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Completed

NCT Number: NCT00167219

Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

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).

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Key information

Conditions

Juvenile Myelomonocytic Leukemia Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myelomonocytic, Juvenile Myelodysplastic-Myeloproliferative Diseases Neoplasms Neoplasms by Histologic Type

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Stem cell transplant

Biological

Transplantation on Day 0.

Other names: Bone marrow transplantation

Preparative Regimen

Drug
  • Busulfan
  • Cyclophosphamide
  • Mesna
  • Melphalan
  • Anti-thymocyte Globulin (ATG)

Primary outcomes

  1. Determine Probability of Long-term Disease Free Survival in JMML

    Time frame: at 1 year after transplant

Secondary outcomes

  1. Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.

    Time frame: at 1 year after transplant

    Measured by AGVHD Grades 2 - 4.

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Official study title

Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Important dates

Study start
1999
Primary completion
2022
Study completion
2023
First posted
Sep 14, 2005
Registry last updated
Nov 7, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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