MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
|ClinicalTrials.gov Identifier: NCT00794508|
Recruitment Status : Completed
First Posted : November 20, 2008
Results First Posted : May 30, 2016
Last Update Posted : March 1, 2018
|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
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|