NCT06879847Recruiting
A Single-arm, Prospective Study of TBI + BUMEL As a Conditioning Regimen for Salvage HSCT in Patients with R/R AML
A Single-arm, Prospective Clinical Study on the Efficacy of Total Body Irradiation Combined with Busulfan and Melphalan As Conditioning Regimen for Patients with Relapsed/Refractory Acute Myeloid Leukemia Undergoing Salvage HSCT
Source: ClinicalTrials.gov · Last updated 17 Mar 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
Acute myeloid leukemia (AML) is one of the hematologic malignancies, for which patients typically undergo chemotherapy to achieve complete remission. However, approximately 30% of patients fail to respond to initial treatment, and many experience relapse after achieving remission. For patients with relapsed or refractory AML, allogeneic hematopoietic stem cell transplantation (HSCT) offers a potentially curative option. Sibling-matched HSCT has demonstrated a disease-free survival rate of 20-30%, while unrelated donor transplants yield an overall survival rate of approximately 22%. Haploidentical transplantation is a viable alternative for patients lacking a sibling donor. A 2019 study involving 1,693 patients with relapsed/refractory (R/R) AML revealed that haploidentical transplants yielded outcomes comparable to other transplant modalities, including HLA-matched and 9/10 matched unrelated donor transplants, thus supporting haploidentical transplantation as a viable therapeutic option. The conditioning regimen is a critical component of the transplantation. In China, the modified BU/CY conditioning regimen, which combines busulfan (BU) and cyclophosphamide (CTX), is widely utilized for tumor cytoreduction and immunosuppression. Some centers also employ post-transplant cyclophosphamide (PTCy) to mitigate the risk of graft-versus-host disease (GVHD). Despite advances, relapse remains a significant challenge. Optimizing conditioning regimens to enhance tumor cell targeting and achieve deeper remission is crucial. Additionally, many patients are unfit due to prior chemotherapy, infections, and organ dysfunction, which may make them unable to tolerate high-intensity conditioning. Recent studies suggest that melphalan (MEL)-based conditioning regimens may offer advantages over CTX-based protocols. While total body irradiation (TBI) has been traditionally used in conditioning for HSCT, it is associated with considerable organ toxicity. A low-dose TBI regimen combined with BU+MEL represents a promising conditioning regimen for R/R AML. In preliminary studies, 7 patients treated with this regimen successfully achieved hematopoietic stem cell engraftment. Building on these results, a clinical study is planned to evaluate further the safety and efficacy of the TBI+BUMEL (IBM) conditioning regimen in relapsed/refractory AML, with a focus on improving engraftment rates, reducing relapse rates, minimizing GVHD incidence, and enhancing overall survival outcomes.
- Status (registry)
- Recruiting
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- First submitted
- 11 Mar 2025
- Start date
- 1 Dec 2024
- Primary completion date
- 1 Nov 2027
- Lead sponsor
- The First Affiliated Hospital of Soochow University
- Diseases
- Acute myeloid leukemia, Childhood cancers
- NCT number
- NCT06879847
What you commit to
- Study type: Interventional
- Planned participants: 40
- From 1 Dec 2024 to the primary completion date, 1 Nov 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patients had serious adverse reactions to investigational drugs such as allergies;
- Patients received Class II or higher surgery within 4 weeks prior to enrollment;
Other illness
- Patients with a history of immunodeficiency, or other acquired or congenital diseases, immunodeficiency diseases, and a history of organ transplantation;
- Patients with hypertension, ventricular arrhythmia requiring clinical intervention, acute coronary syndrome, congestive heart failure, stroke, or other grade III or higher cardiovascular events within 6 months;
- Patient has an active and difficult-to-control infection, including but not limited to active fungal, bacterial, or viral infections that require systemic treatment, such as active HIV, hepatitis B or C;
- Other conditions where the investigator deems the patient unsuitable for inclusion.
Other
- Patient has active central nervous system leukemia infiltration;
- Pregnant or lactating patients;
- Patient is currently participating in another clinical studies;
Who the study is looking for
- Age between 14 and 70 years (inclusive of the age limits);
- Patients diagnosed with relapsed/refractory (R/R) AML, meeting the World Health Organization (WHO) 2016 AML diagnostic criteria, must meet one of the following definitions:
- Relapsed AML: Leukemic cells reappear in peripheral blood or bone marrow blasts \>5% after achieving complete remission (CR, CRi) (excluding other causes such as bone marrow regeneration following consolidation chemotherapy), or extramedullary leukemic infiltration occurs.
- Refractory AML:Initial cases that do not respond to two courses of standard treatment. Relapse within 12 months after consolidation therapy. Relapse after 12 months with no response to conventional chemotherapy. Two or more relapses. Persistent extramedullary leukemia.
- Heart, liver, and kidney function must meet the following criteria:
- Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 3× the upper limit of normal (ULN); Total bilirubin ≤ 3× ULN; Serum creatinine ≤ 2× ULN or creatinine clearance ≥ 40 mL/min; Left ventricular ejection fraction (LVEF), as measured by echocardiography or multi-gated acquisition (MUGA) scan, must be within the normal range (\>50%).
- Availability of a suitable allogeneic donor;
- Life expectancy of ≥3 months;
- Karnofsky Performance Status (KPS) ≥ 60%, Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2;
- The patient understands the study protocol and voluntarily signs the informed consent form.
Age in the registry: 14 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hematology Department, The First Affiliated Hospital of Soochow University Coordinator of this site? Confirm its status | Suzhou | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.