Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

NCT ID: NCT00167219

Last Updated: 2023-11-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1999-11-18
• Study Completion Date: 2023-08-09

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.

Conditions

Juvenile Myelomonocytic Leukemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Intent-to-Treat

Patients receiving study regimen.

Group Type: EXPERIMENTAL

Stem Cell Transplant

Intervention Type: BIOLOGICAL

Transplantation on Day 0.

Preparative Regimen

Intervention Type: DRUG

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

Interventions

Stem Cell Transplant

Transplantation on Day 0.

Intervention Type: BIOLOGICAL

Preparative Regimen

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

Intervention Type: DRUG

Other Intervention Names

Bone marrow transplantation

Eligibility Criteria

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.

• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Margaret MacMillan, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

MT1999-20

Identifier Type: OTHER

Identifier Source: secondary_id

9911M24961

Identifier Type: OTHER

Identifier Source: secondary_id

1999LS073

Identifier Type: -

Identifier Source: org_study_id

NCT00262756

Identifier Type: -

Identifier Source: nct_alias