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.|
|ClinicalTrials.gov Identifier: NCT00794508|
Recruitment Status : Completed
First Posted : November 20, 2008
Results First Posted : May 30, 2016
Last Update Posted : April 23, 2021
|Condition or disease||Intervention/treatment||Phase|
|Severe Combined Immunodeficiency||Biological: ADA gene transfer||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan|
|Study Start Date :||November 2008|
|Actual Primary Completion Date :||December 2014|
|Actual Study Completion Date :||January 2015|
Experimental: Retroviral-mediated ADA gene transfer
Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow.
Biological: ADA gene transfer
Autologous CD34+ cells transduced with the retroviral vector MND-ADA, carrying the human ADA gene.
- Number of Participants With Adverse Events [ Time Frame: 2 years ]Examine the safety of the procedure: harvesting bone marrow, isolating CD34+ hematopoietic stem/progenitor cells, performing ex vivo gene transduction with the MND-ADA gamma-retroviral vector, giving 90 mg/m2 busulfan to "make space" in the bone marrow to aid engraftment, and re-infusing the autologous gene-modified cells.
- Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood [ Time Frame: 2 years ]As measured by quantitative polymerase chain reaction in peripheral blood cells separated into mononuclear and granulocyte fractions.
- Number of Participants Reaching the Normal Range of ADA Enzyme Activity [ Time Frame: 2 years ]As measured by ADA enzyme activity in peripheral blood mononuclear cells
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00794508
|United States, California|
|University of California, Los Angeles|
|Los Angeles, California, United States, 90095|
|Principal Investigator:||Donald B. Kohn, M.D.||University of California, Los Angeles|