Conditions: Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Keywords: Chronic inflammatory demyelinating polyradiculoneuropathy CIDP UCB7665 rozanolixizumab
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 34
Healthy volunteers: no

Brief summary:
The purpose of the study is to evaluate clinical efficacy of rozanolixizumab as a treatment for subjects with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

Detailed description:

Arms:
arm 1: Subjects will be randomized to receive predefined subcutaneous doses of rozanolixizumab at a specified frequency arm 2: Subjects will be randomized to receive predefined subcutaneous doses of placebo at a specified frequency

Interventions: intervention 1: Rozanolixizumab intervention 2: Placebo
Intervention descriptions: intervention 1: Subjects will receive rozanolixizumab in a specified sequence during the treatment period. intervention 2: Subjects will receive placebo in a specified sequence during the treatment period.

Protocol document:
Prot_002.pdf: 
UCB 
 
09 Jul 2019 
Clinical Study Protocol 
rozanolixizumab 
CIDP01 
 
 
 
Confidential 
Page 1 of 128 
 
PROTOCOL CIDP01 AMENDMENT 2 
A MULTICENTER, RANDOMIZED, SUBJECT-BLIND, 
INVESTIGATOR-BLIND, PLACEBO-CONTROLLED, 
PARALLEL-GROUP STUDY EVALUATING THE EFFICACY, 
SAFETY, AND TOLERABILITY OF ROZANOLIXIZUMAB IN 
SUBJECTS WITH CHRONIC INFLAMMATORY 
DEMYELINATING POLYRADICULONEUROPATHY (CIDP) 
 
PHASE 2A 
 
EudraCT Number: 2016-002411-17 
 
IND Number: 134848 
Sponsor: 
UCB Biopharma SPRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Protocol/Amendment number 
Date 
Type of amendment 
Final Protocol 
23 Mar 2017 
Not applicable 
Protocol Amendment 1 
15 Nov 2018 
Substantial 
Protocol Amendment 1.1 (France) 
23 Apr 2019 
Nonsubstantial 
Protocol Amendment 2 
09 Jul 2019 
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 
CIDP01 
 
 
 
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Page 2 of 128 
STUDY CONTACT INFORMATION 
Sponsor 
UCB Biopharma SPRL 
Allée de la Recherche 60 
1070 Brussels 
BELGIUM 
Coordinating Investigator 
Name: 
 MD PhD 
Affiliation: 
 
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 SPRL 
Allée de la Recherche, 60 
B – 1070 Brussels 
BELGIUM 
Phone: 
 
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Clinical Study Protocol 
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CIDP01 
 
 
 
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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 
CIDP01 
 
 
 
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Page 4 of 128 
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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Clinical Study Protocol 
rozanolixizumab 
CIDP01 
 
 
 
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Page 5 of 128 
TABLE OF CONTENTS 
TABLE OF CONTENTS ........................................................................................................... 5 
LIST OF TABLES ..................................................................................................................... 9 
LIST OF FIGURES ................................................................................................................. 10 
LIST OF ABBREVIATIONS .................................................................................................. 11 
1 
SUMMARY ..................................................................................................................... 15 
2 
INTRODUCTION ........................................................................................................... 17 
3 
STUDY OBJECTIVES .................................................................................................... 19 
3.1 Primary objective ............................................................................................................. 19 
3.2 Secondary objectives ....................................................................................................... 19 
3.3 Exploratory objectives ..................................................................................................... 20 
4 
STUDY VARIABLES ..................................................................................................... 20 
4.1 Efficacy variables ............................................................................................................. 20 
4.1.1 
Primary efficacy variable ..................................................................................... 20 
4.1.2 
Other efficacy variables ....................................................................................... 20 
4.2 Other variables ................................................................................................................. 21 
4.2.1 
Pharmacokinetic variable ..................................................................................... 21 
4.2.2 
Pharmacodynamic variables ................................................................................ 22 
4.2.3 
Exploratory pharmacogenetics variables ............................................................. 22 
4.2.4 
Exploratory RNA, proteins, and metabolites variables ....................................... 22 
4.2.5 
Immunological variables ..................................................................................... 22 
4.2.6 
Safety variables .................................................................................................... 23 
5 
STUDY DESIGN ............................................................................................................. 23 
5.1 Study description ............................................................................................................. 23 
5.1.1 
Study duration per subject ................................................................................... 25 
5.1.2 
Planned number of subjects and sites .................................................................. 26 
5.1.3 
Anticipated regions and countries ....................................................................... 26 
5.2 Schedule of study assessments ......................................................................................... 26 
5.2.1 
Additional study assessments .............................................................................. 33 
5.3 Schematic diagram ........................................................................................................... 35 
5.4 Rationale for study design and selection of dose ............................................................. 35 
5.4.1 
Pathophysiology of CIDP .................................................................................... 35 
5.4.2 
Mechanism of action of rozanolixizumab ........................................................... 36 
5.4.3 
Choice of study design and endpoints ................................................................. 36 
5.4.4 
Rationale for dose selection ................................................................................. 36 
5.4.5 
Justification for additional genomic analysis ...................................................... 38 
6 
SELECTION AND WITHDRAWAL OF SUBJECTS ................................................... 38 
6.1 Inclusion criteria .............................................................................................................. 38 
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CIDP01 
 
 
 
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6.2 Exclusion criteria ............................................................................................................. 40 
6.2.1 
Exclusion criteria related to study participation .................................................. 40 
6.2.2 
Exclusion criteria related to CIDP diagnosis ....................................................... 40 
6.2.3 
Exclusion criteria related to health status/safety of the subject ........................... 40 
6.2.4 
Exclusion criteria related to the IMP/concomitant medications/procedures ....... 42 
6.3 Rescreening of subjects .................................................................................................... 43 
6.4 Withdrawal criteria .......................................................................................................... 44 
6.4.1 
Potential drug-induced liver injury IMP discontinuation criteria ........................ 45 
7 
STUDY TREATMENTS ................................................................................................. 46 
7.1 Description of investigational medicinal products .......................................................... 46 
7.2 Treatments to be administered ......................................................................................... 46 
7.3 Labeling ........................................................................................................................... 48 
7.4 Handling and storage requirements.................................................................................. 48 
7.5 Drug accountability .......................................................................................................... 49 
7.6 Procedures for monitoring subject compliance ................................................................ 49 
7.7 Concomitant medications/treatments ............................................................................... 49 
7.7.1 
Permitted concomitant treatments (medications and therapies) .......................... 49 
7.7.2 
Prohibited concomitant treatments (medications and therapies) ......................... 50 
7.7.3 
Rescue medication ............................................................................................... 51 
7.8 Blinding ............................................................................................................................ 51 
7.8.1 
Procedures for maintaining and breaking the treatment blind ............................. 51 
7.8.1.1 
Maintenance of study treatment blind ....................................................... 51 
7.8.1.2 
Breaking the treatment blind in an emergency situation ........................... 52 
7.9 Randomization and numbering of subjects ...................................................................... 52 
8 
STUDY PROCEDURES BY VISIT ............................................................................... 52 
8.1 Screening Period (Day -35 to Day -1) ............................................................................. 53 
8.1.1 
Screening Visit 1 (Day -35 to Day -14) ............................................................... 53 
8.2 Treatment Period Visits 2 to 16 (Day 1 to Day 78) ......................................................... 54 
8.2.1 
Visit 2 (Day 1, Baseline) ..................................................................................... 56 
8.2.2 
Visit 3 Telephone Call and Home Visit (Day 3) ................................................. 57 
8.2.3 
Visit 4 (Day 8) ..................................................................................................... 58 
8.2.4 
Visit 5 Telephone Call and Home Visit (Day 10) ............................................... 59 
8.2.5 
Visit 6 (Day 15) ................................................................................................... 60 
8.2.6 
Visit 7 Telephone Call and Home Visit (Day 17) ............................................... 61 
8.2.7 
Visits 8 (Day 22) through 16 (Day 78) inclusive ................................................ 62 
8.3 12-Week Observation Period Visits 17 to 21 (Day 85 to Day 162) ................................ 63 
8.3.1 
Visits 17 (Day 85) through 20 (Day 141) inclusive ............................................ 67 
8.3.2 
Visit 21 (Day 162) Final Visit ............................................................................. 68 
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Clinical Study Protocol 
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CIDP01 
 
 
 
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8.3.3 
Stabilization Visits ............................................................................................... 69 
8.4 PEOT and Final Visit ....................................................................................................... 70 
8.5 Unscheduled Visit/Telephone call ................................................................................... 70 
8.6 Open-label extension study .............................................................................................. 71 
9 
ASSESSMENT OF EFFICACY ...................................................................................... 71 
9.1 iRODS .............................................................................................................................. 71 
9.2 INCAT ............................................................................................................................. 72 
9.3 Grip strength reported by site personnel .......................................................................... 72 
9.4 Patient-reported grip strength .......................................................................................... 72 
9.5 Patient-reported outcomes ............................................................................................... 73 
9.5.1 
CIDP PRO instrument ......................................................................................... 73 
9.5.2 
Fatigue ................................................................................................................. 73 
9.5.3 
Patient Global Impressions .................................................................................. 73 
9.6 RT-MRC .......................................................................................................................... 74 
9.7 Subject exit interview ...................................................................................................... 74 
10 ASSESSMENT OF PHARMACOKINETIC/ PHARMACODYNAMIC AND 
PHARMACOGENOMIC VARIABLES ......................................................................... 74 
10.1 Pharmacokinetic variables ............................................................................................... 74 
10.2 Pharmacodynamic variables ............................................................................................ 75 
10.3 Exploratory pharmacogenomic variables......................................................................... 75 
11 ASSESSMENT OF IMMUNOLOGICAL VARIABLES ............................................... 75 
12 ASSESSMENT OF SAFETY .......................................................................................... 76 
12.1 Adverse events ................................................................................................................. 76 
12.1.1 
Definitions ........................................................................................................... 76 
12.1.1.1 
Adverse event ............................................................................................ 76 
12.1.1.2 
Serious adverse event ................................................................................ 77 
12.1.1.3 
Adverse events of special interest ............................................................. 78 
12.1.1.4 
Adverse events of interest ......................................................................... 78 
12.1.2 
Procedures for reporting and recording adverse events ....................................... 78 
12.1.2.1 
Description of adverse events .................................................................... 78 
12.1.2.2 
Rule for repetition of an adverse event...................................................... 79 
12.1.2.3 
Additional procedures for reporting serious adverse events ..................... 79 
12.1.2.4 
Immediate reporting of adverse events...................................................... 80 
12.1.3 
Follow up of adverse events ................................................................................ 80 
12.1.4 
Pregnancy ............................................................................................................ 80 
12.1.5 
Suspected transmission of an infectious agent via a medicinal product .............. 81 
12.1.6 
Overdose of investigational medicinal product ................................................... 81 
12.1.7 
Safety signal detection ......................................................................................... 81 
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CIDP01 
 
 
 
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12.1.8 
Suicidality ............................................................................................................ 82 
12.1.9 
Hypersensitivity and adverse reactions ............................................................... 82 
12.1.10 
Management of headache .................................................................................... 83 
12.1.11 
Management of moderate or severe diarrhea ....................................................... 84 
12.2 Laboratory measurements ................................................................................................ 84 
12.2.1 
Evaluation of PDILI ............................................................................................ 86 
12.2.1.1 
Consultation with Medical Monitor and local hepatologist ...................... 89 
12.2.1.2 
Immediate action: determination of IMP discontinuation ......................... 89 
12.2.1.3 
Testing: identification/exclusion of alternative etiology ........................... 90 
12.2.1.4 
Follow-up evaluation ................................................................................. 92 
12.3 Other safety measurements .............................................................................................. 92 
12.3.1 
Pregnancy testing ................................................................................................. 92 
12.3.2 
Vital signs ............................................................................................................ 93 
12.3.3 
Body weight and height ....................................................................................... 93 
12.3.4 
Physical examination ........................................................................................... 93 
12.3.4.1 
Full physical examination ......................................................................... 93 
12.3.4.2 
Brief physical examination ........................................................................ 94 
12.3.5 
Neurological examination .................................................................................... 94 
12.3.6 
12-lead ECG ........................................................................................................ 94 
12.3.7 
Assessment and management of TB and TB risk factors .................................... 95 
13 STUDY MANAGEMENT AND ADMINISTRATION ................................................. 97 
13.1 Adherence to protocol ...................................................................................................... 97 
13.2 Monitoring ....................................................................................................................... 97 
13.2.1 
Definition of source data ..................................................................................... 97 
13.2.1.1 
Apps ........................................................................................................... 98 
13.2.2 
Source data verification ....................................................................................... 98 
13.3 Data handling ................................................................................................................... 98 
13.3.1 
Case Report form completion .............................................................................. 98 
13.3.2 
Database entry and reconciliation ........................................................................ 99 
13.3.3 
Subject Screening and Enrollment log/Subject Identification Code list.............. 99 
13.4 Termination of the study .................................................................................................. 99 
13.5 Archiving and data retention ............................................................................................ 99 
13.6 Audit and inspection ...................................................................................................... 100 
13.7 Good Clinical Practice ................................................................................................... 100 
14 STATISTICS ................................................................................................................. 100 
14.1 Definition of analysis sets .............................................................................................. 100 
14.1.1 
Enrolled Set ....................................................................................................... 100 
14.1.2 
Randomized Set ................................................................................................. 100 
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CIDP01 
 
 
 
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14.1.3 
Safety Set ........................................................................................................... 100 
14.1.4 
Full Analysis Set ................................................................................................ 101 
14.1.5 
Per-Protocol Set ................................................................................................. 101 
14.1.6 
Pharmacokinetic Per-Protocol Set ..................................................................... 101 
14.1.7 
Pharmacodynamic Per-Protocol Set .................................................................. 101 
14.2 General statistical considerations ................................................................................... 101 
14.3 Planned efficacy analyses .............................................................................................. 102 
14.3.1 
Analysis of the primary efficacy variable .......................................................... 102 
14.3.2 
Other efficacy analyses ...................................................................................... 102 
14.4 Other analyses ................................................................................................................ 103 
14.4.1 
Safety analyses ................................................................................................... 103 
14.4.2 
PK analyses ........................................................................................................ 103 
14.4.3 
PD analyses ........................................................................................................ 104 
14.4.4 
Immunological analyses .................................................................................... 104 
14.4.5 
Subject exit interview ........................................................................................ 104 
14.5 Handling of protocol deviations ..................................................................................... 104 
14.6 Handling of dropouts or missing data ............................................................................ 104 
14.7 Planned interim analysis and data monitoring ............................................................... 105 
14.8 Determination of sample size ......................................................................................... 105 
15 ETHICS AND REGULATORY REQUIREMENTS .................................................... 106 
15.1 Informed consent ........................................................................................................... 106 
15.2 Subject identification cards ............................................................................................ 106 
15.3 Institutional Review Boards and Independent Ethics Committees ................................ 106 
15.4 Subject privacy ............................................................................................................... 107 
15.5 Protocol amendments ..................................................................................................... 108 
16 FINANCE, INSURANCE, AND PUBLICATION ....................................................... 108 
17 REFERENCES .............................................................................................................. 108 
18 APPENDICES ............................................................................................................... 110 
18.1 Suggested management guidelines for infusion reactions ............................................. 110 
18.2 Diagnosis of anaphylactic reactions ............................................................................... 110 
18.3 Protocol amendment 1 ................................................................................................... 111 
18.4 Protocol amendment 1.1 ................................................................................................ 111 
18.5 Protocol amendment 2 ................................................................................................... 111 
19 DECLARATION AND SIGNATURE OF INVESTIGATOR ..................................... 127 
20 SPONSOR DECLARATION ........................................................................................ 128 
 
LIST OF TABLES 
Table 5‒1: 
Schedule of Assessments ................................................................................. 27 
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Clinical Study Protocol 
rozanolixizumab 
CIDP01 
 
 
 
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Table 5-2: 
Additional study assessments .......................................................................... 34 
Table 6-1: 
Time restrictions required prior to Randomization Visit (Visit 2) for 
immunosuppressants, biologics and other therapies ........................................ 43 
Table 7-1: 
IMP doses to be administered (equivalent to approximately 
 by 
body weight ...................................................................................................... 47 
Table 7-2: 
IMP doses to be administered (equivalent to approximately 
) by 
body weight ...................................................................................................... 47 
Table 7-3: 
Permitted concomitant treatments .................................................................... 50 
Table 8‒1: 
Management of Observation and Stabilization Periods ................................... 64 
Table 10-1: 
Serial blood sampling for rozanolixizumab concentration .............................. 74 
Table 12-1: 
Anticipated serious adverse events for CIDP population ................................ 78 
Table 12-2: 
Infusion-related reaction grading according to the NCI Common 
Terminology Criteria for adverse events version 4.03 (Jun 2013) .................. 83 
Table 12-3: 
Laboratory measurements ................................................................................ 85 
Table 12-4: 
Required investigations and follow up for PDILI............................................ 87 
Table 12-5: 
PDILI laboratory measurements ...................................................................... 91 
Table 12-6: 
PDILI information to be collected ................................................................... 92 
Table 18-1: 
Suggested management guidelines for infusion reactions ............................. 110 
 
LIST OF FIGURES 
Figure 5-1: 
Schematic diagram ........................................................................................... 35 
 
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rozanolixizumab 
CIDP01 
 
 
 
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LIST OF ABBREVIATIONS 
 
 
ADL 
activities of daily living 
AE 
adverse event 
ALP 
alkaline phosphatase 
ALT 
alanine aminotransferase 
ANCOVA 
analysis of covariance 
AST 
aspartate aminotransferase 
AUC 
area under the curve 
BP 
blood pressure 
CDMS 
clinical data management system 
CIDP 
chronic inflammatory demyelinating polyradiculoneuropathy 
Cmax 
maximum concentration 
CPM 
Clinical Project Manager 
CPMP 
Committee for Proprietary Medicinal Products 
CRO 
contract research organization 
C-SSRS 
Columbia Suicide Severity Rating Scale 
CTCAE 
Common Terminology Criteria for Adverse Events 
DMC 
Data Monitoring Committee 
DNA 
deoxyribonucleic acid 
ECG 
electrocardiogram 
eCRF 
electronic Case Report form 
EFNS 
European Federation of Neurological Societies 
ES 
Enrolled Set 
EudraCT 
European Union Drug Regulating Authorities Clinical Trials 
FAS 
Full Analysis Set 
FcRn 
Fc receptor 
FV 
Final Visit 
GCP 
Good Clinical Practice 
HBcAb 
hepatitis B core antibody 
HBsAg 
hepatitis B surface antigen 
HBsAb 
hepatitis B surface antibody test 
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rozanolixizumab 
CIDP01 
 
 
 
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HCAb 
hepatitis C antibody 
HIV 
human immunodeficiency virus 
HIV1 
human immunodeficiency virus antibody 1 
HIV2 
human immunodeficiency virus antibody 2 
hsCRP 
high sensitivity C-Reactive protein 
IA 
immunoabsorption 
IB 
Investigator’s Brochure 
ICH 
International Council for Harmonisation 
IEC 
Independent Ethics Committee 
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 
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 
LOCF 
last observation carried forward 
logit 
log odds unit 
LTB 
latent tuberculosis  
LTBI 
latent tuberculosis infection 
MG 
myasthenia gravis 
MHC 
major histocompatibility complex 
MCID-SE 
minimum clinically important differences-standard error 
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rozanolixizumab 
CIDP01 
 
 
 
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miRNA 
microRNA 
MMRM 
mixed model repeated measures 
MRC 
Medical Research Council 
mRNA 
messenger RNA 
NTMB 
nontuberculosis mycobacterium 
NTMBI 
nontuberculosis mycobacterium infection 
NF-L 
neurofilament light chain 
OLE 
Open-Label Extension  
ONLS 
Overall Neuropathy Limitation scale 
PD 
pharmacodynamic 
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 
PK 
pharmacokinetic 
PK-PPS 
Pharmacokinetic Per-Protocol Set 
PLEX 
plasma exchange 
PNS 
Peripheral Nerve Society 
PPS 
Per-Protocol Set 
PRO 
patient-reported outcome 
PS 
Patient Safety 
QTc 
QT-interval corrected for heart rate 
RNA 
ribonucleic acid 
RT-MRC 
Rasch-built, modified interval Medical Research Council scale 
RS 
Randomized Set 
SAE 
serious adverse event 
SAP 
Statistical Analysis Plan 
sc 
subcutaneous(ly) 
SCIg 
subcutaneous immunoglobulin 
SD 
standard deviation 
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CIDP01 
 
 
 
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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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CIDP01 
 
 
 
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1 
SUMMARY 
CIDP01 is a Phase 2A, multicenter, randomized, subject-blind, investigator-blind, 
placebo-controlled, parallel-group study with the primary objective of evaluating the clinical 
efficacy of rozanolixizumab (UCB7665) as a treatment for subjects with chronic inflammatory 
demyelinating polyradiculoneuropathy (CIDP). Secondary objectives include evaluating the 
safety and tolerability of rozanolixizumab subcutaneous (sc) infusion in subjects with CIDP, and 
assessing the effect of rozanolixizumab as measured by the total immunoglobulin G (IgG) 
concentrations in serum. Eligible subjects will be adults who have a confirmed diagnosis of 
definite or probable CIDP according to the 2010 European Federation of Neurological 
Societies/Peripheral Nerve Society (EFNS/PNS) criteria for CIDP, have been shown to be 
immunoglobulin (Ig)-dependent within 18 months before Screening and who have remained on a 
stable dosage and fixed interval of immunoglobulin therapy for at least 4 months prior to 
Screening. 
Subjects will be randomized to 1 of 2 treatment arms: rozanolixizumab 
 sc or placebo sc 
in a ratio of 1:1. For exact doses to be administered, refer to Section 7.2. Subjects will receive 
12 weekly doses of investigational medicinal product (IMP). The maximum duration of the study 
per subject 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). The study is planned to be conducted in approximately 24 
sites globally. Approximately 34 subjects will be randomized to ensure at least 30 subjects are 
evaluable for the primary efficacy analysis. 
All subjects completing the Treatment Period (ie, all visits performed without relapse) will be 
offered the possibility to enter in the Open-Label Extension (OLE) study, CIDP04, and be treated 
with rozanolixizumab. If they enter in the OLE, Visit 17 will be the last study visit in CIDP01. If 
the subjects wish to test whether they still need treatment, they will continue the Observation 
Period without standard of care (SOC) treatment (ie, Ig treatment; subcutaneous immunoglobulin 
[SCIg] and intravenous immunoglobulin [IVIg]). The subjects have also the opportunity to return 
immediately to SOC for the duration of the Observation Period. 
If the subject relapses during the Treatment Period or Observation Period (according to the 
medical judgement of the investigator supported by eg, subject’ score on inflammatory 
Rasch-built Overall Disability Scale (iRODS), Inflammatory Neuropathy Cause and Treatment 
(INCAT), or maximum grip strength (assessed by site personnel), the subject will return to the 
SOC as rescue medication at the time of relapse and will be stabilized over a period of 2 weeks 
up to maximum 12 weeks starting from relapse visit, ie, premature end of treatment (PEOT) or 
later visit. During the stabilization, visits should be performed every 1 to 3 weeks at the 
discretion of the investigator until the subject is stabilized. 
At the end of the stabilization, a subject who completed the Treatment Period without a CIDP 
relapse will be offered the possibility to enter the OLE (CIDP04) and be treated with open-label 
rozanolixizumab from that moment onwards. The subject will complete the Final Visit (FV) 
(V21) and enter the OLE on the same day. If a subject does not wish to enter the OLE, the 
subject will continue their SOC treatment after stabilization. They will continue the stabilization 
visit schedule (every 1 to 3 weeks) until they have a follow-up of 12 weeks after last IMP dose. 
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CIDP01 
 
 
 
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Subjects who relapsed during the Treatment Period will only be able to enter the OLE once all 
subjects have completed the study, the study is unblinded and it is confirmed they were on the 
placebo arm in CIDP01. At the end of stabilization, they will continue their SOC treatment after 
stabilization. They will continue the stabilization visit schedule (every 1 to 3 weeks) until they 
have a follow-up of 12 weeks after last IMP dose.  
The primary efficacy variable is the change from Baseline to Week 13 (Day 85) in iRODS score. 
Other efficacy variables will include: 
 Subject experienced CIDP relapse (iRODS) up to Week 13 (Day 85) after first treatment and 
time to CIDP relapse (iRODS) at each scheduled assessment during the Treatment Period, 
where 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. Values and change from Baseline in iRODS scores at each scheduled assessment 
during the Treatment and Observation Periods will be assessed. 
 Subject experienced CIDP relapse up to Week 13 (Day 85) after first treatment and time to 
CIDP relapse during the Treatment Period will be determined using the adjusted INCAT 
disability score where CIDP relapse is defined as an increase from Baseline of at least 1 point 
in the adjusted INCAT score. Values and change from Baseline in adjusted INCAT score at 
each scheduled assessment during the Treatment and Observation Periods will be assessed. 
 Subject experienced CIDP relapse up to Week 13 (Day 85) after first treatment and time to 
CIDP relapse during the Treatment Period will be determined using maximum grip strength 
assessed by the site personnel, where CIDP relapse is defined as a clinically important 
deterioration from Baseline in grip strength, ie, a decline of >14kPa. 
 Further variables include values and change from Baseline in maximum grip strength score 
recorded by site personnel at each scheduled assessment during the Treatment and 
Observation Periods; values and change from Baseline in daily maximum grip strength score 
recorded by the subject each day during the Treatment and Observation Periods; additional 
patient-reported outcomes (PROs); values and change from Baseline in Rasch-built, modified 
interval Medical Research Council scale (RT-MRC) sum score at each scheduled assessment 
during the Treatment and Observation Periods; and subjects receiving rescue medication and 
time to rescue medication administration. 
Other and exploratory variables include: safety and tolerability variables, pharmacokinetic (PK), 
pharmacodynamic (PD), immunologic variables, exploratory pharmacogenetics variables, and 
exploratory ribonucleic acid (RNA), proteins, and metabolites biomarkers. Plasma concentration 
of rozanolixizumab over time will be assessed as the PK variable. The PD variables are 
minimum and maximum decrease from Baseline in total IgG concentration during the study; 
value and change from Baseline in IgG concentrations at each scheduled assessment during 
Treatment and Observation Periods; value and change from Baseline in IgG subclass 
concentrations at each scheduled assessment during Treatment and Observation Periods; and 
value and change from Baseline in neurofilament light chain (NF-L) levels at each scheduled 
assessment during Treatment and Observation Periods. Immunological variables will also be 
assessed. 
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Safety and tolerability variables will include occurrence of treatment-emergent adverse events 
(TEAEs); TEAEs leading to withdrawal of IMP; vital sign values and changes from Baseline 
(systolic and diastolic blood pressure [BP], temperature, pulse rate, and body weight) at each 
scheduled assessment during Treatment and Observation Periods; 12-lead electrocardiogram 
(ECG) parameters 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 (TB) evaluation; and 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. 
Exploratory analyses of deoxyribonucleic acid (DNA), RNA, proteins, and metabolites 
biomarkers may be performed to understand the cause, progression, and appropriate treatment of 
CIDP. 
A Data Monitoring Committee (DMC) will be established for the study. 
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 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 (MHC)-class-I-like Fc receptor (FcRn) recycles 
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 
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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 subjects in a completed, first-in-human 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). See summaries below for UP0018, TP0001, and MG0002. 
UP0018 is a completed, first-in-human study conducted in 49 healthy subjects. The study 
evaluated the safety, tolerability, PK, and the PD effect on total IgG levels of single ascending 
doses of intravenous (iv) and sc rozanolixizumab. Doses of rozanolixizumab 
 
were administered by both iv and sc routes in 3 cohorts, respectively, as a 
. The 
information from the study is summarized below. 
There were no deaths or serious adverse events (SAEs) reported during UP0018, and no subjects 
discontinued the study due to TEAEs. Rozanolixizumab was tolerated with an acceptable safety 
profile after the single administration of 
iv and 
 sc doses. Four TEAEs with a 
maximum intensity of severe were reported in this study: headache (3 subjects [50.0%]) and 
back pain (1 subject [16.7%]); all of which were reported in the rozanolixizumab 
 iv 
group. For sc administration of rozanolixizumab, the most frequently reported TEAEs were 
headache (5 subjects [27.8%]), and back pain and diarrhea (each reported by 3 subjects [16.7%]). 
No severe adverse events (AEs) were reported following sc administration. Further details on 
both iv and sc administration may be found in the current version of the Investigator’s Brochure 
(IB). The peak and total exposure of rozanolixizumab showed nonlinear increases consistent 
with target-mediated drug disposition. Dose-dependent statistically significant reductions in 
levels of total IgG and dose-dependent reductions in levels of IgG subclasses (IgG 1 to 4) were 
observed after rozanolixizumab was administered by iv or sc routes. 
TP0001 is a completed, Phase 2, multicenter, open-label, multiple-dose, multiple-arm study to 
evaluate the safety, tolerability, and efficacy of rozanolixizumab administered as sc doses of 
 
 in 66 subjects ≥18 years of age with 
persistent or chronic ITP.  
There were no treatment-emergent deaths reported during TP0001, and no subjects discontinued 
from the study or IMP due to TEAEs. There were no treatment-related SAEs reported from the 
study. All TEAEs considered by the investigator to be related to rozanolixizumab were mild to 
moderate in intensity and resolved without clinical sequelae. The majority of the related TEAEs 
did not require treatment. The most common TEAEs were headache (26 subjects [39.4%]), 
diarrhea (8 subjects [12.1%]), and vomiting (6 subjects [9.1%]). Treatment with rozanolixizumab 
was generally 
 and well tolerated with an acceptable safety profile across the treatment 
groups.  
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MG0002 is a completed, multicenter, randomized, investigator-and subject-blind, 
placebo-controlled, treatment sequence study evaluating the safety, tolerability, and efficacy of 
rozanolixizumab in 43 subjects with moderate to severe MG. 
 
 
 
 
 
There were no deaths reported during MG0002. One subject (2.3%) discontinued from the study 
due to an AE (MG crisis). In addition, 3 subjects experienced TEAEs (headache) that led to 
discontinuation of IMP, but the subjects remained in the study. Six subjects (14.0%) reported a 
total of 7 serious TEAEs. One subject in the Placebo-Rozanolixizumab 
Group 
experienced a serious TEAE of headache that was considered by the investigator to be related to 
the IMP. With the exception of 1 serious TEAE of ulna fracture, all other serious TEAEs were in 
the System Organ Class of Nervous system disorders. Three subjects (7.0%) reported serious 
TEAEs of MG (the studied indication), and 1 subject (2.3%) reported a serious TEAE of MG 
crisis. The most common TEAEs were headache (23 subjects [53.5%]), diarrhea (11 subjects 
[25.6%]), and nausea (6 subjects [14.0%]). Overall, repeated administrations of rozanolixizumab 
at dose levels of 
and 
 were generally 
 and well tolerated, with an 
acceptable safety profile. 
The underlying hypothesis of the current study is that reducing the concentration of pathogenic 
IgG in patients with CIDP will improve the symptoms of CIDP and prevent long-term 
progression. Thus, rozanolixizumab may provide an important novel therapeutic approach to 
address the need for effective treatment of CIDP. 
The current study, CIDP01, will assess the effectiveness of treatment with a weekly dosing 
regimen of rozanolixizumab for 12 weeks (dosing at Visits 2, 4, 6, and Visits 8 through 16) in 
subjects with active CIDP who have been maintained on an immunoglobulin-dependent regimen. 
In addition, this study will assess the safety and tolerability of multiple dosing with 
rozanolixizumab including the development of 
 and potential to impact 
clinical response, and will provide information on the PK and PD of rozanolixizumab in subjects 
with active CIDP. 
Additional information on the development of rozanolixizumab can be found in the current 
version of the IB for rozanolixizumab. 
3 
STUDY OBJECTIVES 
3.1 
Primary objective 
The primary objective of the study is: 
 To evaluate the clinical efficacy of rozanolixizumab as a treatment for subjects with CIDP 
3.2 
Secondary objectives 
The secondary objectives of the study are: 
 To evaluate the safety and tolerability of rozanolixizumab sc infusion in subjects with CIDP 
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 To assess the PD effect of rozanolixizumab as measured by the total IgG concentrations in 
serum 
3.3 
Exploratory objectives 
Exploratory objectives are: 
 To evaluate the effects of rozanolixizumab on the concentration of total protein, albumin, 
α- and β-globulins, IgG subclasses, IgM, IgA, and IgE, serum and plasma complement levels 
 To evaluate the incidence and emergence of 
) with respect to 
immunogenicity and PK and PD 
 To evaluate the effect of rozanolixizumab on complement and cytokines 
 To assess the plasma concentrations of rozanolixizumab administered by sc infusion 
 To assess the PD effect of rozanolixizumab as measured by NF-L in serum 
 To assess the effect of rozanolixizumab on gene and protein expression, and explore the 
relationship between DNA, RNA, protein, and metabolite biomarkers and cause, progression, 
and appropriate treatment of CIDP 
 To assess the effect of rozanolixizumab on CIDP-specific auto-antibody levels 
 To assess the effect of rozanolixizumab on vaccine antibody levels (
) 
4 
STUDY VARIABLES 
4.1 
Efficacy variables 
4.1.1 
Primary efficacy variable 
The primary efficacy variable is the: 
 Change from Baseline to Week 13 (Day 85) in iRODS score 
4.1.2 
Other 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 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  
Other efficacy variables will include: 
 Subject experienced CIDP relapse (iRODS) up to Week 13 (Day 85) after first treatment 
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 Time to CIDP relapse (iRODS) during the Treatment Period 
 Values and change from Baseline in iRODS scores at each scheduled assessment during the 
Treatment and Observation Periods 
 Subject experienced CIDP relapse (adjusted INCAT) up to Week 13 (Day 85) after first 
treatment 
 Time to CIDP relapse (adjusted INCAT) during the Treatment Period 
 Values and change from Baseline in adjusted INCAT score at each scheduled assessment 
during the Treatment and Observation Periods 
 Subject experienced CIDP relapse (maximum grip strength as assessed by site personnel) 
up to Week 13 (Day 85) after first treatment 
 Time to CIDP relapse (maximum grip strength as assessed by site personnel) during the 
Treatment Period 
 Values and 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 change from Baseline in daily maximum grip strength score (maximum of 
3 assessments) taken by the subject at the same time each day during the Treatment and 
Observation Periods 
 Values and change from Baseline in RT-MRC sum score at each scheduled assessment 
during the Treatment and Observation Periods 
 Values and change from Baseline in fatigue at each scheduled assessment during the 
Treatment and Observation Periods 
 Values and change from Baseline in CIDP PRO instrument 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) at each scheduled assessment during the 
Treatment and Observations Periods 
 Subjects receiving rescue medication during Treatment Period 
 Time to rescue medication administration during Treatment Period 
4.2 
Other variables 
4.2.1 
Pharmacokinetic variable 
The PK variable is: 
 Plasma concentration of rozanolixizumab at each scheduled assessment during the Treatment 
Period 
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4.2.2 
Pharmacodynamic variables 
The PD variables are: 
 Minimum value and maximum decrease (absolute and percentage) from Baseline in total 
serum IgG concentration during the study 
 Value and change (absolute and percentage) from Baseline in total serum IgG concentrations 
at each scheduled assessment during Treatment and Observation Periods 
 Value and change (absolute and percentage) from Baseline in serum IgG subclass 
concentrations at each scheduled assessment during Treatment and Observation Periods 
 Value and change (absolute and percentage) from Baseline in NF-L levels at each scheduled 
assessment during Treatment and Observation Periods 
4.2.3 
Exploratory pharmacogenetics variables 
The exploratory pharmacogenetics variables are: 
 Genetic and epigenetic changes that may be measured to understand the cause, progression, 
and appropriate treatment of CIDP 
4.2.4 
Exploratory RNA, proteins, and metabolites variables 
The exploratory RNA, proteins, and metabolites variables are: 
 RNA, proteins, and metabolites changes that may be measured to understand the cause, 
progression, and appropriate treatment of CIDP 
 Exploratory biomarkers such as but not limited 
 
 
 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 
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 
 
 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 
 
during Treatment and Observation Periods 
4.2.6 
Safety variables 
The safety variables for the study are: 
 Occurrence of TEAEs 
 TEAEs leading to withdrawal of IMP 
 Vital sign values and changes from Baseline (systolic and diastolic BP, temperature, pulse 
rate, and body weight) at each scheduled assessment during Treatment and Observation 
Periods 
 12-lead 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 
5 
STUDY DESIGN 
5.1 
Study description 
This is a Phase 2A, multicenter, randomized, subject-blind, investigator-blind, 
placebo-controlled, parallel-group study evaluating the efficacy, safety, and tolerability of 
rozanolixizumab for treatment of subjects with CIDP. Eligible subjects will be adults who have a 
confirmed diagnosis of definite or probable CIDP according to the EFNS/PNS criteria for CIDP, 
have been shown to be dependent on treatment with immunoglobulin and who have remained on 
a stable dosing regimen of immunoglobulin therapy for at least 4 months prior to Screening. 
 Treatment Arm 1: 
 
 
 Treatment Arm 2: 
 
 
See Section 7.2 for details on treatments to be administered. 
Subjects will be randomized in a ratio of 1:1 to the 2 treatment arms. The randomization will 
take place on the day of first treatment with IMP. The randomization will be stratified according 
to previous route of administration for Ig treatment (ie, SCIg and IVIg). 
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The dose level of 
may be reduced to 
 (or equivalent placebo) for an individual 
subject based on their tolerability (see Section 7.2 for details). For example, if a subject 
experiences a severe headache which does not respond to headache treatment (see 
Section 12.1.10), a decision to decrease future doses to 
may be made. The decision to 
decrease the dose will be made following a discussion between the investigator and the UCB 
study physician. In addition, the decision to decrease the dose for the remainder of the subjects 
may be made following a DMC recommendation. 
The study consists of: 
 Screening Period of between 2 and 5 weeks duration. The purpose of the Screening Period is 
to evaluate and confirm the subject’s eligibility. All subjects must have an Ig dependency 
that has been confirmed within 18 months before Screening. All subjects must be on a 
long-term immunoglobulin maintenance therapy with a stable immunoglobulin infusion 
regimen at consistent intervals to be considered eligible. The Screening Period will be a 
minimum of 2 weeks and generally should not exceed 5 weeks. During the Screening Period, 
the subjects will receive their last dose of immunoglobulin therapy before the randomization 
(see details in Section 8.2). 
 The 11-week Treatment Period begins at the Randomization Visit (Visit 2) on Day 1 to 
review inclusion and exclusion criteria as well as concomitant medications and treatments. 
Subjects who remain eligible will be randomized 1:1 to receive rozanolixizumab 
or 
placebo. All eligible subjects will receive the same treatment at weekly intervals during the 
full duration of the Treatment Period, ending with the last dose of IMP (Visit 16). After 
initial 4 IMP administrations have been performed at the clinic, the subject will have the 
opportunity to be treated at home at every other visit. 
 The 12-week Observation Period (from 1 week up to maximum 24 weeks): 
 All subjects completing the Treatment Period (ie, all visits up to Visit 17 performed 
without a CIDP relapse) will be offered the possibility to enter in the OLE study, 
CIDP04, and be treated with rozanolixizumab. If they enter in the OLE, Visit 17 will be 
the last study visit. The Observation Period will be limited to 1 week for these subjects. 
In all other instances the subjects will perform Visits 18 to 21 for efficacy and for safety 
assessments (see Schedule of Assessments; Table 5‒1). Subjects will either return to the 
clinic/hospital, or, if possible and agreed by both investigator and subject, have home 
visits conducted by certified healthcare professionals for Visits 18 and 20. 
 For subjects who complete the Treatment Period without relapse but choose not to enter 
the OLE directly and wish to first test whether they still need treatment, they will 
continue the Observation Period without SOC treatment. 
 Subjects who do not wish to enter the OLE or test whether they still need treatment after 
having successfully completed the Treatment Period also have the opportunity to return 
immediately to SOC for the duration of the Observation Period. 
 All other subjects (ie, having relapsed or withdrawn from the study during Treatment 
Period) will complete the Observation Period. Subjects can return to their SOC for the 
duration of the Observation Period. 
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 Stabilization: If the subject relapses during the Treatment Period or Observation Period 
(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]), the subject 
will return to the SOC (ie, Ig treatment) as rescue medication at the time of relapse and 
will be stabilized over a period ranging from 2 weeks up to maximum 12 weeks starting 
from relapse visit (eg, PEOT [Visit 17] or later visit). 
During the stabilization, visits should be performed every 1 to 3 weeks at the discretion of 
the investigator until the subject is stabilized. The stabilization may extend the Observation 
Period duration up to a maximum of 24 weeks after the last IMP dose. 
At the end of the stabilization: 
1. A subject who completes the Treatment Period without a CIDP relapse will be offered the 
possibility to enter in the OLE (CIDP04) and be treated with rozanolixizumab from that 
moment onwards. The subject will complete the FV (V21) and enter the OLE on the 
same day. 
2. A subject who completes the Treatment Period and does not wish to enter the OLE 
(CIDP04) will continue their SOC treatment after stabilization and the stabilization visit 
schedule (every 1 to 3 weeks) until they have a follow-up of 12 weeks after last IMP 
dose. The subject will complete the FV (V21) and enter the OLE on the same day. 
3. A subject who relapses during Treatment Period will continue their SOC treatment after 
stabilization. They will continue the stabilization visit schedule (every 1 to 3 weeks) until 
they have a follow-up of 12 weeks after last IMP dose. The subject will complete the FV 
(V21) and end CIDP01 study participation. These subjects 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 was confirmed they received placebo. 
Access to the OLE (CIDP04) is allowed for subjects completing the Treatment Period without 
relapse/withdrawal. Entry can occur immediately upon completion of the Treatment Period or 
after stabilization in case the subject relapsed during Observation Period and did not meet 
withdrawal criteria other than relapse. Subjects who relapse during the Treatment Period will 
have the opportunity to enter the OLE once all subjects have completed the study, the study is 
unblinded, and key results are available. In this case, only the subjects from the placebo arm will 
be allowed to enter the OLE study. 
See Table 8‒1 for details on the different options for subjects in the Observation and 
Stabilization Periods. 
A DMC will be established to monitor safety data during the study (see Section 14.7). 
5.1.1 
Study duration per subject 
The total duration of the study per subject is approximately 28 weeks (up to maximum 
40 weeks), consisting of: 
 Screening Period of a minimum of 2 to a maximum of 5 weeks starting with Screening Visit 
(to occur between Day -35 and Day -14, both inclusive). 
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 Treatment Period (11 weeks) – from first dose of IMP to last dose of IMP. The first treatment 
with IMP will occur at the Randomization Visit (Visit 2) and the dosing should occur as 
seamlessly as possible to continue the momentum of the Baseline therapy (see Section 8.2). 
 Observation Period (12 weeks up to 24 weeks) starting day after last IMP dose until study 
end. 
In case of early withdrawal, the duration of the Treatment Period may be shortened depending on 
the time to withdrawal. The Observation Period may be shortened to 1 week in case a subject 
completes the Treatment Period and enters the OLE study immediately. In case a subject relapses 
during the Observation Period, the subject will be stabilized over a period of minimum 2 weeks 
up to 12 weeks, possibly extending the Observation Period to a maximum of 24 weeks. 
The end of the study is defined as the date of the last visit of the last subject in the study. 
5.1.2 
Planned number of subjects and sites 
To ensure that at least 30 subjects are evaluable for the primary efficacy analysis, a sample size 
of approximately 34 randomized subjects is planned at approximately 24 sites. 
It is expected that approximately 50 subjects need to be screened in order to get 34 randomized if 
the screening failure rate is approximately 30%. 
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 is presented in Table 5‒1. 
 
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Table 5‒1: 
Schedule of Assessments 
 
Assessments 
Scra 
11-Week Treatment Period 
12-Week Observation Period s 
 
Visit No 
1 
2 
3 b 
4 
5 b 
6 
7 b 
8 
9c 
10 
11c 
12 13c 14 15c 
16 
17/ 
PEOT 
18c 
19 
20 c 
21/ 
FV 
Stab 
visitq 
Visit type 
S 
S 
TH 
S 
TH 
S 
TH 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
H 
S 
H 
S 
 
Week 
 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
11 
12 
13 
15 
18 
21 
24 
 
Day 
-35 to  
-14 
1 
BL 
2 
or 
3 
8 
±2 
9 or 
10 
15 
±2 
16 
or 
17 
22 
±2 
29 
±2 
36 
±2 
43 
±2 
50 
±2 
57 
±2 
64 
±2 
71 
±2 
78 
±2 
85 
±2 
99 
±3 
120 
±3 
141 
±3 
162 
±3 
 
Written informed 
consent 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic data 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Verification of 
inclusion and exclusion 
criteria 
X 
X d 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
CIDP history 
(symptoms, date of 
onset, therapy) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Randomization 
 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Withdrawal criteria 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
General medical and 
procedures history 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior and concomitant 
medication 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant medical 
procedures 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Vital signs e 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Body weight, height f 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
Recording of AEs 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
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Clinical Study Protocol 
rozanolixizumab 
CIDP01 
 
 
 
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Page 28 of 128 
Table 5‒1: 
Schedule of Assessments 
 
Assessments 
Scra 
11-Week Treatment Period 
12-Week Observation Period s 
 
Visit No 
1 
2 
3 b 
4 
5 b 
6 
7 b 
8 
9c 
10 
11c 
12 13c 14 15c 
16 
17/ 
PEOT 
18c 
19 
20 c 
21/ 
FV 
Stab 
visitq 
Visit type 
S 
S 
TH 
S 
TH 
S 
TH 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
H 
S 
H 
S 
 
Week 
 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
11 
12 
13 
15 
18 
21 
24 
 
Day 
-35 to  
-14 
1 
BL 
2 
or 
3 
8 
±2 
9 or 
10 
15 
±2 
16 
or 
17 
22 
±2 
29 
±2 
36 
±2 
43 
±2 
50 
±2 
57 
±2 
64 
±2 
71 
±2 
78 
±2 
85 
±2 
99 
±3 
120 
±3 
141 
±3 
162 
±3 
 
Query for suicidality g 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
Fatigue scale 
 
X 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
 
X 
 
 
 
X 
 
CIDP PRO instrument 
 
X 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
 
X 
 
 
 
X 
 
PGIS 
 
X 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
 
X 
 
 
 
X 
 
PGIC 
 
 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
 
X 
 
 
 
X 
 
Full physical 
examination 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
Brief physical 
examination 
 
X 
 
X 
 
X 
 
X 
 
X 
 
 
 
X 
 
 
 
 
X 
 
 
X 
Full neurological 
examination h 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
Brief neurological 
examination 
 
X 
 
X 
 
X 
 
X 
 
X 
 
 
 
X 
 
 
 
 
X 
 
 
X 
12-lead ECG 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
 
 
X 
 
Labs (hematology, 
chemistry, urinalysis) 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
X 
 
X 
X t 
Serology for HIV, 
hepatitis B, and 
hepatitis C i 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Serum pregnancy test 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
 
 
 
 
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CIDP01 
 
 
 
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Table 5‒1: 
Schedule of Assessments 
 
Assessments 
Scra 
11-Week Treatment Period 
12-Week Observation Period s 
 
Visit No 
1 
2 
3 b 
4 
5 b 
6 
7 b 
8 
9c 
10 
11c 
12 13c 14 15c 
16 
17/ 
PEOT 
18c 
19 
20 c 
21/ 
FV 
Stab 
visitq 
Visit type 
S 
S 
TH 
S 
TH 
S 
TH 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
H 
S 
H 
S 
 
Week 
 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
11 
12 
13 
15 
18 
21 
24 
 
Day 
-35 to  
-14 
1 
BL 
2 
or 
3 
8 
±2 
9 or 
10 
15 
±2 
16 
or 
17 
22 
±2 
29 
±2 
36 
±2 
43 
±2 
50 
±2 
57 
±2 
64 
±2 
71 
±2 
78 
±2 
85 
±2 
99 
±3 
120 
±3 
141 
±3 
162 
±3 
 
Urine pregnancy test j 
 
X 
 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
IGRA tuberculosis test 
k 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Tuberculosis Signs and 
Symptoms 
questionnaire 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
 
X 
 
Chest x-ray p 
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 l 
 
X 
X 
X 
X 
X 
X 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
 
 
 
 
Blood sampling for 
DNA analysis 
 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
 
 
 
 
Blood sampling for 
RNA analysis 
 
X 
 
X 
 
X 
 
 
 
X 
 
 
 
 
 
 
X 
 
 
 
 
 
 
 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
 
 
X 
 
Serum complement 
(C3, C4) and plasma 
complement (C3a, 
C5a) m 
 
X 
 
X 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
 
 
 
 
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CIDP01 
 
 
 
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Table 5‒1: 
Schedule of Assessments 
 
Assessments 
Scra 
11-Week Treatment Period 
12-Week Observation Period s 
 
Visit No 
1 
2 
3 b 
4 
5 b 
6 
7 b 
8 
9c 
10 
11c 
12 13c 14 15c 
16 
17/ 
PEOT 
18c 
19 
20 c 
21/ 
FV 
Stab 
visitq 
Visit type 
S 
S 
TH 
S 
TH 
S 
TH 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
H 
S 
H 
S 
 
Week 
 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
11 
12 
13 
15 
18 
21 
24 
 
Day 
-35 to  
-14 
1 
BL 
2 
or 
3 
8 
±2 
9 or 
10 
15 
±2 
16 
or 
17 
22 
±2 
29 
±2 
36 
±2 
43 
±2 
50 
±2 
57 
±2 
64 
±2 
71 
±2 
78 
±2 
85 
±2 
99 
±3 
120 
±3 
141 
±3 
162 
±3 
 
Serum cytokines m 
 
X 
 
X 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
 
 
 
 
Vaccination-specific 
antibody titers (
 
) 
X 
 
 
 
 
 
 
X 
 
 
 
 
 
X 
 
 
 
 
 
 
X 
 
Immunoglobulins 
(total IgG and IgG 
subclasses) 
X 
X 
 
X 
 
X 
X c 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
X 
 
NF-L 
 
X 
 
 
 
X 
 
 
 
 
 
 
 
 
 
X 
 
 
 
 
X 
 
IgA, IgE, IgM 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
 
 
X 
 
CIDP-specific auto-
antibodies 
 
X 
 
X 
 
X 
 
X 
 
 
 
X 
 
 
 
X 
X 
 
 
 
 
 
Blood sampling for 
exploratory biomarker 
analysis n 
 
X 
 
X 
 
 
 
X 
 
 
 
X 
 
 
 
X 
 
 
 
 
 
 
iRODS assessment o 
X 
X 
 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
INCAT assessment 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
Site personnel 
assessment of grip 
strength 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
X 
 
X 
X 
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Table 5‒1: 
Schedule of Assessments 
 
Assessments 
Scra 
11-Week Treatment Period 
12-Week Observation Period s 
 
Visit No 
1 
2 
3 b 
4 
5 b 
6 
7 b 
8 
9c 
10 
11c 
12 13c 14 15c 
16 
17/ 
PEOT 
18c 
19 
20 c 
21/ 
FV 
Stab 
visitq 
Visit type 
S 
S 
TH 
S 
TH 
S 
TH 
S 
H 
S 
H 
S 
H 
S 
H 
S 
S 
H 
S 
H 
S 
 
Week 
 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
11 
12 
13 
15 
18 
21 
24 
 
Day 
-35 to  
-14 
1 
BL 
2 
or 
3 
8 
±2 
9 or 
10 
15 
±2 
16 
or 
17 
22 
±2 
29 
±2 
36 
±2 
43 
±2 
50 
±2 
57 
±2 
64 
±2 
71 
±2 
78 
±2 
85 
±2 
99 
±3 
120 
±3 
141 
±3 
162 
±3 
 
Issue subject e-diary 
for recording of daily 
grip strength 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Review subject e-diary 
for daily assessment of 
grip strength 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
RT-MRC assessment 
X 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
 
 
X 
 
Collect subject e-diary 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
Subject exit interview 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Xr 
 
 
 
X 
 
AE=adverse event; BL=Baseline (predose); C3=complement component 3; C3a=complement component 3a; C4=complement component 4; C5a=complement 
component 5a; CIDP=chronic inflammatory demyelinating polyradiculoneuropathy; C-SSRS=Columbia Suicide Severity Rating Scale; 
DNA=deoxyribonucleic acid; 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; OLE=Open-Label Extension; PEOT=premature end of treatment; PGIC=Patient Global 
Impressions of Change; PGIS=Patient Global Impressions of Severity; PK=pharmacokinetic; PRO=patient-reported outcome; RNA=ribonucleic acid; 
RT-MRC=Rasch-built, modified-interval Medical Research Council scale; S=site visit; Scr=Screening; Stab=stabilization; TH=telephone call and home visit. 
Note: All assessments are to be completed in order specified in Section 8 if possible. The ICF should be completed before any assessment. PROs should be 
conducted in the order specified in Section 9.5 immediately after ICF (where applicable). The laboratory manual will provide further guidance on the order of 
sample collection. 
a Screening Visit will be performed between Day -35 and -14, the Screening Period continues until Day -1. 
b One or two days after the subject’s first 3 doses of IMP, the subject will be telephoned, and a healthcare professional will visit the subject at home to collect PK 
samples and immunoglobulins (total IgG and IgG subclasses) (only at Visit 7). Alternately, the visit can be conducted at the site as deemed necessary by site 
personnel and/or the subject.  
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c The following visits (9, 11, 13, 15, 18, and 20) will be performed by a healthcare professional visiting the subject at his/her home. 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 For eligibility criteria the lab data from Screening will be used. However, the urine pregnancy test at Visit 2 also must be negative for eligibility. 
e On dosing visits prior to Visit 9 vital signs will be measured prior to IMP administration, at the end of the infusion, at 2 and 4 hours after the end of the 
infusion. From Visit 9, vital signs will be measured predose, at the end of the infusion and 2 hours 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. 
f Body weight at screening will be used for calculation of the dose. Height will be assessed only at Screening.  
g A full C-SSRS assessment will be performed only when 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 See requirements in Table 5-2 in case the subject experiences severe headache. 
i Serology includes hepatitis C virus (HCV)-antibodies (Ab)+, hepatitis B virus antibodies (HBsAg and HBcAb), human immunodeficiency virus antibodies 
(HIV1 and HIV2). 
j The pregnancy test must be negative before dosing. Note that the final urinary pregnancy test of the study should be no longer than 90 days after the final dose 
of IMP. 
k The IGRA test will be performed in a central laboratory. Subject should not be dosed until IGRA result is available and negative.  
l At Visits 2, 4, 6, 8, and 10, PK samples should be taken predose and 4 hours postdose for all subjects. At Visits 12, 14, and 16 a predose sample only should be 
taken. At Visits 3, 5, 7 and 17, samples to be taken once during the visit. 
m 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 4 hours postdose at Visits 4, 8, 12, and 16 for all subjects. See Table 5-2 for sampling in case of infusion reactions (with the exception of local site 
infusion reactions).  
n At Visits 2, 4, 8, 12 and 16, samples for exploratory biomarkers should be taken predose. See Table 5-2 for sampling in case of AE of interest.  
o Assessment to be performed before all other assessments at each visit (including the Screening Visit, where possible) except ICF. 
p  If a subject has had a recent radiograph of the chest within 3 months prior to the Screening Visit, it may be used as the Screening chest x-ray. 
q  Stabilization visits should take place in case of the subject relapse. Visits should be performed every 1 to 3 weeks at the discretion of the investigator until 
subject is stabilized. Stabilization may last from 2 weeks up to 12 weeks. At the end of stabilization, subjects who completed the Treatment Period without a 
CIDP relapse can enter the OLE study (CIDP04) immediately; the other subjects continue SOC. Time of required follow-up from last IMP dose to the FV is 
12 weeks minimum. The stabilization visit schedule still applies (every 1 to 3 weeks). 
r  For subject entering in OLE immediately after completion of the Treatment Period.  
s  Observation Period starts the day after Visit 16. 
t  Laboratory samples during stabilization should be scheduled every 4 to 6 weeks. 
 
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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 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 2 hours at Visits 4, 8, 
12, and 16, samples should also be taken 2 hours postdose (see Section 12.1.9 and 
Section 18.1).  
In subjects who experience an infusion reaction at Visits 2, 6, 10, and 14, samples 
should be taken 2 hours postdose and 4 hours postdose (see Section 12.1.9 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 or during a telephone call, 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 12.1.1.4) at 
Visits 8, 12 and 16, 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 12.1.10).  
Full neurological 
examination 
Assessments required for all subjects 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 12.1.10). 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. 
Other 
In subjects 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 12.1.10).  
For subjects who experience moderate or severe diarrhea: 
Stool sample 
assessment 
In subjects 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 12.1.11).  
If the moderate or severe diarrhea is initially reported at a home visit or during a 
telephone call, the subject should be reviewed at the study site as soon as practically 
possible. 
AE=adverse event; CSF=cerebral spinal fluid; CT=computed tomography; LP=lumbar puncture; MRI=magnetic 
resonance imaging. 
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5.3 
Schematic diagram 
A study schematic is presented in Figure 5-1.  
Figure 5-1: Schematic diagram 
 
FV=Final Visit; Ig=immunoglobulin; OLE=open-label extension; PEOT=premature end of treatment; 
sc=subcutaneous; SOC=standard of care; V=Visit 
See Table 8‒1 for details on the management of the Observation and Stabilization Periods.  
5.4 
Rationale for study design and selection of dose 
5.4.1 
Pathophysiology of CIDP 
Production of pathogenic auto-antibodies is a major feature of a number of autoimmune diseases 
often associated with a specific pathomechanism. 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 
PLEX and IA. Identification of the specific antigenic target(s) of the autoimmune antibodies in 
CIDP is expanding with recent immunological techniques. Treatments aimed at reducing the 
quantity of circulating IgG auto-antibodies are being used for primary and secondary therapy of 
autoimmune diseases, particularly where corticosteroid-based immune suppression is not or no 
longer effective. The therapeutic approach of these treatments is based on lowering levels of 
pathogenic auto-antibodies, which represents rational and effective treatment modalities of 
autoimmune diseases. 
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5.4.2 
Mechanism of action of rozanolixizumab 
Rozanolixizumab is a humanized IgG4 monoclonal antibody that is being developed as an 
inhibitor of the activity of FcRn. The FcRn recycles IgG and albumin and transports it 
bidirectionally across epithelial barriers. Recent studies have shown that FcRn rescues both IgG 
and albumin from intracellular lysosomal degradation by recycling it from the sorting endosome 
to the cell surface (Anderson et al, 2006). Rozanolixizumab has been specifically designed to 
block IgG binding to FcRn without blocking the binding and recycling of albumin. By blocking 
the activity of FcRn, rozanolixizumab accelerates the catabolism of IgG antibodies, including 
IgG auto-antibodies. The aim is to reduce the concentration of pathogenic IgG in patients with 
autoimmune diseases mediated by the action of IgG auto-antibodies. 
5.4.3 
Choice of study design and endpoints 
The primary objective of the study is to evaluate the clinical efficacy of rozanolixizumab as a 
treatment for subjects with CIDP. This 2-arm parallel design study will compare the change from 
Baseline to Week 13 (Day 85) in iRODS score in the rozanolixizumab compared to placebo 
treatment groups as the primary efficacy variable. In addition, the study will evaluate efficacy 
using CIDP relapse up to Week 13 (Day 85) after treatment start according to the iRODS, 
adjusted INCAT scores, and maximum grip strength as assessed by site personnel, as well as 
treatment comparison versus placebo for the rozanolixizumab group. Both the iRODS and 
INCAT are measures of disability in CIDP. The INCAT scale was used in the registration study 
for IGIV-C (Gamunex-C; Hughes et al, 2008). The iRODS has demonstrated significantly higher 
responsiveness to the ordinal-based INCAT-Overall Neuropathy Limitation scale (INCAT-
ONLS) and the modified INCAT sensory scale when using the minimum clinically important 
differences using the obtained individual standard error (MCID-SE) method for defining a 
relapse (MCID-SE ≤-1.96) (Vanhoutte et al, 2015; Draak et al, 2014). The randomized, 
placebo-controlled study design is widely accepted as optimal for assessing efficacy. Grip 
strength was assessed in the CIDP ICE (Merkies et al, 2010) and the PATH trials (van Schaik 
et al, 2017) and 14kPa was evaluated to be the MCID for determining a relapse. 
A treatment duration of 11 weeks was judged to be sufficiently long to evaluate a difference 
between rozanolixizumab 
 treatment and placebo (Nobile-Orazio et al, 2015; 
Hughes et al, 2008).  
5.4.4 
Rationale for dose selection 
The dose and regimen of IMP to be used in the current study (rozanolixizumab
 sc) was 
selected based on the results from first-in-human study UP0018, alongside the emerging safety 
data from the MG and ITP studies (MG0002 and TP0001).  
UP0018 was a randomized, subject-blind, investigator-blind, placebo-controlled, single 
dose-escalating study to evaluate the safety and PK and to explore the PD of rozanolixizumab 
doses of 
 in 49 healthy male 
and female subjects. Data indicate that mean absolute decreases in IgG and mean percent change 
from Baseline IgG were greater in the active dose groups (n=6 each) compared to the pooled iv 
and sc placebo group (n=12) with maximum decreases of 49.3% (range: 44.6% to 55.9%) 
observed on Day 6 for a rozanolixizumab 
 iv dose and 42.8% (range: 39.6% to 48.6%) on 
Day 9 for a rozanolixizumab 
sc dose. Rozanolixizumab was tolerated with an acceptable 
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Clinical Study Protocol 
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safety profile after the single administration of a 
sc dose, and all subject-reported TEAEs 
had a maximum intensity of mild or moderate. 
MG0002 is a completed, Phase 2, multicenter, randomized, investigator- and subject-blind, 
placebo-controlled, 2-arm repeat dose, treatment sequence study evaluating the safety and 
efficacy of rozanolixizumab sc 
 in 43 subjects with generalized MG. 
 
 
 
 
 
 Overall, repeated administrations of rozanolixizumab at dose levels of 
 
 sc were generally 
 and well tolerated, with an acceptable safety profile. No new 
safety concerns were identified; a summary of results is included in the IB. 
TP0001 is a completed, Phase 2, multicenter, open-label, multiple-arm study to evaluate the 
safety, tolerability, and efficacy of rozanolixizumab in subjects with primary persistent or 
chronic ITP. The following dose arms were used in the study: 
 Dose Arm 1 (15 subjects): rozanolixizumab 
 
 Dose Arm 2 (15 subjects): rozanolixizumab 
 
 Dose Arm 3 (12 subjects): rozanolixizumab 
 
 Dose Arm 4 (12 subjects): rozanolixizumab 
 
 Dose Arm 5 (12 subjects): rozanolixizumab 
  
 
 
dose was well-tolerated, and a summary of the safety results is included in the IB. However, it 
should be noted that the exposure of rozanolixizumab was lower in subjects with ITP than 
subjects with MG when dosed with the same dose level. 
The dose-exposure-response relationship, with total IgG as the primary endpoint, was 
determined using non-linear mixed effects modeling of the data from UP0018. The derived 
population PK-PD (structural PK-PD model based on that of Lowe [Lowe et al, 2010]) was then 
used to guide, through simulation, the selection of appropriate repeat dose regimens that would 
mimic decreases achieved by plasmapheresis paradigms and result in an IgG reduction of 70% or 
greater. The model-based simulations demonstrate that the chosen dose regimen of 
 sc 
administered weekly for a total of 12 doses are expected to produce maximum mean IgG 
reductions of >70% over the Treatment Period. Based on simulations, it is expected that these 
IgG reductions will be reached rapidly and maintained consistently with the selected dosing 
regimen.  
This study will utilize the same liquid formulation at 
 as was used in the first in 
human study. In the first in human study, individual dose was achieved by diluting the 
formulation prior to administration. Based on the heaviest subject (81kg) at the highest sc dose 
), the maximum concentration given was 
. This change in concentration is 
not predicted to have an impact on local tolerability, however to allow this to be fully assessed 
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the first two infusions in each subject will be given at a slower rate of approximately 
. If 
no tolerability concerns are identified subsequent infusions may be given at rates up to 
. 
5.4.5 
Justification for additional genomic analysis 
CIDP is a significantly disabling disorder. However, evidence for genetic predisposition to 
disease or to response to therapy is limited. No genome wide association studies have been 
completed and unusually for an immune mediated disease, no clear human leukocyte antigen 
association demonstrated. Although reports of genetic findings associated with disease or 
response to therapy are scarce, the role of undiscovered gene variants in pathways relating to 
immune system biology or peripheral nerve function cannot be ruled out. As genome wide 
analysis of CIDP patients becomes feasible and affordable, a clearer understanding of the role of 
genetic and genomic factors in CIDP biology is likely to be forthcoming. 
Through the collection of whole blood, DNA analysis will help facilitate identification and 
characterization of genetic and/or epigenetic components of CIDP and will lead to important 
clues into the pathogenesis of disease and possibly advance understanding of drug response 
phenotypes. 
Gene transcription signatures associated with CIDP biology and response of CIDP patients to 
IVIg therapy have been generated from whole blood messenger RNA (mRNA) microarray 
analysis. Such profiles may provide molecular insight into disease biology and activity and can 
facilitate patient stratification via gene expression panels predictive of therapeutic response and 
clinical outcomes. 
MicroRNAs (miRNAs) are short (19-25 nucleotides) evolutionarily conserved single-stranded 
RNA molecules that regulate the expression of genes involved in diverse biological processes. 
The effect of miRNA on mRNA is mediated through the binding of the miRNA to the target 
mRNA ribonucleoprotein complex resulting in altered expression and decreased protein 
translation. 
Collection of blood for analysis of RNA, proteins, and metabolites will facilitate insight into the 
molecular etiology of CIDP at the genomic level and may enable identification of candidate 
markers for treatment effect and safety, and assessment of the feasibility of patient stratification. 
In summary, the DNA, RNA, proteins, and metabolites elements of CIDP require further 
elucidation to understand the cause, progression, and appropriate treatment of CIDP. Through 
the collection of blood DNA, RNA, serum, and plasma samples, these analyses will help enable 
further investigation of this complex disease. 
6 
SELECTION AND WITHDRAWAL OF SUBJECTS 
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 is signed and dated by the subject. 
2. The subject is considered reliable and capable of adhering to the protocol visit schedule, or 
medication intake according to the judgment of the investigator. 
3. Subject is ≥18 years of age at Visit 1 (Screening). 
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4. Subject has a documented definite or probable diagnosis of CIDP according to the 
EFNS/PNS criteria 2010. 
5. Subject has an Ig-dependency confirmed by clinical examination during therapy or upon 
interruption or reduction of therapy within 18 months prior to Screening and documented in 
medical history (ie, that a decrease or withdrawal of immunoglobulin was attempted that 
resulted in a clinically relevant decrease in function). 
6. Subject is on a stable dosage (not more than ±20% deviation) for SCIg or IVIg and a fixed 
interval for at least 4 months of either treatment, eg, once weekly ±2 days for SCIg or every 
2 to 6 weeks ±5 days IVIg, respectively, for stability in functioning between dosing. 
7. Female subjects of childbearing potential must have a negative serum pregnancy test at the 
Screening Visit, which is confirmed to be negative by urine testing prior to the first dose of 
IMP at Week 1 (Visit 2) 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 which 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], 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], 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), 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 
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8. Male subjects 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 subject 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. 
6.2 
Exclusion criteria 
6.2.1 
Exclusion criteria related to study participation 
Subjects are not permitted to enroll in the study if any of the following criteria are met: 
1. Subject has previously received treatment in this study or subject has previously been 
exposed to rozanolixizumab. 
2. Subject has participated in another study of an IMP (or a medical device) within the previous 
30 days of Screening Visit or is currently participating in another study of an IMP (or a 
medical device). For experimental biological agents refer to Exclusion Criterion 26. 
6.2.2 
Exclusion criteria related to CIDP diagnosis 
3. Subject has a current diagnosis or has a history of Type 1 or Type 2 diabetes mellitus and/or 
hemoglobin A1c level >6.0%. 
4. Subject with IgM paraproteinemia. 
5. Subjects with known IgM-mediated neuropathy (eg, multifocal motor neuropathy). 
6. 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). 
7. Subjects on an average dose less than 0.4g IgG/kg/month over the past 4 months. 
6.2.3 
Exclusion criteria related to health status/safety of the subject 
8. Female subject who is pregnant or lactating. 
9. Subject has any medical (acute or chronic illness) or psychiatric condition that, in the opinion 
of the investigator, could harm the subject or would compromise the subject's ability to 
participate in this study. 
10. Subject has 12-lead ECG with abnormalities considered to be clinically significant upon 
medical review. 
11. Subject has renal impairment, defined as: 
 Serum creatinine level of ≥1.4mg/dL for females and ≥1.5mg/dL for males at Screening 
Visit. 
12. Subject has an absolute neutrophil count <1500/mm3 (1.5x109/L). 
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13. Subject 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 subject 
has elevations only in total bilirubin that are >ULN and <1.5xULN, fractionate bilirubin to 
identify possible undiagnosed Gilbert’s syndrome (ie, direct bilirubin <35%). 
For randomized subjects 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 subject has >ULN ALT, AST, or ALP that does not meet the exclusion limit at Screening, 
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 subject 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 includes rescreening. 
14. 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. 
15. 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 HIV (anti-HIV1 or anti-HIV2 antibodies), hepatitis B (HBsAg positive or HBcAb 
positive without positive HBsAb), or hepatitis C antibody (HCAb) at the Screening Visit. 
16. Subjects with known TB infection, at high risk of acquiring TB infection, or latent 
tuberculosis infection (LTBI), or current/history of nontuberculosis mycobacteria (NTMB) 
are excluded. 
a. Known TB infection whether present or past is defined as: 
 Active TB infection or clinical signs and symptoms suggestive of TB (pulmonary or 
extrapulmonary). 
 History of active TB infection involving any organ system or findings in other organ 
systems consistent with TB infection. 
 Any historical evidence by radiography or other imaging modalities consistent with 
previously active TB infection.   
b. High risk of acquiring TB infection is defined as: 
 Known exposure to another person with active TB infection within the 3 months prior to 
Screening. 
 Time spent in a healthcare delivery setting or institution where individuals infected with 
TB are housed and where the risk of transmission of infection is high. 
c. LTBI (see Section 12.3 for further details and instructions). 
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d. NTMB is defined as a group of lung infections caused by mycobacteria different from 
mycobacterium tuberculosis infections. 
17. Subject has a family history of primary immunodeficiency. 
18. Subject 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 6 weeks prior to the first dose of IMP. 
19. Subject 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 which has been definitively treated with SOC approaches). 
20. Subject 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 
“yes” answer to numbers 4 or 5 on the screening Columbia Suicide Severity Rating Scale 
(C-SSRS). 
21. Subject 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. 
22. Subject has a history of known inflammatory bowel disease, active diverticular disease, or a 
history of confirmed duodenal, gastric, or esophageal ulceration in the past 6 months. 
6.2.4 
Exclusion criteria related to the IMP/concomitant 
medications/procedures 
23. Subject has a known hypersensitivity to any components of the IMP. 
24. Subject has a history of hyperprolinemia, since L-proline is a constituent of rozanolixizumab. 
25. Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends 
to have a live vaccination during the course of the study or within 7 weeks following the final 
dose of IMP. 
26. Subject has received any experimental biological agent 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). 
27. Subject has had prior treatment with rituximab, ofatumumab, or ocrelizumab in the 6 months 
prior to the Baseline Visit or subject 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. 
28. Subject has been treated with immunosuppressants, biologics, 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 Randomization Visit (Visit 2) for 
immunosuppressants, biologics and other therapies 
Generic name 
Time restrictions required prior to Randomization 
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; mabs=monocolonal antibodies; 
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 subjects 
For subjects, otherwise fully eligible but not able to be randomized 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 subject has 1 isolated test result in the exclusionary range which 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 
Subjects are free to withdraw from the study at any time, without prejudice to their continued 
care. 
Subjects MUST be withdrawn from the study if any of the following events occur: 
1. Subject withdraws his/her consent. 
2. Subject becomes pregnant during the study, as confirmed by a positive pregnancy test. 
Subjects must discontinue IMP if any of the following events occur: 
1. Subject develops an illness that would interfere with his/her continued participation. 
 Subject has a serious infective episode requiring hospitalization or iv antibiotic therapy 
(including but not limited to bacteremia or sepsis, bacterial meningitis, osteomyelitis or 
septic arthritis, bacterial pneumonia, or visceral abscess). 
 Subject has an AE of severe infusion reaction requiring corticosteroid and/or epinephrine 
therapy (see Section 12.1.9). 
 Subject has an AE of severe anaphylactic reaction requiring corticosteroid and/or epinephrine 
therapy. 
 Subject 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 12.3.7 for further details and instructions). 
 If a nontuberculosis mycobacterium infection (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. Subject 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 subject 
should be referred immediately to a Mental Healthcare Professional. 
3. The sponsor or a regulatory agency requests withdrawal of the subject. 
4. Subject needs or takes PLEX, dexamethasone or rituximab. 
5. Subject is treated with rescue medication and/or relapses during the Treatment Period (refer 
to Section 7.7.3). 
Subjects may be discontinued from IMP at the discretion of the investigator, Medical Monitor, 
and Study Physician if any of the following events occur: 
1. Subject takes prohibited concomitant medications during the Treatment Period as defined in 
this protocol (refer to Section 7.7.2). 
2. Subject experiences a severe AE of headache which is considered related to the IMP in the 
opinion of the investigator (see Section 12.1.10). 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 
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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 to 
(or its placebo equivalent) if the headache persists despite symptomatic treatment 
(refer to Section 12.1.10 for details on the medical management of headaches and Section 7.2 
for treatment to be administered). 
3. Subject experiences severe AE of gastrointestinal disturbance which is considered related to 
the IMP in the opinion of the investigator. Following an event of a severe gastrointestinal 
disturbance, a subject may continue in the study if they are willing to do so and the 
investigator and Medical Monitor agree that the subject’s continuation in the study poses no 
significant risk 
Subjects 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. Subject 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 subject 
should discontinue IMP. 
Subjects who withdraw from the study or discontinue IMP during the Treatment Period should 
complete the assessments outlined for Visit 17/PEOT (see Table 5‒1 and Section 8.4). Subjects 
who withdraw from the study and who are not entering the OLE will be encouraged to return to 
the clinic for all visits during the Observation Period (which should occur every 2 or 3 weeks 
(±3 days) following their final dose of IMP) and the Final Visit (scheduled 12 weeks after final 
dose of IMP). The subjects can return to their SOC during the Observation Period. 
Investigators should attempt to obtain information on subjects in the case of withdrawal. 
For subjects considered as lost to follow up, the investigator should try (at least 1 phone call and 
1 written message to the subject), 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 subject, must be recorded in the source documents. The eCRF must 
document the primary reason for withdrawal or discontinuation, plus whether or not the blind 
was broken, with the reason and date for withdrawal, discontinuation, and/or unblinding. 
Investigators should contact the Medical Monitor, whenever possible, to discuss the withdrawal 
of a subject in advance. 
Subjects who withdraw from the study will not be replaced. 
6.4.1 
Potential drug-induced liver injury IMP discontinuation criteria 
Subjects 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. 
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The PDILI criteria below require immediate and permanent discontinuation of IMP: 
 Subjects 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: 
 Subjects 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 subjects may resume IMP 
administration after discussion with the responsible UCB physician, but only when the 
requirements for rechallenge with IMP as provided in Section 12.2.1.2.1 are followed. 
The PDILI criterion below allows for subjects to continue on IMP at the discretion of the 
investigator. 
 Subjects 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 12.2.1. If subjects are unable to 
comply with the applicable monitoring schedule, IMP must be discontinued immediately. 
Investigators should attempt to obtain information on subjects in the case of IMP discontinuation 
to complete the final evaluation. Subjects 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 subject withdrawal (if 
applicable), must be recorded in the source documents. The eCRF must document the primary 
reason for IMP discontinuation. 
7 
STUDY TREATMENTS 
7.1 
Description of investigational medicinal products 
 
 
 
 
 for 
sc administration is supplied from a commercial source. 
Details of the IMPs and their specifications are provided in the IMP Handling Manual. 
7.2 
Treatments to be administered 
Eligible subjects will be randomized 1:1 to receive rozanolixizumab 
or placebo 
) by sc infusion as follows:  
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 Subjects in Treatment Arm 1 will receive 1 sc dose of rozanolixizumab 
 (see 
Table 7-1) weekly for 12 weeks (Visits 2, 4, and 6, and Visit 8 through Visit 16). 
 Subjects in Treatment Arm 2 will receive 1 sc dose of placebo weekly for 12 weeks (Visits 2, 
4, and 6, and Visit 8 through Visit 16). 
The interval between 2 consecutive dosing should at least 5 days. 
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 
3mL 
≥49 to <63kg 
4mL 
≥63 to <77kg 
5mL 
≥77 to <91kg 
6mL 
≥91 to <105kg 
7mL 
≥105 to <119kg 
8mL 
≥119 to <133kg 
9mL 
≥133 to <147kg 
10mL 
≥147 to <161kg 
11mL 
≥161 to 170kg 
12mL 
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.  
In case of tolerability issues (eg, severe headache) and if symptomatic headache medication 
(eg, acetylsalicylic acid 1000mg) is not sufficient, the dose of IMP could be lowered to 
 
(see Table 7-2) (or equivalent placebo) during the next visit when the investigator, Medical 
Monitor, and Study Physician agree that the dose reduction poses no significant risk for the 
subject (see Section 12.1.10).  
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 
2mL 
≥49 to <69kg 
3mL 
≥69 to <89kg 
4mL 
≥89 to <109kg 
5mL 
≥109 to <129kg 
6mL 
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Table 7-2: 
IMP doses to be administered (equivalent to approximately 7mg/kg) 
by body weight 
Body weight 
ranges 
IMP doses to be administered (equivalent to 
approximately 
 
IMP volume to be 
administered 
≥129 to <149kg 
7mL 
≥149 to <169kg 
8mL 
≥169 to 170kg 
9mL 
IMP= investigational medicinal product  
The IMP will be administered at the clinic or in a home setting (Visits 9, 11, 13, and 15) (see 
Table 5‒1). Home administration is subject to a set of conditions (see Section 8.2) to ensure 
subject safety. 
The IMP will be administered as a sc infusion using a syringe pump. 
 For the first 2 doses, 
 
 
. 
 For the subsequent doses, 
 
 
 
 
 
 
 
. The chronology of all infusion related events should be recorded 
accurately in the source data and eCRF. 
The subject’s body weight at Screening Visit will be used for the dose calculation. The dose will 
be constant for a subject throughout the duration of the study, and will not be adjusted during the 
treatment. 
The exact procedure for dose preparation according to treatment arm and body weight will be 
provided in the IMP Handling Manual. 
7.3 
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. 
7.4 
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. 
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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 (ie, every workday), 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. 
In case of home dosing, the IMP will be transferred to the subject's home. The related process 
will also be described in the IMP Handling Manual. 
7.5 
Drug accountability 
A Drug Accountability form will be used to record IMP dispensing and return information on a 
by-subject basis and will serve as source documentation during the course of the study. Details of 
any IMP lost, damaged (due to breakage or wastage), not used, partially used, disposed of at the 
study site, or returned to the sponsor or designee must also be recorded on the appropriate forms. 
All supplies and pharmacy documentation must be made available throughout the study for UCB 
(or designee) to review. 
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. 
7.6 
Procedures for monitoring subject compliance 
Subject 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. 
7.7 
Concomitant medications/treatments 
7.7.1 
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 treatments 
Permitted medications 
Dose 
Comment 
Oral corticosteroids 
(prednisolone) 
≤30mg/day 
Methotrexate 
≤30mg/week 
Mycophenolate mofetil 
≤3g/day 
Cyclosporin a 
≤5mg/kg/day for unmodified 
≤4mg/kg/day for modified 
(microemulsion) 
Azathioprine 
≤3mg/kg/day 
Tacrolimus b 
≤5mg/day 
 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 subject is not 
above the recommended therapeutic range. 
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 
. 
7.7.2 
Prohibited concomitant treatments (medications and therapies) 
The following medications are prohibited during the Treatment Period: 
 IVIg and SCIg (refer to Section 7.7.3) 
 All biologics including rituximab 
 Cyclophosphamide 
 Pimecrolimus 
 IPP-201101 (Lupuzor™) 
 PLEX 
 IA 
 Systemic dexamethasone 
If a subject 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 subject should discontinue IMP (see 
Section 6.4). 
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If a subject is discontinued from IMP they should be encouraged to complete Visit 17/PEOT and 
complete the Observation Period (see Table 5‒1 and Section 6.4). 
7.7.3 
Rescue medication 
If, at any time during the Treatment and Observation Period, the subject relapses according to the 
predefined criteria for relapse as specified in Section 4.1.2 using the subject’s score on iRODS, 
INCAT, or maximum grip strength (assessed by site personnel), and supported by the medical 
judgement of the investigator, then rescue medication must be considered and the subject will be 
withdrawn from IMP (see Section 6.4). The subject will return to the SOC Ig treatment (eg, IVIg 
of 2g/kg) as rescue medication at the time of relapse at the discretion of the investigator. The 
subject will have the opportunity to be stabilized over a period of 2 weeks to maximum 12 weeks 
starting from the relapse visit (ie, PEOT or later visit; see Section 8.3). Once stabilized, the 
subject who completed the Treatment Period with no CIDP relapse, will be offered the 
possibility to enter in the OLE (CIDP04) and be treated with rozanolixizumab from that moment 
onward. The subject will complete the FV (V21) and enter the OLE on the same day. If a subject 
relapses during Treatment Period or does not wish to enter the OLE, the subject will continue 
their SOC treatment after stabilization. They will continue the stabilization visit schedule (every 
1 to 3 weeks) until they have a follow-up of 12 weeks after last IMP dose. 
7.8 
Blinding 
7.8.1 
Procedures for maintaining and breaking the treatment blind 
7.8.1.1 
Maintenance of study treatment blind 
 
 
 
All subject treatment details, ie, rozanolixizumab 
 sc or placebo, will be allocated and 
maintained by the interactive response technology (IRT) system. 
Study site pharmacists or other suitably qualified site personnel who are responsible for 
preparation of IMP treatments and any necessary assistants will have access to treatment 
allocations for individual subjects via the IRT. The unblinded pharmacy monitors from the 
contract research organization (CRO), and the Clinical Supply Manager will also have access to 
the treatment allocations and to the drug accountability records, if applicable. 
Laboratory results that could unblind the site personnel will not be disclosed to sites and will be 
reviewed accredited unblinded study team. 
The following individuals will, as necessary, have access to the randomization code as indicated: 
 Sponsor Drug Safety staff as needed for reporting SAEs to regulatory authorities. 
 Sponsor and/or CRO staff supporting preparation of the data outputs for the DMC review 
and/or any interim efficacy analyses. 
 The laboratories involved in the detection of PK, PD, and immunological variables. 
 A Quantitative Clinical Pharmacologist/Modeling and Simulation Scientist. 
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7.8.1.2 
Breaking the treatment blind in an emergency situation 
In the event of an emergency, it will be possible to determine to which treatment arm and dose 
the subject has been allocated by contacting the IRT. All sites will be provided with details of 
how to contact the system for code breaking at the start of the study. The Medical Monitor or 
equivalent should be consulted prior to unblinding, whenever possible. The study blind should 
not be broken except in a medical emergency, where knowledge of the IMP received would 
affect the treatment of the emergency. 
The Clinical Project Manager (CPM) will be informed immediately via the IRT when a code is 
broken, but will remain blinded to specific treatment information. Any unblinding of the IMP 
performed by the investigator must be recorded in the source documents and on the Study 
Termination eCRF page. 
7.9 
Randomization and numbering of subjects 
An IRT will be used for assigning eligible subjects to a treatment regimen (as applicable) based 
on a predetermined production randomization and/or packaging schedule which will be produced 
by the IRT vendor. The randomization will be stratified according to previous route of 
administration for Ig treatment (ie, SCIg and IVIg). The IRT will generate individual 
assignments for subject kits of IMP, as appropriate, according to the visit schedule. 
To enroll a subject (Visit 1), the investigator or designee will contact the IRT and provide brief 
details about the subject to be enrolled. Each subject will receive a 5-digit number assigned at 
Screening that serves as the subject identifier throughout the study. The subject number will be 
required in all communication between the investigator or designee and the IRT regarding a 
particular subject. Subject numbers and kit numbers will be tracked via the IRT. 
To randomize a subject, the investigator or designee will contact the IRT and provide brief 
details about the subject to be randomized. The IRT will allocate kit numbers to the subject 
based on the subject number during the course of the study. Randomization numbers will be 
automatically transferred from the IRT to the clinical database. 
The randomization window is described in Section 8.2. 
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. All assessments are to be completed in the recommended order if 
possible. The Informed Consent form (ICF) should be completed before any assessment. The 
PROs should be conducted in the order specified in Section 9.5 immediately after ICF (where 
applicable). The laboratory manual will provide further guidance on the order of sample 
collection. Additional assessments which 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 Section 8. 
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8.1 
Screening Period (Day -35 to Day -1) 
The Screening Period will be a minimum of 2 weeks and generally should not exceed 5 weeks. 
The purpose of the Screening Period is to evaluate and confirm the subject’s eligibility. All 
subjects must have an Ig dependency that has been confirmed within 18 months before 
Screening. All subjects must be established on a long-term immunoglobulin maintenance therapy 
with a stable immunoglobulin infusion regimen at consistent intervals to be considered eligible. 
Stable dose regimen is defined as subjects remaining on the same dose (not more than ±20% 
deviation), and fixed treatment interval for at least 4 months, eg, receiving treatment once 
weekly (±2 days) or every 2 to 6 weeks (±5 days) for SCIg or IVIg, respectively, for stability in 
functioning between dosing. 
The subject’s diagnosis, disability, impairment, and past treatments including documentation of 
dependency on immunoglobulin treatment, current treatment with immunoglobulin at a 
consistent dosage regimen, and other previous and concomitant medications and treatments will 
be evaluated at the Screening Visit. 
8.1.1 
Screening Visit 1 (Day -35 to Day -14) 
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 subject 
 iRODS assessment – to be performed before any other assessment after the consenting 
process 
 Query for suicidality (C-SSRS) 
 Tuberculosis Signs and Symptoms questionnaire 
 Obtain a chest radiograph, unless one has been obtained 3 months prior to the Screening Visit 
 Record subject demographics 
 Verify (to the extent possible with available Screening Visit 1 information) that subject 
fulfills all the inclusion criteria and none of the exclusion criteria 
 General medical and procedures history 
 Prior medication 
 Concomitant medical procedures 
 CIDP history (symptoms, date of onset, therapy) 
 Vital signs (taken once during the visit) 
 Body weight (Screening weight will be used for calculation of the dose) and height 
 12-lead ECG 
 Full physical examination 
 Full neurological examination 
 Recording of AEs 
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 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, human immunodeficiency virus 1 (HIV1) and human 
immunodeficiency virus 2 antibodies (HIV2)], and interferon gamma release assay 
[IGRA] for active and latent tuberculosis [LTB]) 
 Serum pregnancy test for women of childbearing potential 
 Immunoglobulins (total IgG, IgG subclasses) 
 Vaccination-specific antibody titers (
 
 
 
 Urine sample for urinalysis 
 INCAT assessment 
 RT-MRC assessment 
 Assessment of grip strength by the site personnel 
 Issue subject e-diary for daily record of grip strength 
 Provide an appointment to the next visits (see Section 8.2 for details related to scheduling of 
the next visit) 
At the end of the Screening Visit, subjects will be issued a subject e-diary in which they will 
record daily grip strength from the Screening Visit until the end of study participation. Subjects 
should be reminded to bring their diaries with them to each clinic visit and to have them 
available at home nursing visits. 
8.2 
Treatment Period Visits 2 to 16 (Day 1 to Day 78) 
The 11-week Treatment Period begins at the Randomization Visit (Visit 2) on Day 1 to review 
inclusion and exclusion criteria as well as concomitant medications and treatments. Subjects who 
remain eligible will be randomized 1:1 to receive rozanolixizumab 
 or placebo. All 
eligible subjects will receive the same treatment allocation for the duration of the study. 
 For subjects on an IVIg 3- to 6-week regimen, randomization 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 Randomization Visit could be conducted up to the date of the next 
planned IVIg dose (ie, 1 week after the preferred date). 
 Subjects on a 2-week IVIg treatment regimen will continue on their regular schedule for 
1 further IVIg treatment until randomization. Randomization will occur 1 week before the 
planned IVIg dose. However, if that specific date is not possible, the Randomization Visit 
could be conducted up to the date of the next planned IVIg dose (ie, 1 week after the 
preferred date). 
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 Subjects on a weekly SCIg treatment regimen will continue on their regular schedule for at 
least 1 further SCIg treatment until randomization. Randomization should occur on the day of 
a planned dose (according to the baseline SCIg regimen). However, if that specific date is not 
possible, the Randomization Visit should be within a window ±2 days of that date. 
This will ensure continuation of treatment so that subjects do not relapse prior to start of study 
treatment. 
For the 11-week Treatment Period, visits will occur weekly from Visit 2 to Visit 16. 
Three additional visits (Visits 3, 5, and 7) will be performed 1 or 2 days after each of the first 
3 IMP administrations to monitor safety and collect samples for PK/PD. 
At the Randomization Visit (Visit 2), and at Visit 4, Visit 6, and Visit 8 through Visit 16, 
subjects will receive IMP by a sc infusion. Other study-related assessments and procedures will 
be conducted as described in the following sections. For site Visits 2, 4, 6, 8, 10, 12 and 16, 
subjects will be required to remain in the clinic for at least 4 hours following the end of the sc 
infusion to monitor the safety of the subject and collect appropriate blood samples. Subjects may 
leave the clinic thereafter provided the investigator has no safety concerns. For all other visits, 
the visit length should be limited to 2 hours post dose if the investigator has no safety concerns. 
Visit duration may be extended in case of safety concerns (see Table 5-2). 
Visits 3, 5, and 7 are nondosing, safety monitoring visits in which the subject will be telephoned 
by the site personnel at home. Samples for PK sampling (Visits 3, 5, and 7) and Igs (Visit 7) will 
be performed as part of the home nursing visits (ie, a healthcare professional visiting the subject 
at his/her home). Alternately, Visits 3, 5, and 7 can be conducted at the site as deemed necessary 
by site personnel and/or the subject. 
Dosing Visits 9, 11, 13, and 15 are home nursing visits to be conducted by fully trained 
healthcare professionals (one unblinded for IMP preparation and one blinded for the other 
assessments) visiting the subject at his/her home. Alternatively, these visits can be conducted at 
the site as deemed necessary by site personnel and/or subject. 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 subject is willing to be dosed and monitored at home over a 2-hour period by a home 
nurse. 
 The subject has shown good acute tolerability to previous administrations of IMP (namely 
they must have had no moderate or severe infusion reactions, or other AEs which the 
investigator considers could increase the risk of home administration). 
 The subject does not require specific medical supervision based on their 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). 
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 The subject’s home allows rapid access to emergency treatment if required (ie, the subject 
must not live so remotely that a reasonable arrival time of an ambulance could not be 
predicted). 
 The investigator is contactable to support the health care provider (HCP) if needed. 
 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. This checklist will be shared with the UCB study physician and reviewed before IMP 
administration in a home setting can take place. 
If at any time during the Treatment Period, the subject relapses according to the predefined 
criteria for relapse as specified in Section 4.1.2 using the subject’s score on iRODS, INCAT, or 
maximum grip strength (assessed by site personnel), and supported by the medical judgement of 
the investigator, then rescue medication must be considered and the subject must be withdrawn 
from IMP (see Section 6.4 and Section 7.7.3). The subject will return to the SOC (ie, Ig 
treatment) as rescue medication at the time of relapse and will be stabilized over a period of 2 
weeks to maximum 12 weeks starting from PEOT (Visit 17). At the end of the stabilization, the 
subject will continue SOC. The time required for follow-up since last IMP dose is 12 weeks. The 
stabilization visit schedule still applies (every 1 to 3 weeks) if the subject has not reached 12 
weeks after the last IMP dose at the end of stabilization. The subjects in the placebo arm will be 
offered the possibility to enter in the OLE and be treated with rozanolixizumab once all subjects 
have completed the CIDP01 study, the study is unblinded, and key results are available. 
8.2.1 
Visit 2 (Day 1, Baseline) 
The following procedures and assessments will be performed at Visit 2 (Day 1, Baseline): 
 PROs should be conducted as the first assessments in the study in the following order: 
 iRODS assessment – to be performed before any other assessment 
 CIDP PRO instrument 
 Fatigue scale 
 PGIS 
 Query for suicidality (predose) 
 Verification of inclusion/exclusion criteria (including results of laboratory samples, ECG, 
chest x-ray performed since Visit 1). 
 Assessment of withdrawal criteria (see Section 6.4) 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Vital signs (taken predose, at the end of the infusion, at 2 hours and at 4 hours after the end 
of the infusion) 
 Body weight (predose) 
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 12-lead ECG (predose) 
 Recording of AEs 
 Brief physical examination (predose) 
 Brief neurological examination (predose) 
 Contact IRT and randomize the subject (upon confirmation of eligibility) 
 Administration of IMP 
 Urine sample for urinalysis (predose) 
 Urine pregnancy test for women of childbearing potential (which must be confirmed negative 
prior to subject 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: 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) 
 Clinical laboratory tests (ie, hematology, clinical chemistry) 
 Serum cytokines 
 Immunoglobulins (total IgG, IgG subclasses) 
 Neurofilament light chain 
 Exploratory biomarker samples 
 RNA samples 
 CIDP-specific auto-antibodies 
 IgA, IgM, IgE 
 Whole blood for exploratory genetic (DNA) 
 PK of rozanolixizumab (predose and 4 hours postdose) 
 
 
 INCAT assessment (predose) 
 RT-MRC assessment (predose) 
 Assessment of grip strength by site personnel (predose) 
 Review subject e-diary for daily assessment of grip strength 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
8.2.2 
Visit 3 Telephone Call and Home Visit (Day 3) 
Visit 3 consists of a telephone call from the site personnel and a home nursing visit performed 
after 1 or 2 days of the previous IMP administration. The home nursing visit will be conducted 
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by a healthcare professional visiting the subject at his/her home. Alternately, this visit can be 
conducted at the site as deemed necessary by site personnel and/or subject. 
The subject will be telephoned at home and the following assessments performed: 
 Subjects will be assessed to see if they meet any of the study withdrawal criteria 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Recording of AEs 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
At home, the following assessments will be performed: 
 Vital signs (taken once during the visit) 
 Blood sample for: 
 PK of rozanolixizumab 
8.2.3 
Visit 4 (Day 8) 
At Visit 4 the following assessments will be performed: 
 iRODS assessment– to be performed before any other assessment 
 Query for suicidality (predose) 
 Subjects will be assessed to see if they meet any of the study withdrawal criteria 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Vital signs (taken predose, at the end of the infusion, at 2 hours and at 4 hours 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 subject dosing) 
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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: 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) (predose and 4 hours 
postdose) 
 Clinical laboratory tests (ie, hematology, clinical chemistry) 
 Serum cytokines (predose and 4 hours postdose) 
 Immunoglobulins (total IgG, IgG subclasses) 
 Exploratory biomarker analysis 
 CIDP-specific auto-antibodies 
 IgA, IgM, IgE 
 RNA samples 
 PK of rozanolixizumab (predose and 4 hours postdose) 
 
 
 INCAT assessment (predose) 
 RT-MRC assessment (predose) 
 Assessment of grip strength by the site personnel (predose) 
 Review subject e-diary for daily assessment of grip strength 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
8.2.4 
Visit 5 Telephone Call and Home Visit (Day 10) 
Visit 5 is a telephone call from the site with a home nursing visit performed after 1 or 2 days of 
the previous IMP administration. The home nursing visit will be conducted by a healthcare 
professional visiting the subject at his/her home. Alternately, this visit can be conducted at the 
site as deemed necessary by site personnel and/or subject. 
The subject will be telephoned at home and the following assessments performed: 
 Subjects will be assessed to see if they meet any of the study withdrawal criteria 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Recording of AEs 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
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At home, the following assessments will be performed: 
 Vital signs (taken once during the visit) 
 Blood sample for: 
 PK of rozanolixizumab 
8.2.5 
Visit 6 (Day 15) 
At Visit 6 the following assessments will be performed: 
 iRODS assessment– to be performed before any other assessment 
 Query for suicidality (predose) 
 Assessment of study withdrawal criteria 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Vital signs (taken predose, at the end of the infusion, at 2 hours and at 4 hours 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 subject 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, clinical chemistry) 
 Immunoglobulins (total IgG, IgG subclasses) 
 Neurofilament light chain 
 CIDP-specific auto-antibodies 
 IgA, IgM, IgE 
 RNA samples 
 PK of rozanolixizumab (predose and 4 hours postdose) 
 
 
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 INCAT assessment (predose) 
 RT-MRC assessment (predose) 
 Assessment of grip strength by the site personnel (predose) 
 Review subject e-diary for daily assessment of grip strength 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
8.2.6 
Visit 7 Telephone Call and Home Visit (Day 17) 
Visit 7 is a telephone call from the site with a home nursing visit performed after 1 or 2 days of 
the previous IMP administration. The home nursing visit will be conducted by a healthcare 
professional visiting the subject at his/her home. Alternately, this visit can be conducted at the 
site as deemed necessary by site personnel and/or subject. 
The subject will be telephoned at home and the following assessments performed: 
 Subjects will be assessed to see if they meet any of the study withdrawal criteria 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Recording of AEs 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
At home, the following assessments will be performed: 
 Vital signs (taken once during the visit) 
 Blood sample for: 
 Immunoglobulins (total IgG, IgG subclasses) 
 PK of rozanolixizumab 
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8.2.7 
Visits 8 (Day 22) through 16 (Day 78) inclusive 
All visits and IMP administrations (during the Treatment Period) are performed at 1-week 
intervals. For site Visits 8, 10, 12 and 16, subjects will be required to remain in the clinic for at 
least 4 hours following the end of the sc infusion to monitor the safety of the subject and collect 
appropriate blood samples. Subjects may leave the clinic thereafter provided the investigator has 
no safety concerns. For all other visits, the visit length should be limited to 2 hours post dose if 
the investigator has no safety concerns. Visit duration may be extended in case of safety 
concerns (see Table 5-2). Dosing Visits 9, 11, 13, and 15 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. Home dosing will only be 
undertaken if no tolerability issues have been identified on previous dosing occasions in that 
subject (see full details in Section 8.2). 
The following assessments and procedures will be performed at each visit (unless specified 
otherwise): 
 PROs should be conducted as the first assessments in the study in the following order: 
 iRODS assessment– to be performed before any other assessment 
 CIDP PRO instrument (Visits 10 and 14 only) 
 Fatigue scale (Visits 10 and 14 only) 
 PGIS (Visits 10 and 14 only) 
 PGIC (Visits 10 and 14 only) 
 Query for suicidality (predose) (Visits 8, 10, 12, 14, and 16 only) 
 Subjects will be assessed to see if they meet any of the study withdrawal criteria 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Vital signs (taken predose, at the end of infusion, at 2 hours after infusion end) 
 Vital signs (at 4 hours after infusion end) (Visit 8 only) 
 12-lead ECG (Visits 8, 10, 12, 14, and 16 only) 
 Recording of AEs 
 Brief physical examination (Visits 8, 10, and 14 only) 
 Brief neurological examination (Visits 8, 10, and 14 only) 
 Contact IRT 
 Administration of IMP 
 Urine sample for urinalysis (predose) (Visits 8, 10, 12, 14, and 16 only) 
 Urine pregnancy test for women of childbearing potential (which must be confirmed negative 
prior to subject dosing) 
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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: 
 Serum complement (C3, C4) and plasma complement (C3a, C5a) (predose and 4 hours 
postdose) (Visits 8, 12, and 16 only) 
 Clinical laboratory tests (ie, hematology, clinical chemistry) (Visits 8, 10, 12, 14, and 16 
only) 
 Serum cytokines (predose and 4 hours postdose) (Visits 8, 12, and 16 only) 
 Immunoglobulins (total IgG, IgG subclasses) 
 Neurofilament light chain (Visit 16 only) 
 Exploratory biomarker analysis (Visits 8, 12, and 16 only) 
 CIDP-specific auto-antibodies (Visits 8, 12, and 16 only)  
 IgA, IgM, IgE (Visits 8, 10, 12, 14, and 16 only) 
 RNA samples (Visit 10 only) 
 PK of rozanolixizumab (predose and 4 hours postdose) (Visits 8 and 10 only) 
 PK of rozanolixizumab (predose) (Visits 12, 14, and 16 only) 
 Vaccination-specific antibody titers (
) (Visits 8 and 14 only) 
 
 (Visits 8, 10, 12, 14, and 16 only) 
 Serum pregnancy test for women of childbearing potential (Visit 16 only) 
 INCAT assessment (predose) (Visits 8, 10, 12, 14, and 16 only) 
 RT-MRC assessment (predose) (Visits 8, 10, 12, 14, and 16 only) 
 Assessment of grip strength by the site personnel (predose) (Visits 8, 10, 12, 14, and 16 only) 
 Review subject e-diary for daily assessment of grip strength (Visits 8, 10, 12, 14, and 16 
only) 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
8.3 
12-Week Observation Period Visits 17 to 21 (Day 85 to Day 162) 
Following the final dose of rozanolixizumab or placebo at Day 78 (Visit 16), 5 subsequent visits 
will be scheduled over 12 weeks (Visit 17 through Visit 21) to collect safety and efficacy data 
for study related outcome measures and procedures. Visit 18 and Visit 20 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. 
Visit 17 will be the last study visit for the subjects entering immediately in the OLE study upon 
completing all visits in the Treatment Period. 
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All subjects not entering the OLE immediately should perform all visits of the Observation 
Period, including the Final Visit (scheduled 12 weeks after the final dose of IMP). 
If at any time during the Observation Period, the subject relapses according to the predefined 
criteria for relapse as specified in Section 4.1.2 using the subject’s score on iRODS, INCAT, or 
maximum grip strength (assessed by site personnel), and supported by the medical judgement of 
the investigator, then rescue medication must be considered. The subject will return to the SOC 
(ie, Ig treatment) as rescue medication at the time of relapse and will be stabilized over a period 
ranging from 2 weeks to maximum 12 weeks starting from time of relapse. During the 
stabilization, visits should be performed every 1 to 3 weeks at the discretion of the investigator 
until the subject is stabilized. The stabilization may extend the Observation Period duration up to 
a maximum of 24 weeks after last IMP dose. At the end of the stabilization, the subjects who 
complete the Treatment Period without a CIDP relapse will be offered the possibility to enter in 
the OLE immediately and be treated with rozanolixizumab. Their last visit in the study will be 
the FV, scheduled at the end of stabilization and enter the OLE on the same day. Assessments 
performed at the FV will not be repeated in the OLE. 
If the subject does not wish to/cannot enter the OLE, the subject will continue treatment with 
SOC. The time required for follow-up since last IMP dose is 12 weeks. The stabilization visit 
schedule still applies (every 1 to 3 weeks) if the subject has not reached 12 weeks post last IMP 
dose at the end of stabilization. 
Table 8‒1 summarizes the options for the subject to proceed in the Observation Period. 
Table 8‒1: 
Management of Observation and Stabilization Periods 
Subject status 
Stabilization 
with rescue 
medication? 
Ability to 
enter OLE? 
End of study CIDP01 
Subject completes 
Treatment Period 
without CIDP 
relapse (all visits 
performed until 
Visit 17) 
NA 
yes 
 Last Visit=Visit 17=Entry visit in OLE 
(CIDP04)  
NA 
no 
If the subject wants to return immediately to SOC: 
 
Subject returns to treatment with SOC (eg, 
IVIG or SCIG) during Observation Period.  
 
Time of required follow-up since last IMP dose 
is 12 weeks.  
 Subject attends Visit 17 to Visit 21 
(FV=last visit). 
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Table 8‒1: 
Management of Observation and Stabilization Periods 
Subject status 
Stabilization 
with rescue 
medication? 
Ability to 
enter OLE? 
End of study CIDP01 
NA 
Maybe later 
If the subject wants to test whether he/she still needs 
treatment: 
 
Subject continues Observation Period without 
SOC until maximum of 12 weeks post last IMP 
dose. Subject attends Visit 17 to Visit 21 (FV).  
 
If the subject relapses and wants to enter the 
OLE, please refer to line ** 
Subject relapses 
during Treatment 
Period (before 
Visit 17) 
yes 
Maybe later 
 
Subject attends PEOT Visit. 
 
IMP is stopped. 
 
Conduct stabilization for a minimum of 2 weeks 
and a maximum of 12 weeks with rescue 
medication. Visits are scheduled every 1 to 3 
weeks. 
 
Subject continues SOC after stabilization. Time 
of required follow-up since last IMP 
administration is 12 weeks. The stabilization 
visit schedule still applies (every 1 to 3 weeks). 
 
FV (V21)=Last visit after stabilization visits. 
 
Entry in OLE (CIDP04) for subjects from the 
placebo arm once all subjects have completed 
the study, unblinding is performed, and key 
results are available. 
yes 
no 
 
Subject attends PEOT Visit. 
 
IMP is stopped. 
 
Conduct stabilization for a minimum of 2 weeks 
and a maximum of 12 weeks with rescue 
medication. Visits are scheduled every 1 to 
3 weeks. 
 
Subject continues SOC after stabilization. Time 
of required follow-up since last IMP dose is 12 
weeks. The stabilization visit schedule still 
applies (every 1 to 3 weeks). 
 
FV (V21)= Last visit after stabilization visits. 
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Table 8‒1: 
Management of Observation and Stabilization Periods 
Subject status 
Stabilization 
with rescue 
medication? 
Ability to 
enter OLE? 
End of study CIDP01 
** 
CIDP relapse 
during 
Observation 
Period (after Visit 
17) for subjects 
who completed 
Treatment Period 
yes 
yes 
 
Subject starts stabilization for minimum of 
2 weeks and maximum 12 weeks with rescue 
medication. Visits are scheduled every 1 to 
3 weeks. 
 
If relapse happens towards the end of the 
12-week Observation Period, overall timeframe 
(ie, since PEOT, including IMP free and 
stabilization period) until entry in OLE can be 
up to 24 weeks from last IMP dose.  
 
FV (V21)=Last visit after stabilization 
visits=Entry visit in OLE (CIDP04). 
yes 
no 
 
Subject starts stabilization for a minimum of 
2 weeks and a maximum of 12 weeks with 
rescue medication. Visits are scheduled every 1 
to 3 weeks. 
 
Subject continues SOC after stabilization. Time 
of required follow-up since last IMP dose is 12 
weeks. Stabilization visit schedule still applies 
(every 1 to 3 weeks). 
 
FV (V21)=Last visit after stabilization visits. 
Withdrawal for 
other reasons than 
CIDP relapse 
(before Visit 17) 
no 
no 
 
Subject attends PEOT Visit 
 
IMP is stopped 
 
Subject returns to treatment with SOC (eg, IVIg 
or SCIg) during Observation Period 
 
Time of required follow-up since last IMP dose 
is 12 weeks  
 
Subject attends Visit 17 to Visit 21 (FV=Final 
Visit) 
CIDP=chronic inflammatory demyelinating polyradiculoneuropathy; FV=Final Visit; ICF=informed consent form; 
IMP=investigational medicinal product; IVIg=intravenous immunoglobulin; OLE=Open-Label Extension; 
PEOT=premature end of treatment; SCIg=subcutaneous immunoglobulin; SOC=standard of care 
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8.3.1 
Visits 17 (Day 85) through 20 (Day 141) inclusive 
The following assessments and procedures will be performed at each visit of the Observation 
Period (unless specified otherwise): 
 PROs should be conducted as the first assessments in the study in the following order: 
 iRODS assessment– to be performed before any other assessment 
 CIDP PRO instrument (Visit 17 only) 
 Fatigue scale (Visit 17 only) 
 PGIS (Visit 17 only) 
 PGIC (Visit 17 only) 
 Query for suicidality 
 Assessment of the study withdrawal criteria 
 Tuberculosis Signs and Symptoms questionnaire (Visit 17 only) 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Vital signs (taken once during each visit) 
 Body weight (Visit 17 only) 
 12-lead ECG (Visit 17 only) 
 Recording of AEs 
 Brief physical examination (Visit 19 only) 
 Full neurological examination (Visit 17 only) 
 Brief neurological examination (Visit 19 only) 
 Full physical examination (Visit 17 only) 
 Contact IRT (Visit 17 only) 
 Urine sample for urinalysis (Visits 17 and 19 only) 
 Urine pregnancy test 
 Blood sample (the laboratory manual will provide guidance on the order of sample 
collection) for: 
 Clinical laboratory tests (ie, hematology, clinical chemistry) (Visits 17 and 19 only) 
 Immunoglobulins (total IgG, IgG subclasses) (Visit 17 only) 
 CIDP-specific auto-antibodies (Visit 17 only) 
 IgA, IgM, IgE (Visit 17 only) 
 Whole blood for exploratory genetic (DNA) (Visit 17 only) 
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 RNA samples (Visit 17 only) 
 PK of rozanolixizumab (Visit 17 only) 
 
(Visit 17 only) 
 INCAT assessment 
 RT-MRC assessment (Visit 17 only) 
 Assessment of grip strength by site personnel (Visit 17 and Visit 19 only) 
 Review subject e-diary for daily assessment of grip strength 
 Subject exit interview (Visit 17 only for subjects entering in OLE on completion of 
Treatment Period) 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
8.3.2 
Visit 21 (Day 162) Final Visit 
The following assessments and procedures will be performed at the Final Visit (Visit 21): 
 PROs should be conducted as the first assessments in the study in the following order: 
 iRODS assessment – to be performed before any other assessment 
 CIDP PRO instrument 
 Fatigue scale 
 PGIS 
 PGIC 
 Query for suicidality 
 Tuberculosis Signs and Symptoms questionnaire 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Vital signs (taken once during the visit) 
 Body weight 
 12-lead ECG 
 Recording of AEs 
 Full physical examination 
 Full neurological examination 
 Contact IRT 
 Urine sample for urinalysis 
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 Urine pregnancy test 
 Blood sample (the laboratory manual will provide guidance on the order of sample 
collection) for: 
 Immunoglobulins (total IgG, IgG subclasses) 
 Neurofilament light chain 
 IGA, IgM, IgE 
 Clinical laboratory tests (ie, hematology, clinical chemistry) 
 
 
 Vaccination-specific antibody titers (
 
 INCAT assessment 
 RT-MRC assessment 
 Assessment of grip strength by site personnel 
 Review subject e-diary for daily assessment of grip strength 
 Collect subject's daily grip strength e-diary 
 Subject exit interview 
8.3.3 
Stabilization Visits 
During the stabilization, visits should be performed every 1 to 3 weeks at the discretion of the 
investigator until the subject is stabilized. 
The following assessments and procedures will be performed at each visit of the Observation 
Period (unless specified otherwise): 
 PROs should be conducted as the first assessments in the study in the following order: 
 iRODS assessment– to be performed before any other assessment 
 Query for suicidality 
 Assessment of the study withdrawal criteria 
 Prior and concomitant medication 
 Concomitant medical procedures 
 Vital signs (taken once during each visit) 
 Recording of AEs 
 Brief physical examination 
 Brief neurological examination 
 Urine sample for urinalysis 
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 Urine pregnancy test 
 Blood sample (every 4 to 6 weeks only) (the laboratory manual will provide guidance on the 
order of sample collection) for: 
 Clinical laboratory tests (ie, hematology, clinical chemistry) 
 INCAT assessment 
 Assessment of grip strength by site personnel 
 Review subject e-diary for daily assessment of grip strength 
Subjects should be reminded to continue to record daily grip strength in their diaries and to bring 
their e-diary with them to their next study visit. 
8.4 
PEOT and Final Visit 
All subjects 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. 
Subjects 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 subject is not willing to attend the visits in the Observation Period, the subject should 
still be strongly encouraged to attend at least the PEOT Visit and the FV. 
The FV should be scheduled 12 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 17 and 
Visit 21, respectively. 
In the event a subject enters the OLE immediately after Visit 17, Visit 17 will be the final study 
visit and there will be no FV. 
In the event a subject experiences a relapse, the subject will be stabilized; stabilization visits will 
be conducted from the time of relapse. Stabilization duration may range from 2 weeks to 
12 weeks after the time of relapse. Once stabilized, the subject will continue SOC onwards. 
Subjects not entering the OLE following stabilization should perform the FV after minimum 
12 weeks or maximum 24 weeks post last IMP dose. Management of the stabilization period is 
summarized in Table 8‒1. 
After the end of the study, the subjects not entering the OLE study should resume treatment 
according to their standard care as per the recommendation of their physician. 
8.5 
Unscheduled Visit/Telephone call 
At any time, a subject may have an unscheduled study visit/telephone call if the investigator 
and/or the subject 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/telephone call, any information on AEs, etc, 
should be collected in the source documents and recorded in the appropriate section of the eCRF. 
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8.6 
Open-label extension study 
Subjects from this study have the option of taking rozanolixizumab during an OLE study 
(CIDP04) provided they have completed the Treatment Period without a CIDP relapse. The last 
study visit in CIDP01 will be either Visit 17 (immediate entry on completion of the Treatment 
Period) or FV (for subjects who continued the Observation Period, relapsed during this period 
and were then stabilized before entry in the OLE). 
Subjects from the placebo arm who relapsed during the Treatment Period will have the option to 
enter the OLE at the study completion, ie, when all subjects have completed study CIDP01, the 
study is unblinded, and key results are available. 
The decision to enter the OLE study must be made by the subject in consultation with the 
investigator. This decision must consider the potential risks of long-term exposure to 
rozanolixizumab and the potential benefits and risks of other treatment options available. 
9 
ASSESSMENT OF EFFICACY 
9.1 
iRODS 
The iRODS questionnaire should be completed by the subject prior to dosing and before any 
discussion of their current health status with the treating physician or any other study 
assessments. Subjects will complete the iRODS questionnaire at the time points detailed in the 
schedule of study assessments (Table 5‒1). The questionnaire will be given to the subject to 
complete and will then be recollected and checked, for completeness only, by study personnel 
other than the treating physician. Subjects should complete the questionnaires themselves, 
unaided, in a quiet location. 
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 
(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). 
Subjects will be asked to complete the iRODS questionnaire at every study visit except for 
nondosing Visits 3, 5, and 7. 
In addition to the primary efficacy variable in which subjects will be assessed for the change 
from Baseline to Week 13 (Day 85) in their iRODS score, subjects will also be assessed for 
change from Baseline in iRODS scores at each scheduled assessment during the Treatment and 
Observation Periods. Other efficacy variables will also include subject 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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9.2 
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 subjects’ adjusted INCAT score at every 
study visit according to schedule of assessments (see 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. 
Subject experienced CIDP relapse (adjusted INCAT) (defined as an increase from Baseline of at 
least 1 point in their adjusted INCAT score) up to Week 13 (Day 85) 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. 
9.3 
Grip strength reported by site personnel 
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 subject, 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. 
Subject 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 13 (Day 85) 
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. 
9.4 
Patient-reported grip strength 
During the study, subjects will keep e-diaries to record daily grip strength from the Screening 
Visit until the end of study participation. Subjects should be reminded to bring their diaries with 
them to each clinic visit and to have them available at home nursing visits. Subjects will be 
trained by the site personnel and receive a manual describing how to assess grip strength using a 
standardized tool. At approximately the same time every day, the maximum grip strength, the 
peak pressure generated by the subject, will be assessed 3 times, with at least 30sec between 
measurements, using the dominant hand. Subjects will be asked to complete their diaries each 
day and record the values for each of the 3 assessments. 
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For variable derivation, see Section 9.3. 
9.5 
Patient-reported outcomes 
The PRO assessments should be administered in the following order: iRODS, CIDP PRO 
instrument, Fatigue, PGIS, PGIC, and exit interview (Final Visit). Refer to Section 9.1 for detail 
on iRODS. 
Subjects will complete PROs and participate in a subject exit interview 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 subject themselves 
in a quiet place. 
9.5.1 
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 
0-10 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 0-100 scoring will be provided for both domain scores with higher score 
reflecting higher severity. 
9.5.2 
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 CIDP01, transformed interval 0-100 scoring will 
be provided for each domain score with higher scores reflecting higher levels of fatigue. 
9.5.3 
Patient Global Impressions 
Patient global impressions will be assessed at the time points detailed in the schedule of study 
assessments (Table 5‒1). 
Subjects 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”. 
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Subjects 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. 
9.6 
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. 
The RT-MRC sum score was developed based on a Rasch analysis of the original MRC grading 
system (Merkies et al, 2010; Merkies et al, 2002; Vanhoutte et al, 2012). 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. 
9.7 
Subject exit interview 
At the Final Visit, qualitative interviews will be conducted by a study nurse/study personnel 
using a semi-structured interview guide. The aim of the interview is to collect the subject's 
experience with CIDP in terms of fatigue, pain, and physical sensations symptoms and impact, 
and the perceived changes during the course of the study. The extent to which the exploratory 
fatigue, pain, and physical sensation items and scales used fully captured their experience will 
also be assessed. The interviews will be audio-recorded. If a subject leaves the study 
prematurely, he/she will also be invited to participate in an exit interview at the Final Visit. 
10 
ASSESSMENT OF PHARMACOKINETIC/ 
PHARMACODYNAMIC AND PHARMACOGENOMIC 
VARIABLES 
10.1 
Pharmacokinetic variables 
The plasma concentration of rozanolixizumab will be characterized. Blood samples will be 
drawn by qualified personnel according to the schedule in Table 10-1. 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 10-1: Serial blood sampling for rozanolixizumab concentration 
Matrix 
Time after start of sc infusion 
Plasma 
Predose and postdose at 4 hours after finishing the infusion on Visits 2, 4, 6, 8, and 10 
Predose on Visits 12, 14, and 16 
Nondosing Visits 3, 5, 7, and 17: samples to be taken once during the visit 
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Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
10.2 
Pharmacodynamic variables 
Pharmacodynamic variables are defined in Section 4.2.2. Neurofilament light chain levels are a 
marker of neuronal cell damage. 
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. 
10.3 
Exploratory pharmacogenomic variables 
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 4 hours postdose 
in case of AE of interest (Table 5-2). The time and date of collection will be recorded in the 
eCRF. 
For RNA, blood samples will be collected at Baseline (Visit 2) and Visits 4, 6, 10, and 
Visit 17/PEOT. For each RNA blood sample, a volume of 2.5mL whole blood is needed. 
For protein and metabolite exploratory biomarkers, blood samples will be collected at Baseline 
(Visit 2) and Visits 4, 8, 12, and 16. 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 or genomic analysis will only ever be related to 
the exploration of the underlying causes of CIDP in patients, related biology, and drug response. 
Justification for additional genomic analyses is detailed in Section 5.4.5. 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. 
11 
ASSESSMENT OF IMMUNOLOGICAL VARIABLES 
The following immunological assessments will be performed according to the schedule of study 
assessments (Table 5‒1). For all immunological assessments, blood samples will be collected 
predose 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 will also be taken 4 hours postdose at Visits 4, 8, 12 and 16. 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. 
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Instructions pertaining to sample collection, processing, storage, labeling, and shipping are 
provided in the laboratory manual for this study. 
 Serum immunoglobulin concentration over time 
 IgA 
 IgE 
 IgM 
 Serum complement levels over time 
 C3 
 C4 
 Plasma complement levels over time 
 C3a 
 C5a 
 Plasma 
 
 Serum cytokines 
12 
ASSESSMENT OF SAFETY 
12.1 
Adverse events 
12.1.1 
Definitions 
12.1.1.1 
Adverse event 
An AE is any untoward medical occurrence in a patient or clinical investigation subject 
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 Informed Consent form), 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 subject’s history or the Baseline Period. 
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12.1.1.2 
Serious adverse event 
Once it is determined that a subject 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) 
 Important medical event that, based upon appropriate medical judgment, may jeopardize the 
patient or subject 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 12.2], 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 subject 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 
subject 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.) 
12.1.1.2.1 
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. 
This list does not change the investigator’s obligation to report all SAEs (including Anticipated 
SAEs) as detailed in Section 12.1.2.3. 
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Table 12-1: 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 12.1.1.2). 
12.1.1.3 
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 
subject. 
12.1.1.4 
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 24 hours, regardless of seriousness, using the fax 
and email details for AEs of interest and independent of SAE reporting. 
Additional assessments which may be required in case of AEs of interest are presented in 
Table 5-2. 
12.1.2 
Procedures for reporting and recording adverse events 
The subject 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) 
employed in the study. 
12.1.2.1 
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 subject’s own words 
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on his/her own records 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. 
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. 
12.1.2.2 
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 
12.1.2.3 
Additional procedures for reporting serious adverse events 
If an SAE is reported, UCB must be informed within 24 hours 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 
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 24 hours. 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. 
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12.1.2.4 
Immediate reporting of adverse events 
The following AEs must be reported immediately using the SAE Report Form according to the 
procedure in Section 12.1.2.3: 
 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 12.1.1.3) 
 AE of interest (see Section 12.1.1.4) 
 Confirmed LTBI, active TB, and NTMBI (see Section 12.3.7) 
12.1.3 
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 subject 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 12.2.1.4. 
If an AE is ongoing at the end of the study for a subject, 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 subject 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 12 weeks 
after the subject 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. 
12.1.4 
Pregnancy 
If an investigator is notified that a subject 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 subject should be withdrawn from the study as soon as 
pregnancy is known (by positive pregnancy test), and the following should be completed: 
 The subject should return for a PEOT Visit. 
 The subject should immediately stop the intake of the IMP or be down-titrated as instructed 
at the PEOT Visit.  
 A Final Visit should be scheduled 12 weeks after the subject has discontinued her IMP. 
The investigator must inform the subject 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 
report on the health of the mother and of the child. Every reasonable attempt should be made to 
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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 subject is lost to follow up and/or refuses to give information, written documentation of 
attempts to contact the subject 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 subject enrolled in a clinical study becomes pregnant, the 
investigator or designee is asked to contact the subject 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 Report form. 
12.1.5 
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. 
12.1.6 
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). 
12.1.7 
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 subjects, 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. 
12.1.8 
Suicidality 
At Screening, the investigator will query each subject 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 subject 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 subject 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 subject 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 subject must be withdrawn 
and immediately referred to a Mental Healthcare Professional. Details of case must be 
documented by the investigator (PI or investigator physician, not site staff conducting the 
C-SSRS) and provided to UCB via the SAE reporting process. 
12.1.9 
Hypersensitivity and adverse reactions 
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 12-2. In the event of a severe or life-threatening (ie, Grade 3 or 4) infusion reaction, the 
subject must permanently discontinue IMP (see Section 6.4) and be managed as described in 
Section 18.1. 
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Table 12-2: Infusion-related reaction grading according to the NCI Common 
Terminology Criteria for adverse events version 4.03 (Jun 2013) 
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 ≤24 hours 
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; NSAIDs= non-steroidal anti-inflammatory drugs 
Data source: Doessegger et al, 2015 
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 subjects 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. 
12.1.10 
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 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, 
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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. 
Details of neurological examination to be performed are provided in Section 12.3.5. The 
Headache Questionnaire will be provided in the study procedures manual. 
12.1.11 
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. 
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. Analysis of stool samples will be performed locally. In addition, collection of blood 
samples for assessment of exploratory biomarkers is required for subjects with moderate to 
severe gastrointestinal disturbances including diarrhea (Table 5-2). 
Treatment of diarrhea will be provided as clinically indicated according to the local guidelines. 
12.2 
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 subjects. All parameters will be 
assessed by the designated central laboratory with the exception of the urine pregnancy 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 12-3. 
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Table 12-3: 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 at Screening and Visit 16 only for women of childbearing potential. 
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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12.2.1 
Evaluation of PDILI 
The PDILI IMP discontinuation criteria for this study are provided in Section 6.4.1, with the 
accompanying required follow-up investigation and monitoring detailed below. All PDILI events 
must be reported as an AE and reported to the study site and sponsor within 24 hours 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 12.1.1.2). 
Evaluation of PDILI consists of the diagnostic testing and continued monitoring included in 
Table 12-4 (specific tests dependent on laboratory results and corresponding symptoms) and 
consultation with a local hepatologist (if applicable; discussed in Section 12.2.1.1). 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 12.2.1.4). 
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 6.4.1), 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 12.2.1.2.1 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 12-4 summarizes the approach to investigate PDILI. 
 
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Table 12-4: 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 subject 
discussed with 
Medical Monitor 
ASAP. 
Immediate, 
permanent IMP 
discontinuation. 
Essential: Must 
have repeat liver 
chemistry values 
and additional 
testing completed 
ASAP (see 
Section 12.2.1.3); 
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 12.2.1.2). 
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 12.2.1.3). 
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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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 subject also has ≥2xULN ALP, the possibility of an indication of biliary obstruction should be discussed with the Medical Monitor. 
c Details provided in Section 12.2.1.1. 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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12.2.1.1 
Consultation with Medical Monitor and local hepatologist 
Potential drug-induced liver injury events require notification of the Medical Monitor within 
24 hours (eg, by laboratory alert), and the subject must be discussed with the Medical Monitor as 
soon as possible. If required, the subject 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 12.2.1.3) and SAE report (if applicable). 
12.2.1.2 
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 12-4 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. 
12.2.1.2.1 
IMP restart/rechallenge (if applicable) 
Rechallenge with a substance potentially causing drug-induced liver injury is dangerous, may be 
fatal, and must not occur. 
Subjects who are immediately discontinued from IMP due to having met certain criteria for 
PDILI (as described in Section 6.4.1 and Table 12-4), 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 12.2.1.3 and 
Section 12.2.1.4 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 subject. 
 The subject has shown clear therapeutic benefit from the IMP. 
 Subject’s ALT or AST elevations do not exceed ≥3xULN. 
 Subject’s total bilirubin is <1.5xULN. 
 Subject 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 subject. 
 Subject agrees to the investigator-recommended monitoring plan. 
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12.2.1.3 
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 12-5 (laboratory measurements) and Table 12-6 (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 subject 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 12-5: 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 subjects 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 12-6: 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 
12.2.1.4 
Follow-up evaluation 
Potential drug-induced liver injury events require follow-up monitoring as described in  
Table 12-4. 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. 
12.3 
Other safety measurements 
12.3.1 
Pregnancy testing 
Pregnancy testing will consist of serum testing at the Screening Visit and Visit 16 and 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 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 
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administered to all female subjects of childbearing potential, regardless of their use of birth 
control. 
12.3.2 
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: 
 PR 
 Systolic/diastolic BP 
 Temperature (oral preferred, ear or axillary allowed) 
Subjects should be sitting for 5 minutes prior and during the collection of BP and PR 
measurements. On dosing days prior to Visit 9, vital signs will be measured prior to IMP 
administration, at the end of the infusion, at 2 hours and 4 hours after the end of the infusion. 
From Visit 9, vital signs will be measured predose, at the end of the infusion and at 2 hours after 
the end of infusion only. The same schedule of vital signs monitoring will be used even if IMP is 
administered at home. 
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. 
12.3.3 
Body weight and height 
The subject’s body weight will be determined at Screening, Visit 2, Visit 17/PEOT, and at Final 
Visit (Visit 21). Body weight at Screening will be used for calculating the dose for 
administration. Height is measured at Screening only. 
12.3.4 
Physical examination 
12.3.4.1 
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, Visit 17/PEOT, and Final Visit. At all other visits (where 
physical examination is performed), an abbreviated 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: 
 General appearance 
 Ear, nose, and throat 
 Eyes 
 Hair and skin 
 Respiratory 
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 Cardiovascular 
 Gastrointestinal 
 Musculoskeletal 
 Hepatic 
 Neurological examination (see also Section 12.3.5) 
 Mental status 
12.3.4.2 
Brief physical examination 
The following body systems will be examined as a part of the brief physical examination at the 
visits outlined in the schedule of assessments (Table 5‒1): 
 General appearance 
 Ear, nose, and throat 
 Eyes 
 Skin 
 Respiratory 
 Gastrointestinal 
 Neurological (focused assessment of sensitivity and power) 
12.3.5 
Neurological examination 
A full neurological examination should be performed at Screening Visit, Visit 17/PEOT and 
Final Visit. In addition, full neurological examination should be performed for any subject who 
experiences severe headache (see Section 12.1.10 and Table 5-2). A full neurological assessment 
will include: (1) General appearance, including posture, motor activity and meningeal signs and, 
if indicated, the 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 examination will include a selected assessment of the following: cognition, 
general, reflexes, muscle strength, and coordination/cerebellar function. 
12.3.6 
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. Subjects 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 subject should be read by 
the same reader, if possible. Findings will be recorded in the eCRF. 
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For the QTc, the following correction formula will be applied: 
Fridericia’s correction: QTc = QT/RR0.33 
12.3.7 
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 
considering 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 6.2.3 Exclusion Criterion 16 and Section 6.4 Withdrawal 
Criteria). The following are the key considerations of these procedures: 
TB tests at Screening 
The IGRA, chest x-ray, and TB 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 subjects to enroll in this study. 
 Subjects 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 subject may 
not be randomized to IMP and, if already randomized, must undergo appropriate study 
specified withdrawal procedures. The retest must be done during the protocol-defined 
Screening window. 
 Subjects 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. Subjects 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 6.2.3). 
 A plain posteroanterior chest x-ray must be performed in the Screening Period unless one has 
been performed within 3 months prior to the Screening Visit. The chest x-ray (or, if done, 
computed axial tomography of the chest) must be clear of signs of TB infection (previous or 
current) before first IMP dose. 
Monitoring for TB during the study 
Subjects will be monitored for signs/symptoms of TB using routine pharmacovigilance measures 
for AEs. Subjects reporting AEs related to signs/symptoms of TB will be evaluated for LTB and 
active TB according to the local medical practice guidelines. 
Subjects 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 
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NTMB must be reported to the Sponsor immediately regardless of seriousness using the SAE 
Report Form. Additional information received by the investigator should be provided to the 
Sponsor within 24 hours of awareness. 
Once withdrawn from study treatment, subjects should return for the PEOT, complete all early 
withdrawal assessments, and complete the follow-up visits. 
TB tests at Final Visit 
Subjects 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 the Final/PEOT Visit. See the 
“TB 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 subject’s history. 
Common symptoms with which the subject 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. 
Chest x-ray for TB 
A plain posteroanterior chest x-ray must be performed in the Screening Period unless one has 
been performed within 3 months prior to the Screening Visit. The chest x-ray (or, if done, 
computed axial tomography of the chest) must be clear of signs of TB infection (previous or 
current) before first IMP administration. All chest imaging (particularly x-rays) should be 
available for review by the investigator before randomization of the subject. The chest x-ray 
should be repeated only if the TB test was confirmed positive or any further evidence is 
suggestive of potential lung TB infection (eg, exposure). Radiographic findings suggestive of 
inactive TB or active TB may include but are not limited to: apical fibrosis, pleural thickening, 
pulmonary nodules, fibrotic scars, calcified granulomas, upper lobe infiltrates, cavitations and 
pleural effusions, calcified lung nodules, calcified hilar lymph nodes, and pericardial 
calcification. 
The chest imaging must be negative for any old or recent TB infection as determined by a 
qualified radiologist and/or pulmonary physician. Any new clinically significant findings post 
Baseline on chest x-ray must be documented in the source documents and the eCRF as an AE. 
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 subjects who may 
require therapy for TB. A subject 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 subject has 
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latent or active TB (see Section 6.2.3, Exclusion Criterion 16). 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 subject has either LTB or active TB infection. 
LTB infection, active TB or other NTBM identified during study 
During the study, subjects who develop evidence of LTB infection or active TB or NMTB must 
immediately stop further administration of IMP 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. 
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 
study subjects 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 CPM 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 eCRFs 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 eCRFs, 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 eCRFs are not considered 
acceptable source documents. 
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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 subject’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). 
13.2.1.1 
Apps 
The data recorded in myUCB 4me is not intended to be used to influence the treatment decisions 
of subjects during the conduct of this study. Rather, the data will be analyzed at the end of the 
study as part of the efficacy measurements (see Section 9). 
Furthermore, this app is designed to only record data associated with the subject’s disease state, 
and therefore it is neither designed nor intended to be used to collect or report safety-related 
information about the subject. See Section 12 that describes the procedures for handling 
safety-related information. 
To ensure the confidence in the reliability, quality, and integrity of the data, several security 
measures have been put in place. myUCB 4me itself is protected by a Personal Identification 
Number (PIN) that is known only to the subject participating in CIDP01. 
All data entered by the subject is fully encrypted within myUCB 4me and its transmission to the 
database. 
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 
source documents (eg, subject 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 
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. 
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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, except for comment fields, which are verified by a second person. The data 
are entered into the eCRFs 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 
Subject Screening and Enrollment log/Subject Identification Code list 
The subject’s screening and enrollment will be recorded in the Subject Screening and Enrollment 
Log. 
The investigator will keep a Subject Identification Code list. This list remains with the 
investigator and is used for unambiguous identification of each subject. 
The subject’s consent and enrollment in the study must be recorded in the subject’s medical 
record. These data should identify the study and document the dates of the subject’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. 
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 eCRFs, 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. These documents should be retained 
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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 
subjects enrolled have been protected, that enrolled subjects (ie, signing consent and undergoing 
study procedures) are appropriate for the study, and that all data relevant for the evaluation of the 
IMP 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. 
14 
STATISTICS 
A description of statistical methods follows and will be described in more detail in the Statistical 
Analysis Plan (SAP), which will be finalized prior to unblinding. 
14.1 
Definition of analysis sets 
Seven analysis sets will be defined for this study: the Enrolled Set (ES), the Randomized Set 
(RS), the Safety Set (SS), the Full Analysis Set (FAS), the Per-Protocol Set (PPS), the 
Pharmacokinetic Per-Protocol Set (PK-PPS), and the Pharmacodynamic-Per Protocol Set 
(PD-PPS). 
14.1.1 
Enrolled Set 
The Enrolled Set (ES) will consist of all subjects who have given informed consent. 
14.1.2 
Randomized Set 
The Randomized Set (RS) will consist of all subjects randomized into the study. 
14.1.3 
Safety Set 
The Safety Set (SS) will consist of all subjects in the RS, who have received at least 1 dose of 
IMP. 
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Safety variables will be analyzed using the SS. 
It is expected that subjects will receive treatment as randomized and hence safety analyses will 
be based on the randomized treatment group. However, if after unblinding it is determined that a 
subject has received treatment different to the treatment they were randomized to, then for safety 
analyses the subject will be allocated to the actual treatment they received. 
14.1.4 
Full Analysis Set 
The Full Analysis Set (FAS) will consist of all subjects in the SS, who have a Baseline and least 
1 post-Baseline iRODS measurement. 
The FAS is the primary analysis set for efficacy analyses. Even in the case of mistreatment, 
subjects will be analyzed as treated. As for the SS, in the case of mistreatment subjects will be 
primarily analyzed as treated. However, if applicable, sensitivity analyses will also be performed 
according to the randomized treatment group. 
14.1.5 
Per-Protocol Set 
The Per-Protocol Set (PPS) is a subset of the FAS, consisting of those subjects who had no 
important protocol deviation affecting the primary efficacy variable, as confirmed during a 
preanalysis data review meeting conducted prior to study unblinding. Post-Baseline deviations 
will not necessarily lead to total exclusion of a subject from the PPS but may lead to exclusion of 
specific data. 
Analysis of the primary efficacy variable will be repeated using the PPS. 
14.1.6 
Pharmacokinetic Per-Protocol Set 
The Pharmacokinetic Per-Protocol Set (PK-PPS) is a subset of the SS, consisting of those 
subjects who had no important protocol deviation affecting the plasma concentration of 
rozanolixizumab. Post-Baseline deviations will not necessarily lead to total exclusion of a 
subject from the PK-PPS but may lead to exclusion of specific data. 
14.1.7 
Pharmacodynamic Per-Protocol Set 
The Pharmacodynamic Per-Protocol Set (PD-PPS) is a subset of the FAS, consisting of those 
subjects 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 subject 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 treatment group (Placebo and rozanolixizumab) and 
visit (where applicable) with the statistics mean, standard deviation (SD), median, minimum, and 
maximum. Categorical variables will be summarized by treatment group and visit (where 
applicable) with frequency counts and percentages. 
Unless stated otherwise, all statistical tests will be 1-sided and conducted at 0.05 alpha levels. 
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. 
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14.3 
Planned efficacy analyses 
14.3.1 
Analysis of the primary efficacy variable 
The primary efficacy variable is the change from Baseline to Week 13 (Day 85) in the iRODS 
score. 
The primary analysis of the primary variable will be based on a mixed model repeated measures 
(MMRM) analysis of covariance (ANCOVA) that includes terms for treatment group, the 
Baseline iRODS score, the prior Ig therapy, and the interaction between treatment group and 
week. The model will define subject as a random effect and utilize an unstructured covariance 
pattern. 
An estimate for the placebo-adjusted treatment effect with a 2-sided 90% confidence interval 
(CI) will be produced. 
For subjects who prematurely withdraw for any reason before Week 13 (Day 85), data collected 
during the PEOT Visit will be used to impute iRODS at the next visit. Since the analysis is based 
on the MMRM approach, visits beyond the PEOT Visit will not be imputed. 
Different sensitivity analyses to evaluate the effect of missing observations on the primary 
analysis findings will be done. The first sensitivity analysis will be performed in the same way as 
the primary analysis, however for the PPS. A further analysis will utilize the last observation 
carried forward (LOCF) approach; missing values will be replaced by the last observed 
post-Baseline value of the variable and the analysis will be performed on the resulting dataset. 
An additional sensitivity analysis will be performed excluding any subject who received a mean 
dose more than ±10% from the intended dose. 
14.3.2 
Other efficacy analyses 
For the CIDP relapse (Yes/No) up to Week 13 (Day 85) after first treatment according to 
iRODS, adjusted INCAT, and maximum grip strength assessed by the site personnel scores, 
treatment comparison versus placebo for the rozanolixizumab group (differences in CIDP relapse 
rates) will be performed using a standard 1-sided Wald asymptotic test with a 5% alpha level. 
The corresponding 2-sided 90% CI for the difference will be constructed using the asymptotic 
standard error (asymptotic Wald confidence limits). Sensitivity analyses for relapse will be 
performed and described in the SAP. 
For the analysis, subjects who withdraw for any reason before Week 13 (Day 85) or who have 
missing data at Week 13 (Day 85) will be considered as relapsed. The same is true for subjects 
administered an Ig infusion rescue medication during this time interval. 
Kaplan-Meier estimates and log-rank tests will be used to compare the treatment difference for 
the time to CIDP relapse (according to iRODS, adjusted INCAT scores, and maximum grip 
strength assessed by site personnel). 
The statistical methods applied for the variables derived from rescue medication (subject 
received rescue medication [Yes/No] and time to rescue medication administration) will be the 
same as those for the CIDP relapse and time to CIDP relapse. 
For the change from Baseline at each scheduled assessment during the Treatment and 
Observation Period, iRODS score, adjusted INCAT score, maximum grip strength assessed by 
the site personnel score, and RT-MRC sum score, treatment differences will be compared by 
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ANCOVAs adjusted for Baseline measurement and prior Ig therapy. Analyses will be performed 
for the FAS utilizing the observed cases. 
The results of the newly developed PROs (CIDP PRO instrument and fatigue scale) will be listed 
and frequency tables will be provided at an item level. A psychometric analysis plan will be 
created, which will detail all the exploratory and correlation analyses between the newly 
developed PROs and clinical variables. The PROs will also be subject to a Rasch analysis. The 
results of the psychometric analyses will be reported separately from the clinical study report 
(CSR). 
14.4 
Other analyses 
14.4.1 
Safety analyses 
The incidence of subjects with TEAEs will be determined by treatment group. 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 treatment group and system organ class. Additional 
tables will summarize TEAEs by maximum severity and causal relationship with 
rozanolixizumab, as judged by the investigator, per treatment group. 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 by treatment group. 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 by treatment group 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 by treatment 
group and 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 treatment group and 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 subjects who complete a headache 
questionnaire, undergo stool sampling, or lumbar puncture, etc. 
14.4.2 
PK analyses 
Pharmacokinetic variables of rozanolixizumab like AUC (area under the curve), Cmax (maximum 
concentration) cannot be derived, since blood sampling will be performed at 1 time point 
post-dosing per visit only. Thus, PK is restricted to concentration data. 
In contrast to the general descriptive display, concentration data will be summarized by treatment 
group, actual dose received, and time point using the number of available observations, mean, 
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median, SD, minimum, maximum, geometric mean (and associated 95% confidence intervals), 
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 
PD analyses 
For all PD variables, descriptive statistics for the value, change from Baseline, and/or percentage 
change from Baseline will be tabulated by treatment group, actual dose received, and time point. 
The PD variables will include serum IgG, IgG subclass, and neurofilament light chain 
concentrations. 
Population PK analyses and PK/PD analyses may be conducted for the PD variables of interest. 
All such PK and PK/PD analyses will be described in a separate Data Analysis Plan (DAP) 
however results will not be reported in the CSR. 
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 by treatment group and visit using descriptive statistics. 
The 
 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 
treatment group and visit to inform on incidence and emergence of 
 figures will also be 
presented. 
Further details will be provided in the SAP. 
14.4.5 
Subject exit interview 
The anonymous audio recordings will be analyzed by a third party and reported outside the 
clinical study report. 
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 subjects 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 subjects who prematurely withdraw for any reason before Week 13 (Day 85), data collected 
during the PEOT Visit will be used to impute iRODS at the next visit. Since the primary analysis 
of the primary variable is based on the MMRM approach, visits beyond the PEOT Visit will not 
be imputed. Missing data in-between visits will be covered by the MMRM approach. 
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All imputation of missing or partial dates for safety assessments, as well as detailed handling of 
missing efficacy data, will be detailed in the SAP. 
14.7 
Planned interim analysis and data monitoring 
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 subjects, regulatory 
authorities, and IRBs/IECs will be informed appropriately and as early as possible (see safety 
signal detection in Section 12.1.7). 
At least 4 interim analyses will be performed. 
The first interim analysis will be conducted after 8 subjects have received their first 4 sc 
infusions and have attended the next visit (Visit 9). The second interim analysis will be 
conducted after 8 subjects (receiving 12 sc infusions) have attended the first visit of the 
Observation Period (ie, Visit 17, the visit 1 week after last dosing). The third interim analysis 
will take place after data from 8 additional subjects are available, ie, after 16 subjects have 
attended the first visit of the Observation Period. Each time, the DMC will assess the safety of 
rozanolixizumab based on an unblinded data analysis. The safety and PD variables to be used 
and decision rules will be specified in the DMC charter. During these reviews recruitment will 
not be stopped. If the observed safety or PD of the data from of interim analyses 1 and 2 is not as 
expected, the DMC may recommend to either lower or increase the dose. The implementation of 
such changes would be handled via a substantial protocol amendment. 
The analyses will be described in a separate Interim Statistical Analysis Plan. The first 3 interim 
analyses will utilize all safety, PK and PD data available at the time of the cut off (eg, possibly 
additional visits and/or subjects). For all interim analyses, the data to be analyzed should be as 
clean as possible; however, the database will not be locked and a snapshot will be taken. 
The fourth and final interim analysis will be performed once all subjects have attended Visit 17, 
the first visit of the Observation Period (ie, Day 85). This interim analysis will provide, at a 
minimum, the results for the primary variable “Change from Baseline to Week 13 (Day 85) in 
iRODS score”. 
If needed, subsequent DMC meetings will take place. The timing of further interim analyses and 
reviews of the data by the DMC will be decided by the DMC. 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. 
14.8 
Determination of sample size 
Due to the pilot character of the study, the sample size is mainly chosen based on the practical 
limitations (eg, to recruit sufficient subjects in a reasonable time). In addition, currently no data 
of results is available in the literature for the primary variable. Thus, the sample size 
considerations are only based on assumptions for the effect size index (difference in group 
means/SD). When the sample size in each group is 
 a 1-sided 5.0% t-test for the comparison 
of rozanolixizumab and placebo will provide 80% power to detect a statistically significant 
difference in case there is a large effect of rozanolixizumab with an effect size index of 
 In 
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case of a medium index (
) the power already decreases to 40%. It will be assumed that 
 
of the randomized subjects cannot be utilized for the efficacy analysis; hence 34 subjects will be 
randomized. 
15 
ETHICS AND REGULATORY REQUIREMENTS 
15.1 
Informed consent 
Subject’s informed consents 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 subject in both oral and written form by the investigator (or 
designee). Each subject will have the opportunity to discuss the study and its alternatives with 
the investigator. 
Prior to participation in the study, the Informed Consent form(s) should be signed and personally 
dated by the subject, or his/her legal representative, and by the person who conducted the 
informed consent discussion (investigator or designee). The subject or his/her legal 
representative must receive a copy of the signed and dated Informed Consent form. As part of 
the consent process, each subject must consent to direct access to his/her medical records for 
study-related monitoring, auditing, IRB/IEC review, and regulatory inspection. 
If the Informed Consent form is amended during the study, the investigator (or the sponsor, if 
applicable) must follow all applicable regulatory requirements pertaining to the approval of the 
amended Informed Consent form by the IRB/IEC and use of the amended form. 
All studies conducted at centers in the United States must include the use of a Health Insurance 
Portability and Accountability Act Authorization form. 
The subject may withdraw his/her consent to participate in the study at any time. A subject is 
considered as enrolled in the study when he/she has signed the Informed Consent form. An eCRF 
must not be started, nor may any study specific procedure be performed for a given subject, 
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 
Subject identification cards 
Upon signing the Informed Consent and Assent form (as applicable), the subject or legal 
representative will be provided with a subject identification card in the language of the subject. 
The investigator will fill in the subject identifying information and medical emergency contact 
information. The investigator will instruct the subject 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. 
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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, Informed 
Consent form, IB, investigator’s curriculum vitae (if applicable), advertisement (if applicable), 
and all other subject-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 subjects or others, and any protocol deviations, 
to eliminate immediate hazards to subjects. 
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 subjects. 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 subject risk involved, but no less than 
once per year. The investigator should provide a final report to the IRB/IEC following study 
completion. 
UCB (or its representative) will communicate safety information to the appropriate regulatory 
authorities and all active investigators in accordance with applicable regulatory requirements. 
The appropriate IRB/IEC will also be informed by the investigator or the sponsor, as specified by 
the applicable regulatory requirements in each concerned country. Where applicable, 
investigators are to provide the sponsor (or its representative) with evidence of such IRB/IEC 
notification. 
15.4 
Subject privacy 
UCB staff (or designee) will affirm and uphold the subject’s confidentiality. Throughout this 
study, all data forwarded to UCB (or designee) will be identified only by the subject 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 
subject’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 subject’s study participation, and autopsy reports for deaths 
occurring during the study). 
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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 
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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 infusion rate to 
. 
 
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. 
Infuse 
. 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 5 minutes until back to Baseline. 
Wait 20 minutes. 
 
 
 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 
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) 
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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 
subject (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) 
3. Reduced blood pressure after exposure to known allergen for that subject (minutes to several 
hours): Systolic BP of less than 90mmHg or greater than 30% decrease from the subject’s 
Baseline systolic BP value. 
18.3 
Protocol amendment 1 
Rationale for the amendment 
The CIDP01 protocol was originally finalized by UCB in Mar 2017, but was not submitted to 
any regulatory authorities nor IRBs/IECs. The current protocol has been amended to incorporate 
changes to the study’s conduct and analysis to better align with the current understanding of 
available data from completed and ongoing studies with rozanolixizumab. Given that the 
protocol has been updated subsequent to original protocol finalization at UCB, the current 
protocol is considered an amendment per UCB policy. A summary of changes section is not 
included since the original protocol was not previously submitted to any regulatory authorities. 
18.4 
Protocol amendment 1.1 
Rationale for the amendment 
This is a local amendment for France. The changes implemented in this amendment (modifying 
the timeframe for use of contraception post study completion and the final urinary pregnancy 
test) have been incorporated into and are detailed in global Protocol Amendment 2 below. 
18.5 
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 
about actual doses administered to the subjects. Updates to the status of the other 
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rozanolixizumab studies have been included. The visit windows and the scheduling of the 
randomization visit in view of the Ig treatment at study start have been clarified. 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 #3 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.  
In addition, some administrative changes have been made. 
  
Modifications and changes 
Specific changes 
Change #1 
Section 1 Summary, second paragraph 
Subjects will be randomized to 1 of 2 treatment arms: rozanolixizumab 
sc or placebo sc 
in a ratio of 1:1. Subjects will receive 12 weekly doses of investigational medicinal product 
(IMP). The maximum duration of the study per subject 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). The study 
is planned to be conducted in approximately 24 sites globally. Approximately 34 subjects will be 
randomized to ensure at least 30 subjects are evaluable for the primary efficacy analysis. 
Has been changed to: 
Subjects will be randomized to 1 of 2 treatment arms: rozanolixizumab 
 sc or placebo sc 
in a ratio of 1:1. For exact doses to be administered, refer to Section 7.2. Subjects will receive 
12 weekly doses of investigational medicinal product (IMP). The maximum duration of the study 
per subject 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). The study is planned to be conducted in approximately 24 
sites globally. Approximately 34 subjects will be randomized to ensure at least 30 subjects are 
evaluable for the primary efficacy analysis. 
Change #2 
Section 2 Introduction, fifth paragraph, final sentence 
To date, rozanolixizumab has been administered to healthy subjects in a completed, first in-
human study (UP0018, detailed below), 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). 
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Has been changed to: 
To date, rozanolixizumab has been administered to healthy subjects in a completed, first-in-
human study (UP0018, detailed below), 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). See summaries below for UP0018, TP0001, 
and MG0002. 
Change #3 
Section 2 Introduction 
The following text has been added: 
TP0001 is a completed, Phase 2, multicenter, open-label, multiple-dose, multiple-arm study 
to evaluate the safety, tolerability, and efficacy of rozanolixizumab administered as sc doses 
of 
 
), in 66 subjects ≥18 years 
of age with persistent or chronic ITP.  
There were no treatment-emergent deaths reported during TP0001, and no subjects 
discontinued from the study or IMP due to TEAEs. There were no treatment-related SAEs 
reported from the study. All TEAEs considered by the investigator to be related to 
rozanolixizumab were mild to moderate in intensity and resolved without clinical sequelae. 
The majority of the related TEAEs did not require treatment. The most common TEAEs 
were headache (26 subjects [39.4%]), diarrhea (8 subjects [12.1%]), and vomiting (6 
subjects [9.1%]). Treatment with rozanolixizumab was generally 
 and well tolerated 
with an acceptable safety profile across the treatment groups.  
MG0002 is a completed, multicenter, randomized, investigator-and subject-blind, 
placebo-controlled, treatment sequence study evaluating the safety, tolerability, and 
efficacy of rozanolixizumab in 43 subjects with moderate to severe MG. 
 
 
 
 
 
 
There were no deaths reported during MG0002. One subject (2.3%) discontinued from the 
study due to an AE (MG crisis). In addition, 3 subjects experienced TEAEs (headache) that 
led to discontinuation of IMP, but the subjects remained in the study. Six subjects (14.0%) 
reported a total of 7 serious TEAEs. One subject in the Placebo-Rozanolixizumab 
 Group experienced a serious TEAE of headache that was considered by the 
investigator to be related to the IMP. With the exception of 1 serious TEAE of ulna 
fracture, all other serious TEAEs were in the System Organ Class of Nervous system 
disorders. Three subjects (7.0%) reported serious TEAEs of MG (the studied indication), 
and 1 subject (2.3%) reported a serious TEAE of MG crisis. The most common TEAEs 
were headache (23 subjects [53.5%]), diarrhea (11 subjects [25.6%]), and nausea 
(6 subjects [14.0%]). Overall, repeated administrations of rozanolixizumab at dose levels of 
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 sc were generally 
 and well tolerated, with an acceptable safety 
profile. 
Change #4 
Section 2 Introduction, final paragraph 
Additional information on the development of rozanolixizumab, including data from the ongoing 
clinical studies in ITP subjects (TP0001) and MG subjects (MG0002), can be found in the 
current version of the IB for rozanolixizumab. 
Has been changed to: 
Additional information on the development of rozanolixizumab, including data from the ongoing 
clinical studies in ITP subjects (TP0001) and MG subjects (MG0002), can be found in the 
current version of the IB for rozanolixizumab. 
Change #5 
Section 5.1 Study description 
The following text has been added: 
See Section 7.2 for details on treatments to be administered. 
Change #6 
Section 5.1 Study description, third paragraph 
The dose level of 
 may be reduced to 
 (or equivalent placebo) for an individual 
subject based on their tolerability. For example, if a subject experiences a severe headache which 
does not respond to headache treatment (see Section 12.1.10), a decision to decrease future doses 
to 7mg/kg may be made. The decision to decrease the dose will be made following a discussion 
between the investigator and the UCB study physician. In addition, the decision to decrease the 
dose for the remainder of the subjects may be made following a DMC recommendation. 
Has been changed to: 
The dose level of 
 may be reduced to 
 (or equivalent placebo) for an individual 
subject based on their tolerability (see Section 7.2 for details). For example, if a subject 
experiences a severe headache which does not respond to headache treatment (see 
Section 12.1.10), a decision to decrease future doses to 
 may be made. The decision to 
decrease the dose will be made following a discussion between the investigator and the UCB 
study physician. In addition, the decision to decrease the dose for the remainder of the subjects 
may be made following a DMC recommendation. 
Change #7 
Table 5-1 Schedule of Assessments, Days 3, 10, and 17 column headings 
Visit No 
1 
2 
3 b 
4 
5 b 
6 
7 b 
8 
9c 
… 
Visit type 
S 
S 
TH 
S 
TH 
S 
TH 
S 
H 
… 
Week 
 
1 
2 
3 
4 
5 
… 
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Day 
-35 to  
-14 
1 
BL 
3 
8 
±2 
10 
15 
±2 
17 
22 
±2 
29 
±2 
… 
Have been changed to: 
Visit No 
1 
2 
3 b 
4 
5 b 
6 
7 b 
8 
9c 
… 
Visit type 
S 
S 
TH 
S 
TH 
S 
TH 
S 
H 
… 
Week 
 
1 
2 
3 
4 
5 
… 
Day 
-35 to  
-14 
1 
BL 
2 or 3 
8 
±2 
9 or 10 
15 
±2 
16 or 
17 
22 
±2 
29 
±2 
… 
Change #8 
Table 5-1 Schedule of Assessments, footnotes b and j 
b Two days after the subject’s first 3 doses of IMP, the subject will be telephoned and a healthcare professional will 
visit the subject at home to collect PK samples and immunoglobulins (total IgG and IgG subclasses) (only at 
Visit 7). Alternately, the visit can be conducted at the site as deemed necessary by site personnel and/or the 
subject.  
j The pregnancy test must be negative before dosing. Note that the final urinary pregnancy test of the study should 
be no longer than 60 days after the final dose of IMP. 
Have been changed to: 
b One or two days after the subject’s first 3 doses of IMP, the subject will be telephoned, and a healthcare 
professional will visit the subject at home to collect PK samples and immunoglobulins (total IgG and IgG 
subclasses) (only at Visit 7). Alternately, the visit can be conducted at the site as deemed necessary by site 
personnel and/or the subject. 
j The pregnancy test must be negative before dosing. Note that the final urinary pregnancy test of the study should 
be no longer than 90 days after the final dose of IMP. 
Change #9 
Section 5.4.4 Rationale for dose selection, paragraphs 3 through 5 
MG0002 is an ongoing, Phase 2, multicenter, randomized, investigator- and subject-blind, 
placebo-controlled, 2-arm repeat dose, treatment sequence study evaluating the safety and 
efficacy of rozanolixizumab sc 
) in 43 subjects with generalized MG. 
 
 
 
 
 
 
Overall, repeated administrations of rozanolixizumab at dose levels of 
 sc 
have been generally 
 and well tolerated, with an acceptable safety profile. No new safety 
concerns have been identified to date; a summary of results is included in the IB. 
TP0001 is an ongoing, Phase 2, multicenter, open-label, multiple-arm study to evaluate the 
safety, tolerability, and efficacy of rozanolixizumab in subjects with primary persistent or 
chronic ITP. The following dose arms are being used in the study: 
 Dose Arm 1 (15 subjects): rozanolixizumab 
sc (
) 
 Dose Arm 2 (15 subjects): rozanolixizumab 
sc 
) 
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 Dose Arm 3 (6 to 12 subjects): rozanolixizumab 
 sc (
) 
 Dose Arm 4 (6 to 12 subjects): rozanolixizumab
 sc (
) 
 Dose Arm 5 (6 to 12 subjects): rozanolixizumab 
g sc (
)  
 
. Based on an interim 
analysis of 55 subjects, the 
dose was well-tolerated according to data available to date 
and a summary of the safety results is included in the IB. However, it should be noted that the 
exposure of rozanolixizumab was lower in subjects with ITP than subjects with MG when dosed 
with the same dose level. 
Have been changed to: 
MG0002 is a completed, Phase 2, multicenter, randomized, investigator- and subject-blind, 
placebo-controlled, 2-arm repeat dose, treatment sequence study evaluating the safety and 
efficacy of rozanolixizumab sc (
 in 43 subjects with generalized MG. 
 
 
 
 
 
 Overall, repeated administrations of rozanolixizumab at dose levels of 
 
 sc were generally 
 and well tolerated, with an acceptable safety profile. No new 
safety concerns were identified; a summary of results is included in the IB. 
TP0001 is a completed, Phase 2, multicenter, open-label, multiple-arm study to evaluate the 
safety, tolerability, and efficacy of rozanolixizumab in subjects with primary persistent or 
chronic ITP. The following dose arms were used in the study: 
 Dose Arm 1 (15 subjects): rozanolixizumab 
 sc (
) 
 Dose Arm 2 (15 subjects): rozanolixizumab
 sc (
) 
 Dose Arm 3 (6 to 12 subjects): rozanolixizumab 
g sc (
) 
 Dose Arm 4 (6 to 12 subjects): rozanolixizumab 
 sc 
 
 Dose Arm 5 (6 to 12 subjects): rozanolixizumab 
 sc 
  
 
s. Based on an 
interim analysis of 55 subjects, The 
 dose was well-tolerated, according to data 
available to date and a summary of the safety results is included in the IB. However, it should be 
noted that the exposure of rozanolixizumab was lower in subjects with ITP than subjects with 
MG when dosed with the same dose level. 
Change #10 
Section 5.4.4 Rationale for dose selection, final paragraph, final 
2 sentences 
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This change in concentration is not predicted to have an impact on local tolerability, however to 
allow this to be fully assessed the first two infusions in each subject will be given at a slower rate 
). If no tolerability concerns are identified subsequent infusions may be given at 
. 
Have been changed to: 
This change in concentration is not predicted to have an impact on local tolerability, however to 
allow this to be fully assessed the first two infusions in each subject will be given at a slower rate 
of approximately 
. If no tolerability concerns are identified subsequent infusions may be 
given at rates up to 
. 
Change #11 
Section 6.1 Inclusion criteria, criterion 7, second paragraph, first sentence 
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. 
Has been changed to: 
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. 
Change #12 
Section 6.2.2 Exclusion criteria related to CIDP diagnosis, criterion 3 
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 #13 
Section 6.2.3 Exclusion criteria related to health status/safety of the 
subject, criterion 14 
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 #14 
Section 6.4 Withdrawal criteria, Subject may be discontinued from IMP 
section, criterion 2 
Subject experiences a severe AE of headache which 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 
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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 to 
 (or its placebo equivalent) 
if the headache persists despite symptomatic treatment (refer to Section 12.1.10 for details on the 
medical management of headaches). 
Has been changed to: 
Subject experiences a severe AE of headache which is considered related to the IMP in the 
opinion of the investigator (see Section 12.1.10). 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 to 
 (or its 
placebo equivalent) if the headache persists despite symptomatic treatment (refer to 
Section 12.1.10 for details on the medical management of headaches and Section 7.2 for 
treatment to be administered). 
Change #15 
Section 7.2 Treatments to be administered, first paragraph, first bullet 
 Subjects in Treatment Arm 2 will receive 1 sc dose of placebo weekly for 12 weeks (Visits 2, 
4, and 6, and Visit 8 through Visit 16). 
 Has been changed to: 
 Subjects in Treatment Arm 1 will receive 1 sc dose of rozanolixizumab 
(see 
Table 7-1) weekly for 12 weeks (Visits 2, 4, and 6, and Visit 8 through Visit 16). 
Change #16 
Section 7.2 Treatments to be administered 
The following table has been added: 
Table 7-1: 
IMP doses to be administered (equivalent to approximately 10mg/kg) 
by body weight 
Body weight ranges 
IMP doses to be administered (equivalent to 
approximately 
) 
IMP volume to be 
administered 
≥40 to <49kg 
3mL 
≥49 to <63kg 
4mL 
≥63 to <77kg 
5mL 
≥77 to <91kg 
6mL 
≥91 to <105kg 
7mL 
≥105 to <119kg 
8mL 
≥119 to <133kg 
9mL 
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Table 7-1: 
IMP doses to be administered (equivalent to approximately 10mg/kg) 
by body weight 
Body weight ranges 
IMP doses to be administered (equivalent to 
approximately 
) 
IMP volume to be 
administered 
≥133 to <147kg 
10mL 
≥147 to <161kg 
11mL 
≥161 to 170kg 
12mL 
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. 
Change #17 
Section 7.2 Treatments to be administered, third paragraph 
In case of tolerability issues (eg, severe headache) and if symptomatic headache medication 
(eg, acetylsalicylic acid 1000mg) is not sufficient, the dose of IMP could be lowered to 
 
(or equivalent placebo) during the next visit when the investigator, Medical Monitor and Study 
Physician agree that the dose reduction poses no significant risk for the subject (see 
Section 12.1.10). 
Has been changed to: 
In case of tolerability issues (eg, severe headache) and if symptomatic headache medication 
(eg, acetylsalicylic acid 1000mg) is not sufficient, the dose of IMP could be lowered to 
 
(see Table 7-2) (or equivalent placebo) during the next visit when the investigator, Medical 
Monitor, and Study Physician agree that the dose reduction poses no significant risk for the 
subject (see Section 12.1.10).  
Change #18 
Section 7.2 Treatments to be administered 
The following table has been added: 
Table 7-2: 
IMP doses to be administered (equivalent to approximately 7mg/kg) 
by body weight 
Body weight ranges 
IMP doses to be administered (equivalent to 
approximately 
) 
IMP volume to be 
administered 
≥40 to <49kg 
2mL 
≥49 to <69kg 
3mL 
≥69 to <89kg 
4mL 
≥89 to <109kg 
5mL 
≥109 to <129kg 
6mL 
≥129 to <149kg 
7mL 
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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 
≥149 to <169kg 
8mL 
≥169 to 170kg 
9mL 
IMP= investigational medicinal product  
Change #19 
Section 7.2 Treatments to be administered, fifth paragraph 
The IMP will be administered as a sc infusion using a syringe pump. 
 For the first 2 doses, 
 
 
 
 For the subsequent doses, 
 
 
 
 
Has been changed to: 
The IMP will be administered as a sc infusion using a syringe pump. 
 For the first 2 doses, 
 
 
 
 For the subsequent doses, 
 
 
 
 
Change #20 
Section 7.7.1 Permitted concomitant treatments (medications and 
therapies) 
The following text 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 
 
. 
Change #21 
Section 7.7.3 Rescue medication, first sentence 
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If, at any time during the Treatment and 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 
and the subject will be withdrawn from IMP (see Section 6.4). 
Has been changed to: 
If, at any time during the Treatment and Observation Period, the subject relapses according to the 
predefined criteria for relapse as specified in Section 4.1.2 using the subject’s score on 
iRODS, INCAT, or maximum grip strength (assessed by site personnel), and supported by the 
medical judgement of the investigator, then rescue medication must be considered and the 
subject will be withdrawn from IMP (see Section 6.4). 
Change #22 
Section 8.1.1 Screening Visit 1 (Day -35 to Day -14), first 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 #23 
Section 8.2 Treatment Period Visits 2 to 16 (Day 1 to Day 78), first paragraph, first 2 
bullets 
 For subjects on an IVIg 3- to 6-week regimen, randomization 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 Randomization Visit could be up to 1 week after that date. 
 Subjects on a 2-week IVIg treatment regimen will continue on their regular schedule for 
1 further IVIg treatment until randomization. Randomization will occur 1 week before the 
planned IVIg dose. However, if that specific date is not possible, the Randomization Visit 
could be up to 1 week after that date. 
Has been changed to: 
 For subjects on an IVIg 3- to 6-week regimen, randomization 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 Randomization Visit could be conducted up to the date of the next 
planned IVIg dose (ie, 1 week after the preferred date). 
 Subjects on a 2-week IVIg treatment regimen will continue on their regular schedule for 
1 further IVIg treatment until randomization. Randomization will occur 1 week before the 
planned IVIg dose. However, if that specific date is not possible, the Randomization Visit 
could be conducted up to the date of the next planned IVIg dose (ie, 1 week after the 
preferred date). 
Change #24 
Section 8.2 Treatment Period Visits 2 to 16 (Day 1 to Day 78), third paragraph 
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For the 11-week Treatment Period, visits will occur weekly from Visit 2 to Visit 16. 
Three additional visits (Visits 3, 5 and 7) will be performed 1 day after each of the first 3 IMP 
administrations to monitor safety and collect samples for PK/PD. 
Has been changed to: 
For the 11-week Treatment Period, visits will occur weekly from Visit 2 to Visit 16. 
Three additional visits (Visits 3, 5, and 7) will be performed 1 or 2 days after each of the first 
3 IMP administrations to monitor safety and collect samples for PK/PD. 
Change #25 
Section 8.2 Treatment Period Visits 2 to 16 (Day 1 to Day 78), final 
paragraph, first sentence 
If at any time during the Treatment 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 and the 
subject must be withdrawn from IMP (see Section 6.4 and Section 7.7.3). 
Has been changed to: 
If at any time during the Treatment Period, the subject relapses according to the predefined 
criteria for relapse as specified in Section 4.1.2 using the subject’s score on iRODS, INCAT, 
or maximum grip strength (assessed by site personnel), and supported by the medical 
judgement of the investigator, then rescue medication must be considered and the subject must 
be withdrawn from IMP (see Section 6.4 and Section 7.7.3). 
Change #26 
Section 8.2.2 Visit 3 Telephone Call and Home Visit (Day 3), first paragraph, 
first sentence 
Visit 3 consists of a telephone call from the site personnel and a home nursing visit performed 
within 2 days of the previous IMP administration. 
Has been changed to: 
Visit 3 consists of a telephone call from the site personnel and a home nursing visit performed 
after 1 or 2 days of the previous IMP administration. 
Change #27 
Section 8.2.4 Visit 5 Telephone Call and Home Visit (Day 10), first 
paragraph, first sentence 
Visit 5 is a telephone call from the site with a home nursing visit performed within 2 days of the 
previous IMP administration. 
Has been changed to: 
Visit 5 is a telephone call from the site with a home nursing visit performed after 1 or 2 days of 
the previous IMP administration. 
Change #28 
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Clinical Study Protocol 
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CIDP01 
 
 
 
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Page 123 of 128 
Section 8.2.6 Visit 7 Telephone Call and Home Visit (Day 17), first 
paragraph, first sentence 
Visit 7 is a telephone call from the site with a home nursing visit performed within 2 days of the 
previous IMP administration. 
Has been changed to: 
Visit 7 is a telephone call from the site with a home nursing visit performed after 1 or 2 days of 
the previous IMP administration. 
Change #29 
Section 8.3 12-Week Observation Period Visits 17 to 21 (Day 85 to Day 162), 
fourth paragraph, first sentence 
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. 
Has been changed to: 
If at any time during the Observation Period, the subject relapses according to the predefined 
criteria for relapse as specified in Section 4.1.2 using the subject’s score on iRODS, INCAT, 
or maximum grip strength (assessed by site personnel), and supported by the medical 
judgement of the investigator, then rescue medication must be considered. 
Change #30 
Section 9.2 INCAT, second paragraph 
The investigator (or qualified personnel) will record the subjects’ adjusted INCAT score at every 
study visit according to schedule of assessments (see Table 5‒1). 
Has been changed to: 
The investigator (or qualified personnel) will record the subjects’ adjusted INCAT score at every 
study visit according to schedule of assessments (see 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 #31 
Section 12.1.6 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, >
 
 should be recorded in the eCRF. 
Change #32 
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Clinical Study Protocol 
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CIDP01 
 
 
 
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Table 12-3 Laboratory measurements, footnote e 
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: 
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. 
Change #33 
Section 14.3.1 Analysis of the primary efficacy variable, fifth paragraph 
Different sensitivity analyses to evaluate the effect of missing observations on the primary 
analysis findings will be done. The first sensitivity analysis will be performed in the same way as 
the primary analysis, however for the PPS. A further analysis will utilize the last observation 
carried forward (LOCF) approach; missing values will be replaced by the last observed 
post-Baseline value of the variable and the analysis will be performed on the resulting dataset. 
Has been changed to: 
Different sensitivity analyses to evaluate the effect of missing observations on the primary 
analysis findings will be done. The first sensitivity analysis will be performed in the same way as 
the primary analysis, however for the PPS. A further analysis will utilize the last observation 
carried forward (LOCF) approach; missing values will be replaced by the last observed 
post-Baseline value of the variable and the analysis will be performed on the resulting dataset. 
An additional sensitivity analysis will be performed excluding any subject who received a 
mean dose more than ±10% from the intended dose. 
Change #34 
Section 14.4.1 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 #35 
Section 14.4.2 PK analyses, second paragraph, first sentence 
In contrast to the general descriptive display, concentration data will be summarized by treatment 
group and time point using the number of available observations, mean, median, SD, minimum, 
maximum, geometric mean (and associated 95% confidence intervals), 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 treatment 
group, actual dose received, and time point using the number of available observations, mean, 
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CIDP01 
 
 
 
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Page 125 of 128 
median, SD, minimum, maximum, geometric mean (and associated 95% confidence intervals), 
and geometric coefficient of variation (assuming log-normally distributed data). 
Change #36 
Section 14.4.3 PD analyses, first paragraph 
For all PD variables, descriptive statistics for the value, change from Baseline, and/or percentage 
change from Baseline will be tabulated by treatment group and 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 treatment group, actual dose received, and time 
point. 
Change #37 
Section 14.5 Handling of protocol deviations, first 3 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. 
Has 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 #38 
Section 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 #39 
Table 18-1 Suggested management guidelines for infusion reactions, first 2 reactions 
Type of reaction 
Sponsor recommendations for management 
Acute – Mild 
eg, flushing, dizziness, 
headache, sweating, 
palpitations, nausea 
Slow infusion rate to 
 
Infuse 
 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 10 minutes until back to Baseline. 
 
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Clinical Study Protocol 
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CIDP01 
 
 
 
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Page 126 of 128 
Type of reaction 
Sponsor recommendations for management 
, 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. 
Infuse 
 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 5 minutes until back to Baseline. 
Wait 20 minutes. 
 
 
 following this suggested regimen: 
Restart infusion at 
. 
 
, 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 
 
Infuse 
. 
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. 
Infuse 
 
Administer antihistamine iv/im. 
Administer paracetamol 1g orally. 
Monitor vital signs every 5 minutes until back to Baseline. 
Wait 20 minutes. 
 
 
 following this suggested regimen: 
Restart infusion 
 
, as tolerated until intended 
dose has been given. 
 
 as tolerated until 
intended dose has been given. 
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Clinical Study Protocol 
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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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CIDP01 
 
 
 
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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:
CIDP01-protocol-amendment-2
Version:
1. 0
Document Number:
CLIN-000136862
Title:
CIDP01 protocol amendment 2
Approved Date:
10 Jul 2019
Document Approvals
Approval
Verdict: Approved
Name: 
Capacity: Clinical
Date of Signature: 10-Jul-2019 14:57:27 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Subject Matter Expert
Date of Signature: 10-Jul-2019 15:10:38 GMT+0000
Approval
Verdict: Approved
Name: 
Capacity: Clinical
Date of Signature: 10-Jul-2019 15:43:51 GMT+0000
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SAP_001.pdf: 
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08 Apr 2021 
Statistical Analysis Plan 
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CIDP01 
 
 
 
Confidential 
Page 1 of 61 
  
STATISTICAL ANALYSIS PLAN  
Study: CIDP01 
Product: rozanolixizumab 
A MULTICENTER, RANDOMIZED, SUBJECT-BLIND, INVESTIGATOR BLIND, 
PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY EVALUATING THE 
EFFICACY, SAFETY, AND TOLERABILITY OF ROZANOLIXIZUMAB IN SUBJECTS 
WITH CHRONIC INFLAMMATORY DEMYELINATING 
POLYRADICULONEUROPATHY (CIDP) 
 
 
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. 
SAP/Amendment Number Date 
Final SAP 3.0 
08 Apr 2021 
 
 
 
 
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Statistical Analysis Plan 
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CIDP01 
 
 
 
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Page 2 of 61 
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 objectives ....................................................................................... 9 
2.1.3 
Exploratory objectives ..................................................................................... 9 
2.2 
Study variable(s) ............................................................................................ .. ..  10 
2.2.1 
Efficacy variable ................................................................................. ... ...... 10 
2.2.1.1 
Primary efficacy variable ........................................................ ........... 10 
2.2.1.2 
Other efficacy variable ................................................ .. ... . ..... .. ... 10 
2.2.2 
Other variables .................................................................... .. ........ ... .......... 11 
2.2.2.1 
Pharmacokinetic variable ................................... . .......... ................... 11 
2.2.2.2 
Pharmacodynamic variables ......................... . .. ....... . .. .................... 11 
2.2.2.3 
Exploratory pharmacogenetics variables . ......... . ......................... 12 
2.2.2.4 
Exploratory RNA, proteins, and metabolites variables ....................... 12 
2.2.3 
Immunological variables ........ .... ........ .............. ...................................... 12 
2.2.4 
Safety variables ................. ............... ... ............ ........................................... 12 
2.3 
Study design and conduct ... ...... ........ .. .. ...... ... ................................................ 13 
2.4 
Determination of sample size ............ ........ ... ..................................................... 15 
3 
DATA ANALYSIS CONSIDERATIONS .. . ........................................................... 15 
3.1 
General presentation of summaries and analyses .................................................. 15 
3.2 
General study level definitions ... ......................................................................... 16 
3.2.1 
Analysis time points .... .. ............................................................................ 16 
3.2.1.1 
Relative day .... .................................................................................... 16 
3.2.2 
Study periods .... . . ....................................................................................... 16 
3.3 
Definition of Baseline values ................................................................................. 17 
3.4 
Protocol deviations................................................................................................. 18 
3.5 
Analysis sets .......................................................................................................... 18 
3.5.1 
Enrolled Set ................................................................................................... 18 
3 5.2 
Randomized Set ............................................................................................. 18 
3.5.3 
Safety Set ....................................................................................................... 18 
3.5.4 
Full Analysis Set ............................................................................................ 19 
3.5.5 
Modified Full Analysis Set ............................................................................ 19 
3.5.6 
Per Protocol Set ............................................................................................. 19 
3.5.7 
Pharmacokinetic Per-Protocol Set ................................................................. 19 
3.5.8 
Pharmacodynamic Per-Protocol Set .............................................................. 19 
3.6 
Treatment assignment and treatment groups ......................................................... 20 
3.7 
Center pooling strategy .......................................................................................... 20 
3.8 
Coding dictionaries ................................................................................................ 20 
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3.9 
Changes to protocol-defined analyses ................................................................... 20 
3.9.1 
Changes related to COVID-19 ....................................................................... 21 
3.9.2 
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 .............................................................................. . .. ... 23 
4.2.5 
Rozanolixizumab concentration data ............................................... ... ......... 23 
4.2.6 
Electrocardiogram data .............................................................. . ......... . .. 23 
4.2.7 
Dates and Times .................................................................. . ......... . .. ....... 23 
4.2.8 
Handling of repeated and unscheduled measurements ..... .............. ............ 25 
4.3 
Handling of measurements obtained at the premature end of treatment visit ........ 26 
4.4 
Interim analyses and data monitoring .......... ............... ........... .. ......................... 26 
4.4.1 
Timing of interim analyses ............ ............... . .......... .. . ............................. 27 
4.4.2 
Data required for interim analyses .. ........ . ............ . ................................... 27 
4.5 
Multicenter studies ........................... ....... . . ....... . .......................................... 27 
4.6 
Multiple comparisons/multiplicity ......... .. ....... .. ................................................ 27 
4.7 
Use of an efficacy subset of 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 
Participant disposition .......... .. ............................................................................ 27 
5.2 
Protocol deviations. . ....... .. .. ............................................................................... 28 
6 
DEMOGRAPHICS AND OTHER BASELINE CHARACTERISTICS .................. 28 
6.1 
Demographics ......... . ............................................................................................ 29 
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 ............................................................... 31 
7 
MEASUREMENTS OF TREATMENT COMPLIANCE ......................................... 31 
8 
EFFICACY ANALYSES .......................................................................................... 31 
8.1 
Statistical analysis of the primary efficacy variable .............................................. 32 
8.1.1 
Derivations of primary efficacy variable ....................................................... 32 
8.1.2 
Analysis of the primary efficacy variable ...................................................... 32 
8.1.2.1 
Presentation of primary efficacy variable ........................................... 33 
8.1.3 
Supportive and sensitivity analyses of the primary efficacy variable ........... 33 
8.1.3.1 
Per-protocol analysis ........................................................................... 33 
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Statistical Analysis Plan 
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8.1.3.2 
LOCF ................................................................................................... 33 
8.1.3.3 
MFAS .................................................................................................. 33 
8.2 
Statistical analysis of the other efficacy variables ................................................. 34 
8.2.1 
CIDP relapse, MMRM and summary tables will be performed on the FAS 
and repeated for the PPS for iRODS, adjusted INCAT and clinician reported 
grip strength.CIDP relapse (iRODS) ............................................................. 34 
8.2.1.1 
Analysis of time to CIDP Relapse (iRODS) ....................................... 34 
8.2.2 
Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) and 
CIDP relapse .................................................................................................. 34 
8.2.3 
Maximum grip strength as assessed by site personnel and CIDP relapse .. .. 35 
8.2.4 
Patient-reported grip strength .............................................................. .. .....  36 
8.2.5 
CIDP relapse ................................................................................ ............... 37 
8.2.6 
RT-MRC sum score ................................................................ .. ............ ...... 37 
8.2.7 
CIDP PRO and Fatigue scale ............................................ . .. ...... .. ............. 38 
8.2.8 
PGIS and PGIC ............................................................ .. ........... ................ 38 
8.2.9 
Use of rescue medication ....................................... .. ....... ..... . .................... 39 
9 
PHARMACOKINETICS AND PHARMACODYNAMICS . .. . ............................ 39 
9.1 
Pharmacokinetics ................................
. ........... ... ....... . .. ................................ 39 
9.2 
Pharmacodynamics ........................ .... ......... . ......... ........................................... 40 
9.2.1 
Total Serum IgG ............... ................ .. ......... .. ........................................... 40 
9.2.2 
Neurofilament light chain .. ........ ... . ........ . ................................................. 40 
9.3 
Immunological analyses... . . ....... ............ . ...................................................... 40 
9.3.1 
Immunoglobulins ........... ............. ... ............................................................ 40 
9.3.2 
 ... .. ........ .. .................................................................. 41 
9.3.3 
Serum complement levels and plasma complement levels ............................ 42 
9.3.4 
Cytokines ....... . . .......... ... ........................................................................... 43 
9.3.5 
CIDP-specific auto-antibodies ....................................................................... 43 
9.3.6 
 ................................ 43 
10 SAFETY ANALYSES ............................................................................................... 43 
10.1 
Extent of exposure ................................................................................................. 43 
10.2 
Adverse events ....................................................................................................... 44 
10.3 
Clinical laboratory evaluations .............................................................................. 46 
10.3.1 
Potential drug-induced liver injury ................................................................ 47 
10.4 
Vital signs, physical findings, and other observations related to safety ................ 48 
10.4.1 
Vital signs ...................................................................................................... 48 
10.4.2 
Electrocardiograms ........................................................................................ 49 
10.4.3 
Suicidality ...................................................................................................... 50 
10.4.4 
Other safety variables .................................................................................... 50 
11 OTHER ANALYSES ................................................................................................ 51 
12 REFERENCES .......................................................................................................... 51 
13 APPENDICES ........................................................................................................... 52 
13.1 
Conversion of iRODS scores ................................................................................. 52 
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Statistical Analysis Plan 
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CIDP01 
 
 
 
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13.2 
Marked abnormality criteria for Rozanolixizumab program ................................. 54 
14 AMENDMENT(S) TO THE STATISTICAL ANALYSIS PLAN (SAP) ................ 56 
14.1 
Amendment 1 ......................................................................................................... 56 
14.1.1 
Rationale ........................................................................................................ 56 
14.1.2 
Changes .......................................................................................................... 56 
14.2 
Amendment 2 ......................................................................................................... 59 
14.2.1 
Rationale ........................................................................................................ 59 
14.2.2 
Changes .......................................................................................................... 59 
14.2.2.1 
Change 1 ........................................................................................... .. 59 
14.2.2.2 
Change 2 ..................................................................................... . .. ... 59 
14.2.2.3 
Change 3 ................................................................................ ... ......... 59 
14.2.2.4 
Change 4 .......................................................................... . ......... . .. 59 
14.2.2.5 
Change 5 .................................................................... . ......... . .. ....... 59 
14.2.2.6 
Change 6 ................................................................. .............. ............ 59 
14.2.2.7 
Change 7 ......................................................... . . ....... .. ................... 59 
14.2.2.8 
Change 8 .................................... ............... ........... .. ......................... 59 
14.2.2.9 
Change 9 .............................. ............... . .......... .. . ............................. 59 
14.2.2.10 
Change 10 ........................ .... ........ . ............ . ................................... 59 
14.2.2.11 
Change 11 ...................... ....... . . ....... . .......................................... 60 
14.2.2.12 
Change 12 .............. . .. ......... .. ....... .. ................................................ 60 
STATISTICAL ANALYSIS PLAN SIGNATURE PAGE ............................................... 61 
 
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Statistical Analysis Plan 
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CIDP01 
 
 
 
Confidential 
Page 6 of 61 
LIST OF ABBREVIATIONS 
 
 
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 
ANCOVA 
Analysis of covariance 
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 
Contract research organization 
CTCAE 
Common Terminology Criteria for Adverse Events 
CSP 
Clinical study protocol 
CSR 
Clinical study report 
C-SSRS 
Columbia Suicide Severity Rating Scale 
CV 
Coefficient of variation 
DBP 
Diastolic blood pressure 
DEM 
Data evaluation meeting 
DMC 
Data Monitoring Committee 
DNA 
Deoxyribonucleic acid 
ECG 
Electrocardiogram 
eCRF 
Electronic 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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Statistical Analysis Plan 
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CIDP01 
 
 
 
Confidential 
Page 7 of 61 
FCS 
Fully conditional specification 
FDA 
Food and Drug Administration 
FV 
Final Visit 
geoCV 
Geometric coefficient of variation 
geoMean 
Geometric mean 
GGT 
Gamma glutamyltransferase 
HDL 
High density lioprotein 
HIV 
Human immunodeficiency virus 
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 
LOCF 
Last observation carried forward 
logit 
Log odds unit 
LS 
Least square 
MA 
Markedly abnormal 
MAR  
Missing at random 
MCID-SE  
Minimum clinically important differences-standard error 
MedDRA 
Medical Dictionary for Regulatory Activities 
MFAS 
Modified full analysis set 
MMRM 
Mixed model repeated measures 
MNAR 
Missing not at random 
MRC 
Medical Research Council 
MRD 
Minimum required dilution 
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CIDP01 
 
 
 
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n 
Number of participants 
NF-L 
Neurofilament light chain 
OLE 
Open-Label Extension 
PD 
Pharmacodynamic 
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 
PPS 
Per-Protocol Set 
PT 
Preferred term 
QTcF 
QT corrected for heart rate using Fridericia’s formula 
RBC 
Red blood cell 
RNA 
Ribonucleic acid 
RT-MRC 
Rasch-built, modified interval Medical Research Council scale 
RS 
Randomized Set 
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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Statistical Analysis Plan 
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CIDP01 
 
 
 
Confidential 
Page 9 of 61 
1 
INTRODUCTION 
The purpose of this statistical analysis plan (SAP) is to provide all information that is 
necessary to perform the required statistical analyses of study CIDP01. It also defines the 
summary tables, figures and listings (TFLs) to be included in the final Clinical Study Report 
(CSR) according to the protocol. 
This final analysis SAP is based upon, and assumes familiarity, with the following 
documents: 
• Protocol Amendment 1: 15 Nov 2018 
• Protocol Amendment 2: 09 Jul 2019 
• Adverse Events of Focus (AEoF) for the rozanolixizumab Program: v1.3, 04 Aug 2020 
Unless 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: 2016-002411-17). 
If a future protocol amendment necessitates a substantial change to the statistical analysis of 
the study data, this SAP may be amended accordingly. If, after database lock, additional 
analyses are required to supplement the planned analyses described in this SAP, those 
unplanned analyses will not be described in an amended SAP but they will be described in a 
separate statistical analysis plan. However, if analysis definitions have to be modified or 
updated, a SAP amendment will be required. 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 evaluate the clinical efficacy of rozanolixizumab as a treatment for subjects with 
chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) 
2.1.2 
Secondary objectives 
The secondary objectives of the study are: 
• To evaluate the safety and tolerability of rozanolixizumab subcutaneous (sc) infusion in 
subjects with CIDP 
• To assess the pharmacodynamic (PD) effect of rozanolixizumab as measured by the total 
immunoglobulin G (IgG) concentrations in serum 
2.1.3 
Exploratory objectives 
Exploratory objectives are: 
• To evaluate the effects of rozanolixizumab on the concentration of total protein, albumin, 
α- and β-globulins, IgG subclasses, immunoglobulin M (IgM), immunoglobulin A (IgA), 
and immunoglobulin E (IgE), serum and plasma complement levels 
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Statistical Analysis Plan 
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• To evaluate the incidence and emergence of 
 with respect to 
immunogenicity and pharmacokinetic (PK) and PD 
• To evaluate the effect of rozanolixizumab on complement and cytokines 
• To assess the plasma concentrations of rozanolixizumab administered by sc infusion 
• To assess the PD effect of rozanolixizumab as measured by neurofilament light chain 
(NF-L) in serum 
• To assess the effect of rozanolixizumab on gene and protein expression, and explore the 
relationship between deoxyribonucleic acid (DNA), ribonucleic acid (RNA), protein, and 
metabolite biomarkers and cause, progression, and appropriate treatment of CIDP 
• To assess the effect of rozanolixizumab on CIDP-specific auto-antibody levels 
• To assess the effect of rozanolixizumab on vaccine antibody levels (
 
s) 
2.2 
Study variable(s) 
2.2.1 
Efficacy variable 
2.2.1.1 
Primary efficacy variable 
The primary efficacy variable is the: 
• Change from Baseline to Week 13 (Day 85) in inflammatory Rasch-built Overall 
Disability Scale (iRODS) score 
2.2.1.2 
Other efficacy variable 
Preliminary definitions linked to assessment of relapse: 
• CIDP relapse (iRODS) is defined as a clinically important deterioration from Baseline in 
iRODS score, ie, a 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 
Other efficacy variables will include: 
• Subject experienced CIDP relapse (iRODS) up to Week 13 (Day 85) after first treatment 
• Time to CIDP relapse (iRODS) during the Treatment Period 
• Values and change from Baseline in iRODS scores at each scheduled assessment during 
the Treatment and Observation Periods 
• Subject experienced CIDP relapse (adjusted INCAT) up to Week 13 (Day 85) after first 
treatment 
• Time to CIDP relapse (adjusted INCAT) during the Treatment Period 
• Values and change from Baseline in adjusted INCAT score at each scheduled assessment 
during the Treatment and Observation Periods 
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Statistical Analysis Plan 
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• Subject experienced CIDP relapse (maximum grip strength as assessed by site 
personnel) up to Week 13 (Day 85) after first treatment 
• Time to CIDP relapse (maximum grip strength as assessed by site personnel) during 
the Treatment Period 
• Values and 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 change from Baseline in daily maximum grip strength score (maximum of 3 
assessments) taken by the subject at the same time each day during the Treatment and 
Observation Periods 
• Values and change from Baseline in Rasch-built, modified interval Medical Research 
Council scale (RT-MRC) sum score at each scheduled assessment during the Treatment 
and Observation Periods 
• Values and change from Baseline in fatigue at each scheduled assessment during the 
Treatment and Observation Periods 
• Values and change from Baseline in CIDP patient reported outcome (PRO) instrument 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) at each scheduled assessment during the 
Treatment and Observations Periods 
• Subjects receiving rescue medication during Treatment Period 
• Time to rescue medication administration during Treatment Period 
2.2.2 
Other variables 
2.2.2.1 
Pharmacokinetic variable 
The PK variable is: 
• Plasma concentration of rozanolixizumab at each scheduled assessment during the 
Treatment Period 
2.2.2.2 
Pharmacodynamic variables 
The PD variables are: 
• Minimum value and maximum decrease (absolute and percentage) from Baseline in total 
serum IgG concentration during the study 
• Value and change (absolute and percentage) from Baseline in total serum IgG 
concentrations at each scheduled assessment during Treatment and Observation Periods 
• Value and change (absolute and percentage) from Baseline in serum IgG subclass 
concentrations at each scheduled assessment during Treatment and Observation Periods 
• Value and change (absolute and percentage) from Baseline in NF-L levels at each 
scheduled assessment during Treatment and Observation Periods 
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2.2.2.3 
Exploratory pharmacogenetics variables 
The exploratory pharmacogenetics variables are: 
• Genetic and epigenetic changes that may be measured to understand the cause, 
progression, and appropriate treatment of CIDP 
2.2.2.4 
Exploratory RNA, proteins, and metabolites variables 
The exploratory RNA, proteins, and metabolites variables are: 
• RNA, proteins, 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 
 
 
• 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. 
2.2.3 
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 
• 
 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 
 
 during Treatment and Observation Periods 
2.2.4 
Safety variables 
The safety variables for the study are: 
• Occurrence of treatment-emergent adverse events (TEAEs) 
• TEAEs leading to withdrawal of investigational medicinal product (IMP)  
• Vital sign values and changes from Baseline (systolic and diastolic blood pressure (BP), 
temperature, pulse rate, and body weight) at each scheduled assessment during Treatment 
and Observation Periods 
• 12-lead electrocardiogram (ECG) values and change from Baseline at each scheduled 
assessment during Treatment and Observation Periods 
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Statistical Analysis Plan 
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• 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 
• Serum and urine pregnancy 
• Columbia Suicide Severity Rating Scale (C-SSRS)  
• Headache questionnaire 
2.3 
Study design and conduct 
This is a Phase 2A, multicenter, randomized, subject-blind, investigator-blind, placebo-
controlled, parallel-group study evaluating the efficacy, safety, and tolerability of 
rozanolixizumab for treatment of subjects with CIDP. Eligible subjects will be adults who 
have a confirmed diagnosis of definite or probable CIDP according to the European 
Federation of Neurological Societies (EFNS)/ Peripheral Nerve Society (PNS) criteria for 
CIDP, have been shown to be dependent on treatment with immunoglobulin and who have 
remained on a stable dosing regimen of immunoglobulin therapy for at least 4 months prior to 
Screening. 
• Treatment Arm 1: rozanolixizumab 
 
 
• Treatment Arm 2: placebo sc 
 
 
Subjects will be randomized in a ratio of 1:1 to the 2 treatment arms. The randomization will 
take place on the day of first treatment with investigational medicinal product (IMP). The 
randomization will be stratified according to previous route of administration for Ig treatment 
(ie, subcutaneous immunoglobulin (SCIg) and intravenous immunoglobulin (IVIg)). 
The dose level of 
 may be reduced to 
 (or equivalent placebo) for an 
individual subject based on their tolerability. 
The study consists of: 
• Screening Period of between 2 and 5 weeks duration 
• The 11-week Treatment Period 
• The 12-week Observation Period (from 1 week up to maximum 24 weeks): 
− All subjects completing the Treatment Period (ie, all visits up to Visit 17 performed 
without a CIDP relapse) will be offered the possibility to enter in the Open-Label 
Extension (OLE) study, CIDP04, and be treated with rozanolixizumab. If they enter in 
the OLE, Visit 17 will be the last study visit. The Observation Period will be limited to 
1 week for these subjects. 
In all other instances the subjects will perform Visits 18 to 21 for efficacy and for 
safety assessments. Subjects will either return to the clinic/hospital, or, if possible and 
agreed by both investigator and subject, have home visits conducted by certified 
healthcare professionals for Visits 18 and 20. 
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− For subjects who complete the Treatment Period without relapse but choose not to 
enter the OLE directly and wish to first test whether they still need treatment, they will 
continue the Observation Period without standard of care treatment. 
− Subjects who do not wish to enter the OLE or test whether they still need treatment 
after having successfully completed the Treatment Period also have the opportunity to 
return immediately to standard of care for the duration of the Observation Period. 
− All other subjects (ie, having relapsed or withdrawn from the study during Treatment 
Period) will complete the Observation Period. Subjects can return to their standard of 
care for the duration of the Observation Period. 
− Stabilization: If the subject relapses during the Treatment Period or Observation Period 
(according to the medical judgement of the investigator supported by eg, subject’s 
score on iRODS, Inflammatory Neuropathy Cause and Treatment (INCAT), or 
maximum grip strength [assessed by site personnel]), the subject will return to the 
standard of care (ie, Ig treatment) as rescue medication at the time of relapse and will 
be stabilized over a period ranging from 2 weeks up to maximum 12 weeks starting 
from relapse visit (eg, premature end of treatment (PEOT) [Visit 17] or later visit). 
During the stabilization, visits should be performed every 1 to 3 weeks at the discretion of the 
investigator until the subject is stabilized. The stabilization may extend the Observation 
Period duration up to a maximum of 24 weeks after the last IMP dose.  
At the end of the stabilization: 
1. A subject who completes the Treatment Period without a CIDP relapse will be offered the 
possibility to enter in the OLE (CIDP04) and be treated with rozanolixizumab from that 
moment onwards. The subject will complete the Final Visit (FV) (V21) and enter the 
OLE on the same day. 
2. A subject who completes the Treatment Period and does not wish to enter the OLE 
(CIDP04) will continue their standard of care treatment after stabilization and the 
stabilization visit schedule (every 1 to 3 weeks) until they have a follow-up of 12 weeks 
after last IMP dose. The subject will complete the FV (V21) and enter the OLE on the 
same day.  
3. A subject who relapses during Treatment Period will continue their standard of care 
treatment after stabilization  They will continue the stabilization visit schedule (every 1 to 
3 weeks) until they have a follow-up of 12 weeks after last IMP dose. The subject will 
complete the FV (V21) and end CIDP01 study participation. These subjects 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 was confirmed they received placebo. 
Access to the OLE (CIDP04) is allowed for subjects completing the Treatment Period 
without relapse/withdrawal. Entry can occur immediately upon completion of the Treatment 
Period or after stabilization in case the subject relapsed during Observation Period and did 
not meet withdrawal criteria other than relapse. Subjects who relapse during the Treatment 
Period will have the opportunity to enter the OLE once all subjects have completed the study, 
the study is unblinded, and key results are available. In this case, only the subjects from the 
placebo arm will be allowed to enter the OLE study. 
Further details regarding the study design are provided in Section 5 of the clinical study 
protocol (CSP). 
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2.4 
Determination of sample size 
Due to the pilot character of the study, the sample size is chosen based on both the statistical 
considerations and practical limitations (eg, to recruit sufficient subjects in a reasonable time 
to achieve the objectives of the study) and to establish whether a positive trend towards active 
treatment can be established. For this reason, a one-sided exploratory test is considered 
appropriate in this setting.   
Since no data is currently publicly available on the primary outcome measure in this 
indication, the sample size is based on assumptions about the likely effect size. Thus, the 
sample size considerations are only based on assumptions for the effect size index (difference 
in group means/SD). When the sample size in each group is 15, a 1-sided 5.0% t-test for the 
comparison of rozanolixizumab and placebo will provide 80% power to detect a statistically 
significant difference in case there is a large effect of rozanolixizumab with an effect size 
index of 0.93. In case of a medium index (0.52) the power already decreases to 40%. It will 
be assumed that 10% of the randomized subjects cannot be utilized for the efficacy analysis; 
hence 34 subjects will be randomized. 
3 
DATA ANALYSIS CONSIDERATIONS 
3.1 
General presentation of summaries and analyses 
Statistical evaluation for the final analyses will be performed by PAREXEL. The 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 (Placebo and 
rozanolixizumab) and visit (where applicable) with the statistics mean, standard deviation 
(SD), median, minimum, and maximum. Categorical variables will be summarized by 
treatment group and 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: 
• For values where all 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 
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• If no 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. 
Analyses will be performed by treatment group and for all participants where stated. The 
treatment groups will be displayed as follows in the TFLs: 
• Treatment group 1: Placebo 
• Treatment group 2: Rozanolixizumab 
Participants with the reduced dose due to tolerability issues will be mapped under the same 
treatment groups. These participants will be flagged in the listings. For the Observation 
period the same treatment groups will be displayed. 
 
Data listings containing all documented data and all calculated data, as required for the 
variables and analysis applicable to the final analyses, will be generated. 
The visit type information (home or site visit) will be included into the listings. 
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. 
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] 
For events or measurements occurring after the date of the last sc infusion, relative day will 
be prefixed with ‘+’ in the data listings and 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 participant data listing. In such cases, relative day should be 
presented as ‘- -’ in the participant data listings. 
3.2.2 
Study periods 
The total duration of the study per subject is approximately 28 weeks (up to maximum 40 
weeks), consisting of: 
• Screening Period of a minimum of 2 to a maximum of 5 weeks starting with Screening 
Visit (to occur between Day -35 and Day -14, both inclusive). 
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• Treatment Period (11 weeks) – from first dose of IMP to last dose of IMP. The first 
treatment with IMP will occur at the Randomization Visit (Visit 2) and the dosing should 
occur as seamlessly as possible to continue the momentum of the Baseline therapy. 
• Observation Period (12 weeks up to 24 weeks) starting day after last IMP dose until study 
end. 
In case of early withdrawal, the duration of the Treatment Period may be shortened 
depending on the time to withdrawal. The Observation Period may be shortened to 1 week in 
case a subject completes the Treatment Period and enters the OLE study immediately. In case 
a subject relapses during the Observation Period, the subject will be stabilized over a period 
of minimum 2 weeks up to 12 weeks, possibly extending the Observation Period to a 
maximum of 24 weeks. 
The end of the study is defined as the date of the last visit of the last subject in the study. 
3.3 
Definition of Baseline values 
Baseline will be the last available pre-dose value prior to the first infusion of study drug in 
the Treatment Period, or if missing, the Screening values. Assessments with the same date as 
the first infusion, for which time was not planned to be captured, can be acceptable as 
Baseline assessments. 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. 
Table 3‒1: Expected Baseline 
Measurement 
Definition of Baseline 
Safety data: 
• 
Safety Lab (Clinical chemistry, 
Hematology, hsCRP, Urinalysis) 
• 
ECG 
• 
Vital signs 
• 
Total protein, albumin, α- and β-globulins 
Safety labs, vital signs, total protein, albumin, α- 
and β-globulin: Baseline visit (Day 1), if missing 
Screening visit. Pre-dose value at Baseline visit 
will be used for the vital signs. 
 
ECG: mean of triplicate values of Baseline visit. If 
missing Baseline visit (Day 1), mean of triplicate 
values of Screening visit. 
Efficacy data: 
• 
iRODS 
• 
INCAT 
• 
Grip strength (assessed by site personnel 
and patient reported) 
• 
RT-MRC and original MRC 
• 
Fatigue score 
• 
CIDP PRO 
• 
PGIS 
• 
PGIC 
Baseline visit (Day 1), if missing Screening visit 
 
For patient reported grip strength, Baseline value 
will be defined as the mean of three daily 
maximum grip strength scores taken by a 
participant during the last three non-missing days 
prior to the first IMP. Missing values will be 
handled as described in Section 4.2.1. 
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Table 3‒1: Expected Baseline 
Measurement 
Definition of Baseline 
Immunological variables: 
• 
IgA, IgE, IgM 
• 
Serum complement levels (C3, C4),  
• 
Plasma compliment (C3a, C5a)  
• 
 
• 
Cytokines 
• 
 
 
Baseline visit (Day 1), except for 
 
 where 
baseline is Screening visit 
PD variables: 
• 
Total serum IgG,  
• 
NF-L levels 
Total serum IgG: Baseline visit (Day 1), if missing 
Screening visit 
NF-L: Baseline visit (Day 1) 
; 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 important deviations will be identified and documented prior to unblinding to 
confirm exclusion from analysis sets. 
IPDs potentially related to the Coronavirus Disease 2019 (COVID-19) were 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 
The following analysis sets will be defined for this study:  
3.5.1 
Enrolled Set 
The Enrolled Set (ES) will consist of all participants who have given informed consent. 
3.5.2 
Randomized Set 
The Randomized Set (RS) will consist of all participants randomized into the study. 
3.5.3 
Safety Set 
The Safety Set (SS) will consist of all participants, who have received at least one dose of 
IMP. 
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Safety variables will be analyzed using the SS. 
It is expected that participants will receive treatment as randomized and hence safety analyses 
will be based on the randomized treatment group. However, if after unblinding it is 
determined that a participant has received treatment different to the treatment they were 
randomized to, then for safety analyses the participant will be allocated to the actual 
treatment they received. 
3.5.4 
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 17/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 
and MMRM, but kept in listings. 
The FAS is the primary analysis set for efficacy analyses. In the case of mistreatment, 
participants will be analyzed as treated if the participant received the incorrect treatment for 
the entire study period. In the case of partial mistreatment, patients should be analyzed as 
randomized, but considered as protocol violators. As for the SS, in the case of mistreatment 
participants will be primarily analyzed as treated. However, if applicable, sensitivity analyses 
will also be performed according to the randomized treatment group. 
3.5.5 
Modified Full Analysis Set 
The Modified Full Analysis Set (MFAS) will consist of all participants in the FAS, excluding 
any participant who received a mean dose more than ±10% (ie excluding any participant who 
received a mean dose of IMP >10% or <-10%) from the randomized (intended) dose.  
The definition of the randomized dose as well as details for calculations are provided in 
Section 10.1. 
3.5.6 
Per Protocol Set 
The Per-Protocol Set (PPS) is a subset of the FAS, consisting of those participants who had 
no important protocol deviation affecting the primary efficacy variable, as confirmed during a 
preanalysis data review meeting conducted prior to study unblinding. Post-Baseline 
deviations will not necessarily lead to total exclusion of a participant from the PPS but may 
lead to exclusion of specific data.  
Analysis of the primary efficacy variable will be repeated using the PPS. 
3.5.7 
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. 
3.5.8 
Pharmacodynamic Per-Protocol Set 
The Pharmacodynamic Per-Protocol Set (PD-PPS) is a subset of the FAS, 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.  
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3.6 
Treatment assignment and treatment groups 
Safety and efficacy analyses will be based on the actual treatment participants received. 
Analyses will be performed by treatment groups and for all participants (if applicable) as 
described in Section 3.1. 
3.7 
Center pooling strategy 
It is planned to recruit participants in Europe, United States of America and Canada in this 
study, with possible extension to other regions and countries. There will be no stratification 
by site or country performed. 
3.8 
Coding dictionaries 
Adverse events (AEs) will be coded using version 22.0 or later of the Medical Dictionary for 
Regulatory Activities (MedDRA®).  
Prior and concomitant 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 
• The definition of the SS has been updated to include all subjects who received at least 
one dose of the IMP. 
• FAS definition:  
o Study participants will be analysed as actually treated only if they received the 
incorrect treatment for the entire study period. In case of partial mistreatment, 
the participant will be analyzed as randomized. 
o The FAS definition is clarified being limited to valid primary efficacy results 
up to V17 only as the primary efficacy endpoint is the change in iRODS from 
baseline to V17.  
• 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 valid. 
• The protocol specifies the primary efficacy analysis covariate “prior Ig therapy” in 
section 14.3 1  Prior Ig therapy is defined by the stratification factor route of 
administration of prior Ig therapy. 
• The MFAS was included as an additional analysis set in the SAP and was not part of 
the final clinical study protocol (CSP). This was added as the sensitivity analysis, to 
ensure the robustness of the efficacy analysis results in relation to dose variation, as 
foreseen in the CSP. 
• Hy’s Law, defined in the protocol as  
≥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 
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. 
Protocol deviations will be presented by COVID-19 period (pre- or during). A post- COVID-
19 period may be added if applicable. 
3.9.2 
Interim Analysis 
 An interim analysis was planned according to protocol section 14 7 Planned interim analysis 
and data monitoring: 
“The fourth and final interim analysis will be performed once all subjects have attended 
Visit 17, the first visit of the Observation Period (ie, Day 85). This interim analysis will 
provide, at a minimum, the results for the primary variable “Change from Baseline to 
Week 13 (Day 85) in iRODS score.“ 
The event of all participants having attended Visit 17 is expected to coincide with the event 
of the last participant attending his/her last visit in CIDP01, resulting in database lock and the 
final analysis. With both triggers resulting in similar data, the interim analysis specified in the 
Interim Analysis SAP is not expected to provide additional value and will not be performed. 
4 
STATISTICAL/ANALYTICAL ISSUES 
4.1 
Adjustments for covariates 
The regression analysis model for efficacy evaluation will adjust for the following covariates: 
• Baseline score 
• Route of administration of prior Ig therapy 
4.2 
Handling of dropouts or missing data 
 
4.2 1 
Efficacy data 
For participants who prematurely withdraw for any reason before Week 13 (Day 85), data 
collected during the PEOT Visit will be used to impute iRODS, INCAT and grip strength at 
the closest missing visit. In case a participant discontinues the study prematurely, the 
assessment of the PEOT visit will be mapped to closest missing visit according to the visit 
schedule utilizing the relative day in study to a maximum of day 85 (Visit 17). However, if a 
participant receives rescue medication prior to the PEOT visit, the assessment will be 
regarded invalid and only listed. Assessments on the same day as first intake of rescue 
medication is acceptable for analysis.  
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Since the analysis is based on the mixed model repeated measures (MMRM) approach, visits 
beyond the PEOT Visit will not be imputed. Missing data in-between visits will be covered 
by the MMRM approach. 
For additional analyses of iRODS score to account for missing data, last observation carried 
forward (LOCF) and multiple imputation will be applied to address the issue of missing 
scores (see Section 8.1.3). There will be no imputation of missing individual item scores in 
the summaries for the iRODS; only to the overall iRODS score. 
For the analysis of relapse data, participants who withdraw from the study for any reason 
before Week 13 (Day 85) or who have missing data at Week 13 (Day 85) will be considered 
as relapsed and analysed as such. The same is true for participants administered an Ig 
infusion rescue medication during this time interval. 
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 and the issue will be covered by the MMRM approach. 
 
Sensitivity analyses will be performed for the primary efficacy parameter, as described in 
Section 8.1.3. 
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 
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 calculated 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 calculated. 
4.2.3 
Pharmacodynamic data 
Measurements BLQ are not anticipated for the total serum IgG data. In the event that any 
BLQ measurements are received, these will be regarded as missing for the calculation of 
descriptive statistics and changes from Baseline. 
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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 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 Day 1, 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. In the event that 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 
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• 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 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. 
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 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 participants or the date of 
discontinuation for 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 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 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 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 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. 
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: 
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• 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 
original values (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 the 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 
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 (including efficacy, 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 efficacy, 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 12-Weeks Observation period, will be 
mapped to the corresponding planned visit. Assessments on these visits will be flagged in the 
data listing. 
4.4 
Interim analyses and data monitoring 
A Data Monitoring Commitee (DMC) will be established to monitor mainly safety data 
during the study. The efficacy data will be assessed during the third interim analysis. The 
interim analyses will be unblinded and thus the live randomization code will be utilized in the 
datasets and TFLs.  
Details about the Interim analyses will be provided in a separate DMC SAP. 
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4.4.1 
Timing of interim analyses 
The following interim analyses are performed: 
• The first interim analysis will be conducted after 8 participants have received their first 4 
subcutaneous (sc) infusions and have attended the next visit (Visit 9). Based on this 
unblinded analysis the PK, PD and safety of rozanolixizumab will be assessed by the 
DMC 
• The second interim analysis will be conducted after 8 participants (receiving 12 sc 
infusions) have attended the first visit of the Observation Period (ie, Visit 17, the visit 1 
week after last dosing). Based on this unblinded analysis the PK, PD and safety of 
rozanolixizumab will be assessed by the DMC 
• The third interim analysis will take place after data from 8 additional participants are 
available, ie, after 16 participants have attended the first visit of the Observation Period. 
Based on this unblinded analysis the PK, PD and safety of rozanolixizumab will be 
assessed by the DMC 
− Following DMC recommendations from the second interim analysis, listings and 
descriptive summary statistics for iRODS, INCAT and site personnel reported grip 
strength will be included in the third interim analysis 
•  A fourth interim analysis was planned, but will not be performed. Please see 
Section 3.9.2 for details.  
If needed, subsequent DMC meetings will take place. The timing of further interim analyses 
and reviews of the data by the DMC will be decided by the DMC. Ad hoc DMC meetings can 
be held for reasons determined appropriate by the sponsor. 
4.4.2 
Data required for interim analyses 
The analyses and data required for review are described in the 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 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 
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 
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reason for discontinuation, discontinuation due to AEs, 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 (RS) 
• Participant analysis sets (ES) 
Participant disposition will be listed by treatment group based on the ES, and will include the 
date of informed consent, date of randomization, date and time of first and last dose of IMP, 
date of premature termination and primary reason, and date of final contact (if applicable).  
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 will be listed and plotted in a Kaplan-Meier-Curve for all 
discontinuations versus discontinuation due to COVID-19. 
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 RS, and will include the 
deviation type and description. The number and percentage of participants in the RS with 
IPDs will be summarized overall if appropriate. The denominator for percentages will be the 
number of participants in the RS  
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 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 RS. 
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 
 
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6.1 
Demographics 
A by-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, 
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 will be 
summarized for the RS. 
Body mass index in kg/m2 is calculated based on the height (in m) and the weight (in kg) 
measured at the Screening visit using the following formula: 
BMI (kg/m2) = weight (kg)/[height (m)] 2 
The BMI will be reported 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 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 Randomization visit will be listed and 
summarized for the RS by treatment group and for all participants. Listing will include the 
reported term, preferred term, dose, route of administration, frequency, formulation, start/stop 
dates and duration (in days). For definition of prior treatments see Section 6.4.1. 
Duration will be calculated as follows: 
Duration (in days) = ((End date of Ig treatment date – Start date of Ig treatment) +1) 
In addition, the following variables will be listed and summarized at baseline for the RS 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)  
• Duration of disease (years)  
Duration of disease (years) will be calculated as: 
𝐷𝑖𝑠𝑒𝑎𝑠𝑒 𝑑𝑢𝑟𝑎𝑡𝑖𝑜𝑛
= 𝐷𝑎𝑡𝑒 𝑜𝑓 𝑟𝑎𝑛𝑑𝑜𝑚𝑖𝑧𝑎𝑡𝑖𝑜𝑛−𝐷𝑎𝑡𝑒 𝑜𝑓 𝑖𝑛𝑖𝑡𝑖𝑎𝑙 𝐶𝐼𝐷𝑃 𝑑𝑖𝑎𝑔𝑛𝑜𝑠𝑖𝑠+ 1
365.25
 
If the date of initial CIDP diagnosis is partial, it should be imputed to the most 
recent feasible date (ie, last day of the month if only day is missing, or the last day 
of the year if day and month are missing). Note that if the date of randomization is 
missing then the duration of disease will be derived using the date of screening. 
6.3 
Medical and procedures history 
Past medications will include any medications that started and stopped before the first 
administration of IMP. Medical history will be listed and summarized for the RS by treatment 
group and will include the MedDRA system organ class (SOC), high level term (HLT) and 
preferred term (PT). Procedure history and concomitant medical procedures will be listed 
based on the RS. 
History of CIDP will be listed for RS. 
6.4 
Prior and concomitant medications 
Prior and concomitant medications will be listed and summarized for the RS by treatment 
group and for all 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). 
• Prior medications will include any medications that started before the first 
administration of IMP. 
• Prior and concomitant medications will include any medications that started prior to 
dosing and continued after. 
• Concomitant medications will include any medications that have been taken at least 
once after the first administration of IMP during the Treatment and/or Observation 
Period. 
6.4.1 
Prior medication definition 
Prior medications will include any medications that started prior to the first administration of 
IMP. This includes any medications that started and stopped prior to dosing as well as those 
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which started prior to dosing and continued after (classified as prior and concomitant 
medications). 
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) during the Dosing 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 17/PEOT (non
inclusively). This includes medications that started prior to the first IMP and continued 
during Treatment Period and those that continued into the Observation Period. 
• Observation Period: a medication will be assigned to Observation Period if it has been 
taken at least once between day 85 (Visit 17/PEOT) and the Final visit or end of study 
participation. 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, 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 (i.e. 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 analyses of the efficacy variables will be performed on the FAS. Additional outputs may 
be repeated for the PPS or the MFAS where indicated. Listings will be based on the SS. 
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Treatment group assignment for efficacy analysis will be according to the actual treatment 
regimen. 
All efficacy analyses are to be interpreted as exploratory analyses. No p-values will be 
presented. 
Any efficacy measurements are considered invalid, if the participant received rescue 
medication before the assessment. Assessments collected at the same day as first intake of 
rescue medication are considered valid. Respective measurements will not be used for 
summary tables and MMRM, but kept in listings. This includes iRODS, INCAT, clinician 
reported grip strength, RT-MRC, CIDP PRO and Fatigue, PGIS, PGIC and the patient 
reported grip strength as derived in Section 8.2.4. 
8.1 
Statistical analysis of the primary efficacy variable 
The primary efficacy variable described in Section 2.2.1.1 will be analyzed using the logit-
transformed iRODS score to compute the change from Baseline to Week 13 (Day 85).  
8.1.1 
Derivations of primary efficacy variable 
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 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 provided in tables and key results will be presented in transformed and original 
units to aid interpretation based on the raw scores. 
8.1.2 
Analysis of the primary efficacy variable 
The primary analysis will be based on a MMRM analysis (fixed effect terms: treatment 
group, the Baseline location iRODS score, route of administration of prior Ig therapy (as 
captured in the eCRF Prior and Concomitant Medication form), assessment week (at pre-
specified levels determined by the visit schedule) and the treatment by week interaction and 
study participant as a random effect, and utilize an unstructured covariance matrix).  
The Kenward-Roger approximation will be used to estimate denominator degrees of freedom. 
In case of non-convergence, the model will be fitted using the following covariance matrices 
until convergence is met: Heterogeneous Toeplitz, Heterogeneous autoregressive, Toeplitz, 
Autoregressive.  
SAS proc MIXED will be used: 
a. Subject ID, treatment group, prior Ig therapy and visit will be set as classes 
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b. The model will be specified with covariates baseline score, treatment group, prior 
Ig therapy, visit and treatment*visit 
c. The repetition variable is visit. 
d. Subject ID is set as random factor. 
e. Covariance structure as described above. 
f. LS means, differences and confidence limits for treatment*visit are requested. 
An estimate for the placebo-adjusted treatment effect with 90% CI will be produced on logit 
location scale, on the raw scores as well as centile metric scores. 
Imputation rules are given in Section 4.2.1. 
8.1.2.1 
Presentation of primary efficacy variable 
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.  
The summary of the primary MMRM procedure results at each week, i.e. Least Square (LS) 
Means along with their standard error (SE), LS Mean difference and its 2-sided 90% CI 
corresponding to the treatment group difference at each week, will also be presented in the 
descriptive statistics for raw sum score, logit location and centile metric score. 
Mean change from Baseline including 90% CI of the centile metric score will be plotted 
against timepoint for both treatments. 
8.1.3 
Supportive and sensitivity analyses of the primary efficacy 
variable 
Different sensitivity analyses to evaluate the effect of missing observations on the primary 
analysis findings will be performed. This applies to summary of observed results and MMRM 
for iRODS.  
8.1.3.1 
Per-protocol analysis 
The first supportive analysis will be performed in the same way as the primary analysis, using 
the PPS.  
8.1.3.2 
LOCF 
A further analysis will utilize the LOCF approach; a missing value will be replaced by the 
most recent previously observed post-Baseline value of the variable up to day 85 and the 
analysis will be performed for the FAS. In case a participant discontinues the study 
prematurely, the assessment of the PEOT visit will be mapped to next missing visit according 
to the visit schedule utilizing the relative day in study to a maximum of day 85 (Visit 17) 
before applying LOCF. 
8.1.3.3 
MFAS 
In addition, a further exploratory sensitivity analysis to ensure the robustness of the results in 
relation to dose variation will be conducted for the primary endpoint, using the MFAS. 
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8.2 
Statistical analysis of the other efficacy variables 
8.2.1 
CIDP relapse, MMRM and summary tables will be performed on 
the FAS and repeated for the PPS for iRODS, adjusted INCAT and 
clinician reported grip strength.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" = 𝑆𝑡𝑎𝑛𝑑𝑎𝑟𝑑𝑖𝑧𝑒𝑑 𝑣𝑎𝑙𝑢𝑒𝑉𝑖𝑠𝑖𝑡−𝑆𝑡𝑎𝑛𝑑𝑎𝑟𝑑𝑖𝑧𝑒𝑑 𝑣𝑎𝑙𝑢𝑒𝑏𝑎𝑠𝑒𝑙𝑖𝑛𝑒
√𝑆𝐸𝑉𝑖𝑠𝑖𝑡
2
+ 𝑆𝐸𝐵𝑎𝑠𝑒𝑙𝑖𝑛𝑒
2
 
Values and changes from Baseline in iRODS scores 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. 
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). 
Analyses will be performed for the FAS utilizing the observed cases. 
Sensitivity analysis for relapse will be performed in the same way, however for the PPS.  
The rule for handling missing data is described in Section 4.2.1. 
8.2.1.1 
Analysis of time to CIDP Relapse (iRODS) 
The time to CIDP relapse (iRODS) is defined as the time from starting treatment (Day 1) to 
achievement of iRODS and will be displayed using a Kaplan-Meier curve.  
Summary tables will include descriptive statistics. 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 withdrawing from the study 
will be handled as relapsed (see Section 4.2.1). 
For the purpose of the analysis participants with no relapse will be censored at the end of the 
study. 
Analyses will be performed for the FAS and repeated for the PPS. 
8.2.2 
Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) 
and CIDP relapse 
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. 
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The investigator (or qualified personnel) will record the participant’s 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’s 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. 
 
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 
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.  
Values and changes from Baseline in adjusted INCAT 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. For the change from Baseline at each 
scheduled assessment during the Treatment and Observation Period, treatment differences 
will be compared using the same MMRM analysis as for the primary endpoint, with the fixed 
categorical effects of treatment group, assessment week (at pre-specified levels determined 
by the visit schedule), the treatment by week interaction and the route of administration of 
prior Ig therapy, as well as continuous fixed covariate of the Baseline value and participant as 
a random effect. Analyses will be performed for the FAS utilizing the observed cases and 
repeated for the PPS. 
INCAT score measurements will be listed by treatment group. 
The statistical methods applied to CIDP relapse (adjusted INCAT) and time to CIDP relapse 
(adjusted INCAT) will be the same as for CIDP relapse (iRODS), see Sections 8.2.1 and 
8.2.1.1.  
The rule for handling missing data is described in Section 4.2.1. 
Mean change from Baseline in adjusted INCAT score, including 90% CI will be plotted 
against timepoint for both treatments. 
8.2.3 
Maximum grip strength as assessed by site personnel and CIDP 
relapse 
Grip strength is assessed by qualified site personnel each scheduled assessment. Qualified 
site personnel will be trained regarding obtaining and recording measurements. At each 
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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. 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 by treatment group during the Treatment and Observation Periods. 
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. 
Values and changes from Baseline in maximum grip strength score taken by site personnel 
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.  
For the change from Baseline in maximum grip strength at each scheduled assessment during 
the Treatment and Observation Period, treatment differences will be compared using the 
same MMRM analysis as for the primary endpoint, with the fixed categorical effects of 
treatment group, assessment week (at pre-specified levels determined by the visit schedule), 
the treatment by week interaction and the route of administration of prior Ig therapy, as well 
as continuous fixed covariate of the Baseline value, and participant as a random effect. 
Analyses will be performed for the FAS utilizing the observed cases and repeated for the 
PPS. 
Values of grip strength score taken by site personnel will be listed by treatment group. All 3 
assessments taken at the scheduled assessments will be listed and the maximum per 
scheduled assessment will be flagged. 
The statistical methods applied to CIDP relapse (maximum grip strength as assessed by site 
personnel) and time to CIDP relapse (maximum grip strength as assessed by site personnel) 
will be the same as for CIDP relapse (iRODS), see Sections 8.2.1 and 8.2.1.1. The rule for 
handling missing data is described in Section 4.2.1. 
The analyses on CIDP relapse and time to CIDP relapse will be repeated with a CIDP relapse 
definition based on a decline of >8kPa  
Mean change from Baseline in maximum grip strength score, including 90% CI will be 
plotted against timepoint for both treatments. 
8.2.4 
Patient-reported grip strength 
During the study, participants keep e-diaries to record daily grip strength from the Screening 
Visit until the end of study participation. Participants should be reminded to bring their 
diaries with them to each clinic visit and to have them available at home nursing visits. 
Participants will be trained by the site personnel and receive a manual describing how to 
assess grip strength using a standardized tool. At approximately the same time every day, the 
maximum grip strength, the peak pressure generated by the participant, will be assessed 3 
times, with at least 30sec between measurements, using the dominant hand. Participants will 
be asked to complete their diaries each day and record the values for each of the 3 
assessments. 
For the purpose of the analysis, the mean of the maximum of the 3 daily assessments taken 
during the last three non-missing days prior to the scheduled visit will be used. Missing data 
will be handled as described in Section 4.2.1.  
Values and changes from Baseline in mean daily maximum grip strength score taken by the 
participant will be summarized by treatment group and for all participants and listed by 
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treatment group at each scheduled visit during the Treatment and Observation Period. The 
treatment differences of change from baseline in the derived grip strength score will be 
compared using the same MMRM analysis as for the primary endpoint, with the fixed 
categorical effects of treatment group, assessment week (at pre-specified levels determined 
by the visit schedule), the treatment by week interaction and the route of administration of 
prior Ig therapy, as well as continuous fixed covariate of the Baseline value, and participant 
as a random effect. Analyses will be performed for the FAS utilizing the observed cases and 
repeated for the PPS. 
Values of grip strength score taken by the participant as well as derived scores will be listed 
by treatment group. All 3 daily assessments taken at the scheduled assessments will be listed 
and the maximum per scheduled assessment will be flagged. 
In addition to the above the daily maximum values will be displayed graphically over time, 
i.e. each treatment group’s mean value and 90% CI of the maximum of the participants’ three 
daily grip strength measurements will be displayed versus the relative day. 
8.2.5 
CIDP relapse 
The event of CIDP relapse is defined by 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 8.2.1, 8.2.2 and 8.2.3 and ‘other’ as 
captured in the CRF by the investigator. It will be described and analysed in the same way as 
the individual relapse definitions. This will be done for the FAS and the PPS. 
 
8.2.6 
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. 
For the change from Baseline at each scheduled assessment during the Treatment and 
Observation Period, treatment differences will be compared  using the same MMRM analysis 
as for the primary endpoint, with the fixed categorical effects of treatment group, assessment 
week (at pre-specified levels determined by the visit schedule), the treatment by week 
interaction and the route of administration of prior Ig therapy, as well as continuous fixed 
covariate of the Baseline value, and participant as a random effect. Analyses will be 
performed for the FAS utilizing the observed cases. 
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8.2.7 
CIDP PRO and Fatigue scale 
The CIDP PRO instrument consists of 2 domain scales;  
• Pain Severity numeric rating scale 
• 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. 
Following psychometric evaluation and refinement (eg, reduction in number of items) of 
these domain scales by using data generated in CIDP01, transformed interval 0-100 scoring 
will be provided for each domain score with higher scores reflecting higher levels of fatigue. 
A psychometric analysis plan will be created, which will detail all the exploratory and 
correlation analyses between the newly developed PROs and clinical variables. The PROs 
will also be participant to a Rasch analysis. The results of the psychometric analyses will be 
reported separately from the clinical study report (CSR). This analysis is not in scope of this 
SAP.  
8.2.8 
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 subjects 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. 
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8.2.9 
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), see Sections 8.2.1 and 8.2.1 1. The 
rule for handling missing data is described in Section 4.2.7 . 
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 treatment rozanolixizumab by exposure (%) 
categories (see section 10.1) and time point 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.  
Concentration data will be summarized for treatment rozanolixizumab by time point and 
 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 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 participant, individual concentration versus time (week) profiles will be presented 
graphically in linear and semi-logarithmic scale. In addition, combined individual (spaghetti 
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plots) concentration versus time profiles will be presented in linear and semi-logarithmic 
scale with all participants overlaid on the same figure.  
All figures will include the LLOQ on the semi-logarithmic plots. 
9.2 
Pharmacodynamics 
The analysis of the 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 for absolute values, changes from 
Baseline and percentage changes from Baseline.  
Further descriptive summaries (including n, arithmetic mean, median, SD, minimum and 
maximum) will be presented by treatment group, timepoint and 
 status (6 categories; see 
Section 9.3.2) for absolute values, changes from Baseline and percentage changes from 
Baseline. 
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 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. In the event that a decrease from Baseline in IgG is not 
observed in a given participant, the maximum decrease will be reported as the smallest 
increase from Baseline. 
9.2.2 
Neurofilament light chain 
NF-L levels in serum will be listed by treatment group and timepoint including changes from 
Baseline. Descriptive summaries will be presented by treatment group and timepoint for 
absolute values, changes from Baseline and percentage changes from Baseline. 
Participant NF-L levels in serum will be plotted against planned time. Mean NF-L levels, 
mean change from Baseline and percentage change from Baseline will be plotted against 
planned time by treatment group. 
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 treatment group and timepoint 
including changes from Baseline. Descriptive summaries will be presented by treatment 
group and timepoint for both absolute values and changes from Baseline. 
Individual figures over time (absolute value) will be presented by participant, with all 
variables overlaid on the same plot and separate plots each participant. 
Any values that are BLQ or ALQ will be handled as described in Section 4.2.4. 
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9.3.2 
 
 will be measured using a three-tiered assay approach: 
screening assay, confirmatory assay and titration assay. The results for the 
 
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 immunodeplet on 
positive). Samples that are confirmed as positive will be evaluated in a titration assay to 
quantify the 
 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 
 sample status should be determined for each visit where samples were taken for 
 analysis: 
- Sample values that are either negative screen or positive screen and negative 
immunodepletion will be defined as 
 negative 
- Sample values that are positive screen and positive immunodepletion will be defined as 
 positive. 
There will be six 
 participant status classifications   
(1) For participants who are negative at baseline, and antibody negative at all sampling points 
post treatment (including FV)- pre 
 negative- treatment induced 
 negative  
(2) For participants who are negative at baseline, and antibody positive at any sampling point 
post treatment (including FV)- pre 
 negative- treatment induced 
 positive. If a 
participant has a missing pre-treatment sample (either missing or insufficient volume) at 
baseline with one or more 
 positive post-treatment samples will be also classified as 
pre 
 negative- treatment induced 
 positive   
(3) For participants who are positive at baseline, and antibody negative at all sampling points 
post treatment (including FV)- pre 
 positive- treatment reduced 
 
(4) For 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. ≤ then a 
predefined fold difference from the baseline value)- pre 
 positive - treatment 
unaffected 
  
(5) For 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 
 positive- treatment boosted 
 positive. 
(6) For participants who have a positive pre-treatment sample and some post-treatment 
samples are missing, while other post-treatment samples are 
 negative, the 
participant will be classed as inconclusive. 
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•  A participant will be classified as having treatment-induced 
 positivity when 
meeting one of the following criteria: 
1. The Baseline result is 
 negative and at least one post-Baseline timepoint is 
 positive 
2.  The Baseline result is 
 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) 
The available 
 sample results will be listed. The listing will include rozanolixizumab 
concentration, 
 sample result 
 negative or positive), 
together with the confirmatory result (% inhibition) and 
 titer for results that are 
positive. In addition, the time since the previous administration of 
IMP will be reported (in days). 
A study participant will be considered  
• Overall 
 Positive if having at least one scheduled assessment that is confirmed 
positive during the treatment period (ie, classifications (2) to (6)). 
• Overall 
 Negative if having no scheduled assessments that are confirmed 
positive at any time in the treatment period (ie, classification (1)). 
Number and percentage of 
 positive and negative participants at the time of each 
scheduled assessment and overall will be summarized based on the SS. 
The first occurrence of treatment-induced 
 positivity (based on the definitions above) 
will be summarized (number and percentage of participants) at each post-Baseline scheduled 
assessment, based on the SS. This tabulation will count the number of participants at each 
post-Baseline visit who fulfill at least one of the above defined criteria for treatment-induced 
positivity; participants will be counted in the numerator based on the earliest visit at which 
one of these criteria is fulfilled. At other visits, 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 
 status by the six categories, defined above, will be tabulated by treatment group, 
with an additional category "total treatment induced" combining participants who are pre 
 negative- treatment induced 
 positive, participants with a missing pre-treatment 
sample (either missing or insufficient volume) at baseline with one or more 
 positive 
post-treatment samples and participants pre 
 positive- treatment boosted 
 positive. 
The prevalence of immunogenicity, defined as (cumulative) proportion of participants having 
positive immunodepletion 
 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 
 titer results as well as 
 
titer results and percentage change from baseline in Total IgG by the six 
 classes 
described above will be presented. Spaghetti plots of 
 titer versus time will be presented 
for all 
 positive 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. Descriptive summaries will 
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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. 
9.3.4 
Cytokines 
Serum cytokines will be listed by treatment group and time point, changes from Baseline will 
be added to the listing. Descriptive summaries will be presented by treatment group and time 
point for both absolute values and changes from Baseline, both for all participants and for 
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 
 
 will be listed by treatment group and 
time point. Descriptive summaries 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. 
10 
SAFETY ANALYSES 
All safety analyses will be presented using the SS  Listings will be presented by treatment 
group and participant; tabulations will be presented by visit/timepoint (if applicable) and 
treatment group. 
10.1 
Extent of exposure 
Eligible participants will be randomized 1:1 to receive rozanolixizumab 
 or placebo. 
Randomized (intended) dose will be calculated based on participant’s weight at screening as 
follows: 
Randomized dose (mg)= weight at screening (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% 
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The exposure categories will be used for the definition of the MFAS (Section 3.5.5) as well 
as for 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 or 
any unresolved event already present before the first administration of IMP that worsens in 
intensity following exposure to treatment until 8 weeks following the last administration of 
IMP. Adverse events starting before the first administration of IMP 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: 
1. 
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 on Day 1, up to Visit 17/PEOT (non-inclusively). 
2. 
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 17/PEOT till 8 weeks 
after administration of the last IMP. 
• The Observation Period’s TEAEs will be stratified by TEAEs of 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 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 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 
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• 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 
• 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 participants, percentages of participants in parentheses 
and the number of events. A participant who has multiple events in the same SOC, HLT and 
PT will be counted only once in the 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 active 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 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: 
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• Headache using the UCB-defined search criteria. 
• Gastrointestinal disturbances using the UCB-defined search criteria. 
• Hypersensitivity reactions using the criteria SMQ=’Hypersensitivity’ 
• 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 13.2. 
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 final 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 
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Table 10‒1: 
Laboratory measurements 
Category 
Panel 
Variable 
 
White blood cell 
differential 
Absolute counts: ANC, basophils, eosinophils, ALC, 
monocytes 
Percentages: neutrophils/leukocytes, 
basophils/leukocytes, eosinophils/leukocytes, 
lymphocytes/leukocytes, monocytes/leukocytes 
 
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 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, >10 x ULN, >20 x ULN 
• Alanine aminotransferase (ALT): >3 x ULN, >5 x ULN, >10 x ULN, >20 x ULN 
• AST or ALT: >3 x ULN, >5 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 
• Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≥5x Upper limit of 
normal (ULN)   
• (ALT or AST increase ≥3x ULN) and Total bilirubin ≥2x ULN 
• AST or ALT ≥ 3 x ULN 
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• 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 >3x ULN) and Total bilirubin >2x ULN and ALP <2 x ULN (Hy’s 
Law) 
• (ALT or AST ≥3x ULN) and (eCRF question for “Is the subject exhibiting symptoms of 
hypersensitivity” = Yes or eCRF question for “Is the subject exhibiting symptoms of 
Hepatitis” = Yes at the same date +/-2 days) 
• (ALT or AST) ≥3xULN (and ≥2x Baseline) and <5xULN and Total bilirubin <2xULN 
and (eCRF question for “Is the subject exhibiting symptoms of hypersensitivity”= No and 
eCRF question for “Is the subject exhibiting symptoms of Hepatitis”= No at the same 
date +/-2 days) 
• (ALT or AST) ≥5xULN (and ≥2x Baseline) and Total bilirubin <2xULN (eCRF question 
for “Is the subject exhibiting symptoms of hypersensitivity”= No and eCRF question for 
“Is the subject exhibiting symptoms of Hepatitis”= No at the same date +/ 2 days) 
The laboratory results listing will flag 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-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 Table 10‒2. 
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, participants who missed treatments will not be 
summarized  
Table 10‒2: 
MA Criteria for Vital Signs 
Variable 
Unit 
Low 
High 
Systolic blood pressurea 
mmHg 
≤ 90 and a decrease from 
Baseline of ≥20 
≥ 180 and an increase from 
Baseline of ≥20 
Diastolic blood pressurea 
mmHg 
<50 and a decrease from 
Baseline of ≥15 
>105 and an increase from 
Baseline of ≥ 15 
Pulse ratea 
bpm 
<50 and a decrease from 
Baseline of ≥15 
>120 and an increase from 
Baseline of ≥15 
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Table 10‒2: 
MA Criteria for Vital Signs 
Variable 
Unit 
Low 
High 
Temperature 
°C 
 
>101 0F (38.3 0C) 
Body Weight 
1 
≥ 10% decrease from Baseline 
≥ 10% increase from Baseline 
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 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 
• QT corrected for heart rate using Fridericia’s formula (QTcF = QT/(RR^1/3)) 
The individual measurements and the mean of the triplicate measurements will be reported in 
the by-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 timepoint. The listing will also include a flag for values identified as MA/PCS as 
defined by the following criteria. 
Table  10‒3: 
MA/PCS criteria for Electrocardiograms 
Parameter 
Abnormality Criteria 
QT interval (ms) 
 
>=500ms 
>=60ms increase from Baseline 
QTc(F) (ms) 
>=500ms 
>=60ms increase from Baseline 
PR interval (ms) 
Treatment-emergent value >200ms 
QRS interval (ms) 
Treatment-emergent value >100ms 
Heart rate (bpm) 
<50bpm 
>120bpm 
 
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). 
Summarization will be as treated, participants who missed treatments will not be 
summarized. 
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: 
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• <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 participant has a positive response to the suicidal ideation query. Results of suicidal 
ideation and C-SSRS will be listed by treatment group, 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 Chest X-Ray will be listed  
Results of interferon-gamma release assay (IGRA) tuberculosis test will be listed. 
Physical and neurological examination findings will be listed. 
Results of serology testing for human immunodeficiency virus (HIV), Hepatitis B and 
Hepatitis C will be listed. 
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). 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 
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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 RNA, proteins, and metabolites variables samples date collection will be listed. 
For the presentation of the data for the interim analyses, each participant will be presented 
separately in the form of a patient profile which will include the following information: 
• Participant demographics (age, gender, race, BMI, body weight at Screening) 
• Treatment group 
• Medical history (including procedure history) 
• Prior and concomitant medication 
• Drug administration (including whether performed at site or at home) 
• AEs 
• Safety laboratory variables (individual figures over time by panel [hematology, 
clinical chemistry, coagulation] and variable) 
• Vital signs variables (individual figures over time by variable) 
• ECG variables (individual figures over time by variable) 
• IgG (individual figures over time for observed values and changes from Baseline) 
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 
16 
-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 
32 
1.36 
55 
0.45 
33 
1.58 
57 
0.46 
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Page 53 of 61 
R-ODS summed 
raw score 
Rasch person location 
centile metric 
Corresponding SE 
(logits) 
SE= 0.4248 – 0.0016*location + 
0.0140*location2 
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 
  
 
 
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Page 54 of 61 
13.2 
Marked abnormality criteria for Rozanolixizumab program 
The following criteria will be applied in the determination of marked abnormalities for 
laboratory assessment values for the clinical studies within the Rozanolixizumab clinical 
development program. These values are based on the CTCAE criteria version 5, 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  
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 
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 
<40 mg/dL; <2.2 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 
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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 
Immunoglobulin 
G (IgG) 
g/L 
g/L 
≤ 1 g/L 
Hematology 
Hemoglobin  
g/dL 
 
g/L 
<8.0 g/dL; <80 g/L  
Hematology 
WBC 
(Leukocytes) 
109/L 
109/L  
Low: <2.0 x 109/L  
High:  >100 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   
Hematology 
Platelets 
109/L 
109/L  
<50.0 x 109/L  
 
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Page 56 of 61 
14 
AMENDMENT(S) TO THE STATISTICAL ANALYSIS PLAN 
(SAP) 
14.1 
Amendment 1 
14.1.1 
Rationale 
Following the clinical study protocol amendment 2, the SAP was updated to reflect these 
changes and add detail to some analysis descriptions in this document. Changes were made to 
harmonize safety results across the IMP’s program. 
14.1.2 
Changes 
 
Change #1 
Section 3.4, 3.9, 4.9, 5.2, 6: 
COVID-19 protocol deviations were introduced and will be listed and summarized. 
Demographics, baseline characteristics and prior and concomitant medical conditions will be 
summarized by COVID-19 period. 
 
Change #2 
Section 3.5.3: 
The Safety Set has been updated to include all subjects who received at least one dose of the 
IMP. 
 
Change #3 
Section 3.5.4, 3.9: 
FAS definition: Study participants will be analysed as actually treated only if they received 
the incorrect treatment for the entire study period. In case of partial mistreatment, the 
participant will be analyzed as randomized. 
 
Change #4 
Section 3.5.5 and 8.1.4: 
A modified full analysis set has been introduced to analyze the sensitivity to IMP dose 
variations. 
 
Change #5 
Section 4.2.1 and 8.2.4: 
Patient reported grip strength at a visit is calculated as the mean of three daily maximum grip 
strength scores so data can be analyzed by visit and will be analyzed using MMRM. 
 
Change #6 
Section 6.1: 
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Region, country, body weight categories at screening added to demographics. 
 
 
Change #7 
Section 6.2: 
Duration of disease (years) has been added to baseline characteristics. 
 
Change #8 
Section 7: 
Treatment compliance is determined by percent of actual to planned dose administered  
 
Change #9 
Section 8.1.2: 
Definition of fixed effects of the MMRM have been clarified  In case of non-convergence of 
the model, a sequence for alternative covariance matrix assumptions has been defined. 
 
Change #10 
Section 8.1.2.1: 
The MMRM estimates will be summarized. 
 
Change #11 
Section 8.1.3: 
The description of MI and LOCF sensitivity analyses have been detailed. P-values will not be 
displayed for sensitivity analyses. 
 
Change #12 
Section 8.2.2: 
The adjusted INCAT score derivation has been detailed and is also analyzed using MMRM. 
A graphic representation will be created. 
 
Change #13 
Section 8.2.3: 
Maximum grip strength as assessed by site personnel is also analyzed using MMRM. A 
graphic representation will be created.   
 
Change #14 
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Section 8.2.6: 
RT-MRC ANCOVA model was aligned with primary MMRM. 
 
Change #15 
Section 8.2.7: 
“Following psychometric evaluation and refinement (eg, reduction in items) of these domains 
scales by using data generated in CIDP01, transformed interval 0-100 scoring will be 
provided for both domain scores with higher score reflecting higher severity” has been 
deleted, as this is not in scope of this analysis. Sum scores will be set to missing, in case of 
missing individual items. 
 
Change #16 
Section 8.2.9: 
Identification of rescue medication has been clarified and time to first administration will be 
summarized. 
 
Change #17 
Section 10.1: 
The extent of exposure to study treatment is categorized by percent deviation from 
randomized dose. 
 
Change #18 
Section 10.2: 
TEAEs occurring in the observation period will not be stratified by stabilization period, but 
by participants being under standard of care treatment when the TEAE occurs. 
Specific AEs of focus will be summarized separately. 
 
Change #19 
Section 10.3.1: 
The instructions for assessment of PDILI were made clearer. 
 
Change #20 
Section 10.4.1: 
Blood pressure MA/PCS definitions were changed to be in harmony with other 
Rozanolixizumab studies. 
 
 
 
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Change #21 
Section 11: 
A description of contents of patient profiles was added. 
14.2 
Amendment 2 
14.2.1 
Rationale 
Adaptations reflect findings in a blinded data evaluation meeting and review of planned 
outputs. 
14.2.2 
Changes 
14.2.2.1 
Change 1 
Sections 3.5.4 and 3.9, 4.2.1 and 8: 
The FAS definition was clarified and efficacy assessments following intake of rescue 
medication are not eligible for analysis. 
14.2.2.2 
Change 2 
Some COVID-19 related analyses were considered not valuable and removed. This includes 
removal of potentially COVID-19 impacted data and subgroup assessments based on 
COVID-19 period. 
14.2.2.3 
Change 3 
Definition of Hy’s Law updated. 
14.2.2.4 
Change 4 
The fourth interim analysis was canceled not providing additional value. 
14.2.2.5 
Change 5 
Exploratory statistical inference tests were removed to prevent overinterpretation of potential 
statistical significance. No p-values will be reported. 
14.2.2.6 
Change 6 
Sensitivity analysis via multiple imputation was removed due to the low sample size. 
14.2.2.7 
Change 7 
It was clarified that relapse, MMRM and summary tables for iRODS, adjusted INCAT and 
clinician reported grip strength will be performed for the FAS and PPS. 
14.2.2.8 
Change 8 
Efficacy listings will be based on the SS, not the FAS, to present all relevant data available. 
14.2.2.9 
Change 9 
Summary presentation of CIDP PRO Fatigue, PGIS and PGIC were corrected to reflect the 
scales of the assessments adequately. 
14.2.2.10 
Change 10 
A PDILI criterion has been corrected (text for symptoms missing). 
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14.2.2.11 
Change 11 
ECG MA criteria table has been corrected (missing text). An IgG MA criterion has been 
added. 
14.2.2.12 
Change 12 
CIDP-specific auto-antibodies will not be analyzed in scope of this SAP. 
 
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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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