Evaluating the Safety and Effectiveness of an Umbilical Cord Blood Stem Cell Transplant (BMT CTN 0604)
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)
3 other identifiers
interventional
54
1 country
16
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Dec 2008
Longer than P75 for phase_2
16 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2008
CompletedFirst Submitted
Initial submission to the registry
March 17, 2009
CompletedFirst Posted
Study publicly available on registry
March 18, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2013
CompletedResults Posted
Study results publicly available
August 28, 2015
CompletedDecember 14, 2022
December 1, 2022
2.3 years
March 17, 2009
May 20, 2015
December 12, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall Survival at 180 Days From the Time of Transplant
Measured at Month 6 and Year 1
Secondary Outcomes (11)
Neutrophil Recovery
Measured at Days 28, 56, 90, and 100
Primary Graft Failure
Measured at Day 100
Secondary Graft Failure
Measured at Day 100
Platelet Recovery to 20K
Measured at Days 56, 90, and 100
Donor Cell Engraftment
Measured at Day 56
- +6 more secondary outcomes
Study Arms (1)
Umbilical Cord Blood Transplantation
EXPERIMENTALParticipants will receive a double unit Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen, GVHD prophylaxis.
Interventions
The transplant preparative regimen is listed below. The - sign is the number of days before the transplant. * Fludarabine: 40 mg/m\^2 intravenously (IV) on Days -6, -5, -4, -3, and -2 * Cyclophosphamide: 50 mg/kg IV on Day -6 * Total body irradiation: 200 centigray (cGy) on Day -1
GVHD prophylaxis regimen will consist of: * Cyclosporine: beginning on Day -3 with the dose adjusted to maintain a level of 200-400 mg/mL * MMF: 1 gram IV three times a day (TID) if greater than 50 kg, or 15 mg/kg IV TID if less than 50 kg beginning on Day -3; continued until Day 30 or 7 days after engraftment, whichever day is later Day 0 is the day of the infusion of the umbilical cord blood graft units, which will be obtained from partially HLA-matched unrelated donors. Beginning on Day 1, participants will receive G-CSF 5 mcg/kg/day until absolute neutrophil count (ANC) is greater than or equal to 2,000/mm\^3 for three consecutive measurements, each on different days.
Eligibility Criteria
You may qualify if:
- Participants must be 21 to 70 years old; participants 1 to 21 years old are also eligible if they are ineligible for BMT CTN #0501 (NCT00412360)
- Each unit must supply a minimum of 1.5 x 10\^7/kg pre-cryopreserved nucleated cell dose
- Participants must have two partially human leucocyte antigen (HLA)-matched umbilical cord blood units. Each unit must match at a minimum of 4 of 6 at HLA-A, -B, -DRB1 loci with the recipient. This may include 0 to 2 antigen mismatches at each A or B (at the antigen level) or DRB1 (at the allele level) loci. Each unit must be a 4 to 6 HLA-A, B, and DRB1 antigen matched to each other, not necessarily at the same loci as with the recipient. All typing will be done using molecular typing. Though molecular level typing will be available, a match is defined at intermediate resolution for HLA-A and -B and at high resolution for -DRB1 for this study. An adult unrelated donor search is not required for a person to be eligible for this study if the clinical situation dictates an urgent transplant. Clinical urgency is defined as 6 to 8 weeks from referral to transplant center or low likelihood of finding a matched, unrelated donor.
- Must have received cytotoxic chemotherapy within 3 months of the consent date (measured from the start date of chemotherapy)
- Acute leukemias (includes T lymphoblastic lymphoma) in the second or subsequent complete remission (CR)
- Burkitt's lymphoma in the second or subsequent CR
- Lymphoma
- Patients with adequate physical function, as measured by the following:
- Heart: left ventricular ejection fraction at rest greater than 35%, or shortening fraction greater than 25%
- Liver: bilirubin less than or equal to 2.5 mg/dL and alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase less than or equal to five times the upper limit of normal
- Kidney: serum creatinine within normal range for age, or if serum creatinine is outside the normal range for age, then kidney function (creatinine clearance or glomerular filtration rate (GFR) greater than 40 mL/min/1.73m\^2
- Lungs: forced expiratory volume in one second (FEV1), forced vital capacity (FVC), and carbon monoxide diffusing capacity (DLCO) greater than 50% predicted (corrected for hemoglobin). If unable to perform pulmonary function tests, then oxygen (O2) saturation must be greater than 92% on room air.
You may not qualify if:
- Have an HLA-matched, related, or 7 or 8/8 HLA allele matched (HLA-A, -B, -Cw, -DRB1) related donor able to donate
- Had an autologous hematopoietic stem cell transplant in the 3 months before study entry
- Pregnant or breastfeeding
- Evidence of HIV infection or known HIV positive serology
- Current uncontrolled bacterial, viral, or fungal infection (i.e., currently taking medication with evidence of progression of clinical symptoms or radiologic findings)
- Prior allogeneic hematopoietic stem cell transplant
- History of primary idiopathic myelofibrosis
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Medical College of Wisconsinlead
- National Heart, Lung, and Blood Institute (NHLBI)collaborator
- Blood and Marrow Transplant Clinical Trials Networkcollaborator
- National Cancer Institute (NCI)collaborator
- National Marrow Donor Programcollaborator
Study Sites (16)
City of Hope National Medical Center
Duarte, California, 91010, United States
University of Florida College of Medicine, Shands
Gainesville, Florida, 32610-3633, United States
H. Lee Moffitt Cancer Center
Tampa, Florida, 33624, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242-1009, United States
University of Kansas Hospital
Kansas City, Kansas, 66160, United States
Dana-Farber Cancer Institute (DFCI), Brigham & Women's Hospital
Boston, Massachusetts, 02114, United States
Dana-Farber Cancer Institute (DFCI), Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Washington University, Barnes Jewish Hospital
St Louis, Missouri, 63110, United States
Weill Cornell Medical College, NY Presbyterian Hospital
New York, New York, 10065, United States
Ohio State, Arthur G. James Cancer Hospital
Columbus, Ohio, 43210, United States
University of Pennsylvania Cancer Center
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Texas Transplant Institute
San Antonio, Texas, 78229, United States
Virginia Commonwealth University, Medical College of Virginia (MCV) Hospital
Richmond, Virginia, 23298, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792-5156, United States
Related Publications (2)
Brunstein CG, Fuchs EJ, Carter SL, Karanes C, Costa LJ, Wu J, Devine SM, Wingard JR, Aljitawi OS, Cutler CS, Jagasia MH, Ballen KK, Eapen M, O'Donnell PV; Blood and Marrow Transplant Clinical Trials Network. Alternative donor transplantation after reduced intensity conditioning: results of parallel phase 2 trials using partially HLA-mismatched related bone marrow or unrelated double umbilical cord blood grafts. Blood. 2011 Jul 14;118(2):282-8. doi: 10.1182/blood-2011-03-344853. Epub 2011 Apr 28.
PMID: 21527516RESULTEapen M, O'Donnell P, Brunstein CG, Wu J, Barowski K, Mendizabal A, Fuchs EJ. Mismatched related and unrelated donors for allogeneic hematopoietic cell transplantation for adults with hematologic malignancies. Biol Blood Marrow Transplant. 2014 Oct;20(10):1485-92. doi: 10.1016/j.bbmt.2014.05.015. Epub 2014 May 23.
PMID: 24862638RESULT
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Adam Mendizabal
- Organization
- The EMMES Corporation
Study Officials
- STUDY DIRECTOR
Mary Horowitz, MD, MS
Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 17, 2009
First Posted
March 18, 2009
Study Start
December 1, 2008
Primary Completion
April 1, 2011
Study Completion
November 1, 2013
Last Updated
December 14, 2022
Results First Posted
August 28, 2015
Record last verified: 2022-12
Data Sharing
- IPD Sharing
- Will not share
Findings will be published in a manuscript.