Condition: Adenosine Deaminase Severe Combined Immune Deficiency · Sponsor: University of California, Los Angeles
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
This description comes directly from the study's public registry record.
Satiro De Oliveira, MD · 1-310-825-6708 · sdeoliveira@mednet.ucla.edu
Augustine Fernandes, PhD · 1-310-267-4948 · afernandes@mednet.ucla.edu
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| University of California, Los Angeles (UCLA) | Los Angeles, California, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05432310