Clinical Trial: Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: 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 Summary:
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.
Sponsor: Masonic Cancer Center, University of Minnesota
Current Primary Outcome: Determine probability of long-term disease free survival in JMML [ Time Frame: at 1 year after transplant ]
Original Primary Outcome: Evaluate long-term DFS in JMML using a common preparative regimen
Current Secondary Outcome: 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 ]
Original Secondary Outcome: Secondary outcome measures are the incidence of neutrophil engraftment, GVHD, regimen-related toxicity, and relapse.
Information By: Masonic Cancer Center, University of Minnesota
Dates:
Date Received: September 9, 2005
Date Started: November 18, 1999
Date Completion: December 2020
Last Updated: February 17, 2017
Last Verified: February 2017
