A Phase II Study Evaluating the Safety and Efficacy of MGTA-145 in Combination With Plerixafor for the Mobilization and Transplantation of HLA-Matched Donor Hematopoietic Stem Cells in Recipients With Hematological Malignancies
Phase: PHASE2
Conditions: Related Donors Donating PBSC to a Family Member, Healthy Donors, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome
Sponsor: Ensoma

 
 
 
Protocol 145-ADS-202 
 
A Phase II Study Evaluating the Safety and Efficacy of 
MGTA-145 in Combination with Plerixafor for the Mobilization 
and Transplantation of HLA-Matched Donor Hematopoietic 
Stem Cells in Recipients with Hematological Malignancies  
 
Resource for Clinical Investigation in Blood and Marrow Transplantation and 
Magenta Therapeutics, Inc. 
 
IND Number: 139251 
 
Version 2.0 
23 Sep 2020 
 
Sponsor Representatives 
Steven Devine, MD1 
John C. Davis Jr., MD, MPH, MS2 
 
 
 
Sponsored by Magenta Therapeutics and the National Marrow Donor Program® 
(NMDP) 
 
 
 
 
 
 
 
 
 
 
1 
Center for International Blood and Marrow Transplant Research 
(CIBMTR)/NMDP 
2 
Magenta Therapeutics, Inc.  

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Contact Information 
Role 
Contact  
Protocol Chairperson 
and Co- Sponsor 
Steven Devine, MD 
NMDP/CIBMTR 
sdevine2@nmdp.org  
763-406-8239 
500 N 5th St. 
Minneapolis, MN 55401 
Co- Sponsor 
John C. Davis Jr., MD, MPH, MS  
Magenta Therapeutics 
jdavis@magentatx.com 
415-297-7006  
100 Technology Square 
Cambridge, MA 02139 
CIBMTR Medical 
Monitor 
Linda J. Burns, MD 
CIBMTR/Medical College of Wisconsin 
lburns@mcw.edu 
414-805-0700 
Vendors/Labs 
A list of vendors/laboratories involved in the trial is maintained by 
the sponsor. A complete listing of applicable vendors will be 
provided to investigational sites.  
 
 
 

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SPONSOR SIGNATURE PAGE 
 
I, the undersigned, have read this protocol and agree that it contains all necessary information 
required to conduct the study. 
 
 
 
Magenta Therapeutics 
 
 
 
______________________________________ 
John C. Davis Jr., MD, MPH, MS  
Chief Medical Officer 
 
 
 
___________________ 
Date 
 
 
 
 
National Marrow Donor Program 
 
 
 
______________________________________ 
Steven Devine, MD 
Chief Medical Officer 
Associate Scientific Director – CIBMTR  
 
 
 
___________________ 
Date 
 
 
 
 
 
 
 
 
 
 

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INVESTIGATOR’S AGREEMENT 
 
The signature below constitutes the approval of this protocol and provides the necessary 
assurances that this trial will be conducted according to all stipulations of the protocol, including 
all statements regarding confidentiality, and according to local legal and regulatory requirements 
and applicable U.S. federal regulations and ICH guidelines. 
 
 
 
 
 
______________________________________ 
Printed Name of Investigator 
 
 
 
______________________________________ 
Signature of Investigator 
 
 
 
___________________ 
Date 

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TABLE OF CONTENTS 
SPONSOR SIGNATURE PAGE ........................................................................................................................... III 
1 
PROTOCOL SUMMARY ..............................................................................................................................10 
1.1 
SYNOPSIS .............................................................................................................................................................. 10 
1.2 
SCHEDULE OF ACTIVITIES (SOA) .......................................................................................................................... 16 
1.2.1 
Assessments of Donors ............................................................................................................................ 16 
1.2.2 
Assessments for Recipients ..................................................................................................................... 17 
2 
INTRODUCTION ...........................................................................................................................................18 
2.1 
STUDY RATIONALE................................................................................................................................................. 18 
2.2 
BACKGROUND ........................................................................................................................................................ 18 
2.2.1 
Defining the optimal allograft source: bone marrow versus peripheral blood ................................... 18 
2.2.2 
Experience of Donors Mobilized with G-CSF ........................................................................................ 19 
2.2.3 
Mechanism of G-CSF Induced Stem Cell Mobilization: Critical Role of CXCR4 and SDF-1 
Interaction ................................................................................................................................................... 19 
2.2.4 
Plerixafor is a Potent and Selective Antagonist of the CXCR4/SDF1 Interaction ............................ 20 
2.2.5 
Plerixafor Chemistry .................................................................................................................................. 20 
2.2.6 
MGTA-145 Background and Chemistry .................................................................................................. 20 
2.2.7 
Pharmacokinetics of Plerixafor ................................................................................................................ 21 
2.2.8 
Pharmacokinetics of MGTA-145 .............................................................................................................. 21 
2.2.9 
Pharmacodynamics of MGTA-145 .......................................................................................................... 22 
2.2.10 
Clinical studies evaluating safety of plerixafor ....................................................................................... 23 
2.2.11 
Clinical studies evaluating safety of MGTA-145 .................................................................................... 25 
2.2.12 
Rationale for studying MGTA-145 + plerixafor mobilized allografts following myeloablative 
conditioning ................................................................................................................................................. 25 
2.2.13 
Rationale for post-transplant growth factor administration .................................................................. 26 
2.3 
RISK/BENEFIT ASSESSMENT.................................................................................................................................. 26 
2.3.1 
Known Potential Risks .............................................................................................................................. 26 
2.3.2 
Known Potential Benefits .......................................................................................................................... 26 
2.3.3 
Assessment of Potential Risk and Benefits ........................................................................................... 26 
3 
OBJECTIVES ...............................................................................................................................................26 
3.1 
PRIMARY OBJECTIVE ............................................................................................................................................. 26 
3.2 
SECONDARY OBJECTIVES ...................................................................................................................................... 27 
3.2.1 
Donor Related ............................................................................................................................................ 27 
3.2.2 
Recipient Related ...................................................................................................................................... 27 
3.3 
EXPLORATORY OBJECTIVES .................................................................................................................................. 27 
3.3.1 
Graft Related .............................................................................................................................................. 27 
3.3.2 
Recipient Related ...................................................................................................................................... 27 
3.3.3 
Donor Related ............................................................................................................................................ 27 
4 
STUDY DESIGN ...........................................................................................................................................28 
4.1 
OVERALL DESIGN .................................................................................................................................................. 28 
4.2 
JUSTIFICATION FOR DOSE ...................................................................................................................................... 28 
4.3 
END OF STUDY DEFINITION.................................................................................................................................... 28 
5 
STUDY POPULATION .................................................................................................................................28 
5.1 
ELIGIBILITY CRITERIA ............................................................................................................................................. 28 
5.1.1 
Donor Inclusion Criteria ............................................................................................................................ 28 
5.1.2 
Donor Exclusion Criteria ........................................................................................................................... 29 
5.1.3 
Recipient Inclusion Criteria ....................................................................................................................... 29 
5.1.4 
Recipient Exclusion Criteria ..................................................................................................................... 29 
5.2 
SCREEN FAILURES ................................................................................................................................................. 30 
5.3 
STRATEGIES FOR RECRUITMENT AND RETENTION ................................................................................................. 30 
5.3.1 
Related Donors .......................................................................................................................................... 30 

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5.3.2 
Unrelated Donors ....................................................................................................................................... 30 
6 
SUBJECT ACCOUNTABILITY .....................................................................................................................30 
6.1 
POINT OF ENROLLMENT ......................................................................................................................................... 30 
6.2 
WITHDRAWAL ......................................................................................................................................................... 30 
6.3 
SUBJECT STATUS AND CLASSIFICATION ................................................................................................................ 31 
7 
STUDY INTERVENTION ..............................................................................................................................31 
7.1 
STUDY INTERVENTION(S) ADMINISTRATION ........................................................................................................... 31 
7.1.1 
Donor Treatment Plan ............................................................................................................................... 31 
7.1.2 
Recipient Treatment Plan ......................................................................................................................... 34 
7.1.3 
Supportive Care ......................................................................................................................................... 35 
7.1.4 
Dosing Administration ............................................................................................................................... 36 
7.2 
STUDY DRUGS ....................................................................................................................................................... 36 
7.2.1 
Description .................................................................................................................................................. 36 
7.2.2 
Packaging and Labeling ........................................................................................................................... 36 
7.2.3 
Product Storage and Stability................................................................................................................... 36 
7.2.4 
Preparation and Administration ............................................................................................................... 37 
7.3 
CONCOMITANT THERAPY ........................................................................................................................................ 37 
8 
STUDY INTERVENTION DISCONTINUATION AND SUBJECT DISCONTINUATION/WITHDRAWAL .....38 
8.1 
DISCONTINUATION OF STUDY INTERVENTION ........................................................................................................ 38 
8.2 
STUDY-WIDE SAFETY MONITORING RULES ........................................................................................................... 38 
8.3 
SUBJECT DISCONTINUATION/WITHDRAWAL FROM THE STUDY .............................................................................. 39 
8.4 
LOST TO FOLLOW-UP ............................................................................................................................................ 40 
9 
STUDY ASSESSMENTS AND PROCEDURES ...........................................................................................40 
9.1 
DONOR SCREENING ............................................................................................................................................... 40 
9.1.1 
Unrelated Donors ....................................................................................................................................... 41 
9.2 
DONOR FOLLOW-UP .............................................................................................................................................. 41 
9.3 
RECIPIENT SCREENING .......................................................................................................................................... 41 
9.4 
RECIPIENT FOLLOW-UP ......................................................................................................................................... 42 
9.5 
CRITERIA FOR ENGRAFTMENT FAILURE AND TRANSPLANTATION OF SECOND STEM CELL ALLOGRAFT .................... 43 
9.6 
DEFINITIONS FOR SAFETY, EFFICACY AND PK ASSESSMENTS .............................................................................. 44 
9.6.1 
Primary Endpoint: Clinically adequate allograft within one apheresis collection .............................. 44 
9.6.2 
Secondary Endpoint: Clinically desirable allograft in one apheresis collection ................................ 44 
9.6.3 
Secondary Endpoint: Clinically adequate allograft overall ................................................................... 44 
9.6.4 
Secondary Endpoint: Assessment of AE in Donors .............................................................................. 44 
9.6.5 
Secondary Endpoint: Assessment of AEs related to Allograft............................................................. 44 
9.6.6 
Secondary Endpoint: Neutrophil Recovery ............................................................................................ 44 
9.6.7 
Secondary Endpoint: Platelet Recovery ................................................................................................. 44 
9.6.8 
Secondary Endpoint: Graft Failure .......................................................................................................... 44 
9.6.9 
Secondary Endpoint: Acute GVHD ......................................................................................................... 45 
9.6.10 
Secondary Endpoint: Chronic GVHD ...................................................................................................... 45 
9.6.11 
Secondary Endpoint: Treatment-Related Mortality (TRM) ................................................................... 45 
9.6.12 
Secondary Endpoint: Overall Survival .................................................................................................... 45 
9.6.13 
Secondary Endpoint: Determination of Relapse or Disease Progression ......................................... 45 
9.6.14 
Secondary Endpoint: Progression-Free Survival .................................................................................. 45 
9.6.15 
Exploratory Endpoint: Graft composition ................................................................................................ 45 
9.6.16 
Exploratory Endpoint: Colony Formation Assay .................................................................................... 45 
9.6.17 
Exploratory Endpoint: Chimerism ............................................................................................................ 45 
9.6.18 
Exploratory Endpoint: Immune Reconstitution ...................................................................................... 46 
9.6.19 
Exploratory Endpoint: CMV Reactivation ............................................................................................... 46 
9.6.20 
Exploratory Endpoint: MGTA-145 PK ..................................................................................................... 46 
9.6.21 
Exploratory Endpoint: Immunogenicity ................................................................................................... 46 
9.6.22 
Exploratory Endpoint: Donor Experience ............................................................................................... 46 
 
 

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9.7 
DONOR SURVEY ASSESSMENTS ............................................................................................................................ 46 
9.7.1 
Brief Pain Inventory ................................................................................................................................... 47 
9.7.2 
PROMIS domains ...................................................................................................................................... 47 
9.7.3 
EQ-5D .......................................................................................................................................................... 48 
9.7.4 
Work Productivity and Activity Impairment (WPAI) ............................................................................... 48 
9.7.5 
Healthcare Resource Use (HRU) ............................................................................................................ 48 
9.7.6 
Donor Experience ...................................................................................................................................... 48 
9.8 
ADVERSE EVENTS AND SERIOUS ADVERSE EVENTS ............................................................................................. 48 
9.8.1 
Definition of Adverse Events (AE) ........................................................................................................... 48 
9.8.2 
Definition of Serious Adverse Events (SAE) .......................................................................................... 49 
9.8.3 
Classification of an Adverse Event .......................................................................................................... 49 
9.8.4 
Time Period and Frequency for Adverse Event Assessment and Follow-Up ................................... 50 
9.8.5 
Adverse Event Reporting .......................................................................................................................... 51 
9.8.6 
Adverse Events of Special Interest ......................................................................................................... 52 
9.8.7 
Reporting of Pregnancy ............................................................................................................................ 52 
10 
LABORATORY ASSESSEMENTS ..........................................................................................................53 
11 
STATISTICAL CONSIDERATIONS..........................................................................................................54 
11.1 
STATISTICAL HYPOTHESES ............................................................................................................................... 54 
11.2 
SAMPLE SIZE DETERMINATION .......................................................................................................................... 54 
11.2.1 
Planned Interim Analyses ......................................................................................................................... 55 
11.3 
POPULATIONS FOR ANALYSES ........................................................................................................................... 55 
11.4 
STATISTICAL ANALYSES .................................................................................................................................... 55 
11.4.1 
General Approach ...................................................................................................................................... 55 
11.4.2 
Baseline Descriptive Statistics ................................................................................................................. 55 
11.4.3 
Analysis of the Primary Efficacy Endpoint(s) ......................................................................................... 56 
11.4.4 
Analysis of the Secondary Endpoint(s) ................................................................................................... 56 
11.4.5 
Exploratory Analyses................................................................................................................................. 57 
12 
SUPPORTING DOCUMENTATION AND OPERATIONAL CONSIDERATIONS .....................................58 
12.1 
REGULATORY, ETHICAL, AND STUDY OVERSIGHT CONSIDERATIONS ................................................................ 58 
12.1.1 
Institutional Review Board Approval ....................................................................................................... 58 
12.1.2 
Informed Consent Process ....................................................................................................................... 59 
12.1.3 
Study Discontinuation and Closure ......................................................................................................... 60 
12.1.4 
Investigator Responsibilities ..................................................................................................................... 60 
12.1.5 
Delegation of Responsibility ..................................................................................................................... 61 
12.1.6 
Confidentiality and Privacy ....................................................................................................................... 61 
12.1.7 
Future use of Stored Specimens and Data ............................................................................................ 61 
12.1.8 
Safety Oversight ........................................................................................................................................ 61 
12.1.9 
Clinical Monitoring ..................................................................................................................................... 61 
12.1.10 
Data Handling and Record Keeping ................................................................................................... 62 
12.1.11 
Protocol Deviations ............................................................................................................................... 62 
12.1.12 
Publication and Data Sharing Policy................................................................................................... 63 
12.1.13 
Conflict of Interest Policy ...................................................................................................................... 63 
12.2 
ADDITIONAL CONSIDERATIONS .......................................................................................................................... 63 
12.2.1 
Donor Product Qualification ..................................................................................................................... 63 
12.3 
PUBLICATION POLICY ........................................................................................................................................ 63 
12.4 
ABBREVIATIONS ................................................................................................................................................. 64 
13 
REFERENCES ..........................................................................................................................................66 
14 
APPENDICES ...........................................................................................................................................69 
A. 
KARNOFSKY PERFORMANCE SCALE .......................................................................................................................... 69 
B. 
HEMATOPOIETIC CELL TRANSPLANT COMORBIDITY INDEX (HCT-CI) SCORING ........................................................ 70 
C. 
ACUTE GVHD GRADING ............................................................................................................................................ 71 
D. 
CHRONIC GVHD GRADING ....................................................................................................................................... 73 
E. 
DETERMINATION OF RELAPSE OR DISEASE PROGRESSION ....................................................................................... 75 

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F. 
DONOR SURVEY ASSESSMENTS ................................................................................................................................ 79 
G. 
ADVERSE EVENT DEFINITIONS AND GRADING (CTCAE V5.0) .................................................................................. 92 
H. 
GUIDANCE ON REVIEWING AND REPORTING UNANTICIPATED PROBLEMS ................................................................. 93 
 
 

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STATEMENT OF COMPLIANCE 
 
The trial will be carried out in accordance with International Conference on Harmonisation 
Good Clinical Practice (ICH GCP) and the United States (US) Code of Federal Regulations 
(CFR) applicable to clinical studies (45 CFR Part 46, 21 CFR Part 50, 21 CFR Part 54, 21 
CFR Part 56, 21 CFR Part 312, and/or 21 CFR Part 812).   
The protocol, informed consent form(s), recruitment materials, and all subject materials will 
be submitted to the National Marrow Donor Program (NMDP) Institutional Review Board 
(IRB) for review and approval for sites relying on the NMDP IRB as their IRB of record. 
Documentation of local IRB submissions will be collected for those sites relying on local 
IRBs. IRB approval of both the protocol and the consent form must be obtained before any 
subject is enrolled. Any amendment to the protocol and consent will require review and 
approval by the IRB before the changes are implemented to the study with a determination 
for re-consent for previously enrolled subjects.  
  
 
 

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1 
PROTOCOL SUMMARY 
1.1 Synopsis 
Protocol Title 
(Full) 
A Phase II Study Evaluating the Safety and Efficacy of MGTA-145 in 
Combination with Plerixafor for the Mobilization and Transplantation of 
HLA-Matched Donor Hematopoietic Stem Cells in Recipients with 
Hematological Malignancies 
Protocol Title 
(Short) 
MGTA-145 in Combination with Plerixafor for the Mobilization and 
Transplantation of HLA-Matched Donor Hematopoietic Stem Cells in 
Recipients with Hematological Malignancies 
Study 
Objective(s) 
Primary Hypothesis: Human leukocyte antigen (HLA) matched 
donors receiving injections of plerixafor at 240 μg/kg combined with 
MGTA-145 0.015 mg/kg will safely mobilize sufficient CD34+ cells (at 
least 2.0 x 106 CD34+ cells/kg actual recipient weight) following one 
day of leukapheresis to support hematopoietic stem cell 
transplantation (HSCT). The hematopoietic cells mobilized by MGTA-
145 + plerixafor will be functional and will result in prompt and durable 
hematopoietic recovery following transplantation into HLA-matched 
siblings and matched unrelated recipients with hematological 
malignancies.  
Primary Objectives: To determine the proportion of donors whose 
cells can be successfully mobilized and collected with a sufficient 
CD34+ cell dose using same-day, single-dose MGTA-145 + plerixafor 
as the mobilizing agents. Donor mobilization following MGTA-145 + 
plerixafor will be considered successful if ≥ 2.0 x 106 CD34+ cells/kg 
actual recipient weight are collected in one leukapheresis collection. 
All donors receiving MGTA-145 + plerixafor will be included in the 
analysis of the primary objective.  
Secondary Objectives:  
Donor related  
1. To determine the proportion of donors whose cells can be 
successfully mobilized and collected with a target CD34+ cell dose 
of at least 4.0 x 106 CD34+ cells/kg actual recipient weight in one 
apheresis collection 
2. To ascertain the incidence and severity of acute adverse events 
(AEs) before and during apheresis experienced by donors 
receiving MGTA-145 + plerixafor  
3. To characterize the adverse effects experienced by donors 
receiving MGTA-145 + plerixafor  

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Recipient Related  
1. To determine the incidence of and kinetics of neutrophil and 
platelet recovery after transplantation of hematopoietic cells 
mobilized with MGTA-145 + plerixafor  
2. To determine the incidence of primary and secondary graft failure 
after transplantation of hematopoietic cells mobilized with MGTA-
145 + plerixafor  
3. To determine the incidence and severity of acute and chronic graft 
versus host disease (GVHD) after transplantation of hematopoietic 
cells mobilized with MGTA-145 + plerixafor  
4. To determine the incidence of treatment-related mortality and 
disease relapse/progression after transplantation of hematopoietic 
cells mobilized with MGTA-145 + plerixafor  
5. To determine the probability of progression-free and overall 
survival after transplantation of hematopoietic cells mobilized with 
MGTA-145 + plerixafor  
6. To characterize the adverse effects experienced by recipients 
receiving grafts mobilized by MGTA-145 + plerixafor  
Study Population  The donor population for the study includes those subjects undergoing 
peripheral blood mobilization and collection of grafts for allogeneic 
HSCT.  
The recipient population for the study includes those subjects with 
acute myelogenous leukemia (AML), acute lymphoblastic leukemia 
(ALL) or myelodysplastic syndrome (MDS) receiving a hematopoietic 
stem cell transplant using the product obtained from a donor who 
underwent mobilization using MGTA-145 in combination with 
plerixafor.  
Study 
Design/Phase 
This is a Phase II, open-label, multicenter, prospective study of 
MGTA-145 + plerixafor mobilized HLA-matched sibling and matched 
unrelated donor allografts for myeloablative HSCT in recipients with 
hematological malignancies. Donors will undergo 1 or 2 days of 
mobilization and apheresis. The target collection is at least 4.0 x 106 
CD34+ cells/kg actual recipient weight. Donors who achieve this target 
in one day will not undergo a second day of mobilization and 
apheresis. Donors who collect < 4.0 x 106 CD34+ cells/kg actual 
recipient weight will undergo a second day of mobilization and 
apheresis. AEs will be reported using event terms and grading for 
severity per the Common Terminology Criteria for Adverse Events 
(CTCAE) version 5. Donors will be contacted at Week 1, Month 1, and 
Month 6 to assess the donor’s medical condition. Donors will have 
blood work at Month 1. The design utilizes established Center for 
International Blood and Marrow Transplant Research (CIBMTR) 
transplant recipient follow up procedures to capture transplant 
outcome data. NMDP will implement the study within its clinical 
investigation group and with Resource for Clinical Investigation in 

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Blood and Marrow Transplantation (RCI BMT) Data Safety Monitoring 
Board (DSMB) oversight.  
Treatment 
Description 
Donor: Eligible donors will receive subcutaneous plerixafor at 240 
μg/kg actual donor weight followed by MGTA-145 infusion 
approximately 2 hours later and commence apheresis as soon as 
feasible thereafter. Some donors will require a second dose of MGTA-
145 + plerixafor and apheresis the day after the first collection to reach 
the target CD34+ cell collection goal. The target CD34+ cell dose is ≥ 
4.0 x 106/kg actual recipient weight with a minimum of ≥ 2.0 x 106/kg 
actual recipient weight.  
Recipient: Eligible recipients will undergo conditioning using a 
myeloablative regimen after an adequate allograft has been collected 
from the donor. Allograft will be cryopreserved and stored until 
transplant Day 0. GVHD prophylaxis may include post-transplant 
cyclophosphamide or a calcineurin phosphatase inhibitor in 
combination with methotrexate, mycophenolate mofetil, or sirolimus in 
accordance with local institutional guidelines. Recipients will receive 
G-CSF 5 μg/kg/day starting day +7 until absolute neutrophil count 
(ANC) > 1.5 x 109/L for two consecutive days.  
Planned Number 
of Subjects 
Approximately 28 donors. With a 5% drop out prior to transplant, it is 
anticipated that this will lead to 28 donors evaluable for mobilization 
endpoints and 26-27 recipients available for transplant follow up. The 
study employs a two-stage Simon minimax design in which 13 donors 
are enrolled in stage 1 and 15 donors are enrolled in stage 2.  
Planned Number 
of Sites  
Up to 10 
Method of 
Assigning 
Patients to 
Treatment 
Single arm, non-randomized 
 
Follow-up 
Schedule 
Donors will be followed from Day 1 (Baseline, MGTA-145 infusion) 
through 6-months post-mobilization. Donors will also complete survey 
assessments from prior to mobilization through 1-month post-
mobilization. 
Recipients will be followed from Day 0 (Day of Transplant) through 
1-year post-transplant. Recipients will be asked to co-enroll on the 
CIBMTR Outcomes Database Protocol. This study will require CRF-
track data collection for those subjects that consent to CIBMTR 
Outcomes research. 
Study Duration 
2.5 years (1.5 years enrollment + up to 1-year follow-up) 

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Participation 
Duration 
6 months for donors 
1 year for recipients 
Key Inclusion 
Criteria 
Donor Inclusion Criteria:  
1. Donor medical suitability and eligibility will be determined following 
Institution or NMDP/Be The Match standards 
2. Age 18-65 years old at the time of signing informed consent  
3. 8/8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer 
unrelated donor 
4. Fulfill Institution or NMDP/Be The Match criteria to serve as a 
mobilized blood cell donor  
5. Serum creatinine < 1.5 x institution upper limit of normal (ULN) or 
estimated creatinine clearance (CRCL) > 50 mL/min using the 
Modification of Diet in Renal Disease Study (MDRD) equation or 
similar method 
Recipient Inclusion Criteria:  
1. At least 18 years old at the time of signing informed consent  
2. Has an available 8/8 (HLA- A, B, C, and DRB1) HLA-matched 
sibling or volunteer unrelated donor willing to donate peripheral 
blood stem cells (PBSC) for transplant  
3. Fulfill additional individual Transplant Center Criteria for transplant 
beyond NMDP/Be The Match criteria 
4. One of the following diagnoses:  
• 
Acute myelogenous leukemia (AML) in 1st remission or 
beyond with ≤ 5% marrow blasts and no circulating blasts.  
Documentation of bone marrow assessment will be accepted 
within 45 days prior to the date of consent.  
• 
Acute lymphoblastic leukemia (ALL) in 1st remission or 
beyond with ≤ 5% marrow blasts and no circulating blasts. 
Documentation of bone marrow assessment will be accepted 
within 45 days prior to the date of consent.  
• 
Patients with myelodysplasia (MDS) with no circulating blasts 
and with less than 10% blasts in the bone marrow (higher 
blast percentage allowed in MDS due to lack of differences in 
outcomes with < 5% or 5-10% blasts in MDS). Documentation 
of bone marrow assessment will be accepted within 45 days 
prior to the date of consent.  
5. Cardiac function: Left ventricular ejection fraction at least 45% 
based on most recent echocardiogram or MUGA results obtained 
via standard of care 
6. Estimated creatinine clearance acceptable per local institutional 
guidelines  

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7. Pulmonary function: diffusing capacity of the lungs for carbon 
monoxide (DLCO) corrected for hemoglobin at least 50% and 
forced expiratory volume in first second (FEV1) predicted at least 
50% based on most recent DLCO results obtained via standard of 
care 
8. Liver function acceptable per local institutional guidelines  
9. Karnofsky performance status (KPS) of 70% or greater 
10. Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) 
score of 4 or less 
Key Exclusion 
Criteria 
Donor Exclusion Criteria:  
1. Donor unwilling or unable to give informed consent, or unable to 
comply with the protocol including required follow-up and testing  
2. Donor already enrolled on another investigational agent study  
3. Pregnant or breastfeeding females, sexually active female and 
male donors not willing or able to use adequate contraception, or 
males who do not agree to refrain from donating sperm, from the 
time of consent through 3 months after treatment with 
MGTA-145 + plerixafor 
Recipient Exclusion Criteria:  
1. Subject unwilling or unable to give informed consent, or unable to 
comply with the protocol including required follow-up and testing  
2. Subject whose donor does not meet the eligibility criteria and is a 
screen fail 
3. Subjects with a prior allogeneic transplant  
4. Subjects with active, uncontrolled infection at the time of the 
transplant preparative regimen  
5. Pregnant or breastfeeding females, sexually active female or male 
subjects not willing or able to use adequate contraception, or 
males who do not agree to refrain from donating sperm, from the 
time of consent through 3 months after PBSC infusion 
6. Subjects with clinical evidence of active Central Nervous System 
(CNS) tumor involvement as evidenced by documented disease 
on examination of spinal fluid or MRI within 45 days of start of 
conditioning  
7. A condition, which, in the opinion of the clinical investigator, would 
interfere with the evaluation of primary and secondary endpoints  
8. Planned treatment with a new investigational agent from the time 
of transplant through 30 days post-transplant 

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Statistical Methods  
Primary 
Statistical 
Analysis Plan 
The primary endpoint of this study is the collection of a clinically 
adequate allograft in one apheresis session.  A donor is considered 
successful for this endpoint if ≥ 2.0 x 106 CD34+ cells/kg actual 
recipient weight are collected in one leukapheresis collection using 
MGTA-145 + plerixafor. 
The proportion of donors who mobilize ≥ 2.0 x 106 CD34+ cells/kg 
actual recipient weight in one leukapheresis collection will be 
estimated along with a 90% confidence interval for consistency with 
the one-sided type I error rate of 5%. A 95% confidence interval will 
also be provided. The primary analysis will use the Donor Treated 
Population, where donors who do not undergo apheresis will be 
considered a failure for this endpoint.   
Sample Size 
Justification 
The sample size for this study is 28 donors receiving at least one dose 
of the combination regimen of MGTA-145 + plerixafor. It is expected 
that 90% of donors treated with MGTA-145 + plerixafor will mobilize 
≥ 2.0 x 106 CD34+ cells/kg within one leukapheresis collection.  
MGTA-145 + plerixafor is considered an unacceptable mobilizing 
agent if fewer than 70% of donors achieve this cell-dose threshold 
within one leukapheresis collection.  
Using a two-stage Simon minimax design, assuming the true 
successful collection probability of 90%, 28 donors provide 85% power 
to reject the probability of successful collection of 70% or lower using 
a one-sided Type I error α=0.05. This is equivalent to testing the null 
hypothesis H0: p ≤ 0.70 against the alternative hypothesis H1: p > 
0.70 with 85% power to contrast a successful collection probability of 
90% vs. 70%. 

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1.2 Schedule of Activities (SOA)  
1.2.1 
Assessments of Donors  
Study Visit 
Screening1 
Day 1 
(Baseline)2 
Day 1 
(Plerixafor 
Dosing)2 
Day 1 (MGTA-
145 Dosing)2 
Post-LA, Day 
1 
Day 3-43 
Week 14 
Month 14 
Month 64 
End of Study 
Study Day 
-30 - 1 
1 
1 
1 
1 
3-4 
7 (±2 days) 
28 (±7 days) 
180 (±14 days) 
Informed Consent 
X 
 
 
 
 
 
 
 
 
Medical History and Medications 
X 
X 
 
 
 
 
 
 
 
Physical Exam, Height and Weight 
X5 
X6 
 
 
 
 
 
 
 
Vital Signs7 
X 
 
X8 
X9 
X 
 
 
 
 
KPS 
X 
 
 
 
 
 
 
 
 
ECG 
X 
 
 
 
 
 
 
 
 
CBC with differential and platelet count 
X 
X6 
 
 
X 
 
 
X 
 
Serum Creatinine & PT/PTT 
X 
 
 
 
 
 
 
 
 
Serum Pregnancy Test10 
X 
X10 
 
 
 
 
 
 
 
Urinalysis 
X 
 
 
 
 
 
 
 
 
Infectious disease markers11 
X 
 
 
 
 
 
 
 
 
ABO/Rh Typing 
X 
 
 
 
 
 
 
 
 
Chimerism  
X 
 
 
 
 
 
 
 
 
Donor Survey Assessments12 
 
X12 
 
 
X 
X 
 
X 
 
ADA Sample 
 
X6 
 
 
 
 
 
X 
 
Flow cytometric analysis for CD34+ cells 
 
X6 
 
X13 
X14 
 
 
 
 
Plerixafor Dosing 
 
 
X15 
 
 
 
 
 
 
PK  
 
 
 
X16 
 
 
 
 
 
MGTA-145 Infusion 
 
 
 
X17 
 
 
 
 
 
Leukapheresis (LA) 
 
 
 
X18 
 
 
 
 
 
Colony Forming Unit 
 
 
 
 
X14 
 
 
 
 
Graft Immunophenotyping 
 
 
 
 
X14 
 
 
 
 
Medical assessment (Virtual visit) 
 
 
 
 
 
 
X 
X 
X 
AEs/SAEs19 
X 
 
1 Screening occurs up to 30 days prior to the start of stem cell mobilization; screening and determination of eligibility of unrelated donors is managed by Be The Match Donor Services. 
2 If donor undergoes a second day of mobilization and apheresis, all Day 1 assessments must be repeated and donors will follow the same schedule (i.e., Day 7 will remain Day 7)  
3 Donor survey visit only. CIBMTR SRG will contact donors. 
4 Study site staff will contact donors at Week 1, Month 1, and Month 6 for virtual visits to assess donor medical condition. Donors will have blood work at Month 1. 
5 Height only required at Screening 
6 To be obtained prior to dosing 
7 Vital signs include blood pressure, pulse, respiration rate and temperature 
8 Vital signs and AEs monitored prior to plerixafor dosing; and 30 minutes (±10 minutes) and 60 minutes (±10 minutes)) following injection; and per local institutional guidelines 
9 Vital signs and AEs monitored prior to MGTA-145 dosing; during infusion; and 30 minutes (±10 minutes), 60 minutes (±10 minutes, 120 minutes (±20 minutes), and 240 minutes (±20 minutes) following 
start of infusion 
10 For females of childbearing potential. Day 1 test can be performed up to 48 hours prior to dosing 
11 Infectious disease markers collected as required by FDA 
12 CIBMTR SRG will contact donors to complete survey assessments. Baseline donor survey assessment to be administered up to 14 days prior to Day 1. See Table 9.7 
13 To be collected after MGTA-145 infusion but prior to apheresis 
14 Sample will come from the apheresis product 
15 Plerixafor will be administered subcutaneously at 240 µg/kg/actual donor weight approximately 2 hours prior to planned leukapheresis 
16 PK samples to be collected prior to MGTA-145 infusion (within 60 minutes of the start of the infusion) and 15-30 minutes after start of infusion 
17 MGTA-145 administered at dose of 0.015 mg/kg via IV infusion over 3 minutes approximately 2 hours after plerixafor; the infusion time may range from 1 to 10 minutes if clinically indicated 
18 As soon as feasible after MGTA-145 infusion (approximately 30 minutes post infusion), begin apheresis to process at least 4 times blood volume, but no more than the institutional limit. 
19 SAEs to be reported from informed consent through 6 months post mobilization. AEs to be collected from informed consent through 72 hours post mobilization. See Table 9.8 

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1.2.2 
Assessments for Recipients 
Study Visit 
Screening1 
Day 0 
Days 
1-212 
Day 28 
± 3 days 
Day 56 
± 7 days 
Day 100 
± 7 days 
Day 180 
± 14 days 
Day 365 
± 14 days 
Informed Consent 
X 
 
 
 
 
 
 
 
Medical History and Medications3 
X 
 
 
 
 
 
 
 
Physical Exam and Vital Signs4 
X 
X5 
X 
X 
X 
X 
X 
X 
Height and Weight 
X 
 
 
 
 
 
 
 
KPS  
X 
 
 
 
 
 
X 
X 
CBC with differential and platelet count 
X 
X5 
X6 
X7 
X8 
X8 
X 
X 
Serum creatinine/AST/ALT/LDH/Total 
bilirubin  
X 
X5 
X9 
X 
X 
X 
X 
X 
Serum Pregnancy Test  
X10 
 
 
 
 
 
 
 
Urinalysis  
X 
 
 
 
 
 
 
 
Infectious disease markers11 
X12 
 
 
 
 
 
 
 
ABO/Rh Typing 
X12 
 
 
 
 
 
 
 
Chimerism  
X12 
 
 
X 
 
X 
X 
X 
Disease specific staging and assessment 
X 
 
 
 
 
 
 
X 
Assessment of HCT-CI score 
X 
 
 
 
 
 
 
 
Bone marrow assessment 
X13 
 
 
 
 
 
 
X14 
Allograft Infusion 
 
X 
 
 
 
 
 
 
GVHD Assessment 15 
 
 
X 
X 
X 
X 
X 
X 
Immune Reconstitution  
 
 
 
X 
 
X 
X 
X 
AEs/SAEs16 
 
X 
 
1 Screening occurs up to 45 days prior to the start of the conditioning regimen 
2 Visits to occur Day +1 through neutrophil recovery or Day +21 (whichever is first) 
3 Includes most recent echocardiogram or MUGA and DLCO results obtained via standard of care 
4 Vital signs include blood pressure, pulse, respiration rate, and temperature 
5 To be obtained prior to receiving allograft 
6 CBC with differential and platelet counts to be performed daily until neutrophil recovery occurs; in the event that neutrophil recovery occurs but not platelet recovery, a CBC should continue 
to be obtained daily until platelet count recovery or Day +21 (whichever is first) 
7 If platelet and/or neutrophil recovery has not occurred at this time, CBC to be performed 3 times per week until recovery 
8 If platelet and/or neutrophil recovery has not occurred at this time, CBC to be performed 2 times per week until recovery 
9 Blood chemistries performed 2 times per week 
10 For females of childbearing potential 
11 Infectious disease markers collected as required by FDA 
12 May be performed earlier than 45 days prior to the start of the conditioning regimen 
13 Documentation of bone marrow assessment will be accepted within 45 days prior to the date of consent 
14 Bone marrow aspirate and biopsy required if leukocyte count is outside of the normal laboratory range at the transplant center and/or platelet count is < 100 x 109/L 
15 Recipients will be assessed for or the development of acute GVHD weekly at Days 1-100 and for chronic GVHD at Days 100, 180, and 365 and more frequently as clinically indicated 
16 SAEs to be reported from Day 0 through 1-year post-transplant; Related AEs to be collected from Day 0 through 1-year post-transplant  

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2 
INTRODUCTION  
2.1  Study Rationale 
Subjects with protocol-specified advanced hematological malignancies who are appropriate candidates 
for hematopoietic stem cell transplantation (HSCT) and who have an HLA-identical (8/8 HLA antigen 
matched) sibling donor or 8/8 matched unrelated donor will be eligible for this study. If the subject and 
donor are eligible and provide informed consent, the donors will be mobilized with plerixafor 
subcutaneously at a dose of 240 μg/kg actual donor weight, followed approximately 2 hours later by an 
intravenous infusion of 0.015 mg/kg MGTA-145. The donors will undergo leukapheresis commencing as 
soon as feasible after MGTA-145 dosing. At the end of the leukapheresis, the collected graft will be 
analyzed by standard flow cytometry for CD34+ cell content. If the graft contains ≥ 4.0 x 106 CD34+ 
cells/kg actual recipient weight after the first leukapheresis collection, the mobilization will be considered 
clinically successful and complete. If the graft contains < 4.0 x 106 CD34+ cells/kg actual recipient weight 
after the first leukapheresis collection, the donor will undergo a second day of plerixafor and MGTA-145 
infusion mobilization and apheresis.  
If the sum of the grafts collected after two days is ≥ 2.0 x 106/kg of actual recipient weight, the 
mobilization will have achieved the minimum dose and will be considered completed and clinically 
successful. Allografts will be cryopreserved prior to infusion. If the donor fails to mobilize a graft 
containing at least 2.0 x 106/kg CD34+ cells/kg of actual recipient weight after two leukapheresis 
procedures, mobilization will be considered unsuccessful. The center will be allowed to use that graft 
(regardless of the total CD34+ cell dose) at their discretion, and the recipient will be followed. 
Alternatively, the treating center may choose to remobilize the donor with granulocyte colony stimulating 
factor (G-CSF) or collect bone marrow for transplantation as a supplement or replacement for the MGTA-
145 + plerixafor mobilized graft.  
For recipients for whom > 2.0 x 106 CD34+ cells/kg were collected from the donor following MGTA-145 + 
plerixafor, the collected allograft will be used as the primary allograft for transplantation. Recipients will be 
followed until one-year post-transplant and donors will be followed until six-months post-mobilization for 
important secondary endpoints.  
2.2 Background 
2.2.1 
Defining the optimal allograft source: bone marrow versus peripheral blood  
G-CSF-based mobilization is a relatively safe and effective alternative to bone marrow harvesting for 
collection of hematopoietic stem cells (HSC) prior to allografting.1-3 Several randomized trials performed 
in recipients of HLA-matched sibling allografts have been reported comparing G-CSF mobilized 
peripheral blood (MPB) to bone marrow (BM).4-9 These trials demonstrate that G-CSF MPB is associated 
with more rapid hematopoietic recovery, similar rates of acute graft versus host disease (GVHD), and 
higher rates of chronic GVHD compared to BM.10,11 Early regimen-related mortality appears to be lower 
with MPB, particularly in recipients with advanced hematological malignancies. Overall survival appears 
to be improved in recipients with advanced stage disease transplanted using MPB versus BM. Thus, in 
virtually all US transplant centers, G-CSF MPB has largely supplanted harvested BM in recipients 
receiving HLA-identical sibling transplantation. Similarly, the majority of volunteer unrelated donor 
allografts collected for adults with hematological malignancies are from MPB in preference to BM. A 
randomized trial of MPB vs. BM showed similar 2-year survival between groups with a higher chronic 
GVHD incidence with MPB grafts (53% vs 41%) but lower graft failure with MPB (3% vs 9%).12 
Despite the increasing utilization of MPB, the dose of CD34+ cells required to optimize transplant 
outcomes is presently unknown. While it appears that grafts containing CD34+ cell doses > 2.0 x 106/kg 
recipient weight promote prompt and durable recovery, some studies suggest that CD34+ cell doses > 
8.0 x 106/kg may result in a higher risk of both acute and chronic GVHD.13,14 The mechanisms underlying 
this relationship are uncertain, and not all studies are in agreement.8,15,16 Nevertheless, the optimal 

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cellular composition of a G-CSF MPB allograft necessary to promote reliable recovery without increasing 
the risk of GVHD or other complications remains to be determined.  
2.2.2 
Experience of Donors Mobilized with G-CSF  
In addition to improving hematopoietic recovery, it was theorized that G-CSF based stem cell mobilization 
would reduce the morbidity associated with donor stem cell harvesting, primarily by replacing a painful 
surgical procedure with a simple outpatient apheresis. Early reports suggested G-CSF based mobilization 
was relatively safe and well tolerated by the majority of donors.1-3,17 More recent data suggest that while 
virtually all donors complete the stem cell mobilization and collection procedure, many G-CSF mobilized 
donors experience significant albeit transient morbidity following G-CSF stimulation.18-21 In a retrospective 
study performed using data from the International Bone Marrow Transplant Registry (IBMTR) and 
European Bone Marrow Transplant (EBMT) Group, 1321 donors undergoing Peripheral Blood (PB) 
mobilization and leukapheresis (LA) were analyzed.19 In order to achieve a target CD34+ cell dose set by 
the transplant programs, over 60% of donors required more than one LA procedure, and 15% greater 
than two. Surprisingly, approximately 20% of donors required placement of central venous catheter 
(CVC). The overall rate of serious complications associated with G-CSF MPB donation was 1.1%, higher 
in comparison to the 0.5% rate observed following bone marrow donation. Two recent prospective 
studies performed concurrently with the randomized trials found similar overall rates of complications 
comparing BM to G-CSF MPB donation.20,21 Pain, fatigue, and anxiety were the most frequently reported 
adverse events (AEs). Rowley et al. noted that pain experienced by G-CSF stimulated donors worsened 
with each day of treatment and peaked at the time of Leukapheresis (LA).20 In contrast to the crescendo 
nature of the pain experienced by the G-CSF mobilized donors, pain and discomfort experienced by BM 
donors was clearly related to anesthesia and multiple needle punctures on the day of donation. The 
symptom burden reported with both methods of stem cell procurement was similar, with pain a prominent 
symptom in both groups. Equivalent mean levels of maximal pain, average pain, and pain duration 
throughout the day were observed in both groups. These findings were essentially corroborated by a 
similar study performed as part of a French randomized trial.21 Pulsipher performed a similar analysis of 
volunteer unrelated donors receiving five to six days of G-CSF for stem cell mobilization and found that 
57% of donors experience moderate (48%) or severe (9%) bone pain and overall 38% experience grade 
2 or 3 toxicity.22 Unfortunately, there are few data available regarding the impact of inconvenience, loss of 
work days, and economic cost incurred during the 4-6 day process of G-CSF based mobilization.  
In summary, G-CSF MPB donation is relatively safe but is associated with significant morbidity, similar in 
overall intensity in comparison to BM donation but with a different distribution of peak intensity. The 
leukapheresis procedure itself has not been associated with significant morbidity in most studies unless 
a CVC is required. Morbidity increases with each day of G-CSF administration. While the available data 
suggest that G-CSF MPB donation is associated with lower risk of serious adverse events (SAEs) 
relative to BM, it remains associated with significant morbidity and inconvenience. Accordingly, a less 
toxic, more rapid, yet efficient method for the collection of MPB from related and volunteer donors is 
desirable and would represent a clear advance.  
2.2.3 
Mechanism of G-CSF Induced Stem Cell Mobilization: Critical Role of CXCR4 and SDF-1 
Interaction  
Many groups have had a longstanding interest in elucidating the mechanistic basis for G-CSF induced 
HSC mobilization. Most studies suggest that G-CSF induces HSC mobilization indirectly through its 
effects on proteases elaborated by marrow neutrophils, resulting in cleavage of adhesive interactions 
(e.g., VLA-4/VCAM-1) and remodeling of matrix proteins within the bone marrow microenvironment.23-26 
Recent data suggest the interaction between the CXC chemokine receptor CXCR4 and its ligand stromal 
derived factor-1 (SDF-1) plays a key role in stem cell mobilization.23-26 CXCR4 is a member of the large 
family of seven transmembrane domain receptors coupled to heterotrimeric G1 proteins. Binding with its 
only known ligand SDF-1 (also known as CXCL12) results in activation of multiple signal transduction 
pathways ultimately triggering chemotaxis. Targeted disruption of either molecule is lethal in mice, 

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resulting in failure of HSC migration from liver to bone marrow, defects in B- lymphopoiesis, and 
cerebellar dysgenesis.27-29 Interactions between SDF-1 and CXCR4 critically regulate the homing and 
migration of human SCID repopulating cells from BM, umbilical cord blood (UCB), and G-CSF MPB 
transplanted into NOD/SCID recipients.23,30 Efficient mobilization of murine stem and progenitor cells is 
observed following injection of adenovirus expressing SDF-1 or after injection of methionine-SDF-1, both 
resulting in a gradient of SDF-1 from BM to PB.31Treatment of mice and primates with sulfated 
polysaccharides (e.g. fucoidan) results in a rapid increase in circulating SDF-1 and subsequent stem cell 
mobilization.32 Lapidot and colleagues have suggested a model wherein G-CSF stimulation induces 
proteases, notably neutrophil elastase and perhaps other matrix metalloproteinases, that markedly 
reduce local bone marrow levels of SDF-1, resulting in the egress of hematopoietic stem and progenitor 
cells from the BM into the PB.26,33 Taken together, mounting evidence supports a primary role for the 
CXCR4/SDF-1 axis in both murine and human stem and progenitor cell mobilization and suggests this 
interaction provides a novel target to induce the migration of HSC and progenitors from BM into PB.  
2.2.4 
Plerixafor is a Potent and Selective Antagonist of the CXCR4/SDF1 Interaction  
The bicyclam derivative plerixafor was first described for its potent and selective inhibition of human 
immunodeficiency virus (HIV) type 1 and 2 replication through binding to the chemokine receptor CXCR4, 
used by T- tropic HIV for entry into the cell.34-39 Plerixafor also potently, specifically, and reversibly blocks 
the binding of CXCR4 with its only known natural ligand, SDF-1; it has no effect on other cell surface 
chemokine receptors.34,40-42 Initial clinical trials of plerixafor evaluated its safety and efficacy in the 
treatment of patients with HIV-1 infection.43 In these studies, transient increases in white blood cell counts 
were observed immediately following injection of plerixafor. Similar findings were also noted in healthy 
volunteers.44 These observations prompted studies to evaluate the effects of plerixafor on mobilization of 
hematopoietic stem and progenitor cells. 
2.2.5 
Plerixafor Chemistry  
Plerixafor is a bicyclam of the following chemical composition 1,1'-[1,4- phenylenebis(methylene)]-bis-
1,4,8,11-tetraazacyclotetradecane. Its structure is shown in Figure 1.  
2.2.6 
MGTA-145 Background and Chemistry 
Magenta Therapeutics is developing MGTA-145 as a potential alternative to G-CSF for the mobilization of 
CD34+ cells when used in combination with plerixafor. MGTA-145 (aka Gro-Beta Truncate or GroβT) is a 
CXCR2 agonist. Preclinical studies in mice, rats, and non-human primates demonstrated that MGTA-145, 
when administered simultaneously with plerixafor, leads to rapid and robust mobilization of CD34+ cells, 
CD34+CD90+CD45RA cells, and hematopoietic progenitor cells. When GroβT (SB-251353; also known 
as Garnocestim manufactured by Glaxo SmithKline Beecham) was clinically tested in healthy volunteers 
as a monotherapy, a statistically significant increase in mobilization of HSCs was observed.45 In addition, 
preclinical studies in mice, rats, and non-human primates demonstrated that MGTA-145 administered 
Figure 1. PLERIXAFOR chemical structure 

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with plerixafor leads to rapid and robust mobilization of CD34+, CD34+CD90+CD45RA- cells, and 
hematopoietic progenitor cells within minutes to hours. 
Company or Laboratory Codes 
MGTA-145, GROβT acetate salt, GROβT. Its structure is shown in Figure 2.  
 
 
 
2.2.7 
Pharmacokinetics of Plerixafor  
The pharmacokinetic behavior of plerixafor following intravenous administration is characterized by 
elimination from the plasma in a bi-exponential manner with a terminal elimination half-life of 
approximately 3 hours following a single dose. Plerixafor absorption following subcutaneous 
administration is rapid and essentially complete, with peak plasma levels occurring within 0.5–1 hour of 
dosing. Plerixafor is extensively protein bound to both human serum albumin and 1-acid glycoprotein; 
however, protein binding does not appear to have a major influence on toxicity. Saturation of protein 
binding sites may occur at plasma plerixafor concentrations in excess of those likely to be achieved in 
any ongoing or planned clinical studies. Findings from animal studies performed using 14C-labelled- 
plerixafor suggest that the parent molecule is the major circulating form of the drug in plasma, with a 
number of potential metabolites noted in urine.  
2.2.8 
Pharmacokinetics of MGTA-145 
The pharmacokinetics of MGTA-145 following administration in combination with plerixafor was evaluated 
in the Phase I 145-HV-101 study and are summarized in Figure 3. The Cmax and Area Under the Curve 
(AUC) of MGTA-145 increased along with dose linearly in the dosing range of 0.0075 mg/kg – 0.3 mg/kg. 
The mean Cmax of MGTA-145 ranged from 34.5 μg/L to 1469.3 μg/L, and the mean area under the 
concentration-time curve from time zero to time of last measurable concentration (AUClast ) of MGTA-145 
ranged from 17.6 h*μg/L to 2290.7 h*μg/L. Co-administration of plerixafor did not change the 
pharmacokinetics (PK) of MGTA-145. Accumulation was not observed following the second of two daily 
doses of MGTA-145. 
Figure 2:  
MGTA-145  
Amino Acid 
Sequence 

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2.2.9 
Pharmacodynamics of MGTA-145 
MGTA-145 is a protein that is chemotactic for neutrophils, which express its cognate chemokine receptor, 
CXCR2. It was expected that administration of MGTA-145 as monotherapy would lead to mobilization of 
neutrophils with a corresponding increase in white blood cell (WBC) counts. Plerixafor is also known to 
mobilize WBCs including neutrophils, lymphocytes, monocytes, basophils and eosinophils. Based on this, 
it was anticipated that MGTA-145 in combination with plerixafor would result in a cumulative increase in 
the same cell types. 
MGTA-145 monotherapy induced immediate and significant mobilization of neutrophils at the lowest dose 
tested, 0.0075 mg/kg (Figure 4A). Suppression of neutrophil mobilization was observed at MGTA-145 
doses greater than 0.03 mg/kg. The mechanism of neutrophil suppression is thought in part to be due to 
saturation of plasma MGTA-145 levels with increasing doses, resulting in loss of the chemotactic gradient 
necessary for neutrophil mobilization. MGTA-145 induced rapid downregulation of CXCR2 expression on 
neutrophils at all dose levels tested demonstrating direct target interaction (Figure 4B). Re-expression of 
the receptor was dose-dependent with the higher doses of MGTA-145 resulting in a prolonged period of 
downregulation. Re-expression of the receptor at 0.075 – 0.3 mg/kg (along with continued 
pharmacokinetic exposure) correlates with the rebound of neutrophil mobilization observed at the higher 
doses of MGTA-145.  
 
 
 
 
 
 
 
 
Figure 3. Pharmacokinetics of two daily doses of 
MGTA-145 administered in combination with 
plerixafor. All data expressed as mean ± standard 
deviation. 
0
2
4
6
1
1 0
1 0 0
1 ,0 0 0
2 4
2 6
2 8
3 0
H o u r s P o s t M G T A -1 4 5  A d m in is tr a tio n
M e a n  P la sm a  D r u g  C o n c e n tr a tio n
(n g /m L )
0 .0 7  m g /k g  M G T A -1 4 5  +  p le rix a fo r
0 .0 3  m g /k g  M G T A -1 4 5  +  p le rix a fo r
L L O Q
C

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0
2
4
6
8
1 0
1 2
1 4
0
5
1 0
1 5
2 0
2 5
H o u r s  P o s t A d m in is tr a tio n
N e u tr o p h ils (x 1 0
6/m L )
2 4
P la c e b o
0 .0 3  m g /k g  M G T A -1 4 5
0 .0 7 5  m g /k g  M G T A -1 4 5
0 .1 5  m g /k g  M G T A -1 4 5
0 .3  m g /k g  M G T A -1 4 5
0 .0 1 5  m g /k g  M G T A -1 4 5
0 .0 0 7 5  m g /k g  M G T A -1 4 5
4 8
0
2
4
6
8
1 0
1 2
1 4
0
2 5
5 0
7 5
1 0 0
1 2 5
1 5 0
H o u r s  P o s t A d m in is tr a tio n
C X C R 2  E x p ressio n
(%  o f b a se lin e )
2 4
A
B
 
A. Neutrophil mobilization following a single dose of MGTA-145 monotherapy or placebo. The shaded 
region represents the normal reference range. B. Relative CXCR2 expression in peripheral blood 
neutrophils following a single dose of MGTA-145 monotherapy or placebo. All data expressed as mean ± 
standard deviation.  
Administration of MGTA-145 two hours after plerixafor led to a median peak WBC count of 36,500 
WBC/µL and 33,550 WBC/µL when MGTA-145 was dosed at 0.015 mg/kg or 0.03 mg/kg, respectively. 
By comparison, the median peak WBC count observed in subjects dosed with placebo + plerixafor in Part 
B was 23,400 WBC per microliter.  This increase was transient, returned to normal range by 24 hours 
post dosing, and was significantly lower than the threshold for leukocytosis (≥ 100,000 WBC/µL) noted in 
the NEUPOGEN® (filgrastim, G-CSF) package insert as precautionary for a patient to undergo dose 
modification (NEUPOGEN Package Insert46). The increased WBC counts observed in subjects dosed 
with MGTA-145 + plerixafor compared to plerixafor alone were predominantly the result of increased 
neutrophil and monocyte mobilization, with a minimal increase in lymphocyte, basophil, and eosinophil 
counts. Similar to what was observed in response to MGTA-145 monotherapy, increasing MGTA-145 
dose above 0.03 mg/kg suppressed neutrophil mobilization when administered in combination with 
plerixafor. 
2.2.10 
Clinical studies evaluating safety of plerixafor 
A phase I study [PLERIXAFOR 98-01] established the initial safety and pharmacokinetic profiles for 
plerixafor.44 Intravenous doses of 10 to 80 µg/kg and subcutaneous doses of 40 and 80 µg/kg were 
administered to healthy volunteers and were generally safe and well tolerated. Maximal increases in 
WBC (approximately three times baseline) were observed at the 40 and 80 µg/kg dose levels in both 
intravenous and subcutaneous populations. 
Figure 4. Effect of MGTA-145 Monotherapy on 
Neutrophil Mobilization and CXCR2 Expression 

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2.2.10.1 
Use of Plerixafor in patients with lymphoma and multiple myeloma  
A phase II trial showed significant enhancement of HSC mobilization when plerixafor was combined with 
G-CSF in patients with non-Hodgkin lymphoma (NHL) and multiple myeloma.47 Two phase III studies 
comparing Hematopoietic Stem and Progenitor Cells (HSPC) mobilization with G-CSF alone to G-CSF 
combined with plerixafor demonstrated a highly significant benefit for the combined arm.48,49 Importantly, 
short-term administration of plerixafor has been shown to be well tolerated. Long-term follow up shows 
no concerns with graft durability or disease progression.50 Plerixafor is U.S. Food and Drug 
Administration (FDA) approved for use in combination with G-CSF in patients with NHL and multiple 
myeloma undergoing stem/progenitor cell mobilization in anticipation of high dose chemotherapy and 
autologous transplantation.  
2.2.10.2 
Use of Plerixafor in related stem cell donors  
Plerixafor is a safe and more rapid stem cell mobilizing agent for healthy volunteer sibling donors. A 
study performed at Washington University demonstrated the safety and efficacy of this concept in 
healthy sibling donors.51 Twenty-five sibling donors were treated with plerixafor at a dose of 240 µg/kg by 
subcutaneous injection, followed 4 hours later by leukapheresis. Successful mobilization was defined as 
a minimum leukapheresis yield of > 2.0 x 106/kg CD34+ cell/kg actual recipient body weight. If the 
minimal yield was not achieved following first leukapheresis, the identical procedure of plerixafor 
administration and repeat leukapheresis was attempted on day 3. One donor receiving plerixafor never 
underwent leukapheresis because of vaso-vagal episodes associated with peripheral line placement. Of 
the remaining 24 donors, 16 (66%) mobilized at least the minimum required number of CD34+ cells after 
first leukapheresis. Seven out of the 8 donors who did not collect sufficient quantity of CD34+ cells 
underwent repeat plerixafor administration on day 3, with 6 such donors mobilizing the target CD34+ cell 
dose. Overall, of the 24 donors undergoing 1 or 2 days of leukapheresis following plerixafor, two did not 
achieve a minimum CD34+ dose of 2.0 x 106/kg, for a failure rate of 8.3%. None of the donors 
experienced any grade 3-4 toxicities. Common grade 1 toxicities were lightheadedness, nausea, 
bloating, flatulence, injection site discomfort, perioral paresthesias, loose stools, and headaches. A total 
of 20 patients received allografts mobilized by plerixafor. All recipients engrafted promptly, with median 
time to neutrophil and platelet recovery of 10 and 12 days, respectively. In this study plerixafor mobilized 
grafts contained significantly greater number of CD3+ cells, compared to G-CSF mobilized grafts. 
However, despite the increased CD3+ content of the allograft, the rate of grade II-IV acute GVHD in the 
recipients (42%) was not more than expected. Interestingly, the cumulative incidence of chronic GVHD 
at 18 months was about 30%, suggesting a possible lowering of the risk of chronic GVHD compared to 
G-CSF mobilized grafts. The group at Washington University then performed a larger dose finding study 
and determined that the 240 μg/kg dose of plerixafor and subcutaneous route of administration 
possessed the best combination of safety and efficacy in normal donors.52  
In 2019, Chen et al reported a phase II study (n=64) of subcutaneous plerixafor to mobilize matched 
related donors for allogeneic transplant for hematologic malignancies.53 The primary objective was to 
determine the proportion of donors who were successfully mobilized: defined as collection of ≥ 2.0 x 106 
CD34+ cells per kg recipient weight in up to 2 apheresis sessions. Recipients subsequently received 
reduced intensity (RIC; n=33) or myeloablative (MAC; n=30) conditioning. Sixty-three of 64 (98%) donors 
achieved the primary objective. The median CD34+ cell dose per kilogram recipient weight collected 
within 2 days was 4.7 (0.9-9.6). Plerixafor was well tolerated with only grade 1 or 2 drug-related AEs 
noted. Bone pain was not observed. Plerixafor-mobilized grafts engrafted promptly. One-year 
progression-free and overall survivals were 53% (95% confidence interval [CI], 36-71%) and 63% (95% 
CI, 46-79%) for MAC and 64% (95% CI, 47-79%) and 70% (95% CI, 53-84%) for RIC recipients, 
respectively. Donor toxicity was reduced relative to G-CSF mobilized related donors. This study 
confirmed the feasibility of using single agent plerixafor for mobilization, but only 27% of donors achieved 
the target of 4 x 106 CD34+ cells/kg in one apheresis session. Five percent of donors required 3 

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apheresis sessions. Although feasible, there is opportunity to improve on the mobilization efficacy of 
plerixafor mobilization. 
2.2.11 
Clinical studies evaluating safety of MGTA-145 
Please see the Investigator’s Brochure (IB) for comprehensive safety information.  
Safety of MGTA-145 was evaluated in the phase I 145-HV-101 study in which 79 healthy volunteers were 
dosed with MGTA-145 out of 107 volunteers enrolled in the study in 4 parts: Part A: single dose MGTA-
145 monotherapy or placebo; Part B: single dose MGTA-145 + plerixafor or placebo; Part C: 2 
consecutive daily doses of MGTA-145 + plerixafor or placebo; Part D: single dose MGTA-145 + plerixafor 
followed by apheresis cell collection.  
Treatment emergent adverse events (TEAEs) overall were generally mild, without an apparent trend 
across dose levels or study part. The most consistently observed adverse event associated with MGTA-
145 infusion was Grade 1 bone pain, most often occurring in the back that begins during the 10-minute 
infusion and generally ends 10-15 minutes later. Specifically, 48 of 102 subjects overall experienced a 
TEAE of back pain. All AEs of back pain were considered to be mild (grade 1) in severity with the 
exception of one AE of back pain, reported as moderate (grade 2) in severity. In Part A of the study, 
grade 1 back pain was reported for 19 of the 24 subjects in the cohorts administered MGTA-145 alone; 
the event was not reported for any subject in the pooled placebo group. In Part B of the study, a total of 
22 subjects in the MGTA-145 + plerixafor group and 2 subjects in the pooled placebo + plerixafor group 
experienced an AE of back pain. One subject who received MGTA-145 0.075 mg/kg + plerixafor 240 
μg/kg, experienced grade 2 (moderate) back pain after initiation of the infusion. MGTA-145 was 
administered 2 hours following plerixafor. Treatment was interrupted after 5 minutes and the AE of back 
pain resolved shortly after stopping the infusion. Of note, grade 2 back pain was not seen with MGTA-
145 doses of 0.07 and 0.15 mg/kg in combination with plerixafor or at monotherapy doses up to 0.3 
mg/kg. 
The known toxicities of plerixafor were observed and generally assessed as mild. Overall, MGTA-145, 
when given in combination with plerixafor, showed no increase in severity or frequency of adverse effects 
expected with plerixafor. 
Safety was also evaluated in study 145-RI-102, an open-label, single-arm, single-dose study to evaluate 
the PK and safety and tolerability of MGTA-145 in subjects with normal estimated glomerular filtration 
rate (GFR) and varying degrees of renal impairment. MGTA-145 was administered at the 0.07 mg/kg 
dose level without plerixafor. The study (n=23) evaluated 6 subjects with normal renal function, 8 subjects 
with mildly decreased GFR, and 9 subjects with moderately decreased GFR. Eighteen subjects 
experienced a TEAE of back pain. Nine of 18 (50%) subjects experienced an AE of back pain which was 
moderate in severity. Although no subjects experienced an AE that led to early study discontinuation, 
study treatment was withdrawn in 4 subjects who experienced an AE of back pain during the 10-minute 
infusion. All study subjects recovered from the AE of back pain. The incidence of AEs of back pain was 
similar across groups regardless of the baseline GFR status. 
2.2.12 
Rationale for studying MGTA-145 + plerixafor mobilized allografts following myeloablative 
conditioning  
Establishing proof of concept for successful engraftment is a key objective of the first allogeneic 
transplant study with MGTA-145 + plerixafor mobilized HSCs. Although the use of RIC has become a 
standard procedure in most transplant centers across the country, following engraftment and preliminary 
GVHD outcomes in a heterogeneous group of RIC and MAC transplants will increase variability of these 
outcomes for a relatively small proof of concept study. Proof of principle that a plerixafor-mobilized 
allograft can lead to prompt recovery in recipients receiving RIC has already been established in the 
Chen et al study.54 The present study should permit us to generate sufficient safety and efficacy data of 
MGTA-145 + plerixafor mobilized allografts that may be generalizable to other allogeneic transplant 

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donors and provide a rationale for comparison to G-CSF based mobilization. At the present time, this 
study will not evaluate the use of MGTA-145 + plerixafor-mobilized allografts in recipients scheduled to 
receive non-myeloablative (NMA) conditioning since these recipients will be more difficult to evaluate for 
neutrophil recovery. Hence, we propose a single stratum phase II study of MGTA-145 at 15 µg/kg 
intravenous infusion plus plerixafor administered at 240 µg/kg subcutaneously for mobilization in healthy 
sibling donors of recipients undergoing MAC allogeneic transplantation.  
2.2.13 
Rationale for post-transplant growth factor administration  
We will administer G-CSF beginning day +7 in recipients of MAC transplantation since G-CSF was used 
in all recipients in the original Washington University study and in most recipients in the Chen et al 
study.51,53 While the use of myeloid growth factors following allogeneic transplantation varies widely 
between centers, a recent CIBMTR study did show acceleration of neutrophil recovery without any 
increase in the risk of acute or chronic GVHD.54 
2.3 Risk/Benefit Assessment 
2.3.1 
Known Potential Risks 
The most commonly encountered AEs observed in subjects treated with MGTA-145 and plerixafor were 
back pain/musculoskeletal pain, diarrhea, nausea, dizziness, abdominal pain and headache. Clinically, 
an important risk during administration of MGTA-145 includes AEs of back pain occurring within 10 
minutes of study treatment administration.  
There is no known risk for the combination of MGTA-145 and plerixafor. Results following co-
administration of MGTA-145 and plerixafor did not demonstrate any additive toxicological effect, 
indicating no further risk posed by the addition of MGTA-145 to plerixafor administration despite the 
synergistic pharmacological effect. 
2.3.2 
Known Potential Benefits 
Mobilization with MGTA-145 + plerixafor offers the potential for same-day mobilization and apheresis cell 
collection, as compared to 4-5 days of G-CSF for mobilization. The shorter duration of mobilization has 
the potential to cause less interference with usual activities. G-CSF causes bone pain in a majority of 
donors for up to one week. Although MGTA-145 causes bone pain, it generally lasts < 15 minutes. Thus, 
there is the potential for a reduction in the cumulative pain morbidity associated with mobilization. The 
combination of less interference with daily life combined with decrease total pain may increase donor 
willingness to undergo mobilization and apheresis, thereby increasing the pool of potential allogeneic 
donors.  
2.3.3 
Assessment of Potential Risk and Benefits 
The overall safety profile of MGTA-145 is acceptable, and combination therapy does not substantially 
increase the risks of side effects. Thus, the potential additional benefits of combination therapy are likely 
to outweigh the risk of AEs experienced during MGTA-145 treatment. Back pain is an important risk for 
MGTA-145 because it has led to discontinuation of infusion in phase I studies. Based on the available 
safety data for MGTA-145, the benefit-risk profile of the medicinal product remains favorable.  
3 
OBJECTIVES  
3.1 Primary Objective 
To determine the proportion of donors whose cells can be successfully mobilized and collected with a 
sufficient CD34+ cell dose using same-day, single-dose MGTA-145 + plerixafor as the mobilizing agents. 
Donor mobilization following MGTA-145 + plerixafor will be considered successful if ≥ 2.0 x 106 CD34+ 
cells/kg actual recipient weight are collected in one leukapheresis collection. All donors receiving MGTA 
145 + plerixafor will be included in the analysis of the primary objective. 

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3.2 Secondary Objectives 
3.2.1 
Donor Related 
3.2.1.1 
To determine the proportion of donors whose cells can be successfully mobilized 
and collected with a target CD34+ cell dose of at least 4.0 x 106 CD34+ cells/kg actual 
recipient weight in one apheresis collection 
3.2.1.2 
To ascertain the incidence and severity of acute adverse events (AEs) before and 
during apheresis experienced by donors receiving MGTA-145 + plerixafor  
3.2.1.3 
To characterize the adverse effects experienced by donors receiving MGTA-145 + 
plerixafor 
3.2.2 
Recipient Related 
3.2.2.1 
To determine the incidence of and kinetics of neutrophil and platelet recovery after 
transplantation of hematopoietic cells mobilized with MGTA-145 + plerixafor  
3.2.2.2 
To determine the incidence of primary and secondary graft failure after 
transplantation of hematopoietic cells mobilized with MGTA-145 + plerixafor  
3.2.2.3 
To determine the incidence and severity of acute and chronic graft versus host 
disease (GVHD) after transplantation of hematopoietic cells mobilized with MGTA-145 + 
plerixafor 
3.2.2.4 
To determine the incidence of treatment-related mortality and disease 
relapse/progression after transplantation of hematopoietic cells mobilized with MGTA-145 + 
plerixafor  
3.2.2.5 
To determine the probability of progression-free and overall survival after 
transplantation of hematopoietic cells mobilized with MGTA 145 + plerixafor 
3.2.2.6 
To characterize the adverse effects experienced by recipients receiving grafts 
mobilized by MGTA-145 + plerixafor 
3.3 Exploratory Objectives 
3.3.1 
Graft Related 
3.3.1.1 
To describe the cellular composition of allografts mobilized with MGTA-145 + 
plerixafor (stem/progenitor cells, T/B/NK-cells, colony forming units)  
3.3.2 
Recipient Related 
3.3.2.1 
To describe T-cell (CD3+) and myeloid chimerism after transplantation of 
hematopoietic cells mobilized with MGTA-145 + plerixafor  
3.3.2.2 
To assess the rate and quality of immune reconstitution as evidenced by peripheral 
blood immunophenotype after transplantation of hematopoietic cells mobilized with MGTA-
145 + plerixafor  
3.3.2.3 
To determine the incidence of CMV reactivation after transplantation of 
hematopoietic cells mobilized with MGTA-145 + plerixafor in cytomegalovirus (CMV) 
seropositive recipients 
3.3.3 
Donor Related 
3.3.3.1 
To characterize the PK of MGTA-145 
3.3.3.2 
To assess treatment related Anti-drug Antibody (ADA) 

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3.3.3.3 
To describe donation experience of PBSC donors mobilized with MGTA-145 + 
plerixafor 
4 
STUDY DESIGN 
4.1 Overall Design 
This is a Phase II, open-label, multicenter, prospective study of MGTA-145 + plerixafor-mobilized HLA-
matched sibling and matched unrelated donor allografts for myeloablative HSCT in recipients with 
hematological malignancies. This study will establish the safety and efficacy of the combination of MGTA-
145 infusion and subcutaneous plerixafor for HSC mobilization. Donor-recipient pairs will be recruited for 
this study. Collection and transplant outcomes will be assessed. AEs will be reported using event terms 
and grading for severity per the Common Terminology Criteria for Adverse Events (CTCAE) version 5. 
Safety monitoring rules related to donor and recipient AEs are in place, which may result in a Data Safety 
Monitoring Board (DSMB) evaluation as detailed in Section 8.2.  
4.2 Justification for Dose 
Optimal doses from the 145-HV-101 Phase I study were 0.015 mg/kg and 0.03 mg/kg. The dose in 
humans of 0.03 mg/kg represents a 1500-fold for Cmax and 2300-fold for AUC relative to exposures in the 
NOAEL for macaques in the definitive repeat-dose toxicology study. In Study 145-HV-101, 6 dose 
cohorts of MGTA-145 infusion alone were evaluated in Part A, and no dose limiting toxicities (DLTs) were 
observed with dosing up to 0.3 mg/kg. In Part B of the study, MGTA-145 was administered in conjunction 
with plerixafor. When administered immediately after plerixafor, there were no DLTs observed at the 0.03, 
0.07, and 0.15 mg/kg doses. When MGTA-145 was dosed 2 hours after plerixafor, the maximum 
tolerated dose (MTD) was 0.07 mg/kg, with an MTD defined as a single CTCAE grade 2 event. 
Pharmacodynamic assessment of CD34 cell mobilization showed apheresis collection yields of CD34+ 
and CD34+CD90+CD45RA- cells were highest with the 0.03 and 0.015 mg/kg doses, respectively. The 
0.015 mg/kg dose was selected since it is the lowest dose that maximizes apheresis cell yield.  
4.3 End of Study Definition  
The study is considered complete when the last subject has completed their last study visit. 
5 
STUDY POPULATION 
The donor population for the study includes those subjects undergoing peripheral blood mobilization and 
collection of grafts for allogeneic HSCT.  
The recipient population for the study includes those subjects with AML, ALL or MDS receiving a 
hematopoietic stem cell transplant using the product obtained from a donor who underwent mobilization 
using MGTA-145 in combination with plerixafor.  
Once the recipient has qualified for the study, the intended donor (related or unrelated) must meet all 
eligibility criteria for the study in order for the recipient to complete enrollment. The recipient will remain 
as ‘screening’ until the donor is enrolled on the study. The recipient may commence conditioning after a 
clinically successful allograft has been collected from the donor. 
5.1 Eligibility Criteria 
5.1.1 
Donor Inclusion Criteria 
1. 
Donor medical suitability and eligibility will be determined following Institution or National 
Marrow Donor Program (NMDP)/Be The Match standards  
2. 
Age 18-65 years old at the time of signing informed consent 
3. 
8/8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer unrelated donor 
4. 
Fulfill Institution or NMDP/Be The Match criteria to serve as a mobilized blood cell donor 

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5. 
Serum creatinine < 1.5 x institution ULN or estimated creatinine clearance (CRCL) > 50 mL/min 
using the Modification of Diet in Renal Disease Study (MDRD) equation or similar method 
5.1.2 
Donor Exclusion Criteria 
1. 
Donor unwilling or unable to give informed consent, or unable to comply with the protocol 
including required follow-up and testing  
2. 
Donor already enrolled on another investigational agent study  
3. 
Pregnant or breastfeeding females, sexually active female or male donors not willing or able to 
use adequate contraception, or males who do not agree to refrain from donating sperm, from 
the time of consent through 3 months after treatment with MGTA-145 + plerixafor 
5.1.3 
Recipient Inclusion Criteria 
1. 
At least 18 years old at the time of signing informed consent  
2. 
Has an available 8/8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer unrelated 
donor willing to donate peripheral blood stem cells (PBSC) for transplant  
3. 
Fulfill additional individual Transplant Center Criteria for transplant beyond NMDP/Be The 
Match criteria 
4. 
One of the following diagnoses:  
o Acute myelogenous leukemia (AML) in 1st remission or beyond with ≤ 5% marrow blasts 
and no circulating blasts.  Documentation of bone marrow assessment will be accepted 
within 45 days prior to the date of consent. 
o Acute lymphoblastic leukemia (ALL) in 1st remission or beyond with ≤ 5% marrow blasts and 
no circulating blasts. Documentation of bone marrow assessment will be accepted within 45 
days prior to the date of consent.  
o Patients with myelodysplasia (MDS) with no circulating blasts and with less than 10% blasts 
in the bone marrow (higher blast percentage allowed in MDS due to lack of differences in 
outcomes with < 5% or 5-10% blasts in MDS). Documentation of bone marrow assessment 
will be accepted within 45 days prior to the date of consent. 
5. Cardiac function: Left ventricular ejection fraction at least 45% based on most recent 
echocardiogram or MUGA results obtained via standard of care 
6. Estimated creatinine clearance acceptable per local institutional guidelines  
7. Pulmonary function: diffusing capacity of the lungs for carbon monoxide (DLCO) corrected for 
hemoglobin at least 50% and forced expiratory volume in first second (FEV1) predicted at least 
50% based on most recent DLCO results obtained via standard of care 
8. Liver function acceptable per local institutional guidelines  
9. Karnofsky performance status (KPS) of 70% or greater (See Appendix A) 
10. Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score of 4 or less (See Appendix 
B) 
5.1.4 
Recipient Exclusion Criteria 
1. Subject unwilling or unable to give informed consent, or unable to comply with the protocol 
including required follow-up and testing  
2. Subject whose donor does not meet the eligibility criteria and is a screen fail 

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3. Subjects with a prior allogeneic transplant  
4. Subjects with active, uncontrolled infection at the time of the transplant preparative regimen  
5. Pregnant or breastfeeding females, sexually active female or male subjects not willing or able to 
use adequate contraception, or males who do not agree to refrain from donating sperm, from the 
time of consent through 3 months after PBSC infusion 
6. Subjects with clinical evidence of active Central Nervous System (CNS) tumor involvement as 
evidenced by documented disease on examination of spinal fluid or MRI within 45 days of start of 
conditioning  
7. A condition, which, in the opinion of the clinical investigator, would interfere with the evaluation of 
primary and secondary endpoints  
8. Planned treatment with a new investigational agent from the time of transplant through 30 days 
post-transplant 
5.2 Screen Failures 
Screen failures are defined as subjects who consent to participate in the clinical trial but are not 
subsequently entered in the study. A minimal set of screen failure information may be collected, such as 
demographics, screen failure details, eligibility criteria, and any SAEs.  
5.3 Strategies for Recruitment and Retention 
Potential subjects will be identified at participating transplant centers in the course of clinical care for 
allogeneic HSCT.  
5.3.1 
Related Donors 
Transplant centers will be responsible for managing study procedures for related donors. Potential 
donors must be evaluated for suitability and eligibility. 
5.3.2 
Unrelated Donors 
Be The Match will manage all unrelated donor contact, informed consent sessions, donor medical 
suitability and eligibility determinations, donor sample collections and donor hematopoietic progenitor cell 
apheresis (HPC(A)) collections. Only donors who provide written informed consent and who meet the 
protocol requirements will be permitted to donate an HPC(A) product for this protocol. 
6 
SUBJECT ACCOUNTABILITY 
6.1 Point of Enrollment 
A donor subject is considered enrolled when the informed consent form has been signed and the subject 
is confirmed to meet eligibility criteria for the study. A recipient subject is considered enrolled when the 
informed consent form has been signed, the subject is confirmed to meet eligibility criteria for the study, 
and the intended donor has met the criteria for enrollment on the study. Recipient subjects will remain in 
screening status until intended donor has been successfully enrolled on the study. Subjects with 
documented informed consent to participate in research and meeting all eligibility criteria for the study 
must be enrolled in the Medidata Rave® electronic data capture (EDC) application prior to initiating any 
study-specific activity.  
6.2 Withdrawal 
Subjects have the right to withdraw consent for study participation at any time and for any reason. 
Subject data collected up to withdrawal of consent will be retained and included in the analysis of the 
study. An explanation of why the subject is withdrawing from the study must be documented in a Study 
Exit form in the Medidata Rave® EDC application. All subjects who withdraw from the study with ongoing 

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SAEs or AEs of at least possible relatedness must be followed until resolution of the event, death, or until 
the site Principal Investigator concludes that the event is stable with no further improvement anticipated. 
Subjects lost to follow-up must be reported on the Study Exit form in the Medidata Rave® EDC 
application (see Section 8.3). 
6.3 Subject Status and Classification 
The following subject classifications will be utilized for this protocol: 
Important: Once the recipient has qualified for the study, the intended donor (related or unrelated) must 
meet all eligibility criteria for the study in order for the recipient to complete enrollment. The recipient will 
remain as ‘screening’ until the donor is enrolled on the study.  The recipient may commence conditioning 
after a clinically successful allograft has been collected from the donor. 
• 
Screening: A subject who is being evaluated for study participation.  
• 
Screen Failure: A subject who has signed the informed consent but is found to not meet eligibility 
criteria. These subjects do not count against the enrollment ceiling. 
• 
Consented, Eligible, Enrolled but not Treated: A subject who signs the informed consent, meets 
eligibility criteria but then does not undergo the main study procedure. The original Informed 
Consent form and screening documentation for these subjects should be maintained in the site’s 
files. There are no follow-up requirements for these subjects. These subjects do not count against 
the enrollment ceiling. 
• 
Enrolled, treated: A donor subject who is consented, meets eligibility and receives treatment per 
the study protocol. A recipient subject who is consented, meets eligibility and whose intended 
donor has met the criteria for enrollment on the study. Both donor and recipient subjects are 
followed in accordance with the follow-up schedule and included in all analyses of safety and 
efficacy. 
• 
Early Terminated: A subject who enrolled on the study and subsequently met a criterion for 
discontinuation from the study. 
• 
Completed: A donor subject who receives treatment and completes the 6-month follow-up visit or 
contact, or a recipient subject who receives treatment and completes the 1-year follow-up visit or 
contact 
7 
STUDY INTERVENTION 
 
7.1 Study Intervention(s) Administration 
7.1.1 
Donor Treatment Plan   
7.1.1.1 
Plerixafor and MGTA-145 Administration  
Plerixafor  
On the first day of mobilization, the donor will arrive in the morning and the following procedures will be 
performed prior to study drug administration:  
• 
Review of medical history and current medications 
• 
Physical exam 
• 
Measurement of weight in kg  
• 
Vital signs including blood pressure, pulse, respiratory rate, and temperature  
• 
Serum pregnancy test for female donors of childbearing potential; Note: pregnancy test can be 
performed up to 48 hours prior to dosing 

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• 
Complete blood count (CBC) with automated differential and platelet count  
• 
Peripheral blood sample to be collected for CD34+ cell analysis by flow cytometry 
• 
Anti-drug Antibody (ADA) sample collected 
• 
Plerixafor will be administered subcutaneously at 240 µg/kg actual donor weight approximately 2 
hours prior to planned leukapheresis. Donors will be observed for 15-30 minutes after dosing. 
Only administer plerixafor in a monitored setting when personnel and therapies are immediately 
available for the treatment of anaphylaxis and other hypersensitivity reactions.  
The following procedures will be performed after dosing of plerixafor:   
• 
Vital signs monitored post plerixafor injection at 30 minutes (±10 minutes), 60 minutes (±10 
minutes) and per local institutional guidelines 
• 
Monitor donor for hypersensitivity reactions including anaphylaxis.  
• 
Monitor donor for vasovagal reaction. The majority of vasovagal events occur within an hour of 
plerixafor administration.  
• 
It is suggested that donors remain recumbent for the first hour after plerixafor administration with 
access to a bedside commode. The investigator should consider the clinical status and relevant 
laboratory parameters of each donor and consider extending the observation and recumbent 
period as warranted on a case-by-case basis.  
• 
If donors choose to get up during the observation period after plerixafor administration when 
recumbency is recommended, it is suggested that orthostatic (supine, sitting and standing) blood 
pressure and pulse be collected before they get up, and also at any time donors experience 
symptoms consistent with a vasovagal reaction (e.g., orthostatic hypotension, bradycardia, 
lightheadedness, fainting). Should syncope, bradycardia, orthostasis or lightheadedness occur, 
an electrocardiogram (ECG) should be obtained.  
• 
Should symptomatic orthostasis occur (a decrease in > 20 mm Hg systolic or > 10 mm Hg 
diastolic within 3 minutes of standing), it is recommended that the donor remain recumbent until 
orthostasis resolves and hydration should be administered if clinically acceptable.  
• 
Donors will be assessed by clinic and/or apheresis staff for evidence of AEs related to plerixafor 
during the injection and at 30 minutes (±10 minutes) and 60 minutes (±10 minutes) following 
administration of plerixafor. Any AEs observed will be noted in the Medidata Rave® EDC 
application by the staff assessing the donor. If any CTCAE grade 3 or greater AE is observed, the 
clinical staff (e.g. attending physician, site Principal Investigator, mid-level providers) will be 
contacted immediately for further instruction. If an AE requires any type of intervention, the 
requirement for and nature of that intervention should be clearly documented in the Medidata 
Rave® EDC application.   
MGTA-145  
• 
PK sample to be collected prior to MGTA-145 infusion (within 60 minutes of the start of the 
infusion)  
• 
Vital signs monitored prior to MGTA-145 dosing, during infusion and 30 minutes (±10 minutes), 
60 minutes (±10 minutes, 120 minutes (±20 minutes), and 240 minutes (±20 minutes) following 
start of infusion  
• 
MGTA-145 will be administered at a dose of 0.015 mg/kg intravenously over 3 minutes, starting 
approximately 2 hours after plerixafor. The infusion time may range from 1 to 10 minutes as 
clinically indicated.  

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• 
PK sample to be collected 15-30 minutes after start of infusion  
• 
Donors will be assessed by clinic and/or apheresis staff for evidence of AEs related to MGTA-145 
during the infusion and at 30 minutes (±10 minutes), 60 minutes (±10 minutes, 120 minutes (±20 
minutes), and 240 minutes (±20 minutes) following start of infusion. Any AEs observed will be 
noted in the Medidata Rave® EDC application by the staff assessing the donor. If any CTCAE 
grade 3 or greater AE is observed, the clinical staff (e.g. attending physician, site Principal 
Investigator, mid-level provider) will be contacted immediately for further instruction. If an AE 
requires any type of intervention, the requirement for and nature of that intervention should be 
clearly documented in the Medidata Rave® EDC application.  
• 
After MGTA-145 infusion but prior to leukapheresis, a peripheral blood sample for CD34+ cell 
analysis by flow cytometry will be collected. 
• 
As soon as feasible after MGTA-145 infusion (approximately 30 minutes following the 
administration of MGTA-145), begin leukapheresis procedure to process at least 4 times blood 
volume, but no more than the institutional limit.  
• 
Following the completion of leukapheresis, the following will be done: 
• 
Vital signs 
• 
Collect sample for CBC with automated differential and platelet count  
• 
Collect sample for CD34+ cell analysis by flow cytometry from the apheresis product 
• 
Collect sample for graft immunophenotyping from the apheresis product 
• 
Collect sample for colony forming unit testing from the apheresis product 
• 
Both during and immediately following completion of leukapheresis, donor will be assessed for 
evidence of AEs. Any AEs observed will be noted on the day of collection form by the staff 
assessing the donor. If any CTCAE grade 3 or greater AE is observed, the clinical staff (attending 
physician, site Principal Investigator, mid-level provider) will be contacted immediately for further 
instruction. It is possible that grade 3 or greater AE could be entirely related to the leukapheresis 
procedure rather than MGTA-145 or plerixafor, but this will ultimately be determined by the 
investigator at the site. Otherwise, the donor may be discharged from the leukapheresis center.  
• 
Leukapheresis product must be cryopreserved until the recipient is ready to receive the product.  
7.1.1.2 
Target Collection 
The target CD34+ cell dose for the donor allograft collected following MGTA + plerixafor is ≥ 4.0 x 106/kg 
actual recipient weight. The leukapheresis should target 4-5 blood volumes.  
If the target CD34+ cell dose of ≥ 4.0 x 106/kg actual recipient weight is met after the first leukapheresis, 
no further collections will be performed. Otherwise, the donor will return the following morning and the 
same procedures described above will be repeated in order to achieve the target CD34+ cell dose. The 
second leukapheresis session should also be targeted to process 4-5 blood volumes just as with the first 
leukapheresis session, as long as it can be done safely. If the sum of the two collected leukapheresis 
products is ≥ 2.0 x 106 CD34+ cells/kg actual recipient weight, collection will be completed.  
If the donor mobilizes ≥ 2.0 x 106 CD34+ cells/kg actual recipient weight after one or two days of MGTA-
145 + plerixafor administration, the treating centers are strongly encouraged to use this as the sole graft 
for the recipient in order to keep the recipient on study and evaluate for important secondary endpoints.  
In the event that the donor fails to mobilize at least 2.0 x 106 CD34+ cells/kg actual recipient weight after 
two days of treatment with MGTA-145 + plerixafor, the center will be allowed to use that graft (regardless 
of the total CD34+ cell dose) at their discretion, and the recipient will be followed. Alternatively, in this 
scenario the treating center may choose to remobilize the donor with G-CSF or collect bone marrow for 
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Each leukapheresis product (that is, from each day of collection) must be analyzed for CD34+ and CD3+ 
cell content, normalized to recipient actual body weight, prior to cryopreservation. In addition, exploratory 
immunophenotypic analysis and colony forming unit assays will be performed. Following thawing, an 
analysis of CD34+ cell viability must also be performed.  
7.1.1.3 
Monitoring donors for adverse events  
Adverse Events will be monitored and collected on donors beginning from the time of signing informed 
consent and up to 72 hours following dosing with MGTA-145 and plerixafor.  The donor will be closely 
monitored during treatment and mobilization with MGTA-145 + plerixafor for AEs. Refer to Section 9.8 
for Adverse Event Reporting Requirements. 
To date in Magenta studies, the most commonly encountered AEs observed in plerixafor treated healthy 
donors include injection site erythema, headache, paresthesias, nausea, diarrhea, and flatulence. The 
CTCAE grade of toxicities observed have been mild (grade 1) or moderate (grade 2). The most 
commonly encountered AEs observed in subjects treated with MGTA-145 and plerixafor were back 
pain/musculoskeletal pain, diarrhea, nausea, dizziness, abdominal pain and headache as well as 
transient bone pain, predominantly in the lower back, that begins during infusion and lasts < 20 minutes. 
The CTCAE grade has been mild (grade 1) or moderate (grade 2).  
7.1.1.4 
 Cryopreservation and Thawing of Collected Product  
To ensure that an adequate Hematopoietic Progenitor Cells (HPC) product is available prior to 
admission of the recipient and start of the preparative regimen, the HPC products must be collected and 
cryopreserved according to the local institutional guidelines. Collection by apheresis and 
cryopreservation are performed using validated facility procedures. A sample adequate to perform post-
thaw viability testing must also be cryopreserved. Cryopreserved products and samples must be stored 
in the liquid or vapor phase of liquid nitrogen freezers until completion of the required product testing. 
HPC products must be tested at the time of collection for total nucleated cell count (TNC), CD34, CD3 
and red blood cell count (RBC) content according to local procedures. The subject may commence 
conditioning as long as a CD34+ dose of ≥ 2.0 x 106/kg actual recipient weight has been collected. Since 
the cellular content (e.g., neutrophils) of HPC products has the potential to impact how well cells survive 
the cryopreservation process, a sample of the cryopreserved product must be thawed and tested to 
determine the percent recovery of viable CD34+ cells. Upon thawing of the cryopreserved sample, it is 
recommended that the sample contain at least a viable CD34+ count of 2.0 x 106/kg actual recipient 
weight cells to proceed with the transplant.   
Products are to be thawed for infusion according to validated facility procedures. Due to the potential for 
hemolysis due to both the freeze thaw process and major ABO incompatibility, sites should consider 
either washing products at the time of thaw or splitting the infusion over multiple days or morning and 
evening if the volume of RBCs to be infused exceeds the institutional RBC maximum, regardless of ABO 
compatibility.  
7.1.1.5 
Transport and Shipment of Product  
If product transportation or shipment is required, product may be shipped fresh and cryopreserved at the 
transplant center destination. Product shipment will follow NMDP standard shipping procedures.  
7.1.2 
Recipient Treatment Plan  
7.1.2.1 
Administration of conditioning to the subject  
The following conditioning regimens can be used for subjects enrolled on this trial:  
Myeloablative (one of five general regimens):  
• 
Busulfan (≥ 9 mg/kg orally (PO) or intravenously (IV) total) with fludarabine  
• 
Busulfan (≥ 9 mg/kg orally (PO) or intravenously (IV) total) with cyclophosphamide  

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• 
Total body irradiation (≥ 1000 cGy) plus etoposide  
• 
Total body irradiation (≥ 500 cGy) plus cyclophosphamide  
• 
Melphalan 140 mg/m2 with fludarabine 
Details of the conditioning regimen are at the treating center’s discretion as long as the backbone 
requirements for the regimen as specified above are met. Centers using pharmacokinetically targeted 
busulfan dosing should target an AUC of at least 4,000 +/-10% µM*min per day for 4 days. 
7.1.2.2 
GVHD Prophylaxis  
All recipients must receive pharmacological GVHD prophylaxis. Choice of agents for GVHD prophylaxis 
will also be left to the discretion of the treating center provided no monoclonal (e.g., alemtuzumab) anti-
T-cell antibodies or any form of ex vivo T-cell depletion is employed. GVHD prophylaxis may include 
post-transplant cyclophosphamide or a calcineurin phosphatase inhibitor in combination with 
methotrexate, mycophenolate mofetil, or sirolimus in accordance with local institutional guidelines. 
Tapering of immunosuppressive agents will be per local institutional guidelines. In general, it is 
recommended that tapering of immunosuppression not be started, in the absence of disease 
progression, until days 90-100 post-transplant since the risks for and tempo of GVHD following MGTA-
145 + plerixafor-mobilized allografts are not completely understood.  
7.1.2.3 
Stem Cell Transplantation (Day 0)  
Recipients who have a clinically successful allograft (≥ 2.0 x 106 CD34+ cells/kg actual recipient weight) 
collected following mobilization with MGTA-145 + plerixafor will undergo the testing described below 
and will receive the MGTA-145 + plerixafor mobilized allograft on Day 0.  
• 
Physical examination  
• 
CBC with differential and platelet count  
• 
Serum creatinine/AST/ALT/LDH/Total bilirubin  
• 
Vital signs will be monitored 1 hour prior to the allograft infusion and then approximately 15 
minutes, 30 minutes, 1 hour, 2 hours, and 4 hours post infusion  
7.1.3 
Supportive Care  
Supportive care will be given in keeping with local institutional guidelines.  
7.1.3.1 
Growth Factors  
G-CSF will be administered at a dose of 5 µg/kg/day actual recipient weight (may be rounded to the 
nearest vial size) by subcutaneous injection starting day +7 and continuing until the absolute neutrophil 
count (ANC) is > 1.5 x 109/L for 2 consecutive days.   
7.1.3.2 
Blood Products  
Transfusion thresholds for blood product support will be consistent with local institutional guidelines. All 
cellular blood products will be irradiated. Recipients who are CMV negative will receive CMV negative or 
leuko-reduced blood products from study entry. Blood products will be recorded as a concomitant 
medication. 
7.1.3.3 
Prophylaxis Against Infections  
Prophylaxis against infections during the peritransplant period will be done according to local 
institutional guidelines.  
7.1.3.4 
Intravenous Immune Globulin (IVIG)  
IVIG administration will be left to local institutional guidelines.  

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7.1.3.5 
ABO Incompatibility  
All recipients with ABO incompatibility should be evaluated and treated per local institutional guidelines.  
7.1.3.6 
Anti-seizure Prophylaxis  
Phenytoin, levetiracetam (Keppra), or a benzodiazepine may be used with busulfan in accordance with 
local institutional guidelines. 
7.1.4 
Dosing Administration  
Donors will be administered both investigational interventions, plerixafor and MGTA-145, described 
below.  
7.2 Study Drugs 
7.2.1 
Description 
7.2.1.1 
Plerixafor 
Plerixafor (injection) is a sterile, preservative-free, clear, colorless to pale yellow, isotonic solution for 
subcutaneous injection. Each mL of the sterile solution contains 20 mg of plerixafor. Each single-use vial 
is filled to deliver 1.2 mL of the sterile solution that contains 24 mg of plerixafor and 5.9 mg of sodium 
chloride in Water for Injection adjusted to a pH of 6.0 to 7.5 with hydrochloric acid and with sodium 
hydroxide, if required.  
7.2.1.2 
MGTA-145 
MGTA-145 (Gro-beta truncate or GROβT) is a synthetically manufactured naturally occurring four amino 
acid-truncated variant of the immunomodulatory protein CXCL2. 
MGTA-145 is formulated at 20 mg/mL in 20 mM sodium acetate, 150 mM sodium chloride, 0.02% (w/v) 
Polysorbate 80 at pH 4.0. MGTA-145 is supplied in a single-dose 2 mL Type I amber glass vial. Each vial 
contains not less than 1 mL. 
7.2.2 
Packaging and Labeling 
7.2.2.1 
Plerixafor 
Commercially available plerixafor will be supplied by the study site in the commercially approved 
packaging and labeling. 
7.2.2.2 
MGTA-145 
MGTA-145 is manufactured, packaged, and labelled according to Good Manufacturing Practice 
guidelines and applicable laws or regulations. The study drug is suitably packaged in such a way 
as to protect it from deterioration during transport and storage. 
MGTA-145 is supplied in single-use amber vials packaged in cartons. Each vial is labeled as required 
per country requirements. Each vial is filled to deliver 1.0 mL of 20 mg/mL solution containing 20 mg 
MGTA-145. 
7.2.3 
Product Storage and Stability 
7.2.3.1 
Plerixafor  
Plerixafor should be stored according to the manufacturer’s instructions. 
7.2.3.2 
MGTA-145 
Vials of MGTA-145 should be stored at -20°C ± 5°C in the carton to protect from light. 

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7.2.4 
 Preparation and Administration 
7.2.4.1 
Plerixafor  
Plerixafor is supplied as a ready-to-use formulation. The contents of the vial must be transferred to a 
suitable syringe for administration. Vials should be inspected visually for particulate matter and 
discoloration prior to administration and should not be used if there is particulate matter or if the solution 
is discolored.  
Plerixafor should be administered subcutaneously per the manufacturer’s instructions. Plerixafor will be 
administered subcutaneously at 240 µg/kg actual donor weight approximately 2 hours prior to planned 
leukapheresis. Plerixafor will be administered up to two times, once on each of two consecutive days.    
7.2.4.2 
MGTA-145 
Vials of MGTA-145 should be thawed overnight at 2°C – 8°C prior to use and are stable at 2°C – 8°C for 
up to 30 days. Once thawed, MGTA-145 should not be refrozen. 
Vials should be inspected visually for particulate matter and discoloration prior to administration and should 
not be used if there is particulate matter or if the solution is discolored. 
MGTA-145 should be diluted in 0.9% saline in accordance with the study protocol and Pharmacy Manual. 
Once prepared, MGTA-145 may be stored for no more than 8 hours at room temperature prior to 
administration.  
Approximately 2 hours after plerixafor administration MGTA-145 will be administered intravenously at 
0.015 mg/kg actual donor weight over 3 minutes. The infusion time may range from 1 to 10 minutes as 
clinically indicated.  MGTA-145 will be administered up to two times, once on each of the two 
consecutive days. 
The Investigator or designee is responsible for recording the details of the intravenous infusion of MGTA 
145 in the Medidata Rave® EDC application. 
1. The investigator or designee must confirm appropriate temperature conditions have been 
maintained during transit for all study intervention received and any discrepancies are reported 
and resolved before use of the study intervention. 
2. Only subjects enrolled in the study may receive study intervention and only authorized site staff 
may supply or administer study intervention. All study intervention must be stored in a secure, 
environmentally controlled, and monitored area in accordance with the labeled storage conditions 
with access limited to the investigator and authorized site staff. 
3. The investigator, institution, or the head of the medical institution (where applicable) is responsible 
for study intervention accountability, reconciliation, and record maintenance (i.e., receipt, 
reconciliation, and final disposition records). 
4. Further guidance and information for the final disposition of unused study interventions are 
provided in the Pharmacy Manual. 
7.3 Concomitant therapy 
Donors may continue all medications in compliance with local apheresis center mobilization and 
apheresis local institutional guidelines. Premedication and/or treatment for possible AEs related to 
MGTA-145 and plerixafor are permitted and should be documented in the Medidata Rave® EDC 
application.  
Prohibited medications for donors include concomitant use of Granulocyte-macrophage colony-
stimulating factor (GM-CSF), G-CSF and investigational products. 
There are no prohibited medications for recipients.  

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8 
STUDY INTERVENTION DISCONTINUATION AND SUBJECT 
DISCONTINUATION/WITHDRAWAL 
8.1 Discontinuation of Study Intervention 
In the event that the donor fails to mobilize at least 2.0 x 106 CD34+ cells/kg actual recipient weight after 
two days of treatment with MGTA-145 + plerixafor, the center will be allowed to use that graft (regardless 
of the total CD34+ cell dose) at their discretion, and the recipient will be followed. Alternatively, in this 
scenario the treating center may choose to remobilize the donor with G-CSF or collect bone marrow for 
transplantation as a supplement or replacement for the MGTA-145 + plerixafor mobilized graft.  
8.2 Study-wide Safety Monitoring Rules 
Monitoring of Donor safety: Donors will be monitored closely for AEs up to 72 hours following infusion.  If 
a CTCAE grade 3 or greater AE believed to be at least possibly related to plerixafor or MGTA-145 is 
observed in any of the donors, the AE must be entered in Medidata Rave® EDC application within 24 
hours of learning of the event. This will notify the CIBMTR Medical Monitor who will consult with Magenta, 
the NMDP Protocol Chair and the DSMB Study Chairperson. At that time, a decision will be made as to 
whether the DSMB needs to meet to review the AE further and whether the study enrollment should be 
suspended, at least temporarily until the DSMB meeting. Refer to Section 9.8 for Adverse Event 
Reporting Requirements.  
Monitoring of Recipient safety: Three safety monitoring rules will be utilized to monitor recipient safety. 
The CIBMTR Medical Monitor will review the relevant data on a weekly basis to assess whether any 
stopping rules have been met. If a stopping rule as described below is triggered, the DSMB will be 
notified, and enrollment will be paused while the DSMB conducts a review of the safety data. The safety 
monitoring rules serve as a trigger for consultation with the DSMB for additional review and would not 
mandate automatic closure of study enrollment. Since all events being monitored are expected to occur 
with some frequency in the general transplant population, a sequential hypothesis testing framework will 
be utilized to identify whether the event rate is exceeding what is expected.  The following safety 
endpoints and null hypothesis values will be used for sequential testing.   
• 
The first monitoring rule is based on primary graft failure, which is expected to be no higher than 
3% of recipients by Day 28. 
• 
The second monitoring rule is based on treatment-related mortality (TRM), which is expected to 
be no higher than 15% of recipients by Day 100. 
• 
The third monitoring rule is based on grades 3-4 acute GVHD (aGVHD), which is expected to be 
no higher than 10% of recipients by Day 100. 
Monitoring rules for recipients and their operating characteristics were obtained using the R package 
‘clinfun’, using a 5% one-sided type I error rate across the sequential tests for each event.  These are 
described in Table 8.2.   
  
 
 
 
 
 
 
 
 
*Stopping guideline is triggered if ≥ x recipients out of n experience events described  
Table 8.2 Monitoring 
Guidelines among 
Recipients Receiving 
Allografts Successfully 
Mobilized by MGTA-145 + 
Plerixafor 
Graft Failure at day 28 
Number of recipients (n)  
2-9 
10-23 
24-28 
 
 
 
Stopping boundary (x)  
2 
3 
4 
 
 
 
TRM by day 100 
Number of recipients (n)  
4-8 
9-11 
12-15 
16-19 
20-24 
25-28 
Stopping boundary (x)  
4 
5 
6 
7 
8 
9 
aGVHD grade 3-4 
Number of recipients (n)  
3-6 
7-11 
12-17 
18-22 
23-28 
 
Stopping boundary (x)  
3 
4 
5 
6 
7 
 

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The graft failure pausing rule has a 5% chance of being triggered if the true graft failure rate is 3%, and 
an 85% chance of being triggered if the true graft failure rate is 18%. If the true graft failure rate is 18%, 
the stopping rule will be triggered on average after approximately 13 subjects are evaluable for the graft 
failure endpoint. Similarly, the TRM pausing rule has a 5% chance of being triggered if the true TRM rate 
is 15%, and an 89% chance of being triggered if the true TRM rate is 40%. If the true TRM rate is 40%, 
the stopping rule will be triggered on average after approximately 14 subjects are evaluable for the TRM 
endpoint. Finally, the grade 3-4 aGVHD pausing rule has a 5% chance of being triggered if the true rate 
is 10%, and an 83% chance of being triggered if the true rate is 30%. If the true grade 3-4 aGVHD rate is 
30%, the stopping rule will be triggered on average after approximately 15 subjects are evaluable for the 
grade 3-4 aGVHD endpoint.  
Triggering a safety monitoring rule does not mean discontinuation from the study for subjects currently 
enrolled, and remaining study procedures should be completed as indicated by the study protocol, 
especially for efficacy and safety endpoints, namely, to capture AE, SAE, and unanticipated problems 
(UPs). Refer to Appendix G and Appendix H for more information. If a clinically significant finding is 
identified after enrollment, the investigator or qualified designee will determine if any change in subject 
management is needed.  
8.3 Subject Discontinuation/Withdrawal from the Study 
Subjects are free to withdraw from participation in the study at any time upon request. If the subject 
wishes to withdraw, data collected up to that time point for inclusion in the study will be retained. If a 
subject withdraws prematurely after HSCT, all data normally collected at the end of study visit should be 
gathered at the time of premature discontinuation and reported in the forms in the Medidata Rave® EDC 
application.  
Off-study criteria: The subject is no longer followed. No study-specific evaluations, procedures, or tests 
are performed after date the subject was considered to have met off-study criteria. 
An investigator may discontinue or withdraw a subject from the study for the following reasons:  
• 
Concerns for recipient or donor safety 
• 
Noncompliance with study procedures or obligations 
• 
Required use of a prohibited medication 
• 
Transplant is cancelled, subject (recipient) reason 
• 
Transplant is cancelled (recipient), insufficient number of cells obtained 
• 
A subject meets an exclusion criterion (either newly developed or not previously recognized) that 
precludes further study participation.  
• 
The subject is lost to follow-up 
• 
Discontinued per medical discretion of the site Principal Investigator or Medical Monitor, such as if 
any clinical AE, laboratory abnormality, or other medical condition or situation occurs such that 
continued participation in the study would not be in the best interest of the subject  
• 
Subject withdraws consent 
• 
Death 
 
Off-protocol criteria: The subject is followed for clinical endpoint data capture only. No study-specific 
evaluations, procedures, or tests will be performed after date the subject was considered to have met off-
protocol criteria. 
• 
Subject received a subsequent HSCT and/or donor cellular infusion (DLI) 
• 
Subject experiences disease relapse 
 
The reason for subject discontinuation or withdrawal from the study will be recorded in the Medidata 
Rave® EDC application and documented in the medical record. All subjects who withdraw from the study 
with ongoing SAEs or AEs of at least possible relatedness must be followed until resolution of the event, 

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death, or until the site Principal Investigator concludes that the event is stable with no further 
improvement anticipated. 
Subjects who sign the informed consent form but do not receive the study medications may be replaced. 
Subjects who sign the informed consent form, and receive the study medications, and subsequently 
withdraw, or are withdrawn or discontinued from the study, will not be replaced. 
 
8.4 Lost to Follow-Up 
A subject will be considered lost to follow-up if the subject fails to be available for or return for a 
scheduled visit and is unable to be contacted by the study site staff.  Subjects lost to follow-up must be 
reported in the Medidata Rave® EDC application. 
The following actions must be taken if a subject fails to be available for a virtual visit or return to the clinic 
or laboratory for a required study visit:  
• 
The site will attempt to contact the subject and reschedule the missed visit and counsel the 
subject on the importance of maintaining the assigned visit schedule and ascertain if the subject 
wishes to and/or should continue in the study.  
• 
Before a subject is deemed lost-to-follow-up, the investigator or designee will make every effort to 
regain contact with the subject (where possible, 3 telephone calls and, if necessary, a certified 
letter to the subject’s last known mailing address or local equivalent methods).These contact 
attempts should be documented in the subject’s medical record or study file.   
• 
Should the subject continue to be unreachable, he or she will be considered to have withdrawn 
from the study with a primary reason of lost to follow-up. 
Post-collection donor survey assessments are administered centrally by the CIBMTR Survey Research 
Group. If the Survey Research Group is unable to contact a subject for a specific survey time point, after 
several phone and email contact attempts, they will code that survey assessment as Lost to Follow-Up. 
The subject will not be exited from the study and the Survey Research Group will continue attempting to 
reach the subject for subsequent survey time points. 
 
9 
 STUDY ASSESSMENTS AND PROCEDURES 
9.1  Donor Screening  
Within 30 days prior to start of stem cell mobilization with plerixafor and MGTA-145, the donor will 
undergo inclusion/exclusion criteria testing to include the following:  
• 
Written informed consent covering the complete study procedures must be obtained before any 
study procedure is performed.  
• 
Medical history review and collection of any current medications  
• 
Physical exam including vital signs (blood pressure, pulse, respiratory rate, and temperature), 
height, weight  
• 
KPS 
• 
Electrocardiogram  
• 
CBC with differential and platelet count  
• 
Serum Creatinine  
• 
Prothrombin Time (PT)/ Partial thromboplastin time (PTT)  
• 
Serum pregnancy for female donors of childbearing potential 
• 
Urinalysis (dipstick and microscopic examination) 
• 
Infectious disease markers as required by FDA  
• 
ABO/Rh Typing  
• 
Chimerism sample (e.g. VNTR or other method in use at treating institution)  
 

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9.1.1 
Unrelated Donors 
Be The Match will manage all aspects of volunteer unrelated donor participation on the study. Standard 
operating procedures for approaching and evaluating potential donors for HSC donation will be followed. 
Volunteer unrelated donors will participate in an information session with a Be The Match Donor Services 
representative via telephone regarding HSC donation and will be approached for participation in this 
study. If the donor is willing to proceed, the donor will participate in an informed consent session with a 
Be The Match Medical Services representative. Verbal consent will be obtained to proceed with the Be 
The Match donor eligibility evaluations. Study specific screening assessments will not start until the donor 
signs the study specific informed consent form and returns the consent to Be The Match, where the 
signed consent form will be retained. 
If the donor at any point does not meet the requirements for donation, they will be withdrawn from the 
study and the reason will be documented.  
Donors that are determined to be acceptable to continue participation will be scheduled for an HPC(A) 
collection at a study-qualified Be The Match apheresis center.   
 
9.2  Donor Follow-up  
Donors will be contacted for a virtual visit (with telephone backup if video is not available) by study site 
staff (clinic or apheresis center) for related donors, or by a Be The Match Donor Services representative 
for unrelated donors, at Week 1, Month 1, and Month 6. At the Week 1 visit, the Donors will be asked 
non-leading questions about whether any AEs have occurred and whether any new medications were 
required. At the Week 1, Month 1, and Month 6 visits. Donors will be asked whether any SAEs have 
occurred. Donors will also be instructed to report any important safety events throughout the study to the 
clinic where the donation procedure occurred. 
At Month 1, laboratory sample collections will be ordered by CIBMTR staff. Study sites will be responsible 
for communicating visit requirements with subjects. Donors will be given instruction by the study site on 
where to go to have samples collected. 
At Days 3-4, and Month 1, donor survey assessments will be conducted by the CIBMTR Survey 
Research Group staff, as described in Section 9.7.   
9.3  Recipient Screening  
Within 45 days of the start of the conditioning regimen, the subject will undergo inclusion/exclusion 
criteria testing to include the following:  
• 
Written informed consent covering the complete study procedures must be obtained before any 
study procedure is performed. In order for the recipient to continue on the study, the intended 
donor will have to consent as well (see Section 9.1). 
• 
Medical history review and collection of any current medications 
• 
Physical exam including vital signs (blood pressure, pulse, respiratory rate, and temperature) 
• 
Height and Weight 
• 
KPS 
• 
CBC with differential and platelet count  
• 
Blood chemistries to assess renal/hepatic function per local institutional guidelines, including 
serum creatine, AST, ALT, LDH and total bilirubin 
• 
Serum pregnancy for female subjects of childbearing potential  
• 
Urinalysis  
• 
Assessment of HCT-CI score  
• 
Disease specific staging and assessment  
• 
Bone marrow assessment if not done within 45 days prior to the date of consent 
 

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The following tests are required but may be performed earlier than 45 days prior to the start of the 
conditioning regimen:  
• 
Infectious disease markers as required by FDA 
• 
ABO/Rh Typing  
• 
Chimerism sample (e.g. VNTR or other method in use at treating institution)  
• 
Most recent echocardiogram or MUGA results obtained via standard of care 
• 
Most recent DLCO results obtained via standard of care 
 
9.4  Recipient Follow-up  
The following assessments should be made at the time points listed. The study visits reflect standard 
follow-up for an allogeneic HSCT subject.  
Day + 1 through neutrophil recovery or Day + 21 (whichever is first):  
• 
Daily physical exam with vital signs (blood pressure, pulse, respiratory rate, and temperature)  
• 
CBC with differential and platelet count: daily until neutrophil recovery occurs (neutrophil 
recovery is defined in Section 9.6.6) Note: in the event that neutrophil recovery occurs but not 
platelet recovery, a CBC should continue to be obtained daily until platelet count recovery or 
Day + 21 (whichever is first) 
• 
Blood chemistries (serum creatinine, AST, ALT, LDH, total bilirubin) twice weekly  
• 
Daily acute GVHD assessment  
• 
Recipients will receive G-CSF 5 μg/kg/day subcutaneously starting day +7 until ANC > 1.5 x 
109/L for 2 consecutive days 
• 
Daily assessment for AEs  
Note: Only AEs with relatedness of possible, probable, or definite need to be entered in 
Medidata Rave® EDC application  
 
Day 28 (± 3 days):  
• 
Physical exam including vital signs (blood pressure, pulse, respiratory rate, and temperature) 
• 
CBC with differential and platelet count  
Note: If platelet and/or neutrophil recovery has not occurred at this time, continue testing 
three times weekly until recovery  
• 
Blood chemistries (serum creatinine, AST, ALT, LDH, total bilirubin)  
• 
Chimerism sample  
• 
Immune reconstitution sample 
• 
Assessment for adverse events  
• 
Acute GVHD assessment  
• 
Note: Only AEs with relatedness of possible, probable, or definite need to be entered in 
Medidata Rave® EDC application 
 
Day 56 (± 7 days): 
• 
Physical exam including vital signs (blood pressure, pulse, respiratory rate, and temperature) 
• 
CBC with differential and platelet count  
Note: If platelet and /or neutrophil recovery has not occurred, continue testing two times 
weekly until recovery  
• 
Blood chemistries (serum creatinine, AST, ALT, LDH, total bilirubin)  
• 
Acute GVHD assessment 
• 
Assessment for adverse events  
Note: Only AEs with relatedness of possible, probable, or definite need to be entered in 
Medidata Rave® EDC application 
 

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Day 100 (± 7 days):   
• 
Physical exam with vital signs (blood pressure, pulse, respiratory rate, and temperature) 
• 
CBC with differential and platelet count  
Note: If platelet and /or neutrophil recovery has not occurred, continue testing two times 
weekly until recovery  
• 
Blood chemistries (serum creatinine, AST, ALT, LDH, total bilirubin)  
• 
Acute and/or chronic GVHD assessment  
• 
Chimerism sample 
• 
Immune reconstitution sample 
• 
Assessment for adverse events  
Note: Only AEs with relatedness of possible, probable, or definite need to be entered in 
Medidata Rave® EDC application 
 
Day 180 (± 14 days):   
• 
Physical Exam including vital signs (blood pressure, pulse, respiratory rate, and temperature) 
• 
KPS 
• 
CBC and differential with platelet count  
• 
Blood chemistries (serum creatinine, AST, ALT, LDH, total bilirubin)  
• 
Acute and/or chronic GVHD assessment  
• 
Chimerism sample 
• 
Immune reconstitution sample 
• 
Assessment for adverse events  
Note: Only AEs with relatedness of possible, probable, or definite need to be entered in 
Medidata Rave® EDC application 
 
 Day 365 (± 14 days):  
• 
Physical Exam including vital signs (blood pressure, pulse, respiratory rate, and temperature) 
• 
KPS 
• 
CBC and differential with platelet count  
• 
Blood chemistries (serum creatinine, AST, ALT, LDH, total bilirubin)  
• 
Acute and/or chronic GVHD assessment  
• 
Chimerism sample 
• 
Immune reconstitution sample 
• 
Disease appropriate staging reevaluation is recommended  
• 
Bone marrow aspirate and biopsy required if leukocyte count is outside of the normal 
laboratory range at the transplant center and/or platelet count is < 100 x 109/L 
• 
Assessment for adverse events  
Note: Only AEs with relatedness of possible, probable, or definite need to be entered in 
Medidata Rave® EDC application  
 
9.5 Criteria for engraftment failure and transplantation of second stem cell allograft  
If any recipient receiving the MGTA-145 + plerixafor mobilized allograft does not have evidence of an 
ANC of at least 0.5 x 109/L by Day + 28 following myeloablative conditioning and transplantation, plans 
for a second allograft should be made (e.g., G-CSF mobilized allograft, bone marrow harvest) unless 
there is a clear reason for failure to achieve an ANC > 0.5 x 109/L such as CMV viremia, an active 
bacterial, other viral, or fungal infection, or the concomitant use of myelosuppressive drugs believed to be 
a likely cause of the neutropenia. If these plans are being made, the Medical Monitor should be notified. 

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9.6 Definitions for Safety, Efficacy and PK Assessments  
9.6.1 
Primary Endpoint: Clinically adequate allograft within one apheresis collection 
For the purposes of this study, a clinically adequate allograft is defined as a graft that contains ≥ 2.0 x 
106 CD34+ cells/kg recipient weight collected. The primary endpoint is defined as achieving this clinically 
adequate allograft in one apheresis session. 
9.6.2 
Secondary Endpoint: Clinically desirable allograft in one apheresis collection 
This secondary endpoint is defined as achieving a graft that contains ≥ 4.0 x 106 CD34+ cells/kg recipient 
weight collected in one apheresis session.  
9.6.3 
Secondary Endpoint: Clinically adequate allograft overall 
This secondary endpoint is defined as achieving a graft that contains ≥ 2.0 x 106 CD34+ cells/kg recipient 
weight collected across all apheresis sessions.   
9.6.4 
Secondary Endpoint: Assessment of AE in Donors  
Assessment of AE in donors will be done during the infusion and 30 minutes (±10 minutes), 60 minutes 
(±10 minutes) after the administration of plerixafor and 30 minutes (±10 minutes), 60 minutes (±10 
minutes, 120 minutes (±20 minutes), and 240 minutes (±20 minutes) following start of MGTA-145 
infusion. In addition to monitoring the donor on the days of collection, donors will be contacted at Week 
1, Month 1, and Month 6 to assess the donor’s medical condition.  
Back pain is an AE of special interest, and information on the date and time of onset, date and time of 
resolution, concomitant medications and supportive care used to treat the back pain, action taken with 
study drug, peak severity of the pain on a 0-10 numeric rating scale, relationship to study drug will be 
assessed and documented in Medidata Rave® EDC application. 
9.6.5 
Secondary Endpoint: Assessment of AEs related to Allograft  
Assessment of AEs related to the allograft will include measurement of the recipient’s blood pressure, 
pulse, respiration rate and temperature one hour prior to the allograft infusion and then approximately 15 
minutes, 30 minutes, 1 hour, 2 hours, and 4 hours post infusion.  
9.6.6 
Secondary Endpoint: Neutrophil Recovery  
Time to neutrophil recovery is measured by determining the first of 3 consecutive daily measurements of 
neutrophil count ≥ 0.5 x109/L following conditioning regimen induced nadir. Although unlikely, recipients 
who do not experience a drop in the neutrophil count < 0.5 x109/L will not be evaluable for neutrophil 
recovery but will be evaluable for graft failure based on the degree of donor cell chimerism at Day +28.  
9.6.7 
Secondary Endpoint: Platelet Recovery  
Platelet recovery is defined as the first day when the platelet count is ≥ 20 × 109/L measured by at least 
3 consecutive laboratory values that show that level was achieved and maintained. The recipient should 
not have any platelet transfusions in the 7 days prior to the date selected for achieving ≥20 × 109/L and 
the date should be the first of 3 consecutive lab values tested on different days.  
9.6.8 
Secondary Endpoint: Graft Failure  
Primary graft failure is defined as failure to experience neutrophil recovery by Day +28 following a 
conditioning regimen induced neutrophil nadir < 0.5 x109/L. For recipients not experiencing a fall in the 
neutrophil count to < 0.5 x109/L following conditioning, graft failure will be defined as < 5% donor cell 
chimerism in the myeloid and CD3+ cell compartments by Day +28. Secondary graft failure will be 
defined as a fall in the neutrophil count after primary engraftment to < 0.5 x109/L sustained for more than 
three days that cannot be attributed to other causes such as drugs, infection, GVHD, etc., is not 

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responsive to G-CSF or GM-CSF, and is associated with a fall in donor chimerism to < 5%. Fall in donor 
chimerism to less than 5% following primary engraftment will also be considered secondary graft failure 
even in the absence of a fall in the peripheral blood counts. 
9.6.9 
Secondary Endpoint: Acute GVHD  
Mount Sinai Acute GVHD International Consortium (MAGIC) clinical criteria (see Appendix C) and 
histological grading of skin, liver or gastrointestinal pathology and confidence levels where possible will 
be used to establish and grade acute GVHD. In the first 100 days after transplantation recipients will be 
assessed by a transplant physician or designee (e.g. mid-level provider) for the development of acute 
GVHD approximately weekly.  
9.6.10 
Secondary Endpoint: Chronic GVHD  
Chronic GVHD will be diagnosed and graded according to the NIH consensus criteria (Appendix D) and 
treated with standard or experimental immunosuppressive therapy as deemed appropriate by the 
transplant center. Recipients will be assessed for chronic GVHD at Days 100, 180, and 365, and more 
frequently if clinically indicated.  
9.6.11 
Secondary Endpoint: Treatment-Related Mortality (TRM)  
TRM is defined as death in recipients without relapse or progression of their disease. Non-medical, 
accidental causes of death, e.g. natural disasters, are not considered TRM.  
9.6.12 
Secondary Endpoint: Overall Survival  
Survival will be measured by assessing if the recipient remains alive by visual observation or telephone 
call. For recipients lost to follow-up, public means of assessing survival may be employed such as the 
National Death Index.  
9.6.13 
Secondary Endpoint: Determination of Relapse or Disease Progression  
Disease relapse occurs in subjects who entered HSCT in complete remission (CR); progression occurs 
in those with existing disease at transplant who meet criteria for progressive disease post-HSCT. A 
recipient will be considered relapsed when there is a recurrence of the original malignant disease after 
transplantation. This will be determined locally by tests and procedures in place at each participating 
institution. Date of relapse/progression is defined as the date at which the first observation of 
hematologic, radiographic, or cytogenetic changes which herald progression/relapse is made. See 
Appendix E for definitions.  
9.6.14 
Secondary Endpoint: Progression-Free Survival 
Death or relapse/disease progression will be an event, and the time to the event will be determined as 
the time from transplant to the first event.  Patients alive and relapse/progression-free will be censored at 
last contact.   
9.6.15 
Exploratory Endpoint: Graft composition 
Graft composition will be defined phenotypically by molecular cell markers to stratify leukocyte subsets. 
9.6.16 
Exploratory Endpoint: Colony Formation Assay 
Colony formation assay is defined as number of functional progenitor cells that form colonies in 
methylcellulose. 
9.6.17 
Exploratory Endpoint: Chimerism  
Chimerism is defined as leukocyte subset percentages that are donor vs. recipient over time. 

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9.6.18 
Exploratory Endpoint: Immune Reconstitution 
Immune reconstitution is defined as counts of leukocyte subsets over time, with respect to normal ranges. 
9.6.19 
Exploratory Endpoint: CMV Reactivation  
CMV reactivation will be defined as a positive test for CMV viremia as determined by an antigenemia 
assay or quantitative PCR (whichever is in use at each individual institution) that results in the 
administration of antiviral treatment directed against CMV.  
9.6.20 
Exploratory Endpoint: MGTA-145 PK 
A validated assay will be used to determine MGTA-145 plasma concentrations. The exact date and time 
(hours and minutes) of the sampling time and infusion start and end times will be recorded. 
9.6.21 
Exploratory Endpoint: Immunogenicity  
A validated assay to determine the formation of ADA will be used. 
9.6.22 
Exploratory Endpoint: Donor Experience  
Donor survey assessments are described in more detail in Section 9.7. 
9.7 Donor Survey Assessments 
The CIBMTR Survey Research Group (SRG) centrally administers all donor survey assessments. At the 
time a donor consents to participate in the study, the SRG is notified and then adds that subject to 
CIBMTR’s electronic Patient Reported Outcomes (ePRO) system for data collection tracking. Contact 
information for unrelated donor subjects is accessed in NMDP databases. Contact information for related 
donor subjects will be obtained from transplant centers and maintained in a confidential manner. 
 
The SRG will contact the donor by phone to confirm contact information, remind them of survey 
assessment time points and confirm how they want to receive surveys going forward (paper or 
electronic). The same method of survey collection (paper or electronic) will be used for each Donor 
throughout the study. 
 
Up to 14 days prior to Day 1, the SRG will contact the donor to administer the baseline survey 
assessments.  
 
For the Day 1 donor survey assessments, donors will be asked to complete the surveys anytime from 60 
minutes after the apheresis process is completed until the end of that day (e.g., midnight). Unique links 
and an email template for electronic survey administration and PDFs of paper versions of the survey will 
be provided by SRG to HSC collection centers, if needed. Day 1 survey assessments will be repeated if 
there is a second day of collection. HSC collection center staff will securely email or fax completed paper 
surveys to SRG for data entry, as applicable.  
 
The SRG will contact the donor via email, phone and/or mail to collect donor survey assessments at Day 
3-4 and Month 1. The SRG will follow-up with non-responders to minimize missing surveys.  
 
 
 

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Table 9.7: Schedule of Donor Survey Assessments 
Survey1 
Items Minutes to 
complete 
Baseline 
Day -14 to Day 
0 
Post-treatment 
Day 12, 3 
Day 3-44 
Month 1 
Day 28 (+/-7) 
Brief Pain Inventory 
15 
5 
X 
X 
X 
X 
PROMIS Fatigue 
8 
1-2 
X 
X 
X 
X 
PROMIS 
(Gastrointestinal, 
Diarrhea) 
6 
1-2 
X 
X 
X 
X 
PROMIS Phys. Function 
10 
1-2 
X 
X 
X 
X 
PROMIS Anxiety 
4 
1-2 
X 
X 
 
X 
PROMIS Depression 
4 
1-2 
X 
X 
 
X 
PROMIS Sleep 
disturbance 
4 
1-2 
X 
X 
X 
X 
EQ-5D 
5 
1 
X 
X 
X 
X 
Work Productivity and 
Activity Impairment 
6 
1 
 
 
X 
 
Healthcare Resource 
Use (HRU) utilization 
6 
1-2 
 
 
X 
 
Donor Experience 
6 
1-2 
 
 
 
X 
Total items 
 
 
46-86 
46-86 
50-74 
52-92 
Total minutes 
 
 
12-18 
12-18 
13-17 
14-21 
1- To be completed in the order listed  
2- Repeat if 2 days of mobilization and donation 
3- To be completed from 60 minutes after apheresis is completed until the end of that day (e.g., midnight) 
4- 72-96 hours after last donation 
9.7.1 
Brief Pain Inventory 
The 15-item Brief Pain Inventory (BPI) assesses severity of pain and its impact on functioning. The BPI 
will be included on the donor assessment survey at baseline, post-treatment (Day 1), Day 3-4, and 
Day 28.  
9.7.2 
PROMIS domains 
Six PROMIS domains – Fatigue, Gastrointestinal (Diarrhea), Sleep disturbance, Physical function, 
Anxiety and Depression - will be used to measure detailed functioning and symptom burden for donors. 
They will be evaluated at Baseline, post-treatment (Day 1), Day 3-4 and Day 28. Note: Anxiety and 
Depression will not be collected at Day 3-4. 
 
When delivered on paper, the domains will be delivered in 8-item (Fatigue), 10-item (Physical Function), 
6-item (Gastrointestinal), and 4-item (Anxiety, Depression, Sleep disturbance) short forms. When 
delivered electronically, most domains will be delivered as Computer Adaptive Tests (CAT), in which the 
questions a person answers are tailored to that person. Each response is used to further refine the 
questions a participant receives, and thus the participant’s score, for that domain. The PROMIS CAT item 
banks for a domain typically involve 4-12 items. The first item administered is usually in the middle of the 
range of function or severity for that domain. After a participant responds, an estimated score is 
calculated. The PROMIS CAT algorithm then selects the best item in the item bank for refining the 
estimated score and recalculates the participant’s score as they continue responding. The PROMIS CAT 
continues to administer items until a specified level of measurement precision is reached, or the 
maximum number of 12 items per measure have been administered. Studies have shown that the 
average number of items delivered in a CAT domain is 5-8.55 The Gastrointestinal domain is not available 
in CAT format. 
 

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The Gastrointestinal, Fatigue and Sleep Disturbance domains have a 7-day recall period. At the 3-4 day 
post-treatment time point, these domains will be delivered as short forms, and modified to a 24-hour 
recall period. 
9.7.3 
EQ-5D 
The EQ-5D is a 5-domain questionnaire measuring mobility, self-care, usual activities, pain/discomfort 
and anxiety/depression. This questionnaire will be used to measure donors’ overall quality of life at 
baseline, post-treatment (Day 1), Day 3-4 and Day 28. 
9.7.4 
Work Productivity and Activity Impairment (WPAI) 
The 6-item Work Productivity and Activity Impairment (WPAI) Questionnaire measures work productivity 
and activity impairment during a 7-day recall period. It yields four scores: absenteeism (proportion of work 
hours missed), presenteeism (degree that health is affected productivity while working), overall work 
impairment (linear combination of absenteeism and presenteeism), and activity impairment (degree to 
which health is affected regular activities). The WPAI will be collected at Day 3-4.  
9.7.5 
Healthcare Resource Use (HRU) 
A donor survey assessment will include a questionnaire of healthcare resource use at Day 3-4 to capture 
healthcare resources utilized.  
9.7.6 
Donor Experience 
A donor experience questionnaire that asks about time and travel burden and overall satisfaction with the 
donation experience will be collected at Day 28. This questionnaire will be used to understand travel 
burden and donation satisfaction.  
 
9.8 Adverse Events and Serious Adverse Events 
9.8.1 
Definition of Adverse Events (AE) 
An adverse event (AE) is any untoward medical occurrence regardless of causality assessment. An 
adverse event can be an unfavorable and unintended sign (including an abnormal laboratory finding), 
symptom, syndrome or disease associated with or occurring during the use of an investigational product 
whether or not considered related to the investigational product. 
 
Medical conditions present at screening (i.e., before the study treatment is administered) are not AEs. 
These medical conditions should be adequately documented on the subject study chart and in Medidata 
Rave® EDC application as Medical History. However, medical conditions present at baseline that worsen 
in intensity or frequency during the treatment or post treatment periods should be considered and 
recorded as AEs in the Medidata Rave® EDC application. 
 
For Donors, all AEs and SAEs will be collected beginning from the signing of the informed consent form. 
AEs will then be collected through 72 hours post infusion of MGTA-145 and SAEs will be collected 
through the 6-month follow-up time point as outlined in the schedule of assessments. 
 
For Recipients, all SAEs and all related AEs, will be collected from Day 0 through the 1-year follow-up 
time point as outlined in the schedule of assessments. 
 
 

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9.8.2 
Definition of Serious Adverse Events (SAE) 
A serious adverse event (SAE) is any medical occurrence at any dose that meets the following criteria:   
1. Death.  
2. Life-threatening situation. 
3. In patient hospitalization or prolongation of existing hospitalization.  
4. Persistent or significant disability/incapacity or substantial disruption of the ability to conduct 
normal life functions. 
5. Congenital anomaly/birth defect. 
6. A significant medical condition which, without urgent medical intervention, would lead to one 
of the above outcomes.  
 
Clarification on SAEs:  
• 
All deaths, regardless of cause, must be reported for subjects on study and for deaths occurring 
within 30 days of last study evaluation, whichever is longer.  
• 
“Immediately life-threatening” means that the subject was at immediate risk of death from the 
event as it occurred. This does not include an event that might have led to death, if it had 
occurred with greater severity.  
• 
Complications that occur during hospitalizations are AEs. If a complication prolongs 
hospitalization, it is an SAE. Note that the hospital prolongation should be clearly and directly 
attributable to the event.  
• 
“Inpatient hospitalization” means the subject has been formally admitted to a hospital for medical 
reasons, for any length of time. This may or may not be overnight. This does not refer to 
evaluation in an Emergency Department without admission to the hospital. This does not include 
planned inpatient hospitalizations for transplant or other elective procedures.  
• 
Subjects undergoing HSCT are frequently hospitalized after the initial transplant hospitalization. 
Subsequent hospitalizations for reasons that meet criteria for SAEs should be reported as an 
SAE.  
 
The investigator should attempt to establish a diagnosis of the event based on signs, symptoms and/or 
other clinical information. In such cases, the diagnosis should be documented as the AE and/or SAE and 
not the individual signs/symptoms. 
9.8.3 
Classification of an Adverse Event 
The site Principal Investigator will assess each AE for duration, severity, seriousness, and relatedness of 
the event to the investigational products. 
9.8.3.1 
Severity of Event 
AEs will be reported using event terms and grading for severity per the CTCAEv5.0. The CTCAE includes 
a grading (severity) scale for each AE term:  
 
Grade    
• 
0 – No AE or within normal limits  
• 
1 – Mild AE  
• 
2 – Moderate AE 
• 
3 – Severe AE  
• 
4 – Life-threatening or disabling AE  
• 
5 – Fatal AE 
 

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9.8.3.2 
Relationship to Study Product 
Attribution of the event to the investigational product may be characterized as follows:  
• 
Definite – The AE is clearly related to the study procedure/treatment(s).  
• 
Probable – The AE is likely related to the study procedure/treatment(s). 
o 
The AE is not likely to be caused by the subject’s underlying medical condition or other 
concomitant therapy, and the nature of the AE or the temporal relationship between the 
onset of the AE and study procedure/treatment administration lead the investigator to 
believe that there is a reasonable chance of causal relationship. 
• 
Possible – The AE may be related to the study procedure/treatment(s). 
o 
The AE could be attributed to the subject’s underlying medical condition or other 
concomitant therapy, but the nature of the AE or the temporal relationship between the 
onset of the AE and study procedure/treatment administration lead the investigator to 
believe that there could be a causal relationship.  
• 
Unlikely – The AE is doubtfully related to the study procedure/treatment(s).  
• 
Unrelated – The AE is clearly NOT related to the study procedure/treatment(s). 
o 
The AE is most plausibly explained by the subject’s underlying medical condition or 
other concomitant therapy, or the AE has no plausible biological relationship to study 
procedure/treatment.  
 
For reporting purposes, an AE should be regarded as definitely or probably related to the regimen if the 
investigator believes that at least one of following criteria are met:  
 
• 
There is a clinically plausible time sequence between onset of the AE and the administration of 
the study treatment;  
• 
There is a biologically plausible mechanism for the treatment causing or contributing to the AE;  
• 
The AE cannot be attributed solely to concurrent/underlying illness, other drugs, or procedures 
9.8.3.3 
Expectedness 
• 
Expected Adverse Events are those listed in the Reference Safety Information of the 
Investigators Brochure for MGTA-145 or in the Package Insert for Plerixafor.  
• 
Unexpected Adverse Events are those events the nature of which, severity, or frequency are not 
consistent with the known or foreseeable risk of AEs associated with the research procedures 
described in the protocol-related documents. Adverse events that are reflective of the patient’s 
pre-existing condition need not be reported.  
9.8.4 
Time Period and Frequency for Adverse Event Assessment and Follow-Up 
Safety events for donors and recipients will be reported and collected as indicated in Table 9.8.    
 
 
 

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Table 9.8: Reporting Adverse Events 
Subject 
Category 
Severity 
Grade 
Relatedness 
Reporting 
Timelines 
Study Site Reporting 
Requirement 
Donor 
SAE 
All grades 
All 
attributions 
From time of 
informed consent 
through 6-months 
post mobilization 
Within 24 hours of learning 
of the event; Site is to report 
to Sponsor using SAE form1 
AE 
≥ Grade 3 AEs 
All 
attributions 
From time of 
informed consent 
through 72 hours 
post mobilization 
Within 24 hours of learning 
of the event; Site is to enter 
AE in Medidata Rave® EDC 
application  
AE 
Grade 1, 
Grade 2 
All 
attributions 
From time of 
informed consent 
through 72 hours 
post mobilization 
Site is to enter AE in 
Medidata Rave® EDC 
application within standard 
timeline 
Recipient 
SAE 
All grades 
All 
attributions 
From Day 0 through 
1-year 
post-transplant 
Within 24 hours of learning 
of the event; Site is to report 
to Sponsor using SAE form1 
AE 
All grades 
Possible, 
Probable, or 
Definite2 
From Day 0 through 
1-year 
post-transplant 
Site is to enter AE in 
Medidata Rave® EDC 
application within standard 
timeline 
1Submit death summaries and/or autopsy reports when SAE is Fatal along with the SAE form, which should include potential contributing causes 
of death; AE form should include potential contributing causes of death. Study site must also complete all follow up forms through date of death, 
and study exit form. 
2AEs that are attributed as Unlikely or Unrelated to study product do not need to be reported.  
All treatment related SAEs or AEs and Adverse Events of Special Interest (AESIs) should be followed 
until resolution of the event, death, or until the investigator concludes that the event is stable with no 
further improvement anticipated.  Any SAE occurring after the reporting period must be promptly reported 
if a causal relationship to the study drugs is suspected.   
The CIBMTR Medical Monitor will be notified by the Medidata Rave® EDC application if any CTCAE 
grade 3 or greater AE believed to be at least possibly related to plerixafor or MGTA-145 is observed in 
any of the donors. 
The CIBMTR Medical Monitor will review reported AEs, GVHD, infection, and primary graft failure events 
on a weekly basis to assess whether any safety monitoring rules have been met that should be referred 
to the DSMB (See Section 8.2). 
9.8.5 
Adverse Event Reporting 
AEs occurring in donors and recipients will be reported using event terms and grading for severity per the 
CTCAE v5.0 (See Appendix G).  
All SAEs, regardless of relationship to study treatment, must be reported to the Sponsor within 24 hours 
of the Investigator becoming aware of the event.  Initial and Follow up SAE notification should be made 
by emailing or faxing the SAE report form to the email or fax number provided on the SAE report form. 
The Sponsor is responsible for submitting reports of SAEs associated with the use of the study drugs to 
the appropriate Regulatory Authority (i.e., US Food and Drug Administration) and Investigators in 
accordance with applicable regulations and guidelines.   
It is the responsibility of the Investigator to notify the IRB of all SAEs that occur at his or her site.  
Investigators will be notified of all Suspected Unexpected Serious Adverse Reactions (SUSARs 7/15 Day 
Safety Reports) that occur during any clinical studies that are using the investigative compound.  Each 
site is responsible for notifying their IRB of these additional SUSARs in accordance with local regulations. 

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9.8.6 
Adverse Events of Special Interest 
An Adverse Event of Special Interest (AESI) is a category of adverse event specific to this trial. Back 
pain occurring within 24 hours of administration of MGTA-145, regardless of severity or relationship to 
study drug, will be captured as an AESI. The location, severity based on CTCAE v5.0, time of onset, 
duration of back pain, relationship to study drug, action taken with study drug, outcome, and any 
treatment administered will be captured in the Medidata Rave® EDC application. 
9.8.7 
Reporting of Pregnancy 
All donors must agree to an effective means of birth control while on study treatment and for at least 3 
months following plerixafor and MGTA-145 treatment (including both female subjects of childbearing 
potential and male subjects with partners of childbearing potential). All recipients must agree to an 
effective means of birth control for at least 3 months following PBSC infusion (including both female 
subjects of childbearing potential and male subjects with partners of childbearing potential).  
Female subjects of childbearing potential are defined as women who have not undergone hysterectomy 
or bilateral oophorectomy or are not naturally postmenopausal (defined as ≥ 12 consecutive months 
without menstrual bleeding). Effective birth control includes: (a) vasectomy or vasectomized partner (b) 
hormonal contraception (for example, birth control pills, intravaginal birth control, transdermal birth control 
injectable birth control or implantable birth control) or an intrauterine device plus one barrier method; or 
(c) two barrier methods. Effective barrier methods are male and female condoms, diaphragms, and 
spermicides (creams or gels that contain a chemical to kill sperm). All male subjects will refrain from 
sperm donation from the time of signing this consent form until 3 months after treatment with MGTA-145 
and plerixafor (Donors) or until 3 months after PBSC infusion (Recipients). For subjects using a hormonal 
contraceptive method, information about any interaction of plerixafor or MGTA-145 with hormonal 
contraceptives is not known.  
  
During the study, all women of childbearing potential and female partners of male subjects should be 
instructed to contact the Investigator immediately if they suspect they might be pregnant (e.g. missed or 
late menstrual cycle). If a pregnancy occurs, the pregnant woman should be referred to an obstetrician/ 
gynecologist experienced in reproductive toxicity for further evaluation and counseling.   
 
The Pregnancy must be reported to the Sponsor within 24 hours of the Investigator’s knowledge of the 
pregnancy using a Pregnancy Report Form.   
 
The Investigator will follow the pregnant woman until completion of the pregnancy and must notify the 
Sponsor of the outcome within 24 hours of the Investigator’s knowledge of the pregnancy outcome using 
a Pregnancy Outcome Form. This notification includes pregnancies resulting in live, “normal” births.  
  
 

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10 LABORATORY ASSESSEMENTS 
The following tables list the laboratory tests that will be performed for the donors and recipients on the 
study. 
Table 10.1 (Donor) 
HEMATOLOGY1 
CHEMISTRY1 
Hemoglobin 
Hematocrit  
Erythrocyte Count (Red Blood Cells [RBC]) 
Mean Cell Volume 
Leukocytes (White Blood Cells [WBC])  
Absolute Counts Of:  
• 
Neutrophils  
• 
Lymphocytes  
• 
Monocytes  
• 
Eosinophils  
• 
Basophils  
• 
Platelets 
Serum Creatinine 
 
OTHER1 
URINALYSIS1 
PT/PTT 
Microscopic evaluation  
Serum pregnancy test 
 
Infectious disease markers  
EXPLORATORY2 
ABO/Rh Typing 
PK  
Chimerism analysis   
ADA  
Flow cytometric analysis for CD34+ cells3 
Colony Forming Unit 
 
Graft Immunophenotyping  
1- Samples sent to local laboratory for analysis; the hematology samples collected at Month 1 may be analyzed at a laboratory  
coordinated by CIBMTR staff   
2- Samples sent to central laboratory for analysis 
3- Samples sent to local laboratory and central laboratory for analysis 
 
Table 10.2 (Recipient) 
HEMATOLOGY1 
CHEMISTRY1 
Hemoglobin 
Hematocrit  
Erythrocyte Count (Red Blood Cells [RBC]) 
Mean Cell Volume 
Leukocytes (White Blood Cells [WBC])  
Absolute Counts Of:  
• 
Neutrophils  
• 
Lymphocytes  
• 
Monocytes  
• 
Eosinophils  
• 
Basophils  
• 
Platelets 
Leukoblasts 
Serum Creatinine 
Aspartate Aminotransferase (AST) 
Alanine Aminotransferase (ALT) 
Lactate Dehydrogenase (LDH)  
Total Bilirubin  
 
 
OTHER1 
URINALYSIS1 
Serum pregnancy test 
Microscopic evaluation  
Infectious disease markers  
 
ABO/Rh Typing 
EXPLORATORY2 
Chimerism analysis   
Immune Reconstitution 
1- Samples sent to local laboratory for analysis 
2- Samples sent to central laboratory for analysis 
 

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11 STATISTICAL CONSIDERATIONS 
11.1  Statistical Hypotheses 
The study is designed as a Phase II, multicenter, prospective trial to evaluate the safety and efficacy of 
subcutaneous plerixafor and MGTA-145 for the mobilization and transplantation of HLA-matched sibling 
and matched unrelated donor allografts in recipients with hematological malignancies.  This study is 
designed as a Simon minimax two-stage trial.56 
  
The target enrollment is 28 donors receiving at least one dose of the combination regimen of MGTA-145 
+ plerixafor. The number of recipients may vary if the mobilization is unsuccessful in some donors.   
1. Accrual  
It is estimated that 18 months of accrual will be necessary to enroll the targeted sample size. The trial will 
remain open until the last recipient enrolled has been followed for one year.  
2. Randomization  
There will be no randomization in this study.  At enrollment, each center will declare whether or not a 
recipient is intended to receive a myeloablative regimen.  
3. Primary Endpoint  
The primary endpoint of this study is the collection of sufficient CD34+ cells using MGTA-145 + plerixafor 
as the mobilizing agent. A donor is considered successful for this endpoint if ≥ 2.0 x 106 CD34+ cells/kg 
recipient weight are collected in one leukapheresis collection using MGTA-145 + plerixafor.  
11.2   Sample Size Determination 
The sample size for this study is 28 donors receiving at least one dose of the combination regimen of 
MGTA-145 + plerixafor. It is expected that 90% of donors treated with MGTA-145 + plerixafor will 
mobilize ≥ 2.0 x 106 CD34+ cells/kg within one leukapheresis collection.  MGTA-145 + plerixafor is 
considered an unacceptable mobilizing agent if fewer than 70% of donors achieve this cell-dose 
threshold within one leukapheresis collection.  
  
Using a two-stage Simon minimax design, assuming the true successful collection probability of 90%, 28 
donors provide 85% power to reject the probability of successful collection of 70% or lower using a one-
sided Type I error α=0.05. This is equivalent to testing the null hypothesis H0: p ≤ 0.70 against the 
alternative hypothesis H1: p > 0.70 with 85% power to contrast a successful collection probability of 90% 
vs. 70%.  
  
In this design, 13 donors are accrued in the first stage. If 9 or fewer donors mobilize sufficient cells within 
one leukapheresis collection, the study will be terminated due to inadequate response. Otherwise, accrue 
15 additional donors in stage 2. At the end of this stage, if at least 24 out of 28 donors successfully 
mobilize ≥ 2.0 x 106 CD34+ cells/kg within one leukapheresis collections, it will be concluded that MGTA-
145 + plerixafor is effective and can be recommended for further investigation.  
  
 
 

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Table 11.2 shows the power to reject successful collection probability of 70% or lower for other plausible 
true probability of successful collection using the same design.  
 
True probability of successful 
collection (p1)  
Power  
0.85  
 58% 
0.90  
 85% 
0.95  
 99% 
 
11.2.1 
 
Planned Interim Analyses 
There will be no interim analysis for efficacy. Interim analyses for futility will be induced by the two-stage 
design as described in Section 11.2. Safety monitoring is described in Section 8.2. 
 
11.3  Populations for Analyses 
• 
Donor treated Population: Includes all donors receiving any exposure to both parts of the 
combination regimen of MGTA-145 + plerixafor. This population will be used for the primary donor 
efficacy analysis and for safety analyses of the donor. 
• 
Donor Per Protocol Population: Includes all donors receiving both products of the combination 
regimen at the right doses and who meet the requirement of the apheresis volume collected. 
Donors who have major protocol deviations that impact the primary or secondary objectives of the 
study will be excluded from this population. This population will be used for a per protocol analysis 
of the donor outcomes. 
• 
Plerixafor-only Population: Includes all donors receiving exposure to plerixafor and did not receive 
any MGTA-145. This population will be followed for safety purposes. 
• 
Recipient Analysis Population: Includes all enrolled subjects receiving a MGTA-145 + plerixafor-
mobilized allograft. This will be used for the primary analysis of the recipient outcomes 
• 
Recipient Eligible Analysis Population: Includes all subjects receiving a MGTA-145 + plerixafor-
mobilized allograft who continue to meet eligibility requirements at the time of infusion. This will be 
used for a sensitivity analysis of the recipient outcomes, in the event that more than 10% of the 
subjects has changed eligibility between enrollment and transplant so that they would no longer 
be eligible if reassessed.   
11.4   Statistical Analyses 
11.4.1 
General Approach 
Counts and percentages will be used to describe categorical variables, while the number of subjects (N), 
median, mean, standard deviation, and range will be used to summarize continuous variables. Missing 
data will not be imputed due to the small sample size of this study.  Unless otherwise specified, 
confidence intervals will be done using 95% confidence level, and hypothesis testing will be done using a 
two-sided 5% significance level. All individual data will be presented in listings.  
11.4.2 
Baseline Descriptive Statistics 
Demographic and baseline characteristics will be summarized for all donors and recipients.  Donor 
characteristics to be examined are: age, gender, weight, race/ethnicity, CMV status.  Recipient 
characteristics to be examined are: age, gender, weight, race/ethnicity, KPS, disease status at transplant, 
time from diagnosis to transplantation, cytogenetics at diagnosis, conditioning regimen, GVHD 
prophylaxis, graft source and recipient CMV status.  
Table 11.2 Power and Operating 
Characteristics for Plausible 
Combinations of Primary Endpoint  

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11.4.3 
Analysis of the Primary Efficacy Endpoint(s) 
The primary endpoint of this study is the collection of a clinically adequate allograft in one apheresis 
session.  A donor is considered successful for this endpoint if ≥ 2.0 x 106 CD34+ cells/kg actual recipient 
weight are collected in one leukapheresis collection using MGTA-145 + plerixafor. 
A test of the null hypothesis that the rate of providing a clinically adequate allograft within one apheresis 
collection is ≤ 70% will be done using the Simon 2-stage design as described in Section 11.2, with a one-
sided type I error of 5%. The proportion of donors who mobilize ≥ 2.0 x 106 CD34+ cells/kg actual 
recipient weight in one leukapheresis collection will be estimated along with a 90% confidence interval for 
consistency with the one-sided type I error rate of 5%. A 95% confidence interval will also be provided.  
The primary analysis will use the Donor Treated Population, where donors who do not undergo apheresis 
will be considered a failure for this endpoint.  A secondary analysis of this outcome in the donor per 
protocol population will also be conducted using similar methods. 
11.4.4 
Analysis of the Secondary Endpoint(s) 
Clinically desirable allograft in one apheresis collection  
The proportion of donors who mobilize ≥ 4.0 x 106 CD34+ cells/kg actual recipient weight in one 
leukapheresis collection will be estimated along with both 90% and 95% confidence intervals. The 
analysis will use the Donor Treated Population, where donors who do not undergo apheresis will be 
considered a failure for this endpoint. A secondary analysis of this outcome in the donor per protocol 
population will also be conducted using similar methods. 
 
Clinically adequate allograft overall 
The proportion of donors who mobilize ≥ 2.0 x 106 CD34+ cells/kg actual recipient weight across all 
leukapheresis collection will be estimated along with both 90% and 95% confidence intervals. The 
analysis will use the Donor Treated Population, where donors who do not undergo apheresis will be 
considered a failure for this endpoint. A secondary analysis of this outcome in the donor per protocol 
population will also be conducted using similar methods. 
Incidence of Donor AEs  
Type and severity of AEs experienced at various timepoints during and after infusion of plerixafor and 
after administration of MGTA-145, during apheresis, and at one week, one, and six months post 
donation will be tabulated in the donor treated population.  Incidence of AEs will be summarized along 
with both 90% and 95% confidence intervals. Adverse Events of Special Interest (AESI) will also be 
summarized and detailed in a listing. AEs experienced in the plerixafor only population will also be 
listed.   
Time to Neutrophil Recovery  
Incidence of neutrophil recovery will be estimated using the cumulative incidence function treating death 
and relapse prior to recovery as the competing risk.  Probability of neutrophil recovery at Day 28 will be 
calculated along with both 90% and 95% confidence intervals for the recipient analysis population.  
Time to Platelet Recovery  
Incidence of platelet recovery will be estimated for the recipient analysis population using the 
cumulative incidence function treating death prior to platelet recovery as the competing risk. 90% and 
95% confidence intervals will be provided. 
Primary and Secondary Graft Failure  
The frequency and proportion of recipients experiencing graft failure by Day 28 and the proportion of 
recipients who have engrafted who subsequently experience secondary graft failure will be described 
with 90% and 95% confidence intervals for the recipient analysis population. 

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Acute Graft versus Host Disease of Grades 2-4 and 3-4  
Incidence of aGVHD will be estimated using the cumulative incidence function treating death prior to 
aGVHD as the competing risk. The incidence of aGVHD with 90% and 95% confidence intervals will be 
estimated at Day 100 for the recipient analysis population.  
Chronic Graft versus Host Disease  
Incidence of cGVHD will be estimated using the cumulative incidence function treating death prior to 
cGVHD as the competing risk. The incidence of cGVHD with 90% and 95% confidence intervals will be 
estimated at Day 100, 6 and 12 months for the recipient analysis population.  
Treatment Related Mortality (TRM)  
The event is death without disease relapse or progression.  Incidence of TRM will be estimated using 
the cumulative incidence function treating relapse/progression as the competing risk.  The incidence of 
TRM with 90% and 95% confidence intervals will be estimated at Day 100 for the recipient analysis 
population.  
Relapse/Progression  
The event is relapse or progression.  Incidence of relapse/progression in subjects receiving grafts 
mobilized with MGTA-145 + plerixafor will be estimated using the cumulative incidence function treating 
death in remission as the competing risk. Estimates and 90% and 95% confidence intervals will be 
provided at 1 year for the recipient analysis population. 
Progression-Free Survival  
The event is relapse/progression or death. The time to this event is the time from transplant to 
relapse/progression, death, loss to follow-up, or end of study whichever comes first. Progression–free 
survival will be estimated using the Kaplan-Meier estimator for the recipient analysis population. 
Estimates and 90% and 95% confidence intervals will be provided at 1 year. 
Overall Survival  
The event is death from any cause.  The time to this event is the time from transplant to death, loss to 
follow-up, or end of study whichever comes first. Overall survival will be estimated using the Kaplan-
Meier estimator for the recipient analysis population. Estimates and 90% and 95% confidence intervals 
will be provided at 1 year.  
 
Incidence of AEs related to the Allograft 
Type and severity of AEs experienced by the recipient related to the allograft will be tabulated in the 
recipient analysis population.  Incidence of AEs related to the allograft will be summarized along with 
the 90% and 95% confidence intervals.  
 
11.4.5 
 Exploratory Analyses 
Donor Chimerism  
Donor and host cells will be measured at Day 28, 100, 180 and at one-year post-transplant. The degree 
of donor chimerism will be summarized at each time point using descriptive statistics. The proportions of 
recipients with mixed (5-95% donor cells), full (> 95%), or graft rejection (< 5%) will be summarized. 
This analysis will be conducted in the recipient analysis population.  
Colony Formation 
Number of functional progenitor cells that form in methylcellulose (defined as total CFU-GM, CFU-
GEMM, CFU-E, and BFU-E) will be measured. Data will be summarized using descriptive statistics. 

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Immunologic Reconstitution  
Immune reconstitution assays which will include a quantitation of absolute numbers of CD3+, 
CD3+CD4+, CD3+CD8+, CD19+ and CD3-CD56+ cells in recipients on Day 28, 100, 180 and at one-
year post-transplant.  These will be summarized at each time point using descriptive statistics for the 
recipient analysis population. 
Incidence of CMV Reactivation  
Incidence of CMV reactivation will be estimated using the cumulative incidence function with death prior 
to CMV reactivation as the competing risk, in the recipient analysis population. 
Cellular Composition of Allografts  
The proportions of CD34+, CD3+, CD3+CD8+, CD3+CD4+, CD19+, CD34+C90+CD45RA- and CD3-
CD56+ cells in the allografts collected will be summarized using descriptive statistics for grafts 
mobilized with MGTA-145 + plerixafor.  
 
PK Analysis 
Individual Concentration-time data will be listed. Summary statistics of concentration data will be 
presented at each timepoint. Noncompartmental analyses will not be performed.  Data will be analyzed 
as collected in the donor treated population. 
 
Immunogenicity  
The number and percentage of positive ADAs will be presented at Day 28 for the donor treated 
population. 
 
Donor Survey Assessments 
Donor survey assessments (BPI, PROMIS and EQ-5D) will be described longitudinally using means 
and SDs. Linear mixed models will be used to describe the trajectory of recovery over time. The WPAI, 
HRU and donor experience surveys will be presented descriptively. 
12 SUPPORTING DOCUMENTATION AND OPERATIONAL CONSIDERATIONS 
12.1   Regulatory, Ethical, and Study oversight Considerations 
12.1.1 
Institutional Review Board Approval 
Prior to gaining Approval-to-Enroll status, the investigational site will provide to the Sponsor/Sponsor 
representative documentation verifying of IRB approval.  
A copy of the written IRB approval and Informed Consent Form (ICF), must be received by the Sponsor 
before recruitment of subjects into the study and shipment of investigational product.  
Annual IRB approval and renewals will be obtained throughout the duration of the study as required by 
local/central IRB requirements. Copies of the Investigator’s reports and the IRB continuance of approval 
must be provided to the sponsor. 
12.1.1.1 
Recipients and Related Donors 
The IRB of Record for each site (whether sites are relying on a local IRB or central IRB, such as the 
National Marrow Donor Program IRB) will be responsible for the review and continuing oversight of 
protocol procedures that relate to recipient and related donor subjects consented to the study.  
12.1.1.2 
NMDP Unrelated Donors 
The National Marrow Donor Program IRB will have sole responsibility for the review and continuing 
oversight of protocol procedures that relate only to NMDP unrelated donors, including unrelated donor 
consent forms. Local IRB’s do not hold any jurisdiction over unrelated donors obtained through the 

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NMDP, as NMDP Donor Centers participating in this protocol rely on NMDP IRB for this research 
oversight. 
12.1.2 
Informed Consent Process  
Informed consent is a process that is initiated prior to the individual’s agreeing to participate in the study 
and continuing throughout the individual’s study participation. Subject participation is voluntary. Informed 
Consent is required from each subject or his/her legally authorized representative. The Investigator is 
responsible for ensuring that Informed Consent is obtained prior to any study-required procedures and/or 
testing, or data collection.  
The obtaining and documentation of Informed Consent must be in accordance with the principles of the 
Declaration of Helsinki, any applicable national regulations, or Regulatory authority body, as applicable. 
The ICF must be approved by the IRB of Record. 
The ICF must be in a language understandable to the subject. Privacy language for compliance with 
Health Insurance Portability and Accountability Act (HIPAA) regulations shall be included in the body of 
the form or as a separate form, as applicable. 
12.1.2.1 
Consent Procedures and Documentation 
The process of obtaining Informed Consent shall at a minimum include the following steps, as well as any 
other steps required by applicable laws, rules, regulations and guidelines:  
• 
Be conducted by the site Principal Investigator or designee authorized/delegated to conduct the 
process, or a Be The Match Donor Services representative,  
• 
Include a description of all aspects of the clinical study that are relevant to the subject’s decision 
to participate throughout the clinical study,  
• 
Avoid any coercion of or undue influence of subjects to participate,  
• 
Not waive or appear to waive subject’s legal rights,  
• 
Use native language that is non-technical and understandable to the subject or his/her legal 
representative,  
• 
Provide ample time for the subject to consider participation and ask questions if necessary,  
• 
Ensure important new information is provided to new and existing subjects throughout the clinical 
study.  
Consent forms describing in detail the study procedures and possible risks and benefits of the research 
are given to the subject. The investigator will explain the research study to the subject and answer any 
questions that may arise, and the subject will sign the informed consent document prior to any 
procedures being done specifically for the study. The subject may withdraw consent at any time 
throughout the course of the trial, and a copy of the informed consent document will be given to the 
subject. The rights and welfare of the subject will be protected by emphasizing that the quality of medical 
care will not be adversely affected by declining to participate in this study. A copy of the signed consent 
form and documentation of the informed consent discussion will be filed in the subject’s medical record. 
If new information becomes available that can significantly affect a subject's future health and medical 
care, that information shall be provided to the affected subject(s) in written form via a revised ICF or, in 
some situations, enrolled subjects may be requested to sign and date an addendum to the ICF. In 
addition to new significant information during the course of a study, other situations may necessitate 
revision of the ICF, such as if there are amendments to the applicable laws, protocol, a change in site 
Principal Investigator, administrative changes, or following annual review by the IRB. The new version of 
the ICF must be approved by the IRB. The IRB will determine the subject population to be re-consented. 

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12.1.3 
Study Discontinuation and Closure 
The study may be discontinued early by the Sponsor for valid scientific or administrative reasons and 
reasons related to the protection of subjects. Written notification, documenting the reason for study 
suspension or termination, will be provided by the suspending or terminating party to investigators, IRBs 
and regulatory authorities, as applicable. Study subjects will be contacted, as applicable, and be informed 
of changes to study visit schedule.  
12.1.4 
 Investigator Responsibilities 
The Principal Investigator of an investigational site is responsible for ensuring that the study is conducted 
in accordance with the clinical study agreement/rider, applicable sections of 21 CFR, the clinical study 
protocol, ethical principles that have their origins in the Declaration of Helsinki, any conditions of approval 
imposed by the reviewing IRB, and prevailing local and/or country laws and/or regulations, whichever 
affords the greater protection to the subject. 
The Principal Investigator’s responsibilities include, but are not limited to, the following:  
• 
Provide his/her qualifications and experience to assume responsibility for the proper conduct of 
the study and that of key members of the site team through up-to-date curriculum vitae or other 
relevant documentation and disclose potential conflicts of interest, including financial, that may 
interfere with the conduct of the clinical study or interpretation of results. 
• 
Make no changes in or deviate from this protocol, except to protect the life and physical well-being 
of a subject in an emergency; document and explain any deviation from the approved protocol 
that occurred during the course of the clinical investigation. 
• 
Create and maintain source documents throughout the clinical study and ensure their availability 
with direct access during monitoring visits or audits; ensure that all clinical-investigation-related 
records are retained per requirements. 
• 
Ensure the accuracy, completeness, legibility, and timeliness of the data reported to the sponsor 
in the CRFs and in all required reports. 
• 
Record, report, and assess (seriousness and relationship) every adverse event as applicable per 
the protocol. 
• 
Allow the sponsor/sponsor representative to perform monitoring and auditing activities and be 
accessible to the clinical research monitor or auditor and respond to questions during monitoring 
visits or audit(s). 
• 
Allow and support regulatory authorities and the IRB when performing auditing activities. 
• 
Ensure that informed consent is obtained in accordance with applicable laws, this protocol and 
lRB requirements. 
• 
Provide adequate medical care to a subject during and after a subject’s participation in a clinical 
study in the case of adverse events, as described in the ICF. 
• 
Make all reasonable efforts to ascertain the reason(s) for a subject’s premature withdrawal from 
clinical investigation while fully respecting the subject’s rights. 
• 
Ensure that an adequate investigation site team and facilities exist and are maintained and 
documented during the clinical investigation. 

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12.1.5 
Delegation of Responsibility 
When specific tasks are delegated by an investigator, including but not limited to conducting the informed 
consent process, the Principal Investigator is responsible for providing appropriate training and adequate 
supervision of those to whom tasks are delegated. The investigator is accountable for regulatory 
violations resulting from failure to adequately supervise the conduct of the clinical study. 
12.1.6 
Confidentiality and Privacy 
Subject confidentiality is strictly held in trust by the participating investigators, their staff, and the 
sponsor(s) and their agents. This confidentiality is extended to cover testing of biological samples in 
addition to the clinical information relating to participating subjects. 
The study protocol, documentation, data and all other information generated will be held in strict 
confidence. No information concerning the study, or the data will be released to any unauthorized third 
party, without prior written approval of the sponsor.  
The study monitor, or other authorized representatives of the sponsor, may inspect all documents and 
records required to be maintained by the Investigator, including but not limited to, medical records (office, 
clinic or hospital) and pharmacy records for the subjects in this study. The clinical study site will permit 
access to such records. 
12.1.7 
 Future use of Stored Specimens and Data 
Donors will be provided with an optional ICF for an additional blood sample to be obtained during the Day 
28 blood draw. This blood sample will be stored and used for future medical and/or scientific research 
projects that are outside of the current study purpose and objectives.  
12.1.8 
Safety Oversight 
12.1.8.1 
Data Safety Monitoring Board 
Safety oversight will be under the direction of the RCI BMT DSMB composed of individuals with the 
appropriate expertise. Members of the DSMB should be independent from the study conduct and free of 
conflict of interest, or measures should be in place to minimize perceived conflict of interest. All DSMB 
recommendations will be communicated to the appropriate representatives of Magenta and NMDP as 
defined in the charter.  
 
The DSMB will meet per requirements of its charter to assess safety data and determine whether any 
subject safety problems necessitate protocol modifications or discontinuation of the trial. The DSMB will 
also meet on an ad hoc basis if safety monitoring rules are met (see Section 8.2) or if unexpected safety 
events occur that may necessitate study suspension or closure. The DSMB will determine if additional 
review is required and make recommendations to the study team concerning continuation of the study. 
The DSMB will discontinue the review of outcomes when this protocol is closed to accrual.  
12.1.9 
 Clinical Monitoring 
The Investigator will permit study-related on-site, remote, and/or centralized monitoring visits by 
representatives of CIBMTR or designees, and regulatory inspection(s) to ensure proper conduct of the 
study and compliance with the protocol. Access will be provided to the facilities where the study took 
place, to source documents, to data collection forms, and to all other study documents. It is important that 
the site Principal Investigator and relevant study personnel are available during on-site monitoring visits 
or audits and that sufficient time is devoted to the process. 
The monitor should have access to laboratory test reports and other subject records needed to verify the 
entries in the Medidata Rave® EDC application. The investigator [or designee] agrees to cooperate with 

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the monitor to ensure that any problems detected in the course of these monitoring visits are resolved.  
Details regarding monitoring can be found in the study monitoring plan. 
Following written Standard Operating Procedures (SOPs), the monitors will verify that the clinical trial is 
conducted and data are generated and biological specimens are collected, documented (recorded), and 
reported in compliance with the protocol, ICH GCP, and applicable regulatory requirements (e.g., Good 
Laboratory Practices (GLP), Good Manufacturing Practices (GMP)).   
12.1.10  Data Handling and Record Keeping 
12.1.10.1 
Data Collection and Management Responsibilities 
Database backups are performed regularly. 
Protocol-specific study data will be collected within Medidata Rave® EDC application. Medidata Rave® 
software and database have been designed to meet regulatory compliance (21 CFR Part 11) as part of a 
validated system compliant with laws and regulations applicable to the conduct of clinical studies 
pertaining to the use of electronic records and signatures. All changes made to the clinical data will be 
captured in an electronic audit trail and available for review by the sponsor or its representative. For each 
subject enrolled, appropriate electronic case report forms (eCRFs) will be completed. These include 
study-specific eCRFs within the study specific electronic data capture system. The Investigator or 
delegate provides his/her electronic signature on the appropriate eCRFs in compliance with local 
regulations. Data recorded in the eCRF derived from source documents should be consistent with the 
data recorded on the source documents.   
The data collection forms for the subjects enrolled on this study include the standard CIBMTR data 
collection forms in the FormsNet3 Recipient module, and study-specific CIBMTR data collection forms in 
the study specific electronic data capture system, Medidata Rave. Monitoring for this study is limited to 
source document verification (SDV) of the data within Medidata Rave. 
Many important data elements for the study are collected on the standard reporting forms within 
FormsNet3 and therefore timely and accurate completion of these forms is essential. Centers must 
continue to do standard CIBMTR follow-up reporting on these subjects beyond the study time point of 6 
months post-HSCT, per CIBMTR reporting requirements. Recipient subjects will be approached for co-
enrollment to the CIBMTR Outcomes Database protocol. If consented, CIBMTR comprehensive report 
forms (CRF-track) will be required in FormsNet3. 
12.1.10.2 
Study Records Retention 
The Investigator must maintain, at the investigative site, all essential study documentation relating to the 
study for a period of 3 years after the last marketing application approval or, if not approved, 3 years 
following the discontinuance of the test article for investigation. If it becomes necessary for Magenta or 
the regulatory authority to review any documentation relating to the study, the Investigator must permit 
access to such records. 
The Principal Investigator or his/her designee will take measures to ensure that these essential 
documents are not accidentally damaged or destroyed. If for any reason the Principal Investigator or 
his/her designee withdraws responsibility for maintaining these essential documents, custody must be 
transferred to an individual who will assume responsibility and Magenta must receive written notification 
of this custodial change. Sites are required to inform Magenta in writing where paper or electronic files 
are maintained in case files are stored off site and are not readily available. 
12.1.11 Protocol Deviations 
A protocol deviation is any noncompliance with the clinical trial protocol or ICH GCP. The noncompliance 
may be either on the part of the subject, the investigator, or the study site staff. As a result of deviations, 
corrective actions may be developed by the site and implemented promptly. 

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These practices are consistent with Good Clinical Practice (GCP ICH E6) Sections: 
• 
Compliance with Protocol, sections 4.5.1, 4.5.2, and 4.5.3 
• 
Quality Assurance and Quality Control, section 5.1.1 
• 
Noncompliance, sections 5.20.1, and 5.20.2 
An Investigator must not make any changes or deviate from this protocol, except to protect the life and 
physical well-being of a subject in an emergency. An Investigator shall notify the sponsor and the 
reviewing IRB (per local requirements) of any deviation from the investigational plan to protect the life or 
physical well-being of a subject in an emergency and those deviations which affect the scientific integrity 
of the clinical investigation.  
Deviations will be reviewed and evaluated on an ongoing basis and, as necessary, appropriate corrective 
and preventive actions will be put into place by the sponsor. 
12.1.12 Publication and Data Sharing Policy 
Following completion of the study, the study team may publish research results in a scientific journal with 
any interim analysis presented as experience is gained. The International Committee of Medical Journal 
Editors (ICMJE) member journals has adopted a trials-registration policy as a condition for publication. 
This policy requires that all clinical trials be registered in a public trials registry such as 
www.ClinicalTrials.gov, which is sponsored by the National Library of Medicine. 
12.1.13 Conflict of Interest Policy 
The independence of this study from any actual or perceived influence is critical. Therefore, any actual 
conflict of interest of persons who have a role in the design, conduct, analysis, publication, or any aspect 
of this trial will be disclosed and managed. Furthermore, persons who have a perceived conflict of 
interest will be required to have such conflicts managed in a way that is appropriate to their participation 
in the design and conduct of this trial. The study leadership in conjunction with the RCI BMT has 
established policies and procedures for all study group members to disclose all conflicts of interest and 
will establish a mechanism for the management of all reported dualities of interest. 
 
12.2  Additional Considerations 
12.2.1 
Donor Product Qualification  
Any processes covered by the FDA’s 21 CFR part 1271, including donor qualification and good tissue 
practice, are the responsibility of the transplant center, and are out of the scope for this study.  
 
12.3  Publication Policy 
Magenta assures that the key design elements of this protocol will be posted in a publicly accessible 
database such as www.ClinicalTrials.gov. In addition, upon study completion and finalization of the study 
report, the results of this study (regardless of study outcomes) will be either submitted for publication 
and/or posted in a publicly accessible database of clinical study results. Magenta requires disclosures of 
its involvements as a sponsor or financial supporter in any publication or presentation relating to a 
Magenta study or its results. 
 
 

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12.4  Abbreviations 
 
ABO/RH 
Blood type  
ADA 
Anti-drug Antibody 
AE  
Adverse Event 
AESI 
Adverse Event of Special Interest 
aGVHD 
Acute graft versus host disease 
ALL 
Acute Lymphoblastic Leukemia 
ALT 
Alanine aminotransferase 
AML 
Acute Myelogenous Leukemia 
ANC 
Absolute Neutrophil Count 
AST 
Aspartate aminotransferase  
AUC 
Area under the curve 
BM 
Bone Marrow 
CBC 
Complete blood count 
CFR 
Code of Federal Regulations 
CIBMTR 
Center for International Blood and Marrow Transplant Research 
CRCL 
Creatinine Clearance 
CMV 
Cytomegalovirus 
CNS 
Central Nervous System 
CR 
Complete Remission 
CTCAE 
Common Terminology Criteria for Adverse Events 
CVC 
Central Venous Catheter 
DLCO 
Diffusing capacity of the lungs for carbon monoxide 
DLI 
Donor Cellular Infusion 
DLT 
Dose limiting toxicities 
DSMB 
Data Safety Monitoring Board 
EBMT 
European Bone Marrow Transplant group 
ECG 
Electrocardiogram  
eCRF 
Electronic Case Report Forms 
EDC 
Electronic Data Capture 
FEV1 
Forced expiratory volume in first second 
GCP 
Good Clinical Practice 
G-CSF 
Granulocyte colony-stimulating factor 
GFR 
Glomerular filtration rate 
GLP 
Good Laboratory Practices 
GM-CSF 
Granulocyte-macrophage colony-stimulating factor 
GMP 
Good Manufacturing Practices 
GVHD 
Graft versus host disease 
HCT-CI 
Hematopoietic Cell Transplantation-Comorbidity Index 
HIPAA 
Health Insurance Portability and Accountability Act   
HIV 
Human immunodeficiency virus 
HLA 
Human Leukocyte Antigen 
HPC 
Hematopoietic Progenitor Cells 
HPC(A) 
Hematopoietic Progenitor Cell Apheresis 
HRU 
Healthcare Resource Utilization 
HSC 
Hematopoietic Stem Cells 
HSCT 
Hematopoietic Stem Cell Transplantation 

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HSPC 
Hematopoietic Stem and Progenitor Cells 
IB 
Investigators Brochure 
IBMTR 
International Blood and Marrow Transplant Registry 
ICF 
Informed Consent Form 
ICH 
International Conference on Harmonisation 
ICMJE 
International Committee of Medical Journal Editors 
IRB 
Institutional Review Board 
IVIG 
Intravenous Immune Globulin 
KPS 
Karnofsky performance status 
LA 
Leukapheresis 
LDH 
Lactate dehydrogenase 
MAC 
Myeloablative Conditioning 
MDRD 
Modification of Diet in Renal Disease 
MDS 
Myelodysplastic Syndrome 
MPB 
Mobilized Peripheral Blood 
MTD 
Maximum tolerated dose 
NHL 
Non-Hodgkin lymphoma 
NMA 
Non-myeloablative Conditioning  
NMDP 
National Marrow Donor Program 
PB 
Peripheral Blood 
PBSC 
Peripheral Blood Stem Cells 
PK 
Pharmacokinetic  
PT 
Prothrombin Time 
PTT 
Partial thromboplastin time  
RBC 
Red blood cell count 
RIC 
Reduced Intensity Conditioning 
SAE 
Serious Adverse Event 
SOA 
Schedule of Activities 
SOP 
Standard Operating Procedure 
SUSAR 
Suspected Unexpected Serious Adverse Reaction 
TEAEs 
Treatment emergent adverse events 
TNC 
Total Nucleated Cell count 
TRM 
Treatment-Related Mortality 
UCB 
Umbilical cord blood 
ULN 
Upper Limit of Normal 
US 
United States 
WBC 
White Blood Cell 
WPAI 
Work Productivity and Activity Impairment 
 
 
 

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13 REFERENCES 
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Körbling M, Huh YO, Durett A, et al. Allogeneic blood stem cell transplantation: peripheralization 
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Bensinger WI, Martin PJ, Storer B, et al. Transplantation of bone marrow as compared with 
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Couban S, Simpson DR, Barnett MJ, et al. A randomized multicenter comparison of bone marrow 
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Kaiser U, Uebelacker I, Abel U, et al. Randomized study to evaluate the use of high-dose therapy 
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Vigorito AC, Azevedo WM, Marques JF, et al. A randomised, prospective comparison of 
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10. 
Cutler C, Giri S, Jeyapalan S, Paniagua D, Viswanathan A, Antin JH. Acute and chronic graft-
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a meta-analysis. J Clin Oncol. 2001;19(16):3685-3691. 
11. 
Allogeneic peripheral blood stem-cell compared with bone marrow transplantation in the 
management of hematologic malignancies: an individual patient data meta-analysis of nine 
randomized trials. J Clin Oncol. 2005;23(22):5074-5087. 
12. 
Anasetti C, Logan BR, Lee SJ, et al. Peripheral-blood stem cells versus bone marrow from 
unrelated donors. N Engl J Med. 2012;367(16):1487-1496. 
13. 
Zaucha JM, Gooley T, Bensinger WI, et al. CD34 cell dose in granulocyte colony-stimulating 
factor-mobilized peripheral blood mononuclear cell grafts affects engraftment kinetics and 
development of extensive chronic graft-versus-host disease after human leukocyte antigen-
identical sibling transplantation. Blood. 2001;98(12):3221-3227. 
14. 
Przepiorka D, Weisdorf D, Martin P, et al. 1994 Consensus Conference on Acute GVHD Grading. 
Bone Marrow Transplant. 1995;15(6):825-828. 
15. 
Nakamura R, Bahceci E, Read EJ, et al. Transplant dose of CD34(+) and CD3(+) cells predicts 
outcome in patients with haematological malignancies undergoing T cell-depleted peripheral 
blood stem cell transplants with delayed donor lymphocyte add-back. Br J Haematol. 
2001;115(1):95-104. 
16. 
Bahçeci E, Read EJ, Leitman S, et al. CD34+ cell dose predicts relapse and survival after T-cell-
depleted HLA-identical haematopoietic stem cell transplantation (HSCT) for haematological 
malignancies. Br J Haematol. 2000;108(2):408-414. 
17. 
Anderlini P, Körbling M, Dale D, et al. Allogeneic blood stem cell transplantation: considerations 
for donors. Blood. 1997;90(3):903-908. 

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18. 
Körbling M, Anderlini P. Peripheral blood stem cell versus bone marrow allotransplantation: does 
the source of hematopoietic stem cells matter? Blood. 2001;98(10):2900-2908. 
19. 
Anderlini P, Rizzo JD, Nugent ML, Schmitz N, Champlin RE, Horowitz MM. Peripheral blood stem 
cell donation: an analysis from the International Bone Marrow Transplant Registry (IBMTR) and 
European Group for Blood and Marrow Transplant (EBMT) databases. Bone Marrow Transplant. 
2001;27(7):689-692. 
20. 
Rowley SD, Donaldson G, Lilleby K, Bensinger WI, Appelbaum FR. Experiences of donors 
enrolled in a randomized study of allogeneic bone marrow or peripheral blood stem cell 
transplantation. Blood. 2001;97(9):2541-2548. 
21. 
Fortanier C, Kuentz M, Sutton L, et al. Healthy sibling donor anxiety and pain during bone marrow 
or peripheral blood stem cell harvesting for allogeneic transplantation: results of a randomised 
study. Bone Marrow Transplant. 2002;29(2):145-149. 
22. 
Sisler IY, Koehler E, Koyama T, et al. Impact of conditioning regimen in allogeneic hematopoetic 
stem cell transplantation for children with acute myelogenous leukemia beyond first complete 
remission: a pediatric blood and marrow transplant consortium (PBMTC) study. Biol Blood Marrow 
Transplant. 2009;15(12):1620-1627. 
23. 
Lapidot T. Mechanism of human stem cell migration and repopulation of NOD/SCID and B2mnull 
NOD/SCID mice. The role of SDF-1/CXCR4 interactions. Ann N Y Acad Sci. 2001;938:83-95. 
24. 
Lapidot T, Kollet O. The essential roles of the chemokine SDF-1 and its receptor CXCR4 in 
human stem cell homing and repopulation of transplanted immune-deficient NOD/SCID and 
NOD/SCID/B2m(null) mice. Leukemia. 2002;16(10):1992-2003. 
25. 
Lévesque JP, Hendy J, Takamatsu Y, Simmons PJ, Bendall LJ. Disruption of the 
CXCR4/CXCL12 chemotactic interaction during hematopoietic stem cell mobilization induced by 
GCSF or cyclophosphamide. J Clin Invest. 2003;111(2):187-196. 
26. 
Petit I, Szyper-Kravitz M, Nagler A, et al. G-CSF induces stem cell mobilization by decreasing 
bone marrow SDF-1 and up-regulating CXCR4. Nat Immunol. 2002;3(7):687-694. 
27. 
Ma Q, Jones D, Borghesani PR, et al. Impaired B-lymphopoiesis, myelopoiesis, and derailed 
cerebellar neuron migration in CXCR4- and SDF-1-deficient mice. Proc Natl Acad Sci U S A. 
1998;95(16):9448-9453. 
28. 
Tachibana K, Hirota S, Iizasa H, et al. The chemokine receptor CXCR4 is essential for 
vascularization of the gastrointestinal tract. Nature. 1998;393(6685):591-594. 
29. 
Zou YR, Kottmann AH, Kuroda M, Taniuchi I, Littman DR. Function of the chemokine receptor 
CXCR4 in haematopoiesis and in cerebellar development. Nature. 1998;393(6685):595-599. 
30. 
Peled A, Petit I, Kollet O, et al. Dependence of human stem cell engraftment and repopulation of 
NOD/SCID mice on CXCR4. Science. 1999;283(5403):845-848. 
31. 
Hattori K, Heissig B, Tashiro K, et al. Plasma elevation of stromal cell-derived factor-1 induces 
mobilization of mature and immature hematopoietic progenitor and stem cells. Blood. 
2001;97(11):3354-3360. 
32. 
Sweeney EA, Lortat-Jacob H, Priestley GV, Nakamoto B, Papayannopoulou T. Sulfated 
polysaccharides increase plasma levels of SDF-1 in monkeys and mice: involvement in 
mobilization of stem/progenitor cells. Blood. 2002;99(1):44-51. 
33. 
Lapidot T, Petit I. Current understanding of stem cell mobilization: the roles of chemokines, 
proteolytic enzymes, adhesion molecules, cytokines, and stromal cells. Exp Hematol. 
2002;30(9):973-981. 
34. 
Schols D, Esté JA, Henson G, De Clercq E. Bicyclams, a class of potent anti-HIV agents, are 
targeted at the HIV coreceptor fusin/CXCR-4. Antiviral Res. 1997;35(3):147-156. 
35. 
Labrosse B, Brelot A, Heveker N, et al. Determinants for sensitivity of human immunodeficiency 
virus coreceptor CXCR4 to the bicyclam AMD3100. J Virol. 1998;72(8):6381-6388. 
36. 
Schols D, De Clercq E. The simian immunodeficiency virus mnd(GB-1) strain uses CXCR4, not 
CCR5, as coreceptor for entry in human cells. J Gen Virol. 1998;79 ( Pt 9):2203-2205. 

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37. 
Donzella GA, Schols D, Lin SW, et al. AMD3100, a small molecule inhibitor of HIV-1 entry via the 
CXCR4 co-receptor. Nat Med. 1998;4(1):72-77. 
38. 
Egberink HF, De Clercq E, Van Vliet AL, et al. Bicyclams, selective antagonists of the human 
chemokine receptor CXCR4, potently inhibit feline immunodeficiency virus replication. J Virol. 
1999;73(8):6346-6352. 
39. 
Murai M, Yoneyama H, Harada A, et al. Active participation of CCR5(+)CD8(+) T lymphocytes in 
the pathogenesis of liver injury in graft-versus-host disease. J Clin Invest. 1999;104(1):49-57. 
40. 
Hatse S, Princen K, Bridger G, De Clercq E, Schols D. Chemokine receptor inhibition by 
AMD3100 is strictly confined to CXCR4. FEBS Lett. 2002;527(1-3):255-262. 
41. 
Matthys P, Hatse S, Vermeire K, et al. AMD3100, a potent and specific antagonist of the stromal 
cell-derived factor-1 chemokine receptor CXCR4, inhibits autoimmune joint inflammation in IFN-
gamma receptor-deficient mice. J Immunol. 2001;167(8):4686-4692. 
42. 
Gerlach LO, Skerlj RT, Bridger GJ, Schwartz TW. Molecular interactions of cyclam and bicyclam 
non-peptide antagonists with the CXCR4 chemokine receptor. J Biol Chem. 2001;276(17):14153-
14160. 
43. 
De Clercq E. The bicyclam AMD3100 story. Nat Rev Drug Discov. 2003;2(7):581-587. 
44. 
Hendrix CW, Flexner C, MacFarland RT, et al. Pharmacokinetics and safety of AMD-3100, a 
novel antagonist of the CXCR-4 chemokine receptor, in human volunteers. Antimicrob Agents 
Chemother. 2000;44(6):1667-1673. 
45. 
Hoggatt J, Singh P, Tate TA, et al. Rapid Mobilization Reveals a Highly Engraftable 
Hematopoietic Stem Cell. Cell. 2018;172(1-2):191-204.e110. 
46. 
Amgen. NEUPOGEN (filgrastim) injection, for subcutaneous or intravenous. 
https://www.accessdata.fda.gov/drugsatfda_docs/label/2015/103353s5183lbl.pdf. Published 2015. 
Accessed 2020 Aug 13. 
47. 
Flomenberg N, Devine SM, Dipersio JF, et al. The use of AMD3100 plus G-CSF for autologous 
hematopoietic progenitor cell mobilization is superior to G-CSF alone. Blood. 2005;106(5):1867-
1874. 
48. 
Schroeder MA, Rettig MP, Lopez S, et al. Outcomes of Patients Receiving Allogeneic Peripheral 
Blood Stem Cell Products Mobilized with Intravenous Plerixafor. Blood. 2011;118(21):314-314. 
49. 
DiPersio JF, Stadtmauer EA, Nademanee A, et al. Plerixafor and G-CSF versus placebo and G-
CSF to mobilize hematopoietic stem cells for autologous stem cell transplantation in patients with 
multiple myeloma. Blood. 2009;113(23):5720-5726. 
50. 
Micallef IN, Stiff PJ, Nademanee AP, et al. Plerixafor Plus Granulocyte Colony-Stimulating Factor 
for Patients with Non-Hodgkin Lymphoma and Multiple Myeloma: Long-Term Follow-Up Report. 
Biol Blood Marrow Transplant. 2018;24(6):1187-1195. 
51. 
Devine SM, Vij R, Rettig M, et al. Rapid mobilization of functional donor hematopoietic cells 
without G-CSF using AMD3100, an antagonist of the CXCR4/SDF-1 interaction. Blood. 
2008;112(4):990-998. 
52. 
Schroeder MA, Rettig MP, Lopez S, et al. Mobilization of allogeneic peripheral blood stem cell 
donors with intravenous plerixafor mobilizes a unique graft. Blood. 2017;129(19):2680-2692. 
53. 
Chen YB, Le-Rademacher J, Brazauskas R, et al. Plerixafor alone for the mobilization and 
transplantation of HLA-matched sibling donor hematopoietic stem cells. Blood Adv. 
2019;3(6):875-883. 
54. 
Khoury HJ, Loberiza FR, Jr., Ringdén O, et al. Impact of posttransplantation G-CSF on outcomes 
of allogeneic hematopoietic stem cell transplantation. Blood. 2006;107(4):1712-1716. 
55. 
Cella D, Gershon R, Lai JS, Choi S. The future of outcomes measurement: item banking, tailored 
short-forms, and computerized adaptive assessment. Qual Life Res. 2007;16 Suppl 1:133-141. 
56. 
Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials. 1989;10(1):1-
10. 
 

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14 APPENDICES 
A. Karnofsky Performance Scale 
 
KARNOFSKY SCALE, ≥ 16 YEARS 
 
100% – Normal, no complaints, no evidence of disease 
90% – Able to carry on normal activity 
80% – Normal activity with effort 
70% – Cares for self, unable to carry on normal activity or to do active work 
60% – Requires occasional assistance but is able to care for most needs 
50% – Requires considerable assistance and frequent medical care 
40% – Disabled, requires special care and assistance 
30% – Severely disabled, hospitalization indicated, although death not imminent 
20% – Very sick, hospitalization necessary 
10% – Moribund, fatal process progressing rapidly 
* Report performance score only as multiples of 10. 
 
 

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B. Hematopoietic Cell Transplant Comorbidity Index (HCT-CI) Scoring 
The following table can be used to calculate the HCT-CI as developed by Sorror, Blood, 2013 Apr 11; 
121(15): 2854-2863.  
Comorbidities 
HCT-CI scores 
Arrhythmia 
1 
Cardiovascular comorbidity 
1 
Inflammatory bowel disease 
1 
Diabetes or steroid-induced hyperglycemia 
1 
Cerebrovascular disease 
1 
Psychiatric disorder 
1 
Mild hepatic comorbidity 
1 
Obesity 
1 
Infection 
1 
Rheumatologic comorbidity 
2 
Peptic ulcer 
2 
Renal comorbidity 
2 
Moderate pulmonary comorbidity 
2 
Prior malignancy 
3 
Heart valve disease 
3 
Moderate/severe hepatic comorbidity 
3 
Severe pulmonary comorbidity 
3 
Total score = ________ 
 
 
 

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C. Acute GVHD Grading 
The following target organ staging and overall clinical grading criteria should be used when 
completing acute GVHD assessments, utilizing the Mount Sinai Acute GVHD International 
Consortium (MAGIC). 
Harris AC, Young R, Devine S, et al. International, Multicenter Standardization of Acute Graft-versus-Host 
Disease Clinical Data Collection: A Report from the Mount Sinai Acute GVHD International Consortium. Biol Blood 
Marrow Transplant. 2016;22(1):4-10. doi:10.1016/j.bbmt.2015.09.001 
 
Target Organ Staging 
Stage 
Skin (active erythema 
only) 
Liver (bilirubin) 
Upper GI 
Lower GI (stool output/day)3 
0 
No active (erythematous) 
GVHD rash 
< 2 mg/dl 
No or intermittent 
nausea, vomiting 
or anorexia 
Adult: < 500 ml/day or <3 
episodes/day 
Child: < 10 ml/kg/day or <4 
episodes/day 
1 
Maculopapular rash  
<25% BSA1 
2-3 mg/dl2 
Persistent nausea, 
vomiting or 
anorexia4 
Adult: 500–999 ml/day or 3–4 
episodes/day 
Child: 10–19.9 ml/kg/day or 4–6 
episodes/day 
2 
Maculopapular rash  
25-50% BSA1 
3.1-6 mg/dl2 
- 
Adult: 1000–1500 ml/day or 5–7 
episodes/day 
Child: 20 – 30 ml/kg/day or 7–10 
episodes/day 
3 
Maculopapular rash  
>50% BSA1 
6.1-15 mg/dl2 
- 
Adult: >1500 ml/day or >7 
episodes/day 
Child: > 30 ml/kg/day or >10 
episodes/day 
4 
Generalized erythroderma 
(>50% BSA1) plus bullous 
formation and 
desquamation >5% BSA1 
>15 mg/dl2 
- 
Severe abdominal pain with or 
without ileus, or grossly bloody stool 
(regardless of stool volume). 
Overall Clinical Grade
5 
0 
None 
None 
None 
None 
I 
Stage 1-2 
None 
None 
None 
II 
Stage 3 
Stage 1 
Stage 1 
Stage 1 
III 
— 
Stage 2-3 
Stage 1 
Stage 2-3 

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IV6 
Stage 4 
Stage 4 
 
Stage 4 
Confidence Levels 
 
Pathologic evidence 
Clinician assessment 
Treatment for 
acute GVHD 
Comments 
Confirmed Unequivocal pathologic 
evidence of GVHD 
GVHD is the etiology for 
symptoms 
Not applicable 
GVHD is clearly present even if 
other etiologies may co-exist 
simultaneously 
Probable 
Not required 
GVHD most likely 
etiology for symptoms 
(as evidenced by 
treatment being 
provided) 
Yes 
GVHD is most likely present but 
other etiologies may also explain 
the symptoms and there is 
insufficient evidence to make a 
confirmed diagnosis 
Possible 
Not required 
GVHD in differential 
diagnosis (but no 
treatment is being 
provided) 
No 
GVHD may be present, but other 
etiologies are favored to the degree 
that GVHD treatment is not initiated 
Negative 
Unequivocal evidence of a 
diagnosis other than 
GVHD (e.g., drug rash) 
GVHD is not considered 
as an explanation for 
the symptoms 
No and the 
symptoms resolve 
without GVHD 
treatment 
A “negative” biopsy (e.g., normal 
skin) is not unequivocal evidence 
of a diagnosis other than GVHD 
1 Use “Rule of Nines” or burn chart to determine extent of rash. 
2 Range given as total bilirubin. Downgrade one stage if an additional cause of elevated bilirubin has been documented. 
3 Downgrade one stage if an additional cause of diarrhea has been documented. 
4 Persistent nausea with or without histologic evidence of GVHD in the stomach or duodenum. 
5 Criteria for grading given as minimum degree of organ involvement required to confer that grade. 
6 Grade IV may also include lesser organ involvement with an extreme decrease in performance status 
 
 

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D. Chronic GVHD Grading 
NIH Consensus Criteria will be used for GVHD diagnosis and grading.  
Jagasia MH, Greinix HT, Arora M, et al. National Institutes of Health Consensus Development Project on 
Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: I. The 2014 Diagnosis and Staging Working 
Group report. Biol Blood Marrow Transplant. 2015;21(3):389-401.e1. doi:10.1016/j.bbmt.2014.12.001 
 

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E. Determination of Relapse or Disease Progression 
Acute Myelogenous Leukemia (AML) Response Criteria: 
Complete Remission (CR) 
Hematologic complete remission is defined as meeting all of the following response criteria 
for at least four weeks. 
• 
< 5% blasts in the bone marrow 
• 
No blasts with Auer rods 
• 
Normal maturation of all cellular components in the bone marrow 
• 
No extramedullary disease (e.g., CNS, soft tissue disease) 
• 
Neutrophils ≥ 1 x109/L 
• 
Platelets ≥ 100 x109/L 
• 
Transfusion independent 
Complete Remission with Incomplete Hematologic Recovery (CRi) 
Hematologic complete remission with incomplete hematologic recovery is defined as meeting 
all of the following response criteria for at least four weeks: 
• 
< 5% blasts in the bone marrow 
• 
No blasts with Auer rods 
• 
Normal maturation of all cellular components in the bone marrow 
• 
No extramedullary disease (e.g., CNS, soft tissue disease) 
• 
Transfusion independent (Please note, if the physician documents transfusion 
dependence related to treatment and not the patient’s underlying AML, CRi can be 
reported) 
Primary Induction Failure (PIF) 
The patient received treatment for AML but never achieved CR or CRi at anytime. PIF is 
not limited by the number of unsuccessful treatments; this disease status only applies to 
recipients who have never been in CR or CRi. 
Relapse (REL) 
Relapse is defined as the recurrence of disease after CR, meeting one or more of the 
following criteria: 
• 
≥ 5% blasts in the marrow or peripheral blood 
• 
Extramedullary disease 
• 
Disease presence determined by a physician upon clinical assessment 
 
No Treatment 
The recipient was diagnosed with acute leukemia and never received therapeutic agents; include 
patients who have received only supportive therapy, including growth factors and/or blood 
transfusions. 
 
 

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Acute Lymphoblastic Leukemia (ALL) Response Criteria: 
Complete Remission (CR) 
Hematologic complete remission is defined as meeting all of the following response criteria 
for at least four weeks. 
• 
< 5% blasts in the bone marrow 
• 
Normal maturation of all cellular components in the bone marrow 
• 
No extramedullary disease (e.g., CNS, soft tissue disease) 
• 
ANC (absolute neutrophil count) ≥ 1 x 109/L 
• 
Platelets ≥ 100 x 109/L 
• 
Transfusion independent 
Complete Remission with Incomplete Hematologic Recovery (CRi) 
Hematologic complete remission with incomplete hematologic recovery is defined as meeting 
all of the following response criteria for at least four weeks: 
• 
< 5% blasts in the bone marrow 
• 
Normal maturation of all cellular components in the bone marrow 
• 
No extramedullary disease (e.g., CNS, soft tissue disease) 
• 
Transfusion independent (Please note, if the physician documents transfusion 
dependence related to treatment and not the patient’s underlying ALL, CRi can be 
reported) 
Primary Induction Failure (PIF) 
The patient received treatment for ALL but never achieved CR or CRi at anytime. PIF is not 
limited by the number of unsuccessful treatments; this disease status only applies to 
recipients who have never been in CR or CRi. 
Relapse (REL) 
Relapse is defined as the recurrence of disease after CR, meeting at least one of the 
following criteria: 
• 
≥ 5% blasts in the marrow or peripheral blood 
• 
Extramedullary disease 
• 
Disease presence determined by a physician upon clinical assessment 
No Treatment 
The recipient was diagnosed with acute leukemia and never received therapeutic agents; include 
patients who have received only supportive therapy, including growth factors and/or blood 
transfusions. 
 
 
 

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Myelodysplastic Syndrome (MDS) Response Criteria 
Complete Remission (CR) 
Requires all of the following maintained for a minimum of four weeks: 
Bone marrow evaluation: 
• 
< 5% myeloblasts with normal maturation of all cell lines 
 
Peripheral blood evaluation: 
• 
Hemoglobin ≥ 11 g/dL untransfused without erythropoietic support 
• 
ANC ≥ 1 x 109/L without myeloid growth factor support 
• 
Platelets ≥ 100 x 109/L without thrombopoietic support 
• 
0% blasts in blood 
Hematologic Improvement (HI) 
Requires one measurement of the following maintained for at least eight weeks without ongoing 
cytotoxic therapy: 
Hematologic improvement – erythropoietic (HI-E): 
• 
Hemoglobin increase of ≥ 1.5 g/dL untransfused 
or 
• 
For RBC transfusions performed for hemoglobin ≤ 9.0: reduction in RBC units transfused 
in 8 weeks by ≥ 4 units compared to the number of units transfused in the 8 weeks prior to 
treatment 
 
Hematologic improvement – platelets (HI-P): 
• 
For pre-treatment platelet count of ≥ 20 ×109, platelet absolute increase of ≥ 30 ×109 
• 
For pre-treatment platelet count of < 20 ×109, platelet absolute increase of ≥ 20 ×109 and ≥ 
100% increase from pre-treatment level 
 
Hematologic improvement – neutrophils (HI-N): 
• 
Neutrophil count increase of ≥ 100% from pre-treatment level and an absolute increase of 
≥ 0.5 x 109/L 
No Response (NR)/Stable Disease (SD) 
Does not meet the criteria for at least HI, but no evidence of disease progression to AML 
Progression from Hematologic Improvement (Prog from HI) 
Requires at least one of the following in the absence of another explanation (e.g., infection, 
bleeding, ongoing chemotherapy, etc.): 

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• 
≥ 50% reduction from maximum response levels in granulocytes or platelets 
• 
Reduction in hemoglobin by ≥ 1.5 g/dL 
• 
Transfusion dependence 
Note: declining donor chimerism does not meet the criteria for progression. If the above criteria 
for progression have been met, but a hematologic improvement was not previously achieved, 
status is “No Response (NR) / Stable Disease (SD).” 
Relapse from Complete Remission (Rel from CR) 
Requires at least one of the following: 
• 
Return to pre-treatment bone marrow blast percentage 
• 
Decrease of ≥ 50% from maximum response levels in granulocytes or platelets 
• 
Transfusion dependence or hemoglobin level ≥ 1.5 g/dL lower than prior to therapy 
Note: declining donor chimerism does not meet the criteria for relapse. 
Progression to AML 
• 
≥ 20% blasts in the blood or bone marrow 
 
 

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F. Donor Survey Assessments 
Presented here are examples of potential donor survey assessment questions. Not all questions are 
asked at each donor survey time point. Section 9.7 lists the donor survey assessment domains included 
at each time point. 
 
The PROMIS questions presented here represent the short forms that would be included on any paper 
donor survey assessment. If a donor subject completes their surveys online, the PROMIS questionnaires 
will be administered via Computer Adaptive Testing, as described in section 9.7.2 of this protocol. In 
Computer Adaptive Testing, each subject is presented with different questions within each domain, that 
are tailored to how they feel.  
 
Brief Pain Inventory (Short Form) 
 
Throughout our lives, most of us have had pain from time to time (such as minor headaches, 
sprains, and toothaches). Have you had pain other than these everyday kind of pains today? 
□ Yes 
□ No 
 
On the diagram, shade in the areas where you feel pain. Put an X on the area that hurts the most. 
 
 
 
 

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Please rate your pain by selecting the one number that best describes your pain at its worst in the 
last 24 hours. 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
No 
Pain 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Pain as bad as 
you can imagine 
 
Please rate your pain by selecting the one number that best describes your pain at its least in the 
last 24 hours. 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
No 
Pain 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Pain as bad as 
you can imagine 
 
Please rate your pain by selecting the one number that best describes your pain on the average. 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
No 
Pain 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Pain as bad as 
you can imagine 
 
Please rate your pain by selecting the one number that tells how much pain you have right now. 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
No 
Pain 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Pain as bad as 
you can imagine 
 
 
What treatments or medications are you receiving for your pain? 
 
___________________________________________________________________________ 
___________________________________________________________________________ 
___________________________________________________________________________ 
 
In the last 24 hours, how much relief have pain treatments or medications provided? Please select 
the one percentage that shows how much relief you have received. 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0% 
No 
Relief 
10% 
20% 
30% 
40% 
50% 
60% 
70% 
80% 
90% 
100%  
Complete Relief 
 
 
 

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Select the one number that describes how during the past 24 hours, pain has interfered with your: 
General Activity 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
Does not 
interfere 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Completely 
interferes 
Mood 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
Does not 
interfere 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Completely 
interferes 
Walking Ability 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
Does not 
interfere 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Completely 
interferes 
Normal Work (includes both work outside the home and housework) 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
Does not 
interfere 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Completely 
interferes 
Relations with other people 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
Does not 
interfere 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Completely 
interferes 
Sleep 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
Does not 
interfere 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Completely 
interferes 
Enjoyment of life 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
Does not 
interfere 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
Completely 
interferes 
 
 
 
 

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PROMIS Fatigue 
At Day 3-4 time point, the recall period of these questions will be changed from 7 days to 1 day. 
 
Please respond to each question or statement by marking one box per row 
During the past 7 days… 
Not at all 
A little bit 
Somewhat Quite a bit Very much 
I feel fatigued 
□ 
□ 
□ 
□ 
□ 
I have trouble starting things because I 
am tired 
□ 
□ 
□ 
□ 
□ 
 
Please respond to each question or statement by marking one box per row 
In the past 7 days… 
Not at all 
A little bit 
Somewhat Quite a bit Very much 
How run-down did you feel on average? 
□ 
□ 
□ 
□ 
□ 
How fatigued were you on average? 
□ 
□ 
□ 
□ 
□ 
How much were you bothered by your 
fatigue on average? 
□ 
□ 
□ 
□ 
□ 
To what degree did your fatigue interfere 
with your physical functioning? 
□ 
□ 
□ 
□ 
□ 
 
Please respond to each question or statement by marking one box per row 
In the past 7 days… 
Never 
Rarely 
Sometimes 
Often 
Always 
How often did you have to push yourself 
to get things done because of your 
fatigue? 
□ 
□ 
□ 
□ 
□ 
How often did you have trouble finishing 
things because of your fatigue? 
□ 
□ 
□ 
□ 
□ 
 
 
 

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PROMIS Gastrointestinal (Diarrhea) 
Asked at Baseline and Day 28 
 
In the past 7 days, how many days did you have loose or watery stools? 
 
If 1 or more days… 
 
How much did having loose or watery stools interfere with your day-to-day activities?  
 
How much did having loose or watery stools bother you?  
 
 
 
In the past 7 days, how often did you feel like you needed to empty your bowels right away or else 
you would have an accident? 
 
If 1 or more days… 
 
How much did feeling you needed to empty your bowels right away interfere with your day-to-
day activities? 
How much did feeling you needed to empty your bowels right away bother you?  
 
 
Asked at Day 1 and Day 3-4 
 
In the past 24 hours, did you have loose or watery stools? 
□ No 
□ Yes 
 
If yes… 
 
How much did having loose or watery stools interfere with your day-to-day activities?  
How much did having loose or watery stools bother you?  
 
 
In the past 24 hours, did you feel like you needed to empty your bowels right away or else you 
would have an accident? 
□ No 
□ Yes 
 
If Yes… 
 
How much did feeling you needed to empty your bowels right away interfere with your day-to-
day activities? 
 
 

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PROMIS Physical Function 
 
Please respond to each question or statement by marking one box per row 
 
Not at all 
Very little 
Somewhat Quite a lot Cannot do 
Does your health now limit you in doing 
vigorous activities, such as running, lifting 
heavy objects, participating in strenuous 
sports? 
□ 
□ 
□ 
□ 
□ 
Does your health now limit you in walking 
more than a mile (1.6 km)? 
□ 
□ 
□ 
□ 
□ 
Does your health now limit you in climbing 
one flight of stairs? 
□ 
□ 
□ 
□ 
□ 
Does your health now limit you in lifting or 
carrying groceries? 
□ 
□ 
□ 
□ 
□ 
Does your health now limit you in bending, 
kneeling, or stooping? 
□ 
□ 
□ 
□ 
□ 
 
Please respond to each question or statement by marking one box per row 
 
 
Without 
any 
difficulty 
With a little 
difficulty 
With some 
difficulty 
With much 
difficulty 
Cannot do 
Are you able to do chores such as 
vacuuming or yard work? 
□ 
□ 
□ 
□ 
□ 
Are you able to dress yourself, including 
tying shoelaces and buttoning your 
clothes? 
□ 
□ 
□ 
□ 
□ 
Are you able to shampoo your hair? 
□ 
□ 
□ 
□ 
□ 
Are you able to wash and dry your body? 
□ 
□ 
□ 
□ 
□ 
Are you able to sit on and get up from the
toilet? 
□ 
□ 
□ 
□ 
□ 
 
 
 
 

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PROMIS Anxiety 
 
Please respond to each question or statement by marking one box per row 
In the past 7 days… 
Never 
Rarely 
Sometimes 
Often 
Always 
I felt fearful 
□ 
□ 
□ 
□ 
□ 
I found it hard to focus on anything 
other than my anxiety 
□ 
□ 
□ 
□ 
□ 
My worries overwhelmed me 
□ 
□ 
□ 
□ 
□ 
I felt uneasy 
□ 
□ 
□ 
□ 
□ 
 
 
PROMIS Depression 
 
Please respond to each question or statement by marking one box per row 
In the past 7 days… 
Never 
Rarely 
Sometimes 
Often 
Always 
I felt worthless 
□ 
□ 
□ 
□ 
□ 
I felt helpless 
□ 
□ 
□ 
□ 
□ 
I felt depressed 
□ 
□ 
□ 
□ 
□ 
I felt hopeless 
□ 
□ 
□ 
□ 
□ 
 
 
 

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PROMIS Sleep Disturbance 
At Day 3-4 time point, the recall period of these questions will be changed from 7 days to 1 day. 
 
 
Please respond to each question or statement by marking one box per row 
In the past 7 days… 
Very poor 
Poor 
Fair 
Good 
Very good 
My sleep quality was 
□ 
□ 
□ 
□ 
□ 
 
Please respond to each question or statement by marking one box per row 
In the past 7 days… 
Not at all 
A little bit 
Somewhat Quite a bit Very much 
My sleep was refreshing 
□ 
□ 
□ 
□ 
□ 
I had a problem with my sleep 
□ 
□ 
□ 
□ 
□ 
I had difficulty falling asleep 
□ 
□ 
□ 
□ 
□ 
 
 
 

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EQ-5D 
 
Please mark one box in each group below to indicate which statements best describe your state 
of health today. 
Mobility 
□ I have no problems in walking about 
□ I have some problems in walking about 
□ I am confined to bed 
 
Self-Care 
□ I have no problems with self-care 
□ I have some problems washing or dressing myself 
□ I am unable to wash or dress myself 
 
Usual Activities (e.g. work, study, housework, family or leisure activities) 
□ I have no problems with performing my usual activities 
□ I have some problems with performing my usual activities 
□ I am unable to perform my usual activities 
 
Pain / Discomfort 
□ I have no pain or discomfort 
□ I have moderate pain or discomfort 
□ I have extreme pain or discomfort 
 
Anxiety / Depression 
□ I am not anxious or depressed 
□ I am moderately anxious or depressed 
□ I am extremely anxious or depressed 
 
On a scale of zero to one hundred; with the best state of health you can imagine being 100 and 
the worst state you can imagine being 0.  Which number best describes your state of health 
today? 
Please enter a number between 0 and 100: __________ 
 
 
 
 

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Work Productivity and Activity Impairment (WPAI) 
 
The following questions ask about the effect of your stem cell donation process on your ability to work 
and perform regular activities. Please fill in the blanks or select a response as indicated. 
Are you currently employed (working for pay)? 
□ No 
□ Yes 
 
If yes… 
 
During the past seven days, how many hours did you miss from work because of problems 
associated with your stem cell donation process? Include hours you missed on sick days, times 
you went in late, left early, etc., because of your stem cell donation process. Do not include time you 
missed to participate in this study. 
 
__________ hours 
 
During the past seven days, how many hours did you miss from work because of any other 
reason, such as vacation, holidays, time off to participate in this study? 
 
__________ hours 
 
During the past seven days, how many hours did you actually work? 
 
__________ hours 
 
If you have worked any hours during the past seven days… 
During the past seven days how much did your stem cell donation process affect your 
productivity while you were working? 
 
Think about days you were limited in the amount or kind of work you could do, days you 
accomplished less than you would like, or days you could not do your work as carefully as usual. 
If your stem cell donation process affected your work only a little, choose a low number. Choose 
a high number if your stem cell donation process affected your work a great deal. 
 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
 
Stem cell 
donation 
process had no 
effect on work 
 
 
 
 
 
 
 
 
Stem cell donation 
process completely 
prevented me from 
working 
 
 
 
 

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During the past seven days, how much did your stem cell donation process affect your ability 
to do your regular daily activities, other than work at a job? 
By regular activities, we mean the usual activities you do, such as work around the house, shopping, 
child care, exercising, studying, etc. Think about times you were limited in the amount or kind of 
activities you could do and times you accomplished less than you would like. If your stem cell 
donation process affected your activities only a little, choose a low number. Choose a high number if 
your stem cell donation process affected your activities a great deal. 
 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
□ 
0 
1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
 
Stem cell 
donation process 
had no effect on 
my daily activities 
 
 
 
 
 
 
 
 
Stem cell donation 
process completely 
prevented me from 
doing my daily 
activities 
 

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Healthcare Resource Use (HRU) 
 
Since completing your stem cell donation, have you sought any medical care related to your donation? 
Please enter how many visits you have made for each type of medical care listed below.  
 
For each type of medical care that you have had 1 or more visit, please indicate whether the care was related to donation, the reason 
for the visit/s, how far from your home the medical care was, and how much time the visit took.   
 
 
 
Type of medical care 
Number of visits  
enter whole number 
Was any of the care 
related to your donation? 
Reasons for visit/s 
Approximate 
distance from 
your home 
Time spent seeking
this care, including 
travel and waiting 
room time 
General practitioner 
_____ visits 
□ Related to donation 
□ Not related to donation 
 
 
______ miles 
_____ hours 
Medical specialist 
_____ visits 
□ Related to donation 
□ Not related to donation 
 
 
______ miles 
_____ hours 
Urgent Care 
_____ visits 
□ Related to donation 
□ Not related to donation 
 
 
______ miles 
_____ hours 
Emergency room 
_____ visits 
□ Related to donation 
□ Not related to donation 
 
 
______ miles 
_____ hours 
Social worker or Care 
manager 
_____ visits 
□ Related to donation 
□ Not related to donation 
 
 
______ miles 
_____ hours 
Other, please describe: 
______________________
_____ visits 
□ Related to donation 
□ Not related to donation 
 
 
______ miles 
_____ hours 
 
 
 

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Donation experience 
 
Thinking back over your entire donation journey to date, which statement best 
describes your overall satisfaction? 
□ Completely satisfied. I wouldn’t change a thing. 
□ Moderately satisfied. Some things could have gone better, but overall, I was 
satisfied. 
□ Neither satisfied nor unsatisfied. 
□ Moderately unsatisfied. Some things could have gone better, but overall, I was 
unsatisfied. 
□ Extremely unsatisfied. A lot needs to change. 
 
If called to donate again, would you? (Your answer will not affect your status on the 
Registry) 
□ Yes 
□ No 
□ Unsure 
 
Would you recommend donation to a friend or family member? 
□ Yes 
□ No 
□ Unsure 
 
Did you learn or discover anything after your donation that you wish you had known 
earlier? 
__________________________________________________________________________ 
__________________________________________________________________________ 
 
Reflecting on your donation experience overall, are there any changes you would 
recommend? 
__________________________________________________________________________ 
__________________________________________________________________________ 
 
Is there anything else you would like to share with the researchers about your stem 
cell donation experience? 
__________________________________________________________________________ 
__________________________________________________________________________ 
 
 

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G. Adverse Event Definitions and Grading (CTCAE v5.0) 
 
https://ctep.cancer.gov/protocolDevelopment/electronic_applications/docs/CTCAE_v5_Q
uick_Reference_8.5x11.pdf 
 
 
 

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H. Guidance on Reviewing and Reporting Unanticipated Problems 
 
For more information, please see http://www.hhs.gov/ohrp/policy/advevntguid.html#Q1  
Below are pertinent excerpts from the Office for Human Research Protections (OHRP) 
Guidance on Reviewing and Reporting Unanticipated Problems Involving Risks to Subjects. 
1. What are unanticipated problems? 
The phrase “unanticipated problems involving risks to subjects or others” is found but not 
defined in the HHS regulations at 45 CFR part 46.  OHRP considers unanticipated problems, 
in general, to include any incident, experience, or outcome that meets all of the following 
criteria: 
a. unexpected (in terms of nature, severity, or frequency) given (a) the research 
procedures that are described in the protocol-related documents, such as the 
IRB-approved research protocol and informed consent document; and (b) the 
characteristics of the subject population being studied; 
b. related or possibly related to participation in the research (in this guidance 
document, possibly related means there is a reasonable possibility that the 
incident, experience, or outcome may have been caused by the procedures 
involved in the research); and 
c. suggests that the research places subjects or others at a greater risk of harm 
(including physical, psychological, economic, or social harm) than was 
previously known or recognized. 
OHRP recognizes that it may be difficult to determine whether a particular incident, 
experience, or outcome is unexpected and whether it is related or possibly related to 
participation in the research.  
2.  How do you determine which adverse events are unanticipated problems? 
In OHRP’s experience, most IRB members, investigators, and institutional officials understand 
the scope and meaning of the term adverse event in the research context but lack a clear 
understanding of OHRP’s expectations for what, when, and to whom adverse events need to 
be reported as unanticipated problems, given the requirements of the HHS regulations at 45 
CFR part 46.  
The following Venn diagram summarizes the general relationship between adverse events 
and unanticipated problems:  
 
 
 
 

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To determine whether an adverse event is an unanticipated problem, the following questions 
should be asked:  
a. Is the adverse event unexpected? 
b. Is the adverse event related or possibly related to participation in the 
research? 
c. Does the adverse event suggest that the research places subjects or others 
at a greater risk of harm than was previously known or recognized? 
If the answer to all three questions is yes, then the adverse event is an unanticipated 
problem.