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

NCT07588594 | Recruiting Phase 4 DMC

• 1 other identifier: 2025PHD048-001
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 1

Brief Summary

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 see 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.

Conditions

Chronic Myelomonocytic Leukemia (CMML)

Keywords

Chronic Myelomonocytic Leukemia (CMML); Hematopoietic Stem Cell Transplantation (HSCT); Thiotepa

Outcome Measures

Primary Outcomes (1)

• One-year relapse-free survival (RFS) after allogeneic hematopoietic stem cell transplantation

  Time from stem cell infusion to hematologic/extramedullary relapse or death, censored at 12 months for event-free participants.

  At 12 months after allogeneic hematopoietic stem cell transplantation.

Secondary Outcomes (7)

• One-year overall survival (OS)

  At 12 months after allogeneic hematopoietic stem cell transplantation.

• One-year cumulative incidence of relapse (RR)

  At 12 months after allogeneic hematopoietic stem cell transplantation.

• One-year non-relapse mortality (NRM)

  At 12 months after allogeneic hematopoietic stem cell transplantation.

• Regimen toxicity at day +30

  Up to day +30 post-transplant

• Incidence and severity of adverse events (AEs)

  At 12 months after allogeneic hematopoietic stem cell transplantation.

Study Arms (1)

Single Arm: Thiotepa + Busulfan + Fludarabine ± ATG

EXPERIMENTAL

Patients with intermediate-2 or high-risk chronic myelomonocytic leukemia (CMML) per CPSS/CPSS-mol criteria, who are scheduled to receive allogeneic hematopoietic stem cell transplantation (allo-HSCT) with a thiotepa-containing conditioning regimen.

Drug: TBF regimen

Interventions

TBF regimen DRUG

Intravenous thiotepa 5 mg/kg/d on days -11,-10; busulfan 3.2 mg/kg/d on days -8,-7,-6; fludarabine 30 mg/m²/d on days -6 to -2; ATG per donor type (haplo/unrelated: 2.5 mg/kg/d days -5 to -2; MSD: 1.125 mg/kg/d days -5 to -2). Allogeneic stem cells infused on day 0. GVHD prophylaxis: CsA, MMF, MTX per protocol.

Also known as: TBF (Thiotepa, Busulfan, Fludarabine) ± ATG

Single Arm: Thiotepa + Busulfan + Fludarabine ± ATG

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• Previous allogeneic HSCT for CMML that later relapsed.
• Unwilling or unable to receive the study treatment regimen.
• 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.
• History of stroke or intracranial hemorrhage within 6 months before enrollment.
• Known pregnancy (positive urine pregnancy test), or currently breastfeeding.
• 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.
• Any other condition that, in the investigator's judgment, makes the patient unsuitable for study participation.

Sponsors & Collaborators

• Sun Yuqian: lead
• Jiangsu Hengrui Pharmaceutical Co., Ltd.: collaborator

Study Sites (1)

Peking University People's Hospital

Beijing, 100044, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myelomonocytic, Chronic

Interventions

Thiotepa; Busulfan; fludarabine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramides; Organophosphorus Compounds; Organic Chemicals; Triethylenephosphoramide; Aziridines; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Butylene Glycols; Glycols; Alcohols; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Study Officials

• Yuqian Sun, MD

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yuqian Sun, MD

CONTACT

+86 13717833825; sunyuqian83@hotmail.com

Xueyi Luo, MD

CONTACT

+86 13811936698; lll_xxx_yyy@126.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: May 6, 2026
• First Posted: May 15, 2026
• Study Start: February 13, 2026
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): May 1, 2028
• Last Updated: May 15, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)