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Single vs Double Umbilical Cord Blood Transplants in Children With High Risk Leukemia and Myelodysplasia (BMT CTN 0501)

This study has been completed.
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
Information provided by (Responsible Party):
Medical College of Wisconsin
ClinicalTrials.gov Identifier:
NCT00412360
First received: December 14, 2006
Last updated: June 27, 2016
Last verified: May 2016
Results First Received: August 28, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Acute Myelogenous Leukemia
Acute Lymphocytic Leukemia
Chronic Myelogenous Leukemia
Myelodysplastic Syndrome
Natural Killer Cell Lymphoblastic Leukemia/Lymphoma
Interventions: Biological: Single Umbilical Cord Blood Unit Transplant
Biological: Double Umbilical Cord Blood Unit Transplant
Radiation: Total Body Irradiation
Drug: Cyclophosphamide
Drug: Fludarabine
Drug: Cyclosporine A
Drug: Mycophenolate Mofetil

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Single UCB Transplant Single Cord Blood Unit Transplantation: Unrelated donor, single cord blood unit
Double UCB Transplant Double Cord Blood Unit Transplantation: Unrelated donor, double cord blood unit

Participant Flow:   Overall Study
    Single UCB Transplant   Double UCB Transplant
STARTED   113   111 
COMPLETED   112   108 
NOT COMPLETED   1   3 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Analysis based on intent-to-treat.

Reporting Groups
  Description
Single UCB Transplant Single Umbilical Cord Blood Unit Transplantation
Double UCB Transplant Double Umbilical Cord Blood Unit Transplantation
Total Total of all reporting groups

Baseline Measures
   Single UCB Transplant   Double UCB Transplant   Total 
Overall Participants Analyzed 
[Units: Participants]
 113   111   224 
Age 
[Units: Years]
Mean (Standard Deviation)
 10.4  (5.1)   10.4  (5.1)   10.4  (5.1) 
Gender 
[Units: Participants]
     
Female   55   41   96 
Male   58   70   128 
Ethnicity (NIH/OMB) 
[Units: Participants]
     
Hispanic or Latino   22   20   42 
Not Hispanic or Latino   88   88   176 
Unknown or Not Reported   3   3   6 
Race (NIH/OMB) 
[Units: Participants]
     
American Indian or Alaska Native   1   0   1 
Asian   4   5   9 
Native Hawaiian or Other Pacific Islander   0   0   0 
Black or African American   13   11   24 
White   80   85   165 
More than one race   3   3   6 
Unknown or Not Reported   12   7   19 
Primary Disease 
[Units: Participants]
     
Acute Myelogenous Leukemia (AML)   36   38   74 
Acute Lymphoblastic Leukemia (ALL)   61   58   119 
Acute Biphenotypic Leukemia   6   2   8 
Acute Undifferentiated Leukemia   1   0   1 
Myelodysplastic Syndrome (MDS)   5   13   18 
Chronic Myelogenous Leukemia (CML)   1   0   1 
Secondary/Therapy Related AML   3   0   3 
Karnofsky Performance-status Score [1] 
[Units: Participants]
     
100%   58   57   115 
90%   38   43   81 
80%   13   9   22 
70%   4   2   6 
[1] Assesses patient self-perceived global quality of life and functioning (excellent, very good, good, fair, poor), where 100 equals perfect quality of life.
Recipient to First CBU HLA Match [1] 
[Units: Participants]
     
3/6 HLA markers   1   2   3 
4/6 HLA markers   44   33   77 
5/6 HLA markers   51   59   110 
6/6 HLA markers   16   14   30 
[1] Data limited to transplanted patients only.
Minimum HLA Match Score [1] 
[Units: Participants]
     
3/6 HLA markers   1   2   3 
4/6 HLA markers   44   48   92 
5/6 HLA markers   51   46   97 
6/6 HLA markers   16   12   28 
[1] Data limited to transplanted patients only. Minimum HLA match is defined as the minimum match score between the first unit/recipient, second unit/recipient, or first unit/second unit.
Weight at Infusion 
[Units: Kilograms]
Mean (Standard Deviation)
 39.6  (20.2)   38.7  (19.2)   39.15  (19.7) 
Recipient CMV Status [1] 
[Units: Participants]
     
Positive   58   66   124 
Negative   51   39   90 
Inconclusive   1   1   2 
Unknown   2   2   4 
[1] Data limited to transplanted patients only.


  Outcome Measures
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1.  Primary:   Overall Survival   [ Time Frame: Year 1 ]

2.  Secondary:   Disease-free Survival   [ Time Frame: Year 1 ]

3.  Secondary:   Neutrophil Engraftment   [ Time Frame: Day 180 ]

4.  Secondary:   Acute Graft-versus-host Disease (GVHD)   [ Time Frame: Day 100 ]

5.  Secondary:   Chronic GVHD   [ Time Frame: Year 1 ]

6.  Secondary:   Treatment-related Mortality   [ Time Frame: Year 1 ]

7.  Secondary:   Infections   [ Time Frame: Year 2 ]

8.  Secondary:   Relapse   [ Time Frame: Year 1 ]

9.  Secondary:   Platelet Engraftment   [ Time Frame: Day 180 ]

10.  Secondary:   Engraftment Syndrome   [ Time Frame: Day 100 ]

11.  Secondary:   Chimerism   [ Time Frame: 28, 42, 60, 180, 365 days ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

12.  Secondary:   Immune Reconstitution   [ Time Frame: 100 days, 6 months, 1 and 2 years ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Adam Mendizabal
Organization: The EMMES Corporation
phone: 301-251-1161
e-mail: amendizabal@EMMES.com


Publications of Results:

Responsible Party: Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT00412360     History of Changes
Obsolete Identifiers: NCT00429598
Other Study ID Numbers: BMTCTN0501
2U01HL069294 ( US NIH Grant/Contract Award Number )
5U24CA076518 ( US NIH Grant/Contract Award Number )
Study First Received: December 14, 2006
Results First Received: August 28, 2015
Last Updated: June 27, 2016
Health Authority: United States: Federal Government
United States: Institutional Review Board