A Multicenter, Open-Label, Outpatient Study to Evaluate the Safe And Effective Use of a Zilucoplan Auto-Injector Combination Product for Subcutaneous Self-Administration by Study Participants With Generalized Myasthenia Gravis
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Sponsor: UCB Biopharma SRL

UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 1 of 73
A MULTICENTER, OPEN-LABEL, OUTPATIENT STUDY TO 
EVALUATE THE SAFE AND EFFECTIVE USE OF A 
ZILUCOPLAN AUTO-INJECTOR COMBINATION PRODUCT 
FOR SUBCUTANEOUS SELF-ADMINISTRATION BY STUDY 
PARTICIPANTS WITH GENERALIZED MYASTHENIA GRAVIS
PROTOCOL DV0013
PHASE 3B
SHORT TITLE:
An open-label, outpatient study to evaluate the safe and effective use of a zilucoplan 
auto-injector for self-administration by study participants with generalized myasthenia gravis
Sponsor:
UCB Biopharma SRL
Allée de la Recherche 60
1070 Brussels
BELGIUM
Regulatory agency identifying number(s):
EudraCT Number:
Not applicable
EU CT Number:
2023-508287-30-00
EUDAMED Number:
Not applicable
IND Number:
134340
IDE Number:
Not applicable
NCT Number:
Not applicable
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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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 2 of 73
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE
Document History
Document
Date
Type of amendment
Original Protocol
15 Dec 2023
Not applicable
PUBLIC COPY 
This document cannot be used to support any marketing authorization application 
and any extensions or variations thereof.

UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 3 of 73
SERIOUS ADVERSE EVENT AND MEDICATION ERRORS REPORTING
Serious adverse event reporting (24h)
Fax
Europe and Rest of the World: +32 2 386 24 21
US and Canada: +1 800 880 6949 or +1 866 890 3175
Email
Global: DS_ICT@ucb.com (for interventional clinical studies)
REPORTING OF ADVERSE DEVICE EFFECTS (SERIOUS AND 
NONSERIOUS) AND DEVICE DEFICIENCIES
Reporting of adverse device effects (serious and nonserious) and device 
deficiencies for UCB devices used in the study (24h)
eCRF
The mechanism for reporting an adverse device effects and device 
deficiencies to UCB (or its representative) will be the electronic data 
collection tool.
Email
Global: qactscomplaints@ucb.com
Note: In case of a device related event or a device deficiency for 
UCB devices or device constituents these should be captured in the 
electronic data collection tool and an investigational medicinal 
product (IMP) complaint form should be filled.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 4 of 73
TABLE OF CONTENTS
1
PROTOCOL SUMMARY.................................................................................................8
1.1
Synopsis.........................................................................................................................8
1.2
Schema.........................................................................................................................13
1.3
Schedule of Activities..................................................................................................14
2
INTRODUCTION ...........................................................................................................18
2.1
Study rationale .............................................................................................................18
2.2
Background..................................................................................................................18
2.3
Benefit/risk assessment................................................................................................19
3
OBJECTIVES AND ENDPOINTS .................................................................................20
3.1
Estimand/intercurrent event handling rationale and impact on the study....................22
4
STUDY DESIGN.............................................................................................................22
4.1
Overall design..............................................................................................................22
4.2
Scientific rationale for study design ............................................................................23
4.3
Justification for dose....................................................................................................23
4.4
End of study definition.................................................................................................24
5
STUDY POPULATION ..................................................................................................24
5.1
Inclusion criteria ..........................................................................................................24
5.2
Exclusion criteria .........................................................................................................25
5.3
Lifestyle restrictions.....................................................................................................26
5.4
Screen failures..............................................................................................................26
6
STUDY TREATMENT...................................................................................................26
6.1
Treatment administered ...............................................................................................26
6.1.1
Devices ................................................................................................................28
6.2
Preparation, handling, storage, and accountability requirements ................................28
6.2.1
Drug accountability/device accountability ..........................................................28
6.3
Measures to minimize bias: randomization and blinding ............................................29
6.4
Treatment compliance..................................................................................................29
6.5
Concomitant medication(s)/treatment(s) .....................................................................30
6.5.1
Permitted and prohibited concomitant treatments (medications and therapies)..30
6.5.2
Vaccinations ........................................................................................................30
6.6
Dose modification........................................................................................................30
6.7
Criteria for study hold or dosing stoppage...................................................................30
6.8
Treatment after the end of the study ............................................................................31
7
DISCONTINUATION OF STUDY MEDICATION AND PARTICIPANT 
DISCONTINUATION/WITHDRAWAL........................................................................31
7.1
Discontinuation of study medication ...........................................................................31
7.2
Participant discontinuation/withdrawal from the study ...............................................32
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 5 of 73
7.3
Lost to follow up..........................................................................................................32
8
STUDY ASSESSMENTS AND PROCEDURES...........................................................33
8.1
Efficacy assessments....................................................................................................33
8.1.1
Effective self-administration of ZLP-AI .............................................................34
8.1.2
Evaluation of post-use structural and mechanical integrity of ZLP-AI...............34
8.1.3
SIAQ....................................................................................................................34
8.1.4
Patient Preference Question.................................................................................35
8.2
Safety assessments.......................................................................................................35
8.2.1
Physical examination...........................................................................................35
8.2.2
Vital signs ............................................................................................................35
8.2.3
Suicidal risk monitoring ......................................................................................35
8.3
Adverse events and serious adverse events .................................................................36
8.3.1
Time period and frequency for collecting AE and SAE information..................36
8.3.2
Method of detecting AEs and SAEs ....................................................................37
8.3.3
Follow up of AEs and SAEs................................................................................37
8.3.4
Regulatory reporting requirements for SAEs ......................................................37
8.3.5
Pregnancy ............................................................................................................37
8.3.6
Medication errors associated with self-administration........................................38
8.3.7
Adverse events of special monitoring..................................................................38
8.3.8
Device – adverse events (ADEs, UADEs, SAEs, SADEs, and USADEs) and 
device deficiencies...............................................................................................38
8.3.8.1
Time period for detecting ADEs and device deficiencies.........................38
8.3.8.2
Follow up of ADEs and device deficiencies .............................................39
8.3.8.3
Prompt reporting of ADEs and device deficiencies to Sponsor................39
8.3.8.4
Regulatory reporting requirements for ADEs and device deficiencies.....39
8.4
Safety signal detection.................................................................................................39
8.5
Treatment of overdose .................................................................................................40
8.6
Pharmacokinetics.........................................................................................................40
8.7
Biomarkers...................................................................................................................40
8.8
Immunogenicity assessments.......................................................................................40
8.9
Medical resource utilization and health economics.....................................................40
9
STATISTICAL CONSIDERATIONS.............................................................................41
9.1
Definition of analysis sets............................................................................................41
9.2
General statistical considerations.................................................................................41
9.3
Planned efficacy/outcome analyses .............................................................................41
9.3.1
Primary estimand/primary endpoint analyses......................................................41
9.3.2
Secondary estimand/secondary endpoint analyses ..............................................42
9.3.3
Other estimand/other endpoint analyses..............................................................42
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 6 of 73
9.4
Planned safety and other analyses................................................................................42
9.4.1
Other analyses......................................................................................................42
9.4.2
Safety analyses.....................................................................................................42
9.5
Handling of protocol deviations...................................................................................43
9.6
Handling of dropouts or missing data..........................................................................43
9.7
Planned interim analysis and data monitoring.............................................................43
9.8
Determination of sample size.......................................................................................43
10
SUPPORTING DOCUMENTATION AND OPERATIONAL CONSIDERATIONS ..45
10.1
Appendix 1: Regulatory, ethical, and study oversight considerations.........................45
10.1.1
Regulatory and ethical considerations.................................................................45
10.1.2
Financial disclosure .............................................................................................45
10.1.3
Informed consent process ....................................................................................46
10.1.4
Data protection.....................................................................................................46
10.1.5
Committees structure...........................................................................................47
10.1.6
Dissemination of clinical study data....................................................................47
10.1.7
Data quality assurance .........................................................................................47
10.1.7.1
Case report form completion.....................................................................48
10.1.7.2
Apps...........................................................................................................48
10.1.8
Source documents................................................................................................48
10.1.9
Study and site start and closure ...........................................................................49
10.1.10
Publication policy ................................................................................................49
10.2
Appendix 2: Clinical laboratory tests ..........................................................................50
10.3
Appendix 3: Adverse events – Definitions and procedures for recording, evaluating, 
follow up, and reporting...............................................................................................51
10.4
Appendix 4: Contraceptive guidance and collection of pregnancy information .........56
10.5
Appendix 5: Genetics...................................................................................................59
10.6
Appendix 6: Liver safety – suggested actions and follow-up assessments .................60
10.7
Appendix 7: Device AEs, ADEs, SAEs, and device deficiencies: Definition and 
procedures for recording, evaluating, follow up, and reporting ..................................61
10.7.1
Definitions of AE and ADE.................................................................................61
10.7.2
Definitions of SAE, SADE, and USADE............................................................62
10.7.3
Definition of device deficiency ...........................................................................62
10.7.4
Recording and follow up of AE and/or SAE and device deficiencies.................63
10.7.5
Reporting of SAEs...............................................................................................65
10.7.6
Reporting of SADEs............................................................................................65
10.8
Appendix 8: Rapid alert procedures ............................................................................66
10.9
Appendix 9: Country-specific requirements................................................................67
10.10 Appendix 10: Abbreviations and trademarks ..............................................................68
10.11 Appendix 11: Protocol amendment history .................................................................70
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15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 7 of 73
10.12 Appendix 12: Medication errors associated with self-administration .........................71
11
REFERENCES ................................................................................................................72
SPONSOR DECLARATION..................................................................................................73
LIST OF TABLES
Table 1-1:
Schedule of study assessments.........................................................................14
Table 3-1:
Objectives and estimands/endpoints................................................................20
Table 4-1:
Zilucoplan weight-bracketed dosing................................................................24
Table 6-1:
Treatment administered ...................................................................................27
Table 8-1:
Summary table of structural and mechanical integrity tests ............................34
LIST OF FIGURES
Figure 1-1:
Study schematic ...............................................................................................13
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 8 of 73
1
PROTOCOL SUMMARY
1.1
Synopsis
Protocol title:
A multicenter, open-label, outpatient study to evaluate the safe and effective use of a zilucoplan 
auto-injector combination product for subcutaneous self-administration by study participants 
with generalized myasthenia gravis
Short title:
An open-label, outpatient study to evaluate the safe and effective use of a zilucoplan 
auto-injector for self-administration by study participants with generalized myasthenia gravis
Rationale:
Myasthenia gravis (MG) is a serious, sometimes life-threatening, debilitating condition 
associated with numerous symptoms including muscular weakness and fatigue. Some patients 
can manage their symptoms with oral medications such as oral acetylcholinesterase inhibitors, 
corticosteroids, or nonsteroidal immunosuppressants. In addition, intravenous immunoglobulin G 
and plasma exchange treatment could be utilized. For those whose symptoms are not well 
managed by these medications, a potential alternative is long-term treatment with approved 
intravenous (IV) C5 inhibitors (eg, eculizumab or ravulizumab) which have proven effective at 
treating MG symptoms.
Zilucoplan (RA101495; ZLP) was recently approved in the US and EU and is currently available 
as a prefilled syringe (PFS) for subcutaneous injection (sc). The PFS reduces drug administration 
burden and can be used by patients with difficult venous access or who are in underserved or 
rural populations where economic access to IV infusions is prohibitive. However, it is expected 
that there will be a need to have different treatment administration options available for the 
administration of ZLP and that individuals may prefer to have the option to administer their ZLP 
medication using an auto-injector (AI). The AI device may improve the handling of the drug 
administration and improve compliance. DV0013 will, therefore evaluate the safe and effective 
self-administration with the zilucoplan-auto-injector (ZLP-AI) as an administration option.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 9 of 73
Objectives and estimands/endpoints
Objectives
Estimands/endpoints
Primary
x
To evaluate the effectiveness 
of ZLP-AI self-administration
Primary estimand
x
Treatment: Zilucoplan
x
Target Population: Participants aged ≥18 years with gMG, 
already treated with zilucoplan (currently participating in 
MG0011 or treated with commercial zilucoplan)
x
Endpoint: Effective self-administrations of zilucoplan using 
the ZLP-AI from Visit 1 to Visit 8.
Effective self-administration is defined as:

Complete dose delivery: completeness of the delivery as 
confirmed by the Investigator. The entire dose of IMP 
should be completely delivered (ie, the yellow plunger 
that can be seen through the device window should be 
completely depressed)
x
Intercurrent event:

No intercurrent event is defined
x
Population level summary:

Proportion of effective self-administrations overall (total 
number of self-administrations from Visit 1 to Visit 8), 
along with the 95% CI
Secondary
x
To evaluate the effectiveness 
of ZLP-AI self-administration 
using additional effectiveness 
endpoints
Secondary estimands
x
Treatment: Zilucoplan
x
Target Population: Participants aged ≥18 years with gMG, 
already treated with zilucoplan (currently participating in 
MG0011 or treated with commercial zilucoplan)
x
Endpoints:

Effective self-administration of zilucoplan using ZLP-AI 
at Visit 8. Effective self-administration is defined as 
complete dose delivery.

Effective self-administration of zilucoplan using ZLP-AI 
at Visit 1. Effective self-administration is defined as 
complete dose delivery
o
Complete dose delivery: completeness of the delivery 
as confirmed by the Investigator. The entire dose of 
IMP should be completely delivered (ie, the yellow 
plunger that can be seen through the device window 
should be completely depressed)
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 10 of 73
Objectives
Estimands/endpoints
x
Intercurrent event handling:

No intercurrent event is defined
x
Population level summary:

Proportion of participants, along with the 95% CI
x
Evaluate the safety and
tolerability of the ZLP-AI
self-administrations
Secondary endpoint:
x
Occurrence of SAEs, TEAEs, ADEs (serious and nonserious)
from Visit 1 up to the SFU Visit
Other objectives
x
Evaluate the PK of the ZLP-AI
for self-administration
Other PK endpoint:
x
Measurement of plasma concentrations for zilucoplan and
main metabolites 
x
Evaluate the safety and
tolerability of the ZLP-AI
self-administrations
Other safety endpoints:
x
Occurrence of ISRs
x
Occurrence of medication errors associated with adverse
reaction
x
Evaluate the safety of the
ZLP-AI
Other safety endpoint:
x
Occurrence of SADEs related to the use of the ZLP-AI that
would preclude continued use of the ZLP-AI for
self-administration (ie, SADEs and/or SADEs leading to
withdrawal from the DV0013 study)
x
Evaluate the structural and
mechanical integrity of the
ZLP-AI after completion of
self-administration
Other endpoint:
x
Occurrence of used ZLP-AI combination products identified
as having structural or mechanical integrity issues after
completion of self-administration.
x
Investigator observed and reported device deficiencies.
Note: An independent assessment of the structural and
mechanical integrity of all used ZLP-AI devices will
additionally be performed by UCB. The outcome will be
reported separately from the study results.
x
Evaluate the study
participant’s experience of
self-administration with
ZLP-AI as assessed by the
SIAQ©
Other endpoint:
x
Preinjection SIAQ (SIAQ PRE module) domain scores at
Visit 1
x
Postinjection SIAQ (SIAQ POST module) domain scores
following self-administration using the ZLP-AI at Visit 1 and
Visit 8
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 11 of 73
Objectives
Estimands/endpoints
x
Explore the study participant’s 
preference for zilucoplan PFS 
(as used in MG0011 or 
commercial supply) vs 
ZLP-AI following the use of 
the ZLP-AI
Other endpoint:
x
Patient Preference Question at Visit 8
ADE=adverse device effects; CI=confidence interval; gMG=generalized myasthenia gravis; IMP=investigational 
medicinal product; ISR=injection site reaction; PFS=prefilled syringe; PK=pharmacokinetics; SADE=serious 
adverse device effects; SAE=serious adverse event; SFU=Safety Follow-Up; SIAQ=Self-Injection Assessment 
Questionnaire; TEAE=treatment-emergent adverse event; ZLP-AI=zilucoplan-auto-injector
Overall design
DV0013 is a Phase 3b, multicenter, open-label study to evaluate the safe and effective use of a 
ZLP-AI combination product for sc self-administration of ZLP solution (the IMP) by study 
participants with generalized myasthenia gravis (gMG), planned to be conducted in Europe and 
the US.
The study will aim to reflect real-world use scenarios by including participants who are already 
self-administering ZLP using the PFS on a once-daily dosing regimen as part of the MG0011 
long-term open-label extension (OLE) study or who are on commercial ZLP treatment for at 
least 1 month prior to Screening.
DV0013 will consist of 9 visits (2 clinic visits and 7 remote telephone visits) and 
14 self-administrations using the ZLP-AI:
Treatment Period
x
Visit 1 (Screening/Day 1): Each study participant will be provided with training by study 
personnel in self-administration using the ZLP-AI and will receive the written instructions 
for use (IFU). Following the training, the study participant will perform self-administration 
at the clinic using the ZLP-AI.
x
Visit 2 to Visit 7: No additional training will be performed; however, the IFU will be 
available. The study participant will perform self-administration at home using the ZLP-AI. 
These visits will be remote visits using a telephone call. During the call, the Investigator
will ask about the self-administration and document any adverse events and adverse device 
effects (AEs and ADEs), any changes in concomitant medication use, and/or medication 
errors occurring in the course of the self-administration. Telephone visits which would be 
scheduled to occur on a weekend day may be skipped, and the data for the respective days 
(Saturday and Sunday) will be collected on the next clinic business day (Monday).
x
Visit 8: The study participant will perform self-administration at the clinic using the 
ZLP-AI. The Investigator will collect all used devices from participants and will check 
completeness of the delivery for the collected devices. All used devices will be returned to 
UCB for an assessment of structural and mechanical integrity.
x
At Visits 1 and 8 , the Investigator is required to witness and evaluate the study 
participant’s use of the ZLP-AI, document any adverse events (AEs and ADEs) and/or 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 12 of 73
medication errors occurring in the course of the self-administration, and, following the 
completion of the self-administration, perform a visual inspection of the ZLP-AI to check 
that the yellow plunger that can be seen through the viewing window is completely 
depressed.
Safety Follow-Up Visit
x
Visit 9: For all participants, a Safety Follow-up (SFU) via telephone call, 7 days (±3 days) 
after their final study dose using the ZLP-AI, will be required. Any AEs, serious adverse 
events (SAEs), ADEs, or serious adverse device effects (SADEs) will be recorded. 
Participants who were enrolled into DV0013 from MG0011 will roll back into MG0011 on 
the next day following Visit 8 and will continue to be followed up as part of the MG0011 
protocol. Participants who were enrolled following treatment with commercial ZLP 
treatment will continue with their prescribed treatment; for AEs occurring after the SFU 
telephone call and up to 40 days after the last administration, participants will be instructed 
to contact the site or follow the safety reporting according to the label.
Number of participants
Participants from MG0011 or those who are currently being treated with commercial ZLP and 
are on a stable dosing regimen for at least 1 month will be screened at approximately 20 sites in 
the US and Europe in order to enroll at least 25 participants who use the ZLP-AI at Visit 1 and to 
obtain approximately 350 self-administrations. See Section 9.8 for the determination of sample 
size.
Treatment groups and duration
Zilucoplan will be provided in ZLP-AI devices for self-injection using weight-bracketed dosing 
(ie, participants will be provided ZLP-AI devices containing fixed amounts of ZLP based on 
their weight, and each fixed amount will cover a range of participant weights). Participants will 
receive the same weight bracketed dosing as currently received in MG0011 or by prescription.
The study duration for each participant will be approximately 21 days. The end of the study is 
defined as the date of the last SFU phone call for the last participant in the study.
No Data Monitoring Committee will be established for this study.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 13 of 73
1.2
Schema
Figure 1-1:
Study schematic
AI=auto-injector; SFU=Safety Follow-Up
Note: *=Participants roll back into MG0011 or go back to self-administration with commercial supply on the next day after Visit 8.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 14 of 73
1.3
Schedule of Activities
Table 1-1:
Schedule of study assessments
Period
Screening 
Period
Treatment Period
EWb
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X
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history
X
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X
X
X
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X
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X
X
MGFA classification
X
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X
X
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medication
X
X
X
X
X
X
X
X
X
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 15 of 73
Table 1-1:
Schedule of study assessments
Period
Screening 
Period
Treatment Period
EWb
(In-
clinic)
SFU Visitc
(Phone 
call)
Name
Screening 
(In-clinic)
Day 1 
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Day 2 
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Day 4
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and dose date taken in 
MG0011/commercially 
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in DV0013h
X
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preference questioni
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self-administration with 
ZLP-AI device
X
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ZLP with ZLP-AI device
X
X
X
X
X
X
X
X
Study personnel evaluation of 
self-injection and of used 
ZLP-AI devicel
X
X
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 16 of 73
Table 1-1:
Schedule of study assessments
Period
Screening 
Period
Treatment Period
EWb
(In-
clinic)
SFU Visitc
(Phone 
call)
Name
Screening 
(In-clinic)
Day 1 
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Day 2 
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5
6
7
8
9
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4
6
8
10
12
14
(Final 
dose 
+7±3 days)
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postinjection SIAQ (SIAQ 
POST module)j
X
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ZLP-AI devices from each 
participant to send to UCB for 
structural/mechanical integrity 
assessmentm
X
X
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accountability
X
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SADEs, medication errors, 
ISR, and device deficienciesn
X
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plasma concentrationso
ADE=adverse device effect; AE=adverse event; AI=auto-injector; βHCG=beta human chorionic gonadotropin; C-SSRS=Columbia-Suicide Severity Rating 
Scale; EW=early withdrawal; IMP=investigational medicinal product; ISR=injection site reaction; IXRS=interactive response technology; 
MGFA=Myasthenia Gravis Foundation of America; PK=pharmacokinetic; SADE=serious adverse device event; SAE=serious adverse event; SFU=Safety 
Follow-Up; SIAQ=Self-Injection Assessment Questionnaire; ZLP=zilucoplan
a Telephone visits which would be scheduled to occur on a weekend day may be skipped, and the data for the respective days (Saturday and Sunday) will be 
collected on the next clinic business day (Monday).
b If a study participant permanently withdraws from the study, the study participant should return to the clinic for an EW Visit as soon as possible but no later 
than the next scheduled in-clinic visit.
c Study participant will be required to perform a SFU telephone call 7 days (±3 days) after their final study dose of ZLP using the ZLP-AI.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 17 of 73
Table 1-1:
Schedule of study assessments
Period
Screening 
Period
Treatment Period
EWb
(In-
clinic)
SFU Visitc
(Phone 
call)
Name
Screening 
(In-clinic)
Day 1 
(In-clinic)
Day 2 
(Phone 
call)a
Day 4
(Phone 
call)a
Day 6
(Phone 
call)a
Day 8
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call)a
Day 10
(Phone 
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Day 12
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Day 14
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5
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8
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2
4
6
8
10
12
14
(Final 
dose 
+7±3 days)
d Participants may rescreen twice (total of 3 screenings).
e The full physical exam will include examination of the following systems: eyes, hair, and skin; respiratory; cardiovascular; and gastrointestinal systems. The 
Investigator should examine the injection site at each scheduled visit for injection site reactions (eg, bruising, pain, swelling, pruritis, and hematoma). Any 
abnormalities should be reported as AEs. Height and weight will also be measured and recorded at Visit 1. A full physical exam will be performed at Visit 1 
and Visit 8. Any findings will be reported as AEs as appropriate. Any clinically significant changes since the physical examination at Visit 1 will be 
recorded as AEs.
f Vital signs (sitting systolic and diastolic blood pressure, pulse rate, and temperature) are to be measured prior to blood sampling and prior to dosing, where 
applicable.
g For women of childbearing potential, pregnancy testing will consist of urine pregnancy tests at Visits 1 and 8 only. Pregnancy test results must be negative 
prior to administering IMP. Positive urinary test will be exclusionary. Participant may rescreen at a later time if pregnancy not confirmed subsequently with 
a serum test.
h When recording the final dose taken prior to enrolling in DV0013, the time of self-administration should also be recorded. Participants should continue on 
the same weight-based dose that they were self-administering in MG0011 or commercial ZLP dose they were on prior to enrolling in DV0013.
i A single question will be asked at Visit 8 postdose, assessing the study participant's preference for administration method.
j The preinjection SIAQ (SIAQ PRE module version 2.1) will be completed preinjection at Visit 1; the postinjection SIAQ (SIAQ POST module version 2.1) 
will be completed directly following self-injection and no later than 30 minutes postinjection at Visit 1 and Visit 8 during the study Treatment Period.
k In accordance with the draft Food and Drug Administration Suicidal Ideation and Behavior: Prospective Assessment of Occurrence in Clinical Trials 
Guidance (Food and Drug Administration, Guidance for Industry, 2012) study participants will be asked to complete the C-SSRS at study visits as specified.
l At Visits 1 and 8, the Investigator will observe the participant self-administrating with the ZLP-AI device. The used ZLP-AI devices will be visually 
inspected by the Investigator to determine if the entire dose was completely delivered (ie, the yellow plunger that can be seen through the device window is 
completely depressed).
m All used AI devices will be sent to UCB for assessment of structural and mechanical integrity and damage (ie, clear evidence of damage/compromised 
structural integrity—not superficial, cosmetic imperfections).
n Note that ADEs, SADEs, ISRs, and medication errors can only be recorded on the 14 days that the participant is using the ZLP-AI, and device deficiencies 
will also be collected during those 14 days. Device deficiencies will be reported via the eCRF. In addition, sites will fill out a product quality complaint 
form; all product quality complaints will be investigated by UCB.
o A single PK sample will be collected 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 18 of 73
2
INTRODUCTION
2.1
Study rationale
Myasthenia gravis is a serious, sometimes life-threatening, debilitating condition associated with 
numerous symptoms including muscular weakness and fatigue. Some patients can manage their 
symptoms with oral medications such as oral acetylcholinesterase inhibitors, corticosteroids, or 
nonsteroidal immunosuppressants. In addition, intravenous immunoglobulin G and plasma 
exchange treatment could be utilized. For those whose symptoms are not well managed by these 
medications, a potential alternative is long-term treatment with approved IV C5 inhibitors (eg, 
eculizumab or ravulizumab) which have proven effective at treating MG symptoms.
Zilucoplan (RA101495; ZLP) was recently approved in the US and EU and is currently available 
as a PFS for sc injection. The PFS reduces drug administration burden and can be used by 
patients with difficult venous access or who are in underserved or rural populations where 
economic access to IV infusions is prohibitive. However, it is expected that there will be a need 
to have different treatment administration options available for the administration of ZLP and 
that individuals may prefer to have the option to administer their ZLP medication using an AI. 
The AI device may improve the handling of the drug administration and improve compliance. 
DV0013 will, therefore evaluate the safe and effective self-administration with the ZLP-AI as an 
administration option.
2.2
Background
Myasthenia gravis is a rare complement-mediated autoimmune disease characterized by the 
production of autoantibodies targeting proteins that are critical for the normal transmission of 
neurotransmitter signals from nerves to muscles. The prevalence of MG globally is estimated to 
be 12.4 (range 10.6 to 14.5) per 100,000 persons (Salari et al, 2021), hence, MG affects up to 
approximately 760,000 people worldwide. Myasthenia gravis most commonly affects young 
adult women (under 40) and older men (over 60), but it can occur at any age. Epidemiological 
studies reveal an increasing prevalence over the past 50 years, due in part to an increase in the 
frequency of diagnosis in the elderly (Sanders et al, 2016). As the population has aged, the 
average age at onset has increased correspondingly. In 15% of patients with MG, symptoms 
remain confined to the ocular muscles. In approximately 85% of patients, MG progresses beyond 
the ocular muscles to affect multiple muscle groups throughout the body, a condition that is 
typically referred to as gMG (Gilhus, 2016).
Patients with gMG present with muscle weakness that characteristically becomes more severe 
with repeated use and recovers with rest. Symptoms are typically at their mildest in the morning, 
when overnight inactivity enables replenishment of acetylcholine levels in presynaptic motor 
nerve terminals, and worsen during the course of the day. Muscle weakness can be localized to 
specific muscles, but often progresses to more diffuse muscle weakness (Gilhus, 2016; 
Gilhus and Verschuuren, 2015; Chamanza et al, 2010). Generalized MG symptoms can become 
life-threatening when muscle weakness involves the diaphragm and intercostal muscles in the 
chest wall that are responsible for breathing. The most dangerous complication of gMG, known 
as myasthenic crisis, requires hospitalization, intubation, and mechanical ventilation. 
Approximately 15% to 20% of patients with gMG will experience a myasthenic crisis within 
2 years of diagnosis (Ramizuddin, 2014).
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 19 of 73
Zilucoplan is a 15-amino acid macrocyclic peptide complement inhibitor designed for the 
treatment of conditions in which inappropriate activation of C5 has been demonstrated to play a 
role. Zilucoplan binds to C5 with high affinity and prevents its cleavage by C5 convertases into 
the cleavage products C5a and C5b. Inhibition of C5 cleavage prevents the downstream 
assembly and cytolytic activity of the membrane attack complex. Pharmacologically, ZLP has 
demonstrated dose-dependent inhibition of C5a and C5b formation following activation of 
classical or alternative complement pathways, as well as inhibition of red blood cell lysis in the 
serum/plasma from various species.
The clinical efficacy of ZLP in gMG has been shown in a Phase 2 study, MG0009 (previously 
RA101495-02.201), and in a Phase 3 study, MG0010 (previously RA101495-02.301) 
(Howard et al, 2023). In addition, ZLP has shown a favorable safety and tolerability profile 
across all completed studies. Further details are provided in the ZLP Investigator’s 
Brochure (IB).
2.3
Benefit/risk assessment
The primary risk associated with C5 inhibitors as a class is increased susceptibility to infections 
with encapsulated bacteria, such as Neisseria spp. Specifically, this risk is related to suppression 
of the innate immune response and subsequent infections, particularly meningococcal infections. 
It is well established that inhibition of C5 and the terminal complement pathway increases the 
susceptibility to Neisseria spp. infections. Given the known increased risk of meningococcal 
infection (Neisseria. meningitidis) associated with inhibition or inherited deficiency of C5, 
participants in this study will be required to have documentation of N. meningitidis vaccination 
at least 14 days prior to IMP administration, if not vaccinated within 3 years prior to the start of 
treatment. Participants must be vaccinated with a quadrivalent vaccine (where available and in 
accordance with local standard of care) and serogroup B vaccine against meningococcal 
infections (N. meningitidis), and booster vaccinations should be administered in accordance with 
local standard of care. In addition, while on ZLP, study participants must be monitored closely 
for signs and symptoms of N. meningitidis infection, including self-monitoring and caregiver 
monitoring based on detailed instructions about signs and symptoms of possible meningococcal 
infections. To date, no meningococcal infections have been identified with ZLP treatment in 
clinical studies, as the risk of Neisseria infection is well-managed by appropriate prophylactic 
vaccination.
Together, the nonclinical and clinical data generated by investigations with ZLP support the use 
of ZLP in patients with gMG. While both benefits and risks may be associated with the 
administration of any investigational medicine, to date, ZLP has shown good tolerability and a 
favorable safety profile across studies in healthy adult study participants and adult study 
participants with 
, immune-mediated necrotizing myopathy, gMG, 
paroxysmal nocturnal hemoglobinuria, coronavirus Disease 2019 (COVID-19)-associated 
respiratory syndrome, and amyotrophic lateral sclerosis. The bioequivalence of the AI device is 
under investigation in DV0012; however the AI device may improve the handling of the drug 
administration and improve compliance.
Considering the potential benefits, risks, and mitigation measures in place, UCB considers the 
overall benefit/risk to be favorable for participants in this study.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 20 of 73
More detailed information about the known and expected benefits and risks and reasonably 
expected adverse events of ZLP may be found in the IB.
3
OBJECTIVES AND ENDPOINTS
Table 3-1:
Objectives and estimands/endpoints
Objectives
Estimands/endpoints
Primary
x
To evaluate the effectiveness 
of ZLP-AI self-administration
Primary estimand
x
Treatment: Zilucoplan
x
Target Population: Participants aged ≥18 years with gMG, 
already treated with zilucoplan (currently participating in 
MG0011 or treated with commercial zilucoplan)
x
Endpoint: Effective self-administrations of zilucoplan using 
the ZLP-AI from Visit 1 to Visit 8. Effective 
self-administration is defined as:

Complete dose delivery: completeness of the delivery as 
confirmed by the Investigator. The entire dose of IMP 
should be completely delivered (ie, the yellow plunger 
that can be seen through the device window should be 
completely depressed)
x
Intercurrent event:

No intercurrent event is defined
x
Population level summary:

Proportion of effective self-administrations overall (total 
number of self-administrations from Visit 1 to Visit 8), 
along with the 95% CI
Secondary
x
To evaluate the effectiveness 
of ZLP-AI self-administration 
using additional effectiveness 
endpoints
Secondary estimands
x
Treatment: Zilucoplan
x
Target Population: Participants aged ≥18 years with gMG, 
already treated with zilucoplan (currently participating in 
MG0011 or treated with commercial zilucoplan)
x
Endpoints:

Effective self-administration of zilucoplan using ZLP-AI 
at Visit 8. Effective self-administration is defined as 
complete dose delivery.

Effective self-administration of zilucoplan using ZLP-AI 
at Visit 1. Effective self-administration is defined as 
complete dose delivery
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 21 of 73
Table 3-1:
Objectives and estimands/endpoints
Objectives
Estimands/endpoints

Complete dose delivery: completeness of the delivery 
as confirmed by the Investigator. The entire dose of 
IMP should be completely delivered (ie, the yellow 
plunger that can be seen through the device window 
should be completely depressed)
x
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
Proportion of participants, along with the 95% CI
x
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tolerability of the ZLP-AI 
self-administrations
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x
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from Visit 1 up to the SFU Visit
Other objectives
x
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for self-administration
Other PK endpoint:
x
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main metabolites 
x
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tolerability of the ZLP-AI 
self-administrations
Other safety endpoints:
x
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x
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reaction
x
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ZLP-AI
Other safety endpoint:
x
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would preclude continued use of the ZLP-AI for 
self-administration (ie, SADEs and/or SADEs leading to 
withdrawal from the DV0013 study)
x
Evaluate the structural and 
mechanical integrity of the 
ZLP-AI after completion of 
self-administration
Other endpoint:
x
Occurrence of used ZLP-AI combination products identified 
as having structural or mechanical integrity issues after 
completion of self-administration.
x
Investigator observed and reported device deficiencies.
Note: An independent assessment of the structural and 
mechanical integrity of all used ZLP-AI devices will 
additionally be performed by UCB. The outcome will be 
reported separately from the study results.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 22 of 73
Table 3-1:
Objectives and estimands/endpoints
Objectives
Estimands/endpoints
x
Evaluate the study 
participant’s experience of 
self-administration with 
ZLP-AI as assessed by the 
SIAQ©
Other endpoint:
x
Preinjection SIAQ (SIAQ PRE module) domain scores at 
Visit 1
x
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following self-administration using the ZLP-AI at Visit 1 and 
Visit 8
x
Explore the study participant’s 
preference for zilucoplan PFS 
(as used in MG0011 or 
commercial supply) vs 
ZLP-AI following the use of 
the ZLP-AI
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x
Patient Preference Question at Visit 8
ADE=adverse device effect; CI=confidence interval; gMG=generalized myasthenia gravis; IMP=investigational 
medicinal product; ISR=injection site reaction; PFS=prefilled syringe; PK=pharmacokinetics; SADE=serious 
adverse device effect; SAE=serious adverse event; SFU=Safety Follow-Up; SIAQ=Self-Injection Assessment 
Questionnaire; TEAE=treatment-emergent adverse event; ZLP-AI=zilucoplan-auto-injector
3.1
Estimand/intercurrent event handling rationale and impact on 
the study
Only observed data will be analyzed. There will be no special procedures for handling missing 
data. All imputation of missing or partial dates for safety assessments will be detailed in the 
Statistical Analysis Plan.
4
STUDY DESIGN
4.1
Overall design
DV0013 is a Phase 3b, multicenter, open-label study to evaluate the safe and effective use of a 
ZLP-AI combination product for sc self-administration of ZLP solution (the IMP) by study 
participants with gMG, planned to be conducted in Europe and the US.
The study will aim to reflect real-world use scenarios by including participants who are already 
self-administering ZLP using the PFS on a once-daily dosing regimen as part of the MG0011 
long-term OLE study or who are on commercial ZLP treatment for at least 1 month prior to 
Screening.
DV0013 will consist of 9 visits (2 clinic visits and 7 remote telephone visits) and 
14 self-administrations using the ZLP-AI:
Treatment Period
x
Visit 1 (Screening/Day 1 visit): Each study participant will be provided with training by 
study personnel in self-administration using the ZLP-AI and will receive the written IFU. 
Following the training, the study participant will perform self-administration at the clinic 
using the ZLP-AI.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 23 of 73
x
Visit 2 to Visit 7: No additional training will be performed; however, the IFU will be 
available. The study participant will perform self-administration at home using the ZLP-AI. 
These visits will be remote visits using a telephone call. During the call, the Investigator
will ask about the self-administration and document any adverse events (AEs and ADEs), 
any changes in concomitant medication use, and/or medication errors occurring in the 
course of the self-administration. Telephone visits which would be scheduled to occur on a 
weekend day may be skipped, and the data for the respective days (Saturday and Sunday) 
will be collected on the next clinic business day (Monday).
x
Visit 8: The study participant will perform self-administration at the clinic using the 
ZLP-AI. The Investigator will collect all used devices from participants and will check 
completeness of the delivery for the collected devices. All used devices will be returned to 
UCB for an assessment of structural and mechanical integrity.
x
At Visits 1 and 8, the Investigator is required to witness and evaluate the study participant’s 
use of the ZLP-AI, document any adverse events (AEs and ADEs) and/or medication errors 
occurring in the course of the self-administration, and, following the completion of the 
self-administration, perform a visual inspection of the ZLP-AI to check that the yellow 
plunger that can be seen through the viewing window is completely depressed.
Safety Follow-Up Visit
x
Visit 9: For all participants, an SFU via telephone call, 7 days (±3 days) after their final 
study dose using the ZLP-AI, will be required. Any AEs, SAEs, ADEs, or SADEs will be 
recorded. Participants who were enrolled into DV0013 from MG0011 will roll back into 
MG0011 on the next day following Visit 8 and will continue to be followed up as part of 
the MG0011 protocol. Participants who were enrolled following treatment with commercial 
ZLP treatment will continue with their prescribed treatment; for AEs occurring after the 
SFU telephone call and up to 40 days after the last administration, participants will be 
instructed to contact the site or follow the safety reporting according to the label.
The study duration for each participant will be approximately 21 days.
4.2
Scientific rationale for study design
It is expected that there will be a need to have different treatment administration options 
available for the administration of licensed ZLP and that individuals may prefer to have the 
option to administer their ZLP medication using an AI. DV0013 will therefore evaluate the safe 
and effective self-administration with the ZLP-AI as an administration option.
The study will aim to reflect real-world use scenarios by including participants who are already 
self-administering ZLP using the PFS on a once-daily dosing regimen as part of the MG0011 
long-term OLE or who are on commercial ZLP treatment for at least 1 month prior to Screening.
4.3
Justification for dose
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 24 of 73
Table 4-1:
Zilucoplan weight-bracketed dosing
4.4
End of study definition
A participant is considered to have completed the study if he/she has completed all periods of the 
study including the Treatment Period and SFU phone call.
The global end of the study is defined as the date of the last SFU phone call for the last 
participant in the study.
5
STUDY POPULATION
Prospective approval of protocol deviations to recruitment and enrollment criteria, also known as 
protocol waivers or exemptions, is not permitted.
5.1
Inclusion criteria
Participants are eligible to be included in the study only if all of the following criteria apply:
Age
1. Study participant is male or female and must be at least 18 years of age at the time of signing 
the informed consent form (ICF).
Type of participant and disease characteristics
2. Study participant must have a documented diagnosis of gMG, based on study participant's 
history and supported by previous evaluations.
3. Study participant is currently participating in ZLP study MG0011 or is administering 
commercial ZLP on a stable dosing regimen for at least 1month prior to Screening.
4. Study participants on commercial ZLP need to receive ZLP per the approved local labeling.
5. Study participant is considered reliable and capable of adhering to the study protocol 
(eg, able to understand and complete questionnaires and able to adhere to the visit schedule) 
according to the judgement of the Investigator.
6. Study participant is willing and capable of self-administering ZLP using the ZLP-AI 
according to the IFU, ie, does not have any visual, physical, or other disability or impairment 
that interferes with his/her capacity to self-administer; if the participant has a caregiver, 
he/she may assist the participant with the injection.
7. Vaccination with a quadrivalent meningococcal vaccine and, where available, meningococcal 
serotype B vaccine at least 14 days prior to IMP administration, if not vaccinated within 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 25 of 73
3 years prior to the start of treatment. Booster vaccination(s) should also be administered as 
clinically indicated, according to the local standard of care, for participants who have been 
previously vaccinated against Neisseria meningitidis.
8. Female participants of childbearing potential must have a negative urine pregnancy test prior 
to the first dose of study drug.
Sex
9. Male and/or female study participants

A male participant must agree to use contraception as detailed in Appendix 4 
(Section 10.4) during the Treatment Period and for 40 days after the last dose of study 
medication, and refrain from donating sperm during this period.

A female participant is eligible to participate if she is not pregnant (see Appendix 4 
[Section 10.4]), not breastfeeding, and at least 1 of the following conditions applies:
◦
Not a woman of childbearing potential (WOCBP) as defined in Appendix 4 
(Section 10.4)
OR
A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 
(Section 10.4) during the Treatment Period and for 40 days after the last dose of study 
medication.
Informed consent
10. Capable of giving signed informed consent as described in Appendix 1 (Section 10.1), which 
includes compliance with the requirements and restrictions listed in the ICF and in this 
protocol.
5.2
Exclusion criteria
Participants are excluded from the study if any of the following criteria apply:
Medical conditions
1. Study participant has any medical or psychiatric condition that, in the opinion of the 
Investigator, could jeopardize or would compromise the study participant’s ability to 
participate in this study.
2. Female participants who are breastfeeding, pregnant, or plan to become pregnant during the 
study.
3. Study participant has a known hypersensitivity to any components of the study medication 
(and/or an investigational device) as stated in this protocol.
4. Study participant has a clinically relevant active infection or a history of serious infection 
(resulting in hospitalization or requiring intravenous antibiotic treatment) within 6 weeks 
before Visit 1.
5. Study participant has a history of meningococcal disease.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 26 of 73
Prior/concurrent clinical study experience
6. Participant has previously participated in this study or participant has previously been 
assigned to treatment in a study of the medication under investigation in this study (except 
studies MG0009, MG0010, or MG0011, which are not excluded, unless the participant was 
required to withdraw from said studies for a safety reason which could reasonably recur).
7. Participant has participated in another study of an IMP (and/or an investigational device) 
different from ZLP within the previous 3 months or 5 half-lives, whichever is longer, or is 
currently participating in another study of an IMP (and/or an investigational device).
Diagnostic assessments
8. Current unstable liver or biliary disease at Screening (Visit 1), per Investigator assessment, 
defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, 
esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: with exception of 
stable hepatobiliary conditions (including Gilbert's syndrome, asymptomatic gallstones).
5.3
Lifestyle restrictions
There are no lifestyle restrictions during the study.
5.4
Screen failures
Screen failures are defined as participants who consent to participate in the clinical study but are 
not subsequently entered in the study. A minimal set of screen failure information is required to 
ensure transparent reporting of screen failure participants to meet the Consolidated Standards of 
Reporting Trials publishing requirements and to respond to queries from regulatory authorities. 
Minimal information includes demography, screen failure details, eligibility criteria, and any 
SAE.
In case of screen failure, the study participant can be rescreened twice following discussion with 
the UCB Medical Monitor and/or Study Physician.
6
STUDY TREATMENT
Study treatment is defined as any investigational treatment(s), marketed product(s), placebo, or 
medical device(s) intended to be administered to a study participant according to the study 
protocol.
6.1
Treatment administered
The treatment administered in DV0013 is presented in Table 6-1.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 27 of 73
Table 6-1:
Treatment administered
ARM name
ZLP-AI
Intervention name
Zilucoplan
Type
Drug product (15-amino acid macrocyclic synthetic 
peptide) associated with a functional secondary packaging
Dose formulation
Prefilled pen containing the solution for injection (also 
known as the AI)
Unit dose strength(s)
Drug solution 40mg/mL
Dosage level(s)
Three dose levels based on participant body weight:
x
<56kg: 16.6mg ZLP
x
≥56 to <77kg: 23.0mg ZLP
x
≥77kg: 32.4mg ZLP
Frequency: once daily
Route of administration
Subcutaneous injection
Use
Experimental
IMP and NIMP/AxMP
IMP
Sourcing
Provided centrally by the Sponsor
Packaging and labeling
The IMP will be provided in a box that protects from light 
or damage. The IMP is manufactured, packaged, and 
labeled according to GMP guidelines and in accordance 
with all applicable laws or regulations. Each IMP carton 
will be labeled according to individual country 
requirements. Details of the packaging and labeling will be 
provided in the IMP Handling Manual.
Excipients
Isotonic buffered solution of RA101495 sodium (drug 
substance), containing standard excipients
AI=auto-injector; AxMP=auxiliary medicinal product; GMP=Good Manufacturing Practices; 
IMP=investigational medicinal product; NIMP=noninvestigational medicinal product; ZLP=zilucoplan; 
ZLP-AI=zilucoplan-auto-injector
In DV0013, the term IMP refers to the ZLP-AI, which consists of the naked PFS (primary 
packaging containing ZLP drug product used for all Phase 3 studies and in the ZLP safety 
syringe 1mL) inside an auto-injector.
Each participant will be provided with self-injection training and IFU for the ZLP-AI, as 
specified in the Schedule of Activities (SoA) (Section 1.3).
Study participants will be instructed to self-inject sc doses daily at approximately the same time 
each day. Participants may inject study drug into the abdomen (preferred site), front part of the 
thigh, or back part of the upper arm. Participants will be requested to choose a different site each 
time they give themselves an injection; if they wish to use the same injection site, they should 
ensure that it is at least 1 inch (approximately 2.5cm) from the spot previously used.Treatment of 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 28 of 73
the injection site with an anesthetic or other topical products (eg, steroids) prior to dosing is not 
permitted.
Participants will receive the doses of IMP listed in Table 6-1 based on their weight and their 
established dosing regimen in MG0011/commercial supply.
Regardless of the assigned dose regimen, participants will perform a single self-administration 
with the ZLP-AI, as specified in the SoA (Section 1.3) on Days 1 to 14.
6.1.1
Devices
The ZLP-AI-1mL consists of the naked PFS (primary packaging containing ZLP drug product 
used for all Phase 3 studies and in the ZLP safety syringe 1mL) inside an auto-injector. The PFS 
volume will be administered from the same primary container, a 1mL long glass syringe, and a 
29-gauge thin wall half inch stacked needle. The ZLP-AI-1mL is a single dose, disposable, 
nonsterile combination product.
Additional instructions for device use, including the injection angle, are provided in the IFU. 
Participants will be instructed to retain and return all ZLP-AI devices administered at home when 
they return to the clinic, as specified in the SoA (Section 1.3). Devices will be individually 
numbered for tracking purposes.
All device deficiencies (including malfunction use error and inadequate labeling) shall be 
documented and reported by the Investigator throughout the study (see Section 8.3.8) and 
appropriately managed by the Sponsor.
6.2
Preparation, handling, storage, and accountability requirements
The Investigator or designee must confirm that appropriate temperature conditions have been 
maintained during transit for all study treatment received and any discrepancies are reported and 
resolved before use of the study treatment.
Only participants enrolled in the study may receive study treatment and only authorized site staff 
may supply study treatment. All study treatments must be stored in a secure, environmentally 
controlled, and monitored (manual or automated) area in accordance with the labeled storage 
conditions, with access limited to the Investigator and authorized site staff.
The Investigator, institution, or the head of the medical institution (where applicable) is 
responsible for IMP accountability, reconciliation, and record maintenance (ie, receipt, 
reconciliation, and final disposition records).
In case an out-of-range temperature is noted, it must be immediately reported as per instructions 
contained in the IMP Handling Manual.
The Investigator (or designee) will instruct the participant to store the study medication 
following the instructions on the label.
Further guidance and information for the final disposition of unused study treatment are provided 
in the IMP Handling Manual.
6.2.1
Drug accountability/device accountability
The electronic case report form (eCRF) will be used to record study medication dispensing and 
return information on a by-participant basis and will serve as source documentation during the 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 29 of 73
course of the study. Details of any study medication lost, damaged (due to breakage or wastage), 
not used, partially used, disposed of at the study site, or returned to the Sponsor or designee must 
also be recorded on the appropriate forms. All supplies and pharmacy documentation must be 
made available throughout the study for UCB (or designee) to review.
The Investigator (or designee) is responsible for retaining all used, unused, and partially used 
containers of study medication until returned.
The Investigator may assign some of the Investigator’s duties for drug accountability at the study 
site to an appropriate pharmacist/designee.
The Investigator must ensure that the study medication is used only in accordance with the 
protocol.
After completion of the clinical phase of the study, all used (including empty containers/partially 
used), unused, damaged, and/or expired study medication must be reconciled at the site 
according to local laws, regulations, and UCB standard operating procedures (SOPs) and 
returned to UCB for assessment. Investigational medicinal product intended for the study cannot 
be used for any other purpose than that described in this protocol. Assessment of post-use 
structural and mechanical integrity of ZLP-AI will be done at UCB, and data will be reported on 
file (Section 8.1.2).
For further details about drug accountability, refer to the IMP Handling Manual.
6.3
Measures to minimize bias: randomization and blinding
This is an open-label, single-arm study. Therefore, no blinding or randomization is required.
To enroll a study participant (Visit 1), the Investigator or designee will contact the interactive 
response technology (IXRS) and provide brief details about the participant to be enrolled. Each 
participant will receive a 5-digit number assigned at Screening that serves as the participant 
identifier throughout the study. Participants enrolling from MG0011 will receive a unique 5-digit 
number for DV0013 and the participant ID from MG0011 will be recorded in the eCRF. The 
participant number will be required in all communication between the Investigator or designee 
and the IXRS regarding a particular participant. Participant numbers and kit numbers will be 
tracked via the IXRS.
The IXRS will allocate kit numbers to the participant based on the participant number during the 
course of the study.
6.4
Treatment compliance
Participants must return all unused study medication and empty study medication containers as 
specified in the SoA (Section 1.3). Drug accountability must be done in the participant’s 
presence in order to obtain explanations regarding discrepancies in compliance with the dosing 
regimen. Drug accountability must be recorded on the Drug Accountability form.
If a participant is found to be persistently noncompliant, the Sponsor, in conjunction with the 
Investigator, will make a decision as to whether the participant should be withdrawn from the 
study.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 30 of 73
6.5
Concomitant medication(s)/treatment(s)
Concomitant medications include any prescription or over-the-counter medication that is 
ongoing on Day 1 or that is initiated while in this study. All concomitant medications ongoing 
from MG0011 will be documented in the eCRF.
6.5.1
Permitted and prohibited concomitant treatments (medications and 
therapies)
In addition to the permitted and prohibited concomitant treatments described in the local labeling 
for zilucoplan:
x
Treatment of the injection site with an anesthetic or other topical medications (eg, steroids) 
prior to dosing is not permitted.
6.5.2
Vaccinations
Study participants should be up to date with their vaccination schedule, and their vaccination 
history will be reviewed as specified in the SoA (Section 1.3). If vaccination is due, the study 
participant should receive it. Administration of live and nonlive vaccines is allowed during the 
study at the discretion of the Investigator and should be documented. This includes 
administration of nonlive COVID-19 vaccines authorized at the time of issuance of this protocol. 
Current medical and scientific evidence does not suggest that there is any medical or scientific 
reason for restricting SARS-CoV-2 vaccinations in ZLP clinical studies.
If administration of a vaccine is planned, all relevant information (ie, name of vaccine, dose, date 
of administration) should be captured in the eCRF Concomitant Medication Page.
There is a known increased risk of meningococcal infection (N.meningitidis), which is associated 
with the inhibition, or inherited deficiency, of C5. Therefore, to reduce the risk of meningococcal 
infection (N.meningitidis), all study participants must have documentation of vaccination with a 
quadrivalent meningococcal vaccine and, where available, meningococcal serotype B vaccine. 
A study participant must agree to receive a booster vaccination during the study, if clinically 
indicated according to the local standard of care, for participants who have been previously 
vaccinated against N.meningitidis. In addition, while on-treatment with ZLP, participants will be 
monitored closely for signs and symptoms of N.meningitidis infection.
During the study, participants will be counseled and reminded of the early signs and symptoms 
of N.meningitidis infection. A patient safety card detailing the signs and symptoms of infection, 
with instructions to seek immediate medical attention if any such symptoms occur, will be 
provided to each study participant.
6.6
Dose modification
No dose modifications are allowed during the study. Participants should continue on the same 
weight-based dose that they were self-administering in MG0011 or commercial ZLP dose they 
were on prior to enrolling in DV0013.
6.7
Criteria for study hold or dosing stoppage
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, 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 31 of 73
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 Institutional Review Board (IRB)/Independent Ethics Committee (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 or destruction of all unused 
IMP and other material in accordance with UCB procedures for the study.
6.8
Treatment after the end of the study
Participants who were enrolled into DV0013 from MG0011 will roll back into MG0011 and will 
continue to be followed up as part of the MG0011 protocol.
Participants who enrolled in DV0013 from commercial ZLP treatment will roll back to 
self-administration with commercial supply.
7
DISCONTINUATION OF STUDY MEDICATION AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL
7.1
Discontinuation of study medication
Dosing of a study participant must be immediately and permanently discontinued if:
x
Study participant is diagnosed with meningococcal disease
x
In the opinion of the Investigator and/or Sponsor, it is unsafe for the study participant to 
continue dosing with study medication (eg, suspected pancreatitis)
In circumstances where permanent discontinuation from study medication may occur, the 
Investigator is to discuss with the study participant the appropriate processes for discontinuation 
from study medication and must discuss with the study participant the options for continuation of 
the assessments in the SoA (Section 1.3), including different options of follow up (eg, in person, 
by phone/mail, in correspondence/communication with other treating physicians, from the 
review of medical records) and collection of data, including endpoints and AEs. Study 
participants who discontinue study medication should not be automatically removed from the 
study. Whenever safe and feasible, it is imperative that study participants remain on the study to 
ensure safety surveillance and/or collection of outcome data. The Investigator must document the 
level of follow up that is agreed to by the study participant, any changes to the scheduled visits 
and assessments, and the plan for follow up that is agreed to by the participant. In regard to 
changes to scheduled visits, for the level of follow up, it is recommended that the Early 
Withdrawal Visit and the SFU Visit be prioritized. If the study participant does not want to 
continue the study medication and withdraws consent (Section 7.2), the Investigator should 
contact the Medical Monitor, whenever possible, to discuss the withdrawal of a participant in 
advance.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 32 of 73
7.2
Participant discontinuation/withdrawal from the study
Participants are free to withdraw from the study at any time, without prejudice to their continued 
care.
A participant may withdraw from the study at any time at his/her own request or may be 
withdrawn at any time at the discretion of the Investigator for safety, behavioral, compliance, or 
administrative reasons.
If the participant withdraws consent for disclosure of future information, the Sponsor may retain 
and continue to use any data collected before such a withdrawal of consent.
If a participant withdraws from the study, he/she may request destruction of any samples taken 
and not tested, and the Investigator must document this in the site study records.
See the SoA (Section 1.3) for data to be collected at the time of study discontinuation and follow 
up and for any further evaluations that need to be completed.
Participants should be withdrawn from the study if any of the following events occur:
1. Participant develops an illness that would interfere with his/her continued participation.
2. Participant is noncompliant with the study procedures or medications in the opinion of the 
Investigator.
3. Participant withdraws his/her consent.
4. There is confirmation of a pregnancy during the study, as evidenced by a positive pregnancy 
test. If the study participant was enrolled from MG0011, the study participant should roll 
back into MG0011, and MG0011 protocol procedures should be followed. Participants 
recruited from commercial supply should go back to using commercial supply and follow the 
guidance in the regional-approved product label.
5. The Sponsor or a regulatory agency requests withdrawal of the participant.
6. The Investigator is of the opinion that the participant’s continued participation in the study is 
not in the best interest of the participant.
7. Participant experiences a severe or serious injection site reaction (eg, bleeding, bruising, or 
pain) or a SAE that would, in the opinion of the Investigator, preclude the participant’s 
further participation in the study.
Study participants who withdraw from DV0013, ie, study participants who have a medical 
condition or personal preference that precludes further self-administration, must perform a SFU 
telephone call or equivalent 7 days (±3 days) after their final DV0013 dosing visit.
Study participants who withdraw from DV0013 and do not have a safety event that would 
require termination of ZLP treatment may return to MG0011 or to prescribed treatment.
Investigators should contact the Medical Monitor, whenever possible, to discuss the withdrawal 
of a participant in advance.
7.3
Lost to follow up
A participant will be considered lost to follow up if he or she repeatedly fails to return for 
scheduled visits and is unable to be contacted by the study site.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 33 of 73
The following actions must be taken if a participant fails to return to the clinic for a required 
study visit:
x
The site must attempt to contact the participant and reschedule the missed visit as soon as 
possible and counsel the participant on the importance of maintaining the assigned visit 
schedule and ascertain whether or not the participant wishes to and/or should continue in the 
study.
x
Before a participant is deemed lost to follow up, the Investigator or designee must make 
every effort to regain contact with the participant (at least 1 phone call and 1 written message 
to the participant) and document his/her effort (date and summary of the phone call and copy 
of the written message in the source documents) to complete the final evaluation. All results 
of these evaluations and observations, together with a narrative description of the reason(s) 
for removing the participant, must be recorded in the source documents. The eCRF must 
document the primary reason for withdrawal.
Should the participant continue to be unreachable, he/she will be considered to have withdrawn 
from the study with a primary reason of lost to follow up documented in the eCRF.
8
STUDY ASSESSMENTS AND PROCEDURES
Study procedures and their timing are summarized in the SoA (Section 1.3).
Protocol waivers or exemptions are not allowed.
Immediate safety concerns should be discussed with the Sponsor immediately upon occurrence 
or awareness to determine if the participant should continue or discontinue study treatment.
Adherence to the study design requirements, including those specified in the SoA (Section 1.3), 
is essential and required for study conduct.
All screening evaluations must be completed and reviewed to confirm that potential participants 
meet all eligibility criteria. The Investigator will maintain a screening log to record details of all 
participants screened and to confirm eligibility or record reasons for screening failure, as 
applicable.
Procedures conducted as part of the participant’s routine clinical management (eg, blood count)
and obtained before signing of the ICF may be utilized for screening (Visit 1) purposes provided 
the procedures met the protocol-specified criteria and were performed within the time frame 
defined in the SoA (Section 1.3).
The maximum amount of blood collected from each participant over the duration of the study, 
including any extra assessments that may be required, will not exceed approximately 4mL. 
Repeat or unscheduled samples may be taken for safety reasons or for technical issues with the 
samples.
8.1
Efficacy assessments
Planned time points for all efficacy assessments are provided in the SoA (Section 1.3).
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 34 of 73
8.1.1
Effective self-administration of ZLP-AI
Effective self-administration is defined as:
x
Complete dose delivery: completeness of the delivery as confirmed by the Investigator. The 
entire dose of IMP should be completely delivered (ie, the yellow plunger that can be seen 
through the device window should be completely depressed).
8.1.2
Evaluation of post-use structural and mechanical integrity of ZLP-AI
The visual inspection of the used devices will check for structural/mechanical integrity and 
damage (ie, clear evidence of damage/compromised structural integrity and not any superficial, 
cosmetic imperfections). All evaluations will be performed by appropriately trained site staff.
All devices will be retrieved post-use and will be assessed for structural and mechanical integrity 
internally by the technical team at UCB. Results from the assessments will be reported as data on 
file.
The tests to be performed to assess structural and mechanical integrity of ZLP-AI are 
summarized in Table 8-1.
Table 8-1:
Summary table of structural and mechanical integrity tests
Control
Methodology
Acceptance criteria
Integrity of the syringe barrel
Visual inspection
Attribute (pass/fail)
Syringe should be completely 
empty
Visual inspection
Attribute (pass/fail)
Locking mechanism that 
protects the needle is deployed 
and locked
Qualitative inspection
Attribute (pass/fail)
8.1.3
SIAQ
The Self-Injection Assessment Questionnaire (SIAQ) version 2.1 is a self-administered 
Patient-Reported Outcome, which will be used to assess the participants’ self-injection 
experience, including the perceived advantages and the potential limitations of self-injection of a 
sc medication (Keininger and Coteur, 2011).
The preinjection SIAQ (SIAQ PRE module) is composed of 7 items grouped into 3 domains 
(feelings about injection, self-confidence, and satisfaction with the current mode of 
administration). The postinjection SIAQ (SIAQ POST module) is composed of 21 items grouped 
into 6 domains (feelings about injection, self-image, self-confidence, injection site reactions, 
ease of use, and satisfaction with self-injection). Each domain score of both pre- and 
post-modules range from 0 to 10, where higher scores indicate a more positive self-injection 
experience.
The preinjection SIAQ will be completed before self-administration at Visit 1, and the 
postinjection SIAQ will be completed within 30 minutes after each self-injection (ie, at Visit 1 
and Visit 8).
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 35 of 73
8.1.4
Patient Preference Question
The Patient Preference Question is a self-administered, single question that will be asked at 
Visit 8 postdose, assessing the study participant's preference for administration method:
“Think about your experience with the auto-injector you used during this clinical trial compared 
with the prefilled syringe used prior to this clinical trial. All things considered, which injector did 
you prefer? (please select one answer):
x
Auto-injector (used in this clinical study)
x
Prefilled syringe (prior to the clinical study)
x
No preference”
Given the limitations of the study design, this question will not produce robust and generalizable 
patient preference data, but it will generate exploratory data into patient preferences based on 
this sample’s experience with the 2 treatments.
8.2
Safety assessments
Planned time points for all safety assessments are provided in the SoA (Section 1.3).
8.2.1
Physical examination
A full physical examination will include eyes, hair, and skin; and assessments of the respiratory, 
cardiovascular, and gastrointestinal systems. The Investigator should examine the injection site 
at each scheduled visit for injection site reactions (eg, bruising, pain, swelling, pruritis, and 
hematoma). Any abnormalities should be reported as AEs.
Height and weight will also be measured and recorded.
Investigators should pay special attention to clinical signs related to previous serious illnesses.
Clinically relevant findings or worsening of previous findings since the physical examination at 
Visit 1 will be recorded as AEs.
8.2.2
Vital signs
Temperature, pulse rate, and blood pressure will be assessed.
Blood pressure and pulse measurements will be assessed in the sitting position with a completely 
automated device. Manual techniques will be used only if an automated device is not available.
Blood pressure and pulse measurements should be preceded by at least 5 minutes of rest for the 
participant in a quiet setting without distractions (eg, television, cell phones).
Vital signs (to be taken before blood collection for laboratory tests and prior to dosing) will 
consist of 1 pulse and 3 blood pressure measurements (3 consecutive blood pressure readings 
will be recorded at intervals of at least 1 minute). The average of the 3 blood pressure readings 
will be recorded on the eCRF.
8.2.3
Suicidal risk monitoring
Study participants being treated with ZLP should be monitored appropriately and observed
closely for suicidal ideation and behavior or any other unusual changes in behavior. Families and 
caregivers of study participants being treated with ZLP should be instructed to monitor 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 36 of 73
participants for the emergence of unusual changes in behavior, as well as the emergence of 
suicidal ideation and behavior, and to report such symptoms immediately to the Investigator.
Suicidality will be assessed by trained study personnel using the Columbia-Suicide Severity 
Rating Scale (C-SSRS) (Posner et al, 2011; Mundt et al, 2010). The “Screening/Baseline” 
version of the C-SSRS will be completed during the Screening Visit for participants coming 
from commercial ZLP treatment, and the “Since Last Visit” version will be completed at all 
other time points. The “Since Last Visit” version will be completed at all time points for 
participants coming from MG0011 as detailed in the SoA (Section 1.3).
8.3
Adverse events and serious adverse events
The definitions of device-related safety events, ADEs, and SADEs can be found in Appendix 7 
(Section 10.7). Device deficiencies are addressed in Appendix 7 (Section 10.7).
Adverse events will be reported by the participant (or, when appropriate, by a caregiver, 
surrogate, or the participant's legally authorized representative).
The Investigator and any designees are responsible for detecting, documenting, and recording 
events that meet the definition of an AE or SAE and remain responsible for following up AEs 
that are serious, considered related to the study treatment or study procedures, or that caused the 
participant to discontinue the IMP or study (see Section 7).
For results disclosure on public registries (eg, ClinicalTrials.gov), treatment-emergent AEs 
(TEAEs) and treatment emergent SAEs will be published.
8.3.1
Time period and frequency for collecting AE and SAE information
All AEs and SAEs will be collected from the signing of the ICF until the SFU Visit, at the time 
points specified in the SoA (Section 1.3).
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 study medication 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.
All SAEs will be recorded and reported to the Sponsor or designee within 24 hours, as indicated 
in Appendix 3 (Section 10.3). The Investigator will submit any updated SAE data to the Sponsor 
within 24 hours of it being available.
The Investigator is specifically requested to collect and report to UCB (or its representative) any 
SAEs (even if the Investigator is certain that they are in no way associated with the study 
medication) up to 40 days from the end of the study for participants who were enrolled following 
treatment with commercial ZLP treatment or last contact in DV0013 for participants who were 
enrolled from MG0011, and also to inform participants of the need to inform the Investigator of 
any SAE within this period. Serious AEs that the Investigator thinks may be associated with the 
study medication must be reported to UCB regardless of the time between the event and the end 
of the study.
The method of recording, evaluating, and assessing causality of AEs and SAEs and the 
procedures for completing and transmitting SAE reports are provided in Appendix 3 
(Section 10.3).
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 37 of 73
8.3.2
Method of detecting AEs and SAEs
Care will be taken not to introduce bias when detecting AEs and/or SAEs. Open-ended and 
nonleading verbal questioning of the participant is the preferred method to inquire about 
AE occurrences.
8.3.3
Follow up of AEs and SAEs
After the initial AE/SAE report, the Investigator is required to proactively follow each 
participant at subsequent visits/contacts. All SAEs will be followed until resolution, stabilization, 
the Investigator determines that it is no longer clinically significant, the event is otherwise 
explained, or the participant is lost to follow up (as defined in Section 7.3). Further information 
on follow-up procedures is given in Appendix 3 (Section 10.3).
8.3.4
Regulatory reporting requirements for SAEs
Prompt notification of a SAE by the Investigator to the Sponsor is essential so that legal 
obligations and ethical responsibilities towards the safety of participants and the safety of a study 
treatment under clinical investigation are met.
The Sponsor has a legal responsibility to notify both the local regulatory authority and other 
regulatory agencies about the safety of a study treatment under clinical investigation. The 
Sponsor will comply with country-specific regulatory requirements relating to safety reporting to 
the regulatory authority, IRB/IEC, and Investigators.
Investigator safety reports must be prepared for suspected unexpected serious adverse reactions 
according to local regulatory requirements and Sponsor policy and forwarded to Investigators as 
necessary. Suspected unexpected serious adverse reactions reporting will be in adherence to 
requirements of EU pharmacovigilance legislation, CT legislation and guidance, Clinical Trial 
Regulation EU 536/2014; CT-3, and all other applicable local regulations.
An Investigator who receives an Investigator safety report describing a SAE or other specific 
safety information (eg, summary or listing of SAEs) from the Sponsor will review and then file it 
along with the Investigator’s Brochure and will notify the IRB/IEC, if appropriate according to 
local requirements.
8.3.5
Pregnancy
Details of all pregnancies in female participants and female partners of male participants will be 
collected after the start of study treatment and until 40 days after the last dose of study treatment.
If a pregnancy is reported, the Investigator must immediately inform the Sponsor within 24 hours 
of learning of the pregnancy and should follow the procedures outlined in Appendix 4 
(Section 10.4).
The participant should be withdrawn from the study as soon as pregnancy is known (by positive 
pregnancy test), and the following should be completed:
x
The participant should return for an early discontinuation visit.
x
The participant should immediately stop the intake of the study medication or be 
down-titrated as instructed at the early discontinuation visit.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 38 of 73
x
An SFU Visit should be scheduled 7±3 days after the participant has discontinued her study 
medication.
Abnormal pregnancy outcomes (eg, spontaneous abortion, fetal death, stillbirth, congenital 
anomalies, ectopic pregnancy) are considered SAEs.
8.3.6
Medication errors associated with self-administration
The definitions of medication errors can be found in Appendix 12 (Section 10.12). The 
medication errors reported by the study participant (or, when appropriate, by a caregiver, or 
observed by the Investigator) shall be recorded in the paper Medication Error form by the 
Investigator and emailed to the contact found in SERIOUS ADVERSE EVENT AND 
MEDICATION ERRORS REPORTING. All medication errors will be recorded and reported to 
the sponsor or designee within 7 days of awareness. Medication errors that are associated with an 
adverse reaction will be recorded and reported within 24 hours following the SAE recording and 
reporting procedures as indicated in Appendix 3 (Section 10.3) and using the Medication Error 
form.
The Investigator and any designees are responsible for detecting, documenting, and recording 
events that meet the definition of a medication error and remain responsible for following up 
medication errors that are associated with adverse reaction.
8.3.7
Adverse events of special monitoring
To mitigate the risk of infection, study participants will be counseled and reminded of the early 
signs and symptoms of Neisseria meningitidis infection. A patient safety card detailing the signs 
and symptoms of infection, with instructions to seek immediate medical attention if any such 
symptoms occur, will be provided to each study participant.
8.3.8
Device – adverse events (ADEs, UADEs, SAEs, SADEs, and USADEs) 
and device deficiencies
Devices are being provided for use in this study for the purposes of sc ZLP dosing 
(see Section 6.1.1). In order to fulfill regulatory reporting obligations worldwide, the Investigator
is responsible for the detection and documentation of events meeting the definitions of ADEs, 
SADEs, and device deficiency that occur during the study with such devices.
Adverse events will be reported according to the International Organization for Standardization
(ISO) 14155:2020, while recognizing and following requirements including reporting timelines 
specified in other specific laws, regulations, directives, standards; and/or guidelines as 
appropriate and as required by the countries in which the clinical investigation is conducted.
The definitions of an ADE, SADE, unanticipated serious adverse device effect (USADE), and 
device deficiency can be found in Appendix 7 (Section 10.7).
NOTE: Events fulfilling the definition of an AE/SAE will also follow the processes outlined in 
Section 8.3.3 and Appendix 3 (Section 10.3).
8.3.8.1
Time period for detecting ADEs and device deficiencies
Adverse device effects and device deficiencies will be detected, documented, and reported 
during all periods of the study in which the device is used.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 39 of 73
If the Investigator learns of any ADEs or device deficiencies at any time after a participant has 
been discharged from the study, and such event(s) is considered reasonably related to a device 
provided for the study, the Investigator will promptly notify the Sponsor.
The method of documenting ADEs and device deficiencies is provided in Appendix 7 
(Section 10.7).
8.3.8.2
Follow up of ADEs and device deficiencies
Follow up applies to all study participants, including those who discontinue study medication 
and/or the study.
The Investigator is responsible for ensuring that follow up includes any supplemental 
investigations as indicated to elucidate the nature and/or causality of the deficiency.
New or updated information will be recorded on the originally completed form with all changes 
signed and dated by the Investigator.
8.3.8.3
Prompt reporting of ADEs and device deficiencies to Sponsor
Adverse device effects and device deficiencies will be reported to the Sponsor immediately and 
under no circumstances should this exceed 24 hours after the Investigator determines that the 
event meets the protocol definition of an ADE or device deficiency.
The Sponsor will be the contact for the receipt of device deficiency reports.
8.3.8.4
Regulatory reporting requirements for ADEs and device deficiencies
The Investigator will promptly report all ADEs and device deficiencies immediately, and under 
no circumstance should this exceed 24 hours for any medical device provided for use in the study 
in order for the Sponsor to fulfill the legal responsibility to notify appropriate regulatory 
authorities and other entities about certain safety information relating to medical devices being 
used in clinical studies.
The Investigator, or responsible person according to local requirements (eg, the head of the 
medical institution), will comply with the applicable local regulatory requirements relating to the 
reporting of device deficiencies to the IRB/IEC.
8.4
Safety signal detection
Selected data from this study will be reviewed periodically to detect as early as possible any 
safety concern(s) related to the study medication so that Investigators, clinical study participants, 
regulatory authorities, and IRBs/IECs will be informed appropriately and as early as possible.
The Study Physician or medically qualified designee/equivalent will conduct an ongoing review 
of SAEs (including device-related SAEs) and perform ongoing SAE reconciliations in 
collaboration with the Patient Safety representative.
As appropriate for the stage of development and accumulated experience with the study 
medication, medically qualified personnel at UCB may identify additional safety measures 
(eg, AEs or vital signs) for which data will be periodically reviewed during the course of the 
study.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 40 of 73
8.5
Treatment of overdose
Unintentional overdose events are considered Medication Errors and should be reported as 
described in Section 8.3.6 and Section 8.3.7.
For this study, any dose of ZLP greater than the intended daily dose will be considered an 
overdose. Overdose events are only considered AEs or SAEs if there are associated clinical signs 
and symptoms, or if the act of taking the excess study medication itself is an AE or SAE (eg, 
suicide attempt).
It is not anticipated that overdose of ZLP will lead to acute or specific systemic TEAEs. In case 
of overdose, clinically appropriate supportive measures should be instituted as determined by the 
clinical scenario and in consultation with the Medical Monitor.
In the event of an overdose, the Investigator should:
1. Contact the Medical Monitor immediately.
2. Closely monitor the participant for any AE/SAE and laboratory abnormalities.
3. Document the quantity of the excess dose as well as the duration of the overdose in the 
eCRF.
Decisions regarding dose interruptions or modifications will be made by the Investigator in 
consultation with the Medical Monitor based on the clinical evaluation of the participant.
8.6
Pharmacokinetics
Blood samples of approximately 2mL will be collected for measurement of plasma 
concentrations of IMP and its main metabolites (
) as specified in the 
SoA (Section 1.3) Instructions for the collection and handling of biological samples will be 
provided by the Sponsor. The actual date and time (24-hour clock time) of each sample will be 
recorded.
Samples collected for pharmacokinetic (PK) analyses may also be used to evaluate safety or 
efficacy aspects related to concerns arising during or after the study. In addition, surplus PK 
samples may be stored and used for potential future biomarker research (including assay 
development/optimization), but not for future genetic biomarker research.
Any changes in the timing or addition of time points for any planned study assessments must be 
documented and approved by the relevant study team member and then archived in the Sponsor 
and site study files but will not constitute a protocol amendment. The IRB/IEC will be informed 
of any safety issues that require alteration of the safety monitoring scheme or amendment of the 
ICF.
8.7
Biomarkers
Biomarkers are not evaluated in this study.
8.8
Immunogenicity assessments
Immunogenicity is not evaluated in this study.
8.9
Medical resource utilization and health economics
Medical resource utilization and health economics parameters are not evaluated in this study.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 41 of 73
9
STATISTICAL CONSIDERATIONS
A description of statistical methods follows and will be described in more detail in the Statistical 
Analysis Plan.
9.1
Definition of analysis sets
An Enrolled Set will include all participants who have signed an informed consent form.
A Safety Set (SS) will be generated for the administration of ZLP using the ZLP-AI. The SS will 
consist of all study participants in the study who receive at least 1 dose of ZLP with the ZLP-AI 
combination product.
The Pharmacokinetic per-Protocol Set will consist of all study participants in the SS who have at 
least 1 quantifiable PK measurement during the study without important protocol deviations that 
would affect the PK.
9.2
General statistical considerations
Statistical analysis and generation of tables, figures, and study participant data listings will be 
performed using statistical analysis system (SAS®, SAS Institute, Cary, NC, US) Version 9.4 or 
higher using validated program code according to relevant SOP.
9.3
Planned efficacy/outcome analyses
The study will estimate the true population proportion of self-administration-related endpoints 
for ZLP-AI. No formal statistical hypothesis testing will be done.
Summary statistics for continuous variables will include:
x
Number of available observations
x
Mean, standard deviation, minimum, median, and maximum
For categorical variables, the number and proportion of participants, along with the 95% 
confidence interval (CI) based on the Exact Binomial method (when appropriate), will be 
presented.
No imputation of missing data will be done on the primary estimand. As a sensitivity analysis, 
missing data will be imputed as administration failure.
All data recorded in the eCRFs and questionnaires will be listed where appropriate.
All statistical analyses will be descriptive in nature. No inferential statistical analyses are 
planned.
9.3.1
Primary estimand/primary endpoint analyses
The primary outcome variable is defined in Table 3-1.
The proportion of effective self-administrations will be tabulated for the ZLP-AI combination 
product using the SS population. The 95% CIs based on the Exact Binomial method will be 
reported. As a sensitivity analysis, the primary endpoint will also be evaluated, where any 
missing self-administration will be considered as an administration failure. In addition, the 
proportion of effective self-administrations may be described by geographical region as well.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 42 of 73
9.3.2
Secondary estimand/secondary endpoint analyses
The secondary outcome variables are defined in Table 3-1.
The secondary outcome variables will be analyzed using the SS in the same manner as the 
primary outcome variable (Section 9.3.1).
9.3.3
Other estimand/other endpoint analyses
The other outcome variables are defined in Table 3-1.
These variables will be summarized using descriptive statistics, and they will be tabulated for the 
ZLP-AI by geographical region where appropriate using the SS population.
9.4
Planned safety and other analyses
9.4.1
Other analyses
The PK endpoint is trough PK (zilucoplan) levels associated with self-injection using the 
ZLP-AI.
Zilucoplan and main metabolite 
will 
be described descriptively (geomean, 95% CI, coefficient of variation, median, minimum, and 
maximum).
9.4.2
Safety analyses
Safety endpoints are defined in Table 3-1.
x
SAEs, TEAEs, SADEs, and ADEs
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An ADE is an AE related to the use of the AI device. An ADE includes: (1) Adverse 
event resulting from insufficiencies or inadequacies in the IFU, the deployment, the 
installation, the operation, or any malfunction of the device. (2) Adverse event that is a 
result of a use error or intentional misuse of the investigational device.

A SADE is an ADE that has resulted in any of the consequences characteristic of an 
SAE.
x
Medication errors associated with adverse reaction

The definitions of medication errors can be found in Appendix 12 (Section 10.12). The 
medication errors reported by the study participant (or, when appropriate, by a caregiver, 
or observed by the Investigator) will be recorded in the eCRF.
x
Injection site reactions

Treatment-emergent injection site reactions.
Analyses of the safety data will be done on the SS population.
All AE data will be listed. Only TEAEs, ADEs, SADEs, and medication errors associated with 
clinical harm will be included in the summary tables. Treatment-emergent AEs are defined as 
AEs starting after the time of first injection in the study and up to and including 40 days after the 
final dose (or last contact in the study, depending on which occurs first).
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 43 of 73
All AEs will be coded and classified by system organ class, high level term, and preferred term 
according to the Medical Dictionary for Regulatory Activities.
In this study, safety reporting requirements apply to all constituents of the IMP (ZLP-AI 
combination product which includes: zilucoplan solution contained inside the PFS and the 
auto-injector housing the PFS) as per 21 CFR 320. Adverse events related to use of the ZLP-AI 
combination (ADEs) and device deficiencies will be summarized separately. Adverse events will 
be summarized by the frequency and percent of participants having 1 or more of the events in 
question. Planned summaries include overall AEs, AEs by intensity, AEs by relationship to study 
drug, SAEs, AEs leading to withdrawal, AEs leading to death, ADEs, SADEs, and AEs of 
special interest.
Vital signs and physical examination findings will be recorded in the eCRF only at 
Screening/Day 1. Clinically relevant changes in subsequent physical examinations will be 
recorded as AEs.
Physical examination findings at Day 1 will be listed. A by-participant listing of the C-SSRS 
questionnaire data will be provided.
9.5
Handling of protocol deviations
Important protocol deviations are deviations from the protocol which could potentially have a 
meaningful impact on study conduct or on the key safety, efficacy, or other outcomes for an 
individual study participant. The criteria for identifying important protocol deviations and the 
classification of important protocol deviations will be defined within the Important Protocol 
Deviations Template. To the extent feasible, rules for identifying protocol deviations will be 
defined without review of the data and without consideration of the frequency of occurrence of 
such deviations. Whenever possible, criteria for identifying important protocol deviations will be 
implemented algorithmically to ensure consistency in the classification of important protocol 
deviations across all study participants.
All important protocol deviations will be listed by study participant.
9.6
Handling of dropouts or missing data
There will be no special procedures for handling missing data.
As a sensitivity analysis on the primary estimand, missing data will be imputed as administration 
failure.
All imputation of missing or partial dates for safety assessments will be detailed in the Statistical 
Analysis Plan.
9.7
Planned interim analysis and data monitoring
The final DV0013 study analysis and clinical study report will be prepared once all data have 
been collected. No Data Monitoring Committee will be established for this study.
9.8
Determination of sample size
This study will not be powered with respect to any endpoint, and sample size is based on 
practical considerations. The number of participants and their diagnosed conditions are 
summarized below.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 44 of 73
Given the rare diseases framework of the gMG indication and the fact that the device is not life 
supportive, the sample size is based on previous experience and the operating functions of the 
ZLP-AI.
Participants from MG0011 or those who are currently being treated with commercial ZLP and 
are on a stable dosing regimen for at least 1 month will be screened at approximately 20 sites in 
the US and Europe in order to enroll at least 25 participants who use the ZLP-AI at Visit 1 and to 
obtain approximately 350 self-administrations.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 45 of 73
10
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS
10.1
Appendix 1: Regulatory, ethical, and study oversight 
considerations
10.1.1
Regulatory and ethical considerations
The study will be conducted under the auspices of an IRB/IEC, as defined in local regulations, 
International Council for Harmonisation (ICH)-Good Clinical Practice (GCP), and in accordance 
with the ethical principles that have their origin in the Declaration of Helsinki.
The Investigator/UCB will ensure that an appropriately constituted IRB/IEC that complies with 
the requirements of the current ICH-GCP version or applicable country-specific regulations will 
be responsible for the initial and continuing review and approval of the clinical study. Prior to 
initiation of the study, the Investigator/UCB will forward copies of the protocol, Informed 
Consent form, Investigator’s Brochure, Investigator’s curriculum vitae (if applicable), 
advertisement (if applicable), and all other participant-related documents to be used for the study 
to the IRB/IEC for its review and approval.
Before initiating a study, the Investigator will have written and dated full approval from the 
responsible IRB/IEC for the protocol.
The Investigator will also promptly report to the IRB/IEC all changes in the study, all 
unanticipated problems involving risks to participants or others, and any protocol deviations, to 
eliminate immediate hazards to participants.
The Investigator will not make any changes in the study or study conduct without IRB/IEC 
approval, except where necessary to eliminate apparent immediate hazards to the participants. 
For minor changes to a previously approved protocol during the period covered by the original 
approval, it may be possible for the Investigator to obtain an expedited review by the IRB/IEC as 
allowed.
As part of the IRB/IEC requirements for continuing review of approved studies, the Investigator
will be responsible for submitting periodic progress reports to the IRB/IEC (based on IRB/IEC 
requirements) at intervals appropriate to the degree of participant risk involved, but no less than 
once per year. The Investigator should provide a final report to the IRB/IEC following study 
completion.
UCB (or its representative) will communicate safety information to the appropriate regulatory 
authorities and all active Investigators in accordance with applicable regulatory requirements. 
The appropriate IRB/IEC will also be informed by the Investigator or the Sponsor, as specified 
by the applicable regulatory requirements in each concerned country. Where applicable, 
Investigators are to provide the Sponsor (or its representative) with evidence of such IRB/IEC 
notification.
10.1.2
Financial disclosure
Insurance coverage will be handled according to local requirements.
Finance and insurance are addressed in the Investigator and/or contract research organization 
agreements, as applicable.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 46 of 73
10.1.3
Informed consent process
Participant’s informed consent must be obtained and documented in accordance with local 
regulations, ICH-GCP requirements, and the ethical principles that have their origin in the 
principles of the Declaration of Helsinki.
Prior to obtaining informed consent, information should be given in a language and at a level of 
complexity understandable to the participant in both oral and written form by the Investigator (or 
designee). Each participant will have the opportunity to discuss the study and its alternatives 
with the Investigator.
Prior to participation in the study, the Informed Consent form should be signed and personally 
dated by the participant, or his/her legal representative, and by the person who conducted the 
informed consent discussion (Investigator or designee). The participant or his/her legal 
representative must receive a copy of the signed and dated Informed Consent form. As part of 
the consent process, each participant must consent to direct access to his/her medical records for 
study-related monitoring, auditing, IRB/IEC review, and regulatory inspection.
If the Informed Consent form is amended during the study, the Investigator (or the Sponsor, if 
applicable) must follow all applicable regulatory requirements pertaining to the approval of the 
amended Informed Consent form by the IRB/IEC and use of the amended form.
All studies conducted at centers in the United States must include the use of a Health Insurance 
Portability and Accountability Act authorization form.
The participant may withdraw his/her consent to participate in the study at any time. A 
participant is considered as enrolled in the study when he/she has signed the Informed Consent 
form. An eCRF must not be started, nor may any study specific procedure be performed for a 
given participant, without having obtained his/her written consent to participate in the study.
10.1.4
Data protection
UCB staff (or designee) will affirm and uphold the participant’s confidentiality. Throughout this 
study, all data forwarded to UCB (or designee) will be identified only by the participant number 
assigned at Screening.
The Investigator agrees that representatives of UCB, its designee, representatives of the relevant 
IRB/IEC, or representatives of regulatory authorities will be allowed to review that portion of the 
participant’s primary medical records that directly concerns this study (including, but not limited 
to, laboratory test result reports, electrocardiogram (ECG) reports, admission/discharge 
summaries for hospital admissions occurring during a participant’s study participation, and 
autopsy reports for deaths occurring during the study).
The participant must be informed that his/her personal study-related data will be used by the 
Sponsor in accordance with local data protection law. The level of disclosure must also be 
explained to the participant.
The participant must be informed that his/her medical records may be examined by Clinical 
Quality Assurance auditors or other authorized personnel appointed by the Sponsor, by 
appropriate IRB/IEC members, and by inspectors from regulatory authorities.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 47 of 73
The contract between UCB and study sites specifies responsibilities of the parties related to data 
protection, including handling of data security breaches and respective communication and 
cooperation of the parties.
Information technology systems used to collect, process, and store study-related data are secured 
by technical and organizational security measures designed to protect such data against 
accidental or unlawful loss, alteration, or unauthorized disclosure or access.
10.1.5
Committees structure
No Data Monitoring Committee will be established for this study.
10.1.6
Dissemination of clinical study data
All Phase 1 to 4 clinical studies in patients will be registered on ClinicalTrials.gov, with results 
posted after completion of the study.
A plain language summary of the results of all Phase 1 to 4 clinical studies will be developed and 
shared on UCB’s website.
UCB is committed to submitting all Phase 2 to 4 clinical study results, irrespective of outcome, 
for publication in a credible, peer-reviewed journal. While there are some exceptions owing to 
intellectual property considerations in early clinical development, UCB’s policy is also to submit 
Phase 1 study results for publication in a peer-reviewed journal wherever possible.
Data from this study may be requested by qualified researchers 6 months after product approval 
in the US and/or Europe, or global development is discontinued, and 18 months after study 
completion. Investigators may request access to anonymized individual patient-level data and 
redacted study documents which may include analysis-ready datasets, study protocol, annotated 
case report forms, statistical analysis plan, dataset specifications, and clinical study report. Prior 
to use of the data, proposals need to be approved by an independent review panel at 
www.Vivli.org, and a signed data sharing agreement will need to be executed. All documents are 
available in English only, for a prespecified time, typically 12 months, on a password protected 
portal. This plan may change if the risk of reidentifying study participants is determined to be too 
high after the study is completed; in this case and to protect participants, individual patient-level 
data would not be made available.
10.1.7
Data quality assurance
All participant data relating to the study will be recorded on printed or eCRF unless transmitted 
to the Sponsor or designee electronically (eg, laboratory data). The Investigator is responsible for 
verifying that data entries are accurate and correct by physically or electronically signing the 
eCRF.
The Investigator must maintain accurate documentation (source data) that supports the 
information entered in the eCRF.
The Investigator must permit study-related monitoring, audits, IRB/IEC review, and regulatory 
agency inspections and provide direct access to source data documents.
The Sponsor or designee is responsible for the data management of this study, including quality 
checking of the data.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 48 of 73
Study monitors will perform ongoing source data verification to confirm that data entered into 
the eCRF by authorized site personnel are accurate, legible, contemporaneous, original, and 
attributable from source documents; that the safety and rights of participants are being protected; 
and that the study is being conducted in accordance with the currently approved protocol and any 
other study agreements, ICH-GCP, and all applicable regulatory requirements.
All essential documents must be retained by the Investigator for the minimum retention period 
mandatory under the applicable local laws and regulations. The Investigator will contact UCB 
for authorization prior to the destruction of any study records or in the event of accidental loss or 
destruction of any study records. The Investigator will also notify UCB should he/she relocate or 
move the study-related files to a location other than that specified in the Sponsor’s trial master 
file.
Quality tolerance limits will be established for the study using parameters related to participant 
safety reporting and reliability of study results. The parameters will be monitored throughout the 
study to identify systematic issues. Parameters used, parameter values, important deviations from 
the quality tolerance limits, and actions taken will be summarized in the clinical study report.
10.1.7.1
Case report form completion
The Investigator is responsible for prompt reporting of accurate, complete, and legible data in the 
eCRFs and in all required reports.
Any change or correction to the eCRF after saving must be accompanied by a reason for the 
change.
Corrections made after the Investigator’s review and approval (by means of a 
password/electronic signature) will be reapproved by the Investigator.
The Investigator should maintain a list of personnel authorized to enter data into the eCRF.
Detailed instructions will be provided in the eCRF Completion Guidelines.
10.1.7.2
Apps
The questionnaire data collected on the electronic Patient-Reported Outcome (ePRO) diary 
(SIAQ) will be uploaded to a central server database and will be 21 CFR Part 11 compliant. 
Appropriate training will be performed at the study sites. The SIAQ nor ePRO platform are 
intended to be used to influence treatment decisions of participants during conduct of the study 
and is also not intended to be used to collect or report safety-related information about the 
participant. The questionnaire responses will be analyzed at the end of the study.
10.1.8
Source documents
All source documents must be accurate, clear, unambiguous, permanent, and capable of being 
audited. They should be made using some permanent form of recording (ink, typing, printing, 
optical disc). They should not be obscured by correction fluid or have temporary attachments 
(such as removable self-stick notes). Photocopies and/or printouts of CRFs are not considered 
acceptable source documents.
Source documents are original records in which raw data are first recorded. These may include 
hospital/clinic/general practitioner records, charts, diaries, x-rays, laboratory results, printouts, 
pharmacy records, care records, ECG or other printouts, completed scales, quality of life 
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 49 of 73
questionnaires, or video, for example. Source documents should be kept in a secure, limited 
access area.
10.1.9
Study and site start and closure
The start of recruitment
The start of recruitment is the first participant’s first visit and is also the start date of the clinical 
study.
Study/site termination
The Sponsor designee reserves the right to close the study site or terminate the study at any time 
for any reason at the sole discretion of the Sponsor. Study sites will be closed upon study 
completion. A study site is considered closed when all required documents and study supplies 
have been collected and a study-site closure visit has been performed.
The Investigator may initiate study-site closure at any time, provided there is reasonable cause 
and sufficient notice is given in advance of the intended termination.
Reasons for the early closure of a study site by the Sponsor or Investigator may include, but are 
not limited to:
x
Failure of the Investigator to comply with the protocol, the requirements of the IRB/IEC or 
local health authorities, the Sponsor's procedures, or GCP guidelines
x
Inadequate recruitment of participants by the Investigator
x
Discontinuation of further study medication development
10.1.10
Publication policy
The results of this study may be published or presented at scientific meetings. If this is foreseen, 
the Investigator agrees to submit all manuscripts or abstracts to the Sponsor before submission. 
This allows the Sponsor to protect proprietary information and to provide comments.
The Sponsor will comply with the requirements for publication of study results. In accordance 
with standard editorial and ethical practice, the Sponsor will generally support publication of 
multicenter studies only in their entirety and not as individual site data. In this case, a 
coordinating Investigator will be designated by mutual agreement.
Authorship will be determined by mutual agreement and in line with International Committee of 
Medical Journal Editors authorship requirements.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 50 of 73
10.2
Appendix 2: Clinical laboratory tests
Not applicable.
COPY 
This document cannot be used to support any marketing authorization application 
and any extensions or variations thereof.

UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 51 of 73
10.3
Appendix 3: Adverse events – Definitions and procedures for 
recording, evaluating, follow up, and reporting
Definition of AE
AE definition
x
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally 
associated with the use of study medication, whether or not considered related to the study 
medication.
x
NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal 
laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use 
of study medication.
Events meeting the AE definition
x
Any abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety 
assessments (eg, ECG, radiological scans, vital signs measurements), including those that worsen 
from Visit 1, considered clinically significant in the medical and scientific judgment of the 
Investigator (ie, not related to progression of underlying disease).
x
Exacerbation of a chronic or intermittent pre-existing condition, including either an increase in 
frequency and/or intensity of the condition.
x
New conditions detected or diagnosed after study medication administration even though it may 
have been present before the start of the study.
x
Signs, symptoms, or the clinical sequelae of a suspected drug-drug interaction.
x
Signs, symptoms, or the clinical sequelae of a suspected overdose of either study medication or a 
concomitant medication. Overdose per se will not be reported as an AE/SAE unless it is an 
intentional overdose taken with possible suicidal/self-harming intent. Such overdoses should be 
reported regardless of sequelae.
x
"Lack of efficacy" or "failure of expected pharmacological action" per se will not be reported as 
an AE or SAE. Such instances will be captured in the efficacy assessments. However, the signs, 
symptoms, and/or clinical sequelae resulting from lack of efficacy will be reported as AE or SAE 
if they fulfill the definition of an AE or SAE.
Events NOT meeting the AE definition
x
Any clinically significant abnormal laboratory findings or other abnormal safety assessments 
which are associated with the underlying disease, unless judged by the Investigator to be more 
severe than expected for the participant’s condition.
x
The disease/disorder being studied or expected progression, signs, or symptoms of the 
disease/disorder being studied, unless more severe than expected for the study participant’s 
condition.
x
Medical or surgical procedure (eg, endoscopy, appendectomy): the condition that leads to the 
procedure is the AE.
x
Situations in which an untoward medical occurrence did not occur (social and/or convenience 
admission to a hospital).
x
Anticipated day-to-day fluctuations of pre-existing disease(s) or condition(s) present or detected at 
the start of the study that do not worsen.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 52 of 73
Definition of SAE
If an event is not an AE per definition above, then it cannot be a SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease).
A SAE is defined as any untoward medical occurrence that, at any dose:
a.
Results in death
b.
Is life-threatening
The term 'life-threatening' in the definition of 'serious' refers to an event in which the participant was at 
risk of death at the time of the event. It does not refer to an event which hypothetically might have 
caused death if it were more severe.
c.
Requires inpatient hospitalization or prolongation of existing hospitalization
In general, hospitalization signifies that the participant has been detained (usually involving at least an 
overnight stay) at the hospital or emergency ward for observation and/or treatment that would not have 
been appropriate in the physician’s office or outpatient setting. Complications that occur during 
hospitalization are AEs. If a complication prolongs hospitalization or fulfills any other serious criteria, 
the event is serious. When in doubt as to whether “hospitalization” occurred or was necessary, the AE 
should be considered serious.
Hospitalization for elective treatment of a pre-existing condition that did not worsen from Visit 1 is not 
considered an AE.
d.
Results in persistent disability/incapacity
x
The term disability means a substantial disruption of a person’s ability to conduct normal life 
functions.
x
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such as uncomplicated headache, nausea, vomiting, diarrhea, influenza, and accidental trauma 
(eg, sprained ankle) which may interfere with or prevent everyday life functions but do not 
constitute a substantial disruption.
e.
Is a congenital anomaly/birth defect
f.
Important medical events:
x
Medical or scientific judgment should be exercised in deciding whether SAE reporting is 
appropriate in other situations, such as important medical events that may not be immediately 
life-threatening or result in death or hospitalization but may jeopardize the participant or may 
require medical or surgical intervention to prevent 1 of the other outcomes listed in the above 
definition. These events should usually be considered serious.
x
Examples of such events include, but are not limited to, potential Hy’s law, invasive or 
malignant cancers, intensive treatment in an emergency room or at home for allergic 
bronchospasm, blood dyscrasias or convulsions that do not result in hospitalization, or 
development of drug dependency or drug abuse.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 53 of 73
Recording and follow up of AE and/or SAE
AE and SAE recording
x
When an AE/SAE occurs, it is the responsibility of the Investigator to review all 
documentation (eg, hospital progress notes, laboratory reports, and diagnostics reports) related 
to the event.
x
The Investigator will then record all relevant AE/SAE information in the eCRF.
x
It is not acceptable for the Investigator to send photocopies of the participant’s medical records 
to UCB in lieu of completion of the UCB AE/SAE eCRF page.
x
There may be instances when copies of medical records for certain cases are requested by 
UCB. In this case, all participant identifiers, with the exception of the participant number, will 
be redacted on the copies of the medical records before submission to UCB.
x
The Investigator will attempt to establish a diagnosis of the event based on signs, symptoms, 
and/or other clinical information. Whenever possible, the diagnosis (not the individual 
signs/symptoms) will be documented as the AE/SAE.
Assessment of intensity
The Investigator will make an assessment of intensity for each AE and SAE reported during the study 
and assign it to 1 of the following categories:
x
Mild: an event that is easily tolerated by the participant, causing minimal discomfort and not 
interfering with everyday activities.
x
Moderate: an event that causes sufficient discomfort and interferes with normal everyday 
activities.
x
Severe: an event that prevents normal everyday activities. An AE that is assessed as severe 
should not be confused with a SAE. Severe is a category utilized for rating the intensity of an 
event; and both AEs and SAEs can be assessed as severe.
x
An event is defined as ‘serious’ when it meets at least 1 of the predefined outcomes as 
described in the definition of a SAE, NOT when it is rated as severe (eg, a severe AE may be 
either serious or not serious, depending on whether these criteria are also met).
The National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) 
should be used as a supportive standardization instrument to evaluate AEs and SAEs but the final 
intensity grading by the Investigator must be mild, moderate, or severe.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 54 of 73
Assessment of causality
x
The Investigator is obligated to assess the relationship between study medication and each 
occurrence of each AE/SAE.
x
A "reasonable possibility" of a relationship conveys that there are facts, evidence, and/or 
arguments to suggest a causal relationship, rather than a relationship cannot be ruled out.
x
The Investigator will use clinical judgment to determine the relationship.
x
Alternative causes, such as underlying disease(s), concomitant therapy, and other risk factors, as 
well as the temporal relationship of the event to study medication administration will be 
considered and investigated.
x
The Investigator will also consult the IB and/or Product Information, for marketed products, in 
his/her assessment.
x
For each AE/SAE, the Investigator must document in the medical notes that he/she has reviewed 
the AE/SAE and has provided an assessment of causality.
x
There may be situations in which a SAE has occurred and the Investigator has minimal 
information to include in the initial report to UCB. However, it is very important that the 
Investigator always make an assessment of causality for every event before the initial 
transmission of the SAE data to UCB.
x
The Investigator may change his/her opinion of causality in light of follow-up information and 
send a SAE follow-up report with the updated causality assessment.
x
The causality assessment is 1 of the criteria used when determining regulatory reporting 
requirements.
Follow up of AEs and SAEs
x
The Investigator is obligated to perform or arrange for the conduct of supplemental measurements 
and/or evaluations as medically indicated or as requested by UCB to elucidate the nature and/or 
causality of the AE or SAE as fully as possible. This may include additional laboratory tests or 
investigations, histopathological examinations, or consultation with other health care 
professionals.
x
An AE should be followed until it has resolved, has a stable sequelae, the Investigator determines 
that it is no longer clinically significant, or the participant is lost to follow up. This follow-up 
requirement applies to AEs, SAEs, and AEs of special interest.
x
If a participant dies during participation in the study or during a recognized follow-up period, the 
Investigator will provide UCB with a copy of any postmortem findings, including histopathology.
x
New or updated information will be recorded in the originally completed eCRF.
x
The Investigator will submit any updated SAE data to the Sponsor within 24 hours of receipt of 
the information.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 55 of 73
Reporting of SAEs
SAE reporting to UCB via an electronic data collection tool
x
The primary mechanism for reporting a SAE to UCB will be the electronic data collection tool.
x
If the electronic system is unavailable for more than 24 hours, then the site will use the paper SAE 
data collection tool (see next section).
x
The site will enter the SAE data into the electronic system as soon as it becomes available.
x
After the study is completed at a given site, the electronic data collection tool will be taken off-
line to prevent the entry of new data or changes to existing data.
x
If a site receives a report of a new SAE from a study participant or receives updated data on a 
previously reported SAE after the electronic data collection tool has been taken off-line, then the 
site can report this information on a paper SAE form (see next section) or to the Medical Monitor 
by telephone.
x
Contacts for SAE reporting can be found in the SERIOUS ADVERSE EVENT AND 
MEDICATION ERRORS REPORTING section at the front of the protocol.
SAE reporting to UCB via paper CRF
x
Facsimile transmission of the SAE paper CRF is the preferred method to transmit this information 
to the Medical Monitor.
x
In rare circumstances and in the absence of facsimile equipment, notification by telephone is 
acceptable with a copy of the SAE data collection tool sent by overnight mail or courier service.
x
Initial notification via telephone does not replace the need for the Investigator to complete and 
sign the SAE CRF pages within the designated reporting time frames.
x
Contacts for SAE reporting can be found in the SERIOUS ADVERSE EVENT AND 
MEDICATION ERRORS REPORTING section at the front of the protocol.
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15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 56 of 73
10.4
Appendix 4: Contraceptive guidance and collection of 
pregnancy information
Definitions
Woman of childbearing potential
A woman is considered fertile following menarche and until becoming postmenopausal unless 
permanently sterile (see below).
Women in the following categories are not considered WOCBP:
1. Premenarchal
2. Premenopausal female with 1 of the following:

Documented hysterectomy

Documented bilateral salpingectomy

Documented bilateral oophorectomy
Note:  Documentation can come from the site personnel’s review of the participant’s medical 
records, medical examination, or medical history interview.
3. Postmenopausal female

A postmenopausal state is defined as no menses for 12 months without an alternative 
medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal 
range may be used to confirm a postmenopausal state in women not using hormonal 
contraception or hormonal replacement therapy (HRT). However, in the absence of 
12 months of amenorrhea, a single FSH measurement is insufficient.

Females on HRT and whose menopausal status is in doubt will be required to use 1 of the 
nonestrogen hormonal highly effective contraception methods if they wish to continue 
their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation 
of postmenopausal status before study enrollment.
Contraception guidance
Male participants
Male participants with female partners of childbearing potential are eligible to participate if they 
agree to ONE of the following during the Treatment Period and for at least 40 days after the last 
dose of study medication:
x
Are abstinent from penile-vaginal intercourse as their usual and preferred lifestyle (abstinent 
on a long-term and persistent basis) and agree to remain abstinent.
x
Agree to use a male condom when having penile-vaginal intercourse with a woman of 
childbearing potential who is not currently pregnant.
In addition, male participants must refrain from donating sperm for the duration of the study and 
for 40 days after the last dose of study medication.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 57 of 73
Male participants with a pregnant or breastfeeding partner must agree to remain abstinent from 
penile-vaginal intercourse or use a male condom during each episode of penile penetration 
during the Treatment Period and for at least 40 days after the last dose of study medication.
Female participants
Female participants of childbearing potential are eligible to participate if they agree to use a 
highly effective method of contraception consistently and correctly as described in the table 
below.
Highly effective contraceptive methodsa
Highly effective contraceptive methods that are user dependentb
Failure rate of <1% per year when used consistently and correctly.
Combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of 
ovulationc
x
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x
Intravaginal
x
Transdermal
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x
Oral
x
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Highly effective methods that are user independentc
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x
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x
Intrauterine hormone-releasing system (IUS)
x
Bilateral tubal occlusion
Vasectomized partner
A vasectomized partner is a highly effective contraception method provided that the partner is the sole 
male sexual partner of the WOCBP and the absence of sperm has been confirmed. If not, an additional 
highly effective method of contraception should be used.
Sexual abstinence
Sexual abstinence is considered a highly effective method only if defined as refraining from 
heterosexual intercourse during the entire period of risk associated with the study medication. The 
reliability of sexual abstinence needs to be evaluated in relation to the duration of the study and the 
preferred and usual lifestyle of the participant.
NOTES:
a) In case of newly started contraception pills/IUDs, the Investigator should consider the correct timing of 
starting/applying such methods in relation to the menstrual cycle and the manufacturing instruction as to when 
these newly started methods would become effective.
b) Typical use failure rates may differ from those when used consistently and correctly. Use should be consistent 
with local regulations regarding the use of contraceptive methods for participants participating in clinical studies.
c) Hormonal contraception may be susceptible to interaction with the study medication, which may reduce the 
efficacy of the contraceptive method. In this case, 2 highly effective methods of contraception should be utilized 
during the Treatment Period and for at least 40 days after the last dose of study medication.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 58 of 73
Pregnancy testing
x
WOCBP should only be included after a confirmed menstrual period and a negative highly 
sensitive urine pregnancy test.
x
Additional pregnancy testing will be performed as described in the SoA (Section 1.3).
x
Pregnancy testing will be performed whenever a menstrual cycle is missed or when 
pregnancy is otherwise suspected.
x
Pregnancy testing, with a sensitivity of 25mIU/mL will be performed.
Male participants with partners who become pregnant
x
The Investigator will attempt to collect pregnancy information on any male participant’s 
female partner who becomes pregnant while the male participant is in this study. This applies 
only to male participants who receive study medication.
x
After obtaining the necessary signed informed consent from the pregnant female partner 
directly, the Investigator will record pregnancy information on the appropriate form and 
submit it to the Sponsor within 24 hours of learning of the partner’s pregnancy. The female 
partner will also be followed to determine the outcome of the pregnancy. Information on the 
status of the mother and child will be forwarded to the Sponsor. Generally, the follow up will 
be at least 12 months after the delivery date. Any termination of the pregnancy will be 
reported regardless of fetal status (presence or absence of anomalies) or indication for the 
procedure.
Female participants who become pregnant
x
Any female participant who becomes pregnant while participating in the study will be 
withdrawn from the study.
x
The Investigator will collect pregnancy information on any female participant who becomes 
pregnant while participating in this study. Information will be recorded on the appropriate 
form and submitted to the Sponsor within 24 hours of learning of a participant's pregnancy. 
The participant will be followed to determine the outcome of the pregnancy. The Investigator
will collect follow-up information on the participant and the neonate and the information will 
be forwarded to the Sponsor. Generally, the follow-up will be at least 12 months after the 
delivery date. Any termination of pregnancy will be reported, regardless of fetal status 
(presence or absence of anomalies) or indication for the procedure.
x
While pregnancy itself is not considered to be an AE or SAE, any pregnancy complication or 
elective termination of a pregnancy will be reported as an AE or SAE. A spontaneous 
abortion is always considered to be a SAE and will be reported as such. Any poststudy 
pregnancy-related SAE considered reasonably related to the study medication by the 
Investigator will be reported to the Sponsor as described in Section 8.3.5. While the 
Investigator is not obligated to actively seek this information in former study participants, he 
or she may learn of a SAE through spontaneous reporting.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 59 of 73
10.5
Appendix 5: Genetics
Not applicable.
COPY 
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and any extensions or variations thereof.

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15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 60 of 73
10.6
Appendix 6: Liver safety – suggested actions and follow-up 
assessments
Not applicable.
PUBLIC COPY 
This document cannot be used to support any marketing authorization application 
and any extensions or variations thereof.

UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 61 of 73
10.7
Appendix 7: Device AEs, ADEs, SAEs, and device deficiencies: 
Definition and procedures for recording, evaluating, follow up, 
and reporting
The definitions and procedures detailed in this appendix are in accordance with ISO 14155 and 
the European Medical Device Regulation 2017/745 for clinical device research (if applicable).
Both the Investigator and the Sponsor will comply with all local medical device reporting 
requirements.
The detection and documentation procedures described in this protocol apply to all Sponsor 
medical devices provided for use in the study. See Section 6.1.1 for the description of the 
Sponsor medical device.
10.7.1
Definitions of AE and ADE
AE and ADE Definitions
x
An AE is defined as any untoward medical occurrence, unintended disease or injury, or untoward 
clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, 
whether or not related to the investigational medical device. This definition includes events related 
to the investigational medical device or comparator and events related to the procedures involved 
except for events in users or other persons, which only include events related to investigational 
devices.
x
An ADE is defined as an adverse event related to the use of an investigational medical device. 
This definition includes any adverse events resulting from insufficient or inadequate instructions 
for use, deployment, implantation, installation, or operation, or any malfunction of the 
investigational medical device as well as any event resulting from use error or from intentional 
misuse of the investigational medical device.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 62 of 73
10.7.2
Definitions of SAE, SADE, and USADE
If an event is not an AE per definition above, then it cannot be a SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease).
A SAE is an AE that:
a.
Led to death
b.
Led to serious deterioration in the health of the participant, that either resulted in:
1.
A life-threatening illness or injury. The term life-threatening in the definition of serious refers 
to an event in which the participant was at risk of death at the time of the event. It does not 
refer to an event which hypothetically might have caused death if it were more severe
2.
A permanent impairment of a body structure or a body function
3.
Inpatient or prolonged hospitalization. Planned hospitalization for a pre-existing condition, or a 
procedure required by the protocol, without serious deterioration in health, is not considered a 
SAE
4.
Medical or surgical intervention to prevent life-threatening illness or injury or permanent 
impairment to a body structure or a body function
c.
Led to fetal distress, fetal death, or a congenital abnormality or birth defect
SADE definition
x
A SADE is defined as an adverse device effect that has resulted in any of the consequences 
characteristic of a serious adverse event.
USADE definition
x
An USADE is a serious adverse device effect which by its nature, incidence, severity, or outcome 
has not been identified in the current version of the risk analysis report (see Section 8.3.8).
10.7.3
Definition of device deficiency
Device deficiency definition
x
A device deficiency is an inadequacy of a device with respect to its identity, quality, durability, 
reliability, safety, or performance. Device deficiencies include malfunctions, use errors, and 
inadequate labeling.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 63 of 73
10.7.4
Recording and follow up of AE and/or SAE and device deficiencies
AE, SAE, and device deficiency recording
x
When an AE/SAE/device deficiency occurs, it is the responsibility of the Investigator to review all 
documentation (eg, hospital progress notes, laboratory reports, and diagnostics reports) related to 
the event.
x
The Investigator will then record all relevant AE/SAE/device deficiency information in the 
participant’s medical records, in accordance with the Investigator’s normal clinical practice and on 
the appropriate form of the eCRF.
x
It is not acceptable for the Investigator to send photocopies of the participant’s medical records to 
UCB in lieu of completion of the AE/SAE/device deficiency eCRF page.
x
There may be instances when copies of medical records for certain cases are requested by UCB. In 
this case, all participant identifiers, with the exception of the participant number, will be redacted 
on the copies of the medical records before submission to UCB.
x
The Investigator will attempt to establish a diagnosis of the event based on signs, symptoms, 
and/or other clinical information. Whenever possible, the diagnosis (not the individual 
signs/symptoms) will be documented as the AE/SAE.
x
For device deficiencies, it is very important that the Investigator describes any corrective or 
remedial actions taken to prevent recurrence of the deficiency
o
A remedial action is any action other than routine maintenance or servicing of a medical 
device where such action is necessary to prevent recurrence of a device deficiency. This 
includes any amendment to the device design to prevent recurrence. The Investigator should 
complete a Product Complaint Form for all reported device deficiencies.
Assessment of intensity
The Investigator will make an assessment of intensity for each AE/SAE/device deficiency reported 
during the study and assign it to 1 of the following categories:
x
Mild: An event that is easily tolerated by the participant, causing minimal discomfort and not 
interfering with everyday activities.
x
Moderate: An event that causes sufficient discomfort and interferes with normal everyday 
activities.
x
Severe: An event that prevents normal everyday activities. An AE that is assessed as severe 
should not be confused with a SAE. Severe is a category utilized for rating the intensity of an 
event, and both AEs and SAEs can be assessed as severe.
x
An event is defined as serious when it meets at least 1 of the predefined outcomes as described 
in the definition of a SAE, NOT when it is rated as severe.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 64 of 73
Assessment of causality
x
The Investigator is obligated to assess the relationship between study intervention and each 
occurrence of each AE/SAE/device deficiency.
x
A “reasonable possibility” of a relationship conveys that there are facts, evidence, and/or 
arguments to suggest a causal relationship, rather than a relationship cannot be ruled out.
x
The Investigator will use clinical judgment to determine the relationship.
x
Alternative causes, such as underlying disease(s), concomitant therapy, and other risk factors, as 
well as the temporal relationship of the event to study intervention administration, will be 
considered and investigated.
x
The Investigator will also consult the IB in his/her assessment.
x
For each AE/SAE/device deficiency, the Investigator must document in the medical notes that 
he/she has reviewed the AE/SAE/device deficiency and has provided an assessment of causality.
x
There may be situations in which a SAE has occurred and the Investigator has minimal 
information to include in the initial report to UCB. However, it is very important that the 
Investigator always make an assessment of causality for every event before the initial transmission 
of the SAE data to UCB.
x
The Investigator may change his/her opinion of causality in light of follow-up information and 
send a SAE follow-up report with the updated causality assessment.
x
The causality assessment is 1 of the criteria used when determining regulatory reporting 
requirements.
Follow up of AE/SAE/device deficiency
x
The Investigator is obligated to perform or arrange for the conduct of supplemental measurements 
and/or evaluations as medically indicated or as requested by UCB to elucidate the nature and/or 
causality of the AE/SAE/device deficiency as fully as possible. This may include additional 
laboratory tests or investigations, histopathological examinations, or consultation with other health 
care professionals.
x
If a participant dies during participation in the study or during a recognized follow-up period, the 
Investigator will provide UCB with a copy of any postmortem findings, including histopathology.
x
New or updated information will be recorded in the originally completed eCRF.
x
The Investigator will submit any updated SAE data to UCB within 24 hours of receipt of the 
information.
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 65 of 73
10.7.5
Reporting of SAEs
SAE reporting to UCB via an electronic data collection tool
x
The primary mechanism for reporting a SAE to UCB will be the electronic data collection tool.
x
If the electronic system is unavailable, then the site will use the paper SAE data collection tool 
(see next section) in order to report the event within 24 hours.
x
The site will enter the SAE data into the electronic system as soon as it becomes available.
x
After the study is completed at a given site, the electronic data collection tool will be taken 
off-line to prevent the entry of new data or changes to existing data.
x
If a site receives a report of a new SAE from a study participant or receives updated data on a 
previously reported SAE after the electronic data collection tool has been taken off-line, then the 
site can report this information on a paper SAE form (see next section) or to the Medical Monitor
by telephone.
x
Contacts for SAE reporting can be found in the SERIOUS ADVERSE EVENT AND 
MEDICATION ERRORS REPORTING section at the front of the protocol.
SAE reporting to UCB via paper CRF
x
Facsimile transmission of the SAE paper CRF is the preferred method to transmit this information 
to the Medical Monitor.
x
In rare circumstances and in the absence of facsimile equipment, notification by telephone is 
acceptable with a copy of the SAE data collection tool sent by overnight mail or courier service.
x
Initial notification via telephone does not replace the need for the Investigator to complete and 
sign the SAE CRF pages within the designated reporting time frames.
x
Contacts for SAE reporting can be found in the SERIOUS ADVERSE EVENT AND 
MEDICATION ERRORS REPORTING section at the front of the protocol.
10.7.6
Reporting of SADEs
SADE Reporting to UCB
NOTE: There are additional reporting obligations for device deficiencies that are potentially related to 
SAEs that must fulfill the legal responsibility to notify appropriate regulatory authorities and other 
entities about certain safety information relating to medical devices being used in clinical studies.
x
Any device deficiency that is associated with a SAE must be reported to the Sponsor within 
24 hours after the Investigator determines that the event meets the definition of a device 
deficiency.
x
The Sponsor shall review all device deficiencies and determine and document in writing 
whether they could have led to a SAE. These shall be reported to the regulatory authorities and 
IRBs/IECs as required by national regulations.
x
Contacts for SAE reporting can be found in the SAFETY REPORTING OF ADVERSE 
EVENTS (SERIOUS AND NONSERIOUS) AND DEVICE DEFICIENCES section at the 
front of the protocol.
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15 Dec 2023
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Zilucoplan
DV0013
Confidential
Page 66 of 73
10.8
Appendix 8: Rapid alert procedures
Not applicable.
COPY 
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Zilucoplan
DV0013
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10.9
Appendix 9: Country-specific requirements
Not applicable.
PUBLIC COPY 
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and any extensions or variations thereof.

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15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 68 of 73
10.10
Appendix 10: Abbreviations and trademarks
ADE
adverse device effect
AE
adverse event
AI
auto-injector
apps
applications
CI
confidence interval
COVID-19
Coronavirus Disease 2019
C-SSRS
Columbia-Suicide Severity Rating Scale
ECG
electrocardiogram
eCRF
electronic case report form
ePRO
electronic Patient-Reported Outcome
EudraCT
European Union Drug Regulating Authorities Clinical Trials
FSH
follicle stimulating hormone
GCP
Good Clinical Practice
gMG
generalized myasthenia gravis
HRT
hormone replacement therapy
IB
Investigator’s Brochure
ICF
informed consent form
ICH
International Council for Harmonisation
IDE
Investigational Device Exemption
IEC
Independent Ethics Committee
IFU
instructions for use
IMP
investigational medicinal product
IND
Investigational New Drug
IRB
Institutional Review Board
ISO
International Organization for Standardization
IUD
intrauterine device
IV
intravenous
IXRS
interactive response technology
MG
myasthenia gravis
NCT number
ClinicalTrials.gov identifier
OLE
open-label extension
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UCB
15 Dec 2023
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Zilucoplan
DV0013
Confidential
Page 69 of 73
PK
pharmacokinetic(s)
PFS
prefilled syringe
SADE
serious adverse device effect
SAE
serious adverse event
sc
subcutaneous(ly)
SFU
Safety Follow-Up
SIAQ
Self-Injection Assessment Questionnaire
SoA
Schedule of Activities
SOP
standard operating procedure
SS
Safety Set
TEAE
treatment-emergent adverse event
USADE
unanticipated serious adverse device effect
WOCBP
woman of childbearing potential
ZLP
zilucoplan
ZLP-AI
zilucoplan-auto-injector
PUBLIC COPY 
tial
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ctct
authorization application 
and any extensions or variations thereof.

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Page 70 of 73
10.11
Appendix 11: Protocol amendment history
Not applicable.
COPY 
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and any extensions or variations thereof.

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15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 71 of 73
10.12
Appendix 12: Medication errors associated with 
self-administration
The definitions and procedures detailed in this appendix are in accordance with the 
pharmacovigilance obligations detailed in Title IX of Directive 2001/83/EC and Regulation 
(EC) 726/2004, Chapter 3, Article 28 with regard to the recording, reporting, and assessment of 
suspected adverse reactions (serious and nonserious) associated with an error in prescribing, 
storing, dispensing, preparing for administration, or administering a medicinal product for human 
use authorized in the EU, which have been adapted as per DV0013 protocol specifications. All 
medication errors will be recorded and reported to the sponsor or designee as indicated in 
Section 8.3.6.
Medication Error Definition
Medication error: A medication error is an unintended failure in the drug treatment process that leads 
to, or has the potential to lead to, harm to the patient. A failure in the drug treatment process does not 
refer to lack of efficacy of the drug, rather to human- or process-mediated failures.
The medication error can be further classified based on the factual information on the case, into the 
following categories:
x
Medication errors associated with adverse reaction(s): result from an error (which has 
already occurred or accomplished) in the medication use associated with harm to the study 
participant.
x
Medication errors without harm: result from an error (which has already occurred or 
accomplished) in the medication use associated with no harm to the study participant.
x
Intercepted medication errors (or near miss): in which an intervention (eg, the timely 
check of the incorrect preparation and administration of the study medication) has 
prevented actual harm being caused to the study participant.
x
Potential medication errors: are the recognition of circumstances that could lead to a 
medication error, which may or may not involve a study participant.
The term potential medication error refers to all possible mistakes in the storing, preparation for 
administration, or administration of the IMP by the study participant and may lead to:

A medication error with harm, but without knowing the actual cause

A medication error without harm and without knowing the actual cause

A medication error without harm but with the awareness of the actual cause
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 72 of 73
11
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UCB
15 Dec 2023
Clinical Study Protocol
Zilucoplan
DV0013
Confidential
Page 73 of 73
SPONSOR DECLARATION
I confirm that I have carefully read and understand this protocol and agree to conduct this 
clinical study as outlined in this protocol, according to Clinical Trial Regulation EU 536/2014, 
and according to current Good Clinical Practice.
COPY 
This document cannot be used to support any marketing authorization application 
and any extensions or variations thereof.