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Comparing two treatment regimens for children with Acute Myeloid Leukemia undergoing stem cell transplantation

A Randomized, Multi-Center Phase III Trial Comparing Two Conditioning Regimens (CloFluBu and BuCyMel) in Children With Acute Myeloid Leukemia Undergoing Allogeneic Stem Cell Transplantation.

Phase 3 Interventional Vastra Gotaland Region · NCT05477589

This study is testing two different treatment plans for children with Acute Myeloid Leukemia who are getting stem cell transplants to see which one helps them survive better and have fewer serious side effects.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment170 (estimated)
AgesN/A to 18 Years
SexAll
SponsorVastra Gotaland Region Government
Drugs / interventionschemotherapy, cyclophosphamide, fludarabine
Locations17 sites (Brussels and 16 other locations)
Trial IDNCT05477589 on ClinicalTrials.gov

What this trial studies

This phase 3 clinical trial is a randomized, open-label, multicenter study that compares two different conditioning regimens for pediatric patients with Acute Myeloid Leukemia (AML) who are undergoing allogeneic stem cell transplantation. The trial aims to determine whether a regimen combining one alkylator and two antimetabolites (CloFluBu) leads to better survival outcomes without severe complications compared to a regimen that uses three alkylating agents (BuCyMel). The study includes both an interventional component, where patients are randomly assigned to one of the two treatment arms, and an observational component that tracks outcomes in patients who do not meet the criteria for randomization but consent to data collection.

Who should consider this trial

Good fit: Ideal candidates are children aged 18 years or younger at the time of initial AML diagnosis who are in hematological remission and meet specific transplant criteria.

Not a fit: Patients who are not in complete remission or do not have a suitable donor for transplantation may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could improve survival rates and reduce complications for children with AML undergoing stem cell transplantation.

How similar studies have performed: Other studies have explored conditioning regimens in pediatric AML, but this specific comparison of alkylators versus antimetabolites is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion criteria for randomization part of the study:

* Age ≤18 years at time of initial AML, age ≤ 21 years at transplantation.
* HCT is performed in a study participating center
* All women of childbearing potential who have to have a negative pregnancy test within 2 weeks prior to the start of treatment.
* Signed informed consent.
* Any relapsed AML after initial treatment according to a defined international AML protocol. (NOPHO-DBH AML 2012/new protocol), or AML in first remission with transplant indications and treatment according to national AML protocol (NOPHO-DBH AML 2012 or new protocol).
* In hematological remission, defined as:

\< 5 % leukemic blasts confirmed by flow cytometry (in patients with an informative leukemia associated immunophenotype) in a bone marrow sample taken ≤14 days prior to start of conditioning and no evidence of extramedullary disease, including in CNS and no leukemic blasts in the peripheral blood (verified by flow cytometry in case immature cells are detected in the peripheral blood differential).

-Patients must have a related or unrelated donor fulfilling any of the following criteria: HLA 10/10 allelic matched, identical, sibling BM donor or HLA 10/10 or 9/10 allelic matched related/unrelated BM or PBSC donor orHLA 5-6/6 unrelated or 6-7-8/8 unrelated Cord Blood (UCB)

Inclusion criteria for observation/registration only:

* Diagnosis of acute myeloid leukemia
* Indication for allogeneic stem cell transplantation, as defined by primary treatment protocol or treating physician.
* Age ≤18 years at time of initial AML, age ≤ 21 years at transplantation.
* Not eligible for randomization, either due to lack of consent or not fulfilling inclusion criteria for interventional part of the study.
* Signed informed consent to prospectively register follow-up data.

Exclusion criteria for the randomization part of the study :

* Diagnosis of myelodysplastic syndrome (MDS).
* Diagnosis of juvenile myelomonocytic leukemia (JMML).
* History of previous malignancy (AML diagnosed as secondary cancer).
* Known diagnosis of Fanconi anemia.
* Prior autologous or allogeneic hematopoietic stem cell transplant.
* Planned prophylactic DLI or other immunotherapeutic interventions after HCT that are not included in the upfront protocol, Planned anti-leukemic medication after HCT that are not included in the upfront protocol
* Known intolerance to any of the chemotherapeutic drugs in the protocol.
* Major organ failure precluding administration of planned chemotherapy.
* Patients with uncontrolled bacterial, viral, or fungal infections (currently taking medication and with progression or no clinical improvement) at time of enrollment.
* Severe concomitant disease that does not allow treatment according to the protocol at the investigator's discretion, e.g. malformation syndromes, cardiac malformations, metabolic disorders, renal impairment (\<30% of normal glomerular filtration rate), severe pulmonary, hepatic or cardiac impairment due to toxicity or infection.
* Karnofsky / Lansky score \< 50%
* Females who are pregnant (positive serum or urine βHCG) or breastfeeding.
* Females of childbearing potential or men who have sexual contact with females of childbearing potential unwilling to use effective forms of birth control or abstinence for one year after transplantation.
* Subjects unwilling or unable to comply with the study procedures.

Exclusion criteria for the observational part of the study:

* Diagnosis of Myelodysplastic syndrome (MDS).
* Diagnosis of Juvenile myelomonocytic leukemia (JMML).
* Age above 21 years at time of transplantation
* No consent is given to prospectively register outcome data
* Prior autologous or allogeneic hematopoietic stem cell transplant.

Where this trial is running

Brussels and 16 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Myeloid Leukemia in RemissionStem Cell TransplantationLeukemiaLeukemia, Myeloid, AcuteNeoplasmsHaematopoietic cell transplantationPaediatric
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.