Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

• ClinicalTrials.gov Identifier: NCT00167219
• Recruitment Status: Completed
• First Posted: September 14, 2005
• Last Update Posted: November 3, 2022
• Sponsor: Masonic Cancer Center, University of Minnesota
• Information provided by (Responsible Party): Masonic Cancer Center, University of Minnesota

Study Description

Brief Summary:

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 or disease	Intervention/treatment	Phase
Juvenile Myelomonocytic Leukemia	Biological: Stem Cell Transplant; Drug: Preparative Regimen	Phase 1; Phase 2

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 15 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
• Actual Study Start Date: November 18, 1999
• Actual Primary Completion Date: July 12, 2022
• Actual Study Completion Date: July 12, 2022

Arms and Interventions

Arm	Intervention/treatment
Experimental: Intent-to-Treat; Patients receiving study regimen.	Biological: Stem Cell Transplant; Transplantation on Day 0.; Other Name: Bone marrow transplantation; Drug: Preparative Regimen; Busulfan; Cyclophosphamide; Mesna; Melphalan; Anti-thymocyte Globulin (ATG)

Outcome Measures

Primary Outcome Measures :

1. Determine probability of long-term disease free survival in JMML [ Time Frame: at 1 year after transplant ]

Secondary Outcome Measures :

1. 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 ]

Eligibility Criteria

• Ages Eligible for Study: up to 18 Years (Child, Adult)
• Sexes Eligible for Study: All
• 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

Locations

United States, Minnesota

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States, 55455

Sponsors and Collaborators

Masonic Cancer Center, University of Minnesota

Investigators

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

More Information

• Responsible Party: Masonic Cancer Center, University of Minnesota
• ClinicalTrials.gov Identifier: NCT00167219
• Obsolete Identifiers: NCT00262756
• Other Study ID Numbers: 1999LS073; MT1999-20 ( Other Identifier: Blood and Marrow Transplant Program ); 9911M24961 ( Other Identifier: IRB, University of Minnesota )
• First Posted: September 14, 2005
• Last Update Posted: November 3, 2022
• Last Verified: November 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

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; Chronic Disease; Disease Attributes; Pathologic Processes