Acute Myeloid Leukemia (AML)Phase 2NCT06869265
Thiotepa Combined With Busulfan and Fludarabine Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplantation in Elderly Patients With High-Risk Acute Myeloid Leukemia
A Phase 2 study testing Thiotepa ;busulfan;fludarabine in people with Acute Myeloid Leukemia (AML) and High Risk Acute Myeloid Leukemia(AML), run by Peking University People's Hospital at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 55 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML), High Risk Acute Myeloid Leukemia(AML), Relapsed/Refractory Acute Myeloid Leukemia (AML)
- What is being tested?
- Thiotepa ;busulfan;fludarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2025to 1 Apr 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University People's Hospital
Beijing, Beijing Municipality, 100044
Yu Wang, 861088326001, ywyw3127@sina.com
xiaojun huang
About this study
Official title: 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
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.
Read more
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.
- Conditions
- Acute Myeloid Leukemia (AML), High Risk Acute Myeloid Leukemia(AML), Relapsed/Refractory Acute Myeloid Leukemia (AML)
- Being tested
- Thiotepa ;busulfan;fludarabine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thiotepa ;busulfan;fludarabineDrug
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).
| Study group | Role | What they receive |
|---|---|---|
| thiotepa 5 mg/kg/day ; busulfan 3.2 mg/kg/day ; fludarabine 30 mg/m²/day | Experimental | 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). Receives: Drug: Thiotepa ;busulfan;fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
1-year Relapse-Free Survival,（RFS）
Measured: 1-year
Secondary outcomes (3)
1-year Overall Survival
Measured: 1-year
1-year Relapse Rate
Measured: 1-year
1-year Non-Relapse Mortality
Measured: 1-year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2025
- Study start1 May 2025
- Primary completion1 Apr 2028 (estimated)
- Study completion1 Apr 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06869265
- Sponsor's protocol ID
- 2024PHD035-001
- Status last verified
- Mar 2025
- First submitted
- 2 Mar 2025
- Last updated
- 18 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06869265
- Acronym / sponsor ID
- 2024PHD035-001
- Conditions
- Acute Myeloid Leukemia (AML), High Risk Acute Myeloid Leukemia(AML), Relapsed/Refractory Acute Myeloid Leukemia (AML)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 1 May 2025 (actual)
- Primary completion
- 1 Apr 2028 (estimated)
- Study completion
- 1 Apr 2029 (estimated)
- First posted
- 11 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| thiotepa 5 mg/kg/day ; busulfan 3.2 mg/kg/day ; fludarabine 30 mg/m²/day | Experimental | Drug: Thiotepa ;busulfan;fludarabineEligible 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). |
Who may qualify
- Ages
- 55 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Thiotepa ;busulfan;fludarabineEligible 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). | Drug | thiotepa 5 mg/kg/day ; busulfan 3.2 mg/kg/day ; fludarabine 30 mg/m²/day |
Primary outcome
1-year Relapse-Free Survival,（RFS）
Time frame: 1-year
3 secondary outcomes
1-year Overall Survival
Time frame: 1-year
1-year Relapse Rate
Time frame: 1-year
1-year Non-Relapse Mortality
Time frame: 1-year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thiotepa ;busulfan;fludarabine in people with Acute Myeloid Leukemia (AML) and High Risk Acute Myeloid Leukemia(AML), run by Peking University People's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yu Wang MD (86-8832666, ywyw3172@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1-year Relapse-Free Survival,（RFS） (time frame: 1-year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2025.