Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Duke University
ClinicalTrials.gov Identifier:
NCT00744692
First received: August 28, 2008
Last updated: July 23, 2014
Last verified: July 2014
Results First Received: July 23, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Non Malignant Disorders
Immunodeficiencies
Congenital Marrow Failures
Hemoglobinopathies
Inborn Errors of Metabolism
Sickle Cell
Thalassemia
Lysosomal Storage Disease
Interventions: Biological: Unrelated Umbilical Cord Blood Transplant
Drug: Reduced Intensity Conditioning

  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
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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
RIC Cord Blood Transplant

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning


Participant Flow:   Overall Study
    RIC Cord Blood Transplant  
STARTED     22  
COMPLETED     22  
NOT COMPLETED     0  



  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.
22 subjects undergoing umbilical cord blood transplant

Reporting Groups
  Description
RIC Cord Blood Transplant

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning


Baseline Measures
    RIC Cord Blood Transplant  
Number of Participants  
[units: participants]
  22  
Age [1]
[units: years]
Median ( Full Range )
  2.83  
  ( 0.3 to 8 )  
Age [2]
[units: participants]
 
<=18 years     22  
Between 18 and 65 years     0  
>=65 years     0  
Gender  
[units: participants]
 
Female     9  
Male     13  
[1] Age (in years) on the day of transplant
[2] Age (years) on the day of transplant



  Outcome Measures
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1.  Primary:   Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.   [ Time Frame: 180 days post transplant ]

2.  Secondary:   To Describe the Pace of Neutrophil Recovery   [ Time Frame: 42 days post transplant ]

3.  Secondary:   To Evaluate the Pace of Immune Reconstitution.   [ Time Frame: 1 year post transplant ]

4.  Secondary:   To Determine the Overall Survival at day180 Post-transplant   [ Time Frame: 180 days ]

5.  Secondary:   To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)   [ Time Frame: 100 days post transplant ]

6.  Secondary:   To Describe the Incidence of Grade 3-4 Organ Toxicity   [ Time Frame: 2 years post transplant ]

7.  Secondary:   To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure   [ Time Frame: at least 2 years post transplant ]

8.  Secondary:   To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant   [ Time Frame: 2 years post transplant ]

9.  Secondary:   To Describe the Pace of Platelet Recovery   [ Time Frame: 180 days post transplant ]


  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:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Suhag Parikh
Organization: Duke University Medical Center
phone: 919-668-1100
e-mail: suhag.parikh@dm.duke.edu


No publications provided by Duke University

Publications automatically indexed to this study:

Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT00744692     History of Changes
Other Study ID Numbers: Pro00008753
Study First Received: August 28, 2008
Results First Received: July 23, 2014
Last Updated: July 23, 2014
Health Authority: United States: Institutional Review Board