Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML) - Full Text View

Sponsor:	Masonic Cancer Center, University of Minnesota
Information provided by:	Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:	NCT00167219

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 I Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Resource links provided by NLM:

MedlinePlus related topics: Bone Marrow Transplantation Cancer Leukemia, Adult Acute Leukemia, Adult Chronic Leukemia, Childhood

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 2014
Estimated Primary Completion Date:	December 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Intent-to-Treat: Experimental Patients receiving study regimen.	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.

Arms

Assigned Interventions

Intent-to-Treat: Experimental

Patients receiving study regimen.

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.

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: Margaret MacMillan, M.D.

612-626-2778

macmi002@umn.edu

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 ( MacMillan, Margaret L., MD )
Study ID Numbers:	9911M24961, MT1999-20, 1999LS073
Study First Received:	September 9, 2005
Last Updated:	July 6, 2009
ClinicalTrials.gov Identifier:	NCT00167219 History of Changes
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, Myelomonocytic, Acute
Leukemia
Neoplasms
Neoplasms by Histologic Type
Hematologic Diseases

Leukemia, Myelomonocytic, Chronic
Leukemia, Myeloid
Leukemia, Myelomonocytic, Juvenile
Bone Marrow Diseases
Myelodysplastic-Myeloproliferative Diseases

ClinicalTrials.gov processed this record on November 27, 2009