Efficacy and safety of TmBU conditioning for high-risk leukemia patients

A Prospective, Randomized Controlled Clinical Study on the Efficacy and Safety of the TmBU Regimen Versus mBUCY Regimen for Conditioning Before Allo-HSCT in High-risk or Relapsed/Refractory Acute Leukemia

This study is testing a new treatment approach for high-risk leukemia patients to see if it helps them stay cancer-free after a stem cell transplant better than the current method.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Ages: 15 Years to 65 Years
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (other)
• Locations: 1 site (Suzhou, Jiangsu)
• Trial ID: NCT06816134 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of the TmBU conditioning regimen compared to a modified Bu/Cy regimen in patients with high-risk or relapsed/refractory acute leukemia. It is a prospective, single-center, randomized controlled study involving patients aged 15 to 65 years who are candidates for allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint is the two-year cumulative incidence of relapse after transplantation, while secondary endpoints include overall survival, progression-free survival, non-relapse mortality, and graft-versus-host disease-free relapse-free survival rates.

Who should consider this trial

Good fit: Ideal candidates are patients aged 15-65 with high-risk or relapsed/refractory acute myeloid leukemia or acute lymphoblastic leukemia who meet specific eligibility criteria.

Not a fit: Patients with low-risk leukemia or those outside the age range of 15-65 years may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could improve treatment outcomes and survival rates for patients with high-risk acute leukemia.

How similar studies have performed: Other studies have explored conditioning regimens in leukemia treatment, but the specific TmBU regimen is relatively novel.

Eligibility criteria

Inclusion Criteria:

* Confirmed diagnosis of AML or ALL according to WHO 2022 guideline criteria, with indications for allo-HSCT list below:

  1. Relapsed/primary refractory (definitions refer to NCCN 2025) or genetic high-risk group AML at diagnosis (risk stratification refers to ELN 2022)
  2. High-risk at diagnosis (risk stratification refers to ELN 2022) or MRD positive before transplantation B-ALL
  3. Confirmed diagnosis of T-ALL
  4. 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:

  1. Cardiac NYHA grade ≤ 2, left ventricular ejection fraction ≥55%
  2. Creatinine clearance ≥ 50ml/min
  3. 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
  4. Oxygen saturation \> 92% without oxygen
* Expected survival time ≥ 3 months
* Ability to understand and voluntarily sign the informed consent form

Exclusion Criteria:

* 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

Where this trial is running

Suzhou, Jiangsu

• The First Affiliated Hospital of Soochow University — Suzhou, Jiangsu, China (RECRUITING)

Study contacts

• Principal investigator: Sheng-Li Xue, M.D. — The First Affiliated Hospital of Soochow University
• Study coordinator: Sheng-Li Xue, M.D.

Conditions: Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Transplantation, Stem Cell, Conditioning Therapy