Clinical Trial: Evaluating the Safety and Effectiveness of an Umbilical Cord Blood Stem Cell Transplant (BMT CTN 0604)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multi-Center, Phase II Trial of Non-Myeloablative Conditioning (NST) and Transplantation of Umbilical Cord Blood (UCB) From Unrelated Donors in Patients With Hematologic Malignancies (BMT CTN #0604)

Brief Summary: A bone marrow transplant, which is a type of stem cell transplant, is a treatment option for people with leukemia or lymphoma. Recently, stem cell transplants using umbilical cord blood have become a treatment option for people with these types of cancers. This study will evaluate the effectiveness of a stem cell transplant using umbilical cord blood, along with lower doses of chemotherapy, to treat people with leukemia or lymphoma.

Detailed Summary:

Leukemia and lymphoma are types of blood cancers. Chemotherapy is a common treatment option for people with these types of cancers, but if the cancer does not respond well to chemotherapy, or if the cancer returns, people may need to consider other options. A bone marrow transplant, which is a type of stem cell transplant in which healthy bone marrow is donated to a patient by a related or unrelated donor, is commonly used to treat leukemia and lymphoma. Recently, stem cell transplants using umbilical cord blood have become a viable option to treat these types of cancers. Traditionally, umbilical cord blood, which is the blood left over in the placenta after a baby is born, has been disposed of with the placenta. However, over the past few years, doctors have begun to collect and freeze the umbilical cord blood cells so that they may be used in stem cell transplant procedures at a later time.

Typically, people who are undergoing a stem cell transplant receive high doses of chemotherapy before the transplant to prepare their bodies to accept the donor stem cells. In this study, participants will undergo a new type of stem cell transplant called a nonmyeloablative transplant, which is a reduced intensity method of transplantation that does not require high doses of chemotherapy. The purpose of the study is to examine the safety and effectiveness of a nonmyeloablative stem cell transplant that uses umbilical cord blood as a treatment option for people with leukemia or lymphoma.

This study will enroll people with leukemia or lymphoma. Participants will be admitted to the hospital and will first receive a type of chemotherapy called cyclophosphamide, which will be given intravenously on the sixth day before the transplant. In addition, another type of chemotherapy, fludarabine, will be given intravenously each day for 5 days be
Sponsor: Medical College of Wisconsin

Current Primary Outcome: Overall Survival at 180 Days From the Time of Transplant [ Time Frame: Measured at Month 6 and Year 1 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Neutrophil Recovery [ Time Frame: Measured at Days 28, 56, 90, and 100 ]
    Neutrophil recovery is defined as achieving an absolute neutrophil count ≥ 500/mm3 for three consecutive measurements on different days.
  • Primary Graft Failure [ Time Frame: Measured at Day 100 ]
    Primary graft failure is defined as < 5% donor chimerism on all measurements prior to and day-100.
  • Secondary Graft Failure [ Time Frame: Measured at Day 100 ]
    Secondary graft failure is defined initial recovery followed by neutropenia with < 5% donor chimerism.
  • Platelet Recovery to 20K [ Time Frame: Measured at Days 56, 90, and 100 ]
    Platelet recovery is defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count >20,000/mm3 with no platelet transfusions in the preceding seven days.
  • Donor Cell Engraftment [ Time Frame: Measured at Day 56 ]
    Marrow or Blood Sample. Donor cell engraftment is defined as donor chimerism ≥ 5% on Day ≥ 56 after transplantation. Chimerism should be evaluated on Days ~28, ~56, ~180, and ~365 after transplantation. Chimerism may be evaluated in whole blood or mononuclear fraction.
  • Acute Graft-versus-host Disease (GVHD) [ Time Frame: Measured at Day 100 ]
  • Chronic GVHD [ Time Frame: Measured at Year 1 ]
  • Progression-free Survival [ Time Frame: Measured at Year 1 ]
    Progression-free survival is defined as the minimum time interval to relapse/ recurrence/progression, to death or to last follow-up.
  • Treatment-related Mortality (TRM) [ Time Frame: Measured at 6 months and 1 year ]
  • Incidence of Infections [ Time Frame: Measured at Year 1 ]
    Number of participants that experienced at least one infection.
  • Platelet Recovery to 50K [ Time Frame: Measured at Days 56, 90, and 100 ]
    Platelet recovery is defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count >50,000/mm3 with no platelet transfusions in the preceding seven days.


Original Secondary Outcome:

  • Neutrophil Recovery [ Time Frame: Measured at Days 28 and 42 ]
  • Primary Graft Failure [ Time Frame: Measured at Day 100 ]
  • Secondary Graft Failure [ Time Frame: Measured at Day 100 ]
  • Platelet recovery [ Time Frame: Measured at Days 100 and 180 ]
  • Donor Cell Engraftment [ Time Frame: Measured at Days 28, 56, 100, 180, and 365 ]
  • Acute Graft-versus-host Disease (GVHD) [ Time Frame: Measured at Day 100 ]
  • Chronic GVHD [ Time Frame: Measured at Year 1 ]
  • Progression-free Survival [ Time Frame: Measured at Year 1 ]
  • Treatment-related Mortality (TRM) [ Time Frame: Measured at Day 100 ]
  • Infections [ Time Frame: Measured at Year 1 ]


Information By: Medical College of Wisconsin

Dates:
Date Received: March 17, 2009
Date Started: December 2008
Date Completion:
Last Updated: August 1, 2016
Last Verified: August 2016