Thiotepa-based preparation before stem cell transplant for chronic myelomonocytic leukemia

Trial details

Who runs it

Peking University People's Hospital

Trial code

NCT07588594

Record updated

15 September 2026

Original description

This is a prospective, single-arm clinical study. It aims to evaluate the effectiveness and safety of a conditioning regimen containing thiotepa (in combination with busulfan and fludarabine, with or without ATG) of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with chronic myelomonocytic leukemia (CMML) who have an intermediate-2 or high-risk prognosis.Read full original descriptionShow less

Prospective Single-Arm Clinical Study of Thiotepa-Containing Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in Chronic Myelomonocytic Leukemia

This is a prospective, single-arm clinical study. It aims to evaluate the effectiveness and safety of a conditioning regimen containing thiotepa (in combination with busulfan and fludarabine, with or without ATG) of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with chronic myelomonocytic leukemia (CMML) who have an intermediate-2 or high-risk prognosis.

The main goal is to evaluate 1 year RFS and OS. Other goals include assessing engraftment, overall survival, transplant-related complications, and side effects. A total of 31 participants will be enrolled.

Inclusion: Age ≥ 18 years, any sex/gender; Confirmed diagnosis of chronic myelomonocytic leukemia (CMML) according to the 2022 WHO classification; Intermediate-2 or high-risk CMML based on CPSS or CPSS-mol score, and planned to receive allo-HSCT; Has a suitable hematopoietic stem cell donor:; For haploidentical donor: at least 5/10 HLA match at HLA-A, -B, -C, -DQB1, and -DRB1; For unrelated donor: at least 9/10 HLA match at the same five loci; For matched sibling donor: 10/10 HLA match at the same five loci; Hematopoietic cell transplantation comorbidity index (HCT-CI) ≤ 2, with generally good health and no significant organ abnormalities or major comorbidities; Adequate organ function as defined below:; Left ventricular ejection fraction (LVEF) ≥ 50%, and no uncontrolled tachycardia or bradycardia-tachycardia syndrome; Total bilirubin ≤ 1.5 × upper limit of normal (ULN); ALT ≤ 2 × ULN; AST ≤ 2 × ULN; Serum creatinine ≤ 1.5 × ULN; Baseline oxygen saturation > 92%; Pulmonary function: DLCO (corrected for hemoglobin) ≥ 40%, FEV1 ≥ 50%; Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2; Agrees not to participate in any other interventional study during the treatment period; Willing and able to provide written informed consent, understand the nature, purpose, and procedures of the study, and voluntarily comply with study requirements.

Exclusion: Previous allogeneic HSCT for CMML that later relapsed; History of stroke or intracranial hemorrhage within 6 months before enrollment; Diagnosis of another malignancy within the past 2 years, except for localized skin cancer, superficial bladder cancer, carcinoma in situ of the cervix, breast cancer, or localized prostate cancer (Gleason score ≤ 6) that has been treated with curative intent; Active hepatitis B or C, or chronic active hepatitis; known human immunodeficiency virus (HIV) infection; Active uncontrolled infection, including: hemodynamic instability related to infection, new or worsening signs/symptoms of infection, new infection lesions on imaging, or persistent fever without explanation despite no symptoms/signs; Any other condition that, in the investigator's judgment, makes the patient unsuitable for study participation; Unwilling or unable to receive the study treatment regimen; Known pregnancy (positive urine pregnancy test), or currently breastfeeding.

ClinicalTrials.gov NCT07588594