MDS (Myelodysplastic Syndrome)NCT07588360
Haplo-Cord HSCT for AML/MDS
A study testing haplo-cord HSCT and Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX) in people with MDS (Myelodysplastic Syndrome) and AML (Acute Myeloid Leukemia), run by Fujian Medical University Union Hospital at 4 sites in China.
Trial summary
This study aims to investigate the clinical efficacy of haploidentical-cord blood hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and to analyze the impact of different engraftment patterns (haploidentical engraftment versus cord… Read more about this study
- Who is it for?
- Ages 14 to 60 years
- Conditions the study is for
- MDS (Myelodysplastic Syndrome), AML (Acute Myeloid Leukemia)
- What is being tested?
- haplo-cord HSCT and Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 82 participants
- Where
- 4 sitesChina
- Study dates
- 20 Oct 2025to 30 Jun 2029
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age between 14 and 60 years, with no gender restriction.
- Intermediate- or high-risk AML in first complete remission (CR1).
- AML in second or subsequent complete remission (≥ CR2).
- Relapsed or refractory AML.
- Low-risk AML meeting any of the following: Failure to achieve a ≥3-log reduction in RUNX1::RUNX1T1 transcript level compared with baseline after 2 consolidation cycles, or loss of major molecular remission (MMR) within 6 months; CBFB::MYH11/ABL ratio > 0.1% at any time point after 2 consolidation cycles in patients with CBFB::MYH11-rearranged AML; Presence of D816 KIT mutation in patients with CBFB::MYH11-rearranged AML; Flow cytometry-positive MRD at any time point after 2 consolidation cycles in patients with CEBPA double-mutant AML; Persistently positive MRD after chemotherapy in patients with NPM1-mutated AML.
- Intermediate-2 or high-risk MDS according to the IPSS scoring system.
- Adequate general health status and ability to tolerate hematopoietic stem cell transplantation.
- Provision of signed informed consent and willingness to comply with study-required follow-up and examinations.
Who cannot join
- Prior history of other hematopoietic stem cell transplantation.
- History of ex vivo T-cell-depleted stem cell transplantation.
- Survival duration of less than 1 month after transplantation.
- Severe organ dysfunction, including significant impairment of hepatic, renal, cardiac, or pulmonary function.
- Active severe infection, such as uncontrolled pneumonia, sepsis, or other systemic infections.
- History of severe hypersensitivity reactions to study medications, including cyclophosphamide or anti-thymocyte globulin (ATG).
Show all 10Show fewer
- Presence of severe psychiatric disorders or cognitive impairment that precludes compliance with study treatment and follow-up.
- Pregnant or lactating women.
- Concurrent malignancy of other organ system.
- Any other medical conditions deemed inappropriate for study participation by the treating investigators.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China4 sites, 4 recruiting
- Not yet recruiting
Chinese PLA General Hospital
Beijing
Wenrong Huang, +86 010-68182255, huangwenrong@301hospital.com.cn
- Not yet recruiting
Sichuan Provincial People's Hospital
Chengdu, Sichuan
Xiaobing Huang, 028-87394103, huangxiaobing@med.uestc.edu.cn
- Recruiting
Fujian Medical University Union Hospital
Fuzhou, Fujian, 350001
Nainong Li, MD., 13365910189, nainli@aliyun.com
Nainong Li, MD
- Not yet recruiting
Guangdong Provincial People's Hospital
Guangzhou, Guangdong
Suijing Wu, 020-83827812-62110, suijing_wu5413@163.com
About this study
Official title: Haploidentical Combined With Cord Blood Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes: A Prospective, Multicenter Clinical Study
This study aims to investigate the clinical efficacy of haploidentical-cord blood hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and to analyze the impact of different engraftment patterns (haploidentical engraftment versus cord blood engraftment) on clinical outcomes. By comparing the efficacy of haploidentical-cord blood transplantation in different subtypes of AML and MDS, this research will explore its unique advantages and comparative effectiveness relative to conventional transplantation strategies, so as to provide new evidence for clinical practice.
Specific research objectives I. To evaluate the efficacy of haploidentical-cord blood hematopoietic stem cell transplantation for AML and high-risk MDS, including the speed of hematopoietic recovery, immune tolerance, and long-term survival rates.
Read more
II. To compare the effects of different engraftment patterns (haploidentical engraftment vs. cord blood engraftment) on quality of life, immune tolerance, early complications, and long-term prognosis.
III. To identify the clinical advantages and indications of haploidentical-cord blood transplantation through data analysis, and to provide a theoretical basis for clinical decision-making.
Novelty of the Study I. Innovation in Hematopoietic Stem Cell Infusion Schedule The present study employs a sequential infusion strategy: haploidentical stem cells are infused on Day 0, and umbilical cord blood cells are infused on Day +6 after transplantation.In contrast to the conventional approach used at most domestic and international centers (including the uzhou Protocol), in which both stem cell sources are infused simultaneously on Day 0, the current protocol delays cord blood infusion. This design confers potential advantages for immune reconstitution and long-term cord blood engraftment.
II. Unique Myeloablative Conditioning Regimen
The conditioning regimen used in this study is as follows:
Fludarabine 25 mg/m² for 5 days, Cytarabine 2 mg/m² for 5 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days.
(For patients in complete remission (CR) with negative MRD before transplantation, Fludarabine and Cytarabine are administered for 3 days instead of 5 days.) Distinct from regimens at other centers, our team administers cyclophosphamide within the critical window after haploidentical stem cell infusion but before cord blood infusion, establishing a novel sequential conditioning model. This approach balances myeloablative intensity and immunomodulation, creating a favorable environment for subsequent long-term cord blood engraftment.
III. Engraftment Outcomes and Clinical Value Preliminary clinical experience demonstrates that haplo-cord sequential transplantation following the FA5Cy2Bu3 conditioning regimen combined with low-dose ATG/PTCY can achieve long-term cord blood engraftment in approximately 50% of patients.
By comparison, other domestic protocols (e.g., the Suzhou Protocol) rarely result in sustained cord blood engraftment.
Achievement of long-term cord blood engraftment is clinically meaningful for reducing relapse rates, lowering the incidence and severity of graft-versus-host disease (GVHD), and improving patient prognosis. These outcomes represent a key advantage of the present protocol.
Read the detailed description
Acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) are common and rapidly progressive malignant hematologic disorders associated with poor prognosis without effective intervention. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only first-line therapeutic strategy with curative potential for long-term remission or even definitive cure. However, the efficacy of conventional transplantation approaches is limited by multiple factors, particularly the management of graft-versus-host disease (GVHD) and disease recurrence. Balancing the graft-versus-leukemia (GVL) effect against residual leukemia while effectively mitigating GVHD represents an unresolved challenge in current transplantation strategies.
Although an HLA-matched donor is the optimal choice, access to such donors is restricted by time constraints and availability. In recent years, the widespread use of haploidentical donors has dramatically improved transplant accessibility, enabling nearly all patients to identify a suitable donor. China has emerged as a global leader in the development and application of haploidentical transplantation. Nevertheless, the established Beijing Protocol and the Baltimore post-transplantation cyclophosphamide (PTCY) regimen each have distinct limitations: the former is associated with high engraftment rates and low relapse incidence but a relatively high rate of chronic GVHD (approximately 20%), whereas the latter reduces GVHD risk but carries higher rates of engraftment failure and disease relapse.
Umbilical cord blood transplantation (UCBT) represents an alternative donor source, whose inherent immune properties offer the potential to reduce GVHD while preserving the GVL effect. However, the limited cell dose in single cord blood units results in delayed hematopoietic reconstitution, high infection rates, and elevated early mortality, restricting its broader application in adult patients.
Against this background, transplantation strategies combining the advantages of different donor sources have become a major research focus. The 'haplo-cord hematopoietic stem cell transplantation' strategy, which combines haploidentical peripheral blood stem cells with unrelated umbilical cord blood stem cells, has been proposed. This approach integrates the rapid hematopoietic engraftment of haploidentical stem cells with the immunomodulatory properties of cord blood, thereby accelerating hematopoietic recovery and reducing the risk of acute and chronic GVHD. Furthermore, the incorporation of immunomodulatory agents such as low-dose anti-thymocyte globulin (ATG) and post-transplantation cyclophosphamide (PTCY) has led to simultaneous control of both transplantation-related mortality and relapse.
Domestic investigators reported that patients with relapsed/refractory acute leukemia (r/r-AL) who received combined haplo-HSCT and UCB-assisted transplantation exhibited superior leukemia-free survival and lower relapse rates compared with haploidentical transplantation alone, with 2-year overall survival, progression-free survival, cumulative incidence of relapse, and non-relapse mortality of 35.5%, 35.5%, 25.9%, and 38.0%, respectively. An international study comparing double UCBT (dUCBT) with haplo-cord transplantation for hematologic malignancies demonstrated that haplo-cord transplantation was associated with faster neutrophil and platelet engraftment, lower risks of grade II-IV acute GVHD and chronic GVHD, reduced relapse risk, and superior GVHD- and relapse-free survival compared with dUCBT.
In recent years, with the increasing incidence of AML and MDS, post-transplant relapse has become the primary obstacle to long-term therapeutic success. International multicenter data indicate that disease relapse remains the leading cause of transplant failure (59% for matched sibling donors vs. 51% for unrelated donors), followed by severe GVHD and infection. Although conventional strategies including re-induction chemotherapy, second transplantation, and donor lymphocyte infusion (DLI) have been explored, their efficacy is limited by high relapse rates, severe toxicities, and modest survival benefits, failing to meet current clinical needs.
Therefore, the development of novel transplantation models that enhance GVL while controlling GVHD has become an urgent bottleneck to address. Combined donor transplantation strategies represent one promising approach toward this goal. To date, several countries have conducted exploratory studies of the haplo-cord regimen, reporting faster hematopoietic recovery, lower rejection risk, and reduced relapse rates. Multiple centers in China have also initiated clinical validation with encouraging results.
The applicant's research team has developed and implemented a haplo-cord HSCT protocol based on low-dose ATG plus PTCY, with proven safety and efficacy in clinical practice. Compared with conventional haploidentical or single cord blood transplantation, this regimen achieved significantly higher relapse-free survival in patients with relapsed/refractory disease, effectively reduced the incidence of chronic GVHD, and improved quality of life and survival expectancy, with 2-year overall survival, disease-free survival, and GVHD- and relapse-free survival of 64.9%, 64.5%, and 60.8%, respectively. Notably, nearly half of the patients achieved dominant cord blood engraftment, challenging the traditional view that cord blood serves only an auxiliary role.
In the present prospective multicenter cohort study, we aim to further investigate the long-term efficacy of this combined transplantation strategy in patients with AML and high-risk MDS. By comparing immune reconstitution, relapse rates, GVHD, and other prognostic outcomes between cord blood-dominant and haploidentical-dominant engraftment, we intend to define its optimal indications and establish high-level evidence to optimize hematopoietic stem cell transplantation regimens.
This study aims to investigate the clinical efficacy of haploidentical-cord blood hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and to analyze the impact of different engraftment patterns (haploidentical engraftment versus cord blood engraftment) on clinical outcomes. By comparing the efficacy of haploidentical-cord blood transplantation in different subtypes of AML and MDS, this research will explore its unique advantages and comparative effectiveness relative to conventional transplantation strategies, so as to provide new evidence for clinical practice.
Specific research objectives: 1. To evaluate the efficacy of haploidentical-cord blood hematopoietic stem cell transplantation for AML and high-risk MDS, including the speed of hematopoietic recovery, immune tolerance, and long-term survival rates. 2. To compare the effects of different engraftment patterns (haploidentical engraftment vs. cord blood engraftment) on quality of life, immune tolerance, early complications, and long-term prognosis. 3. To identify the clinical advantages and indications of haploidentical-cord blood transplantation through data analysis, and to provide a theoretical basis for clinical decision-making.
- Being tested
- haplo-cord HSCT, Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX)
- Keywords
- Acute Myeloid Leukemia(AML), Myelodysplastic Syndrome(MDS), haplo-cord HSCT
- Medical subject headings
- Anemia, Refractory, with Excess of Blasts, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
haplo-cord HSCTProcedure or surgery
Patients with disease status in CR and MRD-negative before transplantation received the FA3Cy2Bu3 regimen:
Fludarabine 25 mg/m² for 3 days, Cytarabine 2 mg/m² for 3 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days.
All other patients received the FA5Cy2Bu3 regimen:
Fludarabine 25 mg/m² for 5 days, Cytarabine 2 mg/m² for 5 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days.
Following the conditioning regimen, patients underwent haploidentical-cord blood hematopoietic stem cell transplantation.
Haploidentical hematopoietic stem cells were infused on day 0, and umbilical cord blood hematopoietic stem cells were infused on day 6.
Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX)Drug
Patients with disease status in CR and MRD-negative before transplantation received the FA3Cy2Bu3 regimen:
Fludarabine 25 mg/m² for 3 days, Cytarabine 2 mg/m² for 3 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days.
All other patients received the FA5Cy2Bu3 regimen:
Fludarabine 25 mg/m² for 5 days, Cytarabine 2 mg/m² for 5 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days.
Following the conditioning regimen, patients underwent haploidentical-cord blood hematopoietic stem cell transplantation.
Haploidentical hematopoietic stem cells were infused on day 0, and umbilical cord blood hematopoietic stem cells were infused on day 6.
| Study group | Role | What they receive |
|---|---|---|
| Haplo-Cord HSCT | Experimental | Not described Receives: Procedure: haplo-cord HSCT; Drug: Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival (OS)
Measured: 3 years after transplantation
Secondary outcomes (4)
Progression-Free Survival(PFS)
Measured: 3 years after transplantation
Disease-Free Survival(DFS)
Measured: 3 years after transplantation
GVHD and Relapse-Free Survival(GRFS)
Measured: 3 years after transplantation
Non-Relapse Mortality(NRM)
Measured: 3 years after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2026
- Study start20 Oct 2025
- Primary completion20 Oct 2028 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (18)Papers the sponsor has linked to this study.
- Other trials for MDS (Myelodysplastic Syndrome)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 82 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fujian Medical University Union Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07588360
- Sponsor's protocol ID
- 2025XHYG0034-01
- Status last verified
- Oct 2025
- First submitted
- 28 Apr 2026
- Last updated
- 10 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD that underlie results in a publication.
Publications
Li H, Li X, Chen Y, Li D, Chen X, Zhu Z, Wang Y, Huang J, Chen P, Chen Y, Li N. Sequential Transplantation of Haploidentical Stem Cell and Unrelated Cord Blood With Using ATG/PTCY Increases Survival of Relapsed/Refractory Hematologic Malignancies. Front Immunol. 2021 Nov 4;12:733326. doi: 10.3389/fimmu.2021.733326. eCollection 2021.
PubMed 34804017Jabbour E, O'Brien S, Konopleva M, Kantarjian H. New insights into the pathophysiology and therapy of adult acute lymphoblastic leukemia. Cancer. 2015 Aug 1;121(15):2517-28. doi: 10.1002/cncr.29383. Epub 2015 Apr 17.
PubMed 25891003Terwilliger T, Abdul-Hay M. Acute lymphoblastic leukemia: a comprehensive review and 2017 update. Blood Cancer J. 2017 Jun 30;7(6):e577. doi: 10.1038/bcj.2017.53.
PubMed 28665419Chen EL, Liu HL, Geng LQ, Tang BL, Zhu XY, Yao W, Song KD, Wan X, Sun GY, Qiang P, Fan Q, Zhou ZW, Zheng CC, Zhang L, Zhang XH, Tong J, Sun ZM. [Unrelated cord blood stem cell transplantation for high-risk/refractory childhood acute myeloid leukemia: a clinical analysis of 160 cases]. Zhonghua Xue Ye Xue Za Zhi. 2021 Jul 14;42(7):549-554. doi: 10.3760/cma.j.issn.0253-2727.2021.07.004. Chinese.
PubMed 34455741Malard F, Mohty M. Acute lymphoblastic leukaemia. Lancet. 2020 Apr 4;395(10230):1146-1162. doi: 10.1016/S0140-6736(19)33018-1.
PubMed 32247396Global Burden of Disease Cancer Collaboration; Fitzmaurice C, Akinyemiju TF, Al Lami FH, Alam T, Alizadeh-Navaei R, Allen C, Alsharif U, Alvis-Guzman N, Amini E, Anderson BO, Aremu O, Artaman A, Asgedom SW, Assadi R, Atey TM, Avila-Burgos L, Awasthi A, Ba Saleem HO, Barac A, Bennett JR, Bensenor IM, Bhakta N, Brenner H, Cahuana-Hurtado L, Castaneda-Orjuela CA, Catala-Lopez F, Choi JJ, Christopher DJ, Chung SC, Curado MP, Dandona L, Dandona R, das Neves J, Dey S, Dharmaratne SD, Doku DT, Driscoll TR, Dubey M, Ebrahimi H, Edessa D, El-Khatib Z, Endries AY, Fischer F, Force LM, Foreman KJ, Gebrehiwot SW, Gopalani SV, Grosso G, Gupta R, Gyawali B, Hamadeh RR, Hamidi S, Harvey J, Hassen HY, Hay RJ, Hay SI, Heibati B, Hiluf MK, Horita N, Hosgood HD, Ilesanmi OS, Innos K, Islami F, Jakovljevic MB, Johnson SC, Jonas JB, Kasaeian A, Kassa TD, Khader YS, Khan EA, Khan G, Khang YH, Khosravi MH, Khubchandani J, Kopec JA, Kumar GA, Kutz M, Lad DP, Lafranconi A, Lan Q, Legesse Y, Leigh J, Linn S, Lunevicius R, Majeed A, Malekzadeh R, Malta DC, Mantovani LG, McMahon BJ, Meier T, Melaku YA, Melku M, Memiah P, Mendoza W, Meretoja TJ, Mezgebe HB, Miller TR, Mohammed S, Mokdad AH, Moosazadeh M, Moraga P, Mousavi SM, Nangia V, Nguyen CT, Nong VM, Ogbo FA, Olagunju AT, Pa M, Park EK, Patel T, Pereira DM, Pishgar F, Postma MJ, Pourmalek F, Qorbani M, Rafay A, Rawaf S, Rawaf DL, Roshandel G, Safiri S, Salimzadeh H, Sanabria JR, Santric Milicevic MM, Sartorius B, Satpathy M, Sepanlou SG, Shackelford KA, Shaikh MA, Sharif-Alhoseini M, She J, Shin MJ, Shiue I, Shrime MG, Sinke AH, Sisay M, Sligar A, Sufiyan MB, Sykes BL, Tabares-Seisdedos R, Tessema GA, Topor-Madry R, Tran TT, Tran BX, Ukwaja KN, Vlassov VV, Vollset SE, Weiderpass E, Williams HC, Yimer NB, Yonemoto N, Younis MZ, Murray CJL, Naghavi M. Global, Regional, and National Cancer Incidence, Mortality, Years of Life Lost, Years Lived With Disability, and Disability-Adjusted Life-Years for 29 Cancer Groups, 1990 to 2016: A Systematic Analysis for the Global Burden of Disease Study. JAMA Oncol. 2018 Nov 1;4(11):1553-1568. doi: 10.1001/jamaoncol.2018.2706.
PubMed 29860482MacMillan ML, Weisdorf DJ, Brunstein CG, Cao Q, DeFor TE, Verneris MR, Blazar BR, Wagner JE. Acute graft-versus-host disease after unrelated donor umbilical cord blood transplantation: analysis of risk factors. Blood. 2009 Mar 12;113(11):2410-5. doi: 10.1182/blood-2008-07-163238. Epub 2008 Nov 7.
PubMed 18997171Gomez-Arteaga A, Orfali N, Guarneri D, Cushing MM, Gergis U, Hsu J, Hsu YS, Mayer SA, Phillips AA, Chase SA, Mokhtar AE, Shore TB, Van Besien K. Cord blood transplants supported by unrelated donor CD34+ progenitor cells. Bone Marrow Transplant. 2020 Dec;55(12):2298-2307. doi: 10.1038/s41409-020-0959-5. Epub 2020 Jun 9.
PubMed 32518291Yun HD, Varma A, Hussain MJ, Nathan S, Brunstein C. Clinical Relevance of Immunobiology in Umbilical Cord Blood Transplantation. J Clin Med. 2019 Nov 14;8(11):1968. doi: 10.3390/jcm8111968.
PubMed 31739455Hiwarkar P, Qasim W, Ricciardelli I, Gilmour K, Quezada S, Saudemont A, Amrolia P, Veys P. Cord blood T cells mediate enhanced antitumor effects compared with adult peripheral blood T cells. Blood. 2015 Dec 24;126(26):2882-91. doi: 10.1182/blood-2015-06-654780. Epub 2015 Oct 8.
PubMed 26450984Milano F, Gooley T, Wood B, Woolfrey A, Flowers ME, Doney K, Witherspoon R, Mielcarek M, Deeg JH, Sorror M, Dahlberg A, Sandmaier BM, Salit R, Petersdorf E, Appelbaum FR, Delaney C. Cord-Blood Transplantation in Patients with Minimal Residual Disease. N Engl J Med. 2016 Sep 8;375(10):944-53. doi: 10.1056/NEJMoa1602074.
PubMed 27602666Massoud R, Gagelmann N, Fritzsche-Friedland U, Zeck G, Heidenreich S, Wolschke C, Ayuk F, Christopeit M, Kroger N. Comparison of immune reconstitution between anti-T-lymphocyte globulin and posttransplant cyclophosphamide as acute graft-versus-host disease prophylaxis in allogeneic myeloablative peripheral blood stem cell transplantation. Haematologica. 2022 Apr 1;107(4):857-867. doi: 10.3324/haematol.2020.271445.
PubMed 33832208Battipaglia G, Labopin M, Kroger N, Vitek A, Afanasyev B, Hilgendorf I, Schetelig J, Ganser A, Blaise D, Itala-Remes M, Passweg JR, Bonifazi F, Finke J, Ruggeri A, Nagler A, Mohty M. Posttransplant cyclophosphamide vs antithymocyte globulin in HLA-mismatched unrelated donor transplantation. Blood. 2019 Sep 12;134(11):892-899. doi: 10.1182/blood.2019000487. Epub 2019 Jul 3.
PubMed 31270102Lv M, Chang YJ, Huang XJ. Update of the "Beijing Protocol" haplo-identical hematopoietic stem cell transplantation. Bone Marrow Transplant. 2019 Aug;54(Suppl 2):703-707. doi: 10.1038/s41409-019-0605-2.
PubMed 31431695Xu LP, Wu DP, Han MZ, Huang H, Liu QF, Liu DH, Sun ZM, Xia LH, Chen J, Wang HX, Wang C, Li CF, Lai YR, Wang JM, Zhou DB, Chen H, Song YP, Liu T, Liu KY, Huang XJ. A review of hematopoietic cell transplantation in China: data and trends during 2008-2016. Bone Marrow Transplant. 2017 Nov;52(11):1512-1518. doi: 10.1038/bmt.2017.59. Epub 2017 Apr 24.
PubMed 28436973
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Combined With Cord Blood Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes: A Prospective, Multicenter Clinical Study
- ClinicalTrials.gov ID
- NCT07588360
- Acronym / sponsor ID
- 2025XHYG0034-01
- Keywords
- Acute Myeloid Leukemia(AML), Myelodysplastic Syndrome(MDS), haplo-cord HSCT
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 82 participants (estimated)
- Lead sponsor
- Fujian Medical University Union HospitalUniversity, hospital or other
- Study start
- 20 Oct 2025 (actual)
- Primary completion
- 20 Oct 2028 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 14 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aims to investigate the clinical efficacy of haploidentical-cord blood hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and to analyze the impact of different engraftment patterns (haploidentical engraftment versus cord blood engraftment) on clinical outcomes. By comparing the efficacy of haploidentical-cord blood transplantation in different subtypes of AML and MDS, this research will explore its unique advantages and comparative effectiveness relative to conventional transplantation strategies, so as to provide new evidence for clinical practice.
Specific research objectives I. To evaluate the efficacy of haploidentical-cord blood hematopoietic stem cell transplantation for AML and high-risk MDS, including the speed of hematopoietic recovery, immune tolerance, and long-term survival rates.
II. To compare the effects of different engraftment patterns (haploidentical engraftment vs. cord blood engraftment) on quality of life, immune tolerance, early complications, and long-term prognosis.
III. To identify the clinical advantages and indications of haploidentical-cord blood transplantation through data analysis, and to provide a theoretical basis for clinical decision-making.
Novelty of the Study I. Innovation in Hematopoietic Stem Cell Infusion Schedule The present study employs a sequential infusion strategy: haploidentical stem cells are infused on Day 0, and umbilical cord blood cells are infused on Day +6 after transplantation.In contrast to the conventional approach used at most domestic and international centers (including the uzhou Protocol), in which both stem cell sources are infused simultaneously on Day 0, the current protocol delays cord blood infusion. This design confers potential advantages for immune reconstitution and long-term cord blood engraftment.
II. Unique Myeloablative Conditioning Regimen
The conditioning regimen used in this study is as follows:
Fludarabine 25 mg/m² for 5 days, Cytarabine 2 mg/m² for 5 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days.
(For patients in complete remission (CR) with negative MRD before transplantation, Fludarabine and Cytarabine are administered for 3 days instead of 5 days.) Distinct from regimens at other centers, our team administers cyclophosphamide within the critical window after haploidentical stem cell infusion but before cord blood infusion, establishing a novel sequential conditioning model. This approach balances myeloablative intensity and immunomodulation, creating a favorable environment for subsequent long-term cord blood engraftment.
III. Engraftment Outcomes and Clinical Value Preliminary clinical experience demonstrates that haplo-cord sequential transplantation following the FA5Cy2Bu3 conditioning regimen combined with low-dose ATG/PTCY can achieve long-term cord blood engraftment in approximately 50% of patients.
By comparison, other domestic protocols (e.g., the Suzhou Protocol) rarely result in sustained cord blood engraftment.
Achievement of long-term cord blood engraftment is clinically meaningful for reducing relapse rates, lowering the incidence and severity of graft-versus-host disease (GVHD), and improving patient prognosis. These outcomes represent a key advantage of the present protocol.
Detailed description
Acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) are common and rapidly progressive malignant hematologic disorders associated with poor prognosis without effective intervention. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only first-line therapeutic strategy with curative potential for long-term remission or even definitive cure. However, the efficacy of conventional transplantation approaches is limited by multiple factors, particularly the management of graft-versus-host disease (GVHD) and disease recurrence. Balancing the graft-versus-leukemia (GVL) effect against residual leukemia while effectively mitigating GVHD represents an unresolved challenge in current transplantation strategies.
Although an HLA-matched donor is the optimal choice, access to such donors is restricted by time constraints and availability. In recent years, the widespread use of haploidentical donors has dramatically improved transplant accessibility, enabling nearly all patients to identify a suitable donor. China has emerged as a global leader in the development and application of haploidentical transplantation. Nevertheless, the established Beijing Protocol and the Baltimore post-transplantation cyclophosphamide (PTCY) regimen each have distinct limitations: the former is associated with high engraftment rates and low relapse incidence but a relatively high rate of chronic GVHD (approximately 20%), whereas the latter reduces GVHD risk but carries higher rates of engraftment failure and disease relapse.
Umbilical cord blood transplantation (UCBT) represents an alternative donor source, whose inherent immune properties offer the potential to reduce GVHD while preserving the GVL effect. However, the limited cell dose in single cord blood units results in delayed hematopoietic reconstitution, high infection rates, and elevated early mortality, restricting its broader application in adult patients.
Against this background, transplantation strategies combining the advantages of different donor sources have become a major research focus. The 'haplo-cord hematopoietic stem cell transplantation' strategy, which combines haploidentical peripheral blood stem cells with unrelated umbilical cord blood stem cells, has been proposed. This approach integrates the rapid hematopoietic engraftment of haploidentical stem cells with the immunomodulatory properties of cord blood, thereby accelerating hematopoietic recovery and reducing the risk of acute and chronic GVHD. Furthermore, the incorporation of immunomodulatory agents such as low-dose anti-thymocyte globulin (ATG) and post-transplantation cyclophosphamide (PTCY) has led to simultaneous control of both transplantation-related mortality and relapse.
Domestic investigators reported that patients with relapsed/refractory acute leukemia (r/r-AL) who received combined haplo-HSCT and UCB-assisted transplantation exhibited superior leukemia-free survival and lower relapse rates compared with haploidentical transplantation alone, with 2-year overall survival, progression-free survival, cumulative incidence of relapse, and non-relapse mortality of 35.5%, 35.5%, 25.9%, and 38.0%, respectively. An international study comparing double UCBT (dUCBT) with haplo-cord transplantation for hematologic malignancies demonstrated that haplo-cord transplantation was associated with faster neutrophil and platelet engraftment, lower risks of grade II-IV acute GVHD and chronic GVHD, reduced relapse risk, and superior GVHD- and relapse-free survival compared with dUCBT.
In recent years, with the increasing incidence of AML and MDS, post-transplant relapse has become the primary obstacle to long-term therapeutic success. International multicenter data indicate that disease relapse remains the leading cause of transplant failure (59% for matched sibling donors vs. 51% for unrelated donors), followed by severe GVHD and infection. Although conventional strategies including re-induction chemotherapy, second transplantation, and donor lymphocyte infusion (DLI) have been explored, their efficacy is limited by high relapse rates, severe toxicities, and modest survival benefits, failing to meet current clinical needs.
Therefore, the development of novel transplantation models that enhance GVL while controlling GVHD has become an urgent bottleneck to address. Combined donor transplantation strategies represent one promising approach toward this goal. To date, several countries have conducted exploratory studies of the haplo-cord regimen, reporting faster hematopoietic recovery, lower rejection risk, and reduced relapse rates. Multiple centers in China have also initiated clinical validation with encouraging results.
The applicant's research team has developed and implemented a haplo-cord HSCT protocol based on low-dose ATG plus PTCY, with proven safety and efficacy in clinical practice. Compared with conventional haploidentical or single cord blood transplantation, this regimen achieved significantly higher relapse-free survival in patients with relapsed/refractory disease, effectively reduced the incidence of chronic GVHD, and improved quality of life and survival expectancy, with 2-year overall survival, disease-free survival, and GVHD- and relapse-free survival of 64.9%, 64.5%, and 60.8%, respectively. Notably, nearly half of the patients achieved dominant cord blood engraftment, challenging the traditional view that cord blood serves only an auxiliary role.
In the present prospective multicenter cohort study, we aim to further investigate the long-term efficacy of this combined transplantation strategy in patients with AML and high-risk MDS. By comparing immune reconstitution, relapse rates, GVHD, and other prognostic outcomes between cord blood-dominant and haploidentical-dominant engraftment, we intend to define its optimal indications and establish high-level evidence to optimize hematopoietic stem cell transplantation regimens.
This study aims to investigate the clinical efficacy of haploidentical-cord blood hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and to analyze the impact of different engraftment patterns (haploidentical engraftment versus cord blood engraftment) on clinical outcomes. By comparing the efficacy of haploidentical-cord blood transplantation in different subtypes of AML and MDS, this research will explore its unique advantages and comparative effectiveness relative to conventional transplantation strategies, so as to provide new evidence for clinical practice.
Specific research objectives: 1. To evaluate the efficacy of haploidentical-cord blood hematopoietic stem cell transplantation for AML and high-risk MDS, including the speed of hematopoietic recovery, immune tolerance, and long-term survival rates. 2. To compare the effects of different engraftment patterns (haploidentical engraftment vs. cord blood engraftment) on quality of life, immune tolerance, early complications, and long-term prognosis. 3. To identify the clinical advantages and indications of haploidentical-cord blood transplantation through data analysis, and to provide a theoretical basis for clinical decision-making.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Haplo-Cord HSCT | Experimental | Procedure: haplo-cord HSCT, Drug: Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX) |
Who may qualify
- Ages
- 14 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Age between 14 and 60 years, with no gender restriction.
- Intermediate- or high-risk AML in first complete remission (CR1).
- AML in second or subsequent complete remission (≥ CR2).
- Relapsed or refractory AML.
- Low-risk AML meeting any of the following: Failure to achieve a ≥3-log reduction in RUNX1::RUNX1T1 transcript level compared with baseline after 2 consolidation cycles, or loss of major molecular remission (MMR) within 6 months; CBFB::MYH11/ABL ratio > 0.1% at any time point after 2 consolidation cycles in patients with CBFB::MYH11-rearranged AML; Presence of D816 KIT mutation in patients with CBFB::MYH11-rearranged AML; Flow cytometry-positive MRD at any time point after 2 consolidation cycles in patients with CEBPA double-mutant AML; Persistently positive MRD after chemotherapy in patients with NPM1-mutated AML.
Key exclusion criteria (10 listed)
- Prior history of other hematopoietic stem cell transplantation.
- History of ex vivo T-cell-depleted stem cell transplantation.
- Survival duration of less than 1 month after transplantation.
- Severe organ dysfunction, including significant impairment of hepatic, renal, cardiac, or pulmonary function.
- Active severe infection, such as uncontrolled pneumonia, sepsis, or other systemic infections.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| haplo-cord HSCTPatients with disease status in CR and MRD-negative before transplantation received the FA3Cy2Bu3 regimen: Fludarabine 25 mg/m² for 3 days, Cytarabine 2 mg/m² for 3 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days. All other patients received the FA5Cy2Bu3 regimen: Fludarabine 25 mg/m² for 5 days, Cytarabine 2 mg/m² for 5 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days. Following the conditioning regimen, patients underwent haploidentical-cord blood hematopoietic stem cell transplantation. Haploidentical hematopoietic stem cells were infused on day 0, and umbilical cord blood hematopoietic stem cells were infused on day 6. | Procedure or surgery | Haplo-Cord HSCT |
| Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX)Patients with disease status in CR and MRD-negative before transplantation received the FA3Cy2Bu3 regimen: Fludarabine 25 mg/m² for 3 days, Cytarabine 2 mg/m² for 3 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days. All other patients received the FA5Cy2Bu3 regimen: Fludarabine 25 mg/m² for 5 days, Cytarabine 2 mg/m² for 5 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days. Following the conditioning regimen, patients underwent haploidentical-cord blood hematopoietic stem cell transplantation. Haploidentical hematopoietic stem cells were infused on day 0, and umbilical cord blood hematopoietic stem cells were infused on day 6. | Drug | Haplo-Cord HSCT |
Primary outcome
Overall Survival (OS)
Time frame: 3 years after transplantation
4 secondary outcomes
Progression-Free Survival(PFS)
Time frame: 3 years after transplantation
Disease-Free Survival(DFS)
Time frame: 3 years after transplantation
GVHD and Relapse-Free Survival(GRFS)
Time frame: 3 years after transplantation
Non-Relapse Mortality(NRM)
Time frame: 3 years after transplantation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2026.
- Results publication: PubMed 34804017Li H, Li X, Chen Y, Li D, Chen X, Zhu Z, Wang Y, Huang J, Chen P, Chen Y, Li N. Sequential Transplantation of Haploidentical Stem Cell and Unrelated Cord Blood With Using ATG/PTCY Increases Survival of Relapsed/Refractory Hematologic Malignancies. Front Immunol. 2021 Nov 4;12:733326. doi: 10.3389/fimmu.2021.733326. eCollection 2021.
- Results publication: PubMed 25891003Jabbour E, O'Brien S, Konopleva M, Kantarjian H. New insights into the pathophysiology and therapy of adult acute lymphoblastic leukemia. Cancer. 2015 Aug 1;121(15):2517-28. doi: 10.1002/cncr.29383. Epub 2015 Apr 17.
- Results publication: PubMed 28665419Terwilliger T, Abdul-Hay M. Acute lymphoblastic leukemia: a comprehensive review and 2017 update. Blood Cancer J. 2017 Jun 30;7(6):e577. doi: 10.1038/bcj.2017.53.
- Results publication: PubMed 34455741Chen EL, Liu HL, Geng LQ, Tang BL, Zhu XY, Yao W, Song KD, Wan X, Sun GY, Qiang P, Fan Q, Zhou ZW, Zheng CC, Zhang L, Zhang XH, Tong J, Sun ZM. [Unrelated cord blood stem cell transplantation for high-risk/refractory childhood acute myeloid leukemia: a clinical analysis of 160 cases]. Zhonghua Xue Ye Xue Za Zhi. 2021 Jul 14;42(7):549-554. doi: 10.3760/cma.j.issn.0253-2727.2021.07.004. Chinese.
- Results publication: PubMed 32247396Malard F, Mohty M. Acute lymphoblastic leukaemia. Lancet. 2020 Apr 4;395(10230):1146-1162. doi: 10.1016/S0140-6736(19)33018-1.
- Results publication: PubMed 29860482Global Burden of Disease Cancer Collaboration; Fitzmaurice C, Akinyemiju TF, Al Lami FH, Alam T, Alizadeh-Navaei R, Allen C, Alsharif U, Alvis-Guzman N, Amini E, Anderson BO, Aremu O, Artaman A, Asgedom SW, Assadi R, Atey TM, Avila-Burgos L, Awasthi A, Ba Saleem HO, Barac A, Bennett JR, Bensenor IM, Bhakta N, Brenner H, Cahuana-Hurtado L, Castaneda-Orjuela CA, Catala-Lopez F, Choi JJ, Christopher DJ, Chung SC, Curado MP, Dandona L, Dandona R, das Neves J, Dey S, Dharmaratne SD, Doku DT, Driscoll TR, Dubey M, Ebrahimi H, Edessa D, El-Khatib Z, Endries AY, Fischer F, Force LM, Foreman KJ, Gebrehiwot SW, Gopalani SV, Grosso G, Gupta R, Gyawali B, Hamadeh RR, Hamidi S, Harvey J, Hassen HY, Hay RJ, Hay SI, Heibati B, Hiluf MK, Horita N, Hosgood HD, Ilesanmi OS, Innos K, Islami F, Jakovljevic MB, Johnson SC, Jonas JB, Kasaeian A, Kassa TD, Khader YS, Khan EA, Khan G, Khang YH, Khosravi MH, Khubchandani J, Kopec JA, Kumar GA, Kutz M, Lad DP, Lafranconi A, Lan Q, Legesse Y, Leigh J, Linn S, Lunevicius R, Majeed A, Malekzadeh R, Malta DC, Mantovani LG, McMahon BJ, Meier T, Melaku YA, Melku M, Memiah P, Mendoza W, Meretoja TJ, Mezgebe HB, Miller TR, Mohammed S, Mokdad AH, Moosazadeh M, Moraga P, Mousavi SM, Nangia V, Nguyen CT, Nong VM, Ogbo FA, Olagunju AT, Pa M, Park EK, Patel T, Pereira DM, Pishgar F, Postma MJ, Pourmalek F, Qorbani M, Rafay A, Rawaf S, Rawaf DL, Roshandel G, Safiri S, Salimzadeh H, Sanabria JR, Santric Milicevic MM, Sartorius B, Satpathy M, Sepanlou SG, Shackelford KA, Shaikh MA, Sharif-Alhoseini M, She J, Shin MJ, Shiue I, Shrime MG, Sinke AH, Sisay M, Sligar A, Sufiyan MB, Sykes BL, Tabares-Seisdedos R, Tessema GA, Topor-Madry R, Tran TT, Tran BX, Ukwaja KN, Vlassov VV, Vollset SE, Weiderpass E, Williams HC, Yimer NB, Yonemoto N, Younis MZ, Murray CJL, Naghavi M. Global, Regional, and National Cancer Incidence, Mortality, Years of Life Lost, Years Lived With Disability, and Disability-Adjusted Life-Years for 29 Cancer Groups, 1990 to 2016: A Systematic Analysis for the Global Burden of Disease Study. JAMA Oncol. 2018 Nov 1;4(11):1553-1568. doi: 10.1001/jamaoncol.2018.2706.
- Results publication: PubMed 18997171MacMillan ML, Weisdorf DJ, Brunstein CG, Cao Q, DeFor TE, Verneris MR, Blazar BR, Wagner JE. Acute graft-versus-host disease after unrelated donor umbilical cord blood transplantation: analysis of risk factors. Blood. 2009 Mar 12;113(11):2410-5. doi: 10.1182/blood-2008-07-163238. Epub 2008 Nov 7.
- Results publication: PubMed 32518291Gomez-Arteaga A, Orfali N, Guarneri D, Cushing MM, Gergis U, Hsu J, Hsu YS, Mayer SA, Phillips AA, Chase SA, Mokhtar AE, Shore TB, Van Besien K. Cord blood transplants supported by unrelated donor CD34+ progenitor cells. Bone Marrow Transplant. 2020 Dec;55(12):2298-2307. doi: 10.1038/s41409-020-0959-5. Epub 2020 Jun 9.
- Results publication: PubMed 31739455Yun HD, Varma A, Hussain MJ, Nathan S, Brunstein C. Clinical Relevance of Immunobiology in Umbilical Cord Blood Transplantation. J Clin Med. 2019 Nov 14;8(11):1968. doi: 10.3390/jcm8111968.
- Results publication: PubMed 26450984Hiwarkar P, Qasim W, Ricciardelli I, Gilmour K, Quezada S, Saudemont A, Amrolia P, Veys P. Cord blood T cells mediate enhanced antitumor effects compared with adult peripheral blood T cells. Blood. 2015 Dec 24;126(26):2882-91. doi: 10.1182/blood-2015-06-654780. Epub 2015 Oct 8.
- Results publication: PubMed 27602666Milano F, Gooley T, Wood B, Woolfrey A, Flowers ME, Doney K, Witherspoon R, Mielcarek M, Deeg JH, Sorror M, Dahlberg A, Sandmaier BM, Salit R, Petersdorf E, Appelbaum FR, Delaney C. Cord-Blood Transplantation in Patients with Minimal Residual Disease. N Engl J Med. 2016 Sep 8;375(10):944-53. doi: 10.1056/NEJMoa1602074.
- Results publication: PubMed 33832208Massoud R, Gagelmann N, Fritzsche-Friedland U, Zeck G, Heidenreich S, Wolschke C, Ayuk F, Christopeit M, Kroger N. Comparison of immune reconstitution between anti-T-lymphocyte globulin and posttransplant cyclophosphamide as acute graft-versus-host disease prophylaxis in allogeneic myeloablative peripheral blood stem cell transplantation. Haematologica. 2022 Apr 1;107(4):857-867. doi: 10.3324/haematol.2020.271445.
- Individual participant dataSharing planned: Yes. All IPD that underlie results in a publication.
Related trials for MDS (Myelodysplastic Syndrome)
Recruiting
HEME Home Transfusion Program
Leukemia, Myeloma
RecruitingObservational study
Natural History of Trisomy 8-Associated Autoinflammatory Disease (TRIAD) and Related Disorders
Trisomy 8 Mosaicism, Trisomy 8 Associated Autoinflammatory Disease
RecruitingPhase 2
A Study of TAK-226 for Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes
Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Recruiting
Involvement of CDA and/or dCK Metabolizing Enzymes in the Response to Azacytidine Treatment of Patients With Hematologic Malignancies
Myelodysplastic Syndromes (MDS), Leukemia Acute Myeloid - AML
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing haplo-cord HSCT and Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX) in people with MDS (Myelodysplastic Syndrome) and AML (Acute Myeloid Leukemia), run by Fujian Medical University Union Hospital at 4 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Nainong Li, MD (+86 13365910189, nainli@aliyun.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (OS) (time frame: 3 years after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07588360, which also lists 18 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jun 2026.