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Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

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.
 
ClinicalTrials.gov Identifier: NCT00167219
Recruitment Status : Recruiting
First Posted : September 14, 2005
Last Update Posted : April 22, 2022
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

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.

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Study Type : Interventional  (Clinical Trial)
Estimated 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 : November 18, 1999
Estimated Primary Completion Date : July 28, 2022
Estimated Study Completion Date : July 28, 2022


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)




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 ]


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.

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
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Contact: Lisa Burke, RN 612-273-8482 lburke3@Fairview.org

Locations
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United States, Minnesota
Masonic Cancer Center, University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Lisa Burke    612-273-8482    lburke3@Fairview.org   
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
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Principal Investigator: Margaret MacMillan, MD Masonic Cancer Center, University of Minnesota
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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    Key Record Dates
Last Update Posted: April 22, 2022
Last Verified: April 2022

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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:
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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