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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
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
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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  
Number of Participants  
[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  
Recipient to First CBU HLA Match [2]
[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  
Minimum HLA Match Score [3]
[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  
Weight at Infusion  
[units: kilograms]
Mean (Standard Deviation)
  39.6  (20.2)     38.7  (19.2)     39.15  (19.7)  
Recipient CMV Status [2]
[units: participants]
     
Positive     58     66     124  
Negative     51     39     90  
Inconclusive     1     1     2  
Unknown     2     2     4  
[1] Assesses patient self-perceived global quality of life and functioning (excellent, very good, good, fair, poor), where 100 equals perfect quality of life.
[2] Data limited to transplanted patients only.
[3] 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.



  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
No text entered.


  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