Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia

Status: Recruiting
Phase: Phase 2
Diagnosis: Pediatric Leukemia
NCT ID: NCT01824693 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID: 14-280


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.


Conducting Institutions:
Dana-Farber Cancer Institute

Overall PI:
Leslie Lehmann, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

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
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