Phase 2
N=30
Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT01824693 ↗Enrolled (actual)
30
Serious AEs
7.4%
Results posted
Dec 2018
Primary outcome: Primary: Percent Probability of Event-free Survival (EFS) — 83; 22 percent probability
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Allogeneic Hematopoietic Stem Cell Transplantation (Procedure); Busulfan (Drug); Cyclophosphamide (Drug); Fludarabine Phosphate (Drug); Laboratory Biomarker Analysis (Other); Melphalan (Drug); Mycophenolate Mofetil (Drug); Pharmacological Study (Other); Tacrolimus (Drug)
- Age
- Pediatric, Adult · 0+ yrs
- Sex
- All
- Sponsor
- Children's Oncology Group
- Primary completion
- Dec 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Probability of Event-free Survival (EFS) |
83; 22 | — |
| PRIMARY Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100 |
0; 1 | — |
| SECONDARY Percentage of Participants Who Experience Primary Graft Failure Event Between Arms |
0; 0 | — |
| SECONDARY Percent Probability of 18 Months-relapse Event Between Arms |
17; 55 | — |
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 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.
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) 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
Data sourced from ClinicalTrials.gov (NCT01824693). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.