Clinical trial · Interventional
Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia
A Randomized Phase II Study Comparing Two Different Conditioning Regimens Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) for Children With Juvenile Myelomonocytic Leukemia (JMML)
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This randomized phase II trial studies how well giving busulfan, cyclophosphamide, and melphalan or busulfan and fludarabine phosphate before donor hematopoietic cell transplant works in treating younger patients with juvenile myelomonocytic leukemia. Giving chemotherapy before a donor hematopoietic transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether giving busulfan, cyclophosphamide, and melphalan or busulfan and fludarabine phosphate before a donor stem cell transplant is more effective in treating juvenile myelomonocytic leukemia.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Juvenile Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (9)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Allogeneic Hematopoietic Stem Cell Transplantation | Procedure | — | UNRESOLVED |
| Busulfan | Drug | Busulfan | ALIAS |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Fludarabine Phosphate | Drug | Fludarabine | ALIAS |
| Laboratory Biomarker Analysis | Other | — | UNRESOLVED |
| Melphalan | Drug | Melphalan | ALIAS |
| Mycophenolate Mofetil | Drug | — | UNRESOLVED |
| Pharmacological Study | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Arm I (busulfan, cyclophosphamide, melphalan)
- description
- CONDITIONING REGIMEN: Patients receive busulfan IV QD, every 12 hours, or every 6 hours over 2-3 hours on days -8 to -5, cyclophosphamide IV QD over 60 minutes on days -4 and -3, and melphalan IV over 15-30 minutes on day -1. TRANSPLANT: Patients undergo allogeneic HCT no sooner than 24 hours after the last dose of chemotherapy. Patients receive tacrolimus IV or PO on days -1 to 98 (related donor) or 180 (unrelated donor) and mycophenolate mofetil IV over 2 hours or PO every 8 hours on days 1-30 (related donor) or 45 (unrelated donor).
- interventionNames
- Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
- Drug: Busulfan
- Drug: Cyclophosphamide
- Other: Laboratory Biomarker Analysis
- Drug: Melphalan
- Drug: Mycophenolate Mofetil
- Other: Pharmacological Study
- Drug: Tacrolimus
- type
- EXPERIMENTAL
- label
- Arm II (busulfan, fludarabine phosphate)
- description
- CONDITIONING REGIMEN: Patients receive busulfan as in Arm I and fludarabine phosphate IV over 1 hour on days -5 to -2. TRANSPLANT: Patients undergo allogeneic HCT as in Arm I. Patients receive tacrolimus IV or PO on days -1 to 98 (related donor) or 180 (unrelated donor) and mycophenolate mofetil IV over 2 hours or PO every 8 hours on days 1-30 (related donor) or 45 (unrelated donor).
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Months
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Patients must have a strong clinical suspicion of JMML, based on a modified category 1 of the revised diagnostic criteria; specifically, eligible patients must have all of the following: * Splenomegaly * Absolute monocyte count (AMC) \> 1000/uL * Blasts in peripheral blood (PB)/bone marrow (BM) \< 20% * For the 7-10% of patients without splenomegaly, the diagnostic entry criteria must include all other features described above and at least 2 of the following criteria: * Circulating myeloid precursors * White blood cell (WBC) \> 10,000/uL * Increased fetal hemoglobin (HgbF) for age * Sargramostim (GM-CSF) hypersensitivity OR, patients must have been previously diagnosed with JMML * Patients must be previously untreated with HCT * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met Exclusion Criteria: * Patients with a known germline mutation of PTPN11 (Noonan?s Syndrome) are not eligible * Patients with a known history of NF1 (Neurofibromatosis Type 1) and either * A history of a tumor of the central nervous system (astrocytoma or optic glioma), or * A malignant peripheral nerve sheath tumor with a complete remission of \< 1 year are not eligible * Human immunodeficiency virus (HIV) positive patients are not eligible
References
Publications (0)
Data not yet available