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

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. Identifier: NCT00794508
Recruitment Status : Completed
First Posted : November 20, 2008
Results First Posted : May 30, 2016
Last Update Posted : March 1, 2018
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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Severe Combined Immunodeficiency
Intervention: Biological: ADA gene transfer

  Participant Flow

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
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

  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.

Reporting Groups
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 
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
Count of Participants
<=18 years      10 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
Female      6  60.0% 
Male      4  40.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
American Indian or Alaska Native      0   0.0% 
Asian      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      1  10.0% 
White      7  70.0% 
More than one race      2  20.0% 
Unknown or Not Reported      0   0.0% 
Region of Enrollment 
[Units: Participants]
United States   10 

  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

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

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

Publications automatically indexed to this study by Identifier (NCT Number):

Responsible Party: Donald B. Kohn, M.D., University of California, Los Angeles Identifier: NCT00794508     History of Changes
Other Study ID Numbers: ADA Gene Therapy
1R01FD003005-01 ( U.S. FDA Grant/Contract )
9908-337 ( Other Identifier: OBA-RAC )
First Submitted: November 19, 2008
First Posted: November 20, 2008
Results First Submitted: March 9, 2016
Results First Posted: May 30, 2016
Last Update Posted: March 1, 2018