Official Title: “Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia”

The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT).

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Study Primary Completion Date: December 2013

Prior to transplantation, subjects will receive BUSULFAN via the central venous line, six times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's leukemia. As well, these drugs will destroy the subject's own immune system to help ensure the new bone marrow takes and grows after transplantation. On the day of transplantation, bone marrow or umbilical cord blood from the donor will arrive to the bone marrow transplant unit and be transfused via venous line. These new cells will replace the subject's bone marrow.

Intervention(s) in this Clinical Trial

• Procedure: Stem Cell Transplant
  • As part of the stem-cell transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease, such as cancer. This treatment also kills the healthy stem cells already in the marrow. The transplanted cells from a donor replace the patient's bone marrow and allow the blood counts to recover. Subjects will receive BUSULFAN via the central venous line, six times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day.On the day of transplantation, cells from the donor will arrive to the bone marrow transplant unit and be transfused via venous line.

Primary Measures

• Evaluate long-term DFS in JMML using a common preparative regimen
  • Time Frame: at 1 year after transplant
    Safety Issue?: No

Secondary Measures

• Secondary outcome measures are the incidence of neutrophil engraftment, graft-versus-host disease (GVHD), regimen-related toxicity, and relapse.
  • Time Frame: at 1 year after transplant
    Safety Issue?: No

Inclusion Criteria:

• Patients must have a diagnosis of JMML and fulfill these minimal criteria (International diagnostic criteria for JMML):
• Leukocytosis (> 13,000) with absolute monocytosis (> 1,000)
• The presence of immature myeloid cells in the peripheral blood
• Less than 30% marrow blasts
• Absence of t(9:22) or BCR-ABL transcript
• Adequate major organ function including:
• Cardiac: ejection fraction > 45%
• Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
• Karnofsky performance status > 70% or Lansky score > 50%
• Creatinine must be < 2 x normal for age
• Written informed consent.

Exclusion Criteria:

• Active uncontrolled infection within one week of HCT.

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: N/A

Maximum Age for this Clinical Trial: 18 Years

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Overall Clinical Trial Officials and Contacts

Margaret MacMillan, MD Principal Investigator Masonic Cancer Center, University of Minnesota  

Overall Contact: Margaret MacMillan, M.D. 612-626-2778 macmi002@umn.edu

Information obtained from ClinicalTrials.gov on July 02, 2009

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00167219

Study ID Number: 9911M24961

ClinicalTrials.gov Identifier: NCT00167219

Health Authority: United States: Institutional Review Board