The Safety and Efficacy of the Regimen of Thiotepa + Fludarabine +Granulocyte Colony-Stimulating Factor+ Cytarabine + Busulfan in Single Unrelated Umbilical Cord Blood Transplantation

A Prospective, Single-arm, Single-center, Open-label Clinical Study on the Safety and Efficacy of the Regimen of Thiotepa + Fludarabine +Granulocyte Colony-Stimulating Factor+ Cytarabine + Busulfan in Single Unrelated Umbilical Cord Blood Transplantation for Elderly Patients With Hematologic Malignancies

This trial is a prospective, single-arm, single-center clinical study. A total of 44 elderly (aged 60 to 69 years) patients with hematological malignancies who need to receive a single-unit unrelated umbilical cord blood transplantation (UCBT) are planned to be enrolled. After screening and enrollment, patients are scheduled to receive the following drug treatment regimen: Thiotepa 5 mg/kg every 12 hours on day -8; Granulocyte Colony-Stimulating Factor(G-CSF) 5 ug/kg/day from day -8 to -5; Cytarabine 1.5 g/m2 from day -7 to -5; Fludarabine 30 mg/m2/day from day -7 to -4; Busulfan 0.8 mg/kg every 6 hours from day -4 to -2. Umbilical cord blood hematopoietic stem cells are reinfused on day 0. Routine monitoring of blood routine is conducted, and platelets and red blood cells are transfused when necessary.

Study Overview

• Status: Not yet recruiting
• Conditions: Hematologic Malignancies
• Intervention / Treatment: Drug: Thiotepa; Drug: Fludarabine; Drug: G-CSF; Drug: cytarabine; Drug: busulfan

• Study Type: Interventional
• Enrollment (Estimated): 44
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Aged between 60 and 69 years old (inclusive), gender not restricted.
• Patients with hematological malignancies who plan to receive a single unrelated umbilical cord blood transplantation treatment

• The liver and kidney functions, as well as the heart and lung functions, must meet the following requirements:

  1. Serum creatinine ≤ 1.5 × ULN
  2. Cardiac function: Ejection fraction > 50%
  3. Baseline oxygen saturation > 92%
  4. Total bilirubin ≤ 1.5 × ULN; ALT and AST ≤ 2.5 × ULN
  5. Pulmonary function: DLCO (hemoglobin-corrected) > 65% and FEV1 > 65%
• ECOG PS≤2
• HCT-CI≤2
• Expected survival period ≥ 6 months
• By signing the informed consent form, one voluntarily participates in this study and is willing and able to cooperate with the data collection in this research.

Exclusion Criteria:

• For those who are allergic to the drugs and their related metabolites
• Any unstable systemic diseases: including but not limited to stable angina pectoris, cerebrovascular accident or transient cerebral ischemia (within 3 months before screening), myocardial infarction (within 3 months before screening), congestive heart failure (NYHA classification ≥ 3), severe arrhythmias requiring drug treatment, liver, kidney or metabolic diseases; patients with pulmonary hypertension; or diseases that, in the judgment of the investigator, pose a serious threat to the safety of the patient or affect the patient's ability to complete the study.
• Active and uncontrolled infection: Hemodynamic instability related to the infection, or new symptoms or signs of infection appear, or new infection lesions are found on imaging, or persistent fever without symptoms or signs that cannot rule out the presence of an infection.
• The patient is currently involved in a clinical intervention study
• The patient is unable to understand the nature of the study or has not given informed consent
• Other circumstances where the researcher deems the patient unsuitable for inclusion in this study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Thiotepa + fludarabine + G-CSF + cytarabine + busulfan	Drug: Thiotepa; Thiotepa 5 mg/kg every 12 hours, D-8.; Drug: Fludarabine; Fludarabine 30 mg/m2 per day, from D-7 to -4.; Drug: G-CSF; G-CSF 5 ug/kg per day, from D-8 to D-5.; Drug: cytarabine; Cytarabine 1.5 g/m2, from D-7 to D-5.; Drug: busulfan; Busulfan 0.8 mg/kg, every 6 hours, from D-4 to -2.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
180-day non-recurrence mortality rate	180-day

Secondary Outcome Measures

Outcome Measure	Time Frame
The cumulative implantation rate of neutrophils	One month after the transplantation
The cumulative implantation rate of platelets	One month after the transplantation
The cumulative implantation time of platelets	One month after the transplantation
The cumulative implantation time of neutrophils	One month after the transplantation
The cumulative incidence and severity of acute and chronic graft-versus-host disease (GVHD)	Two years after the transplantation
One-year overall survival rate after transplantation	One year after transplantation
One-year progression-free survival rate after transplantation	One-year after transplantation
The cumulative recurrence rate one year after transplantation	one year after transplantation

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Estimated): February 20, 2026
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2028

Study Registration Dates

• First Submitted: February 9, 2026
• First Submitted That Met QC Criteria: February 26, 2026
• First Posted (Actual): March 2, 2026

Study Record Updates

• Last Update Posted (Actual): March 2, 2026
• Last Update Submitted That Met QC Criteria: February 26, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: Regimen; Unrelated Umbilical Cord Blood Transplantation
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Hematologic Neoplasms; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Sulfur Compounds; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleic Acids, Nucleotides, and Nucleosides; Hydrocarbons, Acyclic; Hydrocarbons; Biological Factors; Carbohydrates; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Alkanes; Alcohols; Butylene Glycols; Glycols; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Sulfonic Acids; Sulfur Acids; Phosphoramides; Organophosphorus Compounds; Nucleosides; Intercellular Signaling Peptides and Proteins; Arabinonucleosides; Glycoproteins; Glycoconjugates; Triethylenephosphoramide; Aziridines; Azirines; Colony-Stimulating Factors; Hematopoietic Cell Growth Factors; Cytokines; Cytarabine; Busulfan; Thiotepa; fludarabine; Granulocyte Colony-Stimulating Factor
• Other Study ID Numbers: IIT2025140

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No