Clinical trial NCT04051944
Conditions: Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Keywords: Chronic inflammatory demyelinating polyradiculoneuropathy CIDP UCB7665 rozanolixizumab
Sponsor: UCB Biopharma SRL
Start date: 2019-08-21; Completion date: 2021-11-10
Enrollment: 21 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
The purpose of the study is to assess long-term safety and tolerability of weekly doses of rozanolixizumab in subjects with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

Detailed description:

Arms: arm 1: Subjects in this arm will receive predefined subcutaneous doses of rozanolixizumab at a specified frequency.
Interventions: intervention 1: Rozanolixizumab
Intervention descriptions: intervention 1: Subjects will receive rozanolixizumab in a specified sequence during the treatment period.

Protocol documents:
Prot_000.pdf: 
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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
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PROTOCOL CIDP04 AMENDMENT 3  
AN OPEN-LABEL EXTENSION STUDY TO INVESTIGATE THE 
LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF 
ROZANOLIXIZUMAB IN SUBJECTS WITH CHRONIC 
INFLAMMATORY DEMYELINATING 
POLYRADICULONEUROPATHY (CIDP) 
PHASE 2A 
 
EudraCT Number: 2018-004392-12 
IND Number: 134848 
 
Sponsor: 
UCB Biopharma SRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Protocol/Amendment number 
Date 
Type of 
amendment 
Final Protocol 
14 Dec 2018 
Not applicable 
Protocol Amendment 1 France 
23 Apr 2019 
Non-substantial 
Protocol Amendment 2 
12 Jul 2019 
Substantial 
Protocol Amendment 3 
16 Mar 2020 
Substantial 
 
Confidential Material 
 
Confidential 
This document is the property of UCB and may not – in full or in part – be passed on, 
reproduced, published, or otherwise used without the express permission of UCB. 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
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STUDY CONTACT INFORMATION 
Sponsor 
UCB Biopharma SRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Coordinating Investigator 
Name: 
 
Affiliation: 
Neuromuscular Diseases Unit – Neuromuscular Lab 
Address: 
Neurology Department 
Hospital de la Santa Creu i Sant Pau 
Pavillion 17, 1st floor 
Sant Antoni Maria Claret 167 
08025 Barcelona 
SPAIN 
Phone: 
 
Sponsor Study Physician 
Name: 
 
Address: 
UCB  
Chemin du Foriest 
1420 Braine-l’Alleud 
BELGIUM 
Phone: 
 
Clinical Project Manager 
Name: 
 
Address: 
UCB BIOPHARMA SRL 
Allée de la Recherche, 60 
B – 1070 Brussels 
BELGIUM 
Phone: 
 


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Rozanolixizumab 
CIDP04 
 
 
 
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Clinical Trial Biostatistician 
Name: 
 
Address: 
UCB BIOSCIENCES GmbH 
Alfred-Nobel-Strasse 10 
40789 Monheim 
GERMANY 
Phone: 
 
Clinical Monitoring Contract Research Organization 
Name: 
PAREXEL International (IRL) Limited 
Address: 
70 Sir John Rogerson’s Quay 
Dublin 2 
IRELAND 
Phone: 
+353 1 473 9500 
Fax: 
+353 1 477 3390 


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Rozanolixizumab 
CIDP04 
 
 
 
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SERIOUS ADVERSE EVENT REPORTING 
Serious adverse event reporting (24h) 
Fax 
Europe and Rest of the World: +32 2 386 6561 
USA and Canada: 011 32 2 386 65 61 
Email 
Global: safetyreportingUCB7665@ucb.com 
 


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Rozanolixizumab 
CIDP04 
 
 
 
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TABLE OF CONTENTS 
LIST OF ABBREVIATIONS .................................................................................................. 11 
1 
SUMMARY ..................................................................................................................... 14 
2 
INTRODUCTION ........................................................................................................... 16 
2.1.1 
Rationale for this study ........................................................................................ 17 
3 
STUDY OBJECTIVES .................................................................................................... 17 
3.1 
Primary objective ......................................................................................................... 17 
3.2 
Secondary objective ..................................................................................................... 17 
3.3 
Exploratory objectives ................................................................................................. 17 
4 
STUDY VARIABLES ..................................................................................................... 18 
4.1 
Safety variables ............................................................................................................ 18 
4.1.1 
Primary safety variables ...................................................................................... 18 
4.1.2 
Other safety variables .......................................................................................... 18 
4.2 
Other variables ............................................................................................................. 19 
4.2.1 
Efficacy variables ................................................................................................ 19 
4.2.2 
Patient-reported outcome variables ..................................................................... 20 
4.2.3 
Pharmacokinetic variable ..................................................................................... 20 
4.2.4 
Pharmacodynamic variables ................................................................................ 20 
4.2.5 
Immunological variables ..................................................................................... 20 
4.2.6 
Exploratory biomarkers ....................................................................................... 21 
5 
STUDY DESIGN ............................................................................................................. 21 
5.1 
Study description ......................................................................................................... 21 
5.1.1 
Study duration per study participant .................................................................... 22 
5.1.2 
Planned number of study participants and sites ................................................... 22 
5.1.3 
Anticipated regions and countries ....................................................................... 23 
5.2 
Schedule of study assessments..................................................................................... 23 
5.2.1 
Additional study assessments .............................................................................. 33 
5.3 
Rationale for study design and selection of dose ......................................................... 35 
6 
SELECTION AND WITHDRAWAL OF STUDY PARTICIPANTS ........................... 35 
6.1 
Inclusion criteria .......................................................................................................... 35 
6.2 
Exclusion criteria ......................................................................................................... 37 
6.2.1 
Additional exclusion criteria for study participants with a gap period between 
the parent study and entry in CIDP04 .................................................................. 38 
6.3 
Rescreening of study participants (only for study participants who had a Screening 
Visit) ............................................................................................................................ 40 
6.4 
Withdrawal criteria ...................................................................................................... 41 
6.4.1 
Temporary discontinuation of IMP ..................................................................... 42 
6.4.1.1 
Serious infection ........................................................................................ 42 


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6.4.1.2 
Hypogammaglobulinemia and non-serious persisting or recurrent 
infection ..................................................................................................... 43 
6.4.1.3 
Hypogammaglobulinemia irrespective of infection .................................. 43 
6.4.2 
Potential drug-induced liver injury IMP discontinuation criteria ........................ 43 
7 
STUDY TREATMENT ................................................................................................... 44 
7.1 
Description of investigational medicinal product ........................................................ 44 
7.2 
Treatment to be administered....................................................................................... 44 
7.3 
Packaging ..................................................................................................................... 46 
7.4 
Labeling ....................................................................................................................... 46 
7.5 
Handling and storage requirements ............................................................................. 46 
7.6 
Drug accountability ...................................................................................................... 46 
7.7 
Procedures for monitoring study participant compliance ............................................ 47 
7.8 
Concomitant medications/treatments ........................................................................... 47 
7.8.1 
Permitted concomitant treatments (medications and therapies) .......................... 47 
7.8.2 
Prohibited concomitant treatments (medications and therapies) ......................... 48 
7.8.3 
Rescue medication ............................................................................................... 49 
7.9 
Blinding........................................................................................................................ 49 
7.10 
Randomization and numbering of study participants .................................................. 49 
8 
STUDY PROCEDURES BY VISIT ............................................................................... 49 
8.1 
Screening, Visit 1 (Weeks -5 to -2) ............................................................................. 50 
8.2 
Treatment Period Part 1, Visits 2 to 25 (Weeks 1 to 24) ............................................. 51 
8.2.1 
Visit 2, Baseline (Week 1) ................................................................................... 52 
8.2.2 
Visit 3 (Week 2) ................................................................................................... 53 
8.2.3 
Visit 4 (Week 3) ................................................................................................... 54 
8.2.4 
Visit 5 (Week 4) ................................................................................................... 55 
8.2.5 
Visit 6 (Week 5) ................................................................................................... 56 
8.2.6 
Visit 7 (Week 6), Home Visit .............................................................................. 57 
8.2.7 
Visit 8 (Week 7) ................................................................................................... 57 
8.2.8 
Visits 9 (Week 8), Home Visit ............................................................................ 58 
8.2.9 
Visits 10, 14, 18, and 22 (Weeks 9,13, 17, and 21) ............................................. 58 
8.2.10 
Home Visits 11 to 13, 15 to 17, 19 to 21, and 23 to 25 (Weeks 10 to 12, 14 to 16, 
18 to 20, and 22 to 24) ......................................................................................... 60 
8.3 
Treatment Period Part 2 (Weeks 25 to 76) ................................................................... 60 
8.3.1 
Additional site visits during maximum 52-week Treatment Period Part 2 
(monthly starting at Week 25) or until availability of an Access Program 
(whichever comes first) ....................................................................................... 61 
8.3.2 
Additional home visits during Treatment Period Part 2 (weekly in between site 
visits starting at Week 26 or until availability of an Access Program (whichever 
comes first) .......................................................................................................... 62 


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8.4 
Observation Period, Visits 26 to 29 (Weeks 25 to 32 study participants completing 
Treatment Period Part 1 only and Weeks 77 to 84 for study participants completing 
Treatment Period Part 1 and Part 2) ............................................................................. 62 
8.4.1 
Visit 26 (Week 25 or Week 77 or PEOT) ............................................................ 63 
8.4.2 
Visits 27 and 28 (Weeks 27 and 29, and at 79 and 81), Home Visits ................. 64 
8.4.3 
Visit 29/FV (Week 32/84) ................................................................................... 64 
8.5 
Premature end of treatment and Final Visit ................................................................. 65 
8.6 
Unscheduled Visit/Telephone call ............................................................................... 66 
9 
ASSESSMENT OF SAFETY .......................................................................................... 66 
9.1 
Adverse events ............................................................................................................. 66 
9.1.1 
Definitions ........................................................................................................... 66 
9.1.1.1 
Adverse event ............................................................................................ 66 
9.1.1.2 
Serious adverse event ................................................................................ 66 
9.1.1.3 
Adverse events of special interest ............................................................. 67 
9.1.1.4 
Adverse events of interest ......................................................................... 68 
9.1.2 
Procedures for reporting and recording adverse events ....................................... 68 
9.1.2.1 
Description of adverse events .................................................................... 68 
9.1.2.2 
Rule for repetition of an adverse event...................................................... 69 
9.1.2.3 
Additional procedures for reporting serious adverse events ..................... 69 
9.1.2.4 
Immediate reporting of adverse events...................................................... 70 
9.1.3 
Follow up of adverse events ................................................................................ 70 
9.1.4 
Pregnancy ............................................................................................................ 70 
9.1.5 
Suspected transmission of an infectious agent via a medicinal product .............. 71 
9.1.6 
Overdose of investigational medicinal product ................................................... 71 
9.1.7 
Safety signal detection ......................................................................................... 71 
9.1.8 
Suicidality ............................................................................................................ 72 
9.1.9 
Hypersensitivity and adverse reactions ............................................................... 72 
9.1.10 
Management of headache .................................................................................... 73 
9.1.11 
Management of moderate or severe diarrhea ....................................................... 74 
9.2 
Laboratory measurements ............................................................................................ 74 
9.2.1 
Evaluation of PDILI ............................................................................................ 77 
9.2.1.1 
Consultation with Medical Monitor and local hepatologist ...................... 80 
9.2.1.2 
Immediate action: determination of IMP discontinuation ......................... 80 
9.2.1.3 
Testing: identification/exclusion of alternative etiology ........................... 81 
9.2.1.4 
Follow-up evaluation ................................................................................. 83 
9.3 
Other safety measurements .......................................................................................... 83 
9.3.1 
Pregnancy testing ................................................................................................. 83 
9.3.2 
Vital signs ............................................................................................................ 84 
9.3.3 
Physical examination ........................................................................................... 84 


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9.3.3.1 
Full physical examination ......................................................................... 84 
9.3.3.2 
Brief physical examination ........................................................................ 84 
9.3.4 
Neurological examination .................................................................................... 85 
9.3.5 
12-lead ECG ........................................................................................................ 85 
9.3.6 
Assessment and management of TB and TB risk factors .................................... 85 
10 ASSESSMENT OF EFFICACY ...................................................................................... 87 
10.1 
iRODS .......................................................................................................................... 87 
10.2 
INCAT ......................................................................................................................... 88 
10.3 
Clinician-reported grip strength ................................................................................... 88 
10.4 
Patient-reported outcomes ........................................................................................... 88 
10.4.1 
CIDP PRO instrument ......................................................................................... 89 
10.4.2 
Fatigue ................................................................................................................. 89 
10.4.3 
Patient Global Impressions .................................................................................. 89 
10.5 
RT-MRC ...................................................................................................................... 89 
11 ASSESSMENT OF PHARMACOKINETIC/ PHARMACODYNAMIC VARIABLES90 
11.1 
Pharmacokinetic variable ............................................................................................. 90 
11.2 
Pharmacodynamic variables ........................................................................................ 90 
12 ASSESSMENT OF IMMUNOLOGICAL VARIABLES ............................................... 90 
12.1 
Assessment of exploratory biomarkers ........................................................................ 91 
13 STUDY MANAGEMENT AND ADMINISTRATION ................................................. 91 
13.1 
Adherence to protocol .................................................................................................. 91 
13.2 
Monitoring ................................................................................................................... 92 
13.2.1 
Definition of source data ..................................................................................... 92 
13.2.2 
Source data verification ....................................................................................... 92 
13.3 
Data handling ............................................................................................................... 93 
13.3.1 
Case Report form completion .............................................................................. 93 
13.3.2 
Database entry and reconciliation ........................................................................ 93 
13.3.3 
Study participant Screening and Enrollment log/Study participant Identification 
Code list ............................................................................................................... 93 
13.4 
Termination of the study .............................................................................................. 93 
13.5 
Archiving and data retention ........................................................................................ 94 
13.6 
Audit and inspection .................................................................................................... 94 
13.7 
Good Clinical Practice ................................................................................................. 94 
14 STATISTICS ................................................................................................................... 95 
14.1 
Definition of analysis sets ............................................................................................ 95 
14.1.1 
Enrolled Set ......................................................................................................... 95 
14.1.2 
Safety Set ............................................................................................................. 95 
14.1.3 
Full Analysis Set .................................................................................................. 95 


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14.1.4 
Pharmacokinetic Per-Protocol Set ....................................................................... 95 
14.1.5 
Pharmacodynamic Per-Protocol Set .................................................................... 95 
14.2 
General statistical considerations ................................................................................. 95 
14.3 
Planned safety analyses................................................................................................ 95 
14.3.1 
Safety analyses ..................................................................................................... 95 
14.4 
Other analyses .............................................................................................................. 96 
14.4.1 
Efficacy analyses ................................................................................................. 96 
14.4.2 
Pharmacokinetic analyses .................................................................................... 97 
14.4.3 
Pharmacodynamic analyses ................................................................................. 97 
14.4.4 
Immunological analyses ...................................................................................... 97 
14.5 
Handling of protocol deviations................................................................................... 97 
14.6 
Handling of dropouts or missing data .......................................................................... 97 
14.7 
Planned interim analysis and data monitoring ............................................................. 98 
14.8 
Determination of sample size ....................................................................................... 98 
15 ETHICS AND REGULATORY REQUIREMENTS ...................................................... 98 
15.1 
Informed consent ......................................................................................................... 98 
15.2 
Study participant identification cards .......................................................................... 99 
15.3 
Institutional Review Boards and Independent Ethics Committees .............................. 99 
15.4 
Study participant privacy ........................................................................................... 100 
15.5 
Protocol amendments ................................................................................................. 100 
16 FINANCE, INSURANCE, AND PUBLICATION ....................................................... 100 
17 REFERENCES .............................................................................................................. 100 
18 APPENDICES ............................................................................................................... 102 
18.1 
Suggested management guidelines for infusion reactions ......................................... 102 
18.2 
Diagnosis of anaphylactic reactions ........................................................................... 103 
18.3 
Protocol amendment 1 ............................................................................................... 104 
18.4 
Protocol amendment 2 ............................................................................................... 106 
18.5 
Protocol amendment 3 ............................................................................................... 120 
19 DECLARATION AND SIGNATURE OF INVESTIGATOR ..................................... 159 
20 SPONSOR DECLARATION ........................................................................................ 160 
 


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LIST OF TABLES 
Table 5-1: 
Schedule of study assessments......................................................................... 24 
Table 5-2: 
Additional study assessments .......................................................................... 34 
Table 6-1: 
Time restrictions required prior to Baseline Visit (Visit 2) for 
immunosuppressants, biologics, and other therapies ....................................... 40 
Table 7-1: 
IMP doses to be administered (equivalent to approximately 
) by 
body weight ...................................................................................................... 45 
Table 7-2: 
IMP doses to be administered (equivalent to approximately 
 by 
body weight ...................................................................................................... 45 
Table 7-3: 
Permitted concomitant medications ................................................................. 48 
Table 9-4: 
Infusion-related reaction grading according to the NCI Common 
Terminology Criteria for adverse events version 5.0 (Jun 2017) .................... 73 
Table 9-5: 
Laboratory measurements ................................................................................ 76 
Table 9-6: 
Required investigations and follow up for PDILI............................................ 78 
Table 9-7: 
PDILI laboratory measurements ...................................................................... 82 
Table 9-8: 
PDILI information to be collected ................................................................... 83 
Table 11-1: 
Serial blood sampling for rozanolixizumab concentration .............................. 90 
Table 18-1: 
Suggested management guidelines for infusion reactions ............................. 102 


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CIDP04 
 
 
 
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LIST OF ABBREVIATIONS 
ADA 
antidrug antibody 
ADL 
activities of daily living 
AE 
adverse event 
ALP 
alkaline phosphatase 
ALT 
alanine aminotransferase 
AST 
aspartate aminotransferase 
AUC 
area under the curve from time 0 to infinity 
BP 
blood pressure 
CDMS 
clinical data management system 
CI 
confidence interval 
CIDP 
chronic inflammatory demyelinating polyradiculoneuropathy 
Cmax 
maximum observed plasma concentration 
CRO 
contract research organization 
C-SSRS 
Columbia Suicide Severity Rating Scale 
CTCAE 
Common Terminology Criteria for Adverse Events 
DMC 
Data Monitoring Committee 
ECG 
electrocardiogram 
eCRF 
electronic Case Report form 
ES 
Enrolled Set 
EudraCT 
European Union Drug Regulating Authorities Clinical Trials 
FAS 
Full Analysis Set 
FcRn 
neonatal Fc receptor  
FIH 
first-in-human 
FV 
Final Visit 
GCP 
Good Clinical Practice 
HIV 
human immunodeficiency virus 
HIV1 
human immunodeficiency virus antibody 1 
HIV2 
human immunodeficiency virus antibody 2 
IA 
immunoabsorption 
IB 
Investigator’s Brochure 
ICF 
Informed Consent form 


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CIDP04 
 
 
 
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ICH 
International Council for Harmonisation 
IEC 
Independent Ethics Committee 
IgA 
immunoglobulin A 
IgE 
immunoglobulin E 
IgG 
immunoglobulin G 
IgM 
immunoglobulin M 
IGRA 
interferon gamma release assay 
IMP 
investigational medicinal product 
INCAT 
Inflammatory Neuropathy Cause and Treatment 
IND 
Investigational New Drug 
IRB 
Institutional Review Board 
iRODS 
inflammatory Rasch-built Overall Disability Scale 
IRT 
interactive response technology 
ITP 
immune thrombocytopenia 
iv 
intravenous(ly) 
IVIg 
intravenous immunoglobulin 
LLOQ 
lower limit of quantification 
logit 
log odds unit 
LTBI 
latent tuberculosis infection 
MCID-SE 
minimum clinically important differences-standard error 
MedDRA 
Medical Dictionary for Regulatory Activities 
MG 
myasthenia gravis 
NF-L 
neurofilament light chain 
NTMBI 
nontuberculosis mycobacterium infection 
OLE 
open-label extension  
PD 
pharmacodynamics 
PDILI 
potential drug-induced liver injury 
PD-PPS 
Pharmacodynamic Per-Protocol Set 
PDS 
Protocol Deviation Specification 
PEOT 
premature end of treatment 
PGIC 
Patient Global Impressions of Change 
PGIS 
Patient Global Impressions of Severity 


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PK 
pharmacokinetic 
PK-PPS 
Pharmacokinetic Per-Protocol Set 
PLEX 
plasma exchange 
PPS 
Per-Protocol Set 
PR 
pulse rate 
PRO 
patient-reported outcome 
PS 
Patient Safety 
QTc 
QT-interval corrected for heart rate 
RT-MRC 
Rasch-built, modified interval Medical Research Council scale 
SAE 
serious adverse event 
SAP 
Statistical Analysis Plan 
sc 
subcutaneous(ly) 
SCIg 
subcutaneous immunoglobulin 
SD 
standard deviation 
SOC 
standard of care 
SOP 
Standard Operating Procedure 
SS 
Safety Set 
TB 
tuberculosis 
TEAE 
treatment-emergent adverse event 
ULN 
upper limit of normal 
WHO 
World Health Organization 


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1 
SUMMARY 
Rozanolixizumab (UCB7665) is a humanized anti-neonatal Fc receptor (FcRn) monoclonal 
antibody that is being developed as an inhibitor of FcRn activity with the aim to reduce the 
concentration of pathogenic immunoglobulin (IgG) in patients with IgG auto-antibody mediated 
diseases such as chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). 
CIDP04 is a Phase 2A, multicenter, single-arm, open-label study with the primary objective of 
evaluating the long-term safety and tolerability of rozanolixizumab when administered as 
 
subcutaneous (sc) infusion in study participants with CIDP. The secondary objective includes 
evaluating the long-term clinical efficacy of 
doses of rozanolixizumab in the study 
participants with CIDP. This open-label study will provide study participants from previous 
rozanolixizumab studies (eg, CIDP01) the opportunity to have continued access to 
rozanolixizumab. 
Access to CIDP04 for study participants coming from CIPD01: Study participants from CIDP01 
who have completed the Treatment Period (ie, all visits up to Visit 17) without a relapse of CIDP 
will be offered the opportunity to be directly enrolled into this open-label extension (OLE) study. 
Study participants from CIDP01 who have experienced a relapse of CIDP (during either the 
Treatment Period or the Observation Period) and have been successfully rescued and stabilized 
with standard of care (SOC) medication may also be eligible to enroll in CIDP04. The study 
participants who completed the Treatment Period will be offered the opportunity to enter directly 
once stabilized. The study participants who relapsed during Treatment Period will be offered 
entry into CIDP04 only once CIDP01 study results are available, after the blind has been broken 
at the end of the study, and it is confirmed they received placebo in CIDP01. 
In case the study participants have a gap period between the end of the parent study and the start 
of this open-label study (ie, last visit in the parent study does not take place on the same day as 
entry of study CIDP04), a Screening Period of 2 to 5 weeks will be applicable to confirm that the 
study participant still meets the eligibility criteria for entry in the open-label study and allow for 
a smooth transition between the treatment used by the study participants during the gap period 
and initiation of rozanolixizumab. The CIDP04 study includes a 24-week Treatment Period, an 
optional additional maximum 52-week Treatment Period followed by an 8-week Observation 
Period. The length of the additional Treatment and Observation Periods may be shortened in case 
of availability of an Access Program. For the Treatment Periods, study participants will have 
weekly visits (either on site or at home) during which they will be dosed with an sc infusion of 
rozanolixizumab, up to 
. The initial dose in the study will be based on the dose the study 
participant has received at the completion of the parent study (eg, CIDP01). For study 
participants who were known to be on placebo in the parent study (eg, CIDP01), a starting dose 
of 
 will be used. Dose may be reduced to 
if 
 is not tolerable (eg, 
headache) and/or recurrent low IgG levels (Treatment Period Part 2). The maximum dose of 
rozanolixizumab in CIDP04 will be 
. For exact doses to be administered, refer to 
Section 7.2. 
The primary safety variable is the occurrence of treatment-emergent adverse events (TEAEs). 
The other safety variables include TEAEs leading to withdrawal of the investigational medicinal 
product (IMP); assessment of values and change from Baseline of: vital signs (systolic and 
diastolic blood pressure [BP], pulse rate [PR], body temperature, and body weight); physical and 


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neurological examination findings; 12-lead electrocardiograms (ECGs); clinical laboratory 
findings (hematology, clinical chemistry, and urinalysis); Tuberculosis Signs and Symptoms 
Questionnaire; and total protein, albumin, α- and β-globulins. The other variables include the 
efficacy variables of changes in study participant’s score on inflammatory Rasch built Overall 
Disability Scale (iRODS), Inflammatory Neuropathy Cause and Treatment (INCAT), maximum 
grip strength (assessed by site personnel), the Rasch-built, modified interval Medical Research 
Council scale (RT-MRC) sum score, and patient-reported outcomes (PROs); pharmacokinetic 
(PK) variable of the plasma concentration of rozanolixizumab; pharmacodynamic (PD) variables 
of changes in concentration of serum IgG, and its subclasses, and neurofilament light chain 
(NF-L) levels in serum; and immunological variables of changes in concentration of serum IgA, 
IgE, and IgM, serum complement levels (C3 and C4) and plasma complement levels (C3a and 
C5a), anti-rozanolixizumab antibodies levels, cytokines levels, CIDP-specific auto-antibody 
levels, and tetanus- and influenza A virus-specific IgG antibodies. 
A total of 
 study participants participating in the parent study CIDP01 may be enrolled in this 
OLE study; enrollment is planned at approximately 24 sites in Europe, United States of America, 
and Canada, with possible extension to other regions and countries. 
A Data Monitoring Committee (DMC) will be established for the study. 


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2 
INTRODUCTION 
Chronic inflammatory demyelinating polyradiculoneuropathy is a rare disorder of the peripheral 
nerves characterized by chronic and progressive onset of weakness, loss of sensation, reflexes, 
reduction or partial block in motor nerve conduction, and often pain and imbalance. Worldwide, 
the prevalence of CIDP ranges from approximately 1 to 9 cases per 100,000 
(Mathey et al, 2015). For many patients, CIDP results in substantial disability, limiting their 
activities resulting in an overall negative impact on quality of life across multiple dimensions. 
Progressive disease and/or relapses can result in difficulty moving arms, hands, legs, and feet, a 
loss in the ability to walk independently or confinement to wheelchairs and reliance on 
caregivers (Mahdi-Rogers et al, 2014). Healthcare costs for CIDP are substantial 
(Guptill et al, 2014; Mahdi-Rogers et al, 2014; Olesen et al, 2012). 
Cellular and humoral immune mechanisms are thought to be involved in the pathogenesis of 
CIDP resulting in inflammatory lesions in the spinal roots, proximal nerve trunks and along the 
peripheral nerves. The essential role of the autoimmune antibodies in mediating this pathology is 
supported by the improvement seen after plasma exchange (PLEX) and immunoabsorption (IA). 
Identification of the specific antigenic target(s) of the autoimmune antibodies in CIDP is 
expanding with recent immunological techniques. 
Although the prognosis of CIDP has markedly improved over the last decades, significant 
disability remains. High-dose steroids, PLEX, IA, and intravenous and subcutaneous 
immunoglobulin (IVIg and SCIg) have all been shown to be effective treatments for CIDP. 
However, comorbid diseases, including hypertension, diabetes, and psychiatric diseases, limit the 
use of high-dose steroids. While PLEX is effective, the procedure is burdensome for patients. 
Immunoglobulin treatment (IVIg and SCIg) is often used as first-line treatment but there remains 
a medical need for improved therapeutic options. 
The neonatal major histocompatibility complex-class-I-like FcRn recycles immunoglobulin G 
(IgG) and albumin from most cells and transports it bi-directionally across epithelial barriers to 
affect systemic and mucosal immunity. It has been 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). Immunoglobulin G has a half-life of approximately 
3 weeks in man. This is achieved by interaction of IgG with the receptor, FcRn. Thus in effect 
FcRn salvages IgG, and blockade of FcRn accelerates removal of endogenous IgG. 
Rozanolixizumab is a humanized anti-FcRn monoclonal antibody that has been specifically 
designed to inhibit IgG binding to FcRn without inhibiting albumin binding to FcRn. 
Rozanolixizumab is being developed as an inhibitor of FcRn activity with the aim to reduce the 
concentration of pathogenic IgG in patients with IgG auto-antibody mediated diseases. 
Rozanolixizumab was derived from a rat antibody with specificity for human FcRn. To date, 
rozanolixizumab has been administered to healthy study participants in a completed first-in-
human (FIH) study (UP0018), a completed Phase 2 study in study participants with primary 
immune thrombocytopenia (ITP) (TP0001, evaluating 
 by sc route 
across 5 cohorts), and a completed Phase 2 study in study participants with myasthenia gravis 
(MG) (MG0002, evaluating 
 by sc route). 
Additional information on the development of rozanolixizumab can be found in the current 
version of the Investigator's Brochure (IB) for rozanolixizumab. 


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The extension of the development program of rozanolixizumab to the indication of CIDP is 
based on the underlying hypothesis that reducing the concentration of pathogenic IgG in patients 
with CIDP will improve the symptoms of CIDP and prevent its long-term progression. CIDP01 
is the first study investigating this indication; it is a Phase 2A, multicenter, randomized, study 
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 active CIDP who have been maintained on an immunoglobulin-dependent 
regimen. In addition, CIDP01 will assess the safety and tolerability of multiple dosing with 
rozanolixizumab including the development of antidrug antibodies (ADA) and potential to 
impact clinical response, and will provide information on the PK and PD of rozanolixizumab in 
study participants with active CIDP. The maximum duration of the study per study participant is 
approximately 28 weeks (up to maximum 40 weeks), consisting of a Screening Period of 
between 2 and 5 weeks duration, an 11-week Treatment Period, and an Observation Period of 12 
weeks (up to 24 weeks). Study participants will be randomized to 1 of 2 treatment arms: 
rozanolixizumab 
sc or placebo sc in a ratio of 1:1. Study participants will receive 
 
 of IMP. 
2.1.1 
Rationale for this study  
CIDP04 will evaluate the long-term safety, tolerability, and efficacy of treatment with 
rozanolixizumab with a 
 dosing regimen for 24 weeks in subjects with active CIDP who 
have been maintained on an immunoglobulin-dependent regimen. An optional extension of the 
treatment period by a maximum of an additional 52 weeks, or until an Access Program or 
equivalent is in place, whichever comes first, will be allowed on the basis of each participant’s 
individual benefit-risk assessment at the end of the first 24-weeks Treatment Period. Study 
participants may choose to self-administer the IMP during Treatment Period Part 2 under 
supervision of a nurse and after having received appropriate training. 
The CIDP04 OLE study will provide the opportunity to assess the long-term safety of 
rozanolixizumab in study participants with CIDP from CIDP01. 
3 
STUDY OBJECTIVES 
3.1 
Primary objective 
The primary objective of the study is to assess long-term safety and tolerability of 
 doses 
of rozanolixizumab in study participants with CIDP. 
3.2 
Secondary objective 
The secondary objective of the study is to assess long-term clinical efficacy of 
 doses of 
rozanolixizumab. 
3.3 
Exploratory objectives 
The exploratory objectives of the study are to assess the PD, PK, and immunological variables in 
the treatment course with rozanolixizumab and to assess whether long-term dosing of 
rozanolixizumab improves PROs. These include: 
 The PD effect of rozanolixizumab as measured by the total IgG concentrations in serum 


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 The effects of rozanolixizumab on the concentrations of total protein, albumin, α- and 
β-globulins, IgG subclasses, IgM, IgA, IgE, and serum and plasma complement levels 
 The incidence and emergence of ADA with respect to immunogenicity and PK and PD 
 The effect of rozanolixizumab on complement and cytokines 
 The plasma concentrations of rozanolixizumab administered by sc infusion 
 The PD effect of rozanolixizumab as measured by NF-L in serum 
 The effect of rozanolixizumab on CIDP-specific auto-antibody levels 
 The effect of rozanolixizumab on vaccine antibody levels (influenza A and tetanus) 
 The effect of rozanolixizumab on exploratory biomarkers, and explore the relationship 
between protein and metabolite biomarkers and cause, progression, and appropriate treatment 
of CIDP 
4 
STUDY VARIABLES 
For all study variables, the Baseline values for study participants who enroll directly into 
CIDP04 will be the Baseline values from the parent study (eg, CIDP01). For study participants 
with a gap period prior to enrollment in CIDP04, details of the analyses of specific Baseline will 
be available in the Statistical Analysis Plan (SAP). 
4.1 
Safety variables 
4.1.1 
Primary safety variables 
The primary safety variable is: 
 Occurrence of TEAEs 
4.1.2 
Other safety variables 
The other safety variables are: 
 TEAEs leading to permanent withdrawal of IMP 
 Vital signs values and changes from Baseline (systolic and diastolic BP, PR, body 
temperature, and body weight) at each scheduled assessment during the Treatment and 
Observation Periods 
 Physical examination findings 
 Neurological examination findings 
 12-lead ECG values and change from Baseline at each scheduled assessment during the 
Treatment and Observation Periods 
 Laboratory values and changes from Baseline at each scheduled assessment during the 
Treatment and Observation Periods (hematology, clinical chemistry, and urinalysis) 
 Tuberculosis Signs and Symptoms Questionnaire at each scheduled assessment during the 
Treatment and Observation Periods 


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 Values and change from Baseline in concentrations of total protein, albumin, α- and 
β-globulins at each scheduled assessment during the Treatment and Observation Periods 
4.2 
Other variables 
4.2.1 
Efficacy variables 
Preliminary definitions linked to assessment of relapse: 
 CIDP relapse (iRODS) is defined as a clinically important deterioration from Baseline in 
iRODS score, ie, a minimum clinically important differences-standard error (MCID-SE) of 
≤-1.96 
 CIDP relapse (adjusted INCAT) is defined as an increase from Baseline of at least 1 point in 
the adjusted INCAT score. The adjusted score is identical to the INCAT disability score 
except for the exclusion of changes in upper limb function from 0 (normal) to 1 (minor 
symptoms) or from 1 to 0 
 CIDP relapse (maximum grip strength as assessed by site personnel) is defined as a clinically 
important deterioration from Baseline in grip strength as measured by site personnel, ie, a 
decline of >14kPa 
The other efficacy variables are: 
 Values and absolute change from Baseline in iRODS scores at each scheduled assessment 
during the Treatment and Observation Periods 
 Study participant experienced CIDP relapse (iRODS) up to Week 25 and Week 77 (where 
applicable) from Baseline 
 Time to CIDP relapse (iRODS) during the Treatment Period from Baseline 
 Study participant experienced CIDP relapse (adjusted INCAT) up to Week 25 and Week 77 
(where applicable) from Baseline 
 Time to CIDP relapse (adjusted INCAT) during the Treatment Periods from Baseline 
 Values and absolute change from Baseline in adjusted INCAT score at each scheduled 
assessment during the Treatment and Observation Periods 
 Study participant experienced CIDP relapse (maximum grip strength as assessed by site 
personnel) up to Week 25 and Week 77 (where applicable) from Baseline 
 Time to CIDP relapse (maximum grip strength as assessed by site personnel) during the 
Treatment Periods from Baseline 
 Values and absolute change from Baseline in maximum grip strength score (maximum of 
3 assessments) taken by site personnel at each scheduled assessment during the Treatment 
and Observation Periods 
 Values and absolute change from Baseline in RT-MRC sum score at each scheduled 
assessment during the Treatment and Observation Periods 
 Study participants receiving rescue medication during Treatment Periods 


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 Time to rescue medication administration during Treatment Periods 
4.2.2 
Patient-reported outcome variables 
The PRO variables are: 
 Values and change from Baseline in fatigue domain scores at each scheduled assessment 
during the Treatment and Observation Periods 
 Values and change from Baseline in CIDP PRO instrument domain scores at each scheduled 
assessment during the Treatment and Observation Periods 
 Values and change from Baseline in Patient Global Impressions of Severity (PGIS) at each 
scheduled assessment during the Treatment and Observation Periods  
 Patient Global Impressions of Change (PGIC) value at each scheduled assessment during the 
Treatment and Observations Periods 
4.2.3 
Pharmacokinetic variable 
The PK variable is the plasma concentration of rozanolixizumab at each scheduled assessment 
during the Treatment Periods. 
4.2.4 
Pharmacodynamic variables  
The PD variables are: 
 Minimum value and maximum decrease (absolute and percentage) from Baseline in total 
serum IgG concentration during the study 
 Values and change (absolute and percentage) from Baseline in total serum IgG 
concentrations at each scheduled assessment during Treatment and Observation Periods 
 Values and change (absolute and percentage) from Baseline in serum IgG subclass 
concentrations at each scheduled assessment during Treatment and Observation Periods 
 Values and change (absolute and percentage) from Baseline in NF-L levels at each scheduled 
assessment during Treatment and Observation Periods 
4.2.5 
Immunological variables 
The immunological variables are: 
 Values and change from Baseline in serum immunoglobulin concentrations (total IgA, IgE, 
and IgM) at each scheduled assessment during Treatment and Observation Periods 
 Values and change from Baseline in serum complement levels (C3 and C4) and plasma 
complement levels (C3a and C5a) at each scheduled assessment during Treatment Period 
 ADA (anti-rozanolixizumab antibodies) status (negative or confirmed positive) and the 
confirmed positive titer at each scheduled assessment during Treatment and Observation 
Periods 
 Values and change from Baseline in cytokines at each scheduled assessment during 
Treatment and Observation Periods 


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 Change in CIDP-specific auto-antibody levels in serum from Baseline during Treatment and 
Observation Periods 
 Values and change from Baseline in tetanus- and influenza A virus-specific IgG antibodies 
during Treatment and Observation Periods 
4.2.6 
Exploratory biomarkers 
The exploratory biomarker variables are: 
 Protein and metabolites changes that may be measured to understand the cause, progression, 
and appropriate treatment of CIDP 
 Exploratory biomarkers such as, but not limited to, 
 
  
 Absolute change from Baseline relating to mechanism of action, disease activity, treatment 
response, and clinical outcome at each scheduled assessment during the Treatment and 
Observation Periods  
5 
STUDY DESIGN 
5.1 
Study description 
This is a Phase 2A, multicenter, single-arm, OLE study to evaluate the safety, tolerability, and 
efficacy of rozanolixizumab during long-term treatment of study participants with CIDP from a 
previous study with rozanolixizumab. 
Access to CIDP04 for study participants coming from CIDP01: Study participants from CIDP01 
who have completed the Treatment Period (ie, all visits up to Visit 17) without a relapse of CIDP 
will be offered the opportunity to be directly enrolled into this OLE study. Study participants will 
enter CIDP04 on the same day as the last study visit in CIDP01: Visit 17. Study participants 
from CIDP01 who have experienced a relapse of CIDP (during either the Treatment Period or 
the Observation Period), have been successfully rescued and stabilized with SOC medication and 
have consented to enter this OLE study, may be assessed for eligibility. The study participants 
who relapsed during the Treatment Period of CIDP01 will be offered entry into CIDP04 only 
once CIDP01 study results are available, after the blind has been broken at the end of the study, 
and it is confirmed they received placebo in CIDP01. 
In case the study participants have a gap period between the end of the parent study and the start 
of this OLE study (ie, last visit in the parent study does not take place on the same day as entry 
of study CIDP04), a Screening Period of 2 to 5 weeks will be applicable to confirm that the study 
participant still meets the eligibility criteria for entry in the OLE study and allow for a smooth 
transition between the treatment used by the study participants during the gap period and the 
initiation of rozanolixizumab. The CIDP04 study includes a first 24-week Treatment Period (Part 
1) followed by a second Treatment Period (Part 2) of up to 52 weeks (or until an Access Program 
or equivalent is in place, whichever comes first). Entry to the Treatment Period (Part 2) will be 
contingent to a favorable individual benefit-risk after careful individual assessment has been 
performed for each study participant completing Treatment Period (Part 1). An 8-week 
Observation Period will be followed in case of premature termination or after completion of the 
Treatment Periods (either at end of Part 1 for study participants not continuing in Part 2 or at end 


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of Part 2). The Observation Period will not be performed in the event of a study participant 
continuing treatment with rozanolixizumab after the end of the study (ie, in case of an Access 
Program), in which case only the PEOT (Visit 26) Visit will be performed.  
During the Treatment Periods (Part 1 and Part 2), study participants will have weekly visits 
(either on site or at home) during which they will be dosed with an sc infusion of 
rozanolixizumab up to 
. The initial dose in the study will be based on the dose the study 
participant has received at the completion of the parent study (eg, CIDP01). For study 
participants who were known to be on placebo in the parent study, a starting dose of 
 
will be used. The maximum dose will be 
. See Section 7.2 for details on treatment to be 
administered. 
During the first 4 weeks of the Treatment Period (Part 1), all visits will be on site. During Weeks 
5 to 8, home visits and on-site visits will be alternating (Weeks 5 and 7 will be on site; Weeks 6 
and 8 will be at home). Starting at Week 9 and through the end of the Treatment Period (Week 
24 and if applicable, Week 76), study participants will have 1 visit on site followed by 3 visits at 
home for every 4-week period. 
After the completion of the Treatment Period(s) (starting at Week 25 for study participants only 
completing Part 1 or from Week 77 for study participants completing both Part 1 and Part 2 or at 
any early timepoint in case of PEOT), study participants will enter the 8-week Observation 
Period where no IMP will be administered. During the Observation Period, study participants 
will have on-site visits at the entry and exit of the Observation Period (Weeks 25/77, and 32/84, 
and home visits at Weeks 27/79 and 29/81). Study participants can return to their SOC during the 
Observation Period. In case the study participant enters an Access Program, no Observation 
Period will be performed with the exception of the PEOT (Visit 26) Visit which will be 
immediately completed. 
At the discretion of the investigator and/or study participant, home visits can be changed to site 
visits (eg, for safety reasons). In case an on-site visit cannot be performed, the next visit will be 
performed at site instead of at home, and all safety assessments that should have been completed 
at the missed visits will be performed. 
The assessments to be completed during each visit are presented in Table 5-1. 
5.1.1 
Study duration per study participant 
The total duration of the study for an individual study participant will be up to 89; this includes a 
2- to 5-week Screening Period, a 24-week Treatment Period (Part 1), an optional maximum 
52-week Treatment Period (Part 2), or until an Access Program or equivalent is in place 
whichever comes first, and an 8-week Observation Period in case the participant does not enter 
in an Access Program (or equivalent). 
The end of the study is defined as the date of the last visit of the last study participant in the 
study. 
5.1.2 
Planned number of study participants and sites 
A total of 
 study participants participating in the parent study CIDP01 may be enrolled in this 
OLE study; enrollment is planned at approximately 24 sites. 


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Depending on the further development of the UCB CIDP program, there may also be an 
opportunity for additional, future CIDP patients to enroll into CIDP04 from other parent studies 
at a later time point. 
5.1.3 
Anticipated regions and countries 
Anticipated regions are Europe, United States of America, and Canada, with possible extension 
to other regions and countries. 
5.2 
Schedule of study assessments 
The schedule of study assessments are provided in Table 5-1. 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr b 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type c 
S 
S 
S 
S 
S 
S 
H d 
S 
H d 
S 
H d 
H d 
H d 
S 
H d 
S 
H d H d 
S 
Written informed consent e 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic data e 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Verification of inclusion 
and exclusion criteria e 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Withdrawal criteria 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Medical history update e 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior and concomitant 
medication 
X 
X f 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant medical 
procedures 
X 
X f 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Vital signs g 
X 
X  
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr b 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type c 
S 
S 
S 
S 
S 
S 
H d 
S 
H d 
S 
H d 
H d 
H d 
S 
H d 
S 
H d H d 
S 
Body weight 
X 
X f 
 
 
 
 
 
 
 
 
 
 
 
X h 
 
X 
 
 
X 
Recording of AEs 
X 
X f 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Query for suicidality i 
X 
X f 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
X 
X 
Fatigue scale 
 
X f 
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
CIDP PRO Instrument 
 
X f 
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
PGIS 
 
X f 
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
PGIC 
 
X f k  
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
Full physical examination 
X 
X f 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Brief physical examination 
 
 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr b 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type c 
S 
S 
S 
S 
S 
S 
H d 
S 
H d 
S 
H d 
H d 
H d 
S 
H d 
S 
H d H d 
S 
Full neurological 
examination l 
X 
X f 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Brief neurological 
examination 
 
 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
 
12-lead ECG 
X 
X f 
X 
X 
X 
 
 
X 
 
X 
 
X 
 
X m 
 
X 
 
 
X 
Labs (hematology, 
chemistry, urinalysis) 
X 
X f 
X 
X 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
 
X 
Serology for HIV, hepatitis 
B, and hepatitis C n 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Serum/urine pregnancy test 
o 
X 
X f 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
X 
X 
IGRA tuberculosis test p 
X 
X f 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr b 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type c 
S 
S 
S 
S 
S 
S 
H d 
S 
H d 
S 
H d 
H d 
H d 
S 
H d 
S 
H d H d 
S 
Tuberculosis Signs and 
Symptoms Questionnaire 
X 
 
 
 
 
 
 
 
 
 
 
 
X q 
X q 
 
 
 
 
X 
Contact IRT 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
X 
Administration of IMP 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X r 
X r 
 
 
 
 
Blood sampling for PK of 
rozanolixizumab s 
 
X f 
 
X 
 
 
 
X 
 
X t 
 
 
 
X m  
 
X 
 
 
 
ADA (Anti-
rozanolixizumab 
antibodies) 
 
X f 
 
X 
 
 
 
X 
 
X t 
 
 
 
X m 
 
X 
 
 
X 
Serum complement (C3, 
C4) and plasma 
complement (C3a, C5a) u 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
X 
 
 
 
 
 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr b 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type c 
S 
S 
S 
S 
S 
S 
H d 
S 
H d 
S 
H d 
H d 
H d 
S 
H d 
S 
H d H d 
S 
Serum cytokines u 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
X 
 
 
 
 
 
Vaccination-specific 
antibody titers (tetanus and 
influenza Type A) 
X 
 
 
 
 
 
 
X 
 
X v 
 
 
 
X m 
 
X 
 
 
 
Immunoglobulins 
(total IgG and IgG 
subclasses) 
X 
X f 
X 
X 
X 
X 
 Xw 
X 
X w 
X 
X w 
X w X w 
X 
X w 
X 
 
 
X 
NF-L 
 
X f 
 
 
 
 
 
X 
 
 
 
 
 
X x 
 
X y 
 
 
 
IgA, IgM, IgE 
 
X f 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X m 
 
X 
 
 
X 
CIDP-specific auto-
antibodies 
 
X f 
 
 
 
X 
 
 
 
X 
 
 
 
X x 
 
X y 
 
 
X x 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr b 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type c 
S 
S 
S 
S 
S 
S 
H d 
S 
H d 
S 
H d 
H d 
H d 
S 
H d 
S 
H d H d 
S 
Blood sampling for 
exploratory biomarker 
analysis z 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
 
 
 
 
 
 
iRODS assessment  aa 
X 
X f 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
X 
INCAT assessment 
X 
X f 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
X 
Assessment of grip strength 
by site personnel 
X 
X f 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
X 
RT-MRC assessment 
X 
X f 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X x 
 
X y 
 
 
X x 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr b 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type c 
S 
S 
S 
S 
S 
S 
H d 
S 
H d 
S 
H d 
H d 
H d 
S 
H d 
S 
H d H d 
S 
ADA=antidrug antibody; AE=adverse event; BL=baseline; CIDP=chronic inflammatory demyelinating polyradiculoneuropathy; C-SSRS=Columbia Suicide 
Severity Rating Scale; ECG=electrocardiogram; FV=Final Visit; H=Home visit; HIV=human immunodeficiency virus; ICF=Informed Consent form; 
IgA=immunoglobulin A; IgE=immunoglobulin E; IgG=immunoglobulin G; IgM=immunoglobulin M; IGRA=interferon gamma release assay; 
IMP=investigational medicinal product; INCAT=Inflammatory Neuropathy Cause and Treatment; iRODS=inflammatory Rasch-built Overall Disability Scale; 
IRT=interactive response technology; NF-L=neurofilament light chain; PEOT=premature end of treatment; PGIC=Patient Global Impressions of Change; 
PGIS=Patient Global Impressions of Severity; PK=pharmacokinetic; PRO=patient-reported outcome; RT-MRC=Rasch-built, modified-interval Medical 
Research Council scale, S=on-site visit; Scr=Screening visit; V=visit 
Note: All assessments are to be completed in the order specified in the protocol or laboratory manual if possible. The ICF should be completed before any 
assessment. The PROs should be conducted in the order specified in Section 10.4 immediately after ICF (where applicable). The laboratory manual will 
provide further guidance on the order of sample collection. 
a The 52-week duration can be shortened or extended in view of the availability of an access program. 
aa Assessment to be performed before all other assessments at each visit except ICF. 
b Only for study participants with a gap period between the parent study and entry in CIDP04. 
c All visits have a ±2-day window. Minimum time between doses must be at least 5 days. 
d The following visits can be performed by a healthcare professional visiting the study participant at his/her home: Visits 11 to 13, 15 to 17, 19 to 21, and 23 to 
25 (Weeks 10 to 12, 14 to 16, 18 to 20, and 22 to 24). Visits in Treatment Period Part 2 will follow the same pattern: one site visit followed by 3 visits 


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conducted at the participant 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 8.2). 
e Will not be repeated at Visit 2 if performed at Visit 1. 
f If entry in CIDP04 (Visit 2) is done on the same day as the last visit of the parent study and in case assessment was performed at last visit of the parent study, 
data from the parent study will be used and corresponding assessments will not be repeated in CIDP04.    
g During site Visits 2, 3, and 4, vital signs will be measured prior to IMP administration, at the end of the infusion, and 2 and 4h after the end of the infusion. 
From Visit 5 until Visit 25, vital signs will be measured predose, at the end of the infusion and 2h after the end of the infusion only. During Treatment Period 
Part 2, vital signs will be measured predose, at the end of the infusion. At nondosing visits, vital signs need only be taken once during the visit. For study 
participants requiring additional assessments due to AEs (see Table 5-2), additional vital sign measurements may be taken based on the timing of the 
assessments. 
h Body weight is collected every 6 months during Treatment Period Part 2 starting at first visit in this period and will be used to select the correct volume for 
dose infusion from these timepoints. 
i A full C-SSRS assessment will be performed only when the study participant has a positive response to the suicidal ideation query. If a study participant has 
active suicidal ideation as confirmed by the answer “Yes” to Question 4 or Question 5 of the C-SSRS assessments, the study participant will be excluded or 
withdrawn from the study and immediately referred to a Mental Healthcare Professional. 
j Only performed every 24 weeks. 
k Not required for study participants who had a Screening Visit. 
l The full neurological examination includes a fundoscopy. In addition to the FV, a full neurological examination should be performed for any study participant 
who experiences severe headache (refer to Section 5.2.1 and Table 5-2). 
m Assessment to be performed every 12 weeks (Weeks 25, 37, 49 and 61).  
n Serology includes hepatitis C virus (HCV)-antibodies (Ab)+, hepatitis B virus antibodies (HBsAg and HBcAb), human immunodeficiency virus antibodies 
(HIV1 and HIV2). 
o In case of immediate entry from the parent study, the serum pregnancy test from the parent study should not be older than 5 weeks prior to entry into this 
CIDP04 study and the urine pregnancy test done at Visit 2 must both be negative before dosing. In case of gap period between the parent study and entry in 
CIDP04, a serum test will be performed at Visit 1 (Screening). Pregnancy testing will consist of urine testing at dosing visits during the Treatment Period Part 
1, monthly urine testing during Treatment Period Part 2 and at each visit during the Observation Period for women of childbearing potential. A positive urine 
pregnancy test must be confirmed using a serum pregnancy test. Note that the final urine pregnancy test of the study should be no longer than 90 days after the 
final dose of IMP.  
p The IGRA test will be performed in a central laboratory.  
q Tuberculosis signs and symptoms Questionnaire must be done at least every 12 weeks. 
r Self-administration may be performed by study participant under the supervision of the home nurse after full training. 
s Trough PK samples should be taken for all study participants receiving rozanolixizumab. At Visits 2, 4, and 8, PK samples should be taken predose and 4h 
postdose for all study participants. At Visits 14, 18, 22 and quarterly at site visits during Treatment Period Part 2, a predose sample only should be taken. At 
Visit 26, samples to be taken once during the visit.  
t Does not apply to Visit 10. 
u Serum complement (C3, C4), plasma complement (C3a, C5a), and serum cytokines should be taken predose at Visit 2 for all study participants. Samples should 
be taken predose and 4h postdose for all study participants at other scheduled visits (Visits 3, 8, 10, 14, 18, 22 and at first (Week 25) and last visit (Week 73) of 
Treatment Period Part 2); if an infusion reaction occurs within the first 2h, refer to Section 5.2.1 and Table 5-2. 


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v Only at Visit 14. 
w In case IgG levels are below 2 and the study participant does not qualify for a withdrawal, IgG levels will be monitored weekly. 
x Assessments to be performed only at the first visit after Visit 25 (ie, 1st visit of Part 2). Visit 29/FV should not performed after Treatment Period 2. 
y Only performed at PEOT visit when starting Observation Period directly after Treatment Period Part 1 
z At Visits 2, 3, 8, 10, 14, 18, and 22, samples for exploratory biomarkers should be taken predose. See Table 5-2 for sampling in case of AE of interest. 
 
 


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5.2.1 
Additional study assessments 
In addition to those detailed in Table 5-1, the assessments in Table 5-2 may be required in case 
of infusion reactions or AEs of interest (severe headache, moderate to severe diarrhea, moderate 
to severe abdominal pain, or moderate to severe vomiting). Note that additional vital sign 
measurements may be taken based on the timing of the assessments. 


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Table 5-2: 
Additional study assessments 
Assessment 
When applicable 
For study participants who experience infusion reactions (excluding local injection site reactions): 
Complements 
and cytokines 
Samples required for all study participants are detailed in the schedule of study 
assessments (Table 5-1). 
In study participants who experience an infusion reaction within the first 2h at Visits 
2, 4, 5, and 6, samples should also be taken 2 and 4 hours postdose (see Section 9.1.9 
and Section 18.1). 
In study participants who experience an infusion reaction within 2 hours at Visits 3, 8, 
10, 14, 18, 22 and “S” visits during Treatment Period Part 2, samples should be taken 
2 hours postdose (see Section 9.1.9 and Section 18.1). 
For study participants who experience an AE of interest, including severe headache, moderate to 
severe diarrhea, moderate to severe abdominal pain, or moderate to severe vomiting: 
If the AE is initially reported at a home visit, the study participant should be reviewed at the study site as 
soon as is practically possible for further investigation. 
Exploratory 
biomarkers 
Samples required for all study participants are detailed in the schedule of study 
assessments (Table 5-1). 
In study participants who experience an AE of interest (as defined in Section 9.1.1.4) 
at Visits 2, 3, 8, 10, 14, 18, 22 and “S” visits during Treatment Period Part 2, samples 
should also be taken 4 hours postdose. 
For study participants who experience severe and/or serious headache: 
Headache 
Questionnaire 
In study participants who report severe and/or serious headache, this assessment will 
be performed daily until resolution (ie, if headache becomes moderate or mild, normal 
collection of AEs should apply) (see Section 9.1.10). 
Full neurological 
examination 
Assessments required for all study participants are detailed in the schedule of study 
assessments (Table 5-1). 
In study participants who report severe and/or serious headache at the clinic visit, a 
full neurological examination (including fundoscopy) should be performed (see 
Section 9.1.10). In study participants who report a severe headache whilst at home, a 
visit to the site for the full neurological examination should be arranged for as soon as 
is practically possible. 
Other 
In study participants who report severe headache, other diagnostic procedures 
including but not limited to CT scan, MRI and/or LP for CSF collection are to be 
performed if indicated at the discretion of the investigator (see Section 9.1.10). 
For study participants who experience moderate or severe diarrhea: 
Stool sample 
assessment 
In study participants who report moderate or severe diarrhea, stool collection and 
analysis will be performed. The frequency of stool sampling will be as clinically 
indicated in the opinion of the investigator. Analysis of stool samples will be 
performed locally (see Section 9.1.11). 
If the moderate or severe diarrhea is initially reported at a home visit or during a 
telephone call, the study participant should be reviewed at the study site as soon as 
practically possible. 


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Table 5-2: 
Additional study assessments 
Assessment 
When applicable 
AE=adverse event; CSF=cerebral spinal fluid; CT=computed tomography; LP=lumbar puncture; MRI=magnetic 
resonance imaging 
5.3 
Rationale for study design and selection of dose 
This OLE study will provide study participants with CIDP who participated in the parent study 
(eg, CIDP01) the opportunity to have continued access to rozanolixizumab. CIDP04 will assess 
the safety, tolerability, and efficacy of a longer-term treatment with rozanolixizumab in these 
study participants. 
The initial dose of rozanolixizumab will be based on the dose the study participant has received 
at the completion of the parent study (eg, CIDP01). For study participants who were known to be 
on placebo in parent study, a starting dose of 
 will be used. The dose may be reduced to 
 is not tolerable (eg, severe headache, Section 9.1.10), or in case of IgG 
levels are <1g/L (see Section 6.4.1.3). The dose and regimen of IMP to be used in the current 
study (rozanolixizumab 
 sc) is based on results from the FIH study UP0018, alongside 
the safety data from the MG and ITP studies (MG0002 and TP0001). CIDP04, similar to the 
parent study, CIDP01, will utilize a liquid formulation at 
 
6 
SELECTION AND WITHDRAWAL OF STUDY 
PARTICIPANTS 
6.1 
Inclusion criteria  
To be eligible to participate in this study, all of the following criteria must be met: 
1. An Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved written 
Informed Consent form (ICF) is signed and dated by the study participant. 
2. Study participant who has completed one of the previous rozanolixizumab study(ies) that 
allow access to the present study. 
For example, for study participants coming from CIDP01 the following criteria will have to 
be met: 
 Study participant has completed the Treatment Period of the parent study with no relapse 
of CIDP disease and has chosen to enter this OLE study directly upon completion of the 
Treatment Period of the parent study, or 
 Study participant has experienced a relapse of CIDP during the parent study (either 
during the Treatment Period or the treatment free Observation Period), has been 
successfully rescued and stabilized with eg, IVIg based on assessment of the investigator 
(maximum of 3 months of stabilization treatment), and has chosen to enter this OLE 
study. Study participants who already relapsed during the Treatment Period of CIDP01 
must have been assigned to placebo treatment during that period in order to be able to 
qualify for enrollment into CIDP04. 


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3. 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, and has been 
compliant with the parent study assessments. 
4. Female study participants of child-bearing potential must have the results of a negative serum 
pregnancy test from either the parent study (not older than 5 weeks) or the Screening Visit 
available at the CIDP04 Baseline (Visit 2), and a urine pregnancy test must be negative when 
study participant enters CIDP04 and prior to further dosing at each study visit thereafter. 
Female study participants of childbearing potential must agree to use a highly effective 
method of birth control, during the study and for a period of 3 months after their final dose of 
IMP. Highly effective forms of birth control are methods that achieve a failure rate of less 
than 1% per year when used consistently and correctly. According to the International 
Council for Harmonisation (ICH) M3 R2, highly effective methods of birth control include: 
 Combined (estrogen- and progesterone-containing) hormonal contraception (oral, 
implant, injectable) associated with inhibition of ovulation (which must be stable for at 
least 1 full month prior to Screening [Visit 1], or must have remained stable during the 
parent study for study participants entering CIDP04 without a Screening Visit, and 
should remain stable during the study). 
 Progesterone-only hormonal contraceptives (oral, implant, injectable) associated with 
inhibition of ovulation (which must be stable for at least 1 full month prior to Screening 
[Visit 1], or must have remained stable during the parent study for study participants 
entering CIDP04 without a Screening Visit, and should remain stable during the study). 
 Progesterone-releasing intrauterine systems or the TCu 380A intrauterine device. 
 Vasectomized partner (provided sole partner and partner has medical proof of surgical 
success). 
 True heterosexual sexual abstinence is an acceptable form of contraception when this is 
in line with the preferred and usual lifestyle of the person. Periodic abstinence 
(eg, calendar, ovulation, symptothermal, postovulation methods), declaration of 
abstinence for the duration of the study, and withdrawal are not acceptable methods of 
contraception. 
 Women not agreeing to use birth control must be of nonchildbearing potential, defined as 
being: 
 Postmenopausal (for at least 2 years before the Screening Visit of CIDP04 or the parent 
study if no Screening Visit is scheduled in CIDP04), verified by serum 
follicle-stimulating hormone level >40mIU/mL at the Screening Visit, or 
 Permanently sterilized (eg, bilateral tubal occlusion, hysterectomy, bilateral 
salpingectomy), or 
 Congenitally sterile 
5. Male study participants with a partner of childbearing potential must be willing to use a 
condom when sexually active during the study and for 3 months after the final administration 
of IMP. In addition, the female partner of childbearing potential of a male study participant 


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must be willing to use a highly effective method of contraception (as above), during the study 
period and for 3 months after the final administration of IMP. Sperm donation is not 
permitted during the study and for 3 months after final administration of IMP. 
6. For study participants entering CIDP04 after a gap period since the parent study: Study 
participant is on a stable dosage (not more than ±20% deviation) for SCIg or IVIg and a fixed 
interval (eg, once weekly ±2 days for SCIg or every 2 to 6 weeks ±5 days IVIg, respectively) 
for stability in functioning between dosing. 
6.2 
Exclusion criteria 
Study participants are not permitted to enroll in the study if any of the following criteria is met: 
1. Study participant is currently participating in another study of an IMP (or a medical device). 
2. Female study participant who is pregnant or lactating, or planning to become pregnant during 
the study and until 2 months following completion of the study. 
3. Study participant has any medical (acute or chronic illness) or psychiatric condition that, in 
the opinion of the investigator, could harm the study participant or would compromise the 
study participant’s ability to participate in this study. 
4. Study participant has 12-lead ECG with abnormalities considered to be clinically significant 
upon medical review. 
5. Study participant has renal impairment, defined as serum creatinine level of ≥1.4mg/dL for 
females and ≥1.5mg/dL for males. 
6. Study participant has an absolute neutrophil count <1500/mm3 (1.5x109/L). 
7. Study participant has >2x upper limit of normal (ULN) of any of the following: alanine 
aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP), 
or >ULN total bilirubin (≥1.5xULN total bilirubin if known Gilbert’s syndrome). If study 
participant has elevations only in total bilirubin that are >ULN and <1.5xULN, fractionate 
bilirubin to identify possible undiagnosed Gilbert’s syndrome (ie, direct bilirubin <35%). 
For 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 
understood and recorded in the electronic Case Report form (eCRF). 
If study participant has >ULN ALT, AST, or ALP that does not meet the exclusion limit at 
the time of entry in CIDP04 (either Screening [Visit1] or Baseline [Visit 2]), repeat the tests, 
if possible, prior to dosing to ensure there is no further ongoing clinically relevant increase. 
In case of a clinically relevant increase, inclusion of the study participant must be discussed 
with the Medical Monitor. 
Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit may be repeated 
once for confirmation. This applies to study participants who had a Screening Visit and 
includes rescreening. 
8. Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, abscess). 


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9. Study participant has active suicidal ideation as indicated by a positive response (“Yes”) to 
either Question 4 or Question 5 of the “Since Last Visit” version of the Columbia Suicide 
Severity Rating Scale (C-SSRS) at Baseline Visit (Visit 2), 
or 
Study participant has a lifetime history of suicide attempt (including an active attempt, 
interrupted attempt, or aborted attempt), or has suicidal ideation in the past 6 months as 
indicated by a positive response (“Yes”) to either Question 4 or Question 5 on the Screening 
C-SSRS. 
The study participant should be referred immediately to a Mental Healthcare Professional 
and must be withdrawn from the study. 
10. Study participant has a known hypersensitivity to any components of rozanolixizumab. 
11. Study participant intends to have a live vaccination during the course of the study or within 7 
weeks following the final dose of rozanolixizumab. 
12. Study participant has an ongoing serious adverse event (SAE) or a medical condition in the 
parent study that the investigator considers to put the study participant at a significantly 
increased risk of participating in CIDP04. 
13. Study participant has any planned elective surgery due to occur during the study dosing 
period which in the opinion of the investigator could interfere with study procedures. 
6.2.1 
Additional exclusion criteria for study participants with a gap period 
between the parent study and entry in CIDP04 
14. Study participant has a current diagnosis or has a history of Type 1 or Type 2 diabetes 
mellitus and/or hemoglobin A1c level >6.0%. 
15. Study participant with IgM paraproteinemia. 
16. Study participant has known IgM-mediated neuropathy (eg, multifocal motor neuropathy). 
17. Study participant has clinical or known evidence of associated systemic diseases that might 
cause neuropathy, including but not limited to connective tissue disease, Lyme disease, 
Castleman's disease and systemic lupus erythematosus, malignant plasma cell dysplasia, or 
treatment with agents that might lead to neuropathy (eg, amiodarone therapy). 
18. Study participant on an average dose less than 0.4g IgG/kg/month over the past 4 months. 
19. Study participant has a history of clinically relevant ongoing chronic infections including but 
not limited to human immunodeficiency virus (HIV), hepatitis B, hepatitis C, or is tested 
positive for human immunodeficiency virus antibody 1 (HIV1), human immunodeficiency 
virus antibody 2 (HIV2), hepatitis B surface antigen, hepatitis B core antibody without 
hepatitis B surface antibody test positive, or hepatitis C antibody at the Screening Visit. 
20. Study participant with known tuberculosis (TB) infection, at high risk of acquiring TB 
infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculosis 
mycobacteria (NTMB) are excluded. 


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21. Study participant has a history of alcohol use disorder or other substance use disorder (as per 
Diagnostic and Statistical Manual of Mental Disorders-5 [American Psychiatric Association, 
2013]) within 12 months of Screening Visit. 
22. Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, abscess) or 
has had a serious infection (resulting in hospitalization or requiring parenteral antibiotic 
treatment) within 4 weeks prior to the first dose of IMP. 
23. Study participant has active neoplastic disease or history of neoplastic disease within 5 years 
of Screening Visit (except for basal or squamous cell carcinoma of the skin or carcinoma in 
situ of the uterine cervix that has been definitively treated with SOC approaches). 
24. Study participant has received a live vaccination within 8 weeks prior to the Baseline Visit. 
25. Study participant has received any experimental biological agent (except rozanolixizumab) 
within or outside of a clinical study in the past 3 months or within 5 half-lives prior to 
Baseline (whichever is longer) (refer to Table 6-1). 
26. Study participant has had prior treatment with rituximab, ofatumumab, or ocrelizumab in the 
6 months prior to the Baseline Visit or study participant has had prior treatment with 
rituximab, ofatumumab, or ocrelizumab in the 12 months prior to Baseline and B cells are 
not within the normal range. 
27. Study participant has been treated with immunosuppressants, biologics (except for 
rozanolixizumab), and other therapies in the recent timeframe as detailed in Table 6-1 OR 
has been on permitted medications detailed in Table 7-3, but has not been on stable dosing 
regimens of those medications as detailed in Table 7-3. 


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Table 6-1: 
Time restrictions required prior to Baseline Visit (Visit 2) for 
immunosuppressants, biologics, and other therapies 
Generic name 
Time restrictions required prior to Baseline Visit 
(regardless of route) 
Immunosuppressants 
Cyclophosphamide 
Pimecrolimus 
Vinca alkaloids (vincristine, vinblastine) 
Biologics (mAbs and fusion proteins) 
Abatacept (CTLA 4-Ig) 
Belimumab 
Golimumab 
Natalizumab 
Ofatumumab 
Rituximab 
Ocrelizumab 
Atacicept (TACI-Ig) 
Veltuzumab 
Other biologics 
Others 
Rigerimod 
PLEX 
CTLA 4-Ig=cytotoxic T lymphocyte-associated molecule-4 immunoglobulin; mAb=monoclonal antibody; 
PLEX=plasma exchange; TACI-Ig=transmembrane activator and calcium modulator and cyclophilin ligand 
interactor-immunoglobulin 
NOTE: For other immunotherapies not included in the table, consult with Medical Monitor prior to enrollment. 
6.3 
Rescreening of study participants (only for study participants 
who had a Screening Visit) 
For study participants otherwise fully eligible but not able to enter the study as planned for 
nonclinical reasons, rescreening may be allowed at the discretion of the investigator, following 
discussion with the sponsor's Medical Monitor and/or Study Physician. 
If a study participant has 1 isolated test result in the exclusionary range that is deemed not being 
clinically significant by the investigator, retesting may be allowed at the discretion of the 
investigator, following discussion with the sponsor's Medical Monitor and/or Study Physician. If 
the normalization of the test result occurs within the Screening Period, then no other screening 
procedures need to be repeated. 


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6.4 
Withdrawal criteria 
Study participants are free to withdraw from the study at any time, without prejudice to their 
continued care. 
Study participants MUST be withdrawn from the study if any of the following events occur: 
1. Study participant withdraws his/her consent. 
2. Study participant becomes pregnant during the study, as confirmed by a positive pregnancy 
test. 
Study participants must permanently discontinue IMP if any of the following events occur: 
1. Study participant develops an illness that would interfere with his/her continued 
participation. 
 Study participant has an AE of severe infusion reaction requiring corticosteroid and/or 
epinephrine therapy (see Section 9.1.9). 
 Study participant has an AE of severe anaphylactic reaction requiring corticosteroid and/or 
epinephrine therapy. 
 Study participant has a TB test that is confirmed positive or any further evidence suggestive 
of potential TB infection (eg, exposure) and further examinations result in a diagnosis of 
active TB or LTBI (refer to Section 9.3.6 for further details and instructions). 
 If an NTMBI is identified during a study, the same withdrawal procedures as those used for 
an active TB infection identified during the study should be followed. 
2. Study participant has active suicidal ideation as indicated by a positive response (Yes) to 
either Question 4 or Question 5 of the “Since Last Visit” version of the C-SSRS. The study 
participant should be referred immediately to a Mental Healthcare Professional. 
3. The sponsor or a regulatory agency requests withdrawal of the study participant. 
4. Study participant needs or takes PLEX, dexamethasone, or rituximab. 
5. Study participant is treated with rescue medication and/or relapses during the Treatment 
Period (refer to Section 7.8.3). 
Study participants may be discontinued from IMP at the discretion of the investigator, Medical 
Monitor, and Study Physician if any of the following events occur: 
1. Study participant takes prohibited concomitant medications during the Treatment Period as 
defined in this protocol (refer to Section 7.8.2). 
2. Study participant experiences a severe AE of headache that is considered related to the IMP 
in the opinion of the investigator (Section 9.1.10). Following an event of a severe headache, a 
study participant may continue participation in the study if the study participant is willing to 
do so and the investigator, Medical Monitor, and Study Physician agree that the study 
participant’s continuation in the study poses no significant risk for the study participant. The 
use of symptomatic headache treatment is allowed at the discretion of the investigator. The 
IMP dose can be reduced if the headache persists despite symptomatic treatment. The IMP 


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dose may be reduced to 
(refer to Section 9.1.10 for details on the management of 
headaches and Section 7.2 for treatment to be administered). 
3. Study participant experiences severe AE of gastrointestinal disturbance that is considered 
related to the IMP in the opinion of the investigator. Following an event of a severe 
gastrointestinal disturbance, a study participant may continue in the study if they are willing 
to do so and the investigator and Medical Monitor agree that the study participant’s 
continuation in the study poses no significant risk. 
Study participants may be withdrawn from the study at the discretion of the investigator, 
Medical Monitor, and Study Physician if any of the following events occur: 
1. Study participant is noncompliant with the study procedures or medications in the opinion of 
the investigator. 
2. In case of intake of prohibited concomitant medication other than IVIg, SCIg, PLEX, 
dexamethasone or rituximab, the investigator will (where possible) discuss with the Medical 
Monitor and/or sponsor Study Physician and a decision will be made whether the study 
participant should discontinue IMP. 
Study participants who withdraw from the study or discontinue IMP during any of the Treatment 
Periods (Part 1 or Part 2) should complete the assessments outlined for Visit 26/premature end of 
treatment (PEOT) (see Table 5-1 and Section 8.4.1). Study participants who withdraw from the 
study will be encouraged to return to the clinic for all visits during the Observation Period 
(which should occur every 2 weeks) following their final dose of IMP) and the Final Visit (FV) 
(scheduled 8 weeks after final dose of IMP). The study participants can return to their SOC 
during the Observation Period. 
Investigators should attempt to obtain information on study participants in the case of 
withdrawal. 
For study participants considered as lost to follow up, the investigator should try (at least 
1 phone call and 1 written message to the study participant), and document his/her effort (date 
and summary of the phone call and copy of the written message in the source documents), to 
complete the final evaluation. All results of these evaluations and observations, together with a 
narrative description of the reason(s) for removing the study participant, must be recorded in the 
source documents. The eCRF must document the primary reason for withdrawal or 
discontinuation, with the reason and date for withdrawal or discontinuation. 
Investigators should contact the Medical Monitor, whenever possible, to discuss the withdrawal 
of a study participant in advance. 
Study participants who withdraw from the study will not be replaced. 
6.4.1 
Temporary discontinuation of IMP 
6.4.1.1 
Serious infection 
Treatment must be discontinued if study participant has a serious infective episode requiring eg, 
hospitalization, or iv antibiotic therapy (including but not limited to bacteremia or sepsis, 
bacterial meningitis, osteomyelitis or septic arthritis, bacterial pneumonia, or visceral abscess). 


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6.4.1.2 
Hypogammaglobulinemia and non-serious persisting or recurrent 
infection 
In the event of a non-serious infection, the Benefit-Risk of continuing treatment with IMP must 
be carefully evaluated by the Investigator in collaboration with the Medical Monitor and the 
Study Physician. Treatment may be temporarily discontinued for the study participant who 
develops a non-serious persisting or recurrent infection with a serum total IgG levels between 
≥1g/L and <2g/L (see Section 7.9). Upon resolution of infection and the IgG levels returning to 
≥2g/L, the study participant may be allowed to restart treatment with IMP (at the same dose for 
study participants in Treatment Period Part 1, or at the same dose or at the reduced dose of 
for study participants in Treatment Period Part 2) at the discretion of the investigator and 
in agreement with Medical Monitor and Study Physician, if the study participant is willing and if 
the benefit-risk remains favorable for the study participant. 
6.4.1.3 
Hypogammaglobulinemia irrespective of infection 
Treatment will be temporarily discontinued for the study participant who develops an event of 
hypogammaglobulinemia with a serum total IgG <1g/L (see Section 7.9) irrespective of 
infection. When the IgG level reaches ≥2g/L, the study participant may be allowed to restart 
treatment with IMP (at the same dose for study participants in Treatment Period Part 1, or at the 
same dose or at the reduced dose of 
for study participants in Treatment Period Part 2) at 
the discretion of the investigator and in agreement with Medical Monitor and Study Physician, if 
the study participant is willing and if the benefit-risk remains favorable for the study participant. 
6.4.2 
Potential drug-induced liver injury IMP discontinuation criteria 
Study participants with potential drug-induced liver injury (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. 
The PDILI criteria below require immediate and permanent discontinuation of IMP: 
 Study participants with either of the following: 
 ALT or AST ≥5xULN 
 ALT or AST ≥3xULN and coexisting total bilirubin ≥2xULN 
The PDILI criterion below requires immediate discontinuation of IMP: 
 Study participants with ALT or AST ≥3xULN who exhibit temporally associated symptoms 
of hepatitis or hypersensitivity. Hepatitis symptoms include fatigue, nausea, vomiting, right 
upper quadrant pain or tenderness. Hypersensitivity symptoms include fever (without clear 
alternative cause), rash, or eosinophilia (ie, >5%). 
If a nondrug-related cause for the symptoms can be confirmed, these study participants may 
resume IMP administration after discussion with the responsible UCB physician, but only when 
the requirements for rechallenge with IMP as provided in Section 9.2.1.2.1 are followed. 


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The PDILI criterion below allows for study participants to continue on IMP at the discretion of 
the investigator. 
 Study participants with ALT or AST ≥3xULN (and ≥2x Baseline) and <5xULN, total 
bilirubin <2xULN, and no eosinophilia (ie, ≤5%), with no fever, rash, or symptoms of 
hepatitis (eg, fatigue, nausea, vomiting, right upper quadrant pain or tenderness). 
Evaluation of PDILI must be initiated as described in Section 9.2.1. If study participants are 
unable to comply with the applicable monitoring schedule, IMP must be discontinued 
immediately. 
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 study 
participant withdrawal (if applicable), must be recorded in the source documents. The eCRF 
must document the primary reason for IMP discontinuation. 
7 
STUDY TREATMENT 
7.1 
Description of investigational medicinal product 
 
 
 
 
Details of the IMP and its specifications are provided in the IMP Handling Manual. 
7.2 
Treatment to be administered 
The initial dose administered will be based on the dose at the time of completion of the 
Treatment Period from the parent study (eg, CIDP01). For subjects who had a gap period 
between the parent study (eg, CIDP01) and CIDP04, a starting dose of 
 will be used (see 
Table 7-1). The dose used in the study may be reduced based on individual tolerability to 
 
(see Table 7-2) (eg, severe headache) but the maximum dose used will be 
The dose 
used during Treatment Period Part 2 may be reduced to 
 in case of recurrent low observed 
IgG levels for a participant. Treatment temporary halt may also be implemented in some 
situation (see Section 6.4.1). 
Eligible study participants will receive rozanolixizumab 
by sc infusion for 24 weeks 
during Treatment Period Part 1 (from Visit 2 to Visit 25) and during an optional additional 
52 weeks during Treatment Period Part 2. Treatment Period 2 may be shortened depending on 
availability of an Access Program or equivalent.  
Study participants may choose to self-administer the IMP during Treatment Period Part 2 under 
supervision of a nurse and after having received appropriate training.  
 


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Table 7-1: 
IMP doses to be administered (equivalent to approximately 
) 
by body weight 
Body weight ranges 
IMP doses to be administered (equivalent to 
approximately 
a) 
IMP volume to be 
administered 
≥40 to <49kg 
 
 
≥49 to <63kg 
 
 
≥63 to <77kg 
 
 
≥77 to <91kg 
 
 
≥91 to <105kg 
 
 
≥105 to <119kg 
 
 
≥119 to <133kg 
 
 
≥133 to <147kg 
 
 
≥147 to <161kg 
 
 
≥161 to 170kg 
 
 
IMP= investigational medicinal product 
a  Doses administered will be ±10% of the intended dose, except for study participants with a body weight of 47 to 
50kg and 63kg. 
Table 7-2: 
IMP doses to be administered (equivalent to approximately 
) 
by body weight 
Body weight ranges 
IMP doses to be administered (equivalent to 
approximately 
) 
IMP volume to be 
administered 
≥40 to <49kg 
 
 
≥49 to <69kg 
 
 
≥69 to <89kg 
 
 
≥89 to <109kg 
 
 
≥109 to <129kg 
 
 
≥129 to <149kg 
 
 
≥149 to <169kg 
 
 
≥169 to 170kg 
 
 
IMP= investigational medicinal product  
The IMP will be administered at the clinic or in a home setting (refer to Table 5-1for details). 
Home administration is study participant to a set of conditions (see Section 8.2) to ensure study 
participant safety. 
The IMP will be administered as a sc infusion using a syringe pump. 
 
 
 


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 The chronology of these events should be recorded accurately in 
the source data and eCRF. 
The study participant’s body weight at entry into CIDP04 (either at Screening [Visit 1] or 
Baseline [Visit 2]) will be used to select the volume for dose infusion throughout Treatment 
Period Part 1. Body weight will be reevaluated at start of the Treatment Period Part 2, and every 
6 months, and will be used to select the correct volume for dose infusion from these timepoints. 
The exact procedure for dose preparation according to body weight will be provided in the IMP 
Handling Manual. 
7.3 
Packaging 
 
 
Details of the IMP and its specifications are provided in the IMP Handling Manual. 
7.4 
Labeling 
Clinical drug supplies will be labeled in accordance with the current ICH guidelines on Good 
Clinical Practice (GCP) and Good Manufacturing Practice and will include any locally required 
statements. If necessary, labels will be translated into the local language. 
7.5 
Handling and storage requirements 
The investigator (or designee) is responsible for the safe and proper storage of IMP at the site. 
Investigational medicinal product stored by the investigator is to be kept in a secured area with 
limited access according to the storage conditions mentioned on the label. 
Appropriate storage conditions must be ensured either by controlling the temperature (eg, room, 
refrigeration unit) or by completion of a temperature log in accordance with local requirements 
on a regular basis (eg, every work day), showing actual and minimum/maximum temperatures 
reached over the time interval. 
In case an out-of-range temperature is noted, it must be immediately reported as per instructions 
contained in the IMP Handling Manual. 
The investigator (or designee) will instruct the study participant to store the IMP following the 
instructions on the label. 
7.6 
Drug accountability  
A Drug Accountability form will be used to record IMP dispensing and return information on a 
by-study 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, 


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disposed of at the study site, or returned to the sponsor or designee must also be recorded on the 
appropriate forms. All supplies and pharmacy documentation must be made available throughout 
the study for UCB (or designee) to review. 
The investigator (or designee) is responsible for retaining all used, unused, and partially used 
containers of IMP until returned or destroyed (all details are provided in the IMP Handling 
Manual). 
The investigator may assign some of the investigator’s duties for drug accountability at the study 
site to an appropriate pharmacist/designee. 
The investigator must ensure that the 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) or returned to UCB (or designee). Investigational medicinal product intended 
for the study cannot be used for any other purpose than that described in this protocol. 
7.7 
Procedures for monitoring study participant compliance 
Study participant compliance will be ensured by the administration of IMP by sc infusion by 
designated site personnel. Drug accountability must be recorded on the Drug Accountability 
form. 
7.8 
Concomitant medications/treatments 
7.8.1 
Permitted concomitant treatments (medications and therapies) 
Concomitant treatments/medications permitted at a stable dose during the course of the study are 
detailed in Table 7-3. 


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Table 7-3: 
Permitted concomitant medications 
Permitted medication 
Allowed dosages 
Comment 
Oral corticosteroids 
(prednisolone) 
≤30mg/day 
Stable 
 prior to entry 
Methotrexate 
≤30mg/week 
 
stable dose 
 prior to entry 
Mycophenolate mofetil 
≤3g/day 
 
stable dose 
 prior to entry 
Cyclosporine a 
≤5mg/kg/day for unmodified 
≤4mg/kg/day for modified 
(microemulsion) 
 
stable dose 
 prior to entry 
Azathioprine 
≤3mg/kg/day 
 
stable dose 
 prior to entry 
Tacrolimus b 
≤5mg/day 
 
stable dose 
 prior to entry 
 a Doses higher than listed are permissible if plasma trough level is ≤300ng/L. 
 b If the total daily weight-based dose is >5mg, then a plasma trough level should be checked to ensure study 
participant is not above the recommended therapeutic range. 
The use of cannabidiols and medicinal marijuana (prescribed by a physician) is also permitted. 
When applicable, the study participant must be on a stable dose of cannabidiols and/or medicinal 
marijuana for 
 prior to Screening Visit and remain stable for the duration of the study. 
7.8.2 
Prohibited concomitant treatments (medications and therapies) 
The following concomitant medications are prohibited during the Treatment Period: 
 IVIg and SCIg (refer to Section 7.8.3) 
 All biologics including rituximab 
 Cyclophosphamide 
 Pimecrolimus 
 IPP-201101 (Lupuzor™) 
 Systemic dexamethasone 
 PLEX 
 IA 
If a study participant requires or takes IVIg, SCIg, PLEX, dexamethasone, or rituximab, they 
must be discontinued from IMP. In all other cases of intake of prohibited concomitant 
medication, the investigator will (where possible) discuss with the Medical Monitor and/or 
Sponsor Study Physician and a decision will be made whether the study participant should 
discontinue IMP (see Section 6.4). 


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If a study participant is discontinued from IMP they should be encouraged to complete Visit 
26/PEOT and complete the Observation Period (see Table 5-1 and Section 6.4). 
7.8.3 
Rescue medication 
If, at any time during this OLE study, a study participant relapses according to the predefined 
criteria for relapse as specified in Section 4.2.1 using the study participant’s score on iRODS, 
INCAT, or maximum grip strength as assessed by the site personnel and supported by the 
medical judgement of the investigator, then rescue therapy must be considered and the study 
participant withdrawn from IMP (see Section 6.4). The study participant will return to the SOC 
Ig treatment (eg, IVIg of 2g/kg) at the investigator's discretion.  
7.9 
Blinding 
Not applicable; this is an open-label study. However, in order to maintain the blind of the parent 
study (eg, CIDP01) study participant’s IgG levels will be blinded to the investigator for the 
initial 4 weeks treatment of every study participant in CIDP04. The duration of 4 weeks may be 
adjusted during the course of the study based on results from CIDP01 and CIDP04. 
7.10 
Randomization and numbering of study participants 
Randomization is not applicable in the current study. Study participants will be identified with 
the study participant number they received in the parent study. 
An interactive response technology (IRT) will generate individual assignments for study 
participants, as appropriate, according to the visit schedule and the dose the study participant 
should receive. 
The study participant number will be required in all communication between the investigator or 
designee and the IRT regarding a particular study participant. 
8 
STUDY PROCEDURES BY VISIT 
Study visits should preferably be conducted at the same time of the day throughout the study. 
Details of the study assessments to be performed at specific time points prior to and after IMP 
administration are provided in Table 5-1. An outline of all assessments performed is provided in 
the following sections. The ICF should be completed before any assessment. The PROs should 
be conducted in the order specified in Section 10.4 immediately after ICF is completed (where 
applicable). The laboratory manual will provide further guidance on the order of sample 
collection. Additional assessments that may be required in case of infusion reactions or AEs of 
interest (severe headache, moderate to severe diarrhea, moderate to severe abdominal pain, or 
moderate to severe vomiting) are provided in Table 5-2; these assessments are not included in 
the by-visit study procedures within this section.  
In case an on-site visit cannot be performed, the subsequent visit will be performed at the site 
instead of at home, and all safety assessments that should have been completed at the missed 
visit will be performed.  


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8.1 
Screening, Visit 1 (Weeks -5 to -2) 
The Screening Visit is only for study participants with a gap period between the parent study and 
entry in CIDP04. 
The following procedures will be performed at the Screening Visit (or as close as possible to the 
Screening date): 
 Informed consent process including obtaining written informed consent from the study 
participant 
 iRODS assessment 
 Query for suicidality (C-SSRS) 
 Tuberculosis Signs and Symptoms Questionnaire 
 Record study participant demographics 
 Verify (to the extent possible with available information at Screening, Visit 1) that study 
participant fulfills all the inclusion criteria and none of the exclusion criteria 
 Medical history update 
 Prior and concomitant medications  
 Concomitant medical procedures 
 Vital signs (taken once during the visit) 
 Body weight (Screening weight will be used for calculation of the infusion dose) 
 Recording of AEs 
 12-lead ECG 
 Full physical examination 
 Full neurological examination 
 Contact IRT 
 Blood sample (the Laboratory Manual will provide guidance on the order of sample 
collection) for: 
 Clinical laboratory tests (ie, hematology, clinical chemistry, serology [including 
hepatitis C and hepatitis B virus-antibodies, HIV1, and HIV2], and interferon gamma 
release assay [IGRA] for active and latent TB) 
 Serum pregnancy test for women of childbearing potential 
 Immunoglobulins (total IgG and IgG subclasses) 
 Vaccination-specific antibody titers (tetanus and influenza Type A) 
 Urine sample for urinalysis 
 INCAT assessment 


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 Assessment of grip strength by site personnel 
 RT-MRC assessment 
 Provide an appointment to the next visit according to the following guidance: 
 For study participants on an IVIg 3- to 6-week regimen, Baseline Visit should occur 1 
week before the next planned IVIg dose (according to the Baseline IVIg regimen). 
However, if that specific date is not possible, the Baseline Visit (Visit 2) could be up to 1 
week after that date. 
 Study participants on a 2-week IVIg treatment regimen will continue on their regular 
schedule for 1 further IVIg treatment until Visit 2. Visit 2 will occur 1 week before the 
planned IVIg dose. However, if that specific date is not possible, Visit 2 could be up to 1 
week after that date. 
 Study participants on a weekly SCIg treatment regimen will continue on their regular 
schedule for at least 1 further SCIg treatment until Visit 2. Visit 2 should occur on the 
day of a planned dose (according to the Baseline SCIg regimen). However, if that specific 
date is not possible, Visit 2 should be within a window ±2 days of that date. 
This will ensure continuation of treatment so that study participants do not relapse prior to 
start of study treatment. 
8.2 
Treatment Period Part 1, Visits 2 to 25 (Weeks 1 to 24) 
During the 24-week Treatment Period, the at-home visits will be conducted by fully trained 
healthcare professional visiting the study participant at his/her home. Alternatively, these visits 
can be conducted at the site as deemed necessary by site personnel and/or study participant. 
Where dosing is done at home, the same safety monitoring schedule will be followed as if in 
clinic. The home nurse will be present during the full duration of the visit. Home visits can be 
conducted in case the following conditions are met: 
 The study participant is willing to be dosed and monitored at home over a 2-hour period by a 
home nurse. 
 The study participant has shown good acute tolerability to previous administrations of IMP 
(namely, he/she must have had no moderate or severe infusion reactions or other AEs that the 
investigator considers could increase the risk of home administration). 
 The study participant does not require specific medical supervision based on his/her medical 
history/condition. 
 The team delivering the home dose must be trained in the identification and management of 
infusion reactions and hypersensitivity and must have access to immediate treatments (eg, an 
EpiPen). 
 The study participant’s home allows rapid access to emergency treatment if required (ie, the 
study participant must not live so remotely that a reasonable arrival time of an ambulance 
could not be predicted). 
 The investigator is contactable to support the healthcare provider if needed. 


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 UCB has not requested to limit the possibility to perform home visits (eg, based on DMC 
recommendation). 
The investigator will be asked to complete a checklist confirming all criteria have been fully 
evaluated and met before the first home dosing visit can take place, and will be reconfirmed in 
case of changes to the study participant’s condition. This checklist will be shared with the UCB 
Study Physician and reviewed before IMP administration in a home setting can take place. 
8.2.1 
Visit 2, Baseline (Week 1) 
The following procedures will be performed at Visit 2 (Week 1). If study participant enters 
CIDP04 at Visit 2 and the assessment was performed at the last visit in the parent study (eg, 
CIDP01) (or at Visit 16 for NF-L), the assessment will not be repeated and will be marked as 
“not to repeat” in the list below: 
 Informed consent process including obtaining written informed consent from the study 
participant (for study participants who did not perform Screening Visit [Visit 1]) 
 PROs should be conducted in the order specified in Section 10.4: 
 iRODS assessment - to be performed before any other assessment (not to repeat) 
 CIDP PRO instrument (not to repeat) 
 Fatigue scale (not to repeat) 
 PGIS (not to repeat) 
 PGIC (not to repeat) - not required for study participants who had a Screening Visit 
 Query for suicidality (not to repeat) 
 Record study participant demographics (for study participants who did not perform 
Screening Visit [Visit 1]) 
 Verify that study participant fulfills all the inclusion criteria and none of the exclusion 
criteria (for study participants who did not perform Screening Visit [Visit 1]) 
 Withdrawal criteria assessment 
 Medical history update (for study participants who did not perform Screening Visit [Visit 1]) 
 Prior and concomitant medications (not to repeat) 
 Concomitant medical procedures (not to repeat) 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2 and 4h after the end 
of the infusion) 
 Body weight assessment (Visit 2 weight will be used for calculation of the infusion dose for 
study participants without screening visit) (not to repeat) 
 Recording of AEs (not to repeat) 
 Full physical examination (not to repeat) 
 Full neurological examination (not to repeat) 


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 12-lead ECG (not to repeat) 
 Blood sample (all samples should be taken predose unless otherwise specified; the 
Laboratory Manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology, clinical chemistry, and IGRA for active and 
latent TB) (not to repeat) 
 PK of rozanolixizumab (predose and 4h postdose) (not to repeat predose sample if 
performed at last visit in the parent study) 
 ADA (anti-rozanolixizumab antibodies) (not to repeat) 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) 
 Serum cytokines (predose) 
 Immunoglobulins (total IgG and IgG subclasses) (not to repeat) 
 IgA, IgM, IgE (not to repeat)  
 CIDP-specific auto-antibodies (not to repeat except if last visit in the parent study was 
FV) 
 NF-L (not to repeat) 
 Exploratory biomarker analysis 
 Serum pregnancy test for women of childbearing potential (not to repeat if performed 
within 5 weeks of entry in CIDP04) 
 Urine sample for urinalysis (not to repeat) 
 Urine pregnancy test for women of childbearing potential (which must be confirmed negative 
prior to study participant dosing) (not to repeat)  
 Contact IRT 
 Administer IMP 
 INCAT assessment (predose) (not to repeat) 
 Assessment of grip strength by site personnel (predose) (not to repeat) 
 RT-MRC assessment (not to repeat) (predose) 
8.2.2 
Visit 3 (Week 2) 
The following procedures will be performed at Visit 3 (Week 2): 
 iRODS assessment - to be performed before any other assessment 
 Query for suicidality (predose) 
 Withdrawal criteria assessment 
 Prior and concomitant medications 


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 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2 and 4h after the end 
of the infusion) 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 12-lead ECG 
 Contact IRT 
 Administration of IMP 
 Urine sample for urinalysis (predose) 
 Urine pregnancy test for women of childbearing potential (which must be confirmed negative 
prior to study participant dosing) 
 Blood sample (all samples should be taken predose unless otherwise specified; the 
Laboratory Manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) (predose and 
4h postdose) 
 Serum cytokines (predose and 4h postdose) 
 Immunoglobulins (total IgG and IgG subclasses) 
 IgA, IgM, IgE 
 Exploratory biomarker analysis 
 INCAT assessment 
 RT-MRC assessment 
 Assessment of grip strength by site personnel 
8.2.3 
Visit 4 (Week 3) 
The following procedures will be performed at Visit 4 (Week 3): 
 iRODS assessment - to be performed before any other assessment  
 Query for suicidality (predose) 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 


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 Vital signs (prior to IMP administration, at the end of the infusion, at 2 and 4h after the end 
of the infusion) 
 12-lead ECG 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 Contact IRT 
 Administration of IMP 
 Urine sample for urinalysis (predose) 
 Urine pregnancy test for women of childbearing potential (which must be confirmed negative 
prior to study participant dosing) 
 Blood sample (all samples should be taken predose unless otherwise specified; the laboratory 
manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 PK of rozanolixizumab (predose and 4h postdose) 
 ADA (anti-rozanolixizumab antibodies) 
 Immunoglobulins (total IgG and IgG subclasses) 
 IgA, IgM, IgE 
 INCAT assessment (predose) 
 RT-MRC assessment (predose) 
 Assessment of grip strength by site personnel 
8.2.4 
Visit 5 (Week 4) 
The following procedures will be performed at Visit 5 (Week 4): 
 iRODS assessment– to be performed before any other assessment 
 Query for suicidality (predose) 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end of the 
infusion) 
 12-lead ECG 
 Recording of AEs 


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 Brief physical examination 
 Brief neurological examination 
 Urine sample for urinalysis and urine pregnancy test 
 Blood sample (all samples should be taken predose unless otherwise specified; the laboratory 
manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 Immunoglobulins (total IgG, IgG subclasses) 
 IgA, IgM, IgE 
 Contact IRT 
 Administer IMP 
 INCAT assessment 
 RT-MRC assessment 
 Assessment of grip strength by site personnel 
8.2.5 
Visit 6 (Week 5) 
The following procedures will be performed at Visit 6 (Week 5): 
 PROs should be conducted in the order specified in Section 10.4: 
 iRODS assessment – to be performed before any other assessment 
 CIDP PRO instrument 
 Fatigue scale 
 PGIS 
 PGIC 
 Query for suicidality 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end of the 
infusion) 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 Urine sample for urinalysis and urine pregnancy test 


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 Blood sample (all samples should be taken predose unless otherwise specified; the laboratory 
manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 Immunoglobulins (total IgG and IgG subclasses) 
 IgA, IgM, IgE 
 CIDP-specific auto-antibodies 
 Contact IRT 
 Administer IMP 
 INCAT assessment 
 RT-MRC assessment 
 Assessment of grip strength by site personnel 
8.2.6 
Visit 7 (Week 6), Home Visit 
The following procedures will be performed at Visit 7 (Week 6) by a healthcare professional 
visiting the study participant at his/her home: 
 Query for suicidality 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end of the 
infusion) 
 Recording of AEs 
 Urine pregnancy test 
 Contact IRT 
 Administer IMP 
8.2.7 
Visit 8 (Week 7) 
The following procedures will be performed at Visit 8 (Week 7): 
 iRODS assessment - to be performed before any other assessment 
 Query for suicidality  
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 


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 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end of the 
infusion) 
 12-lead ECG  
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 Urine sample for urinalysis and urine pregnancy test 
 Blood sample (all samples should be taken predose unless otherwise specified; the laboratory 
manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 PK of rozanolixizumab (predose and 4h postdose) 
 ADA (anti-rozanolixizumab antibodies) 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) (predose and 
4h postdose) 
 Serum cytokines (predose and 4h post dose) 
 Vaccination-specific antibody titers (tetanus and influenza Type A) 
 Immunoglobulins (total IgG and IgG subclasses) 
 NF-L 
 IgA, IgM, IgE 
 Exploratory biomarker analysis 
 Contact IRT 
 Administer IMP 
 INCAT assessment 
 RT-MRC assessment 
 Assessment of grip strength by site personnel 
8.2.8 
Visits 9 (Week 8), Home Visit 
The procedures performed at this visit are the same as performed for Visit 7 (Week 6); refer to 
Section 8.2.6 for details. 
8.2.9 
Visits 10, 14, 18, and 22 (Weeks 9,13, 17, and 21) 
The following procedures will be performed at Visits 10, 14, 18, and 22 (Weeks 9, 13, 17, and 
21): 
 PROs should be conducted in the order specified in Section 10.4: 
 iRODS assessment – to be performed before any other assessment 


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 CIDP PRO instrument 
 Fatigue scale 
 PGIS 
 PGIC 
 Query for suicidality 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end of the 
infusion) 
 12-lead ECG 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 Urine sample for urinalysis and urine pregnancy test 
 Blood sample (all samples should be taken predose unless otherwise specified; the laboratory 
manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 PK of rozanolixizumab (does not apply to Visit 10) 
 ADA (anti-rozanolixizumab antibodies) (does not apply to Visit 10) 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) (predose and 
4h postdose) 
 Serum cytokines (predose and 4h postdose) 
 Vaccination-specific antibody titers (tetanus and influenza Type A) (only at Visit 14) 
 Immunoglobulins (total IgG and IgG subclasses) 
 IgA, IgM, IgE 
 CIDP-specific auto-antibodies 
 Exploratory biomarker analysis 
 Contact IRT 
 Administer IMP 
 INCAT assessment 
 RT-MRC assessment 


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 Assessment of grip strength by site personnel 
8.2.10 
Home Visits 11 to 13, 15 to 17, 19 to 21, and 23 to 25 (Weeks 10 to 12, 
14 to 16, 18 to 20, and 22 to 24) 
The following procedures will be performed at Visits 11 to 13, 15 to 17, 19 to 21, and 23 to 25 
(Weeks 10 to 12, 14 to 16, 18 to 20, and 22 to 24) by a healthcare professional visiting the study 
participant at his/her home: 
 Query for suicidality 
 Tuberculosis Signs and Symptoms Questionnaire (only at Visit 13, 17, 21, and 25) 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end of the 
infusion) 
 Recording of AEs 
 12-lead ECG (only at Visits 12, 16, 20, and 24) 
 Blood sample for clinical laboratory tests (ie, hematology and clinical chemistry) (only at 
Visits 12, 16, 20, and 24) 
 Urine sample for urinalysis (only at Visits 12, 16, 20, and 24) 
 Urine pregnancy test 
 Contact IRT 
 Administer IMP 
8.3 
Treatment Period Part 2 (Weeks 25 to 76) 
Entry into the Treatment Period (Part 2) will be subject to a careful individual assessment of the 
benefit-risk for each study participant completing Treatment Period (Part 1) following a 
discussion with the Sponsor's Medical Monitor and/or Study Physician. The first visit of 
Treatment Period Part 2 will be performed at site at Week 25 followed by 3 weekly visits at the 
study participant’s home. This sequence will be repeated until the end of the Treatment Period 
Part 2. The visits during this period will be numbered sequentially. 
Study participants may choose to self-administer the IMP during Treatment Period 2 under 
supervision of a nurse and after having received appropriate training. 


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8.3.1 
Additional site visits during maximum 52-week Treatment Period Part 
2 (monthly starting at Week 25) or until availability of an Access 
Program (whichever comes first) 
The first visit of Treatment Period Part 2 will be performed on site at Week 25. Site visits will be 
performed monthly. The following procedures will be performed at monthly frequency unless 
otherwise defined: 
 PROs should be conducted in the order specified in Section 10.4: 
 iRODS assessment – to be performed before any other assessment 
 Fatigue scale (every 24 weeks) 
 CIDP PRO instrument (every 24 weeks) 
 PGIS (every 24 weeks) 
 PGIC (every 24 weeks) 
 Query for suicidality 
 Tuberculosis Signs and Symptoms Questionnaire (every 12 weeks) 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion) 
 Body weight (Week 25 and Week 49) 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 12-lead ECG (on-site visits only [Weeks 25, 37, 49, 61]) 
 Blood sample (all samples should be taken predose unless otherwise specified; the laboratory 
manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 PK of rozanolixizumab (on-site visits only [Weeks 25, 37, 49, 61]) 
 ADA (anti-rozanolixizumab antibodies) (on-site visits only [Week 25, 37, 49, 61]) 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) (predose and 
4h postdose) (at Weeks 25 and 73) 
 Serum cytokines (predose and 4h postdose) (at Weeks 25 and 73) 
 Vaccination-specific antibody titers (tetanus and influenza Type A) (on-site visits only 
[Weeks 25, 37, 49, 61]) 
 Immunoglobulins (total IgG and IgG subclasses) 


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 IgA, IgM, IgE (on-site visits only [Weeks 25, 37, 49, 61]) 
 NF-L (only at Week 25) 
 CIDP specific auto-antibodies (only at Week 25) 
 Urine pregnancy test 
 Contact IRT 
 Administer IMP – self-administration by the study participant under the supervision of the 
nurse (to confirm that study participant is proceeding correctly) may be considered provided 
the participant is willing to do so, the participant has been adequately trained by the site 
personnel according to Self-administration training guidance document  
 INCAT assessment 
 Assessment of grip strength by site personnel 
8.3.2 
Additional home visits during Treatment Period Part 2 (weekly in 
between site visits starting at Week 26 or until availability of an 
Access Program (whichever comes first) 
The following procedures will be performed during the Treatment Period Part 2 by a healthcare 
professional visiting the study participant at his or her home. Visits will be performed weekly in 
between monthly site visits (Section 8.3.1): 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion) 
 Recording of AEs 
 Contact IRT 
 Administer IMP (unless IMP is temporarily discontinued (see Section 6.4.1.3 ) – self-
administration by the study participant under the supervision of the home nurse may be 
considered provided the participant is willing to do it and has been adequately trained by the 
site personnel according to Self-administration training guidance document. 
8.4 
Observation Period, Visits 26 to 29 (Weeks 25 to 32 study 
participants completing Treatment Period Part 1 only and 
Weeks 77 to 84 for study participants completing Treatment 
Period Part 1 and Part 2) 
Following the final dose of rozanolixizumab at Week 24 (Visit 25) or Week 76, 4 subsequent 
visits will be scheduled over 8 weeks (Visits 26 to 29) to collect safety and efficacy data for 
study-related outcome measures and procedures unless the study participant continues 
rozanolixizumab treatment under an Access Program or equivalent. In the latter case, only the 
PEOT Visit (Visit 26) will be performed. Visit 27 and Visit 28 are home nursing visits to be 


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conducted by a healthcare professional visiting the study participant at his/her home; these visits 
can be conducted at the site as deemed necessary by site personnel and/or study participant. 
All study participants who do not continue on rozanolixizumab in an Access Program should 
perform all visits of the Observation Period, including the FV (scheduled 8 weeks after the final 
dose of IMP). If at any time during the Observation Period the study participant relapses 
(according to the medical judgement of the investigator supported by, eg, study participant’s 
score on iRODS, INCAT, or maximum grip strength [assessed by site personnel]), then rescue 
medication must be considered. The study participant will return to the SOC (ie, Ig treatment) as 
rescue medication at the time of relapse. 
8.4.1 
Visit 26 (Week 25 or Week 77 or PEOT) 
The following procedures will be performed at Visit 26 (Week 25 or Week 77 or PEOT): 
 PROs should be conducted in the order specified in Section 10.4: 
 iRODS assessment – to be performed before any other assessment 
 Fatigue scale 
 CIDP PRO instrument 
 PGIS 
 PGIC  
 Query for suicidality 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (taken once during the visit) 
 Body weight 
 12-lead ECG 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 Urine sample for urinalysis  
 Urine pregnancy test for women of childbearing potential 
 Blood sample (all samples should be taken predose unless otherwise specified; the laboratory 
manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 PK of rozanolixizumab 
 ADA (anti-rozanolixizumab antibodies) 


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 Immunoglobulins (total IgG, IgG subclasses) 
 NF-L (Visit 26 [Week 25] only or PEOT only if Treatment Period 1 is performed) 
 IgA, IgM, IgE 
 CIDP specific auto-antibodies (Visit 26 [Week 25] only or PEOT only if Treatment 
Period 1 is performed) 
 Vaccination-specific antibody titers (tetanus and influenza Type A) 
 Contact IRT 
 INCAT assessment 
 RT-MRC assessment (Visit 26 [Week 25] only) 
 Assessment of grip strength by site personnel 
8.4.2 
Visits 27 and 28 (Weeks 27 and 29, and at 79 and 81), Home Visits 
The following procedures will be performed at Visits 27 and 28 (Weeks 27 and 29, and at 79 and 
81) by a healthcare professional visiting the study participant at his/her home: 
 Query for suicidality 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (taken once during the visit) 
 Recording of AEs 
 Urine pregnancy test 
8.4.3 
Visit 29/FV (Week 32/84) 
The following procedures will be performed at Visit 29/FV (Week 32/84): 
 PROs should be conducted in the order specified in Section 10.4: 
 iRODS assessment – to be performed before any other assessment 
 Fatigue scale 
 CIDP (PRO instrument) 
 PGIS  
 PGIC 
 Query for suicidality  
 Prior and concomitant medications 
 Concomitant medical procedures 


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 Vital signs (taken once during the visit) 
 Body weight 
 12-lead ECG 
 Recording of AEs 
 Full physical examination 
 Full neurological examination 
 Urine sample for urinalysis  
 Urine pregnancy test for women of childbearing potential 
 Blood sample for (all samples should be taken predose unless otherwise specified; the 
laboratory manual will provide guidance on the order of sample collection): 
 Clinical laboratory tests (ie, hematology, clinical chemistry, and IGRA for active and 
latent TB) 
 ADA (anti-rozanolixizumab antibodies) 
 Immunoglobulins (total IgG and IgG subclasses) 
 IgA, IgM, IgE 
 CIDP-specific auto-antibodies 
 Tuberculosis Signs and Symptoms Questionnaire 
 Contact IRT 
 INCAT assessment 
 RT-MRC assessment 
 Assessment of grip strength by site personnel 
8.5 
Premature end of treatment and Final Visit 
All study participants who withdraw early from the Treatment Period should attend a PEOT 
Visit, which will be scheduled as close as possible to the date of the decision for the study or 
treatment withdrawal. 
Study participants are encouraged to complete all visits of the Observation Period following the 
PEOT Visit by either coming to the clinic or as home nursing visits, if appropriate. 
In case a study participant is not willing to attend the visits in the Observation Period, the study 
participant should still be strongly encouraged to attend at least the PEOT Visit and the FV. 
The FV should be scheduled 8 weeks after their final dose of IMP. 
The assessments to be done at the PEOT Visit and the FV are the same as those at Visit 26 and 
Visit 29, respectively. 


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A study participant entering the Access Program after completion of part or full Treatment 
Period 2 without any relapse will not complete the Observation Period but will only perform the 
PEOT/V26 one week after the last IMP dispensing for the study.  
8.6 
Unscheduled Visit/Telephone call 
At any time, a study participant may have an unscheduled study visit/telephone call if the 
investigator and/or the study participant deem it necessary. An unscheduled visit may be 
conducted due to safety or efficacy reasons and appropriate assessments will be conducted in 
relation to the reason for the visit. All information, including reason for visit, any information on 
AEs, etc, should be collected in the source documents and recorded in the appropriate section of 
the eCRF. 
9 
ASSESSMENT OF SAFETY 
9.1 
Adverse events 
9.1.1 
Definitions 
9.1.1.1 
Adverse event 
An AE is any untoward medical occurrence in a patient or clinical investigation study participant 
administered a pharmaceutical product that does not necessarily have a causal relationship with 
this treatment. An AE can therefore be any unfavorable and unintended sign (including an 
abnormal laboratory finding), symptom, or disease temporally associated with the use of a 
medicinal (investigational) product, whether or not related to the medicinal (investigational) 
product. 
In order to ensure complete safety data collection, all AEs occurring during the study (ie, after 
the signing of the ICF), including any pretreatment and posttreatment periods required by the 
protocol, must be reported in the eCRF even if no IMP was taken but specific study procedures 
were conducted. This includes all AEs not present prior to the initial visit and all AEs that 
recurred or worsened after the initial visit. 
Signs or symptoms of the condition/disease for which the IMP is being studied should be 
recorded as AEs only if their nature changes considerably or their frequency or intensity 
increases in a clinically significant manner as compared to the clinical profile known to the 
investigator from the study participant’s history or the Baseline Period. 
9.1.1.2 
Serious adverse event 
Once it is determined that a study participant experienced an AE, the seriousness of the AE must 
be determined. An SAE must meet 1 or more of the following criteria: 
 Death 
 Life-threatening 
(Life-threatening does not include a reaction that might have caused death had it occurred in 
a more severe form.) 
 Significant or persistent disability/incapacity 
 Congenital anomaly/birth defect (including that occurring in a fetus) 


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 Important medical event that, based upon appropriate medical judgment, may jeopardize the 
patient or study participant and may require medical or surgical intervention to prevent 1 of 
the other outcomes listed in the definition of serious 
(Important medical events may include, but are not limited to, potential Hy’s Law [see 
Section 9.2], allergic bronchospasm requiring intensive treatment in an emergency room or at 
home, blood dyscrasias that do not result in inpatient hospitalization, or the development of 
drug dependency or drug abuse.) 
 Initial inpatient hospitalization or prolongation of hospitalization 
(A patient admitted to a hospital, even if he/she is released on the same day, meets the 
criteria for the initial inpatient hospitalization. An emergency room visit that results in 
admission to the hospital would also qualify for the initial inpatient hospitalization criteria. 
However, emergency room visits that do not result in admission to the hospital would not 
qualify for this criteria and, instead, should be evaluated for 1 of the other criteria in the 
definition of serious [eg, life-threatening adverse experience, important medical event]. 
Hospitalizations for reasons not associated with the occurrence of an AE [eg, preplanned 
surgery or elective surgery for a pre-existing condition that has not worsened or manifested 
in an unusual or uncharacteristic manner] do not qualify for reporting. For example, if a 
study participant has a condition recorded on his/her medical history and later has a 
preplanned surgery for this condition, it is not appropriate to record the surgery or 
hospitalization as an SAE, since there is no AE upon which to assess the serious criteria. 
Please note that, if the pre-existing condition has worsened or manifested in an unusual or 
uncharacteristic manner, this would then qualify as an AE and, if necessary, the seriousness 
of the event would need to be determined.) 
9.1.1.2.1 
Anticipated serious adverse events 
The following Anticipated SAEs, fatigue, infection and headache are anticipated to occur in the 
population studied in this protocol at some frequency that is independent of drug exposure. 
This information does not change the investigator’s obligation to report all SAEs (including 
Anticipated SAEs) as detailed in Section 9.1.2.3. 
9.1.1.3 
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 
study participant. 


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9.1.1.4 
Adverse events of interest 
For rozanolixizumab, AEs of interest that require immediate reporting to UCB are: 
 Severe headache 
 Moderate to severe diarrhea 
 Moderate to severe abdominal pain 
 Moderate to severe vomiting 
These events should be reported to UCB within 24h, regardless of seriousness, by completing the 
eCRF. If the electronic system is unavailable for more than 24 hours, then the site will use the 
paper SAE data collection tool (see Section 9.1.2.3 for the reporting process). 
Additional assessments that may be required in case of AEs of interest are presented in 
Table 5-2. 
9.1.2 
Procedures for reporting and recording adverse events 
The study participant will be given the opportunity to report AEs spontaneously. A general 
prompt will also be given at each study visit to detect AEs. For example: 
“Did you notice anything unusual about your health (since your last visit)?” 
In addition, the investigator should review any self-assessment procedures (eg, e-diary cards) 
employed in the study. 
9.1.2.1 
Description of adverse events 
When recording an AE, the investigator should use the overall diagnosis or syndrome using 
standard medical terminology, rather than recording individual symptoms or signs. The eCRF 
and source documents should be consistent. Any discrepancies between the study participant’s 
own words on his/her own records (eg, e-diary card) and the corresponding medical terminology 
should be clarified in the source documentation. 
Details for completion of the Adverse Event eCRF (including judgment of relationship to IMP) 
are described in the eCRF Completion Guidelines. 
 Mild: An event that is easily tolerated by the participant, causing minimal discomfort and not 
interfering with everyday activities 
 Moderate: An event that causes sufficient discomfort and interferes with normal everyday 
activities 
 Severe: An event that prevents normal everyday activities. An AE that is assessed as severe 
should not be confused with an SAE. Severe is a category utilized for rating the intensity of 
an event; and both AEs and SAEs can be assessed as severe.  
 An event is defined as ‘serious’ when it meets at least 1 of the predefined outcomes as 
described in the definition of an SAE, NOT when it is rated as severe (eg, a severe AE may 
be either serious or not serious, depending on whether these criteria are also met). 


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9.1.2.2 
Rule for repetition of an adverse event 
An increase in the intensity of an AE should lead to the repetition of the AE being reported with: 
 The outcome date of the first AE that is not related to the natural course of the disease being 
the same as the start date of the repeated AE, and the outcome of “worsening” 
 The AE verbatim term being the same for the first and repeated AE, so that the repeated AE 
can be easily identified as the worsening of the first one 
9.1.2.3 
Additional procedures for reporting serious adverse events 
If an SAE is reported, UCB must be informed within 24h of receipt of this information by the 
site. The primary mechanism for reporting an SAE to UCB will be the eCRF (using the eCRF 
SAE page). If the electronic system is unavailable for more than 24 hours, the investigator must 
forward to UCB (or its representative) a duly completed paper SAE data collection tool provided 
by UCB, even if the data are incomplete, or if it is obvious that more data will be needed in order 
to draw any conclusions (see contact information for SAE reporting listed in the Serious Adverse 
Event Reporting section at the front of the protocol). The site will enter the SAE data into the 
electronic system as soon as it becomes available. 
The investigator SAE Report form must be completed in English. 
It is important for the investigator, when completing the SAE data collection tool, to include the 
assessment as to a causal relationship between the SAE and the IMP administration. This insight 
from the investigator is very important for UCB to consider in assessing the safety of the IMP 
and in determining whether the SAE requires reporting to the regulatory authorities in an 
expedited manner. 
Additional information (eg, autopsy or laboratory reports) received by the investigator must be 
provided within 24h. All documents in the local language must be accompanied by a translation 
in English, or the relevant information included in the same document must be summarized in 
the investigator SAE data collection tool. 
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 study participant, and to also inform participating 
study participants of the need to inform the investigator of any SAE within this period. Serious 
AEs that the investigator thinks may be associated with the IMP must be reported to UCB 
regardless of the time between the event and the end of the study. 
Upon receipt of the SAE data collection tool, UCB will perform an assessment of expectedness 
of the reported SAE. The assessment of the expectedness of the SAE is based on the IB. 
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. 


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9.1.2.4 
Immediate reporting of adverse events 
The following AEs must be reported immediately using the SAE data collection tool according 
to the procedure in Section 9.1.2.3: 
 SAE: AE that the investigator classifies as serious by the above definitions regardless of 
causality 
 Suspected transmission of an infectious agent via a medicinal product 
 AE of special interest (see Section 9.1.1.3) 
 AE of interest (see Section 9.1.1.4) 
 Confirmed LTBI, active TB, and NTMBI (see Section 9.3.6) 
9.1.3 
Follow up of adverse events 
An AE should be followed until it has resolved, has a stable sequelae, the investigator determines 
that it is no longer clinically significant, or the study participant is lost to follow up. This follow-
up requirement applies to AEs, SAEs, and AEs of special interest; further details regarding 
follow up of PDILI events is provided in Section 9.2.1.4. 
If an AE is ongoing at the end of the study for a study participant, follow up should be provided 
until resolution/stable level of sequelae is achieved, or until the investigator no longer deems that 
it is clinically significant, or until the study participant is lost to follow up. If no follow up is 
provided, the investigator must provide a justification. The follow up will usually be continued 
for 8 weeks after the study participant has discontinued his/her IMP. 
Information on SAEs obtained after clinical database lock will be captured through the Patient 
Safety (PS) database without limitation of time. 
9.1.4 
Pregnancy 
If an investigator is notified that a study participant has become pregnant after the first intake of 
any IMP, the investigator must immediately notify UCB’s PS department by providing the 
completed Pregnancy Report and Outcome form (for contact details see SAE reporting 
information at the beginning of this protocol). The study participant should be withdrawn from 
the study as soon as pregnancy is known (by positive pregnancy test), and the following should 
be completed: 
 The study participant should return for a PEOT Visit. 
 The study participant should immediately stop the intake of the IMP as instructed at the 
PEOT Visit. 
 An FV should be scheduled 8 weeks after the study participant has discontinued her IMP. 
The investigator must inform the study participant of information currently known about 
potential risks and about available treatment alternatives. 
The pregnancy will be documented on the Pregnancy Report and Outcome form provided to the 
investigator. The progression of the pregnancy and the eventual birth (if applicable) must be 
followed up using the Pregnancy Report and Outcome form in which the investigator has to 


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report on the health of the mother and of the child. Every reasonable attempt should be made to 
follow the health of the child for 30 days after birth for any significant medical issues. In certain 
circumstances, UCB may request that follow up is continued for a period longer than 30 days. If 
the study participant is lost to follow up and/or refuses to give information, written 
documentation of attempts to contact the study participant needs to be provided by the 
investigator and filed at the site. UCB’s PS department is the primary contact for any questions 
related to the data collection for the pregnancy, eventual birth, and follow up. 
In cases where the partner of a male study participant enrolled in a clinical study becomes 
pregnant, the investigator or designee is asked to contact the study participant to request consent 
of the partner via the Partner Pregnancy Consent form that has been approved by the responsible 
IRB/IEC and should be available in the investigator site file. In case of questions about the 
consent process, the investigator may contact the UCB/contract research organization (CRO) 
contract monitor for the study. The investigator will complete the Pregnancy Report and 
Outcome form and send it to UCB’s PS department (for contact details see SAE reporting 
information at the beginning of this protocol) only after the partner has agreed that additional 
information can be captured and has provided the signed Partner Pregnancy Consent form. 
UCB’s PS department is also the primary contact for any questions related to the data collection 
for the partner pregnancy, eventual birth, and follow up. 
A pregnancy becomes a SAE in the following circumstances: miscarriage, abortion (elective or 
spontaneous), unintended pregnancy after hormonal contraceptive failure (if the hormonal 
contraceptive was correctly used), ectopic pregnancy, fetal demise, or any congenital 
anomaly/birth defect of the baby. Those SAEs must be additionally reported using the 
investigator SAE data collection tool. 
9.1.5 
Suspected transmission of an infectious agent via a medicinal 
product 
For the purposes of reporting, any suspected transmission of an infectious agent via a medicinal 
product should be considered as an SAE; such cases must be reported immediately, recorded in 
the AE module of the eCRF, and followed as any other SAE. Any organism, virus, or infectious 
particle (eg, prion protein transmitting transmissible spongiform encephalopathy), pathogenic or 
nonpathogenic, is considered an infectious agent. 
9.1.6 
Overdose of investigational medicinal product 
Excessive dosing (beyond that prescribed in the protocol and including overdose ie, >10% above 
) should be recorded in the eCRF. Any SAE or nonserious AE associated with excessive 
dosing must be followed as any other SAE or nonserious AE. These events are only considered 
AEs or SAEs if there are associated clinical signs and symptoms or if the act of taking the excess 
medicine itself is an AE or SAE (eg, suicide attempt). 
9.1.7 
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. A DMC will 
be responsible for monitoring safety data during the study. Further details are provided in 
Section 14.7. 


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The Study Physician or medically qualified designee/equivalent will conduct an ongoing review 
of SAEs and perform ongoing SAE reconciliations in collaboration with the PS 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. 
9.1.8 
Suicidality 
At Screening, the investigator will query each study participant if he/she has a lifetime history of 
suicide attempt (including an active attempt, interrupted attempt, or aborted attempt) or suicidal 
ideation in the past 6 months. A full C-SSRS “Lifetime recent” assessment will be performed 
only when the study participant has a positive response to this query. This scale will be assessed 
by trained study personnel. When suicide attempt or suicidal ideation is confirmed by a positive 
response (Yes) to either Question 4 or Question 5 of the C-SSRS, the study participant must be 
excluded and immediately referred to a Mental Healthcare Professional. 
At each clinical visit, the investigator must query the suicidal ideation since the last visit. A full 
C-SSRS “Since last visit” assessment will be performed only when the study participant has a 
positive response to this query. When suicide attempt or suicidal ideation is confirmed by a 
positive response (Yes) to either Question 4 or Question 5 of the C-SSRS, the study participant 
must be withdrawn and immediately referred to a Mental Healthcare Professional. Details of the 
case must be documented by the investigator (principal investigator or investigator physician, not 
site staff conducting the C-SSRS) and provided to UCB via the SAE reporting process. 
9.1.9 
Hypersensitivity and adverse reactions 
The grading for infusion-related reactions according to the NCI Common Terminology Criteria 
for Adverse Events (CTCAE) version 5.0 (Jun 2017) is provided in Table 9-4. In the event of a 
severe or life-threatening (ie, Grade 3 or 4) infusion reaction, the study participant must 
permanently discontinue IMP and be managed as described in Section 18.1. 


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Table 9-4: 
Infusion-related reaction grading according to the NCI Common 
Terminology Criteria for adverse events version 5.0 (Jun 2017) 
Grading 
Infusion-related reaction 
1 
Mild transient reaction: infusion interruption not indicated; intervention not indicated 
2 
Moderate: Therapy or infusion interruption indicated but responds promptly to symptomatic 
treatment (for example, antihistamines, NSAIDs, narcotics, iv fluids); prophylactic 
medications indicated for ≤24h 
3 
Severe: Prolonged (eg, not rapidly responsive to symptomatic medication and/or brief 
interruption of infusion); recurrence of symptoms following initial improvement; 
hospitalization indicated for clinical sequelae 
4 
Life-threatening consequences; urgent intervention indicated 
5 
Death 
iv=intravenous; NCI=National Cancer Institute; NSAID=nonsteroidal anti-inflammatory drugs 
 
In case of occurrence of a hypersensitivity reaction (except for local injection site reaction) and 
depending upon its severity, appropriate countermeasures will immediately be taken by the 
investigator. In the event the infusion reaction occurs during a home visit, the home nurse will be 
trained to follow all steps as detailed in the study manual and will liaise with the investigator for 
the proper handling of the case. Serum complement (C3, C4), plasma complement (C3a, C5a), 
and serum cytokine samples must be collected from study participants experiencing infusion 
reactions (Table 5-2). 
If the investigator does not initially choose to discontinue the infusion of IMP and symptoms 
persist or escalate during continued infusion, the infusion should be stopped. In case of any 
severe infusion reaction(s), the infusion of IMP must be stopped immediately and appropriate 
treatment initiated, as necessary, at the discretion of the investigator and in accordance with the 
SOC. When infusion reactions occurred while the IMP has not been stopped, the investigator 
should closely assess the adequacy of home administrations (refer to Section 8.2) and consult 
with UCB Study Physician before considering home administration after the infusion reactions. 
Suspected anaphylactic reactions should be diagnosed using Sampson’s Criteria 
(Sampson et al, 2006) as described in Section 18.2. In the event of an anaphylactic reaction 
meeting Sampson criterion 1 or 2, the infusion must be discontinued immediately and emergency 
resuscitation measures implemented. 
9.1.10 
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.  
Treatment of headaches should be as per national guidelines and take medical history of previous 
headaches, concomitant medication, and co-morbidities (eg, asthma) in consideration. In case of 
continued tolerance issues, and if symptomatic headache medication (eg, acetylsalicylic acid 


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1000mg) is not sufficient, a further step can be to reduce dose of IMP to 
 (see 
Section 7.2). 
Determination of the severity of headache will be consistent with National Cancer Institute 
CTCAE version 5.0. Severe headache is defined as severe pain limiting self-care activities of 
daily living (ADL). Self-care ADL refer to bathing, dressing and undressing, feeding self, using 
the toilet, taking medications. Treatment of headache will be provided as clinically indicated 
according to the national guidelines. 
Study participants experiencing severe and/or serious headache will complete the Headache 
Questionnaire daily until resolution (ie, if headache becomes nonserious, moderate or mild, or 
completely resolved, whichever comes first). At the clinic visit when the severe headache is 
reported, the Headache Questionnaire will be followed by a neurological assessment (including 
fundoscopy). If the severe or serious headache is initially reported at a home visit or during a 
telephone call, the study participant should be reviewed at the study site as soon as is practically 
possible for further investigations. Study participants should be monitored for signs and 
symptoms suggestive of central nervous system involvement and evaluated immediately if other 
causes (eg, meningitis, intracranial bleeding) are suspected. Further neurological workup may be 
performed (if indicated) at the discretion of the investigator 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 or serious headache when possible (Table 5-2). These 
investigations will be performed to further understand the mechanism of headache in these study 
participants. 
Details of neurological examination to be performed are provided in Section 9.3.4. The 
Headache Questionnaire will be provided in the study procedures manual. 
9.1.11 
Management of moderate or severe diarrhea 
Moderate or severe diarrhea is defined as an increase of ≥4 stools per day over Baseline or 
incontinence due to urgency of diarrhea or new/prolonged hospitalization for management of 
diarrhea or limiting self-care ADL or life-threatening consequences requiring urgent medical 
intervention. Determination of the severity of diarrhea will be consistent with CTCAE version 
5.0. 
Stool collection and analysis will be performed for study participants reporting moderate or 
severe diarrhea. The frequency of stool sampling will be as clinically indicated in the opinion of 
the investigator and assessed per local guidance. Analysis of stool samples will be performed 
locally. In addition, collection of blood samples for the assessment of exploratory safety 
biomarkers is required for study participants with severe GI disturbances including diarrhea.  
Treatment of diarrhea will be provided as clinically indicated according to the local guidelines. 
9.2 
Laboratory measurements 
Blood and urine specimens for routine assay of hematology, clinical chemistry, and urinalysis 
testing, as well as pregnancy testing and serology testing will be performed according to the 
schedule of assessments (Table 5-1) to monitor the safety of study participants. All parameters 
will be assessed by the designated central laboratory with the exception of the urine pregnancy 


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test prior to each dose. Specific details regarding the handling and processing of serum 
chemistry, hematology, and urinalysis samples are provided in the study laboratory manual. 
The following laboratory parameters will be measured as detailed in Table 9-5. 


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Table 9-5: 
Laboratory measurements 
Hematology 
Chemistry 
Urinalysis a 
Hemoglobin 
ALP 
pH 
Hematocrit 
ALT 
Protein 
RBC 
AST 
Glucose 
WBC (including differential) 
GGT 
Ketone 
Platelet count 
Total bilirubin b 
Urobilinogen 
 
LDH 
Bilirubin 
 
Creatine kinase 
Blood 
Serology c 
Total protein 
Nitrite 
HBsAg, HBcAb 
Albumin 
Leukocytes 
HCV Ab 
Alpha- and beta–globulins 
 
HIV (anti-HIV1 or anti-HIV2 
antibodies) 
Urea-nitrogen 
 
Tuberculosis 
Creatinine 
Albumin 
 
Triglycerides 
Creatinine 
 
Total-cholesterol 
 
 
LDL cholesterol 
Pregnancy test 
 
HDL cholesterol 
Serum HCG d 
 
Electrolytes (calcium, phosphate, sodium, 
potassium, chloride, and magnesium) 
Urine HCG e 
 
Amylase 
 
 
hsCRP 
 
 
Procalcitonin 
 
 
HBA1c f 
 
Ab=antibody; ALP=alkaline phosphatase; ALT=alanine aminotransferase; AST=aspartate aminotransferase; 
GGT=gamma-glutamyltransferase; HBcAb=hepatitis B core antibody; HBsAg=hepatitis B surface antigen; 
HCG=human chorionic gonadotropin; HCV=hepatitis C virus; HDL=high density lipoprotein; HIV=human 
immunodeficiency virus; hsCRP=high sensitivity C-reactive protein; IGRA=interferon gamma release assay; 
IMP=investigational medicinal product; LDH=lactate dehydrogenase; LDL=low density lipoprotein; RBC=red 
blood cell; WBC=white blood cell 
a Urine microscopy will be performed if urine is positive for protein, blood, nitrite, or leukocytes. 
b Direct bilirubin will also be measured when total bilirubin is elevated. 
c Serology performed at Screening only. An IGRA should be performed for active or latent tuberculosis testing. 
d Serum pregnancy test performed) only for women of childbearing potential when study participant enters CIDP04 
(either at Screening [Visit 1] or Baseline [Visit 2]). 
e Urine pregnancy test (dipstick) for women of childbearing potential performed prior to dosing (and confirmed 
negative) at dosing visits and each visit of the Observation Period. Note that the final urinary pregnancy test of the 
study should be no longer than 90 days after the final dose of IMP. 
f Performed at Screening only. 


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9.2.1 
Evaluation of PDILI 
The PDILI IMP discontinuation criteria for this study are provided in Section 6.4.2, with the 
accompanying required follow-up investigation and monitoring detailed below. All PDILI events 
must be reported as AEs and reported to the study site and sponsor within 24h of learning of their 
occurrence. Any PDILI event that meets the criterion for potential Hy’s Law must be reported as 
an AE of special interest, and, if applicable, also reported as an SAE (see Section 9.1.1.2). 
Evaluation of PDILI consists of the diagnostic testing and continued monitoring included in 
Table 9-6 (specific tests dependent on laboratory results and corresponding symptoms) and 
consultation with a local hepatologist (if applicable; discussed in Section 9.2.1.1). The local 
hepatologist is the expert usually consulted by the treating physician for assessment and 
management of potential hepatic disease. This would usually be a hepatologist, but may be a 
gastroenterologist. Additional investigation and monitoring may be required and adapted based 
on the diagnosis after the cause of the liver injury/abnormality is confirmed (details in 
Section 9.2.1.4). 
The results of all monitoring, including laboratory testing and other testing, should be made 
available to the study site and sponsor. 
All initial tests resulting in abnormal hepatic laboratory values need to be repeated, but 
appropriate medical action must not be delayed waiting for the repeat result. 
If tests are done locally for more rapid results, a concurrent sample should also be sent to the 
central laboratory whenever possible. Medical care decisions are to be made initially using the 
most rapidly available results and a conservative approach must be taken if the results from the 
2 laboratory tests are significantly different. Data from the local and central laboratory are to be 
recorded on the applicable eCRF pages. 
When IMP is discontinued, all concomitant medications and herbal supplements that are not 
medically necessary should also be discontinued. In these cases, the investigator should also 
consider dose reduction for medically necessary concomitant medication and consider changing 
any medically required concomitant medication known to be hepatotoxic to a suitable 
alternative. 
When IMP is stopped due to PDILI (as described in Section 6.4.2), IMP must be permanently 
discontinued unless a subsequent alternative diagnosis fully explains the hepatic findings. If a 
subsequent alternative diagnosis fully explains the hepatic findings, and the requirements 
provided in Section 9.2.1.2.1 are met, rechallenge with IMP may be appropriate. 
Rechallenge with a substance potentially causing drug-induced liver injury is dangerous, may be 
fatal, and must not occur. 
Table 9-6 summarizes the approach to investigate PDILI. 
 


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Table 9-6: 
Required investigations and follow up for PDILI 
Laboratory value 
 
Immediate 
Follow up 
ALT or 
AST 
Total 
bilirubin 
Symptoms a of 
hepatitis or 
hypersensitivity 
Consultation 
requirements 
Actions 
Testing 
Evaluation 
≥3xULN 
≥2xULN b 
NA 
Hepatology consult.c 
Medical Monitor 
must be notified 
within 24 hours 
(eg, by laboratory 
alert) and study 
participant discussed 
with Medical Monitor 
ASAP. 
Immediate, 
permanent IMP 
discontinuation. 
Essential: Must 
have repeat liver 
chemistry values 
and additional 
testing completed 
ASAP (see 
Section 9.2.1.3); 
recommended to 
occur at the site 
with HCP. 
Monitoring of liver chemistry 
values at least twice per week 
until values normalize, stabilize, 
or return to within Baseline 
values.d 
≥3xULN 
NA 
Yes 
Immediate, 
temporary or 
permanent, IMP 
discontinuation. 
≥3xULN 
(and ≥2x 
Baseline) 
and 
<5xULN 
<2xULN 
No 
Discussion with 
Medical Monitor 
required if the 
criterion that allows 
for IMP continuation 
is met. 
Further investigation 
– immediate IMP 
discontinuation not 
required (see 
Section 9.2.1.2). 
Not required 
unless otherwise 
medically 
indicated (at 
discretion of 
investigator). 
≥5xULN 
(and ≥2x 
Baseline) 
<2xULN 
No 
Discussion with 
Medical Monitor 
required. Hepatology 
consult required if 
ALT or AST 
≥8xULN 
Immediate, 
permanent IMP 
discontinuation. 
Essential: Every 
attempt must be 
made to have 
repeat liver 
chemistry values 
and additional 
testing completed 
within 48 hours 
at the site with 
HCP (see 
Section 9.2.1.3). 
Monitoring of liver chemistry 
values at least twice per week 
until values normalize, stabilize, 
or return to within Baseline 
values.d 


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Table 9-6: 
Required investigations and follow up for PDILI 
Laboratory value 
 
Immediate 
Follow up 
ALT or 
AST 
Total 
bilirubin 
Symptoms a of 
hepatitis or 
hypersensitivity 
Consultation 
requirements 
Actions 
Testing 
Evaluation 
ALP=alkaline phosphatase; ALT=alanine aminotransferase; ASAP=as soon as possible; AST=aspartate aminotransferase; HCP=healthcare practitioner; 
IMP=investigational medicinal product; NA=not applicable; PDILI=potential drug-induced liver injury; ULN=upper limit of normal 
a Hepatitis symptoms include fatigue, nausea, vomiting, and right upper quadrant pain or tenderness; hypersensitivity symptoms include eosinophilia (>5%), 
rash, and fever (without clear alternative cause). 
b If the study participant also has ≥2xULN ALP, the possibility of an indication of biliary obstruction should be discussed with the Medical Monitor. 
c Details provided in Section 9.2.1.1. The local hepatologist is the expert usually consulted by the treating physician for assessment and management of potential 
hepatic disease. This would usually be a hepatologist, but may be a gastroenterologist. 
d Unless an alternative monitoring schedule is agreed by the investigator and UCB responsible physician. Determination of stabilization is at the discretion of the 
investigator in consultation with the hepatologist (as applicable) and UCB responsible physician, as needed. 
 


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9.2.1.1 
Consultation with Medical Monitor and local hepatologist 
Potential drug-induced liver injury events require notification of the Medical Monitor within 24h 
(eg, by laboratory alert), and the study participant must be discussed with the Medical Monitor as 
soon as possible. If required, the study participant must also be discussed with the local 
hepatologist. The local hepatologist is the expert usually consulted by the treating physician for 
assessment and management of potential hepatic disease. This would usually be a hepatologist, 
but may be a gastroenterologist. If determined necessary, this discussion should be followed by a 
full hepatology assessment (see Section 9.2.1.3) and SAE report (if applicable). 
9.2.1.2 
Immediate action: determination of IMP discontinuation 
All PDILI events require immediate action, testing, and monitoring. 
The immediate action is dependent on the laboratory values and symptoms of hepatitis or 
hypersensitivity and ranges from continuation of IMP (followed by immediate investigation) to 
immediate and permanent discontinuation (see Section 6.4.1 and Table 9-6 for details). 
When IMP is discontinued, all concomitant medications and herbal supplements that are not 
medically necessary should also be discontinued. The investigator should also consider dose 
reduction for medically necessary concomitant medication and consider changing any medically 
required concomitant medication known to be hepatotoxic to a suitable alternative. 
9.2.1.2.1 
IMP restart/rechallenge (if applicable) 
Rechallenge with a substance potentially causing drug-induced liver injury is dangerous, may be 
fatal, and must not occur. 
Study participants who are immediately discontinued from IMP due to having met certain 
criteria for PDILI (as described in Section 6.4.1 and Table 9-6), but for whom an alternative 
diagnosis is confirmed, can rarely restart IMP. Rechallenge with IMP can occur only if ALL of 
the following requirements are met: 
 The results of additional testing and monitoring described in Section 9.2.1.3 and 
Section 9.2.1.4 confirm a nondrug-related cause for the abnormal hepatic laboratory 
parameters and any associated symptoms (ie, a subsequent alternative diagnosis fully 
explains the hepatic findings). 
 No alternative treatment options are available to the study participant. 
 The study participant has shown clear therapeutic benefit from the IMP. 
 Study participant’s ALT or AST elevations do not exceed ≥3xULN. 
 Study participant’s total bilirubin is <1.5xULN. 
 Study participant has no signs or symptoms of hypersensitivity. 
 The rechallenge is approved by the UCB responsible physician, DMC, and a hepatologist. 
The hepatologist must be external to UCB but may be a member of the DMC. It is 
recommended that the hepatologist be a local hepatology expert or the hepatologist treating 
the study participant. 
 Study participant agrees to the investigator-recommended monitoring plan. 


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9.2.1.3 
Testing: identification/exclusion of alternative etiology 
The measurements and additional information required for the assessment of PDILI events when 
there is a reasonable possibility that they may have been caused by the IMP are detailed in 
Table 9-7 (laboratory measurements) and Table 9-8 (additional information). Results of the 
laboratory measurements and information collected are to be submitted to the sponsor on the 
corresponding eCRF. If the medical history of the study participant indicates a requirement for 
other assessments not included below, these additional assessments should be completed and 
submitted, as applicable. 
All blood samples should be stored, if possible. If tests are done locally for more rapid results, a 
concurrent sample must also be sent to the central laboratory. 


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The following measurements are to be assessed: 
Table 9-7: 
PDILI laboratory measurements 
Virology-
related 
Hepatitis A IgM antibody 
HBsAg 
Hepatitis E IgM antibody 
HBcAb-IgM 
Hepatitis C RNA 
Cytomegalovirus IgM antibody 
Epstein-Barr viral capsid antigen IgM antibody (if unavailable, obtain heterophile 
antibody or monospot testing) 
Immunology 
Anti-nuclear antibody (qualitative and quantitative) 
Anti-smooth muscle antibody (qualitative and quantitative) 
Type 1 anti-liver kidney microsomal antibodies (qualitative and quantitative) 
Hematology 
Eosinophil count 
Urinalysis 
Toxicology screen 
Chemistry 
Amylase 
If total bilirubin ≥1.5xULN, obtain fractionated bilirubin to obtain % direct bilirubin 
Serum CPK and LDH to evaluate possible muscle injury causing transaminase elevation 
Additional 
Prothrombin time/INR a 
Serum pregnancy test 
PK sample 
ALT=alanine aminotransferase; CPK=creatine phosphokinase; HBcAb-IgM=hepatitis B core antibody-IgM; 
HBsAg=hepatitis B surface antigen; IgM=immunoglobulin M; INR=international normalized ratio; LDH=lactate 
dehydrogenase; PDILI=potential drug-induced liver injury; PK=pharmacokinetic; RNA=ribonucleic acid; 
ULN=upper limit of normal 
a Measured only for study participants with ALT >8xULN, elevations in total bilirubin, and symptoms of hepatitis 
or hypersensitivity. Hepatitis symptoms include fatigue, nausea, vomiting, and right upper quadrant pain or 
tenderness; hypersensitivity symptoms include eosinophilia (>5%), rash, and fever (without clear alternative 
cause). 


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The following additional information is to be collected: 
Table 9-8: 
PDILI information to be collected 
New or updated information 
Concomitant prescription and over-the-counter medications (eg, acetaminophen, herbal remedies, 
vitamins); dosages and dates should be included. 
Pertinent medical history, including the following: 
 
History of liver disease (eg, autoimmune hepatitis, nonalcoholic steatohepatitis or other “fatty liver 
disease”) 
 
Adverse reactions to drugs 
 
Allergies 
 
Relevant family history or inheritable disorders (eg, Gilbert’s syndrome, alpha-1 antitrypsin 
deficiency) 
 
Recent travel  
 
Progression of malignancy involving the liver (Note: Metastatic disease to the liver, by itself, should 
not be used as an explanation for significant AST and/or ALT elevations.) 
The appearance or worsening of clinical symptoms of hepatitis or hypersensitivity (eg, fatigue, nausea, 
vomiting, right upper quadrant pain or tenderness, decreased appetite, abdominal pain, jaundice, fever, or 
rash) 
Recent clinically significant hypotension or hypoxemia with compromised cardiopulmonary function 
Alcohol and illicit drug use 
Results of liver imaging or liver biopsy, if done 
Results of any specialist or hepatology consult, if done 
Any postmortem/pathology reports 
ALT=alanine aminotransferase; AST=aspartate aminotransferase; PDILI=potential drug-induced liver injury 
9.2.1.4 
Follow-up evaluation 
Potential drug-induced liver injury events require follow-up monitoring as described in  
Table 9-6. Monitoring should continue until liver chemistry values normalize, stabilize, or return 
to Baseline. Determination of stabilization is at the discretion of the investigator in consultation 
with the hepatologist (as applicable) and UCB responsible physician, as needed. 
9.3 
Other safety measurements 
9.3.1 
Pregnancy testing 
Pregnancy testing will consist of serum testing at the Screening Visit (only for study participants 
with a gap period between the parent study and entry in CIDP04) and Baseline, if serum 
pregnancy test is not older than 5 weeks prior to entry into CIDP04. Pregnancy testing will 
consist of urine testing at dosing visits during the Treatment Period and each visit during the 
Observation Period as indicated in the schedule of study assessments (Table 5-1). The Screening 
Visit serum pregnancy testing results must be negative and should be confirmed by a negative 
urine pregnancy test prior to first dose of IMP. The urine pregnancy test will be performed 


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locally. A negative urine pregnancy test result should be obtained prior to each dose of IMP. A 
positive urine pregnancy test must be confirmed using a serum pregnancy test. Pregnancy tests 
should be administered to all female study participants of childbearing potential, regardless of 
their use of birth control. 
9.3.2 
Vital signs 
Vital signs will be measured at all visits as indicated in the schedule of study assessments 
(Table 5-1). 
Vital signs to be assessed are as follows: 
 Pulse rate (PR) 
 Systolic/diastolic blood pressure (BP) 
 Temperature (oral preferred, ear or axillary allowed) 
Study participants should be sitting for 5 minutes prior and during the collection of BP and PR 
measurements. During site Visits 2, 3, and 4, vital signs will be measured prior to IMP 
administration, at the end of the infusion, and 2 and 4h after the end of the infusion. From Visit 5 
to Visit 25, vital signs will be measured predose, at the end of the infusion and 2h after the end of 
the infusion only. During Treatment Period Part 2, vital signs will be measured predose, and at 
the end of the infusion. At nondosing visits, vital signs need only be taken once during the visit. 
For study participants requiring additional assessments due to AEs (see Table 5-2), additional 
vital signs measurements may be taken based on the timing of the assessments. 
9.3.3 
Physical examination 
9.3.3.1 
Full physical examination 
A physical examination will be performed at the visits specified in the schedule of study 
assessments (Table 5-1) and findings will be recorded in the eCRF. A full physical examination 
will be performed at Screening or Baseline, and the FV (Visit 29). At all other visits (where 
physical examination is performed), an abbreviated (brief) physical examination will be 
conducted. 
Clinically important abnormal changes in subsequent physical examinations will be recorded as 
AEs. Physical examinations must be documented in source documentation. 
The following body systems will be examined as a part of the complete physical examination: 
(1) General appearance; (2) Ear, nose, and throat; (3) Eyes; (4) Hair and skin; (5) Respiratory; 
(6) Cardiovascular; (7) Musculoskeletal; (9) Gastrointestinal; (10) Hepatic; (11) Neurological 
examination (see also Section 9.3.4); and (12) Mental status. 
9.3.3.2 
Brief physical examination 
The following body systems will be examined at the visits specified in the schedule of study 
assessments (Table 5-1) and findings will be recorded in the eCRF. The following body systems 
will be examined as a part of the brief physical examination: (1) General appearance; (2) Ear, 
nose, and throat; (3) Eyes; (4) Skin; (5) Respiratory; (6) Gastrointestinal; and (7) Neurological 
(focused assessment of sensitivity and power). 


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9.3.4 
Neurological examination 
In addition to the Screening or Baseline, and FV (Visit 29), a full neurological examination 
should be performed for any study participant who experiences severe headache (see 
Section 9.1.10 and Table 5-2). A full neurological assessment will include: (1) General 
appearance, including posture, motor activity and meningeal signs and, if indicated, following 
assessments to be done; (2) Cranial nerves examination; (3) Motor system examination, 
including muscle tone and power and sensory system examination – light touch; (4) Reflexes, 
including deep tendon reflexes; (5) Coordination, gait (if possible); and (6) Fundoscopy. 
A brief neurological assessment will include a selected assessment of the following: cognition, 
general, reflexes, muscle strength, and coordination/cerebellar function. 
9.3.5 
12-lead ECG 
A standard 12-lead ECG will be performed at the visits specified in the schedule of study 
assessments (Table 5-1). Care should be taken to assure proper lead placement and quality ECG 
recordings. Study participants should rest in a supine position in a controlled, calm environment 
for at least 15 minutes prior to the recording and should be motionless during the recording. The 
ECG will be performed in triplicate prior to blood collection for assessment of laboratory 
parameters. 
The ECGs will be read at a central site. The PR, RR, QRS, QT, and corrected QT (QTc) intervals 
and heart rate will be recorded. All ECG readings from an individual study participant should be 
read by the same reader, if possible. Findings will be recorded in the eCRF. 
For the QTc, the following correction formula will be applied: 
Fridericia’s correction: QTc = QT/RR0.33 
9.3.6 
Assessment and management of TB and TB risk factors 
With the currently available data, TB is not considered as an important potential or identified risk 
for treatment with rozanolixizumab. As immunomodulation may carry risk of new or activation 
of LTBI, UCB has conservatively developed TB detection and management procedures taking 
into account the most current recommendations of international guidelines (2010 WHO) and 
most recent literature, covering any infection by the mycobacteria TB complex. 
Appropriate rigorous precautions are being taken within this protocol to monitor the risk of TB 
infection in this study (see Section 6.2.1 Exclusion Criterion 20 and Section 6.4 Withdrawal 
Criteria). The following are the key considerations of these procedures: 
TB tests at Screening 
The IGRA and Tuberculosis Questionnaire are required as indicated in schedule of assessments  
(Table 5-1). 
 TB screening is mandatory both before study entry and during the conduct of the study. The 
preferred screening test is the IGRA performed at a central laboratory. 
 The IGRA result must be negative for study participants to enroll in this study. 


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 Study participants who test positive for IGRA test should be excluded from the study and 
referred for appropriate medical evaluation according to the local medical practice 
guidelines. 
 If the result of the IGRA test is indeterminate, the particular IGRA test previously 
performed may be repeated once; if positive or indeterminate on retest, the study 
participant may not be treated with study drug and, if already enrolled, must undergo 
appropriate study specified withdrawal procedures. The retest must be done during the 
protocol-defined Screening window. 
 In addition to IGRA test, study participants will be evaluated for signs and symptoms of 
latent or active TB infection and for risk factors of exposure to TB, using the TB 
Questionnaire, at Screening. Study participants with known TB, at a high risk of acquiring 
TB or with LTBI should be excluded from this study as described in Exclusion Criteria 
(Section 6.2.1). For study participants directly enrolling in CIDP04, no new TB assessment 
will be performed and values of TB assessment performed at Visit 17 and/or FV (Visit 21) of 
CIDP01will be used. 
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. 
Study participants with confirmed LTB or active TB or NTMB infection will be immediately 
withdrawn from the study as described in Section 6.4 Withdrawal Criteria. Confirmed LTB, 
active TB, and NTMB must be reported to the Sponsor immediately regardless of seriousness 
using the SAE data collection tool. Additional information received by the investigator should be 
provided to the Sponsor within 24 hours of awareness. 
Once withdrawn from study treatment, study participants should return for the POET, complete 
all early withdrawal assessments, and complete the follow-up visits. 
TB tests at Final Visit 
Study participants will be evaluated for signs and symptoms of latent or active TB infection and 
for risk factors of exposure to TB, using the Tuberculosis Questionnaire, at the Final /PEOT 
Visit. See the ‘Tuberculosis Signs and Symptoms Questionnaire’ section for further instructions 
on using the questionnaire. 
Signs and symptoms of TB 
The investigator should consider all potential sites of infection when assessing for TB during the 
physical examination, and other evaluations, and based on the study participant’s history. 
Common symptoms with which the study participant may present with include cough, blood in 
sputum, night sweats, lymphadenitis, joint pain/swelling, spinal deformity, headache/confusion, 
abdominal pain mimicking inflammatory bowel disease, frequent or painful urination, scrotal 
mass in men and pelvic inflammatory disease in women as well as other symptoms, or 
nonspecific symptoms. This is not an exhaustive list and unusual presentations should always be 
considered. 


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TB Signs and Symptoms Questionnaire 
The questionnaire “Evaluation of signs and symptoms of tuberculosis” should be used as a 
source document. The questionnaire will assist with the identification of study participants who 
may require therapy for TB. A study participant who answers “Yes” to the question “
 
 
” at Screening is excluded. A “Yes” response to any of the other 
questions within the questionnaire at Screening should trigger further careful assessment to 
determine if study participant has latent or active TB. A “Yes” response to any of the questions 
at the end of the study should trigger further assessments as per local medical guidelines to 
determine if the study participant has either LTB or active TB infection. 
LTB infection, active TB or other NTBM identified during study 
During the study, study participants who develop evidence of LTB infection or active TB or 
NMTB must immediately stop further administration of study drug and will be referred to an 
appropriate medical specialist for further evaluation. 
Confirmed LTB or active TB or NTMB must be reported to the Sponsor immediately as 
described above. 
10 
ASSESSMENT OF EFFICACY 
10.1 
iRODS 
The iRODS Questionnaire should be completed by the study participant prior to dosing and 
before any discussion of their current health status with the treating physician or any other study 
assessments. The questionnaire will be given to the study participant to complete and will then 
be recollected and checked, for completeness only, by study personnel other than the treating 
physician. Study participants should complete the questionnaires themselves, unaided, in a quiet 
location. 
The iRODS is a linearly weighted PRO measure (questionnaire) that specifically captures 
activity and social participation limitations in patients with CIDP. The iRODS scale was 
constructed based on the World Health Organization (WHO) International Classification of 
Functioning, Disability and Health, literature search, and patient interviews (van Nes et al, 2011). 
All 24 items of iRODS meet Rasch model expectations. The questionnaire consists of 24 items 
(including eating, taking a shower, walking a flight of stairs, standing for hours, etc) and assesses 
a patient’s perception of their ability to perform daily and social activities. Patients have 
3 response options: 0=impossible to perform; 1=performed with difficulty; and 2=easily 
performed, performed without difficulty (van Nes et al, 2011). The raw sum scores of iRODS 
(range: 0 to 48) will be translated to log odds units (logits), placing patients’ estimates on the 
same logit scale. For easier interpretation, the person locations can be translated to values 
changing from 0 (most severe activity and social participation restrictions) to 100 (no activity 
and social participation limitations). 
Study participants will be assessed for the change from Baseline to Week 25 in their iRODS 
score and for change from Baseline in iRODS scores at each scheduled assessment during the 
Treatment and Observation Periods. Other efficacy variables will also include study participant 
experienced CIDP relapse (iRODS), defined as a clinically important deterioration, ie, an MCID-
SE ≤-1.96, and time to CIDP relapse (iRODS) during the Treatment Period. 


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Study participants will complete the iRODS Questionnaire at the time points detailed in the 
schedule of study assessments (Table 5-1). 
10.2 
INCAT 
The INCAT disability scale is a 10-point clinician-reported ordinal measure capturing problems 
in daily arm and leg activities and mobility. The measure captures daily activities such as 
dressing the upper part of the body, doing and undoing buttons and zips, washing and brushing 
hair, and handling coins. Each item is scored as being “not affected,” “affected but not 
prevented,” or “prevented.” The leg scale measures problems with walking, taking into account 
the use of aids. The INCAT scale ranges from 0 (no signs of disability) to 10 (most severe 
disability score) (Breiner et al, 2014; Léger et al, 2013; Hughes et al, 2008). For the purposes of 
the present study, the adjusted INCAT disability score will be used. The adjusted score is 
identical to the INCAT disability score except for the exclusion of changes in upper limb 
function from 0 (normal) to 1 (minor symptoms) or from 1 to 0. 
The investigator (or qualified personnel) will record the study participants’ adjusted INCAT 
score at the time points detailed in the schedule of study assessments (Table 5-1). It is 
recommended that the rater of the scale remains the same throughout the duration of the study to 
ensure consistency of the rating. 
Study participant experienced CIDP relapse (adjusted INCAT) (defined as an increase from 
Baseline of at least 1 point in their adjusted INCAT score) up to Week 25 after first treatment 
and time to CIDP relapse (adjusted INCAT) during the Treatment Period will be assessed 
together with the change from Baseline in adjusted INCAT score at each scheduled assessment 
during the Treatment and Observation Periods. 
10.3 
Clinician-reported grip strength 
Grip strength will be assessed by qualified site personnel according to the schedule of study 
assessments (Table 5-1). Qualified site personnel will be trained regarding obtaining and 
recording measurements. At each scheduled visit, the grip strength, generated by the study 
participant, and measured using a standardized tool, will be evaluated 3 times in the dominant 
hand. All 3 assessments will be recorded in the eCRF. For analyses, UCB will utilize the 
maximum of the 3 assessments. Efficacy will be assessed as change from Baseline in maximum 
grip strength score at each scheduled assessment during the Treatment and Observation Periods. 
Study participant experienced CIDP relapse (maximum grip strength) (defined as a clinically 
important deterioration from Baseline in grip strength, ie, a decline of >14kPa) up to Week 25 
after first treatment and time to CIDP relapse (maximum grip strength) during the Treatment 
Period will be assessed together with the change from Baseline in maximum grip strength score 
at each scheduled assessment during the Treatment and Observation Periods. 
10.4 
Patient-reported outcomes 
The PRO assessments should be administered in the following order: iRODS, CIDP PRO 
instrument, fatigue, PGIS, and PGIC. Refer to Section 10.1 for details on iRODS. 
Study participants will complete PROs at the time points detailed in the schedule of study 
assessments (Table 5-1). Study personnel other than the treating physician should administer the 
PROs. The PROs should be completed by the study participant themselves in a quiet place. 


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10.4.1 
CIDP PRO instrument 
Neuropathic Symptoms will be assessed at the time points detailed in the schedule of study 
assessments (Table 5-1). The CIDP PRO instrument consists of 2 domain scales; (1) Pain 
Severity numeric rating scale and (2) Neuropathy scale. Pain Severity consisting of 4 items 
assessing pain severity in the past 7 days and at the time of completion of the instrument, using 
0 to 10 numeric rating scales. Summed total raw score will range between 0 to 40 with higher 
scores reflecting higher levels of pain severity. The Neuropathy scale consists of 17 items rated 
within a 7-day recall period on a 6-point Likert severity scale ranging from “none” to “very 
severe.” The summed total raw score will range from 17 to 102 with higher scores reflecting 
higher levels of pain and physical sensations severity. Following psychometric evaluation and 
refinement (eg, reduction in items) of these domains scales by using data generated in CIDP01, 
transformed interval 0 to 100 scoring will be provided for both domain scores with higher score 
reflecting higher severity. 
10.4.2 
Fatigue 
Fatigue will be assessed at the time points detailed in the schedule of study assessments  
(Table 5-1). The Fatigue instrument consists of 3 domain scales; Physical Fatigue comprising 
16 items; Mental Fatigue comprising 18 items and Fatigability comprising 21 items. All 55 items 
across the 3-domain scales are rated within a 7-day recall period on a 5-point Likert frequency 
scale ranging from “none of the time” to “all of the time.” The summed total raw scores for each 
of the 3 scales will range between 16 and 80; 18 to 90 and 21 to 105 points for the Physical, 
Mental, and Fatigability scales, respectively, with a potential for an exploratory total overall 
score ranging from 55 to 275. Higher scores reflect higher levels of fatigue across all domain 
scale scores. Following psychometric evaluation and refinement (eg, reduction in number of 
items) of these domain scales by using data generated in CIPD01, transformed interval 0 to 100 
scoring will be provided for each domain score with higher scores reflecting higher levels of 
fatigue. 
10.4.3 
Patient Global Impressions 
Patient global impressions will be assessed at the time points detailed in the schedule of study 
assessments (Table 5-1). Both PGI-C and PGI-S will be used as anchors to determine a 
meaningful change threshold for CIDP PRO and fatigue scores.  
Study participants will rate their global impression of CIDP symptom severity and fatigue 
severity respectively, on a 5-point Likert severity scale ranging from ‘none” to “very severe.” 
Study participants will rate their global impression of change (if at all) in CIDP symptom and 
fatigue respectively, on a 7-point Likert severity scale ranging from “very much improved” to 
“very much worse” from Baseline/start of clinical study treatment to the scheduled visits. 
10.5 
RT-MRC 
The RT-MRC will be assessed by qualified site personnel according to the schedule of study 
assessments (Table 5-1). 
The RT-MRC sum score is a summation of the RT-MRC grades in integers of the following 
muscles on each side: upper arm abductors, elbow flexors, wrist extensors, hip flexors, knee 
extensors, and foot dorsal flexors. 


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The RT-MRC sum score was developed based on a Rasch analysis of the original MRC grading 
system (Vanhoutte et al, 2012; Merkies et al, 2010; Merkies et al, 2002). The MRC grades were 
rescored from 6 to 4 response options (0, paralysis; 1, severe weakness; 2, slight weakness; 
3, normal strength) for the RT-MRC in order to acceptably fulfill Rasch model expectations. 
Sum scores range from 36 “normal” to 0 “quadriplegic.” 
Efficacy will be assessed as a change from Baseline in RT-MRC sum score at each scheduled 
assessment during the Treatment and Observation Periods. 
11 
ASSESSMENT OF PHARMACOKINETIC/ 
PHARMACODYNAMIC VARIABLES 
11.1 
Pharmacokinetic variable 
The plasma concentration of rozanolixizumab will be characterized. Blood samples will be 
drawn by qualified personnel according to the schedule in Table 11-1. All blood samples 
collected before dosing with rozanolixizumab at the dosing visits will be drawn at the same time 
as the sampling for standard clinical laboratory tests. The time and date of collection will be 
recorded in the eCRF. 
Table 11-1: Serial blood sampling for rozanolixizumab concentration 
Matrix 
Time after start of sc infusion 
Plasma 
Predose and postdose at 4h after finishing the 
 on Visits 2, 4, and 8. 
Predose samples will be taken at Visits 14, 18, 22 and quarterly at site visits during 
Treatment Period Part 2. 
Nondosing Visit 26, samples to be taken once during the visit 
Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
11.2 
Pharmacodynamic variables 
Pharmacodynamic variables are defined in Section 4.2.4. 
For all PD assessments blood samples will be collected predose (at dosing visits) by qualified 
site personnel at the same time that samples are collected for standard clinical laboratory 
assessments. Blood samples for PD analyses will be drawn according to the schedule of study 
assessments (Table 5-1). The time and date of collection will be recorded in the eCRF. 
Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
12 
ASSESSMENT OF IMMUNOLOGICAL VARIABLES 
The following immunological assessments will be performed according to the schedule of study 
assessments (Table 5-1). 
 Serum immunoglobulin concentration over time: IgA, IgE, and IgM 
 Serum complement levels over time: C3 and C4 


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 Plasma complement levels over time: C3a and C5a 
 Plasma ADA (anti-rozanolixizumab antibodies) 
 Serum cytokines 
For all immunological assessments, blood samples will be collected predose (at dosing visits) by 
qualified site personnel at the same time that samples are collected for standard clinical 
laboratory. Serum complement (C3, C4), plasma complement (C3a, C5a), and serum cytokine 
samples will also be taken 4h postdose at Visits 3, 8, 10, 14, 18, 22 and Weeks 25 and 73 during 
Treatment Period Part 2. Additional samples may be collected in case of infusion reactions (see 
Table 5-2). The time and date of the blood draws will be recorded in the eCRF. Anti-drug 
antibodies samples will also be taken predose at Visits 2, 4, 8, 14, 18, 22, and at Weeks 25, 37, 
49, and 61 during Treatment Period Part 2 and at Visit 26. 
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). 
Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
12.1 
Assessment of exploratory biomarkers 
Collection of these samples will occur according to the schedule of study assessments 
(Table 5-1). Blood samples will be drawn prior to dosing (on dosing visits) at the same time as 
the sampling for clinical laboratory tests. Exploratory samples may be collected 4h postdose in 
case of AE of interest (Table 5-2). The time and date of collection will be recorded in the eCRF. 
For each protein and metabolite exploratory biomarker blood sample, a volume of 8mL of whole 
blood is needed (4mL for serum and 4mL for plasma). 
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 
the underlying causes of CIDP in patients, related biology, and drug response. The nature and 
format of these tentative additional analyses will be determined at a later time. Details on the 
collection, storage, preparation, and shipping of samples will be presented in the Laboratory 
Manual provided separately. Instructions pertaining to sample collection, processing, storage, 
labeling, and shipping are provided in the laboratory manual for this study. Detailed information 
on sample analyses will be provided in a bioanalytical report.  
13 
STUDY MANAGEMENT AND ADMINISTRATION 
13.1 
Adherence to protocol 
The investigator should not deviate from the protocol. However, the investigator should take any 
measure necessary in deviation from or not defined by the protocol in order to protect clinical 


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study  participants from any immediate hazard to their health and safety. In this case, this action 
should be taken immediately, without prior notification of the regulatory authority, IRB/IEC, or 
sponsor. 
After implementation of such measure, the investigator must notify the Clinical Project Manager 
of the sponsor within 24 hours and follow any local regulatory requirements. 
13.2 
Monitoring 
UCB (or designee) will monitor the study to meet the sponsor’s monitoring SOPs, ICH-GCP 
guideline, and applicable regulatory requirements, and to ensure that study initiation, conduct, 
and closure are adequate. Monitoring of the study may be delegated by UCB to a CRO or a 
contract monitor. 
The investigator and his/her staff are expected to cooperate with UCB (or designee) and to be 
available during the monitoring visits to answer questions sufficiently and to provide any 
missing information. The investigator(s)/institution(s) will permit direct access to source 
data/documents for study-related monitoring, audits, IRB/IEC review, and regulatory 
inspection(s). 
The investigator will allow UCB (or designee) to periodically review all CRFs and 
corresponding source documents (eg, hospital and laboratory records for each study participant). 
Monitoring visits will provide UCB (or designee) with the opportunity to evaluate the progress 
of the study, verify the accuracy and completeness of CRFs, ensure that all protocol 
requirements, applicable authorities’ regulations, and investigator’s obligations are being 
fulfilled, and resolve any inconsistencies in the study records. 
13.2.1 
Definition of source data  
All source documents must be accurate, clear, unambiguous, permanent, and capable of being 
audited. They should be made using some permanent form of recording (ink, typing, printing, 
optical disc). They should not be obscured by correction fluid or have temporary attachments 
(such as removable self-stick notes). Photocopies and/or printouts of CRFs are not considered 
acceptable source documents. 
Source documents are original records in which raw data are first recorded. These may include 
hospital/clinic/general practitioner records, charts, diaries, x-rays, laboratory results, printouts, 
pharmacy records, care records, ECG or other printouts, completed scales, or quality of life 
questionnaires, 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 study 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). 
13.2.2 
Source data verification 
Source data verification ensures accuracy and credibility of the data obtained. During monitoring 
visits, reported data are reviewed with regard to being accurate, complete, and verifiable from 


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source documents (eg, study participant files, recordings from automated instruments, tracings 
[ECG], x-ray films, laboratory notes). All data reported on the eCRF should be supported by 
source documents, unless otherwise specified in Section 13.2.1. 
13.3 
Data handling 
13.3.1 
Case Report form completion 
This study is performed using remote data capture. The investigator is responsible for prompt 
reporting of accurate, complete, and legible data in the eCRFs and in all required reports. 
Any change or correction to the eCRF after saving must be accompanied by a reason for the 
change. 
Corrections made after the investigator’s review and approval (by means of a 
password/electronic signature) will be reapproved by the investigator. 
The investigator should maintain a list of personnel authorized to enter data into the eCRF. 
Detailed instructions will be provided in the eCRF Completion Guidelines. 
13.3.2 
Database entry and reconciliation 
Case Report forms/external electronic data will be entered/loaded into a validated electronic 
database using a clinical data management system (CDMS). Computerized data cleaning checks 
will be used in addition to manual review to check for discrepancies and to ensure consistency of 
the data. Case Report form data are entered into the clinical database using independent, 
double-data entry, with the exception of comment fields, which are verified by a second person. 
The data are entered into the electronic CRFs once and are subsequently verified if the study is 
performed using electronic data capture. 
An electronic audit trail system will be maintained within the CDMS to track all data changes in 
the database once the data have been saved initially into the system or electronically loaded. 
Regular backups of the electronic data will be performed. 
13.3.3 
Study participant Screening and Enrollment log/Study participant 
Identification Code list 
The study participant’s screening and enrollment will be recorded in the Study participant 
Screening and Enrollment Log. 
The investigator will keep a Study participant Identification Code list. This list remains with the 
investigator and is used for unambiguous identification of each study participant. 
The study participant’s consent and enrollment in the study must be recorded in the study 
participant’s medical record. These data should identify the study and document the dates of the 
study participant’s participation. 
13.4 
Termination of the study 
UCB reserves the right to temporarily suspend or prematurely discontinue this study either at a 
single site, multiple sites, or at all sites at any time for reasons including, but not limited to, 
safety or ethical issues, inaccurate or incomplete data recording, noncompliance, or 
unsatisfactory enrollment with respect to quality or quantity. 


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If the study is prematurely terminated or suspended, UCB (or its representative) will inform the 
investigators/institutions and the regulatory authority(ies) of the termination or suspension and 
the reason(s) for the termination or suspension, in accordance with applicable regulatory 
requirement(s). The IRB/IEC should also be informed and provided with reason(s) for the 
termination or suspension by the sponsor or by the investigator/institution, as specified by the 
applicable regulatory requirement(s). In addition, arrangements will be made for the return of all 
unused IMP and other material in accordance with UCB procedures for the study. 
13.5 
Archiving and data retention 
The investigator will maintain adequate records for the study, including CRFs, medical records, 
laboratory results, Informed Consent documents, drug dispensing and disposition records, safety 
reports, information regarding participants who discontinued, and other pertinent data. 
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/investigational device. These 
documents should be retained for a longer period, however, if required by the applicable 
regulatory requirement(s) or by an agreement with UCB (Committee for Proprietary Medicinal 
Products [CPMP]/ICH/135/95, 2002 [Section 4.9.5]). The investigator will contact UCB for 
authorization prior to the destruction of any study records or in the event of accidental loss or 
destruction of any study records. The investigator will also notify UCB should he/she relocate or 
move the study-related files to a location other than that specified in the sponsor’s trial master 
file. 
13.6 
Audit and inspection 
The investigator will permit study-related audits mandated by UCB, after reasonable notice, and 
inspections by domestic or foreign regulatory authorities. 
The main purposes of an audit or inspection are to confirm that the rights and well-being of the 
study participants enrolled have been protected, that enrolled study participants (ie, signing 
consent and undergoing study procedures) are appropriate for the study, and that all data relevant 
for the evaluation of the IMP/investigational device have been processed and reported in 
compliance with the planned arrangements, the protocol, investigational site, and IRB/IEC SOPs, 
ICH GCP, and applicable regulatory requirements. 
The investigator will provide direct access to all study documents, source records, and source 
data. If an inspection by a regulatory authority is announced, the investigator will immediately 
inform UCB (or designee). 
13.7 
Good Clinical Practice 
Noncompliance with the protocol, ICH-GCP, or local regulatory requirements by the 
investigator, institution, institution staff, or designees of the sponsor will lead to prompt action 
by UCB to secure compliance. Continued noncompliance may result in the termination of the 
site’s involvement in the study. 


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14 
STATISTICS 
A description of statistical methods follows and will be described in more detail in SAP which 
will be finalized and approved prior to database lock. 
14.1 
Definition of analysis sets 
14.1.1 
Enrolled Set 
The Enrolled Set (ES) will consist of all study participants who have signed the ICF. 
14.1.2 
Safety Set 
The Safety Set (SS) will consist of all enrolled study participants who were administered at least 
1 dose of rozanolixizumab. 
Safety variables will be analyzed using the SS. 
14.1.3 
Full Analysis Set 
The Full Analysis Set (FAS) will consist of all study participants in the SS who have a Baseline 
and least 1 post-Baseline iRODS measurement. 
The FAS is the primary analysis set for efficacy analyses. 
14.1.4 
Pharmacokinetic Per-Protocol Set 
The Pharmacokinetic Per-Protocol Set (PK-PPS) is a subset of the SS, consisting of those study 
participants who had no important protocol deviation affecting the plasma concentration of 
rozanolixizumab. Post-Baseline deviations will not necessarily lead to total exclusion of a study 
participant from the PK-PPS but may lead to exclusion of specific data. 
14.1.5 
Pharmacodynamic Per-Protocol Set 
The Pharmacodynamic Per-Protocol Set (PD-PPS) is a subset of the FAS, consisting of those 
study participants who had no important protocol deviation affecting the serum concentrations of 
total IgG or IgG subclasses. Post-Baseline deviations will not necessarily lead to total exclusion 
of a study participant from the PD-PPS but may lead to exclusion of specific data. 
14.2 
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 by visit (where applicable) with the statistics mean, 
standard deviation (SD), median, minimum, and maximum. Categorical variables will be 
summarized by visit (where applicable) with frequency counts and percentages. 
Data listings containing all documented data and all calculated data will be generated. 
Baseline will be the last nonmissing data collected prior to the first dose of IMP, and 
measurement-specific Baseline values will be defined in the SAP. 
14.3 
Planned safety analyses 
14.3.1 
Safety analyses 
The incidence of study participants with TEAEs will be determined. Furthermore, the absolute 
and relative frequencies for study participants with a given TEAE with respect to the preferred 
term according to the latest available version of the Medical Dictionary for Regulatory Activities 


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(MedDRA®), will be determined within each system organ class. Additional tables will 
summarize TEAEs by maximum severity and causal relationship with rozanolixizumab, as 
judged by the investigator. Adverse events will be categorized by severity according to the 
National Institutes for Health CTCAE version 5.0 grading (National Institutes for Health, 2017). 
In case the CTCAE grading is not available, the intensity (mild/moderate/severe) will be utilized. 
The TEAEs leading to discontinuation of IMP and the serious TEAEs will also be summarized. 
The action taken, time of onset relative to dosing, actual dose received, and duration of each AE 
will be listed only. 
For the continuous laboratory variables, the values and changes from Baseline will be 
descriptively summarized at each time point. For categorized values according to the reference 
range, shift tables from Baseline to each post-Baseline time point will be presented for selected 
variables to be defined in the SAP. Values outside the reference range will be flagged in the data 
listings. 
Descriptive statistics will be presented for ECG value and changes from Baseline over time 
based on the mean of the triplicate assessments at each time point. Categorical analyses for QTc, 
eg, QTc increase >60msec, will also be performed. 
Descriptive statistics will be reported for all vital sign measurements (including BP, PR, 
temperature, and body weight). Measured values and changes from Baseline will be summarized 
by time point. 
Physical and neurological examination findings and the results of any pregnancy testing will be 
presented in listings only. Results will be listed for any study participants who complete a 
headache questionnaire, undergo stool sampling, or lumbar puncture, etc. 
14.4 
Other analyses 
14.4.1 
Efficacy analyses 
For CIDP relapse (iRODS, adjusted INCAT, and maximum grip strength as assessed by site 
personnel) (Yes/No) point estimates the proportion of patients relapsing will be presented along 
with 95% confidence intervals (CIs). 
For time to CIPD relapse (iRODS, adjusted INCAT and maximum grip strength as assessed by 
site personnel), Kaplan Meier estimates and corresponding 95% CIs will be constructed using the 
asymptotic standard error (asymptotic Wald confidence limits). 
Statistical methods for time to rescue mediation administration during treatment period will be 
the same as for time to CIPD relapse. 
For other efficacy variables, including change from Baseline to Week 25 in iRODS score, 
change from Baseline in adjusted INCAT score and change from Baseline in RT-MRC sum 
score, appropriate summary and descriptive statistics will be provided at each time point where 
the data are recorded. 
A sensitivity analysis will be performed excluding any study participant who received a mean 
dose more than ±10% from the intended dose. Additional sensitivity analyses may be carried out 
and will be fully detailed in the SAP. 


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14.4.2 
Pharmacokinetic analyses 
Pharmacokinetic variables of rozanolixizumab like AUC (area under the curve from 0 to infinity) 
and Cmax (maximum observed plasma concentration) cannot be derived, due to the sparse blood 
sampling scheme. Thus, PK is restricted to concentration data. 
In contrast to the general descriptive display, concentration data will be summarized by actual 
dose received and time point using the number of available observations, mean, median, SD, 
minimum, maximum, geometric mean (and associated 95% CIs), and geometric coefficient of 
variation (assuming log-normally distributed data). Values below the lower limit of 
quantification (LLOQ) will be reported with a clear sign indicating that they were below the 
LLOQ. Descriptive statistics of concentrations will be calculated if at least two-thirds of the 
individual data points are quantifiable (≥LLOQ). Individual concentrations of rozanolixizumab 
will also be displayed graphically. 
14.4.3 
Pharmacodynamic analyses 
For all PD variables, descriptive statistics for the value, change from Baseline, and/or percentage 
change from Baseline will be tabulated by actual dose received and time point. 
The PD variables will include serum IgG and IgG subclass and NF-L. 
14.4.4 
Immunological analyses 
All immunologic variables including concentrations of immunoglobulins (IgA, IgE, and IgM), 
and serum (C3 and C4) and plasma (C3a and C5a) complement levels, and serum cytokines will 
be summarized and visit using descriptive statistics. 
The ADA status (negative or confirmed positive) and the changes in confirmed positive titer at 
each scheduled assessment during Treatment and Observation Periods will be summarized by 
visit to inform on incidence and emergence of ADA; figures will also be presented. 
Further details will be provided in the SAP. 
14.5 
Handling of protocol deviations 
Important protocol deviations are identified as part of the data cleaning process in the Protocol 
Deviation Specification (PDS). Ongoing data cleaning meetings will be held throughout the 
duration of the study. Objectives of these meetings include to review and update (if necessary) 
the important protocol deviations in the PDS and discuss criteria for exclusion of study 
participants from analysis populations. Furthermore, overall trends in protocol deviations will be 
discussed at the Data Evaluation Meeting. Through this ongoing data cleaning and evaluation 
process, all decisions regarding important protocol deviations and exclusions from analysis 
populations are made on an ongoing basis and will be finalized before database lock. 
14.6 
Handling of dropouts or missing data 
For study participants who prematurely withdraw from the study, no imputation of missing 
efficacy data will be performed. 
Details of how missing or partial dates for safety assessments will be handled will be provided in 
the SAP.  


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14.7 
Planned interim analysis and data monitoring 
A DMC will systematically monitor and report on the progress, safety, and/or critical efficacy 
endpoints of this study by convening to review the ongoing program safety and efficacy data. 
The DMC will also review the impact of infusion rate on local and systemic tolerability during 
the first 2 doses. 
A DMC charter will define the composition, roles, and responsibilities of the DMC, specify the 
data to be reviewed and determine the procedures to be followed. Ad hoc DMC meetings can be 
held for reasons determined appropriate by the sponsor. 
The deliberations and decisions of the DMC will be formally documented. 
A detailed description of the DMC composition, processes, and responsibilities will be provided 
in a separate DMC charter. 
Two interim analyses will be conducted. Firstly at the time of the lock of the parent study 
CIPD01 and additionally once all study participants have completed Part 1 of the Treatment 
Period in CIDP04 and have either entered into the Treatment Period Part 2 or the Observation 
Period. Details of the planned analyses will be fully outlined in the respective SAPs. 
14.8 
Determination of sample size 
No formal sample size calculation can be performed. All study participants from the parent study 
(eg, CIDP01, with a maximum of 
 study participants to be randomized) eligible for the OLE 
will be included. 
15 
ETHICS AND REGULATORY REQUIREMENTS 
15.1 
Informed consent 
Study 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 study participant in both oral and written form by the 
investigator (or designee). Each study 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, or his/her legal representative, and by the person who conducted the informed 
consent discussion (investigator or designee). The study participant or his/her legal 
representative must receive a copy of the signed and dated ICF. As part of the consent process, 
each study participant must consent to direct access to his/her medical records for study-related 
monitoring, auditing, IRB/IEC review, and regulatory inspection. 
If the ICF is amended during the study, the investigator (or the sponsor, if applicable) must 
follow all applicable regulatory requirements pertaining to the approval of the amended ICF by 
the IRB/IEC and use of the amended form. 
All studies conducted at centers in the United States must include the use of a Health Insurance 
Portability and Accountability Act Authorization form. 


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The study participant may withdraw his/her consent to participate in the study at any time. A 
study 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 study 
participant, without having obtained his/her written consent to participate in the study. 
Any data already generated on the samples will be retained and used, but no further laboratory 
analysis will occur. 
15.2 
Study participant identification cards 
Upon signing the Informed Consent and Assent form (as applicable), the study participant or 
legal representative will be provided with a study participant identification card in the language 
of the study participant. The investigator will fill in the study participant identifying information 
and medical emergency contact information. The investigator will instruct the study participant 
to keep the card with him/her at all times. 
15.3 
Institutional Review Boards and Independent Ethics 
Committees 
The study will be conducted under the auspices of an IRB/IEC, as defined in local regulations, 
ICH-GCP, and in accordance with the ethical principles that have their origin in the Declaration 
of Helsinki. 
The investigator/UCB will ensure that an appropriately constituted IRB/IEC that complies with 
the requirements of the current ICH-GCP version or applicable country-specific regulations will 
be responsible for the initial and continuing review and approval of the clinical study. Prior to 
initiation of the study, the investigator/UCB will forward copies of the protocol, ICF, IB, 
investigator’s curriculum vitae (if applicable), advertisement (if applicable), and all other study 
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 human study participants or others, and any protocol 
deviations, to eliminate immediate hazards to study 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 study 
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 study 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. 


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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. 
15.4 
Study participant privacy 
UCB staff (or designee) will affirm and uphold the study participant’s confidentiality. 
Throughout this study, all data forwarded to UCB (or designee) will be identified only by the 
study participant number assigned at Screening. 
The investigator agrees that representatives of UCB, its designee, representatives of the relevant 
IRB/IEC, or representatives of regulatory authorities will be allowed to review that portion of the 
study 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 study participant’s study participation, and autopsy 
reports for deaths occurring during the study). 
15.5 
Protocol amendments 
Protocol changes may affect the legal and ethical status of the study and may also affect the 
statistical evaluations of sample size and the likelihood of the study fulfilling its primary 
objective. 
Significant changes to the protocol will only be made as an amendment to the protocol and must 
be approved by UCB, the IRB/IEC, and the regulatory authorities (if required), prior to being 
implemented. 
16 
FINANCE, INSURANCE, AND PUBLICATION 
Insurance coverage will be handled according to local requirements. 
Finance, insurance, and publication rights are addressed in the investigator and/or CRO 
agreements, as applicable. 
17 
REFERENCES 
American Psychiatric Association. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. 
Arlington, VA: American Psychiatric Association; 2013.  
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. 
Breiner A, Barnett C, Bril V. INCAT Disability Score: A critical analysis of its measurement 
properties. Muscle Nerve. 2014;50:164-9. 
CPMP/ICH/135/95 Note for guidance on Good Clinical Practice (EMEA) Jul 2002. 
Guptill JT, Bromberg MB, Zhu L, Sharma BK, Thompson AR, Krueger A, et al. Patient 
demographics and health plan paid costs in chronic inflammatory demyelinating polyneuropathy. 
Muscle Nerve. 2014;50(1):47-51. 
Hughes RA, Donofrio P, Bril V, Dalakas MC, Deng C, Hanna K, et al. Intravenous immune 
globulin (10% caprylate-chromatography purified) for the treatment of chronic inflammatory 


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application and any extensions or variations thereof. 
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16 Mar 2020 
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Page 101 of 160 
demyelinating polyradiculoneuropathy (ICE study): a randomised placebo-controlled trial. 
Lancet Neurol. 2008;7(2):136-44. 
Léger JM, De Bleecker JL, Sommer C, Robberecht W, Saarela M, Kamienowski J, et al. 
Efficacy and safety of Privigen(®) in patients with chronic inflammatory demyelinating 
polyneuropathy: results of a prospective, single-arm, open-label Phase III study (the PRIMA 
study). J Peripher Nerv Syst. 2013;18(2):130-40. 
Mahdi-Rogers M, McCrone P, Hughes, RA. Economic costs and quality of life in chronic 
inflammatory neuropathies in southeast England. Eur J Neurol. 2014;21:34-9. 
Mathey EK, Park SB, Hughes RA, Pollard JD, Armati PJ, Barnett MH, et al. Chronic 
inflammatory demyelinating polyradiculoneuropathy: from pathology to phenotype. J Neurol 
Neurosurg Psychiatry. 2015;86(9):973-85. 
Merkies IS, Hughes RA, Donofrio P, Bril V, Dalakas MC, Hanna K, et al. Understanding the 
consequences of chronic inflammatory demyelinating polyradiculoneuropathy from impairments 
to activity and participation restrictions and reduced quality of life: the ICE study. J Peripher 
Nerv Syst. 2010;15:208-15. 
Merkies IS, Schmitz PI, van der Meché FG, Samijn JP, van Doorn PA. Inflammatory 
Neuropathy Cause and Treatment (INCAT) group. Clinimetric evaluation of a new overall 
disability scale in immune mediated polyneuropathies. J Neurol Neurosurg Psychiatry. 
2002;72(5):596-601. 
National Institutes for Health. Common Terminology Criteria for Adverse Events (CTCAE) 
Version 5.0. US Department of Health and Human Services.  
Olesen J, Gustavsson A, Svensson M, Wittchen HU, Jönsson B. The economic cost of brain 
disorders in Europe. Eur J Neurol. 2012;19(1):155-62. 
Sampson HA, Muñoz-Furlong A, Campbell RL, Adkinson NF Jr, Bock SA, Branum A, et al. 
Second symposium on the definition and management of anaphylaxis: summary report - Second 
National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network 
symposium. J Allergy Clin Immunol. 2006;117(2):391-7. 
Vanhoutte EK, Faber CG, van Nes SI, Jacobs BC, van Doorn PA, van Koningsveld R, et al. 
Modifying the Medical Research Council grading system through Rasch analyses. Brain. 
2012;135:1639-49. 
van Nes SI, Vanhoutte EK, van Doorn PA, Hermans M, Bakkers M, Kuitwaard K, et al. 
Rasch-built Overall Disability Scale (R-ODS) for immune-mediated peripheral neuropathies. 
Neurology. 2011;76:337–45. 
World Health Organization, Treatment of Tuberculosis: Guidelines, 4th edition. 2010 
http://www.who.int/tb/publications/tb_treatmentguidelines/en/. 


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18 
APPENDICES 
18.1 
Suggested management guidelines for infusion reactions 
Table 18-1: Suggested management guidelines for infusion reactions 
Type of reaction 
Sponsor recommendations for management 
Acute – Mild 
eg, flushing, dizziness, 
headache, sweating, 
palpitations, nausea 
Slow 
 
 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 10 minutes until back to Baseline. 
 
 as tolerated until intended dose has been given. 
Acute – Moderate 
eg, flushing, chest tightness, 
dyspnea, hypo/hypertension 
(change >20mmHg in 
systolic blood pressure), 
raised temperature, 
palpitations, urticaria 
Stop infusion. 
 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 5 minutes until back to Baseline. 
 
 
 
 following this suggested regimen: 
 
), as tolerated until intended dose 
has been given. 
Acute – Severe 
eg, hypo/hypertension 
(change >40mmHg in 
systolic blood pressure), 
raised temperature with 
rigors, chest tightness, 
dyspnea with wheezing, 
stridor 
Stop infusion definitively. 
Alert crash team. 
Maintain airway, ensure oxygen is available. 
If wheezing, give epinephrine 0.5mg im (0.5mL 1:1000 epinephrine). 
Administer antihistamine iv/im. 
Administer corticosteroids iv. 
Monitor vital signs every 2 minutes until back to Baseline. 
im=intramuscular; iv=intravenous 


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18.2 
Diagnosis of anaphylactic reactions 
Anaphylaxis is highly likely when any of the following 3 criteria is fulfilled 
(Sampson et al, 2006): 
1. Acute onset of an illness (minutes to several hours) with involvement of the skin, mucosal 
tissue, or both (eg, generalized hives, pruritus or flushing, swollen lips-tongue-uvula) 
AND AT LEAST ONE OF THE FOLLOWING 
a. Respiratory compromise (eg, dyspnea, wheeze-bronchospasm, stridor, reduced peak 
expiratory flow [PEF], hypoxemia) 
b. Reduced blood pressure [BP] or associated symptoms of end-organ dysfunction (eg, 
hypotonia [collapse], syncope, incontinence) 
2. Two or more of the following that occur rapidly after exposure to a likely allergen for that 
patient (minutes to several hours): 
a. Involvement of the skin-mucosal tissue (eg, generalized hives, itch-flush, swollen lips 
tongue-uvula) 
b. Respiratory compromise (eg, dyspnea, wheeze-bronchospasm, stridor, reduced PEF, 
hypoxemia) 
c. Reduced BP or associated symptoms (eg, hypotonia [collapse], syncope, incontinence) 
d. Persistent gastrointestinal symptoms (eg, crampy abdominal pain, vomiting) 
Reduced blood pressure 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. 


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18.3 
Protocol amendment 1 
Rationale for the amendment 
The current protocol has been locally amended to align with CIPD01 Protocol Amendment 1.1, 
modifying the timeframe for use of contraception post study completion, and the final urinary 
pregnancy test of the study.  
The following shows the changes made in Protocol Amendment 1 compared to the original 
Protocol, dated 14 Dec 2018. 
Modifications and changes 
Specific changes 
Change #1 
5.2  
Schedule of study assessments 
Table 5-1 Schedule of study assessments; footnote k 
In case of immediate entry from the parent study, the serum pregnancy test from the parent study 
should not be older than 5 weeks prior to entry into this CIDP04 study and the urine pregnancy 
test done at Visit 2 must both be negative before dosing. In case of gap period between the parent 
study and entry in CIDP04, a serum test will be performed at Visit 1 (Screening). Note that the 
final urine pregnancy test of the study should be no longer than 60 days after the final dose of 
IMP. 
Has been changed to:  
In case of immediate entry from the parent study, the serum pregnancy test from the parent study 
should not be older than 5 weeks prior to entry into this CIDP04 study and the urine pregnancy 
test done at Visit 2 must both be negative before dosing. In case of gap period between the parent 
study and entry in CIDP04, a serum test will be performed at Visit 1 (Screening). Note that the 
final urine pregnancy test of the study should be no longer than 6090 days after the final dose of 
IMP. 
Change #2 
6.1  
Inclusion criteria 
4. Female subjects of child-bearing potential must have the results of a negative serum 
pregnancy test from either the parent study (not older than 5 weeks) or the Screening Visit 
available at the CIDP04 Baseline (Visit 2), and a urine pregnancy test must be negative when 
subject enters CIDP04 and prior to further dosing at each study visit thereafter. 
Female subjects of childbearing potential must agree to use a highly effective method of birth 
control, during the study and for a period of 2 months after their final dose of IMP. Highly 
effective forms of birth control are methods that achieve a failure rate of less than 1% per 
year when used consistently and correctly. According to the International Council for 
Harmonisation (ICH) M3 R2, highly effective methods of birth control include: 
 Combined (estrogen- and progesterone-containing) hormonal contraception (oral, 
implant, injectable) associated with inhibition of ovulation (which must be stable for at 


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least 1 full month prior to Screening [Visit 1], or must have remained stable during the 
parent study for subjects entering CIDP04 without a Screening Visit, and should remain 
stable during the study). 
 Progesterone-only hormonal contraceptives (oral, implant, injectable) associated with 
inhibition of ovulation (which must be stable for at least 1 full month prior to Screening 
[Visit 1], or must have remained stable during the parent study for subjects entering 
CIDP04 without a Screening Visit, and should remain stable during the study). 
 Progesterone-releasing intrauterine systems or the TCu 380A intrauterine device. 
 Vasectomized partner (provided sole partner and partner has medical proof of surgical 
success). 
 True heterosexual sexual abstinence is an acceptable form of contraception when this is 
in line with the preferred and usual lifestyle of the person. Periodic abstinence 
(eg, calendar, ovulation, symptothermal, postovulation methods), declaration of 
abstinence for the duration of the study, and withdrawal are not acceptable methods of 
contraception. 
 Women not agreeing to use birth control must be of nonchildbearing potential, defined as 
being: 
 Postmenopausal (for at least 2 years before the Screening Visit of CIDP04 or the parent 
study if no Screening Visit is scheduled in CIDP04), verified by serum 
follicle-stimulating hormone level >40mIU/mL at the Screening Visit, or 
 Permanently sterilized (eg, bilateral tubal occlusion, hysterectomy, bilateral 
salpingectomy), or 
 Congenitally sterile 
Has been changed to:  
4. Female subjects of child-bearing potential must have the results of a negative serum 
pregnancy test from either the parent study (not older than 5 weeks) or the Screening Visit 
available at the CIDP04 Baseline (Visit 2), and a urine pregnancy test must be negative when 
subject enters CIDP04 and prior to further dosing at each study visit thereafter. 
Female subjects of childbearing potential must agree to use a highly effective method of birth 
control, during the study and for a period of 23 months after their final dose of IMP. Highly 
effective forms of birth control are methods that achieve a failure rate of less than 1% per 
year when used consistently and correctly. According to the International Council for 
Harmonisation (ICH) M3 R2, highly effective methods of birth control include: 
 Combined (estrogen- and progesterone-containing) hormonal contraception (oral, 
implant, injectable) associated with inhibition of ovulation (which must be stable for at 
least 1 full month prior to Screening [Visit 1], or must have remained stable during the 
parent study for subjects entering CIDP04 without a Screening Visit, and should remain 
stable during the study). 
 Progesterone-only hormonal contraceptives (oral, implant, injectable) associated with 
inhibition of ovulation (which must be stable for at least 1 full month prior to Screening 


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[Visit 1], or must have remained stable during the parent study for subjects entering 
CIDP04 without a Screening Visit, and should remain stable during the study). 
 Progesterone-releasing intrauterine systems or the TCu 380A intrauterine device. 
 Vasectomized partner (provided sole partner and partner has medical proof of surgical 
success). 
 True heterosexual sexual abstinence is an acceptable form of contraception when this is 
in line with the preferred and usual lifestyle of the person. Periodic abstinence 
(eg, calendar, ovulation, symptothermal, postovulation methods), declaration of 
abstinence for the duration of the study, and withdrawal are not acceptable methods of 
contraception. 
 Women not agreeing to use birth control must be of nonchildbearing potential, defined as 
being: 
 Postmenopausal (for at least 2 years before the Screening Visit of CIDP04 or the parent 
study if no Screening Visit is scheduled in CIDP04), verified by serum 
follicle-stimulating hormone level >40mIU/mL at the Screening Visit, or 
 Permanently sterilized (eg, bilateral tubal occlusion, hysterectomy, bilateral 
salpingectomy), or 
 Congenitally sterile 
Change #3 
9.2 Laboratory measurements 
Table 9-3 Laboratory measurements; footnote e 
Urine pregnancy test (dipstick) for women of childbearing potential performed prior to dosing 
(and confirmed negative) at dosing visits and each visit of the Observation Period. Note that the 
final urinary pregnancy test of the study should be no longer than 60 days after the final dose of 
IMP. 
Has been changed to:  
Urine pregnancy test (dipstick) for women of childbearing potential performed prior to dosing 
(and confirmed negative) at dosing visits and each visit of the Observation Period. Note that the 
final urinary pregnancy test of the study should be no longer than 6090 days after the final dose 
of IMP. 
 
18.4 
Protocol amendment 2 
Rationale for the amendment  
The purpose of this substantial protocol amendment is to provide clarification on the dose of 
investigational product administered (including an allowance of ±10% compared to the 
 
target dose arm), as well as a flexible infusion rate. Sensitivity analyses were introduced to 
account for deviation outside the 10% target dosage and the descriptive analyses will inform 


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about actual doses administered to the subjects. Updates to the status of the other 
rozanolixizumab studies have been included. The predominance of objective criteria over the 
investigator’s judgement has been confirmed for the assessment of CIDP relapse. The timeframe 
of expected use of contraception post-study completion has been extended to 90 days in view of 
the probable half-life of rozanolixizumab. Exclusion criterion #14 has been extended to 
prediabetic condition. The expectation with regards to the use of cannabidiols and medicinal 
marijuana has been clarified in the concomitant medication section. The protocol amendment 
confirms the expectation of a single rater for the INCAT assessment to ensure consistency of the 
rating during the course of the study. The exit interview assessment has been removed from the 
follow-up study; this will allow UCB to confirm the adequacy of this tool using data collected in 
CIDP01 before extending its use in another study. Safety reporting procedures have been 
updated to align with most current UCB practices. 
In addition, some administrative changes have been made. 
Modifications and changes 
Global changes 
Text pertaining to the exit interview has been removed from the entire protocol and is not listed 
in the specific changes. 
Specific changes  
Change #1 
1 Summary; fourth paragraph 
The following sentence has been added to the end of the paragraph: 
For exact doses to be administered, refer to Section 7.2. 
Change #2 
2 Introduction; fifth and sixth paragraphs 
Rozanolixizumab is a humanized anti-FcRn monoclonal antibody that has been specifically 
designed to inhibit IgG binding to FcRn without inhibiting albumin binding to FcRn. 
Rozanolixizumab is being developed as an inhibitor of FcRn activity with the aim to reduce the 
concentration of pathogenic IgG in patients with IgG auto-antibody mediated diseases. 
Rozanolixizumab was derived from a rat antibody with specificity for human FcRn. To date, 
rozanolixizumab has been administered to healthy subjects in a completed first-in-human (FIH) 
study (UP0018), an ongoing Phase 2 study in subjects with primary immune thrombocytopenia 
(ITP) (TP0001, evaluating 
 by sc route across 5 cohorts), and an 
ongoing Phase 2 study in subjects with myasthenia gravis (MG) (MG0002, evaluating 
 
 by sc route). 
Additional information on the development of rozanolixizumab, including data from UP0018 
and the ongoing clinical studies in ITP subjects (TP0001) and MG subjects (MG0002), can be 
found in the current version of the Investigator's Brochure (IB) for rozanolixizumab. 
Have been changed to: 


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Rozanolixizumab is a humanized anti-FcRn monoclonal antibody that has been specifically 
designed to inhibit IgG binding to FcRn without inhibiting albumin binding to FcRn. 
Rozanolixizumab is being developed as an inhibitor of FcRn activity with the aim to reduce the 
concentration of pathogenic IgG in patients with IgG auto-antibody mediated diseases. 
Rozanolixizumab was derived from a rat antibody with specificity for human FcRn. To date, 
rozanolixizumab has been administered to healthy subjects in a completed first-in-human (FIH) 
study (UP0018), a completed Phase 2 study in subjects with primary immune thrombocytopenia 
(ITP) (TP0001, evaluating 
 by sc route across 5 cohorts), and a 
completed Phase 2 study in subjects with myasthenia gravis (MG) (MG0002, evaluating 
 
 by sc route). 
Additional information on the development of rozanolixizumab, including data from UP0018 
and the ongoing clinical studies in ITP subjects (TP0001) and MG subjects (MG0002), can be 
found in the current version of the Investigator's Brochure (IB) for rozanolixizumab. 
Change #3 
5.1 Study description; third paragraph 
The following sentence has been added to the end of the paragraph: 
See Section 7.2 for details on treatment to be administered. 
Change #4 
5.2 Schedule of study assessments 
Table 5-1 Schedule of study assessments; footnote k 
In case of immediate entry from the parent study, the serum pregnancy test from the parent study 
should not be older than 5 weeks prior to entry into this CIDP04 study and the urine pregnancy 
test done at Visit 2 must both be negative before dosing. In case of gap period between the parent 
study and entry in CIDP04, a serum test will be performed at Visit 1 (Screening). Note that the 
final urine pregnancy test of the study should be no longer than 60 days after the final dose of 
IMP. 
Has been changed to: 
In case of immediate entry from the parent study, the serum pregnancy test from the parent study 
should not be older than 5 weeks prior to entry into this CIDP04 study and the urine pregnancy 
test done at Visit 2 must both be negative before dosing. In case of gap period between the parent 
study and entry in CIDP04, a serum test will be performed at Visit 1 (Screening). Pregnancy 
testing will consist of urine testing at dosing visits during the Treatment Period and each 
visit during the Observation Period. A positive urine pregnancy test must be confirmed 
using a serum pregnancy test. Note that the final urine pregnancy test of the study should be no 
longer than 90 days after the final dose of IMP. 
Change #5 
5.2 Schedule of study assessments 
Table 5-1 Schedule of study assessments; footnote r 


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At Visits 2, 4, 8, 10, 14, 18, and 22, samples for exploratory biomarkers should be taken predose. 
See Table 5-2 for sampling in case of AE of interest. 
Has been changed to: 
At Visits 2, 3, 8, 10, 14, 18, and 22, samples for exploratory biomarkers should be taken predose. 
See Table 5-2 for sampling in case of AE of interest. 
Change #6 
5.3 Rationale for study design and selection of dose; second paragraph, fourth sentence 
The dose and regimen of IMP to be used in the current study (rozanolixizumab 
 sc) is 
based on results from the FIH study UP0018, alongside the emerging safety data from the MG 
and ITP studies (MG0002 and TP0001).  
Has been changed to: 
The dose and regimen of IMP to be used in the current study (rozanolixizumab 
 sc) is 
based on results from the FIH study UP0018, alongside the emerging safety data from the MG 
and ITP studies (MG0002 and TP0001).  
Change #7 
6.1  
Inclusion criteria; Criterion #4 
Female subjects of child-bearing potential must have the results of a negative serum pregnancy 
test from either the parent study (not older than 5 weeks) or the Screening Visit available at the 
CIDP04 Baseline (Visit 2), and a urine pregnancy test must be negative when subject enters 
CIDP04 and prior to further dosing at each study visit thereafter. 
Female subjects of childbearing potential must agree to use a highly effective method of birth 
control, during the study and for a period of 2 months after their final dose of IMP. Highly 
effective forms of birth control are methods that achieve a failure rate of less than 1% per year 
when used consistently and correctly. According to the International Council for Harmonisation 
(ICH) M3 R2, highly effective methods of birth control include: 
 Combined (estrogen- and progesterone-containing) hormonal contraception (oral, implant, 
injectable) associated with inhibition of ovulation (which must be stable for at least 1 full 
month prior to Screening [Visit 1], or must have remained stable during the parent study for 
subjects entering CIDP04 without a Screening Visit, and should remain stable during the 
study). 
 Progesterone-only hormonal contraceptives (oral, implant, injectable) associated with 
inhibition of ovulation (which must be stable for at least 1 full month prior to Screening 
[Visit 1], or must have remained stable during the parent study for subjects entering CIDP04 
without a Screening Visit, and should remain stable during the study). 
 Progesterone-releasing intrauterine systems or the TCu 380A intrauterine device. 
 Vasectomized partner (provided sole partner and partner has medical proof of surgical 
success). 


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 True heterosexual sexual abstinence is an acceptable form of contraception when this is in 
line with the preferred and usual lifestyle of the person. Periodic abstinence (eg, calendar, 
ovulation, symptothermal, postovulation methods), declaration of abstinence for the duration 
of the study, and withdrawal are not acceptable methods of contraception. 
 Women not agreeing to use birth control must be of nonchildbearing potential, defined as 
being: 
 Postmenopausal (for at least 2 years before the Screening Visit of CIDP04 or the parent study 
if no Screening Visit is scheduled in CIDP04), verified by serum follicle-stimulating 
hormone level >40mIU/mL at the Screening Visit, or 
 Permanently sterilized (eg, bilateral tubal occlusion, hysterectomy, bilateral salpingectomy), 
or 
 Congenitally sterile 
Has been changed to:  
Female subjects of child-bearing potential must have the results of a negative serum pregnancy 
test from either the parent study (not older than 5 weeks) or the Screening Visit available at the 
CIDP04 Baseline (Visit 2), and a urine pregnancy test must be negative when subject enters 
CIDP04 and prior to further dosing at each study visit thereafter. 
Female subjects of childbearing potential must agree to use a highly effective method of birth 
control, during the study and for a period of 3 months after their final dose of IMP. Highly 
effective forms of birth control are methods that achieve a failure rate of less than 1% per year 
when used consistently and correctly. According to the International Council for Harmonisation 
(ICH) M3 R2, highly effective methods of birth control include: 
 Combined (estrogen- and progesterone-containing) hormonal contraception (oral, implant, 
injectable) associated with inhibition of ovulation (which must be stable for at least 1 full month 
prior to Screening [Visit 1], or must have remained stable during the parent study for subjects 
entering CIDP04 without a Screening Visit, and should remain stable during the study). 
 Progesterone-only hormonal contraceptives (oral, implant, injectable) associated with inhibition 
of ovulation (which must be stable for at least 1 full month prior to Screening [Visit 1], or must 
have remained stable during the parent study for subjects entering CIDP04 without a Screening 
Visit, and should remain stable during the study). 
 Progesterone-releasing intrauterine systems or the TCu 380A intrauterine device. 
 Vasectomized partner (provided sole partner and partner has medical proof of surgical success). 
 True heterosexual sexual abstinence is an acceptable form of contraception when this is in line 
with the preferred and usual lifestyle of the person. Periodic abstinence (eg, calendar, ovulation, 
symptothermal, postovulation methods), declaration of abstinence for the duration of the study, 
and withdrawal are not acceptable methods of contraception. 
 Women not agreeing to use birth control must be of nonchildbearing potential, defined as being: 


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 Postmenopausal (for at least 2 years before the Screening Visit of CIDP04 or the parent study if 
no Screening Visit is scheduled in CIDP04), verified by serum follicle-stimulating hormone level 
>40mIU/mL at the Screening Visit, or 
 Permanently sterilized (eg, bilateral tubal occlusion, hysterectomy, bilateral salpingectomy), or 
 Congenitally sterile 
Change #8 
6.2.1 Additional exclusion criteria for subjects with a gap period between the parent study and 
entry in CIDP04; Criterion #14 
Subject has a current diagnosis or has a history of Type 1 or Type 2 diabetes mellitus. 
Has been changed to: 
Subject has a current diagnosis or has a history of Type 1 or Type 2 diabetes mellitus and/or 
hemoglobin A1c level >6.0%. 
Change #9 
6.2.1 Additional exclusion criteria for subjects with a gap period between the parent study and 
entry in CIDP04; Criterion #21 
Subject has a history of alcohol use disorder or other substance use disorder within 12 months of 
Screening Visit. 
Has been changed to: 
Subject has a history of alcohol use disorder or other substance use disorder (as per Diagnostic 
and Statistical Manual of Mental Disorders-5 [American Psychiatric Association, 2013]) 
within 12 months of Screening Visit. 
Change #10 
6.4 Withdrawal criteria; Subjects may be discontinued from IMP section, Criterion #2 
Subject experiences a severe AE of headache that is considered related to the IMP in the opinion 
of the investigator (Table 5 2). Following an event of a severe headache, a subject may continue 
participation in the study if the subject is willing to do so and the investigator, Medical Monitor, 
and Study Physician agree that the subject’s continuation in the study poses no significant risk 
for the subject. The use of symptomatic headache treatment is allowed at the discretion of the 
investigator. The IMP dose can be reduced if the headache persists despite symptomatic 
treatment. The IMP dose may be reduced to 
 (refer to Section 9.1.10 for details on the 
management of headaches). 
Has been changed to: 
Subject experiences a severe AE of headache that is considered related to the IMP in the opinion 
of the investigator (Section 9.1.10). Following an event of a severe headache, a subject may 
continue participation in the study if the subject is willing to do so and the investigator, Medical 
Monitor, and Study Physician agree that the subject’s continuation in the study poses no 
significant risk for the subject. The use of symptomatic headache treatment is allowed at the 
discretion of the investigator. The IMP dose can be reduced if the headache persists despite 


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symptomatic treatment. The IMP dose may be reduced to 
 (refer to Section 9.1.10 for 
details on the management of headaches and Section 7.2 for treatment to be administered). 
Change #11 
7.2 Treatment to be administered 
The initial dose administered will be based on the dose at the time of completion of the 
Treatment Period from the parent study (eg, CIDP01). For subjects who had a gap period 
between the parent study (eg, CIDP01) and CIDP04, a starting dose of 
 will be used. 
The dose used in the study may be reduced based on individual tolerability to 
(eg, severe 
headache) but the maximum dose used will be 
. 
Has been changed to: 
The initial dose administered will be based on the dose at the time of completion of the 
Treatment Period from the parent study (eg, CIDP01). For subjects who had a gap period 
between the parent study (eg, CIDP01) and CIDP04, a starting dose of 
 will be used (see 
Table 7-1). The dose used in the study may be reduced based on individual tolerability to 
 (see Table 7-2) (eg, severe headache) but the maximum dose used will be 
. 
Change #12 
7.2 Treatment to be administered 
The following 2 tables have been added and the subsequent table has been 
renumbered sequentially: 
Table 7-1: 
IMP doses to be administered (equivalent to approximately 
) 
by body weight 
Body weight ranges 
IMP doses to be administered (equivalent to 
approximately 
a) 
IMP volume to be 
administered 
≥40 to <49kg 
 
 
≥49 to <63kg 
 
 
≥63 to <77kg 
 
 
≥77 to <91kg 
 
 
≥91 to <105kg 
 
 
≥105 to <119kg 
 
 
≥119 to <133kg 
 
 
≥133 to <147kg 
 
 
≥147 to <161kg 
 
 
≥161 to 170kg 
 
 
IMP= investigational medicinal product 
a  Doses administered will be ±10% of the intended dose, except for subjects with a body weight of 47 to 50kg 
and 63kg. 


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Table 7-2: 
IMP doses to be administered (equivalent to approximately 
) 
by body weight 
Body weight ranges 
IMP doses to be administered (equivalent to 
approximately 
) 
IMP volume to be 
administered 
≥40 to <49kg 
 
 
≥49 to <69kg 
 
 
≥69 to <89kg 
 
 
≥89 to <109kg 
 
 
≥109 to <129kg 
 
 
≥129 to <149kg 
 
 
≥149 to <169kg 
 
 
≥169 to 170kg 
 
 
IMP= investigational medicinal product  
Change #13 
7.2 Treatment to be administered; fifth paragraph 
The IMP will be administered as a sc infusion using a syringe pump. 
 
 
 
 
. 
Has been changed to: 
The IMP will be administered as a sc infusion using a syringe pump. 
 
 
 
 
 
 
Change #14 
7.8.1 Permitted concomitant treatments (medications and therapies) 
The following paragraph has been added: 
The use of cannabidiols and medicinal marijuana (prescribed by a physician) is also 
permitted. When applicable, the subject must be on a stable dose of cannabidiols and/or 
medicinal marijuana for 
 prior to Screening Visit and remain stable for the 
duration of the study. 
Change #15 
7.8.3 Rescue medication; first sentence 


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If, at any time during this OLE study, a subject relapses according to the medical judgement of 
the investigator supported by, eg, subject’s score on iRODS, INCAT, or maximum grip strength 
as assessed by the site personnel, then rescue therapy must be considered and the subject 
withdrawn from IMP (see Section 6.4). 
Has been changed to: 
If, at any time during this OLE study, a subject relapses according to the predefined criteria for 
relapse as specified in Section 4.2.1 using the subject’s score on iRODS, INCAT, or maximum 
grip strength as assessed by the site personnel and supported by the medical judgement of the 
investigator, then rescue therapy must be considered and the subject withdrawn from IMP (see 
Section 6.4). 
Change #16 
8.1 Screening, Visit 1 (Weeks -5 to -2); second sentence  
The following procedures will be performed at the Screening Visit: 
Has been changed to: 
The following procedures will be performed at the Screening Visit (or as close as possible to 
the Screening date): 
Change #17 
9.1.1.4 Adverse events of interest; second paragraph 
These events should be reported to UCB within 24h, regardless of seriousness, using the fax and 
email details for AEs of interest and independent of SAE reporting. 
Has been changed to: 
These events should be reported to UCB within 24h, regardless of seriousness, by completing 
the eCRF. If the electronic system is unavailable for more than 24 hours, then the site will 
use the paper SAE data collection tool (see Section 9.1.2.3 for the reporting process). 
Change #18 
9.1.2.3 Additional procedures for reporting serious adverse events 
If an SAE is reported, UCB must be informed within 24h of receipt of this information by the 
site (see contact information for SAE reporting listed in the Serious Adverse Event Reporting 
section at the front of the protocol). The investigator must forward to UCB (or its representative) 
a duly completed “Investigator SAE Report Form for Development Drug” (SAE report form) 
provided by UCB, even if the data are incomplete, or if it is obvious that more data will be 
needed in order to draw any conclusions. Information recorded on this form will be entered into 
the global safety database. 
An investigator SAE report form will be provided to the investigator. The investigator SAE 
Report form must be completed in English. 
It is important for the investigator, when completing the SAE report form, to include the 
assessment as to a causal relationship between the SAE and the IMP administration. This insight 


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from the investigator is very important for UCB to consider in assessing the safety of the IMP 
and in determining whether the SAE requires reporting to the regulatory authorities in an 
expedited manner. 
Additional information (eg, autopsy or laboratory reports) received by the investigator must be 
provided within 24h. All documents in the local language must be accompanied by a translation 
in English, or the relevant information included in the same document must be summarized in 
the investigator SAE report form. 
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 subject, and to also inform participating subjects 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. 
Upon receipt of the SAE report form, UCB will perform an assessment of expectedness of the 
reported SAE. The assessment of the expectedness of the SAE is based on the IB. 
Has been changed to: 
If an SAE is reported, UCB must be informed within 24h of receipt of this information by the 
site. The primary mechanism for reporting an SAE to UCB will be the eCRF (using the 
eCRF SAE page). If the electronic system is unavailable for more than 24 hours, the 
investigator must forward to UCB (or its representative) a duly completed paper SAE data 
collection tool provided by UCB, even if the data are incomplete, or if it is obvious that more 
data will be needed in order to draw any conclusions (see contact information for SAE 
reporting listed in the Serious Adverse Event Reporting section at the front of the 
protocol). The site will enter the SAE data into the electronic system as soon as it becomes 
available. 
An investigator SAE report form will be provided to the investigator. The investigator SAE 
Report form must be completed in English. 
It is important for the investigator, when completing the SAE data collection tool, to include the 
assessment as to a causal relationship between the SAE and the IMP administration. This insight 
from the investigator is very important for UCB to consider in assessing the safety of the IMP 
and in determining whether the SAE requires reporting to the regulatory authorities in an 
expedited manner. 
Additional information (eg, autopsy or laboratory reports) received by the investigator must be 
provided within 24h. All documents in the local language must be accompanied by a translation 
in English, or the relevant information included in the same document must be summarized in 
the investigator SAE data collection tool. 
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 subject, and to also inform participating subjects 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. 


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Upon receipt of the SAE data collection tool, UCB will perform an assessment of expectedness 
of the reported SAE. The assessment of the expectedness of the SAE is based on the IB. 
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 subject 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. 
Change #19 
9.1.2.4 Immediate reporting of adverse events; first sentence 
The following AEs must be reported immediately using the SAE Report Form according to the 
procedure in Section 9.1.2.3: 
Has been changed to: 
The following AEs must be reported immediately using the SAE data collection tool according 
to the procedure in Section 9.1.2.3: 
Change #20 
9.1.4 Pregnancy; final paragraph, final sentence 
Those SAEs must be additionally reported using the investigator SAE Report form. 
Has been changed to: 
Those SAEs must be additionally reported using the investigator SAE data collection tool. 
Change #21 
9.1.6 Overdose of investigational medicinal product; first sentence 
Excessive dosing (beyond that prescribed in the protocol and including overdose) should be 
recorded in the eCRF 
Has been changed to: 
Excessive dosing (beyond that prescribed in the protocol and including overdose ie, >10% 
above 
) should be recorded in the eCRF. 
Change #22 
9.2 Laboratory measurements 
Table 9-3 Laboratory measurements; footnote e 
Urine pregnancy test (dipstick) for women of childbearing potential performed prior to dosing 
(and confirmed negative) at dosing visits and each visit of the Observation Period. Note that the 
final urinary pregnancy test of the study should be no longer than 60 days after the final dose of 
IMP. 
Has been changed to:  


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Urine pregnancy test (dipstick) for women of childbearing potential performed prior to dosing 
(and confirmed negative) at dosing visits and each visit of the Observation Period. Note that the 
final urinary pregnancy test of the study should be no longer than 90 days after the final dose of 
IMP. 
Change #23 
9.3.6 Assessment and management of TB and TB risk factors; Monitoring for TB during the 
study, second paragraph, second sentence  
Confirmed LTB, active TB, and NTMB must be reported to the Sponsor immediately regardless 
of seriousness using the SAE Report Form. 
Has been changed to: 
Confirmed LTB, active TB, and NTMB must be reported to the Sponsor immediately regardless 
of seriousness using the SAE data collection tool. 
Change #24 
10.2 INCAT; second paragraph 
The investigator (or qualified personnel) will record the subjects’ adjusted INCAT score at the 
time points detailed in the schedule of study assessments (Table 5-1). 
Has been changed to: 
The investigator (or qualified personnel) will record the subjects’ adjusted INCAT score at the 
time points detailed in the schedule of study assessments (Table 5-1). It is recommended that 
the rater of the scale remains the same throughout the duration of the study to ensure 
consistency of the rating. 
Change #25 
14.3.1 Safety analyses; first paragraph, final sentence 
The action taken, time of onset relative to dosing, and duration of each AE will be listed only. 
Has been changed to: 
The action taken, time of onset relative to dosing, actual dose received, and duration of each AE 
will be listed only. 
Change #26 
14.4.1 Efficacy analyses; fifth paragraph 
Additional sensitivity analyses may be carried out and will be fully detailed in the SAP. 
Has been changed to: 
A sensitivity analysis will be performed excluding any subject who received a mean dose 
more than ±10% from the intended dose. Additional sensitivity analyses may be carried out 
and will be fully detailed in the SAP. 
Change #27 
14.4.2 Pharmacokinetic analyses; second paragraph, first sentence 


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In contrast to the general descriptive display, concentration data will be summarized by time 
point using the number of available observations, mean, median, SD, minimum, maximum, 
geometric mean (and associated 95% CIs), and geometric coefficient of variation (assuming log-
normally distributed data). 
Has been changed to: 
In contrast to the general descriptive display, concentration data will be summarized by actual 
dose received and time point using the number of available observations, mean, median, SD, 
minimum, maximum, geometric mean (and associated 95% CIs), and geometric coefficient of 
variation (assuming log-normally distributed data). 
Change #28 
14.4.3 Pharmacodynamic analyses; first sentence 
For all PD variables, descriptive statistics for the value, change from Baseline, and/or percentage 
change from Baseline will be tabulated by time point. 
Has been changed to: 
For all PD variables, descriptive statistics for the value, change from Baseline, and/or percentage 
change from Baseline will be tabulated by actual dose received and time point. 
Change #29 
14.5 
Handling of protocol deviations; first and third sentences 
Important protocol deviations are identified as part of the data cleaning process in the Data 
Cleaning Plan (DCP). Ongoing data cleaning meetings will be held throughout the duration of 
the study. Objectives of these meetings include to review and update (if necessary) the important 
protocol deviations in the DCP and discuss criteria for exclusion of subjects from analysis 
populations. 
Have been changed to: 
Important protocol deviations are identified as part of the data cleaning process in the Protocol 
Deviation Specification (PDS). Ongoing data cleaning meetings will be held throughout the 
duration of the study. Objectives of these meetings include to review and update (if necessary) 
the important protocol deviations in the PDS and discuss criteria for exclusion of subjects from 
analysis populations. 
Change #30 
17 References 
The following reference has been added: 
American Psychiatric Association. Diagnostic and Statistical Manual of Mental Disorders. 5th 
ed. Arlington, VA: American Psychiatric Association; 2013. 
Change #31 
18 Appendices 
Table 18-1 Suggested management guidelines for infusion reactions, first 2 reactions 


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Type of reaction 
Sponsor recommendations for management 
Acute – Mild 
eg, flushing, dizziness, 
headache, sweating, 
palpitations, nausea 
Slow 
 
Infuse 0.9% sodium chloride 500 to 1000mL/h iv. 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 10 minutes until back to Baseline. 
 
 
as 
tolerated until intended dose has been given. 
Acute – Moderate 
eg, flushing, chest tightness, 
dyspnea, hypo/hypertension 
(change >20mmHg in 
systolic blood pressure), 
raised temperature, 
palpitations, urticaria 
Stop infusion. 
. 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 5 minutes until back to Baseline. 
. 
 
 
 following this suggested regimen: 
Restart 
 
 
as tolerated until 
intended dose has been given. 
Have been changed to: 
Type of reaction 
Sponsor recommendations for management 
Acute – Mild 
eg, flushing, dizziness, 
headache, sweating, 
palpitations, nausea 
Slow infusion rate to 
 
. 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 10 minutes until back to Baseline. 
 
. 
Acute – Moderate 
eg, flushing, chest tightness, 
dyspnea, hypo/hypertension 
(change >20mmHg in 
systolic blood pressure), 
raised temperature, 
palpitations, urticaria 
Stop infusion. 
 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 5 minutes until back to Baseline. 
 
 
 
 following this suggested regimen: 
 


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Type of reaction 
Sponsor recommendations for management 
 intended 
dose has been given. 
 
 
 
 
18.5 
Protocol amendment 3 
Rationale for the amendment  
The purpose of this substantial protocol amendment is to assess the safety and key efficacy of 
rozanolixizumab during an optional additional treatment period (Treatment Period Part 2) of up 
to 52 weeks (or until an Access Program or equivalent is available for rozanolixizumab, 
whichever comes first) on the basis of each study participant’s individual benefit-risk assessment 
at the end of the first 24-week Treatment Period. This amendment is the opportunity to update 
the new legal entity of UCB effective since December 2019. Additional wording pertaining study 
participants being able to self-administer IMP after appropriate training has been included. One 
exclusion criteria has been clarified. The management of hypogammaglobulinemia has been 
adjusted for better control. An additional interim analysis has been added at the time of lock of 
the parent study and another at the end of the first Treatment Period (24 weeks) matching the 
original timepoint of results availability.  
The following shows the changes made in Protocol Amendment 3 compared to the Protocol 
Amendment 2, dated 12 Jul 2019. 
Modifications and changes 
Global changes 
Where applicable, the sponsor company name has changed from “UCB Biopharma SPRL” to 
“UCB Biopharma SRL” since the name of the legal form of the entity UCB Biopharma has 
changed into “société à responsabilité limitée” abbreviated “SRL. This change is not listed in the 
specific changes. 
The term “subject” has been replaced with “study participant” where it has been used in 
reference to an individual who participates in a clinical study and is not listed in the specific 
changes. 
Due to the additional Treatment Period in the study, where applicable, the term “Period” has 
been changed to “Periods” and is not listed in the specific changes. 
Where “anti-rozanolixizumab antibodies” has been used in the protocol, the abbreviation “ADA” 
has been added where applicable and is not listed in the specific changes.  
Specific changes 
Change #1 
Study contact information, sponsor study physician 


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Name: 
 
Address: 
UCB  
Chemin du Foriest 
1420 Braine-l’Alleud 
BELGIUM 
Phone: 
 
Has been changed to: 
Name: 
 
Address: 
UCB  
Chemin du Foriest 
1420 Braine-l’Alleud 
BELGIUM 
Phone: 
 
 
Change #2 
1 Summary; third paragraph 
In case the subjects have a gap period between the end of the parent study and the start of this 
open-label study (ie, last visit in the parent study does not take place on the same day as entry of 
study CIDP04), a Screening Period of 2 to 5 weeks will be applicable to confirm that the subject 
still meets the eligibility criteria for entry in the open-label study and allow for a smooth 
transition between the treatment used by the subjects during the gap period and initiation of 
rozanolixizumab. The CIDP04 study includes a 24-week Treatment Period followed by an 
8-week Observation Period. For the 24-week Treatment Period, subjects will have weekly visits 
(either on site or at home) during which they will be dosed with an sc infusion of 
rozanolixizumab, up to 
 The initial dose will be based on the dose the subject has 
received at the completion of the parent study (eg, CIDP01). For subjects who were known to be 
on placebo in the parent study (eg, CIDP01), a starting dose of 
 will be used. Dose may 
be reduced to 
 if 
 is not tolerable (eg, headache). The maximum dose of 
rozanolixizumab in CIDP04 will be 
 For exact doses to be administered, refer to 
Section 7.2. 
Has been changed to: 
In case the study participants have a gap period between the end of the parent study and the start 
of this open-label study (ie, last visit in the parent study does not take place on the same day as 
entry of study CIDP04), a Screening Period of 2 to 5 weeks will be applicable to confirm that the 
study participant still meets the eligibility criteria for entry in the open-label study and allow for 
a smooth transition between the treatment used by the study participants during the gap period 
and initiation of rozanolixizumab. The CIDP04 study includes a 24-week Treatment Period, an 
optional additional maximum 52-week Treatment Period followed by an 8-week Observation 


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Period. The length of the additional Treatment and Observation Periods may be shortened 
adjusted in case of availability of an Access Program. For the 24-week Treatment Periods, 
study participants will have weekly visits (either on site or at home) during which they will be 
dosed with an sc infusion of rozanolixizumab, up to 
. The initial dose in the study will 
be based on the dose the study participant has received at the completion of the parent study (eg, 
CIDP01). For study participants who were known to be on placebo in the parent study (eg, 
CIDP01), a starting dose of 
will be used. Dose may be reduced to 
 if 
 
is not tolerable (eg, headache) and/or recurrent low IgG levels  (Treatment Period Part 2). 
The maximum dose of rozanolixizumab in CIDP04 will be 
 For exact doses to be 
administered, refer to Section. 
Change #3 
2.1.1 Rationale for this study; first paragraph 
The following sentence has been added to the end of the paragraph: 
An optional extension of the treatment period by a maximum of an additional 52 weeks, or 
until an Access Program or equivalent is in place, whichever comes first, will be allowed on 
the basis of each participant’s individual benefit-risk assessment at the end of the first 24-
weeks Treatment Period. Study participants may choose to self-administer the IMP during 
Treatment Period Part 2 under supervision of a nurse and after having received 
appropriate training. 
Change #4 
4.2.1 Efficacy variables; selected other efficacy variables 
 Absolute change from Baseline to Week 25 in iRODS score 
 Subject experienced CIDP relapse (iRODS) up to Week 25 from Baseline 
 Values and absolute change from Baseline in iRODS scores at each scheduled assessment 
during the Treatment and Observation Periods 
 Time to CIDP relapse (adjusted INCAT) during the Treatment Periods from Baseline 
 Subject experienced CIDP relapse (maximum grip strength as assessed by site personnel) up 
to Week 25 from Baseline 
Has been changed to: 
 Absolute change from Baseline to Week 25 in iRODS score 
 Values and absolute change from Baseline in iRODS scores at each scheduled assessment 
during the Treatment and Observation Periods 
 Study participant experienced CIDP relapse (iRODS) up to Week 25 and 77 (where 
applicable) from Baseline 
 Study participant experienced CIDP relapse (adjusted INCAT) up to Week 25 and 77 
(where applicable) from Baseline 


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 Study participant experienced CIDP relapse (maximum grip strength as assessed by site 
personnel) up to Week 25 and 77 (where applicable) from Baseline 
Change #5 
4.2.2 Efficacy variables; other efficacy variables 
The following variables have new wording: 
 Values and change from Baseline in fatigue domain scores at each scheduled assessment 
during the Treatment and Observation Periods 
 Values and change from Baseline in CIDP PRO instrument domain scores at each scheduled 
assessment during the Treatment and Observation Periods 
 Patient Global Impressions of Change (PGIC) value at each scheduled assessment during 
Treatment and Observation Periods 
Change #6 
5.1 Study description; third, fourth, fifth and sixth paragraph 
In case the subjects have a gap period between the end of the parent study and the start of this 
OLE study (ie, last visit in the parent study does not take place on the same day as entry of study 
CIDP04), a Screening Period of 2 to 5 weeks will be applicable to confirm that the subject still 
meets the eligibility criteria for entry in the OLE study and allow for a smooth transition between 
the treatment used by the study participants during the gap period and the initiation of 
rozanolixizumab. The CIDP04 study includes a first 24-week Treatment Period followed by an 
8-week Observation Period. For the 24-week Treatment Period, subjects will have weekly visits 
(either on site or at home) during which they will be dosed with an sc infusion of 
rozanolixizumab up to 
 The initial dose will be based on the dose the subject has 
received at the completion of the parent study (eg, CIDP01). For subjects who were known to be 
on placebo in the parent study, a starting dose of 
 will be used. The maximum dose will 
be 
 See Section 7.2 for details on treatment to be administered. 
During the first 4 weeks of the Treatment Period, all visits will be on site. During Weeks 5 to 8, 
home visits and on-site visits will be alternating (Weeks 5 and 7 will be on site; Weeks 6 and 8 
will be at home). Starting at Week 9 and through the end of the Treatment Period (Week 24), 
subjects will have 1 visit on site followed by 3 visits at home for every 4-week period. 
Starting at Week 25, subjects will enter the 8-week Observation Period where no IMP will be 
administered. During the Observation Period, study participants will have on-site visits at 
Weeks 25, and 32, and home visits at Weeks 27 and 29). Subjects can return to their SOC during 
the Observation Period (Section 6.4). 
At the discretion of the investigator and/or subjects, home visits can be changed to site visits 
(eg, for safety reasons). 
Has been changed to: 
In case the study participants have a gap period between the end of the parent study and the start 
of this OLE study (ie, last visit in the parent study does not take place on the same day as entry 


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of study CIDP04), a Screening Period of 2 to 5 weeks will be applicable to confirm that the study 
participant still meets the eligibility criteria for entry in the OLE study and allow for a smooth 
transition between the treatment used by the study participants during the gap period and the 
initiation of rozanolixizumab. The CIDP04 study includes a first 24-week Treatment Period 
(Part 1) followed by a second Treatment Period (Part 2) of 52 weeks. Entry to the 
Treatment Period (Part 2) will be contingent to a favorable individual benefit-risk after 
careful individual assessment has been performed for each study participant completing 
Treatment Period (Part 1). An 8-week Observation Period will be followed in case of 
premature termination or after completion of the Treatment Periods (either at end of Part 
1 for study participants not continuing in Part 2 or at end of Part 2), study participants will 
have weekly visits (either on site or at home) during which they will be dosed with an sc infusion 
of rozanolixizumab up to 
. The initial dose will be based on the dose the study 
participant has received at the completion of the parent study (eg, CIDP01) or at the completion 
of CIDP04 Part 1 for study participants entering CIDP04 Part 2. For study participants who 
were known to be on placebo in the parent study, a starting dose of 
will be used. The 
maximum dose will be 
. See Section 7.2 for details on treatment to be administered. 
During the Treatment Periods (Part 1 and Part 2), study participants all visits will be on 
site. During Weeks 5 to 8, home visits and on-site visits will be alternating (Weeks 5 and 7 will 
be on site; Weeks 6 and 8 will be at home). Starting at Week 9 and through the end of the 
Treatment Period (Week 24 and if applicable, Week 76), study participants will have 1 visit on 
site followed by 3 visits at home for every 4-week period. 
After the completion of the Treatment Period(s) (starting at Week 25 for study participants 
only completing Part 1 or from Week 77 for study participants completing both Part 1 and 
Part 2 or at any early timepoint in case of PEOT), study participants will enter the 8-week 
Observation Period where no IMP will be administered. During the Observation Period, study 
participants will have on-site visits at the entry and exit of the Observation Period 
(Weeks 25/77, and 32/84, and home visits at Weeks 27/79 and 29/81). Study participants can 
return to their SOC during the Observation Period (Section 6.4). In case the study participant 
enters an Access Program, no Observation Period will be performed with the exception of 
the PEOT (Visit 26) Visit which will be immediately completed. 
At the discretion of the investigator and/or subject, home visits can be changed to site visits 
(eg, for safety reasons). In case an on-site visit cannot performed, the next visit will be 
performed at site instead of at home, and all safety assessments that should have been 
completed at the missed visits will be performed. 
Change #7 
5.1.1 Study duration per subject; first paragraph 
The total duration of the study for an individual subject is up to 37 weeks; this includes a 
2- to 5-week Screening Period, a 24-week Treatment Period and an 8-week Observation Period. 
Has been changed to: 
The total duration of the study for an individual study participant will be up to 37 weeks is 89 
weeks; this includes a 2- to 5-week Screening Period, a 24-week Treatment Period (Part 1), an 


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optional maximum 52-week Treatment Period (Part 2) or until an Access Program or 
equivalent is in place whichever comes first, and an 8-week Observation Period in case the 
participant does not enter in an Access Program (or equivalent). 
Change #8 
5.2 Schedule of study assessments; Table 5-1 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period 
8-Week Observation 
Period 
Visit 
1 
Scr a 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
25 
27 
29 
32 
Visit 
type a 
S 
S 
S 
S 
S 
S 
H a 
S 
H c 
S 
H c 
H c 
H c 
S 
H c H c 
S 
Written informed consent a 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic data d 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Verification of inclusion and 
exclusion criteria d 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Withdrawal criteria 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Medical history update d 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior and concomitant 
medication 
X 
X a 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant medical 
procedures 
X 
X e 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Vital signs a 
X 
X  
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Body weight 
X 
X e 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
X 
Recording of AEs 
X 
X e 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Query for suicidality a 
X 
X e 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period 
8-Week Observation 
Period 
Visit 
1 
Scr a 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
25 
27 
29 
32 
Visit 
type a 
S 
S 
S 
S 
S 
S 
H a 
S 
H c 
S 
H c 
H c 
H c 
S 
H c H c 
S 
Fatigue scale 
 
X e 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
X 
CIDP PRO Instrument 
 
X e 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
X 
PGIS 
 
X e 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
X 
PGIC 
 
X e a 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
X 
Full physical examination 
X 
X e 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Brief physical examination 
 
 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
 
Full neurological examinationa 
X 
X e 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Brief neurological examination 
 
 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
 
12-lead ECG 
X 
X e 
X 
X 
X 
 
 
X 
 
X 
 
X 
 
X 
 
 
X 
Labs (hematology, chemistry, 
urinalysis) 
X 
X e 
X 
X 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
 
X 
Serology for HIV, hepatitis B, 
and hepatitis C a 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Serum/urine pregnancy test a 
X 
X e 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 128 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period 
8-Week Observation 
Period 
Visit 
1 
Scr a 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
25 
27 
29 
32 
Visit 
type a 
S 
S 
S 
S 
S 
S 
H a 
S 
H c 
S 
H c 
H c 
H c 
S 
H c H c 
S 
IGRA tuberculosis test a 
X 
X e 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Tuberculosis Signs and 
Symptoms Questionnaire 
X 
 
 
 
 
 
 
 
 
 
 
 
X a 
 
 
 
X 
Contact IRT 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
X 
Administration of IMP 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
Blood sampling for PK of 
rozanolixizumab a 
 
X e 
 
X 
 
 
 
X 
 
X a 
 
 
 
X 
 
 
 
Anti-rozanolixizumab 
antibodies 
 
X e 
 
X 
 
 
 
X 
 
X o 
 
 
 
X 
 
 
X 
Serum complement (C3, C4) 
and plasma complement (C3a, 
C5a) a 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
 
 
 
 
Serum cytokines p 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
 
 
 
 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 129 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period 
8-Week Observation 
Period 
Visit 
1 
Scr a 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
25 
27 
29 
32 
Visit 
type a 
S 
S 
S 
S 
S 
S 
H a 
S 
H c 
S 
H c 
H c 
H c 
S 
H c H c 
S 
Vaccination-specific antibody 
titers (tetanus and influenza 
Type A) 
X 
 
 
 
 
 
 
X 
 
X a 
 
 
 
X 
 
 
 
Immunoglobulins 
(total IgG and IgG subclasses) 
X 
X e 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
X 
NF-L 
 
X e 
 
 
 
 
 
X 
 
 
 
 
 
X 
 
 
 
IgA, IgM, IgE 
 
X e 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
X 
CIDP-specific auto-antibodies 
 
X e 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
X 
Blood sampling for exploratory 
biomarker analysis a 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
 
 
 
 
iRODS assessment a 
X 
X e 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
X 
INCAT assessment 
X 
X e 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
X 
Assessment of grip strength by 
site personnel 
X 
X e 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
X 
RT-MRC assessment 
X 
X e 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
 
X 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 130 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period 
8-Week Observation 
Period 
Visit 
1 
Scr a 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
25 
27 
29 
32 
Visit 
type a 
S 
S 
S 
S 
S 
S 
H a 
S 
H c 
S 
H c 
H c 
H c 
S 
H c H c 
S 
ADA=antidrug antibody; AE=adverse event; BL=baseline; CIDP=chronic inflammatory demyelinating polyradiculoneuropathy; C-SSRS=Columbia Suicide 
Severity Rating Scale; ECG=electrocardiogram; FV=Final Visit; H=Home visit; HIV=human immunodeficiency virus; ICF=Informed Consent form; 
IgA=immunoglobulin A; IgE=immunoglobulin E; IgG=immunoglobulin G; IgM=immunoglobulin M; IGRA=interferon gamma release assay; 
IMP=investigational medicinal product; INCAT=Inflammatory Neuropathy Cause and Treatment; iRODS=inflammatory Rasch-built Overall Disability Scale; 
IRT=interactive response technology; NF-L=neurofilament light chain; PEOT=premature end of treatment; PGIC=Patient Global Impressions of Change; 
PGIS=Patient Global Impressions of Severity; PK=pharmacokinetic; PRO=patient-reported outcome; RT-MRC=Rasch-built, modified-interval Medical 
Research Council scale, S=on-site visit; Scr=Screening visit; V=visit 
Note: All assessments are to be completed in the order specified in the protocol or laboratory manual if possible. The ICF should be completed before any 
assessment. The PROs should be conducted in the order specified in Section 10.4 immediately after ICF (where applicable). The laboratory manual will 
provide further guidance on the order of sample collection. 
a Only for subjects with a gap period between the parent study and entry in CIDP04. 
b All visits have a ±2-day window. Minimum time between doses must be at least 5 days. 
c The following visits can be performed by a healthcare professional visiting the subject at his/her home: Visits 11 to 13, 15 to 17, 19 to 21, and 23 to 25 
(Weeks 10 to 12, 14 to 16, 18 to 20, and 22 to 24). Alternately, the visits can be conducted at the site as deemed necessary by site and/or subject. Feasibility of 
IMP dosing in a home setting will have to be confirmed before the visit is conducted (see Section 8.2). 
d Will not be repeated at Visit 2 if performed at Visit 1. 
e If entry in CIDP04 (Visit 2) is done on the same day as the last visit of the parent study and in case assessment was performed at last visit of the parent study, 
data from the parent study will be used and corresponding assessments will not be repeated in CIDP04.    
f During site Visits 2, 3, and 4, vital signs will be measured prior to IMP administration, at the end of the infusion, and 2 and 4h after the end of the infusion. 
From Visit 5, vital signs will be measured predose, at the end of the infusion and 2h after the end of the infusion only. At nondosing visits, vital signs need only 
be taken once during the visit. For subjects requiring additional assessments due to AEs (see Table 5-2), additional vital sign measurements may be taken based 
on the timing of the assessments. 


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Clinical Study Protocol 
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CIDP04 
 
 
 
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Page 131 of 160 
g A full C-SSRS assessment will be performed only when the subject has a positive response to the suicidal ideation query. If a subject has active suicidal 
ideation as confirmed by the answer “Yes” to Question 4 or Question 5 of the C-SSRS assessments, the subject will be excluded or withdrawn from the study 
and immediately referred to a Mental Healthcare Professional. 
h Not required for subjects who had a Screening Visit. 
i The full neurological examination includes a fundoscopy. In addition to the FV, a full neurological examination should be performed for any subject who 
experiences severe headache (refer to Section 5.2.1 and Table 5-2). 
j Serology includes hepatitis C virus (HCV)-antibodies (Ab)+, hepatitis B virus antibodies (HBsAg and HBcAb), human immunodeficiency virus antibodies 
(HIV1 and HIV2). 
k In case of immediate entry from the parent study, the serum pregnancy test from the parent study should not be older than 5 weeks prior to entry into this 
CIDP04 study and the urine pregnancy test done at Visit 2 must both be negative before dosing. In case of gap period between the parent study and entry in 
CIDP04, a serum test will be performed at Visit 1 (Screening). Pregnancy testing will consist of urine testing at dosing visits during the Treatment Period and 
each visit during the Observation Period. A positive urine pregnancy test must be confirmed using a serum pregnancy test. Note that the final urine pregnancy 
test of the study should be no longer than 90 days after the final dose of IMP.  
l The IGRA test will be performed in a central laboratory.  
m Tuberculosis signs and symptoms Questionnaire must be done at least every 12 weeks. 
n Trough PK samples should be taken for all subjects receiving rozanolixizumab. At Visits 2, 4, and 8, PK samples should be taken predose and 4h postdose for 
all subjects. At Visits 14, 18, and 22, a predose sample only should be taken. At Visit 26, samples to be taken once during the visit.  
o Does not apply to Visit 10. 
p Serum complement (C3, C4), plasma complement (C3a, C5a), and serum cytokines should be taken predose at Visit 2 for all subjects. Samples should be taken 
predose and 4h postdose for all subjects at other scheduled visits (Visits 3, 8, 10, 14, 18, and 22); if an infusion reaction occurs within the first 2h, refer to 
Section 5.2.1 and Table 5-2. 
q Only at Visit 14. 
r At Visits 2, 3, 8, 10, 14, 18, and 22, samples for exploratory biomarkers should be taken predose. See Table 5-2 for sampling in case of AE of interest. 
s Assessment to be performed before all other assessments at each visit except ICF. 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
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Page 132 of 160 
Has been changed to: 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr 
ab 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type bc 
S 
S 
S 
S 
S 
S 
H 
cd 
S 
H 
cd 
S 
H cd H cd H cd 
S 
H cd 
S 
H 
cd 
H 
cd 
S 
Written informed consent de 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic data de 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Verification of inclusion 
and exclusion criteria de 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Withdrawal criteria 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Medical history update de 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior and concomitant 
medication 
X 
X ef 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant medical 
procedures 
X 
X ef 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 133 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr 
ab 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type bc 
S 
S 
S 
S 
S 
S 
H 
cd 
S 
H 
cd 
S 
H cd H cd H cd 
S 
H cd 
S 
H 
cd 
H 
cd 
S 
Vital signs fg 
X 
X  
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Body weight 
X 
X ef 
 
 
 
 
 
 
 
 
 
 
 
X h 
 
X 
 
 
X 
Recording of AEs 
X 
X ef 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Query for suicidality g i 
X 
X ef 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
X 
X 
Fatigue scale 
 
X ef 
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
CIDP PRO Instrument 
 
 
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
PGIS 
 
X ef 
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
PGIC 
 
X ef hk  
 
 
 
X 
 
 
 
X 
 
 
 
X j 
 
X 
 
 
X 
Full physical examination 
X 
X ef 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Brief physical examination 
 
 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 134 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr 
ab 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type bc 
S 
S 
S 
S 
S 
S 
H 
cd 
S 
H 
cd 
S 
H cd H cd H cd 
S 
H cd 
S 
H 
cd 
H 
cd 
S 
Full neurological 
examination l 
X 
X ef 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Brief neurological 
examination 
 
 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
 
12-lead ECG 
X 
X ef 
X 
X 
X 
 
 
X 
 
X 
 
X 
 
X m 
 
X 
 
 
X 
Labs (hematology, 
chemistry, urinalysis) 
X 
X ef 
X 
X 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
 
X 
Serology for HIV, hepatitis 
B, and hepatitis C jn 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Serum/urine pregnancy test 
ko 
X 
X ef 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
X 
X 
IGRA tuberculosis test l p 
X 
X ef 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 135 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr 
ab 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type bc 
S 
S 
S 
S 
S 
S 
H 
cd 
S 
H 
cd 
S 
H cd H cd H cd 
S 
H cd 
S 
H 
cd 
H 
cd 
S 
Tuberculosis Signs and 
Symptoms Questionnaire 
X 
 
 
 
 
 
 
 
 
 
 
 
X 
mq 
X q 
 
 
 
 
X 
Contact IRT 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
X 
Administration of IMP 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X r 
X r 
 
 
 
 
Blood sampling for PK of 
rozanolixizumab ns 
 
X ef 
 
X 
 
 
 
X 
 
X t 
 
 
 
X m  
 
X 
 
 
 
ADA (Anti-
rozanolixizumab 
antibodies) 
 
X ef 
 
X 
 
 
 
X 
 
X ot 
 
 
 
X m 
 
X 
 
 
X 
Serum complement (C3, 
C4) and plasma 
complement (C3a, C5a) pu 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
X 
 
 
 
 
 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 136 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr 
ab 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type bc 
S 
S 
S 
S 
S 
S 
H 
cd 
S 
H 
cd 
S 
H cd H cd H cd 
S 
H cd 
S 
H 
cd 
H 
cd 
S 
Serum cytokines pu 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
X 
 
 
 
 
 
Vaccination-specific 
antibody titers (tetanus and 
influenza Type A) 
X 
 
 
 
 
 
 
X 
 
X qv 
 
 
 
X m 
 
X 
 
 
 
Immunoglobulins 
(total IgG and IgG 
subclasses) 
X 
X ef 
X 
X 
X 
X 
 Xw 
X 
X w 
X 
X w 
X w X w 
X 
X w 
X 
 
 
X 
NF-L 
 
X ef 
 
 
 
 
 
X 
 
 
 
 
 
X x 
 
X y 
 
 
 
IgA, IgM, IgE 
 
X ef 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X m 
 
X 
 
 
X 
CIDP-specific auto-
antibodies 
 
X ef 
 
 
 
X 
 
 
 
X 
 
 
 
X x 
 
X y 
 
 
X x 


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Clinical Study Protocol 
Rozanolixizumab 
CIDP04 
 
 
 
Confidential 
Page 137 of 160 
Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr 
ab 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type bc 
S 
S 
S 
S 
S 
S 
H 
cd 
S 
H 
cd 
S 
H cd H cd H cd 
S 
H cd 
S 
H 
cd 
H 
cd 
S 
Blood sampling for 
exploratory biomarker 
analysis rz 
 
X 
X 
 
 
 
 
X 
 
X 
 
 
 
 
 
 
 
 
 
iRODS assessment saa 
X 
X ef 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
X 
INCAT assessment 
X 
X ef 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
X 
Assessment of grip strength 
by site personnel 
X 
X ef 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X 
 
X 
 
 
X 
RT-MRC assessment 
X 
X ef 
X 
X 
X 
X 
 
X 
 
X 
 
 
 
X x 
 
X y 
 
 
X x 


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Table 5-1: 
Schedule of study assessments 
Assessments 
 
 
24-Week Treatment Period (Part 1) 
52-Week 
Additional 
Treatment 
Period (Part 
2 - Optional) 
a 
8-Week Observation 
Period (Optional) 
Visit 
1 
Scr 
ab 
2 
BL 
3 
4 
5 
6 
7 
8 
9 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
13 
17 
21 
25 
Additional 
Visits 
numbered 
sequentially 
26 
PEOT 27 
28 
29 
FV 
Week 
-5 to  
-2 
1 
2 
3 
4 
5 
6 
7 
8 
9 
13 
17 
21 
10 
14 
18 
22 
11 
15 
19 
23 
12 
16 
20 
24 
Every 
4 
weeks 
Weekly 
in bet-
ween 
site  
visits 
25 or 
77 
27 
or 
79 
29 
or 
81 
32 
or 
84 
Visit 
type bc 
S 
S 
S 
S 
S 
S 
H 
cd 
S 
H 
cd 
S 
H cd H cd H cd 
S 
H cd 
S 
H 
cd 
H 
cd 
S 
ADA=antidrug antibody; AE=adverse event; BL=baseline; CIDP=chronic inflammatory demyelinating polyradiculoneuropathy; C-SSRS=Columbia Suicide 
Severity Rating Scale; ECG=electrocardiogram; FV=Final Visit; H=Home visit; HIV=human immunodeficiency virus; ICF=Informed Consent form; 
IgA=immunoglobulin A; IgE=immunoglobulin E; IgG=immunoglobulin G; IgM=immunoglobulin M; IGRA=interferon gamma release assay; 
IMP=investigational medicinal product; INCAT=Inflammatory Neuropathy Cause and Treatment; iRODS=inflammatory Rasch-built Overall Disability Scale; 
IRT=interactive response technology; NF-L=neurofilament light chain; PEOT=premature end of treatment; PGIC=Patient Global Impressions of Change; 
PGIS=Patient Global Impressions of Severity; PK=pharmacokinetic; PRO=patient-reported outcome; RT-MRC=Rasch-built, modified-interval Medical 
Research Council scale, S=on-site visit; Scr=Screening visit; V=visit 
Note: All assessments are to be completed in the order specified in the protocol or laboratory manual if possible. The ICF should be completed before any 
assessment. The PROs should be conducted in the order specified in Section 10.4 immediately after ICF (where applicable). The laboratory manual will 
provide further guidance on the order of sample collection. 
a The 52-week duration can be shortened or extended in view of the availability of an access program. 
aa Assessment to be performed before all other assessments at each visit except ICF. 
b Only for study participants with a gap period between the parent study and entry in CIDP04. 
c All visits have a ±2-day window. Minimum time between doses must be at least 5 days. 
d The following visits can be performed by a healthcare professional visiting the study participant at his/her home: Visits 11 to 13, 15 to 17, 19 to 21, and 23 to 
25 (Weeks 10 to 12, 14 to 16, 18 to 20, and 22 to 24). Visits in Treatment Period Part 2 will follow the same pattern: one site visit followed by 3 visits 


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conducted at the participant 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 8.2). 
e Will not be repeated at Visit 2 if performed at Visit 1. 
f If entry in CIDP04 (Visit 2) is done on the same day as the last visit of the parent study and in case assessment was performed at last visit of the parent study, 
data from the parent study will be used and corresponding assessments will not be repeated in CIDP04.    
g During site Visits 2, 3, and 4, vital signs will be measured prior to IMP administration, at the end of the infusion, and 2 and 4h after the end of the infusion. 
From Visit 5 until Visit 25, vital signs will be measured predose, at the end of the infusion and 2h after the end of the infusion only. During Treatment Period 
Part 2, vital signs will be measured predose, at the end of the infusion. At nondosing visits, vital signs need only be taken once during the visit. For study 
participants requiring additional assessments due to AEs (see Table 5-2), additional vital sign measurements may be taken based on the timing of the 
assessments. 
h Body weight is collected every 6 months during Treatment Period Part 2 starting at first visit in this period and will be used to select the correct 
volume for dose infusion from these timepoints. 
i A full C-SSRS assessment will be performed only when the study participant has a positive response to the suicidal ideation query. If a study participant has 
active suicidal ideation as confirmed by the answer “Yes” to Question 4 or Question 5 of the C-SSRS assessments, the study participant will be excluded or 
withdrawn from the study and immediately referred to a Mental Healthcare Professional. 
j Only performed every 24 weeks. 
k Not required for study participants who had a Screening Visit. 
l The full neurological examination includes a fundoscopy. In addition to the FV, a full neurological examination should be performed for any study participant 
who experiences severe headache (refer to Section 5.2.1 and Table 5-2). 
m Assessment to be performed every 12 weeks (Weeks 25, 37, 49 and 61).  
n Serology includes hepatitis C virus (HCV)-antibodies (Ab)+, hepatitis B virus antibodies (HBsAg and HBcAb), human immunodeficiency virus antibodies 
(HIV1 and HIV2). 
o In case of immediate entry from the parent study, the serum pregnancy test from the parent study should not be older than 5 weeks prior to entry into this 
CIDP04 study and the urine pregnancy test done at Visit 2 must both be negative before dosing. In case of gap period between the parent study and entry in 
CIDP04, a serum test will be performed at Visit 1 (Screening). Pregnancy testing will consist of urine testing at dosing visits during the Treatment Period Part 
1, monthly urine testing during Treatment Period Part 2 and at each visit during the Observation Period for women of childbearing potential. A positive urine 
pregnancy test must be confirmed using a serum pregnancy test. Note that the final urine pregnancy test of the study should be no longer than 90 days after the 
final dose of IMP.  
p The IGRA test will be performed in a central laboratory.  
q Tuberculosis signs and symptoms Questionnaire must be done at least every 12 weeks. 
r Self-administration may be performed by study participant under the supervision of the home nurse after full training. 
s Trough PK samples should be taken for all study participants receiving rozanolixizumab. At Visits 2, 4, and 8, PK samples should be taken predose and 4h 
postdose for all study participants. At Visits 14, 18, 22 and quarterly at site visits during Treatment Period Part 2, a predose sample only should be taken. 
At Visit 26, samples to be taken once during the visit.  
t Does not apply to Visit 10. 
u Serum complement (C3, C4), plasma complement (C3a, C5a), and serum cytokines should be taken predose at Visit 2 for all study participants. Samples should 
be taken predose and 4h postdose for all study participants at other scheduled visits (Visits 3, 8, 10, 14, 18, 22 and at first (Week 25) and last visit (Week 73) of 
Treatment Period Part 2); if an infusion reaction occurs within the first 2h, refer to Section 5.2.1 and Table 5-2. 


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v Only at Visit 14. 
w In case IgG levels are below 2 and the study participant does not qualify for a withdrawal, IgG levels will be monitored weekly. 
x Assessments to be performed only at the first visit after Visit 25 (ie, 1st visit of Part 2). Visit 29/FV should not performed after Treatment Period 2. 
y Only performed at PEOT visit when starting Observation Period directly after Treatment Period Part 1 
z At Visits 2, 3, 8, 10, 14, 18, and 22, samples for exploratory biomarkers should be taken predose. See Table 5-2 for sampling in case of AE of interest. 
 


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Change #9 
5.2.1 Additional study assessments; Table 5-2; complements and cytokines, and exploratory 
biomarkers rows only  
Table 5-2: 
Additional study assessments 
Assessment 
When applicable 
For subjects who experience infusion reactions (excluding local injection site reactions): 
Complements 
and cytokines 
Samples required for all subjects are detailed in the schedule of study assessments 
(Table 5-1). 
In subjects who experience an infusion reaction within the first 2h at Visits 2, 4, 5, and 
6, samples should also be taken 2 and 4 hours postdose (see Section 9.1.9 and 
Section 18.1). 
In subjects who experience an infusion reaction within 2 hours at Visits 3, 8, 10, 14, 
18, and 22, samples should be taken 2 hours postdose (see Section 9.1.9 and 
Section 18.1). 
For subjects who experience an AE of interest, including severe headache, moderate to severe diarrhea, 
moderate to severe abdominal pain, or moderate to severe vomiting: 
If the AE is initially reported at a home visit, the subject should be reviewed at the study site as soon as is 
practically possible for further investigation. 
Exploratory 
biomarkers 
Samples required for all subjects are detailed in the schedule of study assessments 
(Table 5-1). 
In subjects who experience an AE of interest (as defined in Section 9.1.14) at Visits 2, 
3, 8, 10, 14, 18, and 22, samples should also be taken 4 hours postdose. 
For subjects who experience severe headache: 
Headache 
Questionnaire 
In subjects who report severe headache, this assessment will be performed daily until 
resolution (ie, if headache becomes moderate or mild, normal collection of AEs 
should apply) (see Section 9.1.10). 
Full neurological 
examination 
Assessments required for all study participants are detailed in the schedule of study 
assessments Table 5-1  
In subjects who report severe headache at the clinic visit, a full neurological 
examination (including fundoscopy) should be performed (see Section 9.1.10). In 
subjects who report a severe headache whilst at home, a visit to the site for the full 
neurological examination should be arranged for as soon as is practically possible. 


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Has been changed to: 
Table 5-2: 
Additional study assessments 
Assessment 
When applicable 
For study participants who experience infusion reactions (excluding local injection site reactions): 
Complements 
and cytokines 
Samples required for all study participants are detailed in the schedule of study 
assessments (Table 5-1). 
In study participants who experience an infusion reaction within the first 2h at Visits 
2, 4, 5, and 6, samples should also be taken 2 and 4 hours postdose (see Section 9.1.9 
and Section 18.1). 
In study participants who experience an infusion reaction within 2 hours at Visits 3, 8, 
10, 14, 18, and 22 and “S” visits during Treatment Period Part 2, samples should 
be taken 2 hours postdose (see Section 9.1.9 and Section 18.1). 
For study participants who experience an AE of interest, including severe headache, moderate to severe 
diarrhea, moderate to severe abdominal pain, or moderate to severe vomiting: 
If the AE is initially reported at a home visit, the study participant should be reviewed at the study site as 
soon as is practically possible for further investigation. 
Exploratory 
biomarkers 
Samples required for all subjects are detailed in the schedule of study assessments 
(Table 5-1). 
In subjects who experience an AE of interest (as defined in Section 9.1.14) at Visits 2, 
3, 8, 10, 14, 18, and 22 and “S” visits during Treatment Period Part 2, samples 
should also be taken 4 hours postdose. 
For study participants who experience severe and/or serious headache: 
Headache 
Questionnaire 
In study participants who report severe and/or serious headache, this assessment will 
be performed daily until resolution (ie, if headache becomes moderate or mild, normal 
collection of AEs should apply) (see Section 9.1.10). 
Full neurological 
examination 
Assessments required for all study participants are detailed in the schedule of study 
assessments (Table 5-1 ). 
In study participants who report severe and/or serious headache at the clinic visit, a 
full neurological examination (including fundoscopy) should be performed (see 
Section 9.1.10). In study participants who report a severe headache whilst at home, a 
visit to the site for the full neurological examination should be arranged for as soon as 
is practically possible. 
Change #10 
5.3 Rationale for study design and selection of dose; second paragraph 
The initial dose of rozanolixizumab will be based on the dose the subject has received at the 
completion of the parent study (eg, CIDP01). For subjects who were known to be on placebo in 
parent study, a starting dose of 
 will be used. The dose may be reduced to 
if 
 is not tolerable (eg, severe headache, Section 9.1.10). The dose and regimen of IMP to 
be used in the current study (rozanolixizumab 
 sc) is based on results from the FIH 
study UP0018, alongside the safety data from the MG and ITP studies (MG0002 and TP0001). 
CIDP04, similar to the parent study, CIDP01, will utilize a liquid formulation at 
. 


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Has been changed to: 
The initial dose of rozanolixizumab will be based on the dose the study participant has received 
at the completion of the parent study (eg, CIDP01). For study participants who were known to be 
on placebo in parent study, a starting dose of 
 will be used. The dose may be reduced to 
 if 
 is not tolerable (eg, severe headache, Section 9.1.10), or in case of IgG 
levels are <1g/L (see Section 6.4.1.3). The dose and regimen of IMP to be used in the current 
study (rozanolixizumab 
 sc) is based on results from the FIH study UP0018, alongside 
the safety data from the MG and ITP studies (MG0002 and TP0001). CIDP04, similar to the 
parent study, CIDP01, will utilize a liquid formulation at 
 
Change #11 
6.2 Exclusion Criteria, criterion #19 
Subject has a history of clinically relevant ongoing chronic infections including but not limited to 
human immunodeficiency virus (HIV), hepatitis B, hepatitis C, or is tested positive for human 
immunodeficiency virus antibody 1 (HIV1), human immunodeficiency virus antibody 2 (HIV2), 
hepatitis B surface antigen, or hepatitis C antibody at the Screening Visit. 
Has been changed to: 
Study participant has a history of clinically relevant ongoing chronic infections including but not 
limited to human immunodeficiency virus (HIV), hepatitis B, hepatitis C, or is tested positive for 
human immunodeficiency virus antibody 1 (HIV1), human immunodeficiency virus antibody 2 
(HIV2), hepatitis B surface antigen, hepatitis B core antibody without hepatitis B surface 
antibody test positive, or hepatitis C antibody at the Screening Visit. 
Change #12 
6.4.1.1 Serious infection 
Treatment must be temporarily discontinued if subject has a serious infective episode requiring 
eg, hospitalization, or iv antibiotic therapy (including but not limited to bacteremia or sepsis, 
bacterial meningitis, osteomyelitis or septic arthritis, bacterial pneumonia, or visceral abscess). 
Upon resolution of such an event, the subject may restart treatment with IMP at the discretion of 
the investigator and in agreement with Medical Monitor and Study Physician, if the subject is 
willing and if the benefit-risk remains favorable for the subject. 
Has been changed to: 
Treatment must be temporarily discontinued if study participant has a serious infective episode 
requiring eg, hospitalization, or iv antibiotic therapy (including but not limited to bacteremia or 
sepsis, bacterial meningitis, osteomyelitis or septic arthritis, bacterial pneumonia, or visceral 
abscess). Upon resolution of such an event, the subject may restart treatment with IMP at the 
discretion of the investigator and in agreement with Medical Monitor and Study Physician, if the 
subject is willing and if the benefit-risk remains favorable for the subject. 


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Change #13 
6.4.1.2 Hypogammaglobulinemia and non-serious persisting or recurrent infection 
Treatment may be temporarily discontinued for the subject who develops an event of non-serious 
infection, which is persisting or recurrent, and a serum total IgG ≤2 g/L (see Section 7.9). Upon 
resolution of such an event, the subject may restart treatment with IMP at the discretion of the 
investigator and in agreement with Medical Monitor and Study Physician, if the subject is 
willing and if the benefit-risk remains favorable for the subject.  
Has been changed to: 
In the event of a non-serious infection, the Benefit-Risk of continuing treatment with IMP 
must be carefully evaluated by the Investigator in collaboration with the Medical Monitor 
and the Study Physician. Treatment may be temporarily discontinued for the subject who 
develops a an event of non-serious infection, which is persisting or recurrent infection with a, 
and a serum total IgG levels between ≥1 and <≤2g/L (see Section 7.9). Upon resolution of such 
an event infection and the IgG levels returning to ≥2g/L, the study participant may restart 
treatment with IMP (at the same dose for study participants in Treatment Period Part 1, or 
at the same dose or at the reduced dose of 
for study participants in Treatment 
Period Part 2) at the discretion of the investigator and in agreement with Medical Monitor and 
Study Physician at a dose level of 
 for study participants in TP Part 2, if the subject is 
willing and if the benefit-risk remains favorable for the study participant.  
Change #14 
6.4.1.3 Hypogammaglobulinemia irrespective of infection 
Treatment may will be temporarily discontinued for the subject who develops an event of 
hypogammaglobulinemia with a serum total IgG <1g/L (see Section 7.9) regardless of event of 
infection. When the IgG level reaches ≥1g/L, the subject may be allowed to continue treatment 
with IMP at the discretion of the investigator and in agreement with Medical Monitor and Study 
Physician, if the subject is willing and if the benefit-risk remains favorable for the subject. The 
dose of the IMP may be reduced to 
 (Section 7.2) 
Has been changed to: 
Treatment may will be temporarily discontinued for the study participant who develops an event 
of hypogammaglobulinemia with a serum total IgG <1g/L (see Section 7.9) regardless of event 
irrespective of infection. When the IgG level reaches ≥12g/L, the study participant may be 
allowed to restart continue treatment with IMP (at the same dose for study participants in 
Treatment Period Part 1, or at the same dose or at the reduced dose of 
 for study 
participants in Treatment Period Part 2) at the discretion of the investigator and in agreement 
with Medical Monitor and Study Physician, at a dose level of 
 for study participants in 
Treatment Period Part 2, if the study participant is willing and if the benefit-risk remains 
favorable for the study participant. 


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Change #15 
7.2 Treatment to be administered; first, second and sixth paragraph  
The initial dose administered will be based on the dose at the time of completion of the 
Treatment Period from the parent study (eg, CIDP01). For subjects who had a gap period 
between the parent study (eg, CIDP01) and CIDP04, a starting dose of 
will be used (see 
Table 7-1). The dose used in the study may be reduced based on individual tolerability to 
 
(see Table 7-2) (eg, severe headache) but the maximum dose used will be 
. 
Eligible subjects will receive rozanolixizumab 
 by sc infusion for 24  weeks during 
Treatment Period Part 1 (from Visit 2 to Visit 25) and during an additional 52 weeks during 
Treatment Period Part 2.  
The subject’s body weight at entry into CIDP04 (either at Screening [Visit 1] or Baseline 
[Visit 2]) will be used for the dose calculation throughout the duration of the study. 
Has been changed to: 
The initial dose administered will be based on the dose at the time of completion of the 
Treatment Period from the parent study (eg, CIDP01). For study participants who had a gap 
period between the parent study (eg, CIDP01) and CIDP04, a starting dose of 
will be 
used (see Table 7-1). The dose used in the study may be reduced based on individual tolerability 
to 
(see Table 7-2) (eg, severe headache) but the maximum dose used will be 
 
The dose used during Treatment Period Part 2 may be reduced to 
 in case of 
recurrent low observed IgG levels for a participant. Treatment temporary halt may also be 
implemented in some situation (see Section 6.4.1). 
Eligible study participants will receive rozanolixizumab 
 by sc infusion for 24 weeks 
during Treatment Period Part 1 (from Visit 2 to Visit 25) and during an additional 52 weeks 
during Treatment Period Part 2. Treatment Period Part 2 may be shortened depending on 
availability of an Access Program or equivalent. 
Study participants may choose to self-administer the IMP during Treatment Period Part 2 
under supervision of a nurse and after having received appropriate training. 
The study participant’s body weight at entry into CIDP04 (either at Screening [Visit 1] or 
Baseline [Visit 2]) will be used for the dose calculation throughout the duration of the study 
Treatment Period Part 1. Body weight will be reevaluated at start of the Treatment Period 
Part 2, and again 6 months later, and will be used to select the correct volume for dose 
infusion from these timepoints. 
Change #16 
7.5 Handling and storage requirements; second paragraph 
Appropriate storage conditions must be ensured either by controlling the temperature (eg, room, 
refrigeration unit) or by completion of a temperature log in accordance with local requirements 
on a regular basis (eg, once a week), showing actual and minimum/maximum temperatures 
reached over the time interval. 


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Has been changed to: 
Appropriate storage conditions must be ensured either by controlling the temperature (eg, room, 
refrigeration unit) or by completion of a temperature log in accordance with local requirements 
on a regular basis (eg, once a week every work day), showing actual and minimum/maximum 
temperatures reached over the time interval. 
Change #17 
8 Study Procedures by Visit 
The following paragraph has been added: 
In case an on-site visit cannot be performed, the subsequent visit will be performed at the site 
instead of at home, and all safety assessments that should have been completed at the missed 
visit will be performed. 
Change #18 
8.2 Treatment Period, Visits 2 to 25 (Weeks 1 to 24); last paragraph 
The investigator will be asked to complete a checklist confirming all criteria have been fully 
evaluated either at Screening (Visit 1) or Baseline (Visit 2), and be confirmed in case of changes 
to the subject’s condition. This checklist will be shared with the UCB Study Physician and 
reviewed before IMP administration in a home setting can take place. 
Has been changed to: 
8.2 Treatment Period Part 1, Visits 2 to 25 (Weeks 1 to 24); last paragraph 
The investigator will be asked to complete a checklist confirming all criteria have been fully 
evaluated either at Screening (Visit 1) or Baseline (Visit 2)and met before the first home 
dosing visit can take place, and will be reconfirmed in case of changes to the study 
participant’s condition. This checklist will be shared with the UCB Study Physician and 
reviewed before IMP administration in a home setting can take place. 
Change #19 
The following sections have been added:  
8.3 Treatment Period Part 2 (Weeks 25 to 76) 
Entry into the Treatment Period (Part 2) will be subject to a careful individual assessment 
of the benefit-risk for each study participant completing Treatment Period (Part 1) 
following a discussion with the Sponsor's Medical Monitor and/or Study Physician. The 
first visit of Treatment Period Part 2 will be performed at site at Week 25 followed by 3 
weekly visits at the study participant’s home. This sequence will be repeated until the end 
of the Treatment Period Part 2. The visits during this period will be numbered 
sequentially. 
Study participants may choose to self-administer the IMP during Treatment Period 2 
under supervision of a nurse and after having received appropriate training. 


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8.3.1 Additional site visits during maximum 52-week Treatment Period Part 2 (monthly 
starting at Week 25) or until availability of an Access Program (whichever comes first) 
The first visit of Treatment Period Part 2 will be performed on site at Week 26. Site visits 
will be performed monthly. The following procedures will be performed at monthly 
frequency unless otherwise defined: 
 PROs should be conducted in the order specified in Section 10.4: 
 iRODS assessment – to be performed before any other assessment 
 Fatigue scale (every 24 weeks) 
 CIDP PRO instrument (every 24 weeks) 
 PGIS (every 24 weeks) 
 PGIC (every 24 weeks) 
 Query for suicidality 
 Tuberculosis Signs and Symptoms Questionnaire (every 12 weeks) 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end 
of the infusion) 
 Body weight (Week 25 and Week 49) 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 12-lead ECG (on-site visits only [Weeks 25, 37, 49, 61]) 
 Blood sample (all samples should be taken predose unless otherwise specified; the 
laboratory manual will provide guidance on the order of sample collection) for: 
 Clinical laboratory tests (ie, hematology and clinical chemistry) 
 PK of rozanolixizumab (on-site visits only [Weeks 25, 37, 49, 61]) 
 ADA (anti-rozanolixizumab antibodies) (on-site visits only [Weeks 25, 37, 49, 61]) 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) (predose and 
4h postdose) (at Weeks 25 and 73) 
 Serum cytokines (predose and 4h postdose) (at Weeks 25 and 73) 
 Vaccination-specific antibody titers (tetanus and influenza Type A) (on-site visits 
only [Weeks 25, 37, 49, 61]) 


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 Immunoglobulins (total IgG and IgG subclasses) 
 IgA, IgM, IgE (on-site visits only [Weeks 25, 37, 49, 61]) 
 NF-L (only at Week 25) 
 CIDP specific auto-antibodies (only at Week 25) 
 Urine pregnancy test 
 Contact IRT 
 Administer IMP – self-administration by the study participant under the supervision of 
the nurse (to confirm that study participant is proceeding correctly) may be considered 
provided the participant is willing to do so, the participant has been adequately trained 
by the site personnel according to Self-administration training guidance document  
 INCAT assessment 
 Assessment of grip strength by site personnel 
8.3.2 Additional home visits during Treatment Period Part 2 (weekly in between site visits 
starting at Week 26 
The following procedures will be performed during the Treatment Period Part 2 by a 
healthcare professional visiting the study participant at his or her home. Visits will be 
performed weekly in between monthly site visits (Section 8.3.1): 
 Withdrawal criteria assessment 
 Prior and concomitant medications 
 Concomitant medical procedures 
 Vital signs (prior to IMP administration, at the end of the infusion, at 2h after the end 
of the infusion) 
 Recording of AEs 
 Contact IRT 
 Administer IMP (unless IMP is temporarily discontinued (see Section 6.4.1.3) – self-
administration by the study participant under the supervision of the home nurse may 
be considered provided the participant is willing to do it and has been adequately 
trained by the site personnel according to Self-administration training guidance 
document. 
Change #20 
8.3 Observation Period, Visits 26 to 29 (Weeks 25 to 32); heading and first paragraph 
Following the final dose of rozanolixizumab at Week 24 (Visit 25), 4 subsequent visits will be 
scheduled over 8 weeks (Visits 26 to 29) to collect safety and efficacy data for study-related 
outcome measures and procedures. Visit 27 and Visit 28 are home nursing visits to be conducted 
by a healthcare professional visiting the subject at his/her home; these visits can be conducted at 
the site as deemed necessary by site personnel and/or subject.  


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All subjects should perform all visits of the Observation Period, including the FV (scheduled 
8 weeks after the final dose of IMP). If at any time during the Observation Period the subject 
relapses (according to the medical judgement of the investigator supported by, eg, subject’s score 
on iRODS, INCAT, or maximum grip strength [assessed by site personnel]), then rescue 
medication must be considered. The subject will return to the SOC (ie, Ig treatment) as rescue 
medication at the time of relapse. 
Has been changed to: 
8.4 Observation Period, Visits 26 to 29 (Weeks 25 to 32 study participants completing 
Treatment Period Part 1 only and Weeks 77 to 84 for study participants completing 
Treatment Period Part 1 and Part 2) 
Following the final dose of rozanolixizumab at Week 24 (Visit 25) or Week 76, 4 subsequent 
visits will be scheduled over 8 weeks (Visits 26 to 29) to collect safety and efficacy data for 
study-related outcome measures and procedures unless the study participant continues 
rozanolixizumab treatment under an Access Program or equivalent. In the latter case, only 
the PEOT Visit (Visit 26) will be performed. Visit 27 and Visit 28 are home nursing visits to 
be conducted by a healthcare professional visiting the study participant at his/her home; these 
visits can be conducted at the site as deemed necessary by site personnel and/or study participant. 
All study participants who do not continue on rozanolixizumab in an Access Program should 
perform all visits of the Observation Period, including the FV (scheduled 8 weeks after the final 
dose of IMP). If at any time during the Observation Period the study participant relapses 
(according to the medical judgement of the investigator supported by, eg, study participant’s 
score on iRODS, INCAT, or maximum grip strength [assessed by site personnel]), then rescue 
medication must be considered. The study participant will return to the SOC (ie, Ig treatment) as 
rescue medication at the time of relapse. 
Change #21 
8.3.1 Visit 26 (Week 25/PEOT); first sentence and specific assessments  
The following procedures will be performed at Visit 26 (Week 25/PEOT): 
 Pregnancy test  
 Anti-rozanolixizumab antibodies 
 NF-L (Visit 26 [Week 25] only) 
 CIDP specific auto-antibodies (Visit 26 [Week 25] only) 
 RT-MRC assessment (Visit 26 [Week 25] only) 
Has been changed to: 
8.34.1 Visit 26 (Week 25 or Week 77 or PEOT)  
The following procedures will be performed at Visit 26 (Week 25 or Week 77 or /PEOT): 
 Urine Ppregnancy test for women of childbearing potential 
 Anti ADA (anti-rozanolixizumab antibodies) 


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  
 NF-L (Visit 26 [Week 25] only or PEOT only if Treatment Period 1 is performed) 
 CIDP specific auto-antibodies (Visit 26 [Week 25] only or PEOT only if Treatment 
Period 1 is performed) 
 RT-MRC assessment (Visit 26 [Week 25] only) 
Change #22 
8.3.2 Visit 27 and 28 (Weeks 27 and 29), Home Visits; first sentence  
The following procedures will be performed at Visits 27 and 28 (Weeks 27 and 29, and at 79 and 
81) by a healthcare professional visiting the subject at his/her home: 
Has been changed to: 
8.34.2 Visit 27 and 28 (Weeks 27 and 29, and at 79 and 81), Home Visits; first sentence  
The following procedures will be performed at Visits 27 and 28 (Weeks 27 and 29, and at 79 
and 81) by a healthcare professional visiting the subject at his/her home: 
Change #23 
8.3.3 Visit 29 (Week 32/FV); first sentence  
The following procedures will be performed at Visit 29 (Week 32/FV): 
 Urine sample for urinalysis and urine pregnancy test 
 Anti-rozanolixizumab antibodies 
Has been changed to: 
8.34.3 Visit 29/FV (Week 32/FV84)  
The following procedures will be performed at Visit 29/FV (Week 32/FV84): 
 Urine sample for urinalysis and urine pregnancy test 
 Urine pregnancy test for women of childbearing potential 
 Anti ADA (anti-rozanolixizumab antibodies) 
Change #24 
8.5 Premature end of treatment and Final Visit 
The following paragraph has been added:  
A study participant entering the Access Program after completion of part or full 
Treatment Period 2 without any relapse will not complete the Observation Period but will 
only perform the PEOT/V26 one week after the last IMP dispensing for the study. 


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Change #25 
9.1.1.2.1 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 9-1). 
This list does not change the investigator’s obligation to report all SAEs (including Anticipated 
SAEs) as detailed in Section 9.1.2.3. 
Table 9-1: 
Anticipated serious adverse events for CIDP population 
Neurologic diseases a 
Muscle weakness, numbness and pain in the extremities, impaired balance, 
difficulty walking 
a The investigator should report these adverse events as serious adverse events only if they meet criteria for 
seriousness (see Section 9.1.1.2). 
Has been changed to: 
The following Anticipated SAEs, fatigue, infection and headache are anticipated to occur in 
the population studied in this protocol at some frequency that is independent of drug exposure 
(Table 9-1). 
This list information does not change the investigator’s obligation to report all SAEs (including 
Anticipated SAEs) as detailed in Section 9.1.2.3. 
Table 9-1: 
Anticipated serious adverse events for CIDP population 
Neurologic diseases a 
Muscle weakness, numbness and pain in the extremities, impaired balance, 
difficulty walking 
a The investigator should report these adverse events as serious adverse events only if they meet criteria for 
seriousness (see Section 9.1.1.2). 
Change #26 
9.1.2.1 Description of adverse events; third paragraph  
For recording an AE, Common Terminology Criteria for Adverse Events (CTCAE) will be used, 
and only if it is impossible to assess severity using CTCAE, then AE intensity will be assessed 
using a scale of mild, moderate, or severe. 
Has been changed to: 
For recording an AE, Common Terminology Criteria for Adverse Events (CTCAE) will be used, 
and only if it is impossible to assess severity using CTCAE, then AE intensity will be assessed 
using a scale of mild, moderate, or severe. 
 Mild: An event that is easily tolerated by the participant, causing minimal discomfort 
and not interfering with everyday activities 
 Moderate: An event that causes sufficient discomfort and interferes with normal 
everyday activities 


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 Severe: An event that prevents normal everyday activities. An AE that is assessed as 
severe should not be confused with an SAE. Severe is a category utilized for rating the 
intensity of an event; and both AEs and SAEs can be assessed as severe.  
 An event is defined as ‘serious’ when it meets at least 1 of the predefined outcomes as 
described in the definition of an SAE, NOT when it is rated as severe (eg, a severe AE 
may be either serious or not serious, depending on whether these criteria are also met). 
Change #27 
9.1.4 Pregnancy; first paragraph, second bullet point 
The study participant should immediately stop the intake of the IMP or be down-titrated as 
instructed at the PEOT Visit. 
Has been changed to: 
The study participant should immediately stop the intake of the IMP or be down-titrated as 
instructed at the PEOT Visit. 
Change #28 
9.1.9 Hypersensitivity and adverse reactions; first paragraph and Table 9-2 
The grading for infusion-related reactions according to the NCI Common Terminology Criteria 
for Adverse Events version 4.03 (Jun 2010) (Doessegger and Banholzer, 2015) is provided in 
Table 9-2. In the event of a severe or life-threatening (ie, Grade 3 or 4) infusion reaction, the 
subject must permanently discontinue IMP and be managed as described in Section 18.1. 
Table 9-2: Infusion-related reaction grading according to the NCI Common Terminology 
Criteria for adverse events version 4.03 (Jun 2010) 
Grading 
Infusion-related reaction 
1 
Mild transient reaction: infusion interruption not indicated; intervention not indicated 
2 
Moderate: Therapy or infusion interruption indicated but responds promptly to symptomatic 
treatment (for example, antihistamines, NSAIDs, narcotics, iv fluids); prophylactic 
medications indicated for ≤24h 
3 
Severe: Prolonged; recurrence of symptoms following initial improvement; hospitalization 
indicated for clinical sequelae 
4 
Life-threatening consequences; urgent intervention indicated 
5 
Death 
iv=intravenous; NCI=National Cancer Institute; NSAID=nonsteroidal anti-inflammatory drugs 
Data source: Doessegger et al, 2015 
Has been changed to: 
The grading for infusion-related reactions according to the NCI Common Terminology Criteria 
for Adverse Events (CTCAE) version 4.035.0 (Jun 20102017) (Doessegger and Banholzer, 
2015) is provided in Table 9-2. In the event of a severe or life-threatening (ie, Grade 3 or 4) 


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infusion reaction, the study participant must permanently discontinue IMP and be managed as 
described in Section 18.1. 
Table 9-2: Infusion-related reaction grading according to the NCI Common Terminology 
Criteria for adverse events version 4.035.0 (Jun 20102017) 
Grading 
Infusion-related reaction 
1 
Mild transient reaction: infusion interruption not indicated; intervention not indicated 
2 
Moderate: Therapy or infusion interruption indicated but responds promptly to symptomatic 
treatment (for example, antihistamines, NSAIDs, narcotics, iv fluids); prophylactic 
medications indicated for ≤24h 
3 
Severe: Prolonged (eg, not rapidly responsive to symptomatic medication and/or brief 
interruption of infusion); recurrence of symptoms following initial improvement; 
hospitalization indicated for clinical sequelae 
4 
Life-threatening consequences; urgent intervention indicated 
5 
Death 
iv=intravenous; NCI=National Cancer Institute; NSAID=nonsteroidal anti-inflammatory drugs 
Data source: Doessegger et al, 2015 
Change #29 
9.1.10 Management of headache  
Treatment of headaches should be as per national guidelines and take medical history of previous 
headaches, concomitant medication, and co-morbidities (eg, asthma) in consideration. In case of 
continued tolerance issues, and if symptomatic headache medication (eg, acetylsalicylic acid 
1000mg) is not sufficient, a further step can be to reduce dose of IMP to 
(see Section 
7.2). 
Severe headache is defined as severe pain limiting self-care activities of daily living (ADL) or 
new/prolonged hospitalization for management of headache or life-threatening consequences 
requiring urgent medical intervention. Self-care ADL refer to bathing, dressing and undressing, 
feeding self, using the toilet, taking medications. Treatment of headache will be provided as 
clinically indicated according to the national guidelines. 
Subjects experiencing severe headache will complete the Headache Questionnaire daily until 
resolution (ie, if headache becomes moderate or mild, normal collection of AEs should apply). 
At the clinic visit when the severe headache is reported, the Headache Questionnaire will be 
followed by a neurological assessment (including fundoscopy). If the severe headache is initially 
reported at a home visit or during a telephone call, the subject should be reviewed at the study 
site as soon as is practically possible for further investigations. Further workup will be performed 
at the discretion of the investigator and may include, eg, a computed tomography scan, magnetic 
resonance imaging and/or a lumbar puncture for cerebral spinal fluid collection. In addition, 
samples for exploratory biomarkers should be taken for subjects experiencing severe headache 
Table 5-2). These investigations will be performed to further understand the mechanism of 
headache in these subjects. 


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Details of neurological examination to be performed are provided in Section 9.3.4. The 
Headache Questionnaire will be provided in the study procedures manual. 
Has been changed to: 
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. 
Treatment of headaches should be as per national guidelines and take medical history of previous 
headaches, concomitant medication, and co-morbidities (eg, asthma) in consideration. In case of 
continued tolerance issues, and if symptomatic headache medication (eg, acetylsalicylic acid 
1000mg) is not sufficient, a further step can be to reduce dose of IMP to 
(see Section 
7.2). 
Determination of the severity of headache will be consistent with National Cancer Institute 
CTCAE version 5.0. Severe headache is defined as severe pain limiting self-care activities of 
daily living (ADL) or new/prolonged hospitalization for management of headache or life-
threatening consequences requiring urgent medical intervention. Self-care ADL refer to bathing, 
dressing and undressing, feeding self, using the toilet, taking medications. Treatment of headache 
will be provided as clinically indicated according to the national guidelines. 
Study participants experiencing severe and/or serious headache will complete the Headache 
Questionnaire daily until resolution (ie, if headache becomes nonserious, moderate or mild, or 
completely resolved, whichever comes first normal collection of AEs should apply). At the 
clinic visit when the severe headache is reported, the Headache Questionnaire will be followed 
by a neurological assessment (including fundoscopy). If the severe or serious headache is 
initially reported at a home visit or during a telephone call, the study participant should be 
reviewed at the study site as soon as is practically possible for further investigations. Study 
participants should be monitored for signs and symptoms suggestive of central nervous 
system involvement and evaluated immediately if other causes (eg, meningitis, intracranial 
bleeding) are suspected. Further neurological workup willmay be performed (if indicated) at 
the discretion of the investigator and may include, eg, 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 collectedtaken for study participants 
experiencing severe or serious headache when possible (Table 5-2). These investigations will 
be performed to further understand the mechanism of headache in these study participants. 
Details of neurological examination to be performed are provided in Section 9.3.4. The 
Headache Questionnaire will be provided in the study procedures manual.  
Change #30 
9.1.11 Management of moderate or severe diarrhea; first and second paragraph  
Moderate or severe diarrhea is defined as an increase of ≥4 stools per day over Baseline or 
incontinence due to urgency of diarrhea or new/prolonged hospitalization for management of 
diarrhea or limiting self-care ADL or life-threatening consequences requiring urgent medical 
intervention. 


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Stool collection and analysis will be performed for subject reporting moderate or severe diarrhea. 
The frequency of stool sampling will be as clinically indicated in the opinion of the investigator. 
Analysis of stool samples will be performed locally.  
Has been changed to: 
Moderate or severe diarrhea is defined as an increase of ≥4 stools per day over Baseline or 
incontinence due to urgency of diarrhea or new/prolonged hospitalization for management of 
diarrhea or limiting self-care ADL or life-threatening consequences requiring urgent medical 
intervention. Determination of the severity of diarrhea will be consistent with CTCAE 
version 5.0. 
Stool collection and analysis will be performed for subjects reporting moderate or severe 
diarrhea. The frequency of stool sampling will be as clinically indicated in the opinion of the 
investigator and assessed per local guidance. Analysis of stool samples will be performed 
locally. In addition, collection of blood samples for the assessment of exploratory safety 
biomarkers is required for study participants with severe GI disturbances including 
diarrhea. 
Change #31 
9.3.2 Vital signs, second paragraph 
Subjects should be sitting for 5 minutes prior and during the collection of BP and PR 
measurements. During site Visits 2, 3, and 4, vital signs will be measured prior to IMP 
administration, at the end of the infusion, and 2 and 4h after the end of the infusion. From 
Visit 5, vital signs will be measured predose, at the end of the infusion and 2h after the end of the 
infusion only. At nondosing visits, vital signs need only be taken once during the visit. 
Has been changed to: 
Study participants should be sitting for 5 minutes prior and during the collection of BP and PR 
measurements. During site Visits 2, 3, and 4, vital signs will be measured prior to IMP 
administration, at the end of the infusion, and 2 and 4h after the end of the infusion. From Visit 5 
to Visit 25, vital signs will be measured predose, at the end of the infusion and 2h after the end 
of the infusion only. During Treatment Period Part 2, vital signs will be measured predose, 
and at the end of the infusion. At nondosing visits, vital signs need only be taken once during 
the visit. 
Change #32 
10.4.3 Patient Global Impressions 
The following sentence has been added:  
Both PGI-C and PGI-S will be used as anchors to determine a meaningful change 
threshold for CIDP PRO and fatigue scores. 


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Change #33 
11.1 Pharmacokinetic variable; Table 11-1 
Table 11-1: Serial blood sampling for rozanolixizumab concentration 
Matrix 
Time after start of sc infusion 
Plasma 
Predose and postdose at 4h after finishing the 
 on Visits 2, 4, and 8. 
Predose samples will be taken at Visits 14, 18, 22. 
Nondosing Visit 26, samples to be taken once during the visit 
Has been changed to: 
Table 11-1: Serial blood sampling for rozanolixizumab concentration 
Matrix 
Time after start of sc infusion 
Plasma 
Predose and postdose at 4h after finishing the 
 on Visits 2, 4, and 8. 
Predose samples will be taken at Visits 14, 18, and 22 and monthly at site visits during 
Treatment Period Part 2. 
Nondosing Visit 26, samples to be taken once during the visit 
Change #34 
12 Assessment of immunological variables; second and third paragraph 
For all immunological assessments, blood samples will be collected predose (at dosing visits) by 
qualified site personnel at the same time that samples are collected for standard clinical 
laboratory. Serum complement (C3, C4), plasma complement (C3a, C5a), and serum cytokine 
samples will also be taken 4h postdose at Visits 3, 8, 10, 14, 18, and 22. Additional samples may 
be collected in case of infusion reactions (see Table 5-2).  
Has been changed to: 
For all immunological assessments, blood samples will be collected predose (at dosing visits) by 
qualified site personnel at the same time that samples are collected for standard clinical 
laboratory. Serum complement (C3, C4), plasma complement (C3a, C5a), and serum cytokine 
samples will also be taken 4h postdose at Visits 3, 8, 10, 14, 18, and 22 and quarterly at site 
visit during Treatment Period Part 2. Additional samples may be collected in case of infusion 
reactions (see Table 5-2). The time and date of the blood draws will be recorded in the eCRF. 
Anti-drug antibodies samples will also be taken predose at Visits 2,  4, 8, 14, 18, 22, 
monthly at site visits during Treatment Period Part 2 and at Visit 26. 
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 


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will be evaluated in a titration assay to quantify the ADA level and will be reported as titer 
(reciprocal dilution factor including MRD). 
Change #35 
14.3.1 Safety analyses; first paragraph 
The incidence of subjects with TEAEs will be determined. Furthermore, the absolute and relative 
frequencies for subjects with a given TEAE with respect to the preferred term according to the 
latest available version of the Medical Dictionary for Regulatory Activities (MedDRA®), will be 
determined within each system organ class. Additional tables will summarize TEAEs by 
maximum severity and causal relationship with rozanolixizumab, as judged by the investigator. 
Adverse events will be categorized by severity according to the National Institutes for Health 
CTCAE version 4.03 grading (National Institutes for Health, 2009). In case the CTCAE grading 
is not available, the intensity (mild/moderate/severe) will be utilized. The TEAEs leading to 
discontinuation of IMP and the serious TEAEs will also be summarized. The action taken, time 
of onset relative to dosing, actual dose received, and duration of each AE will be listed only. 
Has been changed to: 
The incidence of study participants with TEAEs will be determined. Furthermore, the absolute 
and relative frequencies for study participants with a given TEAE with respect to the preferred 
term according to the latest available version of the Medical Dictionary for Regulatory Activities 
(MedDRA®), will be determined within each system organ class. Additional tables will 
summarize TEAEs by maximum severity and causal relationship with rozanolixizumab, as 
judged by the investigator. Adverse events will be categorized by severity according to the 
National Institutes for Health CTCAE version 5.0 4.03 grading (National Institutes for Health, 
20092017). In case the CTCAE grading is not available, the intensity (mild/moderate/severe) 
will be utilized. The TEAEs leading to discontinuation of IMP and the serious TEAEs will also 
be summarized. The action taken, time of onset relative to dosing, actual dose received, and 
duration of each AE will be listed only. 
Change #36 
14.4.2 Pharmacokinetic variables, first paragraph 
Pharmacokinetic variables of rozanolixizumab like AUC (area under the curve from 0 to infinity) 
and Cmax (maximum observed plasma concentration) cannot be derived, since blood sampling 
will be performed at 1 time point post dosing per visit only. Thus, PK is restricted to 
concentration data. 
Has been changed to: 
Pharmacokinetic variables of rozanolixizumab like AUC (area under the curve from 0 to infinity) 
and Cmax (maximum observed plasma concentration) cannot be derived, since due to blood 
sampling will be performed at 1 time point post dosing per visit only scheme. Thus, PK is 
restricted to concentration data. 
Change #37 
14.7 Planned interim analysis and data monitoring 
The following paragraph has been added: 


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Two interim analyses will be conducted. Firstly at the time of the lock of the parent study 
CIPD01 and additionally once all study participants have completed Part 1 of the 
Treatment Period in CIDP04 and have either entered into the Treatment Period Part 2 or 
the Observation Period.  Details of the planned analyses will be fully outlined in the 
respective SAPs. 
Change #38 
14.8 Determination of sample size 
No formal sample size calculation can be performed. All subjects from the parent study 
(eg, CIDP01) eligible for the OLE will be included. 
Has been changed to: 
No formal sample size calculation can be performed. All study participants from the parent study 
(eg, CIDP01, with a maximum of 
study participants to be randomized) eligible for the 
OLE will be included 
Change #39 
17 References 
The following reference has been removed: 
Doessegger L, Banholzer ML. Clinical development methodology for infusion-related reactions 
with monoclonal antibodies. Clin Transl Immunology. 2015;4(7):e39. 


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19 
DECLARATION AND SIGNATURE OF INVESTIGATOR 
I confirm that I have carefully read and understood this protocol and agree to conduct this 
clinical study as outlined in this protocol, according to current Good Clinical Practice and local 
laws and requirements. 
I will ensure that all subinvestigators and other staff members read and understand all aspects of 
this protocol. 
I have received and read all study-related information provided to me. 
The objectives and content of this protocol as well as the results deriving from it will be treated 
confidentially, and will not be made available to third parties without prior authorization by 
UCB. 
All rights of publication of the results reside with UCB, unless other agreements were made in a 
separate contract. 
Investigator: 
 
 
 
 
Printed name 
 
Date/Signature 
 


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20 
SPONSOR DECLARATION 
I confirm that I have carefully read and understand this protocol and agree to conduct this 
clinical study as outlined in this protocol and according to current Good Clinical Practice. 


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Approval Signatures
Name:
cidp04-protocol-amendment-3
Version:
2. 0
Document Number:
CLIN-000148900
Title:
CIDP04 protocol amendment 3
Approved Date:
17 Mar 2020
Document Approvals
Approval
Verdict: Approved
Name:
Capacity: Medical
Date of Signature: 17-Mar-2020 07:51:13 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Subject Matter Expert
Date of Signature: 17-Mar-2020 10:04:54 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Clinical
Date of Signature: 17-Mar-2020 10:24:26 GMT+0000

SAP_001.pdf: 
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Statistical Analysis Plan 
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STATISTICAL ANALYSIS PLAN  
Study: CIDP04 
Product: Rozanolixizumab 
AN OPEN-LABEL EXTENSION STUDY TO INVESTIGATE THE LONG-TERM SAFETY, 
TOLERABILITY, AND EFFICACY OF ROZANOLIXIZUMAB IN SUBJECTS WITH 
CHRONIC INFLAMMATORY DEMYELINATING POLYRADICULONEUROPATHY 
(CIDP) 
 
SAP/Amendment Number Date 
Final SAP 1.0 
08 Jun 2021 
 
 
 
 
Confidentiality Statement 
Confidential 
This document is the property of UCB and may not – in full or in part – be passed on, 
reproduced, published, or otherwise used without the express permission of UCB. 
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TABLE OF CONTENTS 
LIST OF ABBREVIATIONS .................................................................................................... 6 
1 
INTRODUCTION ............................................................................................................. 9 
2 
PROTOCOL SUMMARY ................................................................................................. 9 
2.1 
Study objectives ............................................................................................................. 9 
2.1.1 
Primary objective ................................................................................................... 9 
2.1.2 
Secondary objective ............................................................................................... 9 
2.1.3 
Exploratory objectives ........................................................................................... 9 
2.2 
Study variables ............................................................................................................. 10 
2.2.1 
Safety variables .................................................................................................... 10 
2.2.1.1 
Primary safety variable .............................................................................. 10 
2.2.1.2 
Other safety variables ................................................................................ 10 
2.2.2 
Other variables ..................................................................................................... 11 
2.2.2.1 
Efficacy variables ...................................................................................... 11 
2.2.2.2 
Patient-reported outcome variables ........................................................... 12 
2.2.2.3 
Pharmacokinetic variable .......................................................................... 12 
2.2.2.4 
Pharmacodynamic variables ...................................................................... 12 
2.2.2.5 
Immunological variables ........................................................................... 12 
2.2.2.6 
Exploratory biomarkers ............................................................................. 12 
2.3 
Study design and conduct ............................................................................................ 13 
2.4 
Determination of sample size ....................................................................................... 14 
3 
DATA ANALYSIS CONSIDERATIONS ...................................................................... 14 
3.1 
General presentation of summaries and analyses ........................................................ 14 
3.2 
General study level definitions .................................................................................... 15 
3.2.1 
Analysis time points ............................................................................................ 15 
3.2.1.1 
Relative day ............................................................................................... 15 
3.2.1.2 
Study periods ............................................................................................. 16 
3.3 
Definition of Baseline values ....................................................................................... 16 
3.4 
Protocol deviations....................................................................................................... 18 
3.5 
Analysis sets................................................................................................................. 18 
3.5.1 
Enrolled Set ......................................................................................................... 18 
3.5.2 
Safety Set ............................................................................................................. 18 
3.5.3 
Full Analysis Set .................................................................................................. 19 
3.5.4 
Modified Full Analysis Set .................................................................................. 19 
3.5.5 
Pharmacokinetic Per-Protocol Set ....................................................................... 19 
3.5.6 
Pharmacodynamic Per-Protocol Set .................................................................... 19 
3.6 
Treatment assignment and treatment groups ............................................................... 19 
3.7 
Center pooling strategy ................................................................................................ 20 
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3.8 
Coding dictionaries ...................................................................................................... 20 
3.9 
Changes to protocol-defined analyses ......................................................................... 20 
3.9.1 
Changes related to COVID-19 ............................................................................. 21 
3.9.2 
With the event of the COVID-19 pandemic, an additional protocol deviation 
category (related/not related to COVID-19) has been introduced as well as an 
eCRF page "COVID-19 Impact" to identify visits and procedures which may 
have been impacted. These will be presented in the analysis results as described 
in Section 5.2.Interim Analysis ........................................................................... 21 
4 
STATISTICAL/ANALYTICAL ISSUES ....................................................................... 21 
4.1 
Adjustments for covariates .......................................................................................... 21 
4.2 
Handling of dropouts or missing data .......................................................................... 21 
4.2.1 
Efficacy data ........................................................................................................ 21 
4.2.2 
Safety Laboratory data ......................................................................................... 22 
4.2.3 
Pharmacodynamic data ........................................................................................ 22 
4.2.4 
Immunological data ............................................................................................. 22 
4.2.5 
Rozanolixizumab concentration data ................................................................... 22 
4.2.6 
Electrocardiogram data ........................................................................................ 23 
4.2.7 
Dates and Times .................................................................................................. 23 
4.2.8 
Handling of repeated and unscheduled measurements ........................................ 26 
4.3 
Handling of measurements obtained at the premature end of treatment visit .............. 26 
4.4 
Data monitoring committee (DMC)............................................................................. 26 
4.4.1 
Timing of DMC ................................................................................................... 27 
4.4.2 
Data required for DMC ........................................................................................ 27 
4.5 
Multicenter studies ....................................................................................................... 27 
4.6 
Multiple comparisons/multiplicity ............................................................................... 27 
4.7 
Use of an efficacy subset of study participants ............................................................ 27 
4.8 
Active-control studies intended to show equivalence .................................................. 27 
4.9 
Examination of subgroups ........................................................................................... 27 
5 
STUDY POPULATION CHARACTERISTICS ............................................................. 27 
5.1 
Study participant disposition........................................................................................ 27 
5.2 
Protocol deviations....................................................................................................... 28 
6 
DEMOGRAPHICS AND OTHER BASELINE CHARACTERISTICS ........................ 28 
6.1 
Demographics .............................................................................................................. 28 
6.2 
Other Baseline characteristics ...................................................................................... 29 
6.3 
Medical and procedures history ................................................................................... 30 
6.4 
Prior and concomitant medications .............................................................................. 30 
6.4.1 
Prior medication definition .................................................................................. 30 
6.4.2 
Concomitant medication definition ..................................................................... 30 
7 
MEASUREMENTS OF TREATMENT COMPLIANCE ............................................... 31 
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8 
EFFICACY ANALYSES ................................................................................................ 31 
8.1 
iRODS .......................................................................................................................... 31 
8.1.1 
iRODS scores ....................................................................................................... 31 
8.1.2 
CIDP relapse (iRODS) ........................................................................................ 32 
8.2 
Adjusted INCAT .......................................................................................................... 33 
8.2.1 
Adjusted INCAT score ........................................................................................ 33 
8.2.2 
CIDP relapse (INCAT) ........................................................................................ 34 
8.3 
Maximum Grip Strength (Assessed by Site Personnel) ............................................... 34 
8.3.1 
Maximum Grip Strength (Assessed by Site Personnel) ...................................... 34 
8.3.2 
CIDP relapse (Maximum Grip Strength, 14 kPa) ................................................ 34 
8.3.3 
CIDP relapse (Maximum Grip Strength, 8 kPa) .................................................. 34 
8.4 
CIDP Relapse ............................................................................................................... 35 
8.5 
Use of rescue medication ............................................................................................. 35 
8.6 
RT-MRC sum score ..................................................................................................... 35 
8.6.1 
CIDP PRO and Fatigue scale ............................................................................... 35 
8.6.2 
PGIS and PGIC .................................................................................................... 36 
9 
PHARMACOKINETICS AND PHARMACODYNAMICS .......................................... 36 
9.1 
Pharmacokinetics ......................................................................................................... 36 
9.2 
Pharmacodynamics ...................................................................................................... 37 
9.2.1 
Total Serum IgG .................................................................................................. 37 
9.3 
Immunological analyses............................................................................................... 38 
9.3.1 
Immunoglobulins ................................................................................................. 38 
9.3.2 
Anti-drug antibodies ............................................................................................ 38 
9.3.3 
Serum complement levels and plasma complement levels .................................. 40 
9.3.4 
Cytokines ............................................................................................................. 40 
9.3.5 
CIDP-specific auto-antibodies ............................................................................. 40 
9.3.6 
Tetanus-and influenza A virus-specific IgG antibodies ...................................... 40 
10 SAFETY ANALYSES ..................................................................................................... 41 
10.1 
Extent of exposure ....................................................................................................... 41 
10.2 
Adverse events ............................................................................................................. 41 
10.3 
Clinical laboratory evaluations .................................................................................... 44 
10.3.1 
Potential drug-induced liver injury ...................................................................... 45 
10.4 
Vital signs, physical findings, and other observations related to safety ...................... 46 
10.4.1 
Vital signs ............................................................................................................ 46 
10.4.2 
Electrocardiograms .............................................................................................. 46 
10.4.3 
Suicidality ............................................................................................................ 47 
10.4.4 
Other safety variables .......................................................................................... 47 
11 OTHER ANALYSES ...................................................................................................... 48 
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12 REFERENCES ................................................................................................................ 49 
13 APPENDICES ................................................................................................................. 50 
13.1 
Conversion of iRODS scores ....................................................................................... 50 
13.2 
MA criteria for Rozanolixizumab program ................................................................. 51 
STATISTICAL ANALYSIS PLAN SIGNATURE PAGE ..................................................... 55 
 
 
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LIST OF ABBREVIATIONS 
ADA 
Anti-drug antibody 
ADaM 
Analysis Dataset Model 
AE 
Adverse event 
AEoF 
Adverse event of focus 
ALC 
Absolute lymphocyte count 
ALP 
Alkaline phosphatase 
ALQ 
Above the limit of quantification 
ALT 
Alanine aminotransferase 
ANC 
Absolute neutrophil count 
AST 
Aspartate aminotransferase 
AUC 
Area under the curve 
BLQ 
Below the limit of quantification 
BMI 
Body mass index 
BP 
Blood pressure 
CDISC 
Clinical Data Interchange Standards Consortium 
CI 
Confidence interval 
CIDP 
Chronic inflammatory demyelinating polyradiculoneuropathy 
COVID-19 
Coronavirus Disease of 2019 
Cmax 
Maximum concentration 
CRO 
Ccontract research organization 
CTCAE 
Common Terminology Criteria for Adverse Events 
CSP 
Cclinical study protocol 
CSR 
Clinical study report 
C-SSRS 
Columbia Suicide Severity Rating Scale 
CV 
Ccoefficient of variation 
DBP 
Diastolic blood pressure 
DMC 
Data Monitoring Committee 
ECG 
Eelectrocardiogram 
eCRF 
Eelectronic Case Report Form 
EFNS 
European Federation of Neurological Societies 
ES 
Enrolled Set 
EudraCT 
European Union Drug Regulating Authorities Clinical Trials 
FAS 
Full Analysis Set 
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FDA 
Food and Drug Administration 
FV 
Final Visit 
geoCV 
Geometric coefficient of variation 
geoMean 
Geometric mean 
GGT 
Gamma glutamyltransferase 
HDL 
High density lioprotein 
HLT 
High level term 
hsCRP 
High sensitivity C-reactive protein 
ICH 
International Council for Harmonisation  
Ig 
Immunoglobulin 
IgA 
Immunoglobulin A 
IgE 
Immunoglobulin E 
IgG 
Immunoglobulin G 
IgM 
Immunoglobulin M 
IGRA 
Interferon-gamma release assay 
IMP 
Investigational medicinal product 
INCAT 
Inflammatory Neuropathy Cause and Treatment 
IPD 
Important protocol deviation 
iRODS 
Inflammatory Rasch-built Overall Disability Scale 
IVIg 
Intravenous immunoglobulin 
LDH 
Lactate dehydrogenase 
LDL 
Low density lipoprotein 
LLOQ 
Lower limit of quantification 
logit 
Log odds unit 
MA 
Markedly abnormal 
MCID-SE  
Minimum clinically important differences-standard error 
MedDRA 
Medical Dictionary for Regulatory Activities 
MFAS 
Modified full analysis set 
MRC 
Medical Research Council 
MRD 
Minimum required dilution 
n 
Number of study participants 
NF-L 
Neurofilament light chain 
OLE 
Open-Label Extension 
PD 
Pharmacodynamic 
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PDILI 
Potential drug-induced liver injury 
PD-PPS 
Pharmacodynamic Per-Protocol Set 
PEOT 
Premature end of treatment 
PGIC 
Patient Global Impressions of Change 
PGIS 
Patient Global Impressions of Severity 
PK 
Pharmacokinetic 
PK-PPS 
Pharmacokinetic Per-Protocol Set 
PNS 
Peripheral Nerve Society 
PT 
Preferred term 
QTcF 
QT corrected for heart rate using Fridericia’s formula 
RBC 
Red blood cell 
RNA 
Ribonucleic acid 
SAE 
Serious adverse event 
SAP 
Statistical Analysis Plan 
SBP 
Systolic blood pressure 
sc 
Subcutaneous 
SCIg 
Subcutaneous immunoglobulin 
SD 
Standard deviation 
SE 
Standard error 
SOC 
System organ class 
SS 
Safety Set 
TEAE 
Treatment-emergent adverse event 
TFLs 
Tables, figures and listings 
ULN 
Upper limit of normal 
USA 
United States of America 
WBC 
White blood cell 
WHODD 
World Health Organization Drug Dictionary 
 
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1 
INTRODUCTION 
The purpose of this statistical analysis plan (SAP) is to provide all information necessary to 
perform the required statistical analyses of study CIDP04. It defines the summary tables, figures, 
and listings (TFLs) to be included in the Clinical Study Report (CSR).  
This SAP is based upon, and assumes familiarity, with the following documents: 
• Final Protocol: 14 Dec 2018 
• Protocol Amendment 2: 12 Jul 2019 
• Protocol Amendment 3: 16 Mar 2020 
Unless otherwise specified below, the study will be analyzed as described in the most recent 
version of the protocol (European Union Drug Regulating Authorities Clinical Trials [EudraCT]-
Number: 2018-004392-12). 
If a future protocol amendment necessitates a substantial change to the statistical analysis of the 
study data, this SAP may be amended accordingly. Changes to the analysis from the protocol are 
documented in section 3.9.  
The content of this SAP is compatible with the International Council for Harmonisation 
(ICH)/Food and Drug Administration (FDA) E9 Guidance documents. 
UCB is the Sponsor and PAREXEL is the Contract Research Organization (CRO) for this study. 
2 
PROTOCOL SUMMARY 
2.1 
Study objectives 
2.1.1 
Primary objective 
The primary objective of the study is to assess long-term safety and tolerability of weekly doses 
of rozanolixizumab in study participants with chronic inflammatory demyelinating 
polyradiculoneuropathy (CIDP). 
2.1.2 
Secondary objective 
The secondary objective of the study is to assess long-term clinical efficacy of weekly doses of 
rozanolixizumab. 
2.1.3 
Exploratory objectives 
The exploratory objectives of the study are to assess the PD, PK, and immunological variables in 
the treatment course with rozanolixizumab and to assess whether long-term dosing of 
rozanolixizumab improves patient-reported outcomes (PROs). These include: 
• The PD effect of rozanolixizumab as measured by the total immunoglobulin G (IgG) 
concentrations in serum 
• The effects of rozanolixizumab on the concentrations of total protein, albumin, α- and β 
globulins, IgG subclasses, immunoglobulin M (IgM), immunoglobulin A (IgA), 
immunoglobulin E (IgE), and serum and plasma complement levels 
• The incidence and emergence of anti-drug antibody (ADA) with respect to immunogenicity 
and PK and PD 
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• The effect of rozanolixizumab on complement and cytokines 
• The plasma concentrations of rozanolixizumab administered by subcutaneous (sc) infusion 
• The PD effect of rozanolixizumab as measured by neurofilament light chain (NF-L) in serum 
• The effect of rozanolixizumab on CIDP-specific auto-antibody levels 
• The effect of rozanolixizumab on vaccine antibody levels (influenza A and tetanus) 
• The effect of rozanolixizumab on exploratory biomarkers, and explore the relationship 
between protein and metabolite biomarkers and cause, progression, and appropriate treatment 
of CIDP 
2.2 
Study variables 
For all study variables, the Baseline values for study participants who enroll directly into 
CIDP04 will be the Baseline values from the parent study (eg, CIDP01). For study participants 
with a gap period prior to enrollment in CIDP04, details of the analyses of specific Baseline are 
defined in Section 3.3. 
2.2.1 
Safety variables 
2.2.1.1 
Primary safety variable 
The primary safety variable is: 
Occurrence of treatment-emergent adverse events (TEAEs) 
2.2.1.2 
Other safety variables 
The other safety variables are: 
• TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)  
• Vital sign values and changes from Baseline (systolic and diastolic blood pressure (BP), 
pulse rate (PR), body temperature, and body weight) at each scheduled assessment during 
Treatment and Observation Periods 
• Physical examination findings 
• Neurological examination findings 
• 12-lead electrocardiogram (ECG) values and change from Baseline at each scheduled 
assessment during Treatment and Observation Periods 
• Laboratory values and changes from Baseline at each scheduled assessment during 
Treatment and Observation Periods (hematology, clinical chemistry and urinalysis) 
• Tuberculosis Signs and Symptoms Questionnaire at each scheduled assessment during 
Treatment and Observation Periods 
• Values and change from Baseline in concentrations of total protein, albumin, α- and β-
globulins at each scheduled assessment during Treatment and Observation Periods 
 
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2.2.2 
Other variables 
2.2.2.1 
Efficacy variables 
Preliminary definitions linked to assessment of relapse are: 
• CIDP relapse (Inflammatory Rasch‐Built Overall Disability Scale [iRODS]) is defined as a 
clinically important deterioration from Baseline in iRODS score, ie, a minimum clinically 
important differences-standard error (MCID-SE) of ≤-1.96 
• CIDP relapse (adjusted Inflammatory Neuropathy Cause and Treatment [INCAT]) is defined 
as an increase from Baseline of at least 1 point in the adjusted INCAT score. The adjusted 
score is identical to the INCAT disability score except for the exclusion of changes in upper 
limb function from 0 (normal) to 1 (minor symptoms) or from 1 to 0  
• CIDP relapse (maximum grip strength as assessed by site personnel) is defined as a clinically 
important deterioration from Baseline in grip strength as measured by site personnel, ie, a 
decline of >14kPa  
The other efficacy variables are: 
• Values and absolute change from Baseline in iRODS scores at each scheduled assessment 
during the Treatment and Observation Periods 
• Study participant experienced CIDP relapse (iRODS) up to Week 25 and Week 77 (where 
applicable) from Baseline 
• Time to CIDP relapse (iRODS) during the Treatment Period from Baseline 
• Study participant experienced CIDP relapse (adjusted INCAT) up to Week 25 and Week 77 
(where applicable) from Baseline 
• Time to CIDP relapse (adjusted INCAT) during the Treatment Periods from Baseline 
• Values and absolute change from Baseline in adjusted INCAT score at each scheduled 
assessment during the Treatment and Observation Periods 
• Study participant experienced CIDP relapse (maximum grip strength as assessed by site 
personnel) up to Week 25 and Week 77 (where applicable) from Baseline 
• Time to CIDP relapse (maximum grip strength as assessed by site personnel) during the 
Treatment Periods from Baseline 
• Values and absolute change from Baseline in maximum grip strength score (maximum of 
assessments) taken by site personnel at each scheduled assessment during the Treatment and 
Observation Periods 
• Values and absolute change from Baseline in RT-MRC sum score at each scheduled 
assessment during the Treatment and Observation Periods 
• Study participants receiving rescue medication during Treatment Periods 
• Time to rescue medication administration during Treatment Periods 
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2.2.2.2 
Patient-reported outcome variables 
• Values and change from Baseline in fatigue domain scores at each scheduled assessment 
during the Treatment and Observation Periods 
• Values and change from Baseline in CIDP patient-reported outcome (PRO) instrument 
domain scores at each scheduled assessment during the Treatment and Observation Periods 
• Values and change from Baseline in Patient Global Impressions of Severity (PGIS) at each 
scheduled assessment during the Treatment and Observation Periods 
• Patient Global Impressions of Change (PGIC) value at each scheduled assessment during the 
Treatment and Observations Periods 
2.2.2.3 
Pharmacokinetic variable 
Plasma concentration of rozanolixizumab at each scheduled assessment during the Treatment 
Periods. 
2.2.2.4 
Pharmacodynamic variables 
• Minimum value and maximum decrease (absolute and percentage) from Baseline in total 
serum IgG concentration during the study 
• Values and change (absolute and percentage) from Baseline in total serum IgG 
concentrations at each scheduled assessment during Treatment and Observation Periods 
• Values and change (absolute and percentage) from Baseline in serum IgG subclass 
concentrations at each scheduled assessment during Treatment and Observation Periods 
• Values and change (absolute and percentage) from Baseline in NF-L levels at each scheduled 
assessment during Treatment and Observation Periods 
2.2.2.5 
Immunological variables 
• Values and change from Baseline in serum immunoglobulin concentrations (total IgA, IgE, 
and IgM) at each scheduled assessment during Treatment and Observation Periods 
• Values and change from Baseline in serum complement levels (C3 and C4) and plasma 
complement levels (C3a and C5a) at each scheduled assessment during Treatment Period 
• ADA (anti-rozanolixizumab antibodies) status (negative or confirmed positive) and the 
confirmed positive titer at each scheduled assessment during Treatment and Observation 
Periods 
• Values and change from Baseline in cytokines at each scheduled assessment during 
Treatment and Observation Periods 
• Change in CIDP-specific auto-antibody levels in serum from Baseline during Treatment and 
Observation Periods (This variable is not in scope of this analysis and reported separately.) 
• Values and change from Baseline in Tetanus- and influenza A virus-specific IgG antibodies 
during Treatment and Observation Periods 
2.2.2.6 
Exploratory biomarkers 
The exploratory biomarkers are: 
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• Protein and metabolites changes that may be measured to understand the cause, progression, 
and appropriate treatment of CIDP 
• Exploratory biomarkers such as, but not limited to, B-cell activating factor and Circulating 
Immune Complexes may be measured to evaluate the effect of rozanolixizumab 
• Absolute change from Baseline relating to mechanism of action, disease activity, treatment 
response, and clinical outcome at each scheduled assessment during the Treatment and 
Observation Periods 
These variables are not in scope of this analysis and reported separately. 
2.3 
Study design and conduct 
This is a Phase 2A, multicenter, single-arm, open-label extension OLE study to evaluate the 
safety, tolerability, and efficacy of rozanolixizumab during long-term treatment of study 
participants with CIDP from a previous study with rozanolixizumab. 
Access to CIDP04 for study participants coming from CIDP01: Study participants from CIDP01 
who have completed the Treatment Period (ie, all visits up to Visit 17) without a relapse of CIDP 
will be offered the opportunity to be directly enrolled into this OLE study. Study participants will 
enter CIDP04 on the same day as the last study visit in CIDP01: Visit 17. Study participants 
from CIDP01 who have experienced a relapse of CIDP (during either the Treatment Period or 
the Observation Period), have been successfully rescued and stabilized with SOC medication and 
have consented to enter this OLE study, may be assessed for eligibility. The study participants 
who relapsed during the Treatment Period of CIDP01 will be offered entry into CIDP04 only 
once CIDP01 study results are available, after the blind has been broken at the end of the study, 
and it is confirmed they received placebo in CIDP01. 
In case the study participants have a gap period between the end of the parent study and the start 
of this OLE study (ie, last visit in the parent study does not take place on the same day as entry 
of study CIDP04), a Screening Period of 2 to 5 weeks will be applicable to confirm that the study 
participant still meets the eligibility criteria for entry in the OLE study and allow for a smooth 
transition between the treatment used by the study participants during the gap period and the 
initiation of rozanolixizumab. The CIDP04 study includes a first 24-week Treatment Period (Part 
1) followed by a second Treatment Period (Part 2) of up to 52 weeks (or until an Access Program 
or equivalent is in place, whichever comes first). Entry to the Treatment Period (Part 2) will be 
contingent to a favorable individual benefit-risk after careful individual assessment has been 
performed for each study participant completing Treatment Period (Part 1). An 8-week 
Observation Period will be followed in case of premature termination or after completion of the 
Treatment Periods (either at end of Part 1 for study participants not continuing in Part 2 or at end 
of Part 2). The Observation Period will not be performed in the event of a study participant 
continuing treatment with rozanolixizumab after the end of the study (ie, in case of an Access 
Program), in which case only the premature end of treatment (PEOT) (Visit 26) Visit will be 
performed. 
During the Treatment Periods (Part 1 and Part 2), study participants will have weekly visits 
(either on site or at home) during which they will be dosed with a sc infusion of rozanolixizumab 
up to 
. The initial dose in the study will be based on the dose the study participant has 
received at the completion of the parent study (eg, CIDP01). For study participants who were 
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known to be on placebo in the parent study, a starting dose of 
 will be used. The 
maximum dose will be 
. See Section 7.2 in the protocol for details on treatment to be 
administered. 
During the first 4 weeks of the Treatment Period (Part 1), all visits will be on site. During Weeks 
5 to 8, home visits and on-site visits will be alternating (Weeks 5 and 7 will be on site; Weeks 6 
and 8 will be at home). Starting at Week 9 and through the end of the Treatment Period (Week 
24 and if applicable, Week 76), study participants will have 1 visit on site followed by 3 visits at 
home for every 4-week period.  
After the completion of the Treatment Period(s) (starting at Week 25 for study participants only 
completing Part 1 or from Week 77 for study participants completing both Part 1 and Part 2 or at 
any early timepoint in case of PEOT), study participants will enter the 8-week Observation 
Period where no IMP will be administered. During the Observation Period, study participants 
will have on-site visits at the entry and exit of the Observation Period (Weeks 25/77, and 32/84, 
and home visits at Weeks 27/79 and 29/81). Study participants can return to their SOC during the 
Observation Period. In case the study participant enters an Access Program, no Observation 
Period will be performed with the exception of the PEOT (Visit 26) Visit which will be 
immediately completed.  
At the discretion of the investigator and/or study participant, home visits can be changed to site 
visits (eg, for safety reasons). In case an on-site visit cannot be performed, the next visit will be 
performed at site instead of at home, and all safety assessments that should have been completed 
at the missed visits will be performed. 
The assessments to be completed during each visit are presented in Table 5.1 in the protocol. 
2.4 
Determination of sample size 
No formal sample size calculation has been performed since this is an open label extension 
study. All study participants from the parent study (eg, CIDP01) eligible for the OLE will be 
included. 
3 
DATA ANALYSIS CONSIDERATIONS 
3.1 
General presentation of summaries and analyses 
Statistical evaluation for the analyses will be performed by PAREXEL. Analysis Dataset Model 
(ADaM) will adhere to Clinical Data Interchange Standards Consortium (CDISC) guidance 
documents and follow their UCB interpretation. 
All analyses will be performed using SAS version 9.4 or higher (SAS Institute, Cary, North 
Carolina, USA). Continuous variables will be summarized by treatment group (newly treated in 
CIPD04 or previously treated in CIPD01) and by visit (where applicable) with the statistics 
mean, standard deviation (SD), median, minimum, and maximum. Categorical variables will be 
summarized by treatment group and by visit (where applicable) with frequency counts and 
percentages. Geometric coefficient of variation (geoCV), geometric mean (geoMean) and 95% 
Confidence Interval (CI) for the geoMean will also be presented in the descriptive statistics for 
the PK concentration.  
When reporting relative frequencies or other percentage values, the following rules apply: 
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• For values where all study participants fulfill certain criteria, the percentage value will be 
displayed as 100 
• For values where the absolute frequency is zero, there will be no percentage displayed at all 
• All other percentage displays will use 1 decimal place 
• Percentages displayed based on continuous data (eg, percentage changes from baseline) will 
be displayed to 1 decimal place. 
When reporting descriptive statistics, the following rules will apply in general: 
• n will be an integer 
• Mean (arithmetic and geometric), SD and median will use 1 decimal place more than the 
original data 
• Coefficient of variation and geoCV will be reported as a percentage to 1 decimal place 
• Minimum and maximum will be reported using the same number of decimal places as the 
original value 
• If no study participants have data at a given timepoint, for example, then only n=0 will be 
presented. If n<3, then only n, minimum and maximum will be presented. If n=3, then only 
n, mean, median, minimum and maximum will be presented. The other descriptive statistics 
will be left blank.  
Data listings containing all documented data and all calculated data, as required for the variables 
and analysis applicable to the analyses, will be generated. 
The visit type information (home or site visit) will be included in the listings. 
If entry in CIDP04 (Visit 2) is done on the same day as the last visit of the parent study 
(CIDP01) and in case an assessment was performed at last visit of the parent study, data from the 
parent study will be used in the TFLs for Visit 2/Week 1. 
3.2 
General study level definitions 
3.2.1 
Analysis time points 
3.2.1.1 
Relative day 
Relative day for an event will be derived with the date of the first sc infusion of IMP as 
reference. For study participants treated with rozanolixizumab in parent study (eg CIDP01) the 
date of the first sc infusion of IMP administered at the parent study will be used as reference and 
be considered as the date of the first intake of IMP. For study participants treated with placebo 
during the parent study, date of first intake of IMP will be date of first IMP in CIDP04.  
Relative days for an event of measurement occurring before the date of first sc infusion are 
calculated as follows: 
 
Relative Day = [(Event Date – Date of First Infusion)] 
For events or measurements occurring before the date of the first sc infusion, relative day will be 
prefixed with ‘-’ in the data listings. 
The relative day for an event or measurement occurring on or after the reference date to the date 
of the last infusion is calculated as follows: 
 
Relative Day = [(Event Date - Date of First Infusion) + 1] 
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For events or measurements occurring after the date of the last sc infusion, relative day will be 
prefixed with ‘+’ in the data listings. Relative day will be calculated as follows: 
 
Relative Day = + [(Event Date – Date of Last Infusion)] 
There is no relative Day 0. Relative day is not calculated for partial dates in cases where relative 
day is shown in a study participant data listing. In such cases, relative day should be presented as 
‘- -’ in the study participant data listings. 
3.2.1.2 
Study periods 
The total duration of the study for an individual study participant will be up to 89 weeks; this 
includes a 2- to 5-week Screening Period, a 24-week Treatment Period (Part 1), an optional 
maximum 52-week Treatment Period (Part 2), or until an Access Program or equivalent is in 
place whichever comes first, and an 8-week Observation Period in case the participant does not 
enter in an Access Program (or equivalent). Detailed definitions of those periods are as follows: 
• Screening Period is applicable only for study participants with a gap period (ie, last visit in 
the parent study does not take place on the same day as entry of study CIDP04) between 
CIDP01 and CIDP04. It will start from the Screening visit to the day before Visit 2, when the 
first IMP is administered.  
• Treatment Period Part 1 will start from the IMP administered at Visit 2 to the last IMP 
administered in Treatment Period Part 1, ie. Visit 25 (Weeks 1 to 24).  
• The optional Treatment Period Part 2 will start after Visit 25 (non-inclusively) and last up to 
52 weeks (Weeks 25 to 76) to the last IMP administration visit in Treatment Period Part 2. 
• The Observation Period will start after the last IMP administration before Visit 26/PEOT 
(non-inclusively) and end with the Final Visit (FV/Visit 29). 
 
 
Please note that Visit 26 can either refer to week 25, if a participant participates only in 
Treatment Period part 1, or week 77, if a participant also completes Treatment Period part 2. 
The end of the study is defined as the date of the last visit of the last study participant in the 
study. 
3.3 
Definition of Baseline values 
For all study variables, the Baseline values for study participants treated with rozanolixizumab in 
the parent study (ie, CIDP01) will be the Baseline values from the parent study. For study 
participants treated with placebo in the parent study, the Baseline values will be the ones present 
at Baseline in CIDP04 (Visit 2 or if missing, the Screening Visit value (if applicable)). Note this 
Visit 2 value may be corresponding to the last value registered into CIDP01 as these may be the 
same visit. 
Scheduled or unscheduled measurements can be used as the Baseline value. Expected 
measurement-specific Baseline timepoints are presented in Table 3‒1. If an unscheduled 
measurement occurs after the planned baseline measurement timepoint but before dosing, then 
the unscheduled measurement will be used as the baseline value. 
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Table 3‒1: 
Expected Baseline 
Measurement 
Definition of Baseline 
Safety data: 
• 
Safety Lab (Clinical chemistry, 
Hematology, Urinalysis) 
• 
ECG 
• 
Vital signs 
• 
Total protein, albumin, α- and β-globulins 
For study participants treated with rozanolixizumab 
in parent study CIDP01: 
Safety labs, vital signs, total protein, albumin, α- 
and β-globulin: Baseline visit (Day 1) of parent 
study, if missing Screening visit of parent study 
(pre-dose value , if applicable). 
 
For study participants treated with placebo in the 
parent study: 
Safety labs, vital signs, total protein, albumin, α- 
and β-globulin: Baseline visit (Day 1) in CIDP04, 
if missing Screening visit (if applicable) (pre-dose 
value, if applicable). 
 
ECG: mean of triplicate values of Baseline visit 
(Day 1) in CIDP04. If missing Baseline visit, mean 
of triplicate values of Screening visit (if 
applicable). 
 
Efficacy data 
• 
iRODS 
• 
INCAT 
• 
Grip strength (assessed by site personnel) 
• 
RT-MRC and original MRC 
• 
Fatigue score 
• 
CIDP PRO 
• 
PGIS 
• 
PGIC 
For study participants treated with rozanolixizumab 
in parent study CIDP01: 
Baseline visit (Day 1) in CIDP01, if missing 
Screening visit 
 
For study participants treated with placebo in the 
parent study: 
Baseline visit (Day 1) in CIDP04, if missing 
Screening visit (where applicable) 
Immunological variables: 
• 
IgA, IgE, IgM 
• 
Serum complement levels (C3, C4),  
• 
Plasma complement (C3a, C5a)  
• 
ADA 
• 
Cytokines 
• 
Tetanus- and influenza A virus-specific 
IgG antibodies 
For study participants treated with rozanolixizumab 
in parent study CIDP01, refer to baseline definition 
in SAP for the parent study (ie Baseline visit 
(Day 1) of parent study, except for Tetanus- and 
influenza A virus-specific IgG antibodies where 
baseline is Screening visit of parent study). 
 
For study participants treated with placebo in the 
parent study : 
Baseline visit (Day 1) in CIDP04, except for 
Tetanus- and influenza A virus-specific IgG 
antibodies where baseline is Screening visit if 
applicable 
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Table 3‒1: 
Expected Baseline 
Measurement 
Definition of Baseline 
Pharmacodynamical variables: 
• 
Total serum IgG 
• 
NF-L levels 
 
For study participants treated with rozanolixizumab 
in parent study CIDP01, refer to baseline definition 
in SAP for the parent study (ie for Total serum IgG: 
Baseline visit (Day 1) of parent study, if missing 
Screening visit of parent study. For study 
participants treated with placebo in the parent 
study: 
Total serum IgG: Baseline visit (Day 1) in CIDP04, 
if missing Screening visit (where applicable) 
NF-L: Baseline visit (Week 1) in CIDP04 
ADA=anti-drug antibody; C3=complement component 3; C3a=complement component 3a; C4= complement 
component 4; C5a= complement component 5a; CIDP=chronic inflammatory demyelinating 
polyradiculoneuropathy; CIDP PRO=CIDP PRO instrument; ECG=electrocardiogram; IgG=immunoglobulin G; 
IgA=immunoglobulin A; IgE=immunoglobulin E; IgM=immunoglobulin M; INCAT=Inflammatory Neuropathy 
Cause and Treatment; iRODS=inflammatory Rasch-built Overall Disability Scale; NF-L= neurofilament light chain; 
PGIC=Patient Global Impressions of Change ;  PGIS=Patient Global Impressions of Severity; RT-MRC=Rasch-
built, modified-interval Medical Research Council scale. 
3.4 
Protocol deviations 
Important protocol deviations (IPDs) are deviations from the protocol which potentially could 
have a meaningful impact on study conduct or on the primary efficacy, key safety, or PK/PD 
outcomes (if applicable) for an individual participant. The criteria for identifying important 
protocol deviations will be defined in the protocol deviations specifications. Important protocol 
deviations will be reviewed as part of the ongoing data cleaning process and data evaluation. All 
IPDs will be identified and documented prior to unblinding to confirm exclusion from analysis 
sets. 
IPDs potentially related to the Coronavirus Disease 2019 (COVID-19) will be identified via a 
specific eCRF page "COVID-19 Impact" per visit with relationship: confirmed, suspected 
infection, general circumstances around or any other deviation from the protocol due to COVID-
19. These deviations will also be reviewed separately as part of the ongoing data cleaning 
process. 
3.5 
Analysis sets 
3.5.1 
Enrolled Set 
The Enrolled Set (ES) will consist of all study participants who have signed the informed 
consent form. 
3.5.2 
Safety Set 
The Safety Set (SS) will consist of all enrolled study participants who were administered at least 
one dose of rozanolixizumab in CIDP04. 
All safety variables will be analyzed using the SS. 
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3.5.3 
Full Analysis Set 
The Full Analysis Set (FAS) will consist of all participants in the SS, who have a Baseline and at 
least one valid post-Baseline iRODS measurement up to Visit 26/PEOT (inclusively). 
Measurements are considered invalid if the participant received rescue medication before the 
assessment of iRODS. Assessments collected at the same day as first intake of rescue medication 
are considered valid. Invalid measurements will not be used for summary tables, but kept in 
listings. 
3.5.4 
Modified Full Analysis Set 
The Modified Full Analysis Set (MFAS) will consist of all participants in the FAS, excluding 
any participant who either:  
• received a mean actual dose of IMP deviating >10% or <-10% from the total planned dose 
• missed more than two administrations in a period of three months. 
The definition of the dose as well as details for calculations are provided in Section Error! 
Reference source not found.. 
3.5.5 
Pharmacokinetic Per-Protocol Set 
The Pharmacokinetic Per-Protocol Set (PK-PPS) is a subset of the SS, consisting of those 
participants who had no important protocol deviation affecting the plasma concentration of 
rozanolixizumab. Post-Baseline deviations will not necessarily lead to total exclusion of a 
participant from the PK-PPS but may lead to exclusion of specific data. 
3.5.6 
Pharmacodynamic Per-Protocol Set 
The Pharmacodynamic Per-Protocol Set (PD-PPS) is a subset of the SS, consisting of those 
participants who had no important protocol deviation affecting the serum concentrations of total 
IgG, IgG subclasses, or neurofilament light chain. Post-Baseline deviations will not necessarily 
lead to total exclusion of a participant from the PD-PPS but may lead to exclusion of specific 
data. 
3.6 
Treatment assignment and treatment groups 
All study participants will receive rozanolixizumab in the OLE.  
Analyses will be performed by treatment group and for all study participants where stated. The 
treatment groups will be displayed as follows in the TFLs: 
• Treatment group 1: Newly treated (ie, study participants treated with placebo in parent study) 
• Treatment group 2: Previously treated (ie, study participants treated with rozanolixizumab in 
parent study) 
Participants with the reduced dose due to tolerability issues will be mapped under the same 
treatment groups. These study participants will be flagged in the listings. For the Observation 
period the same treatment groups will be displayed. 
It is expected that study participants will have received treatment as randomized in CIDP01 and 
hence safety and efficacy analyses will be based on the randomized treatment group in CIDP01. 
However, if after unblinding of CIDP01 it is determined that a study participant has received 
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treatment different to the treatment they were randomized to, then for safety analyses the study 
participant will be allocated according to the actual treatment they received. 
3.7 
Center pooling strategy 
It is planned to recruit study participants in Europe, United States of America and Canada in this 
study, with possible extension to other regions and countries. No stratification by site or country 
will be performed. 
3.8 
Coding dictionaries 
Adverse events (AEs) will be coded using version 23.1 or later of the Medical Dictionary for 
Regulatory Activities (MedDRA®).  
Medications will be coded according to version Sep 2017 or later of the World Health 
Organization Drug Dictionary (WHODD). Medical procedures will not be coded. If newer 
dictionary versions become available, data will be coded according to the approved internal 
standard dictionary management processes. 
3.9 
Changes to protocol-defined analyses 
 
• CIDP relapse (maximum grip strength as assessed by site personnel) is defined as a clinically 
important deterioration from Baseline in grip strength as measured by site personnel, ie, a 
decline of >14kPa. Additionally, relapses defined by decline of >8kPa will be investigated. 
• The FAS definition is clarified to include at least one valid post baseline iRODS assessment 
result. 
• The PD-PPS will be handled as a subset of the SS instead of the FAS as defined in the 
protocol to not exclude data due to missing efficacy results. 
• To prevent bias in efficacy evaluation, an efficacy assessment is considered invalid, if that 
assessment follows the first intake of rescue medication. Assessments on the same day as 
first intake of rescue medication are considered valid.  
• The MFAS is included as an additional analysis set in the SAP and was not part of the final 
clinical study protocol (CSP). This was added as a sensitivity analysis, to ensure the 
robustness of the efficacy results in relation to dose variation. 
• Elevated liver function tests and potential drug induced liver injury criteria are harmonized 
across the rozanolixizumab program. Protocol-defined criteria are updated to match UCB's 
standard evaluations. Please refer to Section 10.3.1 for a list of the criteria that will be 
evaluated. 
≥3xULN ALT or AST with coexisting ≥2xULN total bilirubin in the absence of 
≥2xULN ALP  
 
will instead be defined as 
 
 
 
(ALT or AST increase >3xULN) and Total bilirubin >2xULN and ALP <2xULN  
 
to match program standards and general medical definitions.   
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• The study variable “Change in CIDP-specific auto-antibody levels in serum from Baseline 
during Treatment and Observation Periods” will not be analyzed in scope of this SAP. 
Sampling dates are captured in the CRF and will be listed. The samples’ results will not be 
part of this study’s database and instead analyzed and reported separately. The same is true 
for exploratory biomarkers RNA, proteins and metabolites variables. 
3.9.1 
Changes related to COVID-19 
3.9.2 
With the event of the COVID-19 pandemic, an additional protocol 
deviation category (related/not related to COVID-19) has been 
introduced as well as an eCRF page "COVID-19 Impact" to identify 
visits and procedures which may have been impacted. These will be 
presented in the analysis results as described in Section 5.2.Interim 
Analysis 
Per protocol section 14.7:  
“Two interim analyses will be conducted. Firstly at the time of the lock of the parent study 
CIPD01 and additionally once all study participants have completed Part 1 of the Treatment 
Period in CIDP04 and have either entered into the Treatment Period Part 2 or the 
Observation Period. Details of the planned analyses will be fully outlined in the respective 
SAPs.” 
The events "lock of the parent study" and "all study participants have completed Part 1 of the 
Treatment Period in CIDP04" are expected to be close together and not differ enough in 
presented data to provide benefit by additional analysis. Therefore, these two interim analyses 
will not be performed. 
4 
STATISTICAL/ANALYTICAL ISSUES 
4.1 
Adjustments for covariates 
Not applicable. 
4.2 
Handling of dropouts or missing data 
4.2.1 
Efficacy data 
For study participants who prematurely withdraw for any reason before Visit 26, data collected 
during the PEOT Visit will be mapped to the closest missing visit according to the visit schedule 
utilizing the relative day in study to a maximum of Visit 26 (end of treatment period). However, 
if a participant receives rescue medication prior to the PEOT visit, any efficacy assessment post 
intake will be regarded invalid and only listed. Assessments on the same day as first intake of 
rescue medication are acceptable for analysis. 
Study participants administered an Ig infusion rescue medication during the treatment phase will 
be considered relapsed even in case no relapse was documented. Missing relapse data, i.e. data 
for any relapse event definition, will be considered missing. In case of drop-out, if no relapse 
occurs or rescue medication is taken until PEOT visit (inclusively), the subject will be censored 
for time-to-event analysis at the date of PEOT visit.  
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Participants are asked to record three grip strength score assessments at each day. The efficacy 
parameter will be derived from grip strength scores taken by a participant up to five days prior to 
the first IMP (for Baseline value) or visit date (for post Baseline visit value): 
1. Maximum of grip strength assessments available at a particular day 
2. Mean of maximum grip strength over most recent three non-missing days - up to a 
maximum of five days 
3. Missing values: In case all three assessments of a day are missing, the maximum grip 
strength score will be set to missing and will not be used for the derived score. In this 
case the preceding day’s maximum grip strength score will be used instead, but no later 
than the fifth day prior to the first IMP or visit date respectively. In case data is entirely 
missing during the last five days prior to the visit, the derived score will be set to missing. 
4.2.2 
Safety Laboratory data 
Measurements below the limit of quantification (BLQ) will be imputed with half of the lower 
limit of quantification (LLOQ) for the purpose of calculating change from Baseline and the 
respective descriptive statistics. 
Measurements above the limit of quantification (ALQ), if applicable, will be imputed to the 
upper quantification limit for the calculation of changes from baseline and descriptive statistics. 
These rules will be applied to all safety laboratory data including clinical chemistry and 
urinalysis. 
Descriptive statistics will be displayed if at most 33% of the individual data points at a timepoint 
are missing or are either not quantifiable (<LLOQ) or ALQ. Values that are BLQ will be 
replaced by the numerical value of the LLOQ/2 in this instance and values that are ALQ will be 
imputed to the value of the upper quantification limit. If more than 33% of the individual data 
points at a timepoint are missing or not quantifiable (BLQ and/or ALQ), no descriptive statistics 
will be presented. 
4.2.3 
Pharmacodynamic data 
Measurements BLQ are not anticipated for the total serum IgG data. If any BLQ measurements 
are received, these will be regarded as missing for the presentation of descriptive statistics and 
changes from Baseline. 
Descriptive statistics will be calculated if at most 33% of the individual data points at a timepoint 
are missing or are either not quantifiable (<LLOQ) or ALQ as described in Section 4.2.2. 
4.2.4 
Immunological data 
The rules for handling BLQ or ALQ measurements for all immunological data will be as 
described in Section 4.2.2. 
Descriptive statistics will be calculated if at most 33% of the individual data points at a timepoint 
are missing or are either not quantifiable (<LLOQ) or ALQ as described in Section 4.2.2. 
4.2.5 
Rozanolixizumab concentration data 
Measurements that are BLQ will be imputed with half of the LLOQ for the purpose of 
calculating the geoMean and its 95% CI, the geoCV, the arithmetic mean, and SD for summaries 
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and figures. If any summary value (geoMean, arithmetic mean, lower CI level or minimum) is 
lower than LLOQ, then ‘BLQ’ will be displayed. 
For the individual figures, any concentrations that are BLQ will be considered as missing, with 
the exception of predose BLQ measurements on Week 1/ Visit 2 for study participants treated 
with placebo in the parent study, which will be imputed with zero for linear scale plots. 
Additional rules for PK data summaries are provided in Section 9.1. 
4.2.6 
Electrocardiogram data 
For the ECG data, all calculations of changes from Baseline and descriptive statistics will be 
based on the mean of the triplicate assessments at each timepoint. If there are not 3 available 
measurements at a given timepoint, the mean will be calculated based on the number of 
measurements for which data is provided. 
4.2.7 
Dates and Times 
Partial dates may be imputed for the following reasons: 
• Classification of AEs as treatment-emergent 
• Classification of medications and procedures as prior or concomitant 
• Derivation of time to use of rescue medication 
Imputed dates will not be shown in the listings; all dates will be displayed as reported in the 
database. 
The following rules will be applied for partial start dates: 
• If only the month and year are specified and the month and year of dosing is not the same as 
the month and year of the start date then use the 1st of the month, or the date of screening if 
this is later; 
• If only the month and year are specified and the month and year of dosing is the same as the 
month and year of the start date, then use the date of dosing. If this results in an imputed start 
date that is after the specified end date, then use the 1st of the month, or the date of screening 
if this is later; 
• If only the year is specified, and the year of dosing is not the same as the year of the start date 
then use January 01 of the year of the start date or the date of screening if this is later; 
• If only the year is specified, and the year of dosing is the same as the year of the start date, 
then use the date of first dosing. If this results in an imputed start date that is after the 
specified end date, then use January 01 of the year of the start date, or the date of screening if 
this is later; 
• If the start date is completely unknown, then use the date of dosing. If this results in an 
imputed start date that is after the specified end date, then use January 01 of the year of the 
end date, or the date of screening if this is later. 
Missing start times will be imputed as 00:00h or with the time of dosing for events occurring on 
the date of IMP administration in case of missing hour and minute. Otherwise start times with 
only missing minutes will be imputed with :00. 
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Start and end time is not recorded for concomitant medications, thus no imputations for missing 
times will be performed. Any medication with a start date on the first dosing date, will be 
assumed to be concomitant. 
The following rules will be applied for partial stop dates: 
• If only the month and year are specified, then use the last day of the month; 
• If only the year is specified, then use December 31 of the known year; 
• If the stop date is completely unknown, do not impute the stop date 
Missing or partially missing date and/or times will be imputed as described in Table 4‒1 for the 
calculation of duration of each AE. Adverse event duration is computed in and reported in day 
and time format: xx d hh:mm. 
Table 4‒1: 
Calculation rules for duration of AEs 
Data availability 
Onset 
date/time 
Outcome 
date/time 
Calculation rules 
Complete data 
D1/T1 
D2/T2 
Duration = [(D2 – D1)*24 + (T2 – T1)]/24 d 
End time missing 
D1/T1 
D2/-- 
End time is substituted by time 23:59h (=23.98 in 
decimal format) 
Duration = <[(D2 – D1)*24 + (23.98 – T1)]/24 d 
Start time missing 
D1/-- 
D2/T2 
Onset time is substituted by time 00:00h. 
Duration = <[(D2 – D1)*24 + T2]/24 d 
Start and end time 
missing 
D1/-- 
D2/-- 
Duration = <D2 – D1 + 1 
Start day and time 
missing 
--/-- 
D2/T2 
Duration = <[(D2 – D0)*24 + (T2 – T0)]/24 d 
For a study participant in the SS, D0 and T0 are the date 
and time of first administration of UCB7665 and for 
screen failures, D0 is the date of the screening visit and 
T0 = 00:00h 
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Table 4‒1: 
Calculation rules for duration of AEs 
Data availability 
Onset 
date/time 
Outcome 
date/time 
Calculation rules 
End day and time 
missing 
D1/T1 
--/-- 
For ongoing AE: 
Duration = >Discharge day – D1 d OR 
Duration = >Data cut-off day – D1 d 
For resolved AE: 
Duration = <Discharge day – D1 d OR 
Duration = < Data cut-off day – D1 d 
Where discharge refers to the date of the end of study 
visit for completed study participants or the date of 
discontinuation for study participants that were 
withdrawn. 
For any AEs with known start date/time after the date of 
discontinuation, the date of last contact will be used as 
the discharge day. 
For study participants still ongoing in the study at the 
time of the data cut-off, and for whom no discharge date 
is available, the date of the data cut-off will be used 
instead of the discharge date. 
Start and end date 
missing 
--/-- 
--/-- 
For ongoing AE: 
Duration = >Discharge day – D0 d OR 
Duration = >Data cut-off day – D0 d 
For resolved AE: 
Duration = <Discharge day – D0 d OR 
Duration = < Data cut-off day – D0 d 
For a study participant in the SS, D0 and T0 are the date 
and time of first administration of UCB7665 and for 
screen failures, D0 is the date of the screening visit and 
T0 = 00:00h. 
Discharge refers to the date of the end of study visit or 
the date of discontinuation for study participants that 
were withdrawn. 
For any AEs with known start date/time after the date of 
discontinuation, the date of last contact will be used as 
the discharge day. 
For study participants still ongoing in the study at the 
time of the data cut-off, and for whom no discharge date 
is available, the date of the data cut-off will be used 
instead of the discharge date. 
D0/1/2=Date0/1/2; T0/1/2=Time0/1/2; d=day. 
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4.2.8 
Handling of repeated and unscheduled measurements 
All repeated and unscheduled measurements will be presented in the listings where applicable. 
The following general rules will apply to all repeated and unscheduled measurements: 
• For repeated or unscheduled measurements obtained prior to dosing the latest value (which 
may be scheduled or unscheduled) will be used in the calculation of the descriptive statistics 
at each visit (ie, Screening and/or Baseline); 
• For repeated or unscheduled measurements obtained at the designated Baseline visit, the 
latest value (which may be scheduled or unscheduled) will be defined as the Baseline 
provided that the measurement occurred prior to dosing; 
• For repeated or unscheduled measurements taken at any timepoint after dosing, the first 
original value (if non-missing) will be used in the calculation of changes from Baseline and 
for the descriptive statistics (ie, in summaries by timepoint). Unscheduled and repeated 
measurements will generally not be included in summaries by timepoint after dosing. 
The above rules also apply to triplicate measurements obtained for the ECG data. 
4.3 
Handling of measurements obtained at the premature end of 
treatment visit 
Study participants who withdraw from the study prematurely will be encouraged to return for the 
PEOT visit. The following rules will apply with regard to the inclusion of the results obtained at 
the PEOT in the descriptive summaries: 
• Any measurements (safety, immunological and PD assessments) conducted at the PEOT 
should be included in the summaries for the respective scheduled visit, if the PEOT occurs at 
the time of the next scheduled visit (within the tolerance window specified in the protocol); 
• If the PEOT does not correspond to the day of a scheduled visit, the safety and other relevant 
assessments of the PEOT should be mapped to the nearest scheduled visit, relative to the 
Baseline visit date, following the last scheduled visit where assessments are available; 
• If the date of the PEOT is equidistant between 2 scheduled visits at which no scheduled 
assessments were performed, the assessments from the PEOT will be mapped to the earliest 
of these visits. 
Different domains from the same PEOT may be re-assigned to different study visits depending 
on the study schedule of assessments, ie, measurements obtained at the PEOT can only be 
mapped to a scheduled visit at which the respective assessment was intended to be measured. 
Assessments from the PEOT that are mapped to scheduled visits will be flagged in the data 
listings. 
Visits after the PEOT following the schedule of the 8-Weeks Observation period, will be mapped 
to the corresponding planned visit. Assessments on these visits will be flagged in the data listing. 
4.4 
Data monitoring committee (DMC) 
A DMC will systematically monitor and report on the progress, safety, and/or critical efficacy 
endpoints of this study by convening to review the ongoing safety and efficacy data. The DMC 
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will also review the impact of infusion rate on local and systemic tolerability during the first 2 
doses. 
4.4.1 
Timing of DMC 
DMC meetings for CIDP04 will take place in conjunction with CIDP01 DMC meetings. Once 
CIDP01 is completed, additional meetings for CIDP04 may be organized as agreed with the 
DMC members. 
4.4.2 
Data required for DMC 
The analyses and data required for DMC review are described in a separate DMC SAP and 
include all data specified in the DMC Charter. 
4.5 
Multicenter studies 
Individual center results will not be displayed. 
4.6 
Multiple comparisons/multiplicity 
Not applicable. 
4.7 
Use of an efficacy subset of study participants 
Not applicable. 
4.8 
Active-control studies intended to show equivalence 
Not applicable. 
4.9 
Examination of subgroups 
Not applicable. 
5 
STUDY POPULATION CHARACTERISTICS 
5.1 
Study participant disposition 
The number of participants who were enrolled, dosed, participants included in each analysis set, 
and participants who completed or prematurely discontinued the study, as well as the reason for 
discontinuation, and discontinuation due to AEs (categorised as fatal, non-fatal, other) will be 
presented by treatment group and overall. Screen failure reasons will be summarized, based on 
the ES. 
In addition, the following listings will be presented by treatment group: 
• Participant disposition (ES) 
• Study discontinuation (ES) 
• Visit dates (SS) 
• Participant analysis sets (ES) 
Study participant disposition will be listed based on the ES by treatment group, and will include 
the date of informed consent, date and time of first and last dose of IMP, date of premature 
termination and primary reason, and date of final contact (if applicable).  
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The first dose of IMP will be the first IMP infusion in CIDP01 for study participants treated with 
rozanolixizumab in CIDP01 or the first dose of rozanolixizumab in CIDP04 for the study 
participants under placebo in CIDP01.  
The listing of study discontinuation will include the reason for discontinuation, period of 
discontinuation, with dose and number of days on that dose and the total number of days on 
study medication. 
The number of days on IMP will be calculated as follows: 
Number of days on IMP=(Date of Last Dose Received)-(Date of First Dose Received)+1 
Time to discontinuation of study participation will be listed and plotted in a Kaplan-Meier-Curve 
for all discontinuations versus discontinuation due to COVID-19. The plot will be based on the 
randomized set. 
Reasons for screening failures, disposition and discontinuation reasons will also be summarized 
based on the respective event’s date relative to the COVID-19 period. 
5.2 
Protocol deviations 
Important protocol deviations (IPDs) will be identified and classified by the deviation types 
identified in the IPD document. A listing of all IPDs identified at the Data evaluation meetings 
(DEMs) will be presented for all participants in the SS, and will include the deviation type and 
description. The number and percentage of participants in the SS with IPDs will be summarized 
overall if appropriate. The denominator for percentages will be the number of participants in the 
SS. 
A separate listing will present the captured data from the "COVID-19 Impact" eCRF form. 
Frequencies of impact categories by visit will also be presented for each COVID-19 relatedness 
category (suspected, confirmed, general or other reasons).  
Important protocol deviations will be summarized by COVID-19 period and relationship to 
COVID-19 (any, related, not related). Number and percentage of participants with these protocol 
deviations will be summarized for the SS. 
Important protocol deviations will also be presented by COVID-19 period (pre- or during). A 
post- COVID-19 period may be added if applicable. 
Start of the pandemic will be the date the WHO recognized COVID-19 as pandemic, 20 March 
2020. The cut-off date for the post- subgroup level will be defined prior to database lock if 
applicable, an update to the pre-COVID-19 cut-off date may be performed. Details of the 
analyses will be specified in the following sections. 
A date is considered pre COVID-19 up to the day before the start date, so 19 March 2020. 
6 
DEMOGRAPHICS AND OTHER BASELINE 
CHARACTERISTICS 
6.1 
Demographics 
A by-study participant listing of Baseline demographics will be presented by treatment group for 
the ES. This will include the year of birth (if available), age (in years), gender, race, ethnicity, 
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height (in cm), weight (in kg) and body mass index (BMI), region and country. The age will be 
directly entered into the study database and will not be re-calculated for the statistical analysis. 
Childbearing potential will be listed for the ES. 
All demographic characteristics, except date of birth, obtained at the Screening visit or at Visit 2 
in case of missing values will be summarized for the SS. 
Body mass index in kg/m2 is calculated based on the height (in m) and the weight (in kg) 
measured either at the Screening visit (Visit 1) or baseline visit (Visit 2) of CIDP04 using the 
following formula: 
BMI (kg/m2) = weight (kg)/[height (m)] 2. 
The BMI will be rounded to 1 decimal place. 
Age will be classified into categories based on requirements for European Union Drug 
Regulating Authorities Clinical Trials (EudraCT) and clinicaltrials.gov reporting.  
For the EudraCT reporting, the age categories will include: 
• 18 to <65 years 
• 65 to <85 years 
• ≥85 years 
For the clinicaltrials.gov reporting, the age categories will include: 
• ≤18 years 
• 19 to <65 years 
• ≥65 years 
Body weight at screening or Visit 2 of CIDP04 will be summarized in the following categories: 
• <50 kg 
• 50 to <70 kg 
• 70 to <100 kg 
• ≥100 kg 
6.2 
Other Baseline characteristics 
The prior Ig treatments taken four months before the Baseline visit in CIDP04 will be listed for 
the SS by treatment group for all study participants and will include the reported term, preferred 
term (PT), dose, route of administration, frequency, formulation, start/stop dates and duration (in 
days) and if it was a rescue treatment in the parent study or not. For definition of prior treatments 
see Section 6.4.1. 
Duration will be calculated as follows: 
Duration (in days) = (End date of Ig treatment– Start date of Ig treatment)+1 
In addition, the following variables will be listed and summarized at baseline for the SS by 
treatment group: 
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• iRODS score (raw sum score, logit locations and centile metric scores) 
• INCAT 
• Grip strength (assessed by site personnel and patient reported grip strength) 
• Immunological variables (IgG [including total IgG and IgG subclasses], IgM, IgA, 
IgE) 
• Cytokines 
• Serum (C3 and C4) and plasma complement levels (C3a and C5a)  
6.3 
Medical and procedures history 
Medical history updates, not reported in CIDP01, will be listed for the SS by treatment group and 
will include the MedDRA system organ class (SOC), high level term (HLT) and preferred term 
(PT).  
Procedure history updates (not reported in CIDP01) and concomitant medical procedures will be 
listed based on the SS. 
6.4 
Prior and concomitant medications 
Prior and concomitant medications will be listed for the SS by treatment group and for all study 
participants and will include the WHODD Anatomical Main Group [Level 1], Therapeutic 
Subgroup [Level 2], PT and reported term. The listing will also include the dose per intake and 
unit, frequency, formulation, route of administration, indication, category (prior and 
concomitant, prior, or concomitant) and both start and end date (or ongoing, if applicable). 
6.4.1 
Prior medication definition 
Prior medications will include any medications that started prior to the first administration of 
IMP for study participants with a gap period between the study CIDP01 and CIDP04. This 
includes any medications that started and stopped prior to dosing in CIDP04 as well as those 
which started prior to dosing and continued after (classified as prior and concomitant 
medications) if a gap period was present. For study participants who entered the study on the last 
visit day of CIDP01 prior medications will not be defined.  
6.4.2 
Concomitant medication definition 
Concomitant medications will include any medication that has been taken at least once (on or 
after the first administration of IMP of CIDP04 study) during the Treatment Period and/or the 
Observation Period. 
Any medication that started prior to the first administration of IMP and continued after dosing 
will be classified as prior and concomitant. Any medication that started on or after the first 
administration of IMP will be classified as concomitant only. 
Any medications with missing dates and/or times will be handled as described in Section 4.2.7 in 
order to classify them as prior or concomitant. 
The following rules will be used to assign concomitant medications to the study periods: 
• Treatment Period: a medication will be assigned to Treatment Period if it has been taken at 
least once between the first administration of IMP until Visit 26/PEOT (non-inclusively). 
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This includes medications that started prior to the first IMP and continued during Treatment 
Period. 
• Observation Period: a medication will be assigned to Observation Period if it has been taken 
at least once between Visit 26/PEOT (inclusively) and the FV. This includes medications that 
started prior to the Observation Period. 
Medications taken during both study period will be listed once, but the study period will indicate 
both study periods. 
7 
MEASUREMENTS OF TREATMENT COMPLIANCE 
The study medication will be administered and monitored by the investigator or designee. Any 
dosing deviations (eg, incomplete infusion volume administered, infusion temporarily 
interrupted, infusion discontinued) will be discussed at the DEM and any actions taken regarding 
the analyses will be documented accordingly and discussed in the CSR. 
All drug administration details (including exposure category described in Section 10.1, date, start 
and stop time of infusion, interruptions, discontinuations, total planned volume and actual 
volume delivered, planned and actual infusion rates (where applicable), location of infusion site) 
will be listed. The reasons for any interruptions or discontinuations will be included in the 
listing. The duration of the infusion, in minutes, and compliance will also be calculated and 
presented. 
Compliance, percent of planned dose administered, will be calculated based on the planned and 
actual volume delivered as follows: 
Percent of Planned dose administered = (Total actual volume delivered (mL)/Total planned 
volume (mL))*100. 
Actual volume delivered and total planned volume (ie, according to Section 7.2 of protocol) will 
be captured in the eCRF. In case of interruption the sum of actual volumes delivered, and the 
total planned volume recorded into the eCRF will be used for the calculation. 
There will be no specific analysis of compliance. 
8 
EFFICACY ANALYSES 
All summaries of efficacy data will be performed on the FAS. Additional outputs are repeated 
for the MFAS where indicated. Listings will be based on the SS. Treatment group assignment for 
efficacy data presentation will be according to the actual treatment regimen. 
8.1 
iRODS 
8.1.1 
iRODS scores 
The iRODS is a linearly weighted patient-reported outcome measure (questionnaire) that 
specifically captures activity and social participation limitations in patients with CIDP. The 
iRODS scale was constructed based on the World Health Organization (WHO) International 
Classification of Functioning, Disability and Health, literature search, and patient interviews.  
All 24 items of iRODS meet Rasch model expectations. The questionnaire consists of 24 items 
(including eating, taking a shower, walking a flight of stairs, standing for hours, etc.) and 
assesses a patient’s perception of their ability to perform daily and social activities. Patients have 
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3 response options: 0=impossible to perform; 1=performed with difficulty; and 2=easily 
performed, performed without difficulty. 
The raw sum scores of iRODS (range 0 to 48) is an ordinal scale and does not allow for easy 
interpretation of differences. For this reason, the ordinal scale will be translated to log odds units 
(logits) scale using the nomogram in Section 13.1, placing patients’ estimates on the same logit 
scale. The primary analysis and iRODS summary will be performed on logit locations. For easier 
interpretation the person locations (logits) will be translated to centile metric scores changing 
from 0 (most severe activity and social participation restrictions) to 100 (no activity and social 
participation limitations).  
Both transformed and untransformed data will be listed and summarized by treatment group for 
each scheduled assessment during the Treatment and Observation Period.  
The individual items of the iRODS and overall score will be listed by treatment group and 
timepoint using the SS. Overall scores will be displayed including raw sum score, logit locations 
and centile metric scores as well as changes from Baseline in raw sum score, logit locations and 
centile metric scores. Descriptive summaries will be presented by treatment group and timepoint 
for both absolute values and changes from Baseline based on the FAS. Raw sum score summary 
will show only n, median, minimum, maximum. In addition, frequencies of raw scores by each 
iRODS questionnaire item (eating, taking a shower, walking a flight of stairs, standing for hours, 
etc.) will be calculated.  
Mean change from Baseline including 90% CI of the centile metric score will be plotted against 
timepoint for both treatments. 
The summary tables will be repeated based on the MFAS. 
8.1.2 
CIDP relapse (iRODS) 
CIDP relapse (iRODS) is defined as a clinically important deterioration from Baseline in iRODS 
score, i.e., a standardized change from baseline smaller than or equal to the minimum clinically 
important differences-standard error (MCID-SE) of ≤-1.96.  
This standardized change from baseline refers to individual person-level responsiveness 
calculated by individual change divided by their corresponding SE of difference at a given 
timepoint. [Va15] 
"𝑀𝐶𝐼𝐷−𝑆𝐸" = 𝐿𝑜𝑔𝑖𝑡 𝑙𝑜𝑐𝑎𝑡𝑖𝑜𝑛𝑉𝑖𝑠𝑖𝑡−𝐿𝑜𝑔𝑖𝑡 𝑙𝑜𝑐𝑎𝑡𝑖𝑜𝑛𝑏𝑎𝑠𝑒𝑙𝑖𝑛𝑒
√𝑆𝐸𝑉𝑖𝑠𝑖𝑡
2
+ 𝑆𝐸𝐵𝑎𝑠𝑒𝑙𝑖𝑛𝑒
2
 
 
The point estimate and the corresponding 2-sided 90% CI for the difference between placebo and 
rozimab CIDP relapse rates up to week 13 will be reported. The CI will be constructed using the 
asymptotic standard error (asymptotic Wald confidence limits).  
The time to CIDP relapse (iRODS) is defined as the time from Baseline (Visit (Day 1) in 
CIDP01 for study participants treated with rozanolixizumab in the parent study or Baseline Visit 
(Day 1) in CIDP04 if study participants received placebo in the parent study) to achievement of 
iRODS and will be displayed using a Kaplan-Meier curve. Study participants withdrawing from 
the study will be censored unless relapsed or receiving rescue medication, which also is 
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interpreted as relapse. The point estimate for the median number of days to CIDP relapse with 
90% CIs will be given if estimable. A summary including the number and percentage of 
censored participants, the number of relapsed participants and total participants by treatments 
and in total will be given. 
Participants who did not experience a relapse and are ongoing at the time of a data snapshot will 
be censored at the time of their last available assessment. 
Relapse and time to relapse will be listed. Analyses will be performed for the FAS utilizing the 
observed cases. The relapse tables and figures will be generated for relapses up to Treatment 
Period Part 1 and for the entire Treatment Period (Part 1 and Part 2). 
8.2 
Adjusted INCAT 
8.2.1 
Adjusted INCAT score 
The INCAT disability scale is a 10-point clinician-reported ordinal measure capturing problems 
in daily arm and leg activities and mobility. The measure captures daily activities such as 
dressing the upper part of the body, doing and undoing buttons and zips, washing and brushing 
hair, and handling coins. Each item is scored as being “not affected”, “affected but not 
prevented”, or “prevented”. The leg scale measures problems with walking, considering the use 
of aids.  
The investigator (or qualified personnel) will record the participants’ INCAT arm and leg 
disability score at each scheduled assessment.  
The INCAT score is the sum of the eCRF-captured arm disability classification (from 0 to 5) and 
leg disability classification (from 0 to 5). The INCAT scale ranges from 0 (no signs of disability) 
to 10 (most severe disability score). 
If there is only one point deterioration/improvement in the participant upper limb score (arm 
disability score), ie, from 0 (normal) to 1 (minor symptoms) or from 1 to 0, this difference should 
not be considered as a significant worsening/improvement and the related difference between 
baseline and the respective visit should be ignored when evaluating the adjusted INCAT score 
for the visit in the following way: 
For each visit's INCAT score, the adjusted INCAT is  
a. adjusted INCAT score = INCAT score - 1, if the arm disability score for this participant is 0 
at baseline and 1 at the respective visit 
b. adjusted INCAT score = INCAT score +1, if the arm disability score for this participant is 1 
at baseline and 0 at the respective visit 
c. adjusted INCAT score = INCAT score in any other case. 
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Baseline 
score 
Visit 
score 
Total adjusted INCAT score 
Arm 
disability 
score  
0 
1 
Total adjusted INCAT score = Total visit INCAT score – 1 
1 
0 
Total adjusted INCAT score = Total visit INCAT score + 1 
Any other case 
No adjustment 
Raw and adjusted INCAT scores will be listed. Adjusted INCAT scores will be summarized by 
treatment group for each scheduled assessment during the Treatment and Observation Period 
together with changes from baseline. 
Mean change from Baseline in adjusted INCAT score, including 90% CI will be plotted against 
timepoint for both treatments. 
8.2.2 
CIDP relapse (INCAT) 
CIDP relapse (adjusted INCAT) is defined as an increase from Baseline of at least 1 point in the 
adjusted INCAT score. 
The statistical methods and presentations applied to CIDP relapse (adjusted INCAT) and time to 
CIDP relapse (adjusted INCAT) will be the same as for CIDP relapse and time to CIDP relapse 
(iRODS), see Section 8.1.2. 
8.3 
Maximum Grip Strength (Assessed by Site Personnel) 
8.3.1 
Maximum Grip Strength (Assessed by Site Personnel) 
Grip strength is assessed by qualified site personnel at each scheduled assessment. Qualified site 
personnel will be trained regarding obtaining and recording measurements. At each scheduled 
visit, the grip strength, generated by the participant, and measured using a standardized tool, will 
be evaluated 3 times in the dominant hand. All 3 assessments will be recorded in the eCRF and 
listed. For analyses, UCB will utilize the maximum of the 3 assessments, which will be flagged 
in listings. 
Maximum grip strength as assessed by site personnel will be listed and summarized by treatment 
group for each scheduled assessment during the Treatment and Observation Period together with 
changes from baseline.  
8.3.2 
CIDP relapse (Maximum Grip Strength, 14 kPa) 
CIDP relapse (maximum grip strength as assessed by site personnel, 14 kPa) is defined as a 
clinically important deterioration from Baseline in grip strength as measured by site personnel, 
ie, a decline of >14kPa. 
The statistical methods and presentations applied to CIDP relapse (Maximum Grip Strength, 14 
kPa)) and time to CIDP relapse (Maximum Grip Strength, 14 kPa) will be the same as for CIDP 
relapse and time to CIDP relapse (iRODS), see Section 8.1.2. 
8.3.3 
CIDP relapse (Maximum Grip Strength, 8 kPa) 
CIDP relapse (maximum grip strength as assessed by site personnel, 8 kPa) is defined as a 
clinically important deterioration from Baseline in grip strength as measured by site personnel, 
ie, a decline of >8 kPa. 
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The statistical methods and presentations applied to CIDP relapse (Maximum Grip Strength, 8 
kPa) and time to CIDP relapse (Maximum Grip Strength, 8 kPa) will be the same as for CIDP 
relapse and time to CIDP relapse (iRODS), see Section 8.1.2. 
8.4 
CIDP Relapse 
The event of CIDP relapse is defined by first occurrence of either relapse across the 3 calculated 
criteria: iRODS relapse, adjusted INCAT relapse and maximum grip strength as assessed by site 
personnel (14 kPa) relapse as defined in sections 8.1.2, 8.2.2 and 8.3.2 and "other" as captured in 
the eCRF by the investigator. It will be described and analyzed in the same way as the individual 
relapse definitions.  
The specified reason for "other" relapse will be included in the listing of CIDP relapse. 
A listing will present if a participant experienced a CIDP relapse, CIDP relapse (iRODS), CIDP 
relapse (INCAT), CIDP relapse (Maximum Grip Strength, 14 kPa), CIDP relapse (Maximum 
Grip Strength, 8 kPa) or any other relapse reported by the investigator. 
8.5 
Use of rescue medication 
The percentage of participants with concomitant medication identified as rescue medication 
[Yes/No] in the eCRF and time to first rescue medication administration will be summarized by 
treatment group and for all participants and listed by treatment group at each scheduled 
assessment during the Treatment and Observation Period. 
If a participant receives rescue medication at multiple times, the relative day of the first 
occurrence is used to define the time to rescue medication. 
The statistical methods applied to use of rescue medication and time to use of rescue medication 
will be the same as for CIDP relapse (iRODS) summary tables. 
8.6 
RT-MRC sum score 
The RT-MRC sum score is a summation of the RT-MRC grades in integers of the following 
muscles on each side: upper arm abductors, elbow flexors, wrist extensors, hip flexors, knee 
extensors, and foot dorsal flexors. 
The RT-MRC sum score was developed based on a Rasch analysis of the original MRC grading 
system ([Me10]; [Me02]; [Va12]). The original MRC sum score grades from 0 to 5 (0 = No 
movement, no contraction; 1 = Visible contraction without movement; 2 = Movement, but only 
with gravity eliminated; 3 = Movement against gravity; 4 = Movement against resistance, but 
weaker than normal; 5 = Normal Strength). The MRC grades were rescored from 6 to 4 response 
options (0, paralysis; 1, severe weakness; 2, slight weakness; 3, normal strength) for the RT-
MRC in order to acceptably fulfill Rasch model expectations. Sum scores range from 36 
“normal” to 0 “quadriplegic”.  
Values and changes from Baseline in original MRC sum score and in RT-MRC sum score will 
be summarized by treatment group and for all participants and listed by treatment group at each 
scheduled assessment during the Treatment and Observation Period. 
8.6.1 
CIDP PRO and Fatigue scale 
The CIDP PRO instrument consists of 2 domain scales:  
• Pain Severity numeric rating scale 
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• Neuropathy scale.  
 
Pain Severity consisting of 4 items assessing pain severity in the past 7 days and at the time of 
completion of the instrument, using 0-10 numeric rating scales. Summed total raw score will 
range between 0 to 40 with higher scores reflecting higher levels of pain severity.  
The Neuropathy scale consists of 17 items rated within a 7-day recall period on a 6-point Likert 
severity scale ranging from “none” to “very severe”. The summed total raw score will range 
from 17 to 102 with higher scores reflecting higher levels of pain and physical sensations 
severity.  
The Fatigue instrument consists of 3 domain scales; Physical Fatigue comprising 16 items; 
Mental Fatigue comprising 18 items and Fatigability comprising 21 items. All 55 items across 
the 3 domain scales are rated within a 7-day recall period on a 5-point Likert frequency scale 
ranging from “none of the time” to “all of the time”. The summed total raw scores for each of the 
3 scales will range between 16 and 80; 18 to 90 and 21 to 105 points for the Physical, Mental, 
and Fatigability, scales respectively with a potential for an exploratory total overall score ranging 
from 55 to 275. Higher scores reflect higher levels of fatigue across all domain scale scores.  
Values and changes from Baseline in CIDP-PRO instrument and in fatigue scores will be listed 
by treatment group at each scheduled assessment during the Treatment and Observation Period. 
Summary tables will be provided at an item level. If items are missing, the sum will be set to 
missing. 
8.6.2 
PGIS and PGIC 
Participants will rate their global impression of CIDP symptom severity and fatigue severity 
respectively, on a 5-point Likert severity scale ranging from “none” to “very severe”. 
Participants will rate their global impression of change (if at all) in CIDP symptom and fatigue 
respectively, on a 7-point Likert severity scale ranging from “very much improved” to “very 
much worse” from Baseline/start of clinical study treatment to the scheduled visits.  
Frequencies of study participants scoring in the respective categories  in Patient Global 
Impressions of Severity (PGIS) and changes in frequencies from Baseline  will be presented via 
a shift table by visit per treatment group and listed by treatment group at each scheduled 
assessment during the Treatment and Observation Periods. 
Patient Global Impressions of Change (PGIC) will be summarized via frequencies of each 
response by visit and treatment group and listed by treatment group at each scheduled assessment 
during the Treatment and Observations Periods.  
9 
PHARMACOKINETICS AND PHARMACODYNAMICS 
9.1 
Pharmacokinetics 
Pharmacokinetic variables of rozanolixizumab like AUC (area under the curve), Cmax (maximum 
concentration) cannot be derived, since blood sampling will be performed at 1 time point post-
dosing per visit only. Thus, PK is restricted to concentration data. 
Concentration data will be summarized for rozanolixizumab by exposure (%) categories and time 
point using the number of available observations, mean, median, SD, minimum, maximum, 
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geoMean, (and associated 95% CI), and geoCV% (assuming log-normally distributed data) 
based on the PK-PPS. Definitions and rules for the exposure (%) categories are provided in 
Section 10.1. 
Concentration data will be summarized for treatment rozanolixizumab by time point and ADA 
status (6 classifications; see Section 9.3.2) using the number of available observations, mean, 
median, SD, minimum, maximum, geoMean, (and associated 95% confidence intervals), and 
geoCV (assuming log-normally distributed data) based on the PK-PPS.  
Individual concentrations of rozanolixizumab as well as planned and actual dose administered 
will be listed by treatment group for the SS and summarized for the PK-PPS, and will include the 
actual sampling time in days relative to the previous dose.  
The following rules will apply for PK data listings and summaries: 
• Values below the LLOQ will be reported as BLQ. 
• Descriptive statistics of concentrations will be calculated if at most 33% of the individual 
data points at a timepoint are missing or are not quantifiable (<LLOQ). Values that are BLQ 
will be replaced by the numerical value of the LLOQ/2 in this instance. However, if n≤3, 
then only n, minimum and maximum will be presented as summary statistics. The other 
descriptive statistics will be left blank. If more than 33% of data points is missing then no 
summary statistics are given, irrespective of n. 
• If no study participants have data, only n=0 will be presented. The other descriptive statistics 
will be left blank. 
• The 95% CI lower and 95% CI upper should be left blank if the SD (or equivalently, the 
geoCV) is 0. 
• The geoCV will be calculated using the following formula where SD is the standard 
deviation from the log-transformed data 
geoCV (%) = sqrt[(exp (SD^2) – 1)] x 100. 
For each study participant, individual concentration versus time (week) profiles will be presented 
graphically in linear and semi-logarithmic scale. In addition, combined individual (spaghetti 
plots) concentration versus time profiles will be presented in linear and semi-logarithmic scale 
with all study participants overlaid on the same figure.  
All figures will include the LLOQ on the semi-logarithmic plots. 
9.2 
Pharmacodynamics 
The analysis of PD data will be performed on the PD-PPS. All listings will be presented for the 
SS. 
9.2.1 
Total Serum IgG 
Total Serum IgG concentrations and IgG subclasses will be listed by treatment group and 
timepoint including absolute changes from Baseline and percentage changes from Baseline. 
Descriptive summaries (including n, arithmetic mean, median, SD, minimum and maximum) 
will be presented by treatment group and timepoint and ADA status (6 categories; see 
Section 9.3.2) for absolute values, changes from Baseline and percentage changes from Baseline.  
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Study participant total Serum IgG will be plotted against planned time. Mean Serum IgG, mean 
change from Baseline and percentage change from Baseline will be plotted against planned time 
by treatment group. 
For each study participant, the minimum value and maximum decrease (absolute and percentage) 
from Baseline in total serum IgG concentration during the study (using all available data up to 
cut-off point) will be listed. The minimum value and maximum decrease will be summarized by 
treatment group. If a decrease from Baseline in IgG is not observed in a given study participant, 
the maximum decrease will be reported as the smallest increase from Baseline. 
9.3 
Immunological analyses 
All analyses described in this section will be based on the SS. 
9.3.1 
Immunoglobulins 
Immunoglobulins (IgA, IgE and IgM) will be listed by timepoint including changes from 
Baseline. Descriptive summaries will be presented by timepoint for both absolute values and 
changes from Baseline. 
Individual figures over time (absolute value) will be presented by study participant, with all 
variables overlaid on the same plot and separate plots each study participant. 
Any values that are BLQ or ALQ will be handled as described in Section 4.2.4. 
9.3.2 
Anti-drug antibodies 
Anti-rozanolixizumab antibodies will be measured using a three-tiered assay approach: screening 
assay, confirmatory assay and titration assay. The results for the ADA measurements will be 
listed by treatment group and timepoint based on the SS, including the Screening assay, 
confirmatory assay and titer (if applicable). 
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 minimum required dilution (MRD)). 
Screening, confirmatory and titer cut points of the respective assays will be determined by the 
bioanalytical laboratory.  
The ADA status should be determined for each visit where samples were taken for ADA 
analysis: 
• Sample values that are either negative screen or positive screen and negative 
immunodepletion will be defined as ADA negative 
• Sample values that are positive screen and positive immunodepletion will be defined as 
ADA positive. 
There will be six study participant classifications:  
1. For study participants who are negative at baseline, and antibody negative at all sampling 
points post treatment (including FV)- pre ADA negative- treatment induced ADA negative  
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2. For study participants who are negative at baseline, and antibody positive at any sampling 
point post treatment (including FV)- pre ADA negative- treatment induced ADA positive. If 
a study participant has a missing pre-treatment sample (either missing or insufficient volume) 
at baseline with one or more ADA positive post-treatment samples will be also classified as 
pre ADA negative- treatment induced ADA positive   
3. For study participants who are positive at baseline, and antibody negative at all sampling 
points post treatment (including FV)- pre ADA positive- treatment reduced ADA 
4. For study participants who are positive at baseline, and are positive at any sampling point 
post treatment (including FV) with titer values of the same magnitude as baseline (i.e. ≤ than 
a predefined fold difference from the baseline value)- pre ADA positive - treatment 
unaffected ADA 
5. For study participants who are positive at baseline, and are positive at any sampling point 
post treatment (including FV) with increased titer values compared to baseline (above a 
predefined fold difference increase from baseline value which will be defined within the 
validation of the assay and will be included in the TFLs and/or SAP when available)- pre 
ADA positive- treatment boosted ADA positive. 
6. For Study participants who have a positive pre-treatment sample and some post-treatment 
samples are missing, while other post-treatment samples are ADA negative, the study 
participant will be classed as inconclusive. 
A study participant will be classified as having treatment-induced ADA positivity when meeting 
one of the following criteria: 
1. The Baseline result is ADA negative and at least one post-Baseline timepoint is ADA 
positive 
2. The Baseline result is ADA positive and at least one post-Baseline measurement shows a 
pre-defined fold increase in titer from the Baseline value (the fold increase from Baseline 
required to meet this criterion will be defined with the development of the assay and will 
be included in the TFLs) 
A study participant will be considered: 
• Overall ADA Positive if having at least one scheduled assessment that is confirmed positive 
during the treatment period (ie. classifications 2 to 6). 
• Overall ADA Negative if having no scheduled assessments that are confirmed positive at any 
time in the treatment period (ie. classification 1). 
The available ADA results will be listed. The listing will include rozanolixizumab concentration, 
ADA result (anti-rozanolixizumab antibody negative or positive), together with the confirmatory 
result (% inhibition) and ADA titer for results that are anti-rozanolixizumab antibody positive. In 
addition, the time since the previous administration of IMP will be reported (in days). 
Number and percentage of ADA positive and negative study participants at the time of each visit 
and overall will be summarized by treatment group based on the SS. 
The first occurrence of treatment-induced ADA positivity (based on the definitions above) will 
be summarized (number and percentage of study participants) at each post-Baseline visit, based 
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on the SS. This tabulation will count the number of study participants at each post-Baseline visit 
who fulfill at least one of the above defined criteria for treatment-induced positivity; study 
participants will be counted in the numerator based on the earliest visit at which one of these 
criteria is fulfilled. At other visits, study participants will be counted in the denominator 
(assuming a measurement is available). For all tabulations, percentages will be based on the 
number of observations at each visit. 
The ADA status by the six categories, defined above, will be tabulated by treatment group, with 
an additional category "total treatment induced" combining study participants who are pre ADA 
negative- treatment induced ADA positive, study participants with a missing pre-treatment 
sample (either missing or insufficient volume) at baseline with one or more ADA positive post-
treatment samples and study participants pre ADA positive- treatment boosted ADA positive. 
The prevalence of immunogenicity, defined as (cumulative) proportion of study participants 
having positive immunodepletion ADA samples at any point up to and including that time point 
will be summarized by treatment group based on the SS. Missing samples will not be included in 
the denominator. 
Individual plots of rozanolixizumab concentrations and ADA titer results as well as ADA titer 
results and percentage change from baseline in Total IgG by the six ADA classes described 
above will be presented. Spaghetti plots of ADA titer versus time will be presented for all ADA 
positive study participants based on the SS. 
9.3.3 
Serum complement levels and plasma complement levels 
Serum (C3 and C4) and plasma (C3a and C5a) complement variables will be listed by treatment 
group and time point including changes from Baseline. Summaries using descriptive statistics 
will be presented by treatment group and by time point for both absolute values and changes 
from Baseline. 
Any values are that BLQ or ALQ will be handled as described in Section 4.2.4. 
9.3.4 
Cytokines 
Serum cytokines will be listed by treatment group and by time point, changes from Baseline will 
be added to the listing. Summaries using descriptive statistics will be presented by treatment 
group and by time point for both absolute values and changes from Baseline, both for all study 
participants and for study participants experiencing infusion reactions. 
Any values are that BLQ or ALQ will be handled as described in Section 4.2.4. 
9.3.5 
CIDP-specific auto-antibodies 
CIDP-specific auto-antibody sampling information captured in the eCRF will be listed. No 
results will be analyzed. 
9.3.6 
Tetanus-and influenza A virus-specific IgG antibodies 
Tetanus-and influenza A virus-specific IgG antibodies will be listed by treatment group and time 
point. Summaries using descriptive statistics will be presented by treatment group and time point 
for both absolute values and changes from Baseline. 
Any values are that BLQ or ALQ will be handled as described in Section 4.2.4. 
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10 
SAFETY ANALYSES 
All safety analyses will be presented using the SS. Listings will be presented by treatment group 
and study participant; tabulations will be presented by treatment group and visit/timepoint (if 
applicable). 
10.1 
Extent of exposure 
The initial dose administered will be based on the dose at the time of completion of the 
Treatment Period from the parent study (eg, CIDP01). For study participants who had a gap 
period between the parent study (eg, CIDP01) and CIDP04, a starting dose of 
 will be 
used (see protocol Table 7-1).  
The intended dose will be calculated based on the study participant’s weight at screening (where 
applicable) or Baseline in CIDP04 for Treatment Period Part 1. For Treatment Period Part 2, 
body weight is collected every 6 months starting at first visit in this period and the most recent 
weight will be used to select the correct volume for dose infusion from these timepoints. The 
calculation is done as follows for the applicable weight: 
Intended dose (mg) = weight (kg)*
 
The IMP solution contains 
of rozanolixizumab. Actual dose in mg at Visit (i) will be 
calculated as follows: 
Actual dose at Visit (i) (mg) = Total actual volume delivered at Visit (i) (mL)*
 
where actual volume delivered is collected in eCRF, and in case of interruption it should be 
calculated as the sum of actual volumes delivered. 
Overall exposure (%) will be calculated as mean exposure (%) at each visit as follows: 
     Overall exposure (%) = mean([Actual dose at Visit (i) (mg)/ Randomized dose (mg) -1]*100). 
Overall exposure will be categorized as follows: 
• < -10% 
• ≥ -10% and ≤ +10% 
• > +10% 
The exposure categories will be used for the definition of the MFAS as well as the representation 
of Pharmacokinetic data (Section 9.1). 
10.2 
Adverse events 
Adverse events will be recorded from the time of informed consent until study completion. All 
AEs will be coded (Section 3.8) and categorized by relationship to rozanolixizumab. 
In addition, AEs will be classified according to the Common Terminology Criteria for Adverse 
Events (CTCAE) Version 5.0 for severity. For any AEs where it is not possible to provide a 
CTCAE grading, the events will be assessed using a standard intensity classification (mild, 
moderate and severe). 
A TEAE is defined as any event that was not present prior the first administration of IMP in 
CIDP04 study or any unresolved event already present before the first administration of IMP in 
CIDP04 study that worsens in intensity following exposure to treatment until 8 weeks following 
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the last administration of IMP in CIDP04 study. Adverse events starting before the first 
administration of IMP in CIDP04 study or later than 8 weeks after the administration of the last 
IMP will not be considered TEAEs. Such events will be listed only. 
Where dates are missing or partially missing, AEs will be assumed to be treatment-emergent 
unless evidence exists that does not allow the AE to be treatment-emergent. Handling of missing 
dates/times for classification of AEs as TEAEs is described in Section 4.2.7. 
The following rules will be used to assign a TEAE to the study periods: 
• Treatment Period: a TEAE will be assigned to the Treatment Period for the tabulations if 
the start date/time of the event is at the time of or after the first administration of IMP in 
CIDP04 study on Week 1, up to Visit 26/PEOT (non-inclusively). 
• Observation Period: a TEAE will be assigned to the Observation Period for the tabulations 
if the start date/time of the event is at or after Visit 26/PEOT till 8 weeks after administration 
of the last IMP in CIDP04 study.  
− The Observation Period’s TEAEs will be stratified by TEAEs of study participants who 
received/did not receive standard of care treatment (i.e. Ig treatment) between start of the 
Observation Period and occurrence of the TEAE. 
The number and percentage of study participants who experience TEAEs will be summarized by 
Treatment Period, Observation Period (standard of care treatment status identified by WHODD 
J06BA01 and J06BA02) and Overall, with respective treatment group. The following summaries 
will be presented: 
• Incidence of TEAEs (overview including number and percentage of study participants with 
any TEAEs, serious TEAEs, discontinuations due to TEAEs, related TEAEs, TEAEs with 
CTCAE Grade 3 and above [or rated as ‘severe’ for events with no CTCAE classification], 
and deaths; event counts will also be included) 
• Incidence of TEAEs by SOC, HLT and PT 
• Incidence of TEAEs during the Dosing Period by SOC, HLT and PT 
• Incidence of serious TEAEs by SOC, HLT and PT 
• Incidence of non-serious TEAEs by SOC, HLT and PT 
• Incidence of AEs of special interest by SOC, HLT and PT 
• Incidence of AEs of interest by SOC, HLT and PT 
• Incidence of TEAEs by relationship, SOC, HLT and PT 
• Incidence of TEAEs by maximum relationship, SOC, HLT and PT 
• Incidence of serious TEAEs by relationship, SOC, HLT and PT 
• Incidence of non-serious TEAEs by relationship, SOC, HLT and PT 
• Incidence of fatal TEAEs by relationship, SOC, HLT and PT 
• Incidence of TEAEs by maximum intensity (mild, moderate and severe), SOC, HLT and PT 
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• Incidence of non-serious TEAEs above threshold of 5% of participants by SOC and PT 
• Incidence of non-serious TEAEs above threshold of 5% of participants by relationship, SOC 
and PT 
• Discontinuation due to AEs 
Summary tables will contain counts of study participants, percentages of study participants in 
parentheses and the number of events. A study participant who has multiple events in the same 
SOC, HLT and PT will be counted only once in the study participant counts but all events will be 
included. 
Adverse event summaries will be ordered by alphabetical SOC, alphabetical HLT within SOC 
and decreasing frequency in the treatment group. 
In summaries including intensity, the CTCAE categories will be summarized according to the 
 following categories: 
• Grade 1: Mild 
• Grade 2: Moderate 
• Grade 3: Severe 
• Grade 4: Severe 
• Grade 5: Severe 
• not gradable 
In the case both category types are captured and the mapping to a standard intensity 
classification per above rule results in a class different from the standard intensity classification 
given in the eCRF, the worst case will be used.  
These will be tabulated together with the AEs that were not classified according to CTCAE 
criteria i.e., all Grade 1 AEs as per CTCAE criteria will be included in the ‘mild’ category 
together with those AEs classified as mild as per the ‘standard’ intensity classification. 
A listing will be presented by treatment group and study participant for all AEs. This will include 
the onset date/time and outcome date/time of the event (including relative days), the AE 
duration, days since first dose of IMP, days since most recent dose of IMP, actual dose received, 
study period, pattern of event, severity/intensity, relationship, action taken and outcome. In 
addition, the listing will flag AEs that led to discontinuation, TEAEs, serious adverse events 
(SAEs), AEs of interest, AEs of special interest and infusion reactions. 
AEs of focus (ie, TEAEs that are a review focus for rozanolixizumab or the participant 
population) will be summarized, in separate tables, detailing all TEAEs meeting the following 
criteria described below: 
• Headache using the UCB-defined search criteria. 
• Gastrointestinal disturbances using the UCB-defined search criteria. 
• Hypersensitivity reactions using the criteria SMQ=’Hypersensitivity’ 
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• Anaphylactic reactions using the criteria SMQ=‘Anaphylactic reaction’ and the UCB-defined 
search criteria. 
• Injection site reactions using the UCB-defined search criteria. 
• Infusion reaction collected on the eCRF AE page. 
• Opportunistic infections using the UCB-defined search criteria. 
• Reductions in albumin and plasma proteins using the UCB-defined search criteria. 
• Effects on the kidney using the criteria SMQ=‘Acute renal failure’ 
• Drug related hepatic disorders using the criteria SMQ=’Drug related hepatic disorders - 
comprehensive search’. 
For each of the above mentioned AEs of focus the following summaries will be presented: 
• Incidence of all AEoFs by SOC, HLT and PT 
• Incidence of all AEoFs by Relationship, SOC, HLT and PT 
• Incidence of all serious AEoFs by SOC, HLT and PT 
• Incidence of all AEoFs by Maximum Intensity, SOC, HLT and PT 
UCB definitions for AEoF are found in a separate document “Adverse Events (AEs) of Focus for 
the rozanolixizumab Program” version 1.3, dated 04 Aug 2020. 
10.3 
Clinical laboratory evaluations 
Laboratory data (chemistry, hematology and urinalysis) and changes from Baseline (if 
applicable) for numeric variables will be listed by treatment group and timepoint. Any laboratory 
measurements that are BLQ or ALQ will be handled as described in Section 4.2.2. Values 
outside the reference range for the numeric variables will be flagged in the listings. The reference 
ranges will also be reported in the listings. In addition, the listings will include a flag for values 
identified as markedly abnormal MA as defined by the criteria outlined in Section 13.2Error! 
Reference source not found.. 
Chemistry and hematology parameters will be summarized by treatment group for both absolute 
values and changes from Baseline.  
The laboratory variables to be included in the analysis are presented in Table 10‒1. 
Table 10‒1: Laboratory measurements 
Category 
Panel 
Variable 
Hematology 
Red blood cell 
RBC count, hemoglobin, hematocrit 
 
White blood cell 
WBC count 
 
White blood cell 
differential 
Absolute counts: ANC, basophils, eosinophils, ALC, 
monocytes 
Percentages: neutrophils/leukocytes, 
basophils/leukocytes, eosinophils/leukocytes, 
lymphocytes/leukocytes, monocytes/leukocytes 
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Table 10‒1: Laboratory measurements 
Category 
Panel 
Variable 
 
Platelet 
Platelet count 
Chemistry 
Liver function 
ALP, ALT, AST, GGT, total bilirubina, LDH 
 
Enzymes 
Creatine kinase, amylase 
 
Proteins 
Total protein, albumin, alpha- and beta- globulins, 
hsCRP 
 
Kidney Function 
Urea- nitrogen, creatinine 
 
Lipids 
Triglycerides, total cholesterol, LDL cholesterol, HDL 
cholesterol,  
 
Electrolytes 
Calcium, phosphate, sodium, potassium, chloride, and 
magnesium 
 
Hormones 
Procalcitonin 
Urinalysisb 
Dipstick 
pH, nitrite, protein, glucose, ketones, urobilinogen, 
bilirubin, blood,  
 
Urine Sediment 
Leukocytes 
 
Quantitative 
Albumin, creatinine, protein 
ALC=absolute lymphocyte count; ALP=alkaline phosphatase; ALT=alanine aminotransferase; ANC=absolute 
neutrophil count; AST=aspartate aminotransferase; GGT=gamma-glutamyltransferase; HDL=high density 
lipoprotein; hsCRP=high sensitivity C-Reactive Protein; LDH=lactate dehydrogenase; LDL=low density 
lipoprotein; RBC=red blood cell; WBC=white blood cell 
a Direct bilirubin will also be measured when total bilirubin is elevated. 
b Urine microscopy will be performed if urine is positive for protein, blood, nitrite, or leukocytes. 
10.3.1 
Potential drug-induced liver injury 
A separate table will present study participants who meet one or more of the following potential 
drug-induced liver injury (PDILI) criteria at any visit: 
• Aspartate aminotransferase (AST): >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x 
ULN 
• Alanine aminotransferase (ALT): >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x ULN 
• AST or ALT: >3 x ULN, >5 x ULN, >8 x ULN, >10 x ULN, >20 x ULN 
• Total bilirubin (TBL): >1.5 x ULN, >2 x ULN 
• Alkaline phosphatase (ALP) >1.5 x ULN 
• AST or ALT >3 x ULN, with TBL >1.5 x ULN 
• AST or ALT >3 x ULN, with TBL >2 x ULN 
• (ALT or AST increase >3 x ULN) and Total bilirubin >2 x ULN and ALP <2 x ULN (Hy’s 
Law) 
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• Incidence of potential hepatotoxicity with symptoms potentially associated with hepatitis or 
hypersensitivity (participants meeting laboratory criteria for pDILI for at least 1 visit and 
reporting at least 1 symptom potentially associated with hepatitis or hypersensitivity 
according to the Investigator on the pDILI CRF.) 
• Incidence of potential hepatotoxicity with no symptoms potentially associated with hepatitis 
or hypersensitivity (participants meeting laboratory criteria for pDILI for at least 1 visit and 
not reporting any symptom potentially related to hepatitis or hypersensitivity according to the 
Investigator pDILI CRF.) 
The laboratory results listing will flag study participants who are experiencing PDILI including 
Hy’s Law. 
10.4 
Vital signs, physical findings, and other observations related to 
safety 
10.4.1 
Vital signs 
The following vital signs measurements will be obtained: 
• Pulse rate 
• Systolic and diastolic blood pressure 
• Body weight 
• Temperature (oral preferred, ear or axillary permitted) 
A by-study participant listing of all vital sign measurements and change from Baseline will be 
presented by treatment group and timepoint. The listing will include a flag for measurements 
identified as markedly abnormal MA as calculated by the criteria outlined in Section 13.2Error! 
Reference source not found.. 
Descriptive statistics will be reported for all vital sign measurements. Measured values and 
changes from Baseline will be summarized by vital signs variables, timepoint and treatment 
group. 
Summarization will be as treated, study participants who missed treatments will not be 
summarized. Repeated and unscheduled measurements will be handled as described in 
Section 4.2.8. 
10.4.2 
Electrocardiograms  
Standard 12-lead ECG recordings will be taken in triplicate with the study participant resting in 
the supine position for at least 15 minutes and before obtaining any blood samples for 
assessments of laboratory variables. The following variables will be reported: 
• Heart rate 
• RR interval 
• PQ/PR interval 
• QRS duration 
• QT interval 
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• QT corrected for heart rate using Fridericia’s formula (𝑄𝑇𝑐𝐹 =  𝑄𝑇/√𝑅𝑅
3
) 
The individual measurements and the mean of the triplicate measurements will be reported in the 
by-study participant listings. The listing will also include the change from Baseline (based on the 
mean of the triplicate measurements at each timepoint) and will be presented by treatment group 
and by timepoint. The listing will also include a flag for values identified as MA as defined by 
the criteria outlined in Section 13.2Error! Reference source not found.. 
Summarization will be as treated, study participants who missed treatments will not be 
summarized. 
Measured values and changes from Baseline will be summarized by treatment group at each 
timepoint and by ECG variable (based on the mean of the triplicate values at each timepoint). 
The following cut-points in QTcF (raw data and change from Baseline) will be summarized 
categorically by treatment group (number and percentage of participants) and visit/timepoint. 
The denominator for the percentages will be the number of participants with a non-missing 
measurement for the variable at the specific visit/timepoint. 
Raw QTcF data: 
• <450msec 
• ≥450msec to <480msec 
• ≥480msec to <500msec 
• ≥500msec 
Change from Baseline QTcF: 
• <30msec 
• ≥30ms to <60msec 
• ≥60msec 
Electrocardiogram findings for the individual triplicate measurements will be listed separately. 
Repeated and unscheduled measurements will be handled as described in Section 4.2.8. 
In the event that the complete set of triplicate measurements is not available at a specified 
timepoint the data will be handled as described in Section 4.2.6. 
10.4.3 
Suicidality 
A full Columbia Suicide Severity Rating Scale (C-SSRS) assessment will be performed only 
when study participant has a positive response to the suicidal ideation query. Results of suicidal 
ideation and C-SSRS will be listed by treatment group, study participant and time point. 
10.4.4 
Other safety variables 
Results of Serum and Urine pregnancy test will be listed. 
Results of tuberculosis signs and symptoms questionnaire will be listed. 
Results of interferon-gamma release assay (IGRA) tuberculosis test will be listed. 
Physical and neurological examination findings will be listed. 
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Results of serology testing for human immunodeficiency virus (HIV), Hepatitis B and Hepatitis 
C will be listed. 
Study participants experiencing severe headache will complete the Headache Questionnaire daily 
until resolution (ie, if headache becomes moderate or mild, normal collection of AEs should 
apply). The results of the headache questionnaire will be listed for each study participant. 
At the clinic visit when the severe headache is reported, the Headache Questionnaire will be 
followed by a neurological assessment (including fundoscopy). If the severe headache is initially 
reported at a home visit or during a telephone call, the participant should be reviewed at the 
study site as soon as is practically possible for further investigations. Further workup will be 
performed at the discretion of the investigator and may include, eg, a computed tomography 
scan, magnetic resonance imaging and/or a lumbar puncture for cerebral spinal fluid collection. 
In addition, samples for exploratory biomarkers should be taken for participants experiencing 
severe headache (Table 5-2 of the protocol). These investigations will be performed to further 
understand the mechanism of headache in these participants. 
The results of the headache questionnaire, neurological examination and any additional tests 
performed (CT scan, fundoscopy and LP for CSF collection) will be listed for each participant 
having experienced severe headache. No summary tabulations will be provided for these 
assessments 
Stool collection and analysis will be performed for participants reporting moderate or severe 
diarrhea. Results of the analysis will be listed for each participant. No summary tabulation will 
be provided for this assessment. 
11 
OTHER ANALYSES  
Exploratory pharmacogenetics variables samples date/timepoint collection will be listed. 
Exploratory proteins, and metabolites variables samples date collection will be listed. 
 
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12 
REFERENCES 
[Va15] Peripheral neuropathy outcome measures standardisation (PeriNomS) study part 2 : 
getting consensus. / Vanhoutte, E.K. Maastricht : Datawyse / Universitaire Pers Maastricht, 
2015. 249 p. 
[Me02] Merkies IS, Schmitz PI, van der Meché FG, Samijn JP, van Doorn PA. Inflammatory 
Neuropathy Cause and Treatment (INCAT) group. Clinimetric evaluation of a new overall 
disability scale in immune mediated polyneuropathies. J Neurol Neurosurg Psychiatry. 2002; 
72(5):596-601. 
[Me10] Merkies IS, Hughes RA, Donofrio P, Bril V, Dalakas MC, Hanna K, et al. 
Understanding the consequences of chronic inflammatory demyelinating polyradiculoneuropathy 
from impairments to activity and participation restrictions and reduced quality of life: the ICE 
study. J Peripher Nerv Syst. 2010;15(3):208-15. 
[Va12] Vanhoutte EK, Faber CG, van Nes SI, Jacobs BC, van Doorn PA, van Koningsveld R, et 
al. Modifying the Medical Research Council grading system through Rasch analyses. Brain. 
2012;135:1639-49. 
Adverse Events of Focus (AEoF) for the rozanolixizumab Program, version 1.3, 04 Aug 2020 
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13 
APPENDICES 
13.1 
Conversion of iRODS scores 
Table 13‒1: Nomogram 
R-ODS summed 
raw score 
Rasch person location 
centile metric 
Corresponding SE 
(logits) 
SE= 0.4248 – 0.0016*location + 
0.0140*location2 
0 
-6.95 
0 
1.11 
1 
-6.03 
6 
0.94 
2 
-5.36 
11 
0.84 
3 
-4.87 
14 
0.76 
4 
-4.48 
16 
0.71 
5 
-4.14 
19 
0.67 
6 
-3.84 
21 
0.64 
7 
-3.57 
22 
0.61 
8 
-3.32 
24 
0.58 
9 
-3.09 
26 
0.56 
10 
-2.87 
27 
0.54 
11 
-2.66 
28 
0.53 
12 
-2.46 
30 
0.51 
13 
-2.26 
31 
0.5 
14 
-2.07 
32 
0.49 
15 
-1.88 
34 
0.48 
e 
-1.70 
35 
0.47 
17 
-1.52 
36 
0.46 
18 
-1.33 
37 
0.45 
19 
-1.15 
39 
0.45 
20 
-0.97 
40 
0.44 
21 
-0.79 
41 
0.43 
22 
-0.61 
42 
0.43 
23 
-0.42 
43 
0.43 
24 
-0.24 
45 
0.43 
25 
-0.05 
46 
0.42 
26 
0.14 
47 
0.42 
27 
0.34 
48 
0.43 
28 
0.53 
50 
0.43 
29 
0.73 
51 
0.43 
30 
0.94 
52 
0.44 
31 
1.15 
54 
0.44 
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Table 13‒1: Nomogram 
R-ODS summed 
raw score 
Rasch person location 
centile metric 
Corresponding SE 
(logits) 
SE= 0.4248 – 0.0016*location + 
0.0140*location2 
32 
1.36 
55 
0.45 
33 
1.58 
57 
0.46 
34 
1.81 
58 
0.47 
35 
2.04 
60 
0.48 
36 
2.28 
61 
0.49 
37 
2.54 
63 
0.51 
38 
2.80 
65 
0.53 
39 
3.09 
67 
0.55 
40 
3.40 
69 
0.58 
41 
3.74 
71 
0.61 
42 
4.11 
73 
0.65 
43 
4.54 
76 
0.71 
44 
5.03 
80 
0.77 
45 
5.59 
83 
0.85 
46 
6.25 
88 
0.96 
47 
7.07 
93 
1.11 
48 
8.11 
100 
1.33 
 
13.2 
MA criteria for Rozanolixizumab program 
The following criteria will be applied in the determination of marked abnormalities for 
laboratory assessment values. They are based on Version 5 of the Common Terminology Criteria 
for Adverse Events (CTCAE) grade 3 or higher criteria unless otherwise noted.  
Table 13-2: 
MA  Values for Rozanolixizumab Clinical Development Program Based on 
CTCAE Grades 
Domain 
 
Parameter 
Unit 
(conventional) 
Unit 
(standard) 
Marked Abnormality criteria 
Chemistry 
AST (SGOT) 
U/L 
U/L 
>5.0 x ULN  
 
Chemistry 
ALT (SGPT) 
U/L 
U/L 
>5.0 x ULN  
Chemistry 
ALP (Alkaline 
Phosphatase) 
U/L 
U/L 
>5.0 x ULN  
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Table 13-2: 
MA  Values for Rozanolixizumab Clinical Development Program Based on 
CTCAE Grades 
Domain 
 
Parameter 
Unit 
(conventional) 
Unit 
(standard) 
Marked Abnormality criteria 
Chemistry 
GGT (Gamma 
Glutamyl 
Transferase) 
U/L 
U/L 
>5.0 x ULN  
Chemistry 
Bilirubin (Total) 
mg/dL 
umol/L 
>3.0 x ULN if Baseline value is 
normal; 
>3.0 x Baseline value if Baseline is 
abnormal  
Chemistry 
Albumin 
g/dL 
g/L 
<2 g/dL; <20 g/L  
Chemistry 
Creatinine 
mg/dL 
umol/L 
>3.0 x ULN 
Chemistry 
Estimate 
glomerular 
filtrate rate 
(eGFR)1 
mL/min/1.73 m2 
mL/min/1.73 
m2 
eGFR <29 mL/min/1.73 m2 
Chemistry 
C reactive 
protein (CRP)2 
mg/L 
mg/L 
>10 mg/L 
Chemistry 
Calcium 
mg/dL 
mmol/L 
Low: Corrected serum calcium of 
<6.0 mg/dL; <1.5 mmol/L 
Chemistry 
High: Corrected serum calcium of 
>12.5 mg/dL; >3.1 mmol/L 
Chemistry 
IgG 
g/L 
g/L 
≤ 1 g/L 
Chemistry 
Potassium 
mmol/L 
mmol/L 
Low: <2.5 mmol/L 
High: >6.0 mmol/L 
Chemistry 
Sodium 
mmol/L 
mmol/L 
Low: <125 mmol/L  
High: >155 mmol/L 
Chemistry 
Glucose 
mg/dL 
mmol/L 
Low: <40 mg/dL; <2.2 mmol/L  
High: > 250 mg/dL; >13.9 mmol/L 
Chemistry 
Total 
Cholesterol 
mg/dL 
mmol/L 
>400 /dl; >10.34 mmol/L  
Chemistry 
Triglycerides 
mg/dL 
mmol/L 
>500 mg/dl; >5.7 mmol/L 
Hematology 
Hemoglobin  
g/dL 
 
g/L 
<8.0 g/dL; <80 g/L  
Hematology 
WBC 
(Leukocytes) 4 
109/L 
109/L  
Low: <2.0 x 109/L  
High:  >30 x 109/L  
Hematology 
Lymphocytes 
Absolute 
109/L 
109/L  
Low:  <0.5 x 109/L  
High: >20 x 109/L 
Hematology 
Neutrophils 
Absolute  
109/L 
109/L  
 <1.0 x 109/L   
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Table 13-2: 
MA  Values for Rozanolixizumab Clinical Development Program Based on 
CTCAE Grades 
Domain 
 
Parameter 
Unit 
(conventional) 
Unit 
(standard) 
Marked Abnormality criteria 
Hematology 
Platelets 
109/L 
109/L  
<50.0 x 109/L  
Vital Signs 
Pulse Rate 
bpm 
bpm 
≤50 and a decrease from Baseline 
of ≥15 
≥120 and an increase from Baseline 
of ≥15 
Vital Signs 
Systolic Blood 
Pressure 
mmHg 
mmHg 
≤ 90 and a decrease from Baseline 
of ≥20 
≥ 180 and an increase from 
Baseline of ≥20 
Vital Signs 
Diastolic Blood 
Pressure 
mmHg 
mmHg 
≤50 and a decrease from Baseline 
of ≥15 
≥105 and an increase from Baseline 
of ≥ 15 
Vital Signs 
Temperature 
°F 
°C 
>101 °F (38.3 °C) 
Vital Signs 
Body Weight 
 
 
≥ 10% decrease from Baseline 
≥ 10% increase from Baseline 
ECG 
QT interval 
ms 
ms 
>500ms 
≥60ms increase from Baseline 
ECG 
QTc(F) 
ms 
ms 
≥500ms 
 
≥60ms increase from Baseline 
ECG 
PR interval 
ms 
ms  
Treatment-emergent value >200ms 
ECG 
QRS interval 
ms 
ms 
Treatment-emergent value >100ms 
ECG 
Heart rate 
bpm 
bpm 
<50bpm 
>120bpm 
Abbreviations: ALT= alanine aminotransferase; AST = aspartate aminotransferase; dL = deciliter; GGT: gamma-
glutamyltransferase; L = liter; mg = milligram; mmol = millimoles; μg = microgram; U = unit; ULN = upper limit 
of normal 
Note: Marked abnormality criteria are defined by Grade 3 or higher events according to the Common Terminology 
for Adverse Events (CTCAE), Version 5.0, November 17, 2017 unless otherwise noted.  
1eGFR is calculated using the Chronic Kidney Disease Epidemiology Collaboration or CKD-EPI formula 
(https://qxmd.com/calculate/calculator_251/egfr-using-ckd-epi) which is eGFR = 141 * min(Scr/κ,1)α * 
max(Scr/κ, 1)-1.209 * 0.993Age * 1.018 [if female] * 1.159 [if black];  where Scr is serum creatinine (mg/dL), κ is 0.7 
for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/κ or 
1, and max indicates the maximum of Scr/κ or 1. For derivation from values in standard units (umol/L) the κ 
values are 61.9 for females and 79.6 for males. 
2Includes CRP and High Sensitivity (HS) CRP. Reference for marked abnormality criteria: Nehring, S.M.; Goyal, 
A.; Patel, B.C. (2020). StatPearls Publishing, web link: https://www.ncbi.nlm.nih.gov/books/NBK441843/. 
3Immunoglobulin G criterion based on immunodeficiency literature and noted in RLZ study protocols. 
4WBC (Leukocytes) markedly abnormal high criterion is not based on Version 5 CTCAE Grade 3 or higher criteria. 
Due to the mechanism of action of RLZ, the safety alert is related to infection risk which would be identified by a 
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lower cut-point than the standard which is related to acute leukemias. A markedly abnormal high cut-point >30 x 
109/L is applied to flag leukocytosis (George 2012). 
 
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STATISTICAL ANALYSIS PLAN SIGNATURE PAGE 
This document has been reviewed and approved per the Review and Approval of Clinical 
Documents Standard Operating Procedures. Signatures indicate that the final version of the 
Statistical Analysis Plan (SAP) or amended SAP is released for execution. 
 
 
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Approval Signatures
Name:
CIDP04-SAP-Final1.0_2021-06-08
Version:
1. 0
Document Number:
CLIN-000172996
Title:
Statistical Analysis Plan
Approved Date:
10 Jun 2021
Document Approvals
Approval
Verdict: Approved
Name: 
Capacity: Document Author
Date of Signature: 09-Jun-2021 12:22:11 GMT+0000
Approval
Verdict: Approved
Name:
Capacity: Clinical
Date of Signature: 10-Jun-2021 13:40:30 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Subject Matter Expert
Date of Signature: 10-Jun-2021 14:08:00 GMT+0000
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