Safety and Feasibility of Sulforaphane to Promote Early Haematopoietic Recovery After Cord Blood Transplantation A Single-arm, Open, Single-centre Clinical Study
Phase: NA
Conditions: ALL (Acute B-Lymphoblastic Leukemia), AML (Acute Myelogenous Leukemia, MDS (Myelodysplastic Syndrome)
Sponsor: Institute of Hematology & Blood Diseases Hospital, China

 
 
 
 
 
Title: A Single-Arm, Open-Label, Single-Center 
Phase I-II Study of the Safety and Feasibility of 
Sulforaphane in Promoting Early Hematopoietic 
Recovery After Umbilical Cord Blood 
Transplantation 
 
 
NCT Number: SFN-UCBT-2025 
Document Date: 7th July 2025 
 
 
 
 
 
 
 

 
Clinical Study Protocol 
Protocol Title 
A Single-Arm, Open-Label, Single-Center Phase I-II 
Clinical Study on the Safety and Feasibility of 
Sulforaphane in Promoting Early Hematopoietic 
Recovery Following Umbilical Cord Blood 
Transplantation 
Research 
Institution 
Institute of Hematology & Blood Diseases Hospital, 
Chinese Academy of Medical Sciences & Peking Union 
Medical College 
Telephone 
+86-22-23909095 
Contact Address 
288 Nanjing Road, Heping District, Tianjin, China 
Version Number 
2.0 
Version Date 
2025-07-07 
 
Confidentiality Statement 
This document contains confidential information of the Institute of Hematology 
& Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking 
Union Medical College. It is to be used solely for the purpose of this clinical 
study. It shall not be disclosed to any individual other than the participating 
researchers and members of the Institutional Review Board (IRB). Without the 
prior written consent of the Institute of Hematology & Blood Diseases Hospital, 
Chinese Academy of Medical Sciences & Peking Union Medical College, this 
information shall not be used for any purpose other than the evaluation or 
conduct of this clinical study. 
 
 
 

 
 
Investigator's Statement 
I hereby confirm that I have thoroughly reviewed this study protocol and 
acknowledge that it contains all necessary components for the proper conduct 
of the study. I fully understand my responsibilities associated with this 
protocol.I agree to perform all related duties in strict compliance with Chinese 
laws and regulations, the Declaration of Helsinki, Good Clinical Practice (GCP) 
guidelines, and this study protocol. I affirm that no procedures outlined in this 
study will be initiated until the protocol has received approval from the Ethics 
Committee and informed consent has been obtained from the subject. Any 
modifications to the protocol require prior approval by the Ethics Committee 
before implementation, except when immediate action is necessary to 
eliminate an immediate hazard to the subject's safety, rights, and welfare. I 
understand and commit to adhering to all requirements regarding the 
maintenance and confidentiality of original source data. 
 
Principal Investigator：                         Date： 
 
Clinical Research Unit: Institute of Hematology & Blood Diseases Hospital, 
Chinese Academy of Medical Sciences & Peking Union Medical College 
 
 
 
 
 
 
 
 

 
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Table of Contents 
 
Abbreviations ............................................................................................................................ 1 
1. Synopsis ................................................................................................................................ 3 
2. Research Background ......................................................................................................... 9 
3. Study Objectives ................................................................................................................ 10 
4. Study Design ....................................................................................................................... 11 
5. Study Population ................................................................................................................ 11 
5.1 Inclusion Criteria .......................................................................................................... 11 
5.2 Exclusion Criteria ......................................................................................................... 11 
5.3 Criteria for Withdrawal and Discontinuation ............................................................. 13 
5.4 Criteria for Exclusion from Analysis .......................................................................... 14 
6. Investigational Product ...................................................................................................... 14 
7. Dosing Regimen ................................................................................................................. 14 
8. Prohibited Medications ...................................................................................................... 15 
9. Transplant Protocol ............................................................................................................ 15 
10. Other/Concomitant Therapies ......................................................................................... 16 
11. Study Schedule ................................................................................................................. 16 
11.1 Screening Period (Approximately 2 Weeks) ............................................................ 16 
11.2 Treatment Period ........................................................................................................ 17 
11.3 Follow-up Period ......................................................................................................... 17 
12. Endpoints .......................................................................................................................... 18 
12.1 Efficacy Endpoints ..................................................................................................... 18 
12.2 Safety Endpoints ........................................................................................................ 18 
13. Adverse Events and Serious Adverse Events ............................................................... 19 
13.1 Adverse Events ........................................................................................................... 19 
13.2 Serious Adverse Events (SAEs) ................................................................................ 21 
14. Data Management ............................................................................................................. 22 
15. Statistical Analysis ........................................................................................................... 22 
15.1 Analysis Sets .............................................................................................................. 22 
15.2 Statistical Methods ..................................................................................................... 23 
16. Ethics ................................................................................................................................. 23 
16.1 Ethical Requirements ................................................................................................. 23 
16.2 Informed Consent ....................................................................................................... 23 
16.3 Subject Confidentiality ............................................................................................... 24 
17. Study Management ........................................................................................................... 24 
17.1 Training ........................................................................................................................ 24 
17.2 Quality Control and Quality Assurance .................................................................... 25 
18. Anticipated Study Timeline and Completion Date ........................................................ 25 
19. Confidentiality and Publication of Results .................................................................... 25 
20. References ........................................................................................................................ 25 
21. Appendic ........................................................................................................................... 25 

 
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Trial Flow Chart....................................................................................................................... 26 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 
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Abbreviations 
Abbreviation/Acronym 
Term 
AE 
Adverse Event 
ALL 
Acute Lymphoblastic Leukemia 
ALT 
Alanine Aminotransferase 
AML 
Acute Myeloid Leukemia 
Ara-C 
Cytarabine 
AST 
Aspartate Aminotransferase 
Bu 
Busulfan 
CR 
Complete response 
CRF 
Case report form 
CsA 
Cyclosporine A 
CY 
Cyclophosphamide 
DFS 
Disease-free survival 
EBV 
Epstein - Barr Virus 
ECOG 
Eastern Cooperative Oncology Group 
FAS 
Full Analysis Set 
FEV1 
Forced Expiratory Volume in 1 second 
Flu 
Fludarabine 
FVC 
Forced Vital Capacity 
GRFS 
GVHD-free and relapse-free survival 
GVHD 
Graft-versus-host disease 
HBsAg 
Hepatitis B Surface Antigen 
HBV 
Hepatitis B virus 
HCMV 
Human Cytomegalovirus 
HCV 
Hepatitis C Virus 
HIV 
Human Immunodeficiency Virus 
HLA 
Human Leukocyte Antigen 
HSC 
Hematopoietic stem cells 
HSPC 
Hematopoietic stem cell transplantation 
LVEF 
Left Ventricular Ejection Fraction 
MDS 
Myelodysplastic Syndromes 
Mel 
Melphalan 
MMF 
Mycophenolate Mofetil 
NIH 
National Institutes of Health 
NR 
No response 
NRM 
Non-relapse mortality 
NYHA 
New York Heart Association 
OS 
Overall survival 
PR 
Partial response 
RFS 
Relapse-free survival 

 
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SAE 
Severe adverse event 
SFN 
Sulforaphane 
SS 
Safety Set 
TP-Ab 
Treponema Pallidum Antibody 
TRM 
Transplant - related mortality 
UCB 
Umbilicol cord blood 
UCBT 
Umbilicol cord blood transplantation 
ULN 
Upper Limits of Nomal 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 
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1 Synopsis 
Date of Protocol 
Synopsis 
2025-07-07 
Study Dietary 
Supplement Name 
Antioxidant Sulforaphane 
Clinical Trial Phase 
I-II 
Study Title 
A Single-Arm, Open-Label, Single-Center Phase 
I-II Clinical Study on the Safety and Feasibility of 
Sulforaphane in Promoting Early Hematopoietic 
Recovery Following Umbilical Cord Blood 
Transplantation 
Planned Study 
Period 
2025-09-01 to 2027-12-31 
Study Objectives: 
Phase I: To evaluate the safety and feasibility of oral sulforaphane 
administration during the peri-infusion period in adults undergoing UCBT. 
Phase II: To demonstrate the non-inferiority of neutrophil recovery time in 
patients receiving umbilical cord blood (UCB) units cryopreserved for ≥10 
years with concomitant sulforaphane administration, compared to the 
efficacy observed in patients receiving UCB units cryopreserved for <10 
years without sulforaphane. 
Study Population: 
1. Patients with high-risk hematologic malignancies: including AML, ALL, 
and high-risk MDS; 
2. Age and Sex: ≥18 years old, no gender restriction; 
3. Karnofsky score ≥70%, and Eastern Cooperative Oncology Group 
(ECOG) performance status ≤2. 
Planned Sample Size: 
Phase I will enroll 4 patients. Upon meeting the safety evaluation criteria in 
Phase I, Phase II will enroll 32 patients for observation. 
Study Design Overview: 
Umbilical cord blood (UCB) is rich in hematopoietic stem/progenitor cells 
and immune cells, and is used for transplantation in various hematologic 
diseases due to advantages such as lower HLA matching requirements and 
fewer transplant-related complications. As of March 2025, over 280,000 
public UCB units are cryopreserved in 7 (8) public cord blood banks in 
China, with units cryopreserved for ≥10 years accounting for 26%, raising 

 
4
concerns regarding their clinical utility. Our preliminary studies indicate that 
long-term cryopreservation impairs mitochondrial function in UCB 
hematopoietic stem/progenitor cells, leading to reduced reconstitution 
capacity and impaired megakaryocytic differentiation. Intervention with the 
antioxidant sulforaphane (SFN) can partially rescue this 
cryopreservation-induced functional impairment. These findings are based 
on studies in immunodeficient animals. Whether SFN intervention can 
promote hematopoietic reconstitution after transplantation using long-term 
cryopreserved (≥10 years) UCB in patients urgently requires clinical 
investigation. This project proposes a single-arm, open-label, single-center 
Phase I-II clinical study to evaluate the safety and feasibility of the dietary 
supplement SFN in promoting early hematopoietic recovery after UCBT. It 
aims to assess the safety and feasibility of SFN use during the peri-infusion 
period in adult transplant patients receiving long-term cryopreserved UCB, 
and to elucidate the impact of peri-infusion SFN use on neutrophil 
engraftment. This project will provide scientific guidance for promoting the 
clinical application of long-term cryopreserved UCB, and key data for 
optimizing UCBT strategies and expanding its application scope. 
Inclusion Criteria （subjects suitable for inclusion in this study must meet 
all of the following criteria）: 
1. Patients with high-risk hematologic malignancies: including AML, ALL, 
and high-risk MDS diagnosed according to World Health Organization 
(WHO) criteria; 
2. Age and Sex: ≥18 years old, no gender restriction; 
3. Karnofsky score ≥70%, and Eastern Cooperative Oncology Group 
(ECOG) performance status ≤2; 
4. Selection of unrelated umbilical cord blood: Donor-recipient HLA 
high-resolution match ≥4/6, 7/10, and post-thaw CD34+ cell count ≥0.83 
× 105/kg (recipient body weight). Furthermore, only UCB units 
cryopreserved for ≥10 years meeting the above criteria are available 
from Chinese public cord blood stem cell banks for the patient. 

 
5
Exclusion Criteria (subjects who meet any of the following criteria will be 
excluded from participation in this study): 
1. Patients with positive results for the following pathogens: Human 
Immunodeficiency Virus (HIV-1/2), Human Cytomegalovirus 
(HCMV-DNA), Epstein-Barr Virus (EBV-DNA), Hepatitis B (Hepatitis B 
surface antigen [HBsAg] positive or Hepatitis B virus DNA [HBV-DNA] 
positive), Hepatitis C antibody (HCV-Ab) positive, or Treponema 
pallidum antibody (TP-Ab) positive; 
2. Active bacterial, viral, fungal, or parasitic infections judged by the 
investigator to be clinically significant at screening; 
3. Availability of an HLA-identical willing donor and eligibility for allogeneic 
hematopoietic stem cell transplantation from that donor; 
4. Prior history of gene therapy or allogeneic hematopoietic stem cell 
transplantation; 
5. First-degree relatives with known or suspected familial cancer syndromes 
(including but not limited to hereditary breast and ovarian cancer 
syndrome, Lynch syndrome, familial adenomatous polyposis, etc.); 
6. Diagnosis of a major psychiatric disorder or predisposition that would 
severely impair the ability to participate in the clinical study; or history of 
psychotropic drug abuse unable to abstain; 
7. History of major organ impairment, including: 
(1) Hepatic impairment: Liver function tests indicating AST or ALT > 3 × 
ULN; total serum bilirubin > 2.5 × ULN; or if consistent with Gilbert's 
syndrome, total bilirubin > 3 × ULN and direct bilirubin > 2.5 × ULN; 
history of bridging fibrosis, cirrhosis, or presence of active hepatitis. 
(2) Cardiac impairment: Left ventricular ejection fraction (LVEF) <45% 
at screening; New York Heart Association (NYHA) Class III or IV 
congestive heart failure; severe arrhythmia requiring treatment; 
uncontrolled hypertension (systolic blood pressure > 160 mmHg 
and/or diastolic blood pressure > 100 mmHg despite 
antihypertensive medication), or history of hypertensive emergency, 
hypertensive encephalopathy, or unstable angina; myocardial 
infarction, coronary artery bypass graft, peripheral artery bypass 
graft/implantation surgery, or stent placement within 12 months prior 
to enrollment; clinically significant valvular heart disease; estimated 
glomerular filtration rate (eGFR) <60 mL/min/1.73m². 
(3) Pulmonary impairment: FEV1/FVC <60% and/or diffusing capacity 
<60% of predicted value; evidence of clinically significant pulmonary 
hypertension requiring medical intervention. 
8. Uncorrectable coagulation dysfunction or history of severe hemorrhagic 
disorder; 
9. Any other condition considered by the physician to render the subject 
unsuitable for hematopoietic stem cell transplantation; 

 
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10. Known allergy to the investigational product or its components; 
11. Participation in another interventional clinical study within 3 months prior 
to screening or current participation; 
12. Administration of live vaccines within 6 weeks prior to screening; 
13. Pregnant or lactating women; 
14. Poor compliance with the study protocol expected from the subject; 
15. Any other condition considered by the investigator to make the subject 
unsuitable for participation in this clinical trial; 
16. Unwillingness to provide pre-existing diagnostic evidence or undergo 
bone marrow examination before treatment, and unwillingness to 
undergo bone marrow and blood examinations after treatment; 
17. Diagnosis of acute promyelocytic leukemia; 
18. Clinically significant gastrointestinal abnormalities that may affect the 
intake, transport, or absorption of SFN (e.g., inability to swallow, chronic 
diarrhea, intestinal obstruction, etc.), or subjects with total gastrectomy; 
19. Subjects with a definite tendency for gastrointestinal bleeding, whom the 
investigator considers at risk for major gastrointestinal hemorrhage; 
20. History of solid organ transplantation. 
Investigational Product: 
Name of Dietary Supplement: Antioxidant Sulforaphane 
Dosage Form: Tablet 
Specification: 30 mg/tablet 
Route of Administration: Oral administration, two tablets each time, three 
times daily, taken with meals or after meals (recommended to be taken with 
a fat-containing meal). 
Source of Dietary Supplement: A sulforaphane-yielding supplement 
containing glucoraphanin and active myrosinase, manufactured by 
Nutramax Laboratories Consumer Care, USA. Brand Name: Avmacol® 
Extra Strength. Detailed component information is provided in the protocol 
appendices. To avoid confounding factors, the dietary supplement used in 
this study protocol will be from a single production batch. 

 
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Dosing Regimen: 
Administration from day -1 (one day before transplantation) to day +7 
(seven days after transplantation). 
Concomitant Therapy: 
1. During the study period, concomitant medical conditions and any adverse 
events (AEs) should be actively managed after evaluating the 
relationship between the AE and the investigational product. 
Prophylactic treatment may also be administered if necessary (Nausea 
and Vomiting: Nausea and vomiting may occur in most subjects during 
chemotherapy. Appropriate antiemetic therapy is recommended to 
prevent or alleviate nausea and vomiting. Prophylactic use of 
antiemetics, such as prochlorperazine or ondansetron, is recommended 
during chemotherapy. Increased monitoring of electrolytes and 
electrocardiogram is advised during medication use). 
2. Unrelated umbilical cord blood transplantation. 
Prohibited Medications: / 
Follow-up Duration: 
Treatment Period: Approximately ±10 days 
Completion of Primary Endpoint Assessment: 2 months post-transplantation 
Follow-up Period: 1 year 
Criteria for Discontinuation of Investigational Product:  
Discontinuation of investigational product does not equate to withdrawal 
from the study. Subjects who discontinue the investigational product should 
continue to complete subsequent study visits as required by the protocol 
(unless they meet criteria for study withdrawal). Study treatment must be 
discontinued if any of the following criteria are met: 
1. The subject is unwilling to continue receiving the investigational product. 
In accordance with the Declaration of Helsinki and the informed consent 
form, the subject has the right to withdraw at any stage of the trial 
without affecting their future medical care and rights. 
2. Occurrence of an adverse event that does not improve or is intolerable 
after optimal medical treatment, and the investigator judges that 
discontinuation of treatment is necessary. 
3. Subjects who discontinue the study are required to complete all 
examinations for the primary endpoints (the investigator should confirm 
whether subjects who withdraw consent are willing to complete all 
procedures of the final visit). 
4. For all subjects who withdraw from the study due to adverse events or 
laboratory abnormalities, follow-up must be conducted until the 

 
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symptoms (or laboratory values) return to their pre-study state or are 
deemed non-clinically significant, and the results recorded in the CRF. 
5. Criteria for subject study discontinuation also include loss to follow-up. If 
a subject misses a visit without withdrawing consent, the investigator 
should make every effort to contact the subject and record the contact 
attempts in the CRF. The investigator has the right to discontinue a 
subject's participation in the study if it is judged that continuation poses 
an unacceptable risk. 
Criteria for Exclusion from Analysis: 
Failure to receive the planned dose of SFN (<50% of the planned dose) 
Study Endpoints: 
Primary Endpoints: 
(1) Safety: Incidence of adverse events, including non-hematological 
adverse events of Grade 3 or higher. 
(2) Efficacy: Time to neutrophil recovery after UCBT. 
Secondary Endpoints: 
(1) Time to platelet engraftment. 
(2) Relationship between CD34+ cell dose and trilineage engraftment. 
(3) Incidence of acute and chronic GVHD. 
(4) Incidence of primary and secondary graft failure. 
(5) Incidence of severe (Grade 3 or higher) infections. 
(6) Transplant-related mortality, relapse rate, duration of response, 1-year 
disease-free survival, overall survival (OS), and GVHD-free relapse-free 
survival (GRFS). 
(7) Incidence of pre-engraftment syndrome. 
(8) Length of hospital stay (length of stay, number of days with fever, 
number of days on parenteral nutrition within the first 100 days 
post-transplant). 
(9) Immune reconstitution (kinetics of immune and hematopoietic stem cell 
recovery). 
(10) 
Incidence of oral intolerance and vomiting. 
(11) 
Volume of red blood cell transfusions and volume of platelet 
transfusions. 
All patients will be followed up until 1 year after transplantation. 
Sample Size Determination and Statistical Methods: 
(1) Basis for Sample Size Determination: 
Based on clinical practice, Phase I will enroll 4 adult patients to evaluate the 

 
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safety and feasibility of oral sulforaphane during the peri-infusion period of 
UCBT. The Phase II objective is to demonstrate the non-inferiority of 
neutrophil recovery time in patients receiving UCB units cryopreserved for 
≥10 years with sulforaphane, compared to the efficacy in patients receiving 
UCB units cryopreserved for <10 years without sulforaphane. Historical data 
indicate a mean neutrophil recovery time of 18 days (standard error: 4 days) 
in patients receiving UCB cryopreserved for <10 years without 
sulforaphane. The expected neutrophil recovery time for patients receiving 
UCB cryopreserved for ≥10 years with sulforaphane is 18 days. Recovery 
time not exceeding 20 days is considered non-inferior to the effect observed 
with UCB cryopreserved for <10 years. Using a one-sided, one-arm 
non-inferiority z-test, with a Type I error (α) of 0.025 and Type II error (β) of 
0.2 (power=80%), the Phase II study requires a total of 32 adult patients. 
(2) Statistical Analysis Methods: 
Quantitative data will be described using medians (interquartile range) or 
mean ± standard deviation. Comparative analyses will employ the t-test or 
Wilcoxon test based on whether the data conform to a normal distribution. 
Qualitative data will be described using absolute numbers/frequencies, with 
comparative analyses using the chi-square test or Fisher's exact test. The 
analysis of post-transplant blood cell recovery time will utilize the Fine-Gray 
competing risks model to account for early death and primary graft failure as 
competing events, reporting the subdistribution hazard ratio (sHR). 
Between-group comparisons for cumulative incidence will be performed 
using Gray's test. 
 
2 Research Background 
Hematopoietic stem cell (HSC) transplantation represents a potentially 
curative therapy for a variety of hematologic disorders. The primary clinical 
sources of HSCs include bone marrow, mobilized peripheral blood, and 
umbilical cord blood (UCB). Among these, UCB offers distinct advantages 
such as rapid availability, less stringent HLA matching requirements, lower 
incidence of severe chronic graft-versus-host disease (GVHD), and a 
theoretically lower risk of latent viral transmission. Consequently, the 
application of UCB transplantation (UCBT) has been progressively 
expanding. 
Globally, over 8 million UCB units are stored in both public and private 
banks, with China's inventory exceeding 2.4 million units. Despite this 
substantial reserve, the clinical utilization rate remains disproportionately 
low, with only approximately 80,000 reported UCBT cases worldwide and 
about 42,000 in China, translating to an overall usage rate of less than 2%. 
A significant contributing factor to this underutilization is clinician 
apprehension regarding the efficacy of long-term cryopreserved units. Data 
from Chinese public cord blood banks indicate that as of March 2025, from a 

 
1
total inventory exceeding 280,000 units, more than 70,000 (approximately 
26%) have been cryopreserved for 10 years or longer. This substantial 
portion of the inventory represents a critical, yet underused, resource. 
While some studies suggest that UCB units cryopreserved for extended 
periods (up to 19 years) retain engraftment potential, a prevailing 
preference for shorter-term cryopreserved units persists in clinical practice, 
largely based on empirical observation rather than robust scientific evidence. 
Our preliminary investigations have systematically demonstrated that the 
cryopreservation process and duration can impair the hematopoietic 
reconstitution capacity of UCB-derived hematopoietic stem and progenitor 
cells (HSPCs), potentially due to accumulated damage such as 
mitochondrial dysfunction and oxidative stress, while sparing immunologic 
function. Notably, we have found that treatment with the antioxidant 
sulforaphane (SFN) can partially rescue this functional impairment in 
preclinical models. 
Sulforaphane (C6H11NOS2), an isothiocyanate derived from its precursor 
glucoraphanin found in cruciferous vegetables, is a potent inducer of 
cytoprotective enzymes via the Nrf2 pathway. Its antioxidant and potential 
cytoprotective properties make it a compelling candidate for mitigating 
cryopreservation-induced cell damage. Commercially available 
SFN-containing supplements have been utilized in other registered clinical 
trials, supporting its safety profile for human consumption. 
This clinical study is therefore designed to translate these preclinical 
findings into a clinical setting. We hypothesize that SFN administration in 
the peri-transplant period to adult patients undergoing UCBT with long-term 
cryopreserved (≥10 years) UCB will promote early hematopoietic recovery, 
achieving engraftment rates comparable to those observed with 
shorter-term cryopreserved units. Confirming this hypothesis would 
significantly increase the available donor pool by "reawakening" this 
dormant inventory, thereby enhancing patient access to this potentially 
life-saving therapy and maximizing the return on investment in public cord 
blood banking. This trial aims to provide the necessary clinical evidence to 
address a critical unmet need in the field of transplantation medicine. 
 
3 Study Objectives 
Primary Objectives: 
(1) To determine the safety and feasibility of SFN use during the 
peri-infusion period of UCBT in adults. 
(2) To determine the impact of SFN use during the peri-infusion period 

 
1
of UCBT on hematopoietic recovery, particularly neutrophil 
engraftment. 
Secondary Objectives: 
(1) To establish a high-quality clinical cohort of adult patients 
transplanted with long-term cryopreserved umbilical cord blood. 
 
4 Study Design 
This is a single-arm, open-label, single-center study. Phase I will enroll 4 
patients. Upon meeting the safety evaluation criteria in Phase I, Phase II 
will enroll 32 patients for observation. 
 
5 Study Population 
5.1 Inclusion Criteria 
Subjects must meet all of the following criteria to be eligible for the study: 
(1) Patients with high-risk hematologic malignancies: including Acute 
Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), or 
high-risk Myelodysplastic Syndromes (MDS) diagnosed according 
to World Health Organization (WHO) criteria. 
(2) Age and Sex: ≥18 years old, no gender restriction. 
(3) Karnofsky Performance Status (KPS) score ≥70%, and Eastern 
Cooperative Oncology Group (ECOG) performance status of ≤2. 
(4) Selection of Unrelated Donor Umbilical Cord Blood (UCB): The UCB 
unit must meet the following criteria: donor-recipient high-resolution 
HLA match ≥4/6 or 7/10, and a post-thaw CD34+ cell dose ≥0.83 × 
10^5/kg (recipient body weight). Furthermore, only UCB units 
cryopreserved for ≥10 years meeting these criteria are available for 
the patient from Chinese public cord blood stem cell banks. 
5.2 Exclusion Criteria 
Subjects who meet any of the following criteria will be excluded from 
participation: 
(1) Positive test results for any of the following: Human 
Immunodeficiency Virus (HIV-1/2), detectable Human 
Cytomegalovirus DNA (HCMV-DNA), detectable Epstein-Barr Virus 

 
1
DNA (EBV-DNA), Hepatitis B (positive for Hepatitis B surface 
antigen [HBsAg] or detectable Hepatitis B virus DNA [HBV-DNA]), 
positive Hepatitis C antibody (HCV-Ab), or positive Treponema 
pallidum antibody (TP-Ab). 
(2) Active bacterial, viral, fungal, or parasitic infection judged by the 
investigator to be clinically significant at the time of screening. 
(3) Availability of an HLA-identical willing donor and eligibility for 
allogeneic hematopoietic stem cell transplantation from that donor. 
(4) Prior receipt of gene therapy or allogeneic hematopoietic stem cell 
transplantation. 
(5) First-degree relative(s) with a known or suspected hereditary cancer 
syndrome (including, but not limited to, hereditary breast and 
ovarian cancer syndrome, Lynch syndrome, familial adenomatous 
polyposis, etc.). 
(6) Diagnosis of a major psychiatric disorder or condition that would 
severely impair the ability to participate in the clinical study; or 
history of substance abuse related to psychotropic drugs and 
inability to abstain. 
(7) History of significant organ dysfunction, including:  
1) Hepatic: Liver function tests indicating AST or ALT > 3 × Upper 
Limit of Normal (ULN); total serum bilirubin > 2.5 × ULN; or, if 
consistent with Gilbert's syndrome, total bilirubin > 3 × ULN with 
direct bilirubin > 2.5 × ULN; history of bridging hepatic fibrosis, 
cirrhosis, or presence of active hepatitis. 
2) Cardiac: Left ventricular ejection fraction (LVEF) <45% at 
screening; New York Heart Association (NYHA) Class III or IV 
congestive heart failure; severe arrhythmia requiring treatment; 
uncontrolled hypertension (systolic blood pressure > 160 mmHg 
and/or diastolic blood pressure > 100 mmHg despite 
antihypertensive medication), or history of hypertensive 
emergency, hypertensive encephalopathy, or unstable angina; 
myocardial infarction, coronary artery bypass grafting, 
peripheral artery bypass graft/implantation surgery, or stent 
placement within 12 months prior to enrollment; clinically 
significant valvular heart disease; estimated glomerular filtration 
rate (eGFR) <60 mL/min/1.73m². 
3) Pulmonary: FEV1/FVC ratio <60% and/or diffusing capacity for 
carbon monoxide (DLCO) <60% of predicted value; evidence of 
clinically significant pulmonary hypertension requiring medical 

 
1
intervention. 
(8) Uncorrectable coagulation dysfunction or history of a severe 
hemorrhagic disorder. 
(9) Any other condition considered by the physician to render the 
subject unsuitable for hematopoietic stem cell transplantation. 
(10) 
Known allergy or hypersensitivity to the investigational product 
(sulforaphane) or any of its components. 
(11) 
Participation in another interventional clinical study within 3 
months prior to screening or current participation. 
(12) 
Administration of live attenuated vaccines within 6 weeks prior 
to screening. 
(13) 
Pregnant or lactating women. 
(14) 
Poor compliance with the study protocol expected from the 
subject, as judged by the investigator. 
(15) 
Any other condition considered by the investigator to make the 
subject unsuitable for participation in this clinical trial. 
(16) 
Unwillingness to provide valid pre-existing diagnostic evidence 
or undergo bone marrow examination before treatment, and/or 
unwillingness to undergo bone marrow and blood examinations 
after treatment. 
(17) 
Diagnosis of acute promyelocytic leukemia (APL). 
(18) 
Clinically significant gastrointestinal abnormalities that may 
affect drug intake, transport, or absorption (e.g., inability to swallow, 
chronic diarrhea, intestinal obstruction, etc.), or subjects who have 
undergone total gastrectomy. 
(19) 
Subjects with a definite tendency for gastrointestinal bleeding, 
whom the investigator judges to be at risk for major gastrointestinal 
hemorrhage. 
(20) 
History of solid organ transplantation. 
5.3 Criteria for Withdrawal and Discontinuation 
(1) Subjects may withdraw their informed consent and leave the study 
at any time if they are unwilling to continue participation. Subjects 
deciding to withdraw should notify the investigator promptly. 
(2) The reason for withdrawal must be recorded in the Case Report 
Form (CRF), including the date of and reason for withdrawal. 

 
1
(3) Subjects who discontinue the study are required to complete all 
procedures specified for the final visit (the investigator should 
confirm whether subjects who withdraw consent are willing to 
complete all final visit procedures). 
(4) All subjects who withdraw due to adverse events or laboratory 
abnormalities must be followed up until the symptoms (or laboratory 
values) return to their pre-study state or are deemed non-clinically 
significant. The results of this follow-up must be recorded in the 
CRF. 
(5) Criteria for study discontinuation also include loss to follow-up. If a 
subject misses a scheduled visit without formally withdrawing 
consent, the investigator must make reasonable efforts to contact 
the subject and document these contact attempts in the CRF. The 
investigator has the right to discontinue a subject's participation if it 
is judged that continuation poses an unacceptable risk to the 
subject. 
5.4 Criteria for Exclusion from Analysis 
Failure to receive the planned dose of SFN (defined as receiving <50% of 
the total planned dose). 
 
6 Investigational Product 
Name of Dietary Supplement: Antioxidant Sulforaphane 
Dosage Form and Strength: 30 mg/tablet 
Route of Administration: Oral administration, two tablets each time, three 
times daily, to be taken with meals or after meals (recommended to be taken 
with a fat-containing meal). 
Source of Dietary Supplement: A sulforaphane-yielding supplement 
containing glucoraphanin and active myrosinase, manufactured by 
Nutramax Laboratories Consumer Care, USA. Brand Name: Avmacol® 
Extra Strength. Detailed component information is provided in the protocol 
appendices. To minimize variability, the dietary supplement used in this 
study will be from a single manufacturing batch. 
 
7 Dosing Regimen 
(1) Administration starts on day -1 (one day before transplantation) and 

 
1
continues until day +7 (seven days after transplantation). 
If vomiting occurs after drug intake and intact tablets are visible in the 
vomitus, the dose should be re-administered based on the number of 
tablets identified. 
 
8 Prohibited Medications 
/ 
 
9 Transplant Protocol 
Conditioning Regimen: A dose-stratified design is used, adjusting 
intensity based on patient age and comorbidities: 
(1) For patients aged ≤50 years and ECOG ≤1: Myeloablative 
regimen (Flu/Bu/Cy or Flu/Bu/Mel): 
1) Flu/Bu/Cy: Fludarabine (Flu) 30mg/m² daily for 4 days; Busulfan 
(BU) 0.8mg/kg every 6 hours for 4 days; Cyclophosphamide (Cy) 
60mg/kg daily for 2 days. 
2) Flu/Bu/Mel: Flu and Bu as above; Melphalan (Mel) 
80-100mg/m² daily for 1 day. 
(2) For patients aged >50 years or ECOG ≥2: Myeloablative regimen 
(Flu/Ara-C/Bu/Mel): 
1) Flu 30mg/m² daily for 4 days; Granulocyte Colony-Stimulating 
Factor (G-CSF) 5μg/kg daily for 3 days; Cytarabine (Ara-c) 
1.5-2.0g/m² daily for 3 days; Bu 0.8mg/kg every 6 hours for 3 
days; Mel 80-100mg/m² daily for 1 days. 
For patients with a history of meningeal leukemia, high-risk factors for 
meningeal leukemia, or extramedullary disease: Add Semustine (250 
mg/m²) or Thiotepa (8-10 mg/kg daily for 1 day). 
For patients with active disease (not in remission) at transplantation: 
Add Decitabine (20 mg/m²/day for 3 days) or Thiotepa (8-10 mg/kg daily 
for 1 day). 
 
GVHD Prophylaxis: Cyclosporine A (CsA) combined with short-course 
Mycophenolate Mofetil (MMF). 

 
1
(1) CsA: Starting on day -1 at 2.5 mg/kg/day via continuous 24-hour 
intravenous infusion, targeting a blood concentration of 200-300 
ng/mL. After granulocyte engraftment, switch to oral 
administration at twice the intravenous dose. CsA tapering 
begins at 2 months post-transplant based on disease relapse 
risk, GVHD, and infection status, with discontinuation planned 
at 5-6 months post-transplant. 
(2) MMF: 30 mg/kg/day (in two divided doses), starting on day +1. 
Tapering begins after granulocyte engraftment, with gradual 
discontinuation by 3-4 months post-transplant. 
 
Other Supportive Care: 
Other supportive care measures will be administered according to the 
institution's standard transplant protocols. 
 
10 Other/Concomitant Therapies 
(1) Transfusion Support: All blood products used during the transplant 
process must be irradiated. 
(2) Treatment of Underlying Conditions: e.g., antihypertensive 
medication for hypertensive patients; antiviral therapy for patients 
with chronic hepatitis B with regular monitoring of liver function and 
HBV-DNA levels. 
(3) Other Disease-Related Supportive Therapies. 
(4) From the date of signing the informed consent form until the final 
study visit, the investigator must record all treatments for underlying 
conditions, supportive care, and concomitant medications (including 
start/stop dates and indications) in the subject's source documents 
and the CRF. 
 
11 Study Schedule 
11.1 Screening Period (Approximately 2 Weeks) 
(1) Medical History Collection: 
1) Medication history prior to disease onset. 

 
1
2) Recent history of infections. 
3) Pregnancy status. 
4) Prior history of transplantation. 
(2) Standard Pre-Transplant Workup: 
1) Bone marrow examination (bone marrow aspirate/biopsy, 
immunophenotyping, cytogenetics, molecular studies, etc.). 
2) Complete blood count with reticulocytes, comprehensive 
biochemistry panel, coagulation profile, thyroid function tests, 
urinalysis, stool routine plus occult blood test, immunoglobulin 
levels, immune function assessments (T, B, NK cells, TCR 
repertoire, etc.). 
3) Pulmonary function tests, cardiac ultrasound, abdominal 
ultrasound, chest CT/X-ray, electrocardiogram, head MRI, etc. 
4) Pathogen testing: HIV-1/2, HBsAg, HBV-DNA, HCV-Ab, TP-Ab, 
CMV-DNA, EBV-DNA, etc. 
5) Multidisciplinary team assessment. 
11.2 Treatment Period 
(1) Pre-transplant Baseline: Collect samples for serum cytokines, 
immune cell subsets, etc. 
(2) Day 0 to 1 Year Post-Transplant: Monitor complete blood count daily 
post-transplant until neutrophil/platelet/reticulocyte engraftment, 
then periodically thereafter. Donor chimerism assessments will be 
performed on days +3, +5, +7, +14, +21, +28, and at months 2, 3, 6, 
9, and 12 post-transplant to monitor dynamic changes. 
11.3 Follow-up Period 
(1) Assessments at Months 1, 2, 3, 4, 6, 9, and 12 post-transplant: 
Immune cell subsets, acute GVHD grading (Glucksberg/IBMTR 
criteria), chronic GVHD assessment (NIH criteria), monitoring of 
CMV/EBV viral load, infection surveillance, etc. 
(2) All patients will be followed until 1 year post-transplant: Outcomes 
including non-relapse mortality (NRM), disease-free survival (DFS), 
overall survival (OS), and GVHD-free relapse-free survival (GRFS) 
will be calculated. 
 

 
1
12 Endpoints 
12.1 Efficacy Endpoints 
(1) Neutrophil Engraftment: The first of three consecutive days with an 
absolute neutrophil count (ANC) ≥ 0.5 × 10⁹/L after transplantation. 
(2) Platelet Engraftment: The first of seven consecutive days with a 
platelet count ≥ 20 × 10⁹/L without transfusion support after 
transplantation. 
(3) Primary Graft Failure (PGF): Failure to achieve neutrophil recovery 
(as defined above) by day +28, or donor chimerism <5% in whole 
blood or bone marrow at day +28. 
(4) Relapse: Reappearance of ≥5% blasts in the bone marrow after 
achieving complete remission (CR); or detection of blasts in the 
peripheral blood on two separate occasions at least one week apart; 
or development of new extramedullary disease. 
(5) Transplant-Related Mortality (TRM): Death from any cause other 
than relapse of the underlying disease. 
(6) Overall Survival (OS): The time from the initiation of study treatment 
(transplantation) to death from any cause. 
(7) Relapse-Free Survival (RFS): The time from the date of first 
achieving CR to the date of disease relapse or death from any 
cause, whichever occurs first. 
(8) Disease-Free Survival (DFS): The time from the date of achieving 
CR after transplantation to the date of disease relapse or death from 
any cause, whichever occurs first. 
(9) GVHD-free, Relapse-Free Survival (GRFS): The time from 
transplantation to the occurrence of any of the following events: 
grades III-IV acute GVHD, chronic GVHD requiring systemic 
treatment, disease relapse, or death from any cause, whichever 
occurs first. 
12.2 Safety Endpoints 
(1) Tolerance of SFN in UCBT patients and incidence of grade 3-4 
non-hematological toxicities. Dose modifications or interruptions will 
be determined based on grading according to CTCAE (Common 
Terminology Criteria for Adverse Events). 
(2) Transplant-related complications: Incidence of acute GVHD, 

 
1
incidence of infections, and TRM. 
 
13 Adverse Events and Serious Adverse Events 
All Adverse Events (AEs) occurring from the time of signing the informed 
consent form until the final study visit will be recorded. AEs will be graded 
and recorded according to the NCI CTCAE version 5.0. 
13.1 Adverse Events 
13.1.1 Definition of an Adverse Event 
An Adverse Event (AE) is any untoward medical occurrence in a subject 
administered a pharmaceutical product or undergoing a medical 
procedure, which does not necessarily have a causal relationship with this 
treatment or procedure. An AE can therefore be any unfavorable and 
unintended sign (including an abnormal laboratory finding), symptom, or 
disease temporally associated with the subject's participation in the 
research, whether or not considered related to the research intervention. 
This includes any new event or worsening of a pre-existing condition 
relative to the baseline state. Adverse Events include Serious Adverse 
Events (SAEs), non-serious AEs, and abnormal laboratory findings. 
 
13.1.2 Collection and Recording of Adverse Events 
All AEs occurring from the date of signing the informed consent form until 
the final study visit must be recorded. The investigator should report all 
AEs using concise medical terminology. 
 
13.1.3 Criteria for Determining the Severity of Adverse Events 
The severity of AEs will be graded using the National Cancer Institute's 
Common Terminology Criteria for Adverse Events (CTCAE) version 5.0, 
on a scale from Grade 1 (mild) to Grade 5 (death related to AE). 
Grading 
Criteria 
Grade 1 
Mild; asymptomatic or mild symptoms; clinical or diagnostic 
observations only; intervention not indicated. 
Grade 2 
Moderate; minimal, local, or noninvasive intervention 
indicated; limiting age-appropriate instrumental Activities of 
Daily Living (IADL)*. 

 
2
Grade 3 
Severe or medically significant but not immediately 
life-threatening; hospitalization or prolongation of 
hospitalization indicated; disabling; limiting self-care Activities 
of Daily Living (ADL)†. 
Grade 4 
Life-threatening consequences; urgent intervention indicated. 
Grade 5 
Death related to Adverse Event. 
*Instrumental Activities of Daily Living (IADL) refer to activities such as cooking, 
shopping, using the telephone, managing money, etc. 
†Self-care Activities of Daily Living (ADL) refer to activities such as bathing, 
dressing and undressing, eating, using the toilet, taking medications, and not 
bedridden. 
Note: It is important to distinguish between the severity of an adverse event and its 
intensity. The term "severe" is used to describe intensity, which does not 
necessarily qualify an event as a Serious Adverse Event (SAE). For example, a 
headache may be intense ("severe" in intensity) but is not an SAE unless it meets 
the specific SAE criteria. 
 
13.1.4 Criteria for Assessing the Relationship between an Adverse 
Event and the Investigational Product 
The investigator shall analyze the relationship between an adverse event 
and the investigational product based on the principles of causality 
assessment, categorized into five classes: "Definitely Related," "Probably 
Related," "Possibly Related," "Definitely Not Related," and "Cannot Be 
Determined." Events judged as "Definitely Related," "Probably Related," 
"Possibly Related," or "Cannot Be Determined" are considered Adverse 
Drug Reactions (ADRs). The investigator must record all ADRs using 
medical terminology. 
Causality 
Reasonable 
Temporal 
Relationshi
p? 
Consistent 
with Known 
Adverse 
Reaction 
Profile 
Alternativ
e Etiology 
Plausible 
Definitely Related 
Yes 
Yes 
Yes 
Probably Related 
Yes 
Yes/No 
Uncertain 
Possibly Related 
No 
No 
Uncertain 
Definitely Not 
Related 
No 
No 
Yes 

 
2
Cannot Be 
Determined 
Essential information for assessment is 
unavailable 
 
13.2 Serious Adverse Events (SAEs) 
13.2.1 Definition of a Serious Adverse Event:  
A Serious Adverse Event (SAE) is any adverse event that meets one or 
more of the following criteria: 
(1) Results in death or is life-threatening; 
(2) Requires inpatient hospitalization or prolongation of existing 
hospitalization; 
(3) Results in persistent or significant disability/incapacity; 
(4) Is a congenital anomaly/birth defect; 
(5) Is another medically significant event that, based on appropriate 
medical judgment, may jeopardize the subject's health and may 
require medical or surgical intervention to prevent one of the 
outcomes listed above. 
 
The following hospitalizations are generally not considered SAEs: 
(1) Hospitalization due to disease progression in subjects who have 
withdrawn or dropped out from the study; 
(2) Pre-planned hospitalizations for routine treatment or observation of 
the condition under investigation, without worsening of the 
condition; 
(3) Hospitalizations that were scheduled prior to the subject's 
enrollment in the study, with no worsening of pre-existing 
symptoms; 
(4) Treatment in an emergency room or outpatient setting that does not 
meet any of the SAE criteria above and does not result in 
hospitalization; 
(5) Pregnancy itself is not an SAE. However, it must be reported on the 
SAE form or a dedicated pregnancy form and followed up to 
document the outcome, including spontaneous abortion, elective 
termination, birth details, and the presence or absence of congenital 
anomalies. 

 
2
 
13.2.2 Reporting of Serious Adverse Events 
If a subject experiences a Grade 5 (fatal) SAE during the study period 
(from the date of signing the informed consent until the final visit) that is 
assessed by the investigator as possibly related to the investigational 
product, the investigator must complete an SAE report form. This report 
must be submitted to the local Institutional Review Board (IRB)/Ethics 
Committee (EC) at the study site within 24 hours of the investigator 
becoming aware of the event. The report must be signed and dated by the 
investigator. 
 
Medical Ethics Committee of Institute of Hematology & Blood Diseases 
Hospital, CAMS & PUMC 
Telephone: +86-22-23909095 
Email: ec@ihcams.ac.cn 
Address: 288 Nanjing Road, Tianjin, China 
 
14 Data Management 
Case Report Forms (CRFs) will be used for data collection in this study. 
The research site will designate specific personnel to enter data from the 
CRFs into the study database. A separate designated person will verify the 
consistency between the database entries and the source CRF data. 
The data manager will check the data in the database against the clinical 
study protocol. Any discrepancies or queries will be documented in a Data 
Query Form, which will be sent to the investigator for resolution. All Data 
Query Forms shall be retained. 
The data manager will be responsible for exporting the final, cleaned data 
from the database for statistical analysis. 
 
15 Statistical Analysis 
15.1 Analysis Sets 
Full Analysis Set (FAS): Includes data from all enrolled subjects. Used for 
reporting compliance and all baseline characteristics. The primary efficacy 

 
2
analysis will be based on the FAS. 
Safety Set (SS): Includes all enrolled subjects who received at least one 
dose of the study intervention and have at least one post-treatment safety 
assessment. Safety analyses will be based on the SS. 
15.2 Statistical Methods 
Quantitative data will be described using medians (interquartile range) or 
mean ± standard deviation. Comparative analyses will employ the Student's 
t-test or the Wilcoxon rank-sum test based on the normality of the data 
distribution. Qualitative data will be described using absolute numbers and 
frequencies, with comparative analyses using the Chi-square test or 
Fisher's exact test, as appropriate. The cumulative incidence of 
post-transplant hematological recovery will be estimated using the 
Fine-Gray competing risks model to account for competing events (e.g., 
early death, graft failure). Comparisons of cumulative incidence curves 
between groups will be performed using Gray's test. 
 
16 Ethics 
16.1 Ethical Requirements 
The conduct of this study will adhere to the principles of the current 
version of the Declaration of Helsinki (2013), relevant regulations, and the 
approval opinions of the Ethics Committee. 
Before the study begins, the investigator must obtain written approval 
from the relevant regulatory bodies for the study protocol, informed 
consent form (ICF), subject recruitment procedures, and any other written 
information to be provided to subjects. During the study, any amendments 
to the protocol, ICF, etc., must receive written approval from the relevant 
regulatory bodies before implementation. 
 
16.2 Informed Consent 
The investigator or their authorized representative is responsible for 
explaining the study background, the pharmacological characteristics of 
the investigational product, the study procedures, and the potential 
benefits and risks of participation to each potential subject (if applicable), 
and/or the subject's parent(s)/legal guardian(s). Written informed consent, 
signed and dated by the subject (if applicable), the subject's 
parent(s)/legal guardian(s), and the investigating physician, must be 

 
2
obtained before any study-specific procedures are performed (i.e., before 
screening examinations). 
The final ICF text shall include: the study purpose, procedures, subject 
responsibilities, foreseeable benefits, risks, and inconveniences; 
availability of treatment for study-related injury; access to study data and 
confidentiality of subject information. 
The ICF must be approved in writing by the relevant regulatory bodies and 
written in a language understandable to the subject (if applicable) and/or 
the subject's parent(s)/legal guardian(s). The subject (if applicable), the 
subject's parent(s)/legal guardian(s), and the investigator obtaining 
consent must all sign and date the ICF. The original signed ICF shall be 
retained by both the investigator and the subject (or their guardian). If 
significant new information relevant to the study emerges, a revised ICF 
must be prepared, submitted for regulatory approval, and re-consent must 
be obtained. 
For subjects who cannot personally participate in the consent process, 
written informed consent must be obtained from the subject's 
parent(s)/legal guardian(s), and all study procedures must be fully 
explained to them. 
 
16.3 Subject Confidentiality 
The investigator is responsible for maintaining subject anonymity. Subject 
identification on the CRF or other study documents shall use only subject 
initials, numbers, and/or a unique code, not the subject's name. The 
investigator must maintain a confidential subject identification list that links 
the subject's code to their name and contact information. The investigator 
must ensure the confidentiality of all documents that could identify 
individual subjects. 
 
17 Study Management 
17.1 Training 
Prior to the initiation of the clinical study, the investigators shall receive 
training on the study protocol. They must thoroughly read and understand 
the content of this clinical study protocol, unify data recording methods 
and assessment criteria, and strictly adhere to the protocol during 
conduct. 

 
2
17.2 Quality Control and Quality Assurance 
All observations and abnormal findings obtained during the clinical study 
must be promptly and carefully verified and recorded to ensure data 
reliability. The investigator will enter the information required by the 
protocol into the CRFs. The accuracy and completeness of the CRF 
entries will be verified by monitors. 
 
18 Anticipated Study Timeline and Completion Date 
The anticipated study timeline is as follows: 
September 2025 - December 2025: Complete enrollment of 4 patients in 
Phase I and conduct a preliminary evaluation of the safety and feasibility 
of SFN use during the peri-transplant period. 
December 2025 - December 2026: Complete enrollment of 32 patients in 
Phase II and evaluate the impact of peri-transplant SFN use on 
hematopoietic engraftment parameters. 
First Subject Enrolled: September 2025 
Last Subject Completed: December 2027 
Data Management, Statistical Analysis, and Clinical Study Report: Clinical 
data from all subjects will be collected by December 2027 for statistical 
analysis to elucidate the safety and feasibility of peri-transplant SFN use 
and its impact on hematopoietic engraftment. 
 
19 Confidentiality and Publication of Results 
The investigators shall maintain the confidentiality of all information and 
data related to this study. Research results or materials shall not be cited 
or published without the consent of the sponsoring institution. 
 
20 References 
 
21 Appendic

 
2
 
Trial Flow Chart 
Process 
 
Screenin
g Period 
Conditioning 
Regimen 
Period 
Transplantation Time (SFN Administration Time) 
Safety Follow 
- up 
Survival Follow - 
up 
 
D - 28 to 
D - 8 
D - 7 
to D - 
2 
D-1 
D0 
(+1) 
D1 
(+2) 
D2 
(+3) 
D3 
(+4) 
D4 
(+5
) 
D5 
(+6) 
D6 
(+7
) 
D7 
(+8) 
D8 to D30 
D14 
D21 
D28 
30 days after 
transplantation 
30 days, 60 days, 
90 days, 120 
days, 180 days, 
270 days, 365 
days after 
transplantation 
Time Window (days) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Baseline Data 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Sign 
Informed 
Consent Form 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographic Data 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Medical 
History 
and 
Treatment 
History 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusio
n Criteria 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Laboratory 
Tests 
and 
Other 
Examinations 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Pulmonary 
Function 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Electrocardiogram 
(ECG) 
X 
 
If necessary 
 
 
 
 
 

 
2
Cardiac ultrasound 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Chest CT/X - ray 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Abdominal 
ultrasound 
X 
 
If necessary 
 
 
 
 
 
Complete 
Blood 
Count 
+ 
Reticulocyte 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X （Tested 
daily 
before 
three 
- 
lineage 
engraftment） 
X 
X 
X 
 
 
Blood 
Biochemistry 
X 
 
X 
 
X 
 
X 
 
X 
 
X 
X 
 
 
 
 
 
Thyroid Function 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Coagulation 
Function 
X 
If necessary 
 
 
 
 
 
Routine Urine Test 
X 
If necessary 
 
 
 
 
 
Routine Stool Test 
X 
If necessary 
 
 
 
 
 
Etiological 
Detection (HIV - 
1/2, HBsAg, HBV - 
DNA, HCV - Ab, 
TP - Ab) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
CMV - DNA 
X 
 
 
 
X 
 
X 
 
X 
 
X 
 
 
 
 
 
 
EBV - DNA 
X 
 
 
 
X 
 
X 
 
X 
 
X 
 
 
 
 
 
 
Immunoglobulin 
X 
 
 
 
 
 
 
 
 
 
X 
X 
X 
 
X 
 
 

 
2
Immune Function 
(T, B, NK, TCR, 
etc.) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
X 
Other 
Clinical 
Evaluations 
and 
Examinations 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Karnofsky Score 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
HCT - CI 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
ECOG Score 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Physical 
Examination 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
 
Vital Signs 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
 
Body Weight 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
 
GF 
 
 
 
 
 
 
 
 
 
 
 
X 
X 
 
X 
 
 
GVHD 
 
 
 
 
 
 
 
 
 
 
 
X 
X 
 
X 
 
X 
PES 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
 
 
Infection 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
X 
Bone 
Marrow 
Examination 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 
2
Bone 
Marrow 
Morphology 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
X 
Immunophenotypi
ng 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
X 
Karyotype 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
X 
Minimal 
Residual 
Disease 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
X 
Chimerism 
X 
 
 
 
 
 
 
 
 
X 
 
 
X 
X 
X 
 
X 
Patient - Reported 
Outcome 
(PRO) 
Scale Filling 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
EQ - 5D - 5L Scale 
X 
 
 
X 
 
X 
 
X 
 
X 
 
 
X 
 
X 
 
 
Medication 
and 
Subject 
Management 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
SFN 
Administration 
 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
Concomitant 
Medication/Treatm
ent 
 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
Adverse Events 
 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
Survival Follow - 
up 
 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
X 
 
Notes: 

 
3
1) 
Medical History and Treatment History: Document all prior diagnoses and treatments, including findings from the initial diagnosis and relevant 
therapeutic history. This includes medical history and treatments other than AML/ALL/MDS, such as: prior history of cardiac disease (e.g., heart failure, 
coronary artery disease, angina pectoris), history of other malignancies (if applicable, details of treatment history should be collected whenever 
possible), thrombotic events within 6 months prior to randomization, hypertension, history of psychoactive substance abuse, history of recent live 
vaccination and any planned vaccinations, and history of organ transplantation. 
2) 
Baseline Assessments: All baseline results must be obtained within 28 days prior to randomization. For abdominal ultrasound, cardiac ultrasound, and 
chest CT or X-ray, results acknowledged by the Investigator as valid are acceptable. 
3) 
Microbiological Serology/PCR Testing: Must include testing for Human Immunodeficiency Virus (HIV-1/2), Human Cytomegalovirus (HCMV-DNA), 
Epstein-Barr Virus (EBV-DNA), Hepatitis B (positive Hepatitis B surface antigen [HBsAg] or detectable Hepatitis B virus DNA [HBV-DNA]), Hepatitis C 
antibody (HCV-Ab), and Treponema pallidum antibody (TP-Ab). 
4) 
Blood Pregnancy Test: Required only for female subjects of childbearing potential. 
5) 
Bone Marrow Examinations: Include bone marrow cytomorphology, immunophenotyping, chromosome karyotype, bone marrow pathology, and 
assessment for Minimal Residual Disease (MRD). 
⚫ 
Bone Marrow Cytomorphology: Must be sent to the central laboratory for analysis. The sample used for screening should be collected after the last 
prior therapy and within 28 days prior to randomization (historical slides meeting these criteria are acceptable; samples outside this window are not 
permitted – a new sample must be collected if the window is exceeded). 
⚫ 
Immunophenotyping and Chromosome Karyotype (Screening): For the screening period, results from tests performed after the last prior therapy and 
within 28 days prior to randomization may be accepted if acknowledged by the Investigator (results from Grade A tertiary hospitals or qualified 
third-party laboratories are acceptable). Immunophenotyping is only required during the screening period. Bone marrow samples are required in 
principle; however, peripheral blood may be substituted if no bone marrow sample is available and the Investigator has sufficient grounds to deem it 
adequate for analysis. Enrollment confirmation may proceed without awaiting this result if the Investigator confirms the diagnosis based on available 
information. 
⚫ 
Chromosome Karyotype: Is required only during screening (Note: enrollment confirmation does not require this result to be available) and at the time of 
definitive treatment discontinuation. Bone marrow samples are required for testing; peripheral blood may be used only if a bone marrow sample cannot 
be obtained. 
⚫ 
Minimal Residual Disease (MRD): Is required only during the treatment period and at the time of definitive treatment discontinuation (not during 

 
3
screening). All MRD assessments must be sent to the central laboratory for analysis. 
6) 
Concomitant Medications/Therapies: The collection period for concomitant medications/therapies spans from 28 days prior to randomization until the 
end of the safety follow-up period (within 30 ± 7 days after the last dose of study drug). All concomitant medications/therapies used by the subject and 
the reasons for their use must be fully documented in the source records and the electronic Case Report Form (eCRF). After the safety follow-up period 
ends, only concomitant medications and therapies related to Adverse Events (AEs) or Serious Adverse Events (SAEs) associated with the 
investigational product are recorded. 
7) 
Survival Follow-up: Beginning 30 days after the subject's last dose of study drug, survival follow-up should be conducted every 90 days (±14 days). For 
subjects who are randomized but do not receive study treatment, the start date for follow-up is the randomization date. Any new anti-leukemia 
treatments initiated should be recorded. Follow-up continues until the subject reaches the mortality endpoint, is lost to follow-up, the study is completed, 
or the sponsor terminates the study. 
 
Adverse Events of Special Interest 
1) 
Gastrointestinal Intolerance: Nausea, vomiting, bloating, abdominal pain, diarrhea, constipation. Record their frequency, severity, and relationship to 
dosing time (e.g., related to fasting/post-meal administration?). 
2) 
Allergic or Hypersensitivity Reactions: New onset rash, urticaria, skin itching, etc. 
 
Parameters to be Monitored 
 
1) 
Laboratory Tests: Complete blood count (CBC), blood biochemistry, urinalysis, stool routine. 
2) 
Vital Signs and Physical Examination: Weight, temperature, blood pressure, respiration, heart rate. Pay attention to skin (rash), abdomen (tenderness), 
etc. 
3) 
Patient-Reported Outcomes (PROs) and Compliance Records: 
4) 
Record the subject's daily intake of SFN, any missed doses, and any subjectively reported symptoms (e.g., mild nausea, fatigue). 

 
3
5) 
Standardized Questionnaires: Use instruments such as the Gastrointestinal Symptom Rating Scale (GSRS) or the Patient-Reported Outcomes version 
of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) to quantify the severity and frequency of gastrointestinal or other symptoms.