Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

Sponsor
Masonic Cancer Center, University of Minnesota (Other)
Overall Status
Recruiting
CT.gov ID
NCT00167219
Collaborator
(none)
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Study Details

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
  • 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
Anticipated Enrollment :
20 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 :
Nov 18, 1999
Anticipated Primary Completion Date :
Jul 28, 2022
Anticipated Study Completion Date :
Jul 28, 2022

Arms and Interventions

Arm Intervention/Treatment
Experimental: Intent-to-Treat

Patients receiving study regimen.

Biological: Stem Cell Transplant
Transplantation on Day 0.
Other Names:
  • 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 [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. [at 1 year after transplant]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Masonic Cancer Center, University of Minnesota
    ClinicalTrials.gov Identifier:
    NCT00167219
    Other Study ID Numbers:
    • 1999LS073
    • MT1999-20
    • 9911M24961
    • NCT00262756
    First Posted:
    Sep 14, 2005
    Last Update Posted:
    Apr 22, 2022
    Last Verified:
    Apr 1, 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
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Apr 22, 2022