Busulfan, Cyclophosphamide, Melphalan, and Donor Stem Cell Transplant in Treating Young Patients For Juvenile Myelomonocytic Leukemia
Information source: National Cancer Institute (NCI)
Information obtained from ClinicalTrials.gov on November 03, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Leukemia
Intervention: anti-thymocyte globulin (Drug); busulfan (Drug); cyclophosphamide (Drug); cyclosporine (Drug); isotretinoin (Drug); melphalan (Drug); methotrexate (Drug); mycophenolate mofetil (Drug); allogeneic bone marrow transplantation (Procedure); umbilical cord blood transplantation (Procedure)
Phase: Phase 2
Status: Recruiting
Sponsored by: Masonic Cancer Center, University of Minnesota Official(s) and/or principal investigator(s):
Margaret L. MacMillan, MD, Study Chair, Affiliation: Masonic Cancer Center, University of Minnesota
Summary
RATIONALE: Giving chemotherapy drugs, such as busulfan, cyclophosphamide, and melphalan,
before a donor bone marrow transplant or an umbilical cord transplant helps stop the growth
of cancer cells. It also helps 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.
PURPOSE: This phase II trial is studying how well giving busulfan and cyclophosphamide
together with melphalan works in treating young patients who are undergoing a donor bone
marrow transplant or an umbilical cord transplant for juvenile myelomonocytic leukemia.
Clinical Details
Official title: Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
Study design: Treatment, Open Label
Primary outcome: Disease-free survival at 1 year post transplantation
Secondary outcome: Incidence of neutrophil engraftment, graft-versus-host disease (GVHD), regimen-related toxicity, and relapse post transplantation.
Detailed description:
OBJECTIVES:
Primary
- Determine the long-term, disease-free survival of children with juvenile myelomonocytic
leukemia treated with busulfan, cyclophosphamide, and melphalan followed by allogeneic
hematopoietic cell transplantation.
Secondary
- Determine the probability of engraftment in patients treated with this regimen.
- Determine the incidence and severity of acute and chronic graft-versus-host disease in
patients treated with this regimen.
- Determine the incidence of regimen-related toxicity in these patients.
- Determine the incidence of relapse in patients treated with this regimen.
OUTLINE:
- Preparative therapy: Patients receive busulfan IV over 2 hours every 6 hours on days -7 to - 4, cyclophosphamide IV over 2 hours on days -3 and -2, and melphalan IV over 30 minutes on day - 1. Patients who undergo umbilical cord blood transplantation also receive anti-thymocyte globulin IV over 4-6 hours twice daily on days - 3 to -1.
- Allogeneic hematopoietic stem cell transplantation: Patients undergo allogeneic bone
marrow or umbilical cord blood transplantation on day 0.
- Post-transplant therapy: Patients receive cyclosporine IV 2-3 times daily on days -3 to
60 followed by a taper until approximately day 100. Patients who have an umbilical cord
blood donor also receive mycophenolate mofetil IV on days 5-19. Patients who have a bone
marrow donor also receive methotrexate IV on days 1, 3, 6, and 11. All patients receive
oral isotretinoin once a day beginning on day 60 and continuing for 1 year.
After completion of study treatment, patients are followed periodically for survival.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.
Eligibility
Minimum age: N/A.
Maximum age: 17 Years.
Gender(s): Both.
Criteria:
DISEASE CHARACTERISTICS:
- Diagnosis of juvenile myelomonocytic leukemia (JMML) meeting the following criteria:
- Leukocytosis (> 13,000/mm^3) with absolute monocytosis (> 1,000/mm^3)
- Presence of immature myeloid cells in the peripheral blood
- Less than 30% marrow blasts
- Absence of t(9: 22) or BCR-ABL transcript
- Must have a HLA-matched, related or unrelated donor available, determined by class I antigens (HLA-A and - B) and class II antigens (DRB1 or DR [if parental typing
available]), meeting one of the following criteria:
- 6/6 or 5/6 antigen match for bone marrow
- 6/6, 5/6, or 4/6 antigen match for umbilical cord blood
PATIENT CHARACTERISTICS:
Performance status
- Karnofsky 70-100% OR
- Lansky 50-100%
Life expectancy
- Not specified
Hematopoietic
- See Disease Characteristics
Hepatic
- No clinical evidence of hepatic failure (e. g., coagulopathy or ascites)
Renal
- Creatinine < 2 times normal
Cardiovascular
- LVEF ≥ 45% by MUGA or echocardiography
Other
- No active uncontrolled infection
PRIOR CONCURRENT THERAPY:
- Not specified
Locations and Contacts
Masonic Cancer Center at University of Minnesota, Minneapolis, Minnesota 55455, United States; Recruiting
Clinical Trials Office - Masonic Cancer Center at University o, Phone: 612-624-2620
Additional Information
Clinical trial summary from the National Cancer Institute's PDQ® database
Starting date: November 1999
Last updated: October 24, 2008
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