Thiotepa Combined With Busulfan and Fludarabine Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplantation in Elderly Patients With High-Risk Acute Myeloid Leukemia

Phase 2

Recruiting

• Conditions: Acute Myeloid Leukemia (AML); High Risk Acute Myeloid Leukemia(AML); Relapsed/Refractory Acute Myeloid Leukemia (AML)
• Interventions: Drug: Thiotepa ;busulfan;fludarabine

• Registration Number: NCT06869265
• Lead Sponsor: Peking University People's Hospital

Brief Summary

Abstract:

This clinical study is designed to evaluate the efficacy and safety of a thiotepa, busulfan, and fludarabine (TBF) conditioning regimen for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in elderly patients (≥55 years) with high-risk acute myeloid leukemia (AML). A total of 56 eligible patients will be enrolled in this prospective, single-arm, multicenter trial.

Study Design:

This is a multicenter, single-arm, prospective clinical trial. Eligible patients will receive the following conditioning regimen: thiotepa 5 mg/kg/day on Days -10 to -9; busulfan 3.2 mg/kg/day on Days -8 to -6; fludarabine 30 mg/m²/day on Days -6 to -2; and rabbit anti-thymocyte globulin (rATG) 1.5-2.5 mg/kg/day on Days -5 to -2. Hematopoietic stem cell infusion will be performed on Day 0. Graft-versus-host disease (GVHD) prophylaxis will include cyclosporine A (CsA), mycophenolate mofetil (MMF), and methotrexate (MTX). The primary endpoint of this study is 1-year relapse-free survival (RFS).

Inclusion Criteria:

Age 55-70 years (inclusive). Diagnosis of high-risk AML , include relapsed/refractory AML or CR MRD+ or ELN22 adverse risk group .Relapsed/refractory AML defined as at least one of the following: recurrence of leukemia cells in peripheral blood or bone marrow blasts \>5% after complete remission (CR), failure to achieve remission after two courses of standard induction therapy, relapse within 12 months after consolidation therapy, relapse after 12 months that is refractory to conventional chemotherapy, multiple relapses, or persistent extramedullary leukemia）.

Scheduled to undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).

Adequate organ function. Eastern Cooperative Oncology Group (ECOG) performance status ≤2. Written informed consent obtained.

Exclusion Criteria:

Unwillingness or refusal to accept the study treatment protocol. Presence of donor-specific anti-HLA antibodies. Known human immunodeficiency virus (HIV) infection. Active hepatitis B or C, or chronic active hepatitis. Uncontrolled active infection at the time of enrollment. Any other condition deemed by the investigator as unsuitable for study inclusion.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-03
• Study First Submitted: 2025-03-02
• Study First Submitted QC: 2025-03-05
• Study First Posted: 2025-03-11
• Study Start: 2025-05-01
• Last Update Submitted: 2025-05-15
• Last Update Posted: 2025-05-18
• Primary Completion: 2028-04-01
• Study Completion: 2029-04-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 56

Inclusion Criteria

1. Age 55-70 years (inclusive).
2. Diagnosis of high-risk AML , include relapsed/refractory AML or CR MRD+ or ELN22 adverse risk group . Relapsed/refractory AML defined as at least one of the following: recurrence of leukemia cells in peripheral blood or bone marrow blasts >5% after complete remission (CR), failure to achieve remission after two courses of standard induction therapy, relapse within 12 months after consolidation therapy, relapse after 12 months that is refractory to conventional chemotherapy, multiple relapses, or persistent extramedullary leukemia .
3. Scheduled to undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).
4. Adequate organ function.
5. Eastern Cooperative Oncology Group (ECOG) performance status ≤2.
6. Written informed consent obtained.

Exclusion Criteria

1. Unwillingness or refusal to accept the study treatment protocol.
2. Presence of donor-specific anti-HLA antibodies.
3. Known human immunodeficiency virus (HIV) infection.
4. Active hepatitis B or C, or chronic active hepatitis.
5. Uncontrolled active infection at the time of enrollment.
6. Any other condition deemed by the investigator as unsuitable for study inclusion.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
thiotepa 5 mg/kg/day ; busulfan 3.2 mg/kg/day ; fludarabine 30 mg/m²/day	Thiotepa ;busulfan;fludarabine	Eligible patients will receive the following conditioning regimen: thiotepa 5 mg/kg/day on Days -10 to -9; busulfan 3.2 mg/kg/day on Days -8 to -6; fludarabine 30 mg/m²/day on Days -6 to -2; and rabbit anti-thymocyte globulin (rATG) 1.5-2.5 mg/kg/day on Days -5 to -2. Hematopoietic stem cell infusion will be performed on Day 0. Graft-versus-host disease (GVHD) prophylaxis will include cyclosporine A (CsA), mycophenolate mofetil (MMF), and methotrexate (MTX).

Primary Outcome Measures

Name	Time	Method
1-year Relapse-Free Survival,（RFS）	1-year

Secondary Outcome Measures

Name	Time	Method
1-year Overall Survival	1-year
1-year Relapse Rate	1-year
1-year Non-Relapse Mortality	1-year

Trial Locations

Locations (1)

Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China