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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Paediatric Stem Cell Transplant and Immune Deficiency, Department of Pediatric and Adolescent Medicine, Section 4072, Rigshospitalet University Hospital of Copenhagen
  Copenhagen, Denmark Recruiting
• Division of Hematology, Oncology, and Stem Cell Transplantation, The New Children's Hospital, Helsinki University Hospital
  Helsinki, Finland Recruiting
• Princess Máxima Center for Pediatric Oncology
  Utrecht, Netherlands Recruiting
• Department of Pediatric Hematology and Oncology, Oslo University HospitalOslo University Hospital
  Oslo, Norway Recruiting
• Queen Silvia Children's Hospital, Sahlgrenska University Hospital
  Gothenburg, Sweden Recruiting
• Barncancercentrum, avdelning 64, Skane University Hospital
  Lund, Sweden Recruiting

Study contacts

• Principal investigator: Birgitta Versluys, MD, Phd — Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands
• Study coordinator: Karin Mellgren, Prof. MD
• Email: karin.mellgren@vgregion.se
• Phone: +46 (0)31 3421000

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.