MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

This study has been completed.
Sponsor:
Collaborators:
FDA Office of Orphan Products Development
National Institutes of Health (NIH)
Information provided by (Responsible Party):
Donald B. Kohn, M.D., University of California, Los Angeles
ClinicalTrials.gov Identifier:
NCT00794508
First received: November 19, 2008
Last updated: April 22, 2016
Last verified: April 2016
Results First Received: March 9, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Severe Combined Immunodeficiency
Intervention: Biological: ADA gene transfer

  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
Gamma-retroviral-mediated ADA Gene Transfer

Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow.

ADA gene transfer: Autologous CD34+ cells transduced with the gamma-retroviral vector, MND-ADA, carrying the human ADA gene.


Participant Flow:   Overall Study
    Gamma-retroviral-mediated ADA Gene Transfer  
STARTED     10  
COMPLETED     10  
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.
Pre-treatment

Reporting Groups
  Description
Experimental Retroviral-mediated ADA Gene Transfer Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow

Baseline Measures
    Experimental Retroviral-mediated ADA Gene Transfer  
Number of Participants  
[units: participants]
  10  
Age  
[units: participants]
 
<=18 years     10  
Between 18 and 65 years     0  
>=65 years     0  
Gender  
[units: participants]
 
Female     6  
Male     4  
Race (NIH/OMB)  
[units: participants]
 
American Indian or Alaska Native     0  
Asian     0  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     1  
White     7  
More than one race     2  
Unknown or Not Reported     0  
Region of Enrollment  
[units: participants]
 
United States     10  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Participants With Adverse Events   [ Time Frame: 2 years ]

2.  Secondary:   Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood   [ Time Frame: 2 years ]

3.  Secondary:   Number of Participants Reaching the Normal Range of ADA Enzyme Activity   [ Time Frame: 2 years ]


  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: Dr. Donald B. Kohn
Organization: University of California Los Angeles
phone: 310-794-1964
e-mail: dkohn1@mednet.ucla.edu


Publications:

Responsible Party: Donald B. Kohn, M.D., University of California, Los Angeles
ClinicalTrials.gov Identifier: NCT00794508     History of Changes
Other Study ID Numbers: ADA Gene Therapy
1R01FD003005-01 ( US NIH Grant/Contract Award Number )
9908-337 ( Other Identifier: OBA-RAC )
Study First Received: November 19, 2008
Results First Received: March 9, 2016
Last Updated: April 22, 2016
Health Authority: United States: Food and Drug Administration