Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information

Alternate Title

Busulfan, Cyclophosphamide, Melphalan, and Donor Stem Cell Transplant in Treating Young Patients For Juvenile Myelomonocytic Leukemia

Basic Trial Information

Phase Type Status Age Sponsor Protocol IDs Phase II Treatment Active Under 18 NCI UMN-1999LS073 UMN-MT1999-20, NCT00262756

Objectives

Primary

1. 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

1. Determine the probability of engraftment in patients treated with this regimen.
2. Determine the incidence and severity of acute and chronic graft-versus-host disease in patients treated with this regimen.
3. Determine the incidence of regimen-related toxicity in these patients.
4. Determine the incidence of relapse in patients treated with this regimen.

Entry Criteria

Disease Characteristics:

• Diagnosis of juvenile myelomonocytic leukemia (JMML) meeting the following criteria:
  • Leukocytosis (> 13,000/mm³) with absolute monocytosis (> 1,000/mm³)
  • 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

Prior/Concurrent Therapy:

• Not specified

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

Expected Enrollment

A total of 10 patients will be accrued for this study.

Outcomes

Disease-free survival at 1 year post transplantation

Incidence of neutrophil engraftment, graft-versus-host disease (GVHD), regimen-related toxicity, and relapse post transplantation.

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.

Trial Contact Information

Trial Lead Organizations

Masonic Cancer Center at University of Minnesota

Margaret MacMillan, MD, Protocol chair		Ph: 612-626-2778; 888-226-2376 Email: macmi002@umn.edu

U.S.A.
Minnesota
	Minneapolis
	Masonic Cancer Center at University of Minnesota
		Clinical Trials Office - Masonic Cancer Center at University of Minnesota	Ph:  612-624-2620

Registry Information
Official Title		Hematopoietic Cell Transplantation in Children with Juvenile Myelomonocytic Leukemia
Trial Start Date		1999-11-18
Trial Completion Date		2013-08-01 (estimated)
Registered in ClinicalTrials.gov		NCT00262756
Date Submitted to PDQ		2005-09-13
Information Last Verified		2008-10-23
NCI Grant/Contract Number		CA77598

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