Status:
COMPLETED
Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia
Lead Sponsor:
Children's Oncology Group
Collaborating Sponsors:
National Cancer Institute (NCI)
Conditions:
Juvenile Myelomonocytic Leukemia
Eligibility:
All Genders
3-18 years
Phase:
PHASE2
Brief Summary
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 youn...
Detailed Description
PRIMARY OBJECTIVES: I. To compare ? in a randomized fashion ? the day 100 treatment related mortality (TRM) incidence for two myeloablative conditioning regimens, busulfan-fludarabine (fludarabine ph...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
June 24 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 31 2017
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT01824693
Start Date
June 24 2013
End Date
December 31 2017
Last Update
December 5 2018
Active Locations (50)
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1
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
2
Phoenix Childrens Hospital
Phoenix, Arizona, United States, 85016
3
City of Hope Comprehensive Cancer Center
Duarte, California, United States, 91010
4
Mattel Children's Hospital UCLA
Los Angeles, California, United States, 90095