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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
  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 Biological: Stem Cell Transplant Drug: Preparative Regimen Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Resource links provided by NLM:


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 ]

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 ]

Estimated Enrollment: 20
Actual Study Start Date: November 18, 1999
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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)

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

Information from the National Library of Medicine

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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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00167219


Contacts
Contact: Kim Nelson, RN 612-273-2925 knelso62@fairview.org

Locations
United States, Minnesota
Masonic Cancer Center, University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Kim Nelson    612-273-2925    knelso62@fairview.org   
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     History of Changes
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 Submitted: September 9, 2005
First Posted: September 14, 2005
Last Update Posted: December 5, 2017
Last Verified: December 2017

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