Single vs Double Umbilical Cord Blood Transplants in Children With High Risk Leukemia and Myelodysplasia (BMT CTN 0501)
Multi-center, Open Label, Randomized Trial Comparing Single Versus Double Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia (BMT CTN #0501)
3 other identifiers
interventional
224
3 countries
38
Brief Summary
This study is a Phase III, randomized, open-label, multi-center, prospective study of single umbilical cord blood (UCB) transplantation versus double UCB transplantation in pediatric patients with hematologic malignancies.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Dec 2006
Longer than P75 for phase_3
38 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, 2006
CompletedFirst Submitted
Initial submission to the registry
December 14, 2006
CompletedFirst Posted
Study publicly available on registry
December 18, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2014
CompletedResults Posted
Study results publicly available
December 21, 2015
CompletedOctober 28, 2021
October 1, 2021
7.3 years
December 14, 2006
August 28, 2015
October 13, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Participants With Overall Survival
Overall survival is defined as survival of death from any cause.
1 year post-randomization
Secondary Outcomes (9)
Percentage of Participants With Disease-free Survival
1 year post-randomization
Percentage of Participants With Neutrophil and Platelet Engraftment
Days 42 and 100
Time to Neutrophil and Platelet Engraftment
2 years post-transplant
Percentage of Participants With Acute Graft-versus-host Disease (GVHD)
Day 100 post-randomization
Percentage of Participants With Chronic GVHD
1 year post-randomization
- +4 more secondary outcomes
Study Arms (2)
Single Cord Blood Transplant
EXPERIMENTALUnrelated donor, single umbilical cord blood unit transplant; conditioning regimen: Total Body Irradiation/cyclophosphamide/fludarabine; GVHD prophylaxis: Cyclosporine A/Mycophenolate Mofetil
Double Cord Blood Transplant
EXPERIMENTALUnrelated donor, double umbilical cord blood unit transplant; Conditioning regimen: Total Body Irradiation/cyclophosphamide/fludarabine; GVHD prophylaxis: Cyclosporine A/Mycophenolate Mofetil
Interventions
Unrelated donor, single umbilical cord blood unit; conditioning regimen: TBI/cyclophosphamide/fludarabine; GVHD prophylaxis: cyclosporine/MMF
Unrelated donor, double umbilical cord blood unit; Conditioning regimen: TBI/cyclophosphamide/fludarabine; GVHD prophylaxis: cyclosporine/MMF
The TBI will be delivered from either a linear accelerator or cobalt source at a dose rate of between 4 and 26 cGy/minute using energies of between 1 and 25 MV.
Cyclophosphamide 60 mg/kg/day will be administered as a 2 hour intravenous infusion with a high volume fluid flush on Days -3 and -2.
Fludarabine 25 mg/m2/day will be administered over 30-60 minutes intravenous infusion on Days -10 through -8. Fludarabine will not be dose adjusted for body weight.
CSA will be administered beginning on Day -3 and doses will be adjusted to maintain a level of 200-400 ng/mL by TDX method (or 100-250 ng/mL by Tandem MS or equivalent level for other CSA testing methods). CSA can be administered per institutional practice.
MMF will be given at a dose of 1 gram IV q 8 hours if \> 50 kg or 15 mg/kg IV q 8 hours if \< 50 kg beginning the morning of Day -3.
Eligibility Criteria
You may qualify if:
- Two partially HLA-matched UCB units. Units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient, and the units must be HLA-matched at 3 of 6 HLA- A, B, DRB1 loci with each other (using same resolution of molecular typing as indicated above). Two appropriately HLA-matched units must be available such that one unit delivers a pre-cryopreserved nucleated cell dose of at least 2.5 x 10\^7 per kilogram and the second unit at least 1.5 x 10\^7 per kilogram.
- Acute myelogenous leukemia (AML) at the following stages:
- High risk first complete remission (CR1), defined as the following:
- Having preceding myelodysplasia (MDS)
- High risk cytogenetics (high risk cytogenetics: del (5q) -5, -7, abn (3q), t (6;9) complex karyotype \[at least 5 abnormalities\],)the presence of a high FLT3 ITD-AR (\> 0.4)
- Requiring more than 1 cycle of chemotherapy to obtain complete remission (CR);
- FAB M6
- Second or greater CR
- First relapse with less than 25% blasts in bone marrow
- Morphologic complete remission with incomplete blood count recovery
- Therapy-related AML for which prior malignancy has been in remission for at least 12 months
- Acute lymphocytic leukemia (ALL) at the following stages:
- High risk first remission, defined as one of the following conditions:
- Philadelphia chromosome-positive adult lymphoblastic leukemia (Ph+ ALL)
- Mixed lineage leukemia (MLL) rearrangement with slow early response (defined as having M2 \[5-25% blasts\] or M3 \[more than 25% blasts on bone marrow examination on Day 14 of induction therapy\])
- +23 more criteria
You may not qualify if:
- Pregnant (β-positive human chorionic gonadotropin \[HCG\]) or breastfeeding
- Evidence of HIV infection or HIV positive serology
- Current uncontrolled bacterial, viral, or fungal infection (currently taking medication and progression of clinical symptoms)
- Autologous transplant less than 12 months prior to enrollment
- Prior autologous transplant for the disease for which the UCB transplant will be performed
- Prior allogeneic hematopoietic stem cell transplant
- Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
- Inability to receive TBI
- Requirement of supplemental oxygen
- HLA-matched related donor able to donate
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 (38)
University of Alabama
Birmingham, Alabama, 35294, United States
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
City of Hope National Medical Center
Duarte, California, 91010, United States
Childrens Hospital at Oakland
Oakland, California, 94609, United States
UCSD/Rady Childrens Hospital
San Diego, California, 92123, United States
University of California, San Francisco (Peds)
San Francisco, California, 94143, United States
The Children's Hospital of Denver
Denver, Colorado, 80218, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
University of Florida College of Medicine (Shands)
Gainesville, Florida, 32610, United States
Nemours Childrens Clinic
Jacksonville, Florida, 32207, United States
University of Miami
Miami, Florida, 33136, United States
All Children's Hospital
St. Petersburg, Florida, 33710, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322-1062, United States
Indiana University Medical Center
Indianapolis, Indiana, 46202, United States
University of Louisville/Kosiar Children's Hospital
Louisville, Kentucky, 40202, United States
Children's of New Orleans
New Orleans, Louisiana, 70118, United States
DFCI/Children's Hospital of Boston
Boston, Massachusetts, 02115, United States
University of Michigan Medical Center
Ann Arbor, Michigan, 48109, United States
Karmanos Cancer Institute/Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
University of Mississippi
Jackson, Mississippi, 39216, United States
Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64108, United States
New York Medical College
Valhalla, New York, 10595, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205-2696, United States
Oregon Health Sciences University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-7610, United States
Children's Medical Center of Dallas
Dallas, Texas, 75235, United States
Cook Childrens Medical Center
Fort Worth, Texas, 76104, United States
Texas Transplant Institute
San Antonio, Texas, 78229, United States
Utah BMT/University of Utah Medical School
Salt Lake City, Utah, 84132, United States
Virgina Commonwealth University
Richmond, Virginia, 23298, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53211, United States
Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
BC Cancer Agency
Vancouver, British Columbia, V5Z 4E3, Canada
Related Publications (2)
Shulman HM, Sullivan KM, Weiden PL, McDonald GB, Striker GE, Sale GE, Hackman R, Tsoi MS, Storb R, Thomas ED. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med. 1980 Aug;69(2):204-17. doi: 10.1016/0002-9343(80)90380-0.
PMID: 6996481BACKGROUNDWagner JE Jr, Eapen M, Carter S, Wang Y, Schultz KR, Wall DA, Bunin N, Delaney C, Haut P, Margolis D, Peres E, Verneris MR, Walters M, Horowitz MM, Kurtzberg J; Blood and Marrow Transplant Clinical Trials Network. One-unit versus two-unit cord-blood transplantation for hematologic cancers. N Engl J Med. 2014 Oct 30;371(18):1685-94. doi: 10.1056/NEJMoa1405584.
PMID: 25354103RESULT
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention 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
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 14, 2006
First Posted
December 18, 2006
Study Start
December 1, 2006
Primary Completion
March 1, 2014
Study Completion
October 1, 2014
Last Updated
October 28, 2021
Results First Posted
December 21, 2015
Record last verified: 2021-10
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- Within 6 months of official study closure at participating sites.
- Access Criteria
- Available to the public.
Findings were published in a manuscript.