Multicenter, Retrospective Data Collection of Routine Clinical Use With the Spectra Optia® Apheresis System for White Blood Cell Depletion
Conditions: Leukocytosis
Sponsor: Terumo BCT



Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
Page 1 of 10 
 
 
Retrospective Data Collection Protocol 
Amendment 1.0 
 
Protocol Title: 
Multicenter, Retrospective Data Collection of Routine Clinical Use 
with the Spectra Optia® Apheresis System for White Blood Cell 
Depletion  
 
Device: 
Spectra Optia® Apheresis System for WBC Depletion 
 
Sponsor: 
Terumo BCT Europe NV 
Ikaroslaan 41 
1930 Zaventem 
Belgium  
 
Sponsor Contact: 
Heather Pidcoke, MD, PhD 
Director, Clinical Safety and PRT Scientific Affairs 
Phone:  +1 (303) 231-4805 
Email:  Heather.Pidcoke@terumobct.com 
 
 
 
 
 
Version/Date: 
Original version 1.0/18 Feb 2014 
Amendment 1.0/28 Sep 2016 
 
 
 
 
 
Confidentiality Statement 
The information contained in this document, particularly unpublished data, is the property or under 
control of Terumo BCT Inc. and is provided to you in confidence as an investigator, potential 
investigator, or consultant, for review by you, your staff, and an applicable Institutional Review 
Board/Ethics Committee.  The information is only to be used by you in connection with authorized 
clinical studies of the investigational device described in the protocol.  You will not disclose any of the 
information to others without written authorization from Terumo BCT Inc. except to the extent 
necessary to obtain informed consent from those persons who may participate in the clinical trial. 

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
Page 3 of 10 
 
AMENDMENT 1.0 SUMMARY OF CHANGES 
Protocol Title: 
Multicenter, Retrospective Data Collection of Routine Clinical 
Use with the Spectra Optia® Apheresis System for White 
Blood Cell Depletion   
Protocol Number: 
CTS-5043 
Version/Date: 
Original version 1.0/18 Feb 2014 
Amendment 1.0/28 Sep 2016 
Rationale: 
This amendment adds to the protocol original version 1.0, 
dated 18 February 2014. 
 
This amendment was made to address regulatory agency 
feedback and to extend the duration of safety data collection 
following the apheresis procedure.  
Changes from  
Original Version 1.0: 
Previously during this retrospective study, safety data were 
collected during the White Blood Cell Depletion (WBCD) 
apheresis procedure and for up 2 hours post-procedure.  
 
With this amendment, safety data (eg, adverse events [AE], 
blood product transfusions, and clinical laboratory results, as 
available) will be collected for the 24-hour time period 
following the WBCD procedure. No other changes are made 
to study conduct, subject enrollment, or data analysis. 
 
 

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
Page 4 of 10 
 
SYNOPSIS 
Protocol Title: 
Multicenter, Retrospective Data Collection of Routine Clinical Use with the Spectra 
Optia® Apheresis System for White Blood Cell Depletion  
Protocol 
Number: 
CTS-5043 
Target 
Population: 
Patients who have undergone WBCD via the Spectra Optia Apheresis System which 
is likely to include patients with severe leukocytosis in acute leukemia (acute 
myeloid leukemia [AML], acute lymphoblastic leukemia [ALL], or in chronic 
myeloid leukemia [CML]) or to prevent tumor lysis syndrome. 
Device 
Description: 
The Spectra Optia System for WBCD is comprised of (1) hardware (the actual 
apheresis machine) and a removable centrifuge filler (2) a sterile single-use 
disposable tubing set (the Spectra Optia IDL set) and (3) embedded software V7.2 or 
higher for WBCD. 
Intended Use: 
The Spectra Optia Apheresis System, a blood component separator, is intended for 
use in therapeutic apheresis and cell collections and may be used to perform WBCD. 
Objective: 
To gather a broader knowledge and information from routine clinical use on the 
performance and safety of WBCD via the Spectra Optia Apheresis System. 
Outcome 
Measures: 
1. Percent decrease in white blood cell (WBC) count in patient blood following 
apheresis procedure. 
2. Percent of processed WBC which are collected, i.e. Collection efficiency for 
WBC achieved by Spectra Optia.   
3. Adverse events during the WBCD apheresis procedure and for up 24 hours 
post-procedure. 
Inclusion 
Criteria: 
Patients having received a minimum of 1 WBCD procedure via the Spectra Optia 
Apheresis System. 
Protocol 
Design: 
This is a multicenter, retrospective data collection to evaluate the in routine use 
performance and safety of WBCD performed via the Spectra Optia Apheresis 
System. 
Data Collection 
Duration: 
Routine use data will be collected retrospectively from November 2011 to December 
2013. 
Statistical 
Methodology: 
Patient demographics and performance parameters will be summarized with the 
mean, standard deviation, median, and range for continuous variables and with 
frequencies and percentages for discrete variables. 

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
Page 5 of 10 
 
TABLE OF CONTENTS 
CLINICAL INVESTIGATION PLAN APPROVAL ...............................................................2 
AMENDMENT 1.0 SUMMARY OF CHANGES ....................................................................3 
SYNOPSIS .................................................................................................................................4 
TABLE OF CONTENTS ...........................................................................................................5 
LIST OF ABBREVIATIONS ....................................................................................................6 
1.0 INTRODUCTION .............................................................................................................7 
1.1 
Rationale for Amendment 1.0 ..................................................................................7 
2.0 DATA COLLECTION ......................................................................................................8 
2.1 
Ethics Committee .....................................................................................................8 
2.2 
Informed Consent.....................................................................................................8 
2.3 
Data Handling and Record keeping .........................................................................8 
2.4 
Data for Collection ...................................................................................................8 
APPENDIX 1    INVESTIGATOR SIGNATURE ..................................................................10 

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
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LIST OF ABBREVIATIONS 
 
ALL 
Acute lymphoblastic leukemia 
AML 
Acute myeloid leukemia 
AE 
Adverse Event 
CML 
Chronic myeloid leukemia 
CRF 
Case Report Form 
EC 
Ethics Committee 
INR 
International Normalized Ratio 
PT 
Prothrombin time 
SAE 
Serious Adverse Event 
WBC 
White blood cell 
WBCD White blood cell depletion 
 

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
Page 7 of 10 
 
1.0 INTRODUCTION 
White blood cell depletion (WBCD) by apheresis procedures have been conducted in patients for 
the past 30 years to rapidly reduce the concentration of white blood cells (WBCs) that have 
proliferated to levels in the circulation that could cause serious injury. However, these depletion 
procedures are practiced infrequently and are performed emergently in patients at risk of 
life-threatening complications due to high WBC counts. For these reasons, prospective, 
controlled trials are not feasible and have not been done.   
 
The purpose of this retrospective data collection is to gather routine use data to further support 
the use of the Spectra Optia Apheresis System for WBCD procedures, which has been in clinical 
use in Europe since November 2011. Additionally, the objective of this retrospective data 
collection is to gather a broader knowledge and information from routine clinical use on the 
performance and safety of WBCD via the Spectra Optia® Apheresis System. 
1.1 
Rationale for Amendment 1.0 
The protocol original version 1.0 (dated 18 Feb 2014) was conducted and the device 
performance and safety data were summarized, including retrospective safety data for 2 hours 
following the apheresis procedure. Upon consideration of study results by the US Food and Drug 
Administration, regulators requested retrospective safety data not just for the 2 hours following 
the apheresis procedure, but also for the full 24 hours following the apheresis procedure. 
 
This amendment was made to address regulatory agency feedback and to extend the duration of 
safety data collection following the apheresis procedure. With this amendment, safety data (eg, 
adverse events [AEs], blood product transfusions, and clinical laboratory results, as available) 
will be collected for the full 24-hour time period following the WBCD procedure, as requested 
by the US Food and Drug Administration. Under protocol version 1.0 (dated 18 Feb 2014), 
safety data were collected for the first 2 hours following apheresis; this amendment will collect 
safety data in the 22-hour time period from 2 hours postapheresis until 24 hours postapheresis. 
 
This protocol amendment does not make changes to any of the following topics defined in the 
protocol original version (dated 18 Feb 2014): the study population, the definition of AEs and 
serious adverse events (SAEs), AE characterizations (eg, severity, action taken, relationship, 
outcome, SAE criteria), data analysis, or study management procedures.  
 
This amendment does collect the following new information:  
• Collection of AEs in the 22-hour time period from 2 hours postapheresis until 24 hours 
postapheresis 
• Additional characterization of AE relationship to underlying disease or treatment for 
underlying disease (eg, chemotherapy) 
• Reason for transfusion of blood products, if transfusions were administered in the 
24-hour period postapheresis 
• Postapheresis clinical laboratory results, as available     

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
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2.0   DATA COLLECTION 
2.1 
Ethics Committee 
The Institution’s Ethics Committee (EC), or other committee functioning in a similar capacity, 
will review and approve the retrospective data collection protocol amendment. After approval by 
the EC, documentation of approval will be sent to Terumo BCT before any data are collected for 
this study. 
2.2 
Informed Consent 
Obtaining patient consent retrospectively and in this very sick patient population is not feasible. 
Therefore, to protect patient privacy, patient identifier information (e.g., name, initials, birth 
date, and race) will not be collected in this retrospective data collection study. An EC waiver to 
collect these de-identified data will be obtained.   
2.3 
Data Handling and Record keeping 
All required data for this protocol will be gathered from subject medical records and recorded in 
unique Case Report Forms (CRFs) designed to specifically capture safety data for the 24-hour 
time period following the WBCD procedure. All data must be recorded in English.   
2.4 
Data for Collection 
Clinical routine-use data have been collected retrospectively, between November 2011 and 
December 2013, from centers experienced with the Spectra Optia System’s WBCD procedure. 
 
With this amendment, additional safety data will be collected for the 22-hour time period from 
2 hours postapheresis until 24 hours postapheresis.  
 
Specifically, the following new data will be collected: 
1. Adverse event data for AEs that occurred during the 24-hour time period following the 
WBCD procedure (eg, date/time of start and end, ongoing, severity, action taken relationship 
to device/apheresis procedure/underlying disease/treatment for underlying disease, outcome, 
SAE, unexpected adverse device effect). 
 
Note: The Investigator at each site will document his/her opinion of the relationship of the 
event to the procedures or underlying disease. The criteria below, in addition to good clinical 
judgment, should be used in determining relatedness. 
 
Not Related:  The event is clearly related to factors other than the study device and/or 
procedure(s), such as the subject’s clinical state. 
 
Possibly Related:  The event follows a reasonable temporal sequence from the time of study 
treatment administration/ procedure, and/or follows a known response pattern to study 
device/procedure(s) but could have been produced by other factors, such as the subject’s 
clinical state or other therapeutic interventions. 

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
Page 9 of 10 
 
 
Probably Related:  The event follows a reasonable temporal sequence from the time of study 
device/procedure(s) and cannot be reasonably explained by other factors, such as the 
subject’s clinical state or therapeutic interventions. 
 
Definitely Related:  The event follows a reasonable temporal sequence from the time of study 
device/procedure(s), and follows a known response pattern, and cannot be reasonably 
explained by other factors. In addition, the event occurs immediately following study 
procedure(s), and/or improves on stopping the study procedure, and/or reappears on 
resumption of study procedure(s). 
  
2. Blood products transfused during the 24-hour time period following the WBCD procedure 
(eg, reason [AE or underlying disease], blood product, number of units, date/time of start and 
end). 
 
3. Postapheresis clinical laboratory results, as available (eg, prothrombin time [PT], 
international normalized ratio [INR], WBC count, platelet count, hematocrit, red blood cell 
count). 

Spectra Optia® Apheresis System 
 
Terumo BCT 
Retrospective Data Collection Protocol Amendment:  CTS-5043 
Amendment 1.0 
 
 
 
 
 
Confidential 
Page 10 of 10 
 
APPENDIX 1    INVESTIGATOR SIGNATURE 
 
Protocol Title: 
Multicenter, Retrospective Data Collection of Routine Clinical Use 
with the Spectra Optia® Apheresis System for White Blood Cell 
Depletion (WBCD)  
 
 
Protocol Number: 
 
Version/Date: 
 
CTS-5043 
 
Original version 1.0/18 Feb 2014 
Amendment 1.0/28 Sep 2016 
I have read the clinical investigation plan, including all appendices, and I agree that it contains 
all necessary details for my staff and me to conduct this study as described. I will conduct this 
study in compliance with the clinical investigation plan, Good Clinical Practices, and all 
applicable regulations. I will make a reasonable effort to complete the study within the time 
designated. 
I will provide all study personnel under my supervision with copies of the clinical investigation 
plan and grant access to all information provided by Terumo BCT. I will discuss this material 
with all study personnel under my supervision to ensure that they are fully informed about the 
study. 
 
 
 
 
Principal Investigator Name (Printed) 
 
Signature 
 
 
 
 
 
 
Date