Study on the Efficacy and Safety of the TmBU Conditioning Regimen in High-risk or Relapsed/Refractory Acute Leukemia

NCT06816134 | Recruiting Phase 2

• 1 other identifier: SZConditioning01
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

This project is a prospective, single-center, randomized controlled clinical study. The subjects were high-risk or relapsed/refractory AML or ALL patients aged ≤ 65 years diagnosed by bone marrow cell morphology, immunology, genetics and therapeutic efficacy evaluation. The TmBU scheme or modified Bu/Cy (mBuCy) scheme was used for pretreatment in allo-HSCT. The primary endpoint of the study was the 2-year cumulative incidence of relapse (CIR) after allo-HSCT, and the secondary endpoints were 2-year overall survival rate (OS), progressing-free survival rate (PFS), non-relapse mortality rate (NRM), graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) rate.

Conditions

Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Transplantation, Stem Cell; Conditioning Therapy

Outcome Measures

Primary Outcomes (1)

• Cumulative incidence of relapse（CIR）

  It is measured the date from complete remission after transplantation to hematological relapse or molecular relapse was recorded. Patients who had no relapse at the last follow-up were considered as censored data, and non-relapse death was regarded as a competing risk event.

  2 years

Secondary Outcomes (6)

• Time period for hematopoietic reconstruction

  24 weeks

• Incidence of acute graft-versus-host disease (aGVHD)

  100 days

• Progressing-free survival（PFS）

  2 years

• Overall survival（OS）

  2 years

• Non-relapse mortality（NRM）

  2 years

Study Arms (2)

TmBu conditioning Regimen Group

EXPERIMENTAL

TmBU conditioning regimen（TT 7mg/kg -8\~-7d; Ara-C 2g/m2/d -6d; CDA 5 mg/m2/d -6d; Bu 3.2 mg/kg/d from -5 to -3d）

Drug: TmBU conditioning Regimen

mBUCY conditioning Regimen Group

ACTIVE COMPARATOR

mBuCy conditioning regimen（CCNU 250mg/m2/d -9d, Ara-C 2g/m2/d -8d; CDA 5 mg/m2/d -8d; Busulfan 3.2 mg/kg/d from -7 to -5 days; Cyclophosphamide 1.8 mg/m2/d from -4 to -3 days）

Drug: mBUCY conditioning regimen

Interventions

TmBU conditioning Regimen DRUG

TmBU conditioning Regimen and GVHD Prophylaxis of Haploid transplantation (Haplo-HSCT) and unrelated donor transplantation (UDT) : Cyclosporine A (CsA) + Mycophenolate mofetil dispersible tablets (MMF) + short-term low-dose methotrexate (MTX) + rabbit anti-human antithymocyte globulin (ATG) or GVHD Prophylaxis of Matched Sibling Donor Hematopoietic Stem Cell Transplantation (MSD-HSCT)：Cyclosporine A (CsA) + short-term low-dose methotrexate (MTX), then stem cells are infused into patient's blood.

Also known as: Experimental Group

TmBu conditioning Regimen Group

mBUCY conditioning regimen DRUG

mBUCY conditioning Regimen and GVHD Prophylaxis of Haploid transplantation (Haplo-HSCT) and unrelated donor transplantation (UDT): Cyclosporine A (CsA) + Mycophenolate mofetil dispersible tablets (MMF) + short-term low-dose methotrexate (MTX) + rabbit anti-human antithymocyte globulin (ATG) or GVHD Prophylaxis of Matched Sibling Donor Hematopoietic Stem Cell Transplantation (MSD-HSCT)：Cyclosporine A (CsA) + short-term low-dose methotrexate (MTX), then stem cells are infused into patient's blood.

Also known as: Control Group

mBUCY conditioning Regimen Group

Eligibility Criteria

• Age: 15 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of AML or ALL according to WHO 2022 guideline criteria, with indications for allo-HSCT list below:
• Relapsed/primary refractory (definitions refer to NCCN 2025) or genetic high-risk group AML at diagnosis (risk stratification refers to ELN 2022)
• High-risk at diagnosis (risk stratification refers to ELN 2022) or MRD positive before transplantation B-ALL
• Confirmed diagnosis of T-ALL
• History of central nervous system leukemia (CNSL) or histopathologically confirmed extramedullary manifestation (EMD) during the course of the AML or ALL
• Age 15-65 years old (≤ 65 years old)
• HCT-CI score \< 2 points ECOG 0-2 points
• Adequate organ function:
• Cardiac NYHA grade ≤ 2, left ventricular ejection fraction ≥55%
• Creatinine clearance ≥ 50ml/min
• ALT and AST ≤ 2.5 times the upper limit of the normal range, and total bilirubin ≤ 1.5 times the upper limit of the normal range
• Oxygen saturation \> 92% without oxygen
• Expected survival time ≥ 3 months
• Ability to understand and voluntarily sign the informed consent form

You may not qualify if:

• With other malignant tumors and have received any treatment for this tumor within the past 3 years
• Previous or current other CNS disease (such as epilepsy, generalized seizure disorder, paresis, aphasia, stroke, severe brain injuries, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, or psychosis) or any CNS-related autoimmune disease
• HIV/Syphilis infection or uncontrolled active other infections (bacteria or fungus or virus is included)
• With active hepatitis B or hepatitis C infection
• Patients received cardiac angioplasty or stent implantation within 12 months before signing the informed consent form, or have symptoms requiring medical treatment for coronary heart disease
• With primary immunodeficiency or active autoimmune disease
• Previous history of severe immediate hypersensitivity reactions to any of the drugs to be used in this study
• Received a live vaccine within 6 weeks prior to screening
• Pregnant, lactating females and patients of childbearing potential who are unwilling to use contraception
• Inability to cooperate with the requirements of study, treatment and monitoring due to psychiatric illness or other conditions
• Patients not suitable for the study according to the investigator's assessment

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions

Control Groups

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods

Study Officials

• Sheng-Li Xue, M.D.

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sheng-Li Xue, M.D.

CONTACT

008651267781139; slxue@suda.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof.

Study Record Dates

• First Submitted: February 4, 2025
• First Posted: February 10, 2025
• Study Start: January 30, 2025
• Primary Completion (Estimated): January 30, 2028
• Study Completion (Estimated): January 30, 2030
• Last Updated: April 23, 2026

Record last verified: 2026-03

Locations

CN (1)