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Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

This study is currently recruiting participants.
Verified December 2017 by Masonic Cancer Center, University of Minnesota
Sponsor:
ClinicalTrials.gov Identifier:
NCT00167219
First Posted: September 14, 2005
Last Update Posted: December 5, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
September 9, 2005
September 14, 2005
December 5, 2017
November 18, 1999
December 2020   (Final data collection date for primary outcome measure)
Determine probability of long-term disease free survival in JMML [ Time Frame: at 1 year after transplant ]
Evaluate long-term DFS in JMML using a common preparative regimen
Complete list of historical versions of study NCT00167219 on ClinicalTrials.gov Archive Site
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 ]
Secondary outcome measures are the incidence of neutrophil engraftment, GVHD, regimen-related toxicity, and relapse.
Not Provided
Not Provided
 
Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
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).

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.

Interventional
Phase 1
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Juvenile Myelomonocytic Leukemia
  • Biological: Stem Cell Transplant
    Transplantation on Day 0.
    Other Name: Bone marrow transplantation
  • Drug: Preparative Regimen
    • Busulfan
    • Cyclophosphamide
    • Mesna
    • Melphalan
    • Anti-thymocyte Globulin (ATG)
Experimental: Intent-to-Treat
Patients receiving study regimen.
Interventions:
  • Biological: Stem Cell Transplant
  • Drug: Preparative Regimen
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
20
December 2020
December 2020   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
up to 18 Years   (Child, Adult)
No
Contact: Kim Nelson, RN 612-273-2925 knelso62@fairview.org
United States
 
 
NCT00167219
1999LS073
MT1999-20 ( Other Identifier: Blood and Marrow Transplant Program )
9911M24961 ( Other Identifier: IRB, University of Minnesota )
Yes
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
Not Provided
Masonic Cancer Center, University of Minnesota
Masonic Cancer Center, University of Minnesota
Not Provided
Principal Investigator: Margaret MacMillan, MD Masonic Cancer Center, University of Minnesota
Masonic Cancer Center, University of Minnesota
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP