Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML) - Tabular View

Tracking Information

First Received Date ^ICMJE

September 9, 2005

Last Updated Date

July 6, 2009

Start Date ^ICMJE

December 1999

Estimated Primary Completion Date

December 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Determine probability of long-term disease free survival in JMML [ Time Frame: at 1 year after transplant ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Evaluate long-term DFS in JMML using a common preparative regimen

Change History

Complete list of historical versions of study NCT00167219 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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 ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Secondary outcome measures are the incidence of neutrophil engraftment, GVHD, regimen-related toxicity, and relapse.

Descriptive Information

Brief Title ^ICMJE

Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

Official Title ^ICMJE

Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

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 Phase

Phase I, Phase II

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Condition ^ICMJE

Juvenile Myelomonocytic Leukemia

Intervention ^ICMJE

Procedure: Stem Cell Transplant

Study Arms / Comparison Groups

Experimental: Patients receiving study regimen.

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

May 2014

Estimated Primary Completion Date

December 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Gender

Both

Ages

up to 18 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Margaret MacMillan, M.D.

612-626-2778

macmi002@umn.edu

Location Countries ^ICMJE

United States

Administrative Information

NCT ID ^ICMJE

NCT00167219

Responsible Party

MacMillan, Margaret L., MD, Masonic Cancer Center, University of Minnesota

Study ID Numbers ^ICMJE

9911M24961, MT1999-20, 1999LS073

Study Sponsor ^ICMJE

Masonic Cancer Center, University of Minnesota

Collaborators ^ICMJE

Investigators ^ICMJE

Principal Investigator:

Margaret MacMillan, MD

Masonic Cancer Center, University of Minnesota

Information Provided By

Masonic Cancer Center, University of Minnesota

Verification Date

July 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP