Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

Phase 1

Completed

• Conditions: Juvenile Myelomonocytic Leukemia
• Interventions: Biological: Stem Cell Transplant; Drug: Preparative Regimen

• Registration Number: NCT00167219
• Lead Sponsor: 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).

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.

Study Timeline

• Study Start: 1999-11-18
• Study First Submitted: 2005-09-09
• Study First Submitted QC: 2005-09-09
• Study First Posted: 2005-09-14
• Primary Completion: 2022-07-12
• Study Completion: 2023-08-09
• Results First Submitted: 2023-09-11
• Status Verified: 2023-11
• Results First Submitted QC: 2023-11-03
• Last Update Submitted: 2023-11-03
• Results First Posted: 2023-11-07
• Last Update Posted: 2023-11-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Intent-to-Treat	Preparative Regimen	Patients receiving study regimen.
Intent-to-Treat	Stem Cell Transplant	Patients receiving study regimen.

Primary Outcome Measures

Name	Time	Method
Determine Probability of Long-term Disease Free Survival in JMML	at 1 year after transplant

Secondary Outcome Measures

Name	Time	Method
Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.	at 1 year after transplant	Measured by AGVHD Grades 2 - 4.

Trial Locations

Locations (1)

Masonic Cancer Center, University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States