Thiotepa, busulfan, and fludarabine treatment for elderly patients with high-risk acute myeloid leukemia

A Multicenter, Single-Arm, Prospective Clinical Study of Thiotepa Combined With Busulfan and Fludarabine Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplantation in Elderly Patients With High-Risk Acute Myeloid Leukemia

This study is testing a new treatment plan using thiotepa, busulfan, and fludarabine to see if it helps older patients with high-risk acute myeloid leukemia stay cancer-free after a stem cell transplant.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 56 (estimated)
• Ages: 55 Years to 70 Years
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Drugs / interventions: chemotherapy, methotrexate, fludarabine
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06869265 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the safety and efficacy of a conditioning regimen involving thiotepa, busulfan, and fludarabine for haploidentical hematopoietic stem cell transplantation in elderly patients aged 55 and older with high-risk acute myeloid leukemia. The trial is a multicenter, single-arm, prospective study that aims to enroll 56 eligible patients. Participants will receive a specific treatment regimen followed by stem cell infusion, with the primary goal of assessing 1-year relapse-free survival rates.

Who should consider this trial

Good fit: Ideal candidates are elderly patients aged 55-70 with high-risk acute myeloid leukemia, including those who are relapsed or refractory.

Not a fit: Patients who are unwilling to accept the study treatment protocol or have specific contraindications, such as donor-specific anti-HLA antibodies, may not benefit from this study.

Why it matters

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

How similar studies have performed: While this approach is being tested in this specific population, similar conditioning regimens have shown promise in other studies involving hematopoietic stem cell transplantation.

Eligibility criteria

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.

Where this trial is running

Beijing, Beijing Municipality

• Peking University People's Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: Yu Wang MD

Conditions Acute Myeloid Leukemia; High Risk Acute Myeloid Leukemia; Relapsed/Refractory Acute Myeloid Leukemia