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

Completed · Phase 2

Conditions studied: Juvenile Myelomonocytic Leukemia

In brief

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.

Key facts

Study ID

NCT01824693

Run by

Children's Oncology Group

People needed

30

Starts

2013-06-24

Expected to finish

2017-12-31

Last updated by the study team

2018-12-05

Who can join

Age: 0 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Children's Hospital of Alabama — Birmingham, Alabama, United States
• Phoenix Childrens Hospital — Phoenix, Arizona, United States
• City of Hope Comprehensive Cancer Center — Duarte, California, United States
• Mattel Children's Hospital UCLA — Los Angeles, California, United States
• Children's Hospital of Orange County — Orange, California, United States
• Rady Children's Hospital - San Diego — San Diego, California, United States
• UCSF Medical Center-Parnassus — San Francisco, California, United States
• UCSF Medical Center-Mission Bay — San Francisco, California, United States
• Alfred I duPont Hospital for Children — Wilmington, Delaware, United States
• Nemours Children's Clinic-Jacksonville — Jacksonville, Florida, United States
• Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States
• Children's Healthcare of Atlanta - Egleston — Atlanta, Georgia, United States
• Lurie Children's Hospital-Chicago — Chicago, Illinois, United States
• Riley Hospital for Children — Indianapolis, Indiana, United States
• University of Iowa/Holden Comprehensive Cancer Center — Iowa City, Iowa, United States
• Norton Children's Hospital — Louisville, Kentucky, United States
• Johns Hopkins University/Sidney Kimmel Cancer Center — Baltimore, Maryland, United States
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States
• Wayne State University/Karmanos Cancer Institute — Detroit, Michigan, United States
• University of Minnesota/Masonic Cancer Center — Minneapolis, Minnesota, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• The Childrens Mercy Hospital — Kansas City, Missouri, United States
• Cardinal Glennon Children's Medical Center — St Louis, Missouri, United States
• University of Nebraska Medical Center — Omaha, Nebraska, United States
• Hackensack University Medical Center — Hackensack, New Jersey, United States

Full record on ClinicalTrials.gov

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