Clinical Trials /

Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

NCT00167219

Description:

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).

Related Conditions:
  • Juvenile Myelomonocytic Leukemia
Recruiting Status:

Completed

Phase:

Phase 1/Phase 2

URL:

https://clinicaltrials.gov/show/NCT00167219

Trial Eligibility

Document

Title

  • Brief Title: Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
  • Official Title: Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Clinical Trial IDs

  • ORG STUDY ID: 1999LS073
  • SECONDARY ID: MT1999-20
  • SECONDARY ID: 9911M24961
  • NCT ID: NCT00167219
  • NCT ALIAS: NCT00262756

Conditions

  • Juvenile Myelomonocytic Leukemia

Interventions

DrugSynonymsArms
Stem Cell TransplantBone marrow transplantationIntent-to-Treat
Preparative RegimenIntent-to-Treat

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).

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.

Trial Arms

NameTypeDescriptionInterventions
Intent-to-TreatExperimentalPatients receiving study regimen.
  • Stem Cell Transplant
  • Preparative Regimen

Eligibility 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.
Maximum Eligible Age:18 Years
Minimum Eligible Age:N/A
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Determine probability of long-term disease free survival in JMML
Time Frame:at 1 year after transplant
Safety Issue:
Description:

Secondary Outcome Measures

Measure: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
Safety Issue:
Description:

Details

Phase:Phase 1/Phase 2
Primary Purpose:Interventional
Overall Status:Completed
Lead Sponsor:Masonic Cancer Center, University of Minnesota

Trial Keywords

  • Stem cell transplant
  • long term survival
  • retinoic acid

Last Updated

August 10, 2021