NCT00167219

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Nov 1999

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 18, 1999

Completed
5.8 years until next milestone

First Submitted

Initial submission to the registry

September 9, 2005

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 14, 2005

Completed
16.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 12, 2022

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

August 9, 2023

Completed
3 months until next milestone

Results Posted

Study results publicly available

November 7, 2023

Completed
Last Updated

November 7, 2023

Status Verified

November 1, 2023

Enrollment Period

22.7 years

First QC Date

September 9, 2005

Results QC Date

September 11, 2023

Last Update Submit

November 3, 2023

Conditions

Juvenile Myelomonocytic Leukemia

Keywords

Stem cell transplantlong term survivalretinoic acid

Outcome Measures

Primary Outcomes (1)

  • Determine Probability of Long-term Disease Free Survival in JMML

    at 1 year after transplant

Secondary Outcomes (1)

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

    at 1 year after transplant

Study Arms (1)

Intent-to-Treat

EXPERIMENTAL

Patients receiving study regimen.

Biological: Stem Cell TransplantDrug: Preparative Regimen

Interventions

Stem Cell TransplantBIOLOGICAL

Transplantation on Day 0.

Also known as: Bone marrow transplantation
Intent-to-Treat
Preparative RegimenDRUG

* Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)

Intent-to-Treat

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • Active uncontrolled infection within one week of HCT.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Leukemia, Myelomonocytic, Juvenile

Interventions

Stem Cell TransplantationBone Marrow Transplantation

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsMyelodysplastic-Myeloproliferative DiseasesBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Cell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, OperativeTissue Transplantation

Results Point of Contact

Title
Dr. Margaret MacMillan, MD
Organization
University of Minnesota, Masonic Cancer Center
macmi002@umn.edu
+1 612-626-2961

Study Officials

  • Margaret MacMillan, MD

    Masonic Cancer Center, University of Minnesota

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 9, 2005

First Posted

September 14, 2005

Study Start

November 18, 1999

Primary Completion

July 12, 2022

Study Completion

August 9, 2023

Last Updated

November 7, 2023

Results First Posted

November 7, 2023

Record last verified: 2023-11

Locations

US(1)