Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

Juvenile Myelomonocytic Leukemia Clinical Trial

Official title:

Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00167219
• Other study ID #: 1999LS073
• Secondary ID: MT1999-209911M24
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: November 18, 1999
• Est. completion date: August 9, 2023

Study information

• Verified date: November 2023
• Source: Masonic Cancer Center, University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Other known NCT identifiers

• NCT00262756

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: August 9, 2023
• Est. primary completion date: July 12, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Juvenile Myelomonocytic Leukemia
• Leukemia
• Leukemia, Myelomonocytic, Acute
• Leukemia, Myelomonocytic, Chronic
• Leukemia, Myelomonocytic, Juvenile

Intervention

Biological:
• Stem Cell Transplant
Transplantation on Day 0.

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

Locations

Country	Name	City	State
United States	Masonic Cancer Center, University of Minnesota	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Determine Probability of Long-term Disease Free Survival in JMML		at 1 year after transplant
Secondary	Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.	Measured by AGVHD Grades 2 - 4.	at 1 year after transplant