A Phase III Randomized Trial of G-CSF Stimulated Bone Marrow vs. Conventional Bone Marrow as a Stem Cell Source In Matched Sibling Donor Transplantation
PRIMARY OBJECTIVE:
I. Compare improvement in event-free survival of patients with hematologic cancer or other
diseases undergoing filgrastim (G-CSF)-stimulated bone marrow transplantation (BMT) vs
conventional BMT.
SECONDARY OBJECTIVES:
I. Compare the incidence and time to engraftment in patients treated with these regimens.
II. Compare rates of acute and chronic graft-vs-host disease (GVHD) in patients treated with
these regimens.
III. Correlate incidence of acute and chronic GVHD with absolute T-cell numbers, Th1 vs Th2
profile of T cells, dendritic cell populations, and T-regulatory cell content.
IV. Assess the impact of G-CSF-stimulated BMT as a stem cell source on hospital stay and
treatment-related mortality at day 100 in patients treated with this regimen.
OUTLINE: This is a randomized, multicenter study. Patients are stratified according to risk
(high vs intermediate vs standard).
CONDITIONING REGIMEN: Co-enrolled on COG-ASCT0431 or COG-AAML0531; Patients receive a
conditioning regimen as defined on that treatment study.
ACUTE LYMPHOBLASTIC LEUKEMIA (ALL): Patients undergo total-body irradiation (TBI) twice
daily on days -8 to -6. Patients receive thiotepa IV on days -5 and -4 and high-dose
cyclophosphamide IV over 1 hour on days -3 and -2. Some patients with CNS leukemia or very
high-risk ALL in first complete remission receive cranial radiotherapy.
ACUTE MYELOID LEUKEMIA, JUVENILE MYELOMONOCYTIC, CHRONIC MYELOGENOUS LEUKEMIA, OR
MYELODYSPLASTIC SYNDROMES: (myeloid malignancies) Patients receive busulfan IV over 2 hours
every 6 hours on days -9 to -6 and high-dose cyclophosphamide IV over 1 hour on days -5 to
-2.
GRAFT-VS-HOST DISEASE (GVHD) PROPHYLAXIS: Co-enrolled on COG-ASCT0431 or COG-AAML0531:
Patients undergo GVHD prophylaxis as defined on that treatment study.
ALL: Patients receive tacrolimus IV or orally beginning on day -2 and continuing until day
42, followed by a taper until day 98. Patients also receive methotrexate IV on days 1, 3,
and 6.
MYELOID MALIGNANCIES: Patients receive cyclosporine IV continuously or orally beginning on
day -1 and continuing until day 42 or day 50, followed by a taper for 8-16 weeks. Patients
also receive methotrexate IV on days 1, 3, 6, and 11.
ALLOGENEIC BONE MARROW TRANSPLANTATION (BMT): Patients are randomized to 1 of 2
transplantation arms.
ARM I: Patients undergo filgrastim (G-CSF) -stimulated allogeneic BMT on day 0.
ARM II: Patients undergo conventional allogeneic BMT on day 0.
After completion of study treatment, patients are followed at 1 year and then annually for
5-10 years.
Interventional
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Event-free survival
Analyzed using the stratified log-rank test.
2 years
No
Stephan A. Grupp
Principal Investigator
Children's Oncology Group
United States: Institutional Review Board
ASCT0631
NCT00450450
December 2007
Name | Location |
---|---|
Johns Hopkins University | Baltimore, Maryland 21205 |
Roswell Park Cancer Institute | Buffalo, New York 14263 |
Children's Hospital of Philadelphia | Philadelphia, Pennsylvania 19104 |
Washington University School of Medicine | Saint Louis, Missouri 63110 |
Vanderbilt-Ingram Cancer Center | Nashville, Tennessee 37232-6838 |
New York Medical College | Valhalla, New York 10595 |
University of Nebraska Medical Center | Omaha, Nebraska 68198-3330 |
Hackensack University Medical Center | Hackensack, New Jersey 07601 |
Kosair Children's Hospital | Louisville, Kentucky 40202-3830 |
Primary Children's Medical Center | Salt Lake City, Utah 84113-1100 |
Nationwide Children's Hospital | Columbus, Ohio 43205-2696 |
University of North Carolina | Chapel Hill, North Carolina 27599 |
Indiana University Medical Center | Indianapolis, Indiana 46202 |
University of Texas Southwestern Medical Center | Dallas, Texas |
Childrens Memorial Hospital | Chicago, Illinois 60614 |
Children's Oncology Group | Arcadia, California 91006-3776 |
C S Mott Children's Hospital | Ann Arbor, Michigan 48109 |
Riley Hospital for Children | Indianapolis, Indiana 46202 |
The Childrens Mercy Hospital | Kansas City, Missouri 64108 |
Children's Hospital Colorado | Aurora, Colorado 80045 |
University of California San Francisco Medical Center-Parnassus | San Francisco, California 94143 |