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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00412360
Recruitment Status : Completed
First Posted : December 18, 2006
Results First Posted : December 21, 2015
Last Update Posted : November 28, 2017
Sponsor:
Collaborators:
Information provided by (Responsible Party):

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Single Group Assignment;   Masking: None (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

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
No text entered.

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

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) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      55  48.7%      41  36.9%      96  42.9% 
Male      58  51.3%      70  63.1%      128  57.1% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      22  19.5%      20  18.0%      42  18.8% 
Not Hispanic or Latino      88  77.9%      88  79.3%      176  78.6% 
Unknown or Not Reported      3   2.7%      3   2.7%      6   2.7% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      1   0.9%      0   0.0%      1   0.4% 
Asian      4   3.5%      5   4.5%      9   4.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      13  11.5%      11   9.9%      24  10.7% 
White      80  70.8%      85  76.6%      165  73.7% 
More than one race      3   2.7%      3   2.7%      6   2.7% 
Unknown or Not Reported      12  10.6%      7   6.3%      19   8.5% 
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

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.  

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


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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
No text entered.


  More Information

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 ( U.S. NIH Grant/Contract )
5U24CA076518 ( U.S. NIH Grant/Contract )
First Submitted: December 14, 2006
First Posted: December 18, 2006
Results First Submitted: August 28, 2015
Results First Posted: December 21, 2015
Last Update Posted: November 28, 2017