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Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

This study is currently recruiting participants.
Verified December 2012 by Masonic Cancer Center, University of Minnesota
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00167219
First received: September 9, 2005
Last updated: December 17, 2012
Last verified: December 2012
History of Changes
  Purpose

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).


Condition Intervention Phase
Juvenile Myelomonocytic Leukemia
 Procedure: Stem Cell Transplant
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:
  • Determine probability of long-term disease free survival in JMML [ Time Frame: at 1 year after transplant ] [ Designated as safety issue: No ]

Secondary Outcome 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 ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: December 1999
Estimated Study Completion Date: May 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intent-to-Treat
Patients receiving study regimen.
 Procedure: Stem Cell Transplant
As part of the stem-cell transplant process, patients receive high doses of chemotherapy 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.
Other Name: Bone marrow transplantation

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00167219

Contacts
Contact: Patricia Kleinke, RN 612-273-0857 pkleink1@fairview.org

Locations
United States, Minnesota
Masonic Cancer Center, University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Margaret MacMillan, MD     612-626-2778     macmi002@umn.edu    
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Margaret MacMillan, MD Masonic Cancer Center, University of Minnesota
  More Information

No publications provided

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00167219     History of Changes
Obsolete Identifiers: NCT00262756
Other Study ID Numbers: 1999LS073, MT1999-20, 9911M24961
Study First Received: September 9, 2005
Last Updated: December 17, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by Masonic Cancer Center, University of Minnesota:
Stem cell transplant
long term survival
retinoic acid

Additional relevant MeSH terms:
Leukemia
Leukemia, Myelomonocytic, Acute
Leukemia, Myelomonocytic, Chronic
Leukemia, Myelomonocytic, Juvenile
Neoplasms by Histologic Type
 Neoplasms
Leukemia, Myeloid
Myelodysplastic-Myeloproliferative Diseases
Bone Marrow Diseases
Hematologic Diseases

ClinicalTrials.gov processed this record on May 23, 2013