident or malfunction that occur during 
the study with such devices.  
Adverse events will be reported according to the ISO 14155:2011, 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.  
NOTE: Events fulfilling the definition of an AE/SAE will also follow the processes outlined in 
Section 8.2.3 and Appendix 3 (Section 10.3) of the protocol. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 100 of 199 
Time period for detecting medical device deficiencies 
Medical device deficiency or malfunction of the device that results in a reportable event will be 
detected, documented, and reported during all periods of the study in which the medical device is 
used.  
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. 
Follow-up of medical 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. 
Prompt reporting of medical device deficiencies to sponsor 
Device deficiencies will be reported to the sponsor within 24 hours after the investigator 
determines that the event meets the protocol definition of a medical device deficiency. 
The Adverse Event and Device Deficiency Report Form will be sent to the sponsor by email. If 
email is unavailable, then fax should be utilized. 
The sponsor will be the contact for the receipt of device deficiency reports. 
Regulatory reporting requirements for medical device deficiencies 
The investigator will promptly report all device deficiencies occurring with any medical device 
provided for use in the study in order for the sponsor to fulfill the legal responsibility to notify 
appropriate regulatory authorities and other entities about certain safety information relating to 
medical devices being used in clinical studies. 
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/IEC. 
Medical Device AEs (ADEs, UADEs, SAEs, SADEs, and USADEs) 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. 
Both the investigator and the sponsor will comply with all local medical device reporting 
requirements. 
The detection and documentation procedures described in this protocol apply to all sponsor 
medical devices provided for use in the study.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 101 of 199 
Definition of AE and ADE 
AE and ADE Definition  
• 
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally 
associated with the use of IMP, whether or not considered related to the IMP. 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 IMP.  
• 
An adverse device effect (ADE) is defined as an adverse event related to the use of an 
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. 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. 
Definition of SAE, and SADE  
If an event is not an AE per definition above, then it cannot be an SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease). 
An SAE is an AE that: 
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 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 102 of 199 
Medical or scientific judgment should be exercised in deciding whether SAE reporting is 
appropriate in other situations such as important medical events that may not be immediately life-
threatening or result in death or hospitalization but may jeopardize the participant or may require 
medical or surgical intervention to prevent one of the other outcomes listed in the above definition. 
These events should usually be considered serious. 
Examples of such events include, but are not limited to, potential Hy’s law, invasive or malignant 
cancers, intensive treatment in an emergency room or at home for allergic bronchospasm, blood 
dyscrasias or convulsions that do not result in hospitalization, or development of drug dependency 
or drug abuse. 
SADE definition 
• 
A SADE is defined as an adverse device effect that has resulted in any of the consequences 
characteristic of a serious adverse event. 
Definition of Device Deficiency 
Device Deficiency definition 
• 
A device deficiency is an inadequacy of an investigational medical device with respect to its 
identity, quality, durability, reliability, safety, or performance. Device deficiencies include 
malfunctions, use errors, and inadequate labeling. 
Recording and Follow-Up of AE and/or SAE and Device Deficiencies 
AE, SAE and Device Deficiency Recording 
• 
When an AE/SAE/device deficiency 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/device deficiency information in the 
participant’s medical records, in accordance with the investigator’s normal clinical practice and 
on the appropriate form of 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 AE/SAE/device deficiency 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. 
• 
For device deficiencies, it is very important that the investigator describes any corrective or 
remedial actions taken to prevent recurrence of the deficiency 
o A remedial action is any action other than routine maintenance or servicing of a 
medical device where such action is necessary to prevent recurrence of a device 
deficiency. This includes any amendment to the device design to prevent recurrence. 
The investigator should inform the sponsor for all reported device deficiencies.  
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 103 of 199 
Follow-up of AE/SAE/device deficiency 
• 
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/SAE/device deficiency as fully as possible. This may 
include additional laboratory tests or investigations, histopathological examinations, or 
consultation with other health care professionals. 
• 
If a participant dies during participation in the study or during a recognized follow-up period, 
the investigator will provide UCB with a copy of any post-mortem findings including 
histopathology.  
• 
New or updated information will be recorded in the originally completed CRF. 
• 
The investigator will submit any updated SAE data to UCB within 24 hours of receipt of the 
information. 
Reporting of SAEs 
SAE Reporting to UCB via an Electronic Data Collection Tool 
• 
The primary mechanism for reporting an SAE to UCB will be the electronic data collection 
tool. 
• 
If the electronic system is unavailable, then the site will use the paper SAE data collection tool 
(see next section) in order to report the event within 24 hours. 
• 
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) or to UCB by 
telephone. 
• 
Contacts for SAE reporting can be found in this protocol. 
Reporting of SADEs 
SADE Reporting to UCB  
NOTE: There are additional reporting obligations for medical device deficiencies that are potentially 
related to SAEs that must fulfil the legal responsibility to notify appropriate regulatory authorities and 
other entities about certain safety information relating to medical devices being used in clinical studies.  
• 
Any device deficiency that is associated with an SAE must be reported to the sponsor within 
24 hours after the investigator determines that the event meets the definition of a device 
deficiency using the paper form provided by the sponsor. 
• 
The sponsor shall review all device deficiencies and determine and document in writing 
whether they could have led to an SAE. These shall be reported to the regulatory authorities 
and IRBs/IECs as required by national regulations. 
• 
Contacts for SAE reporting can be found in this protocol. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 104 of 199 
Reporting of AE and/or Device Deficiencies  
AE and/or Device Deficiencies Reporting to UCB  
NOTE: There are additional reporting obligations for medical device deficiencies that must fulfil the 
legal responsibility to notify appropriate regulatory authorities and other entities about certain safety 
information relating to medical devices being used in clinical studies.  
• 
Any AE and/or device deficiencies must be reported to the Sponsor within 24 hours after the 
Investigator determines that the event meets the definition of a device deficiency using the 
paper form provided by the Sponsor. 
• 
The sponsor shall review all device deficiencies and determine and document in writing 
whether they meet device reporting requirement.  
• 
Contacts for SAE reporting can be found in this protocol. 
10.7.1 
Countries where self-administration will be offered as an option 
Self-administration will be offered as an option to the following countries: 
• Austria 
• Belgium 
• Canada 
• France 
• Germany  
• Italy 
• Japan 
• Poland 
• Russian Federation 
• Spain 
• Ukraine 
• United Kingdom 
• United States 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 105 of 199 
10.8 
Appendix 8: Rapid alert procedures 
Not applicable. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 106 of 199 
10.9 
Appendix 9: Abbreviations and trademarks 
ADA 
antidrug antibody 
ADE 
adverse device effect 
ADL 
activities of daily living 
AE 
adverse event 
AESM 
adverse event of special monitoring 
ALP 
alkaline phosphatase 
ALT 
alanine aminotransferase 
anti-d 
anti-Rho 
AST 
aspartate aminotransferase 
BP 
blood pressure 
CDC 
Centers for Disease Control and Prevention 
CRF 
Case Report form 
CRO 
contract research organization 
CRP 
c-reactive protein  
ECG 
electrocardiogram 
eCRF 
electronic Case Report form 
eqv 
equivalent 
EOS 
End of Study 
EQ VAS 
EuroQoL visual analog scale 
EQ-5D-5L 
European Quality of Life-5 Dimension 5 Levels 
EW 
Early Withdrawal 
FcRn 
neonatal Fc receptor 
FSH 
follicle-stimulating hormone 
GCP 
Good Clinical Practice 
GI 
gastrointestinal 
HCP 
healthcare professional 
HRQoL 
health-related quality of life 
HRT 
hormone replacement therapy 
IB 
Investigator's Brochure 
ICF 
Informed Consent form 
ICH 
International Council for Harmonisation 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 107 of 199 
IDMC 
Independent Data Monitoring Committee 
IEC 
Independent Ethics Committee 
Ig 
immunoglobulin  
IGRA 
interferon-gamma release assay 
IMP 
investigational medicinal product 
IRB 
Institutional Review Board 
IRT 
interactive response technology 
ITP 
immune thrombocytopenia 
ITP-BAT 
ITP-specific Bleeding Assessment Tool 
ITP-PAQ 
ITP-Patient Assessment Questionnaire 
iv 
intravenous 
IVIg 
intravenous immunoglobulin 
K-PD 
kinetic-pharmacodynamic 
LLOQ 
lower limit of quantification 
LTB 
latent tuberculosis 
LTBI 
latent tuberculosis infection 
MAP 
managed access program 
MG 
myasthenia gravis 
NTMBI 
nontuberculous mycobacterial infection 
OLE 
open-label extension 
PD 
pharmacodynamic 
PDILI 
potential drug-induced liver injury 
PEX 
plasma exchange 
PGI-C 
Patient Global Impression of Change 
PGI-S 
Patient Global Impression of Severity 
PK 
pharmacokinetic 
PRO 
patient-reported outcome 
Q2W 
every 2 weeks 
QoL 
quality of life 
QTc 
corrected QT interval 
QW 
every week 
RO 
Receptor Occupancy 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 108 of 199 
SADE 
serious adverse device effects 
SAE 
serious adverse event 
SIAQ 
self-injection assessment questionnaire 
SAP 
Statistical Analysis Plan 
sc 
subcutaneous 
SF-36 
Short-Form 36-Item Health Survey 
SFU 
Safety Follow-Up 
SMOG 
Skin-Visible Mucosa-Internal Organs tool 
SS 
Safety Set 
TB 
tuberculosis 
TEAE 
treatment-emergent adverse event 
TPO-RA 
thrombopoietin-receptor agonists 
UADE 
unanticipated adverse device effect  
USADE 
unanticipated serious adverse device effect 
ULN 
upper limit of normal 
VAS 
visual analog scale 
WHO 
World Health Organization 
WOCBP 
woman of childbearing potential 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 109 of 199 
10.10 
Appendix 10: ITP-BAT 
Grading of bleeding symptoms at presentation and at each subsequent evaluation.1 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
SKIN 
 
 
 
 
 
Petechiae (does not 
include steroid–induced 
or senile purpura) 
 No 
 Less than or equal 
to 10 in a patient’s 
palm-sized area3 in the 
most affected body 
area4 
 Any number if 
reported by the patient 
 More than 10 in a 
patient’s palm-sized 
area or more than 5 in at 
least 2 patient’s palm-
sized areas located in at 
least 2 different body 
areas4, one above and 
one below the belt (in 
the most affected body 
areas) 4 
 More than 50, if 
scattered both above 
and below the belt 
 
Ecchymoses 
 None or up to 
2 in the same body 
area4, but smaller 
than a patient’s 
palm-sized area, if 
a) spontaneous or 
b) disproportionate 
to trauma/ 
constriction5 
 3 or more in the 
same body area4, but 
all smaller than a 
patient’s palm-sized 
area, if 
a) spontaneous or  
b) disproportionate to 
trauma/constriction5 
 At least 2 in two 
different body areas4, 
smaller than a patient’s 
palm-sized area, if 
a) spontaneous or  
b) disproportionate to 
trauma/constriction5 
 From 1 to 5 larger 
than a patient’s 
palm-sized area, if 
a) spontaneous or  
b) disproportionate to 
trauma/constriction5 
with or without smaller 
ones 
 More than 5 larger 
than a patient’s palm-
sized area, if 
a) spontaneous or  
b) disproportionate to 
trauma/ constriction5 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 110 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
 Any number and 
size if reported by the 
patient 
Subcutaneous 
hematomas 
 No 
 1 smaller than a 
patient’s palm-sized 
area 
 Any number and 
size if reported by the 
patient 
 2 smaller than a 
patient’s palm-sized 
area, spontaneous  
 2 smaller than a 
patient’s palm-sized 
area, disproportionate to 
trauma5 
 More than 2 
smaller or at least 1 
larger than a patient’s 
palm-sized area, 
spontaneous  
 More than 2 
smaller or at least 1 
larger than a patient’s 
palm-sized area, 
disproportionate to 
trauma5 
 
Bleeding from minor 
wounds6 
 No 
 Lasting <5 min 
 Any episode if 
reported by the patient 
 Lasting >5 min or 
interfering with daily 
activities 
 Requiring 
protracted medical 
observation at the 
time of this visit  
 Medical report 
describing patient’s 
evaluation by a 
physician 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 111 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
MUCOSAL 
 
 
 
 
 
Epistaxis7 
 No 
 Lasting <5 min 
 Any episode if 
reported by the patient 
 Lasting >5 min or 
interfering with daily 
activities 
 Packing or 
cauterization or 
in-hospital evaluation 
at the time of this visit 
 Medical report 
describing packing or 
cauterization or in-
hospital evaluation 
 RBC 
transfusion or 
Hb drop >2g/dL 
Oral cavity – gum 
bleeding7 
 No 
 Lasting <5 min 
 Any episode if 
reported by the patient 
 Lasting >5 min or 
interfering with daily 
activities 
 Requiring 
protracted medical 
observation at the 
time of this visit  
 Medical report 
describing patient’s 
evaluation by a 
physician 
 
Oral cavity – 
hemorrhagic bullae or 
blisters 
 No 
 Less than 3 
 Any number if 
reported by the patient 
 From 3 to 10 but no 
difficulty with 
mastication 
 More than 10 or 
more than 5 if 
difficulty with 
mastication 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 112 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
Oral cavity - bleeding 
from bites to lips & 
tongue or after 
deciduous teeth loss 
 No 
 Lasting <5 min 
 Any episode if 
reported by the patient 
 Lasting >5 min or 
interfering with daily 
activities 
 Interventions to 
ensure hemostasis or 
in-hospital evaluation 
at the time of this visit 
 Medical report 
describing 
interventions to 
ensure hemostasis or 
in-hospital evaluation 
 
Subconjunctival 
hemorrhage  
(not due to conjunctival 
disease) 
 No 
 Petechiae/ 
hemorrhage partially 
involving one eye 
 Any episode if 
reported by the patient 
 Petechiae/ 
hemorrhage partially 
involving both eyes, or 
diffuse hemorrhage in 
one eye 
 Diffuse 
hemorrhage in both 
eyes 
 
ORGAN (and internal 
mucosae) 
 
 
 
 
 
Gastrointestinal 
bleeding not explained 
by visible mucosal 
bleeding or lesion: 
Hematemesis, 
Melena, 
Hematochezia, 
Rectorrhagia 
 No 
 Any episode if 
reported by the patient 
 Present at the visit  
 Described in a 
medical report  
 Requiring 
endoscopy8 or other 
therapeutic 
procedures or in-
hospital evaluation at 
the time of this visit 
 Medical report 
prescribing 
endoscopy8 or other 
therapeutic 
procedures or in-
hospital evaluation 
 RBC 
transfusion or 
Hb drop >2g/dL 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 113 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
Lung bleeding 
Hemoptysis 
Tracheobronchial 
bleeding 
 No 
 Any episode if 
reported by the patient 
 Present at this visit 
 Described in a 
medical report 
 Requiring 
bronchoscopy8 or 
other therapeutic 
procedures or in-
hospital evaluation at 
the time of this visit 
 An equivalent 
episode if described 
in a medical report 
 RBC 
transfusion or 
Hb drop >2g/dL 
Hematuria 
 No 
 Any episode if 
reported by the patient 
 Microscopic (lab 
analysis) 
 Macroscopic  
 Described in a 
medical report 
 Macroscopic, and 
requiring cystoscopy8 
or other therapeutic 
procedures or in-
hospital evaluation at 
the time of this visit 
 An equivalent 
episode if described 
in a medical report 
 RBC 
transfusion or 
Hb drop >2g/dL 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 114 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
Menorrhagia 
(compared to pre-ITP 
or to a phase of disease 
with normal platelet 
count)9 
 No 
 Doubling nr. of 
pads or tampons in last 
cycle compared to pre-
ITP or to a phase of 
disease with normal 
platelet count 
 Score >100 using 
PBAC in the last 
cycle, if normal score 
in pre-ITP cycles or in 
a phase of disease with 
normal platelet count 
 Changing pads more 
frequently than every 2 
hrs. or clot and flooding 
 Requiring combined 
treatment with 
antifibrinolytics and 
hormonal therapy or 
gynecological 
investigation (either at 
this visit or described in 
a medical report) 
 Acute 
menorrhagia requiring 
hospital admission or 
endometrial ablation 
(either at this visit or 
described in a medical 
report) 
 RBC 
transfusion or 
Hb drop >2g/dL 
Intramuscular 
hematomas 
(only if diagnosed by a 
physician with an 
objective method) 
 No 
 Post trauma, 
diagnosed at this visit, 
if judged 
disproportionate to 
trauma 
 An equivalent 
episode if described in 
a medical report 
 Spontaneous, 
diagnosed at this visit 
 An equivalent 
episode if described in a 
medical report 
 Spontaneous or 
post trauma (if judged 
disproportionate to 
trauma) diagnosed at 
this visit and 
requiring hospital 
admission or surgical 
intervention 
 An equivalent 
episode if described 
in a medical report 
 RBC 
transfusion or 
Hb drop >2g/dL 
Hemarthrosis 
(only if diagnosed by a 
physician with an 
objective method) 
 No  
 Post trauma, 
diagnosed at this visit, 
function conserved or 
minimally impaired, if 
judged 
 Spontaneous, 
diagnosed at this visit, 
function conserved or 
minimally impaired 
 Spontaneous or 
post trauma (if judged 
disproportionate to 
trauma), diagnosed at 
this visit and 
requiring 
 Spontaneous 
or post trauma 
(if judged 
disproportionate 
to trauma) 
diagnosed at this 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 115 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
disproportionate to 
trauma 
 An equivalent 
episode if described in 
a medical report 
 An equivalent 
episode if described in a 
medical report 
immobilization or 
joint aspiration 
 An equivalent 
episode if described 
in a medical report 
visit and 
requiring 
surgical 
intervention 
 An 
equivalent 
episode if 
described in a 
medical report 
Ocular bleeding 
(only if diagnosed by a 
physician with an 
objective method) 
 No 
 
 Any post trauma 
vitreous or retinal 
hemorrhage involving 
one or both eyes with or 
without 
impaired/blurred vision 
present at this visit if 
judged disproportionate 
to trauma 
 An equivalent 
episode if described in a 
medical report 
 Spontaneous 
vitreous or retinal 
hemorrhage involving 
one or both eyes with 
impaired/blurred 
vision present at this 
visit 
 An equivalent 
episode if described 
in a medical report 
 Spontaneous 
vitreous or 
retinal 
hemorrhage with 
loss of vision in 
one or both eyes 
present at this 
visit 
 An 
equivalent 
episode if 
described in a 
medical report 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 116 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
Intracranial bleeding10: 
intracerebral, 
intraventricular, 
subarachnoidal, 
subdural, extradural 
(only if diagnosed with 
an objective method at 
the visit or described in 
a medical report 
provided by the 
patient) 
 No 
 
 Any post trauma 
event requiring 
hospitalization 
 Any spontaneous 
event requiring 
hospitalization in 
presence of an 
underlying 
intracranial lesion 
 Any 
spontaneous 
event requiring 
hospitalization 
without an 
underlying 
intracranial 
lesion 
Other internal 
bleeding: 
hemoperitoneum 
hemopericardium 
hemothorax 
retroperitoneal bleeding 
hepatic and splenic 
peliosis with organ 
rupture 
retroorbital bleeding 
metrorrhagia 
etc. 
(only if diagnosed with 
an objective method at 
the visit or described in 
a medical report 
 No 
 
 
 Any event 
requiring 
hospitalization <48 
hrs. 
 Any event 
requiring 
hospitalization 
>48 hrs. or RBC 
transfusion or 
Hb drop >2g/dL 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 117 of 199 
Table 10-5: ITP-Bleeding Assessment Tool 
Type of bleeding 
GRADES BASED ON THE WORST INCIDENT EPISODE SINCE LAST VISIT2 
 
0 
1 
2 
3 
4 
provided by the 
patient) 
Hb=hemoglobin; hrs=hours; ITP=immune thrombocytopenia; nr=number; PBAC= Pictorial Blood Loss Assessment Chart; RBC=red blood cell; SMOG=Skin-
Visible Mucosa-Internal Organs tool 
1 In case of study participants examined for the first time, all types of bleeding occurring at the visit and in the 15 days preceding the visit should be considered.  
2 Each type of bleeding should be graded based on the worst bleeding manifestation that occurred during each observation period or in the 15 days preceding the 
first visit. 
3 Study participant’s own palm size is commonly considered to be proportional to body surface area. Palm=the inner surface of the hand stretching between the 
distal crease of the wrist and the bases of the fingers (fingers surface excluded). 
4 Body areas include: face, neck, right and left upper limbs (considered separately), right and left lower limbs (considered separately), trunk, abdomen, and 
recumbent areas (for the ambulatory patient means the area below the knees).  
5 Bleedings considered proportionate to trauma/constriction on a clinical ground should not be reported for skin domain. 
6 Minor wound means superficial skin cuts (eg, by shaving razor, knife, or scissors). 
7 Epistaxis and gum bleeding are also reported in some normal study participants. Thus, a critical judgment is required in grading these manifestations; they 
should be reported only if judged more severe when compared with pre-ITP bleeding, if any. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 118 of 199 
8 Any endoscopic investigations should be considered for grading only if performed for therapeutic purpose and not solely for diagnostic purpose. 
9 In girls at menarche, grade 1 cannot be assigned, lacking comparison with previous cycles. 
10 Intracranial bleeding should always be reported, irrespective of its grade. For example, if a woman had S2 (subcutaneous hematoma) M2 (epistaxis) O3 
(menorrhagia) and an intracranial bleeding grade 2 (post trauma, requiring hospitalization), the SMOG index is S2M2O3 (intracranial 2). If the same 
participant also had intracranial bleeding grade 3, the SMOG index is S2M2O3 (intracranial 3). 
Note: Grading is based on physical examination at the time of the visit by the physician or expert nurse or on study participant’s history supplemented by 
available medical reports. Bleeding manifestations reported by the study participant but not visible at the time of data collection are graded 1. Grade 5 is 
assigned to fatal bleeding. 
Note: Illustrative examples are available on the website of the Hematology Project Foundation (http://itpbat.fondazioneematologia.it/). 
Note: To receive a grade >1, all nonovert skin and non-overt mucosal bleeding (petechiae, ecchymoses, subcutaneous hematomas, vescicles/bullae 
subconjunctival bleeding) should be visible at the time of visit for grading by the physician or expert nurse taking the history.  
Note: For bleeding from minor wounds and overt-mucosal bleeding (epistaxis, gum, bleeding from bites to lips & tongue or after deciduous teeth loss/extraction) 
and all organ bleeding, a medical record describing the symptom or indicating a specific intervention/prescription should be also taken into account for grading. 
Note: Requirement for ITP-specific treatments and anti-fibrinolytics (apart from menorrhagia) was not considered for grading, due to their subjective nature and 
their adoption not only to control actual bleeding but also to reduce the “risk” of impendent or future bleeding. 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 119 of 199 
10.11 
Appendix 11: ITP-PAQ 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 120 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 121 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 122 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 123 of 199 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 124 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 125 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 126 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 127 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 128 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 129 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 130 of 199 
10.12 
Appendix 12: SF-36 Form 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 131 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 132 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 133 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 134 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 135 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 136 of 199 
10.13 
Appendix 13: FATIGUE-PRO Physical Fatigue Scale 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 137 of 199 
10.14 
Appendix 14: EQ-5D-5L 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 138 of 199 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 139 of 199 
10.15 
Appendix 15: PGI-S 
The following question asks you about your current overall ITP symptoms. 
Please check the box that best describes your current situation. 
 
How would you describe your ITP symptoms during the past week? 
 
None 
 
Mild 
 
Moderate 
 
Severe 
 
Very severe 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 140 of 199 
10.16 
Appendix 16: PGI-C 
The following question asks you about your overall symptoms now compared to before starting 
treatment within this clinical study.  
Please check the box that best describes your current situation. 
 
How would you describe your current ITP symptoms compared to before you started 
treatment in this clinical study? 
 
Very much worse 
 
Much worse 
 
A little bit worse 
 
No change 
 
A little bit improved 
 
Much improved 
 
Very much improved 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 141 of 199 
10.17 
Appendix 17: Headache Questionnaire 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 142 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 143 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 144 of 199 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 145 of 199 
10.18 
Appendix 18: Tuberculosis assessment 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 146 of 199 
10.19 
Appendix 19: Sampson Criteria Questionnaire 
Anaphylaxis is highly likely when any of the following 3 criteria is fulfilled 
(Sampson et al, 2006): 
1. Acute onset of an illness (minutes to several hours) with involvement of the skin, mucosal 
tissue, or both (eg, generalized hives, pruritus or flushing, swollen lips-tongue-uvula) 
AND AT LEAST ONE OF THE FOLLOWING 
a. Respiratory compromise (eg, dyspnea, wheeze-bronchospasm, stridor, reduced peak 
expiratory flow [PEF], hypoxemia) 
b. Reduced BP or associated symptoms of end-organ dysfunction (eg, hypotonia [collapse], 
syncope, incontinence) 
2. Two or more of the following that occur rapidly after exposure to a likely allergen for that 
patient (minutes to several hours): 
a. Involvement of the skin-mucosal tissue (eg, generalized hives, itch-flush, swollen 
lips-tongue-uvula) 
b. Respiratory compromise (eg, dyspnea, wheeze-bronchospasm, stridor, reduced PEF, 
hypoxemia) 
c. Reduced BP or associated symptoms (eg, hypotonia [collapse], syncope, incontinence) 
d. Persistent GI symptoms (eg, crampy abdominal pain, vomiting) 
Reduced BP after exposure to known allergen for that study participant (minutes to several 
hours): Systolic BP of less than 90mmHg or greater than 30% decrease from the study 
participant’s Baseline systolic BP value. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 147 of 199 
10.20 
Appendix 20: Management of headache 
Based on current available clinical data, headache is the most commonly reported adverse drug 
reaction in study participants treated with rozanolixizumab. Study participants should be well 
informed of this potential adverse drug reaction and should be instructed on how to manage it.  
Determination of the severity of headache will be consistent with National Cancer Institute 
Terminology Criteria for Adverse Events (CTCAE) version 5.0 (see Appendix 3, Section 10.3). 
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.  
The Headache Questionnaire (Appendix 17, Section 10.17) should be administered daily by a 
health care professional via an interview with any study participant experiencing moderate, 
severe and/or serious headache until resolving or resolution (ie, if headache becomes nonserious, 
mild, or completely resolved, whichever comes first). If the severe or serious headache is 
initially reported at a home visit or during a telephone call, the study participant should be seen 
at the study site as soon as is practically possible for further investigations. Study participants 
should be monitored for signs and symptoms suggestive of central nervous system involvement 
and evaluated immediately if other causes (eg, meningitidis, intracranial bleeding) are suspected. 
Further neurological workup may be performed (if indicated) at the discretion of the investigator 
or the treating physician and may include a computed tomography scan, magnetic resonance 
imaging and/or a lumbar puncture for cerebral spinal fluid collection. In addition, samples for 
exploratory safety biomarkers should be collected for study participants experiencing severe 
headaches or serious headaches when possible. These investigations will be performed to further 
understand the mechanism of headaches in the study participants. 
Treatments must be permanently discontinued if a study participant has a serious headache (SAE 
of headache) that is considered related to the IMP in the opinion of the investigator. If a study 
participant experiences a severe AE of headache that is considered related to the IMP in the 
opinion of the investigator, the dose of IMP may be reduced or the treatment may be temporarily 
put on hold. If the treatment has been put on hold and if deemed appropriate by the investigator 
and agreed upon by the study participant and the sponsor, the study treatment can resume upon 
the resolution of the severe headache event, at the previous dose or at a lower dose. The benefit 
and risk of the treatment should be carefully considered prior to reinitiating the IMP. If a study 
participant experiences a second episode of treatment-related severe headache in the opinion of 
the investigator, the treatment can only be continued upon agreement with the sponsor, 
investigator, and study participant taking benefit and risk into consideration. If the benefit-risk 
balance is not acceptable, then the participant must discontinue the IMP. However, if a 
participant experiences >2 severe headaches that is considered related to the IMP in the opinion 
of the investigator, the treatment must be permanently discontinued.  
Dosing modifications are allowed if judged necessary by the investigator from 
 to 
 or from
to 
 based on specific volume reduction per protocol 
guidance, provided the treatment is effective as evidenced by relevant efficacy measurements 
(eg, platelet count ≥30×109/L).  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 148 of 199 
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 a health care professional. The analgesic can 
be started at the 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 moderate or severe headache after discussion 
with the Medical Monitor. The benefit and risk of continuing treatment with IMP and chronic 
prophylactic treatment with analgesics must be carefully evaluated by the investigator. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 149 of 199 
10.21 
Appendix 21: Management of diarrhea 
Severe (Grade 3) diarrhea is defined as an increase of ≥7 stools per day or hospitalization for 
management of diarrhea or limiting self-care ADL. Determination of the severity of diarrhea will 
be consistent with CTCAE version 5.0 (see Appendix 3, Section 10.3, for further details). 
Diarrhea will be treated as clinically indicated according to the local guidelines. 
Stool samples may be collected for stool analysis to rule out infection for study participants 
reporting severe diarrhea. Stool sampling will be done as clinically indicated in the opinion of 
the investigator and assessed per local guidance. In addition, collection of blood samples for 
assessment of exploratory safety biomarkers is required for study participants with severe GI 
disturbances including diarrhea.  
In study participants experiencing severe treatment-related diarrhea, dosing modifications are 
allowed, if judged necessary by the investigator from 
to 
or from 
 to 
 based on specific volume reduction per protocol guidance, provided the 
treatment is effective as evidenced by relevant efficacy measurements (eg, platelet count 
≥30×109/L). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 150 of 199 
10.22 
Appendix 22: Management of infections and 
hypogammaglobulinemia 
Study participants who have the 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/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 EW Visit, enter the SFU Period and complete the EOS Visit. 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. 
To maintain the study integrity, study site personnel, the UCB and CRO study teams will remain 
blinded to IgG levels until Week 4 (Visit 5). However, to ensure study participants’ safety, serum 
IgG level will be monitored by unblinded Medical Monitors external to UCB until Week 4 
(Visit 5) and by the blinded Medical Monitors starting with the blood samples taken at Week 4 
(Visit 5). In case the serum IgG level decreases <1g/L, as described below and in Table 10-7, the 
unblinded/blinded Medical Monitors will inform the investigator.  
The most recently available IgG value is the trigger for discussions between the 
unblinded/blinded Medical Monitors and the investigator and for any decisions on 
changing/skipping the dose of IMP. All discussions and decisions related to the benefit-risk of 
dose change/skip are to be based on a holistic approach of the study participant’s status, 
including platelet response (eg, study participant’s ITP history, stability of the current response, 
past and current platelet counts), current signs and symptoms of nonserious/serious infection, 
potential risk of acquiring a nonserious/serious infection in the foreseeable future and are at the 
sole discretion of the investigator.  
The investigator must document the respective benefit-risk assessments and decisions in the 
study participant’s source documents.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 151 of 199 
Table 10-7: Management of infections and hypogammaglobulinemia 
IgG value 
Actions  
Outcome 
IgG <1g/La 
Unblinded medical monitor 
informs the investigator 
Investigator decides to either: 
• maintain the dose 
• reduce the dose 
• skip dose  
based on the study 
participant’s status regarding 
platelets response, signs & 
symptoms of nonserious/ 
serious infection, potential 
risk of acquiring a 
nonserious/serious infection 
AE=adverse event; eCRF=electronic case report form 
a Events of hypogammaglobulinemia must not be captured as an AE in the eCRF, to protect the blind.  
Dose adjustments/dose skipped  
The dose of IMP can be reduced from 
equivalent (eqv.) to 
 eqv., from
 
eqv. to 
 (approx. 
), or directly from 
eqv. to 
 (approximately 
), as needed.  
The IgG would be expected to return to values ≥1g/L in up to 3-4 weeks (after a dose 
decrease/skip). The level of IgG recovery will depend on the IgG observed value that triggered 
the intervention and the intra-study participants variability, so that the IgG level may not 
immediately increase, and a delayed effect could be expected.  
The study participant will need to be reassessed on an ongoing basis regarding platelet response 
and risk of infection until the serum IgG returns to values ≥1g/L. The dose may be further 
decreased, or it can be skipped, if needed. 
In case a dose is skipped, IMP administration may be restarted at a lower dose compared to the 
previous dose in order to decrease the likelihood of any further repeated reduction in the IgG 
level <1g/L.  
Ad hoc assessments (eg, additional weekly IgG samples) may be performed to monitor recovery 
of IgG levels.  
Splenectomized Study Participants 
Splenectomised study participants are at a higher risk of infections, especially of overwhelming 
post-splenectomy infections. Irrespective of the IgG level, in case a splenectomised study 
participant develops a (persistent or re-occurring) nonserious infection, the dose of IMP can be 
reduced, or the IMP treatment may be temporarily stopped, when appropriate, at the 
investigator’s discretion. The IMP treatment may be resumed at the previous dose after the 
resolution of the event. The investigator may also reach out to the unblinded/blinded Medical 
Monitors, as needed.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 152 of 199 
Local guidelines regarding antibiotic prophylaxis in splenectomised study participants should be 
followed. Splenectomised study participants should have a prophylactic antibiotic available, 
unless they are already taking an antibiotic as per local guidance.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 153 of 199 
10.23 
Appendix 23: Management of infusion-related reactions or 
hypersensitivity reactions 
Study participants must be closely monitored for reactions during and after the IMP 
administration period. 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 10-8. Definitions of the severity 
of the relevant events will be consistent with CTCAE version 5.0 (see Appendix 3, Section 10.3). 
Table 10-8: Suggested management guidelines for infusion reactions 
Type of reaction 
Suggested action 
Acute – Mild 
Grade 1 
Monitor vital signs every 10 min. 
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 NSAIDs. 
Monitor vital signs initially every 5 min. 
If the reaction improves and upon further assessment it is clear that the 
event is not an anaphylaxis, restart the infusion cautiously. Continue to 
monitor vital signs every 5 min. 
If reaction recurs or worsens to Grade 3, discontinue infusion. 
Acute – Severe 
Grade 3 or 
anaphylaxis 
Discontinue IMP infusion permanently. 
Alert crash team. 
Maintain airway; ensure oxygen is available. 
Administer: 
− Antihistamine iv/im, corticosteroids iv, epinephrine im, and iv 
fluids as appropriate. 
− Monitor vital signs every 2 min. 
− Hospitalize, if condition not improving or worsens. 
− Monitor study participant until symptoms resolve. 
CTCAE=Common Terminology Criteria for Adverse Events; im=intramuscular; IMP=investigational medicinal 
product; iv=intravenous(ly); NSAIDs=Nonsteroidal anti-inflammatory drugs 
Note: Management criteria were adapted from the CTCAE v5.0 (National Cancer Institute, 2017). 
In case of suspected anaphylaxis, the Sampson Criteria (Sampson et al, 2006) should be accessed 
and Appendix 19 (Section 10.19, Sampson Criteria Questionnaire) should be completed. The 
infusion must be discontinued immediately, and emergency resuscitation measures implemented. 
In study participants experiencing an infusion-related reaction or anaphylaxis, blood samples will 
be collected as soon as possible, while the event is ongoing, to investigate the nature of the 
reaction as per Schedule of Activities (Section 1.3). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 154 of 199 
Samples for serum complement s(C3, C4), plasma complements (C3a, C5a) should be collected 
as specified in the Schedule of Activities (Section 1.3). Additional tests such as IgE levels, 
tryptase may be performed when there is a suspicion of Type I or III hypersensitivity reaction. 
The results of all monitoring, including laboratory testing, should be made available to the study 
site and sponsor. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 155 of 199 
10.24 
Appendix 24: SIAQ (infusion version) 
PRE-SIAQ (Infusion version):  
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 156 of 199 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 157 of 199 
POST-SIAQ (Infusion version):  
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 158 of 199 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 159 of 199 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 160 of 199 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 161 of 199 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 162 of 199 
10.25 
Appendix 25: Protocol Amendment History 
Amendment 3 (09 Dec 2021) 
Overall Rationale for the Amendment 
The primary reason for this protocol amendment is to correct errors in Table 1-1 Schedule of 
Assessment at Weeks 19, 23, 27 and 51.  
Additional updates were incorporated to provide further clarity on the protocol or to correct 
errors. 
This amendment is considered to be non-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, Objectives 
and Endpoints 
3 Objectives and endpoints 
Footnote “a” was added to the 
endpoints referring to “Baseline”. 
Updated for consistency.  
1.1 Synopsis, Objectives 
and Endpoints 
3 Objectives and endpoints 
The following secondary objective 
and endpoint were removed:  
• 
To assess the 
pharmacodynamic (PD) effect 
of rozanolixizumab  
• 
Total serum IgG (absolute 
value) and change from 
Baselinea (absolute value and 
percentage) in serum IgG and 
in serum IgG subclass 
concentration at each scheduled 
assessment   
To remove duplication as they will 
be assessed as an exploratory 
objective and endpoint. 
1.1 Synopsis, Overall 
design 
 
4.1 Overall design 
The sentence “In case of a late 
rollover, platelet counts will need 
to be remeasured.” was added.  
The sentence was deleted in error 
in Protocol Amendment 2.  
1.3 Schedule of activities, 
Table 1-1 Schedule of 
activities 
The following assessments were 
added to Weeks 19, 23, 27 and 51: 
Platelet count, vital signs, recording 
of AEs (including hospitalizations), 
serum complements (C3 and C4) 
and plasma complements (C3a and 
C5a), blood collection for 
exploratory biomarker analysis, 
ITP bleeding scale and headache 
questionnaire.  
Updated for consistency and to 
correct errors.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 163 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
Assessments for pre-SIAQ and 
post-SIAQ were added at all dosing 
visits starting at Week 7.  
8.1.5.1 Tuberculosis 
assessment 
A new subsection for monitoring 
for TB during the study was added.  
Added for consistency across ITP 
protocols.  
Amendment 2 (03 Dec 2021) 
Overall Rationale for the Amendment 
The primary reason for this protocol amendment was the recommendation of the external 
Independent Data Monitoring Committee (IDMC) to modify the dosing regimen of the study. 
Additional updates were incorporated to provide further clarity on the protocol or to correct 
errors. 
This amendment is considered to be substantial based on the criteria set forth in Article 10(a) of 
Directive 2001/20/EC of the European Parliament and the Council of the European Union. 
Section # and Name 
Description of Change 
Brief Rationale 
Global 
IMP dosing 
 was 
updated to
.  
 
Updated based on the feedback 
from the IDMC recommending the 
option to increase the frequency in 
dosing, to reduce the potential 
risks associated with low platelet 
counts. 
Global 
Exploratory dosing was removed.  
Reference to maintenance dosing 
was removed. 
 
Updated based on the feedback 
from the IDMC recommending the 
option to increase the frequency in 
dosing, to reduce the potential 
risks associated with low platelet 
counts. Therefore, the exploratory 
arm intended to evaluate wider 
dosing intervals was removed as 
the scientific value is limited. 
Global 
54-week treatment period was 
updated to 52-week treatment 
period. 
Updated to provide clarity and 
consistency with remainder of the 
protocol. 
1.1 Synopsis 
Rationale 
Reference to the starting dose of 
fixed dose equivalent of
 
was removed. 
Deleted as the 
 equivalent 
starting dose is no longer applied 
in the parent studies TP0003 and 
TP0006.  
1.1 Synopsis, Objectives 
and Endpoints 
3 Objectives and endpoints 
 
Objectives corresponding to the 
exploratory dosing were removed. 
Reference to maintenance dosing 
was removed.  
 
 
Updated to provide consistency 
within the protocol due to the 
removal of the exploratory arm. 
 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 164 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
Objectives were reworded from 
“…starting after the second dose of 
rozanolixizumab (Week 4)” to 
“…starting at Week 4.” 
Updated to reflect the changes in 
the dosing frequency. Reference to 
the "second dose" was deleted and 
reference is only made to Week 4. 
1.1 Synopsis, Objectives 
and endpoints 
 
3 Objectives and endpoints 
 
The other safety endpoints was 
updated to delete “α globulin and β 
globulin.” 
The other safety endpoint: 
“Change from Baseline in 
cytokines during the study (for 
study participants experiencing 
infusion reactions or 
hypersensitivity reactions)” was 
deleted. 
The endpoint was removed to 
decrease study complexity, as 
scientific value is limited. 
 
The endpoint was removed as the 
analysis of cytokines is not 
required in the ITP protocols as 
samples have been collected in 
other rozanolixizumab studies and 
no additional information is 
expected from the ITP population. 
1.1 Synopsis, Objectives 
and endpoints 
 
3 Objectives and endpoints 
The following PD secondary 
endpoint was updated as follows: 
“Minimum value in total serum 
IgG and maximum decrease from 
Baseline in total serum IgG 
concentration” was changed to 
“Total serum IgG (absolute value) 
and change from Baseline (absolute 
value and percentage) in serum IgG 
and in serum IgG subclass 
concentration at each scheduled 
assessment” 
Updated as this variable will be 
followed at every scheduled visit 
rather than only reporting the 
lowest value across the duration of 
the study. 
1.1 Synopsis, Objectives 
and endpoints 
 
3 Objectives and endpoints 
 
The following exploratory PD 
endpoints were updated as follows:  
“Minimum value in total serum 
IgG and maximum decrease from 
Baseline in total serum IgG 
concentration” was deleted. 
 
 
 
“Change from Baseline in serum 
IgG and in serum IgG subclass 
concentration over time” was 
updated to “Total serum IgG 
(absolute value) and change from 
Baseline (absolute value and 
percentage) in serum IgG and in 
serum IgG subclass concentration 
at each scheduled assessment.” 
“Change from Baseline in serum 
immunoglobulin concentrations 
 
 
Deleted as this variable will be 
followed at every scheduled visit 
rather than only reporting the 
lowest value across the duration of 
the study. 
 
 
Endpoint was amended to provide 
further details of the endpoint and 
to add clarity. 
 
 
 
 
 
 
Endpoint was amended to provide 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 165 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
(IgA, IgE, IgM)” was updated to 
“Absolute value and change from 
Baseline (absolute value and 
percentage) in serum 
immunoglobulin concentrations 
(IgA, IgE, IgM) at each scheduled 
assessment.” 
“Change from Baseline in ITP-
specific autoantibodies in serum” 
was deleted. 
further details of the endpoint and 
to add clarity. 
 
 
 
 
 
 
 
The endpoint was deleted as the 
results of the analysis will not be 
part of the CSR of this study and 
will be described in a separate 
report. 
1.1 Synopsis, Objectives 
and endpoints 
 
3 Objectives and endpoints 
The objective “To evaluate the 
effects of rozanolixizumab on 
exploratory biomarkers” and 
associated endpoints were 
removed.  
Objective and endpoint were 
removed as the exploratory 
biomarker samples are taken for 
future analysis, with no predefined 
analysis objective and endpoints 
1.1 Synopsis 
Overall design 
 
4.1 Overall design 
 
IMP dosing 
 was 
updated to 
 dosing.  
Rozanolixizumab dose 
administration was updated from 
Week 3, Visit 5 to Week 2, Visit 3. 
Updated upon recommendation of 
the IDMC   
 
Updated to reflect the dosing 
frequency change from 
 
to 
dosing. 
 
1.1 Synopsis, Overall 
design 
 
4.1 Overall design 
 
The visit window upon rollover 
from TP0003 or TP0006 into 
TP0004, has been updated from 1 
week to 3 days.  
The requirement for a SFU visit in 
between or platelet count 
measurement in case of late 
rollover, has been removed.  
The wording on rozanolixizumab 
infusion was updated. 
The timepoints for observation 
after the end of infusion were 
updated.  
Updated for consistency with the 
 dosing regimen. 
1.1 Synopsis, Overall 
design 
 
4.1 Overall design 
The following paragraph was 
added: “Based on feedback from 
the IDMC, 
 dosing was 
implemented in protocol 
amendment 2. Study participants 
being treated with the 
 
dosing regimen will switch to the 
To clarify the dosing for existing 
study participants receiving 
 dosing 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 166 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
 dosing regimen once 
protocol amendment 2 is approved 
at the respective study site.” 
1.1 Synopsis 
Number of study 
participants 
Reference to the interim analysis 
was removed.  
To provide consistency with the 
feeder study protocols. The interim 
analysis in the feeder studies was 
removed.  
1.1 Synopsis 
Treatment groups and 
duration  
 
4.1 Overall design 
The total maximum study duration 
per study participants was updated 
to 59 weeks. 
To provide consistency within the 
protocol due to the change in 
dosing frequency. 
1.2 Schema 
Figure 1-1 TP0004 study 
schematic  
IMP dosing 
 was 
updated to 
 dosing.  
Reference to maintenance dosing 
was removed and changed to 
“treatment period.” 
The treatment period was corrected 
to 52 weeks.  
Week 55 was updated to Week 53. 
The Safety Follow-up Period was 
updated to Week 60.  
The total maximum study duration 
per study participants was updated 
to 59 weeks. 
To provide consistency within the 
protocol due to the change in 
dosing frequency and the removal 
of the exploratory arm.  
1.3 Schedule of Activities  
Table 1-1 Schedule of 
activities  
IMP dosing 
 was 
updated to 
 dosing. IRT 
contacts were updated accordingly. 
Columns related to home visits 
have been grayed out. 
Notes were added to clarify white 
columns are site visits and gray 
columns are optional home visits. 
Additional text was added to state 
“Home IMP administration and 
assessments are optional and can be 
conducted (if approved by 
regulatory agencies) at the sites as 
deemed necessary by site personnel 
and/or the study participant” 
The safety follow-up period was 
amended from W61, Day 420 to 
W60, Day 414. 
Updated to provide consistency 
within the protocol due to the 
change in dosing frequency, last 
dose being at Week 52 and the 
removal of the exploratory arm.  
 
Updated to harmonize the 
assessments and visits with the 
change in dosing regimen.  
 
 
 
 
 
 
 
 
 
 
The visit at Day 5 was removed 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 167 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
A +2 day time window for Week 1, 
Day 3 was added.  
A new footnote “a” was added: 
“The allowed time window for 
assessments is ±5 mins for 
assessments <30 mins and ±15 
mins for assessments >30 mins.” 
Footnote a now b: was updated to 
clarify that home visits, if 
applicable are displayed in the grey 
columns. The following sentence 
was removed: “If applicable, home 
administration of IMP is to be 
conducted unless not approved by 
regulatory agencies.” 
Footnote f now g:  was updated 
from “50 to 200×109/L” to “50 to 
150×109/L and no dose change.” 
Footnote g now h: visit window for 
rollover from TP0003 or TP0006 
was updated to +3 days. 
A new footnote “i” was added: 
“Platelet counts will be taken at the 
indicated home visits; however, the 
IMP dose will be based on the 
previous 4 platelet counts (must be 
stable between 50 to 150×109/L 
and no dose change). If criteria are 
not met, the visit must be 
conducted on site.” 
Footnote h now j: was updated to 
remove triplicate and three ECGs 
and the timepoints for ECG.  
Footnote j now k: vital sign 
assessment timepoints were 
updated. Footnote p now q: relating 
to onsite observation was updated 
accordingly.  
A new footnote “p” was added: 
“Prior to dosing, management of 
infections and 
hypogammaglobulinemia must be 
considered as per Appendix 22, 
Section 10.22.” 
Footnote q relating to the 
Exploratory Treatment Arm was 
deleted.  
therefore the Visit at Day 3 was 
given a visit window. 
 
To clarify the time window for 
assessments.  
 
 
 
Updated for clarity.  
 
 
 
 
 
 
 
Updated for consistency across 
other sections of the protocol. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Updated for clarity.  
 
 
 
 
 
 
 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 168 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
Footnote u now s: was updated to 
remove the 4 hour postdose 
assessments at Baseline and Week 
33.  
Footnote “t” relating to 2 to 5 days 
postdose PK and ADA samples 
was deleted.  
Footnote “u” was updated to add 
“and/or serious.”  
A new footnote “v” was added: If 
total IgG levels are <1g/L, ad hoc 
assessments (eg, additional IgG 
samples) may be performed to 
monitor recovery of IgG levels. See 
Section 10.22.  
Footnote aa now z:  was updated as 
follows: This assessment is 
applicable only to participants 
experiencing moderate, severe 
and/or serious headache (see 
Section 10.20), and will be 
performed daily until resolution (ie, 
if headache becomes mild, normal 
collection of AEs should apply). 
This assessment can also be done 
remotely eg, via phone interview. 
The IGRA TB test and chest x-ray 
were deleted.  
 
 
The 4 hour postdose sample is not 
required.  
 
 
These samples are not required. 
 
Updated for consistency across 
other sections of the protocol.  
 
 
 
 
 
 
 
Updated for consistency with 
Section 10.20. 
 
 
 
 
 
 
 
 
 
Updated for consistency with 
Section 8.1.5 
Table 1-2 Schedule of 
activities – exploratory 
treatment arm (Weeks 25 
to 41) 
Table 1-2 was deleted.  
Updated to provide consistency 
within the protocol due to the 
removal of the exploratory arm.   
2.2. Background  
The background was updated to 
state the removal of the starting 
dose from feeder studies TP0003 
and TP0006. 
CIDP01 was added to the list of 
completed studies.  
 
Brief details about CIDP01 and 
CIDP04 were added. 
A note was added that in 
TP0003/TP0006 protocol 
amendment 3, the starting fixed 
dose equivalent to 
 sc was 
Updated for consistency with the 
change in dosing regimen, and 
with relevant completed or 
ongoing study information. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 169 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
removed due to the change to 
weekly dosing. 
2.3 Benefit/risk assessment  The following cross reference was 
corrected from Section 6.5.1 to 
Section 8.2.9: “If any AEs were to 
occur, they should be handled as 
described in (Section 8.2.9) with 
causality assessment provided for 
both IMP and vaccine.” 
Cross reference was corrected.  
Table 4-1 TP0004 dose 
levels and weight tiers of 
IMP 
The table title was updated from 
‘rozanolixizumab’ to ‘IMP’. 
 
Updated to provide consistency 
within the protocol.  
Table 4-2 Dose 
adjustments of 
rozanolixizumab for 
maintenance treatment arm 
 
Figure 4-1 Dose 
adjustments of 
rozanolixizumab 
Additional instructions were added 
for platelet counts of <10×109/L 
and between ≥30×109/L to 
<50×109/L. 
Updated to provide further clarity 
on when rescue medication is to be 
used.  
Table 4-2 Dose 
adjustments of 
rozanolixizumab for 
maintenance treatment arm 
 
Figure 4-1 Dose 
adjustments of 
rozanolixizumab 
A clarification was added that 
rescue therapy is recommended 
when platelet count reaches 
<30×109/L. An additional row/box 
was added to clarify that platelet 
count <10×109/L instructions 
would be to administer rescue 
therapy. 
The text regarding interindividual 
variable platelet response 
previously in the section body was 
included as footnote ‘b’ to the 
table. 
Updated for clarity 
4.1 Overall design 
Vital signs measurement time 
points for all visits were updated 
and clarified. 
Updated for consistency with the 
change in dosing regimen. 
4.1 Overall design, Table 
4-2, Figure 4-1 
The following text was updated: 
Platelet counts of <10×109/L rescue 
therapy is highly recommended.  
 
Platelet counts of ≥10×109/L to 
<30×109/L (on at least two 
consecutive visits) rescue therapy is 
recommended.  
 
Updated to permit reduction in 
dose earlier in the event of a 
platelet increase given the change 
to a 
 dosing regimen.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 170 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
≥30×109/L to <50×109/L dose level 
can be increased unless the study 
participant is on a maintenance 
dose of 
.  
 
Platelet count of ≥50×109/L to 
≤200×109/L was updated to 
≥50×109/L to 150×109/L.  
 
Platelet count of >200×109/L to 
<400 x109/L was updated to 
150×109/L to <400 x109/L. 
 
For platelet counts of ≥400×109/L, 
instructions were updated to “Once 
the platelet count is ≤150×109/L, 
reinitiate treatment decreased by 
one dose level.” 
 
The text regarding interindividual 
variable platelet response was 
moved as table footnote b. 
4.3 Justification for dose  
A more frequent dosing regimen of 
 equivalent once per week 
is introduced in TP0003/TP0006 
and the  OLE study.  
The 
 equivalent starting 
dose in TP0003/6 is removed from 
the protocol. 
Updated to follow the advice of the 
external IDMC on introducing a 
more frequent dosing regimen to 
reduce the potential risks of low 
platelet counts. 
5.1 Inclusion criteria 
Criterion #1a, now 1b, was updated 
to state Visit 27. 
Updated as per updated visit 
numbering in studies 
TP0003/TP0006.  
5.1 Inclusion criteria  
Inclusion criteria #3 and #4 were 
removed.   
Inclusion criteria 3 and 4 were 
deleted as the criteria are not 
related to eligibility. Inclusion 
criterion 3: Highest permitted 
concomitant treatments are listed 
in table 6-2 and a stable dose is not 
required as an inclusion criterion 
Inclusion criterion 4: Depending 
on the IgG level, the participant 
should be dosed in accordance to 
Appendix 22 and IgG levels are 
not an inclusion criterion. 
5.1 Inclusion criteria 
Inclusion criterion #5, now 5a, was 
updated to correct “or” to “and” 
The sentence was corrected.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 171 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
“as confirmed by a negative 
pregnancy test and not planning to 
get pregnant” 
5.2 Exclusion criteria 
Exclusion criterion #6a, now 6b, 
was updated to remove the bullet 
point relating to ALT, AST, or 
ALP results up to 25% above the 
exclusion limit may be repeated 
once for confirmation. 
Updated for providing additional 
clarity to this exclusion criterion. 
 
5.2 Exclusion criteria 
Exclusion criterion 12 was 
removed. 
No longer required as Baseline 
values are taken from 
TP0003/TP0006.  
6 Study treatments  
Table 6-1 Treatments 
administered  
Exploratory dosing was removed.  
IMP dosing 
 was 
updated to 
 dosing. 
Dosing instructions were updated.  
Updated to provide consistency 
within the protocol.   
6 Study treatments  
Table 6-1 Treatments 
administered 
Dosing instructions: “and Week 53 
measurement” was removed.  
Removed as the Week 53 body 
weight measurement is not used 
for the dose calculation.  
6.2 Measures to minimize 
bias: randomization  
Description of the Exploratory  
Arm was removed.  
Updated to provide consistency 
within the protocol.  
6.4.1 Permitted 
concomitant treatments 
(medications and 
therapies) 
An additional paragraph regarding 
the management of the concomitant 
ITP treatment of the study 
participants rolling over from 
TP0003/TP0006 to TP0004.  
To clarify the management of the 
concomitant ITP treatment of the 
study participants rolling over 
from TP0003/TP0006 to TP0004. 
6.4.1 Permitted 
concomitant treatments 
(medications and 
therapies), Table 6-2 
6.4.3.1 Rescue therapy not 
leading to discontinuation 
Permitted oral corticosteroids dose 
was increased to 20mg/day. 
Allowed dose of oral 
corticosteroids was increased to a 
maximum of 20mg/day for more 
flexibility in the managing the 
concomitant ITP treatment.  
6.4.3.2 Rescue therapy 
leading to discontinuation 
Oral steroids above 10mg/day was 
updated to 20mg/day. 
Updated to provide consistency 
with other changes in the protocol.  
6.5 Dose modification 
Individual requirements for 
splenectomized patients were 
removed. 
Updated for consistency with the 
changes in Section 10.22. 
6.5 Dose modification 
A sentence was added to state that 
Dose modifications or temporary 
IMP treatment discontinuation are 
permitted if serum IgG value 
are<1g/L (Section 10.22). 
Updated for consistency with the 
changes in Section 10.22. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 172 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
6.6 Self-administration  
A new subsection was added 
regarding details for self-
administration 
Updated and introduced as a 
separate section to provide more 
details on the optional IMP self-
administration 
6.7 Home visits   
A new subsection was added 
regarding details for home visits 
Updated to separate the optional 
self-administration from the home 
visits and to provide more details 
on the optional home visits 
7.1.1 Liver chemistry 
stopping criteria  
Figure 7-1 
Figure 7-2 
Cross reference to Appendix 6 was 
corrected to Appendix 5. 
Updated to provide the correct 
reference   
7.1.2 QTc stopping criteria  Triplicate ECG was removed 
Updated to reflect the changes in 
Section 8.1.3. 
7.1.3 Discontinuation of 
IMP due to other adverse 
events or medical 
condition 
The text was updated to clarify that 
the 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. 
Updated to provide clarity  
7.1.4 Temporary IMP 
discontinuation 
Text was updated to clarify that: 
A study participant may 
temporarily discontinue IMP if any 
of the following events occur:  
3. A severe AE of headache that 
is considered related to the 
IMP in the opinion of the 
investigator (Section 10.20).  
4. A splenectomised study 
participant develops a 
(persistent or re-occurring) 
nonserious infection, as per 
investigator’s decision 
(Section 10.22). 
5. Total serum IgG values goes 
<1g/L as per investigator’s 
decision (Section 10.22). As 
IMP treatment is 
administered 
 the 
decision is based on the most 
recently available total IgG 
value. In order to maintain 
 
 
 
 
To be consistent with Section 
20.20. 
 
 
 
To reflect changes in the process of 
managing infections and events of 
hypogammaglobulinemia and for 
consistency with the changes in 
Section 10.22. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 173 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
the blind from the parent 
studies, IgG, IgG subclasses, 
total protein, and albumin 
values will be unblinded 
starting with the blood 
samples taken at Week 4 
(Visit 5).  
7.2 Participant 
discontinuation/withdrawal 
from the study 
#3 was added as follows: 3. An 
adverse event or a medical 
condition as described in Section 
7.1.1, Section 7.1.2 or Section 7.1.3 
occur. 
Added for consistency throughout 
the protocol.  
7.3 Lost to follow-up 
“to complete the final evaluation” 
was deleted from the last 
bulletpoint 
No final evaluation is required for 
a study participant who is lost to 
follow-up. 
8.1.2 Vital signs  
Body temperature was added to the 
list of vital signs to be measured 
before blood samples are taken. 
Updated for further clarity and 
consistency throughout the 
protocol. 
8.1.3 Electrocardiograms 
Text was updated to remove 
references to triplicate 
ECGs:“Triplicate 12 lead ECG” 
ECG was replaced with “A 12-lead 
ECG.” 
No identification of cardio-toxicity 
from non-clinical data, supported 
by lack of signal of cardiac events 
in the rozanolixizumab-
program.  Updated in order to 
decrease the burden to the sites and 
study participants.  
8.1.5 Assessment and 
management of TB and TB 
risk factors 
The text was updated to remove 
“appropriate rigorous” and to add 
that assessment and management of 
TB and TB risk factors should 
follow local or national guidelines.  
Patients with known tuberculosis 
(TB) infection, at high risk of 
acquiring TB infection, or latent 
tuberculosis infection (LTBI), or 
current/history of nontuberculous 
mycobacterial infection (NTMBI) 
are already excluded from 
TP0003/TP0006, therefore the 
sites are expected to follow 
national/local guidelines for the 
management of TB, in case 
needed. 
In addition, the mechanism of 
action of rozanolixizumab is 
expected to have little or no impact 
on the immune response against 
intracellular organisms which is 
involved in controlling the 
infection with Mycobacterium 
tuberculosis.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 174 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
8.1.5.1 Tuberculosis 
assessment  
Sections on physical examination 
for TB, TB assessment by IGRA, 
and TB assessment by chest x-ray 
were removed.  
Updated for consistency with 
Section 8.1.5 
8.1.5.2 Tuberculosis 
management 
Test conversion for IGRA was 
removed. Under Latent TB, text 
regarding 2 indeterminate IGRA 
test results as suggestive of TB 
infection was removed. 
Updated for consistency with 
Section 8.1.5 
8.1.5.2 Tuberculosis 
management 
The definition for LTBI was 
updated.  
The definitions for active TB and 
NTMBI were added.  
LTBI definition updated as per 
WHO and CDC guidelines. Active 
TB and NTMBI definitions as per 
WHO and CDC guidelines were 
added for consistency.  
8.1.5.2 Tuberculosis 
management 
Text pertaining to AE reporting for 
Latent TB was removed.  
Updated for consistency in 
reporting any TB related AEs as 
per Section 8.1.5.1. 
8.2.8 Adverse events of 
special monitoring 
Text was added to state that the 
headache questionnaire should also 
be completed in case of moderate 
headache although moderate 
headache is not considered an 
AESM. 
Updated for consistency with the 
changes in Section 10.20. 
8.4 Treatment of overdose  
The numbered list was changed to 
bullet points.  
Formatting correction.  
8.5.1 Platelet counts 
Reference to the maintenance 
treatment arm was removed. 
The endpoints related to the 
Exploratory Treatment Arm were 
removed. 
Platelet count endpoints were 
updated. 
Updated to provide consistency 
within the protocol 
8.7 Pharmacodynamics 
A sentence was added to state that 
results of the ITP-specific 
autoantibodies analyses will not be 
outlined in the Clinical Study 
Report for this study. 
Analysis of the ITP-specific 
autoantibodies is a lengthy method 
and therefore the results will be 
provided in a separate report. 
8.8 Exploratory 
biomarkers  
α- and β-globulinswere removed.  
Sampling of α- and β-globulins 
was removed to decrease study 
complexity, as scientific value of 
the evaluation is limited. 
8.8 Exploratory 
biomarkers 
The text was updated to add 
“and/or serious.” 
Updated to provide consistency 
within the protocol. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 175 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
8.9 Immunology 
biomarkers  
The serum complements sample 
4 hours postdose at Baseline and 
Visit 23 (Week 33) was removed.  
Updated to provide consistency 
within the protocol. 
8.9 Immunology 
biomarkers 
Serum cytokines were deleted. 
Blood sampling for cytokine 
analysis was removed in the ITP 
protocols as samples have been 
collected in other rozanolixizumab 
studies and no additional 
information is expected from the 
ITP population. 
9.1 Definition of analysis 
sets  
The Exploratory Treatment Arm 
Safety Set was removed.  
Updated to provide consistency 
within the protocol. 
9.3 Estimands  
Safety Set and estimand was 
updated to state “who started 
TP0004 on 
 dosing.” 
Updated to reflect that estimand 
concerns subjects who started on 
 dosing. 
9.3 Estimands 
A further secondary safety 
estimand, also based on “treatment 
policy” was added.  
Updated to reflect the two dosing 
regimens. 
9.4 Planned safety analysis 
The text was updated to add 
“started TP0004 on 
 or 
 dosing” 
Updated to reflect change in 
dosing regimen. 
9.5 Planned 
efficacy/outcome analyses  
The text was updated to add 
“started TP0004 on 
 or 
 dosing” 
Updated to reflect change in 
dosing regimen. 
9.5 Planned 
efficacy/outcome analyses 
The wording for the secondary 
efficacy endpoints were updated.   
Updated to reflect change in 
dosing regimen and the removal of 
exploratory arm. 
9.6.1 Analysis of 
pharmacodynamic 
endpoints 
α- and β-globulins were removed.  
Updated for consistency with 
Section 8.8. Sampling of α- and β-
globulin biomarkers were removed 
to decrease study complexity, as 
scientific value of the evaluation is 
limited. 
9.6.1 Analysis of 
pharmacodynamic 
endpoints 
9.6.2 Analysis of 
pharmacokinetics 
endpoints  
Text was updated to state “by 
dosing regimen” 
Updated to reflect the change in 
dosing regimen. Analysis can be 
done based on 
 or 
 
dosings. 
9.10 Determination of 
sample size 
The text was updated to state “up to 
a maximum of” 
Updated to provide further clarity  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 176 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
10.2 Appendix 2: Clinical 
laboratory tests 
Table 10-1 Protocol-
required safety laboratory 
assessments 
Assessment of % platelet 
reticulocytes and associated 
footnotes were removed.  
Updated to reflect that the analysis 
will not be performed.  
10.2 Appendix 2: Clinical 
laboratory tests 
Table 10-1 Protocol-
required safety laboratory 
assessments 
Low-density lipoprotein (LDL), 
High-density lipoprotein (HDL), 
Total cholesterol and Triglycerides 
were moved under Clinical 
Chemistry. 
Moved under clinical chemistry as 
these will be measured as part of 
clinical chemistry panel.  
10.2 Appendix 2: Clinical 
laboratory tests 
Table 10-1 Protocol-
required safety laboratory 
assessments 
Footnote e was updated as follows: 
“Includes total IgG, IgG subclasses, 
IgA, IgM, and IgE. Results of the 
IgG testing will be unblinded 
starting with the blood samples 
taken at Week 4 (Visit 5 ) in 
TP0004 in order maintain blind of 
from the parent studies. 
To be consistent with Section 22.  
10.5 Appendix 5: Liver 
safety 
Table 10-4 Phase 3-4 liver 
chemistry increased 
monitoring criteria with 
continued IMP 
The cross reference to the liver 
chemistry stopping criteria was 
corrected.  
Updated to provide the correct 
reference. 
10.7, Appendix 7: 
Country-specific 
requirements 
France 
The visit window of +7 days was 
updated to +3 days.  
Updated to align with the updated 
visit window.  
10.7, Appendix 7: 
Country-specific 
requirements 
Japan 
TB assessment and chest x-ray for 
TB were removed.  
Updated for consistency 
throughout the protocol. 
10.7, Appendix 7: 
Country-specific 
requirements 
Japan 
A cross reference was added to 
Table 10-2 and the wording was 
updated.  
Updated for clarity.  
10.7, Appendix 7: 
Country-specific 
requirements 
Section 10.7.1 was added to list the 
countries where self-administration 
will be offered as an option  
Updated to provide further clarity 
on the countries where self-
administration will be offered as an 
option 
10.9 Appendix 9: 
Abbreviations and 
trademarks 
Additional abbreviations and 
definitions were added for: 
General update.  
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 177 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
CDC, eqv, HCP, QW, RO, and 
WHO. 
10.20 Appendix 20: 
Management of headache 
The text was updated to clarify the 
management of IMP administration 
including dose adjustment 
following an AE of headache. 
Updated to reflect the current 
general program guidance for the 
management of headaches. 
10.22 Appendix 22: 
Management of infections 
and 
hypogammaglobulinemia 
The text was updated to clarify the 
management of IMP administration 
including dose adjustment 
following episodes of infections 
and hypogammaglobulinemia. 
The total serum IgG cut-off value 
for informing the investigators has 
been aligned for all study 
participants based on the lack of 
conclusive literature supporting the 
previous approach.  
The investigators are given total 
flexibility in deciding if and how 
to continue the IMP treatment in 
cases of total IgG values < 1g/L, 
based on a holistic approach of the 
study participant’s status (platelets 
response, signs & symptoms of 
nonserious/ serious infection, 
potential risk of acquiring a 
nonserious/ serious infection).  
10.23 Appendix 23: 
Management of infusion 
reactions or 
hypersensitivity reactions 
Serum cytokines were removed. 
Blood cytokine sampling was 
removed as analysis of cytokines is 
not required in the ITP protocols as 
samples have been collected in 
other rozanolixizumab studies and 
no additional information is 
expected from the ITP population. 
10.25. Appendix 25: 
Protocol Amendment 
History  
Protocol amendment 2 (global 
amendment) has been added. 
Appendix no. 26 was corrected to 
no. 25 
General update. 
Throughout 
Minor editorial and formatting 
changes have been made. 
Minor, therefore have not been 
summarized. 
 
 
Amendment 1 (11 May 2021) 
Overall Rationale for the Amendment 
The primary reason for this protocol amendment was to incorporate changes in the objectives, 
endpoints, the statistical analysis section, as well as a specific study objective and endpoint on 
post-vaccination biomarkers in study participants who have received a COVID-19 vaccine. Other 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 178 of 199 
changes include incorporating local protocol amendments (0.1- Japan and 0.2-France only) into 
one global protocol. 
Additional updates were incorporated to provide further clarity on the protocol or to correct 
errors. 
This amendment is considered to be substantial based on the criteria set forth in Article 10(a) of 
Directive 2001/20/EC of the European Parliament and the Council of the European Union. 
Section # and Name 
Description of Change 
Brief Rationale 
Global 
Minor administrative, consistency, 
formatting and typographical 
changes have been made. 
The duration for the treatment 
period was clarified from 52 weeks 
to 54 weeks. 
Updated to provide clarity and be 
consistent with remainder of 
protocol. 
Updated to provide clarity, where 
the 54-week treatment period 
includes a 2-week observation 
period after Week 52. 
Global 
Japan-specific requirements have 
been highlighted throughout the 
protocol and added to Appendix 7, 
Section 10.7. 
In accordance with local 
requirements and actual condition 
of medical treatment in Japan. 
Serious adverse event 
reporting  
For serious adverse event reporting 
(24h), the fax number for Japan has 
been removed.  
For participants in Japan only: 
Additional reporting instructions 
for SAE reporting (investigational 
device) and device deficiency 
reporting specific for Japan have 
been included. 
In accordance with local 
requirements in Japan. Reporting 
instructions are included in the 
event the site uses devices that are 
not approved. a 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
For several objectives, wording on 
maintenance or exploratory dosing 
has been included. 
Updated to provide clarity on 
which study population the 
objective applies to. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
The primary objective has been 
changed to remove the following 
word, "repeated.” 
The following text has been added 
to the safety endpoint: 
Occurrence of TEAEs leading to 
permanent withdrawal of 
investigational medicinal product 
(IMP) (ie, study discontinuation) 
The other safety endpoint specific 
to occurrence of AESM was 
updated to read as “treatment-
emergent AESM”  
Updated to provide further clarity. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 179 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
The following word, "repeated" has 
been removed from the secondary 
and exploratory objectives relating 
to assessment the long-term clinical 
efficacy. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
For several endpoints, "first 
Complete Response" has been 
removed and "during both 
maintenance dosing and 
exploratory dosing" has been 
added. 
Updated to provide clarity with the 
data collected for the study. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
The secondary efficacy endpoint 
was amended to include "over the 
planned 54-week Treatment 
Period" 
Updated to allow flexibility for 
potential calculation based on 
available visits for participants 
prematurely withdrawn from the 
study. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
Footnotes have been updated: 
Footnote b, now a: Treatment cycle 
is defined as the period between 
two consecutive doses, unless 
otherwise noted in the Statistical 
Analysis Plan.  
Footnote b: Baseline definitions for 
the maintenance and exploratory 
arm treatment cycles will be 
detailed in the SAP. 
Updated to provide definitions for 
Treatment cycle and Baseline for 
the Maintenance and Exploratory 
Treatment arms (maintenance and 
exploratory dosing). 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
The other efficacy endpoints have 
been updated to include several 
changes, and two new endpoints, as 
listed below:  
- Stable Response defined as 
platelet count ≥30x109/L and 
absence of bleeding without rescue 
therapy at ≥70% of the visits over 
the planned 54-week Treatment 
Period starting after the second 
dose of rozanolixizumab (Week 4) 
- Cumulative number of weeks 
with platelet counts ≥30x109/L over 
the planned 54-week Treatment 
Period  
- Mean Change from Baseline  in 
platelet count at each visit 
- The rescue medication endpoint 
was changed from Time to first 
Updated as per query from the 
FDA on endpoints related to 
efficacy. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 180 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
rescue medication to "Use of 
rescue medication by visit"  
Additionally, the following 
endpoints were removed:  
- Complete Response, defined as 
platelet count ≥100x109/L, 
confirmed on at least 2 consecutive 
occasions, and absence of bleeding 
- Usage of rescue therapy 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and endpoints 
The following endpoint related to 
ITP bleeding score has been 
changed to "ITP-BAT bleeding 
events and severity by visit" 
Updated to specify the bleeding 
assessment tool to be used and 
clarification of the timepoint. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
A new secondary objective has 
been added:  
To assess the effect of 
rozanolixizumab on study 
participant perceived symptoms 
Updated to reflect that only the 
ITP-PAQ Symptoms domain score 
will be assessed for the secondary 
HRQoL objective. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
The exploratory endpoints related 
to the effect of rozanolixizumab on 
health-related quality of life 
(HRQoL) have been modified. 
The secondary objective related to 
HRQoL has moved to be an 
exploratory objective. 
The following endpoint, Response 
defined as change from Baseline at 
or above the defined threshold for 
ITP-PAQ Symptoms domain score 
has been deleted.  "Change from 
baseline endpoint" was moved to 
the exploratory PRO objective. 
Updated to provide clarity and to 
include missing details from 
previous version of the protocol. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
The secondary endpoints for 
change from Baseline in serum IgG 
subclasses and concentrations have 
been moved to exploratory 
endpoints. 
Minimum absolute IgG value and 
maximum decrease from Baseline 
in total serum IgG concentration 
over time are kept as secondary 
endpoints. Change from baseline in 
total IgG, IgG- subclasses and IgA, 
IgE and IgM over time are as other 
endpoints. 
1.1 Synopsis – Objectives 
and endpoints 
3 Objectives and endpoints 
The secondary objective related to 
reduction in use of steroid has been 
updated to include also other 
concurrent ITP medications, and 
the associated endpoint has been 
Updated to reflect that the analysis 
of changes in concomitant ITP 
therapies will not be limited only 
to corticosteroids. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 181 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
modified to replace "reduction" 
with "change”" 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and endpoints 
1.3 Schedule of Activities, 
Tables 1-1 and 1-2 
The PRO name for Physical 
Fatigue Instrument has been 
changed to the "FATIGUE-PRO 
Physical Fatigue scale.” 
Updated to indicate the correct 
name of the PRO. 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and 
Endpoints 
Secondary and exploratory: 
Additional wording has been 
provided for clarification that the 
minimum value from Baseline in 
total serum IgG concentration 
refers to the absolute IgG value. 
Updated wording to clarify that 
total serum IgG will be measured in 
addition to the IgG subclasses.  
Updated to provide further clarity. a 
 
 
 
 
 
 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and endpoints 
Two new exploratory endpoints 
related to clinical efficacy in study 
participants with first exposure to 
rozanolixizumab have been added.   
Added to specifically analyze 
efficacy in participants that 
switched from placebo to 
rozanolixizumab treatment in the 
OLE study. 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and endpoints 
Exploratory endpoints: In relation 
to Complete Response by visit 
within 1 treatment cycle without 
rescue, the endpoint has been 
amended to "Platelet count 
≥30x109/L by visit within 1 
treatment cycle without rescue 
therapy per treatment cycle"  
Updated for consistency to delete 
Complete Response endpoints and 
to focus on platelet counts 
≥30x109/L and Clinically 
Meaningful Response. 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and endpoints 
A new objective and the associated 
endpoints related to study 
participants experience with 
subcutaneous self-administration 
have been included.  
For participants that are eligible to 
use self-administration this 
questionnaire was added to capture 
their experience. 
1.1 Synopsis – Objectives 
and Endpoints 
Exploratory: 
Changed pneumococci to 
Streptococcus pneumoniae and 
meningococci to Neisseria 
meningitidis 
Corrected spelling of Haemophilus 
influenzae  
Updated for consistency with the 
table in Section 3. a 
 
 
 
Error in original protocol. a 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and endpoints  
The following exploratory endpoint 
has been removed: "Percent change 
from Baseline in vaccination titers 
Vaccination titers against tetanus 
will not be measured in this study. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 182 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
against tetanus in all study 
participants" 
1.1 Synopsis – Objectives 
and Endpoints 
3 Objectives and endpoints 
The objective on assessing clinical 
efficacy in the Exploratory 
Treatment Arm has been amended 
to remove "long-term" and include 
"intermittent.”  
The associated endpoint has been 
updated to remove “average” 
Updated to be consistent with 
study design. 
 
 
 
Updated to ensure clarity with the 
associated objective.   
1.1 Synopsis  
3 Objectives and 
Endpoints 
An additional objective and 
associated endpoint specific to the 
effect of rozanolixizumab on 
COVID-19 biomarkers the has 
been included. 
New objective and endpoint is 
needed for the collection of 
information on post-vaccination 
biomarkers for participants who 
have received the COVID-19 
vaccination.  
1.1 Synopsis, Overall 
Design 
4.1 Overall design 
Additional wording on which 
treatment will the study participants 
receive upon entering the 
Maintenance Treatment arm has 
been included. 
Updated to provide further clarity. 
1.1 Synopsis, Overall 
Design 
4.1.2 Exploratory 
treatment arm 
The following wording "a 
proportion of" and “most recently” 
have been added in reference to 
participants paused from treatment 
in the Maintenance Treatment Arm 
and randomized to the Exploratory 
Treatment Arm. 
Updated to provide further clarity. 
1.1 Synopsis – Overall 
Design 
 
Details have been added to explain 
that a
 total dose 
corresponds to an average 
 
dose in a participant weighing 
70kg. 
Updated to provide further clarity. 
1.1 Synopsis – Overall 
Design 
4.1.2 Exploratory 
treatment arm 
Additional wording added ("ie, no 
need for rescue therapy") to clarify 
criteria for study participant to 
return to Maintenance Treatment 
Arm. 
Updated to provide further clarity. a 
1.1 Synopsis, Overall 
Design 
4.1.1 Maintenance 
treatment arm 
4.1.2 Exploratory 
treatment arm 
6.4.3 Rescue therapy 
The following wording "dose 
and/or type" has been added in 
reference to study participants 
possibly being withdrawn if they 
receive rescue therapy. 
Updated to provide further clarity. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 183 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
1.1 Synopsis, Overall 
Design 
In relation to tolerability issues, the 
following term "side effects" has 
been amended to replace "side" 
with "adverse.”  
Updated to provide clarity and be 
consistent with remainder of the 
protocol. 
1.1 Synopsis, Overall 
Design 
4.1 Overall design 
9.9.2 Data monitoring 
The following sentence has been 
removed: "the IDMC will be the 
same as used for the parent studies 
TP0003 and TP0006, but data from 
each study will be presented 
separately" 
Updated to be consistent with the 
IDMC Charter. 
1.1 Synopsis, Number of 
Participants 
9.10 Determination of 
sample size 
The maximum number of 
participants completing TP0003 or 
TP0006 planned for enrollment into 
TP0004 has reduced from 210 to 
180. 
Number of participants has been 
changed based on revised sample 
size calculation method and 
assumptions in the parent studies, 
TP0003 and TP0006 leading to 
reduction of participants in 
TP0004. 
1.1 Synopsis, Treatment 
Groups and Duration  
4.1 Overall Design 
 
The following wording has been 
added "includes a 2-week 
observation period after Week 52" 
Updated to provide further clarity 
on the total duration of the 
maintenance treatment period. 
1.3 Schedule of Activities, 
Table 1-1 (Maintenance 
treatment arm) 
For participants in Japan only: 
IGRA TB test has been added to 
EOS/EW Visit.  
To confirm safety at study 
termination. a 
1.3 Schedule of Activities, 
Table 1-1 (Maintenance 
treatment arm) 
New assessments specific to 
COVID-19 vaccination samples 
and the associated footnotes have 
been added. 
New details on platelet count 
assessments have been added as a 
new footnote (f).  
Subsequent footnotes have been 
reordered. 
Included as part of the objective on 
post-vaccination biomarkers for 
participants who have received the 
COVID-19 vaccination. 
1.3 Schedule of Activities, 
Table 1-1 (Maintenance 
treatment arm) 
Tetanus titer has been removed.  
Vaccination titers against tetanus 
will not be measured in this study. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 184 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities, 
Table 1-1 (Maintenance 
treatment arm)  
Additional activities have been 
added: Pre-SIAQ and Post-SIAQ 
(Infusion version).  
The following footnotes have been 
added:  
y For study participants who self-
infuse, to be completed once, prior 
to first self-infusion 
z To be completed within 1 hour 
after the first self-infusion and the 
next consecutive self-infusion 
Added participant self-
administration questionnaire to 
capture their experience. 
 
1.3 Schedule of Activities, 
Table 1-1 (Maintenance 
treatment arm) 
Footnote d: Reworded to state that 
the data will be transferred from the 
Week 25 (Visit 28) from TP0003 or 
TP0006.  
Footnote f now g: Updated to 
clarify participants will need a new 
platelet count. 
Footnote g, now h: The following 
wording has been removed: "1 to 2 
minutes" 
Footnote i, now j: Updated to 
include "and prior to discharge 
from clinic/departure of home 
healthcare professional" 
Footnote j, now k: Additional 
laboratory samples (total 
cholesterol, LDL, HDL, and 
triglycerides) will be collected at 
Week 13 and Week 25 only. 
For participants in Japan only: 
Footnote o: Wording pertaining to 
T-SPOT.TB test may be performed 
as an alternative. 
Footnote t, now aa: updated to 
include cross-reference to the 
management of headaches 
Updated to correct timepoint when 
data will be taken from the parent 
studies. a  
 
Updated to provide further clarity.  
 
 
Updated to provide further clarity. 
 
 
Updated to provide further clarity. 
 
 
 
Lipid profile will be assessed 
during the study. 
 
 
 
To add the T-SPOT test as a 
recommended IGRA test in 
addition to the QuantiFERON test. 
a 
Updated to provide further clarity. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 185 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities, 
Table 1-1 (Maintenance 
treatment arm) 
Footnote n, now q: Corrected 
footnote to state that observation 
time of 1 hour is required for the 
next 2 IMP infusions after Visits 1 
and 5, and additional wording was 
added to specify that the infusions 
need to be well tolerated by the 
study participant to reduce 
observation time to 15 minutes for 
the remaining visits until Week 25. 
Additional wording specific to the 
criteria for no post-infusion 
observation periods after Week 25 
has been included.    
Updated to remain consistent with 
the observation requirements and 
the overall study design 
(Section 4.1). 
1.3 Schedule of Activities, 
Table 1-2 (Exploratory 
treatment arm) 
Table header was updated to 
include the correct visit numbers to 
correlate with the weeks. 
Correction of error in footnote b to 
correlate with updates to header 
correction. 
Error in original protocol. a 
1.3 Schedule of Activities, 
Table 1-2 (Exploratory 
treatment arm) 
Added a scheduled visit for platelet 
count (local laboratory) at Week 
35. 
For laboratory parameters, "local" 
was replaced with "central.”  
Error in original protocol. a 
1.3 Schedule of Activities, 
Table 1-2 (Exploratory 
treatment arm) 
Collection of anti-rozanolixizumab 
antibodies (ADA) samples was 
removed at Weeks 29, 37 and 41. 
Additional ADA samples will 
already be collected at scheduled 
visits for plasma concentration of 
rozanolixizumab samples. 
1.3 Schedule of Activities, 
Table 1-2 (Exploratory 
treatment arm) 
Correction of typographical error in 
footnote b, ≥50x109/L and added 
"to ≤200x109/L.” 
Footnote e: Replaced "final" with 
"each" dose of IMP. 
Footnote f now g: The following 
wording has been removed: "1 to 2 
minutes" 
Footnote f: A new footnote specific 
to withdrawal criteria has been 
added.  
Footnote g now h: The following 
wording was added: "and prior to 
discharge from clinic/departure of 
home healthcare professional.” 
Updated for consistency with 
Overall Design in Section 1.1. a 
 
Updated to correct an error. a 
 
Updated to provide further clarity. 
 
 
Updated for consistency with the 
protocol. 
 
Updated to remain consistent with 
the post-infusion observation 
requirements and the overall study 
design (Section 4.1). 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 186 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
Footnote j: A new footnote specific 
to instructions if study participant 
requires IMP dosing. 
Footnote k and m: New footnotes 
specific to platelet count at the time 
of the visit. 
Footnote l: Durations of the post-
dosing observation period have 
been updated. 
 
 
 
Updated to be consistent across the 
Phase 3 rozanolixizumab clinical 
program for an OLE study. 
1.3 Schedule of Activities, 
Table 1-2 (Exploratory 
treatment arm) 
Footnotes l and k, now o and p, 
respectively: postdose has been 
replaced with postevent. 
Updated to correct an error. 
1.3 Schedule of Activities, 
Table 1-2 (Exploratory 
treatment arm) 
Added the following footnote (l) to 
the administration of IMP: 
"Study participants are to stay at 
the study site for 4 hours after the 
first IMP infusion. The observation 
will be 2 hours after the second 
dose, and 1 hour for the remaining 
dosing visits" 
Reordering of footnotes was 
required. 
To provide details of post-infusion 
observation time for consistency 
with Section 4.1. a 
1.3 Schedule of Activities, 
Table 1-2 (Exploratory 
treatment arm) 
Additional activities have been 
added: Pre-SIAQ and Post-SIAQ 
(Infusion version).  
The following footnotes have been 
added:  
q For study participants who self-
infuse, to be completed once, prior 
to first self-infusion 
r To be completed within 1 hour 
after the first and second 
consecutive self-infusion  
Added participant self-
administration questionnaire to 
capture their experience. 
 
2.2 Background 
Additional studies have been 
included to this section.  
"Ongoing" has been removed from 
Study UP0060. 
Updated to be consistent. 
 
The study is complete. 
2.3 Benefit/risk assessment Text on potential risks associated 
with administration of 
rozanolixizumab has been updated. 
New wording on COVID-19 
vaccines has been added. 
Updated to be consistent across the 
Phase 3 rozanolixizumab clinical 
program. 
4.1 Overall Design 
Wording referring to the medical 
monitor decision on whether 
This deletion has been made due to 
the medical monitor’s role in 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 187 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
participants can be enrolled has 
been removed. 
determining a study participant’s 
eligibility in TP0004 not being 
considered valid. 
4.1 Overall Design 
 
Several minor edits were made for 
consistency with other sections in 
the protocol. 
Wording referring to the medical 
monitor’s decision on whether 
participants can be enrolled and 
participants receiving a vaccination 
titer for tetanus have been removed. 
Wording pertaining to the next step 
for to study participants previously 
randomized to placebo has been 
added.  
Post-infusion observation timings 
and instructions for the 
maintenance and exploratory 
treatment arms have been updated. 
 
 
 
Update due to the medical 
monitor’s role in determining a 
study participant’s eligibility not 
considered valid. 
 
Updated for consistency with 
footnote m in Table 1-1. a  
 
 
Updated to be consistent across the 
Phase 3 rozanolixizumab clinical 
program for an OLE study. 
 
4.1 Overall Design 
A new sentence has been added to 
explain that contingency measures 
during a pandemic and other 
exceptional circumstances have 
been included. 
Contingency measures have been 
implemented to ensure study 
participant safety in response to the 
COVID-19 pandemic causing the 
inability for sites to adhere to 
protocol visits and assessments. 
4.1 Overall Design,  
Table 4-1, footer 
4.1.1 Maintenance 
treatment arm 
 
Table header updated to replace 
"starting" with "Exploratory arm.”  
Additional wording has been 
provided for clarification on the 
action taken for study participants 
with on the lowest dose level with a 
platelet count between >200×109/L 
and <400 ×109/L. 
This update has been included to 
consider that no dose reduction to 
less than 
(fixed dose) is set. 
a   
4.1.2 Exploratory 
treatment arm 
This section has been updated to 
include: 
"or the investigator deems not to be 
suitable" in reference to entry to the 
Exploratory Arm, and additional 
information on based on rescue 
therapy.   
Week 23 and Visit 18 have been 
included. 
Updated to provide further clarity. 
4.3 Justification for dose 
The following wording, "then 
quickly reset platelets to the 
desired levels as faster onset of 
Updated to provide further clarity. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 188 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
response" has been replaced with 
"is anticipated to increase platelet 
count to ≥50x10 within a week as.” 
In addition the following sentence 
has been included: "However, if 
any safety concerns are identified 
in the parent studies, TP0003 and 
TP0006, corresponding changes 
will be made in the TP0004 dosing 
scheme" 
5.1 Inclusion criteria 
Criterion #1, now 1a,  has been 
modified to remove "and sponsor" 
from the criterion. 
This deletion has been made as the 
sponsor cannot be involved in 
deciding a study participant’s 
eligibility in TP0004. 
5.2 Exclusion criteria 
Criterion #2 has been removed.  
This deletion has been made due to 
the medical monitor’s role in 
determining a study participant’s 
eligibility in TP0004 not being 
considered valid. 
5.2 Exclusion criteria 
Criterion #6, now 6a: The 
following text has been removed:  
"In case of a clinically relevant 
increase, inclusion of the study 
participant must be discussed with 
the Medical Monitor or designee." 
Criterion #7, now 7a: the criterion 
has been updated to include a 
timepoint.  
This deletion has been made due to 
the medical monitor’s role in 
determining a study participant’s 
eligibility in TP0004 not being 
considered valid. 
 
Updated to provide clarity on 
bilirubin values used at the time of 
eligibility. 
5.2 Exclusion criteria 
Criterion #9, now 9a: the criterion 
has been updated to include 
"major" in reference to planned 
surgery and the duration of 14 
months has been replaced by study 
period. 
Criterion #10, now 10a: the 
criterion has been updated to 
include "at the last available 
assessment in TP0003/TP0006" 
Updated to remain consistent with 
the parent studies. 
 
 
 
 
Updated to provide clarity on lab 
values used at the time of 
eligibility. 
5.2 Exclusion criteria 
Criterion #11, now 11a: The 
following text has been removed:  
"and any queries regarding 
continuation of the study 
participants will have to be 
addressed with the medical 
monitor." 
 
 
This deletion has been made due to  
the medical monitor’s role in 
determining a study participant’s 
eligibility in TP0004. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 189 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
"The ECG contains findings that 
may represent a significant 
abnormality" 
 
This is not applicable and remains 
consistent with the parent studies. 
5.2 Exclusion criteria 
New criteria on renal impairment 
and QT interval (#12 and #13), and  
live vaccination prior to, during or 
after the study (#14) have been 
included. 
Updated to be consistent across the 
Phase 3 rozanolixizumab clinical 
program for an OLE study. 
5.2 Exclusion criteria 
New wording on France-specific 
requirements has been included. 
Updated to align with French 
Ethics Committee query on 
exclusion criteria pertaining the 
French Public Health Code and 
participant’s age at Baseline. b 
6 Study treatments, Table 
6-1 
The description for unit doses, and 
packaging and labelling for the 
maintenance and exploratory arms 
has been updated to delete: "6mL" 
and "no less than 4.0mL extractable 
volume of.” 
Update to reflect the foreseeable 
changes in vial size. 
6.2 Measures to minimize 
bias: randomization and 
blinding 
The following text has been added 
to: The randomization number will 
be incorporated from the IRT into 
the eCRF by automatic data 
transfer. 
Updated to provide further clarity. 
6.4.1 Permitted 
concomitant treatments 
(medications and 
therapies) 
Added "above Baseline (Visit 1, 
TP0004)" to the following 
sentence: 
Any increase in concomitant ITP 
medication above Baseline (Visit 1, 
TP0004) is regarded as rescue 
therapy. 
New text has been added in specific 
to the requirement to collect 
information on COVID-19 
vaccinations in the eCRF. 
Updated to provide further clarity. a 
 
 
 
 
 
 
Information on COVID-19 
vaccination will be required. 
6.4.1 Permitted 
concomitant treatments 
(medications and 
therapies), Table 6-2 
"Prednisolone" changed to 
"prednisone.” Clarification of dose 
for oral corticosteroid.  
Dosing for cyclosporin was 
updated to include modified and 
unmodified doses.  
Addition of Eltrombopag, 
Avatrombopag and Fostamatinib at 
any dose as permitted concomitant 
treatments.  
Updated to clarify permitted 
concomitant medications. a 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 190 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
6.4.1 Permitted 
concomitant treatments 
(medications and 
therapies) 
The following wording has been 
removed: "When applicable, the 
study participant must be on a 
stable dose of cannabidiols and/or 
medicinal marijuana for Visit 1 
(Day 1)" 
No fixed period of stable dose 
before Baseline for this study. 
6.4.2 Prohibited 
concomitant treatments 
(medications and 
therapies) 
Removal of Dexamethasone, and 
addition of Romiplostim as a 
prohibited treatment, and 
clarification for corticosteroids. 
Updated to clarify prohibited 
concomitant medications. a 
6.4.2 Prohibited 
concomitant treatments 
(medications and 
therapies) 
Removal of TPO-RAs as prohibited 
treatment. 
Updated to clarify prohibited 
concomitant medications. a 
6.4.2 Prohibited 
concomitant treatments 
(medications and 
therapies) 
New wording has been included 
explaining the use of prohibited 
concomitant treatment will lead to 
permanent discontinuation from 
IMP.  
Updated for clarification of 
prohibited medications. 
6.4.3 Rescue therapy  
 
Additional wording to following 
sentence has been included: Any 
increase in concomitant ITP 
medication "above Baseline (Visit 
1, TP0004)" is regarded as rescue 
therapy. 
Additional wording in relation to 
systemically administered 
corticosteroids has been included: 
"ie, pulse, oral and iv steroids" as 
well as "are also considered rescue 
therapy.” 
Updated to clarify rescue therapy. a 
6.4.3.1 Rescue therapy not 
leading to discontinuation 
Platelet count measurement was 
corrected from ≥ to <. 
Additional wording in reference to 
the measures of starting 
rozanolixizumab treatment and 
defining a high dose of 
corticosteroids have been included. 
The increase amount for permitted 
concomitant immunosuppressants 
has been corrected from 5% to 
25%. 
Updated to clarify rescue therapy. a 
6.4.3.2 Rescue therapy 
leading to discontinuation 
TPO-RAs was removed, and 
"initiation" was added in reference 
to additional ITP therapy. 
Updated to clarify rescue therapy. a 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 191 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
6.5 Dose modification 
The following recommended dose 
modifications were updated/added: 
- Moderate to severe toxicities 
(Grade 2 and above, as defined by 
Common Terminology Criteria for 
Adverse Events version 5.0) for 
which rozanolixizumab cannot be 
excluded as a cause. 
Wording on potential changes in 
the dosing scheme based on safety 
concerns identified in the parent 
studies has been included. 
Updated to remain consistent 
across the rozanolixizumab 
program. 
6.6 Home visits and 
home/self-dosing 
Sub-heading was changed to 
"Home visits and self-
administration" and additional 
wording has been included 
pertaining to self-administration 
requirements as well as the timing 
of administration.  
The conditions for conducting 
home visits/self-administration 
have been amended.  
Updated to provide clarity and 
remain consistent with study 
design. 
6.7 Treatment after the 
end of the study 
New wording on access to a 
Managed Access Program has been 
included. 
Updated to provide clarity. 
7.1.3 Discontinuation of 
IMP due to other adverse 
events or medical 
condition 
New section was included to detail 
criterion and procedure for any 
discontinuation of IMP due to other 
AEs or medical condition  
Updated to remain consistent with 
Phase 3 ITP clinical program. 
7.1.4 Temporary IMP 
discontinuation 
Section was previously 7.1.3. All 
criteria were amended. 
Additional parameters to be 
unblinded following the second 
dose have been included. 
In addition, details have been added 
for participants that have suspected 
or confirmed COVID-19. 
Updated to provide clarity and 
remain consistent with study 
design and remainder of protocol. 
 
 
Criteria #2 #3, and #5 were 
previously updated in protocol 
amendment 0.1. 
7.1.4 Temporary IMP 
discontinuation 
Clarification that treatment will be 
temporarily discontinued for any 
participant with IgG levels below 
1g/L on any occasion. 
Clarification of formal and 
informal unblinding and the role of 
the unblinded Medical Monitor. 
Updated to clarify when treatment 
discontinuation is required, 
unblinding, and the role of the 
unblinded Medical Monitor. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 192 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
7.2 Participant 
discontinuation/withdrawal 
from the study 
This section was reordered and 
updated to remove repetitive 
information highlighted in Section 
7.1.4. 
Updated to provide clarity and be 
consistent with study design and 
remainder of protocol.  
Correction of error and for 
consistency with parent studies. 
7.3 Lost to follow up 
This section was updated to remove 
repetitive information in relation to 
evaluations and observations. 
To avoid repetition. 
 
8 Study Assessments and 
Procedures 
A new paragraph describing 
contingency measures during a 
pandemic and other exceptional 
circumstances has been included. 
Contingency measures have been 
implemented to ensure study 
participant safety in response to the 
COVID-19 pandemic causing the 
inability for sites to adhere to 
protocol visits and assessments. 
8.1.3 Electrocardiograms 
The following wording has been 
removed: "1 to 2 minutes" 
Updated to provide further clarity. 
8.1.5.1 Tuberculosis 
assessment 
TB signs and symptoms 
questionnaire: "and must receive 
prophylactic LTB infection 
therapy" and "must be withdrawn" 
have been deleted. 
To provide further clarity. 
8.1.5.1 Tuberculosis 
assessment  
If reference to determining if a 
participant has LTBI, wording on 
receiving prophylactic LTBI 
therapy has been removed.  
To provide further clarity on the 
assessment of TB. 
8.1.5.1 Tuberculosis 
assessment  
TB assessment by IGRA:  
Wording on requirements for 
positive or indeterminate IGRA 
tests result has been updated.  
To provide further clarity on the 
assessment of TB.  
8.1.5.2 Tuberculosis 
management 
New text on the recommendation 
for withdrawn participants with 
active TB or NTMB infection to 
enter the SFU Period and attend 
Visit 35 has been included. 
To provide further clarity in line 
with the protocol. 
8.1.6 Splenectomized 
study participants  
The following wording "for the 
whole duration of the study" was 
added in reference to the 
requirement of carrying a 
splenectomy card. 
New wording on the requirement 
for splenectomized study 
participants to have documented 
evidence of vaccinations from 
parent studies has been included.  
Updated to provide further clarity. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 193 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
8.2.5 Pregnancy  
Instructional wording for 
participants with confirmed 
pregnancy has been reordered. 
Updated to provide further clarity. 
8.2.8 Adverse events of 
special monitoring 
A sentence has been added to 
clarify that an AESM is not 
necessarily a serious adverse event 
unless one of the seriousness 
criteria defined in the Appendix 3 
is met. 
Updated to provide further clarity. 
8.2.9 COVID-19 
vaccination  
New subsection has been included 
to ensure AEs considered related to 
COVID-19 are captured.  
Implemented to ensure study 
participant safety in response to the 
COVID-19 pandemic. 
8.5.1 Platelet counts 
Wording on platelet count-related 
endpoints has been updated. 
Updated to be consistent with the 
protocol and reflect the changes 
made for the endpoints. 
8.5.3 Patient Reported 
Outcomes 
The list of PROs has been updated 
to replace the "Physical Fatigue 
Instrument" by the "FATIGUE-
PRO Physical Fatigue scale" and 
"EQ-5D" to "EQ-5D-5L.” 
To indicate the correct name of the 
PRO. 
8.5.3.1 ITP-PAQ 
Additional text on the choice of 
selection as a secondary endpoint 
has been included. 
Updated to be consistent with the 
protocol and reflect the changes 
made for the endpoints 
8.5.3.3 FATIGUE-PRO 
Physical Fatigue Scale 
The "Physical Fatigue Instrument" 
has been replaced by the 
"FATIGUE-PRO Physical Fatigue 
scale.” 
Additional changes were made to 
the description of the FATIGUE-
PRO Physical Fatigue scale. 
To indicate the correct name of the 
PRO. 
8.5.3.7 SIAQ (Infusion 
version) 
An new subsection for an 
additional patient reported outcome 
tool "SIAQ (Infusion version)" has 
been included.  
Added to capture the participants 
experience of  IMP self-
administration. 
8.8 Exploratory 
biomarkers 
Duplicate details pertaining to 
sample collection, processing, 
storage, labeling, and shipping have 
been deleted. 
To remove repetition. 
8.9 Immunological 
biomarkers 
New text has been added in specific 
to blood sampling for the 
measurement of COVID-19 
antibodies. 
COVID-19 antibodies will be 
collected in this study for all 
participants who have received a 
COVID-19 vaccine. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 194 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
9.1 Definition of analysis 
sets 
Definition of analysis sets: The 
Safety Set and Exploratory 
Treatment Arm Set have been 
updated. 
The Pharmacokinetic Per-Protocol 
Set was updated to remove "and no 
important protocol deviations 
affecting the PK variable, as 
confirmed during a pre-analysis 
review of the data prior to database 
lock" 
Updated for further clarity. 
9.2 General statistical 
considerations 
A sentence was added to explain 
that data handling conventions for 
data affected by COVID-19 will be 
detailed fully in the SAP. 
Added to confirm that the potential 
effects of COVID-19 on the data 
analysis will be addressed. 
9.4 Planned safety 
analyses 
This section has been amended to 
remove "8 weeks after the end of 
the Treatment Period" and "in 
study participants who discontinue 
the study or IMP" in reference to 
follow up of exposure to treatment, 
and to include details specific to 
safety analyses.  
Updated for further clarity. 
9.5 Planned 
efficacy/outcome analyses 
Changed "feeder" to "parent" when 
describing TP0003/TP0006 studies. 
New wording has been included to 
provide details and clarification on   
efficacy/outcome analyses. 
Updated for consistency in 
terminology within protocol. a 
Clarification has been provided on 
how assessments will be conducted 
for key efficacy endpoints in study 
TP0004 for patients who received 
rescue medication, especially, near 
the end of the parent studies. a 
9.6.1 Analysis of 
pharmacodynamics 
endpoints 
Total IgG was added to the list of 
included analyses to be considered. 
Updated for to be consistent with 
endpoints listed in the protocol. 
9.6.2 Analysis of 
pharmacokinetics 
endpoints 
The following wording has been 
removed: "stratified by dose 
regimen" and "stratified by 
treatment arm.” 
Updated for further clarity. 
9.7 Handling of protocol 
deviations 
The following wording has been 
removed: "exclusion of participants 
from analysis populations" 
Updated to be consistent with the 
data cleaning plan. 
9.9.1 Interim analysis 
New wording on data cutoffs has 
been included. 
Updated for further clarity. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 195 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
9.9.2 Data monitoring 
Wording on reviewing safety data 
at periodic data reviews has been 
removed. 
Updated to be consistent with the 
IDMC Charter. 
10.1.3 Informed consent 
process 
New wording has been included in 
reference to Japan-specific 
requirements.  
In accordance with local 
requirements in Japan. a  
10.1.5 Committees 
structure 
The following new wording was 
added: "The IDMC will have the 
possibility to unblind the data" 
To be consistent with the rest of 
the protocol. 
 
10.1.6 Data quality 
assurance 
Additional wording on quality 
tolerance limits has been included. 
Correcting an error as this 
information was previously 
omitted from the protocol. 
10.2, Appendix 2: Clinical 
laboratory tests 
Table 10-1: Footnotes have been 
reordered due to addition of 
footnotes b, c, d, e, and g to clarify 
collection timepoints and 
requirements for protocol-required 
safety laboratory assessments. 
Updated for clarification. a 
10.2, Appendix 2: Clinical 
laboratory tests 
Total cholesterol, LDL, HDL and 
triglycerides have been added. 
Table 10-1: Updated wording for 
immunoglobulin footnote g to 
clarify when results are unblinded. 
Glycosylated hemoglobin (HbA1c) 
has been removed.  
Lipid profile will be assessed in 
the study. 
To ensure blinding is consistent 
throughout all studies in the 
rozanolixizumab clinical program.a 
Removed wording to maintain 
consistency within the protocol. 
10.3, Appendix 3: Adverse 
events – definitions and 
procedures for recording, 
evaluating, follow-up, and 
reporting 
Additional wording for follow-up 
of AEs and SAEs has been 
included. 
Updated to ensure guidance for AE 
follow up is consistent with the 
Phase 3 rozanolixizumab clinical 
program. 
10.4, Appendix 4: 
Contraceptive guidance 
and collection of 
pregnancy information, 
Table 10-2 
Wording for vasectomized partner 
has been updated.  
Footnote c has been removed.  
To remain consistent with the 
Phase 3 rozanolixizumab clinical 
program. 
10.5 Appendix 5: Liver 
safety – suggested actions 
and follow-up assessments 
Table 10-3 
 
Clarification of language related to 
acetaminophen contribution to liver 
injury and removal of wording 
pertaining to requirements in 
China. 
The following reference was 
removed: 
James LP, Letzig L, Simpson PM, 
Capparelli E, Roberts DW, Hinson 
To remove wording that was left 
from a previous version and to 
clarify the language for suggested 
actions. a 
Updated to maintain consistency 
within the protocol. a 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 196 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
JA, et al. Pharmacokinetics of 
acetaminophen-protein adducts in 
adults with acetaminophen 
overdose and acute liver failure. 
Drug Metab Dispos. 2009; 
37:1779-84 
10.7, Appendix 7: 
Country-specific 
requirements 
New wording on specific 
requirements for France have been 
included. 
Updated in accordance with 
include local requirements. b 
10.7, Appendix 7: 
Country-specific 
requirements 
New wording on specific 
requirements for Japan have been 
added, including section-specific 
updates and new wording on 
specific requirements for AEs, 
ADEs, SAEs, SADEs, and Device 
Deficiencies associated with an 
investigational/medical device, 
have been added. 
In accordance with local 
requirements and actual condition 
of medical care, as well as sponsor 
requirements and procedures for 
any study using a medical or 
investigational device. a  
10.9 Appendix 9: 
Abbreviations and 
trademarks 
Additional abbreviations and 
definitions were added: ADE, PEX 
and SADE. 
Updated to reflect the parameters 
measured in the study. a 
10.11, Appendix 11: 
ITP-PAQ 
Removed an additional version of 
the ITP-PAQ questionnaire. 
Updated to remove an additional 
version of the questionnaire that 
was accidentally included. a 
10.13, Appendix 13: 
FATIGUE-PRO Physical 
Fatigue scale 
The "Physical Fatigue Instrument" 
has been replaced by the 
"FATIGUE-PRO Physical Fatigue 
scale.” 
Updated name for PRO.  
10.20, Appendix 20: 
Management of headaches 
Text added to clarify that the 
Headache Questionnaire should be 
completed by an HCP via an 
interview with the study 
participant, and treatment may be 
temporarily put on hold if a study 
participant experiences an AE of a 
severe headache that is considered 
related to the IMP in the opinion of 
the investigator, and is not resolved 
prior to the next scheduled IMP. 
To add further clarity. 
10.21, Appendix 21: 
Management of diarrhea 
Definition of severe (grade 3) 
diarrhea has been updated. 
Modification of language regarding 
stool sample collection for 
participants reporting severe 
diarrhea. 
To add further clarity.  
 
To clarify when stool samples may 
be collected. a 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 197 of 199 
Section # and Name 
Description of Change 
Brief Rationale 
10.22 Appendix 22: 
Management of Infections 
and 
Hypogammaglobulinemia 
Modification of language regarding 
the Medical Monitor to clarify they 
may be unblinded. a 
Additionally, the following has 
been included in reference to study 
sites and study team: “remain 
blinded to IgG levels until after the 
first two dose of IMP.”  
In the event of a nonserious 
infection, the unblinded or blinded 
(after the second dose of IMP) 
medical monitor may be consulted.  
The following sentence was 
modified to include "must": 
Treatment must be temporarily 
discontinued for a study participant 
who develops an event of 
hypogammaglobulinemia with a 
serum total IgG of <1g/L 
irrespective of infection. 
Updated to be consistent across the 
Phase 3 rozanolixizumab clinical 
program. 
10.23 Appendix 23: 
Management of infusion 
reactions or 
hypersensitivity reactions 
Modification of language regarding 
potential infusion related reactions. 
Updated to provide further clarity. 
10.24 Appendix 24: SIAQ 
(infusion version) 
A new appendix to include 
examples of the SIAQ 
questionnaires has been added.   
Included for additional clarity and 
details on the questionnaire.  
11 References  
A new reference specific to SIAQ 
has been included.  
General update. 
a This update was previously incorporated to local protocol amendment 0.1 (Japan) 
b This update was previously incorporated to local protocol amendment 0.2 (France)  
 
11 
REFERENCES 
Anderson CL, Chaudhury C, Kim J, Bronson CL, Wani MA, Mohanty S. Perspective - FcRn 
transports albumin: relevance to immunology and medicine. Trends Immunol. 2006;27(7):343-8. 
CPMP/ICH/135/95 Note for guidance on Good Clinical Practice (EMEA) Jul 2002. 
Keininger D and Coteur F. Assessment of self-injection experience in patients with rheumatoid 
arthritis: psychometric validation of the Self-Injection Assessment Questionnaire (SIAQ). Health 
Qual Life Outcomes. 2011;9:2. 
Mathias SD, Bussel JB, George JN, McMillan R, Okano GJ, Nichol JL. A disease-specific 
measure of health-related quality of life for use in adults with immune thrombocytopenic 
purpura: its development and validation. Health Qual Life Outcomes. 2007;5:11. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 198 of 199 
Mathias SD, Gao SK, Rutstein M, Snyder CF, Wu AW, Cella D. Evaluating clinically 
meaningful change on the ITP-PAQ: preliminary estimates of minimal important differences. 
Curr Med Res Opin. 2009;25(2):375-83. 
Neunert C, Lim W, Crowther M, et al. The American Society of Hematology 2011 evidence-
based practice guideline for immune thrombocytopenia. Blood. 2011;117:4190-207. 
Rodeghiero F, Michel M, Gernsheimer T, et al. Standardization of bleeding assessment in 
immune thrombocytopenia: report from the International Working Group. Blood. 
2013;121(14):2596-606. 
Roopenian DC, Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol. 
2007;7(9):715-25. 
Sampson HA, Muñoz-Furlong A, Campbell RL, 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. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

UCB 
 
04 Apr 2022 
Clinical Study Protocol Amendment 4 
Rozanolixizumab 
TP0004 
 
 
 
Confidential 
Page 199 of 199 
SPONSOR DECLARATION 
I confirm that I have carefully read and understand this protocol and agree to conduct this 
clinical study as outlined in this protocol and according to current Good Clinical Practice. 
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.

Approval Signatures
Name:
tp0004-protocol-amendment-4
Version:
1. 0
Document Number:
CLIN-000186061
Title:
tp0004-protocol-amendment-4
Approved Date:
04 Apr 2022
Document Approvals
Approval
Verdict: Approved
Name: 
Capacity: Clinical
Date of Signature: 04-Apr-2022 13:24:45 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Subject Matter Expert
Date of Signature: 04-Apr-2022 13:40:42 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Clinical
Date of Signature: 04-Apr-2022 14:05:09 GMT+0000
PUBLIC COPY 
This document cannot be used to support any marketing authorization 
application and any extensions or variations thereof.